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UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 10-Q
(Mark One)
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QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES AND EXCHANGE ACT OF 1934 |
For the quarterly period ended March 31, 2025
OR
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TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES AND EXCHANGE ACT OF 1934 |
For the transition period from to
Commission File Number: 001-38915
IDEAYA Biosciences, Inc.
(Exact name of registrant as specified in its charter)
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Delaware |
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47-4268251 |
(State or other jurisdiction of incorporation or organization) |
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(I.R.S. Employer Identification No.) |
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5000 Shoreline Court, Suite 300 South San Francisco, California |
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94080 |
(Address of principal executive offices) |
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(Zip Code) |
(650) 443-6209
(telephone number, including area code)
Securities registered pursuant to Section 12(b) of the Act:
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Title of each class |
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Trading Symbol(s) |
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Name of each exchange on which registered |
Common Stock, $0.0001 par value per share |
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IDYA |
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Nasdaq Global Select Market |
Indicate by check mark whether the registrant (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days. Yes ☒ No ☐
Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule 405 of Regulation S-T (§ 232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit such files). Yes ☒ No ☐
Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, smaller reporting company, or an emerging growth company. See the definitions of “large accelerated filer,” “accelerated filer,” “smaller reporting company,” and “emerging growth company” in Rule 12b-2 of the Exchange Act.
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Large accelerated filer |
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Accelerated filer |
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Non-accelerated filer |
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Smaller reporting company |
☐ |
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Emerging growth company |
☐ |
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐
Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act). Yes ☐ No ☒
As of May 2, 2025, the registrant had 87,581,963 shares of common stock, $0.0001 par value per share, outstanding.
NOTE REGARDING FORWARD-LOOKING STATEMENTS
This Quarterly Report on Form 10-Q contains forward-looking statements. We intend such forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act of 1933, as amended (the “Securities Act”), and Section 21E of the Securities Exchange Act of 1934, as amended (the “Exchange Act”). All statements other than statements of historical facts contained in this Form 10-Q, including statements regarding our future results of operations and financial position, business strategy, prospective products, product approvals, research and development costs, timing and likelihood of success, plans and objectives of management for future operations and future results of anticipated products, are forward-looking statements. These statements involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements.
In some cases, you can identify forward-looking statements by terms such as “may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplate,” “believe,” “estimate,” “predict,” “potential” or “continue” or the negative of these terms or other similar expressions. The forward-looking statements in this Quarterly Report on Form 10-Q are only predictions. We have based these forward-looking statements largely on our current expectations and projections about future events and financial trends that we believe may affect our business, financial condition and results of operations. These forward-looking statements speak only as of the date of this Quarterly Report on Form 10-Q and are subject to a number of risks, uncertainties and assumptions described under the sections in this Quarterly Report on Form 10-Q entitled “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results of Operations” and elsewhere in this Quarterly Report on Form 10-Q. These forward-looking statements are subject to numerous risks, including, without limitation, the following:
•the scope, progress, results and costs of developing our product candidates or any other future product candidates, and conducting preclinical studies and clinical trials, including our darovasertib (PKC) Phase 2/3 clinical trials, IDE397 (MAT2A) Phase 1/2 clinical trials, IDE849 (DLL3) Phase 1 clinical trial, IDE275 / GSK959 (Werner Helicase) Phase 1 clinical trial, IDE161 (PARG) Phase 1/2 clinical trial, IDE705 / GSK101 (Pol Theta Helicase) Phase 1 clinical trial, as well as the potential clinical utility and tolerability of our product candidates;
•our clinical and regulatory development plans;
•the scope, progress, results and costs related to the research and development of our precision medicine target and biomarker discovery platform, including costs related to the development of our proprietary libraries and database of tumor genetic information and specific cancer-target dependency networks;
•our expectations about the impact of macroeconomic developments, such as health epidemics or pandemics, macro-economic uncertainties, social unrest, geopolitical hostilities, natural disasters or other catastrophic events, on our business, and operations, including clinical trials, manufacturing suppliers and collaborators, and on our results of operations and financial condition;
•the availability of companion diagnostics for biomarkers associated with our product candidates and any future product candidates, or the cost of coordinating and/or collaborating with certain diagnostic companies for the manufacture and supply of companion diagnostics;
•the timing of and costs involved in obtaining and maintaining regulatory approval (or certification in certain foreign jurisdictions) for any current or future product candidates and companion diagnostics, and any related restrictions, limitations, and/or warnings in the label of an approved product candidate;
•our expectations regarding the potential market size and size of the potential patient populations for darovasertib, IDE397, IDE849, IDE275 / GSK959, IDE161, IDE705 / GSK101, our other product candidates and any future product candidates, if approved for commercial use;
•the timing and amount of any option exercised, milestone, royalty or other payments we may or may not receive pursuant to any current or future collaboration or license agreement, including under the Collaboration, Option and License Agreement with an affiliate of GSK plc, GLAXOSMITHKLINE INTELLECTUAL PROPERTY (NO. 4) LIMITED, or GSK;
•our ability to maintain existing, and establish new, strategic collaborations, licensing or other arrangements and the financial terms of any such agreements, including our Collaboration, Option and License
Agreement with GSK, our Clinical Study Collaboration and Supply Agreement with Gilead Sciences, Inc., our Clinical Trial Collaboration and Supply Agreement with MSD International Business GmbH, our Clinical Trial Collaboration and Supply Agreements with Pfizer Inc., our Clinical Trial Collaboration and Supply Agreement with Amgen Inc., our License Agreement with Novartis, our Option and License Agreement with Cancer Research Technologies Ltd. and the University of Manchester, our Option and License Agreement with Biocytogen Pharmaceuticals (Beijing) Co., Ltd and our License Agreement with Jiangsu Hengrui Pharmaceuticals Co., Ltd.;
•the timing of commencement of future nonclinical studies and clinical trials and research and development programs;
•our ability to acquire, discover, develop and advance product candidates into, and successfully complete, clinical trials;
•our intentions and our ability to establish collaborations and/or partnerships;
•the timing or likelihood of regulatory filings and approvals for our product candidates;
•our commercialization, marketing and manufacturing capabilities and expectations;
•our intentions with respect to the commercialization of our product candidates;
• the pricing and reimbursement of our product candidates, if approved;
•the implementation of our business model and strategic plans for our business, product candidates and technology platforms, including additional indications for which we may pursue;
•the scope of protection we are able to establish and maintain for intellectual property rights covering our product candidates, including the projected terms of patent protection;
•our potential involvement in lawsuits in connection with enforcing our intellectual property rights;
•our potential involvement in third party interference, opposition, derivation or similar proceedings with respect to our patent rights and other challenges to our patent rights and patent infringement claims;
•estimates of our expenses, future revenue, capital requirements, our needs for additional financing and our ability to obtain additional capital;
•our future financial performance; and
•developments and projections relating to our competitors and our industry, including competing therapies and procedures, as well as the competitive position of our product candidates.
Moreover, we operate in an evolving environment. New risk factors and uncertainties may emerge from time to time, and it is not possible for management to predict all risk factors and uncertainties.
Because forward-looking statements are inherently subject to risks and uncertainties, some of which cannot be predicted or quantified and some of which are beyond our control, you should not rely on these forward-looking statements as predictions of future events. The events and circumstances reflected in our forward-looking statements may not occur or be achieved, and actual results could differ materially from those projected in the forward-looking statements. We qualify all of our forward-looking statements by these cautionary statements. Except as required by applicable law, we do not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.
IDEAYA Biosciences, Inc.
Form 10-Q for Quarterly Period Ended March 31, 2025
Table of Contents
Item 1. Financial Statements (UNAUDITED).
IDEAYA Biosciences, Inc.
Condensed Balance Sheets
(in thousands, except share and per share amounts)
(Unaudited)
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March 31, |
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December 31, |
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2025 |
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2024 |
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Assets |
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Current assets |
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Cash and cash equivalents |
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$ |
129,996 |
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$ |
84,378 |
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Short-term marketable securities |
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562,512 |
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591,941 |
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Prepaid expenses and other current assets |
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15,364 |
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13,394 |
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Total current assets |
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707,872 |
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689,713 |
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Restricted cash |
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805 |
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805 |
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Long-term marketable securities |
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358,665 |
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405,832 |
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Property and equipment, net |
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8,871 |
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8,966 |
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Right-of-use assets |
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24,428 |
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18,775 |
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Total assets |
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$ |
1,100,641 |
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$ |
1,124,091 |
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Liabilities and Stockholders’ Equity |
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Current liabilities |
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Accounts payable |
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$ |
17,631 |
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$ |
15,421 |
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Accrued liabilities |
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32,907 |
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30,352 |
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Operating lease liabilities, current |
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308 |
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298 |
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Total current liabilities |
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50,846 |
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46,071 |
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Long-term operating lease liabilities |
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25,660 |
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18,873 |
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Total liabilities |
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76,506 |
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64,944 |
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Commitments and contingencies (Note 6) |
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Stockholders’ equity |
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Preferred stock, $0.0001 par value, 10,000,000 shares authorized as of March 31, 2025 and December 31, 2024; no shares issued and outstanding as of March 31, 2025 and December 31, 2024 |
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— |
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— |
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Common stock, $0.0001 par value, 300,000,000 shares authorized as of March 31, 2025 and December 31, 2024; 87,565,252 and 86,503,509 shares issued and outstanding as of March 31, 2025 and December 31, 2024 |
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9 |
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9 |
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Additional paid-in capital |
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1,717,560 |
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1,681,167 |
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Accumulated other comprehensive income |
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1,585 |
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812 |
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Accumulated deficit |
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(695,019 |
) |
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(622,841 |
) |
Total stockholders’ equity |
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1,024,135 |
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1,059,147 |
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Total liabilities and stockholders’ equity |
|
$ |
1,100,641 |
|
$ |
1,124,091 |
|
The accompanying notes are an integral part of these condensed financial statements.
IDEAYA Biosciences, Inc.
Condensed Statements of Operations and Comprehensive Loss
(in thousands, except share and per share amounts)
(Unaudited)
|
|
|
|
|
|
|
|
|
|
Three Months Ended March 31, |
|
|
|
2025 |
|
2024 |
|
Operating expenses |
|
|
|
|
|
Research and development |
|
|
70,886 |
|
|
42,805 |
|
General and administrative |
|
|
13,503 |
|
|
8,212 |
|
Total operating expenses |
|
|
84,389 |
|
|
51,017 |
|
Loss from operations |
|
|
(84,389 |
) |
|
(51,017 |
) |
Other income |
|
|
|
|
|
Interest income and other income, net |
|
|
12,211 |
|
|
11,445 |
|
Net loss |
|
$ |
(72,178 |
) |
$ |
(39,572 |
) |
Unrealized gains (losses) on marketable securities |
|
|
773 |
|
|
(1,485 |
) |
Comprehensive loss |
|
$ |
(71,405 |
) |
$ |
(41,057 |
) |
Net loss per common share, basic and diluted |
|
$ |
(0.82 |
) |
$ |
(0.53 |
) |
Weighted-average common shares outstanding, basic and diluted |
|
|
88,356,335 |
|
|
75,108,484 |
|
The accompanying notes are an integral part of these condensed financial statements.
IDEAYA Biosciences, Inc.
Condensed Statements of Stockholders’ Equity
(in thousands, except share amounts)
(Unaudited)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Accumulated |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Additional |
|
|
Other |
|
|
|
|
|
Total |
|
|
|
Common Stock |
|
|
Paid-In |
|
|
Comprehensive |
|
|
Accumulated |
|
|
Stockholders' |
|
|
|
Shares |
|
|
Amount |
|
|
Capital |
|
|
Income (Loss) |
|
|
Deficit |
|
|
Equity |
|
Balances as of December 31, 2024 |
|
|
86,503,509 |
|
|
$ |
9 |
|
|
$ |
1,681,167 |
|
|
$ |
812 |
|
|
$ |
(622,841 |
) |
|
$ |
1,059,147 |
|
Issuance of common stock related to at-the-market offering program, net of issuance costs |
|
|
984,000 |
|
|
|
— |
|
|
|
25,022 |
|
|
|
— |
|
|
|
— |
|
|
|
25,022 |
|
Issuance of common stock upon exercise of stock options |
|
|
77,743 |
|
|
|
— |
|
|
|
1,134 |
|
|
|
— |
|
|
|
— |
|
|
|
1,134 |
|
Stock-based compensation |
|
|
— |
|
|
|
— |
|
|
|
10,237 |
|
|
|
— |
|
|
|
— |
|
|
|
10,237 |
|
Other comprehensive income |
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
773 |
|
|
|
— |
|
|
|
773 |
|
Net loss |
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
(72,178 |
) |
|
|
(72,178 |
) |
Balances as of March 31, 2025 |
|
|
87,565,252 |
|
|
$ |
9 |
|
|
$ |
1,717,560 |
|
|
$ |
1,585 |
|
|
$ |
(695,019 |
) |
|
$ |
1,024,135 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Balances as of December 31, 2023 |
|
|
65,039,369 |
|
|
$ |
7 |
|
|
$ |
968,885 |
|
|
$ |
562 |
|
|
$ |
(348,364 |
) |
|
$ |
621,090 |
|
Issuance of common stock related to at-the-market offering program, net of issuance costs |
|
|
9,260,382 |
|
|
|
— |
|
|
|
343,505 |
|
|
|
— |
|
|
|
— |
|
|
|
343,505 |
|
Issuance of common stock upon exercise of stock options |
|
|
464,877 |
|
|
|
— |
|
|
|
5,461 |
|
|
|
— |
|
|
|
— |
|
|
|
5,461 |
|
Stock-based compensation |
|
|
— |
|
|
|
— |
|
|
|
6,312 |
|
|
|
— |
|
|
|
— |
|
|
|
6,312 |
|
Other comprehensive loss |
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
(1,485 |
) |
|
|
— |
|
|
|
(1,485 |
) |
Net loss |
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
(39,572 |
) |
|
|
(39,572 |
) |
Balances as of March 31, 2024 |
|
|
74,764,628 |
|
|
$ |
7 |
|
|
$ |
1,324,163 |
|
|
$ |
(923 |
) |
|
$ |
(387,936 |
) |
|
$ |
935,311 |
|
The accompanying notes are an integral part of these condensed financial statements.
IDEAYA Biosciences, Inc.
Condensed Statements of Cash Flows
(in thousands)
(Unaudited)
|
|
|
|
|
|
|
|
|
|
Three Months Ended March 31, |
|
|
|
2025 |
|
2024 |
|
Cash flows from operating activities |
|
|
|
|
|
Net loss |
|
$ |
(72,178 |
) |
$ |
(39,572 |
) |
Adjustments to reconcile net loss to net cash used in operating activities |
|
|
|
|
|
Depreciation and amortization |
|
|
600 |
|
|
616 |
|
Net accretion of discounts on marketable securities |
|
|
(3,778 |
) |
|
(6,314 |
) |
Stock-based compensation |
|
|
10,237 |
|
|
6,312 |
|
Amortization of right-of-use assets |
|
|
533 |
|
|
466 |
|
Changes in assets and liabilities |
|
|
|
|
|
Prepaid expenses and other assets |
|
|
(1,970 |
) |
|
(3,130 |
) |
Accounts payable |
|
|
2,728 |
|
|
(453 |
) |
Accrued and other liabilities |
|
|
2,875 |
|
|
(1,273 |
) |
Lease liabilities |
|
|
611 |
|
|
(465 |
) |
Net cash used in operating activities |
|
|
(60,342 |
) |
|
(43,813 |
) |
Cash flows from investing activities |
|
|
|
|
|
Purchases of property and equipment, net |
|
|
(1,330 |
) |
|
(1,325 |
) |
Purchases of marketable securities |
|
|
(105,207 |
) |
|
(475,781 |
) |
Maturities of marketable securities |
|
|
186,354 |
|
|
123,135 |
|
Net cash provided by (used in) investing activities |
|
|
79,817 |
|
|
(353,971 |
) |
Cash flows from financing activities |
|
|
|
|
|
Proceeds from issuance of common stock related to at-the-market offering program, net of issuance costs |
|
|
25,009 |
|
|
343,650 |
|
Proceeds from exercise of common stock options |
|
|
1,134 |
|
|
5,461 |
|
Net cash provided by financing activities |
|
|
26,143 |
|
|
349,111 |
|
Net increase (decrease) in cash, cash equivalents and restricted cash |
|
|
45,618 |
|
|
(48,673 |
) |
Cash, cash equivalents and restricted cash |
|
|
|
|
|
Cash, cash equivalents and restricted cash, at beginning of period |
|
|
85,183 |
|
|
157,775 |
|
Cash, cash equivalents and restricted cash, at end of period |
|
$ |
130,801 |
|
$ |
109,102 |
|
|
|
|
|
|
|
Reconciliation of cash, cash equivalents and restricted cash |
|
|
|
|
|
Cash and cash equivalents |
|
$ |
129,996 |
|
$ |
108,345 |
|
Restricted cash |
|
|
805 |
|
|
757 |
|
Cash, cash equivalents and restricted cash |
|
$ |
130,801 |
|
$ |
109,102 |
|
|
|
|
|
|
|
Supplemental disclosure of cash flow information: |
|
|
|
|
|
Cash paid for interest |
|
$ |
— |
|
$ |
12 |
|
|
|
|
|
|
|
Supplemental non-cash investing and financing activities: |
|
|
|
|
|
Right-of-use asset obtained in exchange for a new operating lease liability |
|
|
6,186 |
|
|
— |
|
Purchases of property and equipment in accounts payable and accrued liabilities |
|
|
654 |
|
|
183 |
|
Unpaid at-the-market offering program costs |
|
$ |
— |
|
$ |
145 |
|
The accompanying notes are an integral part of these condensed financial statements.
IDEAYA Biosciences, Inc.
Notes to Condensed Financial Statements (Unaudited)
1. Organization
Description of the Business
IDEAYA Biosciences, Inc. (the “Company”) is a precision medicine oncology company committed to the discovery and development of targeted therapeutics for patient populations selected using molecular diagnostics. The Company is headquartered in South San Francisco, California and was incorporated in the State of Delaware in June 2015. To date, the Company has been primarily engaged in business planning, research, development, recruiting and raising capital.
Follow-On Offering
On July 11, 2024, the Company completed an underwritten public follow-on offering. The offering consisted of 8,355,714 shares of the Company's common stock, par value $0.0001 per share ("common stock"), at an offering price to the public of $35.00 per share, including 1,127,142 shares of common stock upon the exercise in full of the overallotment option by the underwriters, as well as pre-funded warrants to purchase 285,715 shares of common stock at a public offering price of $34.9999 per underlying share, in each case before underwriting discounts and commissions. Pursuant to the offering, the Company received aggregate gross proceeds of approximately $302.4 million, before deducting underwriting discounts and commissions and other offering expenses, resulting in net proceeds of approximately $283.8 million, after deducting underwriting discounts and commissions and other offering expenses.
At-the-Market Offering
On January 19, 2024, the Company entered into a new Open Market Sales Agreement (the “January 2024 Sales Agreement”) with Jefferies relating to an at-the-market offering program under which the Company may offer and sell, from time to time at its sole discretion, shares of common stock having aggregate gross proceeds of up to $350.0 million through Jefferies as sales agent.
During the three months ended March 31, 2025, the Company sold an aggregate of 984,000 shares of common stock for aggregate net proceeds of $25.0 million at a weighted average sales price of approximately $26.00 per share under the at-the-market offering pursuant to the January 2024 Sales Agreement with Jefferies as sales agent. As of March 31, 2025, approximately $156.6 million of common stock remained available to be sold pursuant to the January 2024 Sales Agreement.
The Company may cancel its at-the-market program at any time upon written notice, pursuant to its terms.
Liquidity
The Company has incurred significant losses and negative cash flows from operations in all periods since inception and had an accumulated deficit of $695.0 million as of March 31, 2025.
The Company has financed its operations primarily through the sale and issuance of common stock and the upfront payment and certain milestone payments received from GSK.
To date, none of the Company’s product candidates have been approved for sale, and the Company has not generated any revenue from commercial products since inception. Management expects operating losses to continue and increase for the foreseeable future, as the Company progresses clinical development activities for its lead product candidates. The Company’s prospects are subject to risks, expenses and uncertainties frequently encountered by companies in the biotechnology industry as discussed under Risks and Uncertainties in Note 2. While the Company has been able to raise multiple rounds of financing, there can be no assurance that in the event the Company requires additional financing, such financing will be available on terms which are favorable or at all. Failure to generate sufficient cash flows from operations, raise additional capital or reduce certain discretionary spending would have a material adverse effect on the Company’s ability to achieve its intended business objectives.
As of March 31, 2025, the Company had cash, cash equivalents and marketable securities of approximately $1.05 billion. Management believes that the Company’s current cash, cash equivalents and marketable securities will be sufficient to fund its planned operations for at least 12 months from the date of the issuance of these financial statements.
2. Summary of Significant Accounting Policies
Basis of Presentation
These condensed financial statements have been prepared in accordance with accounting principles generally accepted in the United States of America (“GAAP”) and the rules and regulations of the United States Securities and Exchange Commission (“SEC”) for interim reporting.
Certain information and footnote disclosures normally included in annual financial statements prepared in accordance with GAAP have been condensed or omitted. Accordingly, the unaudited condensed financial statements should be read in conjunction with the audited financial statements and the related notes thereto included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2024 filed with the SEC on February 18, 2025.
Unaudited Condensed Financial Statements
The accompanying financial information for the three months ended March 31, 2025 and March 31, 2024 are unaudited. The unaudited condensed financial statements have been prepared on the same basis as the annual audited financial statements and, in the opinion of management, reflect all adjustments, which include only normal recurring adjustments, necessary to present fairly the Company’s financial position as of March 31, 2025 and December 31, 2024, its results of operations for the three months ended March 31, 2025 and March 31, 2024 and cash flows for the three months ended March 31, 2025 and March 31, 2024. The results for interim periods are not necessarily indicative of the results expected for the full fiscal year or any other periods.
Use of Estimates
The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and disclosure of contingent assets and liabilities as of the date of the financial statements and the reported amounts of revenue and expenses during the reporting period. Such estimates include useful lives of property and equipment, determination of the discount rate for operating leases, accruals for research and development activities, revenue recognition, stock-based compensation, and income taxes. On an ongoing basis, management reviews these estimates and assumptions. Changes in facts and circumstances may alter such estimates and actual results could differ from those estimates.
Risks and Uncertainties
The Company operates in a dynamic and highly competitive industry and is subject to risks and uncertainties common to early-stage companies in the biotechnology industry, including, but not limited to, development by competitors of new technological innovations, protection of proprietary technology, dependence on key personnel, contract manufacturers, contract research organizations and collaboration partners, compliance with government regulations and the need to obtain additional financing to fund operations. Product candidates currently under development will require significant additional research and development efforts, including extensive preclinical studies and clinical trials and regulatory approval, prior to commercialization. These efforts require significant amounts of additional capital, adequate personnel infrastructure and extensive compliance and reporting. The Company believes that changes in any of the following areas could have a material adverse effect on the Company’s future financial position, results of operations, or cash flows: ability to obtain future financing; advances and trends in new technologies and industry standards; results of clinical trials and collaboration activities; regulatory approval and market acceptance of the Company’s products; development of sales channels; certain strategic relationships; litigation or claims against the Company based on intellectual property, patent, product, regulatory, or other factors; and the Company’s ability to attract and retain employees necessary to support its growth.
Products developed by the Company require approvals from the U.S. Food and Drug Administration (“FDA”) or other international regulatory agencies prior to commercial sales. There can be no assurance that the Company’s research and development will be successfully completed, that adequate protection for the Company’s intellectual property will be obtained or maintained, that the products will receive the necessary approvals, or that any approved
products will be commercially viable. If the Company was denied approval, approval was delayed or the Company was unable to maintain approval, it could have a materially adverse impact on the Company. Even if the Company’s product development efforts are successful, it is uncertain when, if ever, the Company will generate revenue from product sales. The Company operates in an environment of rapid change in technology and substantial competition from other pharmaceutical and biotechnology companies. In addition, the Company is dependent upon the services of its employees, consultants and other third parties.
The Company has expended and will continue to expend substantial funds to complete the research, development and clinical testing of product candidates. The Company also will be required to expend additional funds to establish commercial-scale manufacturing arrangements and to provide for the marketing and distribution of products that receive regulatory approval. The Company may require additional funds to commercialize its products. The Company is unable to entirely fund these efforts with its current financial resources. If adequate funds are unavailable on a timely basis from operations or additional sources of financing, the Company may have to delay, reduce the scope of or eliminate one or more of its research or development programs which would materially and adversely affect its business, financial condition and operations.
Concentration of Credit Risk
Financial instruments that potentially subject the Company to a concentration of credit risk consist of cash, cash equivalents and marketable securities. Substantially all the Company’s cash, cash equivalents and marketable securities are held by three financial institutions that management believes are of high credit quality. Such deposits may, at times, exceed federally insured limits.
The Company’s investment policy addresses credit ratings, diversification, and maturity dates.
The Company invests its cash equivalents and marketable securities in money market funds, U.S. government securities, commercial paper, and corporate bonds. The Company limits its credit risk associated with cash equivalents and marketable securities by placing them with banks and institutions it believes are creditworthy and in highly rated investments and, by policy, limits the amount of credit exposure with any one commercial issuer. The Company has not experienced any credit losses on its deposits of cash, cash equivalents or marketable securities.
Summary of Significant Accounting Policies
There have been no material changes in the accounting policies from those disclosed in the financial statements and the related notes included in the Company’s Annual Report on Form 10-K, filed with the SEC on February 18, 2025.
Recent Accounting Pronouncements
From time to time, new accounting pronouncements are issued by the Financial Accounting Standards Board (“FASB”) under its accounting standard codifications (“ASC”) or other standard setting bodies and adopted by the Company as of the specified effective date, unless otherwise discussed below.
New Accounting Pronouncements Adopted
In November 2023, the FASB issued ASU 2023-07, Segment Reporting (Topic 280): Improvements to Reportable Segment Disclosures, which improves reportable segment disclosure requirements through enhanced disclosures about significant segment expenses. These amendments enhance interim disclosure requirements, require disclosure of the title and position of the chief operating decision maker (“CODM”), require disclosure of significant segment expenses that are regularly provided to the CODM, clarify circumstances for disclosure of more than one segment profit or loss measure and require that a public entity that has a single reportable segment provide all disclosures required by ASC 280 and amendments. This ASU update is effective for fiscal years beginning after December 15, 2023 for the Company’s annual report, and interim periods within fiscal years beginning after December 15, 2024. The Company adopted this ASU for the annual report for the fiscal year beginning January 1, 2024 and for this quarterly report for the period beginning January 1, 2025. This ASU resulted in additional disclosures. See Note 13. Segment Information.
New Accounting Pronouncements, Not yet Adopted
In October 2023, the FASB issued ASU 2023-06, Disclosure Improvements: Codification Amendments in Response to the SEC’s Disclosure Update and Simplification Initiative, which modifies the disclosure or presentation requirements related to variety of FASB Accounting Standard Codification topics. The effective date for each amendment will be the date on which the SEC’s removal of that related disclosure from Regulation S-X or Regulation S-K is effective. If by June 30, 2027, the SEC has not removed the applicable requirement from Regulation S-X or Regulation S-K, the pending content of the associated amendment will be removed from the Codification and will not become effective for any entities. The Company is currently evaluating the effect of adopting this ASU.
In December 2023, the FASB issued ASU 2023-09, Improvements to Income Tax Disclosures, which amends the guidance in ASC 740, Income Taxes. The ASU is intended to improve the transparency of income tax disclosures by requiring (1) consistent categories and greater disaggregation of information in the rate reconciliation and (2) income taxes paid disaggregated by jurisdiction. It also includes certain other amendments to improve the effectiveness of income tax disclosures. The ASU’s amendments are effective for public business entities for annual periods beginning after December 15, 2024. Entities are permitted to early adopt the standard “for annual financial statements that have not yet been issued or made available for issuance.” Adoption is either prospectively or retrospectively; the Company will adopt this ASU on a prospective basis. This ASU will likely result in additional disclosures.
In November 2024, the FASB issued ASU 2024-03 - Income Statement - Reporting Comprehensive Income - Expense Disaggregation Disclosures (Subtopic 220-40): Disaggregation of Income Statement Expenses. The ASU requires more detailed disclosures about the types of expenses in commonly presented expense captions such as cost of sales, selling, general and administrative expenses and research and development expenses. This includes separate footnote disclosure for expenses such as purchases of inventory, employee compensation, depreciation, and intangible asset amortization. Public business entities are required to apply the guidance prospectively and may apply it retrospectively. The ASU's amendments are effective for public business entities for annual periods beginning after December 15, 2026, and interim reporting periods beginning after December 15, 2027. Public business entities are required to apply the guidance prospectively and may apply it retrospectively. This ASU will likely result in additional disclosures.
In January 2025, the FASB issued ASU 2025-01 - Income Statement - Reporting Comprehensive Income - Expense Disaggregation Disclosures (Subtopic 220-40): Clarifying the Effective Date. The amendments in this ASU clarify that ASU 2024-03 is effective for public business entities for annual reporting periods beginning after December 15, 2026 and interim reporting periods within annual reporting periods beginning after December 15, 2027. This ASU will likely result in additional disclosures.
3. Fair Value Measurement and Marketable Securities
The Company applies fair value accounting for all financial assets and liabilities and non-financial assets and liabilities that are recognized or disclosed at fair value in the financial statements on a recurring basis. Fair value is an exit price, representing the amount that would be received to sell an asset or paid to transfer a liability in an orderly transaction between market participants. As such, fair value is a market-based measurement that should be determined based on assumptions that market participants would use in pricing an asset or liability. As a basis for considering such assumptions, a three-tier fair value hierarchy has been established, which prioritizes the inputs used in measuring fair value as follows:
Level 1—Observable inputs, such as quoted prices in active markets for identical assets or liabilities at the measurement date.
Level 2—Observable inputs other than Level 1 prices such as quoted prices for similar assets or liabilities, quoted prices in markets that are not active, or other inputs that are observable or can be corroborated by observable market data for substantially the full term of the assets or liabilities.
Level 3—Unobservable inputs which reflect management’s best estimate of what market participants would use in pricing the asset or liability at the measurement date. Consideration is given to the risk inherent in the valuation technique and the risk inherent in the inputs to the model.
In determining fair value, the Company utilizes valuation techniques that maximize the use of observable inputs and minimize the use of unobservable inputs to the extent possible as well as consider counterparty credit risk in its assessment of fair value.
As of March 31, 2025, financial assets measured and recognized at fair value are as follows (in thousands):
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
March 31, 2025 |
|
|
|
|
|
Amortized Cost |
|
|
Gross Unrealized Gains |
|
|
Gross Unrealized Losses |
|
|
Estimated Fair Value |
|
Assets |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
U.S. government securities |
|
Level 2 |
|
$ |
492,737 |
|
|
$ |
1,248 |
|
|
$ |
(85 |
) |
|
$ |
493,900 |
|
Corporate bonds |
|
Level 2 |
|
|
380,731 |
|
|
|
561 |
|
|
|
(126 |
) |
|
|
381,166 |
|
Commercial paper |
|
Level 2 |
|
|
84,457 |
|
|
|
— |
|
|
|
(14 |
) |
|
|
84,443 |
|
Subtotal |
|
|
|
|
957,925 |
|
|
|
1,809 |
|
|
|
(225 |
) |
|
|
959,509 |
|
Money market funds |
|
Level 1 |
|
|
84,521 |
|
|
|
— |
|
|
|
— |
|
|
|
84,521 |
|
Cash |
|
|
|
|
7,143 |
|
|
|
— |
|
|
|
— |
|
|
|
7,143 |
|
Total fair value of assets |
|
|
|
$ |
1,049,589 |
|
|
$ |
1,809 |
|
|
$ |
(225 |
) |
|
$ |
1,051,173 |
|
Included in cash and cash equivalents(1) |
|
|
|
|
130,004 |
|
|
|
— |
|
|
|
(8 |
) |
|
|
129,996 |
|
Included in marketable securities, current(2) |
|
|
|
|
561,567 |
|
|
|
989 |
|
|
|
(44 |
) |
|
|
562,512 |
|
Included in marketable securities, non-current(3) |
|
|
|
|
358,018 |
|
|
|
820 |
|
|
|
(173 |
) |
|
|
358,665 |
|
Total fair value of assets |
|
|
|
$ |
1,049,589 |
|
|
$ |
1,809 |
|
|
$ |
(225 |
) |
|
$ |
1,051,173 |
|
(1) $38.3 million of commercial paper was included in cash and cash equivalents on the condensed balance sheet due to securities with
purchase.
dates within 90 days of maturity dates.
(2) The Company’s short-term marketable securities mature in one year or less.
(3) The Company’s long-term marketable securities mature between one and three years.
As of December 31, 2024, financial assets measured and recognized at fair value are as follows (in thousands):
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
December 31, 2024 |
|
|
|
|
|
Amortized Cost |
|
|
Gross Unrealized Gains |
|
|
Gross Unrealized Losses |
|
|
Estimated Fair Value |
|
Assets |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
U.S. government securities |
|
Level 2 |
|
$ |
552,008 |
|
|
$ |
1,214 |
|
|
$ |
(353 |
) |
|
$ |
552,869 |
|
Corporate bonds |
|
Level 2 |
|
|
363,197 |
|
|
|
357 |
|
|
|
(419 |
) |
|
|
363,135 |
|
Commercial paper |
|
Level 2 |
|
|
89,109 |
|
|
|
21 |
|
|
|
(8 |
) |
|
|
89,122 |
|
Subtotal |
|
|
|
|
1,004,314 |
|
|
|
1,592 |
|
|
|
(780 |
) |
|
|
1,005,126 |
|
Money market funds |
|
Level 1 |
|
|
57,626 |
|
|
|
— |
|
|
|
— |
|
|
|
57,626 |
|
Cash |
|
|
|
|
19,399 |
|
|
|
— |
|
|
|
— |
|
|
|
19,399 |
|
Total fair value of assets |
|
|
|
$ |
1,081,339 |
|
|
$ |
1,592 |
|
|
$ |
(780 |
) |
|
$ |
1,082,151 |
|
Included in cash and cash equivalents(1) |
|
|
|
|
84,379 |
|
|
|
— |
|
|
|
(1 |
) |
|
|
84,378 |
|
Included in marketable securities, current(2) |
|
|
|
|
591,089 |
|
|
|
928 |
|
|
|
(76 |
) |
|
|
591,941 |
|
Included in marketable securities, non-current(3) |
|
|
|
|
405,871 |
|
|
|
664 |
|
|
|
(703 |
) |
|
|
405,832 |
|
Total fair value of assets |
|
|
|
$ |
1,081,339 |
|
|
$ |
1,592 |
|
|
$ |
(780 |
) |
|
$ |
1,082,151 |
|
(1) $7.4 million of commercial paper was included in cash and cash equivalents on the condensed balance sheet due to securities with
purchase.
dates within 90 days of maturity dates.
(2) The Company’s short-term marketable securities mature in one year or less.
(3) The Company’s long-term marketable securities mature between one and three years
As of March 31, 2025 and December 31, 2024, all marketable securities had a remaining maturity of less than three years. There were no financial liabilities measured and recognized at fair value as of March 31, 2025 and December 31, 2024.
As of March 31, 2025 and December 31, 2024, certain securities were held in an unrealized loss position. Based on review of the portfolio of marketable securities and the creditworthiness of the underlying issuer, the Company determined that the decline in fair value below cost did not result from credit-related factors. Additionally, the Company does not intend to sell these securities, nor will it be required to sell before recovery of the amortized cost basis at maturity. As a result, no credit related losses have been recognized for any of the periods presented.
4. Balance Sheet Components
Property and Equipment, Net
Property and equipment, net consisted of the following (in thousands):
|
|
|
|
|
|
|
|
|
|
Useful Life |
|
March 31, |
|
December 31, |
|
|
(In Years) |
|
2025 |
|
2024 |
|
Laboratory equipment |
5 |
|
$ |
13,982 |
|
$ |
13,513 |
|
Computer equipment |
3 |
|
|
503 |
|
|
503 |
|
Software |
3 |
|
|
231 |
|
|
231 |
|
Leasehold improvements |
Shorter of useful life or lease term |
|
|
4,938 |
|
|
4,913 |
|
Furniture and fixtures |
5 |
|
|
1,517 |
|
|
1,517 |
|
Total property and equipment |
|
|
|
21,171 |
|
|
20,677 |
|
Less: Accumulated depreciation and amortization |
|
|
|
(12,300 |
) |
|
(11,711 |
) |
Property and equipment, net |
|
|
$ |
8,871 |
|
$ |
8,966 |
|
Depreciation and amortization expense was $0.6 million and $0.6 million for the three months ended March 31, 2025 and March 31, 2024, respectively.
Accrued Liabilities
Accrued liabilities consisted of the following (in thousands):
|
|
|
|
|
|
|
|
|
|
|
March 31, |
|
|
December 31, |
|
|
|
2025 |
|
|
2024 |
|
Accrued research and development expenses |
|
$ |
25,982 |
|
|
$ |
19,956 |
|
Accrued salaries and benefits |
|
|
4,929 |
|
|
|
8,233 |
|
Legal and professional fees |
|
|
1,366 |
|
|
|
1,213 |
|
Other |
|
|
630 |
|
|
|
950 |
|
Accrued liabilities |
|
$ |
32,907 |
|
|
$ |
30,352 |
|
5. Operating Leases
In June 2023, the Company entered into a lease agreement for approximately 44,000 square feet of laboratory and office facilities at 5000 Shoreline Court, South San Francisco, California. The lease term is 120 months, and the Company has an option to extend the lease term for a total of two consecutive five-year periods. This lease agreement commenced in August 2024.
In May 2024, the Company amended its 5000 Shoreline Court facility lease agreement to expand the size of the original premises by adding approximately 11,321 rentable square feet of additional space. The amendment to the lease term commenced in January 2025.
The Company's lease at 7000 Shoreline Court, South San Francisco, California, expired in September 2024.
In November 2023, the Company entered into a lease agreement for approximately 5,700 square feet of space at 11710 El Camino Real, San Diego, California for corporate office space. The lease commenced in December 2023 and expires in March 2028. The Company has an option to renew the lease for three years.
Future minimum lease payments under operating leases included on the Company's condensed balance sheet are as follows:
|
|
|
|
|
As of March 31, 2025 |
|
Operating Leases |
|
2025 |
|
$ |
290 |
|
2026 |
|
|
1,944 |
|
2027 |
|
|
5,379 |
|
2028 |
|
|
5,248 |
|
2029 |
|
|
5,323 |
|
Thereafter |
|
|
28,464 |
|
Total future minimum lease payments |
|
|
46,648 |
|
Less: imputed interest |
|
|
(20,680 |
) |
Total operating lease liabilities |
|
$ |
25,968 |
|
The following table summarizes other information about the Company’s operating leases:
|
|
|
|
|
|
|
As of |
|
|
March 31, 2025 |
|
December 31, 2024 |
Remaining Lease Term |
|
9.3 |
|
9.4 |
Discount Rate |
|
11.5% |
|
12.6% |
Operating lease costs were $1.2 million and $0.5 million for the three months ended March 31, 2025 and March 31, 2024.
Variable lease costs were $0.6 million and $0.4 million for the three months ended March 31, 2025 and March 31, 2024. Variable lease costs represent additional costs incurred, related to administration, maintenance and property tax costs incurred, which are billed based on both usage and as a percentage of the Company’s share of total square footage.
During the three months ended March 31, 2025 and March 31, 2024, cash paid for amounts included in the measurement of lease liabilities were $0.1 million and $0.5 million, respectively.
6. Commitments and Contingencies
Contingencies
From time to time, the Company may be involved in litigation related to claims that arise in the ordinary course of its business activities. The Company accrues for these matters when it is probable that future expenditures will be made and these expenditures can be reasonably estimated. As of March 31, 2025, the Company does not believe that any such matters, individually or in the aggregate, will have a material adverse effect on the Company’s financial position, results of operations or cash flows.
Indemnification
The Company enters into standard indemnification arrangements in the ordinary course of business with vendors, clinical trial sites and other parties. Pursuant to these arrangements, the Company indemnifies, holds harmless and agrees to reimburse the indemnified parties for losses suffered or incurred by the indemnified party. The term of these indemnification agreements is generally perpetual any time after the execution of the agreement. The maximum potential amount of future payments the Company could be required to make under these arrangements is not determinable. The Company has never incurred costs to defend lawsuits or settle claims related to these indemnification agreements. Accordingly, the Company has not recorded a liability related to such indemnification agreements as of March 31, 2025.
7. Income Taxes
For the three months ended March 31, 2025 and March 31, 2024, the Company did not record a federal or state income tax provision due to its recurring net losses. In addition, the Company has taken a full valuation allowance against its net deferred tax assets as the Company believes it is not more likely than not that the benefit will be realized.
The Company is under audit in California for tax years 2020-2021.
8. Common Stock
As of March 31, 2025 and December 31, 2024, the Company’s certificate of incorporation authorized the Company to issue 300,000,000 shares of common stock at a par value of $0.0001 per share. Each share of common stock is entitled to one vote. The holders of common stock are also entitled to receive dividends whenever funds are legally available and when declared by the Company’s board of directors. As of March 31, 2025 and December 31, 2024, no dividends have been declared to date.
On July 11, 2024, the Company completed an underwritten public follow-on offering. The offering consisted of 8,355,714 shares of common stock at an offering price to the public of $35.00 per share, including 1,127,142 shares of common stock upon the exercise in full of the overallotment option by the underwriters, as well as pre-funded warrants to purchase 285,715 shares of common stock at a public offering price of $34.9999 per underlying share, in each case before underwriting discounts and commissions. Pursuant to the offering, the Company received aggregate gross proceeds of approximately $302.4 million, before deducting underwriting discounts and commissions and other offering expenses, resulting in net proceeds of approximately $283.8 million, after deducting underwriting discounts and commissions and other offering expenses.
As of March 31, 2025, the following aggregate warrants to purchase shares of the Company’s common stock were issued and outstanding:
|
|
|
|
|
|
|
|
|
|
|
Issue Date |
|
Expiration Date |
|
Exercise Price per Share |
|
|
Number of Shares subject to Outstanding Warrants |
|
July 11, 2024 |
|
None |
|
$ |
0.0001 |
|
|
|
285,715 |
|
October 27, 2023 |
|
None |
|
$ |
0.0001 |
|
|
|
319,150 |
|
April 27, 2023 |
|
None |
|
$ |
0.0001 |
|
|
270,270(1) |
|
(1) In September 2024, 1,750,000 shares of common stock subject to outstanding pre-funded warrants were
cashless exercised and 1,749,993 shares of common stock were issued.
The warrants are classified as a component of Stockholders’ Equity within Additional Paid-in-Capital. The warrants
are classified as equity because they are freestanding financial instruments that are legally detachable and separately exercisable from the equity instruments, are immediately exercisable, do not embody an obligation for the Company to repurchase its shares, are indexed to the Company’s common stock and meet the equity classification criteria. The warrants will not expire until they are fully exercised.
The Company had reserved common stock for future issuance as follows:
|
|
|
|
|
|
|
March 31, |
|
December 31, |
|
|
2025 |
|
2024 |
Exercise of outstanding options under the 2015, 2019 and 2023 Plans |
|
10,220,556 |
|
7,737,595 |
Shares available for grant under the 2019 Plan |
|
2,928,995 |
|
1,910,589 |
Shares available for grant under the 2023 Inducement Plan |
|
474,622 |
|
593,592 |
Shares available under the Employee Stock Purchase Plan |
|
2,776,046 |
|
1,911,011 |
Pre-funded warrants issued and outstanding |
|
875,135 |
|
875,135 |
Total |
|
17,275,354 |
|
13,027,922 |
9. Stock-Based Compensation
2023 Inducement Plan
On February 24, 2023, the Company adopted the IDEAYA Biosciences, Inc. 2023 Employment Inducement Award Plan (the “2023 Inducement Plan”), pursuant to which the Company reserved 1,000,000 shares of its common stock to be used exclusively for grants of awards to individuals who were not previously employees or directors of the Company as an inducement material to the individual’s entry into employment with the Company within the meaning of Rule 5635(c)(4) of the Nasdaq Listing Rules. The 2023 Inducement Plan was approved by the Company’s board of directors without stockholder approval in accordance with such rule. Options granted under the 2023 Inducement Plan have a term of 10 years and generally vest over a 4-year period with 1-year cliff vesting.
On June 25, 2024, the Company amended the 2023 Employment Inducement Award Plan, increasing the number of shares available for issuance by 1,000,000.
As of March 31, 2025, the number of shares available for issuance under the 2023 Inducement Plan was 474,622.
2019 Incentive Award Plan
In May 2019, the Company’s board of directors adopted and the Company’s stockholders approved the 2019 Incentive Award Plan (the “2019 Plan”), under which the Company may grant cash and equity-based incentive awards to the Company’s employees, consultants and directors. Following the effectiveness of the 2019 Plan, the Company will not make any further grants under the 2015 Equity Incentive Plan (the “2015 Plan”). However, the 2015 Plan continues to govern the terms and conditions of the outstanding awards granted under it. Shares of common stock subject to awards granted under the 2015 Plan that are forfeited or lapse unexercised and which following the effective date of the 2019 Plan are not issued under the 2015 Plan will be available for issuance under the 2019 Plan.
Options granted under the 2019 Plan may be either incentive stock options (“ISOs”) or nonqualified stock options (“NSOs”). ISOs may be granted only to Company employees (including officers and directors who are also employees). NSOs may be granted to Company employees, directors and consultants.
The 2019 Plan is subject to an annual increase on the first day of each year beginning in 2020 and ending in 2029, equal to the lesser of 4% of the shares outstanding on the last day of the immediately preceding fiscal year, and such smaller number of shares as determined by the Company’s board of directors. Options granted under the 2019 Plan have a term of 10 years (or five years if granted to a 10% stockholder) and generally vest over a 4-year period with 1-year cliff vesting.
As of March 31, 2025, the number of shares available for issuance under the 2019 Plan was 2,928,995.
2015 Equity Incentive Plan
In 2015, the Company established its 2015 Plan which provides for the granting of stock options to employees, directors and consultants of the Company. Options granted under the 2015 Plan may be either ISOs or NSOs.
2019 Employee Stock Purchase Plan
In May 2019, the Company’s board of directors adopted and the Company’s stockholders approved the 2019 Employee Stock Purchase Plan (the “ESPP”). The ESPP provides eligible employees with the opportunity to acquire an ownership interest in the Company through periodic payroll deductions up to 15% of eligible compensation. The offering period is determined by the Company in its discretion but may not exceed 27 months. The per-share purchase price on the applicable exercise date for an offering period is equal to the lesser of 85% of the fair market value of the common stock at either the first business day or last business day of the offering period, provided that no more than 4,000 shares of common stock may be purchased by any one employee during each offering period.
The ESPP is intended to constitute an “employee stock purchase plan” under Section 423(b) of the Internal Revenue Code of 1986, as amended. A total of 195,000 shares of common stock were initially reserved for issuance under the ESPP, subject to an annual increase on January 1 of each year, beginning on January 1, 2020, equal to the lesser of 1% of the shares outstanding on the last day of the immediately preceding fiscal year and such smaller number of shares as may be determined by the Company’s board of directors, provided, however, that no more than 2,500,000 shares may be issued under the ESPP.
As of March 31, 2025, the number of shares available for issuance under the ESPP was 2,776,046. For the three months ended March 31, 2025 and March 31, 2024, the Company recorded $0.2 million and $0.1 million, respectively, of compensation expense related to employee participation in the ESPP.
Stock-Based Compensation Expense
Total stock-based compensation expense recorded related to awards granted to employees and non-employees was as follows (in thousands):
|
|
|
|
|
|
|
|
|
|
Three Months Ended March 31, |
|
|
|
2025 |
|
2024 |
|
Research and development |
|
$ |
6,020 |
|
$ |
3,784 |
|
General and administrative |
|
|
4,217 |
|
|
2,528 |
|
Total stock-based compensation expense |
|
$ |
10,237 |
|
$ |
6,312 |
|
Stock Options
Activity under the Company’s 2015 and 2019 Plans and 2023 Inducement Plan is set forth below:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Outstanding Options |
|
|
|
|
|
|
|
|
|
Shares |
|
|
Weighted- Average Exercise Price |
|
|
Weighted- Average Remaining Contractual Term (Years) |
|
|
Aggregate Intrinsic Value (Millions) |
|
Balance, January 1, 2025 |
|
|
7,737,595 |
|
|
$ |
26.06 |
|
|
|
7.89 |
|
|
$ |
43.01 |
|
Options granted |
|
|
2,673,758 |
|
|
$ |
20.29 |
|
|
|
|
|
|
|
Options exercised |
|
|
(77,743 |
) |
|
$ |
14.59 |
|
|
|
|
|
|
|
Options canceled |
|
|
(113,054 |
) |
|
$ |
26.10 |
|
|
|
|
|
|
|
Balance, March 31, 2025 |
|
|
10,220,556 |
|
|
$ |
24.64 |
|
|
|
8.27 |
|
|
$ |
9.52 |
|
Exercisable as of March 31, 2025 |
|
|
3,898,336 |
|
|
$ |
20.09 |
|
|
|
6.81 |
|
|
$ |
8.59 |
|
Vested and expected to vest as of March 31, 2025 |
|
|
10,220,556 |
|
|
$ |
24.64 |
|
|
|
8.27 |
|
|
$ |
9.52 |
|
The weighted-average grant-date fair value of options granted during the three months ended March 31, 2025 and March 31, 2024 was $14.07 and $33.00 per share, respectively. The aggregate intrinsic value of options exercised for the three months ended March 31, 2025 and March 31, 2024 was $0.6 million and $14.5 million, respectively. Intrinsic values are calculated as the difference between the exercise price of the underlying options and the fair value of the common stock on the date of exercise.
As of March 31, 2025 and December 31, 2024, total unrecognized stock-based compensation expense for stock options was $115.7 million and $90.2 million, respectively, which is expected to be recognized over a weighted-average period of 2.90 years and 2.63 years, respectively.
Black-Scholes Assumptions
The fair values of options were calculated using the assumptions set forth below:
|
|
|
|
|
|
|
Three Months Ended March 31, |
|
|
2025 |
|
2024 |
Expected term |
|
6.1 years |
|
6.1 years |
Expected volatility |
|
75.7% - 76.3% |
|
80.2% - 81.3% |
Risk-free interest rate |
|
4.1% - 4.4% |
|
4.0% - 4.3% |
Dividend yield |
|
0% |
|
0% |
Expected term. The expected term represents the weighted-average period the stock options are expected to remain outstanding and is based on the options’ vesting terms and contractual terms.
Expected Volatility. The expected volatility is based on the Company’s historical stock price volatility. The historical stock price volatility is calculated based on a period of time commensurate with the expected term assumption for each grant.
Risk-Free Interest Rate. The risk-free rate assumption is based on U.S. Treasury instruments whose term was consistent with the expected term of the Company’s stock options.
Expected Dividend Rate. The Company has not paid and does not anticipate paying any dividends in the near future. Accordingly, the Company has estimated the dividend yield to be zero.
The Company accounts for forfeitures as they occur.
Fair Value of Common Stock
The fair value of the Company’s common stock is determined based on the market price on the date of grant.
10. Significant Agreements
GSK Collaboration, Option and License Agreement
In June 2020, the Company entered into the Collaboration, Option and License Agreement (the “GSK Collaboration Agreement”), with an affiliate of GSK plc, GLAXOSMITHKLINE INTELLECTUAL PROPERTY (NO. 4) LIMITED (“GSK”), pursuant to which the Company and GSK have entered into a collaboration for its synthetic lethality programs targeting MAT2A, Pol Theta and Werner Helicase (“Werner” or “WRN”).
Pursuant to the GSK Collaboration Agreement, GSK paid the Company $100.0 million on July 31, 2020. As of March 31, 2025, GSK has made aggregate payments in the amount of $20.0 million for the achievement of certain development and regulatory milestones with respect to Pol Theta and WRN products.
GSK Collaboration - Pol Theta Program
Pursuant to the GSK Collaboration Agreement, GSK holds a global, exclusive license to develop and commercialize Pol Theta products arising out of the Pol Theta program. The Company and GSK collaborated on preclinical research for the Pol Theta program, and GSK is leading clinical development of IDE705 (GSK101) for the Pol Theta program. GSK is responsible for all research and development costs for the Pol Theta program.
In August 2023, the Company earned a $7.0 million payment for a milestone based on acceptance of the IND by the FDA. An earlier preclinical development $3.0 million milestone payment from GSK was achieved in August 2022 in connection with ongoing IND-enabling studies to support the evaluation of IDE705 (GSK101).
The Company has the potential to achieve an additional $10.0 million development milestone upon initiation of Phase 1 clinical dose expansion, as well as potential further aggregate late-stage development and regulatory milestones of up to $465.0 million. Upon commercialization, the Company will be eligible to receive commercial milestones of up to $475.0 million, and tiered royalties on global net sales of GSK101 – ranging from high single-digit to sub-teen double-digit percentages, subject to certain customary reductions.
GSK Collaboration - Werner Helicase Program
Pursuant to the GSK Collaboration Agreement, GSK holds a global, exclusive license to develop and commercialize WRN products arising out of the WRN program. The Company and GSK collaborated on preclinical research for the WRN program, and GSK is sponsoring and leading clinical development of IDE275 (GSK959) for the WRN program, with the Company responsible for 20% and GSK responsible for 80% of such global research and development costs. The cost-sharing percentages will be adjusted based on the actual ratio of U.S. to global profits for WRN products, as measured three and six years after global commercial launch thereof
In October 2023, the Company earned a $3.0 million milestone from GSK in connection with IND-enabling studies. In October 2024, the Company earned a $7.0 million milestone payment for the IND clearance of IDE275 (GSK 959) to enable clinical evaluation.
The Company has the potential to earn up to an additional $10.0 million development milestone upon initiation of Phase 1 clinical dose expansion, as well as potential further aggregate late-stage development and regulatory milestones of up to $465.0 million.
Upon commercialization, the Company will be eligible to receive commercial milestones of up to $475.0 million, 50% of U.S. net profits and tiered royalties on global non-U.S. net sales of the Werner Helicase Inhibitor DC – ranging from high single-digit to sub-teen double-digit percentages, subject to certain customary reductions.
Novartis License Agreement
In September 2018, the Company entered into a License Agreement with Novartis to develop and commercialize Novartis’ LXS196 (also known as IDE196), a Phase 1 protein kinase C (“PKC”) inhibitor, for the treatment of cancers having GNAQ and GNA11 mutations. The Company renamed Novartis’ LXS196 oncology as IDE196, and which has a non-proprietary name of darovasertib. Under the license agreement, Novartis granted to the Company a worldwide, exclusive, sublicensable license to research, develop, manufacture, and commercialize certain defined compounds and products, including IDE196 and certain other PKC inhibitors, as well as companion diagnostic products, collectively referred to as the licensed products, for any purpose. The Company paid Novartis an upfront payment of $2.5 million and issued 263,615 shares of its Series B redeemable convertible preferred stock concurrently with the execution of the license agreement.
In March 2025, the FDA granted Breakthrough Therapy designation (“BTD”) for darovasertib, a potential first-in-class protein kinase C (“PKC”) inhibitor, for the neoadjuvant treatment of adult patients with primary uveal melanoma (“UM”) for whom enucleation has been recommended. Under the license agreement with Novartis, the Company paid Novartis a $1.0 million milestone payment in April 2025.
Subject to completion of certain clinical and regulatory development milestones, the Company agreed to make additional milestone payments in the aggregate of up to $8.0 million, and subject to achievement of certain commercial sales milestones, the Company agreed to make milestone payments in the aggregate of up to $20.0 million. The Company also agreed to pay mid to high single-digit tiered royalty payments based on annual worldwide net sales of licensed products, payable on a licensed product-by-licensed product and country by country basis until the latest of the expiration of the last to expire exclusively licensed patent, the expiration of regulatory exclusivity, and the ten year anniversary of the first commercial sale of such product in such country. The royalty payments are subject to reductions for lack of patent coverage, loss of market exclusivity, and payment obligations for third-party licenses.
The Company owns or controls all commercial rights in its darovasertib program in UM, including in MUM and in primary UM, subject to certain economic obligations pursuant to its exclusive, worldwide license to darovasertib with Novartis.
Pfizer Clinical Trial Collaboration and Supply Agreements
In March 2020, the Company entered into a Clinical Trial Collaboration and Supply Agreement with Pfizer, Inc. (as amended in September 2020, April 2021, September 2021 and May 2023 (the “Pfizer Agreement”). Pursuant to the Pfizer Agreement, Pfizer supplies the Company with their MEK inhibitor, binimetinib, and their cMET inhibitor, crizotinib, to evaluate combinations of darovasertib independently with each of the Pfizer compounds, in patients with tumors harboring activating GNAQ or GNA11 mutations. Under the Pfizer Agreement, the Company is the sponsor of the combination studies and will provide darovasertib and pay for the costs of the combination studies. Pfizer will provide binimetinib and crizotinib for use in the clinical trial at no cost to the Company. The Pfizer Agreement provides that the Company and Pfizer will jointly own clinical data generated from the clinical trial and will also jointly own inventions, if any, relating to the combined use of darovasertib and binimetinib, or independently, to the combined use of darovasertib and crizotinib. The Company and Pfizer have formed a joint development committee responsible for coordinating all regulatory and other activities under the agreement.
In March 2022, the Company and Pfizer entered into a Second Clinical Trial Collaboration and Supply Agreement, as amended in May 2023 (the “Second Pfizer Agreement”), pursuant to which the Company is evaluating darovasertib and crizotinib as a combination therapy in MUM in a planned Phase 2/3 potential registration-enabling clinical trial. Pursuant to the Second Pfizer Agreement, the Company is the sponsor of the combination trial and the Company will provide darovasertib and pay for the costs of the combination trial, and Pfizer will provide crizotinib for the planned combination trial at no cost to the Company for up to an agreed-upon number of MUM patients. The Company and Pfizer will jointly own clinical data from the planned combination trial and all inventions relating to the combined use of darovasertib and crizotinib. The Company and Pfizer have formed a joint development committee responsible for coordinating all regulatory and other activities under the Second Pfizer Agreement.
Separately, in March 2022, the Company and Pfizer also entered into a Third Clinical Trial Collaboration and Supply Agreement (the “Third Pfizer Agreement”), pursuant to which the Company could, subject to preclinical validation and FDA feedback and guidance, evaluate darovasertib and crizotinib, as a combination therapy in
cMET-driven tumors such as NSCLC and/or HCC in a Phase 1 clinical trial. Pursuant to the Third Pfizer Agreement, the Company was the sponsor of the planned combination trial, and the Company would provide darovasertib and pay for the costs of the combination trial. Pfizer would provide crizotinib for the planned combination trial at no cost to the Company. Pursuant to Amendment No. 1 to the Second Pfizer Agreement, as described below, the Company and Pfizer terminated the Third Pfizer Agreement.
In May 2023, the Company continued its relationship with Pfizer by entering into Amendment No. 4 to the Pfizer Agreement relating to the supply of crizotinib in support of this Phase 2 clinical trial, pursuant to which Pfizer will continue to provide the Company with an additional defined quantity of crizotinib at no cost.
Also, in May 2023, the Company expanded its relationship with Pfizer to support the Phase 2/3 registrational trial to evaluate darovasertib and crizotinib as a combination therapy in MUM by entering into Amendment No. 1 to the Second Pfizer Agreement. Under Amendment No. 1 to the Second Pfizer Agreement, Pfizer will provide the Company with a first defined quantity of crizotinib at no cost, as well as an additional second defined quantity of crizotinib at a lump-sum cost. The Third Pfizer Agreement was terminated by the Company and Pfizer under Amendment No. 1 to the Second Pfizer Agreement.
In December 2024, the Company entered into Amendment No. 5 to the Pfizer Agreement for the supply of crizotinib in the Phase 1/2 clinical trial for Pfizer to provide us a defined quantity of crizotinib at defined costs.
Cancer Research UK and University of Manchester Exclusive Option and License Agreement
The Company entered into an exclusive license under the Evaluation, Option and License Agreement with Cancer Research Technologies Ltd., also known as Cancer Research United Kingdom Ltd. (“CRT”), and the University of Manchester, pursuant to which the Company holds an exclusive worldwide license rights covering a broad class of PARG inhibitors.
In January 2022, the Company exercised its option for an exclusive worldwide license covering a broad class of poly (ADP-ribose) glycohydrolase (“PARG”), inhibitors from CRT, and the University of Manchester, and in connection therewith, paid a one-time option exercise fee of £250,000.
In April 2023, the Company incurred an obligation to pay milestone payments in an aggregate amount of £750,000 to CRT based upon the achievement of certain milestones relating to first and second tumor histologies in connection with the Phase 1 portion of the IDE161-001 Phase 1/2 clinical trial in oncologic diseases.
The Company will be obligated to make additional payments to CRT aggregating up to £18.75 million upon the achievement of specific development and regulatory approval events for development of a PARG inhibitor in oncologic diseases, including an aggregate of up to £1.5 million and up to £2.25 million for the achievement of certain Phase 2 and Phase 3 development milestones, respectively, in each case as relating to first and second tumor histologies.
The Company will also pay low single-digit tiered royalties, and potentially also sales-based milestones, to CRT based on net sales of licensed products. In addition, in the event the Company sublicenses the intellectual property, it will also be obligated to pay CRT a specified percentage of any sublicense revenue.
Amgen Clinical Trial Collaboration and Supply Agreement
In July 2022, the Company entered into a clinical trial collaboration and supply agreement with Amgen Inc. (the “Amgen CTCSA”), to clinically evaluate IDE397 in combination with AMG 193, the Amgen investigational MTA-cooperative PRMT5 inhibitor, in patients having MTAP-null solid tumors, in a Phase 1/2 clinical trial. In February 2025, the Company and Amgen mutually agreed to wind down the IDE397 and AMG 193 clinical combination study and will not pursue dose expansion.
Gilead Clinical Study Collaboration and Supply Agreement
In November 2023, the Company entered into a Clinical Study Collaboration and Supply Agreement with Gilead Sciences, Inc. (“Gilead”), (the “Gilead CSCSA”), to clinically evaluate IDE397 in combination with Trodelvy (sacituzumab govitecan-hziy), a Trop-2 directed antibody drug conjugate (“ADC”), in patients having MTAP-deletion urothelial cancer (“UC”), in a Phase 1 clinical trial.
In February 2025, the Company expanded its clinical study collaboration and entered into an additional Clinical Study Collaboration and Supply Agreement with Gilead (the “Second Gilead CSCSA”), to evaluate the IDE397 and Trodelvy combination in MTAP-deletion NSCLC.
The Company is the study sponsor and Gilead will provide the supply of Trodelvy. Gilead will bear internal or external costs incurred in connection with its supply of Trodelvy. The Company will bear all internal and external costs and expenses associated with the conduct of the combination study. The Company and Gilead each retain commercial rights to its respective compounds, including with respect to use as a monotherapy agent or combination agent.
Merck Clinical Trial Collaboration and Supply Agreement
In March 2024, the Company entered into a Clinical Trial Collaboration and Supply Agreement (“Merck CTCSA”), with Merck (known as MSD outside of the United States and Canada) to evaluate the combination of IDE161 with Merck’s anti-PD-1 therapy, KEYTRUDA® (pembrolizumab), in patients with high microsatellite instability (“MSI-High”) and microsatellite stable (“MSS”) endometrial cancer. Pursuant to the Merck CTCSA, the Company is the sponsor of the combination study, and the Company will provide the IDE161 compound and pay for the costs of the combination study. Merck will provide KEYTRUDA at no cost to the Company. The Company and Merck will jointly own clinical data from the combination. Each party retains commercial rights to its respective compounds, including with respect to use as a monotherapy or combination agent.
Biocytogen Option and License Agreement
In July 2024, the Company entered into an Option and License Agreement (the “Biocytogen Option and License Agreement”), pursuant to which Biocytogen Pharmaceuticals (Beijing) Co., Ltd. (“Biocytogen”), granted us an option for an exclusive worldwide license from Biocytogen to develop and commercialize products in connection with a potential first-in-class B7H3/PTK7 topoisomerase-I-inhibitor-payload BsADC program (the “Option”). Under the terms of the Biocytogen Option and License Agreement, the Company paid Biocytogen an upfront fee and an exercise fee totaling $6.5 million upon exercise of the option. The Company is targeting an IND submission to the FDA in the second half of 2025 for IDE034, subject to satisfactory completion of ongoing preclinical and IND-enabling studies.
Biocytogen is eligible to receive additional development and regulatory milestone payments and commercial milestone payments, as well as low to mid single-digit royalties on net sales. Total potential milestone payments equal an aggregate of $400.0 million, including development and regulatory milestone payments of up to $100.0 million. The Company's royalty obligations continue with respect to each country and each product until the later of (i) the date on which such product is no longer covered by certain intellectual property rights in such country and (ii) the 10th anniversary of the first commercial sale of such product in such country.
Hengrui Pharma License Agreement
In December 2024, the Company entered into an exclusive License Agreement (the “Hengrui Pharma License Agreement”) with Jiangsu Hengrui Pharmaceuticals Co., Ltd. (“Hengrui Pharma”), pursuant to which Hengrui Pharma granted the Company an exclusive worldwide license outside of Greater China, for IDE849 (SHR-4849), a potential first-in-class Phase 1 DLL3 TOP1i ADC. In April 2025, the Company received U.S. IND clearance for the initiation of a Phase 1 clinical trial to evaluate IDE849 in solid tumors.
Under the terms of the Hengrui Pharma License Agreement, Hengrui Pharma is eligible to receive upfront and milestone payments totaling $1.045 billion, including a $75.0 million upfront fee, up to $200.0 million in development and regulatory milestone payments, plus commercial success-based milestones. Hengrui Pharma is
also eligible to receive mid-single to low-double digit royalties on net sales outside of Greater China. The Company owns or controls all commercial rights outside of greater China for IDE849.
11. Revenue Recognition
The Company recognizes revenue in accordance with ASC 606 for the GSK Collaboration Agreement (see Note 10, Significant Agreements).
Disaggregation of Revenue
The Company recognized no revenue for the three months ended March 31, 2025 and March 31, 2024.
The Company identified the following six performance obligations associated with the GSK Collaboration Agreement:
(i) Preclinical and Phase 1 Monotherapy clinical research and development services under the MAT2A program (“MAT2A R&D Services”)
(ii) Preclinical research services and the related license to IDEAYA-owned technology under the Pol Theta program (“Pol Theta R&D Services”)
(iii) Preclinical research services and the related license to IDEAYA-owned technology under the WRN program (“WRN R&D Services”)
(iv) Material right associated with the option to license IDEAYA-owned technology under the MAT2A program (“Option”)
(v) Material right associated with the option to license to IDEAYA-owned technology under the MAT2A program to the extent necessary for preclinical activities in preparation for the MAT2A Combination Trial (“Preclinical MAT2A License”)
(vi) Material right associated with the supply of MAT2A product for the MAT2A Combination Trial (“MAT2A Supply”)
The Company completed all performance obligations related to the upfront payment under the GSK Collaboration Agreement as of December 31, 2023. Since December 31, 2023, the Company has no accounts receivable and no contract liabilities related to the GSK Collaboration Agreement. Because the Company completed all performance obligations, future collaboration revenue recognized under the GSK Collaboration Agreement is only related to milestone payments as they are earned.
For the Pol Theta product, the Company achieved and earned a $7.0 million payment for a milestone in August 2023 based on acceptance of the IND by the FDA. An earlier preclinical development $3.0 million milestone payment from GSK was achieved in August 2022 in connection with ongoing IND-enabling studies to support evaluation of IDE705 (GSK101). The Company has the potential to receive an additional $10.0 million milestone payment upon initiation of Phase 1 clinical dose expansion.
For the WRN product, the Company achieved and earned a $7.0 million payment for a milestone in October 2024 based on the acceptance of the IND by the FDA. An earlier preclinical development $3.0 million payment from GSK was achieved in October 2023 in connection with IND-enabling studies for IDE275 (GSK959). The Company has the potential to receive an additional $10.0 million milestone payment upon initiation of Phase 1 clinical dose expansion.
Significant Judgments
In applying ASC 606 to the GSK Collaboration Agreement, the Company made the following judgment that significantly affect the timing and amount of revenue recognition:
(i) Determination of the transaction price, including whether any variable consideration is included at inception of the contract
The transaction price is the amount of consideration that the Company expects to be entitled to in exchange for transferring promised goods or services to the customer. The transaction price must be determined at inception of a contract and may include amounts of variable consideration. However, there is a constraint on inclusion of variable consideration in the transaction price, if there is uncertainty at inception of the contract as to whether such consideration will be recognized in the future.
The decision as to whether or not it is probable that a significant reversal of revenue will occur in the future, depends on the likelihood and magnitude of the reversal and is highly susceptible to factors outside the Company’s influence (for example, the Company cannot determine the outcome of clinical trials; the Company cannot determine if or when the counterparty will initiate or complete clinical trials; and the Company cannot determine if or when an regulatory agency provides any approval). In addition, the uncertainty is not expected to be resolved for a long period and finally, the Company has limited experience in the field. Therefore, at inception of the GSK Collaboration Agreement, development and regulatory milestones were fully constrained and were not included in the transaction price based on the factors noted above.
The Company constrains estimates of other variable consideration, such as reimbursable program costs, to amounts that are not expected to result in a significant revenue reversal in the future. The Company re-evaluates the transaction price, including the estimated variable consideration included in the transaction price and all constrained amounts, in each reporting period and as uncertain events are resolved or other changes in circumstances occur.
12. Net Loss Per Share Attributable to Common Stockholders
The following table sets forth the computation of basic and diluted net loss per share attributable to common stockholders (in thousands, except share and per share data):
|
|
|
|
|
|
|
|
|
|
Three Months Ended March 31, |
|
|
|
2025 |
|
2024 |
|
Numerator: |
|
|
|
|
|
Net loss attributable to common stockholders |
|
$ |
(72,178 |
) |
$ |
(39,572 |
) |
Denominator: |
|
|
|
|
|
Weighted-average common shares outstanding, basic and diluted(1) |
|
|
88,356,335 |
|
|
75,108,484 |
|
Net loss per share attributable to common stockholders, basic and diluted |
|
$ |
(0.82 |
) |
$ |
(0.53 |
) |
(1) The shares underlying the pre-funded warrants to purchase shares of the Company’s common stock have been included in the
calculation of the weighted-average number of shares outstanding, basic and diluted, for the three months ended March 31, 2025.
The following outstanding shares of potentially dilutive securities were excluded from the computation of diluted net loss per share attributable to common stockholders for the periods presented because including them would have been antidilutive:
|
|
|
|
|
|
|
As of March 31, |
|
|
2025 |
|
2024 |
Options to purchase common stock |
|
10,220,556 |
|
7,874,924 |
Total |
|
10,220,556 |
|
7,874,924 |
13. Segment Information
The Company operates and manages its business as one operating and reportable segment, which is the business of research and development for oncology-focused precision medicine. The Company’s chief operating decision maker (“CODM”) is its President and CEO. The Company’s measure of segment profit or loss is net income. For purposes of evaluating performance and allocating resources, the CODM reviews the financial information and evaluates net income against comparable prior periods and the Company’s forecast. All of the Company's long-lived assets are located in the United States.
In addition to the significant expense categories included within net income presented on the Company's condensed statements of operations and comprehensive loss, see below for disaggregated research and development expenses:
|
|
|
|
|
|
|
|
|
|
Three Months Ended |
|
|
|
March 31, 2025 |
|
March 31, 2024 |
|
External clinical development expenses(1): |
|
|
|
|
|
Darovasertib |
|
$ |
23,018 |
|
$ |
10,869 |
|
IDE397(2) |
|
|
3,741 |
|
|
2,926 |
|
IDE161 |
|
|
2,448 |
|
|
2,695 |
|
Personnel related and stock-based compensation |
|
|
15,814 |
|
|
12,254 |
|
Other research and development expenses(3) |
|
|
25,865 |
|
|
14,061 |
|
Total research and development expenses |
|
$ |
70,886 |
|
$ |
42,805 |
|
(1) External clinical development expenses include manufacturing and clinical trial costs. These expenses are primarily for services
provided by external consultants, CMOs and CROs.
(2) IDE397 includes costs from the Amgen CTCSA
(3) Other research and development expenses include manufacturing and clinical trial costs for preclinical and earlier clinical stage
programs. These expenses are primarily for services provided by external consultants, CMOs and CROs.
14. Subsequent Events
The Company evaluated all subsequent events from March 31, 2025 through the filing date of this Quarterly Report on Form 10-Q and determined there were no significant events that required recognition or disclosure.
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations.
The following discussion and analysis of our financial condition and results of operations should be read in conjunction with our financial statements and related notes appearing elsewhere in this Quarterly Report on Form 10-Q. Some of the information contained in this discussion and analysis or set forth elsewhere in this Quarterly Report on Form 10-Q, including information with respect to our plans and strategy for our business, includes forward-looking statements that involve risks and uncertainties. As a result of many factors, including those factors set forth in the “Risk Factors” section of this Quarterly Report on Form 10-Q, our actual results could differ materially from the results described, in or implied, by these forward-looking statements. Please also see the section of this Quarterly Report on Form 10-Q titled “Note Regarding Forward-Looking Statements.”
Overview
We are a precision medicine oncology company committed to the discovery and development of targeted therapeutics for patient populations selected using molecular diagnostics. Our approach integrates small molecule drug discovery with extensive capabilities in identifying and validating translational biomarkers to develop targeted therapies for select patient populations that are most likely to benefit from these targeted therapies. Our small molecule drug discovery expertise includes discovery and development of small molecule therapeutics. We are applying these capabilities and approach to develop a robust pipeline in precision medicine oncology.
Our clinical pipeline includes six potential first-in-class clinical-stage product candidates – darovasertib (PKC), IDE397 (MAT2A), IDE849 (DLL3), IDE275 / GSK959 (Werner Helicase), IDE161 (PARG), and IDE705 / GSK101 (Pol Theta Helicase). We own or control all commercial rights of three of these product candidates: darovasertib, IDE397, and IDE161, and own or control all commercial rights outside of greater China for IDE849. We are also advancing several development candidates, including IDE892, a potential best-in-class MTA-cooperative PRMT5 inhibitor for which we are targeting an investigational new drug, or IND, filing in mid-year 2025; IDE034, a potential first-in-class B7H3/PTK7 topoisomerase-I-inhibitor-payload bispecific antibody drug conjugate, or BsADC, program for which we are targeting an IND filing in the second half of 2025; and IDE574, a potential first-in-class KAT6/7 dual inhibitor program for which we are targeting an IND filing in the second half of 2025. We also have multiple earlier-stage preclinical programs. We have established selective, value-accretive collaborations with leading pharmaceutical companies to support our clinical development activities.
Darovasertib – PKC Inhibitor Clinical Candidate in Uveal Melanoma (UM)
Darovasertib (IDE196) is our most advanced clinical-stage product candidate, which we in-licensed from Novartis. Darovasertib is a potent, selective small molecule inhibitor of protein kinase C, or PKC, which we are developing for genetically defined cancers having GNAQ or GNA11 gene mutations. PKC is a protein kinase that functions downstream of the GTPases GNAQ and GNA11.
We have enrolled over 300 patients as of May 5, 2025, and have opened multiple clinical sites, including international sites, in our potential registration-enabling Phase 2/3 clinical trial, designated as IDE196-002. The purpose of the clinical trial is to evaluate darovasertib in combination with crizotinib, Pfizer’s investigational cMET inhibitor, in patients having metastatic uveal melanoma, or MUM, with human leukocyte antigen-, or HLA-A*02:01 negative, or HLA-A2(-), serotype, as part of a second Clinical Trial Collaboration and Supply Agreement, or the Second Pfizer Agreement, with Pfizer.
In December 2024, we announced the recommendation of a move-forward dose and the completion of the Part 2a dose optimization for the potential registration-enabling Phase 2/3 trial evaluating the combination of darovasertib and crizotinib in the first-line, or 1L, setting in patients with HLA-A2(-) MUM. We are continuing enrollment in Part 2b with the selected optimal dose.
We are targeting a median progression free survival, or PFS, readout for the Phase 2/3 registration-enabling trial of the darovasertib and crizotinib combination in 1L HLA-A2(-) MUM by year-end 2025.
We are enrolling additional HLA-A*02:01 positive, or HLA-A2(+), patients as an independent clinical strategy to address HLA-A2(+) MUM patients, in our ongoing Phase 2 clinical trial, designated as IDE196-001.
We are targeting a median overall survival, or OS, readout from our Phase 2 clinical trial, designated as IDE196-001, in over 40 1L MUM patients at a medical conference in the second half of 2025. The readout will include both 1L HLA-A2(-) and HLA-A2(+) MUM patients.
We are also supporting evaluation of darovasertib as single-agent neoadjuvant and adjuvant therapy in primary uveal melanoma, or UM, in an ongoing investigator-sponsored clinical trial, or IST, captioned as “Neoadjuvant / Adjuvant trial of Darovasertib in Ocular Melanoma,” or NADOM, led by St. Vincent’s Hospital in Sydney with the participation of Alfred Health and the Royal Victorian Eye and Ear Hospital in Melbourne.
We enrolled 95 patients as of December 31, 2024 in our Phase 2 clinical trial, designated as IDE196-009, evaluating darovasertib as single-agent neoadjuvant and adjuvant therapy in patients having primary uveal melanoma, or UM, with ongoing enrollment and multiple clinical sites open. We are targeting two clinical data updates from this Phase 2 clinical trial to be presented at medical conferences in mid-2025 and the second half of 2025. The mid-2025 update will be focused on vision data and the plaque brachytherapy patients, and the update in the second half of 2025 is expected to include over 90 UM patients from both the enucleation and plaque brachytherapy eligible cohorts.
In September 2024, we announced interim clinical data from the ongoing Phase 2 Company-sponsored trial and provided a regulatory update on a potential Phase 3 registration-enabling clinical trial in neoadjuvant UM patients based on a Type C meeting held with the U.S. Food and Drug Administration, or FDA. In April 2025, we provided a further regulatory update on the potential Phase 3 registrational trial design for darovasertib in neoadjuvant UM patients based on a Type D meeting held with the FDA. Based on the Type D meeting, we currently project approximately 520 patients will be randomized 2:1 to receive darovasertib or control in two cohorts. The two cohorts include enucleation-eligible UM patients (n=120) and plaque brachytherapy, or PB,-eligible UM patients (n=400). Primary endpoints, which are supportive of full approval based on the FDA Type D Meeting, include eye preservation rate for enucleation patients and proportion of patients with best corrected visual acuity 15-letter loss from time of randomization and time of completion of PB for the PB cohort, with event-free survival (EFS) as a required secondary endpoint for both cohorts. Commencement of the Phase 3 registration-enabling trial for darovasertib in neoadjuvant UM is targeted for the first half of 2025.
In June 2024, we announced interim clinical data from the ongoing investigator-sponsored Phase 2 trial of darovasertib as neoadjuvant/adjuvant treatment in UM, which was included in an oral presentation at the American Society of Clinical Oncology, or ASCO, 2024 Annual Meeting, and preliminary clinical data from our Phase 2 trial of darovasertib for neoadjuvant UM.
ASCO Clinical Data from Investigator-Sponsored Phase 2 Trial
In our ongoing investigator-sponsored Phase 2 trial of darovasertib as neoadjuvant/adjuvant treatment in UM, 15 patients planned for enucleation with localized UM were treated twice daily with a 300 mg dose of darovasertib in the Phase 2 investigator-sponsored clinical trial as of May 14, 2024. An initial safety cohort of three patients was treated for one month, and the remaining 12 patients were treated in an expansion cohort for up to six months with darovasertib as neoadjuvant treatment prior to their primary intervention (enucleation, plaque brachytherapy or external beam radiotherapy, or EBRT) across three Australian centers. As of May 14, 2024, 13 patients had completed neoadjuvant darovasertib treatment, 11 patients received adjuvant darovasertib treatment after primary treatment of UM, with five patients completing the planned six months of therapy. As of May 14, 2024, 75% (nine out of 12 enucleation patients) had confirmed preservation of the eye, by conversion from planned enucleation to plaque brachytherapy or EBRT, and approximately 67% (eight out of 12 enucleation patients) observed greater than 30% tumor shrinkage (maximum tumor volume change) after six months. Median tumor shrinkage (maximum tumor volume change) in the 12 enucleation patients was approximately 47% after six months. The darovasertib monotherapy neoadjuvant treatment had a manageable adverse event, or AE, profile with no drug-related serious adverse events, or SAEs, observed in the investigator-sponsored Phase 2 trial. Drug-related AEs in the trial were predominantly Grade 1 or Grade 2, and 20% of patients reported at least one drug-related Grade 3 AE.
Company-Sponsored Phase 2 Trial
In September 2024, we provided a clinical data update in which we observed encouraging clinical activity in our Phase 2 Company-sponsored trial. Collectively with the IST trial, the clinical efficacy data from the Phase 2 Company-sponsored trial substantiate clinical proof of concept for the use of darovasertib in the neoadjuvant uveal melanoma setting. The Phase 2 Company-sponsored trial used a data cutoff date of August 15, 2024, with an enrollment cutoff date of May 13, 2024.
We evaluated 31 enucleation and 18 plaque brachytherapy UM patients who were treated with darovasertib neoadjuvant therapy in the Phase 2 Company-sponsored and IST trials. We observed approximately 59%, or 29 of the 49 total evaluable patients, with greater than or equal to 20% ocular tumor shrinkage by product of diameters and approximately 49%, or 24 of the 49 total evaluable patients, with greater than or equal to 30% ocular tumor shrinkage by product of diameters. We also observed an approximately 61% eye preservation rate in enucleation patients. We found evidence predicting visual preservation by reducing the amount of radiation associated with plaque brachytherapy. We observed a manageable AE profile from the Phase 2 Company-sponsored trial. In 38 patients, 11% of patients experienced a Grade 3 or higher AE and 5% of patients experienced a serious AE rate. We also observed a discontinuation rate of 3%. The most common AEs observed included diarrhea, nausea, vomiting and fatigue.
We are pursuing a clinical strategy for darovasertib to broadly address UM, alternatively referred to as ocular melanoma, in both primary and metastatic settings. Greater than 90% of UM patients have tumors harboring GNAQ or GNA11 mutations. There are no FDA-approved systemic therapies for primary UM as either neoadjuvant or adjuvant therapies. There are likewise no FDA-approved therapies for patients having MUM with HLA-A*02:01 negative, or HLA-A2(-), serotype. These primary UM patients and HLA-A2(-) MUM patients collectively represent approximately 85% of all ocular melanoma patients. We have a separate, independent clinical strategy to address HLA-A*02:01 positive, or HLA-A2(+), MUM patients.
The potentially addressable patient population for MUM is estimated to include an annual incidence of approximately 4,500 patients across the United States and Europe. (Neo)Adjuvant UM represents a significant expansion opportunity for darovasertib – with a potential annual incidence of approximately 12,000 patients aggregate in North America, Europe and Australia.
We own or control all commercial rights in our darovasertib program in UM, including in MUM and in primary UM, subject to certain economic obligations pursuant to our exclusive, worldwide license to darovasertib with Novartis.
Darovasertib – Potential Registration-Enabling Clinical Trial in First-Line HLA-A2(-) MUM
The protocol of the Phase 2/3 clinical trial design incorporates guidance and feedback following our Type C meeting with the FDA in March 2023. This protocol includes an integrated Phase 2/3 open-label study-in-study design in first-line MUM patients with an HLA-A2(-) serotype. The clinical trial design employs a Phase 2 portion with median progression free survival, or PFS, as a primary endpoint for potential accelerated approval. Patients enrolled in Phase 2 will continue on treatment within the same study and will be considered, together with additional enrolled patients, to evaluate OS as the primary endpoint of the Phase 3 confirmatory portion of the clinical trial to support a potential full approval.
In the Phase 2 portion of the clinical trial, approximately 260 patients will be randomized on a 2:1 basis for treatment with the darovasertib and crizotinib combination in the treatment arm or investigators choice in the control arm, selected from (a) a combination of ipilimumab (ipi) and nivolumab (nivo), (b) PD1-targeted monotherapy or (c) dacarbazine. The treatment arm of the Phase 2 portion of the clinical trial includes a nested study to confirm the move forward combination dose for the integrated Phase 2/3 clinical trial – including cohorts at the Phase 2 expansion doses of (i) darovasertib 300 mg BID + crizotinib 200 mg BID and (ii) darovasertib 200 mg BID + crizotinib 200 mg BID. Under the nested study design, patients enrolled in the cohort at the move forward dose will be included within the Phase 2/3 registrational clinical trial. The Phase 2 portion of the clinical trial contemplates an efficacy and safety data set of approximately 200 patients randomized 2:1 with the treatment arm at the move forward dose to support a potential accelerated approval based on median PFS by blinded independent central review, or BICR, as a primary endpoint. Accelerated approval is intended to allow for earlier approval of drugs that
treat serious conditions and fill an unmet medical need based on a demonstration of effectiveness on a surrogate endpoint.
Patients enrolled in Phase 2 at the selected dose would continue on treatment and be included in the Phase 3 study analysis, supplemented by enrollment of approximately 120 additional patients into the Phase 3 portion of the clinical trial, with 2:1 randomization on the same basis as the Phase 2 portion. Efficacy data from the Phase 3 could support potential full approval using median OS as a primary endpoint.
In December 2024, we announced the recommendation of a move-forward dose and the completion of the Part 2a dose optimization for the potential registration-enabling Phase 2/3 trial evaluating the combination of darovasertib and crizotinib in the 1L setting in patients with HLA-A2(-) MUM. We are continuing enrollment in Part 2b with the selected optimal dose.
In May 2023, we expanded our relationship with Pfizer to support the Phase 2/3 registrational trial to evaluate darovasertib and crizotinib as a combination therapy in MUM by entering into Amendment No. 1 to the Second Pfizer Agreement. Under Amendment No. 1 to the Second Pfizer Agreement, Pfizer will provide us with a first defined quantity of crizotinib at no cost, as well as an additional second defined quantity of crizotinib at a lump-sum cost.
In December 2024, we entered into Amendment No. 5 to the Pfizer Agreement for the supply of crizotinib in the Phase 1/2 clinical trial for Pfizer to provide us a defined quantity of crizotinib at defined costs.
Prevalence of HLA-A2*02:01 Negative Serotype in MUM
Data from darovasertib clinical trials in MUM demonstrate that approximately 70% of MUM patients with known HLA-A*02:01, or HLA-A2 status were HLA-A2(-). As reported at ESMO 2023, the HLA-A2 status was known in subsets of patients enrolled in clinical trials evaluating darovasertib. Prevalence of HLA-A2(+) and HLA-A2(-) in MUM patients was determined from a first data set of n=149 MUM patients treated with darovasertib as monotherapy or in a combination arm of a clinical trial, and separately in a second data set of n=118 MUM patients treated with the darovasertib and crizotinib combination. These data include 102 of 149 (68%) of patients in the all-treatment subset and 81 of 118 (69%) patients in the darovasertib and crizotinib combination treatment subset.
Darovasertib – Strategy for HLA-A*02:01 Positive MUM
Based on clinical data from the Phase 2 clinical trial evaluating darovasertib and crizotinib in MUM as reported at ESMO 2023, and based on the darovasertib mechanism of action, we anticipate darovasertib will have clinical activity independent of HLA-A2 status in GNAQ/11-mutation cancers.
We are enrolling additional HLA-A2(+) MUM patients as an independent clinical strategy to address HLA-A2(+) MUM patients, in our ongoing Phase 2 clinical trial, designated as IDE196-001. This strategy demonstrates our commitment to fully address the high unmet medical need in MUM. Such clinical trial data from darovasertib and crizotinib combination treatment in HLA-A2(+) MUM could support publication and potential inclusion in NCCN Clinical Practice Guidelines in Oncology.
Darovasertib – Orphan Drug Designation in UM, Fast Track Designation in MUM and Breakthrough Therapy Designation in Neoadjuvant UM
In April 2022, the FDA designated darovasertib as an Orphan Drug in UM, including primary and metastatic disease. Under an Orphan Drug designation, darovasertib may be entitled to certain tax credits for qualifying clinical trial expenses, exemption from certain user fees and, subject to FDA approval of a New Drug Application, or NDA, for darovasertib in UM, eligibility for seven years of statutory marketing exclusivity during which the FDA is prohibited from approving a subsequent same drug for the same rare disease or condition except in limited circumstances, such as a subsequent drug that demonstrates clinical superiority. As an FDA-designated Orphan Drug, darovasertib may also be excluded from certain mandatory price negotiation provisions of the 2022 Inflation Reduction Act, if approved for a single indication only.
In November 2022, the FDA granted Fast Track designation to our development program investigating darovasertib in combination with crizotinib in adult patients being treated for MUM. The Fast Track designation makes our darovasertib and crizotinib development program eligible for various expedited regulatory review processes, including generally more frequent FDA interactions, such as meetings and written communications, potential eligibility for rolling review of a future NDA and potential accelerated approval and priority review of an NDA.
In March 2025, the FDA granted Breakthrough Therapy Designation, or BTD, for darovasertib for the neoadjuvant treatment of adult patients with primary UM for whom enucleation has been recommended. Under the license agreement with Novartis, we paid Novartis a $1.0 million milestone payment in April 2025. BTD is designed to expedite the development and regulatory review of promising therapies for serious or life-threatening conditions where preliminary clinical evidence suggests substantial improvement over existing treatments. The designation facilitates more intensive FDA guidance, cross-disciplinary collaboration, and eligibility for rolling submission and priority review.
Darovasertib – Phase 2 Trials in Neoadjuvant and Adjuvant Therapy in UM
We are clinically evaluating the potential for darovasertib as neoadjuvant or adjuvant therapy, or both, also referred to as (neo)adjuvant therapy, in primary, non-metastatic UM patients. We previously reported preliminary clinical data in the neoadjuvant setting showing evidence of anti-tumor activity that we believe supports further clinical evaluation of darovasertib to determine its potential as a neoadjuvant therapy to either save the eye by avoiding enucleation, or to reduce the tumor thickness in the eye, enabling treatment with less radiation to preserve vision, and as an adjuvant therapy, to potentially extend relapse free survival.
We enrolled 95 patients as of December 31, 2024 in our Phase 2 clinical trial, designated as IDE196-009, evaluating darovasertib as single-agent neoadjuvant and adjuvant therapy in patients having primary UM with ongoing enrollment and multiple clinical sites open. The purpose of the clinical trial is to evaluate single-agent darovasertib as neoadjuvant treatment of primary UM prior to primary interventional treatment of enucleation or radiation therapy and also as adjuvant therapy following the primary treatment. An amendment to the study protocol was submitted to the FDA in July 2024 to enable dosing of darovasertib therapy up to 12 months.
We are additionally supporting evaluation of darovasertib as (neo)adjuvant therapy in primary UM in the ongoing NADOM IST. Pursuant to an as-amended protocol for the NADOM study, UM patients who would otherwise undergo enucleation are instead treated with single agent darovasertib as neoadjuvant treatment for up to six months or maximum benefit. This reflects an increase in potential treatment duration versus the initial approach of one month neoadjuvant therapy, following which these patients will undergo a primary interventional treatment. Patients will subsequently be treated with darovasertib for up to six months as follow-up adjuvant therapy after the primary interventional treatment.
Darovasertib – Potential Registration-Enabling Clinical Trial in Neoadjuvant UM
A Type C meeting was held with the FDA in September 2024 and a Type D meeting was subsequently held in April 2025 for the clinical trial design of a potential Phase 3 registration-enabling clinical trial in neoadjuvant UM patients. For the potential Phase 3 clinical trial, we currently project approximately 520 patients will be randomized 2:1 for treatment with darovasertib in the treatment arm or the control arm. Based on the currently targeted clinical trial design, there will be two cohorts enrolled, including enucleation eligible UM patients and plaque brachytherapy eligible UM patients. For the enucleation cohort, the randomization will be with or without darovasertib as neoadjuvant therapy. For the plaque brachytherapy cohort, the randomization will be darovasertib followed by plaque brachytherapy versus plaque brachytherapy alone.
Based on guidance and information provided in the FDA Type D meeting, we expect that eye preservation rate (exceed lower bound of 10% eye preservation rate with a 95% confidence interval) will be the primary endpoint for enucleation UM patients. Proportion of patients with best corrected visual acuity 15-letter loss from time of randomization and time of completion of PB will be the primary endpoint for plaque brachytherapy UM patients. Vision detriment will be measured by the Early Treatment Diabetic Retinopathy Study (ETDRS) Best-Corrected Visual Acuity (BCVA) of >15-letters lost. No detriment to Event-Free-Survival, or EFS, in the treatment arms will be a required secondary endpoint. No detriment is defined as overlapping confidence intervals.
Additional secondary endpoints include overall response rate (>20% ocular tumor shrinkage by product of diameters), proportion of patients with clinically significant macular edema, proportion of subjects with 20/200 vision loss or worse (legal blindness), and proportion of subjects with reduction of radiation dose of >20% delivered to key eye structures.
The registrational study will enroll UM patients with a high risk for metastatic disease. Patients with moderate to high risk for vision loss will also be enrolled in the plaque brachytherapy eligible UM cohort. Based on the FDA meeting, there is a potential for consideration of a broad indication label in neoadjuvant UM for subjects with low, intermediate and high risk for metastatic disease. We anticipate approximately 18 months of data maturity from the last patient enrolled to an initial readout for no detriment to EFS in the treatment arms. 300mg BID darovasertib will be the move-forward dose for the registrational trial. Commencement of the Phase 3 registration-enabling trial for darovasertib in neoadjuvant UM is targeted for the first half of 2025.
IDE397 – MAT2A Inhibitor in Tumors with MTAP Deletion
IDE397 is a clinical-stage, potent, selective small molecule inhibitor of methionine adenosyltransferase 2a, or MAT2A, which we are developing for patients having solid tumors with MTAP deletion. The prevalence of methylthioadenosine phosphorylase, or MTAP, gene deletion is estimated to be approximately 15% of human solid tumors. MTAP deletion in patient tumors is identified by commercial or institutional next generation sequencing, or NGS, panels or by MTAP immunohistochemistry, or IHC, assay with confirmation by NGS.
MTAP-null cells lack the ability to metabolize 5-methylthioadenosine, or MTA, which is an essential step in a biochemical pathway involved in salvaging the metabolite S-adenosyl methionine, or SAM. Increased levels of MTA partially inhibit the methyltransferase PRMT5 for which SAM is the methyl-donor substrate for methylation of various proteins. This partial inhibition of PRMT5 by increased levels of MTA renders MTAP-null cells more dependent on the activity of MAT2A, an enzyme that is responsible for the synthesis of SAM. Because of this enhanced dependence, loss of MTAP results in synthetic lethality when MAT2A is pharmacologically inhibited.
We have enrolled patients into a Phase 1/2 clinical trial, designated as IDE397-001, to evaluate IDE397 for patients having certain tumors with MTAP gene deletion, with an initial focus in MTAP-deletion urothelial cancer, or UC, and non-small cell lung cancer, or NSCLC. We are proceeding with enrollment of MTAP-deletion patients into a monotherapy Phase 1/2 expansion cohort with an initial focus on high priority solid tumor types, including UC and NSCLC. We have selected a move-forward Phase 2 expansion dose for IDE397 monotherapy, based on AE profile and preliminary clinical efficacy observed, including multiple partial responses by RECIST 1.1.
We own all right, title and interest in and to IDE397 and the MAT2A program, including all worldwide commercial rights thereto.
Company-Sponsored Phase 1/2 Monotherapy Expansion in MTAP-Deletion Urothelial and Lung Cancer
In July 2024, we announced clinical data for the IDE397 Phase 1/2 monotherapy expansion dose demonstrating preliminary clinical efficacy in heavily pre-treated MTAP-deletion UC and NSCLC patients. The patients evaluated had a median of two prior lines of therapy, ranging from one to seven prior lines of treatment. The reported Phase 1/2 clinical data were based on 18 evaluable MTAP-deletion patients, including seven UC patients, four adenocarcinoma squamous NSCLC patients, and seven squamous NSCLC patients at the expansion dose of 30 mg once-a-day, or QD, of IDE397. In the interim update for 18 evaluable patients, with a data analysis cutoff date of June 21, 2024, we reported an overall response rate of approximately 39% (one complete response and six partial responses by RECIST 1.1 evaluation), which included two unconfirmed partial responses (one UC patient that had a 100% tumor reduction in the target lesion at the last CT-scan assessment and one adenocarcinoma squamous NSCLC patient which were both confirmed in our October 2024 update). We also observed a disease control rate of 94%, including one complete response, six partial responses and 10 stable disease by RECIST 1.1 evaluation. In addition, we observed tumor shrinkage in 14 of the 18 evaluable patients. 11 of the evaluable patients are still on treatment and five of the seven responses by RECIST 1.1 evaluation remain in response. We also reported a ctDNA molecular response, or MR, rate of 81%, representing 13 of 16 reportable patients with 50% or greater ctDNA reduction (several quality control failures of patient samples precluded the other patients from MR analysis).
Regarding safety data, we also reported a favorable AE profile at the 30 mg QD expansion dose. Approximately 5.6% of patients experienced a Grade 3 or higher drug-related AE at the 30 mg QD dose, represented by one instance of Grade 3 asthenia, and no drug-related SAEs were observed. We observed no drug-related AEs leading to discontinuations, and one non-evaluable patient discontinued due to rapid clinical progression of cancer fatigue and drug-unrelated AEs in the first cycle of treatment. We anticipate that the favorable AE profile and dosing convenience of a 30 mg QD tablet has the potential to enable long-term dosing and combination development.
In October 2024, we announced Phase 1 expansion data for IDE397 in MTAP-deletion UC and NSCLC patients in a late breaker abstract oral presentation at the 36th edition of the EORTC-NCI-AACR Symposium, or ENA 2024, in Barcelona, Spain. The patients evaluated had a median of two to three prior lines of therapy, ranging from one to seven prior lines of treatment. The reported clinical data were based on 27 evaluable MTAP-deletion patients, including 10 UC patients, nine adenocarcinoma NSCLC patients, and eight squamous NSCLC patients at the expansion dose of 30 mg QD of IDE397. In the update of 27 evaluable patients, with a data analysis cutoff date of August 22, 2024, we reported an overall response rate of approximately 33% (one complete response and eight partial responses by RECIST 1.1 evaluation). Nine of nine responses were confirmed by RECIST 1.1, including four UC patients, of which one was a complete response, three squamous NSCLC patients, and two adenocarcinoma NSCLC patients. Two patients were confirmed after the data cutoff date. We also observed an overall response rate by RECIST 1.1 evaluation by solid tumor type. For MTAP-deletion UC patients, the confirmed overall response rate was 40%, or 4 out of 10 patients, for MTAP-deletion squamous NSCLC patients, the confirmed overall response rate was approximately 38%, or 3 out of 8 patients, and for MTAP-deletion adenocarcinoma NSCLC patients, the confirmed overall response rate was approximately 22%, or 2 out of 9 patients. In addition, we observed a disease control rate of 93%, including one complete response, eight partial responses and 16 stable disease by RECIST 1.1 evaluation, reflecting 25 of 27 evaluable patients with stable disease or better. Of the 27 evaluable patients, 15 are still on treatment. The median duration of treatment has not been reached and is greater than 6.2 months. The median time to response is approximately 2.7 months. The median duration of response and median progression free survival data is still immature. Three UC patients were on treatment greater than 250 days, four squamous NSCLC patients were on treatment greater than 200 days, and three adenocarcinoma NSCLC patients were on treatment greater than 200 days. We also reported a ctDNA MR rate of 81%, representing 17 of 21 reportable patients with 50% or greater ctDNA reduction and approximately 33%, representing 7 of 21 reportable patients, with a deep 90% or greater ctDNA reduction (several quality control failures of patient samples precluded the other patients from MR analysis). All 17 MRs were rapid occurring at the first ctDNA sample analysis.
We continued to report favorable AE profile at the 30 mg QD expansion dose. Approximately 18% of patients experienced a Grade 3 or higher drug-related AE at the 30 mg QD dose, and no drug-related SAEs were observed. No drug-related AEs leading to discontinuations were observed. We anticipate that the favorable AE profile and dosing convenience of a 30 mg QD tablet has the potential to enable long-term dosing and combination development, including with MTA-cooperative PRMT5 inhibitors and topoisomerase payload ADCs.
We are collaborating with Gilead Sciences, Inc., or Gilead, to clinically evaluate IDE397 in combination with Trodelvy (sacituzumab govitecan-hziy), Gilead’s Trop-2 directed antibody drug conjugate, or ADC, in patients having MTAP-deletion UC, in our Phase 1 clinical trial pursuant to a Clinical Study Collaboration and Supply Agreement, or CSCSA, with Gilead.
The first patient was dosed on the Phase 1 trial in June 2024. The Phase 1 clinical trial is evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of IDE397 in combination with Trodelvy in MTAP-deletion UC patients (NCT04794699). Pursuant to the Gilead CSCSA, we and Gilead retain the commercial rights to our respective compounds, including with respect to use as a monotherapy or combination agent. We are the study sponsor and Gilead will provide the supply of Trodelvy. IDE397 monotherapy or in combination with Trodelvy has not been approved by any regulatory agency, and the efficacy and safety of this combination has not been established.
In October 2024, we reported the first preliminary clinical case study of the IDE397 and Trodelvy combination in MTAP-deletion UC at ENA 2024, including a partial response by RECIST 1.1 in a patient case report with a genetic co-alteration of MTAP-deletion and a FGFR3-TACC3 fusion, and rapid and deep first-evaluation MRs with ctDNA reduction of greater than 95% observed. The partial response reported at ENA 2024 has confirmed by RECIST 1.1.
In February 2025, we expanded our clinical study collaboration with Gilead and entered into an additional CSCSA, or the Second Gilead CSCSA, to evaluate the IDE397 and Trodelvy combination in MTAP-deletion NSCLC.
In April 2025, we initiated Phase 1/2 expansion for IDE397 in combination with Trodelvy in MTAP-deletion UC based on preliminary safety and clinical efficacy data.
We were collaborating with Amgen to clinically evaluate IDE397 in combination with AMG 193, the Amgen investigational MTA-cooperative PRMT5 inhibitor, in patients having tumors with MTAP-deletion, in an Amgen-sponsored clinical trial pursuant to our Clinical Trial Collaboration and Supply Agreement with Amgen, or the Amgen CTCSA. In February 2025, we and Amgen mutually agreed to wind down the IDE397 and AMG 193 clinical combination study and will not pursue dose expansion.
In October 2024, we presented a preclinical poster presentation on the antitumor activity by combinatorial inhibition of MAT2A and PRMT5 in MTAP-deleted tumors at ENA 2024. We are targeting to enable our wholly-owned clinical combination of IDE397 and IDE892, our potential best-in-class MTA-cooperative PRMT5 inhibitor development candidate, in the second half of 2025 in MTAP-deletion NSCLC.
There are currently no FDA-approved therapies for patients with MTAP-deletion solid tumors, highlighting the unmet medical need. The priority MTAP-deletion solid tumor types for the IDE397 Phase 1/2 monotherapy program are UC and NSCLC. MTAP-deletion prevalence has been reported at over 15% in NSCLC and over 25% in UC, based on The Cancer Genome Atlas, or TCGA, database. We estimate that the MTAP-deletion annual incidence in the United States in NSCLC and UC is approximately 48,000 patients, based on the 2024 Surveillance, Epidemiology, and End Results database. In addition, there are several potential expansion MTAP-deletion solid tumor types that are also being considered for monotherapy and combination development, including pancreatic, head and neck, gastric, and squamous esophageal cancer, among others. Based on the TCGA database, MTAP-deletion prevalence in pancreatic, head and neck, gastric and squamous esophageal cancer represents an aggregate U.S. annual incidence of approximately 27,000 patients.
We own all rights, title, and interest in and to IDE397 and IDE892, including all worldwide commercial rights thereto.
IDE849 (DLL3) Program with Hengrui Pharma
In December 2024, we entered into an exclusive License Agreement, or the Hengrui Pharma License Agreement, with Jiangsu Hengrui Pharmaceuticals Co., Ltd., or Hengrui Pharma, pursuant to which Hengrui Pharma granted us an exclusive worldwide license outside of Greater China for IDE849 (SHR-4849), a potential first-in-class Phase 1 DLL3 TOP1i ADC. DLL3 has been reported to be expressed in multiple solid tumor types, including in small cell lung cancer, or SCLC, and Neuroendocrine Tumors, or NETs, at approximately 85% and 20-40%, respectively. DLL3 has limited extracellular expression in normal tissues, making it a promising therapeutic target in these tumor types, for which there remains significant unmet medical need.
IDE849 has shown promising antitumor activity in preclinical studies, including tumor regression as a monotherapy in multiple models. IDE849 is currently being evaluated by Hengrui Pharma in an ongoing Phase 1 trial in China in SCLC patients. In the ongoing Phase 1 dose escalation, IDE849 has reached therapeutic dose levels where multiple partial responses have been observed as of the data cutoff date of December 10, 2024. Among 11 evaluable SCLC subjects treated at therapeutic dose levels, 8 partial responses by RECIST 1.1 were observed, resulting in an overall response rate of ~73% (including both confirmed and unconfirmed responses, with all unconfirmed responses pending further evaluation). As of the data cutoff date, treatment related adverse events, or TRAEs were predominantly Grade 1 or 2. The Phase 1 dose escalation is ongoing with no reported drug-related discontinuations, and the maximum tolerated dose has not yet been reached. The most common TRAEs observed were white blood cell count decreased, anemia, neutrophil count decreased, nausea and platelet count decreased. In January 2025, Hengrui Pharma selected expansion doses for their Phase 1 trial. Clinical efficacy and safety data from over 40 SCLC patients in the multi-site open label Phase 1 trial, including the dose escalation and multiple expansion doses, is targeted to be presented at a medical conference in the third quarter of 2025.
In May 2025, we received U.S. IND clearance for the initiation of a Phase 1 clinical trial to evaluate IDE849 in solid tumors. We are targeting to evaluate IDE849 clinically in a multi-site global clinical trial as a monotherapy agent in SCLC and NETs and multiple DLL3 upregulated solid tumor types, and to evaluate IDE849 in a clinical combination with our potential first-in-class PARG inhibitor, IDE161, in the second half of 2025. Based on the FDA guidance, we will begin the Phase 1 study in the United States at a IDE849 starting dose that is equivalent to one of the expansion doses being evaluated in the ongoing Phase 1 study (NCT06443489) by partner Hengrui Pharma, where multiple confirmed partial responses have been observed by RECIST 1.1. We have also published preclinical data demonstrating IDE161 has combination synergy in-vitro and enhances the durability of TOP1-payload ADCs in preclinical in vivo models.
Under the terms of the Hengrui Pharma License Agreement, Hengrui Pharma is eligible to receive upfront and milestone payments totaling $1.045 billion, including a $75.0 million upfront fee, up to $200.0 million in development and regulatory milestone payments, plus commercial success-based milestones. Hengrui Pharma is also eligible to receive mid-single to low-double digit royalties on net sales outside of Greater China.
We own or control all commercial rights outside of greater China for IDE849.
IDE275 (GSK959) - WRN Inhibitor in Tumors with High Microsatellite Instability
We discovered IDE275 (GSK959), our Werner, or WRN, Helicase inhibitor clinical development candidate and evaluated IDE275 (GSK959) in preclinical studies in collaboration with GSK. IDE275 (GSK959) targets WRN for patients having tumors with MSI-High.
WRN protein is a RecQ enzyme involved in the maintenance of genome integrity. Germline loss of function mutations in WRN lead to premature aging and pre-disposition to cancer. Microsatellite instability is a change in the DNA content of a tumor cell in which the number of repeats of microsatellites, short repeated sequences of DNA, differ as cells divide. MSI-High is present in about 15% of gastrointestinal tumor cancers, including in approximately 22% of stomach adenocarcinoma and 16% of colorectal cancer. Tumors with MSI-High are routinely assessed in multiple diagnostic profiling tests.
WRN is a protein having several functional domains, and we have shown that the helicase functional domain of WRN is responsible for this synthetic lethal interaction, as reflected in our publication in Cell Press – iScience, Werner Syndrome Helicase is Required for the Survival of Cancer Cells with Microsatellite Instability (March 2019).
We, in collaboration with GSK, have demonstrated preclinical in vivo efficacy with tumor regression and PD response in a relevant MSI-High model. We have observed selectivity of our Werner Helicase inhibitor and validation of the synthetic lethal relationship to tumors with MSI-High over tumors with MSS based on a lack of in vivo pharmacological response in relevant MSS xenograft models.
We, in collaboration with GSK, received IND clearance for IDE275 (GSK959), a potential first-in-class WRN inhibitor, in October 2024 to enable first-in-human clinical evaluation of IDE275 (GSK959) for patients having tumors with MSI-High. GSK initiated a Phase 1/2 clinical trial for IDE275 (GSK959), following FDA allowance to proceed with the clinical trial. GSK will lead clinical development for the Werner Helicase program. GSK is responsible for 80% of global research and development costs, and we are responsible for 20% of such costs. GSK holds a global, exclusive license to develop and commercialize the Werner Helicase Inhibitor DC.
In April 2025, we, in collaboration with GSK, presented an oral presentation in the New Drugs on the Horizon series and three poster presentations at the American Association for Cancer Research, or AACR, 2025 Annual Meeting, on the potential best-in-class preclinical profile in the MSI-H setting for IDE275 (GSK959), with a unique binding mode from previously reported WRN inhibitors. A Trial in Progress poster for the ongoing SYLVER Phase 1/2 study (NCT06710847) was also presented at AACR 2025.
In October 2023, we achieved and earned a $3.0 million milestone in connection with IND-enabling studies. In October 2024, we earned a $7.0 million milestone payment for the IND clearance of IDE275 (GSK 959). We have
the potential to earn up to an additional $10.0 million milestone payment upon initiation of Phase 1 clinical dose expansion.
We are also eligible to receive further aggregate late-stage development and regulatory milestones of up to $465.0 million. Upon commercialization, we will be eligible to receive commercial milestones of up to $475.0 million, 50% of U.S. net profits and tiered royalties on global non-U.S. net sales of the Werner Helicase Inhibitor DC – ranging from high single-digit to sub-teen double-digit percentages, subject to certain customary reductions.
IDE161 – PARG Inhibitor in Tumors with Homologous Recombination Deficiency
We are evaluating IDE161, a potential first-in-class small molecule poly (ADP-ribose) glycohydrolase, or PARG, inhibitor, in a Phase 1/2 clinical trial, designated as IDE161-001 for patients having tumors with homologous recombination deficiency, or HRD, and potentially other genetic and/or molecular signatures. PARG is a novel target in a clinically validated biological pathway. PARG functions as a regulator of DNA repair in the same biochemical pathway as poly-(ADP-ribose) polymerase, or PARP. PARG hydrolyzes PAR chains that are polymerized by PARP enzymes, completing the PAR cycle. Small molecule inhibitors of PARG result in a dose dependent increase in cellular PAR after DNA damage. PARG is a mechanistically distinct target relative to PARP.
We are progressing with enrollment of patients having tumors with HRD into the Phase 1 expansion portion of the Phase 1/2 clinical trial. We selected an initial Phase 1/2 monotherapy expansion dose for IDE161 in endometrial cancer, based on AE profile and preliminary efficacy observed. In parallel, we are also continuing with Phase 1 monotherapy dose optimization. Our clinical strategy focus for the IDE161 program is on enrollment for the combination with IDE849.
In September 2023, we received Fast Track Designation from the FDA for IDE161 for the treatment of adult patients having advanced or metastatic ovarian cancer with germline or somatic BRCA 1/2 mutations who are platinum resistant and have received prior antiangiogenic and PARP inhibitor therapies and for the treatment of adult patients having advanced or metastatic hormone receptor positive, or HR+, Her2- breast cancer with germline or somatic BRCA 1/2 mutations who have progressed following treatment with at least one line of a hormonal therapy, a CDK4/6 inhibitor therapy and a PARP inhibitor therapy.
Under each Fast Track designation, the IDE161 development program in BRCA1/2 mutant (m) breast and ovarian cancers is eligible for various expedited regulatory review processes, including generally more frequent FDA interactions (e.g., meetings, written communications), potential eligibility for rolling review, accelerated approval, and priority review of a future NDA.
In March 2024, we entered into a Clinical Trial Collaboration and Supply Agreement, or the Merck CTCSA, with Merck (known as MSD outside of the United States and Canada). We are evaluating IDE161 in combination with Merck's anti-PD-1 therapy, KEYTRUDA® (pembrolizumab), in patients with MSI-High, and microsatellite stable, or MSS, endometrial cancer. Under the Merck CTCSA, Merck will provide KEYTRUDA® to us, and we will sponsor the Phase 1 clinical combination trial.
In October 2024, we presented preclinical results on the IDE161 and ADC combination rationale as a poster at ENA 2024.
In December 2024, the first patient was dosed with IDE161 in combination with KEYTRUDA in the Company-sponsored Phase 1 clinical trial. The safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of IDE161 in combination with KEYTRUDA is being evaluated as an arm in IDE161-001 (NCT05787587), a Company-sponsored Phase 1 trial of IDE161 in solid tumors.
In April 2025, we presented preclinical data on immune checkpoint inhibitor (ICI)-driven anti-tumor immunity at AACR 2025.
We are targeting to present preclinical combination mechanism and synergy efficacy data of IDE161 with TOP1-payload based ADCs at a medical conference in the third quarter of 2025.
We entered into an exclusive license under the Evaluation, Option and License Agreement with Cancer Research Technologies Ltd., also known as Cancer Research United Kingdom, or CRT, and the University of Manchester, pursuant to which we hold exclusive worldwide license rights covering a broad class of PARG inhibitors.
In April 2023, we incurred an obligation to pay milestone payments in an aggregate amount of £750,000 to CRT based upon the achievement of certain milestones relating to first and second tumor histologies in connection with the Phase 1 portion of the IDE161-001 Phase 1/2 clinical trial in oncologic diseases. We will be obligated to make additional payments to CRT aggregating up to £18.75 million upon the achievement of specific development and regulatory approval events for development of a PARG inhibitor in oncologic diseases, including an aggregate of up to £1.5 million and up to £2.25 million for the achievement of certain Phase 2 and Phase 3 development milestones, respectively, in each case as relating to first and second tumor histologies.
We own or control all commercial rights in our PARG program, subject to certain economic obligations pursuant to our exclusive, worldwide license to certain PARG inhibitors, including IDE161, with CRT and University of Manchester.
IDE705 (GSK101) – Pol Theta Helicase Inhibitor in tumors with Homologous Recombination Deficiency
We discovered IDE705 (GSK101), our DNA Polymerase Theta, or Pol Theta, Helicase inhibitor clinical development candidate, and evaluated IDE705(GSK101) in preclinical studies in collaboration with GSK. IDE705 (GSK101) targets the helicase domain of the Pol Theta protein for patients having solid tumors with BRCA or other mutations associated with HRD.
Pol Theta is involved in a DNA repair process called microhomology mediated end joining, or MMEJ, that is utilized when homologous recombination mediated repair is compromised, as happens in the case of certain BRCA1 or BRCA2 mutations. The expression of Pol Theta is largely absent in normal cells, but tumor cells harboring double strand break repair defects, such as BRCA1 or BRCA2 mutations, show higher Pol Theta expression and synthetic lethality when Pol Theta is inhibited. Pol Theta is a large protein with two functional domains: a DNA polymerase domain and an ATP-dependent DNA helicase domain, sometimes referred to as an ATPase domain, linked by a RAD51 central region.
GSK is evaluating IDE705 (GSK101) in combination with niraparib, the GSK small molecule inhibitor of PARP for the treatment of patients having tumors with BRCA or other HRD, in a GSK-sponsored Phase 1 clinical trial. GSK has dosed the first patient, and enrollment is ongoing in the dose escalation portion of this study.
GSK is leading clinical development of IDE705 (GSK101) pursuant to the Collaboration, Option and License Agreement with GSK, or GSK Collaboration Agreement. GSK is responsible for all research and development costs for the Pol Theta program.
In August 2023, we earned a $7.0 million payment for a milestone based on acceptance of the IND by the FDA. An earlier preclinical development $3.0 million milestone payment from GSK was achieved in August 2022 in connection with ongoing IND-enabling studies to support the evaluation of IDE705 (GSK101). We have the potential to receive an additional $10.0 million milestone payment upon initiation of Phase 1 clinical dose expansion.
We have the potential to earn further aggregate late-stage development and regulatory milestones of up to $465.0 million. Upon commercialization, we will be eligible to receive commercial milestones of up to $475.0 million, and tiered royalties on global net sales of GSK101 – ranging from high single-digit to sub-teen double-digit percentages, subject to certain customary reductions.
IDE892 – MTA-cooperative PMRT5 inhibitor
In December 2024, we selected IDE892, a potential best-in-class MTA-cooperative PRMT5 inhibitor, as a development candidate. IDE892 was discovered through our iterative physics-based ligand design and optimization platform, and is a highly potent and selective MTA-cooperative PRMT5 inhibitor with best-in-class potential and favorable drug-like properties. IDE892 has demonstrated exceptionally selective antiproliferative activity in
MTAP-deleted tumor cell models and durable complete responses in combination with MAT2A inhibitor IDE397 in challenging MTAP-deletion preclinical models.
IDE892 enables a wholly-owned clinical combination between the PRMT5 and MAT2A mechanisms and delivers potentially greater efficacy in MTAP-deletion solids tumors through this rational combination approach, including favorable potency, selectivity, and synergistic combination potential with MAT2A inhibitor IDE397.
Development of IDE892 is ongoing to support an IND filing to the FDA in mid-year 2025, subject to satisfactory completion of ongoing preclinical and IND-enabling studies.
In April 2025, we presented preclinical data providing insights into metabolite kinetics and PRMT5 dysregulation in MTAP-deficient cancers at AACR 2025.
We are also targeting to enable our wholly-owned clinical combination of IDE397 and IDE892 in the second half of 2025 in MTAP-deletion NSCLC.
IDE034 (B7H3/PTK7) program with Biocytogen
In July 2024, we entered into an Option and License Agreement, or the Biocytogen Option and License Agreement, with Biocytogen Pharmaceuticals (Beijing) Co., Ltd., (Biocytogen, HKEX: 02315), or Biocytogen, pursuant to which Biocytogen granted us an option for an exclusive worldwide license for a potential first-in-class B7H3/PTK7 topoisomerase-I-inhibitor-payload BsADC program, or the Option. B7H3/PTK7 has been found to be co-expressed in multiple solid tumor types, including double-digit percent prevalence in lung, colorectal, and head and neck cancers, among others. Based on preclinical data, the potential first-in-class B7H3/PTK7 topoisomerase-I-inhibitor-payload BsADC program has the potential to be developed as a monotherapy agent and used in combination with multiple programs in our pipeline targeting DDR-based therapies, including our PARG inhibitor IDE161.
In November 2024, we announced the selection of IDE034, a potential first-in-class B7H3/PTK7 topo-I-payload BsADC, as a development candidate and the exercise of the Option. Under the terms of the Biocytogen Option and License Agreement, we paid Biocytogen an upfront fee and an exercise fee for the Option totaling $6.5 million.
We are targeting an IND submission to the FDA in the second half of 2025 for IDE034, subject to satisfactory completion of ongoing preclinical and IND-enabling studies.
Biocytogen is eligible to receive total potential upfront, option exercise and milestone payments equal an aggregate of $406.5 million, including development and regulatory milestone payments of up to $100.0 million. Our royalty obligations continue with respect to each country and each product until the later of (i) the date on which such product is no longer covered by certain intellectual property rights in such country and (ii) the 10th anniversary of the first commercial sale of such product in such country.
IDE574 - KAT6/7 inhibitor
Our development candidate IDE574, is a potential first-in-class KAT6/7 inhibitor which is an equipotent, highly selective, small molecule dual inhibitor of the lysine acetyltransferase (KAT) 6 and 7, both of which have been shown to support cancer cell survival. IND-enabling studies to support the potential clinical evaluation of IDE574 monotherapy in patients with breast and lung cancers with 8p11 amplification are ongoing, as well as additional opportunities in the setting of lineage addiction. Based on our biomarker evaluation, 8p11 amplification prevalence is projected to be approximately 15% in breast cancer and 17.5% in squamous NSCLC.
IDE574 selectively inhibits both KAT6 and KAT7 while sparing other structurally similar KAT molecules. KAT6 and KAT7 are mechanistically intertwined epigenetic modulators of cell identity and lineage commitment programs corrupted by oncogenic transformation. Dual KAT6/7 inhibition with IDE574 delivers robust and durable anti-tumor activity, superior to KAT6 inhibition alone, in preclinical tumor models with 8p11 amplifications, as well as in biomarker selected indications dependent upon lineage-specific transcription factor activity.
In April 2025, we presented preclinical data on dual inhibition’s impact on epigenetics and adaptive drug resistance at AACR 2025.
We are targeting an IND submission to the FDA in the second half of 2025 for IDE574, subject to satisfactory completion of ongoing preclinical and IND-enabling studies.
Next-Generation Precision Medicine Pipeline Programs
We have initiated early preclinical research programs focused on pharmacological inhibition of several new targets, or NTs, for patients with solid tumors characterized by defined biomarkers based on genetic mutations and/or molecular signatures. We believe these research programs have the potential for discovery and development of first-in-class or unique-in-class or best-in-class therapeutics. Collectively, we believe these efforts will further advance our multi-pronged clinical and business strategy. We own or control all commercial rights in our next-generation NT programs.
New Target and Biomarker Discovery Platform
Since the inception of the company, our core research has and continues to be focused on precision medicine oncology, with synthetic lethality as a central tenet. We have invested significantly and continue to invest in capabilities for identification and validation of new precision medicine targets and biomarkers for patient selection. These investments include both additional research personnel and capital investments, which will enhance our capabilities broadly, including in target validation, biological assay development, protein synthesis, structural biology, computational chemistry, and analytical chemistry, among other core functional areas. For targets of interest, we advance our research to discover therapeutic drugs and to further qualify relevant biomarkers.
Collaboration with ATTMOS
We are collaborating with ATTMOS to build a physics-based computational small molecule discovery platform that rapidly unlocks what are currently perceived as undruggable oncology targets. The focus of the partnership is to engineer and optimize a workflow solution for high-throughput absolute binding free energy perturbation predictions (ABFEP) of first-in-class drug candidate molecules. This approach enables application of gold-standard physics-based statistical mechanics calculations of protein-ligand affinities at the scale required for virtual screens and represents what could become the industry's go-to standard for high-speed and high probability-of-success drug hit-finding against structurally-enabled novel biological targets.
The collaboration leverages the Amber molecular dynamics suite as the GPU-accelerated back-end free energy simulation engine. We train and evaluate ABFEP-based active learning cycles based on extensive ground-truth data sets derived from its successful wet-lab drug discovery campaigns against novel targets. These models will be used to screen enormous libraries of synthetically tractable chemical space for accurate and efficient de novo discovery of small molecule ligands for new targets.
Precision Medicine Research Platform
We have established a robust precision medicine research platform with capabilities for identification and validation of new targets and biomarkers, drug discovery and translational biology. Our approach integrates small molecule drug discovery with extensive capabilities in identifying and validating translational biomarkers to develop targeted therapies for select patient populations that are most likely to benefit from these targeted therapies. Our small molecule drug discovery expertise includes discovery and development of small molecule therapeutics.
The drug discovery platform includes our proprietary chemical library, INQUIRE, structure-based drug design enabled by extensive structural biology and crystallography capabilities with over 200,000 chemical compounds, and our proprietary content-based machine-learning engine, HARMONY, providing effective and efficient molecular design and structure-activity-relationship, or SAR, cycles. We have deep research and development expertise in synthetic lethality – which represents an emerging class of precision medicine targets. We are applying these capabilities to develop a robust pipeline in precision medicine oncology.
DECIPHER Dual CRISPER Synthetic Lethality Library – UCSD
We have constructed our DECIPHER Dual CRISPR library for synthetic lethality target and biomarker discovery in collaboration with the University of California, San Diego, and bioinformatics analysis and validation are ongoing. The DECIPHER 1.0 library is focused on DNA Damage Repair targets across various tumor suppressor genes and oncogenes of interest that were selected based on their known prevalence and role in solid tumors, enabling evaluation of approximately 50,000 independent gene knockout combinations of DDR pathway related drug targets across known tumor suppressor genes.
PAGEO Paralogous Gene Evaluation in Ovarian Cancer and Dep Map Consortium – Broad Institute
We have an ongoing strategic collaboration with the Broad Institute focused on synthetic lethality target and biomarker discovery. This collaboration will use the large-scale CRISPR paralog screening platform developed at the laboratory of William R. Sellers, M.D., Core Institute Member, Broad Institute, to evaluate functionally redundant paralogous genes across ovarian cancer subtypes and to generate novel target and biomarker hypotheses. Dr. Sellers, who also serves on our Scientific Advisory Board, is the principal investigator for the strategic collaboration. We have also become a member of the Broad DepMap (Cancer Dependency Map) consortium led by the Broad Institute to further enhance our efforts in bioinformatics and cell-based screening for synthetic lethality target and biomarker discovery and validation.
Small and Medium Enterprise Status from the European Medicines Agency
In June 2024, we were granted Small and Medium Enterprise, or SME, status by the European Medicines Agency, or EMA. This enables us to have access to administrative, regulatory and financial support, including fee reductions for scientific advice and regulatory procedures across all our programs.
Prospectus Supplement - At-the-Market Facility
On January 19, 2024, we entered into a new Open Market Sales Agreement, or the January 2024 Sales Agreement, with Jefferies LLC, or Jefferies, relating to an at-the-market offering program under which we may offer and sell, from time to time at our sole discretion, shares of common stock, par value $0.0001 per share, or common stock, having aggregate gross proceeds of up to $350.0 million through Jefferies as sales agent.
During the three months ended March 31, 2025, we sold an aggregate 984,000 shares of our common stock for aggregate net proceeds of $25.0 million at a weighted average sales price of approximately $26.00 per share under the at-the-market offering pursuant to the January 2024 Sales Agreement with Jefferies as sales agent. As of March 31, 2025, approximately $156.6 million of common stock remained available to be sold pursuant to the January 2024 Sales Agreement.
We may cancel our at-the-market program at any time upon written notice, pursuant to its terms.
2024 July Public Offering and Sale of IDEAYA Common Stock
On July 11, 2024, we completed an underwritten public follow-on offering. The offering consisted of 8,355,714 shares of common stock at an offering price to the public of $35.00 per share, including 1,127,142 shares of common stock upon the exercise in full of the overallotment option by the underwriters, as well as pre-funded warrants to purchase 285,715 shares of common stock at a public offering price of $34.9999 per underlying share, in each case before underwriting discounts and commissions. Pursuant to the offering, we received aggregate gross proceeds of approximately $302.4 million, before deducting underwriting discounts and commissions and other offering expenses, resulting in net proceeds of approximately $283.8 million, after deducting underwriting discounts and commissions and other offering expenses.
Corporate Update
We do not have any products approved for sale and have not generated any product revenue since inception. We have funded our operations primarily through the sale and issuance of common stock and the upfront payment and certain milestone payments received from GSK. As of March 31, 2025, we had cash, cash equivalents and
marketable securities of approximately $1.05 billion, consisting primarily of money market funds, U.S. government securities, commercial paper, and corporate bonds.
Since our inception in June 2015, we have devoted substantially all of our resources to discovering and developing our product candidates. We have incurred significant operating losses to date and expect that our operating expenses will increase significantly as we advance our product candidates through preclinical and clinical development; seek regulatory approval, and prepare for, and, if approved, proceed to commercialization; acquire, discover, validate and develop additional product candidates; obtain, maintain, protect and enforce our intellectual property portfolio; and hire additional personnel. Certain program costs that contribute to our operating expenses have been and/or will be reimbursed by GSK pursuant to the GSK Collaboration Agreement, including 100% of costs we incur for research we perform in connection with the Pol Theta program and 80% of the aggregate program costs incurred by us and GSK for research each of us performs for the Werner Helicase program. We also incur costs in accordance with the Gilead CSCSA and Second CSCSA. Gilead bears internal or external costs incurred in connection with its supply of Trodelvy. We bear all internal and external costs and expenses associated with the conduct of the combination study. We also incur costs in accordance with the Merck CTCSA. Merck provides KEYTRUDA for the study at no cost to us. We bear all internal and external costs and expenses associated with the conduct of the study. We also entered into two in-licensing agreements for ADCs with topoisomerase-I-inhibitor-payloads to enable combinations with our synthetic lethality programs with Hengrui Pharma for IDE849 and Biocytogen for IDE034. See Note 10. Significant Agreements.
Our net losses were $72.2 million and $39.6 million for the three months ended March 31, 2025 and March 31, 2024, respectively. As of March 31, 2025, we had an accumulated deficit of $695.0 million.
Our ability to generate product revenue will depend on the successful development, regulatory approval and eventual commercialization of one or more of our product candidates, ourselves, or for some programs, in collaboration with our strategic partners.
Until such time as we can generate significant revenue from product sales, if ever, we expect to finance our operations through the sale of equity, debt financings, or other capital sources, including potential collaborations with other companies or other strategic transactions. Adequate funding may not be available to us on acceptable terms, or at all. If we fail to raise capital or enter into such agreements as and when needed, we may have to significantly delay, scale back or discontinue the development and commercialization of our product candidates.
As of March 31, 2025, we had cash, cash equivalents and short-term and long-term marketable securities of approximately $1.05 billion.
We believe that our cash, cash equivalents, and short-term and long-term marketable securities will be sufficient to fund our planned operations for at least twelve months from the date of the issuance of our Quarterly Report on Form 10-Q filed May 6, 2025.
These funds will support our efforts through potential achievement of multiple preclinical and clinical milestones across multiple programs.
Components of Operating Results
Collaboration Revenues
To date, we have not generated any revenue from product sales, and we do not expect to generate any revenue from product sales unless and until we are able to initiate a registrational clinical trial, obtain regulatory approval and commercialize one of our product candidates in the future. Our revenue consists exclusively of collaboration revenue under the GSK Collaboration Agreement, including amounts that are recognized related to previously received upfront payments and amounts due and payable to us for research and development services. The amount of revenue recognized related to the GSK Collaboration Agreement, including as related to the previously received upfront payment or to certain development milestone payments, may vary considerably by period and certain components thereof may generally decrease year-over-year as we satisfy remaining performance obligations, for example, relating to the Pol Theta and WRN R&D Services. Since December 31, 2023, we have fully recognized the contract liabilities related to the upfront payment and reimbursements for the research and development performance obligations under the GSK Collaboration Agreement. There are no remaining contract liabilities as of March 31, 2025 as we concluded all the research and development performance obligations under the GSK Collaboration
Agreement. The future revenue recognition is contingent on additional milestones earned, profit sharing and royalties on any net product sales under our collaborations. We expect that any revenue we recognize or generate under the GSK Collaboration Agreement will fluctuate from period to period due to period to period variability in milestone payments and other payments.
Operating Expenses
Research and Development Expenses
Substantially all of our research and development expenses consist of expenses incurred in connection with the discovery and development of our product candidates. These expenses include certain payroll and personnel-related expenses, including salaries, employee benefit costs and stock-based compensation expenses for our research and product development employees, fees to third parties to conduct certain research and development activities on our behalf including fees to CMOs and CROs in support of manufacturing and clinical activity for darovasertib, IDE397, IDE849, IDE275 / GSK959, IDE161, IDE705 / GSK 101, and consulting costs, costs for laboratory supplies, costs for product licenses and allocated overhead, including rent, equipment, depreciation, information technology costs and utilities. We expense both internal and external research and development expenses as they are incurred.
We have entered into various agreements with CMOs and CROs. Our research and development accruals are estimated based on the level of services performed, progress of the studies, including the phase or completion of events, and contracted costs. The estimated costs of research and development provided, but not yet invoiced, are included in accrued liabilities on the condensed balance sheet. If the actual timing of the performance of services or the level of effort varies from the original estimates, we will adjust the accrual accordingly. Payments made to CMOs and CROs under these arrangements in advance of the performance of the related services are recorded as prepaid expenses and other current assets until the services are rendered.
Costs of certain activities, such as preclinical studies, are generally recognized based on an evaluation of the progress to completion of specific tasks. Nonrefundable payments made prior to the receipt of goods or services that will be used or rendered for future research and development activities are deferred and capitalized as prepaid expenses and other current assets on our condensed balance sheet. The capitalized amounts are recognized as expense as the goods are delivered or the related services are performed.
We do not allocate our internal costs by product candidate, including internal costs, such as payroll and other personnel expenses, laboratory supplies and allocated overhead. With respect to internal costs, several of our departments support multiple product candidate research and development programs, and therefore the costs cannot be allocated to a particular product candidate or development program. The following table summarizes our external clinical development expenses by program:
|
|
|
|
|
|
|
|
|
|
Three Months Ended |
|
|
|
March 31, 2025 |
|
December 31, 2024 |
|
External clinical development expenses(1): |
|
|
|
|
|
Darovasertib |
|
$ |
23,018 |
|
$ |
16,354 |
|
IDE397(2) |
|
|
3,741 |
|
|
4,576 |
|
IDE161 |
|
|
2,448 |
|
|
2,555 |
|
Personnel related and stock-based compensation |
|
|
15,814 |
|
|
13,404 |
|
Other research and development expenses(3)(4) |
|
|
25,865 |
|
|
103,294 |
|
Total research and development expenses |
|
$ |
70,886 |
|
$ |
140,183 |
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended |
|
|
|
March 31, 2025 |
|
March 31, 2024 |
|
External clinical development expenses(1): |
|
|
|
|
|
Darovasertib |
|
$ |
23,018 |
|
$ |
10,869 |
|
IDE397(2) |
|
|
3,741 |
|
|
2,926 |
|
IDE161 |
|
|
2,448 |
|
|
2,695 |
|
Personnel related and stock-based compensation |
|
|
15,814 |
|
|
12,254 |
|
Other research and development expenses(3) |
|
|
25,865 |
|
|
14,061 |
|
Total research and development expenses |
|
$ |
70,886 |
|
$ |
42,805 |
|
(1)External clinical development expenses include manufacturing and clinical trial costs. These expenses are primarily for services provided by external consultants, CMOs and CROs.
(2)IDE397 includes costs from Amgen CTCSA.
(3)Other research and development expenses include manufacturing and clinical trial costs for preclinical and earlier clinical stage programs. These expenses are primarily for services provided by external consultants, CMOs and CROs.
(4)Other research and development expenses for the three months ended December 31, 2024 includes the $75.0 million upfront payment under the Hengrui Pharma License Agreement for IDE849.
We are focusing substantially all of our resources on the development of our product candidates. We expect our research and development expenses to increase substantially during the next few years, as we seek to initiate and/or advance clinical trials for our product candidates, complete our clinical program, pursue regulatory approval of our product candidates and prepare for a possible commercial launch. Predicting the timing or the cost to complete our clinical program or validation of our commercial manufacturing and supply processes is difficult and delays may occur because of many factors, including factors outside of our control. For example, if the FDA or other regulatory authorities were to require us to conduct clinical trials beyond those that we currently anticipate, or if we experience significant delays in enrollment in any of our clinical trials, we could be required to expend significant additional financial resources and time on the completion of clinical development. Furthermore, we are unable to predict when or if our product candidates will receive regulatory approval with any certainty.
General and Administrative Expenses
General and administrative expenses consist primarily of payroll and personnel-related expenses, including salaries, employee benefit costs and stock-based compensation expense, professional fees for legal, patent, consulting, accounting and tax services, allocated overhead, including rent, equipment, depreciation, information technology costs and utilities, and other general operating expenses not otherwise classified as research and development expenses.
We anticipate that our general and administrative expenses will increase, as a result of increased personnel costs, including salaries, benefits and stock-based compensation expense, patent costs for our product candidates, expanded infrastructure and higher consulting, legal and accounting services associated with maintaining compliance with our Nasdaq stock exchange listing and requirements of the Securities and Exchange Commission, or the SEC, investor relations costs and director and officer insurance policy premiums associated with being a public company.
Other Income
Interest Income and Other Income, Net
Interest income and other income, net consists primarily of interest income earned on our cash, cash equivalents and marketable securities.
Results of Operations
A discussion regarding our financial condition and results of operations for the three months ended March 31, 2025 compared to the three months ended December 31, 2024 and for the three months ended March 31, 2025 compared to the three months ended March 31, 2024 is presented below.
Comparison of Three Months Ended March 31, 2025 and December 31, 2024
The following table summarizes our results of operations for the periods indicated (in thousands):
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended |
|
|
|
|
|
|
|
March 31, 2025 |
|
December 31, 2024 |
|
Change |
|
% Change |
|
Revenue: |
|
|
|
|
|
|
|
|
|
Collaboration revenue |
|
$ |
— |
|
$ |
7,000 |
|
|
(7,000 |
) |
|
(100 |
%) |
Operating expenses: |
|
|
|
|
|
|
|
|
|
Research and development |
|
|
70,886 |
|
|
140,183 |
|
|
(69,297 |
) |
|
(49 |
%) |
General and administrative |
|
|
13,503 |
|
|
10,955 |
|
|
2,548 |
|
|
23 |
% |
Loss from operations |
|
|
(84,389 |
) |
|
(144,138 |
) |
|
59,749 |
|
|
(41 |
%) |
Interest income and other income, net |
|
|
12,211 |
|
|
13,826 |
|
|
(1,615 |
) |
|
(12 |
%) |
Net loss |
|
$ |
(72,178 |
) |
$ |
(130,312 |
) |
$ |
58,134 |
|
|
(45 |
%) |
Collaboration Revenue
There was no collaboration revenue recognized for the three months ended March 31, 2025 compared to $7.0 million for the three months ended December 31, 2024. The $7.0 million in revenue was earned in October 2024 for the IND clearance of IDE275 (GSK 959), a potential first-in-class WRN inhibitor.
We completed all performance obligations related to the upfront payment under the GSK Collaboration Agreement as of December 31, 2023. Future collaboration revenue recognized under the GSK Collaboration Agreement will be related to future milestone payments as they are earned.
Research and Development Expenses
Research and development expenses decreased by $69.3 million, or 49%, during the three months ended March 31, 2025 compared to the three months ended December 31, 2024 primarily due to the $75.0 million upfront payment under the license agreement for IDE849 with Hengrui Pharma that occurred in December 2024, partially offset by increases in $5.7 million in fees paid to CROs, CMOs, and consultants related to the advancement of our lead product candidates through preclinical and clinical studies.
General and Administrative Expenses
General and administrative expenses increased by $2.5 million, or 23%, during the three months ended March 31, 2025 compared to the three months ended December 31, 2024. The increase in general and administrative expense was primarily due to increases of $1.2 million in personnel-related expenses and $1.3 million in consulting services and legal patent expenses.
Interest Income and Other Income, Net
Interest income decreased by $1.6 million, or 12%, during the three months ended March 31, 2025 compared to the three months ended December 31, 2024, primarily due to lower investment balances.
Comparison of Three Months Ended March 31, 2025 and March 31, 2024
The following table summarizes our results of operations for the periods indicated (in thousands):
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended March 31, |
|
|
|
|
|
|
|
2025 |
|
2024 |
|
Change |
|
% Change |
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
Research and development |
|
|
70,886 |
|
|
42,805 |
|
|
28,081 |
|
|
66 |
% |
General and administrative |
|
|
13,503 |
|
|
8,212 |
|
|
5,291 |
|
|
64 |
% |
Loss from operations |
|
|
(84,389 |
) |
|
(51,017 |
) |
|
(33,372 |
) |
|
65 |
% |
Interest income and other income, net |
|
|
12,211 |
|
|
11,445 |
|
|
766 |
|
|
7 |
% |
Net loss |
|
$ |
(72,178 |
) |
$ |
(39,572 |
) |
$ |
(32,606 |
) |
|
82 |
% |
Collaboration Revenue
There was no collaboration revenue recognized for the three months ended March 31, 2025 and March 31, 2024.
We completed all performance obligations related to the upfront payment under the GSK Collaboration Agreement as of December 31, 2023. Future collaboration revenue recognized under the GSK Collaboration Agreement will be related to future milestone payments as they are earned.
Research and Development Expenses
Research and development expenses increased by $28.0 million, or 66%, during the three months ended March 31, 2025 compared to the three months ended March 31, 2024 primarily due to increases in $22.0 million in fees paid to CROs, CMOs and consultants related to the advancement of our lead product candidates through preclinical and clinical studies, $3.6 million in personnel-related expenses to support our growth, and $2.4 million in costs for laboratory supplies, facilities and information technology costs to support our research and development programs.
General and Administrative Expenses
General and administrative expenses increased by $5.3 million, or 64%, during the three months ended March 31, 2025 compared to the three months ended March 31, 2024. The increase in general and administrative expenses was primarily due to increases of $2.6 million in personnel-related expenses and $2.7 million in consulting and legal patent expenses to support our growth.
Interest Income and Other Income, Net
Interest income increased by $0.8 million, or 7%, during the three months ended March 31, 2025 compared to the three months ended March 31, 2024, primarily due to higher investment balances.
Liquidity and Capital Resources; Plan of Operations
Sources of Liquidity
We have funded our operations primarily through the sale and issuance of common stock and the upfront payment and certain milestone payments received from GSK. As of March 31, 2025, we had cash, cash equivalents and marketable securities of approximately $1.05 billion, consisting primarily of money market funds, U.S. government securities, commercial paper, and corporate bonds.
Material Cash Requirements
We have incurred net losses since our inception. For the three months ended March 31, 2025 and March 31, 2024, we had net losses of $72.2 million and $39.6 million, respectively, and we expect to incur substantial additional
losses in future periods. As of March 31, 2025, we had an accumulated deficit of $695.0 million. Based on our current business plan, we believe that our existing cash, cash equivalents and marketable securities will be sufficient to fund our planned operations for at least the next 12 months from the issuance date of this Quarterly Report on Form 10-Q.
To date, we have not generated any product revenue. We do not expect to generate any meaningful product revenue unless and until we obtain regulatory approval of and commercialize any of our product candidates, and we do not know when, or if, it will occur. We expect to continue to incur significant losses for the foreseeable future, and we expect the losses to increase as we continue the development of, and seek regulatory approvals for our product candidates, and begin to commercialize any approved products. We are subject to all of the risks typically related to the development of new product candidates, and we may encounter unforeseen expenses, difficulties, complications, delays and other unknown factors that may adversely affect our business. Moreover, we expect to incur additional costs associated with operating as a public company.
We will continue to require additional capital to develop our product candidates and fund operations for the foreseeable future. We may seek to raise capital through private or public equity or debt financings, collaboration or other arrangements with corporate sources, or through other sources of financing. Adequate additional funding may not be available to us on acceptable terms or at all. Our failure to raise capital as and when needed would have a negative impact on our financial condition and our ability to pursue our business strategies. We anticipate that we will need to raise substantial additional capital, the requirements for which will depend on many factors, including:
•the scope, timing, rate of progress and costs of our drug discovery, preclinical development activities, laboratory testing and clinical trials for our product candidates;
•the number and scope of clinical programs we decide to pursue;
•the scope and costs of manufacturing development and commercial manufacturing activities;
•the extent to which we acquire or in-license other product candidates and technologies;
•the cost, timing and outcome of regulatory review of our product candidates;
•the costs of preparing, filing and prosecuting patent applications, maintaining and enforcing our intellectual property rights and defending intellectual property-related claims;
•our ability to establish and maintain collaborations on favorable terms, if at all;
•our efforts to enhance operational systems and our ability to attract, hire and retain qualified personnel, including personnel to support the development of our product candidates;
•the costs associated with being a public company; and
•the cost and timing associated with commercializing our product candidates if they receive marketing approval.
A change in the outcome of any of these or other variables with respect to the development of any of our product candidates could significantly change the costs and timing associated with the development of that product candidate. Furthermore, our operating plans may change in the future, and we will continue to require additional capital to meet operational needs and capital requirements associated with such operating plans. If we raise additional funds by issuing equity securities, our stockholders may experience dilution. Any future debt financing into which we enter may impose upon us additional covenants that restrict our operations, including limitations on our ability to incur liens or additional debt, pay dividends, repurchase our common stock, make certain investments or engage in certain merger, consolidation or asset sale transactions.
Any debt financing or additional equity that we raise may contain terms that are not favorable to us or our stockholders. If we are unable to raise additional funds when needed, we may be required to delay, reduce, or terminate some or all of our development programs and clinical trials. We may also be required to sell or license to others rights to our product candidates in certain territories or indications that we would prefer to develop and commercialize ourselves.
In June 2023, we entered into a lease agreement for approximately 44,000 square feet of laboratory and office facilities at 5000 Shoreline Court, South San Francisco, California. The lease term is 120 months, and we have an option to extend the lease term for a total of two consecutive five-year periods. The lease commenced in August 2024.
In May 2024, we amended our 5000 Shoreline Court facility lease agreement to expand the size of the original premises by adding approximately 11,321 rentable square feet of additional space. The amendment to the lease term commenced in January 2025.
In November 2023, we entered into a lease agreement for approximately 5,700 square feet of space at 11710 El Camino Real, San Diego, California for corporate office space. The lease commenced in December 2023 and expires in March 2028. We have an option to renew the lease for three years.
We enter into contracts in the normal course of business with third-party contract organizations for preclinical and clinical studies and testing, manufacture and supply of our preclinical and clinical materials and providing other services and products for operating purposes. These contracts generally provide for termination following a certain period after notice, and therefore, we believe that our non-cancellable obligations under these agreements are not material.
See Notes 5. Operating Leases, 6. Commitments and Contingencies, 7. Income Taxes and 10. Significant Agreements.
Adequate additional funding may not be available to us on acceptable terms or at all.
See the section of this Quarterly Report on Form 10-Q titled “Part I, Item 1A. – Risk Factors” for additional risks associated with our substantial capital requirements.
Off-Balance Sheet Arrangements
We have not entered into any off-balance sheet arrangements as defined in the rules and regulations of the SEC.
Summary Statement of Cash Flows
The following table sets forth the primary sources and uses of cash, cash equivalents, and restricted cash for each of the periods presented below (in thousands):
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Three Months Ended March 31, |
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2025 |
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2024 |
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Net cash provided by (used in): |
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Operating activities |
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$ |
(60,342 |
) |
$ |
(43,813 |
) |
Investing activities |
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79,817 |
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(353,971 |
) |
Financing activities |
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26,143 |
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349,111 |
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Net increase (decrease) in cash, cash equivalents and restricted cash |
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$ |
45,618 |
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$ |
(48,673 |
) |
Cash Flows from Operating Activities
Net cash used in operating activities was $60.3 million for the three months ended March 31, 2025. Cash used in operating activities was primarily due to the use of funds in our operations to develop our product candidates resulting in a net loss of $72.2 million, adjusted for net non-cash charges of $7.6 million and changes in net operating assets and liabilities of $4.2 million. Our non-cash charges consisted of $10.2 million in stock-based compensation, $0.6 million in depreciation and $0.5 million of the amortization of right of use assets, partially offset by $3.8 million accretion of discounts on marketable securities. The net change in our operating assets and liabilities consisted primarily due to cash inflows from $2.7 million in accounts payable, $2.9 million accrued and other liabilities in support of research and manufacturing activities and $0.6 million in lease liabilities, partially offset by outflows of $2.0 million in prepaid and other assets.
Net cash used in operating activities was $43.8 million for the three months ended March 31, 2024. Cash used in operating activities was primarily due to the use of funds in our operations to develop our product candidates resulting in a net loss of $39.6 million, adjusted for net non-cash charges of $1.1 million and changes in net
operating assets and liabilities of $5.3 million. Our non-cash charges consisted of $6.3 million in stock-based compensation, $0.6 million in depreciation and $0.5 million of the amortization of right of use assets, partially offset by $6.3 million accretion of discounts on marketable securities. The net change in our operating assets and liabilities consisted primarily of $3.1 million in prepaid and other assets, $1.3 million accrued and other liabilities due to CRO fees in support of research and manufacturing activities, $0.5 million in lease liabilities, and $0.5 million in accounts payable.
Cash Flows from Investing Activities
Net cash provided by investing activities was $79.8 million for the three months ended March 31, 2025, which consisted primarily of $105.2 million used to purchase marketable securities and $1.3 million used to purchase property and equipment, offset by $186.4 million provided by maturities of marketable securities.
Net cash used in investing activities was $354.0 million for the three months ended March 31, 2024, which consisted primarily of $475.8 million used to purchase marketable securities and $1.3 million used to purchase property and equipment, partially offset by $123.1 million provided by maturities of marketable securities.
Cash Flows from Financing Activities
Net cash provided by financing activities was $26.1 million for the three months ended March 31, 2025, which consisted primarily of $25.0 million in net proceeds from at-the-market offerings and $1.1 million in proceeds from exercise of common stock options.
Net cash provided by financing activities was $349.1 million for the three months ended March 31, 2024, which consisted primarily of $343.7 million of net proceeds from sales under our ATM facility and $5.5 million of proceeds from exercise of common stock options.
Critical Accounting Policies
Our financial statements have been prepared in accordance with U.S. generally accepted accounting principles, or GAAP. The preparation of these financial statements requires us to make estimates and assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities at the date of the financial statements and the reported revenue recognized and expenses incurred during the reporting periods. Our estimates are based on our historical experience and on various other factors that we believe are reasonable under the circumstances, the results of which form the basis for making judgments about the carrying value of assets and liabilities that are not readily apparent from other sources. Actual results may differ from these estimates under different assumptions or conditions. We believe that the accounting policies discussed below are critical to understanding our historical and future performance, as these policies relate to the more significant areas involving management’s judgments and estimates.
For more detail on our critical accounting policies, refer to Note 2 in the unaudited interim condensed financial statements appearing elsewhere in this Quarterly Report on Form 10-Q, and the notes to the financial statements appearing elsewhere in our Annual Report on Form 10-K filed with the SEC on February 18, 2025. For the three months ended March 31, 2025, there were no material changes to our critical accounting policies from those disclosed in our Annual Report on Form 10-K filed with the SEC on February 18, 2025.
Item 3. Quantitative and Qualitative Disclosures About Market Risk.
Interest Rate Sensitivity
The market risk inherent in our financial instruments and in our financial position represents the potential loss arising from adverse changes in interest rates or exchange rates. As of March 31, 2025, we had cash, cash equivalents and marketable securities of approximately $1.05 billion, consisting of bank deposits, interest-bearing money market funds, investments in U.S. government securities, commercial paper, and corporate bonds, for which the fair value would be affected by changes in the general level of U.S. interest rates. Even if the fair value of certain government securities, commercial paper, and corporate bonds is affected by changes in U.S. interest rates, the principal of such instruments will be due to us upon maturity.
The primary objective of our investment activities is to preserve capital to fund our operations. We also seek to maximize income from our investments without assuming significant risk. Because our investments are primarily short-term in duration and our holdings in U.S. government treasury bonds mature prior to our expected need for liquidity, we believe that our exposure to interest rate risk is not significant.
While we are seeing, and expect to continue to see, record inflation due to geopolitical and macroeconomic events, such as the ongoing Ukraine-Russia conflict and related sanctions, the Israel-Hamas conflict, and the banking sector volatility, we do not believe that inflation, or exchange rate fluctuations have had a significant impact on our results of operations for any periods presented herein.
Item 4. Controls and Procedures.
Limitations on Effectiveness of Controls and Procedures
In designing and evaluating our disclosure controls and procedures, management recognizes that any controls and procedures, no matter how well designed and operated, can provide only reasonable assurance of achieving the desired control objectives. In addition, the design of disclosure controls and procedures must reflect the fact that there are resource constraints and that management is required to apply judgment in evaluating the benefits of possible controls and procedures relative to their costs.
Evaluation of Disclosure Controls and Procedures
Our management, with the participation of our Principal Executive Officer and Principal Financial and Accounting Officer, evaluated, as of the end of the period covered by this Quarterly Report on Form 10-Q, the effectiveness of our disclosure controls and procedures (as defined in Rules 13a-15(e) and 15d-15(e) under the Exchange Act. Based on that evaluation, our Principal Executive Officer and Principal Financial and Accounting Officer concluded that our disclosure controls and procedures were effective at the reasonable assurance level as of March 31, 2025.
Changes in Internal Control over Financial Reporting
There was no change in our internal control over financial reporting identified in connection with the evaluation required by Rule 13a-15(e) and 15d-15(e) of the Exchange Act that occurred during the period covered by this Quarterly Report on Form 10-Q that has materially affected, or is reasonably likely to materially affect, our internal control over financial reporting.
PART II—OTHER INFORMATION
Item 1. Legal Proceedings.
From time to time, we may become involved in litigation or other legal proceedings. We are not currently a party to any litigation or legal proceedings that, in the opinion of our management, are likely to have a material adverse effect on our business. Regardless of the outcome, litigation can have an adverse impact on our business, financial condition, results of operations and prospects because of defense and settlement costs, diversion of management resources and other factors.
Item 1A. Risk Factors.
In addition to other information contained elsewhere in this Quarterly Report on Form 10-Q, you should carefully consider the risk factors discussed in Part I, Item 1A. Risk Factors in our Annual Report on Form 10-K filed with the SEC on February 18, 2025, or the Annual Report, which could materially affect our business, financial condition, or future results. As of the date of this Quarterly Report on Form 10-Q, there have been no material changes to the risk factors disclosed in our Annual Report.
Item 2. Unregistered Sales of Equity Securities and Use of Proceeds.
Unregistered Sales of Equity Securities
None.
Use of Proceeds from the Sale of Registered Securities
Not applicable.
Issuer Purchases of Equity Securities
None.
Item 3. Defaults Upon Senior Securities.
Not applicable.
Item 4. Mine Safety Disclosures.
Not applicable.
Item 5. Other information.
Trading Plans
During the three months ended March 31, 2025, no Section 16 officers and directors adopted or terminated contracts, instructions or written plans for the purchase or sale of our securities.
Employment Agreement with Joshua Bleharski, Ph.D.
On February 10, 2025, the Company filed a Current Report on Form 8-K (the “Original Form 8-K”) with the Securities and Exchange Commission announcing the appointment of Joshua Bleharski, Ph.D. as the Company’s Chief Financial Officer. The following description supplements the Original Form 8-K as follows to report the compensation arrangements with Dr. Bleharski pursuant to an employment agreement, effective as of May 1, 2025 (the “Employment Agreement”) by and between the Company and Dr. Bleharski. The Board expects to appoint Dr. Bleharski as the “principal financial officer” of the Company under Section 16(a)-1(f) of the Exchange Act.
The Employment Agreement provides for Dr. Bleharski’s base salary, target cash incentive, benefit plan participation and severance benefits. Pursuant to the Employment Agreement, Dr. Bleharski will receive an annual base salary of $500,000 and a target annual performance bonus amount of 40% of his annual base salary (subject to achievement of certain performance objectives). In the event that Dr. Bleharski’s employment is terminated by the Company without “cause” or he resigns for “good reason” (each, as defined in the Employment Agreement) outside of a Change in Control (as defined in the Employment Agreement), then, subject to timely delivering to us a release of claims, he will be entitled to receive: (i) a lump sum severance payment equal to 75% of his annual base salary; and (ii) payment or reimbursement of continued healthcare coverage for up to 9 months following the date of termination. In the event that Dr. Bleharski’s employment is terminated by the Company without “cause” or he resigns for “good reason” during a period of time that begins three months prior to and ends 12 months following a Change in Control, then, subject to timely delivering to us a release of claims, he will be entitled to receive: (i) a lump sum severance payment equal to his annual base salary and target annual bonus; (ii) payment or reimbursement of continued healthcare coverage for up to 12 months following the date of termination; and (iii) full acceleration of his equity awards.
The foregoing is only a summary description of the terms of the Employment Agreement, does not purpose to be complete and is qualified in its entirety by reference to the Employment Agreement, which will be filed as an exhibit to the Company’s Quarterly Report on Form 10-Q for the fiscal quarter ending June 30, 2025.
Pursuant to the Employment Agreement, the Company expects to grant Dr. Bleharski a stock option to purchase 250,000 shares of the Company’s common stock.
Exhibit Index
Item 6. Exhibits.
Portions of this exhibit have been omitted pursuant to Regulation S-K, Item 601(b)(10) or certain schedules and attachments to this exhibit have been omitted pursuant to Regulation S-K, Item 601(a)(5). Such omitted information is not material and would likely cause competitive harm to the registrant if publicly disclosed.
# Indicates management contract or compensatory plan.
* The certification attached as Exhibit 32.1 that accompanies this Quarterly Report on Form 10-Q is not deemed filed with the SEC and is not to be incorporated by reference into any filing of IDEAYA Biosciences, Inc. under the Securities Act of 1933, as amended, or the Securities Exchange Act of 1934, as amended, whether made before or after the date of this Form 10-Q, irrespective of any general incorporation language contained in such filing.
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned thereto duly authorized.
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IDEAYA Biosciences, Inc. |
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Date: May 6, 2025 |
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By: |
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/s/ Yujiro Hata |
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Yujiro Hata |
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President and Chief Executive Officer |
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(Principal Executive Officer) |
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Date: May 6, 2025 |
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By: |
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/s/ Andres Ruiz Briseno |
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Andres Ruiz Briseno |
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Chief Accounting Officer |
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(Principal Financial and Accounting Officer) |
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