Quarterly Report for Quarter Ending September 30, 2025 (Form 10-Q)

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, development, and commercialization of transformative therapies for cancer. Our approach integrates expertise in small-molecule drug discovery, structural biology and bioinformatics with robust internal capabilities in identifying and validating translational biomarkers to develop tailored, potentially first-in-class targeted therapies aligned to the genetic drivers of disease. We have built a deep pipeline of product candidates focused on synthetic lethality and antibody-drug conjugates, or ADCs, for molecularly defined solid tumor indications. Our clinical development strategy is to evaluate our product candidates in rational combinations, where appropriate, and earlier in the course of disease in the adjuvant and neoadjuvant settings which we believe has the potential to maximize their impact. Our mission is to bring forth the next wave of precision oncology therapies that are more selective, more effective, and deeply personalized with the goal of altering the course of disease and improving clinical outcomes for patients with cancer.

Our clinical pipeline currently consists of seven potential first-in-class product candidates, as follows:

We are also evaluating darovasertib as a single-agent in the neoadjuvant setting of primary UM, where the goal of treatment is to prevent enucleation (surgical eye removal), preserve vision prior to and post-plaque brachytherapy and potentially delay or prevent progression to metastatic disease. In October 2025 at the European Society for Medical Oncology Conference in Berlin, Germany, we presented updated data from more than 90 enucleation-eligible and plaque brachytherapy-eligible patients in our ongoing Phase 2 trial (OptimUM-09). We are currently conducting a randomized Phase 3 trial of darovasertib in the neoadjuvant setting of primary UM (OptimUM-10), which will enroll approximately 450 patients across two cohorts of plaque brachytherapy-eligible and enucleation-eligible patients. Target enrollment in this trial was revised downward from the prior estimate of 520 patients based on additional FDA feedback on the statistical plan (alpha usage) across the two cohorts.

In August 2025, we entered into an exclusive licensing agreement with Les Laboratoires Servier, or Servier, for the regulatory and commercial rights to darovasertib outside of the United States. We received an upfront payment of $210.0 million and are eligible for up to $320.0 million in development, regulatory and commercial-based milestone payments, as well as double-digit royalties on net sales in all territories outside of the United States. Servier will be responsible for the regulatory and commercial activities for darovasertib outside the United States. We and Servier will collaborate on the development of darovasertib and share the associated costs. We retain all rights to darovasertib in the United States.

In addition to our clinical stage pipeline, we are advancing two pre-clinical development programs:

Since our inception, we have invested significantly in capabilities for identifying and validating new precision medicine targets and biomarkers for patient selection and have established multiple research collaborations to support our internal discovery and development efforts. We have also partnered with leading pharmaceutical companies, including GSK, Pfizer and Gilead to support clinical development activities for our pipeline programs.

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 and Servier. As of September 30, 2025, we had cash, cash equivalents and marketable securities of approximately $1.14 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. For information about our specific program costs and expenses, see Note 10. Significant Agreements.

Our net losses were $30.4 million and $144.2 million for the nine months ended September 30, 2025 and September 30, 2024, respectively. As of September 30, 2025, we had an accumulated deficit of $653.3 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 September 30, 2025, we had cash, cash equivalents and short-term and long-term marketable securities of approximately $1.14 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 on November 4, 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 and Servier License Agreement, including amounts that are recognized related to previously received upfront payments, development milestone 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 September 30, 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.

In August 2025, the Company entered into the Servier License Agreement for development and commercial rights to darovasertib in all countries worldwide except for the United States. The Company recognized revenue related to the D&C license performance obligation at a point in time during the three months ended September 30, 2025 upon execution of the contract. The Company recognizes revenue related to amounts allocated to research and development services over time, as the underlying services are performed over the period through the completion of program development activities. The timing of revenue recognition, billings, and cash collections results in accounts receivable, contract assets, and contract liabilities on the balance sheets.

The Company will also recognize revenue from development and regulatory milestones as they are achieved. Future net product sales may also result in royalty payments.

When consideration is received, or such consideration is unconditionally due, from a customer prior to transferring goods or services to the customer under the terms of a contract, a contract asset or contract liability is recorded. Contract liabilities are derecognized as revenue after control of the products or services is transferred to the customer and all revenue recognition criteria have been met.

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, IDE161, IDE275 / GSK959, IDE705 / GSK 101 and IDE892, 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:

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 September 30, 2025 compared to the three months ended June 30, 2025 and the nine months ended September 30, 2025 compared to the nine months ended September 30, 2024 is presented below.

Comparison of Three Months Ended September 30, 2025 and June 30, 2025

The following table summarizes our results of operations for the periods indicated (in thousands):

Collaboration Revenue

Collaboration Revenue was $207.8 million for the three months ended September 30, 2025 as a result of revenue recognized from the Servier License Agreement. See Note 11. Revenue Recognition.

Research and Development Expenses

Research and development expenses increased by $8.8 million, or 12%, during the three months ended September 30, 2025 compared to the three months ended June 30, 2025 primarily due to $8.8 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 $1.8 million, or 12%, during the three months ended September 30, 2025 compared to the three months ended June 30, 2025. The increase in general and administrative expense was primarily due to $1.8 million in consulting and legal patent expenses to support our growth.

Interest Income and Other Income, Net

Interest income decreased by $0.5 million, or 5%, during the three months ended September 30, 2025 compared to the three months ended June 30, 2025 primarily due to lower interest rates.

Comparison of Nine Months Ended September 30, 2025 and September 30, 2024

The following table summarizes our results of operations for the periods indicated (in thousands):

Collaboration Revenue

Collaboration Revenue was $207.8 million for the nine months ended September 30, 2025 as a result of revenue recognized from the Servier License Agreement. See Note 11. Revenue Recognition.

Research and Development Expenses

Research and development expenses increased by $73.6 million, or 48%, during the nine months ended September 30, 2025 compared to the nine months ended September 30, 2024 primarily due to $59.4 million in fees paid to CROs, CMOs, and consultants related to the advancement of our lead product candidates through preclinical and clinical studies, $9.0 million in personnel-related expenses and $5.2 million in costs for laboratory supplies, facilities and software license costs to support our research and development programs.

General and Administrative Expenses

General and administrative expenses increased by $16.1 million, or 57%, during the nine months ended September 30, 2025 compared to the nine months ended September 30, 2024 primarily due to increases in $8.4 million in personnel-related expenses and $7.7 million in consulting services and legal patent expenses to support our growth.

Interest Income and Other Income, Net

Interest income decreased by $4.4 million, or 11%, during the nine months ended September 30, 2025 compared to the nine months ended September 30, 2024 primarily due to lower interest rates.

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 and Servier. As of September 30, 2025, we had cash, cash equivalents and marketable securities of approximately $1.14 billion, consisting primarily of money market funds, U.S. government securities, commercial paper, and corporate bonds.

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 nine months ended September 30, 2025, we sold an aggregate of 984,000 shares of our common stock for an 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 September 30, 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.

Material Cash Requirements

We have primarily incurred net losses since our inception. For the nine months ended September 30, 2025 and September 30, 2024, we had net losses of $30.4 million and $144.2 million, respectively, and we expect to incur substantial additional losses in future periods. As of September 30, 2025, we had an accumulated deficit of $653.3 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:

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.

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.

For more information see Notes 5. Operating Leases, 6. Commitments and Contingencies, 7. Income Taxes and 10. Significant Agreements, each in the unaudited interim condensed financial statements appearing elsewhere in this Quarterly Report on Form 10-Q.

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):

Cash Flows from Operating Activities

Net cash provided by operating activities was $19.4 million for the nine months ended September 30, 2025. Cash provided by operating activities was primarily due to the use of funds in our operations to develop our product candidates, partially offset by the upfront payment from Servier License Agreement resulting in a net loss of $30.4 million, adjusted for net non-cash charges of $29.3 million and changes in net operating assets and liabilities of $20.6 million. Our non-cash charges consisted of $34.3 million in stock-based compensation, $2.0 million in depreciation and $1.4 million of the amortization of right of use assets, partially offset by $8.5 million accretion of discounts on marketable securities. The net change in our operating assets and liabilities consisted primarily due to cash inflows from $3.6 million in accounts payable, $15.2 million accrued and other liabilities in support of the advancement of our lead product candidates, $2.2 million in contract liabilities related to the Servier License Agreement, $1.9 million in lease liabilities, partially offset by outflows of $2.3 million in prepaid and other assets.

Net cash used in operating activities was $125.9 million for the nine months ended September 30, 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 $144.2 million, adjusted for net non-cash charges of $10.1 million and changes in net

operating assets and liabilities of $8.1 million. Our non-cash charges consisted of $25.3 million in stock-based compensation, $1.8 million in depreciation and $1.4 million of the amortization of right of use assets, partially offset by $18.3 million accretion of discounts on marketable securities. The net change in our operating assets and liabilities consisted primarily due to cash inflows from $5.3 million in accounts payable and $8.5 million accrued and other liabilities due to CRO fees in support of research and manufacturing activities, partially offset by outflows of $4.4 million in prepaid and other assets and $1.3 million in lease liabilities.

Cash Flows from Investing Activities

Net cash provided by investing activities was $167.5 million for the nine months ended September 30, 2025, which consisted primarily of $371.8 million used to purchase marketable securities and $2.0 million used to purchase property and equipment, offset by $541.3 million provided by maturities of marketable securities.

Net cash used in investing activities was $305.5 million for the nine months ended September 30, 2024, which consisted primarily of $854.3 million used to purchase marketable securities and $2.8 million used to purchase property and equipment, partially offset by $551.6 million provided by maturities of marketable securities.

Cash Flows from Financing Activities

Net cash provided by financing activities was $27.6 million for the nine months ended September 30, 2025, which consisted primarily of $24.9 million in net proceeds from at-the-market offerings, $1.9 million in proceeds from exercise of common stock options and $0.8 million in proceeds from ESPP purchases.

Net cash provided by financing activities was $674.8 million for the nine months ended September 30, 2024, which consisted primarily of $379.9 million of net proceeds from at-the-market offerings, $274.7 million of proceeds from issuance of common stock upon public offering, $9.4 million of proceeds from issuance of pre-funded warrants, $10.0 million of proceeds from exercise of common stock options and $0.8 million of proceeds from ESPP purchases.

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 nine months ended September 30, 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.