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Verastem, Inc.

For the period ended Jun 30, 2025. Filed Aug 7, 2025.

View on SEC.gov

About this filing

A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.

Jun 30, 2025 · Filed Aug 7, 2025

VSTM$8.41-10.3%Stored close · as of Nov 6, 2026

What changed in Management’s Discussion and Analysis?

Read the current filing

Previous report: 10-Q

Period ended 2025-03-31; filed 2025-05-13.

0001558370-25-007570

Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations

Current report: 10-Q

Period ended 2025-06-30; filed 2025-08-07.

0001558370-25-010757

Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations

The baseline is this issuer’s latest earlier reporting period of the same form that was filed before the current report. A previous 10-Q is a different quarter, not a year-over-year comparison. Amendments are excluded.

Compared 55 previous paragraphs and 79 current paragraphs.

24 unchanged; 30 reworded; 15 added; 0 removed; 1 previous and 10 current paragraphs rearranged.

Tables are not compared: 9 previous and 11 current tables excluded. Dates, whitespace, punctuation and numbering changes are ignored; changes to financial figures remain significant.

A changed paragraph is not evidence that risk increased or decreased. An unchanged paragraph does not establish that risk stayed the same.

Added paragraphs (15)

Current report

At the ASCO 2025 Annual Meeting, GenFleet announced updated data from the dose escalation phase of the Phase 1/2 trial of GFH375 (known as VS-7375 in the U.S.). GenFleet reported that 62 patients were enrolled in the Phase 1 portion of the study in China, receiving oral doses ranging from 100 to 900 mg daily. In the study, 98% of patients had metastatic disease, and 75% had received ≥2 prior lines of therapy. As of the data cutoff of May 16, 2025, 23 efficacy-evaluable patients with PDAC and 12 efficacy-evaluable patients with NSCLC, who received daily dosages of 400 or 600 mg and had at least one post-treatment tumor assessment, achieved an objective response rate (“ORR”) of 52% and a disease control rate (DCR) of 100%, and an ORR of 42% and a DCR of 83%, respectively. As of the cutoff date

Current report

of March 31, 2025, GenFleet reported that there were no dose-limiting toxicities observed across all dose levels (100-900 mg QD), and the treatment-related adverse events (“TRAEs”) were mostly Grade 1/2. The most common TRAEs occurring in at least 20% of patients were diarrhea, nausea, vomiting, and anemia. TRAEs ≥ Grade 3 consisted mainly of decreased neutrophil count (8%) and diarrhea (5%). No TRAE-related deaths were reported.

Current report

*Product Revenue, Net.*We began commercial sales of AVMAPKI FAKZYNJA CO-PACK within the United States in May 2025, following receipt of FDA marketing approval on May 8, 2025. For the three months ended June 30, 2025 (the “2025 Quarter”) we recorded approximately $2.1 million of net product revenue. We had no product revenue during the three months ended June 30, 2024 (the “2024 Quarter”).

Current report

*Sale of COPIKTRA license and related assets revenue.*Sale of COPIKTRA license and related assets revenue for the 2025 Quarter was $0.0 million compared to $10.0 million for the 2024 Quarter. Sale of COPIKTRA license and related assets revenue for the 2024 Quarter was comprised of one sales milestone payment of $10.0 million due to Secura achieving cumulative worldwide net sales of COPIKTRA exceeding $100.0 million during the 2024 Quarter. We received the $10.0 million milestone payment in July 2024.

Current report

*Costs of sales – product.*Costs of sales – product for the 2025 Quarter of approximately $0.3 million consisted of costs associated with the manufacturing of AVMAPKI FAKZYNJA CO-PACK, royalties owed to Chugai, Pfizer, and the Institute for Cancer Research (“ICR”) on such sales, and certain period costs. The Company began capitalizing inventory upon receiving FDA approval for AVMAPKI FAKZYNJA CO-PACK on May 8, 2025. Prior to the FDA approval of AVMAPKI FAKZYNJA CO-PACK, expenses associated with the manufacturing of AVMAPKI FAKZYNJA CO-PACK were recorded as research and development expense. Certain of the costs of AVMAPKI FAKZYNJA CO-PACK units recognized as revenue during the 2025 Quarter, or less than $0.1 million, were expensed prior to obtaining regulatory approval, therefore, are not included in cost of sales- product during this period. We expect cost of sales - product to increase in relation to product revenues as we deplete these inventories. We had no cost of sales – product during the 2024 Quarter.

Current report

*Costs of sales –intangible amortization.*Cost of sales – intangible amortization for the 2025 Quarter of approximately $0.1 million was related to finite-lived intangible assets related to AVMAPKI FAKZYNJA CO-PACK which we recognized and began amortizing in the 2025 Quarter. There was no cost of sales – intangible amortization the 2024 Quarter.

Current report

*Product Revenue, Net.*We began commercial sales of AVMAPKI FAKZYNJA CO-PACK within the United States in May 2025, following receipt of FDA marketing approval on May 8, 2025. For the six months ended June 30, 2025 (the “2025 Period”) we recorded approximately $2.1 million of net product revenue. We had no product revenue during the six months ended June 30, 2024 (the “2024 Period”).

Current report

*Sale of COPIKTRA license and related assets revenue.*Sale of COPIKTRA license and related assets revenue for the 2025 Period was $0.0 million compared to $10.0 million for the 2024 Period. Sale of COPIKTRA license and related assets revenue for the 2024 Period was comprised of one sales milestone payment of $10.0 million due to Secura achieving cumulative worldwide net sales of COPIKTRA exceeding $100.0 million during the 2024 Quarter. We received the $10.0 million milestone payment in July 2024.

Current report

*Costs of sales – product.*Costs of sales – product for the 2025 Period of approximately $0.3 million consisted of costs associated with the manufacturing of AVMAPKI FAKZYNJA CO-PACK, royalties owed to Chugai, Pfizer, and ICR on such sales, and certain period costs. We began capitalizing inventory upon receiving FDA approval for AVMAPKI FAKZYNJA CO-PACK on May 8, 2025. Prior to the FDA approval of AVMAPKI FAKZYNJA CO-PACK, expenses associated with the manufacturing of AVMAPKI FAKZYNJA CO-PACK were recorded as research and development expense. Certain of the costs of AVMAPKI FAKZYNJA CO-PACK units recognized as revenue during the 2025 Period, or less than $0.1 million, were expensed prior to obtaining regulatory approval, therefore, are not included in cost of sales - product during this period. We expect cost of sales - product to increase in relation to product revenues as we deplete these inventories. We had no cost of sales – product during the 2024 Period.

Current report

*Costs of sales –intangible amortization.*Cost of sales – intangible amortization for the 2025 Period of approximately $0.1 million was related to finite-lived intangible assets related to AVMAPKI FAKZYNJA CO-PACK which we recognized and began amortizing in the 2025 Period. There was no cost of sales – intangible amortization the 2024 Period.

Current report

Research and development expense.* *Research and development expense for the Period was $53.9 million compared to $35.8 million for the 2024 Period. The $18.2 million increase in R&D expense from the 2024 period to the

Current report

2025 period was primarily driven by a $6.0 million GenFleet option payment related to VS-7375, a $4.7 million increase in CRO costs, a $2.5 million increase in investigator fees, a $2.3 million increase in personnel costs including non-cash stock compensation, a $2.1 million increase in drug substance and drug product manufacturing costs, and a $0.9 million increase in clinical supply costs. These increases were partially offset by a $0.3 million decrease in investigator-sponsored trial (“IST”) expenses.

Current report

Selling, general and administrative expense. Selling, general and administrative expense for the 2025 Period was $35.7 million compared to $20.6 million for the 2024 Period. The increase of $15.1 million from the 2024 Period to the 2025 Period was primarily driven by an $11.7 million increase in consulting and professional fees, a $3.7 million increase in personnel costs including non-cash stock-based compensation, a $1.1 million financing cost fee related to the Note Purchase Agreement and the 2025 Private Placement, and a $1.1 million increase in travel, conference, and other expenses. These increases were partially offset by a $2.5 million decrease in commercial operations expenses.

Current report

*Interest expense.*Interest expense for the 2025 Period was $0.4 million compared to $2.3 million for the 2024 Period. The decrease of $1.9 million from the 2024 Periodwas primarily driven by termination of the Loan Agreement with Oxford on January 13, 2025 resulting in a reduction of interest expense recorded in the 2025 Period. The Company
Reworded paragraphs (30)

Previous wording

We are a biopharmaceutical company committed to the development and commercialization of new medicines to improve the lives of patients diagnosed withras sarcoma (“RAS”)/ mitogen activated pathway kinase (“MAPK”)pathway-driven cancers. We market AVMAPKI FAKZYNJA CO-PACK (avutometinib capsules; defactinib tablets) in the United States. Our pipeline is focused on novel small molecule drugs that inhibit critical signaling pathways in cancer that promote cancer cell survival and tumor growth, including RAF/mitogen-activated protein kinase kinase (“MEK”) inhibition, FAK inhibition and KRAS G12D inhibition.

Current wording

We are a biopharmaceutical company committed to the development and commercialization of new medicines to improve the lives of patients diagnosed with RAS MAPK pathway-driven cancers. We market AVMAPKI™ FAKZYNJA™ CO-PACK (avutometinib capsules; defactinib tablets) in the United States. Our pipeline is focused on novel small molecule drugs that inhibit critical signaling pathways in cancer that promote cancer cell survival and tumor growth, including RAF/MEK inhibition, FAK inhibition and KRAS G12D inhibition.

Previous wording

On May 8, 2025, the FDA approved AVMAPKI FAKZYNJA CO-PACK for the treatment of adult patients with Kirsten rat sarcoma viral oncogene homolog (“KRAS”) mutant (“KRAS mt”) recurrent LGSOC who received prior systemic therapy. The KRAS mt recurrent LGSOC indication has been granted approval by the FDA under the accelerated approval pathway based on the tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

Current wording

On May 8, 2025, the FDA approved AVMAPKI FAKZYNJA CO-PACK for the treatment of adult patients with “KRAS mt” recurrent LGSOC who received prior systemic therapy. The KRAS mt recurrent LGSOC indication has been granted approval by the FDA under the accelerated approval pathway based on the tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

Previous wording

In the fourth quarter of 2020, we commenced a registration-directed trial investigating avutometinib in combination with defactinib for the treatment of patients with recurrent LGSOC entitled RAMP 201 study. We use the term “RAMP” to refer to our RAF and MEK Program. The RAMP 201 study is an adaptive two-part multicenter, parallel cohort, randomized, open label trial evaluating the efficacy and safety of avutometinib alone and in combination with defactinib in patients with recurrent LGSOC. The primary analysis from both the FRAME trial and RAMP 201 trial are anticipated to be published in the first half of 2025.

Current wording

In the fourth quarter of 2020, we commenced a registration-directed trial investigating avutometinib in combination with defactinib for the treatment of patients with recurrent LGSOC entitled RAMP 201 study. We use the term “RAMP” to refer to our RAF and MEK Program. The RAMP 201 study is an adaptive two-part multicenter, parallel cohort, randomized, open label trial evaluating the efficacy and safety of avutometinib alone and in combination with defactinib in patients with recurrent LGSOC. The primary analysis from both the FRAME trial and RAMP 201 trial has been published.

Previous wording

In December 2023, we announced the initiation of a confirmatory Phase 3 trial to evaluate the combination of avutometinib and defactinib for the treatment of patients with recurrent LGSOC entitled RAMP 301. RAMP 301 is a randomized global confirmatory trial, which is evaluating the efficacy and safety of avutometinib and defactinib versus standard of care chemotherapy or hormonal therapy in patients with recurrent LGSOC with and without a KRAS mutation. RAMP 301 will serve as the confirmatory study required by the FDA for the combination of avutometinib and defactinib for the initial indication of recurrent KRAS mt LGSOC to potentially receive full approval and has the potential to support an expanded indication regardless of KRAS mutation status. RAMP 301 is a global study with enrollment open in the United States, Australia, Canada, Europe United Kingdom, and Korea. Enrollment is on track, and we are targeting full enrollment by the end of 2025.

Current wording

In December 2023, we announced the initiation of a confirmatory Phase 3 trial to evaluate the combination of avutometinib and defactinib for the treatment of patients with recurrent LGSOC entitled RAMP 301. RAMP 301 is a randomized global confirmatory trial, which is evaluating the efficacy and safety of avutometinib and defactinib versus standard of care chemotherapy or hormonal therapy in patients with recurrent LGSOC with and without a KRAS mutation. RAMP 301 will serve as the confirmatory study required by the FDA for the combination of avutometinib and defactinib for the initial indication of recurrent KRAS mt LGSOC to potentially receive full approval and has the potential to support an expanded indication regardless of KRAS mutation status. RAMP 301 is a global study with enrollment open in the United States, Australia, Canada, Europe United Kingdom, New Zealand, Japan, and Korea. Enrollment is on track, and we are targeting full enrollment by the end of 2025.

Previous wording

On May 8, 2025, the FDA approved AVMAPKI FAKZYNJA CO-PACK in advance of its PDUFA date. We have submitted a letter to the National Comprehensive Cancer Network seeking inclusion of avutometinib plus defactinib combination therapy in its LGSOC treatment guidelines. We intend to initiate discussions with other global regulatory authorities, including those in Europe and Japan with the objective of ultimately seeking approval for the combination therapy in additional regions.

Current wording

On May 8, 2025, the FDA approved AVMAPKI FAKZYNJA CO-PACK in advance of its Prescription Drug User Fee Act or PDUFA action date. Following this approval, we submitted a letter to the National Comprehensive Cancer Network (“NCCN”) seeking inclusion of avutometinib plus defactinib combination therapy in its LGSOC treatment guidelines. NCCN included AVMAPKI FAKZYNJA CO-PACK in its LGSOC treatment guidelines consistent with the product’s approved indication. We submitted to NCCN the publications for RAMP 201 and FRAME in support of its consideration of the inclusion of the KRAS wild-type population evaluated in these trials in the NCCN Guidelines in July 2025. The NCCN Committee for Ovarian Cancer will holds its annual meeting in October 2025, and the Company has been informed that its submission will be reviewed at that time.

Previous wording

In October 2024, the Japanese Gynecologic Oncology Group dosed the first patient in a Phase 2 trial called RAMP 201J, evaluating the safety and efficacy of avutometinib in combination with defactinib in recurrent LGSOC in Japan. We expect to report initial data from RAMP 201J in the second half of 2025.

Current wording

In October 2024, the Japanese Gynecologic Oncology Group dosed the first patient in a Phase 2 trial called RAMP 201J, evaluating the safety and efficacy of avutometinib in combination with defactinib in recurrent LGSOC in Japan. We expect to report initial data from RAMP 201J in Q4 2025.

Previous wording

Based on emerging data demonstrating improved tumor regressions in KRAS G12C-mutant NSCLC preclinical models when a FAK inhibitor is combined with a G12C inhibitor and avutometinib, defactinib was added to the RAMP 203 study in new triplet cohorts in 2024. In December 2024, we announced three patients whose cancer previously progressed on a G12C inhibitor have been treated with the triplet combination of sotorasib 960 mg administered daily on a continuous schedule and avutometinib 3.2 mg twice-weekly plus defactinib 200 mg twice-daily. Avutometinib and defactinib are administered on a three out of four weeks schedule.​ There were no dose limiting toxicities observed in the first triplet combination cohort. Planned dose level evaluation cohorts for the triplet combination completed enrollment in first quarter of 2025. We expect to present an interim update of both the doublet and triplet data at a medical meeting in the second half of 2025.

Current wording

Based on emerging data demonstrating improved tumor regressions in KRAS G12C-mutant NSCLC preclinical models when a FAK inhibitor is combined with a G12C inhibitor and avutometinib, defactinib was added to the RAMP 203 study in new triplet cohorts in 2024. In December 2024, we announced three patients whose cancer previously progressed on a G12C inhibitor have been treated with the triplet combination of sotorasib 960 mg administered daily on a continuous schedule and avutometinib 3.2 mg twice-weekly plus defactinib 200 mg twice-daily. Avutometinib and defactinib are administered on a three out of four weeks schedule.​ There were no dose limiting toxicities observed in the first triplet combination cohort. Planned dose level evaluation cohorts for the triplet combination completed enrollment in first quarter of 2025. We expect to present an interim update of safety and efficacy from both the doublet and triplet data in the fourth quarter of 2025.

Previous wording

A dose level “0” has been added to the RAMP 205 study protocol that evaluates 3.2 mg of avutometinib twice a week, 200 mg of defactinib twice a day for three weeks out of every four weeks with 800 mg/m 2 of gemcitabine and 100 mg/m 2 of Nab-paclitaxel on a schedule of day 1, day 8, and day 15. All dose levels have been expanded to 12 patients each, including six additional patients recently enrolled to dose devel 1, where it was reported five out of six patients reported an objective response rate at the American Society of Clinical Oncology (“ASCO”) 2024 Annual Meeting. At dose level 1, of the six additional patients, five remain on therapy and continue to be monitored for response given the initial length of time to response. We completed enrollment in quarter 1 of 2025. Based on the initial safety and efficacy data from these cohorts, dose level 1 or 0 is anticipated to be chosen for expansion. Adverse events across all dose

Current wording

A dose level “0” has been added to the RAMP 205 study protocol that evaluates 3.2 mg of avutometinib twice a week, 200 mg of defactinib twice a day for three weeks out of every four weeks with 800 mg/m 2 of gemcitabine and 100 mg/m 2 of Nab-paclitaxel on a schedule of day 1, day 8, and day 15. All dose levels have been expanded to 12 patients each, including six additional patients recently enrolled to dose devel 1, where it was reported five out of six patients reported an objective response rate at the American Society of Clinical Oncology (“ASCO”) 2024 Annual Meeting. At

Previous wording

cohorts remained generally consistent with the previously announced safety and tolerability profile, and no new safety signals have emerged. We expect to announce data at the 2025 ASCO Annual Meeting and we expect to choose a recommended Phase 2 dose for trial expansion in first half of 2025.

Current wording

dose level 1, of the six additional patients, five remain on therapy and continue to be monitored for response given the initial length of time to response. We completed enrollment in quarter 1 of 2025. In May 2025, we announced as of April 25, 2025, 60 patients (12 per cohort) had been treated in one of five dose regimens with the combination of avutometinib and defactinib with gemcitabine and Nab-paclitaxel in frontline metastatic pancreatic ductal adenocarcinoma (“PDAC”). In the dose level 1 cohort, 12 patients received 2.4 mg of avutometinib twice a week, 200 mg of defactinib twice a day for 3 weeks out of every 4 and 800 mg/m2 of gemcitabine and 125 mg/m2 of Nab-paclitaxel on a schedule of day 1, day 8 and day 15. In dose level 1, 83% (10/12) of patients achieved partial responses (8 confirmed and 2 unconfirmed who remain on treatment). Given these results, we have selected dose level 1 as the recommended phase 2 dose, has met the pre-defined criteria to advance beyond the first stage of the expansion study, and is now enrolling up to 29 patients at this dose level. Adverse events across all dose cohorts remained generally consistent with the previously announced safety and tolerability profile, and no new safety signals have emerged.

Previous wording

In August 2023, we entered into a collaboration and option agreement (the “GenFleet Agreement”) with GenFleet Therapeutics (Shanghai), Inc. (“GenFleet”)pursuant to which GenFleet granted us options to obtain exclusive development and commercialization rights worldwide outside of mainland China, Hong Kong, Macau, and Taiwan for up to three oncology programs targeting RAS pathway driven cancers (the “GenFleet Options”). Under the GenFleet Agreement, we can exercise our GenFleet Options on a program-by-program basis. The collaboration builds on the strengths of both companies in oncology small molecule drug development, enabling us to partner our clinical development and regulatory expertise with GenFleet’s accomplished discovery capabilities. This synergistic collaboration includes our experience and established network of collaborators, including scientific and clinical experts in RAS biology and RAS pathway-driven cancers and GenFleet’s accomplishments with its KRAS G12C inhibitor program. In December 2023, we announced the selection of an oral and selective KRAS G12D (ON/OFF) inhibitor entitled VS-7375 (known as GFH375 in China) with a potential best-in-class profile as the lead program from our collaboration with GenFleet. An investigational new drug (“IND”) application by GenFleet in China for VS-7375 was cleared in June 2024. In July 2024, GenFleet began dosing several patients in a Phase 1/2 trial in China that is evaluating VS-7375 in patients with KRAS G12D-mutated advanced solid tumors. The Phase 1/2 study is being conducted in approximately 20 hospitals in China. The Phase 1 study was used to determine the recommended Phase 2 dose, and the Phase 2 study will further evaluate the efficacy and safety of VS-7375 in patients with advanced solid tumors, such as pancreatic ductal adenocarcinoma, CRC, and NSCLC.

Current wording

In August 2023, we entered into a collaboration and option agreement (the “GenFleet Agreement”) with GenFleet Therapeutics (Shanghai), Inc. (“GenFleet”)pursuant to which GenFleet granted us options to obtain exclusive development and commercialization rights worldwide outside of mainland China, Hong Kong, Macau, and Taiwan for up to three oncology programs targeting RAS pathway driven cancers (the “GenFleet Options”). Under the GenFleet Agreement, we can exercise our GenFleet Options on a program-by-program basis. The collaboration builds on the strengths of both companies in oncology small molecule drug development, enabling us to partner our clinical development and regulatory expertise with GenFleet’s accomplished discovery capabilities. This synergistic collaboration includes our experience and established network of collaborators, including scientific and clinical experts in RAS biology and RAS pathway-driven cancers and GenFleet’s accomplishments with its KRAS G12C inhibitor program. In December 2023, we announced the selection of an oral and selective KRAS G12D (ON/OFF) inhibitor entitled VS-7375 (known as GFH375 in China) with a potential best-in-class profile as the lead program from our collaboration with GenFleet. An investigational new drug (“IND”) application by GenFleet in China for VS-7375 was cleared in June 2024. In July 2024, GenFleet began dosing several patients in a Phase 1/2 trial in China that is evaluating VS-7375 in patients with KRAS G12D-mutated advanced solid tumors. The Phase 1/2 study is being conducted in approximately 20 hospitals in China. The Phase 1 study was used to determine the recommended Phase 2 dose, and the Phase 2 study will further evaluate the efficacy and safety of VS-7375 in patients with advanced solid tumors, such as PDAC, CRC, and NSCLC.

Previous wording

On January 14, 2025, we announced the early exercise of the GenFleet Option with respect to VS-7375. We filed an IND application in the United States for VS-7375 during the first quarter of 2025 and in April 2025 we announced the FDA clearance of the IND. We expect to initiate a Phase 1/2a trial in mid-2025 with plans for multiple expansion cohorts, including combinations, in advanced solid tumors, such as pancreatic cancer, CRC, and NSCLC. Verastem and GenFleet shared preclinical data at American Association for Cancer Research meeting in April 2025. GenFleet expects to share the initial clinical data from the Phase 1 study of VS-7375 in China at the 2025 ASCO Annual Meeting.

Current wording

On January 14, 2025, we announced the early exercise of the GenFleet Option with respect to VS-7375. We filed an IND application in the United States for VS-7375 during the first quarter of 2025. In April 2025 we announced the FDA clearance of the IND, and in June 2025 we announced we dosed our first patient with VS-7375 in our U.S. Phase 1/2a clinical trial evaluating VS-7375 in patients with advanced KRAS G12D solid tumors. We expect to report a preliminary update on the Phase 1 monotherapy dose escalation in Q4 2025. Subject to the results of the Phase 1 monotherapy dose escalation, the Company plans to initiate monotherapy expansion cohorts in both advanced PDAC and NSCLC. Additionally, we expect to initiate the dose escalation cohorts in combination with cetuximab, chemotherapy, and chemotherapy with checkpoint-inhibitor for CRC, PDAC, and NSCLC, respectively, in Q4 2025. Subject to the results of the Phase 1 dose escalation combinations cohorts with VS-7375, the Company plans to initiate a combination expansion cohort in CRC, PDAC, and NSCLC. Verastem and GenFleet shared preclinical data at American Association for Cancer Research meeting in April 2025.

Previous wording

Our operations to date have been focused on organizing and staffing our company, business planning, raising capital, identifying and acquiring potential product candidates, undertaking preclinical studies and clinical trials for our product candidates and initiating U.S. commercial operations following the approval of COPIKTRA through our ownership period ending in September 2020 and in anticipation of the approval of AVMAPKI FAKZYNJA CO-PACK. We have financed our operations to date primarily through public and private offerings of our common stock, pre-funded warrants, and warrants, offerings of convertible notes, sales of common stock under our at-the-market equity offering program, our loan and security agreement executed with Hercules in March 2017, as amended, the Loan Agreement with Oxford, our Note Purchase Agreement, the upfront payments and milestone payments under our license and collaboration agreements with Sanofi, CSPC, and Yakult, the upfront payment and milestone payments received under the Secura APA, and sales of Series B Convertible Preferred Stock. Additionally, from our U.S. commercial launch of COPIKTRA on September 24, 2018, through our ownership period ending in September 2020, we financed a portion of our operations through product revenue.

Current wording

Our operations to date have been focused on organizing and staffing our company, business planning, raising capital, identifying and acquiring potential product candidates, undertaking preclinical studies and clinical trials for our product candidates and initiating U.S. commercial operations following the approval of COPIKTRA through our ownership period ending in September 2020 and in anticipation of the approval of AVMAPKI FAKZYNJA CO-PACK. We have financed our operations to date primarily through public and private offerings of our common stock, pre-funded warrants, and warrants, offerings of convertible notes, sales of common stock under our at-the-market equity offering program, our loan and security agreement executed with Hercules in March 2017, as amended, the Loan Agreement with Oxford, our Note Purchase Agreement, the upfront payments and milestone payments under our license and collaboration agreements with Sanofi, CSPC, and Yakult, the upfront payment and milestone payments received under the Secura APA, and sales of Series B Convertible Preferred Stock. Additionally, from our U.S. commercial launch of COPIKTRA on September 24, 2018, through our ownership period ending in September 2020, we financed a portion of our operations through product revenue. From our U.S. commercial launch of AVMAPKI FAKZYNJA CO-PACK on May 8 th, 2025, we are financing a portion of our operations through product revenue.

Previous wording

As of March 31, 2025, we had an accumulated deficit of $1,007.6 million. Our net loss was $52.1 million, $33.9 million for the three months ended March 31, 2025 and 2024, respectively. As of March 31, 2025, we had cash, cash equivalents, and investments of $117.6 million. In accordance with applicable accounting standards, we evaluated whether there are conditions and events, considered in the aggregate, that raise substantial doubt about our ability to continue as a going concern within 12 months after the date of the issuance of the consolidated financial statements. We anticipate operating losses may continue for the foreseeable future and we continue to incur operating costs to execute

Current wording

As of June 30, 2025, we had an accumulated deficit of $1,033.6 million. Our net loss was $25.9 million, $78.0 million, $8.3 million and $42.1 million for the three and six months ended June 30, 2025 and 2024, respectively. As of June 30, 2025, we had cash, cash equivalents, and investments of $164.3 million. In accordance with applicable accounting standards, we evaluated whether there are conditions and events, considered in the aggregate, that raise substantial doubt about our ability to continue as a going concern within 12 months after the date of the issuance of the consolidated financial statements. We anticipate operating losses may continue for the foreseeable future and we continue to incur operating costs to execute our strategic plan, including costs related to research and development of our product candidates and commercial activities. As a result of the assessment in accordance with the applicable accounting standards, these conditions raise substantial doubt about our ability to continue as a going concern for 12 months after the date the condensed consolidated financial statements are issued.

Previous wording

We believe that several accounting policies are important to understanding our historical and future performance. We refer to these policies as “critical” because these specific areas generally require us to make judgments and estimates about matters that are uncertain at the time we make the estimate, and different estimates—which also would have been reasonable—could have been used, which would have resulted in different financial results.

Current wording

and estimates about matters that are uncertain at the time we make the estimate, and different estimates—which also would have been reasonable—could have been used, which would have resulted in different financial results.

Previous wording

The critical accounting policies we identified in our most recent Annual Report on Form 10-K for the fiscal year ended December 31, 2024, related to revenue recognition, collaborative agreements, accrued and prepaid research and development expenses, and stock-based compensation. During the three months ended March 31, 2025, there were no material changes to our critical accounting policies.

Current wording

The critical accounting policies we identified in our most recent Annual Report on Form 10-K for the fiscal year ended December 31, 2024, related to revenue recognition, collaborative agreements, accrued and prepaid research and development expenses, and stock-based compensation. During the six months ended June 30, 2025, there were no material changes to our critical accounting policies.

Previous wording

*Research and development expense.*Research and development expense for the three months ended March 31, 2025 (the “2025 Quarter”) was $29.1 million compared to $17.7 million for the three months ended March 31, 2024 (the “2024 Quarter”). The $11.4 million increase from the 2024 Quarter to the 2025 Quarter was primarily driven by the $6.0 million GenFleet Option payment with respect to VS-7375, an increase of $2.4 million in contract research organization (“CRO”) costs, an increase of $1.6 million in drug substance and drug product manufacturing costs, an increase of $0.8 million in personnel costs, including non-cash stock compensation, and an increase of $0.6 million in investigator fees.

Current wording

*Research and development expense.*Research and development expense for the 2025 Quarter was $24.8 million compared to $18.1 million for the 2024 Quarter. The $6.7 million increase from the 2024 Quarter to the 2025 Quarter was primarily driven by an increase of $2.4 million in contract research organization (“CRO”) costs, an increase of $2.0 million in investigator fees, an increase of $1.5 million in personnel costs, including non-cash stock compensation, and an increase of $0.8 million in clinical supply costs.

Previous wording

Selling, general and administrative expense. Selling, general and administrative expense for the 2025 Quarter was $15.0 million compared to $10.4 million for the 2024 Quarter. The increase of $4.6 million from the 2024 Quarter to the 2025 Quarter primarily resulted from the increase of $2.1 million of commercialization costs in anticipation of a launch of avutometinib and defactinib in KRAS mt LGSOC, an increase of $1.0 million in personnel costs including non-cash based stock compensation, $0.8 million in financing fees associated with the Note Purchase Agreement, an increase of $0.4 million consulting and professional fees, and an increase of $0.3 million in travel and other costs.

Current wording

Selling, general and administrative expense. Selling, general and administrative expense for the 2025 Quarter was $20.7 million compared to $10.2 million for the 2024 Quarter. The increase of $10.5 million from the 2024 Quarter to the 2025 Quarter primarily resulted from the increase of $8.1 million of consulting and professional fees related to the launch of avutometinib and defactinib in KRAS mt LGSOC, an increase of $2.7 million in personnel costs including non-cash based stock compensation, an increase of $1.0 million in travel and other costs, partially offset by a decrease of $1.3 million in commercial operations expenses.

Previous wording

*Interest income.*Interest income for the 2025 Quarter was $1.0 million compared to $1.4 million for the 2024 Quarter. The decrease of $0.4 million from the 2024 Quarter to the 2025 Quarter in interest income was primarily due to the decrease in investment balances on short term investments and cash equivalents and a decrease in interest rates on securities during each respective quarter.

Current wording

*Interest income.*Interest income for the 2025 Quarter was $0.8 million compared to $1.0 million for the 2024 Quarter. The decrease of $0.2 million from the 2024 Quarter to the 2025 Quarter in interest income was primarily due to the decrease in investment balances on short term investments and cash equivalents and a decrease in interest rates on securities during each respective quarter.

Previous wording

*Interest expense.*Interest expense for the 2025 Quarter was $0.2 million compare to $1.1 million for the 2024 Quarter. The decrease of $0.9 million from the 2024 Quarter was primarily driven by termination of the Loan Agreement with Oxford on January 13, 2025 and therefore there was less interest expense recorded in the 2025 Quarter. The Company has elected to record interest expense associated with the Notes within change in fair value of Notes on the condensed statements of operations and comprehensive loss.

Current wording

*Interest expense.*Interest expense for the 2025 Quarter was $0.2 million compared to $1.1 million for the 2024 Quarter. The $0.9 million decrease from the 2024 Quarter was primarily driven by termination of the Loan Agreement with Oxford on January 13, 2025 resulting in a reduction of interest expense recorded in the 2025 Quarter. The Company has elected to record interest expense associated with the Notes within change in fair value of Notes on the condensed statements of operations and comprehensive loss.

Previous wording

*Change in fair value of warrant liability.*The change in fair value of the warrant liability of $2.4 million expense for the 2025 Quarter was comprised of the mark-to-market adjustment for the liability classified warrants issued as part of the July 2024 Offering. The liability classified warrants increased in value from December 31, 2024 to when 1,166,666 Warrants were exercised in March 2025 and March 31, 2025 primarily driven by an increase in our stock price. There was no warrant liability outstanding during the 2024 Quarter.

Current wording

*Change in fair value of warrant liability.*The change in fair value of the warrant liability of $20.3 million for the 2025 Quarter was comprised of the mark-to-market adjustment for the liability classified warrants issued as part of the July 2024 Offering. The liability classified warrants decreased in value from December 31, 2024 as a result of the exercise of 2,787,499 Warrants during the quarter and a decrease in warrant valuation due to a lower stock price. There was no warrant liability outstanding during the 2024 Quarter.

Previous wording

*Change in fair value of Notes.*We elected the fair value option for the Notes and therefore the changes in fair value, including interest, other than changes that are directly attributable to instrument specific credit risk are recorded as change in fair of Notes in the condensed statements of operations and comprehensive loss. The change in fair value for the 2025 Quarter of $4.4 million was primarily driven by interest on the Notes and a reduction in the risk-free rate during the 2025 Quarter. There were no Notes outstanding in the 2024 Quarter.

Current wording

*Change in fair value of Notes.*We elected the fair value option for the Notes and therefore the changes in fair value, including interest, other than changes that are directly attributable to instrument specific credit risk are recorded as change in fair of Notes in the condensed statements of operations and comprehensive loss. The change in fair value for the 2025 Quarter of $3.0 million was primarily driven by interest on the Notes and a reduction in the risk-free rate during the 2025 Quarter. There were no Notes outstanding in the 2024 Quarter.

Previous wording

The $4.0 million increase in avutometinib+ defactinib – LGSOC is primarily driven by an increase in RAMP 301 trial costs as the trial continues to advance and enroll more patients. The $1.4 million decrease in avutometinib + defactinib – NSCLC is primarily driven by a decrease in RAMP 202 trial costs. The $1.0 million increase in avutometinib + defactinib – pancreatic cancer is primarily driven by fully utilizing the PanCAN Grant in the second quarter of 2024 and therefore there was no offsetting reduction in expense in the 2025 Quarter. The $7.4 million increase in GenFleet related expenses was primarily driven by the $6.0 million GenFleet Option payment with respect to VS-7375.

Current wording

The $8.2 million increase in avutometinib+ defactinib – LGSOC is primarily driven by an increase in RAMP 301 trial costs as the trial continues to advance and enroll more patients. The $1.9 million decrease in avutometinib + defactinib – NSCLC is primarily driven by a decrease in RAMP 202 trial costs. The $1.3 million increase in avutometinib + defactinib – pancreatic cancer is primarily driven by fully utilizing the PanCAN Grant in the second quarter of 2024 and therefore there was no offsetting reduction in expense in the 2025 Period. The $2.7 million decrease in avutometinib and defactinib manufacturing and non-clinical trial specific expenses was driven by a reduction in study activities and safety consulting services. The $9.8 million increase in GenFleet related expenses was primarily driven by the $6.0 million GenFleet Option payment with respect to VS-7375.

Previous wording

*Loss on debt extinguishment.*The loss on debt extinguishment for the 2025 Quarter of $1.8 million represents the loss recognized on early extinguishment of our Loan Agreement. On January 13, 2025, we repaid in full all principal, accrued and unpaid interest, fees, and expenses under the Loan Agreement in an aggregate amount of $42.7 million (the Payoff Amount). The Payoff Amount, excluding accrued interest, exceeded the carrying amount of the Term Loans on January 13, 2025 by $1.8 million which was recorded as a loss on debt extinguishment. There was no loss on debt extinguishment in the 2024 Quarter.

Current wording

*Loss on debt extinguishment.*The loss on debt extinguishment for the 2025 Period of $1.8 million represents the loss recognized on early extinguishment of our Loan Agreement. On January 13, 2025, we repaid in full all principal, accrued and unpaid interest, fees, and expenses under the Loan Agreement in an aggregate amount of $42.7 million (the Payoff Amount). The Payoff Amount, excluding accrued interest, exceeded the carrying amount of the Term Loans on January 13, 2025 by $1.8 million which was recorded as a loss on debt extinguishment. There was no loss on debt extinguishment in the 2024 Period.

Previous wording

*Change in fair value of preferred stock tranche liability.*The change in fair value of the preferred stock tranche liability was $0.0 million for the 2025 Quarter compared to $6.0 million expense for the 2024 Quarter. The change in fair value of preferred stock tranche liability was comprised of the mark-to-market adjustment related to the second tranche right issued as part of the Securities Purchase Agreement. The preferred stock tranche liability expired in July 2024 and therefore was not outstanding during the 2025 Quarter. The fair value of the preferred stock tranche liability increased from $4.2 million at the beginning of the 2024 Quarter to $10.2 million at the end of the 2024 Quarter resulting in $6.0 million expense in the 2024 Quarter.

Current wording

*Change in fair value of preferred stock tranche liability.*The change in fair value of the preferred stock tranche liability was $4.2 million income for the 2024 Period. The change in fair value of preferred stock tranche liability was comprised of the mark-to-market adjustment related to the second tranche right issued as part of the Securities Purchase Agreement. The preferred stock tranche liability expired in July 2024 and therefore was not outstanding during the 2025 Period. The fair value of the preferred stock tranche liability decreased from $4.2 million at the beginning of the 2024 Period to $0.0 million at the end of the 2024 Period resulting in $4.2 million income in the 2024 Period.

Previous wording

As of March 31, 2025, we had $117.6 million of cash, cash equivalents, and investments. We primarily invest our cash, cash equivalents and investments in U.S. Government money market funds, U.S. government agency bonds, corporate bonds and commercial paper of publicly traded companies.

Current wording

As of June 30, 2025, we had $164.3 million of cash, cash equivalents, and investments. We primarily invest our cash, cash equivalents and investments in U.S. Government money market funds, U.S. government agency bonds, corporate bonds and commercial paper of publicly traded companies.

Previous wording

The following table sets forth the primary sources and uses of cash for the 2025 Quarter and the 2024 Quarter (in thousands):

Current wording

The following table sets forth the primary sources and uses of cash for the 2025 Period and the 2024 Period (in thousands):

Previous wording

Operating activities. The use of cash in both periods resulted primarily from our net losses adjusted for non-cash charges and changes in the components of working capital. Our cash outflow from net losses adjusted for non-cash charges and adjustments was $42.9 million and $26.8 million for the 2025 Quarter and the 2024 Quarter, respectively. Non-cash charges and adjustments for the 2025 Quarter were primarily related to non-cash changes in fair value of the Notes, the change in fair value of warrant liability, loss on debt extinguishment and stock-based compensation expense. Non-cash charges and adjustments for the 2024 Quarter were primarily related to change in fair value of preferred stock tranche liability and stock-based compensation expense. Our cash inflow from operating activities due to changes in operating assets and liabilities was $4.3 million for the 2025 Quarter. Our cash outflow from operating activities due to changes in operating assets and liabilities was $1.5 million for the 2024 Quarter. Cash inflow due to changes in operating assets and liabilities for the 2025 Quarter was primarily driven by an increase of $3.2 million in accounts payables and an increase of $2.7 million in accrued expense and other liabilities, partially offset by an increase of $1.6 million in prepaid expenses, other current assets and other assets. Cash outflow due to changes in operating assets and liabilities for the 2024 Quarter was primarily driven by an increase of $0.6 million of prepaid expenses, other current assets and other assets, a decrease of $0.5 million of accrued expenses and other liabilities, a decrease of $0.3 million of deferred liabilities, and an increase of $0.2 million of grant receivable, partially offset by an increase of $0.2 million in accounts payable The increases in both periods in prepaid expenses, other current assets, and other asserts is exclusive of cash

Current wording

Operating activities. The use of cash in both periods resulted primarily from our net losses adjusted for non-cash charges and changes in the components of working capital. Our cash outflow from net losses adjusted for non-cash charges and adjustments was $84.2 million and $43.4 million for the 2025 Period and the 2024 Period, respectively. Non-cash charges and adjustments for the 2025 Period were primarily related to the change in fair value of warrant liability, non-cash changes in fair value of the Notes, loss on debt extinguishment and stock-based compensation expense. Non-cash charges and adjustments for the 2024 Period were primarily related to a change in fair value of preferred stock tranche liability and stock-based compensation expense. Our cash inflow from operating activities due to changes in operating assets and liabilities was $12.9 million for the 2025 Period. Our cash outflow from operating activities due to changes in operating assets and liabilities was $12.5 million for the 2024 Period. Cash inflow due to changes in operating assets and liabilities for the 2025 Period was primarily driven by an increase of $6.6 million in accrued expenses and other liabilities, an increase of $6.3 million in accrued expenses, long term, and an increase of $4.0 million in accounts payable, offset by an increase of $2.1 million in accounts receivable, an increase of $1.2 million in inventory, and an increase of $0.8 million in prepaid expenses, other current assets and other assets. Cash outflow due to changes in operating assets and liabilities for the 2024 Period was primarily driven by an increase of $10.0 million of accounts receivable, $0.8 million of grant receivable, a decrease of $0.7 million of accrued expenses and other liabilities, a decrease of $0.5 million of accounts payable, and a decrease of $0.3 million of deferred liabilities. The increases in both periods in prepaid expenses, other current assets, and other asserts is exclusive of cash received from PanCAN and

Previous wording

received from PanCAN and used on the RAMP 205 study. Cash used in operating activities was $38.7 million and $28.3 million for the 2025 Quarter and the 2024 Quarter, respectively.

Current wording

used on the RAMP 205 study. Cash used in operating activities was $71.3 million and $55.9 million for the 2025 Period and the 2024 Period, respectively.

Previous wording

*Investing activities.*There were no cash inflows or outflows related to investing activities during the 2025 Quarter. The cash provided by investing activities for the 2024 Quarter relates to maturities of investments of $31.0 million, partially offset by a purchase of property and equipment of less than $0.1 million.

Current wording

*Investing activities.*There was no cash used in investing activities for the 2025 Period. The cash provided by investing activities for the 2024 Period relates to maturities of investments of $60.0 million, partially offset by a purchase of property and equipment of less than $0.1 million.

Previous wording

Financing activities. The cash provided by financing activities for the 2025 Quarter represents 75.0 million of proceeds received pursuant to the Note Purchase Agreement, $22.7 million of proceeds received under the August 2021 ATM, $7.4 million of proceeds received pursuant to the Stock Purchase Agreement, $4.1 million of proceeds from the exercise of Warrants, $1.2 million of proceeds received from insurance premium financing, and less than $0.1 million of proceeds received related to our employee stock purchase plan, partially offset by the $42.6 million repayment of our Loan Agreement, and $0.4 million of payments for insurance premium financing. The cash provided by financing activities for the 2024 Quarter represents $1.3 million of proceeds received from insurance premium financing and $0.1 million of proceeds received related to exercise of stock options and our employee stock purchase plan, partially offset by $0.4 million of payments on insurance premium financing and $0.2 million of fees paid to the Lenders to amend our Loan Agreement. Refer toNote 7. Debt to our unaudited condensed consolidated financial statements included in this quarterly report for additional details on the Note Purchase Agreement and Loan Agreement;Note 12. Capital Stock to our unaudited condensed consolidated financial statements included in this quarterly report for additional details on the August 2021 ATM, the Stock Purchase Agreement, and the Warrants;Note 9. Notes Payableto our unaudited condensed consolidated financial statements included in this quarterly report for additional details on the finance agreement with First Insurance related to insurance premium financing and the monthly payments of principal and interest related thereto.

Current wording

Financing activities. The cash provided by financing activities for the 2025 Period represents $75.0 million of proceeds received pursuant to the Note Purchase Agreement, approximately $69.9 million of proceeds received from our 2025 Private Placement, $22.7 million of proceeds received pursuant to the Stock Purchase Agreement, $13.8 million of proceeds from the exercise of Warrants, $1.2 million of proceeds received from insurance premium financing, and less than $0.1 million of proceeds received related to our employee stock purchase plan, partially offset by the $42.6 million repayment of our Loan Agreement, and $0.7 million of payments for insurance premium financing. The cash provided by financing activities for the 2024 Period represents $1.3 million of proceeds received from insurance premium financing and $0.2 million of proceeds received from exercise of stock options and our employee stock purchase plan, partially offset by $0.8 million of payments on insurance premium financing, $0.2 million of payments of deferred issuance costs, and $0.2 million of fees paid to the Lenders to amend our Loan Agreement with Oxford. Refer toNote 10. Debt to our unaudited condensed consolidated financial statements included in this quarterly report for additional details on the Note Purchase Agreement and Loan Agreement;Note 15. Capital Stock to our unaudited condensed consolidated financial statements included in this quarterly report for additional details on the 2025 Private Placement, the Note Purchase Agreement, and the Warrants;Note 12. Notes Payableto our unaudited condensed consolidated financial statements included in this quarterly report for additional details on the finance agreement with First Insurance related to insurance premium financing and the monthly payments of principal and interest related thereto.
Rearranged current paragraphs (10)

Current report — wording carried from elsewhere

We believe that several accounting policies are important to understanding our historical and future performance. We refer to these policies as “critical” because these specific areas generally require us to make judgments

Current report — wording carried from elsewhere

The $4.1 million increase in avutometinib+ defactinib – LGSOC was primarily driven by an increase in RAMP 301 trial costs as the trial continues to advance and enroll more patients. The $0.6 million decrease in avutometinib + defactinib – NSCLC was primarily driven by a decrease in RAMP 202 trial costs. The $1.7 million decrease in avutometinib and defactinib manufacturing and non-clinical trial specific expenses was driven by a reduction in manufacturing and study activities, as well as safety consulting services. The $2.4 million increase in GenFleet related expenses was primarily driven by drug product costs for the 7375-101 study.

Current report — wording carried from elsewhere

*Change in fair value of preferred stock tranche liability.*The change in fair value of the preferred stock tranche liability was $0.0 million for the 2025 Quarter compared to $10.2 million income for the 2024 Quarter. The change in fair value of preferred stock tranche liability was comprised of the mark-to-market adjustment related to the second tranche right issued as part of the Securities Purchase Agreement. The preferred stock tranche liability expired in July 2024 and therefore was not outstanding during the 2025 Quarter. The fair value of the preferred stock tranche liability

Current report — wording carried from elsewhere

decreased from $10.2 million at the beginning of the 2024 Quarter to $0.0 million at the end of the 2024 Quarter resulting in $10.2 million income in the 2024 Quarter.

Current report — wording carried from elsewhere

The table below summarizes our direct research and development expenses for our product/ product candidates/ projects and our unallocated research and development costs for the 2025 Period and the 2024 Period.

Current report — wording carried from elsewhere

Other expense. Other expense for the 2025 Period and the 2024 Period was less than $0.1 million. Other expense for the 2025 Period and 2024 Period was comprised of transaction losses due to changes in foreign currency exchange rates.

Current report — wording carried from elsewhere

*Interest income.*Interest income for the 2025 Period was $1.8 million compared to $2.4 million for the 2024 Period. The decrease of $0.6 million from the 2024 Period to the 2025 Period in interest income was primarily due to the decrease in investment balances on short term investments and cash equivalents and a decrease in interest rates on securities during each respective quarter.

Current report — wording carried from elsewhere

has elected to record interest expense associated with the Notes within change in fair value of Notes on the condensed statements of operations and comprehensive loss.

Current report — wording carried from elsewhere

*Change in fair value of warrant liability.*The change in fair value of the warrant liability of $17.9 million for the 2025 Period was comprised of the mark-to-market adjustment for the liability classified warrants issued as part of the July 2024 Offering. The liability classified warrants decreased in value from December 31, 2024 as a result of the exercise of 2,787,499 Warrants during the period and a decrease in warrant valuation due to a lower stock price. There was no warrant liability outstanding during the 2024 Period.

Current report — wording carried from elsewhere

*Change in fair value of Notes.*We elected the fair value option for the Notes and therefore the changes in fair value, including interest, other than changes that are directly attributable to instrument specific credit risk are recorded as change in fair of Notes in the condensed statements of operations and comprehensive loss. The change in fair value for the 2025 Period of $7.4 million was primarily driven by interest on the Notes and a reduction in the risk-free rate during the 2025 Period. There were no Notes outstanding in the 2024 Period.
Rearranged previous paragraphs (1)

Previous report — wording carried elsewhere

our strategic plan, including costs related to research and development of our product candidates and commercial activities. As a result of the assessment in accordance with the applicable accounting standards, these conditions raise substantial doubt about our ability to continue as a going concern for 12 months after the date the condensed consolidated financial statements are issued.