On March 4, 2026 Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with RAS/MAPK pathway-driven cancers, reported financial results for the three months and full year ended December 31, 2025, and highlighted recent progress.
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"2025 was a transformative year for us, highlighted by the landmark FDA approval of AVMAPKI FAKZYNJA CO-PACK, the only medicine approved to specifically treat KRAS-mutated recurrent LGSOC. The launch is off to a strong start, and this novel-novel combination therapy is gaining positive response across the LGSOC community with gynecologic and medical oncologists in both academic and community settings increasingly turning to it when patients experience a first or subsequent recurrence. We also made considerable progress with VS-7375, our oral, KRAS G12D (ON/OFF) inhibitor with best-in-class potential for solid tumor cancers, clearing multiple dose levels across both monotherapy and cetuximab combination cohorts with no DLTs or major toxicities. Building on the insights from the China data, the tolerability profile that is emerging with VS-7375 in the U.S. has shown meaningful improvement and supports continued dose escalation," said Dan Paterson, president and chief executive officer at Verastem Oncology. "In 2026, our priorities are to continue driving a strong launch of AVMAPKI FAKZYNJA CO-PACK to fuel sustainable growth, while we continue to accelerate the clinical development of VS-7375 and breakout Phase 2 registration-directed clinical trials in pancreatic, lung, and colorectal cancers."
Fourth Quarter 2025 and Recent Highlights
AVMAPKI FAKZYNJA CO-PACK (avutometinib capsules; defactinib tablets) U.S. Launch
AVMAPKI FAKZYNJA CO-PACK generated net product revenues of $17.5 million for the fourth quarter of 2025 and $30.9 million for the full year 2025, following accelerated U.S. Food and Drug Administration (FDA) approval in May 2025, approximately two months ahead of its Prescription Drug User Fee Act (PDUFA) action date of June 30, 2025.
On February 4, 2026, the Company announced updated data for RAMP 201J in Japan with a data cutoff of January 30, 2026. Of the 16 patients enrolled with a median follow-up of 10 months, a confirmed overall response rate (ORR) of 38% (6/16) was achieved by investigator assessment. Among patients with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC), the confirmed ORR was 57% (4/7) and the disease control rate (DCR) was 100% (7/7). Among patients with KRAS wild-type recurrent LGSOC, the confirmed ORR was 22% (2/9) and the DCR was 89% (8/9). Of the 16 patients enrolled, 11 patients remain on treatment. No patients discontinued due to an adverse event. The safety profile was similar to previously reported data outside of Japan. Steady-state exposures of avutometinib and defactinib in the RAMP 201J study were comparable to those seen in RAMP 201.
On February 25, 2026, the annual update of the National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology (NCCN Guidelines) for Ovarian Cancer was released. The Guidelines did not expand the recommendation for avutometinib plus defactinib to include patients with recurrent LGSOC without a KRAS mutation. The Guidelines retained the category 2A recommendation for avutometinib plus defactinib for patients with KRAS-mutated recurrent LGSOC.
"We are disappointed for the patients with KRAS wild-type recurrent LGSOC, who currently have no targeted, FDA-approved treatment options specifically for their disease and face a particularly poor prognosis. Across three separate clinical trials (the FRAME study, RAMP 201, and RAMP 201J) we have observed what we believe are robust objective responses rates for patients with recurrent LGSOC with and without KRAS mutations. We remain committed to advancing the clinical evidence through longer term follow-up analyses from the RAMP 201 study planned for the SGO annual meeting, and completing our ongoing confirmatory RAMP 301 Phase 3 clinical trial, which includes patients with and without KRAS mutations, and look forward to sharing these data with the NCCN and the medical community to support future guideline consideration," said John Hayslip, chief medical officer at Verastem Oncology.
Expected Key Milestones:
Maximize adoption of AVMAPKI FAKZYNJA CO-PACK in the U.S. as the treatment of choice at the earliest recurrence, leveraging its robust clinical data.
Report a topline readout of the primary endpoint in the RAMP 301 trial in mid-2027.
Continue to pursue regulatory paths for potential expansion into Europe and Japan.
VS-7375, an Oral KRAS G12D (ON/OFF) Inhibitor in Advanced Solid Tumors
The Company reported an update on its progress with the VS-7375-101 Phase 1/2 study:
After clearing the 900 mg daily (QD) dose level with no dose-limiting toxicities (DLTs), the dose escalation phase will continue to 1200 mg QD to further interrogate the dose range and characterize the safety, tolerability, and efficacy profile of VS-7375.
The 600 mg QD dose level of VS-7375 in combination with cetuximab was cleared with no DLTs and higher doses are now being evaluated.
In a pharmacokinetics (PK) analysis, doses of VS-7375 at 600 mg QD and above, with feeding and anti-emetic prophylaxis, yielded similar exposures to fasted patients in China. The exposures achieved cover the exposures in preclinical models necessary for maximal anti-tumor efficacy.
As of the January 30, 2026 data cutoff, VS-7375 demonstrated an encouraging safety profile and was generally well-tolerated across all monotherapy dose levels evaluated to date. Patients (n=23) receiving VS-7375 at either 400 mg QD, 600 mg QD or 900 mg QD with a mean duration of therapy of 1.6 months (0.7-5.6), reported no drug related liver function test abnormalities. There was no drug-related neutropenia greater than Grade 2 and rates of nausea, vomiting and diarrhea remained lower than those reported by the Company’s partner in China. No DLTs have been reported to date, and the maximum tolerated dose has not been reached.
Following recent feedback from the FDA, the Company is amending the VS-7375-101 Phase 1/2 protocol to separate out disease-specific Phase 2 registration-directed trials for KRAS G12D mutated 2L pancreatic ductal adenocarcinoma (PDAC) and 2L/3L non-small cell lung cancer (NSCLC) (monotherapy) and 2L+ colorectal cancer (CRC) in combination with cetuximab.
In January 2026, the Company reported updates on its VS-7375-101 trial including that it had cleared the 400 mg QD, 600 mg QD and 900 mg QD dose levels with no DLTs and no major toxicities. The VS-7375 monotherapy expansion cohorts were initiated, and the cohort sizes were expanded in 2L PDAC, 2L/3L NSCLC, and 2L+ other KRAS G12D-mutated solid tumors. In the VS-7375 dose-escalation combination cohort, the 400 mg QD dose was cleared in combination with cetuximab with no DLTs. The combination dose escalation cohorts were initiated in 1L NSCLC and 2L PDAC at the end of 2025.
In October 2025, the Company announced a preliminary update on the Phase 1/2 monotherapy dose escalation trial of VS-7375 in patients with previously treated advanced KRAS G12D mutant solid tumors. In the study, VS-7375 cleared both the 400 mg QD and the 600 mg QD monotherapy doses with no DLTs observed. At the two dose levels evaluated in the U.S. cohort, no nausea, vomiting, or diarrhea greater than Grade 1 were reported. In addition, no new safety signals have been observed relative to earlier data presentations in both PDAC and NSCLC by GenFleet Therapeutics, the Company’s partner in China. Of the five efficacy evaluable patients in the VS-7375-101 study with at least one scan, four out of five patients have had a tumor reduction and were still on treatment.
The Company shared multiple updates from GenFleet and its ongoing evaluation of VS-7375, known as GFH375, in China:
On March 2, 2026, GenFleet announced that GFH375 was granted its first Breakthrough Therapy Designation in China for patients with KRAS G12D-mutated NSCLC who have received prior systemic therapy.
In December 2025, GenFleet announced the initiation of a registrational Phase 3 study for GFH375 in patients with pretreated KRAS G12D-mutated metastatic pancreatic cancer in China.
In October 2025, the Company announced updated data for GFH375 in PDAC featured in a late-breaking oral presentation at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress. GenFleet shared additional analyses of this data set on October 27, 2025:
In a subgroup analysis, 12 patients with 2L PDAC at 600 mg QD achieved an ORR of 58.3% and a DCR of 100%. In the 3L+ setting, 47 PDAC patients receiving 600 mg QD achieved an ORR of 36.2% and a DCR of 95.7%. In the 2L subgroup, the median progression free survival (mPFS) and median overall survival (mOS) have not been reached. An additional analysis of gastrointestinal disorders, hematological toxicities, and liver enzyme abnormalities in 2L+ patients with PDAC (n=66) at 600 mg QD showed no adverse events Grade ≥3 occurred at rates above 8.0%.
In an analysis of pre-treated patients with NSCLC at 600 mg QD, the four-month PFS rate was greater than 75% and the mPFS has not been reached. The median follow-up time was 4.2 months.
In October 2025, GenFleet announced that the first patient has been dosed in a Phase 1b/2 study of GFH375 combined with cetuximab or chemotherapy for advanced solid tumors, including 1L PDAC, in China.
In August 2025, the Company announced data of GFH375 would be featured in a mini oral presentation at the IASLC 2025 World Conference on Lung Cancer (WCLC) on September 8, 2025. At the recommended Phase 2 dose (RP2D) of 600 mg QD, the ORR was 68.8% (11/16) (both confirmed and unconfirmed) and the DCR was 93.8% (15/16). Among the 26 evaluable patients with NSCLC treated across all dose levels, the ORR was 57.7% (15/26) (both confirmed and unconfirmed) and the DCR was 88.5% (23/26).
Expected Key Milestones:
Report early data from the VS-7375-101 trial in 1H 2026.
Select the RP2D with cetuximab and initiate the CRC combination expansion cohort in 1H 2026.
Complete enrollment in combination dose-escalation cohorts in mid-2026.
Complete enrollment in monotherapy expansion cohorts in 2H 2026.
Select the RP2D and plan to initiate the PDAC and NSCLC combination expansion cohorts in 2H 2026.
RAMP 205: Avutometinib Plus Defactinib in Combination with Chemotherapy in 1L Metastatic Pancreatic Cancer
In November 2025, the Company announced that enrollment was completed in the expansion cohort in Q3 2025.
Expected Key Milestone:
Report an update on the safety and efficacy of the RAMP 205 expansion cohort with at least six months of follow-up on all patients in Q2 2026.
Upcoming Presentations
Multiple abstracts were selected for oral and poster presentations at the Society of Gynecologic Oncology (SGO) 2026 Annual Meeting on Women’s Cancer on April 10-13 in Puerto Rico. These presentations will include a late-breaking oral presentation on the long-term analysis of the Phase 2 RAMP 201 trial of avutometinib and defactinib combination in recurrent LGSOC.
Corporate Updates
In December 2025, the Company announced John Johnson, current board member, was appointed to chairman of Verastem’s Board of Directors, and Michael Kauffman, M.D., Ph.D., lead director since 2016, was appointed to president of development of Verastem.
In November 2025, the Company announced it had completed a public offering of over $96.9 million of common stock and pre-funded warrants.
Fourth Quarter 2025 Financial Results
Verastem Oncology ended the fourth quarter of 2025 with cash, cash equivalents, and investments of $205 million. On a pro forma basis, taking into account the net proceeds from the exercise of warrants in January 2026 of $29.4 million, cash, cash equivalents, and investments were $234.4 million as of December 31, 2025. These additional sources of capital along with the existing cash, cash equivalents, and investments and ongoing product revenue provide an expected cash runway into first half of 2027.
Net product revenue for the three months ended December 31, 2025 (the "2025 Quarter") was $17.5 million, compared to no revenue recognized for the three months ended December 31, 2024 (the "2024 Quarter"). The Company began commercial sales of the AVMAPKI FAKZYNJA CO-PACK within the U.S. following receipt of FDA approval in May 2025.
Total operating expenses for the 2025 Quarter were $59.0 million, compared to $31.6 million for the 2024 Quarter. Cost of sales associated with product revenue was $2.9 million for the 2025 Quarter, compared to no cost of sales recognized for the 2024 Quarter.
Research & development expenses for the 2025 Quarter were $31.7 million, compared to $20.8 million for the 2024 Quarter. The increase of $10.9 million, or 52.4%, was primarily due to higher costs incurred for drug substance and drug product manufacturing, contract research organizations, and investigator fees.
Selling, general & administrative expenses for the 2025 Quarter were $24.4 million, compared to $10.8 million for the 2024 Quarter. The increase of $13.6 million, or 125.9%, was primarily due to commercialization costs, including consulting, personnel costs, and professional fees, incurred in connection with the launch of AVMAPKI FAKZYNJA CO-PACK in KRAS-mutated recurrent LGSOC.
Net loss (GAAP basis) for the 2025 Quarter was $32.9 million, or $0.39 per share (basic), compared to $64.6 million, or $1.33 per share (basic and diluted) for the 2024 Quarter.
For the 2025 Quarter, non-GAAP adjusted net loss was $39.8 million, or $0.48 per share (basic) compared to non-GAAP adjusted net loss of $29.3 million, or $0.60 per share (basic), for the 2024 Quarter. Please refer to the GAAP to non-GAAP Reconciliation attached to this press release.
Full-Year 2025 Financial Results
Net product revenue for the year ended December 31, 2025 (the "2025 Period") was $30.9 million, compared to no product revenue recognized for the year ended December 31, 2024 (the "2024 Period"). Sale of COPIKTRA license and related assets revenue was $0.0 million for the 2025 Period, compared to $10.0 million for the 2024 Period. Revenue for the 2024 Period was comprised of one sales milestone payment of $10.0 million due upon Secura Bio achieving cumulative worldwide net sales of COPIKTRA exceeding $100.0 million.
Total operating expenses for the 2025 Period were $201.0 million, compared to $125.0 million for the 2024 Period. Cost of sales associated with product revenue was $5.3 million for the 2025 period, compared to no cost of sales recognized for the 2024 Period.
Research & development expenses for the 2025 Period were $114.6 million, compared to $81.3 million for the 2024 Period. The increase of $33.3 million, or 41.0%, was primarily due to higher costs incurred for contract research organizations, investigator fees, and drug substance and drug product manufacturing.
Selling, general & administrative expenses for the 2025 Period were $81.1 million, compared to $43.6 million for the 2024 Period. The increase of $37.5 million, or 86.0%, was primarily due to commercialization costs, including consulting, personnel costs, and professional fees, incurred in connection with the launch of AVMAPKI FAKZYNJA CO-PACK in KRAS-mutated recurrent LGSOC.
Net loss for the 2025 Period was $209.5 million, or $3.02 per share (basic and diluted), compared to $130.6 million, or $3.66 per share (basic and diluted) for the 2024 period.
For the 2025 Period, non-GAAP adjusted net loss was $163.1 million, or $2.35 per share (basic) compared to non-GAAP adjusted net loss of $107.4 million, or $3.01 per share (basic), for the 2024 Period. Please refer to the GAAP to non-GAAP Reconciliation attached to this press release.
Conference Call and Webcast
Verastem will host a conference call and webcast today at 4:30 p.m. ET to review the fourth quarter and full year 2025 financial results and recent business updates. To access the conference call, please dial (888) 596-4144 (U.S.) or (646) 968-2525 (international) and enter the passcode 7321921 at least 10 minutes prior to the event start time. A live audio webcast of the call, along with accompanying slides, will be available under "Events & Presentations" in the Investor section of the Company’s website, View Source A replay of the webcast will be archived and available following the event.
(Press release, Verastem, MAR 4, 2026, View Source [SID1234663254])