On November 4, 2025 Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with RAS/MAPK pathway-driven cancers, reported business updates and announced financial results for the third quarter ended September 30, 2025.
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"Our performance in Q3, which was the first full quarter since our accelerated approval and launch of AVMAPKI FAKZYNJA CO-PACK, exceeded expectations with net revenue of over $11 million and demonstrated the strength of our growing commercial business and consistent adoption by both academic and community oncologists for the first treatment approved by the FDA specifically for patients with KRAS- mutated recurrent LGSOC," said Dan Paterson, president and chief executive officer of Verastem Oncology. "As we continue to build on this momentum and the fundamentals we have put into place to guide our commercial business, we’re simultaneously advancing our broader strategic priorities, and are very pleased with the progress of our clinical pipeline programs. Particularly for our KRAS G12D (ON/OFF) inhibitor, VS-7375, preliminary safety, tolerability, and anti-tumor activity are promising, and we believe in line as a potential best-in-class option for patients with pancreatic, lung, and other KRAS G12D-mutated solid tumor cancers. As we move ahead with opening the combination cohort with VS-7375 and cetuximab, we look forward to several important data readouts in the first half of 2026 that we believe will further demonstrate the breadth of our RAS/MAPK pathway-driven approach."
Third Quarter 2025 and Recent Updates
AVMAPKI FAKZYNJA CO-PACK (avutometinib in combination with defactinib) U.S. Launch
· Achieved net product revenue of $11.2 million in the first full quarter of the launch.
· Prescriptions for patients are being received from both academic and community centers, including both repeat prescriptions from physicians prescribing to multiple patients and refills for individual patients.
· There has been broad payer coverage and reimbursement since launch.
Avutometinib and Defactinib Combination in LGSOC
· In the ongoing Phase 3 RAMP 301 confirmatory trial, planned enrollment of the targeted 270 patients was completed a full quarter early.
· A pre-planned Interim Analysis (IA) by an Independent Data Monitoring Committee (IDMC) was conducted for RAMP 301, and the IDMC recommended a modest one-time increase in enrollment. Based on the current total enrollment achieved to date, an additional 29 patients will be added across KRAS mutation status. The Company remains blinded to the IA results.
· Preliminary safety and efficacy data from the Phase 2 RAMP 201J trial in Japan was accepted as an E-Poster (EP228/ #371) at the International Gynecologic Cancer Society (IGCS) 2025 Annual Meeting. In the published abstract, with a data extract date of April 11, 2025, no dose limiting toxicities were observed, and avutometinib and defactinib drug exposure levels were comparable to those observed in the global RAMP-201 study. Additional data, including efficacy (response rates) and updated safety will be available on November 5, 2025, when the embargo lifts.
Key Milestone:
· Expect to complete patient enrollment of the IDMC recommended increase in Q1 2026.
VS-7375, an Oral KRAS G12D (ON/OFF) Inhibitor, in Advanced Solid Tumors
· Announced a preliminary update on the Phase 1/2a monotherapy dose escalation trial of VS-7375 in patients with previously treated advanced KRAS G12D mutant solid tumors on Oct. 23, 2025.
o In the study, VS-7375 cleared both the 400 mg daily (QD) and the 600 mg QD monotherapy doses with no dose-limiting toxicities (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 pancreatic ductal adenocarcinoma (PDAC) and non-small cell lung cancer (NSCLC) by our partner, GenFleet Therapeutics, in its ongoing Phase 1/ 2 clinical study in China evaluating VS-7375 (known as GFH375). The Company’s dose escalation study continues with evaluation of the monotherapy 900 mg QD dose level.
o 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 are still on treatment. The remaining patients receiving either the 400 mg QD or 600 mg QD doses have not yet reached their first response assessment.
· The Company also announced it has initiated patient enrollment for the first dose escalation combination cohort evaluating VS-7375 with cetuximab in patients with advanced solid tumors, including colorectal cancer.
· Announced updated data from partner GenFleet Therapeutics’ Phase 1 /2 study of GFH375 in China that was featured in a late-breaking oral presentation at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress on October 19, 2025.
o Among 59 heavily pre-treated patients with PDAC who received one or more prior lines of therapy, an overall response rate (ORR) of 41% was achieved at the monotherapy recommended Phase 2 dose (RP2D) of 600 mg QD. A disease control rate (DCR) of 96.7% (57/59) was also reported with the majority of patients (91.5%) experiencing a reduction in target lesions.
o Overall survival (OS) observed at month four was 92.2%. The median OS was not reached as of the data cutoff, with a median follow-up time of 5.65 months. The median progression-free survival (mPFS) was 5.52 months with a median follow-up time of 5.65 months and a 4-month PFS rate of 78.2%. At evaluation, 31 (47%) patients were still on treatment with the longest duration of treatment eclipsing one year (367 days). The safety profile in PDAC patients was consistent with the previously reported data at recent medical congresses.
· Announced updated data from GenFleet’s Phase 1 /2 study of GFH375 in China that was featured in a mini oral presentation at the IASLC 2025 World Conference on Lung Cancer (WCLC) on September 8, 2025.
o At the 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).
· GenFleet shared the following additional analyses on Oct. 27, 2025, from previously presented data at recent medical congresses evaluating GFH375 in both advanced KRAS G12D mutant PDAC and NSCLC:
o 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 disease control rate (DCR) of 95.7%. In the 2L subgroup, the mPFS and 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 occured at rates above 8.0%.
o In an analysis of pre-treated patients with NSCLC at 600 mg QD, the four-month PFS rate was >75% and the mPFS has not been reached. The median follow-up time was 4.2 months.
· GenFleet also shared that the first patient has been dosed in a Phase 1b/2 study of GFH375 combined with cetuximab or chemotherapy for advanced solid tumors on October 22, 2025.
Key Milestones:
· Plan to initiate the dose escalation cohorts in combination with chemotherapy for PDAC and with chemotherapy plus anti-PD-1 for NSCLC in Q4 2025.
· Plan to report an interim safety and efficacy update on the Phase 1/2a trial of VS-7375 in 1H 2026.
· Expect to select the RP2D and plan to initiate monotherapy expansion cohorts in advanced PDAC, NSCLC, and other KRAS G12D-mutated solid tumors in 1H 2026.
· Expect to select the RP2D and plan to initiate combination expansion cohorts in CRC, PDAC, and NSCLC in 1H 2026.
· Plan to engage with the FDA to discuss our development path forward, including potential registration-directed clinical trials in PDAC and NSCLC in 1H 2026.
RAMP 205: Avutometinib Plus Defactinib in Combination with Chemotherapy in First-Line Metastatic PDAC
· Completed enrollment in the RAMP 205 expansion cohort in Q3 2025.
Key Milestone:
· Expect to report an update on the safety and efficacy of the RAMP 205 expansion cohort with 29 patients at the RP2D in 1H26.
RAMP 203: Avutometinib Plus Defactinib in Combination with a KRAS G12C Inhibitor in NSCLC
· Patients continue to be evaluated in both the doublet and triplet combination cohorts of the study.
Key Milestone:
· Report an interim update on the safety and efficacy results in RAMP 203 from both the doublet and triplet combinations in Q4 2025.
Third Quarter 2025 Financial Results
Net product revenue for the three months ended September 30, 2025 (the "2025 Quarter") was $11.2 million, compared to $0.0 million for the three months ended September 30, 2024 (the "2024 Quarter"). The Company began commercial sales of the AVMAPKI FAKZYNJA CO-PACK within the United States following receipt of FDA approval in May 2025.
Total operating expenses for the 2025 Quarter were $52.0 million, compared to $37.0 million for the 2024 Quarter. Cost of sales associated with product revenue was $1.7 million for the 2025 Quarter, compared to $0.0 for the 2024 Quarter.
Research & development expenses for the 2025 Quarter were $29.0 million, compared to $24.8 million for the 2024 Quarter. The increase of $4.2 million, or 16.9%, was primarily related to increased drug substance and drug product costs, increased contract research organization costs, and increased investigator trial costs.
Selling, general & administrative expenses for the 2025 Quarter were $21.0 million, compared to $12.3 million for the 2024 Quarter. The increase of $8.7 million, or 70.7%, was primarily related to commercialization costs required as part of the launch of AVMAPKI FAKZYNJA CO-PACK in KRAS-mutated recurrent LGSOC. This was comprised of increased consulting, personnel costs, and professional fees.
Net loss (GAAP basis) for the 2025 Quarter was $98.5 million, or $1.35 per share (basic and diluted), compared to $24.0 million, or $0.60 per share (basic and diluted) for the 2024 Quarter.
For the 2025 Quarter, non-GAAP adjusted net loss was $39.4 million, or $0.54 per share (diluted) compared to non-GAAP adjusted net loss of $35.3 million, or $0.88 per share (diluted), for the 2024 Quarter. Please refer to the GAAP to non-GAAP Reconciliation attached to this press release.
Verastem Oncology ended the third quarter of 2025 with cash, cash equivalents and investments of $137.7 million. With existing cash, product revenue, and exercise of cash warrants, Company has expected cash runway into the second half of 2026.
Conference Call and Webcast
Verastem will host a conference call and webcast today at 8:00 a.m. ET to review the third quarter 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 8194537 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, NOV 4, 2025, View Source [SID1234659377])