On May 7, 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 first quarter ended March 31, 2026, and highlighted recent business progress.
Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:
Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing
Schedule Your 30 min Free Demo!
"As we mark the one-year anniversary of the FDA-approval of AVMAPKI FAKZYNJA CO-PACK for KRAS-mutated recurrent low-grade serous ovarian cancer, I am incredibly proud of the progress we have made to deliver a meaningful impact for people living with this rare cancer," said Dan Paterson, president and chief executive officer at Verastem Oncology. "As we build on the foundation we have established with the commercial launch, we also look to continue accelerating VS-7375, our potential best-in-class oral, KRAS G12D (ON/OFF) inhibitor, and are pleased to announce the initiation of three Phase 2 registration-directed trials in pancreatic, non-small cell lung, and colorectal cancers. In an evolving competitive landscape, we remain encouraged by the efficacy data generated by our partner and emerging in the U.S., along with the safety and tolerability profile we are seeing across multiple solid tumor cancers and look forward to sharing data updates throughout this year. Altogether, we believe we are well-positioned to drive strong execution across our commercial launch and expedite VS-7375 development while maintaining disciplined capital management."
First Quarter 2026 and Recent Updates
AVMAPKI FAKZYNJA CO-PACK (avutometinib capsules; defactinib tablets) U.S. Commercial Performance
AVMAPKI FAKZYNJA CO-PACK generated net product revenue of $18.7 million for the first quarter of 2026.
In April, the Company announced the launch of a new healthcare professional and patient marketing campaign, Reimagine Recurrent Low-Grade Serous Ovarian Cancer (LGSOC), to drive awareness of AVMAPKI FAKZYNJA CO-PACK.
In April, the Company announced new two-year median follow up data from the Phase 2 RAMP 201 trial that demonstrated durable benefit of avutometinib plus defactinib across both KRAS mutant and KRAS wild-type recurrent LGSOC patients, with discontinuation rates consistent with the primary analysis, presented at the Society of Gynecologic Oncology 2026 Annual Meeting on Women’s Cancers. A new exposure-response analysis further demonstrated that the approved dose and schedule of avutometinib plus defactinib achieve the optimal therapeutic effect.
In February, the Company announced updated data for RAMP 201J in Japan evaluating the combination in patients with LGSOC with or without a KRAS mutation. As of a data cutoff of January 30, 2026, a confirmed overall response rate (ORR) of 38% (6/16) was achieved. Patients with a KRAS mutation achieved a confirmed ORR of 57% (4/7) and KRAS wild-type patients achieved a confirmed ORR of 22% (2/9). The safety profile was consistent with previously reported data outside of Japan.
Expected Key Milestones:
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
Today, the Company provided an update on its ongoing VS-7375 clinical trials, including branding the trials as the VS-7375 TARGET-D Clinical Trial Program. In the ongoing TARGET-D 101 (VS-7375-101) Phase 1/2 dose escalation, dose expansion and combination-evaluation trial, the Company is now evaluating the 1200 mg daily dose (QD). The Company is also evaluating VS-7375 900 mg QD in combination with cetuximab. The Company expects to finish enrollment across the various expansion cohorts and combination cohorts with chemotherapies in the near term.
In addition, the Company shared updated pharmacokinetic (PK) data that showed the 900 mg QD dose achieves target plasma levels of VS-7375 and provides clear separation from the 600 mg QD dose.
The Company has initiated three Phase 2 registration-directed trials, including:
TARGET-D 201 to evaluate VS-7375 at 900 mg QD both as monotherapy and in combination with cetuximab in patients with second-line pancreatic ductal carcinoma (PDAC). The study is also evaluating VS-7375 and cetuximab in the first-line PDAC setting.
TARGET-D 202 to evaluate VS-7375 at 900 mg QD in patients with advanced non-small cell lung cancer (NSCLC) who have received one to two prior lines of therapy. The study is also evaluating VS-7375 in NSCLC patients with asymptomatic untreated brain metastases.
TARGET-D 203 to evaluate VS-7375 at 900 mg QD as both monotherapy and in combination with EGFR inhibitors, including cetuximab or panitumumab, in previously treated colorectal cancer (CRC). The study will also evaluate VS-7375 in combination with chemotherapy in the first-line setting in patients with metastatic CRC.
In March, the Company announced that several late-breaking and regular abstracts were selected for presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. The abstracts included preclinical data demonstrating sustained tumor regressions with VS-7375 in combination with PRMT5 inhibitors in MTAP-deleted KRAS G12D mutant pancreatic cancer and improved efficacy in comparison to ON-only KRAS G12D or pan-RAS inhibitors.
In March, the Company reported an update on its progress with VS-7375, highlighting progress with its dose-escalation and dose-expansion trial, TARGET-D 101, a PK analysis, and a safety update, which demonstrated VS-7375 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. As of the January 30, 2026 data cutoff, 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. The Company also reported that the U.S. Food and Drug Administration (FDA) requested the Company develop separate Phase 2 trial protocols for their trials in PDAC, NSCLC and CRC.
In January, the Company reported progress from its ongoing TARGET-D 101 trial, with multiple dose levels cleared without dose-limiting toxicities or major toxicities. At that time, monotherapy cohorts and dose escalation combination cohorts were initiated across various KRAS G12D-mutated solid tumors.
The Company shared multiple updates from GenFleet Therapeutics, its partner developing VS-7375, known as GFH375 in China.
In April, GenFleet announced that GFH375 was granted Breakthrough Therapy Designation (BTD) in China for patients with KRAS G12D-mutated metastatic pancreatic cancer who have received at least one prior systemic therapy.
In March, GenFleet announced that GFH375 was granted BTD in China for patients with KRAS G12D-mutated NSCLC who have received prior systemic therapy.
Expected Key Milestones:
Report early data from the TARGET-D 101 trial in 1H 2026.
Report an update on the TARGET-D 101 trial in 2H 2026.
Announce first patient initiated in the TARGET-D 201, TARGET-D 202, and TARGET-D 203 clinical trials in mid-2026.
RAMP 205: Avutometinib Plus Defactinib in Combination with Chemotherapy in 1L Metastatic Pancreatic Cancer
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.
Corporate Updates
The Company announced today the appointment of Daniel Lyons as chief commercial officer. Prior to joining, Mr. Lyons served as the Senior Vice President, Head of Rare Tumors International and Global Head of Market Access at SpringWorks Therapeutics.
First Quarter 2026 Financial Results
Verastem Oncology ended the first quarter of 2026 with cash, cash equivalents, and investments of $181.7 million.
Net product revenue for the three months ended March 31, 2026 (the "2026 Quarter") was $18.7 million, compared to no revenue recognized for the three months ended March 31, 2025 (the "2025 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 2026 Quarter were $63.6 million, compared to $44.2 million for the 2025 Quarter. Cost of sales was $3.1 million for the 2026 Quarter, compared to no cost of sales recognized for the 2025 Quarter.
Research & development expenses for the 2026 Quarter were $38.2 million, compared to $29.2 million for the 2025 Quarter. The increase of $9.0 million, or 31%, was primarily due to higher costs for investigator fees, drug substance manufacturing, drug product manufacturing, clinical supply, and contract research organizations partially offset by the VS-7375 program option exercise fee paid to GenFleet in the 2025 Quarter.
Selling, general & administrative expenses for the 2026 Quarter were $22.3 million, compared to $15.0 million for the 2025 Quarter. The increase of $7.3 million, or 48%, was primarily due to higher costs for personnel and commercial operations.
Net loss (GAAP basis) for the 2026 Quarter was $36.6 million, or $0.37 per share (basic), compared to $52.1 million, or $0.96 per share (basic and diluted) for the 2025 Quarter.
Non-GAAP adjusted net loss for the 2026 Quarter was $42.7 million, or $0.43 per share (basic) compared to non-GAAP adjusted net loss of $42.9 million, or $0.79 per share (basic), for the 2025 Quarter. 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 first quarter 2026 financial results and recent business updates. To access the live audio webcast of the call, along with accompanying slides, please visit the "Events & Presentations" page 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, MAY 7, 2026, View Source [SID1234665350])