Verastem Reports $130.6 Million Net Loss for 2024, Highlights Regulatory Progress and Financial Concerns

Verastem, Inc., a late-stage biopharmaceutical company focused on RAS/MAPK pathway-driven cancers, reported a net loss of $130.6 million for the year ended December 31, 2024, compared to a net loss of $87.4 million in 2023. Revenue for 2024 was $10 million, stemming from a single sales milestone payment of $10 million received in July 2024 from Secura Bio, Inc., following Secura's achievement of over $100 million in worldwide net sales of COPIKTRA. Research and development expenses increased by $19.9 million to $81.3 million in 2024, primarily due to increased costs associated with the RAMP 301 trial and NDA submission for avutometinib and defactinib. Selling, general, and administrative expenses rose by $12.9 million to $43.6 million, largely attributed to increased personnel costs and commercialization preparation expenses.

Significant developments during the year included the completion of a rolling New Drug Application (NDA) submission to the FDA on October 31, 2024, for avutometinib and defactinib in recurrent KRAS mutant low-grade serous ovarian cancer (LGSOC). The FDA accepted the NDA for review under the accelerated approval pathway on December 30, 2024, granting priority review with a Prescription Drug User Fee Act (PDUFA) action date of June 30, 2025. The company also initiated a confirmatory Phase 3 trial (RAMP 301) in December 2023 to evaluate the combination therapy in recurrent LGSOC. In January 2025, Verastem exercised an option to license VS-7375, a KRAS G12D inhibitor, from GenFleet Therapeutics, making a $6 million payment.

Operational highlights included updated results from the RAMP 201 study, presented at the IGCS 2024 Annual Meeting, showing a confirmed overall response rate of 31% in all evaluable patients with measurable disease. Among patients with KRAS mutant LGSOC, the confirmed ORR was 44%, and for those with KRAS wild-type LGSOC, it was 17%. The company also reported initial safety and pharmacokinetic results from the RAMP 203 study, evaluating the combination of avutometinib and LUMAKRAS in KRAS G12C NSCLC, showing a confirmed ORR of 25%. Interim results from the RAMP 205 study, evaluating the combination of avutometinib and defactinib with gemcitabine/nab-paclitaxel in pancreatic cancer, showed a confirmed ORR of 83% in the dose level 1 cohort. As of December 31, 2024, Verastem had 78 full-time equivalent employees.

Verastem's financial statements reflect substantial doubt about its ability to continue as a going concern for the next 12 months. This assessment is based on recurring operating losses, a working capital deficiency, and the uncertainty surrounding future funding sources. The company plans to finance operations through existing cash, potential milestone payments and royalties from Secura, the Note Purchase Agreement, and other strategic financing opportunities. However, the probability of these opportunities is not considered sufficient to alleviate the going concern uncertainty. The company's outlook is contingent upon successful clinical trial outcomes, regulatory approvals, and securing additional funding.