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BOSTON - Verastem Oncology (NASDAQ:VSTM), a clinical-stage biopharmaceutical company with a market capitalization of $230.51 million, reported updated results from the Phase 1/2 FRAME study showing a 42.3% overall response rate (ORR) and 20.1 months median progression-free survival (mPFS) in patients with low-grade serous ovarian cancer (LGSOC), regardless of KRAS mutation status. According to InvestingPro data, the company maintains a healthy liquidity position with a current ratio of 3.5, providing financial flexibility for its clinical programs.
The study, conducted by the Institute of Cancer Research and The Royal Marsden NHS Foundation Trust, demonstrated even stronger results in patients with KRAS-mutated LGSOC, with an ORR of 58.3% and mPFS of 30.8 months.
The FRAME study evaluated the safety and efficacy of avutometinib in combination with defactinib in patients with LGSOC, non-small cell lung cancer, and other solid tumor types. This research provided the foundation for the FDA’s approval of the combination therapy for KRAS-mutated recurrent LGSOC on May 8, 2025.
In the 26 LGSOC patients evaluable for efficacy, researchers observed an ORR of 42.3%. Among the 24 patients whose samples could be sequenced for KRAS mutations, those with KRAS mutations (12 patients) showed an ORR of 58.3% and mPFS of 30.8 months, while those without KRAS mutations (12 patients) had an ORR of 33.3% and mPFS of 8.9 months.
The study also found that 27.3% of patients who had previously received a MEK inhibitor responded to the combination therapy. Additionally, two LGSOC patients with brain metastases showed shrinkage of these metastases at 30 months post-treatment.
The safety profile was manageable, with only one patient (4%) discontinuing treatment due to skin toxicity. The most common adverse events included rash, elevated blood levels of creatine phosphokinase, AST elevation, hyperbilirubinemia, and diarrhea.
The combination therapy works by inhibiting MEK kinase activity while blocking compensatory reactivation of MEK by upstream RAF, with defactinib targeting FAK, a key mediator of drug resistance. Wall Street analysts maintain a bullish outlook on Verastem’s potential, with price targets ranging from $12 to $20 per share, significantly above current trading levels. InvestingPro analysis indicates the stock is currently trading below its Fair Value, suggesting potential upside opportunity.
Verastem is currently conducting RAMP 301, an international Phase 3 confirmatory trial evaluating this combination against standard chemotherapy or hormonal therapy for recurrent LGSOC.
According to the press release, LGSOC affects approximately 6,000-8,000 women in the U.S. and 80,000 worldwide, with about 30% of cases having a KRAS mutation. While the company reported $10 million in revenue over the last twelve months, InvestingPro analysis reveals strong financial fundamentals with more cash than debt on its balance sheet. Investors seeking deeper insights into Verastem’s financial health, growth prospects, and detailed Fair Value analysis can access the comprehensive Pro Research Report, available exclusively to InvestingPro subscribers.
In other recent news, Verastem Oncology has reported significant developments in its ongoing research and trials. The company has dosed the first patient in a U.S. Phase 1/2a clinical trial for VS-7375, an oral KRAS G12D inhibitor, targeting advanced KRAS G12D mutant solid tumors. This trial follows promising results from a Chinese study, with plans to expand into pancreatic and non-small cell lung cancer cohorts. Additionally, Verastem presented positive results from a Phase 1/2 trial of GFH375, with an overall response rate of 52% in pancreatic cancer patients and 42% in non-small cell lung cancer patients. The company is also advancing its RAMP 205 trial, which showed an 83% response rate in metastatic pancreatic cancer, and plans a Phase 3 study in 2026.
Cantor Fitzgerald has reiterated an Overweight rating for Verastem, reflecting confidence in its research efforts. Furthermore, Jefferies has increased Verastem’s price target to $19 following early FDA approval of its avutometinib and defactinib combination therapy for a rare form of ovarian cancer. This approval, ahead of the expected date, marks a milestone for Verastem, potentially representing a $402 million market opportunity in the U.S. These developments underscore Verastem’s commitment to advancing cancer treatments and its strategic direction in oncology.
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