Verastem Oncology, a biopharmaceutical firm dedicated to developing new cancer treatments, has provided an update on its business activities and financial performance for the second quarter ended June 30, 2024.
Dan Paterson, the company’s President and CEO, highlighted significant advancements: “In Q2, we achieved major milestones including the initiation of our rolling NDA submission for the combination of avutometinib and defactinib for recurrent KRAS mutant low-grade serous ovarian cancer. We also released updated topline results from the RAMP 201 trial and shared positive interim findings from our first-line metastatic pancreatic cancer study. Looking ahead, we plan to present mature RAMP 201 data, finalize our NDA submission, and release interim data from our lung cancer programs.”
Second Quarter 2024 Highlights and Recent Developments
Avutometinib and Defactinib Combination for Low-Grade Serous Ovarian Cancer (LGSOC)
- Initiated a rolling New Drug Application (NDA) submission for the combination of avutometinib and defactinib, aimed at accelerated approval for adult patients with recurrent KRAS mutant LGSOC who have undergone at least one prior systemic therapy.
- Announced interim topline data from the RAMP 201 trial, showing strong overall response rates and durable responses with low discontinuation rates due to adverse events, based on a minimum five-month follow-up as of February 2024.
- Plans to complete the rolling NDA submission by including mature safety and efficacy data from the RAMP 201 trial, with 12 months of follow-up, expected in the latter half of 2024.
- Enrollment and site activations for the international Phase 3 RAMP 301 trial are in progress across the U.S., Australia, Europe, and the UK, evaluating the combination against standard chemotherapy or hormonal therapy.
- Ongoing preparations for a potential U.S. launch in 2025 and discussions with European and Japanese regulatory bodies are advancing.
Avutometinib and KRAS G12C Inhibitors for Non-Small Cell Lung Cancer (NSCLC)
- Initiated the RAMP 203 trial, testing a triplet combination of avutometinib, defactinib, and sotorasib in patients who have progressed on KRAS G12C inhibitors.
- Updated interim data from the Phase 1/2 RAMP 203 trial, and initial data from the Phase 1/2 RAMP 204 trial, evaluating avutometinib with adagrasib, are expected in H2 2024.
Avutometinib and Defactinib in First-Line Metastatic Pancreatic Cancer
- Presented early safety and efficacy results from the RAMP 205 trial at the ASCO Annual Meeting on June 1, 2024. As of May 14, 2024, 41 patients were treated, with 83% of those in the first dose cohort achieving a confirmed partial response. One dose-limiting toxicity was observed, but the dose level was cleared following additional patient enrollment.
- The FDA granted Orphan Drug Designation for the combination in July 2024.
- Updated data from the RAMP 205 trial are anticipated in Q1 2025.
GFH375 (VS-7375): Oral KRAS G12D (ON/OFF) Inhibitor
- GenFleet Therapeutics received clearance for the GFH375 (VS-7375) IND application in China in June 2024.
- The first patient was dosed in the Phase 1/2 trial for advanced KRAS G12D-mutated solid tumors in July 2024.
- Plans are underway to initiate studies outside China based on initial dose escalation data from the Phase 1 study.
Corporate Updates
- Appointed John Hayslip, M.D., as Chief Medical Officer in April 2024 and promoted Nate Sanburn to Chief Business Officer in June 2024.
- Raised approximately $51.1 million through a public offering in July 2024 and received a $10 million milestone payment from Secura Bio, Inc. for COPIKTRA sales.
Financial Results for Q2 2024
- Ended Q2 2024 with cash, cash equivalents, and investments totaling $83.4 million. Pro forma, including July 2024 proceeds, the total was $144.5 million, providing a cash runway into early 2025.
- Reported $10.0 million in total revenue, primarily from a milestone payment related to COPIKTRA sales.
- Operating expenses for Q2 2024 were $28.3 million, compared to $20.3 million for Q2 2023. R&D expenses increased to $18.1 million, driven by higher costs for research and consulting. SG&A expenses rose to $10.2 million, reflecting preparations for potential drug launches and increased personnel costs.
- Net loss was $8.3 million ($0.31 per share), down from $24.3 million ($1.37 per share) in Q2 2023.
- Non-GAAP adjusted net loss was $16.5 million ($0.61 per share) compared to $18.8 million ($1.06 per share) in Q2 2023.
