Verastem Oncology Q4 & Full-Year 2024 Results, Business Updates
Verastem Oncology, a biopharmaceutical company dedicated to developing innovative therapies for cancer patients, has announced its financial results for the fourth quarter and full year ending December 31, 2024, along with recent business updates.
Significant Progress in 2024 and Outlook for 2025
Dan Paterson, President and CEO of Verastem Oncology, emphasized the remarkable strides the company made in 2024. Most notably, the FDA accepted its New Drug Application (NDA) for avutometinib combined with defactinib for Priority Review under the accelerated approval pathway for KRAS mutant recurrent low-grade serous ovarian cancer (LGSOC). This milestone sets the stage for a potentially transformative 2025, with the anticipated launch of the first FDA-approved treatment for this indication, alongside advancements in pancreatic cancer, non-small cell lung cancer (NSCLC), and the initiation of a Phase 1/2a study for VS-7375, a KRAS G12D (ON/OFF) inhibitor.
Clinical and Regulatory Highlights
Avutometinib and Defactinib in LGSOC
- On December 30, 2024, the FDA granted Priority Review for the NDA submission of the avutometinib-defactinib combination, assigning a Prescription Drug User Fee Act (PDUFA) action date of June 30, 2025.
- The submission was backed by positive data from the RAMP 201 trial, presented at the International Gynecologic Cancer Society (IGCS) 2024 Annual Meeting. Additional support came from the FRAME Phase 1 trial.
- Commercial preparation activities for the anticipated U.S. launch in mid-2025 are underway.
- Data from the RAMP 201 primary analysis, including subgroup analysis by KRAS mutational status, was presented at the Society of Gynecologic Oncology 2025 Annual Meeting on Women’s Cancer on March 17, 2025. The analysis showed promising responses across patient subgroups.
- The confirmatory Phase 3 RAMP 301 trial, enrolling globally, is on track to complete enrollment by year-end 2025.
- Japan’s Gynecologic Oncology Group (JGOG) dosed the first patient in RAMP201J, a Phase 2 study evaluating the combination therapy in Japan.
Expected 2025 Milestones:
- FDA decision on the NDA submission by June 30, 2025.
- Submission for NCCN guideline inclusion upon approval.
- Publication of primary analyses from FRAME and RAMP 201 in H1 2025.
- Completion of RAMP 301 enrollment by year-end.
- Initial data from RAMP 201J in H2 2025.
- Progress in regulatory pathways for Japan and Europe.
RAMP 205: Avutometinib and Defactinib in Pancreatic Cancer
- A new dose level “0” was introduced to evaluate the combination with gemcitabine and Nab-paclitaxel.
- All dose levels expanded to 12 patients, with promising responses observed.
- Enrollment completion anticipated in Q1 2025.
Expected 2025 Milestones:
- Additional data presentation mid-year 2025.
- Selection of the recommended Phase 2 Dose (RP2D) in H1 2025.
RAMP 203: Avutometinib and Defactinib in NSCLC
- Enrollment in Stage 1 Part B doublet cohort expected to conclude in Q1 2025.
- The triplet combination study with LUMAKRAS™ (sotorasib) and defactinib continues with promising safety data.
Expected 2025 Milestones:
- Interim data update on doublet and triplet regimens in H2 2025.
VS-7375: KRAS G12D (ON/OFF) Inhibitor in Advanced Solid Tumors
- Verastem filed an IND application for VS-7375 in Q1 2025 and exercised its early option to license the candidate from GenFleet.
- Preliminary Phase 1 data from China demonstrated oral bioavailability, a favorable safety profile, and partial responses in pancreatic and lung cancers.
Expected 2025 Milestones:
- Initiation of a U.S.-based Phase 1/2a trial by mid-2025.
- Presentation of preclinical and Phase 1 clinical data in H1 2025.
Corporate Developments
- Strengthened leadership with Matthew E. Ros appointed as Chief Operating Officer in January 2025.
- Financial agreements with Oberland Capital and IQVIA bolster commercialization and R&D funding.
Financial Performance
Fourth Quarter 2024 Results
- Cash, cash equivalents, and investments stood at $88.8 million.
- Pro forma cash, incorporating new financing arrangements, was $151.3 million.
- Total operating expenses were $31.6 million, up from $31.1 million in Q4 2023.
- R&D expenses decreased to $20.8 million from $22.5 million due to lower CRO and drug costs.
- Selling, general & administrative (SG&A) expenses rose to $10.8 million from $8.6 million due to personnel and consulting costs.
- Net loss was $64.6 million ($1.33 per share), compared to $27.4 million ($1.02 per share) in Q4 2023.
- Non-GAAP adjusted net loss was $29.3 million ($0.60 per share) vs. $29.6 million ($1.10 per share) in Q4 2023.
Full-Year 2024 Results
- Total operating expenses increased to $125.0 million from $92.1 million in 2023.
- R&D expenses grew to $81.3 million from $61.4 million due to higher CRO and investigator fees.
- SG&A expenses rose to $43.6 million from $30.7 million due to commercialization investments.
- Net loss was $130.6 million ($3.66 per share) vs. $87.4 million ($3.96 per share) in 2023.
- Non-GAAP adjusted net loss was $107.4 million ($3.01 per share) vs. $85.2 million ($3.86 per share) in 2023.
Market Position and Competitive Landscape
With the anticipated approval and commercialization of avutometinib and defactinib for LGSOC, Verastem is positioning itself as a leader in KRAS-targeted therapies. The competition includes companies developing MEK inhibitors, such as Novartis’ trametinib and Roche’s cobimetinib, but Verastem’s dual approach targeting MEK and FAK signaling pathways sets its therapy apart in terms of efficacy and potential synergistic benefits.
The company’s expansion into pancreatic and lung cancers aligns with growing market demand for novel KRAS inhibitors. Competitors in the space, including Mirati Therapeutics and Amgen, have focused on KRAS G12C inhibitors, whereas Verastem Oncology’s VS-7375 program targeting KRAS G12D offers differentiation and potential advantages in a broader patient population.
