Verastem, Inc. (NASDAQ: VSTM) experienced a notable increase in its share value following the receipt of a significant designation from regulatory authorities. The stock demonstrated a bullish trend on US stock charts, climbing by 6.57% to $2.92 during the after-market session on Monday. With this increase, the -7.43% loss during regular trading hours was recovered.
Table of Contents
Toggle- FDA Approves Orphan Drug Status
- Positive Interim Results and Future Prospects for Verastem
FDA Approves Orphan Drug Status
Verastem stated that, when combined with defactinib, a selective FAK inhibitor, avutometinib, an RAF/MEK clamp, has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA). This classification relates to pancreatic cancer therapy. The acknowledgement highlights the significant unfulfilled medical need for efficient therapies in this area.
Positive Interim Results and Future Prospects for Verastem
At the ASCO 2024 Annual Meeting, Verastem presented preliminary interim findings from the ongoing RAMP 205 trial. This trial is assessing the efficacy of the combination of avutometinib and defactinib alongside standard chemotherapy in first-line metastatic pancreatic cancer. The positive interim results highlight the potential of this therapeutic approach to offer a novel treatment strategy for this challenging disease.
VSTM anticipates releasing updated data from various dose cohorts of the RAMP 205 trial in the first quarter of 2025. As of May 14, 2024, 41 patients had been administered treatment across four dose cohorts, with those in dose cohort 1 showing promising results. Specifically, 83% of patients in this cohort achieved a confirmed partial response with over six months of follow-up.
Furthermore, one dose-limiting toxicity was noted in the dose level 1 cohort, which was subsequently resolved upon the inclusion of additional patients. Of the 26 patients in all groups who had their initial scans, 21 reported a reduction in the target lesions' overall diameter.
The FDA's Orphan Drug Designation offers the possibility of a seven-year market exclusivity following approval for treatments addressing rare diseases affecting fewer than 200,000 people in the US, as well as tax credits for qualifying clinical trials and waivers of certain FDA user fees.