Tag: Verastem Oncology

After a significant regulatory notification, shares of Verastem, Inc. (NASDAQ: VSTM) are up 39.51% to $5.12 as of the recent check on the US stock charts. VSTM’s New Drug Application (NDA) for avutometinib in combination with defactinib has been approved by the US Food and Drug Administration (FDA) via the accelerated approval process.

Innovative Therapy for Uncommon Ovarian Cancer

The suggested treatment is intended for adult patients with recurrent low-grade serous ovarian cancer (LGSOC) who have KRAS mutations. It consists of the oral RAF/MEK clamp avutometinib and the oral FAK inhibitor defactinib. The Prescription Drug User Fee Act or PDUFA action date is planned for June 30, 2025, as a result of the NDA’s October 2024 submission being awarded Priority Review.

For this study, the FDA does not presently have any plans to convene an advisory committee meeting. A major advancement in oncology, if approved, the medicine would be the first FDA-approved treatment especially for recurrent KRAS-mutant LGSOC. 

Clinical Evidence Supporting the Filing

The NDA builds on compelling evidence from the Phase 2 RAMP 201 trial, where the avutometinib-defactinib combination demonstrated a robust overall response rate, confirmed by an independent central review.

These responses were often durable, with patients generally tolerating the treatment well. Supporting data from the FRAME Phase 1 trial further substantiates the efficacy and safety of this innovative approach.

Pathway to Expanded Treatment Options

In parallel with the FDA review process, Verastem is advancing its RAMP 301 Phase 3 trial, enrolling patients with recurrent LGSOC regardless of KRAS mutation status. This confirmatory trial aims to broaden the therapy’s indication, potentially addressing a wider spectrum of patients.

With the FDA’s Priority Review designation, Verastem is positioned to deliver a groundbreaking treatment addressing a critical gap in cancer care. VSTM is actively preparing for a commercial launch anticipated in mid-2025, signaling hope for patients battling this rare disease.

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.

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.