Verastem Oncology, a biopharmaceutical company committed to advancing new medicines for patients, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to avutometinib, a RAF/MEK clamp, in combination with defactinib, a selective FAK inhibitor, for the treatment of pancreatic cancer.
“At the ASCO 2024 Annual Meeting, we reported positive initial interim results from the ongoing RAMP 205 trial evaluating avutometinib and defactinib in combination with standard of care chemotherapy in first-line metastatic pancreatic cancer,” said Dan Paterson, president and chief executive officer of Verastem Oncology. “The FDA Orphan Drug Designation for the combination of avutometinib and defactinib for the treatment of pancreatic cancer recognizes the substantial unmet treatment need for patients with pancreatic cancer. We believe avutometinib and defactinib in combination with standard of care has an opportunity to provide a different approach in treating this challenging cancer. We look forward to reporting updated data from across dose cohorts in the ongoing RAMP 205 trial in the first quarter of 2025.”
At the American Society of Clinical Oncology (ASCO) Annual Meeting in June 2024, Verastem presented initial interim safety and efficacy results from the ongoing RAMP 205 trial of avutometinib and defactinib in combination with current standard of care gemcitabine and nab-paclitaxel in first-line metastatic pancreatic cancer. As of the data cutoff of May 14, 2024, 41 patients had been treated in one of four dose cohort regimens and only patients in dose cohort 1 had a minimum follow up of six months. In the dose level 1 cohort, 83% (5/6) of patients achieved a confirmed partial response with more than six months of follow up at the time of data cutoff. One dose-limiting toxicity (DLT) was observed in the dose level 1 cohort, and the dose level was subsequently cleared after additional patients were enrolled. Of the 26 patients in all cohorts who have had the opportunity to have their first scan while on treatment, 21 have experienced a reduction of the change in target lesion sum of diameters.
FDA Orphan Drug Designation is granted to certain investigational treatments for diseases or conditions that affect fewer than 200,000 people in the United States. Orphan Drug Designation provides benefits to drug developers, including tax credits for qualified clinical trials, exemptions from certain FDA user fees and the potential for seven years of market exclusivity upon approval.
SOURCE: Businesswire