PEP-Therapy’s lead product, PEP-010, a first-in-class bifunctional therapeutic peptide, received Orphan Drug Designation from the FDA for the treatment of pancreatic cancer. The peptide is currently in a Phase Ib clinical trial assessing its safety, tolerability, pharmacokinetics, and preliminary anti-tumor activity in combination with chemotherapy for pancreatic cancer patients. This designation is based on the significant unmet medical need in pancreatic cancer, a disease with limited treatment options and increasing incidence and mortality.
This FDA decision is crucial for advancing pancreatic cancer treatment. Pancreatic cancer is the fourth leading cause of cancer-related deaths in the U.S., with a dismal survival rate for advanced or metastatic cases. Current systemic treatments have seen limited progress, leaving a critical need for innovative therapies. PEP-010 offers a potential new approach by restoring normal apoptosis in cancer cells through a novel mechanism of action involving caspase-9 and PP2A proteins. The Orphan Drug Designation validates the drug’s potential to address this unmet need and encourages further research and development of this promising therapy.
The Orphan Drug Designation provides PEP-Therapy with significant benefits, including tax credits for clinical trials, exemptions from regulatory fees, and potential market exclusivity for seven years post-approval. This status will expedite PEP-010’s development and potentially accelerate its availability to patients. The ongoing Phase Ib trial is currently evaluating PEP-010 in combination with standard chemotherapy regimens, investigating its potential to improve outcomes for patients with this aggressive cancer.
This designation signifies a vital step toward providing a much-needed new treatment option for pancreatic cancer. It allows PEP-Therapy to further investigate PEP-010’s potential to improve the survival and quality of life for patients facing this challenging disease. The positive implications of this designation will likely encourage further investment and collaboration in the development of PEP-010, potentially leading to a significant advancement in the field of pancreatic cancer treatment.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
