Pancreatic Cancer Drug Gets Orphan Status

FDA has granted PEP-Therapy an orphan drug designation for PEP-010 and its used for treating pancreatic cancer. PEP-010 is described by the company as a first-in-class bifunctional therapeutic peptide being evaluated in an ongoing Phase 1b clinical trial in combination with chemotherapy (paclitaxel or gemcitabine) in patients with metastatic pancreatic ductal adenocarcinoma and advanced or metastatic ovarian cancer pancreatic cancer.

PEP-010 “penetrates cells and specifically disrupts the interaction between Caspase-9 and PP2A, two key proteins involved in the apoptotic pathway,” the company says. “PP2A and Caspase-9, when released, restore normal apoptosis in cancer cells.” PEP-Therapy says the drug has demonstrated a good safety and tolerability profile and “first signals of efficacy” in the CLEVer-PEPtide Phase 1a dose escalation clinical trial.