BERG announced the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation to the company's leading product candidate BPM31510, for the treatment of pancreatic cancer.
"The FDA's orphan-drug designation marks an important milestone for BERG," said Niven R. Narain, BERG President and Chief Executive Officer. "We look forward to working closely with the FDA and other regulatory authorities to advance the clinical development of this therapeutic and map out the most effective match for treatment of pancreatic cancer.”
The FDA's orphan-drug designation program provides orphan status to drugs defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases that affect fewer than 200,000 people in the United States. Orphan designation qualifies the sponsor of the drug for certain development incentives, including tax credits for qualified clinical testing, prescription drug user fee exemption and seven-year marketing exclusivity upon FDA approval.
After completion of a Phase 1 clinical trial establishing safety, BERG initiated a Precision Medicine driven Phase 2 clinical trial design for BPM31510-IV alone and in combination with gemcitabine to evaluate the efficacy in patients with advanced pancreatic cancer in the US and Europe. As part of this Phase 2 trial, the Company is driving towards identification and validation of molecular profiles representative of BPM31510 mediated outcomes, which could lead to a diagnostic panel for patient stratification.
Pancreatic cancer is a disease with a high unmet medical need. In 2017, an estimated 53,070 adults in the United States will be diagnosed with pancreatic cancer and about 73% of those people will die within a year of that diagnosis. The majority of pancreatic cancer cases are diagnosed late, at which point the disease is already locally advanced or metastatic. The disease accounts for about 3 percent of all cancers in the US and is the fourth leading cause of cancer death in men and women according to the American Cancer Society.
Cancer cells alter metabolism to generate energy from non-mitochondrial pathways to support uncontrolled growth. This allows the cancer cells to escape molecular mechanisms controlling cell death. BPM31510 is a first in class molecule that specifically targets the dysregulated metabolism observed in cancer. BPM31510, by targeting metabolism in cancer cells, re-engages the mitochondria to generate energy, shifting metabolism to that observed in the normal cell. The effect of BPM31510 on metabolism results in the reactivation of pathways that detect cell damage, triggering apoptosis or programmed cell death.