EB Treatment Candidate BPM 31510 Granted Orphan Drug Designation in US

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to BERG’s BPM 31510 (ubidecarenone), a treatment candidate for patients with epidermolysis bullosa (EB), the company announced.

Medications granted orphan drug designation are intended to treat, diagnose or prevent rare conditions, defined as those affecting fewer than 200,000 people in the U.S. The designation qualifies pharmaceutical companies for development incentives, such as tax credits for clinical trials, fee exemptions, and a seven-year marketing exclusivity after FDA approval.

“This recognition by the FDA for topical BPM 31510 is a major milestone that will support ongoing clinical development of our EB program,” Niven R. Narain, BERG co-founder, president and CEO, said in a press release. “We are committed to the advancement of this asset and look forward to assessing how BPM 31510 may be able to make a difference in the lives of the patients and families dealing with such a life-altering diagnosis.”

Current estimates indicate that EB affects one in every 20,000 births in the U.S., or about 200 children born per year. The disease has no cure or treatment, and may lead to squamous cell carcinoma, the second-most common form of skin cancer.

BPM 31510 is a novel molecule that targets the dysregulated metabolism of cancer cells. These cells are able to generate energy from unconventional sources (meaning not through mitochondria, the powerhouses of cells) in order to support uncontrolled growth and avoid cell death. BPM 31510 restores the mitochondria as the cells’ source of energy, which reactivates pathways that detect cell damage and induce cell death.

“I am excited and proud to be working in partnership with BERG to help address various sub-types of EB and bring hope to patients and their families in need of new treatment options,” said Brett Kopelan, executive director of debra of America, an U.S. nonprofit that funds research and provides  free programs and services to the EB community.

Kopelan also said the team believes that BERG’s innovative drug discovery platform will lead to an EB treatment able to improve patients’ quality of life.

BERG is conducting a Phase 1 clinical trial (NCT02793960) testing a topical form of BPM 31510. The study is assessing the drug’s safety, effectiveness and pharmacological profile in all sub-types of EB. Patient recruitment is ongoing at the University of Miami Department of Dermatology. For information about enrollment, please list this link.

In November 2017, debra honored BERG with a Partners in Progress Award recognizing the company’s efforts in developing therapeutic strategies for EB.

In January, the FDA granted orphan-drug designation to the intravenous formulation of BPM 31510 for the treatment of pancreatic cancer. BERG is currently enrolling participants for a Phase 2 clinical trial (NCT02650804) in advanced pancreatic cancer.

BPM 31510 also is being evaluated as a treatment for glioblastoma, squamous cell carcinoma, and advanced refractory solid tumors.