PellePharm is aiming to launch Phase III trials of the topical hedgehog inhibitor in early 2018.
Less than one year after clinical-stage biotech Pellepharm launched, the company snagged Breakthrough Therapy Designation and Orphan Drug Designation from the U.S. Food and Drug Administration for its topical drug patidegib.
Patidegib was awarded the designations after the company released positive Phase II data valuating the safety and efficacy of patidegib in patients with Gorlin Syndrome, a rare genetic disease that causes patients to develop multiple basal cell carcinomas (BCCs). PellePharm is aiming to launch Phase III trials of the topical hedgehog inhibitor in early 2018.
“These two significant FDA designations mark important steps forward in our development of topical patidegib as a drug with substantial potential to reduce the BCC tumor burden in Gorlin Syndrome patients. From our co-discovery of the underlying genetic defect, through over two decades of preclinical research with hedgehog inhibitors, and our recent clinical trials, our team at PellePharm is deeply committed to delivering a safe and effective therapy for people living with Gorlin Syndrome,” Ervin Epstein, PellePharm’s cofounder and chief medical officer, said in a statement. “We are eager to continue our development program and work closely with the FDA and other regulatory authorities to deliver this potential first-in-class therapy to patients as quickly as possible.”
Phase II results showed that after six months of self-treatment by the 17 patients in the trial, the topical treatment achieved statistically significant results with a complete response demonstrated in 12 tumors across both treatment arms compared with zero tumors in the placebo arm. Currently, there are no FDA-approved therapies for Gorlin Syndrome, and the standard of care for this rare disease is surgery. Patients with severe disease have as many as 30 surgeries per year.
Topical patidegib was developed to provide the efficacy previously demonstrated by oral patidegib in Phase 1 trials, but without the adverse systemic side effects of oral hedgehog inhibitors, the company said.
By awarding the two designations to PellePharm’s topical treatment, the drug development is likely to be accelerated. The FDA’s Breakthrough Therapy Designation is designed to expedite the development and review of drugs that are intended to treat a serious condition, where preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy. The FDA’s Orphan Drug Designation program is designed to incentivize and facilitate the development of drugs for rare diseases that affect fewer than 200,000 people in the U.S. Approximately 10,000 people in the United States, or one in 31,000, are believed to be affected by Gorlin Syndrome.
Jean Pickford, the executive director of the Basal Cell Carcinoma Nevus Syndrome Life Support Network, touted the designations were awarded to patidegib by the FDA. Pickford said people living with BCCNS need additional therapeutic options to help lessen the burden of disease.
