PellePharm announced its launch today, with financing from BridgeBio Pharma. The Menlo Park, Calif.-based company is developing a topical gel to treat basal cell carcinoma, including the tumors that result from Gorlin syndrome.
The company’s topical inhibitor compound, patidegib, acts on the hedgehog pathway – a signaling pathway that transmits cellular information for adult tissue maintenance. Abnormal hedgehog signaling has been correlated with a number of cancers including basal cell carcinoma. PellePharm is studying the topical formulation of patidegib in 2 phase II trials. A study evaluating the gel’s use in patients with Gorlin syndrome just completed enrollment, according to the company.
“It made sense that a potent inhibitor of the hedgehog pathway should provide a therapeutic benefit for patients suffering from Gorlin Syndrome, but we needed an approach that would allow us to target the disease at its source without eliciting harmful toxicity reactions,” PellePharm co-founder Dr. Ervin Epstein said in prepared remarks. “We are hopeful that patidegib will provide the balance of targeted treatment without the adverse events associated with oral formulations.”
“It’s not often that you find a program as attractive as PellePharm’s patidegib,” executive chairman Mark de Souza added. “This investigational treatment has the potential to address a very high unmet need among patients with Gorlin Syndrome, whose current standard of care is to undergo multiple surgeries each year to manage their symptoms. We know that hedgehog pathway inhibitors have therapeutic potential for this condition, and we believe PellePharm’s topical formulation may offer a truly effective treatment option for these patients.”
“PellePharm is really at the intersection of precision oncology and monogenic dermatology – these are the types of assets we look for at BridgeBio Pharma,” BridgeBio Pharma investment committee member Frank McCormick said. “Gorlin Syndrome is a well-researched disease that can be treated directly at its source. We have had the privilege of knowing the founding and operating team of PellePharm for years and look forward to working with them to bring patidegib to patients in need.”