Sol-Gel Acquires Phase 3 Ready Gorlin syndrome Therapy from BridgeBio’s PellePharm

Rare Daily Staff

Israel-based Sol-Gel Technologies said it acquired topically-applied patidegib, a hedgehog signaling pathway blocker, for the treatment of Gorlin syndrome from PellePharm for up to $75 million.

Sol-Gel, which has two dermatology products on the market in the United States launched by its partner Galderma, said the deal broadens its pipeline with a new chemical entity, designated as SGT-610, which has the potential to be the first-ever drug for treatment of Gorlin syndrome.

PellePharm an upfront payment of $4.7 million and total development and NDA acceptance milestones of up to $6 million, and up to $64 million in commercial milestones as well as single digit royalties.

Gorlin syndrome is a genetic disorder caused by one defective copy of the tumor suppressor gene PTCH1. Gorlin syndrome is also called nevoid BCC syndrome because approximately 90 percent of individuals with this syndrome develop multiple basal cell carcinomas (BCCs) by age 35, ranging from a few to many thousands of lesions during a patient’s lifetime. Painful surgical excision is the treatment of choice for BCCs. However, as multiple BCCs continue to evolve, repeated surgical intervention becomes impractical, and therefore, an important consideration in the treatment of Gorlin syndrome is preventing the development of new BCCs.

SGT-610 is intended to prevent new BCC formation in adults with Gorlin syndrome without the accompanying systemic adverse events observed with oral BCC therapies.

The experimental therapy has been granted Orphan Drug designation by the U.S. Food and Drug Administration and European Medicines Agency, as well as Breakthrough Therapy Designation by the FDA. Both FDA and EMA have stated that approval may be supported by a single pivotal phase 3 study.

The planned phase 3 study of SGT-610 will include well-defined modifications to an earlier phase 3 study in which the patidegib arm was found to be as tolerable as the vehicle and the significant adverse events associated with oral hedgehog inhibitors were not observed. These modifications will include selecting patients positive for the PTCH1 mutation (in contrast to the previous study which included symptomatic patients without testing them for the mutation), as well as a requirement for a higher minimum number of BCCs at baseline. Sol-Gel’s phase 3 study is expected to begin in the second half of 2023 with results expected by the end of 2025.

“Rare diseases represent a high margin therapeutic category, and we estimate that SGT-610, if approved by the FDA, has the potential to generate, at peak, annual net sales in excess of $300 million,” Alon Seri-Levy, CEO of Sol-Gel, said. “We believe that the risk/reward of this deal is extremely favorable to Sol-Gel and its shareholders.”

Photo: Alon Seri-Levy, CEO of Sol-Gel