Sol-Gel Technologies acquires Phase 3 Gorlin syndrome candidate patidegib

Sol-Gel Technologies acquired topically-applied patidegib, a hedgehog signaling pathway blocker, for the treatment of Gorlin syndrome from PellePharm. Gorlin syndrome is a rare disease with no currently approved therapies by the FDA or EMA. This new chemical entity, designated as investigational compound SGT-610, has the potential to be the first-ever drug for treatment of Gorlin syndrome. Sol-Gel expects the transaction to close on or about January 30, 2023. Patidegib has been granted Orphan Drug Designation by the FDA and the EMA 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. Sol-Gel estimates that SGT-610, if approved by the FDA, has the potential to generate, at peak, annual net sales in excess of $300M. Gorlin syndrome affects approximately 1 in 31,000 people in the U.S. and is an autosomal dominant genetic disorder, mostly caused by inheritance of one defective copy of the tumor suppressor gene PTCH1. Sol-Gel’s Phase 3 study is expected to begin in the second half of 2023 with results expected by the end of 2025.Sol-Gel will pay PellePharm an upfront payment of $4.7M and total development and NDA acceptance milestones of up to $6.0M, and up to $64.0M in commercial milestones as well as single digit royalties.