Sol-Gel hits LPLV milestone in Phase 3 Gorlin syndrome trial

Sol-Gel Technologies has announced that the last patient has completed the final scheduled visit in its pivotal Phase 3 trial of SGT-610 (patidegib gel, 2%) for Gorlin syndrome. The Ness Ziona-based dermatology company said the last patient last visit (LPLV) milestone clears the way for database lock and statistical analysis, with top-line results expected in late November 2026.

The randomised, double-blind, vehicle-controlled study enrolled 113 patients across a 12-month treatment period, with 102 completing the trial. Patients received either SGT-610 or a vehicle control twice daily. The primary endpoint measures the number of new basal cell carcinoma (BCC) lesions appearing on the face at month 12, while secondary endpoints include the count of new surgically eligible BCCs. All participants, investigators and the company itself remain blinded to treatment assignment pending the analysis.

The programme

SGT-610 holds both orphan drug and breakthrough therapy designations from the US Food and Drug Administration, a combination that could accelerate the regulatory path to approval should the trial succeed. Patidegib, the active compound, is a topical hedgehog pathway inhibitor. Oral hedgehog inhibitors such as vismodegib and sonidegib are already approved for BCC indications, but their systemic side-effect profiles have limited long-term use. Sol-Gel is positioning SGT-610 as a topically delivered alternative that may offer improved tolerability, though no head-to-head data with oral agents has been released.

Executive Chairman and CEO Mori Arkin said that reaching LPLV "brings us closer to understanding its potential to address the significant unmet need in patients with Gorlin syndrome," noting the contribution of patients, investigators and clinical site teams to the study.

Market and regulatory context

Gorlin syndrome, also known as basal cell naevus syndrome, is a rare hereditary condition caused by mutations in the PTCH1 gene that predisposes patients to developing multiple BCCs, often from a young age. Because patients may develop dozens of lesions over a lifetime, a preventive topical therapy addresses a meaningfully different clinical need from the sporadic BCC treatments already on the market.

The BCC treatment landscape is competitive at the late stage. Beyond the approved oral hedgehog inhibitors, several groups have investigated immunotherapy and targeted approaches for advanced BCC, though preventive strategies in hereditary BCC remain a comparatively underexplored regulatory category. The FDA's breakthrough designation for SGT-610 signals agency agreement that the candidate addresses an unmet need, and the orphan designation brings with it seven years of market exclusivity upon approval in the United States.

Sol-Gel also has two FDA-approved dermatology products on the market, TWYNEO and EPSOLAY, providing a commercial infrastructure that could support a future launch. The company separately noted that SGT-610 may represent a longer-term opportunity in high-frequency BCC beyond the Gorlin indication, a broader market that would significantly expand the addressable patient population if a second indication were pursued post-approval.