Sol-Gel Technologies (NASDAQ:SLGL) outlined its development and commercialization plans for SGT-610, a topical patidegib treatment being studied to reduce new basal cell carcinomas (BCCs) in patients with Gorlin syndrome, a rare inherited disorder associated with recurrent skin cancers and other manifestations.
Company management said SGT-610 targets the hedgehog signaling pathway, which is implicated in BCC development. Sol-Gel acquired the asset from PellePharm and is conducting a new Phase III study after PellePharm’s prior trial did not achieve its expected statistical significance.
Trial Design and Timing
Sol-Gel said 184 patients were screened across 41 sites, with 113 enrolled and 102 completing the trial, representing a roughly 10% dropout rate. The company cited several operational changes intended to improve diagnostic consistency and patient retention, including mandatory dermatoscopy, centralized photography review and fewer in-person visits.
Management said topline data are expected “next month,” followed by a planned new drug application submission in April. The company expects a potential approval decision in the second quarter, followed by what it described as a roughly six-month review process and a potential U.S. launch at the beginning of 2028. These timelines reflect company expectations and are subject to regulatory review.
In response to analyst questions, management said the study was initially designed for 90% power based on a post-hoc analysis suggesting an approximately 50% reduction in new BCCs. With 102 trial completers, management said its statisticians believe the study could support a 40% treatment difference and potentially a lower difference if variability is lower than in the earlier study.
Physician and Patient Perspectives
Julie Breneiser, associate director of the Gorlin Syndrome Alliance, described the substantial burden faced by patients with the disease. Breneiser, who said she and her two children have Gorlin syndrome, reported having more than 1,000 BCCs removed over her lifetime. She said currently available approaches, including surgery, topical therapies and oral hedgehog inhibitors, can be painful, disfiguring or difficult to tolerate.
“We have no FDA-approved treatments for any of the manifestations whatsoever,” Breneiser said, adding that patidegib could be “life-changing” if it reduces the need for repeated procedures.
Kavita Sarin, professor of dermatology and director of the Stanford Skin Cancer Genetics Program, said oral hedgehog inhibitors can be effective but often are not suitable for long-term use because of side effects including muscle cramps, taste changes, hair loss, weight loss and fatigue. She said patients with PTCH1 mutations are generally expected to be more responsive to smoothened-targeting hedgehog inhibitors than those with SUFU mutations, which occur downstream of the drug target.
Elizabeth Billingsley, professor of dermatology at Penn State University College of Medicine, said even a 30% reduction in new tumors could be clinically meaningful. For a patient who would otherwise require treatment for 10 new lesions annually, she said such a reduction could mean three fewer procedures, physician visits, scars and days away from work.
- Dr. Sarin said a tolerable treatment reducing new tumors by 30% could be used by many patients with moderate-to-high BCC burdens.
- Dr. Billingsley said she would favor early treatment in patients diagnosed with Gorlin syndrome, given the disease’s lifelong nature.
- Breneiser said patients would likely be willing to use a preventive topical treatment daily if it reduced surgical burden.
Commercial Strategy and Market Opportunity
Sol-Gel estimates there are approximately 11,000 Gorlin syndrome patients in the United States. After excluding patients without significant BCC burdens, pediatric patients and an assumed non-adherent population, the company estimated approximately 6,100 eligible patients. Management’s forecast assumes 65% penetration, or about 4,000 treated patients at peak, supporting a projected $860 million U.S. market opportunity.
The company said it plans to commercialize SGT-610 independently in the United States rather than license the product, citing a concentrated prescriber base of roughly 200 centers of excellence and the potential for a relatively small sales force. It expects to seek commercial partners outside the U.S.
Management also identified potential future opportunities in BCCs occurring beyond the face, high-frequency BCC populations and use of the product to reduce tumor size before surgery. Sol-Gel said it plans to begin a study of patidegib as neoadjuvant therapy for BCCs in sensitive facial areas.
Ervin Epstein, co-founder and consultant to PellePharm, said the broader aim of hedgehog inhibition is to intercept BCC development before lesions become large enough to require treatment. “The earlier people use it, the more likely it is to be successful,” he said.
About Sol-Gel Technologies (NASDAQ:SLGL)
Sol-Gel Technologies Ltd. (NASDAQ: SLGL) is an Israeli specialty pharmaceutical company focused on developing and commercializing topical dermatological treatments. The company uses proprietary silica-based microsponge technology designed to encapsulate and gradually release active pharmaceutical ingredients in the skin.
Its marketed products include TWYNEO, a topical cream combining tretinoin and benzoyl peroxide for the treatment of acne vulgaris, and EPSOLAY, a topical benzoyl peroxide cream for the treatment of inflammatory lesions associated with rosacea.
