Solgel reports today on the purchase of a patidegib product for local administration, for the treatment of Gorlin syndrome from PellePharm. Gorlin syndrome is a rare disease with no treatments approved by the FDA or the EMA. Sol-Gel is expanding its product pipeline with this new chemical product, designated compound SGT-610, which has the potential to be the first treatment for Gorelin syndrome.
patidegib has orphan drug status from the FDA and EMA, as well as breakthrough product status from the FDA. The FDA and EMA have agreed that approval may be granted on the basis of a single pivotal phase 3 trial.
Gorlin syndrome affects approximately 1 in 31 thousand people, and is an autosomal dominant genetic disorder caused mainly by the inheritance of one defective copy of a tumor suppressor gene – PTCH1. The syndrome is also called nevoid BCC since almost 90% of those with the syndrome develop new lesions, ranging from a single number to several thousand during the patient’s lifetime. Painful surgical excision is the prevailing treatment to remove the lesions. However, when the number of lesions continues to develop, repeated surgical intervention becomes impossible and therefore an important consideration in the treatment of Guerlin syndrome is the prevention of the development of new lesions. SGT-610 is designed to prevent the formation of new lesions in adults with Gorlin syndrome, without the side effects associated with oral administration treatments.
The CEO of Sol-Gel Dr. Alon Seri-Levi stated that “rare diseases represent a treatment category with high profitability rates, and we estimate that SGT-610, if approved by the FDA, has the potential to yield, at the peak , annual net sales of over $300 million. We believe the risk/reward equation of this transaction is very good for Sol-Gel and its shareholders. We conducted extensive due diligence on patidegib’s early development plans and consulted with expert clinicians to design a trial A new Phase 3, which we believe can overcome the shortcomings of an early Phase 3 trial, in order to generate the safety and efficacy data necessary to support FDA approval. We have a strong track record of conducting clinical trials of topical dermatological drugs and we look forward to leveraging this experience to advance SGT-610 to FDA approval, With the aim of providing Gorlin syndrome patients with the first treatment, which can prevent the creation of new carcinomas.”
Julie Bernizer, senior director of the Gorlin Syndrome Alliance, stated that “cellular carcinomas are the most burdensome manifestation of Gorlin syndrome patients. The volume of Gorlin syndrome carcinomas and the accompanying need for repeat treatments lead to permanent and significant scarring, anxiety and loss of time from work, school or daily activities others”.
The planned phase 3 trial of SGT-610 will include well-defined changes compared to a previous phase 3 trial, in which the patidegib arm was found to be as well tolerated as the placebo arm, and no serious side effects were observed as with oral administration. These changes will include the selection of patients carrying the PTCH1 mutation (as opposed to the previous trial which included symptomatic patients without testing regarding the mutation carriers), as well as a requirement for a higher minimum number of lesions at the beginning of the trial compared to the previous trial. A phase 3 sol-gel trial is scheduled to begin in the second half of 2023 with results expected by the end of 2025.
Sol-Gel will pay an advance of $4.7 million to PellePharm. In addition, based on the predicted market potential, the company will be required to pay for milestones of overall development and receiving an NDA amounting to up to $6 million, and up to $64 million in commercial milestones alongside single-digit royalties.
Dr. Sri-Levy continued, “SGT-610 is an orphan drug candidate and therefore we are prioritizing the development of SGT-610, alongside the development of SGT-210 for the treatment of rare cutaneous keratoderma and other hyperproliferative skin disorders, which represent opportunities with high profitability rates in the dermatology market , and suspending the development of SGT-310 and SGT-510 for the treatment of psoriasis, a market that has become more crowded and competitive. For these projects we will focus on granting licenses for their foreign development.”
Sol-Gel Chairman of the Board Murray Arkin stated that “Given the team’s great expertise in drug development and collaboration, as well as the certainty gained from connections with the scientific and patient communities, patidegib represents an ideal asset in Sol-Gel’s product pipeline in order to maximize the value to the shareholders”.