• LEO Pharma is committed to$70 million comprised of equity financing and financial R&D support
• PellePharm and its stockholders could receive up to an additional $690 million including merger consideration, and regulatory and commercial milestone payments
• PellePharm's treatment for Gorlin Syndrome has received Orphan designation and Breakthrough Designation from the FDA

Danish dermatology specialists LEO Pharma and California rare disease pioneers PellePharm today announced a strategic development and commercialization collaboration to address unmet medical needs across various skin diseases with no approved treatments, advancing innovation and access to potential therapies for patients with life-altering conditions, such as Gorlin Syndrome and High Frequency Basal Cell Carcinoma (BCC), two distinct and rare forms of skin cancer.

“We are very excited about the partnership with PellePharm, who are pioneers in Gorlin Syndrome and experts in rare skin diseases. Supporting our ambitious 2025 strategy, it marks LEO Pharma’s entrance in rare skin diseases and it offers a unique opportunity to bring the first treatment forward to people suffering from a very severe skin disease for which there currently are no approved therapies,” said Thorsten Thormann, vice president of research at LEO Pharma.

Under the terms of the agreement, LEO Pharma has initially committed $70 million comprised of equity financing and financial R&D support to fund the global Phase 3 trial for patidegib topical gel 2% for the prevention and treatment of Gorlin Syndrome, with LEO Pharma securing an option to acquire all shares in PellePharm. PellePharm and its stockholders could receive up to an additional $690 million including merger consideration, and regulatory and commercial milestone payments. In addition, PellePharm stockholders are eligible to receive a double-digit royalty after achieving certain commercial milestones.

The agreement establishes a joint development committee with PellePharm maintaining responsibility for global development and LEO Pharma supporting in an advising role. Both companies will jointly drive commercialization planning, and Anders Kronborg, chief financial officer of LEO Pharma, will join PellePharm’s board of directors.

''Gorlin Syndrome Group welcomes the news that PellePharm and LEO Pharma are collaborating in developing patidegib topical as a treatment for BCCs. Data from PellePharm’s Phase 2 study suggest patidegib is effective in treating BCCs, with minimal side effects. We hope that by working with LEO, these benefits can be confirmed in a Phase 3 study. An effective topical medication, which can prevent and/or treat BCCs, will avert the need for the painful, disruptive and disfiguring surgeries we currently undergo,'' said Sally Webster, chairperson and Matthew Helbert, trustee of Gorlin Syndrome Group U.K.

Currently, there are no FDA-approved therapies for Gorlin Syndrome, and the standard of care for this rare disease is surgery. Patients with this lifelong, severe disease can have as many as 30 surgeries per year beginning in their mid-teens.

PellePharm’s 2% topical formulation of patidegib aims to be the first approved therapy for the prevention of Gorlin Syndrome, also known as Basal Cell Carcinoma Nevus Syndrome (BCCNS), and has shown early promise in Phase 2 clinical trials treating patients with Gorlin Syndrome and Sporadic BCC in both the U.S. and the U.K.

Patidegib topical gel has shown early promise in a Phase 2 clinical study for the mitigation of BCC tumors in Gorlin Syndrome by blocking the disease at its source within the hedgehog signaling pathway. The topical formulation of patidegib was developed to provide the efficacy previously demonstrated by oral patidegib in Phase 1 trials without the adverse systemic side effects. The gel formulation is stable at room temperature for at least two years, making it a viable potential therapy for ongoing, at-home management of Gorlin Syndrome and High Frequency BCC. PellePharm has received both Orphan Drug Designation and Breakthrough Therapy Designation for patidegib topical gel in Gorlin Syndrome from the FDA.

Approximately 10,000 people in the United States, or one in 31,000, are believed to be affected by Gorlin Syndrome.