PellePharm’s Phase III Skin Disease Trial Is Back on Track Following Agreement With LEO Pharma

Denmark-based LEO Pharma and California rare disease drugmaker Pellepharm entered into a strategic collaboration worth $760 million to address unmet needs in multiple orphan skin diseases, such as Gorlin syndrome and high-frequency basal cell carcinomas (BCCs), two rare forms of skin cancer.

LEO Pharma will provide an infusion of cash to help support development of the global Phase III development trial for patidegib topical gel 2% for the prevention and treatment of Gorlin syndrome. After snagging Breakthrough Therapy Designation and Orphan Drug Designation from the U.S. Food and Drug Administration for patidegib, the Bay Area’s PellePharm had intended to launch Phase III trials this year, but was unable to do so. Now, with the support of LEO Pharma, PellePharm anticipates launching the Phase III trial in the first quarter of 2019.

Patidegib was awarded the FDA designations after positive Phase II data demonstrated the safety and efficacy of patidegib in patients with Gorlin syndrome, a rare genetic disease that causes patients to develop multiple basal cell carcinomas (BCCs). The mid-stage data showed the gel blocked the mitigation of disease-associated tumors at its source in the hedgehog signaling pathway. The gel formulation was developed as a safer alternative to an oral treatment. It is stable at room temperature and maintains its efficacy for more than two years, which PellePharm said makes it a potential therapy for at-home management of Gorlin syndrome and high-frequency BCC.

Currently, there are no therapies for Gorlin syndrome that have been approved by the FDA. The standard of care for this rare disease is surgery and many patients can have more than two dozen disease-related surgeries within a year to address the multiple basal cell carcinomas that often appear on a patient’s face. There are approximately 10,000 people in the United States who are believed to be affected by Gorlin syndrome. PellePharm has hopes that its topical treatment will become the first approved medication in the United States for Gorlin syndrome, which is also known as basal cell carcinoma nevus syndrome (BCCNS).

The treatment is also being aimed at high-frequency BCC, which, like Gorlin syndrome, is a rare disease characterized by the development of an abnormally high number of basal cell carcinomas. The standard of care for this disease is also surgery.

Under 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 III trial for patidegib topical gel 2%. As part of the deal, LEO Pharma secured 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.

Sanuj Ravindran, president and chief executive officer of PellePharm, said his company is founded on a commitment to target rare dermatological diseases at the source. LEO Pharma, he said, is a global leader in medical dermatology and made a “great fit” as development and commercialization partner.

This collaboration puts us on track to commence our pivotal Phase 3 Gorlin syndrome trial in early 2019. Then after the potential merger, we look forward to working with LEO Pharma to address other rare skin diseases with unmet needs,” Ravindran said.

Thorsten Thormann, head of research and development at LEO Pharma, expressed excitement about working with PellePharm, which he called a pioneer in Gorlin syndrome, as well as other 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,” Thormann said in a statement.