Research & Development
PellePharm Completes Enrollment of Pivotal Phase 3 Clinical Trial of Patidegib Topical Gel in Patients with Gorlin Syndrome
3 December 2019 - - US-based late clinical-stage biopharmaceutical company PellePharm, Inc., a BridgeBio Pharma, Inc. (NASDAQ: BBIO) company, has completed enrollment for its pivotal Phase 3 clinical trial of Patidegib Topical Gel 2% vs. vehicle gel in patients with Gorlin Syndrome, the company said.

PellePharm is committed to targeting rare forms of basal cell carcinoma.

The randomised, double-blind, vehicle-controlled Phase 3 trial is evaluating the safety and efficacy of Patidegib Topical Gel 2% applied, twice daily to the face over 12 months vs. vehicle gel.

An open-label safety and tolerability extension study is planned for at least 12 months following the end of the study for patients who complete the Phase 3 trial, and for those who completed the Phase 2 Gorlin Syndrome trial.

As of November 2018, PellePharm and LEO Pharma entered into a strategic collaboration to address the unmet medical needs for rare skin conditions, such as Gorlin Syndrome and High Frequency Basal Cell Carcinoma (HF-BCC).

LEO Pharma is providing resources to PellePharm to fund, amongst other activities, its Phase 3 trial of Patidegib Topical Gel 2% in patients with Gorlin Syndrome under the terms of the agreement.

Patidegib Topical Gel, an investigational treatment, is designed to reduce the BCC tumor burden in patients with Gorlin Syndrome and High Frequency BCC (HF-BCC) by blocking the disease at its source within the hedgehog signaling pathway.

Patidegib Topical Gel has shown early promise in a Phase 2 clinical study for the mitigation of BCC tumors in Gorlin Syndrome.

The topical formulation of Patidegib was developed with a goal of providing the clinical activity previously demonstrated by oral Patidegib in Phase 1 trials and a favorable tolerability profile without the adverse systemic side effects observed with the oral class of hedgehog inhibitors.

The topical gel formulation is stable at room temperature for at least two years, potentially making it an option for ongoing, at-home management of Gorlin Syndrome and HF-BCC.

PellePharm has received both Orphan Drug Designation and Breakthrough Therapy Designation for Patidegib Topical Gel in Gorlin Syndrome from the FDA, as well as Orphan Drug Designation in Gorlin Syndrome from EMA's Committee for Orphan Medicinal Products in the EU.

Gorlin Syndrome is a rare, genetic disease characterised by constitutional, heritable mutations in one allele of the tumor suppressor gene encoding PATCHED1 (PTCH1), which acts as the primary inhibitor of the hedgehog signaling pathway.

This leads to the formation of multiple basal cell carcinomas, often on the face.

With no FDA-approved drugs available for patients with Gorlin Syndrome, the standard of care for treating BCCs is surgery.

People with severe Gorlin Syndrome may have as many as 30 surgeries per year, which can be repetitive, scarring and disfiguring.

Approximately 10,000 people in the United States, or one in 31,000, are believed to be affected by Gorlin Syndrome. Gorlin Syndrome is known by several names, including Gorlin-Goltz Syndrome, Basal Cell Nevus Syndrome and Nevoid Basal Cell Carcinoma Syndrome (NBCCS).

High Frequency BCC, like Gorlin Syndrome, is a rare disease which is characterized by the development of an abnormally high number of BCCs.

Unlike patients with Gorlin Syndrome, patients with HF-BCC are not born with a germline PTCH1 mutation and do not suffer from the other systemic manifestations of Gorlin Syndrome. The standard of care for patients with HF-BCC is surgery.

Founded by specialists in hedgehog pathway signaling, PellePharm, a BridgeBio company, is committed to targeting rare forms of basal cell carcinoma, including Gorlin Syndrome and High Frequency Basal Cell Carcinoma (HF-BCC), at their source.

PellePharm's mission is to improve the quality of life for those suffering from Gorlin Syndrome and HF-BCC by providing an topical gel that could potentially reduce the need for regular, painful and disfiguring surgeries.

Patidegib topical gel is a first-in-class topical formulation of a proprietary hedgehog inhibitor.

LEO Pharma A/S helps people achieve healthy skin. The company is in medical dermatology with a robust R and D pipeline, a wide range of therapies and a pioneering spirit.

Founded in 1908 and owned by the LEO Foundation, LEO Pharma has devoted decades of research and development to advance the science of dermatology, setting new standards of care for people with skin conditions.

LEO Pharma is headquartered in Denmark with a global team of 6,000 people, serving 76m patients in 130 countries.

In 2018, the company generated net sales of DKK 10,410m.

BridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterised genetic diseases at their source.
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