PellePharm Completes Enrollment of Pivotal Phase 3 Clinical Trial of Patidegib Topical Gel in Patients with Gorlin Syndrome
- Trial rapidly enrolled in 8 months
- Trial size expanded to accommodate 24 more patients than planned
“Patients living with Gorlin Syndrome undergo recurring, invasive surgical treatments that result in facial scarring, potential loss of vital facial structure and function, and impact to their quality of life,” said
The randomized, 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.
“This is a very important milestone for PellePharm and the broader Gorlin Syndrome community, and we’re thrilled to be one step closer to our goal of bringing Patidegib Topical Gel to patients living with Gorlin Syndrome, a condition for which there is no
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
About Gorlin Syndrome
Gorlin Syndrome is a rare, genetic disease characterized 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.
About High Frequency Basal Cell Carcinoma (HF-BCC)
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 world leaders 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 easy-to-use 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.
About LEO Pharma
LEO Pharma A/S helps people achieve healthy skin. The company is a leader in medical dermatology with a robust R&D pipeline, a wide range of therapies and a pioneering spirit. Founded in 1908 and owned by the
BridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. BridgeBio was founded in 2015 to identify and advance transformative medicines to treat patients who suffer from Mendelian diseases, which are diseases that arise from defects in a single gene, and cancers with clear genetic drivers. BridgeBio’s pipeline of over 15 development programs includes product candidates ranging from early discovery to late-stage development.
This press release contains forward-looking statements. All statements contained herein other than statements of historical fact constitute forward-looking statements, including statements relating to expectations, plans and prospects regarding PellePharm’s clinical development plans, the clinical and therapeutic potential of Patidegib Topical Gel, the results of clinical trials, the funding by LEO Pharma of PellePharm’s pivotal Phase 3 trial of Patidegib Topical Gel in patients with Gorlin Syndrome and the timing of these events. These forward-looking statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to, PellePharm’s ability to enroll patients in and generate data from the Phase 3 trial, the benefits that will be derived from this trial, the progress and results of the trial, and PellePharm’s ability to maintain its collaboration with LEO Pharma. Moreover, PellePharm operates in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of PellePharm’s management as of the date of this release, and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. All forward-looking statements in this press release are based on information available to PellePharm as of the date hereof, and PellePharm disclaims any obligation to update these forward-looking statements.