PellePharm Initiates Pivotal Phase 3 Clinical Trial of Patidegib Topical Gel in Patients with Gorlin Syndrome

Randomized, 12-Month, Pivotal Trial Evaluating the Safety and Efficacy of Patidegib Topical Gel to Reduce the Disease Burden of Chronic BCCs in Patients with Rare Form of Skin Cancer

SAN FRANCISCO – April 1, 2019 – PellePharm, Inc., a late clinical-stage biopharmaceutical company committed to targeting rare dermatologic conditions at their source, today announced it has dosed the first patient in a pivotal Phase 3 clinical trial of Patidegib Topical Gel, 2%, vs. vehicle gel in patients with Gorlin Syndrome. The Phase 3 study is intended to assess the safety and efficacy of patidegib topical gel for the reduction of disease burden of persistently developing basal cell carcinomas (BCCs) in Gorlin Syndrome, a rare genetic disease that leads to the chronic formation of multiple BCCs. Patients with this severe skin disease, for which there is no U.S. Food and Drug Administration (FDA)-approved therapy, can suffer from tens to hundreds of BCC tumors during their lifetime and often must undergo repetitive and disfiguring surgeries to remove the tumors.

“We are pleased to have initiated our global, multicenter Phase 3 trial to further evaluate patidegib topical gel in patients with Gorlin Syndrome. Based on promising results from our Phase 2 studies, we believe that our topical gel formulation of a proprietary hedgehog inhibitor has the potential to reduce the number of surgically eligible BCCs and reduce the number of surgeries and associated facial scarring in patients with this devastating disease,” said Sanuj K. Ravindran, M.D., president and chief executive officer of PellePharm. “The initiation of this pivotal trial is an important milestone toward our goal of bringing patidegib topical gel to patients as the first FDA-approved therapy for patients with Gorlin Syndrome.”

The randomized, double-blind, vehicle-controlled Phase 3 trial is evaluating the safety and efficacy of Patidegib Topical Gel 2% applied topically, twice daily to the face over 12 months vs. vehicle gel. Approximately 150 adults with Gorlin Syndrome will be randomized 1:1 to patidegib topical gel or vehicle. The primary efficacy endpoint is the number of new surgically eligible BCCs (nSEBs) that develop over the 12-month period in the two treatment arms. Secondary endpoints include the total number of new BCCs over 12 months, percentage of study participants developing >2 facial nSEBs over 12 months; the percentage of participants developing >1 facial nSEBs over 12 months; the number of nSEBs per participant over six and nine months; and change in the Advanced Basal Cell Carcinoma Index (aBCCdex) lesion scale score. An open-label safety and tolerability extension study is planned for at least 12 months for Phase 3 study completers following the end of the study.

“This Phase 3 clinical trial is investigating the potential for topical patidegib to reduce BCC disease burden on the face. The idea that a topical gel could eliminate the need for invasive surgery in Gorlin Syndrome patients holds great promise and may dramatically improve quality of life in this population. I am very excited to be a part of this clinical trial,” said Sean R. Christensen, M.D., Ph.D., assistant professor of dermatology at Yale University School of Medicine.

“Patients with Gorlin Syndrome often must undergo repetitive surgeries to remove BCCs, which can drastically impact their lives,” said Jean Pickford, executive director of the Gorlin Syndrome Alliance. “The initiation of patient recruitment and enrollment in the Phase 3 study of patidegib topical gel is great news as it potentially brings us one step closer to having an easy-to-use chronic topical medication for patients with this life-long and life-altering disease.”

“We are thrilled the Phase 3 clinical trial of patidegib topical gel is under way and open to enrolling patients with Gorlin Syndrome,” said Sally Webster, chairperson of Gorlin Syndrome Group U.K. “New non-surgical treatment options are needed so patients with this debilitating, inherited disease can avoid the need for chronic, painful, disruptive and disfiguring surgeries. We hope the promising initial data from the Phase 2 studies of this investigational treatment will be confirmed in the Phase 3 study.”

In November 2018, PellePharm entered into a strategic development and commercialization collaboration with Danish dermatology specialists LEO Pharma to address unmet medical needs across various skin diseases with no approved treatments, including Gorlin Syndrome and High Frequency Basal Cell Carcinoma (HF-BCC), two rare forms of skin cancer. Under the agreement, LEO Pharma is providing resources to PellePharm to fund its pivotal Phase 3 trial of patidegib topical gel in patients with Gorlin Syndrome.

“We are excited to see initiation of this pivotal trial, which is a major milestone in bringing forward a new innovative treatment in a disease area where the unmet needs are so big. As a company dedicated to improving the lives of people with skin diseases, including rare diseases affecting the skin like Gorlin Syndrome, we are proud to be a partner in developing this important treatment to the patients we serve,” said Thorsten Thormann, vice president of global research at LEO Pharma.

About Patidegib

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 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 from EMA’s Committee for Orphan Medicinal Products in the EU.

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.

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 (BCNS) and Nevoid Basal Cell Carcinoma Syndrome (NBCCS).

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.

About PellePharm

Founded by world leaders in hedgehog pathway signaling, PellePharm, a BridgeBio Company, is committed to targeting rare, genetic dermatological diseases, 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 eliminates the need for regular, painful and disfiguring surgeries. Patidegib topical gel is a first-in-class topical formulation of a proprietary hedgehog inhibitor.

Forward-Looking Statements

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 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.

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