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

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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