– Company Remains on Track to Initiate Phase 3 Clinical Trial in Mid-2018 –
PellePharm, a late clinical-stage biopharmaceutical company committed to targeting rare dermatologic conditions at their source, announced presentation of new data from PellePharm’s clinical and scientific research programs at the International Investigative Dermatology (IID) Meeting in Orlando, FL. The data, being presented in three posters, highlight PellePharm's lead clinical program, patidegib topical gel, in patients with basal cell carcinomas (BCCs) and epidemiological data on sporadic, high frequency BCCs.
“This year at IID, we are pleased to have the opportunity to share our latest clinical experience with patidegib topical gel and continue to be most hopeful about the promise it holds for patients with rare dermatologic diseases,” said Sanuj Ravindran, M.D., president and chief executive officer of PellePharm. “Our presentations will accentuate how PellePharm’s scientific premise and clinical progress are paving the way for us to deliver much needed hope to these patients with no preventative therapeutic options.”
IID Poster Presentations
“The epidemiology and gene sequencing data presented at IID identify patients with high-frequency BCC who, like patients with Gorlin Syndrome, develop multiple BCCs and are in need of safe and effective preventative therapies,” said Ervin Epstein, M.D., co-founder, chief medical officer and director of PellePharm. “With more than 40,000 cases of high-frequency BCC in the adult U.S. population, these results support our goal of working tirelessly to improve the quality of life for those suffering from these rare dermatological diseases and to reduce or even eliminate entirely the need for regular, painful surgeries.”
About Patidegib
Topical patidegib gel has shown early
promise in a phase 2 clinical study for the mitigation of the BCC tumors
in Gorlin Syndrome by blocking the disease at its source within the
hedgehog signaling pathway. Topical patidegib was developed to provide
the efficacy previously demonstrated by oral patidegib in phase 1 trials
without the adverse systemic side effects. Patidegib’s gel formulation
is stable at room temperature for at least two years, making it a viable
potential therapy for ongoing, at-home management of Gorlin Syndrome.
Patidegib has received both Orphan Drug Designation and Breakthrough
Designation from the FDA.
About Gorlin Syndrome
Gorlin Syndrome is a rare, genetic,
disease characterized by mutations in the tumor suppressor gene encoding
PATCHED1 (PTCH1), which acts as the primary inhibitor of the hedgehog
signaling pathway. This leads to hundreds of basal cell carcinomas,
especially on the face.
With no FDA-approved drugs available for Gorlin Syndrome, also known as Basal Cell Carcinoma Nevus Syndrome (BCCNS), the standard of care is surgery. People with severe Gorlin Syndrome may have as many as 30 surgeries per year, which can be repetitive and scarring. 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.
About High Frequency Basal Cell Carcinoma
High Frequency
Basal Cell Carcinoma, like Gorlin Syndrome, is a rare disease which is
characterized by the development of an abnormally high number of BCCs
(three or more BCCs per year). Unlike patients with Gorlin Syndrome,
patients with high frequency 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 high frequency BCC is surgery. Approximately 11,000 adults aged 18 to 64 suffer from high frequency BCC in the United States. In addition, approximately 33,000 adults over the age of 65+ are affected in the United States.
About PellePharm
Founded by world leaders in hedgehog
pathway signaling, PellePharm is a biotechnology company committed to
targeting hedgehog-driven diseases, including Gorlin Syndrome and Basal
Cell Carcinomas (BCCs), at their source. PellePharm’s mission is to
improve the quality of life for those suffering from Gorlin Syndrome and
BCCs by providing an easy-to-use topical solution that eliminates the
need for regular, painful surgeries. Topical patidegib is a
first-in-class topical gel formulation of a proprietary hedgehog
inhibitor.
About BridgeBio Pharma
BridgeBio is a clinical-stage biotech
company developing novel, genetically targeted therapies to improve the
lives of patients. The BridgeBio approach combines a traditional focus
on drug development with a unique corporate model, allowing rapid
translation of early stage science into medicines that treat disease at
its source. Founded in 2015 by a team of industry veterans, the company
has built a robust portfolio of ten transformative drugs ranging from
pre-clinical to late stage development in multiple therapeutic areas
including oncology, cardiology, dermatology and endocrinology. The
company's focus on scientific excellence and rapid execution aims to
translate today's discoveries into tomorrow's medicines.
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Lauren Barbiero
646-564-2156
lbarbiero@purecommunications.com