Company on Track for a Phase 3 Clinical Trial in 2018 

MENLO PARK, CA. – PellePharm, a clinical-stage biopharmaceutical company committed to targeting rare genetic dermatological conditions at the source, today announced that the U.S. Food and Drug Administration (FDA) has granted both Breakthrough Therapy Designation and Orphan Drug Designation to topical patidegib for reduction of the life-long serious clinical morbidity and disease burden of persistently developing basal cell carcinomas (BCCs) in patients with Basal Cell Carcinoma Nevus Syndrome (BCCNS), a rare
genetic disease also known as Gorlin Syndrome.

“These two significant FDA designations mark important steps forward in our development of topical patidegib as a drug with substantial potential to reduce the BCC tumor burden in Gorlin Syndrome patients. From our co-discovery of the underlying genetic defect, through over two decades of preclinical research with hedgehog inhibitors, and our recent clinical trials, our team
at PellePharm is deeply committed to delivering a safe and effective therapy for people living with Gorlin Syndrome,” said Ervin Epstein, MD, cofounder and chief medical officer of PellePharm. “We are eager to continue our development program and work closely with the FDA and other regulatory authorities to deliver this potential first-in-class therapy to patients as quickly as possible.”

The FDA granted PellePharm Breakthrough Therapy Designation based on results from its Phase 2 trial assessing the safety and efficacy of topical patidegib in patients with Gorlin Syndrome. Topline data from this study were announced in July 2017. Breakthrough Therapy Designation is designed to expedite the development and review of drugs that are intended to treat a serious condition, where preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy.

The FDA granted Orphan Drug Designation based on the relatively low prevalence of Gorlin Syndrome. The FDA’s Orphan Drug Designation program is designed to incentivize and facilitate the development of drugs for rare diseases that affect fewer than 200,000 people in the U.S. Approximately 10,000 people in the United States, or one in 31,000, are believed to be
affected by Gorlin Syndrome.

“We are pleased that the FDA recognizes the importance of accelerating the development of topical patidegib for people affected by BCCNS (Gorlin Syndrome),” said Jean Pickford, executive director of the Basal Cell Carcinoma Nevus Syndrome Life Support Network. “People living with BCCNS need additional therapeutic options to help lessen the burden of disease.” Currently, there are no FDA-approved therapies for Gorlin Syndrome, and the standard of care for this rare disease is surgery. Patients with severe disease have as many as 30 surgeries per year.

PellePharm intends to initiate a Phase 3 clinical trial for patidegib in Gorlin Syndrome in 2018

About Patidegib

Topical patidegib gel has shown early promise in a Phase 2 clinical study 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,
but without the adverse systemic side effects of oral hedgehog inhibitors. 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.
Topical patidegib currently is being studied in a United States-based Phase 2 clinical trial for the
treatment of sporadic BCCs.

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 and
sun-exposed areas.

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 BCCNS,
Gorlin-Goltz Syndrome, Basal Cell Nevus Syndrome, or Nevoid Basal Cell Carcinoma
Syndrome.

About PellePharm

Founded by world leaders in hedgehog signaling, PellePharm, a BridgeBio company, is
committed to targeting rare genetic dermatological conditions, including Gorlin Syndrome and
Basal Cell Carcinomas (BCCs), at the source. PellePharm is committed to improving 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.

Media Contact
Kelly Lindenboom
Pure Communications
(617) 763-7281
[email protected] 

PellePharm Announces Topline Results from Phase 2 Study of Topical Patidegib in Gorlin Syndrome Basal Cell Carcinomas and Third Closing of a $20 Million Series B Financing

Company on Track for a Phase 3 Clinical Trial in 1H18 

MENLO PARK, CA. – PellePharm, a clinical-stage biopharmaceutical company committed to targeting hedgehog-driven disorders at the source, today announced topline data from its phase 2 trial evaluating the safety and efficacy of topical patidegib in patients with Gorlin Syndrome, a rare genetic disease that causes patients to develop multiple basal cell carcinomas (BCCs). PellePharm intends to initiate discussions with the FDA, with the goal of initiating a phase 3 clinical trial for patidegib in the first half of 2018. 

The double-blinded, placebo-controlled, randomized, three-arm, UK-based trial evaluated a patidegib topical therapy. Seventeen patients participated in the trial and were self-treated with topical patidegib, a novel hedgehog inhibitor, for six months. There was a statistically significant difference in complete response between treatment and vehicle groups in the per protocol analysis, with complete response demonstrated in 12 tumors across both treatment arms, compared with zero tumors in the vehicle arm. The complete responses were correlated with a meaningful reduction of a biomarker for hedgehog signaling (GLI1). Partial responses were also demonstrated in both treatment groups. Importantly, there were zero detectable levels of patidegib in plasma after topical application of patidegib gel, and patients treated with this topical gel had none of the devastating class-specific systemic side effects observed with oral hedgehog inhibitors. 

“The search for safe and effective chronic mitigation of the tumor burden for patients suffering from Gorlin Syndrome has been my life’s work, and I am encouraged by the safety and efficacy signals indicated by this trial,” said Ervin Epstein, MD, co-founder, president and director of PellePharm. “We very much look forward to further development of this compound, which should reduce greatly the patient need for surgeries by blocking the molecular driver of BCCs.” 

“Having worked closely with those affected by Basal Cell Carcinoma Nevus Syndrome, also known as Gorlin Syndrome, on a daily basis for more than 15 years, I know first-hand how devastating this condition is to quality of life,” said Kristi Burr, founder of the Basal Cell Carcinoma Nevus Syndrome Life Support Network and patient advocate. “PellePharm’s trial represents important progress, bringing hope to people affected by the disease who currently have extremely limited options for addressing their invasive BCCs throughout their lifetime.” 

Currently, there are no FDA-approved drugs to treat Gorlin Syndrome and the standard of care for this rare disease is surgery. 

Additional Corporate Updates

PellePharm confirmed that it has raised an aggregate of $23.5 million to date, with a third closing of a $20 million Series B financing round led by BridgeBio. A prior closing was announced in December 2016. BridgeBio and other current investors are expected to participate in a planned Series C financing to support the company’s Phase 3 clinical trial for patidegib through NDA filing. 

“PellePharm continues to demonstrate patidegib’s potential to expand the treatment landscape for Gorlin Syndrome patients, and we are pleased to see the progress this development candidate is making in the clinic,” said Frank McCormick, Ph.D., co-founder of BridgeBio and former president of the American Association for Cancer Research (AACR). “As PellePharm begins discussions with the FDA about initiating a pivotal phase 3 trial, we believe that this financing will help propel the company through this important step forward for both patients and investors.” 

PellePharm also announced the completion of enrollment for its second phase 2 trial, a double-blinded, dose-escalating, placebo-controlled proof-of-concept clinical trial evaluating topical patidegib gel in patients with sporadic BCCs. Topline data from this trial is expected to be reported in Q4 2017. 

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. 

About Basal Cell Carcinomas (BCCs) and Gorlin Syndrome

Basal cell carcinomas (BCCs) are a type of skin cancer that begins when skin cells develop mutations in their DNA, usually due to UV radiation (sunlight, tanning lamps). They commonly occur in sun-exposed areas like the face and neck and generally in people of European descent with lighter skin and in the elderly and in people with chronic sun exposure. There are more than four million BCCs diagnosed in the United States each year. BCCs are usually treated with surgery, which can leave disfiguring scars. 

Gorlin Syndrome, also known as Basal Cell Carcinoma Nevus Syndrome, nevoid basal cell carcinoma syndrome, Gorlin-Goltz syndrome, is a rare genetic disease in which patients develop many BCCs. Patients with Gorlin Syndrome have heritable 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 BCCs, especially on the face and sun-exposed areas. The standard of care is surgery, as there are no FDA-approved drugs for Gorlin Syndrome. Individuals who have severe Gorlin Syndrome have as many as 30 surgeries per year, many of which can be scarring. 

About PellePharm

PellePharm is developing patidegib, a novel hedgehog pathway inhibitor, to mitigate the tumor burden in patients with basal cell carcinomas (BCCs), including those in Gorlin Syndrome, a devastating rare genetic skin disease. The company is focused on developing and commercializing this treatment to reduce the burden of disease and improve quality of life by potentially reducing invasive and painful surgeries and the scarring that often accompanies them. Topical patidegib is a first-in-class topical gel formulation of a proprietary hedgehog inhibitor exclusively licensed from Infinity Pharmaceuticals. Patidegib is currently being studied in a phase 2 clinical trial for the treatment of sporadic BCCs. 

About BridgeBio Pharma

BridgeBio Pharma develops medicines for genetic diseases with significant unmet need. The company is primarily focused on pre-commercial products, utilizing its advantages in sourcing and operating to find and build value at each stage of development. Founded in 2015, and backed by KKR and Perceptive Life Sciences, BridgeBio Pharma is actively developing multiple clinical and pre-clinical programs.

Media Contact
Kelly Lindenboom
Pure Communications
(617) 763-7281
[email protected] 

PellePharm Launches with Financing from BridgeBio Pharma to Develop Topical Therapy for Basal Cell Carcinomas and Gorlin Syndrome

Enrollment in Key Phase 2 Trial Recently Completed; Data Expected by Second Quarter of 2017

MENLO PARK, CA – PellePharm, a clinical-stage biopharmaceutical company committed to developing patidegib, a topical hedgehog inhibitor to treat basal cell carcinomas (BCCs), including those in Gorlin Syndrome, a devastating orphan disease, announced its launch with financing from BridgeBio Pharma. This financing supports the development of topical patidegib through the completion of two phase 2 clinical trials that are underway, including a clinical trial in Gorlin Syndrome that just completed enrollment. In addition, the company has a drawable pool of capital from BridgeBio Pharma that enables it to finance future clinical trials through registration. 

PellePharm was founded by a team of globally recognized experts in dermatology, oncology and hedgehog signaling and is managed by a team of industry experts. The company is dedicated to finding an effective therapy to manage the extensive tumor burden in Gorlin Syndrome, a rare genetic disease that causes patients to develop multiple BCCs. Gorlin Syndrome affects about 10,000 people in the United States and 15,000 people in the European Union. The disease results from a mutation in a tumor-suppressor gene, which acts as the primary inhibitor of the hedgehog signaling pathway. PellePharm’s founders developed a topical gel formulation of a proprietary hedgehog inhibitor that it exclusively licensed from Infinity Pharmaceuticals to help mitigate certain adverse events observed with oral hedgehog inhibitors.

“It made sense that a potent inhibitor of the hedgehog pathway should provide a therapeutic benefit for patients suffering from Gorlin Syndrome, but we needed an approach that would allow us to target the disease at its source without eliciting harmful toxicity reactions,” said Ervin Epstein, M.D., co-founder of PellePharm. “We are hopeful that patidegib will provide the balance of targeted treatment without the adverse events associated with oral formulations.”

PellePharm’s approach attracted the attention of industry veterans Mark de Souza, Ph.D. and Karl Beutner, M.D., Ph.D. who joined the team. “It’s not often that you find a program as attractive as PellePharm’s patidegib,” said de Souza, executive chairman of PellePharm. “This investigational treatment has the potential to address a very high unmet need among patients with Gorlin Syndrome, whose current standard of care is to undergo multiple surgeries each year to manage their symptoms. We know that hedgehog pathway inhibitors have therapeutic potential for this condition, and we believe PellePharm’s topical formulation may offer a truly effective treatment option for these patients.”

PellePharm is currently conducting two double-blinded, placebo-controlled, randomized, phase 2 clinical trials that are evaluating the safety, tolerability and effect of topical patidegib on both pre-existing tumors and the frequency of the development of new tumors. A U.K.-based study is evaluating 18 patients with Gorlin syndrome to determine whether the product shrinks the tumors present at the beginning of the study and reduces the number of new tumors that develop during the trial. A U.S.-based study is evaluating 36 patients with sporadic BCCs to determine whether tumor diameter decreases after 12 weeks of treatment. Both studies also will evaluate safety and tolerability, as well as a biomarker (GLI-1) of hedgehog signaling. Enrollment in the U.K. trial is complete and topline data is expected in the second quarter of 2017.

“PellePharm is really at the intersection of precision oncology and monogenic dermatology - these are the types of assets we look for at BridgeBio Pharma,” said Frank McCormick, investment committee member of BridgeBio Pharma and board member of PellePharm. “Gorlin Syndrome is a well-researched disease that can be treated directly at its source. We have had the privilege of knowing the founding and operating team of PellePharm for years and look forward to working with them to bring patidegib to patients in need.”

Founders and Scientific Advisory Board Members

PellePharm was founded by a group of globally recognized experts in dermatology, oncology and hedgehog signaling who continue to lend expertise and counsel to the PellePharm management team. Founders and scientific advisory board members include:

 Philip Beachy, Ph.D., professor of biochemistry and developmental biology, Stanford University

 Ervin Epstein, M.D., senior scientist, Children’s Hospital of Oakland Research Institute

 Jean Tang, M.D., Ph.D., associate professor of dermatology, Stanford University

About Basal Cell Carcinomas (BCCs) and Gorlin Syndrome

Basal cell carcinomas (BCCs) are a type of skin cancer that begins when one of the skin cells spontaneously develops a mutation in its DNA, usually due to UV radiation (sunlight, tanning lamps). They commonly occur in sun-exposed areas like the face and neck and generally in people of European descent with lighter skin and in the elderly and in people with chronic sun exposure. There are more than four million BCCs diagnosed in the United States each year. BCCs are usually treated with surgery, which can leave disfiguring scars.

Gorlin Syndrome, also known as nevoid basal cell carcinoma syndrome, is a rare genetic disease where patients develop many BCCs. Patients with Gorlin Syndrome have heritable 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 BCCs, especially on the face and sun-exposed areas. The standard of care is surgery, as there are no FDA-approved drugs for Gorlin Syndrome. Individuals who have severe Gorlin Syndrome have as many as 30 surgeries per year, many of which can be scarring. 

About PellePharm

PellePharm is developing patidegib, a novel hedgehog pathway inhibitor, to treat basal cell carcinomas (BCCs), including those in Gorlin Syndrome, a devastating rare genetic skin disease. The company is focused on developing and commercializing this treatment to reduce the burden of disease and improve quality of life by potentially reducing invasive and painful surgeries and the scarring that often accompanies them. Topical patidegib is a first-in-class topical gel formulation of a proprietary hedgehog inhibitor exclusively licensed from Infinity Pharmaceuticals. Patidegib is currently being studied in two phase 2 clinical trials for the treatment of BCCs and Gorlin Syndrome. 

About BridgeBio Pharma

BridgeBio Pharma develops medicines for genetic diseases with significant unmet need. The company is primarily focused on pre-commercial products, utilizing its advantages in sourcing and operating to find and build value at each stage of development. Founded in 2015, and backed by KKR and Perceptive Life Sciences, BridgeBio Pharma is actively developing multiple clinical and pre-clinical programs.

Media Contact
Katie Engleman
Pure Communications
[email protected]
910-509-3977