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SAN FRANCISCO, June 13, 2018PellePharm, a late clinical-stage biopharmaceutical company committed to targeting rare dermatologic conditions at their source, today announced that the company is scheduled to participate in the Cantor Fitzgerald Dermatology and Aesthetics Summit, Tuesday, June 19, at 2:00 p.m. ET at the Westin Grand Central Hotel in New York, New York.

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.

Contacts

W2Opure
Lauren Barbiero, 646-564-2156
lbarbiero@purecommunications.com

SAN FRANCISCO, May 21, 2018 — PellePharm, a late clinical-stage biopharmaceutical company committed to targeting rare dermatologic conditions at their source, announced that chief executive officer and president of PellePharm, Sanuj K. Ravindran, M.D., will present a company overview at the UBS Global Healthcare Conference: Tuesday, May 22, at 2:30 p.m. ET at the Grand Hyatt New York in New York, NY.

A live webcast of the presentation can be accessed on the company’s website at www.pellepharm.com. A replay of the webcast will be archived on the PellePharm website for at least two weeks following the presentation.

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. 

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Media Contact:

Lauren Barbiero

W2Opure

(646) 564-2156

lbarbiero@purecommunications.com

Company Remains on Track to Initiate Phase 3 Clinical Trial in Mid-2018

SAN FRANCISCO, CA., May 16, 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

  • Anti-BCC efficacy of a topical Hedgehog inhibitor (HHi) without the systemic HHi adverse effects in BCNS patients in a Phase 2 randomized controlled trial
    Poster #139 — Thursday, May 17, 2018 at 11:45 a.m. – 1:45 p.m. ET

This study highlights the recent results from PellePharm’s Phase 2 trial treating patients with Gorlin Syndrome. The company first shared topline data from a Phase 2 study conducted in the UK in July 2017 that showed early promise for topical patidegib for the mitigation of BCC tumors in Gorlin Syndrome. The data being presented at IID include a more recent analysis of that data that shows that patidegib topical gel treated patients developed three-fold fewer new facial BCCs compared to patients treated with vehicle gel for six months (0.4 vs. 1.4 new tumors). Only 16% of patidegib topical gel treated patients developed a new facial BCC compared with 60% in vehicle. PellePharm intends to begin enrollment for a Phase 3 trial starting in July 2018 based on these study results.

  • Prevalence and risk factors for high-frequency basal cell carcinoma in a large commercially insured population in the US

Poster #335 — Thursday, May 17, 2018 at 11:45 a.m. – 1:45 p.m. ET

This study analyzed insurance claims data from 14 million U.S. adults aged 18 to 64 and found that 11,000 patients suffer from high-frequency BCCs. High-frequency BCC patients are a rare subset of patients with sporadic BCCs, who on average experience more than three BCCs annually.

  • Frequent basal cell cancer development is a clinical marker for inherited cancer susceptibility
    Poster #164 — Wednesday, May 16, 2018 at 12:00 p.m. – 2 p.m. ET

This study, conducted at Stanford University Medical Center, sequenced 29 cancer genes in 60 high-frequency BCC patients, defined in the study as the top 5% of patients with any BCC. Approximately 20% of sequenced patients had mutations in DNA repair genes.

“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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Media Contact:

Lauren Barbiero

W2Opure

(646) 564-2156

lbarbiero@purecommunications.com

 

Expanded PellePharm leadership team strengthens operational and regulatory capabilities

PellePharm on track for a Phase 3 clinical trial in mid-2018

MENLO PARK, Calif., January 4, 2017 – PellePharm, a clinical-stage biopharmaceutical company committed to targeting rare genetic dermatological conditions at the source, today announced the appointment of Sanuj K. Ravindran, M.D., to the position of president and chief executive officer. In parallel, Dr. Ravindran will join BridgeBio Pharma, PellePharm’s lead investor, as CEO-in-Residence, to advance its broader orphan dermatology portfolio.

“Dr. Ravindran’s biopharma industry experience and rare disease expertise will offer enormous strategic benefit to PellePharm as we move into our next stage of development,” said Ervin Epstein, M.D., chief medical officer and co-founder of PellePharm. “Having been successful with the management and growth of multiple biopharma companies, Dr. Ravindran brings the right experience to PellePharm so that we may offer topical patidegib to patients as swiftly as possible.”

Dr. Ravindran brings more than 15 years of strategic and operational biopharma experience to PellePharm. Most recently, he was chief business officer at aTyr Pharma (“LIFE”), a clinical stage rare disease-focused biotechnology company, where he led corporate and financial strategy, business development, and investor relations. Prior to that, Dr. Ravindran was senior vice president of corporate development for The Medicines Company (“MDCO”), where he worked to execute multiple transactions totaling more than $2 billion in potential aggregate value. Previously a practicing physician, Dr. Ravindran began his industry career as a venture capitalist for 10 years with Burrill & Company, Radius Ventures, and Asian Healthcare Fund. Dr. Ravindran is trained in Internal Medicine and completed his residency training at Thomas Jefferson University Hospital. Dr. Ravindran received his B.A. from Northwestern University, his M.D. from Jefferson Medical College and his MBA from the Kellogg School of Management.

“I am thrilled to join PellePharm at such an important juncture, as the Company prepares to advance topical patidegib one step closer to patients with Gorlin Syndrome. With PellePharm’s scientific premise, clinical progress, and recently strengthened leadership team, the company is well on its way to meeting its mission of delivering therapies for rare genetic dermatological conditions,” said Dr. Ravindran.

PellePharm today also announced that it has expanded its executive team to enhance regulatory and operational capabilities. Alix Alderman is now vice president of regulatory affairs at PellePharm, and Gerd Kochendoerfer, Ph.D., is vice president of technical operations and program management. Both Ms. Alderman and Dr. Kochendoerfer bring many years of experience in drug development, quality management and global regulatory affairs.

“We are pleased to welcome Dr. Ravindran, Dr. Kochendoerfer and Ms. Alderman,” said Neil Kumar, CEO and co-founder of BridgeBio Pharma. “At this inflection point, having the right team in place, with the collective experience this group brings, enables PellePharm to more ably achieve its goal of helping patients with serious unmet dermatologic conditions.”

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 basal cell carcinomas (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 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
Katie Engleman
Pure Communications
(910) 509-3977
kengleman@w2ogroup.com
###

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.

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.

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.