News

SAN FRANCISCO–PellePharm, Inc., a BridgeBio Pharma, Inc. company, today announced the presentation of various data highlighting product candidate patidegib topical gel for adults with Gorlin syndrome and high-frequency basal cell carcinoma (HF-BCC). The presentations will be made during the 29th Congress of the European Academy of Dermatology and Venereology (EADV), to be held virtually October 29 – 31, 2020.

SAN FRANCISCO – January 8, 2020 – PellePharm, Inc., a BridgeBio Pharma, Inc. (Nasdaq: BBIO) company, today announced it has dosed the first two participants in a Phase 2 clinical trial of Patidegib Topical Gel, 2%, vs. vehicle gel for people with non-Gorlin High-Frequency Basal Cell Carcinoma (HF-BCC). HF-BCC is a rare disease that causes a higher than average number of BCCs to develop, specifically in the facial area. PellePharm is a late clinical-stage biopharmaceutical company committed to targeting rare forms of basal cell carcinoma (BCC).

“There are approximately 35,000 people with HF-BCC in the United States. Their quality of life is significantly altered due to the multiple, invasive surgeries they must undergo during their treatment process,” said Sanuj K. Ravindran, M.D., president and chief executive officer of PellePharm. “Our goal is to provide people living with HF-BCC better, non-surgical options, and we are pleased to have initiated our multicenter Phase 2 trial to further evaluate Patidegib Topical Gel.”

The randomized, double-blinded, stratified, vehicle-controlled Phase 2 trial is evaluating the safety and efficacy of Patidegib Topical Gel 2% applied twice daily to the face over nine months vs. vehicle gel. The primary endpoint of the study is the number of surgically eligible basal cell carcinoma (nSEB) that develop on the face of participants over the nine-month period. The primary endpoint will be assessed by imaging and tracking of BCCs consistently throughout the study in order to identify nSEBs, consistent with the methods employed in the ongoing Phase 3 study of Patidegib topical gel for people living with Gorlin Syndrome. Approximately 40 participants will be enrolled in the Phase 2 trial.

“People with non-Gorlin HF-BCC are phenotypically similar to people with Gorlin Syndrome with respect to their BCCs, but are not born with a germline PTCH1 mutation. Those with HF-BCC are faced with the challenge of frequent BCC surgeries, which can be debilitating, painful and disfiguring, particularly to the face,” said Srikanth Pendyala, M.D., vice president of clinical development at PellePharm. “Due to the success we have found with enrolling our Gorlin Syndrome Phase 3 pivotal trial, we are thrilled about this important Phase 2 study milestone, and hope that by initiating this trial, we are closer to being able to mitigate the significant burden of frequent surgeries for those living with HF-BCC.”

PellePharm recently completed enrollment for its Phase 3 clinical trial of Patidegib Topical Gel 2% for people living with Gorlin Syndrome. PellePharm entered into a strategic collaboration with LEO Pharma in November 2018, which includes an option for LEO Pharma to acquire PellePharm.

About Patidegib

Patidegib Topical Gel, an investigational treatment, is designed to reduce the BCC tumor burden in people living 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 topical 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 in Gorlin Syndrome 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 (BCCs), often on the face.

With no FDA-approved drugs available for people living 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)

HF-BCC, like Gorlin Syndrome, is a rare disease which is characterized by the development of an abnormally high number of basal cell carcinomas (BCCs). Unlike people with Gorlin Syndrome, people with HF-BCC are not born with a germline PTCH1 mutation and do not suffer from the other systemic manifestations of Gorlin Syndrome. The current standard of care for people living with HF-BCC is BCC surgery.

About PellePharm

Founded by world leaders in hedgehog pathway signaling, PellePharm, a BridgeBio company, is committed to targeting rare forms of basal cell carcinoma, 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 could potentially reduce the need for regular, painful and disfiguring surgeries. Patidegib topical gel is a topical formulation of a proprietary hedgehog inhibitor.

About LEO Pharma

LEO Pharma A/S helps people achieve healthy skin. The company is a leader in medical dermatology with a robust R&D pipeline, a wide range of therapies and a pioneering spirit. Founded in 1908 and owned by the LEO Foundation, LEO Pharma has devoted decades of research and development to advance the science of dermatology, setting new standards of care for people with skin conditions. LEO Pharma is headquartered in Denmark with a global team of 6,000 people, serving 76 million patients in 130 countries. In 2018, the company generated net sales of DKK 10,410 million.

About BridgeBio Pharma
BridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. BridgeBio was founded in 2015 to identify and advance transformative medicines to treat patients who suffer from Mendelian diseases, which are diseases that arise from defects in a single gene, and cancers with clear genetic drivers. BridgeBio’s pipeline of over 15 development programs includes product candidates ranging from early discovery to late-stage development. 

BridgeBio Pharma Forward-Looking Statements

This press release contains forward-looking statements. Statements we make in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the “Securities Act”), and Section 21E of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), which are usually identified by the use of words such as “anticipates,” “believes,” “estimates,” “expects,” “intends,” “may,” “plans,” “projects,” “seeks,” “should,” “will,” and variations of such words or similar expressions.  We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act and are making this statement for purposes of complying with those safe harbor provisions. These forward-looking statements, including statements relating to expectations, plans and prospects regarding PellePharm’s clinical development plans, clinical trial designs, 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 people living with Gorlin Syndrome, PellePharm’s Phase 2 clinical trial of Patidegib Topical Gel 2% in people living with High Frequency Basal Cell Carcinoma, enrollment of PellePharm’s Phase 2 clinical trial, and the timing of these events, reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations, strategies and prospects as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved.  Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to, PellePharm’s ability to enroll participants in and generate data from the ongoing Phase 2 and Phase 3 trials, the benefits that will be derived from these trials, the progress and results of the trials, PellePharm’s ability to advance Patidegib Topical Gel in clinical development in accordance with its plans, the timing of these events, and PellePharm’s ability to maintain its collaboration with LEO Pharma, as well as those risks set forth in the Risk Factors section of BridgeBio Pharma Inc.’s most recent Quarterly Report on Form 10-Q and our other SEC filings. 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. Except as required by applicable law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

# # #

Media Contact:

Lauren Barbiero

W2O pure

(646) 564-2156

[email protected]

– Trial rapidly enrolled in 8 months

– Trial size expanded to accommodate 24 more patients than planned

SAN FRANCISCO – December 2, 2019 – PellePharm, Inc., a BridgeBio Pharma, Inc. (Nasdaq: BBIO) company, today announced the completion of enrollment for its pivotal Phase 3 clinical trial of Patidegib Topical Gel 2% vs. vehicle gel in patients with Gorlin Syndrome. PellePharm is a late clinical-stage biopharmaceutical company committed to targeting rare forms of basal cell carcinoma.

“Patients living with Gorlin Syndrome undergo recurring, invasive surgical treatments that result in facial scarring, potential loss of vital facial structure and function, and impact to their quality of life,” said Sanuj K. Ravindran, M.D., president and chief executive officer of PellePharm. “Significant enthusiasm for the trial allowed us to enroll the study quickly, but only after enrolling approximately 16% more patients than originally planned to accommodate the incredible interest from patients. We continue to be encouraged about Patidegib Topical Gel’s potential and are grateful for the overwhelming support of the Gorlin Syndrome community worldwide, including the researchers, clinicians, patients and caregivers working diligently to bring promise to this devastating disease.”

The randomized, double-blind, vehicle-controlled Phase 3 trial is evaluating the safety and efficacy of Patidegib Topical Gel 2% applied, twice daily to the face over 12 months vs. vehicle gel. An open-label safety and tolerability extension study is planned for at least 12 months following the end of the study for patients who complete the Phase 3 trial, and for those who completed the Phase 2 Gorlin Syndrome trial.

“This is a very important milestone for PellePharm and the broader Gorlin Syndrome community, and we’re thrilled to be one step closer to our goal of bringing Patidegib Topical Gel to patients living with Gorlin Syndrome, a condition for which there is no FDA-approved therapy,” said Thorsten Thormann, vice president of global research at LEO Pharma.

As of November 2018, PellePharm and LEO Pharma entered into a strategic collaboration to address the unmet medical needs for rare skin conditions, such as Gorlin Syndrome and High Frequency Basal Cell Carcinoma (HF-BCC). LEO Pharma is providing resources to PellePharm to fund, amongst other activities, its Phase 3 trial of Patidegib Topical Gel 2% in patients with Gorlin Syndrome under the terms of the agreement.

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 topical 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 in Gorlin Syndrome 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 forms of basal cell carcinoma, 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 could potentially reduce the need for regular, painful and disfiguring surgeries. Patidegib topical gel is a first-in-class topical formulation of a proprietary hedgehog inhibitor.

About LEO Pharma

LEO Pharma A/S helps people achieve healthy skin. The company is a leader in medical dermatology with a robust R&D pipeline, a wide range of therapies and a pioneering spirit. Founded in 1908 and owned by the LEO Foundation, LEO Pharma has devoted decades of research and development to advance the science of dermatology, setting new standards of care for people with skin conditions. LEO Pharma is headquartered in Denmark with a global team of 6,000 people, serving 76 million patients in 130 countries. In 2018, the company generated net sales of DKK 10,410 million.

About BridgeBio Pharma
BridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. BridgeBio was founded in 2015 to identify and advance transformative medicines to treat patients who suffer from Mendelian diseases, which are diseases that arise from defects in a single gene, and cancers with clear genetic drivers. BridgeBio’s pipeline of over 15 development programs includes product candidates ranging from early discovery to late-stage development.

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 and generate data from 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.

# # #

Media Contact:

Lauren Barbiero

W2O pure

(646) 564-2156

[email protected]

– 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.

# # #

Media Contact:

Lauren Barbiero

W2O pure

(646) 564-2156

[email protected]

– New production model for cyclopamine uses innovative PCF® technology –

FORT WORTH, Texas–(BUSINESS WIRE)–Phyton Biotech and PellePharm today announced an exclusive partnership to develop a plant cell culture-based process to enable the future renewable production of the raw material for PellePharm’s patidegib, an investigational topical therapy for Gorlin Syndrome and High Frequency Basal Cell Carcinoma (BCC). PellePharm is currently investigating the safety and efficacy of patidegib topical gel for the reduction of surgically-eligible BCCs in Gorlin Syndrome patients in a Phase 3 trial.

The foundation for this joint initiative is Phyton Biotech’s proprietary Plant Cell Fermentation (PCF®) technology, a unique platform that enables renewable, reliable and scalable supply of plant-derived compounds like cyclopamine, the key building block in the synthesis of patidegib. Under its agreement with PellePharm, Phyton Biotech will begin development of the new process immediately.

“We are incredibly excited to partner with PellePharm as pioneers in precision medicine to advance this topical skin cancer treatment to the next level of excellence with our patented PCF® technology,” said Colin Marr, president of Phyton Biotech. “This application of our ‘green chemistry’ process is just the latest example of how Phyton Biotech is advancing traditional manufacturing approaches for high-value phytochemicals and markedly improving patient care.”

Currently, cyclopamine is sourced from the perennial herb Veratrum californicum (corn lily), a member of the Liliaceae plant family found in remote mountainous parts of western North America.

“PellePharm’s partnership with Phyton Biotech underscores our commitment to bring a new therapeutic option to patients with Gorlin Syndrome and other skin cancers,” said Sanuj Ravindran, president and chief executive officer of PellePharm. “As we begin our pivotal Phase 3 clinical trial of patidegib topical gel and prepare for commercial-scale production of patidegib if approved, we anticipate the demand for cyclopamine will grow, spurring the need for a supplemental and more scalable source.”

Gorlin Syndrome, also known as Basal Cell Carcinoma Nevus Syndrome, is a rare, genetic disease that leads to the chronic formation of multiple basal cell carcinomas, often on the face. With no FDA-approved drugs currently available for Gorlin Syndrome, the standard of care is surgery. People with severe Gorlin Syndrome may have as many as 30 or more surgeries per year, which can be repetitive, scarring and disfiguring.

The partnership with PellePharm follows Phyton Biotech’s successful demonstration of its ability to express cyclopamine directly from Veratrum californicum plant cell cultures via PCF®. Further development and full scale-up into commercial-size bioreactors of up to 75 m³ in volume, operated in Phyton Biotech’s facility in Germany, is anticipated to follow the current program.

In addition to this project with PellePharm, Phyton Biotech is working on expanding its patent for the renewable production of alkaloids by Liliaceae cell culture to Europe as it is already granted in the United States and other jurisdictions.

Phyton Biotech’s award-winning green chemistry model focuses on plant cell culture development and offers superior quality, reliability, environmental and cost advantages over other methods of production of complex small molecules, plant-based actives and recombinant proteins.

About Phyton Biotech
Phyton Biotech, a wholly-owned subsidiary of DFB Pharmaceuticals, is the global leader in Plant Cell Fermentation (PCF®) Technology, offering comprehensive services for the development and commercialization of plant-based molecules, extracts and recombinant products, serving the pharmaceutical, Chinese Traditional Medicine, cosmetic, agricultural and food ingredient industries. Using PCF®, Phyton Biotech offers a time, risk and cost-balanced path to commercially viable production processes, overcoming limitations often experienced with traditional plant extraction and chemical synthesis.

As a biotechnology leader with certified GMP facilities in Germany and Canada, Phyton Biotech has a successful track record of developing and implementing innovative contract development solutions for clients around the world. The company is now the world’s largest producer of paclitaxel and docetaxel via PCF®, with the capacity to meet more than one-third of the global demand for these critical active pharmaceutical ingredients. For more information, please visit phytonbiotech.com.

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 (BCC), at their source. PellePharm’s mission is to improve the quality of life for those suffering from Gorlin Syndrome and High Frequency 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.

About Patidegib
Patidegib topical gel has shown early promise in a Phase 2 clinical study for the mitigation of BCC tumors in Gorlin Syndrome by blocking the disease at its source within the hedgehog signaling pathway. The topical formulation of patidegib was developed to provide the efficacy previously demonstrated by oral patidegib in Phase 1 trials without the adverse systemic side effects. The 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 and High Frequency BCC. PellePharm has received both Orphan Drug Designation and Breakthrough Therapy Designation for patidegib topical gel in Gorlin Syndrome from the U.S. Food and Drug Administration (FDA).

Contacts

For Phyton Biotech:
Alex Russell, Principal, Russell Communications
(604) 562-9262 I [email protected]

For PellePharm:
Lauren Barbiero, W2O Group
(646) 564-2156 I [email protected]

###

PellePharm to Present at Piper Jaffray Healthcare Conference

SAN FRANCISCO – November 21, 2018 – PellePharm, a late clinical-stage biopharmaceutical company committed to targeting rare dermatologic conditions at their source, today announced that Sanuj K. Ravindran, M.D., chief executive officer and president, will present at the Piper Jaffray Healthcare Conference on Tuesday, November 27, at 12:30 p.m. EST in New York.

A live webcast of the presentation can be accessed on the company’s website at 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, a BridgeBio Company, is committed to targeting rare, genetic dermatological diseases, including Gorlin Syndrome and High Frequency Basal Cell Carcinoma (BCC), at their source. PellePharm’s mission is to improve the quality of life for those suffering from Gorlin Syndrome and High Frequency BCC by providing an easy-to-use topical gel that eliminates the need for regular, painful surgeries. Patidegib topical gel is a first-in-class topical formulation of a proprietary hedgehog inhibitor.

# # #

Media Contact:
Lauren Barbiero
W2O pure
(646) 564-2156
[email protected]

LEO Pharma and PellePharm Announce $760 Million Collaboration to Advance Innovative Therapies for Rare Skin Diseases

• LEO Pharma A/S acquires minority stake in PellePharm and provides additional non-dilutive capital to fund PellePharm development programs; obtains option to acquire all shares in PellePharm
• PellePharm and stockholders eligible to receive up to an aggregate of US$760 million including equity investment; development and commercialization support; merger consideration; and development, regulatory and commercial milestone payments
• PellePharm on track to commence pivotal Phase 3 trial for Gorlin Syndrome in 1Q2019

 

SAN FRANCISCO & BALLERUP, Denmark – November 20, 2018 – Danish dermatology specialists LEO Pharma and California rare disease pioneers PellePharm today announced a strategic development and commercialization collaboration to address unmet medical needs across various skin diseases with no approved treatments, advancing innovation and access to potential therapies for patients with life-altering conditions, such as Gorlin Syndrome and High Frequency Basal Cell Carcinoma (BCC), two distinct and rare forms of skin cancer.

“We are very excited about the partnership with PellePharm, who are pioneers in Gorlin Syndrome and experts in rare skin diseases. Supporting our ambitious 2025 strategy, it marks LEO Pharma’s entrance in rare skin diseases and it offers a unique opportunity to bring the first treatment forward to people suffering from a very severe skin disease for which there currently are no approved therapies,” said Thorsten Thormann, vice president of research at LEO Pharma.

Under the terms of the agreement, LEO Pharma has initially committed $70 million comprised of equity financing and financial R&D support to fund the global Phase 3 trial for patidegib topical gel 2% for the prevention and treatment of Gorlin Syndrome, with LEO Pharma securing an option to acquire all shares in PellePharm. PellePharm and its stockholders could receive up to an additional $690 million including merger consideration, and regulatory and commercial milestone payments. In addition, PellePharm stockholders are eligible to receive a double-digit royalty after achieving certain commercial milestones.

“Our company is founded on the commitment to targeting rare dermatologic diseases at the source and bringing new groundbreaking treatments forward to patients as efficiently and effectively as possible. As a global leader in medical dermatology, LEO Pharma is a great fit as both a development and commercialization partner. This collaboration puts us on track to commence our pivotal Phase 3 Gorlin Syndrome trial in early 2019. Then after the potential merger, we look forward to working with LEO Pharma to address other rare skin diseases with unmet needs,” said Sanuj Ravindran, president and chief executive officer of PellePharm.

The agreement establishes a joint development committee with PellePharm maintaining responsibility for global development and LEO Pharma supporting in an advising role. Both companies will jointly drive commercialization planning, and Anders Kronborg, chief financial officer of LEO Pharma, will join PellePharm’s board of directors.

”Gorlin Syndrome Group welcomes the news that PellePharm and LEO Pharma are collaborating in developing patidegib topical as a treatment for BCCs. Data from PellePharm’s Phase 2 study suggest patidegib is effective in treating BCCs, with minimal side effects. We hope that by working with LEO, these benefits can be confirmed in a Phase 3 study. An effective topical medication, which can prevent and/or treat BCCs, will avert the need for the painful, disruptive and disfiguring surgeries we currently undergo,” said Sally Webster, chairperson and Matthew Helbert, trustee of Gorlin Syndrome Group U.K.

Currently, there are no FDA-approved therapies for Gorlin Syndrome, and the standard of care for this rare disease is surgery. Patients with this lifelong, severe disease can have as many as 30 surgeries per year beginning in their mid-teens.

PellePharm’s 2% topical formulation of patidegib aims to be the first approved therapy for the prevention of Gorlin Syndrome, also known as Basal Cell Carcinoma Nevus Syndrome (BCCNS), and has shown early promise in Phase 2 clinical trials treating patients with Gorlin Syndrome and Sporadic BCC in both the U.S. and the U.K.

“For more than 18 years, our organization has been dedicated to supporting, educating, and seeking much-needed treatments for people living with Gorlin Syndrome (BCCNS), and yet people with this devastating disease still have no FDA-approved therapies,” said Jean Pickford, executive director of the BCCNS Alliance. “With the Phase 3 study of patidegib beginning recruitment in early 2019, we continue to be encouraged by PellePharm’s dedication to our community, now in partnership with LEO Pharma.”

PellePharm was advised by Rothschild & Co. on this transaction.

About Patidegib

Patidegib topical gel has shown early promise in a Phase 2 clinical study for the mitigation of BCC tumors in Gorlin Syndrome by blocking the disease at its source within the hedgehog signaling pathway. The topical formulation of patidegib was developed to provide the efficacy previously demonstrated by oral patidegib in Phase 1 trials without the adverse systemic side effects. The 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 and High Frequency BCC. PellePharm has received both Orphan Drug Designation and Breakthrough Therapy Designation for patidegib topical gel in Gorlin Syndrome from the FDA.

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 Gorlin Syndrome, the standard of care 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 (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 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.

About LEO Pharma
LEO Pharma helps people achieve healthy skin. By offering care solutions to patients in more than 130 countries globally, LEO Pharma supports people in managing their skin conditions. Founded in 1908 and owned by the LEO Foundation, the healthcare company headquartered in Denmark has devoted decades of research and development to delivering products and solutions to people with skin conditions. In 2017, LEO Pharma employed around 5,200 people worldwide and had sales of 1.4 billion euros. For more information, go to: /

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 (BCC), at their source. PellePharm’s mission is to improve the quality of life for those suffering from Gorlin Syndrome and High Frequency BCC by providing an easy-to-use topical gel that eliminates the need for regular, painful surgeries. Patidegib topical gel is a first-in-class topical 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 seventeen 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.

# # #

LEO Pharma Media Contact:

Henrik Kyndlev

Leo Pharma

+45 3140 6180
[email protected]

PellePharm Media Contact:

Lauren Barbiero

W2O Group

(310) 774-3425

[email protected]

PellePharm Presents Updated Data from Two Phase 2 Studies Demonstrating the Potential of Patidegib Topical Gel to Treat Basal Cell Carcinomas in Patients with Gorlin Syndrome and in Patients with Non-Gorlin Sporadic BCCs

— Study Results Presented at NORD’s Rare Diseases and Orphan Products Breakthrough Summit 2018 —

 

SAN FRANCISCO – October 15, 2018 – PellePharm, a late clinical-stage biopharmaceutical company committed to targeting rare skin conditions at their source, today presented updated clinical data from two Phase 2 studies of patidegib topical gel in a poster session at NORD’s Rare Diseases and Orphan Products Breakthrough Summit 2018 in Washington, D.C. Results of the studies showed clinical clearance and prevention of basal cell carcinoma (BCC) tumors after 6 months of treatment with patidegib topical gel in patients with Gorlin Syndrome, a rare genetic disease; and both clinical and histologic clearance after 3 months of treatment in patients with sporadic, nodular BCCs.

“We are pleased by these positive results showing the potential of patidegib topical gel in preventing new facial BCCs in patients with Gorlin Syndrome, potentially reducing the number of surgeries and resulting facial scarring that patients experience,” said Sanuj Ravindran, M.D., president and chief executive officer of PellePharm. “Based on these Phase 2 findings, we intend to initiate a randomized, one-year registrational Phase 3 trial of patidegib topical gel at the end of 2018 in patients with Gorlin Syndrome, and we also plan to study patidegib topical gel in non-Gorlin patients with high-frequency BCCs.”

Title: Hedgehog Inhibition by Topical Patidegib Reduces Facial BCC Burden in Patients with Gorlin Syndrome

Date and Time: Monday, October 15 – Tuesday, October 16, 2018

Poster Number: #71

The data presented at the NORD Summit are from two Phase 2 trials of topical patidegib gel – a randomized, double-blind, placebo-controlled, study in patients with Gorlin Syndrome in the UK and in a study in patients with sporadic, nodular BCCs in the US. In the Gorlin Phase 2 trial, 17 patients with a total of 85 surgically eligible BCCs (SEBs) applied patidegib topical gel (administered in strengths of 2% and 4%) or a topical vehicle control twice daily for 6 months to their face for prevention and to 5 SEBs for treatment.

The Phase 2 Gorlin trial showed that for prevention of surgically-eligible BCCs (SEBs), patients in the topical control group developed an average of 1.4 new SEBs in 6 months. In contrast, patients treated with patidegib topical gel 2% and 4% developed only 0.4 SEBs in Intent to Treat analysis (p=0.096) and only 0.3 SEBs in Per-Protocol analysis (p=0.008). Additionally, clinical clearance of tumors was observed in 27% (12 out of 45 SEBs) of the patidegib topical gel treated subjects compared to no tumors with clinical clearance in subjects in the vehicle group (N=16 SEBs, P=0.02).

In the U.S. trial, 36 non-Gorlin patients with sporadic, nodular BCCs applied patidegib topical gel 2%, 4%, or topical control to BCCs for 3 months. Use of patidegib topical gel 2% was significantly more effective in clinical and histologic clearance of BCCs after 3 months compared with the topical vehicle gel (p=0.045). This finding correlated with a decrease in hedgehog biomarker in BCC tumors after three months in patients using patidegib topical gel.

Across both studies, patients using topical patidegib gel did not experience any of the significant side effects characteristic of oral hedgehog inhibitors (e.g., hair loss, taste loss or frequent muscle cramps). Patients treated with patidegib topical gel 4% experienced mild skin irritation (e.g., redness, itching and swelling), but those using patidegib topical gel 2% did not.

About Patidegib

Patidegib topical gel has shown early promise in a Phase 2 clinical study for the mitigation of BCC tumors in Gorlin Syndrome by blocking the disease at its source within the hedgehog signaling pathway. Topical patidegib gel 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 Therapy Designation from the FDA.

About Gorlin Syndrome

Gorlin Syndrome is a rare, genetic, disease where patients are born with mutations in 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 Gorlin Syndrome BCCs, the standard of care 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 (BCCs)

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 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.

About PellePharm

Founded by world leaders in hedgehog pathway signaling, PellePharm is a biotechnology company committed to targeting rare, genetic skin diseases, including Gorlin Syndrome and High-Frequency Basal Cell Carcinomas (BCCs), at their source. PellePharm’s mission is to improve the quality of life for those suffering from Gorlin Syndrome and High-Frequency BCCs by providing an easy-to-use topical gel that eliminates the need for regular, painful surgeries. Patidegib topical gel is a first-in-class topical formulation of a proprietary hedgehog inhibitor. 

# # #

Media Contact:

Peter Duckler

W2O Group

(310) 774-3425

[email protected]

SAN FRANCISCO, CA – [September 25, 2018] – PellePharm, a late clinical-stage biopharmaceutical company committed to targeting rare dermatologic conditions at their source, today announced that Sanuj K. Ravindran, M.D., chief executive officer and president, will present at the Cantor Fitzgerald Global Healthcare Conference on Tuesday, October 2, at 4:35 p.m. ET in New York.

A live webcast of the presentation can be accessed on the company’s website at 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 rare, genetic dermatological diseases, including Gorlin Syndrome and High-Frequency Basal Cell Carcinomas (BCCs), at their source. PellePharm’s mission is to improve the quality of life for those suffering from Gorlin Syndrome and High-Frequency 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.

Media Contact:
Lauren Barbiero
W2O pure
(646) 564-2156
[email protected]

 

— Findings show patients with recurring cases of basal cell carcinoma have higher probability of developing other, unrelated cancers —

 

SAN FRANCISCO, August 17, 2018 – PellePharm, a late clinical-stage biopharmaceutical company committed to targeting rare dermatologic conditions at their source, was among the funders of a study published recently in , revealing that individuals having multiple incidences of a common skin cancer, known as basal cell carcinoma (BCC), have a higher inherited risk for developing internal cancers.

“Our team is now able to understand how genetic mutations responsible for repairing DNA damage are linked to basal cell carcinomas, and help physicians begin screening patients with six or more basal cell carcinomas before they develop another unrelated cancer,” said Jean Tang, M.D., Ph.D., co-founder, medical advisor and director of PellePharm.

Preliminary findings from the ten-year ongoing study, performed by researchers at the Stanford University School of Medicine, were published August 9, 2018 in JCI Insight. Researchers found that mutations in genes responsible for repairing DNA are linked to a threefold risk of unrelated cancers in people who develop six or more BCCs during a ten-year period.

“PellePharm is dedicated to improving the quality of life for those suffering from rare dermatological diseases, including Gorlin Syndrome, and through collaborations and support of research, we hope to do just that,” said Sanuj Ravindran, M.D., president and chief executive officer of PellePharm.

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.

Contact
W2Opure
Lauren Barbiero, 646-564-2156
[email protected]

 

SAN FRANCISCO, June 13, 2018–PellePharm, 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
[email protected]

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 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. 

# # #

Media Contact:

Lauren Barbiero

W2Opure

(646) 564-2156

[email protected]

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.

# # #

Media Contact:

Lauren Barbiero

W2Opure

(646) 564-2156

[email protected]

 

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
[email protected]
###

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.