Preclinical Data Demonstrates Potential of Eidos Therapeutics’ Lead Compound for the Treatment for Transythyretin Amyloidosis

Data presented at AHA 2017 Scientific Sessions

PALO ALTO, Calif., Nov. 15, 2017 /PRNewswire/ — Eidos Therapeutics, a subsidiary of BridgeBio Pharma, today announced that recently presented preclinical data highlight the potential of AG10 to treat TTR amyloid cardiomyopathy (ATTR-CM). These data were shared publicly for the first time yesterday as oral podium presentations at the American Heart Association’s (AHA) 2017 Annual Scientific Sessions in Anaheim, CA. Eidos has recently initiated clinical trials to evaluate the potential of AG10 in treating this progressive, fatal disease.

ATTR-CM is a progressive, fatal disease caused by the accumulation of misfolded ATTR fibrils in the heart. Transthyretin (TTR) typically exists as a tetramer in the serum. With certain mutations, the four-part molecule can destabilize and dissociate into individual monomers. The monomers are unstable and can misfold and aggregate as amyloid fibrils. These deposits are toxic to cardiac muscle cells and limit the heart’s ability to fill completely during relaxation, leading to progressive and fatal heart failure.

Data from Eidos’ in vitro and animal studies of AG10 were presented in a rapid-fire oral session by Uma Sinha, Ph.D., Eidos’ chief scientific officer. These experiments demonstrated that AG10 binds native, tetrameric TTR in human and animal serum and prevents its dissociation into disease- causing monomers. Dr. Sinha also presented a preclinical pharmacokinetic evaluation of AG10 that suggests the potential for once daily oral dosing in ATTR patients.

Additionally, in a full oral presentation, Isabella Graef, Eidos’ co-founder and assistant professor of pathology at Stanford University, described biochemical experiments that demonstrated the ability of AG10 to stabilize a series of mutant proteins associated with TTR-CM.

“Taken together, these nonclinical studies clearly demonstrate the potential for AG10 to be orally dosed in patients once daily and result in blood levels of AG10 that completely stabilize the native tetrameric form of TTR,” said Dr. Sinha. “It is well recognized that dissociation of TTR tetramers into monomers is the initial step in the development of ATTR, and that the stability of TTR tetramers is reduced by the disease-promoting mutations examined in Eidos’ studies. By stabilizing TTR tetramers in the blood, AG10 targets ATTR at its source and offers the potential to slow or halt progression of disease.”

About AG10

AG10 is a small molecule that selectively and potently binds to and stabilizes the tetrameric protein transthyretin, preventing its dissociation into disease-causing monomers. AG10 was discovered and initially developed by Eidos’ co-founders Isabella Graef, MD, and Mamoun Alhamadsheh, PhD, at Stanford University and the University of the Pacific, respectively.

About transthyretin amyloid cardiomyopathy (ATTR-CM)

ATTR-CM is a progressive, fatal disease caused by the accumulation of misfolded ATTR fibrils in the heart. These deposits are toxic to cardiac muscle cells and limit the heart’s ability to fill completely during relaxation, leading to progressive and fatal heart failure.

Approximately 240,000 people worldwide suffer from ATTR cardiomyopathy, though the patient population may be underestimated due to its rarity and confusion with more common forms of heart failure. Most patients are diagnosed after age 60, when the disease is already advanced, and may progress rapidly.

Some patients are predisposed to ATTR cardiomyopathy due to mutations in the gene that encodes TTR. The most prevalent pathogenic mutation, V122I TTR, is carried by 3.4% of African Americans.

There are no FDA-approved medications indicated for the treatment of ATTR cardiomyopathy.

About Eidos Therapeutics

Eidos Therapeutics is a clinical stage biopharmaceutical company based in the San Francisco Bay Area. Eidos is developing AG10 as a targeted therapeutic for transthyretin amyloidosis. The company’s singular mission is to improve the lives of patients suffering from this disease. Launched in 2016 after years of research supported by Stanford’s TRAM and SPARK programs, Eidos is led by a team of veteran biotechnology executives.

Together with patients and physicians, the company aims to bring a safe, effective treatment to market as quickly as possible.

About BridgeBio Pharma

BridgeBio is a clinical-stage biotechnology 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 rare diseases at their source.

Founded in 2015 by a team of industry veterans who previously brought more than a dozen products to market, BridgeBio has built a portfolio of ten transformative drug programs that address rare diseases across oncology, cardiology, dermatology and endocrinology. The drugs are in various phases of development, from discovery to late clinical stage.

With a commitment to scientific excellence and rapid execution, BridgeBio aims to translate today’s discoveries into tomorrow’s medicines.

MD Anderson and BridgeBio Pharma Launch Navire Pharma to Develop Targeted Therapy for Patients with Difficult-to-Treat Cancer

HOUSTON and PALO ALTO, Calif., Oct. 4, 2017 /PRNewswire/ — The University of Texas MD Anderson Cancer Center and BridgeBio Pharma today announced the launch of Navire Pharma, a biopharmaceutical company aimed at developing novel small-molecule inhibitors of a tyrosine-protein phosphatase called SHP2 for genetically-driven and treatment-resistant cancer.

BridgeBio has committed $30 million and a team of senior business managers to the company, while MD Anderson, through its Institute for Applied Cancer Sciences (IACS), provides intellectual property and an oncology drug development team to advance SHP2 inhibitors toward clinical studies.

SHP2, encoded by the PTPN11 gene, links growth factor signaling with the downstream RAS/ERK/MAPK pathway to regulate cell growth and division. Over-activity of this pathway, often driven by distinct gene mutations, causes or contributes to many human cancers. Inhibiting SHP2offers a new approach to treat tumors relying on this pathway.

Shafique Virani, M.D., joins BridgeBio as CEO in residence, and will serve as CEO of Navire. Virani was most recently officer and vice president of business development, licensing and M&A at Roche/Genentech where he assumed several leadership roles across the globe over 13 years.

“Navire’s compounds potently bind SHP2 and prevent activation of the protein, blocking its ability to promote tumor growth,” said Virani. “Directly inhibiting SHP2 could provide patients and physicians a new, transformative approach to treat RTK-driven cancers at their source.”

Navire Pharma’s lead compounds were discovered and developed by the IACS team led by Phil Jones, Ph.D., executive director and head of drug discovery at IACS, together with Benjamin G. Neel, M.D., Ph.D., director of the Laura and Isaac Perlmutter Cancer Center at New York University(NYU).

“Resistance mechanisms that cause many patients to stop responding to therapy rely on SHP2activity,” said Jones, a scientific co-founder of Navire. “Our ‘molecular glue’ inhibitory approach inactivates SHP2 and could help overcome these resistance pathways.”

SHP2 also suppresses T-cell activity against growing tumors through regulation of the adaptive immune response by binding to PD-1 and dephosphorylating CD28 and the protein LCK. SHP2inhibition may relieve this negative effect, enhancing the patient’s immune response to fight cancer proliferation.

“SHP2’s role in oncogenic signaling has been known for decades, but effective approaches for inhibiting the protein were only recently discovered,” said Neel, Navire scientific co-founder and chairman of the company’s scientific advisory board. “These novel approaches have demonstrated compelling efficacy in pre-clinical disease models, which we hope will translate into benefit for patients.”

Other members of Navire’s scientific advisory board, comprised of leaders in cell signaling and hematological and solid tumors, include:

  • Kwok Kin-Wong, M.D., Ph.D., chief of Hematology and Medical Oncology, NYU
  • Scott Kopetz, M.D., Ph.D., associate professor, Gastrointestinal Medical Oncology, MD Anderson
  • Frank McCormick, Ph.D., director of the University of California, San Francisco (UCSF) Cancer Center and associate dean of the UCSF School of Medicine, and co-founder, Onyx Pharmaceuticals
  • Lillian Siu, M.D., professor of Medicine, Princess Margaret Cancer Centre, Toronto

BridgeBio also has added pharmaceutical and biotechnology executives to the Navire team, including Uma Sinha, Ph.D., and Brian Metcalf, Ph.D., who together have advanced more than 30 drug candidates into human trials, and have brought nine safe and effective drug products to market. The BridgeBio team will closely collaborate with MD Anderson through IND filing and prosecute subsequent clinical trials.

“Navire combines MD Anderson’s clinicians and drug development scientists with our veteran biotechnology team to create a focused organization to develop SHP2-targeted therapies,” said Neil Kumar, Ph.D., chief executive officer of BridgeBio Pharma. “Together, we aim to bring these novel treatments to patients at the soonest possible opportunity.”

About Navire Pharmaceuticals
Navire Pharma, a subsidiary of BridgeBio Pharma, and in collaboration with the Institute for Applied Cancer Science at MD Anderson, is developing inhibitors of SHP2 as targeted therapeutics for the treatment of multiple cancers. Founded in 2017 with the aim of harnessing advances in understanding of SHP2 signaling to create novel targeted oncology medications, Navire is led by a team of veteran biotechnology executives. Together with patients and physicians, the company aims to bring safe, effective treatments to market as quickly as possible.

About MD Anderson
The University of Texas MD Anderson Cancer Center in Houston ranks as one of the world’s most respected centers focused on cancer patient care, research, education and prevention. The institution’s sole mission is to end cancer for patients and their families around the world. MD Anderson is one of only 47 comprehensive cancer centers designated by the National Cancer Institute (NCI). MD Anderson is ranked No.1 for cancer care in U.S. News & World Report’s “Best Hospitals” survey. It has ranked as one of the nation’s top two hospitals for cancer care since the survey began in 1990, and has ranked first 13 times in the last 16 years. MD Anderson receives a cancer center support grant from the NCI of the National Institutes of Health (P30 CA016672).

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.

Eidos Therapeutics Initiates First Clinical Study for AG10 Targeting Transthyretin Amyloidosis, Appoints Camille Landis as Chief Business Officer

PALO ALTO, Calif., Sept. 28, 2017 /PRNewswire/ — Eidos Therapeutics, a subsidiary of BridgeBio Pharma, today announced dosing of the first healthy adult cohort in the first-in-human, Phase 1 clinical trial of AG10 (NCT03294707). Eidos is developing AG10 as a targeted therapeutic for transthyretin (TTR) amyloidosis (ATTR). Eidos’ first clinical trial will evaluate AG10’s overall safety, tolerability and pharmacokinetic profile. Further, the trial aims to provide evidence of dose-dependent TTR stabilization as proof of therapeutic mechanism. The company also announced the appointment of Camille Landis as chief business officer.

There are no FDA-approved therapies indicated for the treatment of ATTR. This debilitating, progressive and fatal disease causes both cardiomyopathy (heart muscle disease resulting in heart failure) and polyneuropathy (nervous system damage affecting movement, sensation and control of blood pressure and digestion). ATTR is believed to affect at least 250,000 people worldwide and is caused by the accumulation of misfolded and aggregated TTR in affected tissues. AG10’s potent, specific binding and stabilization of TTR suggest the potential to alter the progressive clinical deterioration in patients with ATTR.

“AG10 was rationally designed to potently stabilize TTR, targeting the disease at its source by preventing the formation of disease-causing amyloid fibrils,” said Jonathan Fox, M.D., Ph.D., the company’s president and chief medical officer. “Unlike other TTR stabilizers, AG10’s highly selective binding mechanism mimics the super-stabilizing effects of the disease-suppressing T119M mutation that protects some individuals against ATTR. Our prediction is that AG10 will effectively slow or even halt the clinical progression of the disease. This trial is the first step in a journey to making AG10 a safe and effective therapy for patients with ATTR.”

Following the successful completion of this first clinical trial, Eidos will advance AG10 into subsequent studies in symptomatic patients with ATTR. The initial trial will employ both single and multiple doses and will document dose-related blood concentrations of AG10 and its activity in stabilizing TTR tetramers. The study is designed to provide the data needed for selecting optimal doses for subsequent studies in patients with symptomatic transthyretin amyloid cardiomyopathy (ATTR-CM) and/or polyneuropathy (ATTR-PN).

Camille Landis joins the senior management team of Eidos as chief business officer to lead the company’s corporate development and financing activities. Ms. Landis joins Eidos Therapeutics from Relypsa where she was vice president of corporate development. While at Relypsa, she was responsible for building and leading corporate development, alliance management, and strategic planning. She led the sale process of Relypsa to Galencia for $1.5B and supported the company’s IPO and financing activities which raised over $475M. She also led Relypsa’s global partnering effort that resulted in a $165M licensing agreement with Vifor Fresenius Medical Renal Pharma and a co-detailing agreement with Sanofi. Additionally, Ms. Landis served on Relypsa’s executive leadership team and led the company’s annual strategic planning process. She earned her M.B.A from Haas School of Business and her B.A. in biology from the University of St. Thomas.

“Eidos is pursuing a highly targeted approach to treat this devastating disease,” said Ms. Landis. “The preclinical data is scientifically compelling and strongly supported our initiation of clinical testing. I will strive to help the team build the company to ensure our potentially disease-modifying medicine reaches as many patients as possible in a timely manner.”

About AG10
AG10 is a small molecule that selectively and potently binds and stabilizes the tetrameric protein transthyretin, preventing its dissociation into disease-causing monomers. AG10 was discovered and initially developed by Eidos’ co-founders Isabella Graef, M.D. and Mamoun Alhamadsheh, Ph.D., at Stanford University and the University of the Pacific, respectively.

About transthyretin amyloid cardiomyopathy (ATTR-CM)
ATTR-CM is a progressive, fatal disease caused by the accumulation of misfolded ATTR fibrils in the heart. These deposits are toxic to cardiac muscle cells and limit the heart’s ability to fill completely during relaxation, leading to progressive and fatal heart failure.

Approximately 240,000 people worldwide suffer from ATTR cardiomyopathy, though the patient population may be underestimated due to its rarity and confusion with more common forms of heart failure. Most patients are diagnosed after age 60, when the disease is already advanced and may progress rapidly.

Some patients are predisposed to ATTR cardiomyopathy due to mutations in the gene that encodes TTR. The most prevalent of these mutations, V122I TTR, is carried by 3.4% of African Americans.

There are no FDA-approved medications for the treatment of ATTR cardiomyopathy.

About transthyretin amyloid polyneuropathy (ATTR-PN)
ATTR polyneuropathy is a progressive, fatal disease caused by the accumulation of TTR amyloid in the peripheral nervous system. These deposits cause nerve damage that impairs patients’ ability to move (motor function) and feel (sensory function), and control heart rate, blood pressure and digestion (so-called autonomic nervous function). The consequences can include an inability to walk, abnormal pain sensation and severe malnutrition.

Approximately 10,000 people worldwide suffer from ATTR polyneuropathy, with clusters of affected families sharing the same causal mutation, V30M, in regions of Portugal, Japan and Sweden. The onset of disease is bi-modal, either the fourth decade of life or after age 60, but can progress rapidly.

There are no FDA-approved medications for the treatment of ATTR polyneuropathy.

About Eidos Therapeutics
Eidos Therapeutics is a clinical stage biopharmaceutical company based in the San Francisco Bay Area. Eidos is developing AG10 as a targeted therapeutic for transthyretin amyloidosis. The company’s singular mission is to improve the lives of patients suffering from this disease. Launched in 2016 after years of research supported by Stanford’s TRAM and SPARK programs, Eidos is led by a team of veteran biotechnology executives. Together with patients and physicians, the company aims to bring a safe, effective treatment to market as quickly as possible.

About BridgeBio Pharma
BridgeBio is a clinical-stage biotechnology 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 rare diseases at their source.

Founded in 2015 by a team of industry veterans who previously brought more than a dozen products to market, BridgeBio has built a robust portfolio of nine transformative drug programs that address rare diseases across oncology, cardiology, dermatology and cancer. The drugs are in various phases of development, from pre-clinical to late-stage.

With a commitment to scientific excellence and rapid execution, BridgeBio aims to translate today’s discoveries into tomorrow’s medicines.

Bridgebio Pharma Expands Investor Base, Raising an Additional $135 Million, and Discloses Genetic Disease Pipeline Programs

PALO ALTO, Calif., Sept. 13, 2017 /PRNewswire/ — BridgeBio Pharma, a clinical-stage biopharmaceutical company, today provided further detail on its growing pipeline of genetic disease assets and announced a new financing of $135 million, with potential to access significant additional capital via insiders. New investor Viking Global Investors and existing investor KKR co-led the round and were joined by existing investor Perceptive Advisors alongside new investors AIG, Aisling Capital, Cormorant Capital and Janus Funds. The new financing will be used to support BridgeBio Pharma’s existing programs and establish novel efforts.

“BridgeBio Pharma continues to focus on our mission to create medicines for patients with genetic diseases,” said Neil Kumar, Ph.D., co-founder and chief executive officer of BridgeBio Pharma. “In particular, we work to identify promising, but often overlooked, early stage innovation that can translate into drugs that matter for patients. We are fortunate to be supported by investors who believe in our mission.”

Genetic diseases are conditions for which the pathogenicity can be traced back to distinct somatic or germline mutations. These conditions encompass both Mendelian inherited diseases that tend to be pediatric and well-described cancers with clear genetic drivers. There are at least 7,000 genetic diseases that, in total, affect between 25 and 30 million Americans. There are fewer than 500 drugs approved for these conditions, leaving the vast majority of patients without a viable treatment.  

With an emphasis on early-stage discovery and development, BridgeBio Pharma was formed in 2015 by a team of R&D veterans from biotech and academia. The company seeks to translate novel scientific discoveries from universities, academic medical centers and pharmaceutical research groups into genetically-targeted therapeutics that address the fundamental causes of disease.

“The last few decades have seen significant strides in our understanding of the molecular pathways of inherited diseases and cancers,” said BridgeBio Pharma co-founder and investor Frank McCormick, Ph.D., a professor in the UCSF Helen Diller Family Comprehensive Cancer Center and former scientific co-founder of Onyx Pharmaceuticals. “Targeting disease drivers at their source has proven to be an effective strategy for developing novel and impactful therapies.”

BridgeBio’s ‘hub and spoke’ corporate structure provides centralized capabilities for its diverse portfolio of drug programs which are individually housed subsidiary companies. To date, BridgeBio has launched ten drug programs under its dermatology, oncology, cardiology, neurology and endocrine business units. Targeted indications include transthyretin amyloidosis (ATTR-CM and ATTR-PN), pantothenate kinase-associated neurodegeneration (PKAN), Gorlin syndrome and frequent basal cell carcinomas, dystrophic epidermolysis bullosa (DEB), Darier and Hailey-Hailey diseases, Netherton syndrome, and SHP-2, K-RAS and C-RAF driven cancers.

“Modern drug discovery requires modern business infrastructure,” said company co-founder and investor Andrew W. Lo, Ph.D., the Charles E. and Susan T. Harris Professor at the MIT Sloan School of Management and director of the Laboratory for Financial Engineering. “Despite the terrific scientific innovations we’ve seen in biomedicine, there’s been much less innovation on the corporate side. BridgeBio employs a novel structure that combines portfolio diversification with asset-level focus to sustainably develop drugs for genetic disease.”   

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 rare diseases at their source.

Founded in 2015 by a team of industry veterans who previously brought more than a dozen products to market, BridgeBio has built a robust portfolio of ten transformative drug programs that address rare diseases across oncology, cardiology, dermatology, neurology and endocrinology. The drugs are in various phases of development, from pre-clinical to late-stage.

With a commitment to scientific excellence and rapid execution, BridgeBio aims to translate today’s discoveries into tomorrow’s medicines.

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

MENLO PARK, Calif.–(BUSINESS WIRE)–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.

Contacts

Pure Communications
Kelly Lindenboom, 617-763-7281
klindenboom@purecommunications.com

BridgeBio Pharma Launches Eidos Therapeutics to Develop Targeted Therapy for Fatal Heart Disease

PALO ALTO, Calif., April 27, 2017 /PRNewswire/ — BridgeBio Pharma today announced the launch of Eidos Therapeutics, a biopharmaceutical company developing a novel small-molecule treatment for transthyretin (TTR) amyloidosis. BridgeBio has committed $27 million and seasoned R&D executives to advance Eidos’ lead compound, AG10.

“TTR amyloidosis is a progressive, fatal disease without an FDA-approved therapy. Patients and their families currently rely on supportive treatments that neither address the root cause of the condition nor alter its natural course,” said Jonathan Fox, M.D., Eidos’ president and chief medical officer. “We believe that a safe, effective treatment that halts disease progression is achievable, and that AG10 could fulfill that promise.”

TTR amyloidosis is caused by the accumulation of toxic TTR amyloid deposits in the heart and peripheral nerves. Mutations in the gene that encodes TTR are known to predispose people to the disease and possibly accelerate its progression, although TTR amyloidosis can occur in individuals without mutations as well.

It is estimated that up to 250,000 people worldwide suffer from the cardiac and neurological consequences of this disease. The prevalence of the disease is expected to increase with an aging population, and the development of potential new therapies is driving increased awareness that could lead to earlier diagnoses.

TTR amyloidosis is driven by the destabilization of TTR in the blood, and AG10 targets the disease at its source by stabilizing TTR and preventing toxic amyloid fibrils from forming. This precision medicine approach has the potential to bring a true disease-modifying therapy to patients. AG10 is expected to enter clinical trials in the second half of this year.

AG10 was discovered and initially developed by Eidos’ co-founders Isabella Graef, M.D. and Mamoun Alhamadsheh, Ph.D., through research funded by Stanford Medicine’s SPARK program. Eidos is now led by a veteran team of pharmaceutical and biotechnology executives who together have advanced more than 30 drug candidates into human trials, and have brought more than eight safe and effective drug products to market.

“Eidos is a prime example of the targeted innovation that BridgeBio aims to identify and support,” said Neil Kumar, Ph.D., interim CEO of Eidos and CEO of BridgeBio. “BridgeBio is providing Eidos with the resources and capabilities to efficiently shepherd AG10 through clinical development, translating years of scientific advancement into a therapy that could save thousands of lives.”

BridgeBio Pharma focuses on rapidly translating early-stage science into life-changing drugs for people with serious unmet medical needs. Utilizing partnerships and a lean business model, the company is working to bridge the gap between scientific breakthroughs and the patients who need them most.

About Eidos Therapeutics

Eidos Therapeutics, a subsidiary of BridgeBio Pharma, is developing AG10 as a targeted therapeutic for transthyretin amyloidosis. The company’s singular mission is to improve and prolong the lives of patients suffering from this disease.

Launched in 2016 after years of research supported by Stanford’s SPARK program, Eidos is led by a team of veteran biotechnology executives. Together with patients and physicians, the company aims to bring a safe, effective treatment to market as quickly as possible.

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 eight transformative drugs ranging from pre-clinical to late stage development in multiple therapeutic areas including oncology, cardiology, dermatology and rare disease.

The company’s focus on scientific excellence and rapid execution aims to translate today’s discoveries into tomorrow’s medicines.

BridgeBio Pharma Launches with a Focus on Precision Medicines for Genetic Diseases

PALO ALTO, Calif.–(BUSINESS WIRE)–California-based BridgeBio Pharma has come out of stealth mode to reveal more detail about its novel approach to developing new therapies for genetic diseases. These diseases, often inherited and individually rare, collectively exact devastating effects on tens of millions of patients in the US alone, many of whom are children. The company, formed in 2015, is primarily focused on pre-commercial products, utilizing its advantages in sourcing and operating to find and build value at each stage of development.

BridgeBio Pharma combines a traditional focus on drug products with a new corporate model to tackle a big industry need – translating early stage genetic disease science into drugs that matter for patients. “There’s a lot of money and interest in new technology companies, like those focused on gene editing, and then again for late stage clinical products,” says co-founder and CEO Neil Kumar. “But if you have a small molecule for a single genetic disease that is pre-clinical, it is harder to attract interest. We started BridgeBio to focus on those single product opportunities that target well defined genetic drivers of disease. BridgeBio is set up to move those programs forward efficiently and at scale.”

The company sees itself as a drug product engine as opposed to a novel science platform – “we partner closely with academic and clinical leaders to help move insights they have already made into the clinic. We are not trying to discover new biology so much as to take what is already known and develop therapies from it,” says co-founder Frank McCormick, former co-founder of Onyx Pharmaceuticals. BridgeBio’s team members have collectively been responsible for over a dozen marketed products and include drug development veterans Charles Homcy, Frank McCormick, Philip Reilly, Hoyoung Huh, Uma Sinha, and Robert Zamboni.

BridgeBio’s novel corporate structure was designed in collaboration with MIT Sloan Professor Andrew W. Lo, who is also a founding investor and member of BridgeBio. Rather than forming large platform companies, the team forms lean and focused subsidiaries around individual assets or diseases. These subsidiaries can draw on BridgeBio’s network of world-class genetic disease expertise to complement their internal efforts. This structure is ideal for building value in product-focused investments, and allows for rapid capital reallocation should an asset fail. Lo explains: “We tried to put in place, at the outset, a corporate structure that optimized for focused R&D at the level of each asset but that still provided diversification for investors. This diversification in turn provides more predictable positive outcomes and makes these pre-commercial programs more attractive for a broader pool of capital. Ultimately, the structure also allows for liquidity in ways that are unique as compared with the traditional c-corp or fund structures seen in this industry.”

BridgeBio has seven programs to date, with two in the clinic and hopes to add to its diversified portfolio of assets based on a systematic mapping of the genetic disease landscape. It has deployed over $50 M in R&D commitments in 2016.

Contacts

BridgeBio Pharma
Michael Henderson, 650-665-7924
mh@bridgebio.com

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

MENLO PARK, Calif.–(BUSINESS WIRE)–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.”

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:

“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.

Contacts

Pure Communications
Katie Engleman, 910-509-3977
Katie@purecommunicationsinc.com