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