BridgeBio Pharma launches CoA Therapeutics to Target Coenzyme-A for Rare Genetic Disorders

PALO ALTO, Calif., June 13, 2018 /PRNewswire/ — BridgeBio Pharma today announced the launch of CoA Therapeutics, a biopharmaceutical company developing novel small-molecules designed to increase Coenzyme-A (CoA) levels in genetic disorders where CoA deficiency is implicated. BridgeBio has committed funding to drive the program toward clinical studies. CoA Therapeutics’ lead compound will be entering the clinic in 2019 with a planned initial focus on pantothenate kinase-associated neurodegeneration, or PKAN.

PKAN is an autosomal recessive genetic disorder caused by mutations in the pantothenate kinase 2 (PANK2) gene. The PANK2 enzyme plays a critical role in the synthesis of CoA, which is crucial in energy metabolism and implicated in a large number of developmental disorders. Patients with PKAN either have a complete absence or a significant deficiency of the PANK2 enzyme, which may lead to reduced CoA levels in the brain. There are currently no treatments approved for PKAN.

The CoA Therapeutics’ lead compounds, which were obtained under a license from St. Jude Children’s Research Hospital, were discovered and developed by St. Jude investigators Suzanne Jackowski, Ph.D., Charles Rock, Ph.D., Richard Lee, Ph.D., and Stephen White, Ph.D.

The prevalence of PKAN is estimated to be three in every 1,000,000 people. In the typical form of PKAN, symptoms present before the age of 10, and patients may rapidly experience neuronal degeneration, causing problems with movement, speech and vision.

“PKAN is a progressive, debilitating disease with no current approved therapy, and patients and their families currently rely on supportive treatments, which partially address the symptoms but not the root cause,” said Shafique Virani, M.D., CEO of CoA. “We believe that our novel approach, using a highly brain-penetrant compound to directly target enzyme activity in neurons, can safely increase CoA levels, ease patients’ symptoms and make a meaningful difference in their quality of life. Our novel approach also holds promise for other diseases with defects in CoA metabolism, including the organic acidemias.”

Joining Dr. Virani is Adam Shaywitz, M.D., Ph.D., who will serve as chief medical officer at CoA as well as CMO-in-residence at BridgeBio. Shaywitz was most recently executive director in clinical sciences at BioMarin Pharmaceuticals where he was involved in the design and planning of a number of clinical studies in rare disease including serving as program lead for the natural history and clinical treatment studies in Sanfilippo Syndrome B (MPS IIIB).  

“We are privileged to be working with Drs. Jackowski, Rock, Lee and White, who have been at the forefront of CoA metabolism research in health and disease,” said Neil Kumar, Ph.D., CEO of BridgeBio. “Their novel approach, which relies on increasing activity of the PANK enzyme family, is a great example of an allosteric agonist approach to potentially treat patients who have very few options.”

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 nineteen transformative assets, each housed in its own subsidiary, ranging from pre-clinical to late stage development in multiple therapeutic areas including oncology, cardiology, neurology, dermatology and endocrinology. The company’s focus on scientific excellence and rapid execution aims to translate today’s discoveries into tomorrow’s medicines.

About CoA Therapeutics
CoA Therapeutics, a subsidiary of BridgeBio Pharma, is developing a small molecule as a targeted therapeutic for PKAN. The company’s mission is to improve and prolong the lives of patients suffering from PKAN and other CoA-implicated genetic disorders.

Founded in 2017, CoA Therapeutics 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.

BridgeBio Pharma Enters into Agreement to Acquire Late Stage Therapy for Ultra-Rare Disorder from Alexion; Launches Origin Biosciences to Develop and Commercialize Therapy

PALO ALTO, Calif., June 11, 2018 /PRNewswire/ — BridgeBio Pharma today announced that it has entered into an agreement with Alexion Pharmaceuticals, Inc. to acquire cyclic pyranopterin monophosphate (cPMP; ALXN1101), a synthetic enzyme co-factor therapy for patients with the ultra-rare disease caused by molybdenum cofactor deficiency (MoCD) Type A. In addition, BridgeBio announced that it was launching a new subsidiary, Origin Biosciences, with sufficient capital to support clinical development of ALXN1101 through potential regulatory approval and commercialization.

MoCD is an ultra-rare autosomal recessive inborn error of metabolism. The disease is caused by a mutation in the MOCS1 gene and leads to defective production of cPMP. Clinical signs of MoCD present shortly after birth and progress rapidly. Newborns with MoCD experience difficulty feeding and intractable seizures yet have no approved available therapies. Patients have a median survival of three years, and those who survive often have severe and irreversible injury to their central nervous system.

“Historically, replacing missing or defective proteins has proven highly efficacious for treating loss of function monogenic conditions – in the case of MoCD type A, we are replacing the missing or defective cPMP, providing children with much needed MoCD activity,” said Michael Henderson, M.D., senior vice president of asset acquisition at BridgeBio. “BridgeBio’s team is committed to continuing the development of ALXN1101 for infants born with MoCD Type A deficiency, their families and caregivers.”

ALXN1101 is a synthetic version of cPMP, the missing cofactor causing MoCD Type A. In previous work with a recombinant form of cPMP, 11 patients with MoCD Type A had normalization of biomarkers within two days, eight patients showed some suppression of seizures, and three patients had near-normal development. ALXN1101 has received Breakthrough Therapy designation from the US FDA.

“Patients born with MoCD face a bleak future, and we will do all we can to pursue the development of this exciting compound, which has the potential to replace the missing enzyme,” said Neil Kumar, Ph.D., CEO of BridgeBio. “BridgeBio aims to sustainably pursue even the rarest of diseases, such as MoCD, especially where we can support drug programs that target well described genetic diseases at their source.”

While specific terms of the deal have not been disclosed, BridgeBio has committed sufficient resources to Origin Biosciences to enable clinical development, regulatory approval and to support commercialization of ALXN1101. Alexion will receive additional payments upon the realization of development and sales milestones.

About Origin Biosciences

Origin Biosciences, a subsidiary of BridgeBio Pharma, is a biotechnology company focused on cPMP replacement therapy for patients with molybdenum cofactor deficiency (MoCD) Type A. Origin’s lead candidate will be a synthetic small-molecule replacement of cPMP.

Origin is led by a team of veteran biotechnology executives. Together with patients and physicians, the company aims to bring a safe, effective treatment for MoCD 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 nineteen transformative assets, each housed in its own subsidiary, 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.

PellePharm to Present Topical Patidegib and Skin Cancer Epidemiological Data at the 7th International Investigative Dermatology Meeting

SAN FRANCISCO–(BUSINESS WIRE)–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.

Contacts

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

Eidos Therapeutics Initiates Phase 2 Clinical Trial for AG10 Targeting Transthyretin Amyloidosis Cardiomyopathy

SAN FRANCISCO, May 3, 2018 /PRNewswire/ — Eidos Therapeutics, Inc., a clinical stage biopharmaceutical company developing a novel oral therapy to treat transthyretin (TTR) amyloidosis (ATTR), today announced dosing of the first patient in the Phase 2 clinical trial of AG10 in patients with ATTR cardiomyopathy (NCT03458130).

The Phase 2 trial will enroll approximately 45 symptomatic ATTR cardiomyopathy patients in a randomized, double-blind, placebo-controlled design. The trial will include a minimum of 30% of patients with mutant ATTR cardiomyopathy, with the remainder having wild type ATTR cardiomyopathy.

Patients will be randomized in a 1:1:1 fashion to placebo or one of two different doses of AG10 on a background of stable heart failure therapy. If all doses are well-tolerated, patients will be treated for 28 days.

“ATTR cardiomyopathy represents a significant unmet need with a poor prognosis and limited existing treatment options, and further, the prevalence of the disease is increasing dramatically with improved awareness and novel, non-invasive diagnostic techniques,” said Rodney Falk, M.D., director of the cardiac amyloidosis program at Brigham and Women’s Hospital and associate professor of medicine at Harvard Medical School. “The preclinical and Phase 1 data describing AG10 indicate that it could be a valuable treatment option for these patients, and I am eager to participate in the trial and learn more about AG10’s potential.”

The primary objective of this trial is to evaluate the safety and tolerability of AG10. The trial will also characterize the pharmacokinetics of AG10 administered twice daily. Finally, the trial will measure and confirm TTR stabilization by validated ex vivo assays, namely fluorescent probe exclusion and immunoblotting. As previous clinical studies have demonstrated that increasing levels of TTR stabilization lead to improved clinical benefit, the trial aims to provide clinical proof of concept to support potential subsequent pivotal trials.

This Phase 2 trial follows the successful completion of a Phase 1 trial of AG10 in healthy volunteers, reported at the 16thInternational Symposium on Amyloidosis in Kumamoto, Japan on March 29, 2018. The Phase 1 trial included single ascending dose, multiple ascending dose (MAD) and food effect components. AG10 was found to be well-tolerated at all doses. No serious adverse events were observed in the trial, vital signs and cardiac safety measures revealed no findings of clinical concern, and liver, kidney and hematological parameters were all within normal limits.

“The positive results of our Phase 1 trial of AG10 in healthy volunteers encouraged our rapid advancement into a Phase 2 trial in patients with ATTR cardiomyopathy,” said Jonathan Fox, M.D., Ph.D., the company’s president and chief medical officer. “Our preclinical and Phase 1 data provide evidence that AG10 could be beneficial for both wild type and mutant ATTR cardiomyopathy patients, and this Phase 2 trial aims to provide additional information in these specific populations.”

Pharmacokinetic measurements demonstrated that AG10 is rapidly and consistently absorbed with a terminal half-life of approximately 25 hours. Ex vivo pharmacodynamic assays demonstrated 100% TTR stabilization at peak plasma concentrations and >95% stabilization on average in the final MAD cohort. TTR stabilization measurements were highly correlated between assays and a clear dose-dependency of stabilization was observed.

“Based on genetic evidence and previous clinical trials, we believe that increasing levels of TTR stabilization lead to increased clinical benefit,” said Uma Sinha, Ph.D., the company’s chief scientific officer. “The ex vivo measurements used in our Phase 1 trial demonstrate that AG10 potently binds TTR tetramers and prevents their dissociation into monomers, which is thought to be the rate limiting step in ATTR pathogenesis. These data support our belief that AG10 could become a best-in-class TTR stabilizer treatment for ATTR patients.”

The company expects to report topline results from this Phase 2 trial by the end of 2018. Eidos expects to launch a second Phase 2 trial in patients with ATTR polyneuropathy later this year.

About AG10

AG10 is an orally-administered, small molecule designed to potently stabilize tetrameric TTR, thereby halting at its outset the series of molecular events that give rise to ATTR. Eidos’ approach, based on over 25 years of research, mimics a naturally-occurring genetic rescue mutation that protects high-risk individuals from developing ATTR by stabilizing the TTR tetramer. In fact, the binding of AG10 to tetrameric TTR creates strong molecular bonds at the same locations as the rescue mutation known as T119M, which “super-stabilizes” TTR and has been shown to enhance survival. This specific binding mode underlies AG10’s ability at peak blood concentrations to completely stabilize tetrameric TTR and prevent its dissociation into disease-causing TTR monomers in the bloodstream. Based on data from previous clinical trials in ATTR demonstrating that preventing the generation of TTR monomers from circulating in the bloodstream leads to improved clinical outcomes, Eidos believes that AG10 could be a best-in-class therapy.

About transthyretin amyloidosis (ATTR)

ATTR represents a significant unmet need, with a comparatively large patient population in the context of rare genetic diseases and an inadequate current standard of care. There are three distinct diseases that comprise the ATTR family: wild-type ATTR cardiomyopathy (ATTRwt-CM), mutant ATTR cardiomyopathy (ATTRm-CM), and ATTR polyneuropathy (ATTR-PN). The worldwide prevalence of each disease is approximately 200,000 patients, 40,000 patients and 10,000 patients, respectively, although Eidos believes the cardiomyopathic forms of the disease are significantly underdiagnosed due to non-specific symptoms and a historical reliance on an invasive heart biopsy for diagnosis. Eidos believes that improving

disease awareness and the introduction of a non-invasive, imaging-based diagnostic scan, coupled with appropriate blood tests, are significantly increasing rates of diagnosis for ATTRwt-CM and ATTRm-CM.

All three forms of ATTR are progressive and fatal, and no disease-modifying therapies have been approved by the FDA. For patients with ATTRwt-CM and ATTRm-CM, symptoms usually manifest later in life (age 50+), with median survival of three to five years from diagnosis. ATTR-PN either presents in a patient’s early 30s or later (age 50+), and results in a median life expectancy of five to ten years from diagnosis. Progression of all forms of ATTR causes significant morbidity, impacts productivity and quality of life, and creates a significant economic burden due to the costs associated with progressively greater patient needs for supportive care.

About Eidos Therapeutics

Eidos is a clinical stage biopharmaceutical company focused on addressing the large and growing unmet need in diseases caused by transthyretin (TTR) amyloidosis (ATTR). Eidos seeks to treat this well-defined family of diseases at their collective source by stabilizing TTR, a therapeutic approach that is supported by genetic evidence as well as previous clinical trials. The company’s product candidate, AG10, is an orally-administered small molecule designed to potently stabilize TTR, suggesting a best-in-class treatment with the potential to halt the progression of ATTR. The development of AG10 is led by a proven management team who are responsible for developing over 30 molecules through IND applications and more than 10 approved drugs. Together with patients and physicians, Eidos aims to bring a safe, effective and disease-modifying treatment for ATTR to market as quickly as possible.

Eidos Therapeutics Completes $64M Series B Financing

SAN FRANCISCO, April 5, 2018 /PRNewswire/ — Eidos Therapeutics, Inc., a clinical stage biopharmaceutical company developing a novel oral therapy to treat transthyretin (TTR) amyloidosis (ATTR), today announced a $64.0 million Series B financing. Proceeds from the financing will be used to advance Eidos’ small molecule product candidate, AG10, into Phase 2 clinical trials and to continue preparations for Phase 3 clinical trials. AG10 targets ATTR at its source by potently binding and stabilizing TTR tetramers, the destabilization of which underlies the development of ATTR.

The financing was led by RA Capital Management and included Eidos’ parent company, BridgeBio Pharma, in addition to new investors Janus Henderson, Viking Global Investors, Aisling Capital, Perceptive Advisors, Cormorant Asset Management and Amzak Health Investors. The Series B financing brings the total capital raised by Eidos to approximately $91.0 million.

“Our clinical data demonstrate that AG10 has a safe, well-tolerated profile and is able to stabilize 100% of plasma TTR at peak concentrations and provide average levels of stabilization greater than 95% at steady-state,” said Neil Kumar, PhD, chief executive officer of Eidos. “Given that increasing levels of stabilization have yielded progressively better clinical results in past trials, our near-complete levels of stabilization suggest that AG10 could be a best-in-class solution. We are targeting ATTR at its source by stabilizing TTR, an approach that is validated by genetics and clinical data.”

In connection with the financing, both Rajeev Shah, managing director and portfolio manager at RA Capital, and Eric Aguiar, MD, partner at Aisling Capital, will join Neil Kumar and Hoyoung Huh, MD, PhD, on Eidos’ board of directors.

“ATTR diseases are a large and growing unmet need, and together, they represent one of the largest genetically-defined diseases with inadequate standard of care,” said Rajeev Shah. “We look forward to working with Eidos’ proven management team to bring a disease-modifying treatment for ATTR to market as quickly as possible.”

About AG10

AG10 is an orally-administered, small molecule designed to potently and selectively stabilize tetrameric TTR, thereby halting at its outset the series of molecular events that give rise to ATTR. Eidos’ approach, based on over 25 years of research, mimics a naturally-occurring genetic rescue mutation that protects high-risk individuals from developing ATTR by stabilizing the TTR tetramer. In fact, the binding of AG10 to tetrameric TTR creates strong molecular bonds at the same locations as the rescue mutation known as T119M, which “super-stabilizes” TTR and has been shown to enhance survival. This specific binding mode underlies AG10’s ability at peak blood concentrations to completely stabilize tetrameric TTR and prevent its dissociation into disease-causing TTR monomers in the bloodstream. Given that previous clinical trials in ATTR demonstrate that preventing the generation of TTR monomers from circulating in the bloodstream leads to improved clinical outcomes, Eidos believes that AG10 could be a

best-in-class therapy.

About transthyretin amyloidosis (ATTR)

ATTR represents a significant unmet need, with a comparatively large patient population in the context of rare genetic diseases and an inadequate current standard of care. There are three distinct diseases that comprise the ATTR family: wild-type ATTR cardiomyopathy (ATTRwt-CM), mutant ATTR cardiomyopathy (ATTRm-CM), and ATTR polyneuropathy (ATTR-PN). The worldwide prevalence of each disease is approximately 200,000 patients, 40,000 patients and 10,000 patients, respectively, although the cardiomyopathic forms of the disease are thought to be significantly underdiagnosed due to non-specific symptoms and a historical reliance on heart biopsy to make the diagnosis. Eidos believes that improving disease awareness and the introduction of a non-invasive, imaging-based diagnostic scan, coupled with appropriate blood tests, are significantly increasing rates of diagnosis for ATTRwt-CM and ATTRm-CM.

All three forms of ATTR are progressive and fatal, and no disease-modifying therapies have been approved by the FDA. For patients with ATTRwt-CM and ATTRm-CM, symptoms usually manifest later in life (age 50+), with median survival of 3-5 years from diagnosis. ATTR-PN either presents in a patient’s early 30s or later (age 50+), and results in a median life expectancy of 5-10 years from diagnosis. Progression of all forms of ATTR causes significant morbidity, impacts productivity and quality of life, and creates a significant economic burden due to the costs associated with progressively greater patient needs for supportive care.

About Eidos Therapeutics

Eidos is a clinical stage biopharmaceutical company focused on addressing the large and growing unmet need in diseases caused by transthyretin (TTR) amyloidosis (ATTR). Eidos seeks to treat this well-defined family of diseases at their collective source by stabilizing TTR, a therapeutic approach that is supported by genetic evidence as well as previous clinical trials. The company’s product candidate, AG10, is an orally-administered small molecule designed to potently stabilize TTR, suggesting a best-in-class treatment with the potential to halt the progression of ATTR. The development of AG10 is led by a proven management team who are responsible for developing over 30 molecules through IND applications and more than 10 marketed drugs. Together with patients and physicians, Eidos aims to bring a safe, effective and disease-modifying treatment for ATTR to
market as quickly as possible.

Eidos Therapeutics to Host Symposium on TTR Stabilization at the 16th International Symposium on Amyloidosis

SAN FRANCISCO, March 20, 2018 /PRNewswire/ — Eidos Therapeutics, a clinical stage biopharmaceutical company developing AG10, a novel precision medicine for transthyretin (TTR) amyloidosis (ATTR), today announced that it will host a symposium regarding TTR stabilization at the 16th International Symposium on Amyloidosis in Kumamoto, Japan, on Thursday, March 29 from 7:40-8:40am (JST).

The symposium is titled “TTR Stabilization for ATTR: Past, Present, and Future” and will be chaired by Dr. Mathew Maurer, from Columbia University. The symposium will feature amyloidosis experts including:

  • Dr. Pablo Garcia-Pavia, Hospital Universitario Puerta de Hierro Majadahonda
  • Dr. Morie Gertz, Mayo Clinic
  • Dr. Julian Gillmore, University College London
  • Dr. Stephen Heitner, Oregon Health Sciences University
  • Dr. Daniel Judge, Medical University of South Carolina
  • Dr. Jose Nativi-Nicolau, University of Utah.

“This symposium brings together some of the greatest leaders in the field to discuss the role of TTR stabilizers in treating ATTR,” said Dr. Jonathan Fox, president and chief medical officer of Eidos. “As we advance our potentially best-in-class TTR stabilizer, AG10, into Phase 2 clinical trials, we will continue working closely with clinical experts and investigators, caregivers, and patients to make new treatments for TTR amyloidosis a reality.”

About AG10

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

About transthyretin amyloidosis

Transthyretin (TTR) amyloidosis is a progressive, fatal disease caused by the accumulation of misfolded TTR in multiple organ systems. Mutations in TTR destabilize the protein and predispose individuals to developing TTR amyloidosis, though the disease can also develop in older individuals without genetic mutations. Misfolded TTR is toxic to cells, and aggregated amyloid fibers can cause further organ dysfunction.

Over 250,000 people worldwide suffer from TTR amyloidosis, though the patient population may be underestimated due to its rarity and confusion with more common diseases such as heart failure. There are no FDA-approved medications indicated for the treatment of TTR amyloidosis.

About Eidos Therapeutics

Eidos Therapeutics is a clinical stage biopharmaceutical company based in San Francisco. Eidos is singularly focused on addressing the large and growing unmet need in transthyretin amyloidosis with AG10, a small molecule precision medicine that targets the disease at its source by stabilizing tetrameric transthyretin. Launched in 2016 and based on research at Stanford University, 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.

BridgeBio Pharma Licenses Late-Stage Oncology Drug Infigratinib to Tackle FGFR-Driven Maladies; Establishes New Subsidiary QED Therapeutics with $65 Million in Initial Financing

PALO ALTO, Calif., Jan. 30, 2018 /PRNewswire/ — BridgeBio Pharma today announced that it is has licensed infigratinib (BGJ398), a highly potent and selective inhibitor of the tyrosine kinase receptor FGFR, from Novartis. In addition, BridgeBio announced that it was launching new subsidiary QED Therapeutics to drive development of infigratinib with an initial financial commitment of $65 million.

FGFR has been implicated as a driver mutation across multiple oncologies – including roughly one out of every five cases of cholangiocarcinoma and urothelial carcinoma – and in multiple forms of pediatric skeletal dysplasias, namely achondroplasia, which affects one out of every 20,000 live births.

Infigratinib is currently in a Phase 2 clinical trial for patients with chemotherapy-refractory bile duct cancer (cholangiocarcinoma) containing FGFR2 fusions. Early clinical results, recently published in the Journal of Clinical Oncology, demonstrated that the compound showed meaningful activity in this population. 

“We are committed to moving this compound forward in late-stage development and further proving the strong efficacy in cancer that has already been demonstrated across multiple trials,” said Daniel Hoth, M.D., QED’s chief medical officer, who has devoted over three decades to drug development, including time as chief of the Investigational Drug Branch of the National Cancer Institute (NCI).

Cholangiocarcinoma affects approximately 6,000 to 8,000 patients a year in the United States. Treatment options are limited, and survival rates vary depending on whether cholangiocarcinoma is found on the bile duct branches within the liver versus those outside of the liver.

“Despite immense strides in studying potential drugs in cholangiocarcinoma, there remains significant need to provide options to these patients,” said Stacie Lindsey, president of the Cholangiocarcinoma Foundation. “The patients and caregivers we work with are very hopeful given data already generated with infigratinib, and we are excited that the passionate team at BridgeBio and QED are working to advance this drug.”

BridgeBio co-founder and investor Frank McCormick, Ph.D., head of the NCI’s Ras initiative and former CSO and co-founder of Onyx Pharmaceuticals, remarked “Infigratinib embodies the crux of what we set out to do at BridgeBio: develop targeted therapies for genetically-driven tumors and monogenic disorders.”

In addition to its clinical data in FGFR-driven cancer, infigratinib has demonstrated potential in skeletal dysplasias, including achondroplasia. In the early work published in the Journal of Clinical Investigation, researchers demonstrated that low doses of infigratinib corrected pathological hallmarks of achondroplasia in mouse models.

Neil Kumar, Ph.D., chief executive officer of BridgeBio, noted that with infigratinib, “We have a late-stage, targeted oncology compound that has demonstrated clear efficacy in the clinic. With the same molecule, we have a potential best-in-class therapy to treat achondroplasia at its source.”

While specific terms of the deal have not been disclosed, BridgeBio has committed $65 million in financing to QED, which is inclusive of a substantial upfront payment to Novartis as well as equity in QED. Novartis will also receive additional payments upon the realization of development and sales milestones as well as royalties.

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 fifteen transformative assets, each housed in its own subsidiary, ranging from pre-clinical to late stage development in multiple therapeutic areas including oncology, cardiology, neurology, dermatology and endocrinology. The company’s focus on scientific excellence and rapid execution aims to translate today’s discoveries into tomorrow’s medicines.

About QED Therapeutics 
QED Therapeutics, a subsidiary of BridgeBio Pharma, is a biotechnology company focused on precision medicine for FGFR-driven disorders. Our lead candidate is infigratinib, a best-in-class FGFR kinase inhibitor that has shown meaningful clinical activity in chemotherapy-refractory cholangiocarcinoma with FGFR2 fusions. QED is also evaluating infigratinib in preclinical studies for the treatment of achondroplasia. We plan to develop infigratinib in additional FGFR-driven tumor types and rare disorders.