QED and Parent Company BridgeBio Announce Preclinical Data Supporting Tolerability and Activity of Low-dose Infigratinib in Treating Achondroplasia

Data presented at the American Society of Human Genetics (ASHG) 2019 Annual Meeting

  • Infigratinib showed improvements in nine measures of bone development in the Fgfr3Y367C/+ mouse model of achondroplasia at a low dose
  • Observational study (The PROPEL Trial) is currently enrolling children age 2.5-10 years and planned submission of an investigational new drug application remains on track for 2020

SAN FRANCISCO – October 17, 2019 – QED Therapeutics, Inc. and parent company BridgeBio Pharma, Inc. (NASDAQ: BBIO) announced today the presentation of preclinical data supporting the potential of low-dose infigratinib for the treatment of achondroplasia. The data were presented during the ASHG 2019 Annual Meeting in a poster entitled “Low dose, daily or intermittent administration of infigratinib (BGJ398), a selective FGFR inhibitor, as treatment for achondroplasia in a preclinical mouse model.” 

Achondroplasia is the most common cause of dwarfism and is caused by mutation of the gene for fibroblast growth factor receptor 3 (FGFR3). FGFR3 has been shown to be crucial for the process of bone elongation.1,2 Infigratinib, an investigational agent, binds to FGFR3 and has been previously shown to increase bone growth in preclinical mouse models of achondroplasia.3 The data presented today showed a positive dose-response relationship between infigratinib and bone growth.

“Observing a dose-response relationship is an important step in developing infigratinib as a therapy for achondroplasia,” said Laurence Legeai-Mallet, Ph.D., head of research team at Imagine Institute, INSERM U1163, Université de Paris, who led the research. “We also observed better organization within the hypertrophic zone of the growth plate, which is responsible for bone formation and elongation, indicating that a low dose of infigratinib had a positive effect.”

The preclinical study used a dwarf mouse model (Fgfr3Y367C/+), which mimics achondroplasia. In each of the three dose groups, treatment with infigratinib led to an increase in all nine measures of bone size studied compared to the control group (See figure). At the highest dose examined (0.5 mg/kg/day), treatment with infigratinib demonstrated a statistically significant (P < 0.01) improvement in bone length of 7-14% in the upper limbs, 10% to 17% in the lower limbs and 12% in the foramen magnum (an opening at the base of the skull). No apparent toxicity of infigratinib was noted in this study.

“We are encouraged by this preclinical data supporting the ability of a low dose, or even intermittently dosed, infigratinib to increase the length of the limbs as well as other bones in the skeleton. In particular, we believe the increase in foramen magnum size has significant implications, given that narrowing of this area of the skull can, in some cases, cause cervical spinal cord compression in achondroplastic children,” said Susan Moran, M.D., M.S.C.E., chief medical officer at QED. “These data support our decision to submit an investigational new drug application to the U.S. Food and Drug Administration and initiate a clinical trial in 2020 to investigate the potential to treat children with achondroplasia with infigratinib at doses 10 to 100 times lower than used in oncology indications.”

The full poster can be viewed at www.qedtx.com/ASHG2019/

About QED Therapeutics, Inc.

QED Therapeutics, a subsidiary of BridgeBio Pharma, is a biotechnology company focused on precision medicine for FGFR-driven diseases. Our lead investigational candidate is infigratinib (BGJ398), an orally administered, FGFR1-3 selective tyrosine kinase inhibitor that targets the protein responsible for many genetically-caused skeletal dysplasias. QED is also evaluating infigratinib in clinical studies for cancers driven by mutations in the FGFR1, 2, or 3 genes. We plan to conduct further clinical trials to evaluate the potential for infigratinib to treat people with other FGFR-driven tumor types and rare disorders.

For more information on QED Therapeutics, please visit the company’s website at www.qedtx.com

About BridgeBio Pharma, Inc.

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

About the Imagine Institute:

As the first European center of research, care and education on genetic diseases, the Imagine Institute on the Necker-Enfants malades hospital AP-HP campus aims to understand them and cure them. The Institute brings together 1,000 of the best doctors, researchers and healthcare personnel in a creative architecture of synergies. It is this original continuum of expertise, combined with the proximity of patients, which allows Imagine to make discoveries to benefit the patients. 

Since its creation, Imagine Institute has been supported by six founding Members : AP-HP, Inserm, Université Paris Descartes/Université de Paris, Fondation HP-HP, Mairie de Paris, AFM-Téléthon.

Some 8,000 identified genetic diseases affect 30 million patients in Europe, and nearly 3 million in France, where there are 30,000 new cases each year. Nearly 60% of children that have a consultation leave without a genetic diagnosis, and 90% of genetic diseases do not yet have a curative treatment. Faced with this major issue of public health, the challenge is two-fold: to diagnose and to cure.

www.institutimagine.org

BridgeBio Pharma Forward Looking Statements

This press release contains forward-looking statements.  Statements we make in this press release may include statements which are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the Securities Act), and Section 21E of the Securities Exchange Act of 1934, as amended (the Exchange Act), which are usually identified by the use of words such as “anticipates,” “believes,” “estimates,” “expects,” “intends,” “may,” “plans,” “projects,” “seeks,” “should,” “will,” and variations of such words or similar expressions.  We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Securities Exchange Act and are making this statement for purposes of complying with those safe harbor provisions.  These forward-looking statements, including statements relating to expectations, plans, and prospects regarding QED Therapeutics’ clinical development plans, clinical trial results, timing and completion of clinical trials, competitive environment, and the clinical and therapeutic potential of infigratinib for the treatment of achondroplasia, reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations, strategies and prospects as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved.  Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a variety of risks and factors that are beyond our control including, without limitation, QED Therapeutics’ ability to initiate and continue its planned clinical trials of infigratinib, its ability to advance infigratinib in clinical development, including QED Therapeutics’ plans to file an IND in 2020, and the timing and success of any such continued clinical development, as well as those set forth in the Risk Factors section of BridgeBio Pharma, Inc.’s most recent Quarterly Report on Form 10-Q and BridgeBio’s other SEC filings. Moreover, QED Therapeutics operates in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of QED Therapeutics’ management as of the date of this release and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. All forward-looking statements in this press release are based on information available to us as of the date hereof, and except as required by law, we disclaim any obligation to update these forward-looking statements, whether as a result of new information, future events or otherwise.

_________________________

1Rousseau, F., Bonaventure, J., Legeai-Mallet, L., Pelet, A., Rozet, J.M., Maroteaux, P., Le Merrer, M., Munnich, A. “Mutations in the gene encoding fibroblast growth factor receptor-3 in achondroplasia”. Nature 371 (1994): 252–254.

2Shiang R, Thompson LM, Zhu YZ, Church DM, Fielder TJ, Bocian M, Winokur ST, Wasmuth JJ. “Mutations in the transmembrane domain of FGFR3 cause the most common genetic form of dwarfism, achondroplasia.” Cell 78 (1994): 335–342.

3Komla-Ebri, D., Dambroise, E., Kramer, I., Benoist-Lasselin,C., Kaci, N., Le Gall, C., Martin, L., Busca, P., Barbault, F., Graus-Porta, D., Munnich, A., Kneissel, M., Di Rocco, F., Biosse-Duplan,M., Legeai-Mallet, L. “Tyrosine kinase inhibitor NVP-BGJ398 functionally improves FGFR3-related dwarfism in mouse model”. J Clin Invest 126(5) (2016): 1871-84.

QED Contact:
Carolyn Hawley
Canale Communications
carolyn@canalecomm.com
858-354-3581


BridgeBio Pharma Terminates Merger Process With Its Subsidiary Eidos Therapeutics

Agreement Not Reached Between BridgeBio Pharma and Special Committee of Eidos Therapeutics Independent Directors; BridgeBio No Longer Pursuing Acquisition

PALO ALTO – October 14, 2019 – BridgeBio Pharma, Inc. (NASDAQ: BBIO), a clinical-stage biopharmaceutical company focused on genetic diseases, announced today that it was unable to come to an agreement with the Special Committee formed by its subsidiary Eidos Therapeutics (NASDAQ: EIDX), a clinical-stage biopharmaceutical company developing AG10 for the treatment of transthyretin amyloidosis (ATTR), to acquire the outstanding common stock of Eidos that BridgeBio does not already own (approximately 34% of Eidos’s outstanding shares).

Subsequent to an initial offer of 1.3 BridgeBio shares for each Eidos share, the offer was raised twice, resulting in a final offer equivalent to 1.5 BridgeBio shares for each Eidos share with an option for Eidos shareholders to receive a portion of that consideration in cash. Eidos shareholders were to have three options to receive their consideration: all-stock, mixed consideration of cash and stock, or all-cash subject to proration such that the cash portion of the transaction would not exceed approximately $110 million. The cash consideration was to have been funded by BridgeBio with the use of acquisition financing, and was therefore to have had a neutral-to-positive impact upon BridgeBio’s runway post-close.

“We appreciate the hard work of the Eidos Special Committee in evaluating our proposal,” said Brian Stephenson, Ph.D., CFA, chief financial officer of BridgeBio. “At BridgeBio, we work hard to balance opportunities for doubling down on attractive pipeline programs with preserving our ability to pursue a diversified pipeline, as the latter characteristic is valued deeply by many of our investors. We continue to believe strongly in AG10, of which we retain approximately 66% ownership, but we feel that beyond our final offer there are superior ways for us to deploy capital, both in and outside of our pipeline, to generate benefits for patients and returns for our investors. Going forward, we will continue to focus on creating value for patients and investors alike by efficiently allocating capital to high-return genetic disease projects where the science supports advancing therapeutic candidates as rapidly as possible.”

“With this process now behind us, we will return to operating BridgeBio and Eidos as two companies that share the sole focus of bringing important medicines to patients with genetic disease,” said Neil Kumar, Ph.D., founder and chief executive officer of BridgeBio and chief executive officer of Eidos. “BridgeBio will continue to empower Eidos with critical leadership and infrastructure across the commercial, clinical operations, and executive functions, while Eidos will remain laser-focused on getting AG10, our potentially best-in-class stabilizer for ATTR-CM, to patients. I thank the Special Committee for its hard work and look forward to continuing our efforts around the Phase 3 trial for AG10 and the analysis of data from our Phase 2 Open Label Extension. Eidos and AG10 are a significant part of who we are at BridgeBio, having been part of our plan since we founded the company. We remain incredibly excited about the program and its potential for patients with ATTR cardiomyopathy.”

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

Contacts
Jared Levy/Warren Rizzi/Nate Johnson
Sard Verbinnen & Co
(212) 687-8080
BridgeBio-SVC@sardverb.com

Ivy Brain Tumor Center and BridgeBio Subsidiary QED Therapeutics Announce Collaboration to Advance Cancer Research and Treatment Options

Preclinical study designed to test investigational agent infigratinib for the treatment of glioblastoma.

(PHOENIX, AZ — Sept. 24, 2019) — The Ivy Brain Tumor Center at Barrow Neurological Institute, today announced a new collaboration with QED Therapeutics, Inc., a subsidiary of BridgeBio Pharma, Inc., to investigate the FGFR1-3 tyrosine kinase inhibitor, infigratinib, for the treatment of glioblastoma (GBM).  With the goal of addressing unmet medical needs for those affected by malignant brain cancer, this collaboration will focus on targeting FGFR (fibroblast growth factor receptor) genetic alterations that have been shown to spur growth in malignant tumors.

“Five to seven percent of glioblastoma patients’ tumors are driven by FGFR signaling,” said Dr. Nader Sanai, director of the Ivy Brain Tumor Center. “We believe our collaboration with QED Therapeutics will enable us to test how FGFR-driven GBM tumors respond to infigratinib. If proven effective, we then intend to move forward new combined drug strategies incorporating this target.”

In the preclinical studies, the Ivy Center will employ orthotopically implanted, well-characterized FGFR3 fusion patient-derived xenograft models. This is intended to allow the team to further explore the extent to which the drug crosses the blood-brain barrier and what activity it has in the brain.

“We believe the work we are undertaking with the Ivy Center will provide critical insight to shape our clinical development strategy for this disease,“ said Susan Moran, M.D., M.S.C.E., chief medical officer of QED Therapeutics. “Our hope is that infigratinib will become the backbone of new combination therapies to treat patients with glioblastoma.”

Infigratinib is an orally administered, FGFR1-3 selective tyrosine kinase inhibitor. QED Therapeutics has observed activity that appears to be meaningful in clinical trials for cancers that are driven by errors in the FGFR genes. These include chemotherapy-refractory cholangiocarcinoma with FGFR2 fusions and advanced urothelial carcinoma with FGFR3 genetic alterations.

“The intricacies of the brain have posed significant challenges for brain cancer research and the development of therapies,” said Gary Li, head of translational medicine at QED Therapeutics. “We believe collaborating with the Ivy Brain Tumor Center will enable us to move swiftly and further translational research that we hope will unlock the doors to effective treatment options.”

About Ivy Brain Tumor Center

Ivy Brain Tumor Center at the Barrow Neurological Institute in Phoenix, AZ is a non-profit translational research program that employs a bold, early-phase clinical trials strategy to identify new treatments for aggressive brain tumors, including glioblastoma. The Ivy Center’s Phase 0 clinical trials program is the largest of its kind in the world and enables personalized care in a fraction of the time and cost associated with traditional drug development. Unlike conventional clinical trials focusing on single drugs, its accelerated trials program tests therapeutic combinations matched to individual patients. Learn more at IvyBrainTumorCenter.org. Follow the Ivy Brain Tumor Center on Facebook, Instagram, Twitter and LinkedIn.

About QED Therapeutics, Inc.

QED Therapeutics, a subsidiary of BridgeBio Pharma, Inc., is a biotechnology company focused on precision medicine for FGFR-driven diseases. Our lead investigational candidate is infigratinib (BGJ398), an orally administered, FGFR1-3 selective tyrosine kinase inhibitor that has shown activity that we believe to be meaningful in clinical measures such as overall response rate in patients with chemotherapy-refractory cholangiocarcinoma with FGFR2 fusions and advanced urothelial carcinoma with FGFR3 genomic alterations. QED is also evaluating infigratinib in preclinical studies for the treatment of achondroplasia. We plan to conduct further clinical trials to evaluate the potential for infigratinib to treat patients with other FGFR-driven tumor types and rare disorders.

For more information on QED Therapeutics, please visit the Company’s website at www.qedtx.com.

About BridgeBio Pharma, Inc.

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

BridgeBio Pharma Forward Looking Statements

This press release contains forward-looking statements. All statements contained herein other than statements of historical fact constitute forward-looking statements, including statements relating to expectations, plans, and prospects regarding QED Therapeutics’ clinical development plans, clinical trial results, timing and completion of clinical trials, competitive environment, the success of QED Therapeutics’ collaboration with the Ivy Brain Tumor Center and its impact on QED Therapeutics’ clinical development strategy, and the clinical and therapeutic potential of infigratinib. These forward-looking statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to, QED Therapeutics’ ability to initiate and continue its planned clinical trials of infigratinib, its ability to advance infigratinib in clinical development, the timing and success of any such continued clinical development, and the Ivy Center’s ability to initiate and continue its planned preclinical studies of infigratinib. Moreover, QED Therapeutics operates in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of QED Therapeutics’ management as of the date of this release and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. All forward-looking statements in this press release are based on information available to QED Therapeutics as of the date hereof, and QED Therapeutics disclaims any obligation to update these forward-looking statements.

Media Contacts:

Ivy Brain Tumor Center
Karl Stetson
W2O Group
(206) 445-7506
kstetson@w2ogroup.com

BridgeBio
Alberto Gestri
The Outcast Agency
(415) 820-4820
agestri@theoutcastagency.com

Alexion and BridgeBio Announce Japanese License Agreement for Eidos’ Transthyretin Amyloidosis (ATTR) Investigational Medicine

  • Eidos grants Alexion exclusive license to develop and commercialize AG10 in Japan
  • Phase 3 study of AG10 in ATTR cardiomyopathy underway in U.S. & Europe; Phase 3 trial in ATTR polyneuropathy planned to initiate in second half of 2019
  • Agreement expands Alexion’s amyloidosis portfolio
  • Eidos to receive upfront payment of $25 million and equity investment of $25 million, with potential for additional Japanese-based milestone- & royalty-dependent payments –

BOSTON & SAN FRANCISCO – SEPTEMBER 9, 2019 Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) and BridgeBio Pharma, Inc.’s (NASDAQ:BBIO) subsidiary Eidos Therapeutics, Inc. (NASDAQ:EIDX) today announced an agreement that grants Alexion an exclusive license to develop and commercialize AG10 in Japan. AG10 is a small molecule designed to treat the root cause of transthyretin amyloidosis (ATTR) – destabilized and misfolded transthyretin (TTR) protein – by binding and stabilizing TTR in the blood. Eidos is currently evaluating AG10 in a Phase 3 study in the U.S. and Europe for ATTR cardiomyopathy (ATTR-CM) – a progressive, fatal disease caused by the accumulation of misfolded TTR amyloid in the heart – and plans to begin a Phase 3 study in ATTR polyneuropathy (ATTR-PN) – a progressive, fatal disease caused by the accumulation of misfolded TTR amyloid in the peripheral nervous system.

“There is a significant need for new treatments for TTR amyloidosis. We believe AG10 holds promise in its ability to stabilize TTR and halt disease progression,” said John Orloff, M.D., Executive Vice President and Head of Research & Development at Alexion. “We are excited by the potential to grow our amyloidosis portfolio by partnering with Eidos to expand the development of AG10 to Japan. Alexion has more than 10 years of experience operating there, and we look forward to applying our expertise to bring AG10 to Japanese patients.”

“The Phase 2 study in ATTR-CM suggested that AG10 has the potential to become an important treatment option for the underserved ATTR-CM population. The trial showed that AG10 was generally well-tolerated and resulted in near-complete stabilization of TTR, which is known to be correlated with disease severity in ATTR-CM. In the study, AG10 also normalized serum TTR levels, a prognostic indicator of survival in ATTR patients,” said Jonathan Fox, M.D., Ph.D., President and Chief Medical Officer of Eidos. “We have now begun our Phase 3 program to evaluate the safety and efficacy of AG10 in larger studies. This agreement provides the potential opportunity to help even more patients globally by leveraging Alexion’s significant development and commercial experience to expand the AG10 program into Japan.”

Under the terms of the agreement, Alexion will acquire an exclusive license for the clinical development and commercialization of AG10 in Japan. Eidos will receive an upfront payment of $25 million and an equity investment of $25 million at a premium to the market price upon deal execution, with the potential for additional Japanese-based milestone- and royalty-dependent payments.

About AG10

AG10 is an investigational, orally-administered small molecule designed to potently stabilize tetrameric transthyretin, or TTR, thereby halting at its outset the series of molecular events that give rise to TTR amyloidosis, or ATTR. In a Phase 2 clinical trial in patients with symptomatic ATTR-CM, AG10 was generally well tolerated, demonstrated greater than 90 percent average TTR stabilization at Day 28, and increased serum TTR concentrations, a prognostic indicator of survival in a retrospective study of ATTR-CM patients, in a dose-dependent manner.

AG10 was designed to mimic a naturally-occurring variant of the TTR gene (T119M) that is considered a rescue mutation because co-inheritance has been shown to prevent or ameliorate ATTR in individuals also inheriting a pathogenic, or disease-causing, mutation in the TTR gene. To our knowledge, AG10 is the only TTR stabilizer in development that has been observed to mimic the stabilizing structure of this rescue mutation.

The Phase 3 ATTRibute-CM study of AG10 in patients with ATTR-CM is underway in the United States and Europe. Part A of the study will assess the change from baseline in 6-minute walk distance (6MWD) at 12 months. Part B of the study will evaluate reduction in all-cause mortality and frequency of cardiovascular-related hospitalizations will be evaluated at 30 months. In addition, Eidos plans to initiate a Phase 3 study of AG10 in ATTR polyneuropathy (ATTR-PN) in the second half of 2019.

About Transthyretin Amyloidosis (ATTR)

There is significant medical need in transthyretin amyloidosis (ATTR) given the large patient population and an inadequate current standard of care. ATTR is caused by the destabilization of TTR due to inherited mutations or aging and is commonly divided into three distinct categories: wild-type ATTR cardiomyopathy (ATTRwt-CM), mutant ATTR cardiomyopathy (ATTRm-CM), and ATTR polyneuropathy (ATTR-PN). The worldwide prevalence of each disease is approximately 400,000 patients, 40,000 patients and 10,000 patients, respectively.

All three forms of ATTR are progressive and fatal. For patients with untreated 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 for untreated patients. 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 Alexion

Alexion is a global biopharmaceutical company focused on serving patients and families affected by rare diseases through the discovery, development and commercialization of life-changing therapies. As the global leader in complement biology and inhibition for more than 20 years, Alexion has developed and commercializes two approved complement inhibitors to treat patients with paroxysmal nocturnal hemoglobinuria (PNH) as well as the first and only approved complement inhibitor to treat atypical hemolytic uremic syndrome (aHUS), anti-acetylcholine receptor (AchR) antibody-positive generalized myasthenia gravis (gMG) and neuromyelitis optica spectrum disorder (NMOSD). Alexion also has two highly innovative enzyme replacement therapies for patients with life-threatening and ultra-rare metabolic disorders, hypophosphatasia (HPP) and lysosomal acid lipase deficiency (LAL-D). In addition, the company is developing several mid-to-late-stage therapies, including a second complement inhibitor, a copper-binding agent for Wilson disease and an anti-neonatal Fc receptor (FcRn) antibody for rare Immunoglobulin G (IgG)-mediated diseases as well as several early-stage therapies, including one for light chain (AL) amyloidosis and a second anti-FcRn therapy. Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on the core therapeutic areas of hematology, nephrology, neurology, and metabolic disorders. Alexion has been named to the Forbes’ list of the World’s Most Innovative Companies seven years in a row and is headquartered in Boston, Massachusetts’ Innovation District. The company also has offices around the globe and serves patients in more than 50 countries. This press release and further information about Alexion can be found at: www.alexion.com.

[ALXN-G]

About BridgeBio and Eidos

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

Eidos is a BridgeBio Pharma subsidiary focused on addressing the large and growing unmet need in diseases caused by transthyretin (TTR) amyloidosis (ATTR). Eidos is developing AG10, a potentially disease-modifying therapy for the treatment of ATTR. For more information, please visit www.eidostx.com.

Forward-Looking Statements

This press release includes forward-looking statements. Such forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statements. Examples of forward-looking statements include, among others, statements we make regarding: (i) the therapeutic benefits and commercial potential of AG10 in Japan and elsewhere; (ii) development plans and clinical trial plans related to AG10; (iii) the potential of AG10 for the treatment of ATTR cardiomyopathy, ATTR polyneuropathy and other conditions; (iv) that the rights of Alexion under the license agreement will result in growth of Alexion’s amyloidosis portfolio by partnering with Eidos to expand the development of AG10 to Japan; and (v) the likelihood that AG10 will be approved for commercial sale in Japan. The process by which products such as AG10 could potentially be developed and approved for commercial sale is long and subject to highly significant risks. Applicable risks and uncertainties include: results in early stage clinical trials may not be indicative of full results or results from later stage or larger clinical trials (or in broader patient populations) and do not ensure regulatory approval; the possibility that results of clinical trials are not predictive of safety and efficacy and potency of products (or the failure to adequately operate or manage our clinical trials) which could cause the halt of trials, delays or prevention from making regulatory approval filings or result in denial of regulatory approval of product candidates; unexpected delays in clinical trials; unexpected concerns that may arise from additional data or analysis obtained during clinical trials; delays or failure of product candidates to obtain regulatory approval due to clinical trial results, issues with clinical trial products, unexpected expense or otherwise; as well as those additional risks relating to product development and approval and other risks identified under the heading “Risk Factors” included in Alexion’s, BridgeBio’s and Eidos’ most recent Form 10-Q filings and in their respective other future filings with the SEC. The forward-looking statements contained in this press release reflect Alexion’s, BridgeBio’s and Eidos’ current views with respect to future events. Alexion, BridgeBio and Eidos do not undertake and each specifically disclaims any obligation to update any forward-looking statements, except as required by law.

Alexion Contacts:

Media
Megan Goulart, 857-338-8634
Senior Director, Corporate Communications

Investors
Susan Altschuller, Ph.D., 857-338-8788
Vice President, Investor Relations

Eidos Contacts:

Media
Carolyn Hawley, Canale Communications
619-849-5382, carolyn@canalecomm.com

Investors
John Grimaldi, Burns McClellan
212-213-0006, jgrimaldi@burnsmc.com

BridgeBio Contact:

Media
Alberto Gestri
AGestri@theoutcastagency.com

BridgeBio Pharma’s Origin Biosciences Presents New Data on the Natural History of Molybdenum Cofactor Deficiency (MoCD) Type A at the Society of the Study of Inborn Errors of Metabolism (SSIEM) Conference

  • This landmark study is a comprehensive global survey of the natural history of MoCD Type A
  • Results continue to suggest urgent need for new therapies in MoCD Type A, with median survival of <4 years and rapid disease onset within the first month of life for most patients
  • Continue to anticipate initiating a rolling New Drug Application submission for our first-in-class therapy for MoCD Type A, BBP-870, over the coming months

SAN FRANCISCO, Sept. 05, 2019 (GLOBE NEWSWIRE) — BridgeBio Pharma, Inc. (Nasdaq: BBIO) subsidiary Origin Biosciences announced today the presentation of a study on the natural history of patients with Molybdenum Cofactor Deficiency (MoCD) and isolated sulfite oxidase deficiency (ISOD). The study was presented at the 2019 SSIEM Annual Symposium held in Rotterdam, Netherlands.1

In this comprehensive natural history study, 65 patients with MoCD and ISOD were enrolled from 27 participating centers across 14 countries. The majority of patients had MoCD Type A (n=37), followed by MoCD Type B (n=16) and Other (n=12). Data were collected retrospectively for all patients and prospectively for patients in the living cohort for up to one year. The primary endpoint was survival at 1 year of age.

For MoCD Type A patients, the probability of survival at 1 year of age was 0.72 (0.535-0.845) and median survival was 3.98 years. Nearly all patients (n=57) had first presenting symptoms by Day 28 with median onset for MoCD Type A patients at 2 days. The most common initial symptoms were seizures and feeding difficulties. Other subsequent symptoms included developmental delay, truncal hypotonia with limb hypertonia, and cortical blindness. Median urinary S-sulfocysteine and xanthine concentrations were extremely high, and uric acid levels were drastically low in the first few days of life. Additional biochemical and neuroimaging data were collected. These data were presented during the Disorders of vitamins, cofactors and trace elements poster session at SSIEM. See below for a detailed summary of the data.

“Our findings confirm that the vast majority of MoCD and ISOD cases are severe, rapidly progressive, and neurodegenerative, resulting in significant disability and early lethality in almost all cases from the neonatal age,” said Ronen Spiegel, MD, Clinical Associate Professor, Director of Pediatric B Department, and Head of Metabolic Service, Emek Medical Center, principal investigator and lead author of the study.

Currently there are no approved therapies that alter the course of MoCD or ISOD. BridgeBio Pharma, through its subsidiary Origin Biosciences, is developing BBP-870 (ORGN001), a cPMP substrate replacement therapy that targets MoCD Type A directly at its source. By restoring the natural molybdenum cofactor synthetic pathway, the compound aims to reduce buildup of toxic sulfites and alleviate symptoms in infants and children with MoCD Type A. BBP-870 (ORGN001) has received Orphan Drug Designation in the US and Europe, Rare Pediatric Disease Designation and Breakthrough Therapy Designation from the FDA, and is currently being evaluated in a global Phase 2 clinical trial and a global Phase 2/3 clinical trial (NCT02629393NCT02047461).

Key data from the natural history study:
A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/e270f0a3-80ee-4d0d-9e93-f0a7664270c4

About Molybdenum Cofactor Deficiency (MoCD)

MoCD is a rare, autosomal recessive, inborn error of metabolism caused by disruption in molybdenum cofactor (MoCo) synthesis that is vital for sulfite oxidase (SOX) activity. Patients are often infants with severe encephalopathy and intractable seizures. Disease progression is rapid with a high infant mortality rate.1,2 Those who survive beyond the first few months experience profuse developmental delays and suffer the effects of irreversible neurological damage, including brain atrophy with white matter necrosis, dysmorphic facial features, and spastic paraplegia.1,4 Clinical presentation that can be similar to hypoxic-ischemic encephalopathy (HIE) or other neonatal seizure disorders may lead to misdiagnosis and underdiagnosis.2,3 Immediate testing for elevated sulfite levels and S-sulfocysteine in the urine and very low serum uric acid may help with suspicion of MoCD.2,4

About Origin Biosciences

Origin Biosciences, a subsidiary of BridgeBio Pharma, is a biotechnology company focused on developing and commercializing a treatment for Molybdenum Cofactor Deficiency (MoCD) Type A. 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 Type A to market as quickly as possible.

For more information on Origin Biosciences, please visit the company’s website at www.orgintx.com.

About BridgeBio Pharma

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

BridgeBio Pharma Forward Looking Statements

This press release contains forward-looking statements.  Statements we make in this press release may include statements which are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, which are usually identified by the use of words such as “anticipates,” “believes,” “estimates,” “expects,” “intends,” “may,” “plans,” “projects,” “seeks,” “should,” “will,” and variations of such words or similar expressions.  We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Securities Exchange Act and are making this statement for purposes of complying with those safe harbor provisions.  These forward-looking statements, including statements relating to Origin Biosciences’ clinical development plans, including its plans to initiate a rolling NDA submission for BBP-870 (ORGN001), clinical trial results, timing and completion of clinical trials and regulatory submissions, competitive environment and clinical and therapeutic potential of BBP-870, reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made.  Although we believe that our plans, intentions, expectations, strategies and prospects as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved.  Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a variety of risks and factors that are beyond our control including, without limitation, Origin Biosciences’ ability to continue its planned clinical development and regulatory submissions for BBP-870 and the timing and success of any such continued clinical development and planned regulatory submissions, as well as those set forth in the Risk Factors section of BridgeBio Pharma Inc.’s most recent Quarterly Report on Form 10-Q and our other SEC filings.  Except as required by law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

 [1Spiegel R, Schwahn B, Scribner C L, Confer N. A natural history study of molybdenum cofactor (MoCo) and isolated sulfite oxidase deficiencies (ISOD). https://origintx.com/posters/

2Mechler K, Mountford WK, Hoffmann GF, Ries M. Ultra-orphan diseases: a quantitative analysis of the natural history of molybdenum cofactor deficiency. Genet Med. 2015;17(12):965-970.

3Durmaz MS, Özbakır B. Molybdenum cofactor deficiency: neuroimaging findings. Radiol Case Rep. 2018;13(3):592-595.

4Schwahn BC, Van Spronsen FJ, Belaidi AA, et al. Efficacy and safety of cyclic pyranopterin monophosphate substitution in severe molybdenum cofactor deficiency type A: a prospective cohort study. Lancet. 2015;386(10007):1955-1963.

Contacts

Alberto Gestri
AGestri@theoutcastagency.com

BridgeBio Pharma expands its research into neuromuscular disease to include Limb-Girdle Muscular Dystrophy Type 2i (LGMD2i) with new subsidiary ML Bio Solutions

Provides BridgeBio with leading scientific expertise to develop oral small molecule therapy for LGMD2i

CHARLOTTE, NC and SAN FRANCISCO, CA – September 5, 2019 – BridgeBio Pharma, Inc. (NASDAQ: BBIO) today announced ML Bio Solutions as a new subsidiary dedicated to developing BBP-418, a substrate supplementation therapy for the treatment of Limb-Girdle Muscular Dystrophy Type 2i (LGMD2i). BridgeBio is committing R&D expertise to advance this small molecule oral therapy towards patients and intends to begin a natural history study in the second half of 2019, with interventional trials to initiate as soon as possible thereafter, subject to IND approval. The McColl Lockwood Laboratory for Muscular Dystrophy Research at Atrium Health will continue to support the program alongside BridgeBio.

LGMD2i is an autosomal recessive disease wherein loss-of-function mutations in the FKRP gene lead to decreased glycosylation of alpha-dystroglycan (α-DG). Properly-glycosylated α-DG anchors muscle cells to proteins outside of the cell in the extracellular matrix (ECM) as well as shock absorber proteins inside of the muscle cells, such as dystrophin. In LGMD2i, α-DG has a defective sugar backbone, and thus cannot effectively bind these proteins to stabilize the muscle cell, leading to muscle cell fragility and damage under stress. Damaged and degenerating muscle cells can result in global loss of muscle strength. LGMD2i is a progressive and debilitating disease that affects an estimated 7,000 patients across the US and EU, with more affected globally. It is associated with loss of mobility and ultimately respiratory and cardiac failure, and there is presently no approved treatment.

ML Bio Solutions’ compound BBP-418 aims to bypass the core metabolic defect in LGMD2i by providing a pro-drug [to the other enzymes in the pathway] that can be metabolized into the missing substrate in the α-DG sugar backbone. This therapeutic approach originates from the discovery and translational work of scientific co-founder Dr. Qi Long Lu, director of the McColl-Lockwood Laboratory for Muscular Dystrophy Research at Atrium Health, as published in a Nature Communications article in 2018. A brief overview of the program can be found in the investor section of the BridgeBio website.

“By combining the scientific leadership of the McColl-Lockwood Laboratory with the rare disease development expertise at BridgeBio, we believe we can accelerate the path to making a safe and effective disease-modifying treatment available to LGMD2i patients as soon as possible,” said Luther Lockwood, chairman of the board and President of ML Bio Solutions.

“BBP-418 is a novel approach that targets LGMD2i at the source of disease, and exemplifies the BridgeBio mission of creating life-altering medicines by pairing well-understood genetically- driven phenotypes with treatments whose mechanisms target the sources of disease,” said Uma Sinha, Ph.D, chief scientific officer of BridgeBio Pharma. “We are thrilled to partner with the foremost experts in the field to explore the translation of these important discoveries into clinical impact for patients who currently lack effective treatment options.”

About ML Bio Solutions

ML Bio Solutions, a subsidiary of BridgeBio Pharma based in Charlotte, NC, is a biotechnology company focused on developing a small molecule as an oral substrate supplementation therapy for LGMD2i. ML Bio Solutions is led by a team of veteran biotechnology executives, and together with patients and physicians, aims to bring safe, effective treatments to market as quickly as possible.

About BridgeBio Pharma

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

Forward-Looking Statements

This press release contains forward-looking statements.  Statements we make in this press release may include statements which are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, which are usually identified by the use of words such as “anticipates,” “believes,” “estimates,” “expects,” “intends,” “may,” “plans,” “projects,” “seeks,” “should,” “will,” and variations of such words or similar expressions.  We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Securities Exchange Act and are making this statement for purposes of complying with those safe harbor provisions.  These forward-looking statements, including statements relating to the clinical and therapeutic potential of BBP-418, our intention to initiate a natural history study and our plans to initiate clinical development of BBP-418, and the timing of these events,  reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made.  Although we believe that our plans, intentions, expectations, strategies and prospects as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved.  Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a variety of risks and factors that are beyond our control including, without limitation, our ability to execute our clinical and regulatory strategy on the timelines we anticipate, as well as those set forth in the Risk Factors section of BridgeBio Pharma Inc.’s most recent Quarterly Report on Form 10-Q and our other SEC filings.  Except as required by law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

Contacts

Alberto Gestri
AGestri@theoutcastagency.com

BridgeBio Pharma Announces Pricing of Its Initial Public Offering

PALO ALTO, Calif.– June 26, 2019 – BridgeBio Pharma, Inc. (“BridgeBio”) today announced the pricing of its initial public offering of 20,500,000 shares of its common stock at a price to the public of $17.00 per share, above the range of $14.00 to $16.00. In addition, BridgeBio has granted the underwriters a 30-day option to purchase up to 3,075,000 additional shares of its common stock. The shares are expected to begin trading on the Nasdaq Global Select Market on June 27, 2019, under the ticker symbol “BBIO” and the offering is expected to close on July 1, 2019, subject to customary closing conditions.

J.P. Morgan Securities LLC, Goldman Sachs & Co. LLC, Jefferies LLC, SVB Leerink LLC, KKR Capital Markets LLC, Piper Jaffray & Co., Mizuho Securities USA LLC, BMO Capital Markets Corp. and Raymond James & Associates, Inc. are acting as book-running managers for the offering.

A registration statement relating to the securities being sold in this offering has been filed with and was declared effective by the U.S. Securities and Exchange Commission on June 26, 2019.  This offering is being made only by means of a written prospectus. Copies of the final prospectus may be obtained, when available, from the office of J.P. Morgan Securities LLC, Attention: Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, telephone: 1-866-803-9204 or by emailing prospectus-eq_fi@jpmchase.com; Goldman, Sachs & Co., Attention: Prospectus Department, 200 West Street, New York, NY 10282, telephone: 1-866-471-2526 or by emailing prospectus-ny@ny.email.gs.com; Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, telephone: 1-877-547-6340, or by emailing Prospectus_Department@Jefferies.com; or SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, Massachusetts 01220, telephone: 1-800-808-7525, ext. 6132, or by emailing syndicate@svbleerink.com.

This press release shall not constitute an offer to sell or a solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

About BridgeBio Pharma

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

Contacts

Alberto Gestri
AGestri@theoutcastagency.com

BridgeBio Pharma Announces Filing of Registration Statement for Proposed Initial Public Offering

PALO ALTO, Calif.–(BUSINESS WIRE)–BridgeBio Pharma, Inc., a clinical-stage biopharmaceutical company focused on genetic diseases, today announced that it has filed a registration statement on Form S-1 with the U.S. Securities and Exchange Commission (SEC) relating to a proposed initial public offering of shares of its common stock. The number of shares to be offered and the price range for the proposed offering have not yet been determined. BridgeBio has applied to list its common stock on the Nasdaq Global Market under the symbol “BBIO.”

J.P. Morgan Securities LLC, Goldman Sachs & Co. LLC, Jefferies LLC, SVB Leerink LLC, KKR Capital Markets LLC, Piper Jaffray & Co., Mizuho Securities USA LLC, BMO Capital Markets Corp. and Raymond James & Associates, Inc. will act as book-running managers for the proposed offering.

The proposed offering will be made only by means of a prospectus. Copies of the preliminary prospectus relating to the proposed offering may be obtained, when available, for free by visiting EDGAR on the SEC’s website at www.sec.gov. Alternatively, copies of the prospectus, when available, may be obtained for free from the offices of J.P. Morgan Securities LLC, Attention: Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, telephone: 1-866-803-9204 or by emailing prospectus-eq_fi@jpmchase.com; Goldman, Sachs & Co., Attention: Prospectus Department, 200 West Street, New York, NY 10282, telephone: 1-866-471-2526 or by emailing prospectus-ny@ny.email.gs.com; Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, telephone: 1-877-547-6340, or by emailing Prospectus_Department@Jefferies.com; or SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, Massachusetts 01220, telephone: 1-800-808-7525, ext. 6132, or by emailing syndicate@svbleerink.com. The offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed.

A registration statement relating to these securities has been filed with the SEC but has not yet become effective. These securities may not be sold nor may offers to buy be accepted prior to the time the registration statement becomes effective. This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

About BridgeBio Pharma

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

Contacts

Alberto Gestri
AGestri@theoutcastagency.com

Eidos Therapeutics Initiates Attribute-CM, a Phase 3 Study of AG10 in ATTR-CM with Registrational 12-month Endpoint

ATTRibute-CM study design, which incorporates feedback from the FDA, has the potential to accelerate registration with a 12-month primary endpoint of change in 6-minute walk distance (6MWD), followed by a 30-month endpoint of mortality and cardiovascular-related hospitalizations

Trial sites activated in global Phase 3 study (ATTRibute-CM) of AG10 in patients with transthyretin amyloidosis cardiomyopathy

Eidos to Host Conference Call and Webcast at 8am ET

SAN FRANCISCO, Feb. 27, 2019 (GLOBE NEWSWIRE) — Eidos Therapeutics, Inc. (Eidos) (Nasdaq:EIDX), today announced the initiation and design of its pivotal global Phase 3 trial (ATTRibute-CM) of AG10 in patients with transthyretin (TTR) amyloid cardiomyopathy (ATTR-CM). The design of the ATTRibute-CM study, which incorporates feedback from FDA, includes two potentially registrational endpoints. In Part A, benefit in change from baseline in 6-minute walk distance (6MWD) will be evaluated at 12 months, potentially accelerating the time to registration. In Part B, reduction in all-cause mortality and frequency of cardiovascular-related hospitalizations will be evaluated at 30 months.

“ATTRibute-CM aims to generate registrational evidence that AG10 provides meaningful benefit to ATTR-CM patients. The trial is designed to evaluate preservation of function and quality of life on an accelerated timeframe in addition to evaluating longer-term benefit on mortality and cardiovascular-related hospitalizations,” said Jonathan Fox, MD, PhD, president and chief medical officer of Eidos. “If successful, this trial could lead to an approval of AG10 for the treatment of ATTR-CM, a disease with significant unmet medical need and limited treatment options.”

The ATTRibute-CM study advances clinical development of AG10 following positive results from a Phase 2 trial in subjects with symptomatic ATTR-CM. In that study, AG10 was shown to be generally well-tolerated and significantly increase serum transthyretin, a biomarker associated with survival in a retrospective study of ATTR-CM patients, in a dose-dependent manner. All subjects treated with AG10 achieved normal serum transthyretin levels within 28 days of treatment, even those whose baseline levels were well below normal. Results of this study were presented in a late-breaking Featured Science oral presentation at the Annual Scientific Sessions of the American Heart Association in November 2018. An open-label extension of this study is ongoing.

ATTRibute-CM Design

ATTRibute-CM will enroll approximately 510 subjects with symptomatic ATTR-CM, associated with either wild-type or mutant TTR, with New York Heart Association Class I-III symptoms. Subjects will be randomized 2:1 between treatment (AG10 800 mg) and placebo twice daily. In Part A, change in 6MWD at 12 months will be compared between treatment and placebo groups as the first registrational primary endpoint. In Part B, the study will continue for a total duration of 30 months, at which point all-cause mortality and frequency of cardiovascular-related hospitalizations will be compared between treatment and control groups. Secondary endpoints include quality of life as assessed by the Kansas City Cardiomyopathy Questionnaire, safety parameters, serum TTR levels, and TTR stabilization. In Part B, concomitant use of approved, indicated therapies may be allowed.

Investor Conference Call and Webcast Details
Eidos management will host an investor conference call and webcast 8 am ET to review the Phase 3 trial design. To participate in the conference call, dial +1-844-293-0174 (U.S. toll free) or +1-916-582-3546 (international), conference ID 7365928. The webcast will be available live and for replay on the company’s website at ir.eidostx.com. 

About AG10

AG10 is an investigational, orally-administered small molecule designed to potently stabilize tetrameric transthyretin, or TTR, thereby halting at its outset the series of molecular events that give rise to amyloidosis, or ATTR. In a Phase 2 clinical trial in subjects with symptomatic ATTR-CM, AG10 was generally well tolerated, demonstrated >90% average TTR stabilization at day 28, and increased serum TTR concentrations, a prognostic indicator of survival in a retrospective study of ATTR-CM patients, in a dose-dependent manner. AG10 is currently being studied in an open-label extension of a Phase 2 clinical trial in patients with ATTR-CM and sites are currently being activated for a Phase 3 clinical trial of AG10 in patients with ATTR-CM (ATTRibute-CM).

AG10 was designed to mimic a naturally-occurring variant of the TTR gene (T119M) that is considered a rescue mutation because co-inheritance has been shown to prevent ATTR in individuals also inheriting a pathogenic, or disease-causing, mutation in the TTR gene. To our knowledge, AG10 is the only TTR stabilizer in development that has been observed to mimic the stabilizing structure of this rescue mutation.

About transthyretin amyloidosis (ATTR)

ATTR represents a significant unmet medical need with a large patient population and an inadequate current standard of care. ATTR is caused by the destabilization of TTR due to inherited mutations or aging and is commonly divided into three distinct categories: wild-type ATTR cardiomyopathy (ATTRwt-CM), mutant ATTR cardiomyopathy (ATTRm-CM), and ATTR polyneuropathy (ATTR-PN). The worldwide prevalence of each disease is approximately 400,000 patients, 40,000 patients and 10,000 patients, respectively.

All three forms of ATTR are progressive and fatal. 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 Therapeutics is a clinical stage biopharmaceutical company focused on addressing the large and growing unmet need in diseases caused by transthyretin (TTR) amyloidosis (ATTR). Eidos is developing AG10, a potentially disease-modifying therapy for the treatment of ATTR. For more information, please visit www.eidostx.com.

Forward-Looking Statements

This release includes forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act. All statements other than statements of historical facts, including the statements about the potential therapeutic and clinical benefits of AG10, the potential registrational endpoints in the ATTRibute-CM trial, the potential to accelerate registration of AG10, the design of the ATTRibute-CM trial, our ability to enroll patients in and conduct the ATTRibute-CM trial in accordance with our plans, future clinical and regulatory milestones of AG10, the timing of these events, the indications we intend to pursue and our possible clinical or other business strategies, are forward-looking statements. Forward-looking statements can be identified by terms such as “believes,” “expects,” “plans,” “potential,” “would” or similar expressions and the negative of those terms. These forward-looking statements are based on our management’s current beliefs and assumptions about future events and on information currently available to management.  Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. These risks include, but are not limited to, risks and uncertainties related to: our limited operating history and historical losses, our liquidity to fund the development of AG10 through current and future milestones, our ability to raise additional funding to complete the development of AG10, our dependence on the success of AG10, our ability to enroll patients in the ATTRibute-CM trial, results from our clinical trials and pre-clinical studies and those of third parties working in the same area as our product candidate, our ability to advance AG10 in clinical development in accordance with our plans, and our dependence on third parties in connection with our manufacturing, clinical trials and pre-clinical studies. Additional risks and uncertainties that could affect our future results are included in the section titled “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results of Operations” in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2018, which is available on the SEC’s website at www.sec.gov and our website at eidostx.com. Additional information on potential risks will be made available in other filings that we make from time to time with the SEC. In addition, any forward-looking statements contained in this press release are based on assumptions that we believe to be reasonable as of this date. Except as required by law, we assume no obligation to update these forward-looking statements, or to update the reasons if actual results differ materially from those anticipated in the forward-looking statements.

Media Contact:
Carolyn Hawley
Canale Communications
619-849-5382
Carolyn@canalecomm.com

Investor Contact:
Alex Gray
Burns McClellan
212-213-0006
agray@burnsmc.com

Phoenix Tissue Repair Doses First Patient in Phase 1/2 Clinical Trial of PTR-01 (BBP-589) for Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)

BOSTON, Feb. 22, 2019 /PRNewswire/ — Phoenix Tissue Repair, Inc., a biotechnology company focused on developing transformational disease-modifying treatments for dystrophic epidermolysis bullosa (DEB), today announced that the first patient has been dosed in the Phase 1/2, first in-human trial of PTR-01 (BridgeBio Pharma designation BBP-589), a protein replacement therapy for recessive DEB (RDEB). DEB and RDEB are rare genetic multisystem disorders associated with severe skin blistering, and treatment is currently limited to palliative symptom management.

“Dosing the first patient in the clinic is an important milestone for the development of PTR-01 as a potential treatment for RDEB,” said Dr. Neil Kirby, President and Chief Executive Officer of Phoenix Tissue Repair. “The community of RDEB patients, families and physicians urgently need effective treatments for this devastating disease. With an approach that aims to target the root cause of the disease, we are optimistic that PTR-01 could provide hope to patients and their families.”

DEB is a rare genetic disorder symptomatic from birth that is caused by mutations in the gene encoding collagen type VII protein (C7). Symptoms include extreme skin and mucosal fragility and can also include erosions and scarring of other mucous membranes throughout the body. Severe comorbidities are common, and patients require intensive and constant care. DEB, and RDEB in particular, is associated with a considerable reduction in quality-of-life and life span. There are currently no approved disease-modifying therapies for any form of DEB.

The PTR-01-001 Phase 1/2 trial will enroll 14 patients who will each be dosed over a 10-week period. The primary objective of the trial is to evaluate the safety, tolerability and pharmacokinetics of PTR-01 in RDEB patients. The trial will also assess various secondary endpoints. To learn more about the PTR-01 Phase 1/2 clinical trial, please visit www.clinicaltrials.gov and search the identifier NCT03752905.

About Dystrophic Epidermolysis Bullosa (DEB)

DEB is a rare genetic disorder symptomatic from birth that is caused by mutations in the gene for a protein called collagen type VII (C7). C7 is essential for the formation of anchoring fibrils, structures which connect the uppermost two layers of the skin, the epidermis and dermis. Patients with RDEB tend to have particularly severe symptoms due to severe insufficiency of functional C7. Symptoms include extreme skin and mucosal fragility that presents as recurrent, painful blistering and scarring of the skin; ulcerations of the mouth and tongue, and dental caries. In addition to the cutaneous and oral symptoms, severe forms are associated with erosions and scarring of mucous membranes of the eye, esophagus, genitals, and anus. Joint contractures, mutilating deformities of hands and feet, malnutrition, growth retardation, recurrent infections, and a significantly increased risk for squamous cell carcinoma are also common. There are currently no approved disease-modifying therapies for any form of DEB, and the standard of care focuses on wound and pain management.  

About PTR-01

Phoenix Tissue Repair is advancing an investigational therapy known as PTR-01, a protein replacement therapy which uses a recombinant collagen type VII (rC7) for the potential treatment of RDEB. PTR-01 is designed to be systemically available through intravenous delivery. Phoenix Tissue Repair acquired worldwide rights to PTR-01 in 2017. Preclinical studies of PTR-01 have demonstrated C7 distributes to the basement membrane of the skin, and was observed to restore anchoring fibrils, promote healing and improve survival.

PTR-01 has been granted Orphan Drug Designation by the U.S. Food and Drug Administration and the European Medicines Agency.

About the Phase 1/2 Clinical Trial

The PTR-01-001 trial is a saline-controlled, single-blind, multiple ascending dose, dose-escalation, multi-center study. Patients will be enrolled into one of three cohorts. Cohorts 1, 2 and 3 will consist of two, four and eight patients, respectively. During the Treatment Period a total of three doses of PTR-01 and three doses of saline control will be administered to all patients for a total of six doses in a cross-over design over a 10-week period. The primary objective of the trial is to evaluate the safety, tolerability and pharmacokinetics of PTR-01 in RDEB patients. Additionally, the trial will assess the proof of biologic activity through skin biopsy evaluation of C7 and the presence of anchoring fibrils. Wound healing and clinically meaningful patient reported outcomes will also be evaluated. To learn more about the PTR-01 Phase 1/2 clinical trial, please visit www.clinicaltrials.gov and search the identifier NCT03752905.

About Phoenix Tissue Repair and BridgeBio 

Phoenix Tissue Repair is a Boston-based company that is part of the BridgeBio Pharma LLC (“BridgeBio”) family. BridgeBio is a clinical-stage biopharmaceutical company working to create life-altering medicines that target well-characterized genetic diseases at their source. The BridgeBio approach combines a traditional focus on drug development with a corporate model that is designed to allow rapid translation of early stage science into medicines that treat disease at its source. Founded in 2015 by a team of industry veterans, BridgeBio has built a pipeline of 15 development programs, each housed in its own subsidiary, ranging from pre-clinical to late stage development in multiple therapeutic areas including dermatology, cardiology, oncology and endocrinology. BridgeBio’s focus on scientific excellence and rapid execution aims to translate today’s discoveries into tomorrow’s medicines.

Investor Contact:
LeYi Wang, PhD
Vice President, Business Development 
(650) 391-9740
info@phoenixtissuerepair.com

Media Relations Contact:
Carolyn Hawley
Canale Communications
(619) 849-5382
carolyn@canalecomm.com

SOURCE Phoenix Tissue Repair, Inc.