BridgeBio and Eidos Present Data from Phase 2 Open Label Extension Suggesting Long-term Tolerability of AG10 and Stabilization of Transthyretin Amyloid Cardiomyopathy Disease Measures

AG10 was Well Tolerated with Median 65 Weeks Follow-up since Phase 2 Initiation

Rates of All-Cause Mortality (Including Either Death or Cardiac Transplantation, 8.5%) and Cardiovascular Hospitalization (25.5%) Observed in Exploratory Analysis Were Lower than Rates Observed in Placebo-treated Participants in the ATTR-ACT Study

Near-complete Stabilization of TTR Maintained in Participants Throughout Duration of Study

Serum TTR levels, a Prognostic Indicator of Survival in ATTR-CM Patients, Were Elevated and Maintained in the Normal Range Throughout the Study Duration

Cardiac Biomarkers and Echocardiographic Parameters Were Stable Throughout Trial Duration

Presentation from AHA 2019 Scientific Sessions Available on Company Websites (eidostx.com and bridgebio.com) 

San Francisco, November 16, 2019 — BridgeBio Pharma, Inc. (BridgeBio) (Nasdaq:BBIO) and Eidos Therapeutics, Inc. (Eidos) (Nasdaq:EIDX), today presented positive data from the companies’ ongoing Open Label Extension (OLE) of the Phase 2 clinical trial studying AG10 in patients with symptomatic transthyretin (TTR) amyloidosis cardiomyopathy (ATTR-CM). ATTR-CM is a progressive and fatal disease that is an under recognized cause of heart failure. The data were presented in a late-breaking featured science oral presentation at the American Heart Association (AHA) Scientific Sessions in Philadelphia, Pennsylvania.

Study participants received 800mg of AG10 twice daily during the OLE and were followed for a median of 65 weeks since Phase 2 initiation. AG10 treatment was generally well-tolerated and resulted in near-complete TTR stabilization as measured using established ex vivo assays. Lower rates of mortality (death or cardiac transplantation) and cardiovascular-related hospitalizations were observed in AG10 Phase 2 OLE participants than were reported in a similar population of ATTR-CM patients who received placebo for 15 months in the ATTR-ACT study. Cardiac biomarkers and echocardiographic parameters were stable in patients treated with AG10 in the Phase 2 OLE.

“This update from our Phase 2 OLE demonstrated continued tolerability of AG10 in patients with advanced ATTR-CM. We also observed meaningfully lower rates of mortality and cardiovascular hospitalizations than what would be expected for untreated ATTR-CM patients with similar disease severity,” said Jonathan Fox, M.D., PhD., FACC, president and chief medical officer of Eidos. “These encouraging data continue to support the development of AG10 as a potentially best-in-class treatment for ATTR-CM patients.”

AG10 Phase 2 Open Label Extension Results

The ongoing OLE study enrolled 47 of 49 participants (96%) from the 28-day randomized, placebo-controlled, Phase 2 study of AG10 in ATTR-CM patients with New York Heart Association (NYHA) Class II or III symptoms. Interim analysis of the ongoing study was completed on August 31, 2019 in conjunction with annual regulatory reporting and review, at which time 41 participants remained in the study. Three (6.4%) participants in the OLE had died, two due to disease progression and one due to cervical cancer. Three (6.4%) additional patients enrolled in the study had discontinued treatment, including one participant who underwent cardiac transplantation for their disease.

–          Adverse events reported in the OLE study were generally consistent with the underlying ATTR-CM disease state and no safety signals of potential clinical concern were associated with the administration of AG10 in the study. Forty-six (97.9%) patients experienced a treatment-emergent adverse event reported during the study, with falls, congestive cardiac failure, dyspnea, and acute kidney injury the most commonly reported adverse events. Nineteen (40.4%) participants experienced a treatment-emergent serious adverse event reported during the study, with congestive cardiac failure (10.6%) and acute kidney injury (8.5%) the most commonly reported serious adverse events.

–          The rate of all-cause mortality (including either death or cardiac transplantation, 8.5%) and cardiovascular-related hospitalizations (25.5%) observed in an exploratory analysis of participants in this study following a median of 15 months since Phase 2 initiation were lower than those observed at 15 months in placebo-treated patients in the ATTR-ACT study (all-cause mortality including death or cardiac transplantation, 15.3%; cardiovascular-related hospitalizations, 41.8%).

–          Stabilization of TTR, as measured using established ex vivo assays, was maintained >90% on average at all study visits in actively treated patients.

–          Serum TTR levels, a prognostic indicator of survival in a published cohort of wild-type ATTR-CM patients, were elevated upon AG10 treatment and were maintained in the normal range throughout the study duration. Mean serum TTR levels were increased from baseline by 39% and 56% in wild-type and variant-carrying ATTR-CM patients, respectively, at OLE Visit Day 180.

–          Cardiac biomarkers and echocardiographic parameters were stable during the OLE study. NT-proBNP and TnI were unchanged throughout the course of the study. Echocardiographic parameters, including left ventricular mass and left ventricular stroke volume index, were unchanged during the study.

The presentation of the Phase 2 open label extension data from the American Heart Association (AHA) Scientific Sessions will be available on the company website (eidostx.com and bridgebio.com).

A Phase 3 study of AG10 in ATTR-CM patients (ATTRibute-CM) is currently ongoing. In Part A of the study, change in six-minute walk distance at 12 months will be compared between active treatment and placebo groups as the first registrational primary endpoint. In Part B, all-cause mortality and frequency of cardiovascular-related hospitalizations will be compared between treatment and control groups at 30 months total duration. In Part B, concomitant use of therapies indicated for the treatment of ATTR-CM may be allowed. The study is enrolling at 44 sites across six countries and enrollment for Part A is now projected to complete in the second half of 2020, with top-line data expected in 2021.

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 randomized, placebo-controlled 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. The open label extension of this Phase 2 study identified no safety signals of potential clinical concern associated with administration of AG10 15 months after study initiation. In an exploratory analysis, lower rates of all-cause mortality (including death and cardiac transplantation) and cardiovascular hospitalizations were observed in study participants than in placebo-treated ATTR-CM patients in the ATTR-ACT study. Cardiac biomarkers and echocardiographic parameters were stable in the AG10 Phase 2 OLE.

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. 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 at 30 months. In addition, Eidos plans to initiate a Phase 3 study of AG10 in ATTR polyneuropathy (ATTR-PN) in Q1 2020.

About transthyretin amyloidosis (ATTR)

There is significant medical need in transthyretin amyloidosis (ATTR) given the large patient population and limited 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 estimated to be at least 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 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. For more information, please visit www.bridgebio.com.

About Eidos Therapeutics

Eidos is a BridgeBio Pharma subsidiary focused on addressing the large and growing unmet need 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 of 1934. All statements other than statements of historical facts, including the statements about the potential therapeutic and clinical benefits of AG10, the potential for AG10 to be a best-in-class treatment for ATTR-CM patients, the design of our ongoing Phase 3 ATTRibute-CM trial of AG10, our ability to enroll patients in and conduct the ATTRibute-CM trial and our planned Phase 3 clinical trial of AG10 in ATTR-PN in accordance with our plans, our ability to generate data from and to complete these trials, 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 and our planned Phase 3 clinical trial of AG10 in ATTR-PN, 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, 2019, which is available on the SEC’s website at www.sec.gov and our websites at eidostx.com and bridgebio.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:
Grace Rauh
917-232-5478
Grace.rauh@bridgebio.com

Investor Contact:
John Grimaldi
Burns McClellan
212-213-0006
jgrimaldi@burnsmc.com

BridgeBio Pharma Reports Third Quarter 2019 Financial Results and Highlights Portfolio Progress

-Multiple clinical and pre-clinical milestones achieved across the BridgeBio portfolio

-Delivered pipeline growth with the addition of BBP-418 for limb-girdle muscular dystrophy type 2i

-Ended quarter with $446.1 million in cash, cash equivalents and marketable securities, excluding Eidos

Recent Highlights:

  • BBP-831 – FGFR1-3 inhibitor for achondroplasia: Initiated PROPEL, a prospective observational study in children with achondroplasia, the most common genetic form of short stature (NCT04035811). The study will establish annualized growth velocity (AGV) for each child over a minimum period of six months. PROPEL is designed to provide baseline measurements for children who enroll in PROPEL2, a Phase 2 study of low-dose infigratinib in achondroplasia which is on track to start in 2020. Additionally, in October 2019, new preclinical data supporting tolerability and efficacy of infigratinib in the mouse model of achondroplasia were reported at the American Society of Human Genetics conference (link to poster).
  • BBP-870 – cPMP replacement therapy for MoCD type A: Presented natural history study data at the Society of Inborn Errors of Metabolism Conference (link to poster) in September. These data suggest an urgent need for new therapies in molybdenum cofactor deficiency (MoCD) type A, an often-fatal rare genetic disease, and will be a critical component of the planned new drug application.
  • BBP-812 – Gene therapy candidate for Canavan disease: Opened a natural history study in Canavan disease (treatcanavan.com). Presented preclinical data (link to poster) demonstrating intravenous (IV) dosing of BridgeBio’s experimental therapy for Canavan disease (BBP-812) achieved broad central nervous system delivery; the IV approach is much less invasive compared to intrathecal or intracerebroventricular alternatives.
  • BBP-631 Gene therapy candidate for CAH: Presented preclinical update (link to poster) for gene therapy candidate BBP-631 in congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency wherein IV dosing of non-human primates with BBP-631 resulted in durable delivery and expression of the gene product to the adrenal tissue.
  • BBP-398 – SHP2 inhibitor for treatment-resistant cancer: Presented data (link to poster) highlighting the discovery and preclinical efficacy of BBP-398, a potent and selective SHP2 inhibitor, which is currently being prepared for the submission of an IND in 2020 for evaluation in RTK-driven cancer.
  • BBP-265 (AG10) – TTR stabilizer for ATTR: Granted Alexion Pharmaceuticals, Inc. an exclusive license to develop and commercialize AG10 in Japan for an upfront payment of $25 million and an equity investment of $25 million.
  • Pipeline growth: Announced addition of a new asset, BBP-418, to the pipeline. BBP-418 is a substrate supplementation therapy for the treatment of limb-girdle muscular dystrophy type 2i and is in IND-enabling studies. This condition affects an estimated 7,000 patients in the United States and European Union with no currently approved therapies.
  • Organizational growth: Added two new members to the senior leadership team. Brian Stolz joined as Chief Operating Officer of BridgeBio. Mr. Stolz most recently held the position of Chief People Officer at Activision Blizzard. Yi Ching Yau joined as Chief Accounting Officer of BridgeBio. Prior to joining BridgeBio, Ms. Yau served as the VP of Finance at Nektar Therapeutics.

Upcoming Milestones:

  • BBP-265 (AG10) – TTR stabilizer for ATTR: Plan to present interim analysis of the ongoing Phase 2 open label extension study (NCT03536767) of AG10 in patients with TTR amyloid cardiomyopathy, an inherited form of heart failure, at the American Heart Association 2019 Scientific Sessions in a Late-Breaking Featured Science Oral Presentation. A Phase 3 study of AG10 in ATTR-PN (ATTRibute-PN) is on track to begin in the first quarter of 2020.
  • BBP-870 – cPMP replacement therapy for MoCD type A: On track to initiate a rolling new drug application submission for our first-in-class therapy for molybdenum cofactor deficiency (MoCD) type A, BBP-870, by the end of 2019.
  • BBP-589 – COL7A protein replacement therapy for recessive dystrophic epidermolysis bullosa: Plan to share topline data from the ongoing Phase 1/2 study (NCT03752905) during 2020.
  • BBP-831 (infigratinib) – FGFR1-3 inhibitor for FGFR2+ cholangiocarcinoma: Plan to complete enrollment of the ongoing pivotal Phase 2 study (NCT02150967) in second line cholangiocarcinoma (bile duct cancer) and present updated results at a major oncology meeting in 2020. Remain on track to submit new drug application for FDA approval in 2020.

Third Quarter 2019 Financial Results:

 Cash, Cash Equivalents and Marketable securities

Consolidated cash, cash equivalents and marketable securities, excluding restricted cash, totaled $611.9 million as of September 30, 2019.  Excluding Eidos, BridgeBio’s cash balance as of September 30, 2019 was $446.1 million compared to $162.4 million as of June 30, 2019.  The net change in cash balance of $283.7 million reflects net proceeds received from BridgeBio’s initial public offering of $368.7 million, offset by the repurchase of a non-controlling interest for $26.4 million and approximately $58.6 million primarily for operating expenses.

Operating Expenses

Operating expenses for the three months that ended September 30, 2019 were $81.3 million, as compared to $41.5 million for the same period in the prior year. The increase in operating expenses of approximately $39.8 million was mainly attributable to increased research and development expenses related to the progression of our programs.

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. For more information, please visit www.bridgebio.com.

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 the clinical and therapeutic benefits of our product candidates, our ability to initiate additional preclinical studies and clinical trials, our ability to submit planned regulatory filings for our product candidates, our ability to generate data from our ongoing and planned preclinical studies and clinical trials, 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 continue our planned research and development activities and complete our 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.

Contact:

Alberto Gestri
AGestri@theoutcastagency.com

BridgeBio Pharma Gene Therapy Subsidiaries Present Data Demonstrating Potential in Two Rare Disease Indications at the European Society of Gene and Cell Therapy Conference

  • Preclinical data shows promise for gene therapy candidates for congenital adrenal hyperplasia due to 21-hydroxylase deficiency (BBP-631) and Canavan disease (BBP-812)
  • Natural history clinical study for Canavan disease is currently enrolling
  • IND submissions for both gene therapy product candidates anticipated in 2020

SAN FRANCISCO – October 22, 2019 – BridgeBio Pharma, Inc. (NASDAQ:BBIO) today announced that promising preclinical data from two of its gene therapy-focused subsidiaries – Adrenas Therapeutics and Aspa Therapeutics – will be presented at the European Society of Gene and Cell Therapy (ESGCT) Conference, occurring in Barcelona from October 22 to 25. In addition, the company announced that Aspa Therapeutics is currently conducting a natural history study in children with Canavan disease, CANinform.

Experimental Gene Therapy for Congenital Hyperplasia Shows Durable Transgene Expression

Researchers from BridgeBio subsidiary Adrenas Therapeutics presented new preclinical results from gene therapy candidate BBP-631 in a poster entitled “Durable CYP21A2 Gene Therapy in Non-Human Primates for Treatment of Congenital Adrenal Hyperplasia.” Throughout the study, a total of 20 non-human primates (NHPs) were treated with BBP-631 at one of three intravenous (IV) doses. Vector copy number and transgene mRNA expression in the adrenal glands were analyzed at 4 and 12 weeks post-dosing in the low- and medium-dose arms and at 12 and 24 weeks post-dosing in the high-dose arm. No dose-related adverse events were observed at any of the doses tested at any time point.

Overall, treatment with BBP-631 resulted in high vector copy number (VCN) and mRNA expression in the adrenal gland, suggesting strong tropism and uptake of BBP-631 for the adrenal gland. In the high-dose arm, VCNs were maintained between 12 and 24 weeks. Furthermore, mRNA levels increased between 4 and 12 weeks for the medium dose arm and were consistent between 12 and 24 weeks for the high dose arm. Researchers also saw dose-dependent increases in both VCNs and mRNA levels across the three doses tested.

“Durable expression of a vector in adrenal tissue has presented challenges for the development of gene therapies for adrenal indications,” said Clayton Beard, Ph.D., Senior Vice President, Research and Development at BridgeBio Gene Therapy. “The observed maintenance of the BBP-631 AAV5 vector for as long as 24 weeks in NHPs represents an important step in validating a gene therapy approach to treat 21-hydroxylase deficiency, and we anticipate filing our IND for this indication in 2020.”

Canavan Disease Experimental Therapy Amenable to IV Delivery

In a poster entitled “A Route of Administration Study of BBP-812, an AAV9-based Gene Therapy for the Treatment of Canavan Disease, in Juvenile Cynomolgus Macaques,” scientists from Aspa Therapeutics examine the uptake of and resulting DNA and mRNA expression for BBP-812, an experimental AAV9 vector, in three different administration routes: intravenous (IV), intrathecal (IT) or intracerebroventricular (ICV). Biodistribution of vector genomes and of transgene mRNA was evaluated throughout the central nervous system (CNS) at both 3 and 8 weeks post-administration. No dose-related adverse events were observed at any tested doses at either time period.

Using an IV route of administration, vector copy number and mRNA expression were not only detected broadly throughout the brain and spinal cord, but were generally detected at levels higher than when administered ICV or IT.

“Developing therapies for diseases like Canavan presents particular delivery challenges – namely getting the therapy into the central nervous system,” said Adam Shaywitz, M.D., Ph.D., Chief Medical Officer at BridgeBio Gene Therapy. “In this preclinical study, we observed that broad CNS delivery of our vector is possible using an IV route, which is far less invasive than the IT or ICV routes and therefore points to IV as the preferred route of administration in our clinical program for Canavan disease.”

Aspa Therapeutics is currently enrolling a natural history study of Canavan disease called CANinform. Interested individuals can find information about the study at TreatCanavan.com.

About Adrenas Therapeutics

Adrenas Therapeutics is a BridgeBio Pharma subsidiary developing a gene therapy treatment for congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency, a rare genetic disorder which affects the ability of the adrenal glands to properly function. People with CAH have an impaired ability to produce the hormone cortisol, which can result in life-threatening adrenal crises and significant morbidity throughout life.

About Aspa Therapeutics

Aspa Therapeutics is a BridgeBio Pharma subsidiary focused on developing a gene therapy to treat Canavan disease, an extremely rare genetic disease caused by an inherited mutation of the ASPA gene wherein children affected experience lack of head control, lack of muscle tone (often resulting in floppiness or spasticity) and seizures. Most children are not able to meet developmental milestones, are unable to crawl, walk, sit or talk, and pass away at a young age.

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. For more information, please visit www.bridgebio.com.

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 the clinical and therapeutic benefits of BBP-631 and BBP-812, the potential of BBP-631 to be a meaningful therapy for patients with congenital adrenal hyperplasia with 21-hydroxylase deficiency and the potential for BBP-812 to overcome the challenges associated with other gene therapies’ inability to target the CNS, Adrenas Therapeutics’ and Aspa Therapeutics’ clinical development plans, including their plans to file their respective INDs in 2020, and timing and completion of preclinical studies and regulatory submissions, 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, Adrenas Therapeutics’ and Aspa Therapeutics’ ability to continue its planned development and regulatory submissions for BBP-631 and BBP-812, respectively, 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.

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


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