BridgeBio Pharma To Announce Proof-Of-Concept Data from ADH1 at the Endocrine Society’s 2021 Annual Meeting

SAN FRANCISCO, Feb. 24, 2021 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO), a clinical-stage biopharmaceutical company founded to discover, create, test and deliver meaningful medicines for patients with genetic diseases and cancers with clear genetic drivers, today announced that early results from an ongoing Phase 2 proof-of-concept study of encaleret for the treatment of Autosomal Dominant Hypocalcemia Type 1 (ADH1) will be shared at the upcoming Endocrine Society’s 2021 Annual Meeting (ENDO 2021) taking place virtually from March 20 - 23.

The data are featured in an ePoster presentation titled ‘The Effects of Encaleret (CLTX-305) on Mineral Physiology in ADH1 Demonstrate Proof-of-Concept: Early Results from an Ongoing Phase 2B, Open-Label, Dose-Ranging Study.’ If the development program is successful, encaleret could be the first approved therapy for ADH1, a condition caused by gain of function variants in the calcium-sensing receptor gene estimated to be carried by 12,000 individuals in the United States.

Full ePoster presentation details are listed below, and the full preliminary program is available online at the ENDO 2021 . The presentations will be on display in ENDO 2021’s virtual poster hall starting on March 20 at 11:00 a.m. ET.

BridgeBio will host an investor webcast on March 22 at 8:00 a.m. ET to discuss the proof-of-concept data for encaleret in ADH1.

Additionally at ENDO 2021, BridgeBio will present clinical study designs for its study of low-dose infigratinib, an FGFR1-3 inhibitor, for children with achondroplasia, the most common form of genetic short stature, and for its investigational AAV5 gene therapy candidate for Congenital Adrenal Hyperplasia (CAH). CAH is one of the most prevalent genetic diseases potentially addressable with AAV gene therapy.

BridgeBio ePoster Presentation Details:

The Effects of Encaleret (CLTX-305) on Mineral Physiology in ADH1 Demonstrate Proof-of-Concept: Early Results from an Ongoing Phase 2B, Open-Label, Dose-Ranging Study

Presenter: Rachel Gafni, M.D., Senior Physician of Skeletal Disorders and Mineral Homeostasis of the National Institute of Dental and Craniofacial Research, part of the National Institutes of Health

Poster Session & Number: P08. Parathyroid and Rare Bone Disorders, Abstract #8545

A Phase 2B, Open-Label, Dose-Ranging Study of Encaleret (CLTX-305) in ADH1

Presenter: Rachel Gafni, M.D., Senior Physician of Skeletal Disorders and Mineral Homeostasis of the National Institute of Dental and Craniofacial Research, part of the National Institutes of Health

Poster Session & Number: P08. Parathyroid and Rare Bone Disorders, Abstract #7288

Infigratinib in Children with Achondroplasia (ACH): Design of PROPEL2 – A Phase 2, Open-Label, Dose-Escalation and Dose-Expansion Study

Presenter: Ravi Savarirayan, M.D., Ph.D., Clinical Geneticist and Group Leader of Skeletal Biology and Disease at Murdoch Children’s Research Institute

Poster Session & Number: P34. Disorders of Puberty, Abstract #6897

Design of a Phase 1/2 Open-Label, Dose-Escalation Study of the Safety and Efficacy of Gene Therapy in Adults with Classic Congenital Adrenal Hyperplasia (CAH) Due to 21-hydroxylase Deficiency Through Administration of an Adeno-Associated Virus (AAV) Serotype 5-Based Recombinant Vector Encoding the Human CYP21A2 Gene

Presenter: Deborah Merke, M.D., Senior Investigator, Chief of Pediatric Service and Head of Congenital Disorders at the NIH Clinical Center

Poster Session & Number: P05. Adrenal – Clinical Research Studies, Abstract #8640

Webcast Information

BridgeBio will host a conference call and simultaneous webcast to share updates on the encaleret proof-of-concept data in ADH1 on March 22 at 8:00 a.m. ET. To access this call, dial (800) 379-2666 (U.S.) or (409) 937-8964 (International). Conference ID: 7371879. A link to the webcast may be accessed from the event calendar page of BridgeBio’s website at . A replay of the conference call and webcast will be archived on the Company's website and will be available for at least 30 days following the event. 

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 20 development programs includes product candidates ranging from early discovery to late-stage development. For more information, please visit .

BridgeBio Pharma Forward Looking Statements

This press release contains forward-looking statements. Statements we make in this press release may include statements that 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 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 the preclinical and clinical development plans, clinical trial designs, clinical and therapeutic potential, and strategy of our product candidates, including, but not limited to: the unknown future impact of the COVID-19 pandemic delay on certain clinical trial milestones and/or our operations or operating expenses;; the timing and success of our planned preclinical and clinical development of our development programs, including each of infigratinib, BBP-631 and encaleret; the timing and success of any such continued preclinical and clinical development and planned regulatory submissions, including for each of infigratinib, BBP-631 and encaleret; the potential therapeutic and clinical benefits of each of infigratinib, BBP-631 and encaleret; the potential size of the target patient populations for each of infigratinib, BBP-631 and encaleret; the potential for encaleret to be the first approved therapy for ADH1; our expected runway for cash, cash equivalents and marketable securities; 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 number of risks, uncertainties and assumptions, including, but not limited to: the success of clinical trials, regulatory filings, approvals and/or sales; despite having ongoing interactions with the FDA or other regulatory agencies, the FDA or such other regulatory agencies may not agree with our regulatory approval strategies, components of our filings, such as clinical trial designs, conduct and methodologies, or the sufficiency of data submitted; potential adverse impacts due to the global COVID-19 pandemic such as delays in regulatory review, manufacturing and supply chain interruptions, adverse effects on healthcare systems and disruption of the global economy; and those risks set forth in the Risk Factors section of our most recent quarterly or annual periodic report filed with the SEC and our other SEC filings. Moreover, BridgeBio 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 BridgeBio’s 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. Except as required by applicable law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

Contact:

Grace Rauh

BridgeBio Pharma, Inc.



(917) 232-5478

Source: BridgeBio Pharma, Inc.