BridgeBio Pharma Announces Proof-of-Concept Data of Encaleret in ADH1 at the Endocrine Society’s 2021 Annual Meeting - Seite 2

Baseline and day 5 blood and urine calcium levels are summarized in the figure below:

https://www.globenewswire.com/NewsRoom/AttachmentNg/09f2eee6-0e25-4c52 ...

Throughout this initial period of dose escalation, encaleret was well-tolerated with no serious adverse events and no adverse events of moderate or severe intensity reported. Two participants experienced transient, asymptomatic hypophosphatemia which was the only treatment-related adverse event. The tolerability and consistent mineral responses following encaleret administration in all six ADH1 trial participants demonstrates proof of concept that encaleret may be an efficacious therapy option for ADH1.

The Company intends to meet with regulatory health authorities in 2021 to discuss potential paths to registration prior to initiation of a Phase 3 registrational study in patients with ADH1. If the development program is successful, encaleret could be the first approved therapy option indicated specifically for the treatment of ADH1.

“A close collaboration between world-leading experts in calcium homeostasis at the National Institute of Dental and Craniofacial Research at the NIH and BridgeBio has made possible the development of encaleret for ADH1,” said Jonathan Fox, M.D., Ph.D., Chief Medical Officer of Calcilytix, an affiliate of BridgeBio that is focused on developing encaleret. “We look forward to working with regulators this year to define a potential path to approval for encaleret for the treatment of ADH1 and to exploring its potential use in patients with other forms of hypoparathyroidism.”

At ENDO 2021, BridgeBio also presented clinical study designs for the Phase 2b study of encaleret in ADH1; for its Phase 2 study of low-dose infigratinib, an FGFR1-3 inhibitor, for children with achondroplasia, the most common form of genetic short stature; and for its Phase 1/2 study of its investigational AAV5 gene therapy candidate for congenital adrenal hyperplasia (CAH). CAH is one of the most prevalent genetic diseases that could potentially be addressable with AAV gene therapy.

¹ Dershem et al., Amer Jour of Hum Genetics, 2020

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 [phone] (U.S.) or [phone] (International). Conference ID: 7371879. A link to the webcast may be accessed from the event calendar page of BridgeBio’s website at https://investor.bridgebio.com/. 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.