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BridgeBio Pharma Announces New England Journal of Medicine Publication of Positive Encaleret Proof-of-Concept Phase 2b Results in Patients with Autosomal Dominant Hypocalcemia Type 1 (ADH1)
- In our study, encaleret restored physiologic mineral homeostasis in 13 participants with ADH1, specifically correcting hypocalcemia and reducing
About this update from Bridgebio Pharma, Inc.
[{"type":"text","content":"- In our study, encaleret restored physiologic mineral homeostasis in 13 participants with ADH1, specifically correcting hypocalcemia and reducing hypercalciuria - Rapid and sustained impact of encaleret on mineral homeostasis was observed, specifically in the normalization of blood calcium, urine calcium and parathyroid hormone (PTH) - No serious adverse events were reported with encaleret and no treatment discontinuations or study withdrawals occurred during the trial - The Phase 3 CALIBRATE trial of encaleret in patients with ADH1 is ongoing in six countries with topline data expected in the first half of 2024 - If approved, encaleret could be the first therapy specifically indicated for the treatment of ADH1 PALO ALTO, Calif., Oct. 10, 2023 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, announced that the proof-of-concept Phase 2b data evaluating the effects of orally-administered encaleret on mineral homeostasis in patients with ADH1 were published in the New England Journal of Medicine in partnership with the National Institutes of Health (NIH). “Conventional therapy for ADH1 includes raising the blood calcium levels with calcium supplements and activated vitamin D, taken in multiple doses throughout day. But this burdensome regimen may also increase urine calcium levels above normal, which can damage the kidney, leading to kidney failure in worst-case scenarios. People with ADH1 need better treatments, so they are not constantly walking on a tightrope. It’s been rewarding to observe the robust response in our study participants, some of whom I’ve known for almost 20 years, to this investigational medication that directly targets the underlying cause and appears to restore the calcium balance back towards normal,” said Rachel Gafni, M.D., the principal investigator of the study and a senior research physician in the National Institute of Dental and Craniofacial Research at the NIH. As part of the Phase 2b study, participants completed one or two 5-day inpatient dose-ranging periods, followed by a 24-week outpatient period. Encaleret was administered twice daily with doses adjusted to achieve normal albumin-corrected blood calcium levels and participants stopped taking calcium and vitamin D supple...