BridgeBio’s rare calcium disorder drug scores late-stage trial win

Audio By Carbonatix

(Corrects last name of BridgeBio executive to Outten, from Outen, in paragraph 7)

By Padmanabhan Ananthan

(Reuters) -BridgeBio Pharma’s experimental drug for a rare genetic condition that causes low blood calcium met all the main and secondary goals in a late-stage study, the company said on Wednesday.

In the study, 76% of 67 patients achieved the target levels for both blood and urine calcium after 24 weeks of treatment with encaleret, compared to only 4% when the same patients were on standard treatment earlier in the trial.

Encaleret is a potential treatment for autosomal dominant hypocalcemia type 1 (ADH1), that causes low blood calcium and high urine calcium levels in the body, leading to muscle cramps, spasms, and sometimes kidney issues.

“We plan to move expeditiously to provide this medicine to patients as broadly as possible,” BridgeBio CEO Neil Kumar said in a conference call.

Ninety-one percent of patients taking encaleret increased their natural parathyroid hormone levels to above the lower normal limit, compared to just 7% of those on standard-of-care.

The data positions BridgeBio at the forefront of being the first targeted therapy for this rare genetic disorder to potentially get regulatory approval.

“Our engine is built, our playbook is proven, and we are well prepared to deliver another successful blockbuster launch,” said BridgeBio’s commercial head Matt Outten.

BridgeBio’s flagship drug Attruby, to treat a deadly heart condition known as transthyretin amyloid cardiomyopathy (ATTR-CM), had sales of $71.5 million in the second quarter of 2025.

Evercore ISI analyst Cory Kasimov described the recent encaleret data, along with the previous late-stage readout of its experimental muscle disorder drug, BBP-418, for limb-girdle muscular dystrophy, as a “clear win” that further reinforces the company’s trajectory toward becoming a multi-product biotech firm.

Current standard treatment for ADH1, which has no U.S. FDA approved-therapies, involves calcium supplements and active vitamin D analogs. However, these therapies often increase calcium levels in the urine, which can lead to kidney complications.

Encaleret was well-tolerated with no treatment-related discontinuation, the company said, adding that it plans to file its marketing application for the drug in the first-half of 2026.

(Reporting by Padmanabhan Ananthan in Bengaluru; Editing by Sahal Muhammed and Shailesh Kuber)