(Reuters) -BridgeBio Pharma said on Wednesday that its experimental drug for a rare genetic condition that causes low blood calcium met all the main and secondary goals in a late-stage study, sending its shares up 9.1% in premarket trading.
In a study involving 70 patients, 76% achieved 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.
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.
TD Cowen analysts last week said “encaleret in ADH1 has the highest probability of technical success across BridgeBio’s late stage candidates” and estimated peak global sales of $750 million by 2035.
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)
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