- BridgeBio Pharma (NASDAQ:BBIO) posts early results from an ongoing Phase 2b proof-of-concept, open-label study of encaleret for the treatment of Autosomal Dominant Hypocalcemia Type 1 (ADH1), a rare genetic disorder.
- In the study, participants received sequential, increasing daily doses of encaleret starting at 30 mg while undergoing intensive safety monitoring and frequent blood and urine sampling for biochemical measures.
- Following five days of dosing with encaleret, blood calcium, parathyroid hormone, phosphorus and magnesium were within the normal range on average. Urinary calcium excretion was normal or undetectable in all participants.
- The tolerability and consistent mineral responses following encaleret administration in all six ADH1 trial participants demonstrates proof of concept that the therapy may be an effective option for ADH1, the company said.
- The company intends to meet with regulatory health authorities this year to discuss potential paths to registration prior to initiation of a Phase 3 registrational study.
- Shares up more than 3% premarket.