BridgeBio Pharma BBIO has dosed first patient in its Phase 1 clinical trial of BBP-671, an investigational oral therapy being developed for the potential treatment of conditions caused by coenzyme A (CoA) deficiencies.
The Phase 1 study is a single- and multiple-ascending dose study designed to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of BBP-671.
In this second part of the trial, up to eight propionic acidemia (PA) patients and eight methylmalonic acidemia (MMA) patients will receive BBP-671.
PA and MMA are rare metabolic disorders caused by mutations in genes that impact the development of enzymes that participate in amino acid metabolism, leading to life-threatening metabolic decompensations.
Uma Sinha, Chief Scientific Officer, commented: "We are excited by biomarker changes observed with BBP-671 in a preclinical study using a mouse model for PA, which were associated with improved survival. Individuals diagnosed with PA and MMA and their families currently have no treatment options beyond dietary management and liver and/or kidney transplant, and we hope that BBP-671 may represent a novel therapeutic strategy for these diseases."
Initial data readout from the second part of this clinical trial are expected in the first half of 2023.
Price Action : BridgeBio shares are trading around 5 percent down at $10.10 on Thursday at the time of publication.
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