BridgeBio-Backed CoA Therapeutics Challenges Retrophin In Genetic Disorder PKAN
Emerging Company Profile: CoA Therapeutics plans to start a Phase I study of a CoA modulator in the rare, genetic disorder PKAN during the first quarter of 2020.
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Since raising $135m in 2017 to address rare, monogenic diseases, BridgeBio has launched or purchased 17 companies and built a pipeline of 30-plus candidates. Scrip checked in as the company nears commercialization.
Fosmetpantotenate missed all endpoints in a Phase III trial, so Retrophin will shelve the candidate for the rare disease PKAN. Now, the biotech turns focus to Phase III sparsentan in a pair of indications.
BridgeBio's hub-and-spoke model is similar to the Roivant and Fortress strategies, but with a focus on drug candidates for genetic diseases in need of a bridge to future funding.