4:23 PM
Jun 13, 2018
 |  BC Extra  |  Company News

BridgeBio launches rare genetic disorder subsidiary

BridgeBio Pharma LLC (Palo Alto, Calif.) launched another subsidiary this week, forming CoA Therapeutics Inc. to develop small molecules which increase coenzyme A levels to treat rare genetic disorders.

CoA CEO Shafique Virani told BioCentury that the compounds directly target pantothenate kinase 1 (PANK1) and PANK3 to up-regulate coenzyme A. The candidates were in-licensed from St. Jude Children's Research Hospital (Memphis, Tenn.).

CoA's lead compound is slated to begin clinical testing next year to treat pantothenate kinase-associated neurodegeneration (PKAN). BridgeBio, the sole investor in CoA, will help fund the program. Financial details were not disclosed.

Virani said the company plans to also evaluate its candidates in metabolic disorders.

According to BioCentury's BCIQ database, there are at least two candidates in development to treat PKAN.

Retrophin Inc. (NASDAQ:RTRX) expects top-line data in 2H19 from the Phase III FORT study of fosmetpantotenate (RE-024) in the indication. The product, a phosphopantothenate molecule that restores disruption in coenzyme A biosynthesis caused by a mutant PANK2 gene, has Fast Track and Orphan Drug designation in the U.S.

P-PantSAc (TM-1803) from TM3 Therapeutics B.V. (Leiden, the Netherlands) is in preclinical testing. The candidate is a metabolite in the coenzyme A biosynthesis pathway that overcomes PANK2 enzyme deficiency and has Orphan Drug designation in the EU.

On Monday, BridgeBio announced the launch of subsidiary Origin Biosciences Inc., which gained exclusive, global rights to ALXN1101, a cyclic pyranopterin monophosphate replacement therapy, from Alexion Pharmaceuticals Inc. (NASDAQ:ALXN) (see BioCentury Extra, June 11).

BridgeBio in-licenses preclinical and clinical stage assets from academia and pharma and houses them in individual subsidiaries (see BioCentury, Sept. 15, 2017).

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