Rare Daily Staff
BridgeBio Pharma said it launched CoA Therapeutics, a biopharmaceutical company that will develop small-molecules designed to treat genetic disorders driven by a deficiency in co-enzyme A.
CoA Therapeutics’ lead compound is expected to enter the clinic in 2019 with a planned initial focus on pantothenate kinase-associated neurodegeneration, or PKAN. PKAN is a genetic disorder caused by mutations in the pantothenate kinase 2 (PANK2) gene. The PANK2 enzyme plays a critical role in the synthesis of CoA, which is crucial in energy metabolism and implicated in a number of developmental disorders. In the typical form of PKAN, symptoms present before the age of 10, and patients may rapidly experience neuronal degeneration, causing problems with movement, speech and vision.
Patients with PKAN either have a complete absence or a significant deficiency of the PANK2 enzyme, which may lead to reduced CoA levels in the brain. There are currently no treatments approved for PKAN. The prevalence of PKAN is estimated to be three in every 1,000,000 people.
The CoA Therapeutics’ lead compounds were obtained under a license from St. Jude Children’s Research Hospital where they were discovered and developed.
“We believe that our novel approach, using a highly brain-penetrant compound to directly target enzyme activity in neurons, can safely increase CoA levels, ease patients’ symptoms and make a meaningful difference in their quality of life,” said Shafique Virani, CEO of CoA “Our novel approach also holds promise for other diseases with defects in CoA metabolism, including the organic acidemias.”
The company also named Adam Shaywitz, CMO-in-residence at BridgeBio, as chief medical officer at CoA. Shaywitz was most recently executive director in clinical sciences at BioMarin Pharmaceuticals where he was involved in the design and planning of a number of clinical studies in rare disease including serving as program lead for the natural history and clinical treatment studies in Sanfilippo Syndrome B.
June 13, 2018
Photo: Shafique Virani, CEO of CoA Therapeutics