BridgeBio launches another biotech, focused on gene disorders
- In its second company launch this week, BridgeBio Pharma announced the creation of subsidiary CoA Therapeutics to develop small molecule drugs for genetic disorders linked to coenzyme-A (CoA) deficiencies.
- CoA Therapeutics will be headed up by Shafique Virani, with Adam Shaywitz as CMO. CoA's leading drug candidates were initially discovered by researchers at St. Jude Children's Research Hospital.
- The initial target of unit's lead drug will be pantothenate kinase-associated neurodegeneration (PKAN), and the candidate is expected to move into clinical trials in 2019.
BridgeBio's business strategy is to acquire potential disease-modifying programs in rare and serious genetic disorders and create new subsidiaries to develop each of these assets. Earlier in June, BridgeBio launched a new subsidiary, Origin Biosciences, to develop Alexion's ALXN1101 (cyclic pyranopterin monophosphate) for molybdenum cofactor deficiency Type A, an ultra-rare autosomal recessive inborn error of metabolism.
BridgeBio now has 19 assets at preclinical to late stage development in oncology, cardiology, neurology, dermatology, endocrinology and other areas.
CoA Therapeutics is also targeting a rare, progressive, and debilitating childhood disorder. Pantothenate kinase-associated neurodegeneration, caused by a genetic mutation that leads to reduced coenzyme-A levels, affects around three people in every million. Patients show symptoms of rapid neuronal degeneration before the age of 10, affecting movement, speech and vision. There is no approved therapy on the market, and patients rely on symptomatic treatment that does not affect the root cause of the disorder.
"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. Our novel approach also holds promise for other diseases with defects in CoA metabolism, including the organic acidemias," said Shafique Virani, CEO of CoA, in a statement.
Few companies are developing treatment for PKAN. Retrophin is carrying out a pivotal Phase 3 trial of fosmetpantotenate. That compound crosses the blood-brain barrier where it restores CoA levels, and could be the first approved therapy targeting the underlying cause of PKAN. ApoPharma has assessed an oral solution of Ferriprox (deferiprone) in PKAN, and patients who took part in the study are continuing to receive the drug on a compassionate basis.
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