Benzinga | Nov 17, 2020 08:13AM EST

PTC Therapeutics Announces That PTC518 Has Entered Into Phase 1 Clinical Trial For Huntington's Disease Program

- Initial clinical trial results expected in 1H 2021 -



SOUTH PLAINFIELD, N.J., Nov. 17, 2020 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced the initiation of a Phase 1 clinical trial to evaluate PTC518 in healthy volunteers. PTC518 was identified from PTC's splicing platform and is being developed for the treatment of Huntington's disease (HD). PTC518 is an orally bioavailable molecule with broad central nervous system and systemic distribution and has been designed to target Huntingtin protein expression with high selectivity and specificity. PTC518 has favorable pharmaceutical properties and has demonstrated uniform lowering of the Huntingtin protein throughout the brain in animal models. Currently there are no approved disease modifying therapies for HD.