PTC Therapeutics Begins Clinical Study on Treatment

Shares of PTC Therapeutics were up Wednesday after the company said it started a clinical study evaluating the safety, tolerability and efficacy of oral small molecule RG7916 in infants with type I spinal muscular atrophy.

The company noted it initiated the clinical study together with Roche and the SMA Foundation under a joint development program for spinal muscular atrophy. The clinical study consists of two parts, and commencement of a pivotal portion of the study will trigger a $20 million milestone payment from Roche, PTC added.

SMA is described as a rare genetic disorder which is the leading inherited cause of mortality in infants and young children.

Leave a Comment