PTC gains on early data for SMA candidate

PTC Therapeutics Inc. (NASDAQ:PTCT) added $2.37 (10%) to $27.03 on Monday after it and partner Roche (SIX:ROG; OTCQX:RHHBY) reported preliminary data from the pivotal Phase II FIREFISH trial of RG7916 (RO7034067) to treat infants with spinal muscular atrophy (SMA) type 1. In 13 evaluable patients who received once-daily oral RG7916 in the trial’s dose-finding first part, no patient has lost the ability to swallow and no patient has required a tracheostomy or reached permanent ventilation. Data were presented at the International Scientific Congress on Spinal Muscular Atrophy in Krakow.

RG7916 was well tolerated at all doses with no treatment-related safety findings leading to withdrawal reported. There was also no evidence of retinal toxicity that was previously seen in preclinical monkey studies of the product. PTC said there were two deaths, including a patient with a respiratory tract viral infection on study day 21 and a patient with cardiac and respiratory arrest on study day 236...