BioCentury
ARTICLE | Clinical News

PTC spikes on interim pivotal data for oral SMA candidate

June 18, 2018 10:46 PM UTC

PTC Therapeutics Inc. (NASDAQ:PTCT) gained $10.33 (28%) to $47.88 after reporting updated interim data from part 1 of the pivotal Phase II/III FIREFISH trial showing that oral risdiplam (RG7916) improved motor development scores in infants with spinal muscular atrophy (SMA) type 1.

In 11 evaluable patients in part 1, risdiplam increased Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) scores -- a measure of motor milestone development -- by a median of 14 points from baseline at day 182. Of those patients, 91% had a greater than four-point increase in CHOP-INTEND score from baseline at day 182. ...