PTC Therapeutics Inc. (NASDAQ: PTCT) watched its shares make a massive gain early on Monday after the firm presented updated preliminary results from its study investigating risdiplam (RG7916) in babies with Type 1 spinal muscular atrophy (SMA), at the 22nd Annual SMA Researcher Meeting. The SMA program is a collaboration between PTC, Roche and the SMA Foundation.
The data come from the first part of the study and demonstrated that at Day 182, over 90% of the babies achieved a greater than a four-point increase in the Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) score compared to baseline.
As for the specific results, in the FIREFISH study the median increases in CHOP-INTEND scores were 5.5 points at Day 56, 12.5 points at Day 119 and 14.0 points at Day 182 of treatment. The proportion of patients achieving greater than a four-point increase from baseline in CHOP-INTEND scores was 75% at Day 56, 94% at Day 119 and 91% at Day 182.
The CHOP-INTEND data were further supported by video footage presented demonstrating antigravity movements, the ability to control their head, roll or sit in babies participating in FIREFISH. The second part of the pivotal FIREFISH study is ongoing.
Stuart W. Peltz, Ph.D., CEO of PTC Therapeutics, commented:
We are delighted that up to 6.5-fold increase of protein production has translated into clinical impact for these babies in the FIREFISH study. The survival data and CHOP-INTEND scores are very promising, since babies with Type 1 SMA typically do not experience functional motor milestone improvement based on natural history. We look forward to sharing updates for the programs as the data further develop at upcoming medical meetings.
Shares of PTC Therapeutics were last seen up about 22.8% at $46.10, with a consensus analyst price target of just $24.20 and a 52-week range of $14.56 to $52.95.