On 16th June, PTC Therapeutics, Inc. presented new interim data from part 1 of the Firefish clinical trial which is investigating risdiplam in babies with type 1 SMA. The compound is showing some fantastic results, with part 2 of the study still ongoing. This is a very exciting time for SMA research.
At day 182, the average improvement was 14 points in CHOP-INTEND score and over 90% of the babies achieved a greater than 4 point increase. The results are supported by video footage showing anti gravity movements, the ability to control their head, rolling and sitting.
“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,” said Stuart W. Peltz, Ph.D. Chief Executive Officer of PTC Therapeutics. “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.
Considering that part 1 of the Firefish study was only supposed to be a dose finding study, it is exciting to see that motor function increase was also found. When you consider that all participants received their 1st dose after the age of 5 months and that clinical benefit has been seen at this early stage, it is going to be a very exciting time to see what this oral compound can achieve next.
In addition, risdiplam has been well tolerated at all dose levels and to date there have been no drug-related safety findings leading to withdrawal. Also, no babies have required a tracheostomy or permanent ventilation since study initiation and no baby has lost the ability to swallow.
Risdiplam is a new name of RG7916, an experimental oral molecule for SMA being developed by Roche, PTC Therapeutics and SMA Foundation.
More updates will follow as this study progresses.
