Sarepta Therapeutics Inc. (NASDAQ:SRPT) has announced its two-year study follow up results from four Duchene muscular dystrophy clinical study subjects that received SRP-9001.
SRP-9001 showing a good safety profile after two years of treatment
SRP-9001 is the company’s experimental gene transfer therapy. It is for delivering its micro-dystrophin-encoding gene to muscle tissues for targeted micro-dystrophin protein production. The company presented results at the 25th World Muscle Society International Annual Congress. The results showed that after two years of a one-time SRP-9001 infusion, study participants demonstrated a 7.0 point mean improvement on the North Star Ambulatory Assessment relative to baseline.
Doug Ingram, the CEO and President of Sarepta, said that they are encouraged by the safety profile as well as the enduring treatment response shown so far with the SRP-9001 gene transfer therapy. He added that the consistent results and the function improvement shown over two years boost confidence if the company ahead of Study 102 results. Study 102 is a double-blind, randomized, placebo-controlled SRP-9001 study. Ingram said that the company is working with urgency to deliver the potentially transformative therapy to patients as soon as possible.
Study 101 showed favorable tolerability of the gene therapy
In Study 101 open-label trial, the company enrolled four ambulatory subjects aged between 4 and 7 who were given SRP-9001 infusion at 2×1014 vg/kg dose. Results showed favorable tolerability of the therapy in the subject over the two years. There are only moderate or mild adverse events that occurred within three months of treatment. Interestingly there were no serious adverse events reported or proof of complement activation.
After 90 days of study, participants conformed vector transduction and demonstrated functional improvement based on the NSAA scale as well as reduced levels of creatine kinase. The subjects showed 5.5 points mean increase from baseline to one year following treatment and also 7.0 points mean increase after two years. It is important to note that the NSAA is a certified scale developed for measuring functional motor capabilities in ambulant kids with Duchenne muscular dystrophy, and scores range from 0-34.