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Courtesy of Sarepta Therapeutics
Therapeutic Zarephath share positive results of its ongoing study SRP-9001-102 (study 102) on the efficacy of delandistrogen moxeparvovec in the treatment of Duchenne muscular dystrophy (DMD).
SRP-9001 is an investigational gene transfer treatment that works by delivering the gene encoding micro-dystrophin to muscle tissue with the goal of enhancing the production of micro-dystrophin proteins. The study involved 41 participants aged four to seven years diagnosed with a rare and fatal genetic neuromuscular disease. They were evaluated in a double-blind, randomized 1: 1, placebo-controlled trial at 12 and 48 weeks for expression of microdystrophin and changes in the North Star Ambulatory Assessment (NSAA) score, respectively.
The NSAA is a 17-item assessment procedure that measures functional motor skills in ambulatory patients with Duchenne. It is considered one of the primary endpoints for this trial because it helps researchers track disease progression and treatment results.
The latest results, reflecting part 2 of the study, showed that participants in the placebo crossover group (from part 1) scored 2 more points on the NSAA at week 48. The patient safety profile is also remained consistent with the results of Part 1, with no deaths, study interruptions and serious treatment-related adverse events. Common side effects were also the same as in the first study, which included vomiting. Study 102 is still ongoing at the time of writing.
DMD affects one in 3,500 to 5,000 men worldwide, and symptoms usually appear from infant to toddler. It is caused by a mutation or change in the gene that controls dystrophin production, which eventually results in difficulty walking or standing from a sitting position and muscle weakness. Most patients will need to use a wheelchair full time by the time they become adolescents, which over time results in the loss of the ability to perform activities of daily living.
“The results of Study 102, Part 2 add to the body of evidence for SRP-9001 generated to date – with promising results in several clinical trials and over 80 patients treated, encompassing a wide range of phenotypes. as well as the oldest and heaviest Duchenne patients to date with full-body AAV gene therapy infusion, ”said Doug Ingram, President and CEO of Sarepta, in a statement.
“The totality of the results that we have seen in our multiple trials reinforce our confidence in the potential benefits of this therapy in modifying disease and reinforce our belief in the likelihood of success of EMBARK, our large global phase III placebo-controlled study. currently underway and dosing, ”Ingram added.
Sarepta has announced its intention to market SRP-9001 in the United States after obtaining approval from the United States. Food and drug administration. The company has partnered with rock in December 2019 to combine the latter’s global reach with its gene therapy candidate for Duchenne, with the goal of expanding its presence outside of the United States. National Children’s Hospital.
Details of the interim results were presented at the 40th annual JP Morgan Healthcare conference.