With new results, Sarepta's 2nd gene therapy holds steady – BioPharma Dive

Sarepta has three marketed products treating Duchenne, plus a beefy pipeline of 39 experimental RNA-based programs and genetic medicines for rare diseases. One towers above them all, however: SRP-9001. Its clinical trial setback in January cut the company’s valuation in half as a rival treatment from Pfizer appeared to be taking a lead.
With the path to market looking longer for SRP-9001, other pipeline projects may figure more prominently in Sarepta’s outlook, and the data from the follow-up project, known as SRP-9003, may be reassuring to investors.
SRP-9003 treats a type of muscular dystrophy called Limb-girdle that particularly affects the arms and legs. To do so, it helps replenish the deficiency of a protein called beta-sarcoglycan, the lack of which is thought to trigger the disease.
The first three patients in Sarepta’s early-stage trial were infused with a low dose of the gene therapy, and after 18 months of treatment had improved their score by 5.7 points on a 20-item test that measures their ability to do such tasks as stand up from a chair or stand briefly on one foot. That improvement was sustained at two years, Sarepta reported Thursday.
In a research note, SVB Leerink’s Schwartz wrote that untreated patients would have been expected to decline by 4.6 points over the same time period. Biological measures, such as expression of the beta-sarcoglycan protein, also showed positive signs, although they were short of expression seen in the healthy population.
Three higher-dose patients also showed improvements at one year. Their four-point increase on the functional test was short of the six points seen in the lower-dose group at 12 months, although Schwartz noted that biomarkers indicated the high-dose group may have been less disabled when they received the gene therapy.
The company hasn’t seen any new safety signals. One adverse event was reported in an earlier data release, a patient who was dehydrated from vomiting.
Schwartz wrote that the data could set the stage for a study that could be submitted to regulators to support approval. There are no treatments for limb-girdle muscular dystrophy, so a regulatory pathway will need to be discussed with the Food and Drug Administration, as well as a quality assessment of the batch of gene therapy to be used in that trial. That could make the path forward for limb-girdle gene therapy different than the one Sarepta’s traversing in Duchenne, where there are at least some marketed drugs for the condition.
The next study should begin this year, Schwartz wrote.
Follow Jonathan Gardner on Twitter
Biogen priced its newly approved medicine Aduhelm at an average cost of $56,000 a year, adding affordability to other barriers patients may face. 
Results published in The New England Journal of Medicine offer the first clinical evidence that CRISPR gene editing inside the body can be safe and effective, a culmination of years of scientific research.  
Subscribe to BioPharma Dive to get the must-read news & insights in your inbox.
Topics covered: Pharma, biotech, FDA, gene therapy, clinical trials, drug pricing and much more.
Discover announcements from companies in your industry.
Biogen priced its newly approved medicine Aduhelm at an average cost of $56,000 a year, adding affordability to other barriers patients may face. 
Results published in The New England Journal of Medicine offer the first clinical evidence that CRISPR gene editing inside the body can be safe and effective, a culmination of years of scientific research.  
The free newsletter covering the top industry headlines
Topics covered: Pharma, biotech, FDA, gene therapy, clinical trials, drug pricing and much more.
The free newsletter covering the top industry headlines
Topics covered: Pharma, biotech, FDA, gene therapy, clinical trials, drug pricing and much more.

source