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Sarepta Therapeutics Posts Positive Results in Duchenne Muscular Dystrophy Study

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Sarepta Therapeutics shares traded 8% higher after the company reported its investigational gene therapy SRP-9001, used for treating Duchenne's Muscular Dystrophy, had "positive 12-week expression and safety results."

Developer and marketer of precision genetic medicines for rare diseases Sarepta Therapeutics Inc. (SRPT:NASDAQ) today announced "positive 12-week expression and safety results from the first 11 participants enrolled in Study SRP-9001-103, an open-label study known as ENDEAVOR being conducted in partnership with Roche."

The company stated that these latest results were collected from the first clinical study employing commercially representative material. The firm noted that in the ENDEAVOR study, "SRP-9001 demonstrated robust expression of micro-dystrophin and no new safety signals from prior studies, supporting its potentially differentiated profile for the treatment of Duchenne muscular dystrophy."

Sarepta's President and CEO Doug Ingram remarked "We are delighted by these seminal results from the ENDEAVOR Study, our first trial results with SRP-9001 made by our commercial-scale manufacturing process. These data show strong transduction of the micro-dystrophin gene, resulting in robust expression of the properly localized micro-dystrophin protein, and did so with no new or unexpected safety signals...Armed with these data, we will seek a meeting with the FDA with the goal of rapidly starting our registrational study."

The company advised that in the ENDEAVOR study, 20 patients ranging from four to seven years of age diagnosed with Duchenne's muscular dystrophy were given a single infusion of SRP-9001. The firm noted that after 12 weeks of treatment muscle biopsies were taken from the first 11 trial participants. Sarepta stated that the results showed that these treated patients "demonstrated robust transduction, with mean micro-dystrophin expression of 55.4% of normal, as measured by western blot.

The company further added that muscle dystrophin levels demonstrated a mean of 70.5% muscle fibers expressing micro-dystrophin at 12 weeks with a mean intensity at the sarcolemma of 116.9% compared to normal biopsies, as measured by immunofluorescence. The firm stated that the positive results were statistically significant compared to the respective baseline rates of 12.8% and 41.0% respectively and that mean vector genome copies per nucleus reached 3.87. The company also noted that during the ENDEAVOR trial, SRP-9001's safety profile was consistent with that observed in prior studies using clinical manufacturing process material. The firm added that including the initial 12-week period, each of the patients enrolled in the study will be followed closely for a total of five years.

The company described SRP-9001 (rAAVrh74.MHCK7.micro-dystrophin) as "an investigational gene transfer therapy intended to deliver the micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein."

The report listed that Sarepta is owns the global rights to develop and manufacture SRP-9001 and intends to market SRP-9001 in the U.S. after it obtains the required approval from the FDA. The company entered into a partnership agreement with Roche in 2019 to develop SRP-9001 for Duchenne's patients outside the U.S.

The firm explained that Duchenne muscular dystrophy (DMD) is caused by a change or mutation in the gene that encodes instructions for dystrophin. The rare and fatal neuromuscular genetic disease is found in about one in every 3,500-5,000 males globally. DMD typically shows its first signs in infants and toddlers who often display delays in developmental milestones, such as difficulty in walking, climbing or standing from a sitting position. By the teen years, most patients require full-time use of a wheelchair and the disease eventually progresses to profound loss of muscles preventing, them from carrying out even normal daily personal care. The majority of DMD patients usually succumb to the disease in their twenties or earlier.

Sarepta Therapeutics is based in Cambridge, Mass., and focuses its efforts on developing and commercializing medicine to treatments complex and rare genetic diseases. The company calls itself a leader in addressing both Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs). Overall, Sarepta has greater than 40 active development programs based upon its multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing. The firm is also making efforts to develop treatments for Charcot-Marie-Tooth disease and Mucopolysaccharidosis type IIIA (MPS IIIA), which is also known as Sanfilippo syndrome A.

Sarepta Therapeutics started the day with a market cap of around $6.0 billion with approximately 79.8 million shares outstanding and a short interest of about 8.9%. SRPT shares opened more than 5% higher today at $79.27 (+$4.14, +5.51%) over yesterday's $75.13 closing price. The stock traded today between $77.25 and $85.48 per share and closed for trading at $81.44 (+$6.61, +8.40%).


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