SRPTSarepta Therapeutics Inc (NASDAQ:SRPT) announced the first patient dosed in the phase III clinical trial of SRP-4045 and SRP-4053 in patients with Duchenne muscular dystrophy amenable to exon 45 or 53 skipping.

“We are excited to announce the first patient dosed in our ESSENCE trial of SRP-4045 and SRP-4053, for DMD patients amenable to Exon 45 and 53 skipping “ said Edward Kaye, Sarepta’s chief executive officer. “It is our goal to treat as many patients amenable to exon skipping as possible, and therefore is important to advance our clinical pipeline candidates. We have made great effort to develop a meaningful clinical trial, using our learnings from our previous clinical programs and observations of the natural history of Duchenne.”

The Phase III study, ESSENCE, is a double-blind, placebo-controlled, multi-center study to evaluate the efficacy and safety of SRP-4045 and SRP-4053. Eligible patients with out-of-frame deletion mutations amenable to exon 45 or 53 skipping will be randomized to receive once weekly intravenous (IV) infusions of 30 mg/kg SRP-4045 or 30 mg/kg SRP-4053 respectively (combined-active group, 66 patients) or placebo (33 patients) for up to 96 weeks (the placebo-controlled period of the trial). This will be followed by an open label extension period in which all patients will receive open-label active treatment for up to 96 weeks.

The study will enroll approximately 99 patients aged 7 to 13 years, inclusive, with a minimum target of 45 patients amenable to exon 45 skipping and 45 patients amenable to exon 53 skipping.

Twice as many patients will receive active treatment as will receive placebo. Approximately 66 patients will be randomized to receive active treatment with either SRP-4045 or SRP-4053 (depending on deletion mutation), and 33 patients will be randomized to receive placebo. (Original Source)

Shares of Sarepta closed today at $62.24, up $3.14 or 5.31%. SRPT has a 1-year high of $63.73 and a 1-year low of $8.00. The stock’s 50-day moving average is $32.78 and its 200-day moving average is $22.96.

On the ratings front, Sarepta has been the subject of a number of recent research reports. In a report released today, JMP analyst Liisa Bayko maintained a Buy rating on SRPT, with a price target of $90, which represents a potential upside of 45% from where the stock is currently trading. Separately, on the same day, RBC’s Simos Simeonidis reiterated a Buy rating on the stock and has a price target of $108.

According to, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Liisa Bayko and Simos Simeonidis have a total average return of 10.1% and -33.7% respectively. Bayko has a success rate of 56% and is ranked #416 out of 4185 analysts, while Simeonidis has a success rate of 20% and is ranked #4063.

Overall, 3 research analysts have assigned a Hold rating and 11 research analysts have given a Buy rating to the stock. When considering if perhaps the stock is under or overvalued, the average price target is $72.50 which is 16.5% above where the stock opened today.

Sarepta Therapeutics, Inc. is a biopharmaceutical company, which is engaged in the discovery and development of unique RNA-based therapeutics for the treatment of rare and infectious diseases. The company is primarily focused on rapidly advancing the development of its potentially disease modifying duchenne muscular dystrophy drug candidates.