Sarepta Therapeutics Inc. (NASDAQ: SRPT) shares skyrocketed on Tuesday after the firm presented positive preliminary results from its midstage Duchenne muscular dystrophy (DMD) trial. Specifically, the results came from Sarepta’s Phase 1/2a gene therapy clinical trial assessing AAVrh74.MHCK7.micro-Dystrophin in individuals with DMD.
Essentially, all patients in the study showed robust expression of transduced micro-dystrophin, which is properly localized to the muscle sarcolemma, as measured by immunohistochemistry. The mean gene expression, as measured by percentage of micro-dystrophin positive fibers, was 76.2% and the mean intensity of the fibers was 74.5% compared to normal control.
One of the key highlights was that all patients showed significant decreases of serum creatine kinase (CK) levels, with a mean reduction of CK of over 87% at Day 60. CK is an enzyme associated with muscle damage, and patients with DMD uniformly exhibit high levels of CK.
No serious adverse events were observed in the study.
Doug Ingram, Sarepta’s president and CEO, commented:
As a genetic medicine company, our goal is to work with the world’s leading clinicians and scientists to advance scientific discoveries to the clinic and, ultimately, to therapies that profoundly improve and extend the lives of those living with Duchenne muscular dystrophy and other rare, fatal diseases. Since the discovery of the dystrophin gene in 1986, scientists, clinicians, patient advocates and the biotech ecosystem have tirelessly searched for ways to restore or replace dystrophin and rescue boys with DMD from the damage and early death. Dr. Mendell’s results, if confirmed in additional patients, studies, measures and time points, represent a monumental leap forward in the direction of our goal.
Shares of Sarepta were last seen up about 60% at $169.00, with a consensus analyst price target of $99.65 and a 52-week trading range of $32.36 to $176.50.