Sarepta Therapeutics Inc (NASDAQ: SRPT) is having an overwhelmingly strong day in the market today, and for good reason. The company announced positive preliminary results from a clinical trial assessing a gene therapy candidate. Of course, the data was positive, exciting investors and sending the stock toward the top. Today, we’ll talk about:
- The data;
- what we’re seeing from SRPT as a result; and
- what we’ll be watching for ahead.
SRPT Climbs On Clinical Data
As mentioned above, Sarepta Therapeutics is having an incredibly strong day in the market today after releasing data. In a press release published this morning, the company announced that at its R&D DAy, Jerry Mendell, M.D. of Nationwide Children’s Hosptial presented positive clinical results. The results came from a Phase 1/2a gene therapy clinical trial in which SRPT is assessing AAVrh74.MHCK7.micro-Dystrophin as a treatment for Duchenne muscular dystrophy (DMD).
In the release SRPT said that all patients included in the study showed robust expression of transduced micro-dystrophin. The transduced micro-dystrophin is properly localized in the muscle sarcolemma and was measured by immunohistochemistry. Mean gene expression came in at 76.2% and mean intensity of the fibers came in at 74.5% compared to normal control.
Following the treatment, SRPT said that all biopsies showed robust levels of micro-dystrophin as measured by Western blot. In fact, these biopsies showed a mean of 38.2% when comperd to normal using the company’s method, or 53.7% when compared to normal pursuant to Nationwide Childrend’s quantification of Sarepta’s method that adjust for fat and fibrotic tissue.
Also, all patients showed a significant decrease of serum creatine kinase (CK) levels, with a mean reduction in CK of more than 87% at day 60. Considering that CK is associated with muscle damage in patients with DMD, the drastic reduction is great news. On a safety side of the coin, Sarepta Therapeutics said that no serious adverse events were observed. Two patients did have elevated gamma-glutamyl transferase (GGT) that were resulved with increased steroids that brought these levels back to baseline within a week. In a statement, Doug Ingram, President and CEO at SRPT, had the following to offer:
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.
What We’re Seeing From The Stock
One of the first lessons that we learn when we start to work in the market is that the news causes moves. In the case of Sarepta Therapeutics, the news proved to be overwhelmingly positive. The strong data further validate the company’s innovation in the gene therapy space. So, it’s no surprise to see that the stock is flying in the market this morning. Currently (10:55), SRPT is trading at $161.16 per share after a gain of $55.92 per share or 53.14% thus far today.
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What We’ll Be Watching For Ahead
Moving forward, the CNA Finance team will continue to keep a close eye on SRPT. In particular, we’re interested in following the story surrounding the company’s continued work and innovation in the gene therapy space. Nonetheless, we’ll continue to follow the story closely and bring the news to you as it breaks!
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