Sarepta Therapeutics Inc (NASDAQ: SRPT) is having an incredibly strong start to the trading session this morning, and for good reason. The company announced positive clinical data.
As you could imagine, the news excited investors who are pushing the stock for the top. Today, we’ll talk about:
- The new data;
- what we’re seeing from SRPT stock as a result; and
- what we’ll be watching for ahead.
SRPT Is Climbing On Positive Data
As mentioned above, Sarepta Therapeutics is having an incredibly strong start to the trading session this morning after announcing positive data. In a press release, the company provided the results from three Limb-girdle muscular dystrophy Type 2E clinical trial participants.
The participants that generated the data received MYO-101 as a treatment. The treatment is a novel gene therapy that’s intended to transduce skeletal and cardiac muscle. This is done with a gene that codes for the full-lenght, native beta-SG protein. LGMD2E is caused by the lack of this protein.
In the release, SRPT said that in Cohort 1 of the study, three participants ages 4 through 13, were treated with an infusion of MYO-101. Treatment biopsies were taken at approximately two months.
The company said that all three patients showed robust expression of transduced beta-SG. Moreover, this expression was properly localized in the muscle sarcolemma.
Mean protein expression, properly localized to the sacolemma of the muscle came in at 51%. The pre-defined measure of success for the trial was 20%, showing that the treatment performed at more than two aand a half times this rate.
SRPT also said that two of the participants had elevated liver enzymes. One of these was considered to be a serious adverse event. The company said that both events happened when participants were tapered off oral steriods. However, both also returned to baseline and symptoms resolved quickly following supplemental steroid treatment.
Also, there wer no other clinically significant laboratory findings. Finally, there were no decreases in platelet count observed.
In a statement, Jerry Mendell, M.D., Curran-Peters Chair of Pediatric Research at Nationwide Children’s Hospital and lead investigator on the trial, had the following to offer:
LGMD2E is a devastating neuromuscular disease, currently lacking any treatment options. Results in our first three clinical trial participants are consistent with what we have observed in preclinical models. We look forward to continuing this pivotal trial focused on development of MYO-101 for LGMD2E.
The above statement was followed up by Doug Ingram, President and CEO at SRPT. Here’s what he had to offer:
The positive results in our first MYO-101 cohort strengthen our resolve to build out our gene therapy engine with speed and purpose. Our gene therapy constructs have now produced high levels of expression of the missing protein of interest, and strong results in related biomarkers, in Duchenne and LGMD2E, both cruel, fatal genetic diseases. And these results have potential read through to our other 4 LGMD programs and further validate our gene therapy approach. Our success will come from the talent of our colleagues and our collaboration with the industry’s best and brightest. In that vein, I would like to thank Dr. Jerry Mendell of Nationwide Children’s Hospital for his hard work, ingenuity and extraordinary commitment to those living with rare neuromuscular disease.
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 leads to moves. In the case of Sarepta Therapeutics, the news proved to be incredibly positive.
With the strong clinical data, the company is one step closer to providing a therapy for an ailment that has no current FDA approved options. Should all go well, the potential value here could be incredible.
So, it’s not surprising to see that excited investors are pushing the stock on a run for the top. As is normally the case, our partners at Trade Ideas were the first to alert us to the gains. Currently (10:58), SRPT is trading at $149.29 per share after a gain of $9.25 per share or 6.60% 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 to bring MYO-101 to market. Nonetheless, we’ll continue to follow the story closely and bring the news to you as it breaks!
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