Sarepta Therapeutics SRPT Stock News

Sarepta Therapeutics Inc (NASDAQ: SRPT) is having a great start to Friday’s trading session, up nearly 7% early on. The gains come after the company announced positive functional data from a key clinical study. Here’s what’s going on:

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SRPT Stock Gains On SRP-9003 Data

Sarepta Therapeutics issued a press release this morning announcing positive clinical data. The data came from a Limb-girdle muscular dystrophy Type 2E clinical trial. The trial enrolled three participants who were treated with SRP-9003.

SRP-9003 is an investigational gene therapy. The therapy is designed to transduce skeletal and cardiac muscle with a gene that codes for the full-length, native beta-sarcoglycan protien. It is believed that the lack of this specific protein is the sole cause of LGMD2E.

In the release, SRPT said that three patients were treated with an infusion of SRP-9003. The patients ranged in ages from 4 years old to 13 years old. The company announced that all patients achieved improvements in functional outcomes at nine months.

First and foremost, mean creatine kinase was significantly reduced compared to baseline. This is an enzyme biomarker that is generally associated with muscle damage.

Moreover, SRPT said that all three patients showed improvement from baseline in all functional measures. These measures included:

  • The North Star Assessment for Dysferlinopathy, or NSAD.
  • Time to rise.
  • Four-stair climb.
  • 100-m walk test.
  • 10-meter walk test.

Importantly, SRPT said that the results following treatment are distincly different from what an age-matched, natural history group would predict.

Importantly, Sarepta Therapeutics said that there were no new safety signals observed. As mentioned in the past, two of the three of these patients did show elevated liver enzymes. Also, one of these patients had an associated transient increase in bilirubin.

However, both of these events took place as the patients were tapered off of oral steroids. Also, elevated liver enzymes returned to baseline and symptoms were resolved in both patients following supplemental steroid treatment.

In a statement, Doug Ingram, President and CEO at SRPT, had the following to offer:

We have now observed consistent functional improvements, in addition to high levels of expression of the missing protein of interest and strong results in related biomarkers, in both of our first cohorts for Duchenne muscular dystrophy (SRP-9001) and LGMD2E (SRP-9003). We intend to test one higher dose of SRP-9003 in LGMD2E participants, select our clinical dose and then advance our SRP-9003 program, along with our other five LGMD programs, as rapidly as possible. With the results of our first LGMD2E cohort, Sarepta continues to build its gene therapy engine, an enduring model created to design, develop and bring to the medical and patient community transformative therapies for those living with, and too often dying from, rare genetic disease.

The above statement was followed up by Jerry Mendell, M.D., principal investigator at the Center for Gene Therapy in the Abigail Wexner Research Institute and lead investigator on the study. Here’s what he had to offer:

LGMD2E is a devastating neuromuscular disease with no current treatment options so we are very pleased to observe a functional improvement in study participants who received SRP-9003.

Why This News Is Important

The news released by SRPT this morning proved to be overwhelmingly positive for several reasons. In my view, the three most important of these reasons are below!

Reason #1: LGMD2E Is Met With No Treatment Options

As mentioned by Dr. Mendell in a statement quoted in the press release, LGMD2E is a devastating condition. Unfortunately, the disease is met with no currently approved treatment options.

Ultimately, when a condition is met with little-to-no option for patients and the physicians that take care of them, it is considered a high-value indication. After all, once a company does come up with a positive treatment option, the value of that option to patients and their caregivers is tremendous.

Not only is LGMD2E a severe and debilitating condition, it is also known to reduce life expectancy in some patients. Being able to provide a potential option to these patients wouldn’t just be life changing for them, it would be overwhelmingly valuable for the company that did so.

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Reason #2: Positive Results Mean Further Study Ahead

If you invest in clinical-stage biotechnology companies, you know what tends to happen after positive results are released. At the end of the day, when positive results are announced, they act as a springboard for companies to move into further stages of development.

In the case of SRPT, I would expect nothing less. With positive results released surrounding a severe condition with no currently approved options available, I would imagine that the company is already working on plans to move into the next phase of development.

Importantly, SRP-9003 has been granted Orphan Drug Designation for limb griddle muscular dystrophies. With this designation, the company receives additional support from the FDA and may be able to get on a fast track to approval.

With the positive news in mind, in combination with the drug’s Orphan Drug Designation status, it only makes sense that the company would move quickly to take advantage of the added FDA support and push this drug to market.

This could mean that investors have more than just data driven catalysts to look forward to over the next few years.

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Reason #3: No New Adverse Events

While the last data release surrounding SRP-9003 was positive, there were concerns about adverse events. After all, 2 out of 3 patients saw elevated liver enzymes, one of which was deemed to be a serious adverse event.

With the news that the elevated enzymes were reduced to normal levels through the use of supplemental steroids and that the issue did not pop back up, there aren’t many concerns about adverse events any more.

This is a big deal as adverse events could lead to the end of development and scrapping of new drug candidates.

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