Sarepta Therapeutics shares soar on preliminary gene therapy data

Shares of Sarepta Therapeutics soared more than 50 percent Tuesday after the company presented preliminary data for its experimental treatment for patients with Duchenne muscular dystrophy.

The rare genetic disease causes muscle wasting in addition to also can be fatal before patients turn 30. Symptoms usually start in early childhood, usually between 3 in addition to also 5 years old. This kind of primarily affects boys although in rare cases can affect girls.

Sarepta shared results coming from three children who received the gene therapy in a phase 1/2a clinical trial.

Creatine kinase can be an enzyme found within muscle cells that will can leak into the bloodstream when damage has occurred from the muscle. Elevated levels of This kind of can indicate someone has Duchenne muscular dystrophy, or DMD.

Patients who received Sarepta’s drug showed an average reduction of more than 87 percent in their creatine kinase levels on day 60.

The treatment also produced robust levels of dystrophin, a protein found in muscles that will can be used for movement. The results “meaningfully beat even the highest of expectations,” J.P. Morgan analyst Anupam Rama wrote in a note to clients.

“I have been waiting my entire 49-year career to find a therapy that will dramatically reduces CK levels in addition to also creates significant levels of dystrophin,” Dr. Jerry Mendell of Nationwide Children’s Hospital said in a statement. “Although the data are early in addition to also preliminary, these results, if they persist in addition to also are confirmed in additional patients, will represent an unprecedented advancement from the treatment of DMD.”

Sarepta closed Monday which has a market value of $6.9 billion, according to FactSet. that will rocketed higher to about $10.6 billion during Tuesday’s surge.

The company’s shares have gained nearly 0 percent This kind of year.

Solid Biosciences can be also developing a gene therapy for DMD. Its shares surged 82 percent Tuesday, one day after the Food in addition to also Drug Administration took the company’s program off clinical hold.

Solid has treated just one patient in addition to also said Monday This kind of expects to have data from the second half of 2019.

The NASDAQ’s biotech index was up almost 1.2 percent on Tuesday, on pace for its best day since June 6 when This kind of gained 1.25 percent.

— CNBC’s Meg Tirrell contributed to This kind of report.

A press Discharge summarizing Sarepta’s results follows

CAMBRIDGE, Mass.,June 19, 2018 (GLOBE NEWSWIRE) –Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage biopharmaceutical company focused on the discovery in addition to also development of precision genetic medicine to treat rare neuromuscular diseases, announced that will at the Company’s R&D Day,Jerry Mendell, M.D. of Nationwide Children’s Hospital presented positive preliminary results coming from its Phase 1/2a gene therapy clinical trial assessing AAVrh74.MHCK7.micro-Dystrophin in individuals with Duchenne muscular dystrophy (DMD). Dr. Mendell presented the following preliminary data on the first three patients enrolled from the study:

*All patients showed robust expression of transduced micro-dystrophin, which can be properly localized to the muscle sarcolemma, as measured by immunohistochemistry. Mean gene expression, as measured by percentage of micro-dystrophin positive fibers was 76.2% in addition to also the mean intensity of the fibers was 74.5% compared to normal control.

*All post-treatment biopsies showed robust levels of micro-dystrophin as measured by Western blot, which has a mean of 38.2% compared to normal utilizing Sarepta’s method, or 53.7% compared to normal pursuant to Nationwide Children’s quantification of Sarepta’s method that will adjusts for fat in addition to also fibrotic tissue.

*A mean of 1.6 vector copies per cell nucleus was measured in patients, consistent with the high micro-dystrophin expression levels observed.

*All patients showed significant decreases of serum creatine kinase (CK) levels, which has a mean reduction of CK of over 87% at Day 60. CK can be an enzyme associated with muscle damage in addition to also patients with DMD uniformly exhibit high levels of CK. Indeed, significantly elevated CK can be often used as a preliminary diagnosis tool for DMD, which can be then followed by confirmatory genetic testing.

*No serious adverse events (SAEs) were observed from the study. Two patients had elevated gamma-glutamyl transferase (GGT) that will resolved with increased steroids within a week in addition to also returned to baseline levels. There were no various other significant laboratory findings. Patients had transient nausea generally during the first week of therapy coincident with increased steroid dosing.

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