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Sarepta's Stock Soars on Encouraging Gene Therapy Results

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Sarepta Therapeutics, Inc. SRPT shares soared 37% on Tuesday after it announced promising results from an early-stage study, evaluating its gene therapy as a treatment for patients with Duchenne muscular dystrophy (DMD)

The preliminary data from the first three patients dosed in the phase I/IIa study showed that treatment with the gene therapy, AAVrh74.MHCK7.micro-dystrophin led to a significant increase in dystrophin protein production.

Micro-dystrophin is a muscle protein, which is needed for muscles to function properly. The absence of this protein causes DMD, a rare muscular degenerative disease that mostly affects boys and can be fatal before patients turn 30. Any DMD therapy aims to boost the amount of micro-dystrophin in patients' muscles. Biopsies performed on day 90 showed robust levels of micro-dystrophin in patients treated with the gene therapy achieving a mean level of 38.2% compared to normal levels.

A dramatic reduction in levels of serum creatine kinase (CK), an enzyme associated with muscle damage caused by DMD, was also observed after using the gene therapy. The data showed that treatment with the gene therapy led to a mean reduction of CK of over 87% by day 60. Meanwhile, no serious adverse events (SAEs) were observed in the study.

The data were presented by Jerry Mendell, M.D. of Nationwide Children’s Hospital on Sarepta’s R&D Day.

Dr. Mendell and Sarepta are hopeful that if this transformative data is sustained in additional patients and studies, it can produce a therapy that can dramatically change the course of the DMD disease.

So far this year, Sarepta’s shares have rallied 89.1% against a 7.2% decrease registered by the industry.


However, following the announcement, shares of PTC Therapeutics, Inc. PTCT, which also markets therapies to treat DMD, tumbled nearly 31%. Ironically, shares of PTC Therapeutics had risen almost 28% on Monday after it presented encouraging preliminary data from an early-stage study evaluating its pipeline candidate, risdiplam in babies with type 1 spinal muscular atrophy (“SMA”).

Meanwhile, Solid Biosciences, Inc. SLDB is also developing a gene therapy for DMD. Its shares also surged on Tuesday as the FDA lifted clinical hold on a study on its experimental DMD candidate, SGT-001. (Read more: Solid Surges as FDA Lifts Clinical Hold on DMD Drug)

We remind investors that Pfizer PFE is also developing a gene therapy for the treatment of DMD. An early-stage study was initiated in April.

Sarepta currently carries a Zacks Rank #4 (Sell).

You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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