Sarepta Therapeutics, Inc. SRPT announced encouraging preliminary data from an early-stage study evaluating its gene therapy candidate, SRP-9003, in patients with limb-girdle muscular dystrophy Type 2E (LGMD2E), beating management’s expectation. The study is evaluating single intravenous infusion of two doses of SRP-9003 — 5x10^13 vg/kg and 2x10^14 vg/kg — in separate cohorts in children aged between four and 15 years.
One-year functional data from a low-dose cohort showed that patients who received treatment with SRP-9003 continued to show improvements from baseline across all functional measures.
Preliminary data from high-dose cohort showed mean expression of 72.3% of transduced beta-sarcoglycan protein, compared to 51% for low-dose, as reported in October 2019. Results from the high-dose cohort also exceeded the pre-defined measure of success for the study of 50% positive fibers. Moreover, mean fiber intensity was 73.1% compared to normal control, which was higher compared to 47% for the low-dose cohort. Please note that absence of beta-sarcoglycan protein is the sole cause of progressive degeneration and a shortened lifespan characterized by LGMD2E.
Shares of Sarepta rallied 7.9% on Jun 8 following the promising data readout. In fact, shares of the company have increased 23.7% so far this year compared with the industry’s rise of 7.8%.
Data from the higher dose cohort also showed 89.1% reduction in serum creatine kinase levels compared to baseline. Please note that creatine kinase is an enzyme biomarker strongly associated with muscle damage.
Both doses were well tolerated in patients. None of the patients in either cohorts of the study saw a decrease in platelet count that was outside of normal or any signal of complement activation.
The results from theearly-stage study will help the company to find the most optimal dose of SRP-9003 for pivotal study design.
Sarepta is also progressing well with its lead gene therapy candidate, SRP-9001, as a treatment for Duchenne muscular dystrophy (“DMD”). Encouraging results from the company’s gene therapy studies boosts its leading position in this field as competitors continue to face trouble during development. Pfizer PFE is also developing a gene therapy for treating DMD, but reported underwhelming data last month, which boosted investors’ confidence in Sarepta. Solid Biosciences SLDB is another company developing gene therapy for treating DMD. However, the company’s early-stage study in under clinical hold.
Sarepta has signed a licensing agreement with Roche RHHBY, granting exclusive rights to launch and commercialize SRP-9001 in ex-U.S. markets. This deal will likely boost the prospect of the gene therapy following successful development due to strong presence of Roche in ex-U.S. markets, especially Europe.
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Sarepta currently carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
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