Sarepta Therapeutics, Inc. SRPT announced positive interim data from a phase III study on its duchenne muscular dystrophy (DMD) candidate,casimersen. Data from the ESSENCE study showed that treatment with casimersen led to statistically significant mean increase of dystrophin protein production (as measured by western blot) compared to baseline and placebo.
DMD is a genetic disorder caused by a mutation that prevents the body from producing dystrophin, a protein that muscles need to function properly. Casimersen works through a method called exon skipping, which helps the body's cellular machinery make functional dystrophin. Sarepta already has a marketed drug, Exondys 51 in its portfolio, which is the first approved disease-modifying therapy for DMD in the United States.
In the past year, the company’s shares have increased 60.2% against the industry’s decline of 13.9%.
In the study, 22 patients receiving casimersen displayed an increase in skipping exon 45 over their baseline levels, representing a 100% response rate. The company plans to submit a new drug application (NDA) to the FDA in mid-2019, after holding apre-NDA meeting with the agency.
Sarepta is looking to build its DMD pipeline beyond Exondys 51 by developing other exon-skipping treatments. In fact, the company has about 13 exon-skipping candidates in its pipeline, the most advanced ones being golodirsen and casimersen. In December 2018, the company completed a rolling NDA submission, seeking accelerated approval for golodirsen. A decision is expected by Aug 19, 2019.
The company expects to double the eligible DMD patient population for its marketed products in 2020 compared to 2018, on potential approval of golodirsen and casimersen.
Other companies also involved in developing DMD therapies include Catabasis Pharmaceuticals, Inc. CATB, Summit, PTC Therapeutics, Pfizer Inc. PFE, BioMarin BMRN et al.
Sarepta Therapeutics, Inc. Price
Sarepta Therapeutics, Inc. Price | Sarepta Therapeutics, Inc. Quote
Sarepta currently carries a Zacks Rank #3 (Hold).
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