Shares of Sarepta Therapeutics, Inc. (SRPT - Free Report) increased 17.1% on Jun 28 after Pfizer (PFE - Free Report) announced initial data from its phase Ib study evaluating gene therapy candidate, PF-06939926, as a treatment for Duchenne muscular dystrophy (“DMD”). Serious side effects were observed in two participants in the study.
Two patients dosed with PF-06939926 were hospitalized, one for severe nausea and vomiting and the other for a severe immune response, which resulted in renal complications. Pfizer has stopped further dosing in the study until specific additional safety monitoring obtains all appropriate approvals. Moreover, improvement in dystrophin levels in patients seemed inferior to Sarepta’s gene therapy candidate.
Sarepta is evaluating its micro-Dystrophin gene therapy candidate, SRP-9001, in early- to mid-stage studies. Preliminary data from the studies showed that treatment with SRP-9001 had no adverse effects on patients. A mean of 81.2% of muscle fibers were dystrophin positive after nine months of dosing based on Western blot method. Pfizer’s PF-06939926 achieved a mean of 69% dystrophin positive fibers for the higher dose (3E14 vg/kg) after one year of treatment, based on Pfizer’s proprietary measurement technique.
Although gene therapies of both the companies are in early stage of development, preliminary data suggests better prospects for Sarepta’s candidate. Sarepta also has better knowledge and more experience of the DMD disease due to its string of therapies. We remind investors that Pfizer discontinued clinical development of its DMD candidate, domagrozumab, last year due to its failure in meeting primary endpoints in clinical studies.
DMD is a genetic disorder caused by a mutation that prevents the body from producing dystrophin, a protein that muscles need to function properly.
Shares of Solid Biosciences Inc. (SLDB - Free Report) , another company developing gene therapy for treating DMD, also increased 13.9% last Friday following Pfizer’s setback.
Sarepta’s shares have rallied 39.2% so far this year compared with the industry’s increase of 5.3%.
Sarepta holds a strong position in the DMD segment with its sole marketed drug, Exondys 51 approved for treating DMD patients with certain mutation in their gene. The drug generated sales of $87 million in the first quarter of 2019, up almost 35% year over year and % sequentially.
The company’s another DMD candidate, golodirsen, is under review in the United States. A decision in expected by the end of August. It also has a late-stage DMD candidate, casimersen. In March, Sarepta announced interim data from the phase III study evaluating casimersen. It showed that treatment with the candidate led to statistically significant mean increase of dystrophin protein production compared to baseline and placebo with 100% response rate. A new drug application seeking approval for casimersen is expected to be filed shortly.
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 developing therapies targeting DMD include Wave Life Sciences (WVE - Free Report) and PTC Therapeutics.
Sarepta currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
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