Updated with new information and analysis.
BOSTON ( TheStreet) -- A research abstract containing the GlaxoSmithKline (GSK) drisapersen study results was just released by Cold Spring Harbor Labs in advance of Thursday's meeting. Obviously, these data hold significance for Sarepta Therapeutics (SRPT) and its competing Duchenne muscular dystrophy drug eteplirsen.
I believe the drisapersen data bode well for Sarepta and eteplirsen. Sarepta shares are up 10% to $39.33, so it appears the market agrees with me. I'll have more thoughts on the drisapersen data below, but first, here is the just-released research abstract in full:
DRISAPERSEN TREATMENT FOR DUCHENNE MUSCULAR DYSTROPHY: RESULTS OF A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED CLINICAL TRIALJohn E Kraus1, Claire Wardell1, Katie Rolfe1, Joanna Nakielny1, Naashika Quarcoo1, Lia Liefaard1, Steve Hood1, Allison Morgan2, Afrodite Lourbakos2, Sjef de Kimpe2, Rosamund Wilson2, Giles Campion2 1GlaxoSmithKline, R&D, Research Triangle Park, NC, 27709, 2Prosensa, R&D, Leiden, 2333 CH, Netherlands Introduction: Duchenne muscular dystrophy (DMD) is a progressive neuromuscular disorder, ultimately lethal, caused by the absence of dystrophin protein due to mutations of the dystrophin gene. The aim of oligonucleotide therapy is to manipulate the post-transcriptional splicing process of the premRNA to restore the reading frame of the mRNA, resulting in a shortened dystrophin protein. Drisapersen is a 2'-O-methyl-phosphothioate oligo designed to skip exon 51 in the dystrophin pre-mRNA. Here we report results of an exploratory, unpowered, double blind, placebo-controlled clinical study of drisapersen. Methods: Subjects with DMD met the following inclusion criteria: ≥5 years old; ambulant; corticosteroid-treated; rise from the floor ≤7 sec; and a dystrophin mutation correctable by exon 51 skipping. 53 subjects were randomized to 2 drisapersen dosing regimens or matched placebo (2:1). Dosing regimens: continuous (6mg/kg/wk) and intermittent (10-week cycles of 9 doses at 6mg/kg over 6 wks, and 4 wks off drug). Treatment was administered subcutaneously for 48 weeks. The primary objective was to assess the efficacy (6 Minute Walk Distance
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