Updated with new information and analysis.
BOSTON ( TheStreet) -- A research abstract containing the GlaxoSmithKline (GSK - Get Report) 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 - Get Report) 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