Larger Infarct Volume Associated With Poorer Outcomes, Edavarone Shows Efficacy in ALS, DMD Treatment Demonstrates Proof-Of-Concept


Neurology News Network for the week ending January 2, 2021.

This week Neurology News Network covered a study that examined infarct volume and age on arterial ischemic stroke outcomes, the phase 3 stud of edavarone in patients with amyotrophic lateral sclerosis, and the phase 2 momentum study of SRP-5051 in patients with Duchenne muscular dystrophy.

Welcome to this special edition of Neurology News Network. I’m Marco Meglio. Please excuse our appearance this week as a majority of the US workforce, including the NeurologyLive team, moves to working remote as we come together to help reduce the spread of the novel coronavirus.

Data from a recent study showed that that larger infarct volume and younger age at the time of arterial ischemic stroke (AIS) were associated with poorer outcomes. These associations were modest and the ability to use these characteristics as predictors of outcome is limited, but suggest that infarcts that disrupt critical networks have a disproportionate impact upon outcome after childhood AIS. Infarcts that involved uncinate fasciculus, angular gyrus, insular cortex, or that extended from the cortex to the subcortical nuclei were significantly associated with poorer outcomes. Univariate analysis determined these associations to have odds ratios (ORs) ranging from 1.95 to 3.95. Infarct location was a significant predictor of poor outcome, but this significance disappeared when percentage infarct volume (PIV) was added to the model. Study authors concluded, “This finding suggests the hypothesis that infarcts that damage structures that connect other regions and serve a network function have a disproportionate effect upon function. Future studies of pediatric AIS should examine the effect of network disruption upon outcomes.”

Mitsubishi Tanabe Pharma America recently announced data from a post-hoc analysis of the edaravone phase 3 double-blind trial that examined the use of clinical staging systems to measure disease progression in amyotrophic lateral sclerosis.1 These data showed that The King’s and Milano-Torino (MiToS) staging systems were effective in assessing clinical progression of the disease. The data showed that 42% of patients on edaravone experienced a progression in King’s stage compared to 55.9% on placebo. An analysis of a ≥2-stage progression in MiToS stage showed no difference between treatment and placebo groups during double-blind treatment, but during the open-label period, more rapid progression was noted among patients in the placebo-edaravone arm than among those in the edaravone-edaravone arm.

Sarepta Therapeutics has announced that SRP-5051, its next-generation treatment for patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping, demonstrated proof-of-concept in the phase 2 MOMENTUM study with results supporting continued dose escalation. This was the first clinical data from SRP-5051, a treatment that uses Sarepta’s PPMO technology. Consistently higher tissue exposure, exon-skipping and dystrophin production in patients taking a monthly dose of SRP-5051 was observed in Part A from the multi-ascending dose MOMENTUM study. SRP-5051 was found to be generally well-tolerated across all doses, with no clinical or laboratory findings reported. Sarepta noted its belief that the findings support further clinical development of the agent.

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