No go for ozanezumab treatment in ALS
medwireNews: Ozanezumab has failed to show efficacy for the treatment of amyotrophic lateral sclerosis (ALS) in a phase II study, making neurite outgrowth inhibitor A (Nogo-A) potentially redundant as a treatment target.
Among 152 of 303 patients with ALS randomly assigned to receive 1-hour intravenous infusions of ozanezumab 15 mg/kg every 2 weeks for 46 weeks, the average joint rank analysis score for function (ALS Functional Rating Scale-Revised) and 48-week survival was –14.9. This score is based on patients scoring 0 if no difference in outcome existed, –1 if they had a worse outcome, and +1 if they had a better outcome.
This score was not significantly different, and in fact slightly worse, than the 15.0 points achieved by the 151 patients receiving placebo, despite the desired levels of drug exposure being achieved.
The outcomes were similar for all the secondary endpoints, including overall survival and progression-free survival.
Despite the lack of efficacy, ozanezumab was generally well tolerated. Rates of adverse events including those leading to drug discontinuation were similar in the two groups.
Respiratory failure was the most common serious adverse event, affecting 8% of patients in the ozanezumab group versus 5% in the placebo group, and it was the main contributor to the higher percentage of deaths among patients taking ozanezumab, at 13% versus 11%. There were two deaths considered drug related – bladder transitional cell carcinoma in the ozanezumab group and a cerebrovascular accident in the placebo group.
“These results suggest the futility of further clinical testing of an anti-Nogo-A monoclonal antibody for the treatment of ALS,” say Arseniy Lavrov (GlaxoSmithKline, Uxbridge, UK) and colleagues in The Lancet Neurology.
They conclude that “the identification of new options for ALS treatment remains a priority for clinical research.”
By Lucy Piper
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