Laquinimod Reduces Caudate Volume in Huntington, Assisted Reproductive Technologies Increase In-Hospital Stroke Risk, Omaveloxolone Approved in EU

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Welcome to this special edition of Neurology News Network. I’m Marco Meglio.

Recently published data from the double-blind, placebo-controlled LEGATO-HD study (NCT02215616) showed that treatment with laquinimod did not impact motor symptoms in patients with Huntington disease (HD); however, it did significantly reduce caudate volume loss compared with placebo over a 52-week period. These findings represent the first clinical observations supporting a possible role of neuroinflammation in the pathology of HD that could be modulated by therapeutic intervention. Despite not meeting the primary end point, those on laquinimod 1.0 mg demonstrated a change in caudate volume that differed significantly from placebo. The exploratory MRI volumetric measures of caudate volume loss for the laquinimod 0.5 mg group, and whole brain volume loss, white matter volume loss, and ventricular volume for the laquinimod 0.5 mg and 1.0 mg groups all consistently showed improvements compared with placebo.

In a new retrospective cohort study of population-level data, findings showed an increased risk of any stroke among patients hospitalized for delivery in the United States who used assisted reproductive technologies (ART) compared with nonART users, suggesting an association between ART and stroke among this patient population. Authors recommended clinicians counsel patients who are undergoing ART therapy, regarding potential increased stroke risk, and consider initiating treatments proven to reduce stroke risk before ART. The subtypes of stroke observed in these patients included ischemic stroke, subarachnoid hemorrhage, intracerebral hemorrhage, and cerebral venous thrombosis.

Following a positive recommendation by the Committee for Medicinal Products for Human Use, the European Commission (EC) has approved Biogen’s omaveloxolone (Skyclarys) as the first therapy specific to treat patients with Friedreich ataxia (FA). The therapy is indicated for adults and adolescents aged 16 years and older with the disease. Omaveloxolone, an oral medication, had its efficacy and safety demonstrated in the phase 3 MOXIe part 2 trial, a 48-week, placebo-controlled study. MOXIe included 2 placebo-controlled parts and an open-label extension (OLE). Part 1 was a placebo-controlled, dose-ranging study that enrolled 69 individuals, whereas part 2 was a multicenter, randomized, placebo-controlled clinical trial that enrolled 103 patients at 11 study sites in the US, Europe, and Australia.

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