Evidence Lacking for Pridopidine Efficacy in Huntington's Disease

Although treatment did not meet prespecified efficacy, potential effect on motor phenotype seen

TUESDAY, Nov. 8 (HealthDay News) -- Pridopidine may have some effect on the motor phenotype of Huntington's disease, although it does not reach the prespecificed α level in a phase 3 trial, according to a study published online Nov. 7 in The Lancet Neurology.

Justo Garcia de Yebenes, M.D., from the Hospital Ramón y Cajal in Madrid, Spain, and colleagues investigated the efficacy of pridopidine for treating motor deficit in patients with Huntington's disease (aged 30 years or older). In total, 437 participants were randomized for full analysis: 144, 148, and 145 patients to placebo, 45 mg, and 90 mg per day pridopidine, respectively. Participants had a modified motor score (mMS) of 10 points or greater at baseline; the primary end point was change in the mMS at 26 weeks. Safety and tolerability were also assessed. The α level for the primary analysis was 0.025, and overall was 0.05.

The investigators found that, compared to patients receiving placebo, in the full analysis set those receiving 45 and 90 mg per day of pridopidine had a mean mMS difference of −0.36 points (P = 0.456) and −0.99 (P = 0.042), respectively. In the per-protocol population, compared to patients receiving placebo, those receiving 90 mg per day pridopidine had a mMS reduction of −1.29 points (P = 0.014). Neither dose affected change in the nonmotor end points. Pridopidine was well tolerated.

"This study did not provide evidence of efficacy as measured by the mMS, but a potential effect of pridopidine on the motor phenotype of Huntington's disease merits further investigation," the authors write.

Several authors disclosed financial relationships with pharmaceutical and biomedical companies, including NeuroSearch A/S, which funded the study and manufactures pridopidine.

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