Clinical Catch-up from Last Week: May 5-10

Almost every week, biopharma companies release results from ongoing clinical trials. Here’s a roundup of some of the top clinical trial news from the previous week. AveXis, a Novartis company, released interim data from the Phase I STRONG trial, additional data from the STR1VE trial, and the first data from the Phase III SPR1NT trial for Zolgensma (onasemnogene abeparvovec-xioi) in spinal muscular atrophy (SMA) Type 1. Zolgensma is a gene therapy in development as a one-time infusion for SMA Type 1. It uses a virus vector to deliver a copy of the human SMA gene. SMA is a severe neuromuscular disease noted by the loss of motor neurons, which leads to progressive muscle weakness and paralysis. Neurocrine Biosciences presented data from two Phase III clinical trials of opicapone, a once-daily, oral catechol-O-methyltransferase (COMT) inhibitor for Parkinson’s disease. They found that patients receiving opicapone 50 mg with levodopa, the standard of care, had a significant and sustained improvement in symptoms. Neurocrine reported on two Phase III studies, BIPARK-1 and BIPARK-2. BIPARK-1 looked at about 600 patients with Parkinson’s and motor fluctuations. BIPARK-2 looked at about 400 patients with Parkinson’s and motor fluctuations. They received once-daily doses of opicapone of either 25 mg or 50 mg or placebo for 14 to 15 weeks. The data from these two Phase III trials showed that adding once-daily opicapone to levodopa significantly increased ON time without troublesome dyskinesia.