Risdiplam Shows Positive Results in SMA, Study Trials for DMD Discontinued, Neflamapimod Granted Fast Track for Treatment of Dementia with Lewy Bodies

This week's Neurology News Network covered the SUNFISH trial, which showed positive results in patients with spinal musuclar atrophy, as well as 2 FDA discontinuations of studies that focus on Duchenne muscular dystrophy; The FDA has also granted fast track designation for neflamapimod, a drug intended to treat dementia with lewy bodies.

Marco: Welcome to Neurology News Network. I’m Marco Meglio. Let’s get into the news from this week.

Interim results from part 2 of the phase 2/3 SUNFISH trial of risdiplam show positive results in patients with spinal muscular atrophy (SMA) type 2 or 3, with the study meeting its primary endpoint of change from baseline in the Motor Function Measure 32 (MFM-32) scale after 1 year of treatment with the drug, formerly known as RG7916. After 12 months of treatment, the study met its primary endpoint of change from baseline in MFM-32 compared with placebo. The study affirmed the consistent safety profile of risdiplam from previous trials. No patients experienced any treatment-related safety issues, and there was high tolerability among patients assessed. In addition to SUNFISH, there are 3 other ongoing trials evaluating the efficacy of risdiplam in people with SMA. Specific data from part 2 of the SUNFISH study will be presented at an upcoming medical congress.

Following the occurrence of a serious safety incident, the FDA has placed a hold on Solid Biosciences’ clinical trial for SGT-001, its gene therapy candidate for Duchenne muscular dystrophy. This is the third safety incident in the IGNITE DMD clinical trial that has resulted in a clinical hold since its inception in 2017. Additionally, Roche has discontinued a phase 2/3 trial of RG6206 after a futility analysis showed that the drug was “highly unlikely” to demonstrate clinical benefit in patients with Duchenne muscular dystrophy. The anti-myostatin adnectin protein agent was being studied in the phase 2/3 SPITFIRE trial, as well as the phase 1b/2 THUNDERJET trial. While Roche has seemingly met a blockade in the search for treatment for DMD, there were previous indications made that the recently licensed myostatin inhibitor, could potentially be studied for other neuromuscular disorders besides DMD.

EIP Pharma announced that neflamapimod has been granted fast track designation by the FDA for the treatment of dementia with Lewy bodies. Additionally, they announced the results from the REVERSE-SD study that examined the drug in early stage Alzheimer disease. A phase 2, double-blind, placebo-controlled study is now recruiting, with a target enrollment of 80 participants across 20 sites in the US and 2 in the Netherlands. Patients enrolled will be monitored over the 16-week trial, where they will be assessed on a study-specific Cognate Neuropsychological Test Battery for the primary endpoint. Full results of the REVERSE-SD study will be presented at the upcoming C-TAD meeting in San Diego.

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