Huntington Disease Trial Results; Mind Moments Podcast Now Live; PMM Trial Falls Short

January 4, 2020

Neurology News Network for the week ending January 4, 2020.

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

Topline results from the phase 1b/2a PRECISION-HD2 clinical trial of Wave Life Sciences’ WVE-120102 revealed a statistically significant reduction in mutant huntingtin protein with the highest doses tested in adults with early manifest Huntington disease. The drug is the first allele-selective molecule that preferentially lowers mutant HTT protein by targeting SNP2 in order to maintain levels of healthy or wild-type HTT protein. Wave Life Sciences also disclosed that they observed no difference in neurofilament light chain in CSF between the treatment groups and placebo. Overall, the therapy was safe and well tolerated as single and multiple dosages up to 16 mg. Based on the results, the company plans to add a 32 mg cohort in early 2020.

Episode 5 of the NeurologyLive Mind Moments podcast is now live! The episode, titled "Gut Bacteria and the Brain: An Evolving Role in Epilepsy" features an interview with Daniel Lowenstein, MD, professor of neurology and executive vice chancellor and provost at the University of California, San Francisco, where he previously served as director of the UCSF epilepsy center and epilepsy research laboratory. Lowenstein discusses the latest research on how the gut microbiome plays a role in epilepsy, and how altering the gut microbiota may prove to be a very promising therapeutic option in the future.

Stealth BioTherapeutics has announced that elampretide, its small mitochondrial-targeting tetra-peptide, has failed to achieve the primary end points in the phase 3 MMPOWER-3 clinical trial in primary mitochondrial myopathy. The co-primary end points assessed the changes in the 6-minute walk test and Primary Mitochondrial Myopathy Symptom Assessment total fatigue score. Although these marks were not met, the safety data suggest that treatment with elampretide was well tolerated, with mostly mild to moderate adverse events.

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