122: RNA Therapeutics, Mivelsiran, and Treating Alzheimer Disease

NeurologyLive® Mind Moments®

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice.
In this episode, Sharon Cohen, MD, a behavioral neurologist, sat down to discuss positive data from an ongoing phase 1 study of mivelsiran (Alnylam Pharmaceuticals), the first investigational RNA interference therapeutic targeting amyloid precursor protein for Alzheimer disease (AD). Cohen, who also serves as the medical director of the Toronto Memory Program at the University of Toronto, discussed the potential of RNA therapeutics for treating AD, the unique mechanism of action of mivelsiran, and some of the early promising safety, efficacy, and pharmacokinetic data observed in the phase 1 trial. In addition, Cohen touched upon the idea of how RNA therapeutics could be used in combination with previously approved novel treatments and the benefits mivelsiran brings with no observed amyloid-related imaging abnormalities. Furthermore, the discussion covered some of the potential of this investigational agent, what to expect in the multi-dose part B of the study, and an additional phase 2 study in cerebral amyloid angiopathy.
Looking for more Alzheimer disease/dementia discussion? Check out the NeurologyLive® Alzheimer disease/dementia clinical focus page.
Episode Breakdown:

• 1:15 – Mechanism of action of mivelsiran and how it can be potentially beneficial in AD
• 4:15 – Growing knowledge of RNA therapeutics and their impact on neurologic conditions
• 7:00 – Neurology News Minute
• 10:10 – Phase 1 study data, including efficacy, safety, biomarker, and pharmacokinetic results
• 19:20 – How mivelsiran fits with other approved therapies for AD; future goals and directions of the drug

The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here:

• FDA Clears Indapta Therapeutics’ IND for Cell Therapy IDP-023 in Progressive Multiple Sclerosis

• Muscle-Targeting Therapy Apitegromab Effective in Spinal Muscular Atrophy Over 4 Year Period

• FDA Approves Medtronic’s Deep Brain Stimulation Technology for Asleep Capabilities

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