Introducing The Axon Newsletter from NeurologyLive

Welcome! For those unfamiliar with us, NeurologyLive® is a clinical news publication that provides the latest coverage in neurology of FDA news and the therapeutic pipeline, expert opinions and insights, in-clinic care and advocacy, and major medical meetings throughout the year. Our mission is to deliver quality and relevant information to health care professionals treating neurological diseases to help them achieve the best patient care possible.

Our team spends countless hours compiling this coverage and speaking to the key opinion leaders across the breadth of neurology to bring that info to your fingertips on our website, neurologylive.com. Now, The Axon can be your go-to place to get that insight, right here on LinkedIn.

On the first of each month, we'll bring you a new edition of The Axon highlighting the trending topics and important updates our editorial staff reports on. We'll point you to the news that matters and the specialist insight that can help your clinical practice. Similar to how axons operate as a means of communication within the nervous system, The Axon will seek to keep our readers current.

Of course, we're not planning to leave you hanging for this month, so we've got a couple of things to share from March 2024.

Although it was presented in late February at the ACTRIMS ® Forum in West Palm Beach, Florida, we have to highlight some important data on mesenchymal stem cell treatment in multiple sclerosis—an exciting area of therapeutic development for progressive disease. The data come from a small, open-label interim analysis of a phase 2 trial (NCT02166021), but findings were promising nonetheless, showing maintenance of a continuous reduction of serum NfL and GFAP, as well as beneficial effects reported on cognition, with repeated intrathecal injection of NeuroGenesis’ MSC therapy, NG-01. Lead author Dimitrios Karussis, MD, PhD, of the Hadassah Medical Center in Jerusalem, Israel, told us that the next steps include an extension of the current study with more patients and the preparation of a multicenter trial with MSC-NG01 in progressive MS at 6 sites in the United States.

March 2024 kicked off with the Muscular Dystrophy Association 's Annual Clinical and Scientific Conference running from the third to the sixth. While on the ground in Orlando, Florida, we spoke with more than a dozen clinicians, patients, and advocates in neuromuscular disease, including Brooke Eby , an amyotrophic lateral sclerosis (ALS) advocate who is also a patient, who shared her perspective on ALS and offered insight into ways to incorporate business approaches to improve clinical care and increase enrollment for drug trials in ALS. We also spoke at length about the state of science in neuromuscular medicine with Jeff Chamberlain, PhD, of the University of Washington , who was awarded the MDA Legacy Award at this year's meeting.

We also saw some important data presented at the meeting, including real-world interim data from the phase 4 EVOLVE trial, a confirmatory study assessing the treatment effects of eterplirsen (Exondys 51; Sarepta Therapeutics) in patients with Duchenne muscular dystrophy (DMD) that suggested a persistence of efficacy and consistent safety among a subgroup of nonambulatory patients. Additional open-label data on eterplirsen showed safety in patients as young as 6 months old—with no discernable differences compared with those who were 2-3 years old after 162 weeks. We also covered a post hoc analysis on givinostat (Duvyzat; Italfarmaco SpA)—which received FDA approval for DMD this month—that was consistent with the efficacy displayed in the prespecified analyses and emphasized its potential in a broader context. After givinostat was approved, we spoke with Sharon Hesterlee , PhD, of the MDA, for a special episode of the Mind Moments podcast. You can listen to that episode below:

In other news, Lundbeck announced in March that it will initiate a phase 2 trial, dubbed PROCEED, to evaluate Lu AG09222, a pituitary adenylate cyclase-activating polypeptide (PACAP)-targeting therapy, as a potential preventive for patients with migraine. The double-blind, placebo-controlled, dose-finding study is expected to include 498 patients and is intended to establish the optimal dose for future global pivotal trials. This comes after years of conversation about the potential of PACAP as a therapeutic pathway in migraine, and will hopefully bear fruit when the trial is completed in the second half of 2025.

Finally, we'd be remiss to not introduce the team that will be bringing you this news each month. Meet our team:

Matt Hoffman – Associate Editorial Director

• Matt has covered medical news for MJH Life Sciences since 2017, previously writing for HCPLive®. Previously, he wrote for Science World Report, The Philadelphia Inquirer, and NJ Advance Media. He graduated from Rowan University, where he studied journalism and web editing.

Marco Meglio – Editor

• Marco has been with the team since October 2019. Prior to MJH Life Sciences, he worked in the public relations department for the Somerset Patriots, a minor league baseball team in New Jersey. He graduated from Hofstra University with a degree in journalism and a minor in political science.

Isabella Ciccone, MPH – Assistant Editor

• Isabella has been with the team since September 2022. Prior to joining MJH Life Sciences, she worked as a research assistant in a lab focused on developing a music-based application for people who are living with dementia and their caregivers at Rutgers Institute for Health, Health Care Policy, and Aging Research. She graduated from The College of New Jersey with a Master's in Public Health and a degree in communications.

We hope you've enjoyed this quick intro to The Axon. We'll catch you here on May 1, 2024, for our first full edition!