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, Justin Klee and Josh Cohen, cofounders and cochief executive officers of Amylyx Pharmaceuticals, sat down at the recently concluded AAN Annual Meeting to discuss the results from the pivotal phase 3 PHOENIX trial (NCT) of AMX0035 (Relyvrio) in patients with amyotrophic lateral sclerosis (ALS). The duo provided clarity on the findings, positive takeaways from the disappointing data, and the lessons learned in ALS drug development. In addition, the two provided commentary on the future plans of the therapy in other tauopathies like progressive supranuclear palsy and neurologic conditions like Wolfram syndrome. Furthermore, Klee and Cohen shared thoughts on the company's drug pipeline, including AMX0114, an antisense oligonucleotide in development for patients with ALS.
Looking for more neuromuscular discussion? Check out the NeurologyLive ® Neuromuscular clinical focus page .
Episode Breakdown:

• 1:05– Decision behind removing AMX0035 from market
• 3:00– Lessons from PHOENIX, AMX0035 drug development program
• 6:45– Unraveling PHOENIX study data
• 10:00– Neurology News Minute
• 12:10– Potential of AMX0035 in other tauopathies, neurologic conditions
• 15:00– Outlook of antisense oligonucleotide AMX0114 in ALS

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:

• Diazepam Buccal Film’s Role in Treating Intermittent Pediatric Seizures, with Michael Rogawski, MD, PhD

• Ocrelizumab Gains EU CHMP Positive Opinion for Subcutaneous Formulation to Treat MS

• FDA Approves Sprinkle Formulation of Neurocrine Biosciences’ Valbenazine for Tardive Dyskinesia or Huntington disease Chorea

Thanks for listening to the NeurologyLive ® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to this special episode of the NeurologyLive ® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. For major FDA decisions in the field of neurology, we release short special episodes to offer a snapshot of the news, including the main takeaways for the clinical community, as well as highlights of the efficacy and safety profile of the agent in question.
In this episode, we're covering the recent approval of diazepam buccal film (Libervant; Aquestive Therapeutics) for the treatment of pediatric patients with intermittent, stereotypic episodes of frequent seizure activity (seizure clusters, acute repetitive seizures). Indicated for those between 2 and 5 years of age, the therapy offers patients a compact, easily administered diazepam formulation. The FDA previously granted tentative approval in August 2022 for Libervant for treatment of these patients with epilepsy 12 years of age and older, with U.S. market access for Libervant for this age group of patients subject to the expiration of the existing orphan drug market exclusivity of a previously FDA approved drug scheduled to expire in January 2027. Following the approval, Michael Rogawski, MD, PhD, a distinguished professor of neurology and pharmacology at the University of California Davis Health Medical Center, provided insight on what the decision means for patients and clinicians. Rogawski gave comment on the significance of having a new administration route for diazepam, the safety and feasibility of diazepam buccal film, and some of the major points of emphasis from its clinical program.

For more of NeurologyLive's and Contemporary Pediatrics coverage of diazepam buccal film's approval, head here: FDA approves diazepam for seizure clusters in patients 2 to 5 years

Episode Breakdown:

• 0:20 – Diazepam buccal film approved for intermittent seizures in pediatrics
• 2:00 – Michael Rogawski, MD, PhD, giving reaction to the approval
• 4:10 – Flexibility with multiple diazepam administration routes
• 6:55 – Rogawski on the safety of diazepam buccal film amid high need
• 12:30 – Notable takeaways from the diazepam buccal film trial program

Thanks for listening to the NeurologyLive Mind Moments podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

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, Ian Kremer, executive director of the leaders Engaged on Alzheimer's Disease (LEAD) coalition, spoke about several relevant topics regarding Alzheimer care as new novel therapeutics emerge. He talked about the lessons learned from the recent discontinuation of aducanumab, and the potential and limitations lecanemab (Leqembi; Eisai) and donanemab (Eli Lilly) may bring. Additionally, he provided comments about what matters to patients, the perception of the FDA approval process, and what is considered "clinically meaningful." Furthermore, he gave perspective on ways to improve drug development and emphasized the need for policy decisions to be based on scientific evidence and not by sensationalized headlines.
Looking for more Alzheimer disease/dementia discussion? Check out the NeurologyLive ® Alzheimer disease/dementia clinical focus page .
Episode Breakdown:

• 1:10 – Promise in the Alzheimer field in 2024
• 4:05 – Ways of improving efficiencies with drug develpment
• 9:10 – Discontinuation of aducanumab
• 11:10– Neurology News Minute
• 13:50 – Lessons learned from aducanumab, antiamyloid therapies
• 22:10– Conversations between clinicians and patients surrounding expectations/limitations of antiamyloid therapies and available treatments

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 Agrees to New Specialized Protocol for Phase 3b Study of ALS Agent NurOwn

• FDA Approves Alternate Administration Routes for Antiseizure Medication Cenobamate

• Extended Use of Investigational Agent IPX203 Safe in Parkinson Disease

Thanks for listening to the NeurologyLive ® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

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, Francesco Saccà, MD, PhD, an associate professor of neurology at the University of Naples, discussed an ongoing study assessing the use of dimethyl fumarate, an approved therapy for relapsing multiple sclerosis, in patients with Friedreich ataxia (FA). He spoke on the mechanism of action of the agent and why it can be beneficial in this patient population, as well as how it differs from omaveloxolone (Skyclarys; Biogen), the first approved treatment for FA. In addition, he outlined the study design, the primary end point of frataxin decrease, and what would be considered a "successful" study. Furthermore, he spoke on the reasons behind the trial and what led investigators to this point.
Looking for more ataxia discussion? Check out the NeurologyLive ® ataxia clinical focus page .
Episode Breakdown:

• 1:05 – Origin of the study
• 3:00 – Conduct of the study, end points, goals
• 5:10 – Promising mechanism of action of dimethyl fumarate to treat Friedreich ataxia
• 7:30 – Questions in clinical impact of changes in frataxin
• 10:00 – Neurology News Minute
• 12:40 – What is considered a successful study?
• 14:55– Patient inclusion, demographic makeup of the study
• 16:35– Timeline of the trial and data readout

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 Approves Alexion's Ravulizumab-cwvz for Neuromyelitis Optica Spectrum Disorder
• Epilepsy Agent STK-001 Demonstrates Disease-Modifying Effects in Early Phase Studies of Dravet Syndrome
• Eisai Submits sBLA for Monthly Intravenous Maintenance Dosing of Alzheimer Therapy Lecanemab

Thanks for listening to the NeurologyLive ® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to this special episode of the NeurologyLive ® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. For major FDA decisions in the field of neurology, we release short special episodes to offer a snapshot of the news, including the main takeaways for the clinical community, as well as highlights of the efficacy and safety profile of the agent in question.
In this episode, we're covering the recent approval of givinostat (Duvyzat; Italfarmaco) for the treatment of Duchenne muscular dystrophy (DMD). The therapy, a proprietary histone deacetylase (HDAC) inhibitor, was approved as the first nonsteroidal drug for patients with all genetic variants of DMD. The supporting data for the approval of givinostat comes from the phase 3 EPIDYS trial (NCT02851797). EPIDYS, a randomized, double-blind, placebo-controlled, multicenter study, included 179 ambulant male individuals who were randomly assigned 2:1 to either oral givinostat or placebo for an 18-month treatment period. Following the approval, Sharon Hesterlee, PhD, executive vice president and chief research officer of the Muscular Dystrophy Association, sat down to discuss the significance of the approval and how it changes the care for patients with DMD. She spoke specifically about the mechanism of action of the therapy, its safety profile, and how it may be used with other agents. In addition, she discussed other related topics on gene therapy and unmet needs for this patient population.

For more of NeurologyLive's coverage of givinostat's approval, head here: FDA Approves Italfarmaco's Givinostat for Duchenne Muscular Dystrophy

Episode Breakdown:

• 0:30 – Givinostat approved for Duchenne muscular dystrophy
• 2:00 – Sharon Hesterlee, PhD, on the approval's implications
• 3:35 – Positive downstream effects of the approval
• 4:50 – Hersterlee on the safety profile of givinostat
• 5:40 – Promising outlook of the DMD field
• 6:45 – Overcoming roadblocks involved with gene therapy
• 8:55 – Remaining unmet needs for patients with Duchenne
• 10:15– Closing thoughts on the approval

Thanks for listening to the NeurologyLive Mind Moments podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

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, Daniel Ontaneda, MD, an associate professor of neurology at the Cleveland Clinic Lerner College of Medicine, sat down to discuss the recently concluded Americas Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) Forum. Ontaneda, program committee vice-chair of the meeting, provided insight on a number of notable sessions and discussions that highlighted the recent advances in the MS field. In addition, he talked about the need to innovate clinical trials, ways to tackle progressive MS, and whether drug trials are truly assessing patients at the right time points. Furthermore, he discussed multi-stage trials, the potential role of Bruton tyrosine kinase (BTK) inhibitors, and how this year's meeting differs from previous ones.
Looking for more multiple sclerosis discussion? Check out the NeurologyLive ® multiple sclerosis clinical focus page .
Episode Breakdown:

• 1:05– Overviewing sessions from ACTRIMS Forum
• 13:10– Innovating clinical trial design
• 16:15– 2023 ACTRIMS vs 2024 ACTRIMS
• 20:20– Neurology News Minute
• 23:30– Testing combination approaches for progressive MS
• 26:45– The impact of timing therapeutic interventions

This episode is brought to you by Medical World News , a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com .
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 AdComm to Review Investigational Alzheimer Agent Donanemab and Phase 3 Results

• Amylyx Mulls Over Pulling AMX0035 Following Disappointing Phase 3 PHOENIX Findings

• FDA Issues Complete Response Letter for Long-Acting Form of Glatiramer Acetate for Relapsing Multiple Sclerosis

• Phase 2 PROCEED Trial of PACAP-Targeting Antibody Lu AG09222 in Migraine Announced

Thanks for listening to the NeurologyLive ® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

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, Alcibiades Rodriguez, MD, medical director of the Comprehensive Epilepsy Center—Sleep Center at NYU Langone discussed several sleep-related topics, including the diagnosis of rare sleep disorders and hypersomnias. He spoke on the treatment of obstructive sleep apnea, the importance of adherence to medication, and the ways clinicians can help patients stick to their treatment regimen. Furthermore, he talked about the conversations between patients and clinicians to ensure an accurate diagnosis, as well as the emerging research in the sleep disorder field over the coming years.
Looking for more sleep disorders discussion? Check out the NeurologyLive ® sleep disorders clinical focus page .
Episode Breakdown:

• 1:40 – Conversations needed to differentiate diagnoses
• 4:30 – Appearance of sleep disorders and overlap
• 6:50 – Complications with untreated sleep apnea
• 8:25 – Neurology News Minute
• 10:55 – Ways to improve adherence to medication
• 13:50– Emerging research in the field

This episode is brought to you by Medical World News , a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com .
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:

• Glatiramer Acetate Depot Demonstrates Sustained Longterm Safety Profile as Potential MS Therapy

• Phase 3 DAYBREAK Trial Highlights Long-Term Efficacy of Ozanimod for Relapsing Multiple Sclerosis

• SRP-9001 Improves Duchenne Muscular Dystrophy Disease Trajectory Despite Failing to Meet Primary End Point in Phase 3 EMBARK Trial

Thanks for listening to the NeurologyLive ® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

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, Stephen From, chief executive officer of Aruna Bio, sat down to discuss the company's investigational exosome candidate AB126 and its upcoming phase 1b/2a trial in patients with ischemic stroke. He discussed the mechanism of action of the therapy, the advantages of its anti-inflammatory and neuroprotective properties, and how it will be used with other stroke treatments. Furthermore, he gave insight on the complexities with conducting stroke trials and improving neurodegeneration in poststroke patients.
Looking for more stroke discussion? Check out the NeurologyLive ® stroke clinical focus page .
Episode Breakdown:

• 1:00 – Overviewing phase 1b/2a trial
• 7:05 – Mechanism of action of AB126
• 11:15– Neurology News Minute
• 13:45– Role of AB126 in stroke treatment landscape
• 17:50– Challenges with repairing neurodegeneration in stroke

This episode is brought to you by Medical World News , a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com .
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:

• European Commission Approves Omaveloxolone as First Therapy for Friedreich Ataxia

• AVP-786 Falls Short in Phase 3 Study of Alzheimer Disease Agitation

• FDA Grants Priority Review to Efficacy Supplement for Sarepta Therapeutics’ SRP-9001 Indication Expansion

Thanks for listening to the NeurologyLive ® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

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, Igancio Mata, PhD, sat down to discuss a recently published meta-analysis on the genetic backgrounds of Parkinson disease beyond traditional European populations. Mata, an associate professor in the Genomic Medicine Institute at Cleveland Clinic, provided insight on the newly identified novel loci and how the data adds to previous genome-wide association studies. In addition, he gave thoughts on the possibility of gene therapy and some of the barriers that come with it, as well as the feasibility of incorporating genetic checkpoints into clinical trial inclusion criteria.
Looking for more movement disorder discussion? Check out the NeurologyLive ® movement disorder clinical focus page .
Episode Breakdown:

• 1:10 – Meta-analysis overview
• 3:20 – Significance of results, how they add to the literature
• 6:50 – Feasibility of incorporating genetic screening into clinical trials
• 8:40 – Neurology News Minute
• 11:15 – Realistic expectations and potential with gene therapy
• 15:45 – Next steps in advancing this research

This episode is brought to you by Medical World News , a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com .
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:

• Biogen Walks Away From Aducanumab to Prioritize Lecanemab and Alzheimer Disease Pipeline

• Updated Guidelines Published to Improve Care in Specialized Epilepsy Centers

• FDA Clears IND for Gene Therapy Candidate ETX101 in Dravet Syndrome

Thanks for listening to the NeurologyLive ® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

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, Orrin Devinsky, MD, director of the NYU Langone Comprehensive Epilepsy Center, provided commentary on a recently published study assessing video evidence of sudden unexplained deaths in toddlers. Devinsky, who also serves as a professor of neurology, discussed the importance behind crib cams, the moments captured prior to the deaths assessed, and the involvement of seizure activity even with no prior history. In addition, he spoke on the abnormal sounds and movements of these children, the ways to advance SUDC research, and the next steps in understanding and relaying the data.
Looking for more epilepsy discussion? Check out the NeurologyLive ® epilepsy clinical focus page .
Episode Breakdown:

• 1:10 – Reasons behind studying sudden death in toddlers
• 3:55 – Notable takeaways from trial results
• 8:15 – Advancing ways to research SUDC/SUDEP
• 9:40 – Neurology News Minute
• 12:05 – Realistic ways to lower convulsive activity prior to sleep
• 15:05 – What abnormal movements/noises can ellucidate
• 15:55 – Ways to expand the research further

This episode is brought to you by Medical World News , a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com .
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 Approves Takeda’s Immune Globulin Infusion, Hyqvia, for Chronic Inflammatory Demyelinating Polyneuropathy

• FDA Issues Complete Response Letter for Satsuma’s DHE Nasal Powder STS101 to Treat Acute Migraine

• ALS Agent PrimeC to Advance to Phase 3 Study After Positive PARADIGM Trial Data

Thanks for listening to the NeurologyLive ® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.