NeurologyLive® Mind Moments‪®‬ NeurologyLive

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, Katherine Peters, PhD, a neurologist and neurooncologist at the Preston Robert Tisch Brain Tumor Center at Duke University provided context on a new analysis from the phase 3 INDIGO trial, a study assessing vorasidenib in patients with mutant isocitrate dehydrogenase (mIDH) 1/2 diffuse gliomas. The conversation, which occurred at the 2024 AAN Annual Meeting, covered the covered the therapeutic potential of this agent and its impacts on quality of life, neurocognition, and seizure control. Peters, an expert in the field, provided insight on the mechanism of action of vorasidenib, the added value behind the exploratory analysis, and the next steps in research. Furthermore, she provided context on the patient sample observed and why these data may hold significant weight going forward. 

Looking for more neuromuscular discussion? Check out the NeurologyLive® epilepsy clinical focus page.

Episode Breakdown:



1:05 – Mechanism of action of vorasidenib, a mIDH 1/2 inhibitor

2:00 – Overview of exploratory analysis results

4:20 – Significance of new data, how it adds to previous primary and secondary outcomes

5:50 – Neurology News Minute

8:25 – Next steps for the study and use of vorasidenib

10:35 – Remaining unmet needs for patients with diffuse gliomas



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 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.