2025 NEALS Annual Meeting Booklet by NEALS_Consortium

Annual neals Annual neals meeting meeting 2025 2025

October 7-10, 2025

Clearwater, FL

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Dear NEALS Members & Colleagues,

Welcome Letter Welcome

We are delighted to welcome you to the 24th Annual NEALS Meeting, taking place October 7–10 This year is especially meaningful as we mark the 30th anniversary of the NEALS Consortium – three decades of collaboration, innovation, and commitment to advancing ALS research Together, we have built a foundation of excellence that continues to expand our impact across science, clinical care, and the broader ALS community Fittingly, this year’s program includes a special session, “NEALS at 30: A Legacy of Impact, ” which will reflect on the progress made and the partnerships that have shaped our journey

This year’s program highlights both the strength of our community and the urgency of discovery The Scientific Symposium, “Innovative Approaches and Future Directions for Treating Sporadic ALS,” will set the stage for discussions on emerging science. A keynote talk on CRISPR gene therapy for C9orf72 FTD/ALS and the Hot Topic Session on Gene Therapies: Now and the Future will provide timely insights into the rapidly evolving landscape of ALS treatments. We will also host an Affiliated Trial Session, highlighting study updates from NEALS-affiliated trials. The meeting also brings the return of Stats Corner: Small Trials, Big Decisions in Early Phase ALS Research, offering practical guidance on statistical design, biomarkers, and outcome measures.

Several new elements will enrich the agenda. Lightning Science Talks will feature short presentations of abstracts that will also be highlighted during the poster sessions, offering a snapshot of exciting research in progress. The NEALS ALS Organizations Forum, founded this year, will share updates on how advocacy and research organizations are aligning to strengthen the ALS ecosystem

Our Research Ambassador panel, Partners in Discovery: Research Ambassadors Driving Change, will once again showcase the vital role of individuals with lived experience in advancing ALS research, while member-focused workshops will include the NEALS Respiratory Care and Ventilation Committee Workshop, Outcome Measures training, a cross-committee session on feeding tubes, and revamped Clinical Research Staff Training & Development Workshop; all designed to expand knowledge and strengthen collaboration

We are proud to share that this year we accepted a record 219 abstracts, underscoring the depth and dedication of the ALS research community Several outstanding submissions have been recognized with abstract awards, reflecting the innovation and promise that continue to drive our field forward With over 700 in-person attendees and a robust virtual audience, the meeting continues to be a place where knowledge grows, partnerships form, and momentum builds

In honor of our 30th anniversary, we will debut a new NEALS brand video and share the first NEALS 30th Anniversary Impact Report, reflecting on milestones achieved with, and for, the ALS community. These moments are an opportunity to honor our collective legacy while looking ahead with purpose.

The success of the Annual Meeting rests on the dedication of our community. We extend our deepest gratitude to the Executive Committee, Scientific Advisory Board, PALS/CALS Advisors, Patient Advisory Committee, NEALS Committees, Leadership Working Groups, the Abstract Selection Committee and the NEALS Executive Operations Team. We are also grateful for the collaborations with foundation partners, industry partners, and government agencies. Finally, we sincerely appreciate the work of our Research Ambassadors and the entire community of people living with ALS and their caregivers who centers us and keeps us motivated year after year

Thank you for joining us for this landmark year Together, we are driving forward the science, care, and collaborations that bring hope and impact to the ALS community

With sincere appreciation

Jinsy Andrews, MD, MSc Co-Chair

James Berry, MD, MPH Co-Chair

EXECUTIVE COMMITTEE

Jinsy Andrews, MD, MSc (NEALS Co-Chair)

James Berry, MD, MPH (NEALS Co-Chair)

Leadership and Committees Leadership and Committees

SCIENTIFIC ADVISORY BOARD

Timothy Miller, MD, PhD (SAB Co-Chair)

Jeffrey Rothstein, MD, PhD (SAB Co-Chair)

Richard Bedlack, MD, PhD (Education and Patient Engagement Chair)

Merit Cudkowicz, MD, MSc (Coordinating Center Chair)

Christina Fournier, MD (Investigator Representative)

Kelly Gwathmey, MD (Investigator Representative)

Matthew Harms, MD (Investigator Representative)

Terry Heiman-Patterson, MD (Science Communication and Publication Chair)

Gale Kittle, RN, MPH (Clinical Evaluator Representative)

Shafeeq Ladha, MD (Investigator Representative)

Björn Oskarsson, MD (Investigator Representative)

Michael Robinson, MD (PALS/CALS Advisor)

Jeremy Shefner, MD, PhD (Outcomes/Monitoring Center Chair)

Dave Shulman, MBA (PALS/CALS Advisor)

Zachary Simmons, MD (Science Communication and Publication Chair)

Nicole Turcotte, MPH (Clinical Coordinator Representative)

NEALS FINANCE COMMITTEE

Jinsy Andrews, MD, MSc (NEALS Co-Chair)

James Berry, MD, MPH (NEALS Co-Chair)

Carly Allen, BA (Program Director)

Terry Heiman-Patterson, MD (Science Communication and Publication Chair)

Jeremy Shefner, MD, PhD (Outcomes/Monitoring Center Chair)

Dave Shulman, MBA (PALS/CALS Advisor)

Zachary Simmons, MD (Science Communication and Publication Chair)

Beverly Smits, MBA (Associate Program Director)

GLOBAL RESEARCH & OUTREACH LEADERSHIP WORKING GROUP (GROW)

Jinsy Andrews, MD, MSc

James Berry, MD, MPH

Hristelina Illieva, MD, PhD

Gale Kittle, RN, MPH

Eduardo Locatelli, MD, MPH

Hande Özdinler, PhD

Sabrina Paganoni, MD, PhD

Jeremy Shefner, MD, PhD

Rup Tandan, MD

Senda Ajroud-Driss, MD

Sami Barmada, MD, PhD

Robert Bowser, PhD

Robert Brown, Jr , MD, DPhil (Emeritus SAB Chair)

Merit Cudkowicz, MD, MSc

Christina Fournier, MD, MSc

Jonathan Glass, MD

Matthew Harms, MD

Brent Harris, MD, PhD

Clotilde Lagier-Tourenne, MD, PhD

Nicholas Maragakis, MD

Sabrina Paganoni, MD, PhD

Piera Pasinelli, PhD

Rita Sattler, PhD, MSc

Jeremy Shefner, MD, PhD

NEALS EXECUTIVE OPERATIONS TEAM

Carly Allen, BA (Program Director)

Beverly Smits, MBA (Associate Program Director)

Jennifer Heinly, BS (Administrative Coordinator)

Christina Smith, BS (Communications and Outreach Coordinator)

NEALS INFORMATION & COMMUNITY EDUCATION LEADERSHIP WORKING GROUP (NICE)

Christina Fournier, MD, MSc (Co-Chair)

John Novak, MD (Co-Chair)

Senda Ajroud-Driss, MD

Jinsy Andrews, MD, MSc

James Berry, MD, MPH

I-Hweii Amy Chen, MD, PhD

Lauren Elman, MD

Dominic Ferrey, MD

Stephen Goutman, MD

Kelly Gwathmey MD

Stephen Johnson, MD

Justin Kwan, MD

Margaret A Owegi, DO

Xiaowei W Su, MD, PhD

Leadership and Committees Leadership and Committees

NEALS PATIENT AND CAREGIVER ADVISORY COMMITTEE (PCAC)

Lori Andre

Mandi Bailey

Robert Bohne

Mark Buermann

Elizabeth Fassler

Nicole Johnson

Lori Larson Heller

Timothy Lowrey

Jennifer Peffer

John Peffer

Matt Rochleau

Kevin Robinson

Sridhar Tummala

NEALS COMMITTEE LEADERSHIP

Jinsy Andrews, MD, MSc (UMN Co-Chair)

Jinae Arneklev, MSN, CRNP (Advanced Practice Provider Co-Chair)

Suma Babu, MBBS, MPH (Imaging Co-Chair)

Richard Bedlack, MD, PhD (PEACe Co-Chair)

Robert Bowser, PhD (Biorepository Co-Chair)

Benjamin Rix Brooks, MD (Respiratory Care & Ventilation Co-Chair)

Andrea Charvet, PhD, RDN, LDN (Nutrition Co-Chair)

Stephanie Dobak, MS, RD, LDN, CNSC (Nutrition Co-Chair)

Claire Flaherty, PhD, MS (FTD/FALSCo-Chair)

Christina Fournier, MD (Veterans Affairs Co-Chair)

Laura Foster, MD (Palliative Co-Chair)

Andrew Geronimo, PhD (Technology in ALS Co-Chair)

Kelly Gwathmey, MD (PEACe Co-Chair)

Matthew Harms, MD (FTD/FALS Co-Chair)

Terry Heiman-Patterson, MD (Biorepository Co-Chair)

Lindsay Heyd, BA (RRE Co-Chair)

Stephanie Johnson OTR/L PhD MHS (PT/OT Co-Chair)

Stephen Johnson, MD (Technology in ALS Co-Chair)

Christopher D Lee, MD, MPH (Respiratory Care & Ventilation Co-Chair)

Gabriela Lopes, APRN (Advanced Practice Provider Co-Chair)

Ambereen Mehta, MD, MPH, FAAHPM (Palliative Co-Chair)

Jourdan Milliard, MSN, FNP-C (Advanced Practice Provider Co-Chair)

Lyle Ostrow, MD, PhD (PEACe Co-Chair)

Hande Özdinler, MD (UMN Co-Chair)

Sabrina Paganoni, MD, PhD (UMN Co-Chair)

Gary Pattee, MD (Bulbar Chair)

Erik Pioro, MD, PhD, FRCPC (Imaging Co-Chair)

Brandy Quarles, MPH, CCRC (Research Coordinator Co-Chair)

Colin Quinn, MD, MSPT (Veterans Affairs Co-Chair)

Heidi Runk, CCRC (Research Coordinator Co-Chair)

Danica Sanders, RN (Research Nurse Co-Chair)

Rita Sattler, PhD, MSc (FTD/FALS Co-Chair)

Alex Sherman, MS (Big Data Chair)

Polina Vorobeychik, MPH (Nutrition Co-Chair)

Michele Weaver, PT, DPT, NCS (PT/OT Co-Chair)

Eufrosina Young, MD (Respiratory Care & Ventilation Co-Chair)

200 PORTLAND STREET, FL 5 BOSTON, MA 02114

2025 Annual Meeting 2025 Annual Meeting

THANK YOU TO OUR 2025 ABSTRACT SELECTION COMMITTEE

Terry Heiman-Patterson, MD (Chair)

Frances Marie Aponte Caraballo, MS

Benjamin Rix Brooks, MD

I-Hweii Amy Chen, MD, PhD

Lori Chibnik, PhD, MPH

Dominic Ferrey, MD

Mark Garret, MD

Mukesh Gautam, PhD

Kelly Gwathmey, MD

Astrid Grouls, MD

Moon Han, PhD, MPH, MSc

Daragh Heitzman, MD, FAAN

Justin Kwan, MD

Shafeeq Ladha, MD

Ikjae Lee, MD

Lyle Ostrow, MD, PhD

Gary Pattee, MD

Pramod Pisharady, PhD

Erik Pioro, MD, PhD, FRCPC

Michael Pulley, MD, PhD

Adam Quick, MD

Zachary Simmons, MD

Beverly Smits, MBA

Andrea Swenson, MD

Manisha Thakore-James, MD

Lauren Tabor Gray, PhD, CCC-SLP

David Walk, MD, FAAN

Martina Wiedau, MD

THANK YOU TO OUR 2025 EVENT STAFF

Carly Allen, BA (NEALS)

Michelle Gordon

Jennifer Heinly, BS (NEALS)

Amanda Maidment, MS

Agnes Mulligan, BA

Beverly Smits, MBA (NEALS)

Christina Smith, BA (NEALS)

THANK YOU TO OUR 2025 TRAINING & WORKSHOP FACILITATORS

Clinical Research Staff Training & Development Workshop

Mariah Connolly, BS, CCRA

Meghan Hall, MS, CCRA

Lindsay Heyd, BA

Jennifer Kulesa-Kelley, RN, DNP

Lia Tamburello, BA

Outcome Measures Training

Gale Kittle, RN, MPH

Feeding Tubes and ALS: Multidisciplinary Practical Considerations

Andrea Charvet, PhD, RDN, LDN

Pamela Cazzolli, RN

Stephanie Dobak, MS, RD, LDN, CNSC

Laura Foster, MD

Lauren Tabor Gray, PhD, SLP

Tamara Le Blanc, BSRC-RRT

Ambereen Mehta, MD, MPH

Gary Pattee, MD

Mary Sedarous, MD

Polina Vorobeychik, RDN

RCV Workshop

Benjamin Brooks, MD

Christopher Lee, MD, MPH

Tamara Le Blanc, BSRC-RRT

Eufrosina Young, MD

Pending confirmationof additionalfrom RCV,OM,and MichelleG credentials

Acknowledgment of Support for the NEALS Annual Meeting (from Calaneet)

On behalf of the ALS Association, I want to extend our deep appreciation to NEALS for hosting this year’s Annual Meeting and for your unwavering commitment to advancing research and care for people living with ALS This gathering is more than a convening of experts, it is a testament to what can be achieved when knowledge, innovation, and collaboration come together with urgency and purpose

Our partnership with NEALS dates back to 1996, when we provided the foundational funding to establish NEALS We have provided funding for NEALS over the last three decades because of our shared vision: that we can find new treatments and cures for ALS

We recognize that this is no small task, but through collective thought, scientific rigor, and partnership, we are making meaningful progress Each breakthrough, each idea exchanged, and each collaborative effort moves us closer to changing the trajectory of ALS This will mean people living with ALS have more time, more options, and more hope

Our continuing support of NEALS is part of our plan to make ALS livable and cure it We are focused on accelerating research and technology, optimizing current treatments and care, and reducing the harms of ALS The leadership demonstrated by NEALS is vital to achieving these goals, and we greatly value the role you play in advancing ALS research

We are proud to support and stand alongside NEALS at this Annual Meeting Together, we are not only accelerating research but also reaffirming to people living with ALS and their families that they are not alone, that progress is happening, and that through partnerships like ours, we will make ALS livable and cure it

With gratitude, Calaneet Balas, CEO The ALS Association

THANK YOU TO OUR SUPPORTERS FOR THEIR ONGOING COMMITMENT TO NEALS!

2025 Annual Meeting Agenda*

*Agenda subject to change

MEETING AND BREAKOUT COLOR LEGEND

General Info & General Sessions

Meals & Receptions (open to all attendees)

NEALS Working Group & Committee Meetings (open to NEALS members)

NEALS Affiliated Trial: Study Investigator Meetings (limited to site investigators & study teams)

NEALS Member Training Sessions & Workshops (open to NEALS members)

TUESDAY, OCTOBER 7, 2025

NEALS Member only day NEALS Member only day

8:00a – 5:00p

8:00 – 12:00p

10:00 – 11:00a 11:00 – 12:00p

11:00 – 1:00p

12:00 – 1:00p 12:00 – 1:00p

12:30 – 2:30p

1:00 – 2:00p

Registration (Breakfast on own)

Respiratory Care & Ventilation Committee Workshop Workshop Breakout

Research Coordinator Committee Meeting

Committee Chairs: Heidi Runk, CCRC (Penn State) and Brandy Quarles, MPH, CCRC (Augusta University)

FALS/FTD Committee Meeting

Committee Chairs: Claire Flaherty-Craig, PhD, MS (Penn State), Matthew Harms, MD (Columbia University), and Rita Sattler, PhD (Barrow Neurological Institute)

Lunch

Research Nurse Committee Meeting

Committee Chairs: Danica Sanders, RN (Sean M. Healey & AMG Center for ALS at MGH)

Physical Therapists & Occupational Therapists Committee Meeting

Committee Chairs: Stephanie Johnson OTR/L PhD MHS (Augusta University), and Michele Weaver, PT, DPT, NCS (Corewell Health)

Clinical Research Staff Training & Development Workshop

Advance Practice Provider Committee Meeting

Committee Chairs: Jourdan Milliard, MSN, FNP-C (Barrow Neurological Institute), JinAe Arneklev, MSN, CRNP (Johns Hopkins University), and Gabriela Lopes, APRN (Nova Southeastern University)

Ballroom Foyer

(Opal Sol) Solstice IV (Opal Sol) Solstice III

Sand Dune Sea Watch

Event Pool Lawn

Beach Breeze

Sea Shore Opal Sand

Beach View

All meetings in Opal Sands unless otherwise noted.

2025 Annual Meeting Agenda*

NEALS Member only day

TUESDAY, OCTOBER 7, 2025

NEALS Member only day

(continued)

All meetings in Opal Sands unless otherwise noted.

1:00 – 2:00p

Imaging Committee Meeting

Committee Chairs: Erik Pioro, MD, PhD, FRCPC (University of British Columbia) and Suma Babu, MBBS, MPH (Sean M. Healey & AMG Center for ALS at MGH)

Sand Dune

2:00 – 3:00p

2:30 – 3:30p

3:00 – 7:30p

3:00 – 4:00p

4:00 – 5:00p

5:00 – 6:00p

Palliative Committee Meeting

Committee Chairs: Ambereen Mehta, MD, MPH, FAAHPM (Johns Hopkins University) and Laura Foster, MD (University of Colorado)

Research, Recruitment, and Retention in ALS (RRE) Committee Meeting

Committee Chairs: Lindsay Heyd, BS (Sean M Healey & AMG Center for ALS at MGH) and Sabrina Paganoni, MD, PhD (Sean M. Healey & AMG Center for ALS at MGH)

Refreshment Break

Outcome Measures Training

Technology in ALS Committee Meeting

Committee Chairs: Andrew Geronimo, PhD (Penn State), Sabrina Paganoni, MD, PhD (Sean M Healey & AMG Center for ALS at MGH) and Stephen Johnson, MD (Mayo Clinic Scottsdale)

Nutrition Committee Meeting

Committee Chairs: Andrea Charvet, PhD, RDN, LDN (Nova Southeastern University), Stephanie Dobak, MS, RD, LDN (Jefferson Weinberg ALS Center), and Polina Vorobeychik, MPH (Washington University ALS Center)

Upper Motor Neuron Committee Meeting

Committee Chairs: Sabrina Paganoni, MD, PhD (Sean M. Healey & AMG Center for ALS at MGH), Jinsy Andrews, MD, MSc (New York University), and Hande Özdinler, MD (Northwestern University Feinberg School of Medicine)

Bulbar Committee Meeting

Committee Chair: Gary Pattee, MD (University of Nebraska Medical Center)

Big Data Committee Meeting

Committee Chair: Alexander Sherman, MS (Sean M Healey & AMG Center for ALS at MGH)

Beach Breeze

Sea Side

Ballroom Foyer

Opal Sea

Sand Dune

Sand Dollar

Beach Salons

Sea Side

Opal Sun

2025 Annual Meeting Agenda*

subject to change

NEALS Member only day

TUESDAY, OCTOBER 7, 2025

NEALS Member only day

5:00 – 6:00p

Big Data Committee Meeting

5:00 – 6:00p

6:15 – 7:15p

All meetings in Opal Sands unless otherwise noted.

Committee Chair: Alexander Sherman, MS (Sean M. Healey & AMG Center for ALS at MGH)

Respiratory Care & Ventilation Committee Meeting

Committee Chairs: Christopher D. Lee, MD, MPH (Vanderbilt University), Eufrosina Young, MD (SUNY Upstate) and Benjamin Rix Brooks, MD (Clinical Trials Planning LLC)

· NEALS Information & Community Education (NICE) Working Group

Opal Sand (continued)

Stitching Strength Documentary Screening (External Event)

Opal Sun To be confirmed Sand Dollar

(End of NEALS Member Only day)

2025 Annual Meeting Agenda*

*Agenda subject to change

Available

MEETING AND BREAKOUT COLOR LEGEND

General Info & General Sessions

Meals & Receptions (open to all attendees)

NEALS Working Group & Committee Meetings (open to NEALS members)

Day One Day One

7:00a – 4:30p

7:00 – 8:30a

NEALS Affiliated Trial: Study Investigator Meetings (limited to site investigators & study teams)

NEALS Member Training Sessions & Workshops (open to NEALS members)

WEDNESDAY, OCTOBER 8, 2025

All meetings in Opal Sol unless otherwise noted.

Ballroom Foyer

7:15 – 8:15a Safety, Tolerability, and Exploratory Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants with SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS)

7:15 – 8:15a

Safety, Tolerability, and Exploratory Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants with SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS)

CDC Informational Session: How the National ALS Registry can Support your Research Session Lead: Paul Mehta, MD (National ALS Registry)

8:30 – 10:30aNEALS Co-Chairs

Opening Session: NEALS at 30: A Legacy of Impact

Jinsy Andrews, MD, MSc (Columbia University)

Session Leads: NEALS Co-Chairs

James Berry, MD, MPH (Sean M Healey & AMG Center for ALS at MGH)

Speakers

Equinox I Equinox II & III

Ballroom

Jinsy Andrews, MD, MSc (New York University)

James Berry, MD, MPH (Sean M. Healey & AMG Center for ALS at MGH)

Merit Cudkowicz, MD, MSc (Sean M. Healey & AMG Center for ALS at MGH)

Speakers:

Timothy Miller, MD, PhD (Washington University in St. Louis)

Jeremy Shefner, MD, PhD (Barrow Neurological Institute)

10:30 – 10:50a

10:50 – 12:20p

11:00a

Shubhangi Lal, MS, MBA ((Foundation for the National Institutes of Health ()Collin HCorrigan-Curray, MD, JD (Food & Drug Registration Breakfast

Merit Cudkowicz, MD, MSc (Sean M. Healey & AMG Center for ALS at MGH)

Jeremy Shefner, MD, PhD (Barrow Neurological Institute)

Presentation: ASO Therapies for ALS

Timothy Miller, MD, PhD (Washington University ALS Center)

Break

NEALS Affiliated Trial Session

Session Lead: Jinsy Andrews, MD, MSc (New York University)

Presentations: Healey ALS MyMatch Program Update

Accelerating Medicines Partnership (AMP) ALS

Suma Babu, MBBS, MPH (Sean M. Healey & AMG Center for ALS at MGH)

Ballroom

2025 Annual Meeting Agenda*

Day ONE Day ONE

10:50a – 12:20p

(continued)

WEDNESDAY, OCTOBER 8, 2025

NEALS Affiliated Trial Session (continued)

Session Lead: Jinsy Andrews, MD, MSc (New York University)

John 11:15a 11:30a and Learnings From the Phase 3 Trial Evaluating Sodium Phenylbutyrate and Taurursodiol in Amyotrophic Lateral Sclerosis (PHOENIX)

*Agenda subject to change

All meetings in Opal Sol unless otherwise noted.

Ballroom

EPISOD1 study update on AMT-162 Gene Therapy for Individuals with SOD1 associated ALS

George Apostol, MD (uniQure)

Healey ALS Platform Trial – Adaptations and Next Regimens

Merit Cudkowicz, MD, MSc (Sean M Healey & AMG Center for ALS at MGH)

Sabrina Paganoni, MD, PhD (Healey & AMG Center for ALS)

11:55a

LUMINA Study Update

AYA trial: Double-blind, placebo-controlled trial of RIPK1 inhibitor (SAR443820) in ALS

Lauren Kett, MD, PhD (Amylyx Pharmaceuticals)

Nazem Atassi, MD, MBA (Sanofi)

Sabrina Paganoni, MD, PhD (Sean M. Healey & AMG Center for ALS at MGH)

Merit Cudkowicz, MD, MSc (Healey & AMG Center for ALS)

12:15p

ALL ALS Study Update

ALS: Year 1 Progress and Future Enrollment Goals

James Berry, MD, MPH (Sean M. Healey & AMG Center for ALS at MGH)

James Berry, MD, MPH (Healey & AMG Center for ALS)

Robe

Robert Bowser, PhD (Barrow Neurological Institute)

rt Bowser, PhD (Barrow Neurological Institute)

Lunch

12:20 – 1:45p Y Platform Trial updates what's new for 2025

Merit Cudkowicz, MD, MSc (Healey & AMG Center for ALS)

12:45 – 1:45poA Phase 2A Study of TPN-101, A Nucleoside Reverse Transcriptase Inhibitor, in Patients with C9ORF72-Related

Clene NIH - expanded access program meeting

Andrew Satlin, MD (Transposon Therapeutics)

ALL ALS Meeting

12:45 – 1:45p Ping Progress: Exciting Updates from NEALSAffiliated Trials

Jinsy Andrews, MD, MSc (NEALS Co-Chair; Columbia Universi 12:45 – 3:15p

Feeding Tubes and ALS: Multidisciplinary Practical Considerations Workshop Breakout

1:10 – 2:10p Christophasdfsdfsdf er Coffey, PhD (University of Iowa) 2:00 – 3:05p

NEALS Biorepository Informational Session

Lighting Science Talks

Session Leads: Science Advisory Board Co-Chairs

Timothy Miller, MD, PhD (Washington University ALS Center) and Jeffrey Rothstein, MD, PhD (Johns Hopkins University)

Science Presentations: 3-Minute Talks

Chloroviruses and ALS

Gary Pattee, MD (Neurology Associates, P C )

Presymptomatic gene expression changes in motor neurons in a large animal model of motor neuron degeneration

Stephen Kolb, MD, PhD (Ohio State University)

(Opal Sands)

Pool Event Lawn

Equinox I

Equinox II & III

(Opal Sands) Sea Salons (Opal Sands) Sand Salons

(Opal Sands) Beach Salons

Ballroom

2025 Annual Meeting Agenda*

Day ONE Day ONE

2:00 – 3:05p

(continued)

WEDNESDAY, OCTOBER 8, 2025

Lighting Science Talks (continued)

Session Leads: Science Advisory Board Co-Chairs

Timothy Miller, MD, PhD (Washington University ALS Center) and Jeffrey Rothstein, MD, PhD (Johns Hopkins University)

Science Presentations: 3-Minute Talks (continued)

*Agenda subject to change

All meetings in Opal Sol unless otherwise noted. Ballroom

Phenotypic Drug Screening in Human iPSC-Derived Neurons Identifies Modifiers of Dipeptide Repeat-Induced Toxicity and TDP-43 Pathology in Amyotrophic Lateral Sclerosis/Frontotemporal Dementia

Christine Marques, PhD (Massachusetts General Hospital and Harvard Medical School)

ACE-2223, a novel oral small molecule for non genetically modified ALS, treats both gain- and loss-of-function TDP-43 pathology, extends survival and decreases NfL in vivo and has a favorable preclinical safety profile

Katie Planey, PhD, MBA (Acelot)

Talineuren (TLN): A GM1-Loaded Nanoliposomal Therapeutic Targeting

Mitochondrial Dysfunction and Proteostasis in ALS

Smita Saxena, PhD (University of Missouri)

ALS-associated circular RNA hsa circ 0000119 forms a ribonucleoprotein complex enriched with proteins central to ALS pathophysiology

Kate Smith, MD-PhD Trainee (The University of Alabama at Birmingham)

Audience Q&A

Clinical Presentations: 3-Minute Talks

FUNCtion ALS: A Phase 1/2 trial evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics and exploratory efficacy of TRCN1023, an antisense oligonucleotide restoring UNC13A, in adult persons with Amyotrophic Lateral Sclerosis

Irina Antonijevic, MD, PhD (Trace Neuroscience)

Characterizing the CSF Biomarker Signature of Calpain-2 Activity in ALS and the Biomarker Impact of Calpain-2 Inhibition in a Preclinical Model of ALS

Robert Bowser, PhD (Barrow Neurological Institute)

Impact of Intrathecal delivery of INS1202 AAV9-SOD1-shRNA on hallmarks of neurodegeneration in a murine disease model of ALS and a patient-derived in vitro model

Laura Ferraiuolo, PhD (Insmed Incorporated)

The Effects of Glucagon-Like Peptide-1 Receptor Agonists Medications on Amyotrophic Lateral Sclerosis

Kiran Kumari, MD (Vandalia Health)

2025 Annual Meeting Agenda*

Day ONE Day ONE (continued)

2:00 – 3:05p

WEDNESDAY, OCTOBER 8, 2025

Lighting Science Talks (continued)

Session Leads: Science Advisory Board Co-Chairs

Timothy Miller, MD, PhD (Washington University ALS Center) and Jeffrey Rothstein, MD, PhD (Johns Hopkins University)

Science Presentations: 3-Minute Talks (continued)

*Agenda subject to change

All meetings in Opal Sol unless otherwise noted.

Ballroom

2:00 – 3:00p

3:30 – 4:30p

1:30 – 5:00p

4:30 – 6:30p

Multicenter imaging study in ALS to demonstrate scalability and clinical trial readiness of multimodal MRI: Preliminary data

Pramod Pisharady, PhD (University of Minnesota)

Audience Q&A

LTX-002-101 NeurALS Study Update

Industry Roundtable

Session Lead: Nicholas Maragakis, MD (Johns Hopkins University)

Asa Abeliovich, MD PhD (Leal Therapeutics)

Irina Antonijevic, MD, PhD (Trace Neuroscience)

Ram Miller, MDCM, MS, FRCPC (Novartis)

Shilpa Sambashivan, PhD (Nura Bio)

Bernd-Jan Sanson, MD, PhD (Prilenia)

Audience Q&A

Global Research & Outreach Working Group (GROW) Meeting

Poster Session 1

(End of day 1)

Equinox I

Ballroom

Equinox I & II

(Opal Sands) Ballroom

2025 Annual Meeting Agenda*

Day two Day two

7:00a – 4:15p

7:00 – 8:30a

7:15 – 8:15a

*Agenda subject to change

THURSDAY, OCTOBER 9, 2025

THURSDAY, NOVEMBER 3 2022

Registration

Breakfast

Learnings from EAPs in ALS

Session Leads: Suma Babu, MBBS, MPH (Sean M. Healey & AMG Center for ALS at MGH) and Björn Oskarsson, MD, FAAN (Mayo Clinic)

Trehalose EAP Update

Ibudilast EAP Update

Björn Oskarsson, MD, FAAN (Mayo Clinic)

Sabrina Paganoni, MD, PhD (Sean M. Healey & AMG Center for ALS at MGH)

Trehalose EAP Update

Antisense oligonucleotide jacifusen for FUS-ALS: an investigatorinitiated, multicenter, open-label case series

Matthew Harms, MD (Columbia University)

All meetings in Opal Sol unless otherwise noted.

Ballroom Foyer

Equinox II & III

Sabrina Paganoni, MD, PhD (Sean M Healey & AMG Center for ALS at MGH)

Tofersen EAP Update

Role of EAPs in ASO development for genetic ALS

Matthew Harms, MD (Columbia University)

Robert Bucelli, MD, PhD (Washington University ALS Center)

Tofersen EAP Update

Robert Bucelli, MD, PhD (Washington University ALS Center)

7:15 – 8:15a Anniversary Bike for ALS Initiative

Amylyx A114-001 LUMINA Study Meeting

Jeremy Shefner, MD, PhD (Barrow Neurological Institute)

Day 2 Kickoff

8:30 – 9:00a Committee Spotlight: Updates from the Physical Therapist/Occupational Therapist Committee, Nutrition Committee, Respiratory Care and Ventilation Committee, and Palliative Committee

Session Lead: NEALS Co-Chair

James Berry, MD, MPH (Sean M Healey & AMG Center for ALS at MGH)

9:00 – 10:00aClaire MaacAdam PT, NCS (Massachusetts General Hospital)

Partners in Discovery: Research Ambassadors Driving Change

Michele Weaver PT, DPT (Corewell Health)

Session Lead: Richard Bedlack, MD, PhD (Duke ALS Clinic)

Ambereen Mehta, MD, MPH, FAAHPM (Johns Hopkins)

Stephanie Dobak, MS, RD, LDN, CNSC (Jefferson Weinberg ALS Center)

Robert Bowser, PhD (Barrow Neurological Institute)

Eufrosina Young, MD (SUNY Upstate Medical University)

Terry Heiman-Patterson, MD (Temple University)

Andrea Lytle Peet (ALS Research Ambassador)

David Shulman, MBA (ALS Research Ambassador)

Jean Swidler (ALS Research Ambassador)

Audience Q&A

10:00 – 10:20a

10:20 – 12:20p

Break

Scientific Symposium: Innovative Approaches and Future Directions for Treating Sporadic ALS

Session Leads: Science Advisory Board Co-Chairs

Timothy Miller, MD, PhD (Washington University ALS Center) and

Jeffrey Rothstein, MD, PhD (Johns Hopkins University)

Equinox I

Ballroom

2025 Annual Meeting Agenda*

Day two Day two (continued)

10:20a–12:20p

JohnHansen-Flaschen,MD(PennMedicine)

THURSDAY, OCTOBER 9, 2025

Scientific Symposium: Innovative Approaches and Future Directions for Treating Sporadic ALS (continued) Session Leads: Science Advisory Board Co-Chairs

Timothy Miller, MD, PhD (Washington University ALS Center) and Jeffrey Rothstein, MD, PhD (Johns Hopkins University)

*Agenda subject to change

All meetings in Opal Sol unless otherwise noted.

10:40a

11:10a

Presentations:

All ALS is genetic – is this true and does it matter?

Ammar Al-Chalabi, PhD (King’s College London)

Sporadic ALS iPSNs to understand disease heterogeneity and mechanistic underpinnings

Alyssa Coyne, PhD (Johns Hopkins University)

11:30aandLearningsFromthePhase3TrialEvaluatingSodium PhenylbutyrateandTaurursodiolinAmyotrophicLateralSclerosis (PHOENIX)

Exploring the Genetic Landscape of Nucleoporin Variants in ALS

Julia Kaye, PhD (Gladstone Institutes)

11:50aAYAtrial:Double-blind,placebo-controlledtrialofnhibitor (SAR443820)inALS

Nazem

Atass i,MD,MBA(Sanofi)

TDP-43 loss induces cryptically-spliced proteins in ALS

Sahba Seddighi, MD-PhD Candidate (Johns Hopkins School of Medicine)

12:10pALS:Year1ProgressandFutureEnrollmentGoals

JamesBerry,MD,MPH(Healey&AMGCenterforALS)

Proteomics identifies a panel of plasma proteins that can be used as a diagnostic biomarker of ALS

RobertBow ser,PhD(B arrowNeurologicalInstitute)

Bryan Traynor, MD, PhD (National Institute on Aging)

12:20–2:00pEYPlatformTrialupdateswhat'snewfor2025

Lunch

MeritCudkowicz,MD,MSc(Healey&AMGCenterforALS)

12:45–2:00poAPhase2AStudyofTPN-101,ANucleosideReverse TranscrtaseInhibitor,inPatientswithC9ORF72-Related AndrewSatlin,MD(TransposonTherapeutics) 2:15–3:15pPingProgress:ExcitingUpdatesfromNEALSAffiliatedTrials

HEALEY ALS Platform Trial Study Meeting

Hot Topic Session: Gene Therapies, now and the Future

JinsyAndrews,MD,MSc(NEALSCo-Chair;ColumbiaUniversi

Session Lead: Jonathan Glass, MD (Emory University)

1:10–2:10p Ch ristophasdfsdfsdf 3:20–4:20p

Ballroom (Opal Sands) Pool Event Lawn

Rebecca Ahrens-Nicklas, MD, PhD (Children’s Hospital of Philadelphia)

Robert Brown, MD, DPhil (University of Massachusetts Medical School)

Rebecca Ahrens-Nicklas, MD, PhD (Children’s Hospital of Philadelphia)

Stanley Crooke, MD, PhD (n-Lorem)

Robert Brown, MD, DPhil (University of Massachusetts Medical School)

Stanley Crooke, MD, PhD (n-Lorem)

Stats Corner: Small Trials, Big Decisions in Early Phase ALS Research

Session Lead/Moderator: Lori Chibnik, PhD (MGH Biostatistics)

Presentations:

Eric Macklin, PhD (MGH Biostatistics)

Melanie Quintana, PhD (Berry Consultants)

Peng Sun, PhD (Biogen)

Solstice I Ballroom (Opal Sands) Sea Salons (Opal Sands) Sand Salons m to be confirmed Ballroom

2025 Annual Meeting Agenda*

Day two Day two (continued)

4:20–6:20p

6:20–6:40p

6:40–9:00p

THURSDAY, OCTOBER 9, 2025

Poster Session 2

Group Photo

Annual Beachside Dinner

(End of day 2)

*Agenda subject to change

All meetings in Opal Sol unless otherwise noted.

(Opal Sands) Ballroom (Opal Sands) Lobby

(Opal Sands) Pool Event Lawn

2025 Annual Meeting Agenda*

Day three Day three

7:00a – 12:30p

7:00 – 8:30a

FRIDAY, OCTOBER 10, 2025

THURSDAY, NOVEMBER 3 2022

Registration

2025 Annual Meeting Agenda*

Day three Day three

10:50 – 12:50p

FRIDAY, OCTOBER 10, 2025

THURSDAY, NOVEMBER 3 2022

Abstract Awards

Session Lead: Terry Heiman-Patterson, MD (Temple University)

Basic Science Award

Presented by: Terry Heiman-Patterson, MD (Temple University)

Clinical Investigation Award

Presented by: Terry Heiman-Patterson, MD (Temple University)

Upper Motor Neuron Achievement Awards

Presented by: Christine Hendrickson, ARNP (Spastic Paraplegia Foundation Ambassador)

Platform Presentations

Session Lead: Terry Heiman-Patterson, MD (Temple University)

AAV-mediated gene augmentation therapy for NEK1-ALS

*Agenda subject to change

All meetings in Opal Sol unless otherwise noted.

Ballroom

Katharina Meijboom, PhD (University of Massachusetts Chan Medical School)

Final Results from the Phase 3 VALOR Trial and Open-Label Extension Evaluating Efficacy and Safety of Tofersen in Adults with SOD1-ALS

Timothy Miller, MD, PhD (Washington University ALS Center)

Progressive Upper Motor Neuron Degeneration and Neuroinflammation in TAR4/4 Mice: A New Model for ALS and ALS/FTD

Emel Ulupinar, MD, PhD (Northwestern University)

Autologous Hybrid TREG/Th2 T Stem Cell Therapy (RAPA-501) is Feasible and Safe in Advanced-Stage ALS Patients With Severe Respiratory Insufficiency

James Berry, MD, MPH (Sean M Healey & AMG Center for ALS at MGH)

Ex-Vivo Validation of a Fluorescent Clinical Retinal Tracer to Detect TDP-43 in ALS patients With Applications in FTD and LATE

Alex Huang, MD, PhD (Amydis)

Phase 2b PARADIGM Results Demonstrate Safety, Clinical Signals, and Biomarker Engagement of PrimeC in ALS, Supporting Advancement to Phase 3

Merit Cudkowicz, MD, MSc (Sean M Healey & AMG Center for ALS at MGH)

Audience Q&A

Ballroom

2025 Annual Meeting Agenda*

Day Three Day Three (continued)

FRIDAY, OCTOBER 10, 2025

12:50 – 1:50p

1:10 – 2:10p

1:10 – 2:10p Lunch

COYA 302 Phase 2 / ALSTARS Trial Study Meeting

ALS Organizations Forum (Closed Session)

(End of day 3)

*Agenda subject to change

All meetings in Opal Sol unless otherwise noted.

Ballroom Foyer

Equinox I

Equinox II & III

2025 awards 2025 awards

THE GUPTA FAMILY ENDOWED PRIZE FOR INNOVATION IN ALS CARE

The Gupta Family Endowed Prize for Innovation in ALS Care is a global prize to honor ground-breaking new approaches that lead to exceptional care for individuals living with amyotrophic lateral sclerosis (ALS). This annual prize is awarded to a nominated team that has developed promising new approaches to improving care for people living with ALS The goal of this prize is to encourage idea sharing, innovation, and forward thinking on scalable ongoing and ground-breaking projects that have directly improved ALS patient care.

BASIC SCIENCE

ABSTRACT AWARD

The Basic Science Abstract Award will be given to an outstanding abstract poster presentation focused on basic science.

CLINICAL INVESTIGATION

ABSTRACT AWARD

The Clinical Investigation Abstract Award will be given to an exceptional abstract poster presenter focused on clinical investigation.

DRS

AYEEZ AND SHELENA LALJI & FAMILY ALS STUDENT SCHOLAR AWARD FOR REPAIR AND REGENERATIVE MECHANISMS IN ALS

The Drs. Ayeez and Shelena Lalji & Family ALS Student Scholar Award in Repair Mechanisms in ALS recognizes the brightest young scientists in the field of ALS and facilitates meaningful research to understand the mechanisms underlying axonal and neuronal repair and regeneration in ALS and/or other neurodegenerative disorders that may be translatable to ALS.

UMN ACHIEVEMENT AWARD

The seventh annual UMN Achievement Award will be given to two abstracts focusing on PLS/UMN research. The UMN Achievement Award is supported by the Spastic Paraplegia Foundation (SPF) to promote UMN research and recognize those who contribute significantly to this field.

NEALS Member trainings NEALS Member trainings

RESPIRATORY CARE & VENTILATION COMMITTEE WORKSHOP

During this workshop, participants will engage in live demonstrations covering various aspects of ventilation and respiratory care for ALS patients These include an extended learning experience on the use of portable spirometers, data dashboards for remote monitoring of pulmonary function tests and home ventilation systems Pulmonary experts will showcase airway clearance techniques and equipment, bi-level positive airway pressure support and non-invasive ventilation.

The goal of the workshop is to engage the community of ALS providers for hands-on learning on improvements and innovations in the field of respiratory care This activity provides an opportunity to discuss the current state of diverse practices and goals of achieving ALS respiratory standards of care

CLINICAL RESEARCH STAFF TRAINING & DEVELOPMENT WORKSHOP

The Clinical Research Staff Training & Development Workshop is led by experienced members from the NEALS Consortium Coordinating Centers and will emphasize site management techniques, including study feasibility considerations, study startup and activation, participant recruitment and retention strategies, the informed consent process, ensuring Good Clinical Practice (GCP), Institutional Review Board (IRB) submissions, utilizing an electronic Regulatory Document System (eISF), addressing staffing models needs, and preparation for monitoring visits

Participants who successfully complete this workshop will receive a certificate of completion. This certification reflects a participant's dedication to upholding high standards in clinical research management and may be advantageous for those pursuing continuing education activities

OUTCOME MEASURES TRAINING

The Outcome Measures training is for new and experienced NEALS Clinical Trial Evaluators. The session will focus on training for the core outcomes measures: ALSFRS-R (ALS Functional Rating Scale, Revised), vital capacity, and muscle strength using handheld dynamometry Attendance at this session will fulfill training requirements for new and recertifying evaluators. Updates to outcome measure protocols, as well as hands-on practice and technique checks will be part of this training

FEEDING TUBES AND ALS: MULTIDISCIPLINARY PRACTICAL CONSIDERATIONS

Dive into an enriching learning experience at the NEALS Cross-Committee Workshop, focusing on "Feeding Tubes and ALS: Multidisciplinary Practical Considerations " This hybrid workshop will unite leaders from the NEALS Palliative, Respiratory Care and Ventilation, Nutrition, and Bulbar Committees. Designed for NEALS members, the workshop aims to integrate expertise across these specialties, providing a comprehensive understanding of feeding tube management in ALS care Through cross-disciplinary presentations, group Q&A, and interactive breakout sessions, participants will explore practical applications of feeding tubes, address procedural considerations, and discuss evidence-based best practices to enhance patient outcomes

This collaborative forum will foster cross-committee dialogue, bridging clinical practice with research to develop innovative solutions and improve patient outcomes. Don’t miss this opportunity to engage with peers and contribute to the future of ALS care.

COLLABORATE WITH NEALS

AFFILIATED TRIAL SUPPORT

Governed by a seasoned Leadership Team, NEALS has successfully partnered with industry and academic researchers to conduct high-quality ALS studies for over 25 years and has grown to be the largest ALS research consortium in the world.

Recruitment and retention across NEALS-affiliated studies have been consistently excellent, and NEALS has pioneered a number of innovations aimed at improving clinical trial efficiency. NEALS has over 150 trial-ready sites with more than 1,000 individual members. The members of NEALS are actively engaged in a range of ALS clinical research studies, including clinical trials and observational studies.

Becoming a NEALS-Affiliated Trial can include support for mechanisms such as:

Webinars both for people living with ALS and trial sites

Sharing Study Information with the NEALS membership through emails, announcements, and website content

Email Communication to NEALS Investigators

Engagement with NEALS Committees

Investigator meeting at the Annual NEALS Meeting

Site Selection Support including recommendations from the NEALS Site Selection Committee for trial sites

Community Engagement including interactions with ALS Research Ambassadors, support creating Patient Advisory Boards

MEET WITH THE NEALS SCIENTIFIC ADVISORY BOARD

An experienced Scientific Advisory Board and Executive Committee advises NEALS research activities. Each member brings a depth of experience and a unique perspective to the governance of NEALS.

The NEALS SAB is available to meet with investigators to advise on clinical and pre-clinical projects:

Provides a forum for investigators and industry to evaluate new ideas for drugs, technologies, and trials

Welcomes presentations from industry, NEALS members, and academic partners during scheduled teleconferences

Offers confidential feedback from seasoned ALS scientists and trialists

NEALS can offer a group CDA that includes members of the NEALS Leadership team, and the presentation will remain confidential. Additionally, there will be an opportunity for follow-up discussions after the meeting.

PEACe COMMITTEE

We invite research, industry, and foundation partners to share opportunities with our ALS Research Ambassadors, who are graduates of the ALS Clinical Research Learning Institute (CRLI)® program dedicated to advocating for and contributing to ALS research. Whether you’re seeking patient insights, advisory engagement, or advocacy collaboration, this is a unique opportunity to connect with individuals who are directly impacted by ALS and deeply engaged in the research community.

2025 NEALS ABSTRACT POSTER SESSIONS

POSTER SESSION 1:

WEDNESDAY, OCT 8

4:30 - 6:30 PM

POSTER SESSION 2:

THURSDAY, OCT 9 4:20 - 6:20 PM

Affiliated Trials Affiliated Trials

STUDY NAME 3

A Phase 1-3 Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION363 in Amyotrophic Lateral Sclerosis Patients With Fused in Sarcoma Mutations (FUS-ALS)

PHASE

SPONSOR

Ionis Pharmaceuticals, Inc

STUDY TYPE

Interventional

STUDY NAME 1/2 Biogen

A Phase 1/2 Multiple-Ascending-Dose Study With a Long-Term Open-Label Extension to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Effect on Disease Progression of BIIB105 Administered Intrathecally to Adults With Amyotrophic Lateral Sclerosis With or Without Poly-CAG Expansion in the ATXN2 Gene

PHASE

SPONSOR

STUDY TYPE

Interventional

STUDY NAME 2 Alector

A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AL001 in C9orf72-Associated Amyotrophic Lateral Sclerosis

PHASE

SPONSOR

STUDY TYPE

Interventional

PHASE

STUDY NAME 2 PTC Therapeutics

A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of SAR443820 in Adult Participants With Amyotrophic Lateral Sclerosis, Followed by an Open-label Extension

SPONSOR

STUDY TYPE

Interventional

STUDY NAME PHASE

A Phase 2a Study of TPN-101 in Patients With Amyotrophic Lateral Sclerosis (ALS) and/or Frontotemporal Dementia (FTD) Associated With Hexanucleotide Repeat Expansion in the C9orf72 Gene (C9ORF72 ALS/FTD)

SPONSOR

Transposon Therapeutics, Inc

STUDY TYPE 2

Interventional

STUDY NAME

A Phase 2a, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate Safety, Tolerability, Pharmacodynamic Markers, and Pharmacokinetics of AP-101 in Patients With Familial Amyotrophic Lateral Sclerosis (fALS) and Sporadic Amyotrophic Lateral Sclerosis (sALS)

PHASE

SPONSOR

AL-S Pharma AG

STUDY TYPE

Interventional

STUDY NAME 3

A Phase 3 Global, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Pridopidine in Participants with ALS

STUDY NAME

A Phase 3 Randomized, Placebo-Controlled Trial With a Longitudinal Natural History Run-In and Open-Label Extension to Evaluate BIIB067 Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation

STUDY NAME

A Phase 3, Open-Label Extension of COURAGE-ALS (CY 5031)

PHASE

SPONSOR

Prilenia Therapeutics

STUDY TYPE

Interventional

PHASE

3 Biogen

SPONSOR

STUDY TYPE

Interventional

PHASE

SPONSOR

Cytokinetics

STUDY TYPE

Interventional

STUDY NAME 2

A Phase IIa Trial to Evaluate the Safety, Tolerability, and Biological Activity of LAM-002A (Apilimod Dimesylate Capsules) in C9ORF72-Associated Amyotrophic Lateral Sclerosis

PHASE

SPONSOR

AI Therapeutics

STUDY TYPE

Interventional

STUDY NAME

A Phase III, Randomized, Double-Blind, PlaceboControlled, Multicenter Trial to Evaluate the Safety and Efficacy of AMX0035 Versus Placebo for 48week Treatment of Adult Patients With Amyotrophic Lateral Sclerosis (ALS)

PHASE

SPONSOR

Amylyx Pharmaceuticals Inc

STUDY TYPE

Interventional

STUDY NAME 3 Biogen

A Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BIIB067 Administered to Adult Subjects With ALS and Confirmed Superoxide Dismutase 1 Mutation (VALOR)

PHASE

SPONSOR

STUDY TYPE

Interventional

STUDY NAME N/A

ACCESS ALL ALS Study

PHASE

SPONSOR

NIH/NINDS

STUDY TYPE

Observational

STUDY NAME N/A

ALS MyMatch

ALSTARS Study: A Phase 2 Study of COYA 302 in ALS

PHASE

SPONSOR

Sean M. Healey & AMG Center for ALS at Mass General

STUDY TYPE

Observational

PHASE

STUDY NAME 2 Coya Therapeutics

SPONSOR

STUDY TYPE

Interventional

AMX0114 in Adult Participants With Amyotrophic Lateral Sclerosis (LUMINA)

PHASE

STUDY NAME 1 Amylyx Pharmaceuticals Inc

SPONSOR

STUDY TYPE

Interventional

STUDY NAME 1/2

FUNCtion ALS – A Phase I/II Clinical Trial

PHASE

SPONSOR

Trace Neuroscience

STUDY TYPE

Interventional

STUDY NAME 2/3

HEALEY ALS Platform Trial

PHASE

SPONSOR

Sean M Healey & AMG Center for ALS at Mass General

STUDY TYPE

Interventional

PHASE

STUDY NAME 1 Leal Therapeutics

NeurALS: A First-in-Human, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study of Intrathecally Administered LTX-002 in Adult Participants with Amyotrophic Lateral Sclerosis”

SPONSOR

STUDY TYPE

Interventional

STUDY NAME N/A

PREVENT ALL ALS - Longitudinal Biomarker Study for Participants Who Are Genetically At Risk for Amyotrophic Lateral Sclerosis (ALS)

PHASE

SPONSOR

NIH/NINDS

STUDY TYPE

Observational

STUDY NAME N/A

Radicava® (Edaravone) Findings in Biomarkers

From ALS (REFINE-ALS)

PHASE

SPONSOR

Mitsubishi Tanabe Pharma America

TYPE OF CLINICAL TRIAL

Observational

PHASE

STUDY NAME 1/2 uniQure

Safety, Tolerability, and Exploratory Efficacy Study of Intrathecally Administered Gene Therapy AMT162 in Adult Participants with SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS)

SPONSOR

STUDY TYPE

Interventional

Affiliated EAPs Affiliated EAPs

STUDY NAME

Intermediate-sized Expanded Access Protocol for CNM-Au8 in Amyotrophic Lateral Sclerosis (ALS)

STUDY NAME

Pridopidine in Amyotrophic Lateral Sclerosis (EAP 2)

PHASE

N/A

SPONSOR

NIH/NINDS

STUDY TYPE

Expanded Access

STUDY NAME

RAPA-501 Therapy for ALS Expanded Access Protocol

PHASE

N/A

SPONSOR

NIH/NINDS

STUDY TYPE

Expanded Access

PHASE

N/A

SPONSOR

NIH/NINDS

STUDY TYPE

Expanded Access

2025 NEALS Annual Meeting Booklet

Annual neals Annual neals meeting meeting 2025 2025

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Welcome Letter Welcome

EXECUTIVE COMMITTEE

Leadership and Committees Leadership and Committees

SCIENTIFIC ADVISORY BOARD

NEALS FINANCE COMMITTEE

NEALS EXECUTIVE OPERATIONS TEAM

Leadership and Committees Leadership and Committees

NEALS PATIENT AND CAREGIVER ADVISORY COMMITTEE (PCAC)

NEALS COMMITTEE LEADERSHIP

2025 Annual Meeting 2025 Annual Meeting

Acknowledgment of Support for the NEALS Annual Meeting (from Calaneet)

2025 Annual Meeting Agenda*

MEETING AND BREAKOUT COLOR LEGEND

NEALS Member only day NEALS Member only day

2025 Annual Meeting Agenda*

NEALS Member only day

NEALS Member only day

2025 Annual Meeting Agenda*

NEALS Member only day

NEALS Member only day

2025 Annual Meeting Agenda*

MEETING AND BREAKOUT COLOR LEGEND

Day One Day One

2025 Annual Meeting Agenda*

Day ONE Day ONE

2025 Annual Meeting Agenda*

Day ONE Day ONE

Audience Q&A

2025 Annual Meeting Agenda*

2025 Annual Meeting Agenda*

Day two Day two

2025 Annual Meeting Agenda*

2025 Annual Meeting Agenda*

2025 Annual Meeting Agenda*

2025 Annual Meeting Agenda*

2025 Annual Meeting Agenda*

2025 awards 2025 awards

THE GUPTA FAMILY ENDOWED PRIZE FOR INNOVATION IN ALS CARE

BASIC SCIENCE

DRS

NEALS Member trainings NEALS Member trainings

RESPIRATORY CARE & VENTILATION COMMITTEE WORKSHOP

CLINICAL RESEARCH STAFF TRAINING & DEVELOPMENT WORKSHOP

OUTCOME MEASURES TRAINING

FEEDING TUBES AND ALS: MULTIDISCIPLINARY PRACTICAL CONSIDERATIONS

COLLABORATE WITH NEALS

AFFILIATED TRIAL SUPPORT

MEET WITH THE NEALS SCIENTIFIC ADVISORY BOARD

PEACe COMMITTEE

2025 NEALS ABSTRACT POSTER SESSIONS

Affiliated Trials Affiliated Trials

Affiliated EAPs Affiliated EAPs

NOTES

NOTES

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