WMS 2022 Industry Partners Information Page 1 WMS 2022 Congress 11-15th October 2022 wms2022.com Meet our Industry Partners Version September 2022 WMS 2022 Congress 11th-15th October 2022 wms2022.com
WMS 2022 Industry Partners Information Page 2 WMS 2022 Congress 11-15th October 2022 wms2022.com CONTENTS Welcome 3 Floor Plan 4 Congress Venue Information 5 Congress Supporters 6 Industry Symposia Schedule 7
Many of us will have missed the lively discussions at the poster boards, informal networking at coffee breaks, the opportunity to make new friendships and refresh old ones, and being immersed for 4 days in the latest scientific and clinical developments in the neuromuscular field. Nevertheless, COVID has changed the world and the WMS had to adapt.
With very best wishes,
WMS 2022 Industry Partners Information Page 3 WMS 2022 Congress 11-15th October 2022 wms2022.com
For our first congress in Canada, we decided to offer a hybrid format that would allow colleagues around the world to either join us in person or online. It is great to see more than 1,000 participants did make use of this offer, with a majority choosing to travel to Halifax.
I’m very much looking forward to either interacting with you virtually or to seeing you in Nova Scotia!
VolkerWELCOMEStraub
President of the WMS
The last two congresses, while successful, felt extremely different from the traditional format of WMS congresses.
This is a very special congress for me, as it is the first in-person congress while in post as President of the Society.
Dear members of the World Muscle Society, friends and colleagues,
Welcome to the 27th annual congress of the World Muscle Society in Halifax.
On behalf of the board, our thanks go to our sponsors and exhibitors for their support in enabling this congress to go ahead, as well as our local organising committee and management team for their hard work behind the scenes.
WMS 2022 Industry Partners Information Page 4 WMS 2022 Congress 11-15th October 2022 wms2022.com VENUE FLOOR PLAN Ballroom Level 2, Halifax Convention Centre PostersPosters Escalators up from main entrance Ballroom SpeakerReadyRoom(508) CommsRoom(505) 2m x 3m 2m x 4.5m 2m x Catering6m Table Digital Poster Stage 11 12 13 14 16 13 17 18 19 22 21 6 7 8 9 10 1 2 3 4 5 MeetingsSponsor(501) MeetingsSponsor(502) ExecutiveMeetingBoard 504503 506507 B1 B2 B3 Argyle Suite on Level 1 for additional industry symposia StandNo. Company Name 1 Sarepta 2 Novartis 3 Roche 4 Santhera 5 Amicus 6 Chillibean 7 Astellas Gene Therapies StandNo. Company Name 8 PTC 9 Biogen 10 Sanofi 11 Ithera Medical 12 Ultragenyx 13 Treat-MND 14 Edgewise StandNo. Company Name 15 Regenxbio 16 Dyne Therapeutics 17 Scholar Rock 18 Pfizer 19 UCB 21 Italfarmaco 22 NS Pharma
CONGRESS VENUE
The Halifax Convention Centre is the main conference centre in Halifax, Nova Scotia, Canada. It opened on December 15, 2017 in Downtown Halifax, replacing the older World Trade and Convention Centre. Convention Centre, 1650 Argyle Street, Halifax, Nova Scotia, B3J 0E6, Canada
Halifax
www.halifaxconventioncentre.com
WMS 2022 Industry Partners Information Page 5 WMS 2022 Congress 11-15th October 2022 wms2022.com
WMS 2022 Industry Partners Information Page 6 WMS 2022 Congress 11-15th October 2022 wms2022.com WMS 2022 HYBRID CONGRESS KINDLY SUPPORTED BY PLATINUM GOLD SILVER SPONSORBRONZE
17:30-19:00
The Era of Gene Therapy: Growing Evidence of The Clinical Reality
INDUSTRY SYMPOSIA SCHEDULE
Ballroom Argyle
07:00-08:30
07:30-09:00
Targeting Fast Muscle Myosin: A Novel Approach to Protecting Muscle in the Dystrophinopathies
WMS Industry Symposium 8
WMS 2022 Industry Partners Information Page 7 WMS 2022 Congress 11-15th October 2022 wms2022.com
WMS Industry Symposium 7
Connecting the Dots: Charting New Frontiers in Duchenne
Navigating the changing disease landscape: continuity of care in Duchenne muscular dystrophy
WMS Industry Symposium 5
Day 2 - Wednesday 12th October
Day 3 - Thursday 13th October
WMS Industry Symposium 3
WMS Industry Symposium 6 Rethinking the science of SMA
WMS Industry Symposium 1
Moving the needle: improving outcomes in paediatric SMA and DMD
Day 4 - Friday 14th October
WMS Industry Symposium 2 Shared decision-making in the management of late-onset Pompe disease
WMS Industry Symposium 4 Measuring Progression in FSHD: Implications for Clinical Trials
07:00-08:30
All times Atlantic Daylight Time (ADT) - Local time in Halifax, Canada
Please see: https://www.wms2022.com/page/industry-symposia for other information.
OVERCOMING HURDLES OF GENE THERAPY
GENE THERAPY: THE CLINICAL REALITY
Prof. Francesco Muntoni, M.D., Director and Co-Director, Dubowitz Neuromuscular Centre and MRC Centre for Neuromuscular Diseases, UCL Great Ormond Street Institute of Child Health and Great Ormond Street Hospital for Children (GOSH) Foundation Trust, London, UK
WMS 2022 Industry Partners Information Page 8
Wednesday October 12, 2022, 7:00 – 8:30 AM ADT
7:00 7:05 AM
7:05 7:20 AM
Dr. Giovanni Baranello, M.D., Clinical Associate Professor, Paediatric Neurology/Neuromuscular Disorders, Department of Developmental Neurosciences, UCL GOS Institute of Child Health, London, UK Nicole LaMarca, D.N.P., M.S.N., C.P.N.P., P.M.H.S., Global Medical Director, Data Exploration and Generation Lead SME, Global Medical A airs, Novartis Gene Therapies, USA
This event is supported, in part, by funding from industry. All support is managed in strict accordance with CME/CPD accreditation criteria and standards for commercial support. Industry Sponsored Symposia are organised by industry and not included in the main event CME/CPD credit o ering.
8:15 – 8:30 AM
AGENDA
Discussion moderated by Nicole LaMarca, D.N.P., M.S.N., C.P.N.P., P.M.H.S.
PANEL DISCUSSION AND CLOSING REMARKS
7:45 – 8:15 AM
BULBAR FUNCTION: GENE THERAPY IN PRACTICE
This meeting is sponsored and organized by Novartis Gene Therapies. © 2022 Novartis Gene Therapies, Inc. MED-CON-ZOL-00001-CA 8/22
7:20 – 7:45 AM
Dr. Hernan Gonorazky, M.D. (Chair), Assistant Professor and Director, Faculty of Medicine, The Hospital for Sick Children, Division of Neurology and Neuromuscular Fellowship Program, University of Toronto, Toronto, Canada
Dr. Hernan Gonorazky, M.D.
We look forward to welcoming you to an informative and engaging event.
THE ERA OF GENE THERAPY: GROWING EVIDENCE AND CLINICAL REALITY OF TREATING SMA
Room: Ballroom Halifax Convention Centre Halifax, Nova Scotia, Canada
WELCOME & INTRODUCTION
©2022 SAREPTA THERAPEUTICS. CONFIDENTIAL AND NOT FOR DISTRIBUTION. COPYING OF THIS MATERIAL BY ANY MEANS WITHOUT SAREPTA’S PRIOR WRITTEN CONSENT IS PROHIBITED. C-NP-CAN-0019 07/22 Join an expert-guided exploration of DMD from early diagnosis through disease heterogeneity followed by the utilization of natural history cohorts and disease modeling in clinical trials. Sarepta IndustryTherapeutics’Symposium: Wednesday, 12 October 2022 17:30 - 19:00 ADT Halifax Convention Centre - Ballroom Halifax, Canada Charting New Frontiers in Duchenne Muscular Dystrophy (DMD) Connecting the Dots LouiseModeratorRodino-Klapac, PhD Executive Vice President Head of R&D, Chief Scienti c O cer Sarepta Therapeutics, Inc. Ohio, USA Francesco Muntoni, FRCPCH, FMedSci Director, Dubowitz Neuromuscular Centre Co-Director, MRC Centre for Neuromuscular Diseases UCL Great Ormond Street Institute of Child Health & Great Ormond Street Hospital for Children NHS Foundation Trust London, UK SAREPTA, SAREPTA THERAPEUTICS, and the SAREPTA Helix Logo are trademarks of Sarepta Therapeutics, Inc., registered in the U.S. Patent and Trademark O ce and may be registered in various other jurisdictions. Speakers Craig M. McDonald, MD Department of Physical Medicine & Rehabilitation, Department of Paediatrics Director, MDA Neuromuscular Disease Clinics University of California Davis Health California, USA Emma Ciafaloni, MD, FAAN Co-Director, MDA Neuromuscular Clinic Professor of Neurology and Paediatrics University of Rochester New York, USA
Leo Wang, M.D., Ph.D. Assistant Professor of Neurology University of Washington www.fulcrumtx.com
Wednesday, October 12, 2022
Speakers:
WMS2022 Industry Symposium sponsored by Fulcrum Therapeutics
Sabrina Sacconi, M.D., Ph.D.
Head of the Peripheral Neurology and Muscle Unit Centre Hospitalier Universitaire de Nice
Halifax Convention Centre: Argyle
17:30 - 18:50 ADT / 16:30 - 17:50 EDT
Measuring Progression in FSHD: Implications for Clinical Trials
Judith Dunn, Ph.D. President, Research and Development Fulcrum Therapeutics
WMS 2022 Industry Partners Information Page 10 WMS 2022 Congress 11-15th October 2022 wms2022.com
M-XX-00010007 (Date of preparation: July 2022) *This symposium is a learning activity organised and funded by F. Hoffmann-La Roche Limited, with the DMD section being developed in collaboration with Sarepta Therapeutics, Inc. The views and opinions discussed in this session are those of the presenters, and not of Roche or Sarepta. This symposium is intended for healthcare professionals outside of the US. © 2022 F. Hoffmann-La Roche Ltd. All trademarks mentioned herein are protected by law. www.roche.com
Join us in person or online to discuss the latest evidence and clinical perspectives on the management of neuromuscular diseases, including spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD). We welcome you to our data presentations where we will be sharing insights and updates from our research programs. We also invite you to our satellite symposium*, where presenters will explore recent advances in the treatment of paediatric SMA and DMD. We look forward to seeing you at WMS!
WMS 2022 Industry Partners Information Page 11
Roche at the 27th International Hybrid Annual Congress of the World Muscle Society (WMS), 11–15 October 2022, Halifax, Canada
Roche in outcomes,scienceconditionsneuromuscular–translatingintomeaningfultogether
WMS 2022 Industry Partners Information WMS 2022 Congress 11-15th October 2022 wms2022.com This symposium has been organized and funded by Biogen Global Medical. This session is not included in main conference CME/CPD credit. Biogen products will be discussed at this event, please consult your locally approved information before prescribing nusinersen. Canadian Product Monograph for nusinersen is available from: https://www.biogen.ca/products/SPINRAZA_PM_EN “I became an artist because the most important form of freedom is to realize yourself and to be who you are. I love creating magic, putting something together that is completely unusual and so unexpected that it takes people’s breath away. Something that is ahead of its time. Five steps ahead of what people think.” WMS 2022 Industry Symposium Disease Evolution – Therapeutic Windows – Capacity for Improvement Thursday 13 October, 2022 07:30–09:00 | Argyle oftheRethinkingSuitescienceSMA Dr Didu (Sandi) Kariyawasam Sydney Children’s Hospital, Randwick, NSW, Australia
SMN-restoring therapies are transforming patient outcomes in SMA. However, variability in treatment response at the individual level remains striking. We explore what is known about the genesis, degeneration, and potential resurrection of the motor neuron in SMA, the host and intervention factors that may modify treatment response at the individual level, and the rationale for investigating strategies to optimize these responses.
A light breakfast will be provided from 07:00
Targeting Fast Muscle Myosin Approach to the
Dystrophinopathies YOU ARE INVITED
Friday 14th October 2022 • 7:00-8:30am EST
: A Novel
Protecting Muscle in
to join the WMS2022 Industry Symposium sponsored by Edgewise Therapeutics at the 27th World Muscle Society (WMS) Congress
The symposium will explore how targeting fast muscle myosin could potentially protect muscle in Duchenne and Becker muscular dystrophy.
Dr. Craig McDonald will discuss the clinical course of Becker muscular dystrophy and potential strategies for clinical Dr.trials.Alan Russell will discuss identi cation of a fast myosin modulator to disconnect muscle injury from muscle contraction in dystrophinopathies to circumvent the struc tural stress caused by loss of dystrophin on DMD/BMD. Lastly, Dr. Joanne Donovan will discuss most recent ndings clinical studies with EDG-5506 is a novel, rst-in-class, small molecule for the treatment of dystrophinopathies via this approach, which has potential either as a stand-alone therapy or in combination with dystrophin-targeted thera pies across the spectrum of dystrophinopathies as well as other muscular dystrophies.
After hearing from our speakers, we will open it up for Q&A from the audience.
The damaging consequences of muscle contraction without functional dystrophin will be reviewed by Dr. Lee Sweeney. When dystrophin is absent, muscle contraction generates sarcomeric stress, opening of membrane stress channels, and calcium in ux into the myo ber leading to myo ber degeneration with increases in circulating biomarkers of muscle damage.
"Preferential Fast Fiber Injury in Dystrophinopathies" LEE SWEENEY, PH.D. UF Department of Pharmacology & Therapeutics Gainsville, FL "Mechanical Stress Induced Injury: Changes in Biomarkers of Dystrophic Muscle during Exercise in LGMD and BMD" JOHN VISSING, M.D., PH.D. University of Copenhagen Denmark "Clinical Course Dystrophinopathies"of CRAIG MCDONALD, M.D. UC Davis Sacramento, CA "Targeting Fast Myosin to Decouple Injury from Muscle Contraction in DMD and BMD" ALAN RUSSELL, PH.D. Chief Scientific Officer Edgewise Therapeutics "Clinical Development of EDG-5506 in BMD and DMD" JOANNE DONOVAN, M.D., PH.D. Chief Medical Officer Edgewise Therapeutics ORDER OF SPEAKERS
Dr. John Vissing will discuss the impact of muscle contrac tion and exercise on biomarkers of fast muscle bre damage in dystrophinopathies and other muscular dystrophies.
About the Symposium
WMS 2022 Industry Partners Information Page 14 7:00–7:05 am Welcome Leanne Ward 7:05–7:10 am An introduction to DMD and best practice management of patients Leanne Ward 7:10–7:30 am The patient journey: continuity of care for patients with DMD Craig McDonald 7:30–8:05 am Latest clinical data for atalurena use in patients with nmDMD Eugenio Mercuri 8:05–8:25 am Panel discussion All speakers 8:25–8:30 am Summary and conclusions Leanne Ward aAtaluren is not licensed in Canada. Ataluren is indicated for the treatment of Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 2 years and older in the European Member States and Iceland, Liechtenstein, Norway, Great Britain, Northern Ireland, Kazakhstan, Israel, Republic of Korea, Belarus, Russia and Brazil, and aged 5 years and older in Chile, the Kingdom of Saudi Arabia and Ukraine (under special state registration). In Brazil, the indication is specific to male paediatric patients. The presence of a nonsense mutation in the dystrophin gene should be determined by genetic testing (Translarna Summary of Product Characteristics [SmPC] for the respective countries). DMD, Duchenne muscular dystrophy; nmDMD, nonsense mutation Duchenne muscular dystrophy. This WMS industry symposium is organized and funded by PTC Therapeutics International and is intended for healthcare professionals only. MED-ALL-ATLN-2200011 | Date of preparation: July 2022 diseasetheNavigatingchanginglandscape: continuity of care in Duchennedystrophymuscular Friday 14 October 2022; 7:00–8:30 am ADT Argyle Room, Halifax Convention Centre, Halifax, NS, Canada WMS 2022 industry symposium sponsored by PTC Therapeutics Breakfastwillbeprovided
WMS 2022 Industry Partners Information Page 15 WMS 2022 Congress 11-15th October 2022 wms2022.com
Genetic testing is a fast and direct route to diagnosis Early genetic testing can: Confirm diagnoses Help patients seek the care they need Allow patients to enroll in available clinical trials
Did You
Mitochondrial disorders are among the prevalent group of inherited neurological diseases1
most
References: 1. Gorman SG, Schaefer AM, Ng Y, et al. Prevalence of nuclear and mitochondrial DNA mutations related to adult mitochondrial disease. Ann Neurol. 2015;77(5):753-759. 2. Phillips LH. The epidemiology of myasthenia gravis. Ann NY Acad Sci. Inc. to UCB. ©2022 UCB Inc., Smyrna, GA 30080. All rights reserved. US-TK2D-2200015 Intended for residents of the United States only.
2003;998:407-412.UCBwelcomesZogenix,
An estimated 1 in 5,000 adults has a mitochondrial disorder, the same prevalence rate as myasthenia gravis.1,2
• Physician- or patient-initiated program
Detect Muscular Dystrophy: Comprehensive Neuromuscular Disorders Panel
• 55-gene panel with genes associated with limb-girdle muscular dystrophy and mitochondrial depletion syndromes, including thymidine kinase 2 deficiency (TK2d)
• ~210 genes associated with hereditary neuromuscular conditions (includes select mitochondrial nuclear genes)
If multiple organ systems are involved, consider testing for a mitochondrial disorder. Learn more at MayBeMito.com/#testing
Additional programs are being evaluated. Please check MayBeMito.com/#testing for the latest information.
WMS 2022 Industry Partners Information Page 19 WMS 2022 Congress 11-15th October 2022 wms2022.com
Zogenix sponsors multiple no-cost genetic testing programs to help diagnose mitochondrial disorders
• Physician-initiated program
No-Cost Genetic Testing Program for TK2d
WMS 2022 Industry Partners Information Page 20 AT AMICUS THERAPEUTICS, WE ENCOURAGE AND EMBRACE CONSTANT INNOVATION Amicus Therapeutics, Inc. © 2022 Amicus Therapeutics. All rights reserved. NP-NN-ALL-00050622 A R COMPANYRE At Amicus Therapeutics, we strive to fulfill the bold commitment we’ve made to the rare disease community. This commitment keeps us at the forefront of developing therapies for those who need them most. OUR COMMITMENT As we pursue treatments for devastating rare diseases, we maintain a personal and compassionate focus on patients, their caregivers, and families. OUR TECHNOLOGIES We are leveraging our innovative technology platforms in protein stabilization and targeting to help advance treatments for human genetic diseases.
WMS 2022 Industry Partners Information Page 21 WMS 2022 Congress 11-15th October 2022 wms2022.com www.ryr1.org
WMS 2022 Industry Partners Information Page 22 WMS 2022 Congress SEEKING TO IMPROVE GENETHROUGHLIVESTHECURATIVEPOTENTIALOFTHERAPY OUR COMMITMENT REGENXBIO Inc. is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy using our NAV® Technology Platform. Our gene therapy product candidates are designed to deliver functional genes, enabling the production of therapeutic proteins or antibodies that are intended to impact disease. › For the latest updates on our programs, visit REGENXBIO.COM › Please visit us at our booth #15 ©2022 REGENXBIO Inc. All rights reserved. REGENXBIO.com
WMS 2022 Industry Partners Information Page 23 WMS 2022 Congress 11-15th October 2022 wms2022.com Astellas Gene Therapies is developing potential gene therapies for rare neuromuscular diseases Perry Shieh, MD, PhD University of California, Los Angeles Los Angeles, CA
ASTELLAS and the flying star device are registered trademarks of Astellas Pharma Inc. ©2022 Audentes Therapeutics, Inc. All rights reserved. CA-Corporate-082022-00001. August 2022. Wednesday, October 12, 2022 11:00-13:00, Ballroom Session: New Developments in Congenital Myopathies 2 Please Join our Platform Presentation O.06. Long term outcomes for X-Linked myotubular Myopathy (XLMTM) with gene replacement therapy, resamirigene bilparvovec: preliminary results from ASPIRO THE SAFETY AND EFFICACY OF THE AGENTS UNDER INVESTIGATION HAVE NOT BEEN ESTABLISHED. THERE IS NO GUARANTEE THAT THE AGENTS WILL RECEIVE REGULATORY APPROVAL OR BECOME COMMERCIALLY AVAILABLE FOR USES BEING INVESTIGATED. THESE AGENTS ARE NOT AUTHORIZED FOR SALE IN ANY JURISDICTION. X-linked Myotubular Myopathy (XLMTM) is a
1. Biancalana V, et al. Acta Neuropathol. 2017;134(6):889-904.
2. Graham RJ, et al. Arch Dis Child. 2020;105(4):332-8. 40,000-50,000estimatedcongenitallife-threateningrare,monogenicmyopathywithanincidenceof1innewbornmales.1,2
Did youToknow?learn more: Visit our booth Booth #7 Visit our website AstellasGeneTherapies.com
WMS 2022 Industry Partners Information Page 24
WMS 2022 Industry Partners Information Page 25 2022 Congress October 2022 wms2022.com
WMS 2022 Industry Partners Information Page 27 Navigating the changing disease landscape: continuity of care in Duchenne muscular dystrophy aAtaluren is not licensed in Canada. Ataluren is indicated for the treatment of Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 2 years and older in the European Member States and Iceland, Liechtenstein, Norway, Great Britain, Northern Ireland, Kazakhstan, Israel, Republic of Korea, Belarus, Russia and Brazil, and aged 5 years and older in Chile, the Kingdom of Saudi Arabia and Ukraine (under special state registration). In Brazil, the indication is specific to male paediatric patients. The presence of a nonsense mutation in the dystrophin gene should be determined by genetic testing (Translarna Summary of Product Characteristics [SmPC] for the respective countries). This session is not included in the main event CME/CPD credit offering. This WMS Industry Symposium is organized and funded by PTC Therapeutics International Ltd and is intended for healthcare professionals only. MED-ALL-ATLN-2200061 | Date of preparation: July 2022 Join us to see the latest clinical data on atalurena You are invited to join us on Friday 14 October at 7:00–8:30 am ADT Friday 14 7:00–8:30OctoberamArgyleRoom Prof. Leanne Ward Prof. Craig McDonald Prof. Eugenio Mercuri
WMS 2022 Industry Partners Information Page 28 WMS 11-15 wms2022.com LinkerCOMPONENTS Clinically validated and non cleavable; attached at single conjugate site Centyrin Receptor binding and internalization enables targeting to specific cells siRNA Inhibits production of specific protein involved in disease KEY FEATURES Novel antigen binding protein Highly specific and high affinity Vast library diversity Small size (10 kd), monovalent, stable Low immunogenicity risk Proven drug class Novel Aro Demonstratedchemistryspecificity and potency Long PD effect CENTYRIN siRNA CONJUGATE Aro is developing a new class of highly effective and safe muscle targeted RNA therapeutics Contact us to partner on developing muscle targeted medicines Alex Meltzer (Associate Director, ameltzer@arobiotx.comBD/Strategy)
WMS 2022 Industry Partners Information Page 29 WMS 2022 Congress 11-15th October 2022 wms2022.com Discover how we can meet the needs of patients living with rare FURTHERLET’SdiseasesGO Santhera Pharmaceuticals is pass ionate about develo ping new t r eatments for p atien ts with Duchenne muscular d ystroph y and other r are disorders. Meet us at Booth 4 to learn more about our research, pipeline, and purpose. JOIN US ON SOCIAL MEDIACONTACT US © Santhera Pharmaceuticals. All rights reserved. August 2022. NP-US-NTX-NXP-0006. Santhera Pharmaceuticals (USA), Inc. 25 Corporate Drive, Suite 250 Burlington, MA 01803 USA PHONE: +1 781 365 0301 FAX: +1 781 365 0230 www.santhera.com
WWW.WMS2023.COM #WMS2023 Annual Congress of The World Muscle Society 3-7 October WMS 28 Charleston, USA 3-7 October 2023 Get the date in your diary for the 28th World Muscle Society Congress in Charleston, South Carolina, USA.
