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 Symposia Schedule Programme 7
On behalf of the board, our thanks also 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.
VolkerWELCOMEStraub
Dear members of the World Muscle Society, friends and colleagues, Welcome to the 27th annual congress of the World Muscle Society in Halifax.
Nevertheless, COVID has changed the world and the WMS had to adapt.
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! With very best wishes,
WMS 2022 Industry Partners Information Page 3 WMS 2022 Congress 11-15th October 2022 wms2022.com
President of the WMS
This is a very special congress for me, as it is the first in-person congress while in post as president of the Society.
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.
The last two congresses, while successful, felt extremely different from the traditional format of WMS congresses.
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 Registration PostersPosters Escalators up from main entrance MainTheRoomCongressBallroom Speaker Preparation Room (508) Wellbeing Room (505) 2m x 3m 2m x 4.5m 2m x Catering6m Table Digital Poster Stage12111314161317 21201918768910 1234 5 22 Argyle Suite on Level 1 for furtherCongresssymposium,industryparallelsessionsandpostersessions. StandNo. Company Name 1 Sarepta 2 Novartis 3 Roche 4 Santhera 5 Amicus 6 Chillibean 7 Astellas Gene Therapies 8 PTC StandNo. Company Name 9 Biogen 10 Sanofi 11 Ithera Medical 12 Ultragenyx 13 Treat-MND 14 Edgewise 15 Regenxbio 16 Dyne Therapeutics StandNo. Company Name 17 Scholar Rock 18 Pfizer 19 UCB 2021 Italfarmaco 22 NS Pharma
WMS 2022 Industry Partners Information Page 5 WMS 2022 Congress 11-15th October 2022 wms2022.com 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. Halifax Convention Centre, 1650 Argyle Street, Halifax, Nova Scotia, B3J 0E6, Canada
www.halifaxconventioncentre.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
WMS 2022 Industry Partners Information Page 7 WMS 2022 Congress 11-15th October 2022 wms2022.com Ballroom Argyle Day 2 - Wednesday 12th October 07:00-08:30 WMS Industry Symposium 1 The Era of Gene Therapy: Growing Evidence of The Clinical Reality WMS Industry Symposium 2 Shared decision-making in the management of late-onset Pompe disease 17:30-19:00 WMS Industry Symposium 3 Connecting the Dots: Charting New Frontiers in Duchenne WMS Industry Symposium 4 Measuring Progression in FSHD: Implications for Clinical Trials Day 3 - Thursday 13th October 07:30-09:00 WMS Industry Symposium 5 WMS Industry Symposium 6 Rethinking the science of SMA Day 4 - Friday 14th October 07:00-08:30 WMS Industry Symposium 7 Targeting Fast Muscle Myosin: A Novel Approach to Protecting Muscle in the Dystrophinopathies WMS Industry Symposium 8 Navigating the changing disease landscape: continuity of care in Duchenne muscular dystrophy SYMPOSIUM SCHEDULE PROGRAMME All times Atlantic Standard Time (AST) - Local time in Halifax, Canada
Wednesday October 12, 2022, 7:00 – 8:30 AM ADT Room: Ballroom Halifax Convention Centre Halifax, Nova Scotia, Canada
MED-CON-ZOL-00001-CA 8/22
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. This meeting is sponsored and organized by Novartis Gene Therapies. © 2022 Novartis Gene Therapies, Inc.
WELCOME & INTRODUCTION
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 7:05 7:20 AM
GENE
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 7:45 – 8:15 AM BULBAR FUNCTION: GENE THERAPY IN PRACTICE
Dr. Hernan Gonorazky, M.D. 7:20 – 7:45 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 8:15 – 8:30 AM PANEL DISCUSSION AND CLOSING REMARKS Discussion moderated by Nicole LaMarca, D.N.P., M.S.N., C.P.N.P., P.M.H.S.
7:00 7:05 AM
WMS 2022 Industry Partners Information
OVERCOMING HURDLES OF GENE THERAPY
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THERAPY: THE CLINICAL REALITY
AGENDA
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
©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
WMS 2022 Industry Partners Information Page 10 WMS 2022 Congress 11-15th October 2022 wms2022.com WMS2022 Industry Symposium sponsored by Fulcrum Therapeutics Measuring Progression in FSHD: Implications for Clinical Trials Wednesday, October 12, 2022 17:30 - 18:50 ADT / 16:30 - 17:50 EDT Halifax Convention Centre: Argyle Speakers: Judith Dunn, Ph.D. President, Research and Development Fulcrum Therapeutics Sabrina Sacconi, M.D., Ph.D. Head of the Peripheral Neurology and Muscle Unit Centre Hospitalier Universitaire de Nice Leo Wang, M.D., Ph.D. Assistant Professor of Neurology University of Washington www.fulcrumtx.com
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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
Roche in outcomes,scienceconditionsneuromuscular–translatingintomeaningfultogether
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! Roche at the 27th International Hybrid Annual Congress of the World Muscle Society (WMS), 11–15 October 2022, Halifax, Canada
WMS 2022 Industry Partners Information Page 12 WMS 2022 Congress 11-15th October 2022 wms2022.com Biogen-175750. July 2022
“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
About the Artist
07:30–09:00 | Argyle oftheRethinkingSuitescienceSMA
Phil L. Herold is an internationally recognized artist who describes himself as a “cyberspace expressionist of the 21st century”. Born in Munich in April 1980, and diagnosed with SMA type 2 in early childhood, today Phil designs his large-format works of art on a PC, using a small joystick on his wheelchair. Thus, through only the minimal movement that he is left with, he continues to create, inspire, delight and challenge us. October, 2022
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
Dr Didu (Sandi) Kariyawasam Sydney Children’s Hospital, Randwick, NSW, Australia
Prof. Francesco Muntoni University College London Great Ormond Street Institute of Child Health, London, UK
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
Prof. Charlotte Sumner Johns Hopkins University School of Medicine, Baltimore, MD, USA
WMS 2022 Industry Partners Information Page 13 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 16 WMS 2022 Congress 11-15th October 2022 wms2022.com
WMS 2022 Congress 11-15th October 2022 wms2022.com www.ryr1.org
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. 2003;998:407-412.UCBwelcomesZogenix, Inc. to UCB. ©2022 UCB Inc., Smyrna, GA 30080. All rights reserved. US-TK2D-2200015 Intended for residents of the United States only.
WMS 2022 Industry Partners Information Page 18 WMS 2022 Congress 11-15th October 2022 wms2022.com Did You An estimated 1 in 5,000 adults has a mitochondrial disorder, the same prevalence rate as myasthenia gravis.1,2 Mitochondrial disorders are among the most prevalent group of inherited neurological diseases1 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
WMS 2022 Industry Partners Information Page 19 WMS 2022 Congress 11-15th October 2022 wms2022.com If multiple organ systems are involved, consider testing for a mitochondrial disorder. Learn more at MayBeMito.com/#testing Detect Muscular Dystrophy: Comprehensive Neuromuscular Disorders Panel • Physician- or patient-initiated program • ~210 genes associated with hereditary neuromuscular conditions (includes select mitochondrial nuclear genes) No-Cost Genetic Testing Program for TK2d • Physician-initiated program • 55-gene panel with genes associated with limb-girdle muscular dystrophy and mitochondrial depletion syndromes, including thymidine kinase 2 deficiency (TK2d) Additional programs are being evaluated. Please check MayBeMito.com/#testing for the latest information. Zogenix sponsors multiple no-cost genetic testing programs to help diagnose mitochondrial disorders
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.
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.
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
OUR COMMITMENT
GENETHROUGHLIVESTHECURATIVEPOTENTIALOFTHERAPY clinical-stage
WMS 2022 Industry Partners Information Page 21 WMS 2022 Congress SEEKING TO IMPROVE
OUR COMMITMENT REGENXBIO Inc. is a leading
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 22 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 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. 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 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 23 WMS2023.COM #WMS2023 Annual Congress of The World Muscle Society 3-7 October WMS Charleston,28USA 3-7 October 2023 Get the date in your diary for the 28th World Muscle Society Congress in Charleston, South Carolina, USA.
WMS 2022 Industry Partners Information Page 24 2022 Congress October 2022 wms2022.com
the
in
aAtaluren
Navigating changing disease landscape: continuity of care Duchenne muscular dystrophy 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. Date of preparation: July 2022
MED-ALL-ATLN-2200061 |
WMS 2022 Industry Partners Information Page 26
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
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)
LinkerCOMPONENTS
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WMS 2022 Industry Partners Information Page 28 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
