CORD Rare Drug Conference: June 8-9, 2022 Network of Centres of Excellence Canadian Neuromuscular Network, Western University - Craig Campbell, LHSC

1. -Canadian Neuromuscular Centres-
Towards a centres of excellence network
Craig Campbell MD
Western University and Children’s Hospital LHSC
JUNE 2022 CORD CONFERENCE

2. Key messages….
The Canadian Neuromuscular community is aspiring to be a model rare
disease centres of excellence network
Cross cutting structures and activities support success
Clinical trial facilitation is providing opportunity and removing obstacles

3. Key messages…
The Canadian Neuromuscular community is aspiring to be a model rare
disease centres of excellence network
NMD4C
Cross cutting structures and activities support success
Care and Trial Site Registry
Canadian Neuromuscular Disease Registry
Clinical trial facilitation activities are removing obstacles
Clinical trial concierge

4. Pan-Canadian network that brings
together the leading clinical, scientific,
technical, and patient expertise to
improve care, research, and
collaboration in neuromuscular
disease.
We aim to:
ü Train and educate the next
generation of NMD stakeholders
ü Raise the standard of care for
NMD and access to therapies
across Canada
ü Strengthen biomedical and
clinical infrastructure to build
research capacity

5. Steering Committee:
Hanns Lochmuller
James Dowling
Jody Warman Chardon
Rashmi Kothary
Stacey Lintern
Project Management
Erin Beattie
James Davis
Clinical Trials Working
Group Coordinator:
Boonie Wooten

7. More Clinical Trials

8. Network Partnerships
Patient Organizations
• Meaningful patient partnerships are key:
ü Muscular Dystrophy Canada (MDC) is a co-funder and partner of NMD4C
ü Defeat Duchenne Canada (formerly Jesse’s Journey)
ü Myotonic Dystrophy Foundation
ü Cure SMA Canada
• Strong working relationship: Organizations like MDC and Defeat Duchenne Canada (formerly Jesse's
Journey) execute different activities to best understand patient needs and perspectives
ü MDC has a Data Hub whereby they can share information on clinical trials with Canadians affected by
a broad set of neuromuscular disorders including Duchenne Muscular Dystrophy (DMD), invest in
research that will support clinical trial readiness and provide plain language evidence-based
information
ü Defeat Duchenne Canada (formerly Jesse's Journey) supports families and individuals living with DMD
through education, research, and advocacy.

9. Network Tools
Care and Trial Site Registry
• Established 2007 as part of TREAT-NMD Neuromuscular Network
• Is a searchable repository of neuromuscular centres of excellence
• Since 2008 – CTSR now has 383 registered sites worldwide
• Currently there are 15 Canadian Neuromuscular sites
Benefits of the CTSR
• Helps to promote Canada as viable location for clinical trials, including sites that
might previously have not been considered by industry
• Can match trial site to CNDR registrants
• NMD4C has admin function, and coordinates information exchange between trial
sites across Canada

10. Network Tools
Canadian Neuromuscular Disease Registry
• 36 sites, 62
investigators
• 4600 patients
o 605 DMD (585 active)
o 478 DM (453 active)
o 92 LGMD (92 active)
o 271 SMA (257active)
o Approx. 1600 non-index

11. Network Tools
Canadian Neuromuscular Disease Registry
• Clinical Trial Planning and
Recruitment
• Others: surveys,
advocacy, education
• Collaboration with TREAT-
NMD Global Datasystems
Oversight Committee

12. Network Supports
Clinical Trial Concierge
•The NMD4C Clinical Trial Concierge (CTC) acts as a broker for
all Canadian clinical trial sites supporting the coordination and
exchange of information between sites
•Serves as an impartial resource, not funded by industry or
advocacy groups - for all neuromuscular stakeholders
•Works with investigators to develop solutions to barriers
associated with realizing neuromuscular clinical trials in
Canada

13. Network Supports
Clinical Trial Concierge
Responsive to patients,
caregivers, clinicians and
patient advocacy groups
requests for information
about all aspects of clinical
trial opportunities
Navigates clinical trial
space for patients

14. Network Supports
Clinical Trial Concierge
Responsive to patients,
caregivers, clinicians and
patient advocacy groups
requests for information
about all aspects of clinical
trial opportunities
Navigates clinical trial
space for patients
Administers the CTSR
Liaises with the CNDR
Tracks clinical trial metrics

15. Network Supports
Clinical Trial Concierge
Responsive to patients,
caregivers, clinicians and
patient advocacy groups
requests for information
about all aspects of clinical
trial opportunities
Navigates clinical trial
space for patients
Administers the CTSR
Liaises with the CNDR
Tracks clinical trial metrics
Road map to optimize
individual clinical trial
engagement and
recruitment

16. Network Supports
Clinical Trial Concierge
Responsive to patients,
caregivers, clinicians and
patient advocacy groups
requests for information
about all aspects of clinical
trial opportunities
Navigates clinical trial
space for patients
Administers the CTSR
Liaises with the CNDR
Tracks clinical trial metrics
Identifies clinical trial
possibilities
Facilitates meeting
with industry partners
Tracks clinical trial
assets
Road map to optimize
individual clinical trial
engagement and
recruitment

17. -Canadian Neuromuscular Centres-
Towards a centres of excellence model
THANK YOU
Craig Campbell MD
JUNE 2022

19. How the Canadian Clinical Trial Landscape
Benefits Sponsors
Pediatric and adult clinical trial expertise
• DMD: Acceleron, Catabasis, Fibrogen, FORDMD,
Italfarmaco, GSK, Lily, NS Pharma, Pfizer, Prosensa, PTC,
ReveraGen, Sarepta, Wave
• SMA: Avexis, Biogen, Roche
• DM1: AMO, Harmony
• FSHD: Acceleron, Fulcrum, Roche
Experience and expertise across Canada
• Many pediatric and adult sites, geographic location to
serve all areas
• Experience/trained PTs on disease specific outcome
measures
• Specialized expertise: bone health, genetics and
epigenetics, clinical pharmacology, translational research
Success Stories:
• Phase 1 (Wave, Sarepta) to phase
4 (PTC)
• Meaningful engagement on
planning, analysis, safety
• Partnership on regulatory
pathway
• DSMB: Catabasis, Edgewise, Solid
• CNDR Post marketing surveillance
projects