SMA Clinical Care Center Network / Clinical Data Registry & Clinical Trials Site Readiness for SMA. March 21, 2018

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1 SMA Clinical Care Center Network / Clinical Data Registry & Clinical Trials Site Readiness for SMA March 21, 2018

2 SMA Clinical Care Center Network & Clinical Data Registry Mary Schroth, MD Chief Medical Officer

3 Cure SMA We fund and direct comprehensive research that drives breakthroughs in treatment and care, and we provide families the support they need for today. Impact 115,000 supporters 12,000 members affected by SMA 350 newly diagnosed contacts annually 28 th Annual conference, 1500 attendees Spinraza, first ever FDAapproved therapy for SMA 3

4 Cure SMA Investments $70 Million in research funding More than 28 current research programs diverse therapeutic approaches more than 100 institution partners around the world. Half of all ongoing new drug programs for SMA including Spinraza were funded by Cure SMA. Spinraza is the first ever FDA-approved therapy specifically for SMA, December $9.1 Million in clinical care funding to establish the Cure SMA Care Center Network and Clinical Data Registry (CDR)

5 SMA Care Center Network - Vision 1. Quality improvement of SMA clinical care and disease management leading to creation of evidence to support a robust standard of care for SMA 2. Standardize care across the US to facilitate more rapid therapeutic development. 3. Develop clinical care center capacity to deliver new therapies to individuals with SMA and increase patient access to new treatments.

6 SMA Care Center Network - Goals - Enroll 8 initial SMA Care Centers with CDR capacity - Establish: Center Committee Data Committee - Enroll 10 more Care Centers by year end Enroll 10 more Care Centers - Care Center Network Accreditation process defined Enroll 20 more Care Centers - Care Center Network Accreditation process implemented

7 SMA Care Center Characteristics Established Clinics with desire to partner with Cure SMA to improve SMA care locally and nationally Geographically diverse Interdisciplinary care model across medical specialties and disciplines Pediatric and adult care team partners or potential to achieve partnership Capacity to provide FDA approved therapies for SMA Information Technology team capacity to support the EHR and Cure SMA registry integration

8 How It Works Current SMA Care Centers will be invited to complete a Letter of Interest Centers complete the Letter of Interest, and submit to patientcare@curesma.org Cure SMA will schedule a conference call with the Care Center Champion, Executive and EHR team to determine needed resources to integrate the Care Center EHR with the Cure SMA CDR. Contracts will be completed. Cure SMA will provide funding to the Care Center. Cure SMA CDR partner will work with the care center EHR team to build the integration pathway. Consent processes will be completed. During outpatient visits or hospitalizations, clinicians document in the EHR per institution guidelines. On a predetermined schedule, clinical data is pushed or pulled through secure electronic pathways from the EHR documentation to the Cure SMA CDR. Each center will have access via a web portal, to their submitted clinical data Cure SMA will provide annual reports to centers for benchmarking The CDR will be available for investigator query

9 Clinical Data Registry (CDR) Integrated with the EHRs at Care Centers Uses the Health Level Seven (HL7) Fast Healthcare Interoperability Resources (FHIR) standard in a unique application programming interface (API) developed by Prometheus Research Allows a variety of EHR systems including Epic and Cerner and other cloud based EHRs to submit data directly to registry using standard web technology Alternative EHR data submission formats are available. Data entered into the EHR is pushed or pulled into the CDR Consent process through a central independent IRB Compensation to centers for initial integration process and annually for complete patient data sets Dashboard available to each site for their patient data 9

10 Clinical Data Registry Capacity Benchmarking data comparing each Center to other deidentified Care Centers Data analysis for care outcomes, areas of care variance across centers and opportunities for improvement Linkage with: Other databases Patient reported outcomes data Innovative data collection tools, e.g., wearables Portal for manual data entry to capture newborn screen identified/ newly diagnosed patients from all Care Centers

11 SMA Care Center Network Impact Create evidence for SMA standard of care Resource for: Local patient care services and family support Regional care providers Broader Cure SMA community Industry collaboration Preparation for Clinical Trials

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13 Clinical Care Meeting 2018 SMA Annual Conference June 16, 2018, 1-6 pm following the Researcher Meeting in Dallas at the Hilton Anatole 2 Topic sessions followed by wine reception Topics: Innovations in Clinical Care Impact of New Therapies on Clinical Care Presentation abstracts deadline March 23! Scholarships available by application

14 New Therapies Support Website Directory of New Treatment Network: Spinraza 124 sites in 36 states and DC Contact:

15 Cure SMA PT/OT Mentoring Program Purpose: Opportunity for experienced therapists to teach and train therapists new to SMA Increase patient access to therapists who can perform motor function evaluations Funding: Travel for Mentee or Mentor travel Mentor stipend

16 Clinical Trial Site Readiness for SMA: Objectives, Needs, and Opportunities

17 Goals and Motivation Objectives Identify and pursue opportunities to increase trial site capacity and optimize readiness in the United States Motivation To alleviate challenges related to site capacity and patient access to clinical trial sites, and Better meet the needs of trial sponsors and the SMA patient community as the number of SMA clinical trials in the US increases

18 Current Landscape in SMA Clinical Trials Rosángel Cruz, Associate Research Director

19 Cure SMA Pipeline

20 Seven Investigational Drugs Tested One Approved! For these seven compounds, 20 clinical trials in SMA (Phases I-III) have been conducted, enrolling a total 1,179* participants since Of these, 9 trials have been completed with A combined enrollment of 569 participants An average of 8 patients per each site (range 4-9) 11 trials are ONGOING / currently recruiting (OLEs) through These have enrolled 610 participants to date* Average of 8 patients per site (range 2-15) *As of the end of 2017

21 Needs and Opportunities: Current vs. Prospective Trial Site Needs Projected recruitment need between : 1,084 SMA patients Participating Sites All SMA sites participating in Phase I-III All SMA Sites Available (2018) All SMA Sites Available (2020) CT Sites in Europe & Asia CT Sites in Canada Sites in US 21 35* 55** Total SMA Trial Sites Worldwide * US site capacity would need to more than DOUBLE Projections prepared based on evaluation of pipeline needs: *Assumes readying of 14 existing SMA sites **Assumes addition of 20 new sites starting 3Q2018

22 Perspectives on Conducting SMA Clinical Trials Stephen J. Kolb, MD, PhD Associate Professor, Department of Neurology and Biological Chemistry & Pharmacology Director, Ohio State ALS/MND Multidisciplinary Clinic and Research Program The Ohio State University Wexner Medical Center

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24 What is Unique about Conducting Trials in SMA? The disease itself SMA is one underlying disease with multiple phenotypes (and varied clinical presentation, with mild to severe/life-threatening severity) Multiple outcome measures Multiple outcome measures used to evaluate clinically meaningful changes, depending on SMA type Complex care and follow-up Understanding the natural history of SMA is essential for understanding meaningful change/assessing efficacy Complex, multi-disciplinary care and follow-up Many types of healthcare professionals need to be involved Patients with SMA have many co-morbidities that need to be tracked/ as part of involvement in trials (possible AEs) Adherence to standard of care is essential for robust/reliable trials

25 Achieving Site Readiness - What Does this Mean for Sites? Ensuring that PI/staff have a solid understanding of SMA, its pathophysiology, natural history, outcome measures, etc. is essential Conducting well-run, and effective trials require continued internal coordination, ongoing-communication, flexibility and putting the patient first. Effectively engaging / recruiting patients, parents, caregivers

26 Join Us in Changing the Future of SMA! Rosángel Cruz, Associate Research Director

27 Why Would You Want to Get Involved? Serve your current SMA patients by expanding access to clinical trials Gain valuable knowledge and expertise that may improve the quality of care delivered at your site and facilitate access to delivery of future therapies at your site Potentially attract new patients Change lives and the future of rare disease such as SMA

28 How Cure SMA Will Help You Become Ready To help sites determine whether they have the right ingredients to be successful To ensure that PTs & OTs have the right skills to evaluate SMA patients To help sites understand key considerations involved in running trials, especially in SMA

29 Readiness Evaluation for Clinical Trials Our Goal Help sites ensure site-readiness (experience with SMA, staffing, training, processes, infrastructure) before they engage / are approached by industry sponsors Our Approach We are establishing a two-part readiness evaluation to assess whether new sites have the key ingredients needed to effectively manage clinical trials in SMA. Steps in Evaluation 1. Preliminary evaluation to determine whether sites meet basic criteria necessary to take on a trial 2. In-depth evaluation assessing site infrastructure, staff experience & training, and other factors important to trial readiness

30 Readiness Toolkit Resources will include: Readiness checklist Basics of SMA Overview of recommended staff training (ethical considerations, site operations, SMA-specific outcome measures) Best practices for site coordination Best practices for implementing quality assurance processes (SOPs, Templates) for all aspects of a trials life. Appendix of other resources Toolkit may be expanded over time.

31 Next Steps for Getting Involved Megan Lenz, Director of Marketing and Communications

32 Next-Steps Clinical Trial Readiness Basic Eligibility Criteria If you meet all of the following criteria, Experience evaluating and following patients with SMA Familiarity with motor function outcome measures in SMA Experience conducting trials in any neuromuscular disorder Immediate Next-Step if you are interested in learning more about how to become involved, Contact us let us know via the webinar evaluation form or Getting Started Cure SMA will contact you in early April, after receiving your response with next steps A feasibility survey will be sent to you for completion and to begin assessment process

33 Next-Steps SMA Care Center Network/ Clinical Data Registry For questions regarding the SMA Care Center Network and Clinical Data Registry, please contact For information about the Clinical Care Meeting, please go to For information about the PT/OT Mentoring Program, please contact Page Kirkpatrick at or go to: For information about the Cure SMA New Treatment Site locator go to: or contact:

34 Q & A

35 Thank you!