SMA Summit

SMA Summit on Drug Development, September 28-29, 2007

Summary: SMA Summit on Drug Development (text summary)

Agenda Day 1

Session 1: Opening Remarks (Slides)
John Kissel- Introduction and Overview of Meeting Goals  

Session 2: Scientific Overview (Slides)
Charlotte Sumner- Spinal Muscular Atrophy Biology and Therapeutics Development: An Overview       

Session 3: Overcoming Challenges in Translational Research and Drug Development for SMA (Slides)
Moderated by John Kissel

Tan Nyugen- FDA Office of Orphan Product Development    
Jean Temeck- Regulatory Requirements For Pediatric Drug Development     
Jill Heemskerk- A role for NIH in Translational Research                   

Session 4: Established SMA Clinical Trial Resources (Slides)
Moderated by Susan Iannaccone

Ching Wang- Standard of Clinical Care for Spinal Muscular Atrophy      
Jacqueline Jackson- International Spinal Muscular Atrophy Patient Registry      
Richard Finkel- Parent Survey Data (not posted)        

Existing US SMA Clinical Networks
Susan Iannaccone- AmSMART       
Darryl De Vivo- The PNCR Network       
Kathryn Swoboda- Project Cure SMA      
Existing European SMA Clinical Networks
Enrico Bertini- Treat-NMD Overview      
Michael Rose- NMD Registry of Outcome Measures   
Susan Iannaccone- Natural History of SMA      

Session 5: Clinical Trial Design (Slides)
Moderated by Edward Kaye

Petra Kaufmann- Issues in SMA Clinical Trials      
Alfred Sandrock- Industry Perspective on SMA Clinical Trials (not posted)     
Panel Discussion: Clinical Trial Design Issues
Panelists: Jean-Louis Abitbol, Wilson Bryan, Petra Kaufmann, Alfred Sandrock, Kathryn Swoboda, Ching Wang

Agenda Day 2

Session 6: Regulatory Requirements for Orphan and Pediatric Drug Development (Slides)
Moderated by Tan Nguyen

Agnes Saint Raymond - (Video) Orphan Designation and Paediatric Initiative
Wilson Bryan- Regulatory Issues in SMA Clinical Trials

Session 7: Drug pipeline (Slides)
Moderated by Jill Heemskerk

Jill Heemskerk- Chemical Optimization of Indoprofen for the Treatment of SMA   
Jon Tinsley- Use of a Drosophila SMN Model in a High Throughput Small Molecule Screen 
Leo Liu- Identifying Novel SMA Drug Targets and Drug Candidates    
Lou Barbato- Sodium Phenylbutyrate (NaPB) for the Potential Treatment of SMA (not posted)   
Mark Gurney- Families of SMA - deCODE – Clinical Candidate Selection  
Paul Higgins- Identification of Tetracycline Compounds That Correct Defective SMA Splicing   
Rebecca Pruss- Discovery and Development of TRO19622 for the treatment of SMA     
Zuomei Li- Brain-Penetrant, Isotype-Selective HDAC Inhibitors    
Sergey Paushkin- An Innovative and Integrated Approach to Identify Compounds (not posted)   

Session 8: Evaluating Pre-clinical and Clinical Compounds (Slides)
Moderated by Darryl De Vivo   

Q&A session and Panel Discussion
Panelists: C. Frank Bennett, John McCall, Paul McGonigle, Thomas Meier, Robert Pacifici, Charlotte Sumner

Session 9: Endpoints for SMA Clinical Trials (Slides)
Moderated by Leslie Morrison

Kristin Krosschell- Functional Testing        
Kathryn Swoboda- Electrophysiologic Outcomes CMAP and MUNE      
Marion Main- Strength Testing and Walking in SMA 2&3      
Richard Finkel- Type I-specific Measures (not posted)       
Anita Simonds- Outcomes Measurement: Pulmonary Assessment Type 1&2       
Linda Hynan- Measuring Quality of Life in Patients with Spinal Muscular Atrophy          
Louise Simard- Biomarkers for SMA Clinical Drug Trials       
Q&A and Panel Discussion: Clinically Meaningful Endpoints for SMA Clinical Trials
Panelists: Wilson Bryan, Julaine Florence, David Jacoby, Kathryn Swoboda

Session 10: Conclusions (Slides)
Moderated by Kenneth Fischbeck

Kenneth Fischbeck- Overview of the Meeting        
Panel Discussion: Advancing SMA Drug Development: Challenges, Funding Priorities and Next Steps
Panelists: Petra Kaufmann, Edward Kaye, John Kissel, Langdon Miller, Tan Nguyen, Robert Pacifici

 *Dinner Speaker Presentation in Session 10 Slides*

Neurology Today: SMA Drug Development Gains Momentum Under NINDS Model for Neuro-Research Partnerships. Reprinted with permission from the American Academy of Neurology from Neurology Today, Nov. 6, 2007.