SMA Summit on Drug Development
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
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 W. 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* |
