2008 Family & Professionals Conference

1. 2008 Family & Professionals Conference BEANTOWN: The SMA Revolution

2. Master Plan Everything builds from the base: Conference reflects these stages. COMMUNITY

3. Master Plan Everything builds from the base: Conference reflects these stages. CARE/MANAGEMENT COMMUNITY

4. Master Plan Everything builds from the base: Conference reflects these stages. EXISTING DRUGS CARE/MANAGEMENT COMMUNITY

5. Master Plan Everything builds from the base: Conference reflects these stages. TREATMENT EXISTING DRUGS CARE/MANAGEMENT COMMUNITY

6. Master Plan Everything builds from the base: Conference reflects these stages. CURE TREATMENT EXISTING DRUGS 2008 CARE/MANAGEMENT COMMUNITY

7. Stages To a Treatment and Cure: 1st – Manage – with Care. 2nd – Slow – with Existing Drugs. 3rd – Stop – with New Drugs. 4th – Reverse – with a Cure.

8. 1) SMA Community Orphan Disease: • Everything starts with the community. • Small market – too risky for companies (and government) to get involved at start. • Fundraising – over $40 Million to date. • Over time, if successful, will see transition from Community: • plus Government, • plus Companies.

9. 2008 Conference – SMA Community • Invested over $2 Million since 1990 to bring community together every year. • Networking – Families and Researchers: • Opening, Lunches, Banquet, Carnival, Wonderful Life…… • Informal Opportunities. • Key Aspect - Attractive to the outside. • Legislative update – advocate.

10. 2) Care/Management • Sharing and implementing best practices. • Families and physicians working together. • No effect on underlying cause. • Everyday items – special for SMA.

11. 2) 2008 Conference - Care/Management • Informal sharing is just as important: • Workshops: • Share Your Experiences. • “Talk it Outs” with Al. • Standard of Care – Breathing, Feeding, Movement, Illness: • Workshops: • Respiratory Care I & II, Care for III, Ortho Management.

12. 3) Existing Drugs - Slow • PBA, VPA, HU. • Designed for other diseases: • Urea Cycle, Epilepsy, Sickle Cell. • Cheap and quick to test (relatively): • Generally shown to be safe already. • Long shots – not likely to be very effective . • Builds SMA clinical trials infrastructure.

13. 1. CARNI-VAL Type I 2. Stop SMA 3. Nptune 01 4. Nptune 02 5. Trophos 19622 6. VALIANT 7. Stanford HU Type I 8. HU Taiwan 3) 2008 Conference – Existing Drugs • New General Session: • Saturday – Therapeutic Approaches. • Sunday Q&A: Two Trials Recently Completed: Stanford HU in Type II/III AND Project Cure SMA CARNI-VAL Phase II

14. 4) AND 5) Treatment AND Cure • Failure is the rule: • 9 out of 10 entering clinical trials fail. • Plan and prepare for this: • SMA needs 10 programs into clinic. • Cell replacement: • Even more risky, • But high potential. New Drugs/Treatment Stop Cure Reverse

15. Clinical Development Preclinical: Discovery 4 AND 5) 2008 Conference – Treatment/Cure • Sunday Q&A: • Drug Development Panel • Clinical/Medical Panel FDA Approval Assay Dev. Screening Hit to Lead Lead Optimization Pre-Clinical Safety Clinical Trials Phase I, II, III Target ID Clinical Candidate IND FIH NDA Paratek Tetracyclines Quinazolines

16. Conference reflects these stages Everything builds from the base: CURE TREATMENT EXISTING DRUGS 2008 CARE/MANAGEMENT COMMUNITY

17. Progress 20002008 • Conference Attendees:275

18. Progress 20002008 • Conference Attendees:275 750

19. Progress 20002008 • Conference Attendees:275 750 • Companies in SMA:1

20. Progress 20002008 • Conference Attendees:275 750 • Companies in SMA:1 23

21. Progress 20002008 • Conference Attendees:275 750 • Companies in SMA:1 23 • Drug Discovery Programs:1

22. Progress 20002008 • Conference Attendees:275 750 • Companies in SMA:1 23 • Drug Discovery Programs:1 15

23. Progress 20002008 • Conference Attendees:275 750 • Companies in SMA:1 23 • Drug Discovery Programs:1 15 • Active Clinical Trials: 4

24. Progress 20002008 • Conference Attendees:275 750 • Companies in SMA:1 23 • Drug Discovery Programs:1 15 • Active Clinical Trials: 4 12