Speaker Dr. Annemarie Moseley, CEO of Aggregate Therapeutics (Palo Alto) explores how drug-device combination products are altering the medical practice from development to regulation to treatment. Part of the MaRS Emerging Technologies Event Series. More information on the series can be found here: http://www.marsdd.com/emergingtech/

1. Regenerative Medicine: Impact of Convergence on Drug, Device and Biologics Development Annemarie Moseley, PhD, MD Acting CEO, Aggregate Therapeutics, Inc.

2. It’s a new age….

10. Combination Products Include Any investigational drug, device, or biological product packaged separately that according to its proposed labeling is for use only with another individually specified investigational drug, device, or biological product where both are required to achieve the intended use, indication, or effect. (MiniMed Pump)

11. Designated Agency To determine the agency with primary jurisdiction for the premarket review and regulation of a combination product, will be based on the primary mode of action of the product.

15. Bone marrow stem cells … within 3 months researchers found that the pumping activity had increased…this work, although preliminary, has the potential to revolutionize the treatment of heart failure …. AHA advertisement in Time Magazine, 2004

16. Will one delivery approach fit them all? Bone Marrow Adult Stem Cells Myoblasts

24. Investing in Convergence Device which has been combined with drug? - Is the drug approved? Human safety? - Is the drug delivered via a polymer or other drug delivery vehicle? - Manufacturing GMP vs. existing quality systems and impact on cost and timing - Value proposition?

25. Investing in Convergence Delivery device? - Drug or biologic delivered? - Is there a predicate? - Is there an existing market? - Have you covered delivery of all drugs, biologics etc in IP claims - Value proposition?

26. Investing in Convergence Biologic? - Does the biologic require or have improved efficacy with local delivery? - Is local delivery more cost efficient? - Do distribution channels exist? - Does it need a newly designed delivery system or can it be delivered with existing device?

28. Commercialization Target Product Profile -Indication -Patient Benefit -Asking price/cost of goods - Delivery -Reimbursement Potential It’s never too early!!

29. TIER 1 TIER 2 TIER 3 Pipeline REDUCED RISK THROUGH LEVERAGED PRODUCT APPROACH Synovo-Derm ATX-Derm ATX-CNS Diabetes Synovo-Joint ATX-Ortho Cancer DX Marketed material; Novel ATX cells + New Indication for improved formulation Synovo ATX cells RISK

30. Sg2 Healthcare intelligence 2006 Looking to the future—learning from the past!