Non For Profit Model for Rare Disease Therapy Development

1. Non For Profit Modelfor Rare Disease Therapy Development

2. Therapy Development Process Historically big business Large investment Time to Trial 6 to 8 years Rare disease economically not attractive Rare Disease Therapy Development - Historically

3. Foster collaborations Liaisons between Basic Research and Biotech companies Deliver resources to fill funding gaps in the development process “Managing science” –resources/timeframes/direction/goals Therapy focus – move what is known today forward Political Involvement Typical Rare Disease “Proactive” Advocacy Groups

4. Enzyme Therapy Gene Transfer Stem Cell Therapy Small Molecule Pharmaceuticals High Through-put Screening Standard Therapeutic Options

5. Proof of Concept Investigational New Drug Application (IND) Protocol Development Markers and Endpoints Toxicology Short Term Studies Long Term Studies Pharmacology Chemistry/Manufacturing Dosage and Delivery Studies Therapy Development Process

6. Institutional Review Board (IRB) Informed Consent Forms Approvals Recombinant Advisory Committee (RAC) Food and Drug Administration (FDA) Clinical Trial Initiation Therapy Development Process (cont.)

7. Disease A B C Proof of Concept X X X IND X X X IRB X X X RAC X X X FDA X X X Clinical Trial Initiation X X X Rare Disease Therapy Development Process Commonality

8. Regardless of the therapy we all face the same procedural development issues. Uniting empowers rare diseases What is good for one of us is good for all How do we leverage the commonality? Rare Disease Synergies

9. Non-For-Profit foundation sponsors the therapy development Academic society is the Principle Investigator Non-For-Profit Therapy Development Model

10. Goal is to develop therapies verses commercialization of a drug Quickly take proof of concept from the lab to the clinic NPF assume risk and responsibility Leveraging infrastructure and regulatory knowledge Provide data to companies for future clinical phases for long term solutions Funding sources: foundations, companies, or NIH Cost savings in therapy development for companies to prove concept before large scale investment Business issues not a concern to achieve the NFP goals regarding intellectual properties or patents Non-For-Profit Therapy Development Model Benefits

11. Rare diseases have a platform to move to a clinical program Together we have stronger voice to regulatory committees Quicker to clinic Develop NFP models for each type of therapy We all face the same issues Advancing Rare Diseases to the Clinic in a United Approach