1

FUNDING FROM PATIENTS ASSOCIATIONS

• Patients associations & industry?• Patients organizations have a very strong interest in the

development of drugs for unmet medical needs• Patients organizations: « mum & dad » initiatives or

prominent associations• Funding from these organizations: non-dilutive and/or

dilutive (grants, convertible loans, equity)• Hundreds of fundings observed (US.. and EU)

2

FUNDING FROM PATIENTS ASSOCIATIONS

• Company: Trophos (FR)• Patients association: AFM-Téléthon (FR)• R&D programs: ALS and SMA• Dilutive funding: 3.6M€• Non-dilutive funding: 19M€+• Acquired by Roche (2015): 470M€• AFM-Téléthon gets 43M€ (8M€ upfront + 35M€ milestones)• 2X

3

FUNDING FROM PATIENTS ASSOCIATIONS

• Company: Aurora Biosciences/Vertex Pharmaceuticals (US)• Patients association: Cystic Fibrosis Foundation (US)• R&D program: Cystic Fibrosis• Dilutive funding: -$• Non-dilutive funding: 150M$ (royalty-based grants)• Cystic Fibrosis Foundation’s rights sold to Royalty Pharma (2015)• For a cash payment of.. 3.2B$!• 22X!

4

FUNDING FROM PATIENTS ASSOCIATIONS

• Benefit: access to funding/non-dilutive funding from non-financial organizations (different rationale)

• Benefit: cost of money• Benefit: not only money, but support from patients (enrollment

in clinical trials, discussions with regulation agencies and payors)• Challenge: identify relevant associations• Challenge: industry vs. patients organizations cultures• Challenge: set up syndications (few associations have deep

pockets)

5

FUNDING FROM PATIENTS ASSOCIATIONS

• Patients associations fund drugs, diagnostics & devices

• Patients associations should be included in every funding campaign, particularly in rare diseases, but not only

• Contact: Christian Girard, Consultant christian@orphandrugsindustry.com