Disease affecting < 1/2.000 citizens (EU) Disease affecting
< 200.000 citizens (US) > 7.000 different orphan diseases
< 500 drugs approved for orphan diseases (2014) 6.500+ orphan
diseases have no cure 350M patients worldwide (60M US+EU) A huge
therapeutic need & opportunity do not distribute
Slide 3
Genzyme (US) Alexion Pharmaceuticals (US) BioMarin
Pharmaceutical (US) Orphan Europe (FR) SOBI (SE) 600+ companies
involved (WW) Drug development attrition rate: similar to
non-orphan Death Valley Low awareness & poor understanding Low
prevalence rates Niche markets Despite incentives, VCs reluctance
do not distribute
Slide 4
Individual patients initiatives Patients organizations
emergence Link with scientists and clinicians Link with industry
Patients lobby politicians & governments Patients and their
relatives are stakeholders do not distribute
Slide 5
Association Franaise contre les Myopathies (France) Raised >
1.5B euros since the start of Tlthon Donations go to patients
assistance, research and preclinical/clinical trials > 1B euros
invested in R&D Started a 50M euros VC fund (with CDC) Funds
start-ups do not distribute
Slide 6
RDH12 Fund for Sight (US) 2010: four families met at a Lebers
Congenital Amaurosis scientific meeting Raised >1M US$ Currently
funding US scientists developing a RDH12-targeting gene therapy do
not distribute
Slide 7
ALS Fund (NL) 3 Amyotrophic Lateral Sclerosis patients
gathered, started and funded Treeway, a biotech developing ALS
cures, and Project MinE, a genetic research project 2014: launch of
the ALS Fund, which aims to collect 100M Euros to fund ALS cures do
not distribute
Slide 8
Barth Syndrome Foundation (US) Started in 2000 during an
International Barth Syndrome Conference Funding BEZALIP
(bezafibrate) repurposing with NHLBI Orphan Designation Holder
(2013) do not distribute
Slide 9
AB Science (FR) Formed in 2001 to develop a cure for
Mastocytosis Association Franaise pour les Initiatives de Recherche
sur le Mastocyte et les Mastocytoses funded 650 patients genome
sequencing do not distribute
Slide 10
Solid Ventures (US) Ilan Ganot, a JPMorgan banker, starts Solid
Ventures following his 3-year old son Duchenne diagnosis Solid
Ventures raised 17M US$ in January 2014 Solid Ventures is focused
on acquiring and developing disease-modifying therapies for
Duchenne Muscular Dystrophy do not distribute
Slide 11
Abeona Therapeutics (US) Formed in early 2013 to develop gene
therapies for SanFilippo A and B Raised 750K US$ in a seed round in
December 2013 Investors: The Childrens Medical Research Foundation,
Inc., Team Sanfilippo, Stop Sanfilippo (Spain) and Fondation
Sanfilippo (Switzerland) do not distribute
Slide 12
Targeted projects: early-stage (lead optimization, IND-enabling
preclinical, Phase I) orphan drug candidates Targeted investors:
Patient-Centric investors (ie. patients, families and friends) do
not distribute
Slide 13
Early-stage projects (late discovery IND- enabling studies) =
Death Valley Highest risk level 10.000 preclinical compounds = 1
marketed (not orphan-specific, but still applies) Who wants to
invest here? do not distribute
Slide 14
Patient-Centric investors = highly motivated potential
investors Eagerly looking for a therapeutic return Financial return
expected, but secondary Who may invest: patients, families,
relatives + patients associations Snowball effect expected: 1
patient = 2 parents = 4 grand-parents = siblings = friends do not
distribute
Slide 15
Early-stage, orphan drugs projects sourcing (worldwide
deal-flow) Projects assessment (Go, No-Go, Go-If) Patients
associations targeting International, syndicated equity-
crowdfunding campaigns strategy design Equity-crowdfunding
platforms selection Investor deck material production Social media
buzz-making (key to success) do not distribute
Slide 16
Slide 17
Contact: Christian Girard [email protected] +33 (0)6
67 26 60 92 do not distribute