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ABCrowdFunding AdvisorsAlternative Financing for Orphan Drugs
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Orphan Diseases Context
Disease affecting
< 1/2.000 citizens (EU)
Disease affecting
< 200.000 citizens (US)
> 7.000
different orphan diseases
< 500 drugs
approved for orphan diseases (2015)
6.500+
orphan diseases have no cure
350M
patients worldwide (60M
US+EU)
A huge therapeutic need &
opportunity
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Success Stories and… Issues
Genzyme (US)
Alexion Pharmaceuticals (US)
BioMarin Pharmaceutical (US)
Actelion (CH)
Orphan Europe (FR)
SOBI (SE)
OPi (FR)
ERYTECH (FR)
600+ companies involved (WW)
Drug development attrition rate: slightly similar to non-orphan
Death Valley
Low awareness & poor understanding
Low prevalence rates
(Ultra) niche markets
Despite Orphan Drugs regulationsincentives, VCs uncomfortable
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Current Trend: Patients Empowerment
Individual patients initiatives
Patients organizations emergence
Link with scientists and clinicians
Link with industry
Patients lobby
politicians & governments
Patients and relatives are stakeholders
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Patients Associations & biotechs
Thousands patients organisations worldwide
- Make research grants
- Invest in start-ups
- Finance clinical trials
- Ignite start-ups
Providing non-dilutive and/or dilutive funding
Success track-record (Trophos, Prosensa, CFF/Vertex…)
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Case #1: AFM TELETHON
Association Française contre les Myopathies (FR)
Raised > 1.5B euros since the start of Téléthon
Donations go to patients’ assistance, research and trials
> 1B euros invested in R&D (academic+biotechs)
Started a 50M euros VC fund (with CDC)
Funds start-ups (Trophos, Prosensa, AnagenesisBiotechnologies…)
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Case #2: RDH12 Fund for Sight
RDH12 Fund for Sight (US)
2010: four families met at a Leber’s Congenital Amaurosis scientific meeting
Raised >1M US$
Currently funding US scientists developing a RDH12-targeting gene therapy
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Case #3: ALS Fund
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
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Case #4: Barth Syndrome Foundation
Barth Syndrome Foundation (US)
Started in 2000 during an International Barth Syndrome Conference
Funding BEZALIP (bezafibrate) repurposing with NHLBI
Orphan Designation Holder (2013)
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Case #5: AB Science
AB Science (FR) – public company
Formed in 2001 to develop a cure for Mastocytosis
Association Française pour les Initiatives de Recherche sur le Mastocyte et les Mastocytoses funded 650 patients genome sequencing
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Case #6: Solid Ventures
Solid Ventures (US)
Ilan Ganot, a JPMorgan banker, starts Solid Ventures following his 3-year old son Duchenne (DMD) diagnosis
Solid Ventures raised 17M US$ in January 2014
Focus on acquiring and developing disease-modifyingtherapies for DMD (deal with Pfizer)
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Case #7: Abeona Therapeutics
Abeona Therapeutics (US)
Formed in early 2013 to develop SanFilippo A and B genetherapies – 10x exit in 2015
Raised $750K in seed in 2013 + $3.6M in A Round in 2014
Investors: The Children’s Medical Research Foundation, Inc., Team Sanfilippo, Stop Sanfilippo (Spain), Fondation Sanfilippo (Switzerland)…
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Orphan + Patients = Cure
Targeted projects: early- (lead optimization, IND-enablingpreclinical, Phase I) or later-stage orphan drug candidates
Targeted fund providers: « Patient-Centric » (ie. patients, families, friends, patients organizations and foundations)
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Rationale for Early-Stage Companies
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 at this stage, except patients, relatives, or philanthropists?
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Rationale for Targeted « Investors »
« Patient-Centric » investors = highly motivated potential
Eagerly looking for a therapeutic return
Financial return expected, but secondary
Who may invest: patients, families, relatives + patients associations and foundations
Snowball effect expected: 1 patient = 2 parents +4 grand-parents + siblings + friends…
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What to Expect?
Grants (non-refundable, refundable, royalty-bearing)
Convertible loans
Equity
Support in clinical trials and regulatory process
Support in market access
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Our Process
Early-stage, orphan drugs projects sourcing (worldwide)
Projects assessment (Go, No-Go, Go-If)
Patients associations and foundations targeting
Grant-makers/Investors deck material production/review
Patients associations and foundations approach
Social media campaign (key to success)
Negotiations & closing
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