Role of academic research and spin-off (start-up) companies in drug development and pharma/biotech industry
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Role of academic research and spin-off
(start-up) companies in drug development
and pharma/biotech industry
Péter Ferdinandy, MD, PhD, MBA
Department of Pharmacology and Pharmacotherapy,
Semmelweis University, Budapest, Hungary
Pharmahungary Group, Budapest, Hungary
www.semmelweispharma.com
www.pharmahungary.com
Why pharma industry is a leading one? „The desire to take medicine is perhaps the greatest feature which distinguishes man from animals.” Sir William Osler (1849-1919, founder of the John Hopkins Hospital) • Science for curiosity? • Science for the benefit of the society? - results useful for pharma/biotech industry www.pharmahungary.com www.semmelweispharma.com
No limits of drug development:
what can be a medicine?
1. Small molecules
2. Macromolecules (mostly biologics)
3. Advanced therapy products (2009/120/EC Directive)
Gene therapy medicinal products:
- recombinant nucleic acid for the regulation, repair, substitution,
addition or delation of a specific gene sequence
Cell / tissue therapy medicinal products:
- consist of manipulated cells or tissues
4. The Combination of the above with medical devices
www.pharmahungary.com www.semmelweispharma.com
No limits of drug development:
What can be a medicine?
„DNA nano spider” : A molecular nanorobot dubbed a 'spider'
and labeled with green dyes moves along a DNA track to its
red-labeled goal (Lund et al, Nature, 2010)
„When using
appropriately
designed DNA
origami, the
molecular spiders
autonomously carry
out sequences of
actions such as
'start', 'follow', 'turn'
and 'stop‘”
www.pharmahungary.com www.semmelweispharma.com
No limits of drug development
An example of a drug-device combination: magnetic
nanoparticles to speed up neuroregeneration
NGF
Fe3O4 CORE
Alexa 488
PEI-shell
Magnetic nanoparticle
Magnetic nanoparticles provide
Direction of the ma gnetic field physical guidance to direct
more efficient nerve regeneration
www.semmelweispharma.com Riggio et al, Nanomedicine, 2014
No limits of drug development A drug-device combination for targeted release of drugs www.pharmahungary.com www.semmelweispharma.com
Drug development and pharma industry:
Takes 8-12 years and roughly $1bn to market
Patent protection: only for 25 years
www.pharmahungary.com www.semmelweispharma.com
Drug development and pharma industry:
total cost of drug development
Company drugs R&D Spending/ Total R&D Spending
approved Per Drug ($Bil) 1997-2011 ($Bil)
AstraZeneca 5 11,8 58,9
GlaxoSmithKline 10 8,2 81,7
Sanofi 8 7,9 63,3
Forbes
Roche Holding AG 2012:11 „At $12 billion7,8per drug, inventing
85,8
Pfizer Inc. 14 7,7 108,2
Johnson &medicines
Johnson is15
a pretty unsustainable
5,9 business”
88,3
„At& $3.7
Eli Lilly Co. billion, you11 might just be4,6 able to make50,3money”
Abbott Laboratories 8 4,5 36,0
Merck & Co Inc 16 4,2 67,4
Bristol-Myers Squibb„the
Co. main
11 expense 4,2is failure” 45,7
Novartis AG 21 4,0 83,6
Amgen Inc. 9 3,7 33,2
Sources: Forbes; InnoThink Center For Research In Biomedical Innovation;
Thomson Reuters Fundamentals via FactSet Research Systems, 2012
www.pharmahungary.com www.semmelweispharma.com
Drug development is risky:
Success rate is extremely low (but better than lottery)
IND NDA
High risk of development: efficacy, safety, and affordability
Drug development for rare diseases (orphan disease) - financing problems
www.pharmahungary.com www.semmelweispharma.com
Number of approved new drugs -
stagnating
Nature Rev Drug Discovery 2016 www.semmelweispharma.comThe productivity of pharma/biotech
industry - decliningHow to move forward?
Less risky
Increasing
ways to
success of
develop new
innovations
drugs
www.semmelweispharma.com Modified from Drug Discovery Today, 2012The less risky ways of drug development
Advanced
therapy Drug-
products device
www.semmelweispharma.com Modified from Drug Discovery Today, 2012Drug „repositioning”: new indications for old drugs
(little preclinical development - jump into phase II clinical trials)
New Drug Application:
Marketing uthorization
Phase I Phase II Phase III Phase IV
New indication at
the same dose Observational
range studies
New indication New indication
Human
tolerance (HT) Postmarketing
controlling
Pharmako- studies
kinetics (PK)
Prospective,
randomized,
Quality of life comparative
and pharmaco- trials
economical studies
www.pharmahungary.com www.semmelweispharma.comIncrease the success of innovations: modern
drug development is a cooperative effort
Academic institutions: basic science and
new technologies („omics”, nanotech, in silico
models, etc)
Small innovative R&D companies
(„small biotech”, spin-offs)
R&D service companies: assays, animal
studies, oganizing clinical studies, patent
lawyers, etc
Large pharma companies:
development, financing
Investors: business angels, FFF,
venture capitalists, investments funds
Government regulatory authorities
Health care insurance (government, private)
Hospitals
Doctors www.semmelweispharma.comIncreasing the success rate of innovations:
„translational medicine” - „biomarkers”
Learning phase Confirmation phase
Non-clinical
R&D: clinical phases M
-Translational A
models R
(comorbidities, „Phase 0” Phase I Phase
FázisIIII Phase III K
comedications) E
T
- biomarkers
- large animals
Translational medicine
Proof of
biomarkers
Feedback concept
Optimization
Materials of human originSource of innovation (FDA approved drugs)
Source of Registration Current Source of Registration Current
innovation owner innovation owner
top 20 big phara
medium pharma
small pharma/biotech
other (e.g. academia)
Source: IMSHealth analytics www.semmelweispharma.comInnovation in drug development: role of academic
research and spin-off (start-up) companies
Innovation process: from basic research to marketed product
Basic science
financing (government,
charity, etc)
Basic research IP protection and
technology transfer
Academic of early stage
institution projects
Spin-offs - start-ups
Applied research (SME - small medium enterprise)
Industrial development
Financing
Big pharma - private
Product on the market - publicManagement difficulties at academic institutions and
start-up companies
Early stage R&D company
manager
“Hard qualities” “Soft qualities”
Balanced
management
Business missions, Science values
financial goals
Strict quality Customs, „genious
control, project professor” behaviour,
goals (e.g. ISO, creativity
GLP, GCP)
Company
www.cardiovasc.com output
www.pharmahungary.com www.semmelweispharma.comSummary and conclusions I.
Role of the acedemic sector in pharma industry:
- discovery research
- development of basics of novel technologies
- protecting intellectual property and transfer of technology to industry
- special R&D services ? – administrative and structural challenges?
- clinical development
Role of spin-off companies in pharma/biotech industry:
- create and utilise intellectual property
- further progress of early stage projects to be visible for big pharma
and investors
- tech transfer to big pharma / or growing to become mid size or big
pharma
- R&D services (e.g. non-clinical and clinical CRO, SMO, Phase 1 units,
consultancy, regulatory, medical writing, etc.)
www.pharmahungary.com www.semmelweispharma.comProtection of your intellectual property:
patent and publish
„Research that is not carried out under quality control and not
documented is not done”
Protecting your know-how (keeping secrets)
- no confidentiality, no business with pharma/biotech
industry
- idea and data can be stolen? – data sharing?
Patenting
- only publication? – your idea will never get to the market
- future benefit for you and your institution (more chance
than lottery :-)
Publishing
www.pharmahungary.com www.semmelweispharma.comProductivity in publications:
Central-Eastern EU is among leaders... is it good?
Number of publications per 1 M USD R&D
expenditure in universities and research institutes
(source: NSIOD, Institute for Scientific Information)
Korea 21,8
33
USA 52,8
62,4
Poland 63,8
67,2
Czech Rep. 68,1
76,3
Spain 77,4
84,4
UK 91,5
105
Hungary 107,2
0 20 40 60 80 100 120
www.pharmahungary.com www.semmelweispharma.comWhere are Central-Eastern Europe?: the number of patent applications does not meet with publication productivity www.pharmahungary.com www.semmelweispharma.com
Responsibilities of universities in protecting the
intellectual property
- discovered and
developed at
Semmelweis
University,
Budapest in the
sixties
- on the market in
62 countries
- Direct benefit of
the university?Repositioning for deprenyl and their follow up molecules:
cancer prevention 2016
- new patent, already partnering with big pharma
www.semmelweispharma.comTake home messages
• no limits of drug development – you can be part of it
• confidentiality – think of patents – publish
• quality control system is essential (SOPs, strict
documentation)
• think of being an enterpreneur – „start up” - create jobs
• collaborate with industry
- access to novel technologies
- better publications?
- helps in financing your lab
- my example – listed on highlycited.com 2014 – thanks to industry
driven projects (at least 1/3 of my publications/citations)
www.pharmahungary.com www.semmelweispharma.comwww.pharmahungary.com semmelweis.hu/pharmacology
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