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Strong patent protection is essential for a start-up biotechnology company and can be a valuable company asset. However, it is also expensive, with costs ranging from tens to hundreds of thousands of dollars over time. This session will focus on how to get the most out of your patent dollars. This session presentation is available in audio format here: http://www.marsdd.com/bioent/dec4
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Exploiting EarlyExploiting Early
Staged DiscoveriesStaged DiscoveriesBioEntrepreneurship
Konrad Powell-Jones
Manager, Medical/Biotechnology
December 4, 2006
Innovations at University ofInnovations at University of
Toronto(IUTToronto(IUT))
Established in 1980 (InnovationsFoundation)
• Goal: maximize the impact of > $2million spent every day on research atUT and the affiliated teaching hospitals(such as SWCHSC)
• IUT services now focused on the UTcommunity
• We help researchers and businessescapitalize on unique opportunities
What is Innovation?
The process of making improvements byintroducing something new (OxfordDictionary)
The successful exploitation of new ideas(Dept. of Trade and Industry, UK)
How do you exploit your earlystage discovery?
Protect it (patents,copyright, tradesecrets)
then
Furtherdevelopmentalresearch
then
Out-License it
or
Create a Start-Upcompany
“Ever feel you’re on the verge of an
incredible breakthrough?”
Technology Transfer
ProtectProtect
DiscoveryDiscovery
DevelopDevelop
BusinessBusiness
FinancingFinancing
University
In Canada
60% have TToffices
The Process in Summary
Discovery/Observation
Preparation of Invention Disclosure
Review by I.P. Committee
Decision to
Prepare Patent
Application
YES
NO
File
YES
NO
Preparation of brief description
and preliminary claims
Preliminary Prior
Art Analysis
Assessment
Patentability
Review Proposed Disclosure and
Prior Art with Patent Counsel
Prepare Application
Assess Inventorship
Patent
Counsel
Submission of Provisional Application
YES
NO
Continuations
Divisionals etc.
Additional
Studies
What are theCommercialization Options?
License out to existing company
Start a new company
But what will you encounter…
……in the real world!!!!
The Big Picture
Unmet MedicalNeeds
PayersPerspectives
AgingPopulation
KnowledgeTranslation
R&DProductivity
Biomedical
Discoveries
RegulatoryPerspectives
Escalating R&D
Costs
Innovation Stagnation
Industry Productivity vs. Investment“The Challenge”
Source: 2004 PhRMA Annual Survey, 2003/2004 PAREXEL’S Pharmaceutical Industry Sourcebook
Total R&D Investment ($Billions)
$0
$5
$10
$15
$20
$25
1970
1971
1972
1973
1974
1975
1976
1977
1978
1979
1980
1981
1982
1983
1984
1985
1986
1987
1988
1989
1990
1991
1992
1993
1994
1995
1996
1997
1998
1999
2000
# NCEs
6040200
$30
$35
2001
2002
2003
Development Process: Bottleneck toDelivery of New Products
Net Cost: $802 Million InvestedOver 15 Years
Compound SuccessRates by Stage
16
14
12
10
8
6
4
2
0
Phase II100–300 Patient VolunteersUsed to Look for Efficacyand Side Effects
Phase III1,000–5,000 Patient Volunteers
Used to Monitor AdverseReactions to Long-Term Use
FDA Review ApprovalAdditional Post-
Marketing Testing
Phase I20–80 Healthy Volunteers Used to
Determine Safety and Dosage
Preclinical TestingLaboratory andAnimal Testing
Discovery(2–10 Years)
Years
Source: PhRMA Pharmaceutical Industry Profile 2003, Chapter 1: Increased Length and Complexity of the Research and Development Process. And
DiMasi, JA, Hansen, RW, Grabowski, HG. “The Price of Innovation: new estimates of drug development costs.” J of Health Economics. 2003:22:151-185.
5,000–10,000Screened
5
Enter Clinical
Testing
250Enter Preclinical
Testing
Compound Success
Rates by Stage
5,000–10,000
Screened
11
Approved by the FDAApproved by the FDA
00
22
44
66
88
1010
1212
1414
1616
Patients and Physicians Waiting forTreatments
8% of compounds entering Phase 1 willmake it to market, down from 14 % fifteenyears ago
Cost of development are escalating
Failures due to lack of safety and/orefficacy
Inability to predict failures
Major barriers to address uncommondiseases and explore unproven technologies
News Flash: Lipid Drug StudyHalted
Pfizer’s CETP inhibitor which increases HDLscauses serious adverse events.
Pfizer stops 15,000 patient Torcetrapidstudy, withdraws drug!!
81 patients taking Torcetrapid and Lipitordied cf. 51 taking Lipitor alone
Torcetrapid patients also showed anincrease in angina, CHF and cloggedarteries.
PersonalizedMedicine
! Pharmacogenetics
! New Imaging techniques
! Target therapies
! Responders/non responders
! Markers of drug metabolisms
! Predictive evaluation of safety
! Effective translation to clinicalpractice
Improving Development Sciences
New Biomarkers and SurrogateEndpoints
Safety
biomarkers
X
New imaging
techniques
Efficacy
biomarkers
Patients
Surrogate
endpoints
Predictive
disease
models
Improving Development Sciences
Predictive Animal Models
Advancing Innovative Clinical Trials :Learning trials versus empirical trials
Improving Measurement of PatientResponses :
Variation in individual response
Correlation with biomarkers
Measuring Patient PreferencesIdentify the overall benefits of therapies
“Mind The GAP”
…how do we narrow it?
Hurdles to Commercialization
Invention published prior to patent filing
Prior art
Un-validated discovery
Market too small
Competition too advanced
Market not ready for invention
Possible Solutions
Academic Partnerships and Collaborations
Government funding (CIHR and NSERC)
Industrial Partnerships and Collaborations
Useful Documents
UofT Confidential Disclosure Form
Assignment Agreement
Technology Owner’s Agreement
Confidentiality Agreement
Material Transfer Agreement
Inter-institutional Agreement
Service Agreement
Research Contract
License Agreement
Case Study
Professor Min Zhuo
4 gene targets (pain and fear memory)
In vitro and in vivo data
Patent and literature searches
R&D strategy
Commercialization strategy
Protect intellectual property
Pursue commercialization
Collaborations in place
Mouse models
Medicinal chemistry sessions
SMART HTS Facility
CIHR POP1 Grant
Toxicology studies
NeoBrain Pharma Profile
Focused on small molecule drug discovery fortreating diseases of the cns.
Founded by the Innovations at the Universityof Toronto and Professor Min Zhuo(Physiology UofT).
3 Areas of Indication
acute persistent, neuropathic and chronicinflammatory muscle pain.
delaying opioid tolerance
fear memory and anxiety
Drug Discovery Programs
lead program focused on the inhibition of aspecific neuronal adenylyl cyclase (nAC)isoform. NB001 has been identified as onesuch non-competitive nAC inhibitor.
discovery of inhibitors of a specific proteinkinase, which when combined with opioidsreduces analgesic tolerance to chronictreatment.
drug discovery program focused on treatinganxiety conditions associated withcontextual and auditory fear memory bythe selective inhibition of specific glutamatereceptor subunits.
Proof-of-Concept Data
Mouse models of neuropathic pain
Knockout mouse models
Electrophysiological data
Antisense data
Small molecule data
Behavioural data
Preliminary toxicology data
Models
Example of neuropathic pain
AC inhibition (i.p.) in neuropathic pain
Mechanic
al a
llodynia
(%
Response)
0
20
40
60
80
100
120VehicleNB001 0.1mg/kgNB001 1.1mg/kg
*
Effective for neuropathic pain whenapplied orally in rat!
NB001 orally at 1 mg/kg/3ml
Time
Me
ch
an
ica
l allo
dy
nia
(% re
sp
on
se
)
0
20
40
60
80
100
120
Before 45min 2hr 4hr 6hr
Comparison with other painmedicine
Cumulative effect of gabapentin on withdrawal responses on day 7 after CPN ligation(n=4 C57 mice; vonFrey filamnet 1.65)
Day 7
Nu
mb
er o
f res
po
ns
es
in
5 tria
ls in
%
0
20
40
60
80
100
30 min after
10 mgm
/kg
30 min after
total 30 mgm
/kg
1 hour after
total 30 mgm
/kg
Intellectual Property
-Adenylyl Cyclase program:
US and Chinese patent applications filed onthe specific adenylyl cyclase target and theclass of inhibitory compounds
-Protein Kinase program:
A US patent application filed.
-Fear memory/Anxiety Program:
A US patent application filed
Financial Requirements
Over the next three years, NBPI plans to completeIND-enabling preclinical studies for NB001 for pain,to advance another drug to preclinical developmentfor anxiety and to identify a lead compound for athird indication.
NBPI requires financing of US$5 million to supportoperations through 2009. Financing will be used toadvance the drug discovery programs, executelicenses to its intellectual property, build itsmanagement team, pursue business developmentefforts, and complete a subsequent round offinancing to allow entry into clinical trials.
Ongoing R&D activities
$150,000 awarded from CIHR Proof-of-concept grant
Initial PK and toxicity tests for leadingcompound NB001
RememberRemember
If you have doubts seek advice
Public disclosure of research results isa fundamental act of scholarship
The choice is yours, but publiclydisclosing before you have givenconsideration to patenting andcommercialization may, in the longrun, remove an option you wish youhad kept
Contact InformationContact Information
Konrad Powell-Jones
Phone: 416 - 978 - 5730
Email: [email protected]
Patent, Publish, Prosper!
