Open Innovation in Small Molecule Lead Discovery

“Center for Open Innovation in Drug Discovery”

From Molecules to Business – 25 September 2014

Jac Wijkmans

Take Home Messages

Great need to organize pharma R&D in a different way

Academia hold the key to bridge the innovation gap between

exploratory research & early clinical development

Merge the strengths of academic & industrial drug research

Initiated a feasibility study to create “Center of Open

Innovation in Drug Discovery” to place RU/Radboudumc at

forefront of translational medicine

Similar successful initiatives exist abroad that have proven

to provide a sustainable academic drug discovery enterprise

The Pharma Landscape

Society

‒ Increasing incidence of chronic diseases.

‒ Escalating health care costs

‒ Increased patient awareness

‒ Personalized health care

Industry

‒ Declining productivity

‒ Risk aversion

‒ R&D externalization

‒ Late stage focus

‒ Innovation pull

‒ Driver: Shareholder value

Academia

‒ Austerity measures

‒ Clinical trial experts

‒ Increasingly entrepreneurial

‒ Early stage focus

‒ Valorization push

‒ Driver: Publications & health

Evolving R&D Process

<2005: The ‘good old days’

2005 - present: Shift from R&D to S&D (Search & Development)

IndustryAcademia Pre-clinical candidate

Need to Organize Early R&D Differently

Spin-offCRO #1 CRO #2

CRO #3

?

A:

B:

‒ Too early to out-license

‒ Shortage in funds

‒ Missing skills

‒ No open innovation (recycling of knowledge, molecules, IP)

Issues

Academia Hold the Key, however, …

… while targets, assays, compound synthesis & patients are present, medicinal chemistry capabilities are

critically missing

Frye et al. Nature Rev. Drug Discov. 10, 2011, 409

Medicinal Chemistry

Optimizing Molecular properties to get into Business

IdealNot acceptable

1. Affinity

2. Selectivity

3. Efficacy

4. Solubility

5. Metabolic Stability

6. Protein binding

7. Toxicity

Medicinal Chemistry

Optimizing Molecular properties to get into Business

IdealNot acceptable

1. Affinity

2. Selectivity

3. Efficacy

4. Solubility

5. Metabolic Stability

6. Protein binding

7. Toxicity

Multidimensionaloptimization

paradigm

Empower Academic Drug Research

Academic

biomedical

research

Medicinal

chemistry

capabilities

“Blend academic biomedical research with industry-

standard drug discovery capabilities to valorize innovation”

“Establish a leading force in academic drug discovery”

Center for Open Innovation in Drug

Discovery

COIDD

Creating Value Chains

A Sustainable Pharma R&D Ecosystem

IP

(pre-)clinical candidates

Platform technologies

Spin-offs

Publications

Synergies/cross-overs

Education & training

$$$$$$

Potential projects identified

European Lead FactoryHTS

Center for Open Innovation in Drug Discovery (COIDD)

Incorporate the highest level of drug discovery into

academic research and advance the most exciting scientific

breakthroughs towards the development of drug candidates

suited for clinical studies

Vision

Establish an unique & sustainable pharmaceutical R&D

ecosystem to create new small molecule therapeutics to

improve public health and place RU/Radboudumc at

forefront of translational medicine

Mission

COIDD Core Expertise

MedicinalChemistry

Chemical Biology

Drug discovery

Tool compounds

Project Management & Development

Leads & (pre)clinicalcandidates

New projects

industry-trained professionals

Dedicated facilities

Open Innovation in Practice

New molecular target or

differentiating assay

ClinicalNeed?

amenablefor HTS?

HTS & compound

optimization

Pre-clinical candidate

out-licensing/

partnership

For example:

Revolving funds

A Validated & Sustainable Model!“De-risking investments in new treatments for brain

disorders by blending academic based research with state-of-

the-art drug discovery resources”

“Bringing dedicated medicinal chemistry expertise to bear on

biological targets being investigated by UNC faculty”

A Validated & Sustainable Model!

Vanderbilt Center for Neuroscience Drug Discovery

Founded in 2003, Track Record:

2009: Drug discovery pact with J&J

2010: Research collaboration with Seaside Therapeutics

2011: Partnership Michael J. Fox Foundation

2011: License deal Karuna Pharma

2012: BMS Parkinson R&D collaboration

2013: Licensing & research deal with AstraZeneca

“De-risking investments in new treatments for brain

disorders by blending academic based research with state-of-

the-art drug discovery resources”

A Validated & Sustainable Model!

“Exploiting and transforming innovative

research into pharmaceutical applications in

close cooperation with academic partners”

Lead Discovery Centre Dortmund

Founded in 2007, Track Record:

2011: First LDC lead licensed to Bayer

2013: 2nd LDC lead licensed to Qurient

Drug discovery alliance AstraZeneca

Strategic partnership Merck Serono

2014: Drug discovery alliance Daiichi Sankyo

Embedding in Health Valley Region

Radboud Nanomedicine

Alliance

“HealthyBrain”

Technology centers

Feasibility Study – Next Steps

1. Get buy in and commitment from major stakeholders

2. Determine focus, identify key partners, select projects

3. Learn from “best practices”

4. Further develop business plan (revenue model, financial

requirements, organization, team)

5. Apply for grants, raise capital investments

6. Build team

3Q 4Q 1Q 2Q 3Q

20152014

preparation phase financing phase

Go/NoGo

AccelerateProject

XProject

Y

Start

Conclusions

Great need to organize pharma R&D in a different way

Academia hold the key to bridge the innovation gap

between exploratory research & early clinical development

Merge the strengths of academic & industrial drug research

The COIDD will place RU/Radboudumc at forefront of

translational medicine

Successful & sustainable precedents of COIDD exist but

The Netherlands are falling behind … and we are

determined to change this!

Initiators

Prof. Floris Rutjes

Dr. Thea van Kemenade

Prof. Alain van Gool

Dr. Pedro Hermkens

Dr. Jac Wijkmans