December 16, 2011

Technology Transfer Tactics Webinar:

How your university’s innovations can become a fixture in big pharma’s pipeline

Nurjana Bachman PhD - Business Development Manager

December 16, 2011

Technology Transfer Tactics Webinar:

How big pharma can help you develop innovative therapeutics for patients

Nurjana Bachman PhD - Business Development Manager

Drug identification and optimization

Manufacturing, Marketing and Sales

Clinical Development

One company performed all stages of drug development

Discovery Research, Target Identification

Preclinical Development

Then: Fully-Integrated Pharmaceutical Company

Drug identification and optimization

Manufacturing, Marketing and Sales

Clinical Development

Now: Fully-Integrated Pharmaceutical Network

One company partners to perform all stages of drug development

Discovery Research, Target Identification

Preclinical Development

Academia, other companies, CROs, manufacturing partners

New Pharma Model for Sourcing Ideas

Open Innovation

What does this mean for academia?

Children’s Ranked #1 in US

#1 ranked in:

• Heart and Heart Surgery

• Neurology and Neurosurgery

• Urology

• Orthopedics

• Kidney

• Cancer

#2 ranked in:

• Neonatology

• Diabetes and Endocrine Disorders

• Gastroenterology

#3 ranked in:

• Pulmonary

* US News & World Report, May 2011

World’s Largest Pediatric Research Enterprise

$225M clinical and basic research funding

800,000 sq. feet of research space

40 clinical departments

225 specialized clinical programs

~600,000 inpatient/outpatient visits

8,720 total employees

2 Nobel Prize winners

11 members, Howard Hughes Medical Institute

7 members, National Academy of Sciences

11 members, Institute of Medicine

History of Innovation at Children’s

1869 Children's Hospital Boston opens as a 20-bed facility

1920 Dr. William Ladd devises procedures for correcting various congenital defects

1922 Dr. James Gamble develops a method for IV feeding that saves the lives of thousands of infants at risk of dehydration

1938 Dr. Robert Gross performs the world's first successful surgery to correct a congenital cardiovascular defect

1954 Dr. John Enders wins the Nobel Prize for successfully culturing the polio virus

1971 Dr. Judah Folkman publishes "Tumor angiogenesis: therapeutic implications" in NEJM

1986 Dr. Louis Kunkel identifies the gene for Duchenne muscular dystrophy

1990 Dr. Joseph Murray wins the Nobel Prize for his pioneering work in organ transplantation

From Innovation…

1983 Children's physicians report the first surgical correction of hypoplastic left heart syndrome, a fatal condition.

1986 Dr. Louis Kunkel identifies the gene for Duchenne muscular dystrophy

1990 Dr. Joseph Murray wins the Nobel Prize for his pioneering work in organ transplantation

1998 Dr. Anthony Atala successfully transplants laboratory-grown bladders into dogs, a major advance in the growing field of tissue engineering.

2008 Dr. George Daley discovers how to reprogram human somatic cells to pluripotent stem cells with defined transcription factors.

2008 Dr. Chris Walsh and his colleagues identify several genetic loci associated with Autism

2008 Drs. Vijay Sankaran and Stuart Orkin discover the fetal hemoglobin to adult hemoglobin switch is controlled by BLC11A transcription factor.

Invention Types

Name Company

dmPGE2 (stem cell stimulation)

Von Willebrand Factor

Anti-Neuropilin Antibody

Tissue Engineered Bladder

Pomalidomide

Omegaven

FcRn-Factor VIII

…to Product…

Name Company

Revlimid

Thalidomide

Merge EchoIMS

Neumega

Namenda

Dystrophin Diagnostic

INF2 DNA Sequencing Test

CardioSeal, StarFlex, BioStar

Cannula Needle Set

Patient Communication Board

Plagio Cradle

Quickchange Mutagenesis Kit

Products on the Market Products in Clinical Trials

…and Company

That’s the Bayh-Dole story

So what’s new?

Our Assets

Investigators

Patient samples

Assays

Thought leadership

Patents

Access to alternative funding sources

Translational research infrastructure

Animal models

Expertise

George Daley MD PhD (HHMI)

Marsha Moses PhD

Clifford Woolf, MD, PhD

Christopher Walsh MD PhD (HHMI)

The Academic Medical Center’s Role in Drug Development

Discovery Research, Target Identification

Drug identification and optimization

Manufacturing, Marketing and Sales

Clinical Development

Preclinical Development

IndustryAcademia

Then: Academia a Source

Pharma

Biotech

Academic Medical Center

Foundation

CROVC

Start-up

Other academic Govt

Information, IP

Now: Academia a Development Partner

Pharma

Biotech

Academic Medical Center

Foundation

CROVC

Start-up

Other academic

Govt

New Integrated Functions in the Licensing Office

Business Development

Patents & Licensing

Technology Development

Clinical Trials

Children’s Technology Development Fund

Product

New Company

Post Inc.

Pre Inc.

Prototype Development

Proof of Concept Validation

Licensee

Clinical Trial Validation

Basic Research

TranslationalResearch

Pre-Inc.ŅGapÓ

Opportunity Development

TechnologyDevelopment

FundEach project mentored by

Board member

Technologies selected with

Advisory Board

$50k or $150k awarded for 1 year

product development project with CRO

Research, Target Lead

Manufacturing, Marketing and Sales

Clinical Development

Preclinical Development

The Academic Medical Center’s Role in Drug Development

Patient Treatment

NIH: National Center for Advancing Translational Sciences

Pharma programs: Pfizer, GSK, Eli Lilly, Roche, Novo Nordisk, Sanofi, UCB, Bayer, etc.

Foundations: CF Foundation, LLS, JDRF, Epilepsy Foundation, etc.

Academic programs: e.g. Internal Technology Development Funds

The Academic Medical Center: the new FIPCO?

No– Mission-driven, not profit driven- different incentive structure Multiple funding sources- primary funding from government Without partners, treatments won’t get to patient With more ability, more leverage, and ability to create a better

partnering opportunities Pharma “open innovation” lets us participate in new ways

But– How do we structure it so that it benefits both parties?

Collaboration Partnership Structures

Old way: Sponsored research/licensing: negotiated on a per project basis

New way- on a continuum: From…Company-defined grant programs: application based…

• Company defines access, terms and level of commitment

…To “Strategic Alliances” • Joint Steering Committee decision-making structure• Request for proposals across the institution• Joint project plan development

• Long term commitment

Key Aspects to Consider

Ownership Control (Joint decision-making) Commitment

• Time• Dollars • Really integrating projects into company pipelines• Commitment of internal resources• Ability to get treatment to the patient

Bi-directional knowledge-sharing

Best case scenario: • Specific resources offered • Commitment jointly defined and shared • Information and expertise shared • Value recognized through shared ownership of assets developed• Helps inform our future commercialization efforts

Pharma’s Open Innovation: Advantages to Academia*

Access to complementary expertise and new resources

Forms basis for ongoing communication and relationship

Streamlines negotiations (expectations set up front)

Informs ongoing commercialization efforts

Ability to influence drug development and facilitate getting treatment to patient

* Assumes fair deal structure

How to Become a Fixture in Pharma Pipelines

a.k.a. How pharma can help you develop innovative therapeutics

Understand the breadth of your assets

Understand partners’ needs, goals, resources and limitations

Understand your institution’s needs and limitations

Articulate these to the industry partner

Build long-term relationships

Communication and transparency!

This webinar is sponsored by Merrill DataSite – The Secure Virtual Data Room Solution for the Life Sciences Industry