innovative technology transfer: supporting a strategic delay robert c. bast, jr., m.d. vice...

Innovative Technology Transfer: Supporting a Strategic Delay

Robert C. Bast, Jr., M.D.

Vice President for Translational Research

University of Texas M.D. Anderson Cancer Center

Supporting A Strategic Delay

• In Biomedical Science and Health Care, the Value of new Inventions increases Dramatically if they proceed to Successful Phase I Human Trials before Licensing

• Resources, Champions and Processes must be found to support Early and Mid-Stage Development, Particularly in Today’s Environment where Licensing can be Difficult

• Does JSC need this Capability?

Discovery Drug Development

PreclinicalDevelopment

Clinical Phase I

Phase II Phase III

RegulatoryApprovalTarget

LeadIdentification

Lead Optimization

Market

“GAP 1”Researchfunding

Industryfunding

“GAP 2”

Early-stage development

License or Startup

Funding Gaps

Example Drug Development

“GAP 3”

Definition

• The Funding Gap or The Valley of Death:– Government tends to fund basic research and

a technology may have passed that stage and be too “applied” for further funding

– The private sector will not yet pick up technology because it is too risky (has not been fully “applied” yet)

The Valley Of Death

Valley of Death

Existing Commercialization Resources

Existing Research Resources

Idea Research Fuzzy Front End Product Development Commercialization

From: The PDMA ToolBook 1 for New Product Development

The Valley Of Death

• What are critical needs to bridge the gap– Critical resources

• Money• Expertise• Equipment: i.e. tooling, production, validation, computing,…

– Champions• Supportive experts

– Formal process and pathways• For example protection of IP, regulatory expertise,

standardized processes for development and commercialization,…



Clinical Phase I

Phase II Phase III


LeadIdentification

Lead Optimization

Market


Industryfunding

“GAP 2”


License or Startup

Early-Stage Gap


“GAP 3”

Early-Stage Gap Drugs

• Early gap is typically between promising first cell line and animal test results and further development of compound of interest– Optimization of compound (chemistry)– Lead compound selection– Formulation and pharmacokinetics– Dose ranging– Pre-IND (rodent) toxicology study– Pre-IND FDA call

• Cost range: $50K to $300K

Early-Stage Gap Devices

• Early gap is typically between first idea and concept and creation of functional prototype– Prototype development– Preparation regulatory documentation

Cost range: $50K to $1,000,000

Mid-Stage Gap Drugs

• Mid stage gap is typically between pre-IND and conclusion of a Phase I trial– Second species toxicology study– IND preparation– Quality controls– Manufacturing drug for Phase I trial – Possibly Phase I trial– Costs to license a technology from an University

(license costs and legal fees)

• Cost range: $300K to $3 million

Mid-Stage Gap Devices

• Mid stage gap is typically between prototype development and validation of the device/technology– Validation of function– Preliminary studies with animals or humans (samples)– Regulatory documentation

• Cost range: $100K to $ 2 million



Clinical Phase I

Phase II Phase III


LeadIdentification

Lead Optimization

Market


Industryfunding

“GAP 2”


License or Startup

Late-Stage Gap


“GAP 3”

Late-Stage Gap Drugs

• Typically money to run Phase 2 and 3 clinical trials and comply with all FDA reporting requirements

• Cost range: tens to hundreds of millions• Very complex• Mostly financed by professional investors and

industry

Late-Stage Gap Devices

• Finalizing FDA submission for device clearance • Clinical studies for PreMarket Approvals

(PMA’s) if needed• Set up of manufacturing process• Scale-up manufacturing• Going to market after FDA clearance• Cost range: can be very expensive

Value Increase of Drugs

University Funding Models

• Many universities and academic institutions have launched pre-seed and seed funds to bridge early-stage gaps

• Various models can be found– Managed by universities– Managed by investors– Combined models

Case Study: TRC Fund

• Gap funding by The University of Texas MD Anderson Cancer Center

• TRC = Technology Review Committee• TRC fund managed by Office of Technology

Discovery (Translational Research)• Money comes from philanthropic sources and

operational clinical margin


• Amounts invested: $10K to $250K• Requirements

– Invention Disclosure filed– Has to support hospital’s mission– Has to have commercial potential– Has to be scientifically sound

• Technology Review committee a mix of faculty and external experts – Venture capitalists, lawyers, CEOs biotech


• Inventor has to prepare– Funding request with

• Brief technology and IP summary• Milestones• Timelines• Budgets • 4 pages about technology background, scientific

data, pictures of design or prototype design, description of software, …

– Presentation to TRC committee

Project Flow (7 years)

1532 formal faculty contacts

81 project teams formed

51 projects presented

33 projects funded

$ 2,134,913

Project Flow

14

6

5

8

Aerosolized Lung Immune StimulantDr. Burton Dickey

• New drug to prevent pneumonia in immuno-compromised cancer patients

• Prevent pandemic pneumonia (influenza, SARS, H1N1, etc.)

• Prevent and treat bioterror pneumonia– Plague, anthrax, others

• Startup company “Pulmotect” formed

Aerosolized Lung Immune Stimulant

Aerosolized Lung Immune Stimulant

• Project financed with $ 75,753• Drug under development• Value for MDACC:

– New IP generated– Licensed to startup company Pulmotect

• $ 200K in-kind financing to start-up via AlphaDev

– Grants: total $ 2,835,000• $ 25K (Lehman Brothers Foundation)• $ 10K (Texas Ignition Fund TIF)• $ 50K (NCI Spore)• $ 200K (Living Legends Fund)• $ 300K (Charles Culpeper Award)• $ 250K Texas Emerging Technology Fund (ETF)• $ 2 million (National Institute of Health NIH)

– Several journal publications and abstracts

MRI Visible SeedsDr. Steven Frank

• MRI is superior imaging technology for prostate than ultrasound and CT

• No MRI visible markers available

• Development of MRI marker

• Development of MRI visible brachytherapy seeds for treatment of prostate cancer

MRI Visible Seeds

Seeds visible but unclear anatomy

Seeds visible but CT artifacts

Clear anatomy but no visible seeds

MRI Visible Seeds

MRI Visible Seeds

MRI visible seed

MRI Visible Seeds• Project financed with $ 148,000

• Marker and MRI visible seeds developed

• Value for MDACC:– New IP generated

– Not yet licensed (still underdevelopment)

– Grants: total $ 280,000• $ 225K Frank (Prostate Cancer Foundation)

• $ 55K Frank (Texas Ignition Fund TIF)

– Several journal publications and abstracts

– $2 million external investments for Start-up

Value Creation TRC Projects

2003 to date

License Income (license, option, fees) $ 1,361,470

SBIR’s, STTR’s $ 240,000

Sponsored Research Agreements (SRAs) $ 250,000

Research grants $ 4,773,000

Money saved by MDACC $ 1,322,000

Outside investments $ 4,530,000

Total Value Creation $ 12,476,470 (6XROI)

Other TRC Benefits

New Invention Disclosures as result of funding 14

Patents filed as result of funding 9

Licenses/Options signed 10/4

Startup companies formed / in formation 5/4

Entrepreneurship courses organized and hosted 5

Publications (incl. abstracts) 57

Raised awareness for technology transfer and entrepreneurship Pricele$$



Clinical Phase I

Phase II Phase III


LeadIdentification

Lead Optimization

Market


Industryfunding

“GAP 2”


License or Startup

Mid-Stage Gap


“GAP 3”

Supporting A Strategic Delay

• Does JSC need Additional In-House Capability to develop Inventions Prior to Licensing?

• Can the TMC help to identify Unmet Medical Needs that could be met with NASA Technology?

• Might the TMC Partner with JSC to develop Biomedical Devices and Processes prior to Licensing to the Private Sector?

innovative technology transfer: supporting a strategic delay robert c. bast, jr., m.d. vice...

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