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,…
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
Early-Stage Gap
Example Drug Development
“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
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
Late-Stage Gap
Example Drug Development
“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
Case Study: TRC Fund
• 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
Case Study: TRC Fund
• 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$$
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
Mid-Stage Gap
Example Drug Development
“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?