Personalized Medicine/ Diagnostics/ Chemical-Complex Ecosystems Produce Various Strategies Connecting IP with Products and Services

AIPLA Mid-Winter InstituteJanuary 31, 2013

Natalie Wright Curley, JDManaging Director,Office of Technology Commercialization

MD ANDERSON BACKGROUND

• Established in 1941 by the Texas State Legislature and matching funds from Monroe Dunaway Anderson.

• One of the nation’s original three comprehensive cancer centers designated by the National Cancer Act of 1971.

• One of 41 National Cancer Institute-designated comprehensive cancer centers .

• In 2011, MD Anderson marked its 70-year anniversary and welcomed Ronald DePinho, M.D., as the fourth full-time president in the institution's history.

• Ranked as the best hospital in the nation for cancer care 8 out of the past 10 years by US News and World Report.

• Currently employs more than 19,000 people, including 1,600 faculty.

Our Mission

To eliminate cancer in Texas, the nation and the world through outstanding programs that integrate patient care, research and prevention, and through education for undergraduate and graduate students, trainees, professionals, employees and the public.

Patient Care • FY 2012*115,000 patients seen, nearly 1/3 new patients

Hospital admissions 26,726

Hospital patient days 191,735

Average number of operating beds 616

Outpatient clinic visits, treatments and procedures 1,281,489

Pathology and laboratory medicine procedures 11,619,591

Diagnostic imaging procedures 497,660

Surgery hours 66,241

Active clinical research protocols 1,078

MD Anderson Locations

MD Anderson Radiation Treatment Center in Istanbul at American Hospital (Turkey)

Centro Oncológico MD Anderson International España (Madrid, Spain)

MD Anderson Radiation Treatment Center at

Presbyterian Kaseman Hospital (Albuquerque,

NM)

MD Anderson Cancer Center-Orlando (FL)

Banner MD Anderson Cancer Center (Gilbert, AZ)

MD Anderson Cancer Center Main Campus,

Houston, TX

TEXAS

• In addition to MD Anderson’s main campus in the Texas Medical Center in Houston, multiple regional care centers: Regional Care Center in the Bay Area

(Nassau Bay)

Regional Care Center in Katy

Regional Care Center in Sugar Land

Regional Care Center in The Woodlands• Two research campuses in Bastrop County,

Texas

Research funding • FY 2011Federal grants and contracts $196,753,104

Private industry grants and contracts $68,413,794

Philanthropy and foundations $100,794,491

Internal funding $241,560,330

State funding $40,019,178

Total research funding $647,540,897

Of MD Anderson’s total revenue of $3.7 billionin FY12, only 4.6% was general revenue appropriated by the State of Texas.

Sources of Revenue (in millions)

Net Patient Revenue

Auxillary income

Other Income

Investment and other non-operating income

State-appropriated general revenue

Restricted grants and contracts, philanthropy

• Obtain invention disclosures from faculty;• Evaluate for patentability and commercial

potential;• Oversee patent drafting and prosecution;• Out license discoveries for commercial

development and sale;• Create new companies to commercialize

university technologies;• Goal: generate revenue to contribute back to

mission!

Office of Technology Commercialization

• Patient demand;• Shorter time to market (especially compared to

those difficult therapeutic approvals!!!);• Better technologies at cheaper costs;• Money easy to get;• Patient samples available.Personalized Medicine!

As a result, we saw a surge in “diagnostic discoveries.”

Diagnostics on the Scene!

Before 2010

Therapeutics

Diagnostics and Devices

Other

After 2010

Therapeutics

Diagnostics and Devices

Other

Invention Disclosures (average 134/year)

There is a need, BUT….

• Markers are changing and under debate.  • If new/better biomarkers are identified, how can these be validated and

included on an existing panel? Cost? Regulatory approval?• Tests often stratified by disease type; diagnostic; prognostic; predictive…

not a one size fits all market.• Regulatory uncertainty—not clear what the FDA will require.• Patentability issue…Supreme Court ruling on Prometheus has cast a

cloud over the patentability of diagnostics in general; Myriad to be heard by SC.

• Potential infringement of previously issued patents/FTO.• Reimbursement not certain.• Indicated course of treatment may still not be clear.• Funding is difficult.

Hmmm….Diagnostic “Market” Issues

CURRENT PRACTICEPatient treated based on original biopsy

CHALLENGEPatient outcome is determined by metastatic tumor

Does the primary tumor represent metastatic or recurrent tumor?

Are there subclones in the primary tumor?Are they selected by the metastatic process?Does it matter?

Diagnostic “Technology” Issues

Assay Loss Gain DiscordancePTEN 10% (5/46) 15% (8/46) 25%PIK3CA 8% (4/21) 8% (4/21) 16% marked change 8% (4/21) 12% (6/21) 20%ER (38) 7% (4/38) 0% (0/38) 7%PR 12% (7/38) 3% (2/38) 15%HER2 2% (1/12) 2% (1/12) 4%

*n=51 tumors and 56 metastasesAna Gonzalez; Funda Meric; Kat Hale

Heterogeneity and Molecular Evolution

Primary Metastasis

Breast Cancer

PIK3CA (Exon 9/20 only)

48% discordance between primary and metastasis

Example

Primary WT

Metastasis #1 H1047R

Metastasis #2 E542K

Deep sequencing of primary tumor may identify mutations

HETEROGENEITY: DO DIFFERENT SUBLCONES METASTASIZE TO DIFFERENT SITES

Jensen et al. 2011 E542K

WHAT CLONE FREQUENCY MATTERS FOR PATIENT OUTCOMES????

Drivers and passengers: Specific mutation mattersFunctional genomics program

• How do we know which mutations are functional drivers and which ones are noise? (More of a problem as more genes are examined and we expand to whole exome sequencing.)

• Current offerings only tell us which genes are mutated, not which ones are functional.

Functionality

HOW CAN WE DEVELOP A MODEL THAT ADDRESSES THESE

ISSUES?????• Panels;• Functional information;• Better biopsy technologies;• Improved certainty from regulatory and legal/PTO landscape;• Reimbursement criteria/economics.

Opportunities