Commercializing Innovations at Georgetown University 4/21/2017 Commercializing Innovations at Georgetown University Ruchika Nijhara, Ph.D., MBA, CLP Director Technology Commercialization Georgetown University 3300 Whitehaven Street, NW Harris Building, Suite 1500 Washington, DC 20007 rn86@georgetown.edu HP_presentation_template

Emerging realities in the pharmaceutical industry Rapid escalation of the costs of R&D Emptying product pipelines Global tightening of regulatory policy Patent expirations on most of the blockbuster drugs Challenges to IP by generic companies

Prevailing Wisdom Academia is the leading source of innovative assets. However, many of such assets are under-valued and under-resourced. A huge development gap exists between publicly financed basic research and privately financed clinical development.

Development Gap- One of THE Limiting Factor in Technology Transfer Why does the Gap Exists? Output From Academia Industry’s Requirements Early-stage IP Validated IP Unclear Market Potential Clear Market Potential Incomplete Technology Validation Validated Technology Unproven, High Risk Assets Proven, Low Risk Assets LACK OF RESOURCES (Infrastructure, Expertise and Capital) Development Gap- One of THE Limiting Factor in Technology Transfer

Traditional Models for Technology Maturation in an Academia License to Tech Development Companies Sponsored research using Corporate partner funds Create a Start-up However, financing early stage research is becoming a major challenge

Georgetown University’s strategies to bridge the gap and facilitate commercialization of its innovations

Georgetown University Georgetown College Edmund A. Walsh School of Foreign Service Robert Emmett McDonough School of Business School of Nursing and Health Studies Graduate School of Arts and Sciences School of Medicine Law Center School of Continuing Studies

Hospital, a Medical Center, and a NCI Cancer Center. Technology and Its Development. It’s All Here @ Georgetown University. Georgetown University Medical Center and Medstar Health have a clinical partnership where MedStar Health runs the Georgetown University's clinical enterprise, and Georgetown University runs the education and research enterprises-which include the School of Medicine, the School of Nursing and Health Studies, the Lombardi Comprehensive Cancer Center and the Biomedical Graduate Research Organization. These units continue to be known collectively as Georgetown University Medical Center (GUMC). 1470 Faculty Centered in cura personalis – care for the whole person. http://gumc.georgetown.edu/ The Lombardi Comprehensive Cancer Center is 1 of 39 National Cancer Institute-designated Comprehensive Cancer Center, national leaders in cancer treatment, research and education. http://lombardi.georgetown.edu/

Our Commercial Successes Whole-body CT Scanner T-Wave Alternans Allegra® HPV Vaccine HPV Diagnostic

Development of research into products that benefit the public Our Office’s mission and Obligations Development of research into products that benefit the public Engagement of Georgetown University in the founding & growth of local businesses (economic development) Recruit, reward, and retain faculty A Service to the University, a Partner with the Faculty, and a Business Generating Revenue

Our Office’s mission and Obligations Sometimes misaligned with industry objectives Revenue generation is important, but not primary Creating and communicating knowledge Providing education Well-being of humankind University and federal (Bayh-Dole) policies are important

Responsibilities of the Office of Technology Commercialization Receive Invention Disclosures. Evaluate disclosures. Initiate and follow-up on appropriate type of protection. Market inventions. Negotiate License Agreements. Monitor patent filings and licenses Handle other agreements: Material Transfer Agreements, CDAs, Collaborative Research Agreements IP in Sponsored Research Agreements Inter-institutional Agreements Work with developing new (start-up) companies Outreach/education/local commercial development

Key Considerations in Georgetown’s Licenses Scope of Licensed Technology Economics Assignment Rights Sublicensing Rights Effect of Termination on Sublicenses Diligence Obligations Termination Rights

Economics Typical forms of Consideration Reimbursement of Patent Costs Equity (where University policies permit) Milestones Pre-commercialization Post-commercialization Sublicense Revenue Sharing Royalties Basis of Royalties Scaling of Royalties with Sales Stacking Combination Products

Flexibilities Open to granting non-exclusive know-how license, or other arrangements if work is on-going E.g., Sponsored research or consulting services if work is on-going at the University

Key Elements of Exemplary Practice Appropriately, qualitatively and quantitatively staffed office. Customer-Friendly Orientation-e.g. Efficiency, rapid turnaround Clear Policies and Procedures- e.g. flexible consulting and COI policies, Transparency Supportive University Culture- e.g Tech transfer as a key element of social mission.

Established Infrastructure and Allocated Resources for Translational Research Center for Drug Discovery - The center uses interdisciplinary approach to design, synthesize and test potential drug candidates against novel molecular targets. Access to large, diverse, and growing patient population for clinical trials through clinical partnership with MedStar Clinical Investigators – Provide key translational strategies to demonstrate early proof-of-concept studies in patients.

Center for Drug Discovery Identifies and synthesizes candidate drug compounds for selected molecular targets Validates targets/ compounds in cell models and pre-clinical studies, and Provide supporting experiments, large scale synthesis and consultation for each candidate molecule for submission of an investigational new drug (IND) application

Drug Discovery Infrastructure Capacity 30 projects CHEMICAL BIOLOGY CONSORTIUM IND enabling studies Embedded Model Virtual Screening Medicinal Chemistry Protein Assays Cell Models Preclinical ADME/ Tox Drug Discovery Infrastructure Research Pipeline Lombardi Basic Science Phase 0/ I Clinical Studies Phase II/III Clinical Studies Licenses New companies CRO/Partnerships

Center for Drug Discovery at Georgetown Prioritized Georgetown University Medical Center candidate molecular targets for drug discovery and development Advanced 14 potential drugs through preclinical studies Received National recognition Patents Publications Grants 1 of 11 sites selected for NCI chemical biology consortium International recognition Strategic partnerships in China, Brazil and other countries CDD cultivates and expands translational medicine at GUMC. Developed partnerships with biotechnology companies seeking funding through the Small Business Innovation Research (or SBIR) program. Several multi-investigator collaborations have been developedwith biotechnology companies to advance the discovery and development of novel medicines for the next generation.

RESEARCH PIPELINE EARCH PIPELINE

Criteria for Selecting Projects Potential Project X Criteria (filter) Target validation(justification) siRNA Immunohistochemistry Knockout Mechanistic studies Project Declined or Triaged CDD Assess Project Needs Validating studies Structural models Chemistry Biology Preclinical Models Imaging ADME Virtual Screening Medicinal Protein Assays Cell Models Preclinical Models/Tox Scale-up

Center for Drug Discovery at Georgetown Prioritized Georgetown University Medical Center candidate molecular targets for drug discovery and development Advanced 14 potential drugs through preclinical studies Received National recognition Patents Publications Grants 1 of 11 sites selected for NCI chemical biology consortium International recognition Strategic partnerships in China, Brazil and other countries CDD cultivates and expands translational medicine at GUMC. Developed partnerships with biotechnology companies seeking funding through the Small Business Innovation Research (or SBIR) program. Several multi-investigator collaborations have been developedwith biotechnology companies to advance the discovery and development of novel medicines for the next generation.

Other Resources at Georgetown University Know-How/Assays Technologies developed at Georgetown Research tools and materials Animal models Center for Cellular Reprograming Access to patients and clinicians for clinical trials

Georgetown University’s strategies for value creation Alumni “expert” network Accelerator to mature early stage technologies EIR program with McDonough School of Business Using Interns Strategic research/clinical collaborations with Industry Entrepreneurs 25

Questions? Visit us at http://otc.georgetown.edu | Follow @GUOTC Ruchika Nijhara, Ph.D., MBA, CLP Director Technology Commercialization Georgetown University 3300 Whitehaven Street, NW Harris Building, Suite 1500 Washington, DC 20007 rn86@georgetown.edu