Academic Clinical Trial Do’s & Don’ts for Partnering with Pharma

1. Academic Clinical Trial Do’s & Don’ts for Partnering with Pharma CCAF San Diego, CA April 14, 2014 J. Eric Bubbers, Ph.D.

2. Bona Fides • 20 years experience in pharma industry R&D, Marketing and Business Development • 15 years experience in academic laboratory basic science and human clinical trial research • Multiple industry/academia licensing, acquisition, and R&D agreements negotiated and implemented from both sides of the table. 4/14/14

3. Therapeutic Advances in Childhood Leukemia & Lymphoma Clinical Research Consortium Founded in 2005 to: “Develop Innovative Therapies for Currently Incurable Leukemia and Lymphoma.” CHLA Operating Center Academic Member Institutions TACL Drug Development Companies Community & Philanthropic Organizations 4/14/14

4. TACL Accomplishments Key Achievements • >160 patients have been enrolled and treated on 14 therapeutic clinical research studies since 2006 • All Investigator Initiated Studies • 5 studies supported by pharma partner with study drug • 6 studies supported by pharma partner with drug and funding • Completed 10 pediatric oncology clinical study projects • Only database in the world with 10 years of clinical history and therapeutic outcomes data on over 400 childhood leukemia patients • Currently have 5 ongoing clinical studies • 2 studies under FDA INDs in partnership with pharmaceutical companies • In the last 4 years, >$4 million in funding support; ~ 50% committed from industry sources 4/14/14

5. TACL Value Proposition • Perform trials of new drugs that would otherwise not be studied in pediatric patients • Novel therapies focused on childhood leukemia & lymphoma • Early stage (“first in kids”) clinical studies of relapsed disease • Emphasis on correlative biology studies • Clinical research cooperative of 35 academic institutions in 3 countries • Responsive and nimble; speed to action; low bureaucratic barriers • Centralized operations, administrative, regulatory (CHLA) • Industry, community, philanthropic and family partners • Supportive of, complementary to, Children’s Oncology Group (COG) • Ongoing clinical study collaborations with ITCC in Europe 4/14/14

6. Barriers to Success for Investigator Initiated Clinical Studies Key Steps Along the Way • Finding a Partner • Engaging a Partner • Negotiating with a Partner • Managing a Partner • Ultimate Goal: Successful Partnership Exit (a.k.a. “Happy Ending”) 4/14/14

7. What Pharma Is Good At Hint – It’s Not Early Stage Investigator Initiated Clinical Studies • Drug development and commercialization • Manufacturing facility and process development • Product distribution, marketing & sales • Negotiating and contract management • Project and vendor management • Risk management • Making money 4/14/14

8. What Pharma Is Bad At Or - Why They Need You • Basic research • Disease mechanisms and processes • Treating niche patient populations • Early stage clinical study design • Managing academic collaborations • Taking risks 4/14/14

9. Key Value Drivers for You and Your Pharma Partner What You Can Do for Them • Understand how their drug can best be used therapeutically in your patients • Enable access to world class thought leaders and expertise • Begin establishing a market presence for their drug • At arms length, and relatively inexpensively, manage the high risk of early stage clinical studies • Even if your hypothesis turns out to be incorrect, your partner benefits by avoiding later stage, more expensive clinical studies What They Can Do for You • Investigational drug or device • Funding • Regulatory support • Assay & technical support 4/14/14

10. How to Find & Engage a Pharma Partner Where to Look • Know the class of drug you are interested in testing and search the web for companies developing drugs in that class • Don’t get married to one agent, identify and pursue multiple candidates preferably both marketed and in development • Search literature for company scientists publishing on compounds of interest & establish communication • Find target companies at symposia you are attending, make contact with corporate speakers (business cards are old school and effective), establish communication & follow up • Visit company booths at trade shows; track down your city’s Medical Science Liaison (MSL) • Visit companies web sites and look for IIS pages – least effective but most efficient 4/14/14

11. Negotiating What You Need to Conclude a Successful Agreement • Persistence • A novel and compelling scientific rationale for testing whether a drug is therapeutically beneficial in a particular patient population. • DATA IS KING! • A hypothesis and study design concept that can be successfully and expediently executed in a human clinical trial • A realistic, fact-based budget that covers all the study costs • An experienced academic Research Administration, CTSU / CTO and/or clinical trial consortium (if multi-site study) • Understand the company’s point of view and know how you can add value for them • Know what your walking away point is – be prepared to say no • Persistence 4/14/14

12. Managing the Relationship What to Do After the Deal is Done • Say what you are going to do and then do it (Surprises are bad) • File required reports in a timely and complete manner • If study not going according to plan, know why and communicate revised plan • Be able to account for any expended funds • Be available to speak live with your pharma partner; relationships matter 4/14/14

13. Summary • Finding a partner is a numbers game – the more potential partners you pursue the higher the chance you will be successful • Understand how you can create value for your partner while simultaneously achieving your academic research goals • Companies HATE risk • Have a plan that is achievable with the infrastructure in place to make it happen • Don’t underestimate or undervalue what you have to offer your partner • Be willing to share data and intellectual property under the right terms • Be prepared to walk away from an opportunity if the terms are not sufficient to achieve your projects goals • No deal is better than a bad deal 4/14/14