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

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

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

4 >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 TACL Accomplishments Key Achievements 4/14/14

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

What Pharma Is Good At 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 Hint – It’s Not Early Stage Investigator Initiated Clinical Studies 4/14/14

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

Key Value Drivers for You and Your Pharma Partner 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 You Can Do for Them What They Can Do for You Investigational drug or device Funding Regulatory support Assay & technical support 4/14/14

10 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 How to Find & Engage a Pharma Partner Where to Look 4/14/14

11 Negotiating 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 What You Need to Conclude a Successful Agreement 4/14/14

Managing the Relationship 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 What to Do After the Deal is Done 4/14/14

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