IAMI Meeting Booklet

The Institute forAdvancing Medical Innovation

2011 Meeting of

the Advisory Board

3

I.Meeting Agenda .................................................................5

II.Advisory Board Letter ....................................................7

III.Investment Memos ..............................................................9

Part A, Berkland-O’Brien-Ladner-Sestak ..........................................9Part B, Richter-Wu ....................................................................... 31Part C, Detamore-Berkland-Mohan ..............................................53Part D, Friis-Arnold ......................................................................73Part E, Kieweg-Kim ...................................................................... 97Part F, Desaire-Toumi ................................................................ 119Part G, Roby-Tash-Georg ..........................................................135

IV.Highlighted Collaborations & Partnerships .......................153

V.KU Endowment Presentation ............................................ 159

VI.Meeting Dates 2011-2012 ................................................ 165

VII.Appendix .........................................................................167

Part 1A. Francis S. Collins, MD, PhD, Letter ................................ 167Part 1B. NIH Letter ................................................................... 169Part 2A. KUCTC Technology Transfer & Commercialization Faculty Survey Executive Summary ................................. 173Part 2B. 2011 KUCTC Faculty survey result .................................. 175Part 3. Website Example ..........................................................193Part 4A. Comparative Analysis ................................................... 195Part 4B. Comparison Charts ...................................................... 197

VIII.Note Pages ............................................................... 201

Table of ContentsTable of Contents

5

I. Meeting AgendaI. Meeting Agenda

THE INSTITUTE FOR ADVANCING MEDICAL INNOVATIONMeeting of the Advisory Board

SEPTEMBER 16, 20118:00 A.M. – 3:15 P.M.

CONFERENCE ROOM 307, KU ENDOWMENT4125 RAINBOW BOULEVARD, KANSAS CITY, KANSAS

8:00 – 8:30 A.M.

BREAKFAST & WELCOME Scott Weir, Director of the Institute

8:30 – 10:30 A.M.

INVESTMENT MEMOS

a. Novel Polymer Therapeutics for Iron Chelation TherapyAmy O’Brien-Ladner, M.D. and Joshua Sestak, Ph.D.

b. Development of Clinically Diagnostic Biosensors for Virus DetectionMark Richter, Ph.D. and Judy Wu, Ph.D.

c. Gradient Scaffolds for Regeneration of Osteochondral Defects in a Large Animal ModelNeethu Mohan, Ph.D.

d. Tough Piezoelectric Composite Biomaterial and Spinal Fusion Implant DesignLisa Friis, Ph.D.

10:30 A.M. – 10:40 A.M.

BREAK

10:40 A.M. – 12:10 P.M.

INVESTMENT MEMOSe. Automated Vitrification Device (2nd Generation) and Closed Storage System

Sarah L. Kieweg, Ph.D. and Sam Kim, M.D.f. Protein Production Technology

Heather Desaire, Ph.D. and Melinda Toumi, Ph.D.g. Non-Surgical Sterilant in Female and Male Animals

Katherine Roby, Ph.D. and Joseph Tash, Ph.D.

12:10 P.M. – 12:25 P.M.

BREAK

12:25 P.M. – 1:30 P.M.

WORKING LUNCH – PROJECT DISCUSSION AND DECISIONS

6

1:30 P.M. – 1:50 P.M.

HIGHLIGHTED COLLABORATIONS AND PARTNERSHIPSThe Learning Collaborative

Scott WeirSilvergate Pharmaceuticals

Mike Baltezor

1:50 P.M. – 2:10 P.M.

FINANCIAL UPDATE – LENGTHENING THE RUNWAY Scott Weir

2:10 P.M. – 2:30 P.M.

KU ENDOWMENT PRESENTATION Stephanie Grinage, Vice President for Medical DevelopmentStephanie Carani, Associate Development Director - IAMI

KU Endowment

2:30 P.M. – 3:00 P.M.

EXECUTIVE SESSION Advisory Board Members

3:00 P.M. – 3:15 P.M.

WRAP UP AND NEXT STEPS Scott Weir

Agenda, con’tAgenda, con’t

7

II. Advisory Board LetterII. Advisory Board Letter

30 August 2011

Dear IAMI Advisory Board:

I hope this letter finds you and your families well, and I very much look forward to our upcoming Advisory Board meeting on September 15-16, 2011.

Since we last met, IAMI and the University have accomplished a great deal. A few highlighted achievements related to the mission of IAMI, are as follows:• The Learning Collaborative (TLC) is a unique collaboration between IAMI, the Therapeutics for

Rare and Neglected Diseases Program within NIH, and The Leukemia and Lymphoma Society (LLS). TLC has executed a Cooperative Research and Development Agreement (CRADA) to advance Auranofin, to clinical proof of concept. The CRADA transfers Auranofin development and commercialization responsibilities for the NIH discovery, jointly to KU and LLS, which represents perhaps the first such agreement signed between the NIH and non-profit partners. Additionally, we have received clearance from the FDA to proceed with the clinical proof of concept trial, which will be conducted at KU, The Ohio State University, and the National Heart, Lung and Blood Institute.

• The pediatric, oral, liquid formulation of enalapril, developed in collaboration with Children’s Mercy Hospital and the Institute for Pediatric Innovation, has been licensed to Silvergate Pharmaceuticals. Formal development activities are well underway with a regulatory submission planned for 2012. We are currently negotiating licenses for 6-mercaptopurine and amlodipine pediatric, oral, liquid drug products with Silvergate, as well.

• The University of Kansas became one of 60 members of the Clinical and Translational Science (CTS) centers consortium, funded by the NIH. IAMI, presenting the Novel Methods program, was noted during peer review as a unique capability that our program brings to the CTS centers consortium, which played a key role in securing this $20 million, five-year grant

• IAMI’s success and growing reputation in drug repurposing and rediscovery continues to be recognized nationally. We were one of several academic centers, asked to participate in the NIH-Industry Roundtable – Exploring New Uses for Approved and Abandoned Therapeutics in April, 2011, led by NIH Director Collins. The NIH brought senior R&D leaders from many major pharmaceutical companies together, in hopes of partnering and exploring therapeutic applications of approved and abandoned drugs for rare and neglected diseases. IAMI contributed to specific recommendations created during the two-day meeting, which were developed into formal initiatives.

Enclosed you will find investment memos detailing seven KU faculty research projects which are under consideration for IAMI proof of concept funding. We seek your input and guidance on the scientific and commercial merits of these projects, and whether they warrant IAMI investment.

In addition to faculty project discussions on September 16, we will provide you with brief updates (via presentation) on IAMI organization, strategic partnerships, current and projected financial outlook, and fund-raising strategy. Background materials on these later topics will be provided when you arrive at the meeting. 777

8

We have adequately addressed three of the five improvement areas identified during our February 2011, Advisory Board meeting. They are: focus the mission of IAMI, improve the quality of faculty research proposals and the review process, and define a clear message for fundraising. Work continues on the two remaining areas of improvement: creating a supportive and innovative culture at KU, and, effectively managing previously funded projects for commercial outcomes. We fully intend to make substantial progress in addressing these areas following the September meeting.

Focus on the Mission of IAMI. The focus of IAMI is two-fold: 1) develop KU’s translational research capabilities to achieve bench-to-bedside outcomes, through effective collaborations with industry, academia, government and disease philanthropy organizations, and, 2) to assist KU researchers in transforming basic research into medical innovations, and advancing those technologies to commercial outcomes.

Improve Faculty Research Proposals and the Review Process. Under the leadership of Julie Goonewardene, we have established the Investment Memo process. Investment memos have been prepared on seven projects, which are enclosed. This process has significantly improved the quality of the commercial assessments and commercialization strategies for KU medical innovations. We are building a network of consultants as well, who will provide domain expertise assessments of proposals prior to review by the Advisory Board. One of these consultants, Becky Voorheis, who helped us prepare the seven investment memos enclosed, will participate in our meeting on September 16.

Clear Message for Fundraising. Our fundraising message is strategically aligned with the IAMI mission. In other words, we are focused on two related but distinct strategies, 1) to raise funding in direct support of IAMI’s translational research efforts, and 2) to build a sustainable proof of concept fund, which will be invested in KU researcher medical innovations.

In closing, we very much appreciate your leadership and contributions in establishing IAMI as one of the most successful, if not the most successful, academic proof of concept center in the country.

Sincerely,

Scott J. Weir, Pharm.D., Ph.D.Director, Institute for Advancing Medical Innovation

Letter, con’tLetter, con’t

9

III. Investment Memos, Part AIII. Investment Memos, Part A

KU CONFIDENTIAL

Faculty: Dr. Cory BerklandProject Director: Tyce BrunsDate of Report: August, 2011p g ,

1

Investment Thesis

KU CONFIDENTIAL

Technology OverviewMarket OverviewFinancial AnalysisFit with IAMIStaff Recommendation to the IAMI Board

2

A. BERKLAND O’BRIEN-LADNER SESTAK

999

10

Iron chelating therapeutics represent a drastically underserved medical market. FDA approved therapeutics are marginally effective, suffer from

KU CONFIDENTIAL

significant side effects that limit dose, and require frequent hospital visits for long infusions.

Inconvenience of in-clinic and frequent administration contribute to a 25%+ non-compliance rate among patients.

The current market is ~$1.1B, growing >4% through 2020.

The research team plans to develop 2 safe and effective iron overloadThe research team plans to develop 2 safe and effective iron overload therapies: 1) an IV-infusion alternative to the current sub-Q infusion therapeutic; and 2) a non-absorbed, oral therapeutic.

Clinicians and patients need a less-toxic iron chelator that binds to and removes iron from the body in fewer administrations with less side effects –precisely the product advantages that data indicates this technology will offer.

3

Scientific founders & key personnel• Dr. Cory Berkland, PhD (Associate Professor, Chemical & Petroleum

E i ee i a d Pha ace tical Che i t ) D Ch i tia Scho eichEngineering and Pharmaceutical Chemistry), Dr. Christian Schoneich, PhD (Professor and Chair, Pharmaceutical Chemistry), and Dr. Amy O’Brien-Ladner, MD (Professor, Pulmonary and Critical Care Medicine)

Amount of proposed investment (1Year = $100,000)

Milestones:• Synthesis of iron chelating polymers for oral delivery and injection

Dose ranging toxicity study• Dose ranging toxicity study• Determination of iron blood load reduction for novel polymers vs. the

gold standard

Out-licensing opportunity (not stand-alone company).

4

A. BERKLAND O’BRIEN-LADNER SESTAK, con’t

Investment Memos, part AInvestment Memos, part A

11

KU CONFIDENTIAL

5

Statement of technology

• Novel synthesized polymers which mimic the structure of a high affinity

KU CONFIDENTIAL

iron chelating siderophore produced by bacteria.

How novel is it?

• The side chain conjugated to the polymers proposed has not been described previously to the researchers’ knowledge.

What are its advantages over the current standard of care?

• Improved side effect profile and overall safety.

• More convenient – potential to increase compliance.

• The identified polymers exhibit extremely high iron binding affinity and better iron selectivity than the current gold standard, deferoxamine (DFO).

Iron chelating agents are used to treat iron overload – either hereditary or acquired. Iron is essential for life, but iron overload is toxic.

6

A. BERKLAND O’BRIEN-LADNER SESTAK, con’t

Investment Memos, part AInvestment Memos, part A

1