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Bessor-Brightwaters Pharma New drug development and value creation: uniquely translating university assets to innovative products Joseph A. Boystak, Co-Chairman and Barry A. Berkowitz, PhD, Co-Chairman and CEO We are • Nimble, semi-virtual Pharma development company • At the center of a novel and aligned ecosystem that integrates key constituents • Highly experienced development and business team • Using accelerated development methods to bridge the “Valley of Death” • Optimizing returns relative to risks for our ecosystem stake-holders • Operational and very capital efficient • Developing a portfolio of innovative projects with early and multiple liquidity exits Ecosystem Industry Partners Academic Partners Bessor-Brightwaters Pharma Asset Evaluation and Selection Investors Advisors Foundations Asset Pools Project LLC’s Consolidators/ Acquirers Capital Markets The Problem Pharma/Big Biotech and Patients Pressing, unfulfilled need for innovative compounds Academia Foundations Wealth of projects, but not ready for pharma/big biotech • • Finance Our Solution Bessor-Brightwaters Pharma Early stage venture capital erratic Business models broken • • • • Capital efficient project development POC & pharma-ready drugs Attractive returns to all ecosystem stakeholders Top R & D team/Plus FIPNET Bessor–Brightwaters Pharma Solves Key Challenges • • • • • • • • University and foundation focus Capital efficient Translational, project based Flexible, responsive, integrated structure Efficient/standardized terms Pipeline to mitigate risk, optimize returns Aligned, committed top team Scalable RESULTS Pharma/Big Biotech Products • Flow of innovative new drugs • Multiple and earlier returns/ & types to our ecosystem stakeholders Innovative drugs for patients An Aligned and Fully Integrated and Pharmaceutical (FIPNET) Development Network Technology Experts • Chemistry (AMRI)/Pharmacology • Regulatory/Project Management/Toxicology • Drug Supply/Clinical Development Bessor- Brightwaters Pharma (Semi-virtual/efficient standardized terms) Yale •Launch, accelerate and add value to projects •Development know-how •Business development •Operations management •Fundraising /deals Texas A&M Pipeline Projects: Duke, Univ. of Fla., Univ. Manchester, Others Optimize Returns to Ecosystem Stakeholders Value Proposition: Foundations • Value to Foundations/Disease Societies – Translate projects of HIGH interest to Foundation and their patients to POC; Pharma ready packages; – Provide unique and accelerated means for important drugs to move from the lab “bench” to reach patients. • What? – Form focused teams optimized for results from each project--- with input from Foundation – Active role in selecting projects for Pharma • Why Unique? – Open to both “for profit” and “not for profit” outcomes – Greater alignment of ecosystem stakeholders Value Proposition: University • Our Value to University – – – – • Accelerate University imperative to advance technology and ideas Convert assets to pending products; Prompt, non-biased asset review Highly efficient and accelerated path to proof of concept/value point Added capital, grants and R & D resources for promising assets What? – Form top team dedicated to project; optimized for each step – With License, form project specific LLC’s • Why Unique? – – – – Straightforward, standard licensing terms with non-dilutive terms Maximized return to all stakeholders - Each university receives full benefit of its own success R & D consultation “genius bar” for partners Flexible, renewable and scalable strategy Value Proposition: Pharma and Biotech Companies • Our Value to Companies – Breadth and flow of innovative emerging products to fuel pipeline. • What? – POC and Pharma assets ready for late stage development – Flexibility for Pharma or us to further develop to desired value point. • Why Unique? – Alignment and quality of team selecting and developing projects – Option for risk sharing – Ability to see/review all potential assets from a portfolio of top investigators and universities – Flexibility to enrich portfolio with desired targets, drug leads, or advanced product candidates for any therapeutic area desired Top Team and Network Selects and Develops Opportunities • Team of industry and academic leaders/consultants- “best in class” for each project. • Team nominated by both the Company and PI’s • Our team has been Instrumental in developing 19 drugs on the market – E.g. Tykerb®, Gemzar®, Coreg®, Hycamtin® – Arranon®, Corlopam® • Excellent project management and broad drug development expertise Road to Drug Development Registration Clinical Data Analysis Full Development Rx NDA/BLA Studies in 100-300 Patients (Phase II) Candidate Medicine Tested in 3-10,000 Patients (Phase III) Scale up Candidate Medicine Synthesized Toxicology Phase 1 & POCV in humansrs Phase I Formulations Developed POC/Exploratory Development Project Team and Plans Candidate Pharma cology Synthesis of Compounds Screening Discovery Adapted from J L LaMattina Road to Drug Development: Our Leadership Examples Registration Clinical Data Analysis Full Development Rx™ NDA/BLA Studies in 100-300 Patients (Phase II) Candidate Medicine Tested in 3-10,000 Patients (Phase III) Scale up Candidate Medicine Synthesized Candidate Phase 1 & POCV in humansrs Phase I Formulations Developed POC/Exploratory Development Project Team and Plans Toxicology RJ, HP, SM, WD Pharma cologyt Synthesis of Compounds Screening Discovery BB, MR, TB, EO Adapted from J L LaMattina Road to Drug Development: Our Leadership Examples POC and Pharma ready packages Full Development Studies in 100-300 Patients (Phase II) Registration Clinical Data Analysis Rx® NDA/BLA Candidate Medicine Tested in 3-10,000 Patients (Phase III) Scale up Candidate Medicine Synthesized Toxicology Phase 1 & POCV in humansrs Phase I Candidate Formulations Developed POC/ Exploratory Development Pharma cology Project Team and Plans Synthesis of Compounds Screening Discovery Adapted from J L LaMattina Our R&D Team and Network Team member • Barry A. Berkowitz, PhD (Co-Chairman & CEO) • Neil Spector, MD • Randall K. Johnson, Ph.D. • Homer Pearce, Ph.D. Background Expertise • Biotechnology and big pharma • Pharmaceutical research − Hoffmann LaRoche and development − Smith, Kline & French • Project and company management • Director of Translational Research in Oncology, Duke University • Co-Director of the Experimental Therapeutics Program Duke Cancer Center • Pharmaceutical research and development • Group Research, Glaxo • SmithKline • Director, in vitro and in vivo pharma R & D • Vice President, Eli Lilly • − directed cancer drug research and development Pharmaceutical research and development Pharmaceutical research and development Achievements • Co-Founder and/or CEO: - Myco/Chemgenics (acquired by Millennium) - Fibrogen - New Chemical Entities (acquired by Albany Molecular Research) • Corlopam® program leader • Tykerb® • Arranon® • Hycamtin® • 25 INDs completed • producing 3 NDAs − Gemzar® − Alimta® More R&D Team and Network Team member Background • Tony Barrett, PhD • Sir Derek Barton Professor of Synthesis, Imperial College, UK • Director of the Wolfson Centre for Organic Chemistry in Medical Science • Mark Roffmann, PhD (VP Devel and Reg Affairs) • Glaxo SmithKline • President, Clinsearch (clinical and regulatory CRO) • Eliot H Ohlstein, PhD (VP Devel and Therapeutics) • Senior Vice President of the Cardiovascular Urogenital Center of Excellence in Drug Discovery (CEDD) at GlaxoSmithKline. • Wallace Dairman, P hD • Roche Institute Of Molecular Biology • Senior Toxicologist Hoffmann La Roche Expertise Achievements • Natural product based pharmaceuticals • Analytical and medicinal chemistry • Pioneering pharma industry contributions. Co-founder Argenta Discovery and iThemba Pharmaceuticals • Pharmacology • Regulatory • 30+ years drug development & clinical research experience • Led filings of 4 NDAs and 20+ INDs • Pharmacology • Drug development • PhRMA's Discoverer's Award, for development GSK's Coreg® (carvedilol), • Leadership in taking seven drugs to market • Drug development • Toxicology • Regulatory • Pioneering studies on molecular biology of biogenic amines • Toxicology and regulatory advisor on numerous IND and NDA submissions More R&D Team and Network Team member Background Expertise Achievements • Co-founder Myco/Chemgenics; Novodyme • Chairs/Coordinates NIAID advisory group Infectious Disease • Research and Clinical Development of Antiinfective Drugs Head CNS & Endocrine Research Senior Director Project Management Director Clinical Research Senior Director Quality Assurance • • • • • Project Management Quality Assurance Clinical Development WW Management Experience FDA interactions ∙ Satish Menon, PhD (Director Biotechnology) Allergan- Director Biopharmaceutical Processing Schering Plough • • • Protein Drugs Assay Development GMP Production • Responsible for process development for new biotechnology based drugs · Other Project Specific Advisors Special advisors for each Project Strong input from PI’S • Experts in a specific project • Allows “best in class” project teams • John (Jack) Edwards, MD • Identity Confidential, PhD Chief Infectious Disease. UCLA Medical Center, Harbor Branch; Prof of Medicine UCLA • Project team leader of several Blockbuster drugs •WW leader in Quality Assurance and Project Management Initial Business Advisory Board Team member • Robert Morgan (our CFO) • Marc Goldberg * (Chairman, Business Advisory Board) Background • • • • • PriceWaterhouseCoopers (manager, small business group) Co-Founder NewCoGen (Flagship VenturPartners) Co-founder – Chemgenics, New Chemical Entities BioVentures Investors (co-founder & general partner ) Mass Biotech Council (co-founder) • John Canepa • PathoGenetix (CEO); Stemgent (CFO) • Arthur Anderson (senior partner and head of worldwide life science practice) • Elliot M. Olstein • Carella, Byrne, Cecchi, Olstein, Brody & Agnello, P.C. (Partner) • Patent and licenseing • Jon Soderstrom* • Managing Director, Yale University Medical School, Office of Cooperative Research • Joseph A. Boystak* • • • (Co-Chairman) President and Chief Executive Officer of Brightwaters Capital, LLC, Founder & Managing Director Global Life Sciences, Jefferies & Co University Board Member, UCLA and Johns Hopkins * Board of Directors CASE STUDIES FROM OUR DEVELOPMENT PORTFOLIO Our Initial Therapeutic Areas Under Development • Inflammation • Cancer • Cardiovascular – Renal Other Areas – Case by case for outstanding opportunities – Early review and adoption by Pharma Partner(s) Stem Cells and Stem Cell Proteins • Novel stem cell signaling protein: TSG-6 • New Mechanism of Inflammatory modulation with broad anti-inflammatory indications – initially in ophthalmology • Dr Darwin Prockop, Director Institute of Regenerative Medicine, Texas A & M, PI • POC established, sponsored research, licensed, 12-18 months from IND • Clear metrics of Therapeutic Activity TSG-6 Prevents Corneal Damage Following Injury in Preclinical models: Novel Anti-inflammatory Mechanism Day 3 Day 7 Day 21 PBS rTSG-6 • Project Team includes: B. Berkowitz M. Roffman, D. Prockop, W. Dairman; Plus project specific advisors: Prof Anthony Day (WW leader TSG-6), U Manchester; and Armand Keating (WW leader cancer and biological therapy, past president Am Soc. Hematology). New Generation Vinca for Cancer • Potential 1st in class Therapeutic Index • Preclinical POC shown • Completing Phase 1 clinical trial – Promising Safety Profile (46 patients) – Peripheral Neuropathy a serious side effect of other Vinca’s not apparent to date. • Biomarker identified; NIH interactions • Licensed, partnered with AMRI, for all chemistry and GMP supply in hand thru phase 2 needs. New Drugs for Renal Injury • Innovative project with biomarker/diagnostic and linked therapeutic for personalized medicine – – – – Novel mechanism Meets key need: effective therapies for Renal Failure Gary Desir, MD Yale (Nephrology/Medicine) PI Licensed, preclinical POC shown, 12-18 months to IND; sponsored research • Project team – M. Roffman, E. Ohlstein (former head of CV/Renal GSK), W. Dairman, S, Menon, Gary Desir R & D Genius Bar • University and Foundation Projects frequently “stuck” at translational stage • Biological Target or Drug Lead– What to do next? • Patent? Publish? Proof of Concept? More Resources? License? Bessor-Brightwaters Pharma in synergy with the University or Foundation will provide R & D guidance • “Real time” response and advice on drug discovery, IP and development issues • Questions may come from selected Universities or Foundations where we have established an R & D relationship; optioned/licensed technology to us or emerging technology from other labs/assets at the University or Foundation • Replies will be prompt, coordinated and provided from our expert, integrated drug discovery and development network • No cost or obligations to University or Foundation • IP and confidentiality are protected University Portfolio • Yale • Texas A & M • Emerging relationships: – Duke – Univ. of Florida – Univ. of Manchester (UK) • Others Pending – Initial Focus in Northeast USA We are Open for Business: Universities, Foundations, Pharma, Investors • • • • Ecosystem, development and business model yielding RESULTS Growing network of >20 key advisors and companies Partnering with major universities, including Yale, Texas A&M, Building a pipeline of in-licensing ready additional projects with R & D leaders at five universities; opening discussions with disease foundations • Awarded a competitive grant from the NIH • Sponsored research @ 2 Univ. sites accelerates development • Launched and advancing novel projects in inflammation (12 months to IND), CV/kidney disease (18 months to IND), and cancer (completing Phase 1 clinical trial) THANK YOU AND WE WELCOME FURTHER DISCUSSION Joseph A. Boystak, Co-Chairman and Barry A. Berkowitz, PhD, Co-Chairman and CEO [email protected]