Bessor-Brightwaters Pharma · • Pipeline to mitigate risk, optimize returns • Aligned,...
Transcript of Bessor-Brightwaters Pharma · • Pipeline to mitigate risk, optimize returns • Aligned,...
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
Investors
Asset Evaluation and
Selection Advisors
Academic
Partners
Asset Pools Project LLC’s
Consolidators/
Acquirers
Industry Partners
Foundations
Bessor-Brightwaters
Pharma
Capital Markets
The Problem
Foundations
Pharma/Big Biotech and Patients Pressing, unfulfilled need for innovative compounds
Wealth of projects, but not ready for pharma/big biotech
Finance • Early stage venture capital erratic • Business models broken
Our Solution
Bessor-Brightwaters Pharma
• Capital efficient project development • POC & pharma-ready drugs • Attractive returns to all ecosystem
stakeholders • Top R & D team/Plus FIPNET
Academia
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
Pharma/Big Biotech Products
Innovative drugs for patients
• Flow of innovative new drugs • Multiple and earlier returns/ & types to our ecosystem stakeholders
RESULTS
An Aligned and Fully Integrated and
Pharmaceutical (FIPNET)
Yale
Texas A&M
Bessor- Brightwaters Pharma (Semi-virtual/efficient standardized terms)
•Launch, accelerate and add value to projects •Development know-how •Business development •Operations management •Fundraising /deals
Technology Experts
Development Network • Chemistry (AMRI)/Pharmacology • Regulatory/Project Management/Toxicology • Drug Supply/Clinical Development
Optimize Returns to Ecosystem Stakeholders
Pipeline Projects: Duke, Univ. of Fla., Univ. Manchester, Others
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
Discovery
Full
Development
Registration
Scale up
Candidate Medicine
Synthesized
Project Team
and Plans Synthesis
of Compounds
Pharma
cology
Candidate Formulations
Developed
Toxicology
Screening
Phase 1 & POCV in
humansrs Phase I
Candidate Medicine Tested in
3-10,000 Patients (Phase III)
Studies in 100-300
Patients (Phase II)
Clinical Data
Analysis
Exploratory Development POC/
NDA/BLA
Rx
Adapted from J L LaMattina
Road to Drug Development: Our Leadership
Examples
Discovery
Full
Development
Registration
Scale up
Candidate Medicine
Synthesized
Project Team
and Plans Synthesis
of Compounds
Pharma
cologyt
Candidate Formulations
Developed
Toxicology
Screening
Phase 1 & POCV in
humansrs Phase I
Candidate Medicine Tested in
3-10,000 Patients (Phase III)
Studies in 100-300
Patients (Phase II)
Clinical Data
Analysis
Exploratory Development POC/
NDA/BLA
Rx™
Adapted from J L LaMattina
BB, MR, TB, EO
RJ, HP, SM, WD
Road to Drug Development: Our Leadership
Examples
Discovery
Exploratory Development
Full
Development
Registration
Scale up
Candidate Medicine
Synthesized
Project Team
and Plans
Synthesis
of Compounds
Pharma
cology
Candidate
Formulations
Developed
Toxicology
Screening
Phase 1 & POCV in
humansrs Phase I
Candidate Medicine Tested in
3-10,000 Patients (Phase III)
Studies in 100-300
Patients (Phase II)
Clinical Data
Analysis
POC/
NDA/BLA
Rx®
Adapted from J L LaMattina
POC and Pharma ready
packages
Our R&D Team and Network
Team member Background Expertise Achievements
• Barry A. Berkowitz,
PhD
(Co-Chairman &
CEO)
• Pharmaceutical research
and development
• Project and company
management
• Biotechnology and big pharma
− Hoffmann LaRoche
− Smith, Kline & French
• Director of Translational
Research in Oncology, Duke
University
• Co-Director of the Experimental
Therapeutics Program Duke
Cancer Center
• Tykerb®
• Arranon®
• Neil Spector,
MD
• Pharmaceutical research
and development
• Group Research, Glaxo
SmithKline
• Director, in vitro and in vivo
pharma R & D
• Hycamtin® • Randall K.
Johnson, Ph.D.
• Pharmaceutical research
and development
• Vice President, Eli Lilly
− directed cancer drug research and
development
• Homer Pearce,
Ph.D.
• Pharmaceutical research
and development
• Co-Founder and/or CEO:
- Myco/Chemgenics
(acquired by Millennium)
- Fibrogen
- New Chemical Entities
(acquired by Albany Molecular
Research)
• Corlopam® program leader
• 25 INDs completed
• producing 3 NDAs
− Gemzar®
− Alimta®
More R&D Team and Network
Team member Background Expertise Achievements
• Tony Barrett, PhD • Natural product based
pharmaceuticals
• Analytical and
medicinal chemistry
• 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)
• Pharmacology
• Regulatory
• Glaxo SmithKline
• President, Clinsearch (clinical and
regulatory CRO)
• 30+ years drug development &
clinical research experience
• Led filings of 4 NDAs and 20+
INDs
• Eliot H Ohlstein,
PhD (VP Devel and
Therapeutics)
• Pharmacology
• Drug development
• Wallace Dairman,
P hD • Drug development
• Toxicology
• Regulatory
• Senior Vice President of the
Cardiovascular Urogenital Center of
Excellence in Drug Discovery
(CEDD) at GlaxoSmithKline.
• Roche Institute Of
Molecular Biology
• Senior Toxicologist
Hoffmann La Roche
• PhRMA's Discoverer's Award,
for development GSK's
Coreg® (carvedilol),
• Leadership in taking seven
drugs to market
• Pioneering pharma industry
contributions. Co-founder
Argenta Discovery and iThemba
Pharmaceuticals
• 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
• John (Jack)
Edwards, MD
• Research and Clinical
Development of
Antiinfective Drugs
Chief Infectious Disease. UCLA
Medical Center, Harbor Branch;
Prof of Medicine UCLA
• Identity
Confidential, PhD
• Project Management
• Quality Assurance
• Clinical Development
• WW Management
Experience
• FDA interactions
• Protein Drugs
• Assay Development
• GMP Production
• Experts in a specific
project
• Co-founder
Myco/Chemgenics;
Novodyme
• Chairs/Coordinates NIAID
advisory group Infectious
Disease Head CNS & Endocrine Research
Senior Director Project
Management
Director Clinical Research
Senior Director Quality Assurance
• Project team leader
of several Blockbuster drugs
•WW leader in Quality
Assurance and Project
Management
∙ Satish Menon, PhD
(Director
Biotechnology)
· Other Project
Specific Advisors
Allergan- Director
Biopharmaceutical Processing
Schering Plough
Special advisors for each Project
Strong input from PI’S
• Responsible for process
development for new
biotechnology based drugs
• Allows “best in class”
project teams
Initial Business Advisory Board
Team member Background
• Robert Morgan (our CFO)
• PriceWaterhouseCoopers (manager, small business group)
• Co-Founder NewCoGen (Flagship VenturPartners)
• Co-founder – Chemgenics, New Chemical Entities
• Marc Goldberg *
(Chairman, Business Advisory Board)
• 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*
• Joseph A. Boystak* (Co-Chairman)
• Managing Director, Yale University Medical School, Office of
Cooperative Research
• 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