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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
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Finance
Our Solution
Bessor-Brightwaters Pharma
Early stage venture capital erratic
Business models broken
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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
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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
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Our Value to University
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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
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Why Unique?
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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
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SmithKline
• Director, in vitro and in vivo
pharma R & D
• Vice President, Eli Lilly
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− 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
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Research and Clinical
Development of
Antiinfective Drugs
Head CNS & Endocrine Research
Senior Director Project
Management
Director Clinical Research
Senior Director Quality Assurance
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Project Management
Quality Assurance
Clinical Development
WW Management
Experience
FDA interactions
∙ Satish Menon, PhD
(Director
Biotechnology)
Allergan- Director
Biopharmaceutical Processing
Schering Plough
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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
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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
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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*
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(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
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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
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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]