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

Bberkowitz@B-BPharma.com