Fortress Biotech, Inc. · NASDAQ:FBIO
Fortress Biotech — Coronado Biosciences — Investor Presentation (January 2013)
29 slides from Fortress Biotech's investor presentation, Jan 7, 2013
Slide 1: Developing Unique Products for Autoimmune Diseases and Cancer
Exhibit 99.1 Developing Unique Products for Autoimmune Diseases and Cancer (NASDAQ: CNDO) Harlan F. Weisman, MD Chairman & Chief Executive Officer January 2013
Slide 2: Forward-Looking Statements
Forward-Looking Statements Statements in this presentation that are not descriptions of historical facts are forward-looking statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. We have attempted to identify forward-looking statements
Slide 3: Value Proposition
Value Proposition » Two biologic product candidates in clinical stage development - Focused on autoimmune diseases and cancer immunotherapy - Strong proprietary position » Novel treatments with broad therapeutic applications addressing multi-billion dollar markets » Four efficacy clinical trials com
Slide 4: Coronado Pipeline Overview
Coronado Pipeline Overview TSO (Trichuris suis ova or CNDO-201) Indication Pre-Clinical Phase 1 Phase 2a* Phase 2 Phase 3 Crohn’s Disease Ulcerative Colitis 1Q2013 Multiple Sclerosis Autism Psoriasis 1Q2013 Type-1 Diabetes 1H2013 Psoriatic Arthritis 1H2013 Rheumatoid Arthritis 1H2013 CNDO-109 (Tumor
Slide 5: TSO: Trichuris suis ova (CNDO-201)
TSO: Trichuris suis ova (CNDO-201) » Porcine whipworm ova - Represents a novel approach to treating autoimmune diseases – the “Hygiene Hypothesis” - Natural immunomodulator - regulates T-Reg cells and inflammatory cytokines » Clinical proof of principle established in Inflammatory Bowel Disease and
Slide 6: Rapid Emergence of Autoimmune and Immune Mediated Diseases
Rapid Emergence of Autoimmune and Immune Mediated Diseases » There are >100 immune- mediated diseases affecting 50 million Americans » Second highest cause of chronic disease in United States and number one cause of morbidity in women » In contrast, most of these diseases are rare in less developed
Slide 7: Distribution of Autoimmune Disorders and Helminths
Distribution of Autoimmune Disorders and Helminths Autoimmune disorders incidence Helminths infestation incidence High High Moderate Moderate Low Low Epidemiological data demonstrate: » Various immunological and autoimmune diseases are much less common in the developing world than the industrialized
Slide 8: The Biology Supporting the Hygiene Hypothesis
The Biology Supporting the Hygiene Hypothesis Viral, bacterial and POOR SANITATION, protozoan IMPURE FOOD infections AND CROWDED LIVING CONDITIONS Excess Helminthic Th1 and bacterial exposures Crohn’s Disease, Ulcerative Colitis, Multiple Sclerosis Inhibits & (Prevents) other Excess autoimmune React
Slide 9: Benefits of Trichuris suis ova (TSO)
Benefits of Trichuris suis ova (TSO) Does not multiply in human host Colonization is self-limited in humans No systemic phase No direct transmission Ova stable Oral dosing; 1 tbsp solution taken once every 2 weeks - Clear, odorless, tasteless 9
Slide 10: Effect of TSO in Crohn's Disease
Effect of TSO in Crohn’s Disease Patients • 29 CD patients with and CDAI>220 (mean=296) Methods • Median duration of the disease : 4 yrs • Baseline meds – 5ASA, low dose steroids, 6-MP or Aza, washout of TNF inhibitors • 2500 TSO every 3 weeks for 24 weeks • Remission defined as a CDAI of < 150 poin
Slide 11: Effect of TSO in Ulcerative Colitis
Effect of TSO in Ulcerative Colitis Patients • n = 54 UC patients with a 50 and UCDAI score > 4 points 43.3% Methods p=0.04 • Average score 8.7-8.8 40 • Duration of disease averaged 8 years 30 • 2500 TSO every 2 weeks for 3 months • Most patients refractory to 20 16.7% previous therapy • Response wa
Slide 12: TSO Phase 2 Crohn's Disease Studies
TSO Phase 2 Crohn’s Disease Studies TRUST - I TRUST - II » 12 week study » 12 week, dose ranging study - Double-blind, randomized, - Double-blind, randomized, placebo controlled placebo controlled - TSO 7500 or placebo - TSO 250, 2500, 7500 or placebo - N=220 - nd N=250 (2 interim) - Crohn’s patient
Slide 13: Impact of Parasitic Infections on the Course of Multiple Sclerosis
Impact of Parasitic Infections on the Course of Multiple Sclerosis Correale J, Farez MF. J Neuroimmunol. 2011;233:6 13
Slide 14: Effect of TSO in Multiple Sclerosis
Effect of TSO in Multiple Sclerosis Fleming, et.al., Multiple Sclerosis Journal 2011 14
Slide 15: TSO Pipeline
TSO Pipeline TSO (Trichuris suis ova or CNDO-201) Indication Pre-Clinical Phase 1 Phase 2a* Phase 2 Phase 3 Design Crohn’s Disease Two phase 2 studies ~500pts, DB, PC Ulcerative Colitis 1Q2013 120 pt, DB, PC Ulcerative Colitis (MOA) 18 pt, OL Multiple Sclerosis (US) 16 pt, SB Multiple Sclerosis (EU)
Slide 16: TSO Manufacturing Process
TSO Manufacturing Process Certified specific pathogen-free minipigs API Observation and pathogen testing Quality and pathogen testing Oral Inoculation with OVA from master bank Incubation and sterilization Development of infection Formulation process Harvest OVA from pigs Fill/finish Isolation and p
Slide 17: Acquisition of TSO Manufacturing Rights
Acquisition of TSO Manufacturing Rights » Coronado acquired manufacturing rights from Ovamed GmbH in North America, South America, and Japan (Coronado’s licensed territory) » Establishes control over TSO manufacturing » Facility to be built out in Woburn, MA in 2013, and to supply TSO Phase 3 studie
Slide 18: TSO Intellectual Property
TSO Intellectual Property Three issued US patents entitled ‘Use of Parasitic Biological Agents for Prevention and Control of Autoimmune Disease’ directed to compositions, methods of producing compositions, and methods of autoimmune disease with helminths – exp. 12/2018 Five additional pending patent
Slide 19: TSO Market Opportunity
TSO Market Opportunity The mechanism of action of TSO should, if approved, allow it to be positioned in a variety of autoimmune disorders, including inflammatory bowel diseases and multiple sclerosis as well as other potential disorders such as rheumatoid arthritis and psoriasis. Potential TSO Targe
Slide 20: CNDO-109: Activated Natural Killer Cells
CNDO-109: Activated Natural Killer Cells » NK cells represent the key component of the body’s innate immune surveillance system » Proof of principle established in patients with high-risk refractory or relapsed acute myeloid leukemia (AML) » Activation with CNDO-109 does not require toxic cytokines
Slide 21: CNDO-109 Mechanism of Action
CNDO-109 Mechanism of Action » Activated ex vivo by tumor cell Priming –Signal 1 lysate (CNDO-109) Priming signal Resting CNDO-109 » Effective from autologous or Donor NK Priming receptor allogeneic NK cell source Trigger receptor » Uniquely positioned in patients Triggering S – ignal 2 with “minima
Slide 22: CNDO-109 Phase 1 Study in AML
CNDO-109 Phase 1 Study in AML » Phase 1 investigator sponsored open-label trial » To determine the safety of infusion of allogeneic Tumor-activated NK (TaNK) cells after low dose radiotherapy plus chemotherapy in high-risk relapse or refractory AML patients » Enrolled 8 AML patients - 5 in Complete
Slide 23: CNDO-109 Clinical Development
CNDO-109 Clinical Development » Initiated Phase 1/2 allogeneic clinical trial for the treatment of relapsed AML » Once the dose is selected, plan to initiate a randomized Phase 2 trial - Potential for regulatory approval with single randomized, controlled clinical trial if data are clinically meanin
Slide 24: CNDO-109 Intellectual Property
CNDO-109 Intellectual Property » Core patent - “Method for activating natural killer cells by tumor cell preparation in vitro” - Issued in U.S. - exp. 1/2029 - Issued in Australia – exp. 3/2026 - Pending in Europe, Canada, Japan and India » Patent pending - “Preserved Compositions of Activated NK Ce
Slide 25: CNDO-109 Market Opportunity
CNDO-109 Market Opportunity If Coronado establishes the efficacy of CNDO-109 activated NK cells in the treatment of AML, Coronado believes the market opportunity for CNDO-109 activated NK cell therapy islargedueto the factthat manytypesof tumorsaresensitiveto killingbyactivatedNK cells. G7 Drug G7 M
Slide 26: Key Management and Board Members
Key Management and Board Members Harlan F. Weisman, MD Lucy Lu, MD Chairman and Chief Executive Officer EVP & Chief Financial Officer » Company Group Chairman, Research & Development for » Senior Analyst at Citi Investment Research Pharmaceuticals at JNJ » Over 10 years of biotech equity research ex
Slide 27: Financials
Financials Listed on NASDAQ: CNDO Market Cap as of 1/4/2013 $130M Shares Outstanding 24.4M - Additional 3.6M options and warrants Cash Position as of 9/30/2012 $47.5M 27
Slide 28: Anticipated Upcoming Milestones
Anticipated Upcoming Milestones TSO Initiate Multiple Investigator Initiated Studies 1H 2013 TRUST-II (Falk) Crohn’s study 2nd Interim Results 2H 2013 TRUST-I (CNDO) Crohn’s study Topline Results 2H 2013 CNDO-109 Initiate Multiple Myeloma Study 2H 2013 28
Slide 29: Investment Highlights
Investment Highlights » Two biologic product candidates in clinical stage development - Focused on autoimmune diseases and cancer immunotherapy - Strong proprietary position » Novel treatments with broad therapeutic applications addressing multi-billion dollar markets » Four efficacy clinical trials
Coronado Biosciences — Investor Presentation (January 2013) on 6ix