Fortress Biotech, Inc. - FORM 8-K - EX-99.1 - PRESENTATION OF JANUARY 2013

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8-K - FORM 8-K - Fortress Biotech, Inc.	d462194d8k.htm

Developing Unique Products for Autoimmune

Diseases and Cancer (NASDAQ: CNDO)

January 2013

Harlan F. Weisman, MD

Chairman & Chief Executive Officer

Exhibit 99.1

Statements in this presentation that are not descriptions of historical facts are forward-looking

statements

within

the

meaning

the

“safe

harbor”

provisions

the

Private

Securities

Litigation

Reform Act of 1995. We have attempted to identify forward-looking statements by terminology

including “anticipates,”

“believes,”

“can,”

“continue,”

“could,”

“estimates,”

“expects,”

“intends,”

“may,”

“plans,”

“potential,”

“predicts,”

“should,”

or “will”

or the negative of these terms or other

comparable

terminology.

Forward-looking

statements

are

based

management’s

current

expectations and are subject to risks and uncertainties that could negatively affect our business,

operating results, financial condition and stock price. Factors that could cause actual results to

differ materially from those currently anticipated risks include

those set forth in our SEC filings

including, in particular, risks relating to: our ability to attract, integrate and retain key personnel;

the results of research and development activities; uncertainties relating to preclinical and

clinical testing, financing and strategic agreements and relationships; the early stage of products

under development; our need for substantial additional funds; government regulation; patent

and intellectual property matters; our ability to successfully manufacture TSO in the US;

dependence on third party manufacturers; and competition. We expressly disclaim any

obligation or undertaking to update or revise any statements contained herein to reflect any

change in our expectations or any changes in events, conditions or circumstances after the date

of this presentation.

Forward-Looking Statements

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 completed and multiple additional trials

ongoing

TSO:

Trichuris

suis

ova

(CNDO-201)

Crohn’s

Disease,

Ulcerative

Colitis

(UC)

and

Multiple

Sclerosis

(MS)

CNDO-109:

Tumor

Activated

Cells

relapsed

Acute

Myeloid

Leukemia (AML)

Experienced management team and board of directors

Value Proposition

TSO (Trichuris suis ova or CNDO-201)

Indication

Pre-Clinical

Phase 1

Phase 2a*

Phase 2

Phase 3

Crohn’s Disease

Ulcerative Colitis

Multiple Sclerosis

Autism

Psoriasis

Type-1 Diabetes

Psoriatic Arthritis

Rheumatoid Arthritis

CNDO-109 (Tumor-Activated Natural Killer Cells)

Acute Myeloid Leukemia

Multiple Myeloma

Coronado Pipeline Overview

1Q2013

* TSO Phase 2a studies being conducted as Investigator-Initiated Studies; CNDO-109 Phase 2a AML study is a Phase 1/2 study

1Q2013

1H2013

2H2013

1H2013

Planned

Porcine whipworm ova

Represents

novel

approach

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 Multiple Sclerosis

Phase 2 studies ongoing in Crohn’s disease

Planned studies in multiple additional autoimmune indications

Natural properties suggest strong potential for a safe profile

North and South America and Japanese rights for all indications

TSO: Trichuris suis ova (CNDO-201)

(adapted from Bach, NEJM, 2002)

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 countries

Walsh SJ, Rau LM. Am J Public Health 2000

Faustman, D. Institute of Medicine Report, “Women’s Health Research: Progress,

Pitfalls, and Promise, 2010

Epidemiological data demonstrate:

Various immunological and autoimmune diseases are much less common in

the developing world than the industrialized world

Immigrants

the

industrialized

world

from

the

developing

world

increasingly

develop

immunological

disorders

relation

the

length

time

since arrival in the industrialized world

Distribution of Autoimmune Disorders

and Helminths

Autoimmune disorders incidence

Helminths infestation incidence

High

Moderate

Low

High

Moderate

Low

Weinstock and Elliott, Inflamm Bowel Dis, Jan 2009

POOR SANITATION,

IMPURE FOOD

AND CROWDED

LIVING CONDITIONS

Helminthic

and bacterial

exposures

Viral, bacterial and

protozoan

infections

Crohn’s Disease,

Ulcerative Colitis,

Multiple Sclerosis

other

autoimmune

diseases

(Prevents)

The Biology Supporting the Hygiene

Hypothesis

Inhibits

Excess

Reactivity

Excess

Th1

Elliott & Weinstock, Ann NY Acad Sci, 2012

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

Benefits of Trichuris suis ova (TSO)

Effect of TSO in Crohn’s Disease

Patients

and

Methods

•

29 CD patients with

CDAI>220 (mean=296)

•

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 points

•

Response defined as a

CDAI > 100 point drop from

baseline

Summers, et.al., GUT

2005

Effect of TSO in Ulcerative Colitis

p=0.04

16.7%

43.3%

Placebo

T. suis

Summers, et.al., Gastroenterology 2005

Patients

and

Methods

•

n = 54 UC patients with a

UCDAI score > 4 points

•

Average score 8.7-8.8

•

Duration of disease

averaged 8 years

•

2500 TSO every 2 weeks

for 3 months

•

Most patients refractory to

previous therapy

•

Response was defined as

> 4 point drop

12 week, dose ranging study

Double-blind, randomized,

placebo controlled

TSO 250, 2500, 7500 or placebo

N=250 (2

interim)

Crohn’s patients

•

CDAI = 220-350

•

CRP 2X ULN or Calprotectin 1X

ULN

Outcome –

Remission rates

Interim –

2H 2013

TSO Phase 2 Crohn’s Disease Studies

TRUST

12 week study

Double-blind, randomized,

placebo controlled

TSO 7500 or placebo

N=220

Crohn’s patients

•

CDAI = 220-450

•

Endoscopic evidence of

inflammation

Outcome –

Response rates

Topline data –

2H 2013

Impact of Parasitic Infections on the

Course of Multiple Sclerosis

Correale J, Farez MF.

J Neuroimmunol. 2011;233:6

Effect of TSO in Multiple Sclerosis

Fleming, et.al., Multiple Sclerosis Journal 2011

TSO (Trichuris suis ova or CNDO-201)

Indication

Pre-Clinical

Phase 1

Phase 2a*

Phase 2

Phase 3

Design

Crohn’s Disease

~500pts, DB, PC

Ulcerative Colitis

120 pt, DB, PC

Ulcerative Colitis (MOA)

18 pt, OL

Multiple Sclerosis (US)

16 pt, SB

Multiple Sclerosis (EU)

50 pt, DB, PC

Autism

10 pt, DB, Cross

Psoriasis

20 pt, OL

Type-1 Diabetes

60 pt, DB, PC

Early Intervention

Type-1 Diabetes

150 pt, DB, PC

Prevention

Psoriatic Arthritis

20-30 pt, DB, PC

Rheumatoid Arthritis

50 pt, DB, PC

TSO Pipeline

* TSO Phase 2a studies being conducted

as Investigator-Initiated Studies

1Q2013

Two phase 2 studies

OL = Open-Label DB = Double-Blind,

= Single-Blind PC = Placebo-Controlled

1Q2013

1H2013

Planned

TSO Manufacturing Process

Certified specific pathogen-free minipigs

Observation and pathogen testing

Oral Inoculation with OVA from master bank

Development of infection

Harvest OVA from pigs

Isolation and purification

Processing and pathogen inactivation

API

Quality and pathogen testing

Incubation and sterilization

Formulation process

Fill/finish

DRUG Product

Quality and microbiological testing

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

studies and beyond

OvaMed will continue to supply phase 2 and IIS requirements

Coronado will pay Ovamed a total of $1.5M in three equal installments in Dec.

2014-2016, and a “Manufacturing Fee”

for product made and sold by us in lieu

of the transfer price for manufacture of TSO

The Manufacturing Fee will consist of the greater of (i) a royalty on net sales of

product manufactured by us or (ii) a specified amount per unit, and is subject

to certain adjustments and credits

The agreement contemplates that each of Coronado and Ovamed would act

as Second Source to the other at agreed transfer prices

Acquisition of TSO Manufacturing Rights

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 patents

‘Use

Parasitic

Biological

Agents

for

Disease

Prevention

and

Control’

directed

the

treatment

animals/man

with

Th1

Th2

mediated

autoimmune

disease

–

exp.

11/2023

‘Production

Viable,

Storable

Worm

Egg

Suspension’

directed

process

for

preparation

TSO

using

acid

wash

exp.

3/2028

‘Method for Characterizing the Biological Activity of Helminth Eggs, in particular

Trichuris

Eggs’

–

exp. 5/2029

‘Treatment

with

Helminths’

directed

methods

treating

obesity

and

IBS

exp.

10/2029

‘Compositions

and

Methods

for

Treating

IBD’

directed

method

treating

IBD

contacting

isolated

dendritic/macrophage

cell

with

helminth

–

exp.

~9/2032

* Expiration dates do not include any patent term extension

TSO Intellectual Property

Potential TSO Target

Indications

U.S.

/Japan

Prevalence

U.S.

/Japan Annual

Market Sales

(USD Mil)

Ulcerative Colitis

669,000

$1,300

Crohn’s Disease

534,000

$2,600

Multiple Sclerosis

485,000

$6,400

Sources:

Decision

Resources

2012

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

well

other

potential

disorders

such as rheumatoid arthritis and psoriasis.

TSO Market Opportunity

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 or long-

term culture/expansion, and does not change NK cell phenotypes

Preclinical activity demonstrated in multiple myeloma, breast

cancer, prostate cancer and ovarian cancer

CNDO-109: Activated Natural Killer Cells

Activated ex vivo by tumor cell

lysate (CNDO-109)

Effective from autologous or

allogeneic NK cell source

Uniquely positioned in patients

with “minimal residual disease”

Remains active after

freeze/thaw

CNDO-109 Mechanism of Action

Priming

signal

Priming

receptor

Trigger

receptor

Priming

receptor

Trigger

receptor

Trigger

ligand

Priming

receptor

Trigger

receptor

“Serial Killer”

Tumor lysis

Dead

Tumor

cell

Primed

Donor NK

Primed

Donor NK

Patient’s

tumor

CNDO-109

Resting

Donor NK

Priming –

Signal 1

Triggering –

Signal 2

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 Remission 2 or 3 (CR2 or CR3)

1 patient in partial relapse (PR)

3/5 experienced a longer CR than their previous CR, in addition

PR patient achieved CR

Kottaridis, et al., ASH 2011

CNDO-109 Phase 1 Study in AML

Initiated Phase 1/2 allogeneic clinical trial for the treatment of

relapsed AML

Once

the

dose

selected,

plan

initiate

randomized

Phase

trial

Potential for regulatory approval with single randomized, controlled

clinical trial if data are clinically meaningful and statistically persuasive

Future autologous studies planned in other tumor types (including

multiple myeloma, breast, ovarian and prostate)

CNDO-109 Clinical Development

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 Cells

and Methods of Using the Same”

–

exp. 7/2030

Provisional

application

pending

the

U.S.

“Compositions

and

Methods for Treating Viral Infections”

* Expiration dates do not include any patent term extension

CNDO-109 Intellectual Property

Potential CNDO-109

Target Indications

G7 Drug

Treatable

Population

G7 Market

Sales

(USD Mil)

Mortality

AML

43,500

$165

5 year mortality rate is 85-90% but varies by age

Multiple Myeloma

44,000

$2,870

Stage III: median survival of 29 months

Breast Cancer

494,000

$10,100

Stage IV: 5 year mortality rate is 76%

Ovarian

57,110

$424

Overall 5 year survival rate of 45%

Prostate

500,000

$4,000

Overall

5-year

survival

rate

+99%,

but

leading cause of cancer deaths in men

G7 = U.S., U.K., Germany, France, Italy, Spain, Japan

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

large

due

the

fact

that

many

types

tumors

are

sensitive

killing

activated

cells.

CNDO-109 Market Opportunity

Sources:

Decision

Resources

2011/2012

Harlan

Weisman,

Chairman

and

Chief

Executive

Officer

Company

Group

Chairman,

Research

Development

for

Pharmaceuticals

JNJ

President

Research

Development

Centocor

Over

years

pharmaceutical/biotechnology

experience

Bobby

Sandage,

Jr.,

PhD

President

EVP

CSO

Indevus

Pharmaceuticals,

Inc.

Over

years

pharmaceutical/biotechnology

experience

Noah

Beerman

EVP

Chief

Operating

Officer

President

CEO

RXi

Pharmaceuticals

Over

years

pharmaceutical/biotechnology

experience

Karin

Hehenberger, MD, PhD

EVP

Chief Medical Officer

Senior

management

positions

Juvenile

Diabetes

Research

Foundation

and

JNJ

Diabetes

Over

years

pharmaceutical/biotechnology

experience

Key Management and Board Members

Lucy Lu, MD

EVP & Chief Financial Officer

Senior Analyst at Citi Investment Research

Over 10 years of biotech equity research experience

Eric K. Rowinsky, MD

Vice Chairman

World renown oncologist, former CMO at ImClone, board of

Over 25 years of healthcare experience

Lindsay Rosenwald, MD

Director and Founder

A prolific

and

successful

investor

the

life

sciences

industry

for over 20

years

Biogen/Idec

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

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

Anticipated Upcoming Milestones

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 completed and multiple additional trials

ongoing

TSO:

Trichuris

suis

ova

(CNDO-201)

Crohn’s

Disease,

Ulcerative

Colitis (UC) and Multiple Sclerosis (MS)

CNDO-109:

Tumor

Activated

Cells

relapsed

Acute

Myeloid

Leukemia (AML)

Experienced management team and board of directors

Investment Highlights

Fortress Biotech, Inc. - FORM 8-K - EX-99.1 - PRESENTATION OF JANUARY 2013 - January 7, 2013

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Fortress Biotech, Inc. Reports