Fortress Biotech, Inc. - FORM 8-K - EX-99.1

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8-K - 8-K - Fortress Biotech, Inc.	d649047d8k.htm

NASDAQ: CNDO

December 2013

Lindsay A. Rosenwald, MD

Chairman, President and CEO

Exhibit 99.1

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 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 on 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

Experienced management team and board of directors

In both drug development and business development

Dedicated to maximizing shareholder

value

Business development expertise

Products and technologies acquisitions and licensing

Investment and spin-out opportunities

Creative financing solutions

Two biologic product candidates in clinical stage development

Focused on autoimmune diseases and cancer immunotherapy

Strong proprietary position

Value Proposition

Lindsay A. Rosenwald, MD

Chairman, President and CEO

Prolific and successful investor in the life sciences industry

for over 20 years as Chairman of Paramount BioCapital

Co-Portfolio Manager and Partner of Opus Point Partners

Lucy Lu, MD

EVP & Chief Financial Officer

Senior Analyst at Citi Investment Research

Over 10 years of biotech equity research experience

George C. Avgerinos, PhD

Sr. VP, Biologics Operations

Divisional VP, Global Process and Manufacturing Sciences,

Abbvie

Over 30 years experience in biopharmaceutical process

development including Humira™

process and manufacturing

Key Management and Board Members

Kevin Horgan, MD

Chief Medical Officer

more than 25 years of academic and pharmaceutical experience

SVP and Chief Medical officer at Soligenix

Vice President of Clinical Immunology at Janssen Biotech

Michael S. Weiss

Co-Vice Chairman

Co-Portfolio Manager and Partner of Opus Point Partners, LLC

(since 2009)

Executive Chairman, Interim CEO and President of TG Therapeutics

Chairman and CEO of Keryx Biopharmaceuticals

Eric K. Rowinsky, MD

Co-Vice Chairman

World renown oncologist, former CMO at ImClone, board of

Biogen/Idec

Chief Medical Officer and Head of Research and Development;

Executive Vice President

Stemline Therapeutics

Products and technologies

We intend to pursue product and technology acquisitions and

licenses.

Investment opportunities

We may make controlling and non-controlling investments in

companies with proprietary products and/or technologies where we

believe shareholder value will be enhanced, while preserving capital

and strength of our balance sheet.

Spin-out opportunities

From time to time we may spin out new companies based on current

and new technologies and products that we develop, license or

acquire.

Business Development Strategies

Porcine whipworm ova

Represents

novel

approach

treating

autoimmune

diseases

–

the

“Hygiene Hypothesis”

Natural immunomodulator -

regulates T-Reg cells and inflammatory

cytokines

Planned and ongoing studies in multiple autoimmune indications

including psoriasis, autism, multiple sclerosis, and ulcerative colitis

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)

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

Ova are Microscopic

Benefits of Trichuris suis ova (TSO)

12 week, dose ranging study

TSO 250, 2500, 7500 or placebo

N=240

Crohn’s patients

•

CDAI = 220-350

Outcome –

Discontinued post 2

Interim analysis

TSO Phase 2 Crohn’s Disease Studies

TRUST

12 week study

TSO 7500 or placebo

N=250

Crohn’s patients

•

CDAI = 220-450

•

The randomization was stratified by

disease activity as measured by

CDAI

Outcome –

Did NOT meet overall

primary endpoint of response

Select TSO Trials

* TSO Phase 2a studies being conducted

as Investigator-Initiated Studies

Planned

OL = Open-Label DB = Double-Blind

SB= Single-Blind PC = Placebo-Controlled

*expected

Indication

Pre-Clinical

Phase 1

Phase 2a*

Phase 2

Phase 3

Design

Ulcerative Colitis

120 pt, DB, PC

Ulcerative Colitis (MOA)

18 pt, DB, PC

Multiple Sclerosis (US)

15 pt, SB

Multiple Sclerosis (EU)

50 pt, DB, PC

Autism

10 pt, DB, Cross

Autism

60 pt, DB, PC

Psoriasis

20 pt, OL

1H2014 (*)

Numerous threads of evidence suggest that Autism is an

immune mediated disorder linked to the hygiene hypothesis (HH)

Gene –

environment mismatch is likely responsible for the dramatic

increase in incidence

Increase parallels that of asthma and other HH conditions

Strong evidence for GI issues and immune dysregulation in

Autism patients

Up to 70% of the patients have current or past GI issues, and

endoscopy often shows low level inflammation in these patients

Abnormal cytokines, B cell and T cell subsets, and low levels of

regulatory cytokines

Evidence of activation of the innate immune system in the CNS

Maternal autoimmune diagnosis is a recognized risk factor

Anecdotal evidence and the interim data from Dr. Hollander’s

study point to potential efficacy

Scientific Rationale for TSO for Autism

Ongoing double-blind, placebo-controlled, randomized cross-

over study in 10 adult, high functioning autism patients at

Montefiore Medical Center

In the first 5 patients that completed the study, there was a

statistically significant separation from placebo in favor of TSO

on three measures of disease that reflect a core feature of

autism (the restrictive interests and repetitive behavior domain)

Montefiore-Einstein Rigidity Scale (MERS)

Repetitive Behavior Scale-Revised (RBS-R) Sameness Scale

Social Responsiveness Scale (SRS)-Repetitive Behaviors Scale

TSO is well tolerated

Final results are expected in mid 2014

Pilot Study of TSO in Autism

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

* Expiration dates do not include any patent term extension

TSO Intellectual Property

‘Use of Parasitic Biological Agents for Disease Prevention and Control’ - directed to the

treatment of animals/man with a Th1 or Th2 mediated autoimmune disease – exp.

11/2023

‘Production of a Viable, Storable Worm Egg Suspension’ - directed to a process for

preparation of TSO using an 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 to methods of treating obesity and IBS - exp.

10/2029

‘Compositions and Methods for Treating IBD’ - directed to a method of treating IBD by

contacting an isolated dendritic/macrophage cell with a helminth – exp. ~9/2032

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

Trigger

receptor

Trigger

receptor

Priming

receptor

Priming

receptor

Trigger

receptor

Priming

receptor

Resting

Donor NK

Priming

signal

Trigger

ligand

Priming –

Signal 1

Triggering –

Signal 2

“Serial Killer”

Tumor lysis

Dead

Tumor

cell

Primed

Donor NK

Patient’s

tumor

Primed

Donor NK

CNDO-109

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 Remission at time of study entry

1 patient in partial relapse (PR) at time of study entry

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

PR patient achieved CR

Kottaridis, et al., ASH 2011

CNDO-109 Clinical Development

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

relapsed AML

Future autologous studies in other tumor types are possible (e.g.

multiple myeloma, breast, ovarian and prostate)

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

Financials

Listed on NASDAQ: CNDO

Market Cap as of 12/20/2013

~$75M

Shares Outstanding

35.5M

Additional 4.0 M options and warrants

Additional 3.9 M restricted shares

Cash Position as of 9/30/2013

$106.3M

Total Debt as of 9/30/2013

$15.0M

Accounts Payable as of 9/30/2013

$1.1M

Working Capital as of 9/30/2013

$96.9M

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