A Company Developing Unique Therapies for Autoimmune ... · PDF fileRepurposing and...

Cytocom, Inc – May 2015 - Confidential 1

A Company Developing Unique Therapies

for

Autoimmune Disease and Cancer

Repurposing and Reformulating Existing Therapies

Decreasing time to market, reducing risk


DISCLAIMER THE STATEMENTS MADE IN THIS PRESENTATION MAY INCLUDE FORWARD-LOOKING STATEMENTS REGARDING THE FUTURE OPERATIONS, OPPORTUNITIES OR FINANCIAL PERFORMANCE OF CYTOCOM INC. THESE FORWARD-LOOKING STATEMENTS ARE ONLY ESTIMATIONS BASED UPON THE INFORMATION AVAILABLE TO CYTOCOM, INC., AS OF THE DATE OF THIS PRESENTATION. EXCEPT AS REQUIRED BY LAW, WE EXPRESSLY DISCLAIM ANY RESPONSIBILITY TO PUBLICLY UPDATE OR REVISE OUR FORWARD-LOOKING STATEMENTS, WHETHER AS A RESULT OF NEW INFORMATION, FUTURE EVENTS OR OTHERWISE. THUS, THE FORWARD-LOOKING STATEMENTS HEREIN INVOLVE KNOWN AND UNKNOWN RISKS AND UNCERTAINTIES SUCH THAT ACTUAL FUTURE OPERATIONS, OPPORTUNITIES OR FINANCIAL PERFORMANCE MAY DIFFER MATERIALLY FROM THESE FORWARD-LOOKING STATEMENTS. UNDUE RELIANCE SHOULD NOT BE PLACED ON FORWARD LOOKING STATEMENTS, WHICH SPEAK ONLY AS OF THE DATE HEREOF. ALL FORWARD-LOOKING STATEMENTS CONTAINED HEREIN ARE QUALIFIED IN THEIR ENTIRETY BY THE FOREGOING CAUTIONARY STATEMENTS.

Cytocom, Inc – May 2015 - Confidential 3 Balance Activation

Cytocom, Inc. a specialty biotech company, founded by patients funded by patients, specializing in repurposing of drugs that deploys patented patented therapies focused on the activation and rebalancing of the body’s immune system. Stimulating the body’s immune system remains one of the most promising approaches in the treatment of Cancers, HIV, Autoimmune Diseases, inflammatory conditions and other chronic infectious diseases.

CYTOCOM, INC.


15 Years of R&D leading to a robust platform that delivers

TWO interrelated cytokine immunotherapies—IRT-103 (LDN) and IRT-103 Met- Enkephalin (MENK)

34 Phase 1 & 2 clinical trials 12 indications IRT-103

7 Phase I and II clinical trials 5 indication IRT-101

Pipeline address Multi-Million Dollar Markets

Strong Intellectual Property Portfolio

42 patents issued and 4 pending

Manufacturing at Commercial Scale

IRT-103: formulation completed, cGMP manufacturing agreement

CYTOCOM: BROAD CLINICAL PROGRAM


CYTOCOM IMMUNOTHERAPIES VS

CURRENT THERAPIES

IRT-101 & IRT 103

• Rebalances and activate the immune system

• Shifts the balance between Th1 & Th2 lymphocytes

• Increasing cytotoxic T cells and natural killer (NK) cells

• Regulating cell growth & inhibiting tumor growth

• Fewer opportunistic infections • Single dose daily (IRT-103)

IMMUNOSUPPRESSION DRUGS

• Suppress the body’s immune system

• Regular blood tests are required • Any infections that develop will be

more difficult to treat • These drugs increase the likelihood

of uncontrolled bleeding due to injury or infection.

• Cause cancer with long term use


COMPLETED PHASE II TRIALS: IRT-103

IRT-103 Phase II Clinical

Trials

HIV / AIDS

Cancer

Immune Deficiency

Inflammatory Disease

Crohn’s Disease Fibromyalgia

Multiple Sclerosis

Autism


IRT-103 (LOW DOSE NALTREXONE) An alternative and safe approach for immune dysfunction and the chronic inflammatory issues and cancer

Regulating cell growth & inhibiting tumor growth

increases production of CD4, T cells, Natural killer (NK) cell

Regulates and shifts (Th1 and Th2) and inflammatory cytokines

Decreases TNF-α and other inflammatory cytokines from inflammatory cells

Proof Of Concept: Crohn’s Disease, Fibromyalgia, HIV/AIDs, Autism

IRT-103 exerts its beneficial effects by reducing inflammation and oxidative stress, activating or rebalancing the immune system, and inhibiting cancer cell proliferation. IRT-103 in trials has shown to increase production of cytokines, in particular met-enkelphine that influences the activities of immune system cells and having distinct biochemical effects (e.g. growth factors, neurotrophic factors, antiviral activity, anti-tumor activity, anti-inflammatory and pro-inflammatory effects).


CURRENT CLINICAL PIPELINE: IRT-103

Product Description Preclinical IND-Track Phase I Phase II Phase IIb Phase 3 Regulatory Approval

LDN / IRT-103

Crohn’s Disease ✔ ✔ ✔ ✔ Q4 2015 Q4 2016 Q4 2018

Multiple Sclerosis ✔ ✔ ✔ ✔ Q1 2015 TBD TBD

HIV Associated Cancer ✔ ✔ ✔ ✔

Q4 2015 TBD TBD

Fibromyalgia ✔ ✔ ✔ ✔ Q1 2016 Q2 2016 Q1 2019

Various Cancer ✔ ✔ ✔ ✔ TBD * *

Ulcerative Colitis ✔ ✔ ✔ ✔ TBD * *

Autism ✔ ✔ ✔ ✔ Q3 2016


REGULATORY ESTABLISHED PATHWAY IRT-103 CROHN’S DISEASE

Recognition of Benefit/Risk in the treatment of treatment moderate to server Crohn’s Disease

Orphan DFDA Guidance Path to Phase 3 Efficacy Trials

IND: IND 67442

Submit Final Protocols Phase IIB/III for Adult & Pediatric Crohn’s Disease in parallel Q4 2015

Fast Track Designation Q4 2015 Clinical trials shows IRT-103 to be significantly less toxic then

available therapy


COMPLETED CROHN’S CLINICAL TRIAL

Patient Population Study Design Efficacy Results

Adults with Moderate to Severe Crohn’s CDAI 220-450

Pilot study 17 patients Open Label 4.5 mg po qd 12 wks tmt, 4 wks f/u

Week 12 % response1 = 88% % remission = 47%

Adults with Moderate to Severe Crohn’s CDAI > 220

Phase 2 34 patients (LDN = 18, PBO = 16) RDBPC 4.5 mg po qd (or PBO) 12 wks DB, Â 12 wks OL, 4 wks f/u

Week 12 % response2 •  LDN = 63%, PBO = 33%

Children (6-17 yo) with Moderate to Severe Crohn’s PCDAI > 31

Phase 2 12 patients (LDN = 6, PBO = 6) RDBPC 0.1 mg/kg po qd (or PBO) 8 wks DB, Â 8 wks OL, 4 wks f/u

Week 8 % response1 •  LDN = 67%, PBO = 12%


IRT-103: REGULATORY STATUS

•  FDA Briefing Package Submitted

–  Protocols for a Phase IIB/III study

–  Phase I & II supporting date Crohn’s Disease : IND 67442

–  Phase I 1 Clinical safety data from studies conducted under IND 6744

–  Included Toxicology Studies

•  Type B Meeting held with the FDA

–  Modified protocols Phase IIB/III O1 2015 in parallel

–  505(b)(2) pathway approved

IRT 103 Formulation Developed

•  Oral Adult Dose (.05mg; 1mg, 3mg; 4.5mg/day)

•  Pediatric oral liquid formulation of LDN (0.1 mg/kg/day).

•  Commercial Drug Manufacture Selected

–  cGMP approved

•  Orphan Drug Designation


Estimated Study Timelines FDA Submissions & Interactions

Confidential

3Q 4Q 1Q 2Q 3Q 4Q 1Q 2Q 3Q 4Q

2015 2016 2017

Phase 1 PK

Provide Phase 1 PK

Data

Submit Phase 2 and Phase 3 protocols to

FDA for comment Initiate Phase 2

and Phase 3 clinical trials in

parallel

Obtain Phase 2 data on optimal dose to confirm

Phase 3 study dose

Phase 3 interim analysis (24-weeks post

enrollment of final subject)

If compelling Phase 3 interim analyses data –

request pre-NDA meeting

CROHN’S DISEASE DEVELOPMENT PLAN


IRT-103: ESTABLISHED PATENT PROTECTIONS Expanding immune stimulation to revolutionize the fight against cancers, autoimmune & infectious diseases

Existing Patents:

Crohn’s Disease, Inflammatory bowl diseases, Multiple Sclerosis, HIV/AIDS, adjunct to chemotherapy, chronic fatigue,

neurodegenerative diseases, cancer valid from 2019-2028

Orphan Drug Status Provides 7 year market exclusivity in U.S.

Provides 10 year market exclusivity in E.U. and Japan when obtained

505(b)(2) Designations Provides market exclusivity in the U.S. for between 3 and 7 years

Ongoing Preclinical Work Providing Additional IP Opportunities


COMPLETED CLINICAL & PRE-CLINICAL TRIALS: IRT-101 MET-ENKELPHINE (MENK)

IRT-101 Clinical &

Pre Clinical Trials

HIV / AIDS

Pancreatic Cancer

Immune Deficiency

Inflammatory Disease Hepatoblastoma

Multiple Sclerosis

Kaposi’s


IRT-101 MET-ENKELPHINE Cancer Immunology

increasing proliferation and functional activities of CD4+T-cells and CD8+T-cells

which will play a role in anti-virus and anti-tumor activities

increases production of CD3, CD4, CD8, T cells, Natural killer (NK) cell

increasing secretion of cytokines such as IL-2, TNF, IL-12 and IFN-γ s, forming a modulated and

balanced immunity

The safety and toxicity of MENK has been determined in Humans in Phase II trials

Proof of Concept: Phase II trials with HIV/AIDS and Pancreatic Cancer

IRT-101 met-enkelphine (“MENK or OFG”) is a small peptide that triggers immune response. IRT-101 have been modified for a stronger binding with immune response receptors causing an increase in circulating cytotoxic T cells (CD8+/TH1) and natural killer cells (“NK cells”) IRT-101 triggers a stronger immune response and target tumor growth associated antigens expressed in several cancers, and can be used as an adjunct to chemotherapy to off-set toxic effect


CURRENT CLINICAL PIPELINE IRT-101

Product Description Preclinical IND-Track Phase I Phase II Phase IIb Phase 3 Regulatory Approval

MENK / OGF / IRT-101

Chemotherapy Adjunct ✔ ✔ ✔ ✔ ✔ Q1 2016 2018

Pancreatic Cancer ✔ ✔ ✔ ✔ Q1 2016 Q1 2017 2019

Ovarian Cancer ✔ ✔ ✔ ✔ TBD * *

Hepatocellular Cancer ✔ ✔ ✔ ✔ TBD Q1 2016 2018

HIV/AIDS ✔ ✔ ✔ ✔ Q2 2016 * *


REGULATORY ESTABLISHED DEVELOPMENT PANCARATIC CANCER PATHWAY: IRT-101

Recognition of Benefit/Risk in the treatment of treatment unresectable pancreatic cancer Methionine [Met5]-enkephalin

(MENK)

Orphan DFDA Guidance Path to Phase 3 Efficacy Trials

IND: 50,987

Submit Final Protocols Phase 2 B for pancreatic and hepatic cancer in combination with standard of care chemotherapy.

Fast Track Designation Clinical trials show that IRT-101 is less toxic then available therapy that are the common and causes for discontinuation of treatment.


Survival comparing OGF-treated subjects to untreated controls. OGF-treated patients survived significantly longer than untreated controls. ***p<0.001).

OGF

All patients had metastatic unresectable cancer

PHASE 2 TRIAL: SURVIVAL PANCREATIC CANCER PATIENTS WITH MENK /OGF


IRT-101: REGULATORY STATUS •  FDA Briefing Package Submitted – Included protocols for a Phase IIB/III study

– Included Phase I & II date Pancreatic Cancer: IND 50,987 studies and IRB Protocol No. 26336;

– Included Phase I & 11 Clinical safety data from studies conducted under IND 34,442

– Included Toxicology Studies

•  Type C Meeting FDA

– Submit modified protocols for a Phase IIB O1 2015

– Orphan Drug Designation

•  Potential Study Sites Identified •  Commercial Drug Manufacture Selected

– cGMP approved

Cytocom, Inc – May 2015 - Confidential 20 Confidential

3Q 2015 2Q 2016 3Q 2016 4Q 2016 1Q 2Q 3Q 4Q

2015-2016 2017

Initiate 3-mo GLP Toxicology Studies

(rat & dog) Started

Initiate Phase 1 PK study in healthy volunteers

Submit Phase 2 data to support initiation of

Phase 3

Initiate Phase 3

MENK DEVELOPMENT PLAN: KEY ACTIVITIES *ESTIMATED TIMELINE*

Initiate Phase 2 Randomized

Study

Submit 3-mo GLP Toxicology Reports


ITR-103: ESTABLISHED PATENT PROTECTIONS Expanding immune stimulation to revolutionize the fight against cancers, autoimmune & infectious diseases

Existing Patents:

Methods for Inducing Immune Response, adjunct to Chemotherapy, Pancreatic Cancer, combination therapy with

OGF, curing blood medulla hematopoietic system cancer valid 2023-2028

Orphan Drug Status Provides 7 year market exclusivity in US

Provides 10 year market exclusivity in E.U. and Japan when obtained

Ongoing Preclinical Work Providing Additional IP Opportunities


$212 Billion Market Potentials

Auto-Immune Market US$68 billion by 2014

Cancer Drug Market 2011-2018 to reach US$109 billion.

Adjunct to Chemotherapy US$35 billion a year.


MARKET POTENTIALS EXPLORED

Autoimmune

• Auto Immune Market is significant and the global autoimmune treatment market is expected to be worth US$68 billion by 2014, with an estimated CAGR of 12.7% from 2009 to 2014. Although this market encompasses multiple clinical conditions, three indications, RA, MS and Psoriasis, represent approximately 90% of market sales.

Cancer

• Cancer, with a death toll exceeding that of AIDS (Acquired Immune Deficiency Syndrome), tuberculosis and malaria collectively, accounted for 8.3 million deaths in 2013. With a skyrocketing incidence of 469.6 per 100,000 in the US alone, the oncology market is poised to grow at a substantial CAGR of around seven percent to $109 billion by 2020/

Chemotherapy Adjunct

• Adjunct To Chemotherapy Drugs that unleash the power of the immune system on cancers are generating considerable optimism in industry, but still Andrew Baum thinks analysts are selling them short. In a 22 May, 2013 report, Baum, the London-based head of global health-care research at the investment bank Citi, forecasts that in ten years the drugs will be treating 60% of cancers and earning US$35 billion a year.


RECENT CLOSED IPO: IMMUNOTHERAPY AND ONCOLOGY Date Target C Target Overview Share

Outstanding Transaction

Structure

June 2015

RVT -101 Dementia Axovant Science

Phase 3-ready product for the treatment of moderate-to-severe dementia (GSK licensed the patents )

96,000,000 Option

3,100,000

$315,000,000 IPO Market Valuation

$2.72 billion Dollar

May 2015

NY-ESO-TCE Synovial Sarcoma Multiple Myeloma

Adapatimmue Phase I/2a product in development for synovial and multiple myeloma

70,785,313 Conversion

175,841,800

$191,250,000 IPO Market Valuation $115 Billion Dollar

May 2015

CRS-107, ADU-523, ADU-0741, ADU-214

Aduro Biotech

Pipeline products Phase 2B pancreatic Mesothelioma phase I, ,Prostate lung per-clinical

58,000,000 Options


2 billion Dollar

May 2015

CD 101 IV ,CD 101 COO1, C016 Candidemia, Acute & Recuing VVC and Invasive Aspergillois

Cidara Therapeutics Inc

Pipeline Candidemia phase I and Acute & Recurring VVC phase ½ and Invasive Aspergillosis & Bacterial & Viral per-clinical

13,494,118 Preferred

conversion 89,360,118


$191,000,000


ACQUISITION: IMMUNOTHERAPY AND ONCOLOGY Date Target Acquirer Target Overview Purchase

Price Deal Structure

Apr. 2014

GED-0301 (from Nogra Pharma)

Celgene Phase 3-ready product for the treatment of moderate-to-severe Crohn’s disease

$1.5 billion $710 million upfront, $815 million in milestones, Tiered royalties on net sales

Apr. 2014

ORY-1001 plus Oryzon’s backup programs

Roche Phase I/2a product in development for acute myeloid leukemia

$521 million $21 million upfront, $500 million in milestones

Mar. 2014

Five Prime Therapeutics

Bristol-Myers Squibb

Pipeline products for solid tumors

$350 million $20 million upfront, $9.5 million for research work, $21 million equity stake, $300 million in milestones.

Feb. 2014

CoStim Pharmaceuticals

Novartis Immuno-oncology company developing monoclonal antibody drugs that enable a patient's own immune system to better fight cancer.

Undisclosed Company Acquisition


CYTOCOM OPPORTUNITY

PROMISING PIPELINE

IMMUNOLOGY; CROHN’S DISEASE, MS, FIBROMYOLOGIA , AUTISM, CANCER

ONCOLOGY: PANCARATIC, HEMPATIC, OVERIAN, BREAST & PROSTATE CANCER

ORPHAN DRUG DESIGNATION

7 YEAR EXCLUSIVITY U.S. & 10 YEAR E.U. & JAPAN

HIGHLY EFFICENT PATH TO COMMERLIZATION

505(B)(2) DESIGNATION DECREASES TIME TO MARKET & REDUCING RISK AND COST

REPOSITONING STRATEGY NEW FORMULATION, NEW INDICATION LOW RISK


(888) 613-8802

37 North Orange Ave, Suite 607

Orlando FL 32801

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