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
Cytocom, Inc – May 2015 - Confidential 2
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.
Cytocom, Inc – May 2015 - Confidential 4
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, Inc – May 2015 - Confidential 5
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
Cytocom, Inc – May 2015 - Confidential 6
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
Cytocom, Inc – May 2015 - Confidential 7
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).
Cytocom, Inc – May 2015 - Confidential 8
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
Cytocom, Inc – May 2015 - Confidential 9
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
Cytocom, Inc – May 2015 - Confidential 10
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%
Cytocom, Inc – May 2015 - Confidential 11
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
Cytocom, Inc – May 2015 - Confidential 12
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
Cytocom, Inc – May 2015 - Confidential 13
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
Cytocom, Inc – May 2015 - Confidential 14
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
Cytocom, Inc – May 2015 - Confidential 15
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
Cytocom, Inc – May 2015 - Confidential 16
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 * *
Cytocom, Inc – May 2015 - Confidential 17
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.
Cytocom, Inc – May 2015 - Confidential 18
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
Cytocom, Inc – May 2015 - Confidential 19
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
Cytocom, Inc – May 2015 - Confidential 21
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
Cytocom, Inc – May 2015 - Confidential 22
$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.
Cytocom, Inc – May 2015 - Confidential 23
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.
Cytocom, Inc – May 2015 - Confidential 24
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
$119,000,000 IPO Market Valuation
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
$65,000,000 IPO Market Valuation
$191,000,000
Cytocom, Inc – May 2015 - Confidential 25
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, Inc – May 2015 - Confidential 26
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
Cytocom, Inc – May 2015 - Confidential 27
(888) 613-8802
37 North Orange Ave, Suite 607
Orlando FL 32801
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