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Development of a new class of personalized targeted cancer drugs taken by mouth.
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OncoStem Biotherapeutics LLCDiscovery Stage Pharmaceutical Company
Founded September 2012 Incorporated September 2012
Developing novel small molecule drugs as targeted therapies (‘smart bombs’) against proven drivers of
cancer
Executive Leadership-
Rudolph E Willis, MD. CEO Drexel University
Scott M Kahn, PhD. CSO Columbia University International Cancer Advocacy Network
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OncoStem Biotherapeutics LLCSolutions for Personalized Cancer Care
U.S. cancer statistics – 20121
1,638,910 million new cases 577,190 DIED
European cancer statistics – 20122
3.2 million new cases 1.3 MILLION DIED
1 -CA Cancer J Clin. 2012 Jan-Feb;62(1):10-29. doi: 10.3322/caac.20138. Epub 2012 Jan 4.2- World Health Organization
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OncoStem Biotherapeutics LLCSolutions for Personalized Cancer Care
SYMPTOM-BASED PERSONALIZED
Susanne Harris- Nature Drug Discovery April 2009 Vol. 8, No.4
Cosmas and Damian Healing the SickFrancesco di Stefano Pesellino 1440-1445
We are experiencing a paradigm change in how cancer patients are treated, from a “one size fits all” approach to a scientifically based,
personalized approach based on molecular profiling of individual tumors
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OncoStem Biotherapeutics LLCSolutions for Personalized Cancer Care
RISKASSESSMENT
PREVENTION TARGETEDMONITORING
EARLY DETECTION TESTING
DIAGNOSIS PERSONALIZEDAND TARGETED
THERAPY
RESPONSEMONITORING
Adapted from www.personalizedmedicinecoalition.org
NEW PARADIGM OF PERSONALIZED CANCER TREATMENT
DIAGNOSIS SELECTTHERAPY
SWITCHTHERAPY
AT DISEASEPROGRESSION
OLD PARADIGM- TREATMENT OF DISEASE
Cutting-edge cancer care is now based on molecular profiling
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OncoStem Biotherapeutics LLC
Addressing a need in drug development by combining
the best of academia and industryProblem: Only a limited number of targeted therapies are currently available for cancer patients. Scores of targets remain undrugged.
Problem: Pharma’s nature is the lemming approach, to develop improved versions of existing drugs: e.g. PDE5, PK inhibitors inhibitors, etc.
Problem: Pharma has significantly divested itself of early drug development programs and has turned to academia to identify promising therapies
Problem: The historically conservative nature of funding agencies has handcuffed Academia’s ability to develop new targeted therapies
Oncostem Biotherapeutics tackles these problems providinginvestors with an opportunity to enter the higher risk/high rewardtargeted therapy drug development field on the ground floor.
INNOVATION
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OncoStem Biotherapeutics LLCSolutions for Personalized Cancer Care
Addressing a need in drug development by combining
the best of academia and industry
Problem: Many potential cancer targets remain undrugged. Academia’s forte is target discovery
Pharma’s forte is clinical trial design
Oncostem Biotherapeutics’ forte is translational development
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OncoStem Biotherapeutics LLCSolutions for Personalized Cancer Care
Dr. Willis developed a proprietary algorithm that identifies and ranks potentially druggable oncogenic
drivers of cancer
Vital Oncogene Data Base Characteristics
Over 600 targets of interest identified (OST1-600). Each with some or all of the required vital oncogene characteristics.
Approximately 100 fulfill stringent criteria.
Approximately 50-75 fulfill stringent criteria and have significant supportive literature.
Only a few targets within our database have been seriously investigated for drug production (STATs, NOTCH, NF- kB.)
All others are excellent candidates as drug targets.
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Examples of lead vital oncogene targets
OST-1
Biological process: Transcription regulationPost translation modification: PhosphoproteinLinked to four cancer types
OST-2
Biological process: development, differentiation and lineage commitment. Linked to three cancer types
OncoStem Biotherapeutics LLC
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Oncostem Biotherapeutics’ scientists are proven experts who will spearhead Targeted Therapy and Companion Diagnostic
Assay (CDx) co-development
DiscoveryResearch
Pre-clinicalClinicalPhase I
ClinicalPhase II
ClinicalPhase III
Post approval
BiomarkerSelection
FeasibilityStudies
PrototypeAssay
AnalyticalValidation
ClinicalValidationand Utility
ValidatedCDx Assay
Targeted Therapy Development
Companion Diagnostic Assay Co-Development
Responders, no toxicity
Non-responders
Responders, toxicity
Non-responders, toxicity
CANCER PATIENT POPULATION
BIOMARKER BASED STRATIFICATION TO IDENTIFY TARGETED THERAPY CANDIDATES
STRATEFIED CLINICAL TRIALS
OncoStem Biotherapeutics LLC
CDx Assay
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OncoStem Biotherapeutics LLCSolutions for Personalized Cancer Care
Oncostem’s Blue Ocean
POISED TO START DRUG DEVELOPMENT
Oncostem Bio’s Proprietary Weighted Data Base combines Bioinformatics, Systems Biology, Genomics, Network Theory, and Market Analysis to rank ‘vital oncogenes’ that drive cancer growth.
Disruptive Innovation- The protein products of these vital oncogenes are targets for the development of new chemical small molecule targeted anti-cancer drugs.
Validation- We performed an expert literature review to deliberately dissect the scientific evidence behind our most highly ranked vital oncogenes. Following a careful and detailed market landscape analysis, we selected preferred lead commercial targets.
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Target Market
1. Global market for cancer therapy $47.3 billion in 2008.2. Rise to $110.6 billion by 2013 (CAGR 12.6%)3. Target therapy segment $22.9 billion in 20084. Rise to $69.1 billion by 2013 (CAGR 24.7%)5. Target therapy comprised 45.0% of overall market in 20075. Target therapy market share has increase to 62.5% in 2013
www.bccresearch.com
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OncoStem Biotherapeutics LLCDiscovery Stage Pharmaceutical Company
Competitive Advantage
1. Unique application of Bioinformatics to Systems Biology and Network Theory
2. Proprietary driver cancer gene target database
3. Potential resource pipeline for the production of multiple drugs, lower cost, patient appeal
4. Pursuit of targets not chosen by BioPharma or Biotech companies
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OncoStem Biotherapeutics LLCSolutions for Personalized Cancer Care
Business Model- Drug Development Strategy
Current Top vital oncogene targets identified
Reagents Commercially Available
2013-2014 Screening of libraries, Identification of lead compounds
2014-2016 Detailed analysis of lead compounds,Target specificity, PD, PK, Toxicology, Biochemical modification, animal
studies
2015-2016 Patenting of lead drugsCompanion diagnostics
2016-2017 Pharma [email protected]
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OncoStem Biotherapeutics LLCSolutions for Personalized Cancer Care
Business Model- Funding
Current Privately funded
Near term Angel Investors- Seeking $1.5-3.5M
December 2013 SBIR Phase I application $225,000
Intermediate term SBIR Phase II application $1.0M
Longer term Drug partnerships(3 years out)
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OncoStem Biotherapeutics LLCSolutions for Personalized Cancer Care
Business Model- Estimated Budget years 0-3
Salaries, Advisors, Infrastructure ~$240,000- 400,000/year
Small molecule screen ~$150,000 per target
Drug development, IP costs ~$600,000 per target
One Target: seeking $1.5M investmentThree Targets: seeking $3.5M investment
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OncoStem Biotherapeutics LLCSolutions for Personalized Cancer Care
DATE PARTIES VALUE SUBJECT
April 2013 AlchemiaAstra Zeneca
$240 million Collaboration formulti-target drug
Jan 2012 FORMA TherapeuticsJanssen Biotech
$700 million Collaborative forsmall molecule drugs (metabolism)
Jan 2012 Boehringer IngelheimFORMA Therapeutics
$700 million Collaborative for small molecule drugs
Dec 2011 Janssen BiotechMetamark Genetics
$365 million Collaborative for identification of proprietary cancer targets
High reward potential for successful drug development
Extracted from: Current Agreements
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OncoStem Biotherapeutics LLCSolutions for Personalized Cancer Care
IN SUMMARY
• Independent of academia Maintain benefits of connections at universities Avoid academic overhead Retain full control over IP
• Pioneering experts in oncology, ‘oncogene addiction’, biomarkers, and functional drivers of cancer
• We seek seed support and working capital
• Potential year 3 first exit point following production of first optimized drug lead and out-licensure to BioPharma
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Questions ???
Contacts: Dr. Rudolph Willis 484 273-8367 [email protected]
Dr. Scott Kahn [email protected]
