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OncoTrackerJames Berenson, MDPresident and CEO
November 2014
Institute for Myeloma and Bone Cancer Research (IMBCR) spin-out (founded in 2014)
Our discovery: a marker for multiple myeloma known as BCMA
We developed a simple assay to measure BCMA from a blood sample
BCMA is a better marker than current gold standard for myeloma
Potential initial market opportunity: $559M
Future markets include many common cancers (leukemia, lymphoma)
Revenues will start in 2015
Cash flow positive in 2016
Currently Financing $300k via Convertible Note Finalize assay for testing Organizae Lab for CLIA certification Complete Business Plan for $3mm Share Sale
Overview of OncoTracker
Tumor Markers Patients’ genes and proteins are increasingly being measured to
diagnose and manage their cancers Examples: BRCA1, HER2/neu, Oncotype DX
These markers can help in designing personalized treatment
The Ideal Tumor MarkerTesting requirements:
Easily available source of tissue – e.g., blood sample
Simple and reproducible test Accurate
Clinical requirements:Found in nearly all patientsAccurately correlates with disease to:
Predict patient outcomeMonitor response to treatment
Our Focus: Multiple Myeloma
Common cancer of the bone and bone marrow24,000 new cases annually in
USOver 100,000 patients in US
Bone fractures, pain, kidney failure, etc.
Many frail and elderly patients
Median survival of 7 years
Treating MyelomaMany treatment options available
todayInitial status of disease important in
treatment planRequires a tailored approach based on
prognosis
An accurate and predictive tumor marker has potential to play a major role in this disease
Approach to Treatment Depends on Initial Status
20%
10%
70%
Aggressive Disease - Patients Need Immediate Therapy _x000d_Smoldering - Wait and See ApproachIn Between
Current Treatments Large number of drugs used today: Velcade, Kyprolis, Revlimid,
Thalomid, Pomalyst, Dexamethasone, Medrol, Prednisone, Cytoxan, Doxil, Trisenox, Melphalan, Oncovin, Treanda, and Adriamycin
These are often combined together resulting in many different treatment regimens
Ideally, administer therapy that: Keeps the disease under control
AND Has the least side effects
Treatments are designed based on patients’: Disease status General characteristics (age, overall health, etc.)
Challenges of Treating Myeloma
Three major questions that need to be addressed: Who needs treatment? (Diagnostics) Will it be effective? (Prognostics) How can we track it? (Monitoring)
Answering these questions guides therapy: Starting treatment that is appropriate for the patient Enabling therapy to be modified if necessary
Get off drugs that are not working – reduces exposure to toxic agents Move to alternative therapies
Allowing patients to remain on effective drugs
Tumor markers have the potential to help address these questions and guide treatment
The Gold Standard Myeloma Marker:
M-Protein
A protein secreted by myeloma tumor cells
Testing: simple and straightforward Routine blood
and/or urine sample
Standard assay for measuring proteins
Elevated levels in myeloma patients
M-Protein Has Limitations for Clinical Uses
Diagnosis: 5-10% of patients do not have measurable M-protein
Prognosis: levels do not predict patient outcomes
Monitoring: levels change slowly over timeDifficult to assess current
disease stateProblematic to track cancer
and modify treatment
Our Solution – BCMA
We discovered that a protein, BCMA, was a tumor marker for myeloma Shed from tumor cells into
the blood Correlates with disease status Accurately measures the
current disease status Can be used to quickly
determine response to treatment
Based on research conducted at the IMBCR
Our Solution – BCMA Assay
We developed a simple test to measure BCMA levelsRequires small blood
sampleRoutine testing formatStandard instrumentAccurate and
reproducible results
BCMA Tracks w/ M-Protein Levels in Individual MM Patients
BCMA Can Be Used to Track a Multiple Myeloma Patient without M-Protein
BCMA Levels Predict Treatment Response% factor of control
Serum BCMA Levels are Higher in Multiple Myeloma Patients than MGUS or Controls
BCMA Levels Correlate With Disease Status
Progression Free and Overall Survival Rates in Multiple Myeloma Patients
Median progression free survival following first treatment: 2 years
Importantly, progression-free survival shortens with each subsequent treatmentThus, finding out quickly that treatments are not
working becomes of increasing importance
Median overall survival: 7 years
BCMA Predicts Progression Free Survival During Frontline Treatment
BCMA Predicts Progression Free Survival During Salvage Therapy
BCMA Predicts Overall Survival
Market For Current Diagnostic Tests
Laboratory tests Blood- SPEP, quantitative immunoglobulins, serum free light
chains Urine- UPEP w/ 24-hour urine total protein Routine CBC and chemistries Beta-2-microglobulin
Bone marrow aspirate and biopsy Pathology Genetics- cytogenetics, fluorescent in situ hybridization,
genetic expression profiling
Bone imaging- X-rays, MRI, PET CT scan
Market for Tests to Monitor Myeloma
Routine testsBlood- SPEP, quantitative immunoglobulins, serum
free light chainsUrine- UPEP w/ 24-hour urine total protein
SpecializedNew bone disease- MRI, PET CT scan Bone marrow
How Does it Compare to Other Markers?
Cost Ease of Testing
Diagnosis
Prognosis
Monitoring
M-Protein + + + - +/-
Beta-2 microglobul
in
+ + - - -
Bence Jones protein
+ - - - +/-
Bone marrow biopsy
- - + - -
Radiology - - - - +
BCMA + + + + +
Increasing Size of Cancer Market
Estimated at 5% Per Year
Bill
ions
USD
2014 2015 2016 2017 2018 20190
20
40
60
80
100
120
140
Rapidly Growing Tumor Marker Market
Estimated at 18.1% Per Year
Bill
ions
USD
2014 2015 2016 2017 2018 20190
1
2
3
4
5
6
7
Multiple Myeloma Market
24K new patients per year
100K patients per year
$4.4B market for multiple myeloma drugs in USA
Patients undergo monthly testing to monitor disease
Total of 1.3M evaluations for 100K patients1.3M M-Protein measurements6.5M total tests: battery of 5 tests, including M-
Protein
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