OncoTracker James Berenson, MD President 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 Marker Testing requirements: Easily available source of tissue – e.g., blood sample Simple and reproducible test Accurate Clinical requirements: Found in nearly all patients Accurately correlates with disease to: Predict patient outcome Monitor response to treatment
Our Focus: Multiple Myeloma Common cancer of the bone and bone marrow 24,000 new cases annually in US Over 100,000 patients in US Bone fractures, pain, kidney failure, etc. Many frail and elderly patients Median survival of 7 years
Treating Myeloma Many treatment options available today Initial status of disease important in treatment plan Requires 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
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 time Difficult to assess current disease state Problematic 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 levels Requires small blood sample Routine testing format Standard instrument Accurate 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 treatment Thus, 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 tests Blood- SPEP, quantitative immunoglobulins, serum free light chains Urine- UPEP w/ 24-hour urine total protein Specialized New bone disease- MRI, PET CT scan Bone marrow
How Does it Compare to Other Markers? CostEase of Testing DiagnosisPrognosisMonitoring M-Protein +++-+/- Beta-2 microglobulin Bence Jones protein +---+/- Bone marrow biopsy Radiology BCMA +++++
Increasing Size of Cancer Market Estimated at 5% Per Year Billions USD
Rapidly Growing Tumor Marker Market Estimated at 18.1% Per Year Billions USD
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 patients 1.3M M-Protein measurements 6.5M total tests: battery of 5 tests, including M-Protein