Corporate Presentation
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Biotheranostics Overview
Commercial stage molecular diagnostics company
CLIA and CAP accredited lab based in San Diego,
129 employees
Recently spun-out of bioMérieux; VC backed
Two high value proprietary tests on the market, addressing large unmet medical needs
Only one of three companies to have two tests approved for Medicare coverage through the MolDX process, which are subject to specific coverage criteria set forth in a Local Coverage Determination (LCD).
Entering a period of rapid growth
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Biotheranostics History
2010 2011 2012 2013 2004 2006 2008
CTID obtains Medicare
coverage (LCD)
Key BCI clinical studies
published
Key CTID clinical studies
published
CancerTYPE ID (CTID)
biomarker discovery initiated
Commercial launch of
CTID
Breast Cancer Index (BCI) biomarker discovery initiated
AviaraDx acquired by
bioMérieux & renamed
bioTheranostics
CTID & BCI health
economics studies published
Full commercial launch for
BCI
BCI obtains Medicare
Coverage (LCD)
bioT3 released
2014 2016
Commercial ramp for BCI
2015
$32M funding and spinout from bMX
Our Organic Growth Strategy in a Nutshell
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Awareness & Advocacy Sales
Coverage
High Volume Growth
Increased reimbursement ASP
High Revenue Growth
Commercial Decision impact & health
economic studies to support commercial payers contracts
Guidelines Customer experience (lab ops &
client services)
Expanding BCI’s indications (e.g., chemo prediction, neo adjuvant, metastatic)
Cash collection & appeals effectiveness
Levers of Growth
Our Advanced Molecular Tests Target Large Unmet Medical Needs, Cancer Patient Populations and Markets
~ $800M < 3%
~ $300 M < 5%
~ 170k incident patients per year
~ 500k prevalent pool
of patients
~ 100k incident patients per year for all unclear diagnoses
• Early & late recurrence risk • Likelihood of benefit from
endocrine treatments
• Molecular classifier & diagnosis • Biomarkers linked to targeted first
line treatments for metastatic cancer
Epidemiology US Market Size & Penetration
Source: Mattson-Jack Cancer !Mpact database (2014) 5
Patient Journey for Early-Stage, ER+ Breast Cancer
2 – 4 weeks 0 or 3 mo 0- 5/10 years
Treatments Chemo
Radiation
Chronic endocrine treatment (5-10 years)
Diagnosis
Pre-surgical Chemo
Incidence
~170 k/year
Prevalence between years 3-8
~500k
Oncotype DX™
Surgery
Source: Physician interviews, Mattson-Jack Cancer Mpact database (2014)
-
BCI
Epidemiology (for ER+
early-stage patients)
Long-term treatment
+/-
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Challenging Risk vs. Benefit Profile Of Endocrine Drugs Complicates Decision-Making
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ATLAS Trial1 (Tamoxifen for 10y vs 5y) For every 600 women treated,
16 recurrences were prevented At the cost of 2-3…
5 endometrial cancers 2 pulmonary emboli
MA.17 Trial4 (Extended letrozole vs placebo) For every 600 women treated,
+ Long term side effects and QoL impairment2-3
12 recurrences were prevented At the cost of 5-9…
13 new cases osteoporosis 4 bone fractures 1 thromboembolic event
+ Long term side effects and QoL impairment
1. Davies C et al., Lancet. 2013 ;381(9869):805-16. 2. Nolvadex prescribing information. http://www.fda.gov/downloads/Drugs/DrugSafety/ucm088661.pdf. 3. Conzen, SD. Managing the side effects of tamoxifen. In: UpToDate, Dizon DS (Ed), UpToDate, Waltham, MA. (Accessed on March 11, 2015.) 4. Goss PE et al., N Engl J Med 2003;349. 5.Arimidex prescribing information. http://www1.astrazeneca-us.com/pi/arimidex.pdf 6. Femara prescribing information. https://www.pharma.us.novartis.com/product/pi/pdf/Femara.pdf 7. Aromasin prescribing information. http://labeling.pfizer.com/ShowLabeling.aspx?id=523 8. Fallowfield LJ, et al. Breast Cancer Res Treat. 1999;55(2):189-99. 9. Crew KD, et al. J Clin Oncol. 2007 Sep 1;25(25):3877-83.
Clear Unmet Medical Need
To reliably identify which patients are likely to benefit from extended endocrine therapy and which are not
5 endometrial cancers 2 pulmonary emboli
+ Long term side effects and QoL impairment2-3
13 new cases osteoporosis 4 bone fractures 1 thromboembolic event
+ Long term side effects and QoL impairment
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Sept 2013 Lancet Oncology Editorial
There is only one validated predictive test available today:
“So, is the BCI test ready for prime time in treatment decision making for women who have undergone 5 years of hormonal therapy?
The answer is yes.” “The BCI test has level 1B evidence for this indication.”
Addressing a Key Unmet Medical Need
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BCI Predictive Predicts Likelihood of Benefit
From Hormonal Therapy
BCI Prognostic Assesses Individual Risk of Recurrence from 0-10 years
Initial focus is on the area of greatest unmet need:
Late recurrence risk (5-10 yrs) Benefit from extended endocrine therapy (5-10 yrs)
Second Generation Molecular Diagnostic Test With Two Components
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• Consider completion of endocrine Rx at 5 years • For patients already on extended endocrine Rx, consider stopping ~45%
Low Risk/Low Likelihood of Benefit
• Consider extending endocrine Rx beyond year 5 • For patients beyond year 5 who are off treatment, consider restarting
endocrine Rx • Emphasize importance of compliance and adherence
~30%
High Risk/High Likelihood of Benefit
• Consider additional and other therapeutic approaches for risk reduction and more frequent monitoring ~15%
High Risk/Low Likelihood of Benefit
• Consider continuation of endocrine Rx beyond year 5 if patient is tolerating well and has no concerning comorbidities
~10%
Low Risk/High Likelihood of Benefit
Clinically Actionable Information that can impact her treatment plan
Notes: % of patients based on clinical trials and clinical experience. *For node-negative patients. BCI Prognostic was validated in a cohort that included LN- patients only. Any LN+ patient should be
viewed as higher risk and managed accordingly. For all patients, clinical decisions require incorporation of BCI results with all other clinicopathologic factors
The Breast Cancer Index Protocol
• Individualized risk of late recurrence
• High / Low Risk
• Predictive of likelihood of benefit from extended endocrine therapy
• High or Low (binary result)
RT-PCR Extract RNA
Patient Tissue Sample (FFPE)
Tumor dissection
BCI Prognostic
BCI Predictive (H/I ratio) (1) Biomarker: HoxB13/IL17BR (H/I) gene expression ratio
(2) Biomarker: Molecular Grade Index (MGI)1, which includes 5 cell cycle genes (BUB1B, CENPA, NEK2, RACGAP1, RRM2)
• Algorithm evaluates patient’s gene expression profile
• Interrogates estrogen signaling(1) and proliferative(2) pathways
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BCI Sample Test Report
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Breast Cancer Index Supported by Robust Clinical Evidence
Clinical Utility
Clinical Validity
Analytical Validity
Health Economics
& Outcomes
Technical robustness of biomarker
Pivotal studies (2013) Integration into clinical practice
Health and cost effectiveness
CCR
JNCI
Lancet Oncology
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BCI Pivotal Studies
The BCI clinical study program has demonstrated: • BCI significantly stratified patients
by risk of early and late recurrence • ~60% of patients have a low risk
(<3%) of late recurrence
• BCI significantly stratified patients by risk of early and late recurrence
• Oncotype DX could not predict risk of late recurrence
• Patients categorized as High BCI (H/I) had a significant benefit from extended therapy (67% reduction in risk of recurrence)
• Patients categorized as Low BCI (H/I) did not benefit
Risk Assessment for Early and Late Recurrence: Randomized controlled trial (Stockholm) and Multi-institutional cohorts
(Zhang et al. Clin Cancer Res. 2013)
Improved Performance Compared to Oncotype DX: Head-to-head study in a randomized controlled trial cohort (ATAC)
(Sgroi et al. Lancet Oncol. 2013)
Prediction of Patient Benefit from Extended Endocrine Therapy: Randomized controlled trial cohort (MA.17)
(Sgroi et al. J Nat’l Cancer Inst. 2013)
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Clinical impact of BCI in extended endocrine therapy decision-making
Individual extended endocrine therapy treatment decisions were changed in approximately 26% of cases
Study demonstrated a net decrease in patients on extended endocrine therapy Patient anxiety was reduced in ~50% of cases
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Prospective clinical decision-impact study led by Yale University (N=100 pts) Physicians and Patients completed questionnaires pre- and post-BCI
Treatment Changed
27%
Physician Recommendations for Extended Endocrine Therapy
Sanft T, et al. Breast Cancer Res Treat. 2015
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Actual Data from Clinical Utilization of BCI Confirms Broad Use and large market opportunity
Epidemiology: – Each year ~167k ER+ early stage breast cancer patients are newly
diagnosed – In addition, approximately ~515k patients are already deep in the
journey between years 3-8; about 70% of these still persist on endocrine treatments at ~5 yrs
There are two types of physicians; those that want to use strictly at the anniversary, and others who will also use the test among the prevalent pool of patients The effective market size for BCI ($800M) is a value between these two extremes
Prevalence between years 3-8 years ~ 515k* patients
T= 5 yrs T= 0 @ diagnosis T= 10 yrs
Incidence ~ 167k*
* Epidemiological estimates are for U.S. only in 2015 Source: Kantar Cancer !Mpact database (2014)
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BCI is Uniquely Positioned to Inform Extended Endocrine Decision, Similar to Oncotype DX for Chemo
Diagnosis 5 yrs 10 yrs
Predictive of Chemotherapy
Benefit
Prognostic (at Diagnosis)
Prognostic (Late Recurrence-
specific)
Predictive of Extended Endocrine
Therapy Benefit
Oncotype DX®
(Genomic Health) MammaPrint®
(Agendia) Prosigna™
(Nanostring)
Breast Cancer IndexSM
(Biotheranostics)
Number of Genes 21 70 50 11
Platform RT-PCR Microarray NanoString nCounter RT-PCR
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CUP Wide differential Differential Confirmatory Certain
Diagnosis
Clear Diagnosis
Incidence
Market dynamics
90 – 130K patients 470 – 510K patients
• CancerTYPE ID is the market leader among gene expression tests
– > 23,000 patient tumors analyzed to date
– 1,750 physicians ordered test in last 12 months
– Competitively well positioned vs. main players
• Crowded market • Only ~9% penetration because
solutions not designed for community practices needs
– Some provide incomplete information (e.g., only sequencing)
– Others provide too much information at very high cost
No Yes ~15% ~85%
Source: Mattson-Jack Cancer !Mpact database (2014)
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Addresses the Needs of a Broad Group of Metastatic Patients
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CancerTYPE ID is Supported by Robust Clinical Evidence
Clinical Utility
Clinical Validity
Analytical Validity
Health Economics
& Outcomes
CancerTYPE ID Pivotal Studies
The CancerTYPE ID clinical study program has demonstrated:
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87% accuracy High performance in metastatic
tumors, high grade tumors, and limited tissue biopsies
10% increase in overall accuracy compared to IHC (P = 0.019)
CancerTYPE ID accuracy was ≥IHC/Morphology in all tumor types examined
37% increase in overall survival
Clinical Validity: Blinded, peer-adjudicated, clinical study (N=790) led by 3 centers of excellence (UCLA, MGH, Mayo Clinic)
(Kerr et al. Clin Cancer Res. 2012;18(14):3952-60)
Clinical Utility: Increase in accuracy compared to standard of care IHC in a study led by the City of Hope Nat’l Medical Center
(Weiss et al. J Mol Diagn. 2013;15(2):263-9)
Patient Benefit: Prospective clinical trial led by Sarah Canon Research Institute demonstrated increase in overall survival in patients with cancer of unknown primary (Hainsworth et al. J Clin Oncol. 2013;31(2):217-23)
Clinical utility of CancerTYPE ID is becoming increasingly clear
22 Kim et al. Personalized Medicine Onc., 2013; 2: 68-76 28-site registry study, Data on file (2014)
Tumor type suspected
or in differential Diagnosis 72%
Tumor type not
considered previously 28%
Identified new cancers not considered earlier in ~1/4 patients2
Treatment changed 46% Treatment
unchanged 54%
Changed treatment decision in about half the cases1
Basis: • Results from a 28-site registry study (N = 202)1 • Physician-reported clinical utility study (N = 103)2
Revenue Growth Drivers
2010 - 2013 2014 2015 - 2016 2017 +
+ • Extension of BCI indications • Commercialization ex US
• Cell free DNA assays +
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Summary
Commercial stage molecular diagnostics company focused on cancer
We have two high-value proprietary and uniquely differentiated tests on the market, focused on high unmet medical need areas
Medicare coverage of both tests at prices that recognize their value, subject to specific coverage criteria (LCD)
Entering a rapid-growth phase with increasingly diversified growth drivers
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Back-up slides
Medicare Clinical Coverage Criteria – Breast Cancer Index (BCI) Coverage of the Breast Cancer Index (BCI) is limited to patients that meet the following criteria: Post-menopausal female with non-relapsed, ER+ breast cancer, and Was lymph node negative (LN-), and Is completing five (5) years of tamoxifen
therapy, and Patient must be eligible for consideration of extended endocrine therapy
based on published clinical trial data or practice guidelines, and Physician or patient is concerned about continuing anti-hormonal therapy
because of documented meaningful toxicity or possible significant patient-specific side effects, and
The test results will be discussed with the patient (including the limitations of the testing method, the risks and benefits of either continuing or stopping the therapy based on the test, and current cancer management guidelines).
Medicare Clinical Coverage Criteria – CancerTYPE ID
CancerTYPE ID is covered as a once-in-a-life time benefit. The assay may be used to resolve an unknown primary tumor or to resolve a pathological diagnosis with 2 or more differential diagnoses. In the unlikely event of a second UPC, denied claims can be appealed through standard Medicare protocol. Use of the CancerTYPE ID assay is limited to: Tumors for which a single specific site of origin has not been established or
resolved by the combination of clinicopathologic studies and consultation with pathologists, radiologists and oncologists.
Specimens, such as cytology cell blocks, where limited quantity of the specimen precludes standard pathologic workups
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