Corporate Presentation
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
2
Biotheranostics History
Breast Cancer
Index (BCI)
biomarker
discovery
initiated
2004
AviaraDx
acquired by
bioMérieux &
renamed
bioTheranostics
2006
2008
CancerTYPE ID
(CTID)
biomarker
discovery
initiated
BCI obtains
Medicare
Coverage (LCD)
CTID obtains
Medicare
coverage (LCD)
2010
Commercial
launch of
CTID
2011
Key BCI
clinical studies bioT3
published released
2012
2013
Key CTID
clinical studies
published
2014
$32M funding
and spinout
from bMX
2015
2016
Full
Commercial
commercial
ramp for BCI
launch for
BCI
CTID & BCI
health
economics
studies published
3
Our Organic Growth Strategy in a Nutshell
Levers of 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.,
Awareness
& Advocacy
High
Revenue
Growth
chemo prediction, neo adjuvant, metastatic)
 Cash collection & appeals
effectiveness
Increased
reimbursement
 ASP 
Sales
Coverage
High
Volume
Growth
4
Our Advanced Molecular Tests Target Large Unmet
Medical Needs, Cancer Patient Populations and Markets
Epidemiology
• Molecular classifier & diagnosis
• Biomarkers linked to targeted first
line treatments for metastatic cancer
~ 100k incident
patients per year for
all unclear
diagnoses
~ 170k incident
patients per year
• Early & late recurrence risk
• Likelihood of benefit from
endocrine treatments
Source: Mattson-Jack Cancer !Mpact database (2014)
US Market Size
& Penetration
~ $300 M
< 5%
~ $800M
< 3%
~ 500k prevalent pool
of patients
5
Patient Journey for EarlyStage, ER+ Breast Cancer
2 – 4 weeks
Diagnosis
Pre-surgical
0 or 3 mo
Surgery
Chemo
Oncotype
DX™
Chemo
Treatments
0- 5/10 years
Long-term treatment
BCI
Chronic endocrine
treatment (5-10 years)
+/- Radiation
Epidemiology
(for ER+
early-stage
patients)
Incidence
~170 k/year
Source: Physician interviews, Mattson-Jack Cancer Mpact database (2014)
Prevalence between
years 3-8
~500k
6
Challenging Risk vs. Benefit Profile Of Endocrine
Drugs Complicates Decision-Making
 ATLAS Trial1 (Tamoxifen for 10y vs 5y)
For every 600 women treated,
 16 recurrences were prevented
 MA.17 Trial4 (Extended letrozole vs placebo)
For every 600 women treated,
 12 recurrences were prevented
At the cost of 2-3…
At the cost of 5-9…
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
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.
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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
+ Long term side effects
4 bone fractures
and QoL impairment
1 thromboembolic event
8
Addressing a Key Unmet Medical Need
There is only one validated predictive test available today:
Sept 2013 Lancet Oncology
Editorial
“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.”
9
Second Generation Molecular Diagnostic Test
With Two Components
BCI Prognostic
BCI Predictive
Assesses Individual Risk of
Recurrence from 0-10 years
Predicts Likelihood of Benefit
From Hormonal Therapy
Initial focus is on the area of greatest unmet need:
Benefit from extended
Late recurrence risk (5-10 yrs)
endocrine therapy (5-10 yrs)
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Clinically Actionable Information
that can impact her treatment plan
Low Risk/Low Likelihood of Benefit
~45%
High Risk/High Likelihood of Benefit
~30%
• Consider completion of endocrine Rx at 5 years
• For patients already on extended endocrine Rx, consider stopping
• 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
High Risk/Low Likelihood of Benefit
~15%
• Consider additional and other therapeutic approaches for risk
reduction and more frequent monitoring
Low Risk/High Likelihood of Benefit
~10%
• Consider continuation of endocrine Rx beyond year 5 if patient is
tolerating well and has no concerning comorbidities
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
Patient Tissue
Sample (FFPE)
Tumor dissection
Extract
RNA
RT-PCR
BCI Prognostic
•
•
Individualized risk of late
recurrence
High / Low Risk
BCI Predictive (H/I ratio)
•
•
Predictive of likelihood of benefit
from extended endocrine therapy
High or Low (binary result)
• Algorithm evaluates patient’s
gene expression profile
• Interrogates estrogen signaling(1)
and proliferative(2) pathways
(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)
12
BCI Sample Test Report
13
Breast Cancer Index Supported by Robust
Clinical Evidence
Integration into clinical practice
Pivotal studies (2013)
CCR
JNCI
Lancet
Oncology
Technical robustness of biomarker
Clinical
Utility
Clinical
Validity
Analytical
Validity
Health
Economics
&
Outcomes
Health and cost effectiveness
14
BCI Pivotal Studies
The BCI clinical study program has demonstrated:
 Risk Assessment for Early and
Late Recurrence: Randomized
controlled trial (Stockholm) and Multiinstitutional 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)
• 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
15
Clinical impact of BCI in extended endocrine
therapy decision-making
 Prospective clinical decision-impact study led by Yale University (N=100 pts)
 Physicians and Patients completed questionnaires pre- and post-BCI
Physician Recommendations for Extended Endocrine Therapy
Treatment
Changed
27%
 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
Sanft T, et al. Breast Cancer Res Treat. 2015
16
Actual Data from Clinical Utilization of BCI
Confirms Broad Use and large market opportunity
T= 0 @ diagnosis
Incidence ~ 167k*
T= 5 yrs
T= 10 yrs
Prevalence between
years 3-8 years ~ 515k* patients
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
* Epidemiological estimates are for U.S. only in 2015
Source: Kantar Cancer !Mpact database (2014)
17
BCI is Uniquely Positioned to Inform Extended Endocrine
Decision, Similar to Oncotype DX for Chemo
Diagnosis
5 yrs
Prognostic
(Late Recurrencespecific)
Prognostic
(at Diagnosis)
Predictive of
Chemotherapy
Benefit
Number of Genes
Platform
10 yrs
Predictive of
Extended Endocrine
Therapy Benefit
Oncotype DX®
(Genomic Health)
MammaPrint®
(Agendia)
Prosigna™
(Nanostring)
Breast Cancer
IndexSM
(Biotheranostics)
21
70
50
11
RT-PCR
Microarray
NanoString nCounter
RT-PCR
18
Addresses the Needs of a Broad Group of
Metastatic Patients
~15%
No
~85%
Clear Diagnosis
Yes
+
CUP
Incidence
Wide
differential
Differential
Confirmatory
90 – 130K patients
• CancerTYPE ID is the market
leader among gene expression tests
Market
dynamics
– > 23,000 patient tumors
analyzed to date
– 1,750 physicians ordered test in
last 12 months
– Competitively well positioned
vs. main players
Source: Mattson-Jack Cancer !Mpact database (2014)
Certain
Diagnosis
470 – 510K patients
• 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
19
CancerTYPE ID is Supported by
Robust Clinical Evidence
Clinical
Utility
Clinical
Validity
Analytical
Validity
Health
Economics
&
Outcomes
20
CancerTYPE ID Pivotal Studies
The CancerTYPE ID clinical study program has demonstrated:
 Clinical Validity: Blinded, peeradjudicated, 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
 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
(Hainsworth et al. J Clin Oncol. 2013;31(2):217-23)
21
Clinical utility of CancerTYPE ID is becoming
increasingly clear
Basis:
• Results from a 28-site registry study (N = 202)1
• Physician-reported clinical utility study (N = 103)2
Identified new cancers not considered
earlier in ~1/4 patients2
Tumor type
suspected
or in
differential
Diagnosis
Tumor type
not
considered
previously
28%
Changed treatment decision in about half
the cases1
Treatment
changed
Treatment
unchanged
46%
54%
72%
Kim et al. Personalized Medicine Onc., 2013; 2: 68-76
28-site registry study, Data on file (2014)
22
Revenue Growth Drivers
+
• Extension of BCI indications
• Commercialization ex US
• Cell free DNA assays
+
2010 - 2013
2014
2015 - 2016
2017 +
23
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
24
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 patientspecific 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