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Corporate Update
January 14, 2020
For Investor Communication Purposes Only
Disclaimer
Forward-Looking Statements
The preliminary financial results for the fourth quarter and full year 2019 represent the most current information available to
management and reflect estimates and assumptions. The company's actual results may differ materially from these preliminary results
due to the completion of the company's financial closing procedures, final adjustments and other developments that may arise
between the date of this press release and the time that financial results for the fourth quarter and full year 2019 are finalized.
This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. In
some cases you can identify these statements by forward-looking words, such as “believe,” “may,” “will,” “estimate,” “continue,”
“anticipate,” “intend,” “could,” “would,” “project,” “plan,” “potential,” “seek,” “expect,” “goal,” or the negative or plural of these words or
similar expressions.
These forward-looking statements are subject to a number of risks, uncertainties and assumptions, and new risks emerge from time to
time. These statements include, but are not limited to statements regarding anticipated growth in market size and demand for Andexxa
and potential regulatory approval in additional countries and for additional indications. In light of these risks, uncertainties and
assumptions, the forward-looking events and circumstances discussed in this presentation may not occur and actual results could
differ materially and adversely from those anticipated or implied in the forward-looking statements. Please refer to our Annual Report
on Form 10-K and our most recent Quarterly Report on Form 10-Q that we filed with the SEC for a description of risks and
uncertainties that could impact future results.
Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee that the
future results, levels of activity, performance or events and circumstances reflected in the forward-looking statements will be achieved
or occur. We undertake no obligation to update any forward-looking statements except as required by law.
2
For Investor Communication Purposes Only
Andexxa Q4 Results and Business Trends in 2019
3
4Q19 FY 2019
Andexxa Revenue (M) $28 $111
U.S. $24 $104
Europe $4 $7
New Hospitals Adds 90 425
80% of revenue from reorders in Q4,
up from 78% in the third quarter
For Investor Communication Purposes Only
Fourth quarter sales were impacted primarily by:
• $5 million gross to net adjustment due to return
reserve for short-dated product
• Flat Q/Q demand due to a decrease in
utilization, primarily in Tier 1 accounts
• Lower distributor purchases to manage
inventory
Cash balance at Dec. 31, 2019 ~ $464 million
Andexxa 2020 Growth Drivers
For Investor Communication Purposes Only
Novel
• Only FDA approved Factor Xa reversal agent
• Orphan Drug
• Breakthrough Therapy
• CMS NTAP
• Supported by 18 society guidelines
• JCAHO recommendation for DOAC-specific reversal agent
Long Runway
• No FDA-approved competition
• Strong intellectual property
• Potential label expansion
• Wholly-owned (except Japan)
• Approved in U.S. and Europe
• Large & growing FXa market
Andexxa: Significant Long-Term Growth Opportunity
5
For Investor Communication Purposes Only
Global Potential
2.7 3.5 4.2 5.3 5.7 6.1 6.6 7.2 7.7 8.43.84.9
6.2
7.58.1
8.89.5
10.211.1
11.9
1.5
2.0
2.7
3.63.9
4.24.5
4.9
5.3
5.7
8.0
10.4
13.1
16.417.7
19.120.6
22.3
24.0
26.0
0
5
10
15
20
25
30
2016 2017 2018 2019 2020 2021 2022 2023 2024 2025
FX
aP
atients
in M
illio
ns
Estimated 26 Million FXa Patients by 2025
US EU5 ROE
FXa Inhibitor Use Triples Over 10 Years in U.S. and Europe
6
ROE: AUSTRIA, BELGIUM, BULGARIA, CROATIA, CZECH REPUBLIC, ESTONIA, FINLAND, GREECE, HUNGARY, IRELAND, LATVIA, LITHUANIA, LUXEMBOURG, NETHERLANDS, NORWAY, POLAND, PORTUGAL, ROMANIA,
SLOVAKIA, SLOVENIA, SWEDEN, SWITZERLAND
Sources:
2016 – 2019: *# of patients: estimated based on IMS reported units sold and assumed DOT
2019 – 2024: GlobalData Drug Forecast and Market Analysis for AF, VTE and PAD, 6-yr CAGR of 8% across geographies
For Investor Communication Purposes Only
Andexxa Adopters See Substantial Clinical Differentiation Between Andexxa and PCCs
7
Reversal Agent
Average Rating on
Most Important Factors
in Treatment Selection(on a scale of 1 to 7, 7 being
most important)
*Attribute ratings among HCPs who have used Andexxa
5.7
5.8
5.8
5.8
4.3
4.9
4.6
4.6
4 5 6 7
Strength of clinical data
Onset of action
Hemostatic control
Mortality Reduction
ATTRIBUTE RATING*
PCCs
Andexxa
Average rating
For Investor Communication Purposes Only
Source: Portola Monthly Pulse Study, December 2019
Andexxa Utilization is Largely According to the Label Across Various Bleed Types; HCPs Anticipate Increased Usage of Andexxa
8
For Investor Communication Purposes Only
GI40%
ICH20%
Other40%
Andexxa treated patients( n = 342)
Other includes critical compartment bleeds, etc
Source: Portola Chart Audits, June 2018 – Sept 2019. Portola Q3 ATU, Sept 2019
“Physicians anticipate more
than doubling their use of
Andexxa within a year”
Significant Penetration Opportunity
9
2,100 Target
Accounts
Tier 1Target
Accounts
For Investor Communication Purposes Only
• 2,100 target accounts
• Only approximately 20% penetrated in target accounts
• Only 40% penetrated in Tier 1 accounts
• Additional 3,000+ non-target accounts
• ACC in March – 3 abstracts, including oral presentation showing
the impact of Andexxa on inpatient mortality
• Publication of data demonstrating that 4F-PCC’s are not effective
for reversing Factor Xa related bleeds
• Additional health economics data presentations & publications
• NTAP best practices and education
• Direct support on integration of NTAP into EMR systems
• Reimbursement call center
• Opportunity to educate on patient access and reimbursement in
coordination with pharmacy societies
New Programs in 2020 to Drive Deeper Andexxa Utilization
10
For Investor Communication Purposes Only
Clinical Differentiation
Reimbursement
New Clinical Data at ACC Further Differentiates Andexxa
11
For Investor Communication Purposes Only
Andexxa Abstract Topics
• 3 abstracts, including one oral presentation
• Propensity score matching analysis of 30-day mortality vs. 4F-PCC
• Multi-hospital analysis of in-hospital mortality and length of stay vs. 4F-PCC
• Hospital real world outcomes data; Burden of FXainhibitor bleeds
Unlike Andexxa, 4-Factor PCCs Do Not Restore Thrombin Generation at Therapeutic Levels of Xa Inhibitors
12
Apixaban + 4F-PCC (In-Vitro) Apixaban + Andexanet alfa (In-Vitro)
NormalThrombin Generation
0 1 0 0 2 0 0 3 0 0
0
5 0 0
1 0 0 0
1 5 0 0
2 0 0 0
5 0 0 1 0 0 0 1 5 0 0 2 0 0 0
A p ix a b a n (n g /m L )
ET
P (
nM
*m
in)
0
4 .0
2 .0
A p ixa b a n +
A n d e x a n e t ( M )
Placebo
Andexanet alfaHigh Dose (4µM)
Andexanet alfaLow Dose (2µM)
0 1 0 0 2 0 0 3 0 0
0
1 0 0 0
2 0 0 0
3 0 0 0
4 0 0 0
5 0 0 1 0 0 0 1 5 0 0 2 0 0 0
A p ix a b a n (n g /m L )
ET
P (
nM
*m
in)
A p ixa b a n +
4 F -P C C (U /m L )
1 .0
0 .5
0
Therapeutic range of rivaroxaban
and apixaban seen in bleeding
patients
PCC High Dose 50 U/kg (1.0 U/mL)
PCC Low Dose 25 U/kg (0.5 U/mL)
Placebo
No comparative clinical trials of PCCs and andexanet alfa have been conducted
For Investor Communication Purposes Only
2020 High Impact Clinical, Research, and HEOR Studies
HEORClinical Research
• ICH and Outcomes
• Thrombotic Events and
Restart of Anticoagulation
• Biomarkers: Thrombin
Generation in Bleeding
Associated with FXa
Inhibitors
• In vitro PCC Data
• Anti-FXa Assay used in
ANNEXA-4
• In vitro Andexanet-TFPI
interaction
• EU: Orange Registry Case
Match: 30 day mortality
• Chart Audit 1.0: Inpatient
mortality, LOS, etc.
• German Retrace Study:
Mortality / 30 day outcome
• BIM: Budget impact model
of PCCs v Andexanet
• CEA: Cost effective analysis
of PCCs v Andexanet
For Investor Communication Purposes Only
NTAP Education Opportunity
14
• Only about a quarter of NTAP eligible cases are applying for
reimbursement
• When hospitals apply, 92% result in successful reimbursement
• Partner with IDNs to educate on reimbursement solutions for
NTAP
• Education around proper NTAP utilization can offset budget
impact
For Investor Communication Purposes Only
NTAP Programs Targeting Hospital Pharmacists & Administrators
15
Best Practices
• Share protocols from
hospitals that have
successfully utilized NTAP
reimbursement
• 92% result in successful
reimbursement
Call Center
• Provides broader
coverage for customers
• Support for NTAP and
other reimbursement
opportunities
EMR Integration
• Streamline process of
securing NTAP
• Dedicated field-based
resources to support
customers
• Initial focus on Tier 1
accounts
For Investor Communication Purposes Only
Europe Launch Update
On track with reimbursement
and access processes in key
Wave 1 countries:
• Germany / AMNOG: anticipate
completion in the 2H 2020
• United Kingdom / NICE: on the
agenda for review in late Q1;
publication expected in June 2020
16
For Investor Communication Purposes Only
$4 million in 4Q19 sales;
$7 millionin 2019 sales
Ondexxya available for order in
Germany, Austria, the UK,
the Netherlands, Sweden,
Denmark, and Finland
Initiating
access and
reimbursement planning activity in
Wave 2 countries
Andexxa Label Expansion: Urgent Surgery Update
17
Initiated single arm study that
will enroll 100-200 patients
• Quickest way to gather real world
experience in the surgical setting
• Helps inform study design for
randomized, controlled trial (RCT)
For Investor Communication Purposes Only
Estimate that approximately 60,000 patients each year
taking apixaban or rivaroxaban require urgent surgery
RCT (with approximately 500
patients)
• Informed by data from single arm
study
• Many ANNEXA-I sites could also
serve as sites for this study
Cerdulatinib
For Investor Communication Purposes Only
Significant unmet need for an oral
agent that delivers higher response
rates and a better safety profile
PTCL
3,600 relapsed/
refractory1
FL
9,000 relapsed/
refractory1
ORR with Current Therapies
25% - 30% for R/R PTCL
*Exception is CD30+ ALCL given
CD30-targeted treatment option (Adcetris)
Urgent Need for New Treatment Options for Relapsed/Refractory Patients with PTCL and FL
18
For Investor Communication Purposes Only
1. NIH SEER database – Cancer Stat Facts, 2018; Moskowitz et. al., How I treat the peripheral T-cell lymphomas. Blood. 2014 Apr 24; 123(17): 2636–2644
Significant unmet need for safer,
oral “chemo-free” options in the
relapsed and refractory settings
ORR with Current Therapies
50% - 75% for R/R FL
10+ year overall survival
Cerdulatinib: Differentiated Dual SYK/JAK Inhibitor
20
Follicular Lymphoma Results
• 48% ORR cerdulatinib alone (N=42)
• 76% ORR in combination with
rituximab (N=21)
• 91% ORR with combination in
patients that failed 1-3 prior therapies
(N=11)
AITL Results (N=27)
• ~37% CR
• 52% ORR
• Next step: secure partner and initiate
CELTIC-1 PTCL registration study
Phase 2 Ongoing in B- and T-cell NHL – 250+ Patients Treated Overall
Generally Well-tolerated with Several Patients on Drug > 1 year
For Investor Communication Purposes Only
Data Presented at ASH December 2019:
Cerdulatinib demonstrated good tolerability. The most common Grade 3 or greater adverse events were lipase increase, amylase increase, neutropenia, anemia, diarrhea, sepsis/bacteremia,
and febrile neutropenia. The lipase and amylase changes were generally asymptomatic and not associated with pancreatitis. Additionally, there was no emergence of late-stage colitis, cardiac
or liver abnormalities, or other evidence of cumulative toxicity.
Key Takeaways
For Investor Communication Purposes Only
Andexxa: Best-in-Class Asset in a Large & Growing Market
22
Highly Differentiated• 1st & only FDA & EMA approved FXa reversal agent
• Orphan Drug & Breakthrough Therapy designations
• FDA accelerated approval
• CMS NTAP program
• Recommended by 18 guidelines & Joint Commission
Europe Launch• High physician and hospital interest in Wave 1 countries
• Moving forward with market access and reimbursement
plans in Wave 2 countries
• 2-3x larger FXa patient population vs. U.S.
$2 Billion Opportunity• Significant U.S. penetration opportunity
• Only treated ~3% of FXa bleeding patients in 2019
• 640 ordering accounts vs. 2,100 target accounts and
3,000 non-target accounts
Urgent Surgery Indication• Initiated single-arm study to gather real-world data
• Informs design of RCT
• ~60,000 annual patient opportunity
Factor Xa inhibitor market volume grew by 27% in 2019
For Investor Communication Purposes Only
Questions & Answers
For Investor Communication Purposes Only
Corporate Update
January 14, 2020
For Investor Communication Purposes Only
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