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Cowen 40th Annual Healthcare ConferenceMarch 3, 2020
For Investor Communication Purposes Only
Forward Looking Statements
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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® (coagulation factor Xa (recombinant), inactivated-zhzo), reimbursement opportunity and operational expense guidance. 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 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.
We will be using non-GAAP financial measures, which we believe provide useful information for the understanding of our ongoing business performance. Reconciliations of our financial results and financial guidance are included in our press release. These non-GAAP financial measures should be considered in addition to, but not a substitute for, our GAAP results.
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2019 Accomplishments in U.S. and Europe
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• Completed first full year of Andexxa® launch in the U.S. following the approval of our Gen 2 formulation
• Over 4,000 patients treated in the U.S. in 2019
• Full ANNEXA-4 data set published in The New England Journal of Medicine
• Reimbursement progress: • C-code in the outpatient setting
• Increased NTAP reimbursement in the inpatient setting
• Addition of Andexxa to the VA National Formulary
• EMA approval (April) and launch (July) of Ondexxya® in Europe
• Submitted dossiers to NICE and AMNOG, setting the stage to establish reimbursement in the UK and Germany
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Andexxa Growth Drivers
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Novel
• Only FDA approved Factor Xa (FXa) inhibitor reversal agent• Orphan Drug• Breakthrough Therapy
• CMS NTAP• Supported by 19 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 (Ondexxya)
• Large & growing FXa inhibitor market
• Generic entries to FXa inhibitor market starting in 2023
Multiple Drivers of Growth in 2020 and Beyond
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Global Potential
2.7 3.5 4.2 5.3 5.7 6.1 6.6 7.2 7.7 8.43.84.9
6.27.5 8.1 8.8 9.5 10.2
11.111.9
1.52.0
2.7
3.63.9
4.24.5
4.95.3
5.7
8.0
10.4
13.1
16.417.7
19.120.6
22.324.0
26.0
0
5
10
15
20
25
30
2016 2017 2018 2019 2020 2021 2022 2023 2024 2025
FXa
Pat
ient
s in
Milli
ons
Estimated 26 Million FXa Patients by 2025US EU5 ROE
FXa Inhibitor Use Triples Over 10 Years in U.S. and Europe
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ROE: AUSTRIA, BELGIUM, BULGARIA, CROATIA, CZECH REPUBLIC, ESTONIA, FINLAND, GREECE, HUNGARY, IRELAND, LATVIA, LITHUANIA, LUXEMBOURG, NETHERLANDS, NORWAY, POLAND, PORTUGAL, ROMANIA, SLOVAKIA, SLOVENIA, SWEDEN, SWITZERLANDSources: 2016 – 2019: # of patients: estimated based on IMS reported units sold and assumed DOT2019 – 2024: GlobalData Drug Forecast and Market Analysis for AF, VTE and PAD, 6-yr CAGR of 8% across geographies
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27% CAGR
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Andexxa: Factor Xa Reversal Addressable Patientsin the U.S. & EU by 20251
~150,000Wave 1 European Countries – fXa Related Bleeds2
Current Market Opportunity2 Future Market Opportunities
~190,000
Rest of Europe –fXa Related Bleeds
~150,000
U.S. Patients Admitted with fXa
Related Bleeds
~ 60,000
Label ExpansionUrgent Surgery3
~ 30,000
Label ExpansionEdoxaban/
Enoxaparin3
~ 200,000 -300,000
Generic Factor Xa Inhibitors
1. TRUVEN, Portola projections based on IMS data2. Assumes Portola meets the criteria required for full approval3. Assumes successful clinical trial in urgent surgery patients and FDA approval of potential label expansions
Where we are today
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Significant Penetration Opportunity in U.S.
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2,100 Target
Accounts
Tier 1Target
Accounts
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• 2,100 target accounts, representing 80% of addressable patients
• Only approximately 20% penetrated in target accounts
• Only 40% penetrated in Tier 1 accounts
• Additional 3,000+ non-target accounts
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 FXaInhibitors
• In vitro PCC Data
• Anti-FXa Assay used in ANNEXA-4
• In vitro Andexanet-TFPI interaction
• FXa point of care test
• 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
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New Clinical Data at ACC Further Differentiates Andexxa
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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 FXa
inhibitor bleeds
Unlike Andexxa, 4-Factor PCCs Do Not Restore Thrombin Generation at Therapeutic Levels of Xa Inhibitors
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Apixaban + 4F-PCC (In-Vitro) Apixaban + Andexanet alfa (In-Vitro)
NormalThrombin Generation
0 1 0 0 2 0 0 3 0 00
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
*min
)
0
4 .02 .0
A p ixa b a n +A n d e x a n e t (µ M )
Placebo
Andexanet alfaHigh Dose (4µM)
Andexanet alfaLow Dose (2µM)
0 1 0 0 2 0 0 3 0 00
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
*min
)
A p ixa b a n +4 F -P C C (U /m L )
1 .0
0 .5
0
Therapeutic range of rivaroxabanand 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
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NTAP Education: Reimbursement Opportunity up to 65%
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Best Practices
• 92% result in successful reimbursement
• Share protocols from hospitals that have successfully utilized NTAP 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
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Europe Launch Update
On track with reimbursement and access processes in key
Wave 1 countries:• United Kingdom / NICE: on the
agenda for review in late Q1; publication expected in June 2020• Germany / AMNOG: anticipate
completion in the 2H 2020
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$4.6 million in 4Q19 sales;
$7 millionin 2019 sales
Ondexxya available for order in Wave 1 countries: Germany,
Austria, the UK, the Netherlands, Sweden,
Denmark, and Finland
Initiating access and
reimbursement planning activity in Wave 2 countries including France, Spain and Italy
Andexxa Label Expansion: Urgent Surgery Update
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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)
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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
Financial Highlights
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Focus on Driving Andexxa Growth
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• Investment in life-cycle management to build Andexxa franchise • ANNEXA-I randomized controlled trial (RCT)
• ANNEXA-S single arm study in urgent surgery to generate data and inform RCT
• Use of Andexxa with edoxaban and enoxaparin
• Streamline spend to focus on foundation of our business, Andexxa• Completed internal restructuring in February
• Discontinuing commercialization and partnership efforts for Bevyxxa®
• Will not to initiate CELTIC-1 trial for SYK/JAK inhibitor, cerdulatinib, until a partner is identified
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2019 Financial Highlights (please see press release and 10-K for additional details)
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• Total revenues of $116.6 million led by Andexxa/Ondexxya global product net revenues of $111.5 million
• Total operating expenses of $387.9 million• Non-GAAP total operating expenses of $295.5 million
• Gross margins for 2019 of 85%• R&D expenses of $124.6 million
• Non-GAAP R&D expenses of $96.3 million
• SG&A expenses of $218.9 million• Non-GAAP SG&A expenses of $186.2 million
• Cash, cash equivalents and investments of $466.2 million at Dec. 31, 2019Please see the reconciliation of GAAP to non-GAAP financial measures included in today’s quarterly press release which is posted in the Investor Relations section of our website.
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2020 Operating Expenses Guidance (please see press release and 10-K for additional details)
• Clinical development plan to support life cycle management of Andexxa including:
• Ongoing ANNEXA-I trial • Studies to support label expansion including the
ANNEXA-S study in urgent surgery and the potential use of Andexxa with other FXa inhibitors
• Initiate validation of a second site for Andexxa manufacturing
• Full year of U.S. sales force which was expanded in 2019• Full year of expenses for Ondexxya team in Europe• Market expansion and development activities in our Wave 2
countries in Europe
R&D Expense$105 - $120M
(~$14M in Stock-Based Compensation)
SG&A Expense $235 - $250M
(~$38M in Stock-Based Compensation)
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• Over the next few years, gross margins will be high as we supply customer with product that was already expensed
• Longer-term, we expect global gross margins to be 80-85%Gross Margin
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Key Takeaways
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Andexxa: Best-in-Class Asset in a Large & Growing Market
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Highly Differentiated• 1st & only FDA & EMA approved FXa reversal agent• Orphan Drug & Breakthrough Therapy designations• FDA accelerated approval• CMS NTAP program• Recommended by 19 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.
Significant 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
Label Expansion• Initiated single-arm study to gather real-world data• Informs design of RCT• ~60,000 annual patient opportunity• Use of Andexxa with other FXa inhibitors
Factor Xa inhibitor market volume grew by 27% in 2019
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Cowen 40th Annual Healthcare ConferenceMarch 3, 2020
For Investor Communication Purposes Only