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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


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


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


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


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


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


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


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


• 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


Clinical Differentiation

Reimbursement

New Clinical Data at ACC Further Differentiates Andexxa

11


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


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


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


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


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


$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)


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


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


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


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


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


Questions & Answers


Corporate Update

January 14, 2020


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