Hope for Children with Orphan Liver Diseases

©2021 Albireo Pharma, Inc. All rights reserved

Hope for Children with Orphan Liver Diseases

Corporate Overview (Nasdaq: ALBO)

2©2021 Albireo Pharma, Inc. All rights reserved

Forward Looking Statements This presentation includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include statements, other than statements of historical fact,

regarding, among other things: the timing to achieve revenues, and the amount of such revenues, from sales of odevixibat, should it receive FDA approval for one or more indications; plans for, or progress, scope, cost,

initiation, duration, enrollment, results or timing for availability of results of, development activities, nonclinical studies and clinical trials of Bylvay™(odevixibat) or any other Albireo product candidate or program, such as the

target indication(s) for development or approval, the size, design, population, location, conduct, cost, objective, enrollment, duration or endpoints of any clinical trial, or the timing for initiation or completion of or availability

or reporting of results from any clinical trial, including the long-term open-label extension study for Bylvay in PFIC, the pivotal trial of Bylvay in biliary atresia or the pivotal trial of Bylvay in Alagille syndrome (ALGS), for

submission of any regulatory filing, or for discussions with regulatory authorities; the timing of and our ability to obtain and maintain regulatory approval of any of our product candidates, and any related restrictions,

limitations, or warnings in the label of any approved product candidates; the potential approval and commercialization of Bylvay; the potential for Bylvay to become the first approved drug for PFIC patients; the size of the

PFIC population, the biliary atresia population, the ALGS population or any other disease population for indications that may be targeted by Albireo; the potential benefits or competitive position of Bylvay or any other

Albireo product candidate or program or the commercial opportunity in any target indication; the potential effects of Bylvay on the treatment of PFIC patients and its potential to improve the current standard of care; the

potential issuance of a rare pediatric disease priority review voucher; Albireo’s plans, expectations or future operations, financial position, costs, expenses, uses of cash, capital requirements or our need for additional

financing; or our strategies, prospects, beliefs, intentions, plans, expectations, forecasts or objectives. Words such as “anticipates,” “believes,” “plans,” “expects,” “projects,” “future,” “intends,” “may,” “will,” “should,” “could,”

“estimates,” “predicts,” “potential,” “planned,” “continue,” “guidance,” and similar expressions sometimes identify forward-looking statements.

Any forward-looking statement involves known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to differ materially from those expressed

or implied by such forward-looking statement, and, therefore, investors are cautioned not to place undue reliance on any forward-looking statement. These factors include, but are not limited to: negative impacts of the

COVID-19 pandemic, including on manufacturing, supply, conduct or initiation of clinical trials, or other aspects of our business; whether favorable findings from clinical trials of Bylvay to date, including findings in the

completed Phase 3 clinical trial in PFIC and findings in indications other than PFIC, will be predictive of results from future clinical trials, including the pivotal trial of Bylvay in biliary atresia or the pivotal trial of Bylvay in

ALGS; whether either or both of the United States Food and Drug Administration (FDA) and European Medicines Agency (EMA) will determine that the primary endpoint for their respective evaluations and treatment

duration of the completed Phase 3 clinical trial in patients with PFIC are sufficient to support approval of Bylvay in the United States or the European Union, to treat PFIC, a symptom of PFIC, a specific PFIC subtype(s) or

otherwise; whether the FDA or EMA will complete their respective reviews within the target timelines, including the FDA’s PDUFA goal date, as a potential result of the impact of the COVID-19 pandemic or otherwise; the

outcome and interpretation by regulatory authorities of the ongoing third-party study pooling and analyzing of long-term PFIC patient data; the timing for initiation or completion of, or for availability of data from, clinical trials

of Bylvay, including the pivotal trial of Bylvay in biliary atresia or the pivotal trial of Bylvay in ALGS, and the outcomes of such trials; Albireo’s ability to obtain coverage, pricing or reimbursement for approved products in the

United States or European Union; delays or other challenges in the recruitment of patients for the pivotal trial of Bylvay in biliary atresia or the pivotal trial of Bylvay in ALGS; whether Bylvay will meet the criteria to receive a

rare pediatric disease priority review voucher from the FDA; the competitive environment and commercial opportunity for a potential treatment for PFIC or other orphan pediatric cholestatic liver diseases; the medical

benefit that may be derived from Bylvay, A3907, A2342 or any of the other product candidates; the significant control or influence that EA Pharma has over the commercialization of elobixibat in Japan and the development

and commercialization of elobixibat in EA Pharma’s other licensed territories; our ability to protect and expand our intellectual property; the timing and success of submission, acceptance and approval of regulatory filings;

and our critical accounting policies. These and other risks and uncertainties that we face are described in our most recent Annual Report on Form 10-K and in other filings that we make or have made with the Securities

and Exchange Commission. In addition, market and industry statistics contained in this presentation are based on information available to us that we believe to be reliable but have not independently verified.

All forward-looking statements speak only as of the date this presentation is made and should not be relied upon as representing our views as of any date after this presentation is made. We specifically

disclaim any obligation to update any forward-looking statement, except as required by applicable law. “Albireo” is a trademark of Albireo AB. All other trademarks, service marks, service marks, trade names, logos

and brand names identified in this presentation are the properties of their respective owners.


BylvayTM

(odevixibat)

Strong Ph 3

Results in PFIC

Ph 3 Trials Ongoing in ALGS, BA

Next Generation Bile Acid

Transporter Inhibitors

Strong Financial Foundation with Cash into 2023

Albireo Reaches New Stage In Company Evolution


Growing Albireo: Road to Reach $1 Billion BylvayTM Sales


BylvayTM Aspiration: Road to $1 Billion

LARGE GLOBAL OPPORTUNITY

PREPARED AND READYTO LAUNCH EXPANSION BEYOND PFIC

HIGH CONFIDENCE IN ACCESS


Reaching $1 Billion BylvayTM Sales

PRECLINICAL PHASE 3PHASE 2PHASE 1 APPROVED

Chronic

ConstipationApproved in Japan/Partnered with EA PharmaElobixibat

Adult Liver

Diseases

A3907

Systemic ASBTi

Viral & Cholestatic

Diseases

A2342

Oral NTCPi

Bile Acid

ModulatorsUndisclosed

BylvayTM

(odevixibat)

IBATi

Pediatric Liver

Diseases

PFIC

Biliary Atresia

Alagille Syndrome

Other Cholestatic

Approval and launch expected H2 21

$1B 2nd Half of

the Decade

IBATi = ileal bile acid transport inhibitor, ASBT = apical sodium-dependent bile acid transporter, NTCPi = sodium-taurocholate co-transporting peptide inhibitor


Sustained Growth Through Multiple Catalysts

A3907 Systemic ASBTi adult liver disease

PEDFIC 2: PFIC rollover and expanded cohort

BOLD: Biliary Atresia Phase 3 program

ASSERT: Alagille Syndrome Phase 3 program

PFIC approval, priority review voucher, launch

A2342 Oral NTCP Inhibitor

Novel bile acid modulators

2022H2 21H1 21 2023

Open label

Full site activation


IND-enabling studies

Ph1 Initiation

2024

Topline Data

Topline Data

Ph 1 Topline data Ph 2 Initiation

Ph 1 Initiation Ph 2 Initiation

Candidate identification


Global Patient Opportunity


Overview: Rare Pediatric Cholestatic Liver Diseases

1 Pawlikowska 2010

2 Data on file; Lykavieris et al. Hepatology, 2005

Progressive Familial Intrahepatic

Cholestasis (PFIC)1

Age ~1-2 years, cholestatic, pruritic

Multiple genes, similar symptoms

Inflammation, fibrosis,

cirrhosis, death

Almost no patients survive beyond age

20 without surgical diversion

or liver transplant1

Alagille Syndrome (ALGS)

Age ~4-12 Months, multiple symptoms

Autosomal dominant,

multiple organ impact

Paucity of bile ducts

Many patients may need

a liver transplant

Disease can stabilize

Biliary Atresia2

Age ~2 wk - 3 Months, failure to thrive,

acholic stools, jaundice

Absence of bile ducts

Potential need for liver transplant

Disease can stabilize

Kasai (HPE) surgery may

restore bile flow

~50% of patients have liver

transplant in first 2 years2

Transplant is definitive treatment

Presentation

Cause or Genetic Disorder

Disease Progression

Treatment & Survival


Large Pediatric Global Market Opportunity*

*Top 25 Markets excluding China and India

PrevalenceIndication Available Patients

15K(13K-16K)

PFIC600

(500-700)

1,900(1700-2100)

25K(23K-28K)

Alagille Syndrome1,500

(1400-1600)

3,500(3100-3800)

45K(40K-50K)

Biliary Atresia 2,400

(2200-2700)

8,600(7800-9400)

5K(4K-6K)

Pediatric PSC

~100K(80K-100K)

Total~4,500

(4100-5000)

~14,000(12,600-15,300)

U.S. Ex-U.S.

Global Market Opportunity Size: ~100,000 Patients with Pediatric Cholestatic Liver Disease in Top 25 Countries


Progressive Familial Intrahepatic

Cholestasis (PFIC)


Inadequate Treatment Options for PFIC

1Yang, et al. J Pediatr Gastroenterol Nutr 2009

Off-Label Medications

PEBD Surgery(partial external biliary diversion)1

Liver Transplantation

UDCA

Seeking symptomatic relief

UDCA, rifampicin, cholestyramine …

Bile acid and pruritus reductions

Undesirable external stoma bag

Limited timely organ availability

Need for lifelong immunosuppression

Morbidity and disease recurrence

Seru

m b

ile a

cid

s (

μm

ol/

L)

Time Post PEBD ( Years)

0 1 20

100

200

300

400


NAPPED Natural History Data Provide Strong Rationale for IBATi

*Van Wessel et al. 10.1016/j.hep.2020.02.007, Would be 100%, but one patient died due to complications of multiple PEBD surgeries ** Van Wessel Espghan 2019

NAPPED: Natural Course and Prognosis of PFIC and Effect of Biliary Diversion

Improved native liver survival does not require bile acid normalization

PFIC2 Native Liver

Survival Improvement*

PFIC1 Native Liver

Survival Improvement **

% O

f P

ati

en

tsW

ith

Nati

ve L

iver

Years After Diversion

0

P = 0.001

P < .001

SBA <102 μmol/L

SBA ≥102 μmol/L

0 5 10 15

20

60

100

40

80

% O

f P

ati

en

tsW

ith

Nati

ve L

iver

Years After Diversion

0

P = 0.03

SBA <65 μmol/I

SBA ≥65 μmol/I

0 10

20

60

100

40

80

5


2 4 W E E K S 7 2 W E E K S

PEDFIC 1 & 2: Gold Standard Phase 3 Programs

PEDFIC 2Interim Analyses at Weeks 24 and 48

PEDFIC 1Single Pivotal Trial to Support NDA/MAA Filings

All Odevixibat→

Odevixibat120 µg/kg/day

N = 35

Placebo→


N = 19

OdevixibatNewly enrolled

120 µg/kg/day

N = 17

All enrolled

patientsN = 71

Oral capsule/sprinkle

120 μg/kg/day

PEDFIC 2

Interim analysis

population(≥1 dose of odevixibat)

N = 69

Patients ongoing

in PEDFIC 2N = 66

62 SubjectsOnce daily

Oral capsule/

sprinkle

R

Enrolled but not dosed,

N = 2

Total discontinuations, N = 3

Withdrawal of consent,

N = 2; AE, N = 1

Non-PEDFIC 1 eligible

Cohort 2

Cohort 1 (P1P)

Cohort 1 (P1O)Odevixibat40 µg/kg/day

N = 23


N = 19

PlaceboN = 20

9 6 W E E K S C O L L E C T I V E


Pruritus Improvement: High Statistical Significance

PPAs defined as a scratching score of ≤1 or a ≥1-point drop from baseline on an observer-reported instrument

.

Primary Endpoint: Pruritus Improvement

Pruritus Score Reduction >1 Point

from PEDFIC 1 to PEDFIC 2 Baseline

0

20

40

60

80

LS

Me

an

(S

E)

Pro

po

rtio

n o

f P

PA

s (

%)

Placebo

N = 20

Odevixibat

40 µg/kg/d

N = 23

Odevixibat

120 µg/kg/d

N = 19

All Odevixibat

N = 42

P=0.003

P=0.033 P=0.004

Proportion of Positive Pruritus Assessments (PPA)

0.0

1.0

2.0

3.0

4.0

PEDFIC 1Baseline

PEDFIC 1Weeks 21–24

PEDFIC 2Weeks 1–4



Ob

se

rve

r-R

ep

ort

ed

Sc

ratc

hin

g S

co

re

Odevix 40

Odevix 120

N = 18


0

10

20

30

40

50

60

70

Pati

en

ts (

95%

CI)

Wit

h

Se

rum

Bile

Ac

id R

es

po

nse

(%

)

43.5%

21.1%

33.3%

Placebo

N = 20

Odevixibat

40 µg/kg/d

N = 23

Odevixibat

120 µg/kg/d

N = 19

All Odevixibat

N = 42

P=0.001

P=0.035P=0.003

Serum Bile Acid Responders: High Statistical Significance

1. Serum Bile Acid Response: Serum bile acids ≤70 μmol/L at week 24 or a reduction from baseline to week 24 of ≥70%2. Responder: >70% reduction from baseline or achieving a level of < 70 µmol/L

Increasing Bile Acid Response Observed

With Longer Treatment2

48 weeks

Primary Endpoint: Percent of Patients Achieving Serum Bile Acid Response1

24 weeks

0

10

20

30

40

50

60

70

80

Week 4 Week 12 Weeks 22-24 Week 36 Weeks 46-48

Pro

po

rtio

n o

f B

ile

Ac

id R

es

po

nd

ers

*

Weeks on Odevixibat Treatment (All Doses)

n n=20 n=10

N=68


Bile Acid Levels Normalized in Many Patients

-50

50

150

250

350

450

550

650

PEDFIC 1Baseline

PEDFIC 2Baseline

PEDFIC 2Week 4

PEDFIC 2Week 12


Odevix 40

Odevix 120

Fa

sti

ng

Se

rum

Bile

Ac

id C

on

ce

ntr

ati

on

, μ

mo

l/L

Serum Bile Acid Reduction ≥70% from PEDFIC 1 to PEDFIC 2 Baseline

N = 14


Observed Improvements in Disease Modifying Parameters

Height

-2

-1.5

-1

-0.5

0

0.5

PEDFIC 1ᵃ Baseline PEDFIC 2 Baseline PEDFIC 2 Week 24

Mean

(S

E)

Weig

ht

Z S

co

re

Weight

-2.5

-2

-1.5

-1

-0.5

0


Mean

(S

E)

Heig

ht

Z

Sco

re

0

0.5

1

1.5

2

2.5

3

3.5


Mean

(S

E)

mg

/dL

0

20

40

60

80

100

120


Mean

(S

E),

U/L

Total Bilirubin

Pbo→Odevix

AllOdevix→Odevix

Odevix

Serum Alanine Aminotransferase

Total number of patients (Ns) vary by parameter and time point.

Secondary Endpoints


Observed Improvements in Sleep Parameters

All patients in PEDFIC 2 receive odevixibat; error bars show standard error. aData shown for all patients in PEDFIC 1.Odevix, odevixibat; AllOdevix→Odevix, PEDFIC 2 participants who received odevixibat in the preceding PEDFIC 1 study; Pbo, placebo; Pbo→Odevix,

PEDFIC 2 participants who received placebo in preceding PEDFIC 1 study. Total number of patients (Ns) vary by parameter and time point.

Secondary Endpoints

Pbo→Odevix AllOdevix→Odevix Odevix

0

25

50

75

100

PEDFIC 1ᵃBaseline

PEDFIC 2Baseline

PEDFIC 2Week 24

% of Days Requiring Soothing

-25

0

25

50

75

100

PEDFIC 1ᵃBaseline

PEDFIC 2Baseline


% of Days Needing Help Falling Asleep

0

20

40

60

80

PEDFIC 1ᵃBaseline

PEDFIC 2Baseline


% of Days With Blood Due to Scratching

0

10

20

30

40

PEDFIC 1ᵃBaseline

PEDFIC 2Baseline


% of Days Requiring Medications For Sleep

0

3

6

9

12

PEDFIC 1ᵃBaseline

PEDFIC 2Baseline


Number of Awakenings Per Night

0

25

50

75

100

PEDFIC 1ᵃBaseline

PEDFIC 2Baseline

PEDFIC 2Week 24

% of Days Sleeping with Caregiver


Pruritus Improvement Demonstrated in PFIC 1, 2 & 3

a Reduction from baseline pruritus score (0 to 4 point scale)

Duration of odevixibat treatment 4-112 weeks

High Percentage of Patients Achieved Improvement in Pruritus with Odevixibat Treatment

>1 Point Decrease Deemed Clinically Relevant

PFIC1N = 20

PFIC2N = 52

PFIC3N = 5

95% 80% 100%

Patients with

improved

pruritus score

1.3 1.3 2.1Mean reduction

(points)a


Well Tolerated with Minimal Diarrhea, Minimal to Moderate TEAEs

AE, adverse event; ALT, alanine aminotransferase; AST, aspartate aminotransferase; TEAE, treatment-emergent adverse event.

No deaths or drug-related serious AEs were reported

1 patient in the odevixibat 120 μg/kg/day arm discontinued due to an AE of diarrhea

PEDFIC 1

Patients, N (%)Placebo

N = 20

Odevixibat

40 μg/kg/day

N = 23

Odevixibat

120 μg/kg/day

N = 19

Odevixibat

All doses

N = 42

Any TEAE 17 (85.0) 19 (82.6) 16 (84.2) 35 (83.3)

Mild 6 (30.0) 11 (47.8) 8 (42.1) 19 (45.2)

Moderate 9 (45.0) 7 (30.4) 6 (31.6) 13 (31.0)

Severe 2 (10.0) 1 (4.3) 2 (10.5) 3 (7.1)

Drug-related TEAE 3 (15.0) 7 (30.4) 7 (36.8) 14 (33.3)

Serious TEAEs 5 (25.0) 0 3 (15.8) 3 (7.1)

TEAEs leading to discontinuation 0 0 1 (5.3) 1 (2.4)

Liver-related TEAEs 4 (20.0) 5 (21.7) 6 (31.6) 11 (26.2)

Drug-related TEAEs occurring in 2 or more patients in a group, by preferred term

ALT increased 1 (5.0) 2 (8.7) 2 (10.5) 4 (9.5)

AST increased 1 (5.0) 2 (8.7) 1 (5.3) 3 (7.1)

Blood bilirubin increased 1 (5.0) 2 (8.7) 2 (10.5) 4 (9.5)

Diarrhea/frequent bowel movements 1 (5.0) 2 (8.7) 2 (10.5) 4 (9.5)


Building Blocks for Commercial Success

Commercial

Build-up

Early Access

Programs

Bylvay Global

Brand Name*

Distribution

Network

Regional

PartnershipsCustomer

Insight

Travere Therapeutics

Agreement

*Provisional acceptance by both the FDA and EMA as the brand name for odevixibat.


Alagille Syndrome & Biliary Atresia


ASSERT: Alagille Syndrome Global Pivotal Trial

24-Week Treatment2:1 Randomization

A Phase 3 Double-blind, Randomized, Placebo-controlled Study of the Safety and Efficacy of

Odevixibat in Patients with ALGS

R

PlaceboN= 15


N= 30

~45 SubjectsOral capsule/sprinkle

Once daily

Key Inclusion Criteria:

Patient (of any age) with genetically

confirmed diagnosis of ALGS

History of significant pruritus

Elevated s-BA level

Primary Endpoint

Change from baseline in

scratching to month 6

(weeks 21 to 24) as

measured by the Albireo

ObsRO caregiver instrument

Alagille Syndrome Safety & Efficacy Randomized Trial (ASSERT)

~35 global sites will be initiated

Optional Enrollment Open Label Extension

Safety Follow-up


Serum Bile Acids Post-Kasai Correlated With Improved NLS1

Bile Acids: Significant Impact in Biliary Atresia

1. Harpavat et al. Hepatology. 2020;72(suppl 1):128A–129A.

Serum Bile Acid Reduction Correlated With Improved Native Liver Survival Post-Kasai (HPE) Over 2 Yrs

Low bile acid levels

Time From Kasai to Transplant/Death (months)

Tra

ns

pla

nt-

free

Su

rviv

al

P =0.0006

High bile acid levels


BOLD: Precedent-Setting Biliary Atresia Global Pivotal Trial

24-Month Treatment

Double-Blind, Randomized, Placebo-Controlled Trial to

Evaluate the Efficacy and Safety of Odevixibat in

Children with BA who have undergone Kasai HPE

R

PlaceboN= 100


N= 100~200 SubjectsPost-Kasai HPE

Oral capsule/sprinkle

Once daily

Key Inclusion Criteria:

Clinical Diagnosis of BA

Age at Kasai HPE ≤ 90 days

Randomized within 3 weeks

Rollover CohortExtension Trial

Primary Endpoint

Proportion of patients who are

alive and have not undergone a

liver transplant

FDA/EMA: Single Pivotal

Sufficient to Support Filing

~70 global sites will be initiated

Biliary Atresia and the Use of Odevixibat in Treating Liver Disease (BOLD)


New Oral Bile Acid Modulators


Expansion Beyond BylvayTM

1. Pediatric CLD: Data on file. 2. PSC: Gochunaur; Clinical Liver Disease; 2020; Tabibian; Gastroenterology and Hepatology; 2018; Tanaka; Hepatology Research; 2019, PBC: Lu; Clinical

Gastroenterology and Hepatology; 2018; Marschall; Scientific Reports; 2019; Kumagi; Orphanet; 2008. 3. HBV and HDV: Data on file; Delveinsight Chronic Hepatitis B Virus and Hepatitis D Virus Reports

2020. 4. PBC Linerixibat: Hegade, Lancet 2017, PBC Maralixibat:Mayo, Hepatology 2019, PSC Maralixibat Bowlus, Hepatology 2019 (Suppl), PBC Odevixibat: Al-Dury, Scientific Reports 2018

~100KPediatric CLD1

~600KAdult CLD2

PBC

PSC

~11M Viral3

BA

ALGS

PFIC

High Diarrhea

Adult Ph 2 Data4

IBATi Diarrhea

PBC 64%- 80%

PSC 52%

Reduce Burden on

Large Intestine/Colon

ChallengeOpportunity Solution

Liver & Viral Disease

ASBTi: systemic apical sodium-dependent

bile acid transporter inhibitor

NTCPi: sodium-taurocholate

co-transporting peptide inhibitor


Expansion Beyond BylvayTM: Novel Compounds

High bioavailability

Highly selective

Bile acid excreted in stools and urine

Improved liver histology in NASH model

A3907

Novel Systemic

ASBT Inhibitor

A2342

Novel Oral

NTCP Inhibitor

Blocks entry into the liver

Potential in viral and cholestatic diseases

SubQ Hepcludex™ proof of concept

Gilead purchased €1.15B +.3B CVR


Novel Compounds, Distinct MOAInvestigational compounds with a distinct MOA to regulate bile acid movement


New Approaches Solve IBATi Therapeutic Window Challenges

1. Human recombinant ASBT or NTCP transporters expressed in Chinese hamster ovary cells. Potency of the compounds was assessed based on their ability to inhibit 3H-taurocholic acid uptake. Mean values, N = 3-5.

5

6

7

8

9

10

A3309IBATi

Non-systemic

A3907ASBTi

Systemic

AS0556ASBT/NTCP

Dual

A2342NTCPiOnly

In Vitro Potency of Novel Bile Acid Transport Inhibitors Against ASBT and NTCP1

ASBT NTCP

-Lo

g10

(IC

50)

1 nM

10 nM

100 nM

1000 nM


New A3907: Promising Compound for Adult Liver Diseases

1. 7-d treatment in mice, Mean with SEM, N = 6-8. **P<0.002

2. 10-weeks treatment in diet induced and biopsy confirmed mouse model of Nonalcoholic steatohepatitis. **P<0.001, **P<0.001

Improved Fibrosis Stage vs IBATi2

*** *** **

Higher score

Same score

Pe

rce

nta

ge

Of

An

ima

ls (

%)

0

25

50

75

100

Increases Urinary Bile Acids1

Excretion vs IBATi

0.5

0.4

0.3

0.2

0.1

0.0

Vehicle A3309IBATi

15 mg/kg

A390710 mg/kg

A390730 mg/kg

A3907100 mg/kg

.

**

**

Uri

ne T

ota

l B

ile

Ac

ids

(µ

M)

Pre-Clinical Data


Road to Launch and $1 Billion


Expansion Beyond PFIC: Anticipated First to Market in Most Regions

Current guidance: ASSERT Topline data anticipated in 2022; BOLD Topline data anticipated in 2024

1st PFIC

1st Biliary Atresia

1st ALGS

ROW

1st PFIC

1st Biliary Atresia

Fast Follower ALGS

US

1st PFIC

1st Biliary Atresia

1st ALGS

Europe

After modest PFIC build, marginal

additional costs

Unencumbered global rights, no

royalties/milestone payments

Expansion into rare adult liver diseases


Revenue Uptake Builds Over Time to Profitable Long-Term

Revenue

• 2021 – low single-digit $ millions

• 2nd Half of Decade >$1B annual revenue aspiration

• Unencumbered global rights, with no royalties/milestones

Cash

• Cash runway into 2023 based on budgeted net sales and expenses

• Priority Review Voucher (PRV) eligible upon approval; plan to monetize

• Q1 21 cash and cash equivalents at 3/31/21 of $217.1M

• 2021 op. cash burn $130-$135M


Management Team With Deep Biotech & Pharma Experience

Ron CooperPresident and CEO

Bristol-Myers Squibb

(President of Europe)

Michelle GrahamChief Human

Resources Officer

TESARO, Parexel, Integer,

Bausch + Lomb,


Martha CarterChief Regulatory Officer

Aegerion, Proteon, Trine

Pat Horn, MD, PhD Chief Medical Officer

Orphan Technologies, Dyax,

Tetraphase, Abbott

Jan Mattsson, PhDChief Scientific Officer

(Co-Founder)

AstraZeneca

Jason DuncanChief Legal Officer

and General Counsel

Stallergenes Greer, Sobi,

EMD Serono

Pamela StephensonChief Commercial Officer

Vertex, Pfizer

Simon HarfordChief Financial Officer

Parexel, GlaxoSmithKline,

Eli Lilly

Joan ConnollyChief Technology Officer

Stemline Therapeutics,

ImClone Systems,



Sustained Growth Through Multiple Catalysts

A3907 Systemic ASBTi adult liver disease

PEDFIC 2: PFIC rollover and expanded cohort

BOLD: Biliary Atresia Phase 3 program

ASSERT: Alagille Syndrome Phase 3 program

PFIC approval, priority review voucher, launch

A2342 Oral NTCP Inhibitor

Novel bile acid modulators

2022H2 21H1 21 2023

Open label



IND-enabling studies

Ph1 Initiation

2024

Topline Data

Topline Data

Ph 1 Topline data Ph 2 Initiation

Ph 1 Initiation Ph 2 Initiation

Candidate identification


Hope for Children with Orphan Liver Diseases

Corporate Overview (Nasdaq: ALBO)

Hope for Children with Orphan Liver Diseases

Documents

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