Hope for Children with Orphan Liver Diseases - Through Bile Acid Modulation - Albireo Pharma
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Hope for Children with Orphan Liver Diseases Through Bile Acid Modulation
Cautionary Note Regarding Forward-Looking Statements
This presentation includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, known as the PSLRA. Forward-looking statements include,
among other things, statements, other than historical facts, regarding: the plans for, or progress, scope, cost, duration or results of, clinical trials and nonclinical studies of odevixibat,
elobixibat, A3384 or any of our other product candidates or programs, such as the target indication(s) for development or approval, the size, design, population, conduct, cost, objective or
endpoints of any clinical trial, or the timing for initiation or completion of or availability of results from any clinical trial (including our Phase 3 clinical trial of odevixibat in patients with
progressive familial Intrahepatic cholestasis (PFIC), our planned pivotal trial of odevixibat in biliary atresia or our Phase 2 clinical trial of elobixibat in NAFLD/NASH), for submission of any
regulatory filing, or for meeting 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 size of the PFIC population, the biliary atresia population or any other disease population for
indications that may be targeted by Albireo; the potential benefits or competitive position of odevixibat, elobixibat or any other Albireo product candidate or program or the commercial
opportunity in any target indication; the potential benefits of a rare pediatric disease designation, the potential benefits of a fast track designation, the potential benefits of orphan drug
designation, the pricing of odevixibat if approved; any action by, or decision of, EA Pharma concerning elobixibat or our business relationship; the duration of our cash runway; our future
operations, financial position, revenues, 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,” “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: the designs, endpoints, sizes and
durations for trials that will be required to support approval of odevixibat to treat patients with PFIC or any other orphan pediatric liver disease; whether favorable findings from clinical trials
of odevixibat to date, including findings in patients with diseases other than PFIC, will be predictive of results from the Phase 3 PFIC clinical program for odevixibat in patients with PFIC;
whether either or both of the FDA and EMA will determine that the primary endpoint and duration of the double-blind Phase 3 trial in patients with PFIC is sufficient, even if such primary
endpoint is met with statistical significance, to support approval of odevixibat in the United States or the European Union, to treat PFIC, a symptom of PFIC, a specific PFIC subtype(s) or
otherwise; the outcome and interpretation by regulatory authorities of the ongoing third-party study supported by us pooling and analyzing long-term PFIC patient data; the timing for
initiation or completion of, or for availability of data from, the Phase 3 PFIC clinical program for odevixibat, and the outcomes of the program; and delays or other challenges in the
recruitment of patients for, the double-blind Phase 3 trial of odevixibat; whether odevixibat will meet the criteria to receive a pediatric priority review voucher when applicable; 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
odevixibat, elobixibat, A3384 or any of our other product candidates; the extent to which one or both of our agreements for elobixibat with EA Pharma and HCR generate future nondilutive
income; 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; whether findings from nonclinical studies and clinical trials of IBAT inhibitors will be predictive of future
clinical success for elobixibat or an Albireo bile acid modulator in the treatment of nonalcoholic steatohepatitis (NASH); and the timing and success of submission, acceptance and approval of
regulatory filings. 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.
2
Albireo: Innovative Science + Deep Pipeline
+ Well Capitalized
STRONG
More than a decade of leadership in bile acid modulation
BASIC
Obtained world’s first regulatory approval for IBATi therapy
SCIENCE
ORPHAN
Odevixibat (IBATi) wholly owned, oral QD capsule/sprinkle with MOU patent
PEDIATRIC through 2031/34*, orphan desigs., PRIME, PIP, fast track and PRV eligibility
LIVER LEAD
Elobixibat (IBATi) JP approval CIC; NASH & bile acid malabsorption programs
ASSET
SOLID NASDAQ listed as ALBO; 12.7M outstanding shares
FINANCIAL $143M cash and cash equivalents as of September 30, 2019
POSITION No debt, cash runway into 2021
*Natural expiry/with potential patent term extension (PTE)
3 ©2019 Albireo Pharma, Inc. All rights reserved.
Management Team With Deep Biotech & Pharma Experience
Ron Cooper Jan Mattsson, PhD
President and CEO Chief Scientific Officer
(Co-Founder)
Bristol-Myers Squibb
(President of Europe) AstraZeneca
Pat Horn, MD, PhD Simon Harford
Chief Medical Officer Chief Financial Officer
Parexel, GlaxoSmithKline,
Orphan Technologies, Eli Lilly
Dyax, Tetraphase, Abbott
Pamela Stephenson Martha Carter
Chief Commercial Officer Chief Regulatory Officer
Vertex, Pfizer Aegerion, Proteon, Trine
Michelle Graham Jason Duncan
Chief Human Resources Officer General Counsel
TESARO, Parexel, Integer, Stallergenes Greer, Sobi,
Bausch + Lomb, Bristol-Myers Squibb
EMD Serono
4 ©2019 Albireo Pharma, Inc. All rights reserved.
A Robust Pipeline Targeting Liver and GI
Diseases/Disorders
PRECLINICAL PHASE 1 PHASE 2 PHASE 3 APPROVED
Commercialization
PFIC
Independent
Odevixibat
Planned
Biliary Atresia
Pediatric Liver
Diseases Alagille Syndrome
Other Cholestatic
Chronic
Elobixibat Approved in Japan/Partnered with EA Pharma
Commercialization
Constipation
Planned Partner
Elobixibat NASH
Bile Acid
NASH
Modulators
Bile Acid
A3384 Malabsorption*
*Further advancement of clinical development gated on evaluation of U.S. patents
5 ©2019 Albireo Pharma, Inc. All rights reserved.
Delivering on Our Plan as a Public Company
P
A
T Ph3 Elobixibat Ph2 Elobixibat Ph3 Odevixibat Ph3 Odevixibat Ph2 Elobixibat
I Japan Ph3 Results Odevixibat Japan PFIC Pivotal PFIC Target NASH Start
E Results Approved Start Site Initiation
N Completed
T
S
2016 2017 2018 2019
S Royalty
Equity ATM
T Monetization
NASDAQ Raise Financing
A Equity
~$100M ~$45M ~$21M
K Listing Raise
E ~$30M ~$50M Elobixibat
H Approval
O Elobixibat Milestone
L Milestone Legacy Payment
D Payment Asset ~$11M
E ~$8M Sale
R ~$4.5M PRV
S Eligibility
Odevixibat
6 ©2019 Albireo Pharma, Inc. All rights reserved.
Multiple Planned Milestones
1H'19 2H'19 1H'20 2H'20 2021
Odevixibat
PFIC PEDFIC 1: Phase 3 topline data readout Mid ‘20
Initiate biliary atresia pivotal program 2020
PFIC PEDFIC 2 rollover cohort (Cohort 1) Open label
PFIC PEDFIC 2 expanded cohort (Cohort 2) Open label
Additional pediatric orphan liver diseases 2020
Potential PFIC approval and launch 2021
NASH/PIPELINE
Elobixibat NASH: Initiate Phase 2 trial 2Q’19
Elobixibat NASH: Phase 2 trial topline readout Mid ‘20
Novel bile acid modulators
A3384: Advance clinical development* 2020
*Gated on evaluation of U.S. patent
7 ©2019 Albireo Pharma, Inc. All rights reserved.
Odevixibat:
Multi-Disease Development
Approach
Many Diseases with Cholestasis of the Liver
Cystic Fibrosis-
Intrahepatic Associated Liver Disease
Progressive Familial Cholestasis of
Intrahepatic Pregnancy Primary Biliary
Cholestasis (PFIC) Cholangitis AIDS
Cholangiopathy
Drug-Induced
Cholestasis
Malignancy of Bile Ducts
Biliary Atresia
IG4-associated cholangitis
Alagille Syndrome
Low Phospholipid-
Biliary Associated Cholestasis
Primary Sclerosing Cholangitis Strictures
The diseases above are cholestatic liver disease or may cause cholestasis (EASL Guidelines, J. Hepatol. 2009).
Whether an IBATi could treat the cholestasis in these diseases is unknown.
9 ©2019 Albireo Pharma, Inc. All rights reserved.
Potential Target Indications
~30,000-40,000* patients in the U.S. and EU alone who are lacking an
approved pharmacological treatment
40
35 Alagille 3-5K Genetic disorder, paucity of bile ducts
30 PFIC
Genetic disorders with bile acid build-up in liver
8-10K
25
Thousands
Pediatric
20 PSC Inflammation and scarring of bile ducts
8-10K
15
Biliary
10
Atresia Blocked or absent large bile ducts
15-20K
5
*Estimate derived from literature, primary market research and modeling. Forecast
0 estimates do not include other regional opportunities, such as Saudi Arabia, Turkey,
Bile Acid-Associated Asia, LATAM.
Cholestatic Liver Diseases
10 ©2019 Albireo Pharma, Inc. All rights reserved.What Is PFIC?
Genetic Disease Estimated
Presentation
Disorder Progression Median Survival
Multiple genes, Inflammation Approximately
Age ~1-2 similar symptoms Fibrosis 15 years*
Cirrhosis
Cholestatic/ Death
Pruritic
*Estimates derived from secondary references and primary market research, and may vary greatly based on patient type.
11 ©2019 Albireo Pharma, Inc. All rights reserved.Inadequate Treatment Options for PFIC
Off-Label Medications PEBD Surgery Liver Transplantation
(partial external biliary diversion)1
UDCA
0 1 2
Time Post PEBD ( Years)
Seeking symptomatic relief Bile acid and pruritus reductions Limited timely organ availability
UDCA, rifampicin, cholestyramine, etc. Undesirable external stoma bag Need for lifelong immunosuppression
>20% treatment failure rate2 Morbidity and disease recurrence
1Yang, et al. J Pediatr Gastroenterol Nutr 2009
2Bull, et al. Hepatology Communications 2018
12 ©2019 Albireo Pharma, Inc. All rights reserved.Eleanor’s Story
Admitted to hospital Intense pruritus until ~4yo Liver transplant urgently needed
Severe hematomas Poor-clotting, vit. K deficiency Lengthy hospitalization and recovery,
from immunizations Bloody bed sheets, scratching post-transplant complications
over night
Constant monitoring
Diagnosis Sleep Deprivation Life Post-Transplant
3 months old Pruritus impacts family life, Compromised immune system
PFIC2 confirmed parents’ relationship, Eleanor’s Immunosuppressive medications daily
by genetic test energy, growth and development Higher susceptibility to illnesses
Lengthier recovery
“…Transplant, that was the hardest
thing I’ve ever had to hear… . I kept
thinking ‘I don’t want this. I don’t
want this.’”
- Claire Brinkley, Mother to Eleanor (PFIC Patient)
13 ©2019 Albireo Pharma, Inc. All rights reserved.Odevixibat: A Profile Suitable for Pediatric Use
Once-Daily Dosing
Oral Capsule or Sprinkles
Minimal Systemic Exposure
Favorable Tolerability Profile*
*In Phase 2 clinical trial
14 ©2019 Albireo Pharma, Inc. All rights reserved.NAPPED Natural History Data
Provide Strong Rationale for IBATi
NAPPED: Natural Course and Prognosis of PFIC and Effect of Biliary Diversion*
Improved Native Liver
Survival (NLS) PFIC2
• sBA reduced to ≤118 μmol/L;
95%** NLS at 15 yrs
% Of Patients With Native Liver
• sBA reduced by >70%;
95%** NLS at 15 yrs
P=0.007
• Improved NLS did not require bile
acid normalization
Diversion Surgery vs No Surgery PFIC2
*Van Wessel, EASL 2019
**Would be 100%, but one patient died due to complications of multiple PEBD surgeries.
15 ©2019 Albireo Pharma, Inc. All rights reserved.Odevixibat: Phase 2 Study in Pediatric Cholestatic Liver Disease
Odevixibat doses evaluated (µg/kg), 4 weeks
10 30 60 100 200
PFIC, Biliary Atresia, Alagille Syndrome, Intrahepatic Cholestasis Patients
Open-label, dose-finding study
Primary endpoints: TESAEs and serum bile acid change
Baseline – single test dose – 2-wk washout – 4-wk treatment
Study initially designed with a maximum dose of 300 µg/kg
N=24 (20 unique + 4 retreated)
Oral late breaker EASL’17/Presidential Poster of Distinction AASLD’17
Odevixibat
Phase 2
Pediatric
Study
16 ©2019 Albireo Pharma, Inc. All rights reserved.Primary Efficacy Endpoint:
Reduction Demonstrated in Serum Bile Acids
All Patients PFIC Patients Only*
0
-10
Mean (SEM) % change from
baseline in serum bile acids
-20 -31
-30
-48 -51
-40 -56
-63
-50
-60
*
-70
-80 Bars illustrate Standard Error of the Mean
**
-90
Phase 2 trial was an open-label, dose-finding study of PFIC, biliary atresia, * Excludes PFIC patient with no BSEP function and 17-year-old PFIC
Alagille syndrome, intrahepatic cholestasis patients for four weeks. patient with low baseline sBA. Neither meet inclusion criteria for Phase 3
Odevixibat Primary endpoints: TESAEs and serum bile acid change trial.
Phase 2 N=24 (20 unique + 4 retreated) in five cohorts
Pediatric *Excludes PFIC patient with no BSEP function.
Study **Excludes 17-year-old PFIC patient with low baseline sBA.
Neither meet inclusion criteria for Phase 3 trial.
17 ©2019 Albireo Pharma, Inc. All rights reserved.Statistical Correlation Reinforces Link Between Reductions
of Serum Bile Acids and Pruritus
VAS-Itcha Whitington (itch)b
ap=0.008, r=−0.54, n=23.
bp=0.004, r=−0.58, n=23.
cp=0.006, r=−0.57, n=22.
dp=0.005, r=−0.57, n=22.
PO-SCORAD (itch)c PO-SCORAD (sleep)d
Odevixibat
Phase 2
Pediatric
Study
nFavorable Tolerability Profile in Study
All patients completed treatment; no evidence of diarrhea during 4-week treatment period
No AEs related to treatment during 4-week treatment period
- Most common AEs: pyrexia, ear infections (12.5%)
No SAEs designated as treatment related (2 deemed unrelated)
Decision made not to dose escalate above 200 µg/kg
- Some transaminase elevations at 200 µg/kg
Odevixibat
Phase 2
Pediatric
Study
19 ©2019 Albireo Pharma, Inc. All rights reserved.PEDFIC 1: Phase 3 PFIC Program Summary
Double-Blind, Randomized, Placebo-Controlled, Phase 3 Study to Demonstrate Efficacy and
Safety of Odevixibat in Children with Progressive Familial Intrahepatic Cholestasis
Sites 45 global sites actively recruiting
Key Inclusion Criteria Diagnosis of PFIC1 or 2 confirmed by genotyping
Serum bile acids ≥100 μmol/L
Pruritus ≥2 on 5-point scale
Size (n) ~60 (20 per group)
Dosage (µg/kg/day) 40, 120 in an oral capsule/sprinkle with planned commercial formulation
Treatment Duration 24 weeks
Primary Endpoint – FDA Assessment of change in pruritus
Primary Endpoint – EMA Serum bile acid responder rate (reach ≤70 μmol/L or a reduction of 70%)
Follow-Up – PEDFIC 2 Opportunity to enroll in long-term extension study
FDA/EMA: Single Pivotal Sufficient to Support NDA/MAA Filings
20 ©2019 Albireo Pharma, Inc. All rights reserved.Odevixibat: Expanding Across Multiple Pediatric CLDs
Pediatric
Liver Disease
Franchise
Other Rare CLDs
Biliary Atresia
Alagille
PSC
2020 planned
pivotal initiation Others
PFIC 2020 plan to expand development
Mid-2020 topline Ph.3
PEDFIC 1 results
Estimated U.S./EU populations
21 ©2019 Albireo Pharma, Inc. All rights reserved.What is Biliary Atresia?
Presentation Cause/Impact Treatment Disease Progression
Age ~2-6 weeks Absence of bile Kasai surgery may ~50% of patients
ducts restore bile flow have liver transplant
Acholic stools, in first 2 years1
jaundice, (~80% in first 2 decades2)
Bile flow
hepatomegaly, Transplant
to gut
failure to thrive is definitive #1 cause of
treatment pediatric liver
Hepatic bile
Few pts. pruritic transplants
acid levels
Plan to Initiate Pivotal Trial in 2020
Recently aligned with FDA on key elements of the trial design
Orphan designations in U.S. and EU
1Data on file
2Lykavieris et al. Hepatology, 2005
22 ©2019 Albireo Pharma, Inc. All rights reserved.Low Serum Bile Acids in Biliary Atresia Patients
Associated with Successful Long-Term Outcomes
Odevixibat Ph.2: Lower sBA Post-Kasai BA Pts.
sBA Reduction BA Pts.* Positive Outcomes**
Baseline
43 µM 136 µM 132 µM 100
0 L o w B ile A
in Serum Bile Acids, %
-0.5
Change from Baseline
T w o -Y e a r O u tc o m e s
80 H ig h B ile
-10
(% o f p a tie n ts )
-20 -51 -58
60
-51 -58 -51 40
-30 -58
20
-40
0
-50 ALT GGT P l a te l e ts S p le e n
( ≤ 40 U /L ) ( ≤ 55 U /L ) (≥ 1 5 0 /η L ) (≤ 2 c m b e l o w
c o sta l r e g i o n )
-60
-70 L o w B ile A c id s ( ≤ 4 0 µ M )
1 2 3 H ig h B ile a c id s ( > 4 0 µ M )
Odevixibat Patient
Phase 2
Pediatric
Study *Baumann, EASL 2019 **Data from START trial, Harpavat et al. Hepatology, 2018
Odevixibat dose 30 ug/kg, 4 weeks of treatment
23 ©2019 Albireo Pharma, Inc. All rights reserved.Serum Bile Acids and Pruritus Reduced in Majority
of Patients with Alagille Syndrome
Odevixibat Ph.2: Pruritus Improvement
sBA Reduction ALGS Pts. in Most Patients*
Screening Treatment
“Alagille syndrome causes intractable pruritus
and disfiguring xanthomas because of
retained bile acids and cholesterol.”**
Baumann, EASL 2019
Odevixibat, 10-200 ug/kg, 4 weeks of treatment
*Similar improvements seen on Whitington-Itch and PO-SCORAD scales.
**Emerick and Whitington, Hepatology 2002
24 ©2019 Albireo Pharma, Inc. All rights reserved.Odevixibat: Filing and Launch Readiness
Manufacturing
Establishing supply chain—on track
Registration batches—completed and on stability
Regulatory
Long-term cancer tox and repro tox studies—complete
Supportive Ph.1 program—on track
Commercial
Hired commercial and medical affairs personnel
Developed brand, payor and advocacy plans
25 ©2019 Albireo Pharma, Inc. All rights reserved.Odevixibat Key Market Research Findings
PFIC patients are easily diagnosed, symptomatic disease
Parents with PFIC children are desperate for a drug treatment
Physicians believe significant percentage of PFIC patients seen
in last 5 years are deceased1
Physicians have high “intent to use” if approved, based on
blinded odevixibat profile
Payers recognize rarity, severity and unmet need in PFIC2
1Physician
perception in quantitative market research
2Payer
Primary Research, 4/2019
PFIC Patient Journey, 11/2017
PFIC Physician Quantitative Research, 1/2018
26 ©2019 Albireo Pharma, Inc. All rights reserved.Small, Concentrated Target Prescribing Populations
~100 U.S. Pediatric Hepatologists
# of Ped. # of Ped.
Country
Gastros Heps
USA ~1200 ~100
United
~120 – 400
Kingdom
Italy ~125 – 1300
France ~200 – 500
Germany ~120 – 400
Spain ~325
27 ©2019 Albireo Pharma, Inc. All rights reserved.Albireo Market Access Planning Well Underway
Global market access trends Rare disease market access Albireo progress to date
challenges
Research to understand payer
needs, evidence requirements
Limited epi data
Pressure on payers Experienced market access
Payers seeking L/T data
Explosion of rare disease tx. partner retained
Broader caregiver/societal
Drive for transparency Robust value proposition
benefits often not considered
Innovative pricing models developed and tested
Innovative pricing challenging
Building evidence, messaging
for small populations
and tools
28Unencumbered Global Rights and Strong Patent Estate
Strength of Method-of-Use Patents
WEAK STRONG
Unmet Need New Use New Chemical
Entity (NCE)
Odevixibat
New Chemical Entity, New Use, Major Unmet Need
Pediatric and Orphan Population
Multiple Orange-book listable patents for PFIC and CLDs
3 Patents, 10 Claims Targeted to PFIC
Expiration 2034 (with PTE and pediatric extensions)
Orphan exclusivity in the U.S. and EuropeHigh Unmet Need and Compelling Opportunity
Pediatric Cholestasis: orphan indications with no approved drug
PEBD: strong clinical rationale for potential benefit of IBAT inhibition
Odevixibat: serum bile acids, pruritus, low diarrhea in pediatric Ph.2 study
Exclusivity Position: orphan drug designations (U.S.-7/EU-12* years);
COM 2022/25; MOU for specified cholestatic liver diseases, 2031/34**
Attractive P&L: modest commercial infrastructure required, few target Rx’ers
*Assumes execution of agreed PIP **Natural expiry/with potential PTE
30 ©2019 Albireo Pharma, Inc. All rights reserved.NASH Program
NASH and Cardiovascular Disease (CVD) Are Associated
NAFL/NASH stage 0 NASH stage 1-2 NASH stage 3-4
Liver related
Liver-related diseases
Cause of death
CVD/extrahepatic cancer
Adapted from Sumida and Yoneda, Gastroenterol. 2018
NASH is hepatic manifestation of metabolic syndrome (obesity, diabetes, dyslipidemia).1
Optimal therapy targets underlying metabolic pathology.
Most NAFLD patients die of CVD before reaching end-stage liver disease.2
Optimal therapy targets both liver and CVD risk factors.
NASH is largely asymptomatic disease.2
Optimal therapy must be safe, with limited off-target effects and highly tolerable.
1Sumida and Yoneda. Gastroenterol 2018; 2Banini and Arun. Clin Med Insights Ther. 2016
32 ©2019 Albireo Pharma, Inc. All rights reserved.Bile Acids Elevated in NASH Patients
Elevated Total Primary BA1 Elevated Total Conjugated Primary BA1
p < 0.0001
p < 0.04
p < 0.047
n=24 n=25 n=37 n=24 n=25 n=37
In addition, involved in1,2:
cholesterol levels, insulin sensitivity, liver inflammation, fibrosis
1Puri et al. Hepatology 2017
2Current Diabetes Reports 2011
33 ©2019 Albireo Pharma, Inc. All rights reserved.Strong Rationale for IBATi in NASH
Improved Cholesterol Profile Decreased Insulin Resistance
0. 35,00
*
% change in LDL/H
-3.5
30,00
GLP1 pmol/L
25,00
*
-7.
Ratio
20,00
Placebo
-10.5 15,00
5 mg *
-14.
10,00
10 mg ** *pAlbireo’s Two-Pronged Development Approach In NASH
Efficacy—bile acids, cholesterol, glucose, liver inflammation and fibrosis
Rationale
Oral 1x/day, minimal systemic exposure, low potential for off-target effects
Elobixibat Novel Bile Acid Modulators
Phase 2 Initiated Q2’19 Preclinical
Locally-active IBATi New mechanism of action
Clinical LDL and GLP1 data New chemical structures
>1,500 exposures TPP*: high efficacy, low diarrhea
Long-term safety data Accelerating development
*Aspirational target product profile
35 ©2019 Albireo Pharma, Inc. All rights reserved.Elobixibat NASH Phase 2 Study Summary
Double-Blind, Randomized, PBO-Controlled, Ph.2 Study to Explore Efficacy and Safety of Elobixibat
in Adults with Nonalcoholic Fatty Liver Disease (NAFLD) or Nonalcoholic Steatohepatitis (NASH)
Key Inclusion Criteria Biopsy-confirmed NASH or
Suspected diagnosis of NAFLD/NASH based on metabolic syndrome definitions
LDL >100 mg/dL
≥10% liver fat (MRI)
Size (n) ~46
Dosage 5 mg
Treatment Duration 16 weeks
Primary Endpoint Assessment of change in LDL-C
Secondary Endpoints Assessment of change in liver fat by imaging, ALT and AST
Exploratory Endpoints Assessment of change in: fasting glucose, insulin, homeostatic model assessment-insulin
resistance, HbA1c, free fatty acids; GLP-1, fibroblast growth factor-19 (FGF-19) and C4 levels;
high-sensitivity C-reactive protein (CRP); lanosterol and beta-sitosterol; body weight, BMI, waist
circumference and waist-to-hip ratio; MRI-based measurement of pulse wave velocity to measure
aortic stiffness; Apolipoprotein A1 and Apolipoprotein B
36 ©2019 Albireo Pharma, Inc. All rights reserved.Multiple Upcoming Milestones
Odevixibat biliary atresia: Initiate pivotal program 2020
Odevixibat PFIC: PEDFIC 1 Phase 3 topline data readout Mid-2020
Elobixibat NASH: Phase 2 trial topline data readout Mid-2020
Odevixibat: Expand development into additional pediatric orphan liver diseases 2020
A3384 BAM: Advance clinical development* 2020
Odevixibat PFIC: Approval and launch 2021
*Gated on evaluation of U.S. patent
37 ©2019 Albireo Pharma, Inc. All rights reserved.Hope for Children with Orphan Liver Diseases Through Bile Acid Modulation November 2019
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