Albireo Pharma, Inc. (Nasdaq: ALBO), a rare liver disease company
developing novel bile acid modulators, today provided a business
update and reported financial results for the quarter ended March
31, 2022.
“The global launch of Bylvay continues to go as planned as we
generated excellent quarter over quarter growth of patients on
Bylvay, mirroring the outstanding reception from HCPs and payers,”
said Ron Cooper, President and Chief Executive Officer of Albireo.
“In the upcoming months, we anticipate European launches as we work
with country agencies on pricing and reimbursement, while looking
ahead to a major Phase 3 readout of the ASSERT study in Alagille
syndrome with the goal of making Bylvay available as quickly as
possible to more cholestatic liver patients and families around the
globe.”
Q1 and Upcoming Highlights
- Commercial launch of Bylvay is tracking to global launch
expectations. The global number of reimbursed patients on
commercial drug grew from 53 at the end of 2021 to 87 at the end of
Q1 2022, a 64% quarter-on-quarter growth. Bylvay product revenue,
net was $4.7 million including product revenue, net of $2.8 million
from the United States and $1.9 million from International
markets.
- Bylvay is currently available in
the U.S. and Germany and is expected to become available for sale
in the UK in the coming week. There are ~110 potential rollover
patients on Bylvay as of the end of Q1 2022. The majority are in
ex-U.S. countries which are expected to transfer to reimbursed
commercial drug upon completion of country price listings.
- Dossiers submitted
in 12 European countries for pricing and reimbursement.
- On February 22, 2022, National
Institute for Health and Care Excellence (NICE) issued
guidance that recommended Bylvay for the treatment of all types of
progressive familial intrahepatic cholestasis (PFIC) in people aged
six months and older.
- U.S. payer coverage is strong
across commercial plans, including Anthem, Aetna, Cigna and United
Healthcare.
- The Phase 3 ASSERT study in
Alagille syndrome (ALGS) is fully enrolled as planned and remains
on track to report topline data by the end of 2022. ASSERT is a
gold standard, prospective intervention trial with 35 sites
across North America, Europe, Middle
East and Asia Pacific. The double-blind, randomized,
placebo-controlled trial is designed to evaluate the safety and
efficacy of 120 µg/kg/day Bylvay (odevixibat) for 24 weeks in
relieving pruritus in patients with ALGS. Secondary endpoints will
measure serum bile acid levels and safety and tolerability. The
trial enrolled patients aged 0 to 17 years of age with a
genetically confirmed diagnosis of ALGS.
- Albireo opened an
Expanded Access Program (EAP) for patients with ALGS, which is
available in the U.S. and Europe, to provide access to Bylvay for
patients suffering from ALGS prior to the product’s planned
approval and reimbursement.
- The Phase 3 BOLD
study is the first and only Phase 3 study of an ileal bile acid
transporter (IBAT) inhibitor in biliary atresia with clinical trial
sites spanning the U.S., Europe, Latin American and China. The
study remains on track for topline data in 2024. Biliary atresia is
the most common pediatric cholestatic liver disease with no
approved drug treatment.
- The Company had 18 abstracts
accepted at upcoming medical congresses, including at The
Professional Society for Health Economics and Outcomes Research
(ISPOR), Digestive Disease Week (DDW), The International Liver
CongressTM (EASL) and European Society for Paediatric
Gastroenterology, Hepatology and Nutrition (ESPGHAN). All meetings
are to be held in the second quarter of 2022 with virtual and
in-person healthcare professional (HCP) attendance at scientific
sessions and company booths.
- ISPOR will be held May 14-18 in
Washington, D.C. with data being presented on correlations between
Patient- and Caregiver-Reported Pruritus from PEDFIC 1.
- DDW to be held May 21-24 in San
Diego, CA will highlight data on long-term treatment with Bylvay
improving sleep parameters; sustained improvements in
cholestasis-related parameters in responders, correlations in serum
bile acid reductions, pruritus and sleep disturbances; as well as
efficacy and safety of Bylvay in PFIC patients with prior partial
external biliary diversion (PEBD).
Pipeline: Next Generation Bile Acid
Modulators
- In a preclinical complete bile duct
ligation model of obstructive cholestasis resulting in very high
serum bile acid levels, treatment with A3907 significantly
increased urinary bile acid excretion and reduced serum bile acid
levels. A3907 is the first and only oral systemic apical
sodium-dependent bile acid transporter (ASBT) inhibitor in clinical
development. The Company plans to initiate a Phase 2 study in adult
liver disease by the end of 2022.
- A2342 is the first oral
sodium-taurocholate co-transporting peptide (NTCP) inhibitor in
development for hepatitis B and D. The Company completed
IND-enabling rodent toxicology studies, with no findings of concern
at doses providing wide margins for the expected therapeutic dose.
Non-rodent IND-enabling toxicology studies are due to read out at
the end of Q2 2022. The Company plans to initiate a Phase 1
first-in-human study by the end of 2022.
Corporate
- The Company plans to present at several upcoming investor
conferences, including the H.C. Wainwright 24th Annual Global
Investment Conference, William Blair 42nd Annual Growth Stock
Conference and Jefferies Global Healthcare Conference.
- Bylvay method of use patent term
extension was granted in a number of countries including Italy,
which provides exclusivity into 2036 in Europe.
First Quarter 2022 Financial Results
- Product revenue, net was $4.7 million for the first quarter of
2022 due to Bylvay global sales. Bylvay was approved during the
third quarter of 2021, therefore there was no product revenue for
the first quarter of 2021.
- Royalty revenue was $2.2 million for the first quarter of 2022,
compared with $2.0 million for the first quarter of 2021, an
increase of $0.2 million. The increase relates to higher estimated
royalty revenue which is passed on to HealthCare Royalty
Partners.
- Cost of product revenue was $0.2 million for the first quarter
of 2022. Following approval of Bylvay, certain manufacturing and
quality headcount costs are now included in cost of product
revenue. There were no material costs, as materials related to
current product sold were expensed prior to approval. Bylvay was
approved during the third quarter of 2021, therefore there was no
cost of product revenue for the first quarter of 2021.
- R&D expenses were $21.9 million for the first quarter of
2022 compared with $19.9 million for the first quarter of 2021, an
increase of $2.0 million. The increase in R&D expenses for the
first quarter of 2022 was principally due to expenses related to
clinical and preclinical program activities, personnel expenses
including stock-based compensation and other costs as we continue
to increase our headcount and program activities. The increase in
program activities related to ongoing preclinical trials as well as
the Phase 1 study for A3907 and were partially offset by a decrease
in Bylvay PFIC expenses related to the completion of the PEDFIC 1
study.
- Selling, general and administrative expenses were $16.9 million
for the first quarter of 2022 compared with $15.3 million for the
first quarter of 2021, an increase of $1.6 million. The increase is
attributable to personnel and related expenses as we continue to
increase our headcount, and commercialization activities related to
Bylvay including our sales force and support for global expansion
efforts.
- Net loss for the first quarter of
2022 was $42.4 million, or $(2.19) per share, compared to $43.7
million, or $(2.29) per share for the first quarter of 2021.
- The Company had cash
and cash equivalents of $216.7 million as of March 31, 2022, versus
$248.1 million as of December 31, 2021. The Company expects to have
sufficient cash into 2024 based on current revenue and expense
projections. Bylvay 2022 sales are expected to be a minimum of $30
million.
Conference Call
Albireo will host a conference call and webcast today, May 16 at
4:30 p.m. ET. To access the live conference call by phone, dial
877-407-0792 (domestic) or 201-689-8263 (international), and
provide the access code 13729296. A live audio webcast will be
accessible from the Investors page at ir.albireopharma.com/. To
ensure a timely connection to the webcast, it is recommended that
participants register at least 15 minutes prior to the scheduled
start time. An archived version of the webcast will be available
for replay on the Events & Presentations section of the
Investors page of Albireo’s website for 3 months following the
event.
About Bylvay (odevixibat)
Bylvay is the first drug approved in the U.S. for the treatment
of pruritus in patients 3 months of age and older in all types of
progressive familial intrahepatic cholestasis (PFIC). Limitation of
Use: Bylvay may not be effective in PFIC type 2 patients with
ABCB11 variants resulting in non-functional or complete absence of
bile salt export pump protein (BSEP-3). The European Commission
(EC) and UK Medicines and Healthcare Products Regulatory Agency
(MHRA) have also granted marketing authorization of Bylvay for the
treatment of PFIC in patients aged 6 months or older. Bylvay is
available in Germany and the UK and will be available for sale in
other European countries following pricing and reimbursement
approval. A potent, once-daily, non-systemic ileal bile acid
transport inhibitor, Bylvay acts locally in the small intestine.
Bylvay can be taken as a capsule for patients that are able to
swallow capsules, or opened and sprinkled onto food, which is a
factor of key importance for adherence in a pediatric patient
population. The most common adverse reactions for Bylvay are
diarrhea, liver test abnormalities, vomiting, abdominal pain, and
fat-soluble vitamin deficiency. The medicine can only be obtained
with a prescription. For more information about using Bylvay, see
the package leaflet or contact your doctor or pharmacist. For full
prescribing information, visit www.bylvay.com.
In the U.S. and Europe, Bylvay has orphan exclusivity for its
approved PFIC indications, and orphan designations for the
treatment of ALGS, biliary atresia and primary biliary cholangitis.
Bylvay is being evaluated in the ongoing PEDFIC 2 open-label trial
in patients with PFIC, in the BOLD Phase 3 study for patients with
biliary atresia and the ASSERT Phase 3 study for ALGS.
Important Safety Information
- The most common adverse reactions for Bylvay are diarrhea,
liver test abnormalities, vomiting, abdominal pain, and fat-soluble
vitamin deficiency.
- Liver Test Abnormalities: Patients should obtain baseline liver
tests and monitor during treatment. Dose reduction or treatment
interruption may be required if abnormalities occur. For persistent
or recurrent liver test abnormalities, consider treatment
discontinuation.
- Diarrhea: Treat dehydration. Treatment interruption or
discontinuation may be required for persistent diarrhea.
- Fat-Soluble Vitamin (FSV) Deficiency: Patient should obtain
baseline vitamin levels and monitor during treatment. Supplement if
deficiency is observed. If FSV deficiency persists or worsens
despite FSV supplementation, discontinue treatment.
About ASSERT
ASSERT is a gold standard, prospective intervention trial with
35 sites across North America, Europe, Middle
East and Asia Pacific. The double-blind, randomized,
placebo-controlled trial is designed to evaluate the safety and
efficacy of 120 µg /kg/day Bylvay (odevixibat) for 24 weeks in
relieving pruritus in patients with ALGS. Secondary endpoints will
measure serum bile acid levels and safety and tolerability. The
trial enrolled patients aged 0 to 17 years of age with a
genetically confirmed diagnosis of ALGS. The primary efficacy
endpoint is a change from baseline in scratching to Month 6 (Weeks
21 to 24) as measured by the Albireo ObsRO caregiver instrument.
The key secondary efficacy endpoint is a change in serum bile acid
levels from baseline to the average of Week 20 and Week 24.
After completing the ASSERT trial, study participants have the
option to enroll in an extension study to continue receiving access
to Bylvay while it advances along the regulatory pathway. An EAP
program is also available to provide eligible patients with ALGS
with access to Bylvay (odevixibat) prior to the product’s approval
and reimbursement, subject to authorization by the relevant
competent authority. Learn more about Albireo’s commitment to
access at www.albireopharma.com/responsibility/access/.
EAP for ALGS
Albireo has partnered with Tanner Pharma Group for the ALGS EAP.
Eligible patients with ALGS, that are not in screening in the
ASSERT study, may receive Bylvay on a free-of-charge (FOC) basis,
subject to authorization by the relevant country competent
authority, and meeting of Albireo’s eligibility criteria. If you
are a physician who would like to request ALGS EAP access for your
patient, please send your enquiry to Tanner using
odevixibat@tannerpharma.com, and you will receive a response within
one working day with further information.
About Albireo
Albireo Pharma is a rare disease company focused on the
development of novel bile acid modulators to treat rare pediatric
and adult liver diseases. Albireo’s lead product, Bylvay, was
approved by the U.S. FDA as the first drug for the treatment of
pruritus in all types of progressive familial intrahepatic
cholestasis (PFIC), and it is also being developed to treat other
rare pediatric cholestatic liver diseases with Phase 3 trials in
Alagille syndrome (ALGS) and biliary atresia, as well as Open-label
Extension (OLE) studies for PFIC and ALGS. In Europe, Bylvay has
been approved for the treatment of PFIC with pricing listing in
Germany and guidance from the National Institute for Health and
Care Excellence (NICE) recommending Bylvay for use in the National
Health Service in England, Wales and Northern Ireland. The Company
has also completed a Phase 1 clinical trial for A3907 to advance
development in adult cholestatic liver disease, with IND-enabling
studies progressing with A2342 for viral and cholestatic liver
disease. Albireo was spun out from AstraZeneca in 2008 and is
headquartered in Boston, Massachusetts, with its key operating
subsidiary in Gothenburg, Sweden. For more information on Albireo,
please visit www.albireopharma.com.
Forward-Looking Statements
This press release 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:
Albireo’s commercialization plans; the plans for, or progress,
scope, cost, initiation, duration, enrollment, results or timing
for availability of results of, development of Bylvay, A3907, A2342
or any other Albireo product candidate or program; the PEDFIC 2
open-label trial in patients with PFIC; the pivotal trial for
Bylvay in biliary atresia (BOLD); the pivotal trial for Bylvay in
Alagille syndrome (ASSERT); the Phase 2 study for A3907 the
IND-enabling or clinical studies for A2342; the target
indication(s) for development or approval; 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 BOLD and ASSERT trials, the
Phase 2 study for A3907, and the IND-enabling and clinical studies
for A2342; the impact of the Expanded Access Program; expectations
that biliary atresia is the most common pediatric cholestatic liver
disease with no approved drug treatment; potential regulatory
approval and plans for potential commercialization of Bylvay in
additional countries; the potential benefits or competitive
position of Bylvay or any other Albireo product candidate or
program or the commercial opportunity in any target indication;
future price listings and reimbursement approvals of Bylvay; the
length of time for which Albireo’s cash resources are expected to
be sufficient; or Albireo’s plans, expectations or future
operations, financial position, revenues, costs or
expenses. Albireo often uses words such as “anticipates,”
“believes,” “plans,” “expects,” “projects,” “future,” “intends,”
“may,” “will,” “should,” “could,” “estimates,” “predicts,”
“potential,” “planned,” “continue,” “guidance,” or the negative of
these terms or other similar expressions to identify
forward-looking statements. Actual results, performance or
experience may differ materially from those expressed or implied by
any forward-looking statement as a result of various risks,
uncertainties and other factors, including, but not limited to:
there are no guarantees that Bylvay will be commercially
successful; we may encounter issues, delays or other challenges in
commercializing Bylvay; whether Bylvay receives adequate
reimbursement from third-party payors; the degree to which Bylvay
receives acceptance from patients and physicians for its approved
indication; challenges associated with execution of our sales
activities, which in each case could limit the potential of our
product; challenges associated with supply and distribution
activities, which in each case could limit our sales and the
availability of our product; results achieved in Bylvay in the
treatment of patients with PFIC may be different than observed in
clinical trials, and may vary among patients; potential 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 indications other than PFIC,
will be predictive of results from other clinical trials of Bylvay;
there is no guarantee that Bylvay will be approved in jurisdictions
or for indications beyond the jurisdictions in which or indications
for which Bylvay is currently approved; there is no guarantee that
our other products candidates will be approved; estimates of the
addressable patient population for target indications may prove to
be incorrect; 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 BOLD and ASSERT and the Phase 2 clinical trial of
A3907, and the outcomes of such trials; Albireo’s ability to obtain
coverage, pricing or reimbursement for approved products in the
United States or Europe; delays or other challenges in the
recruitment of patients for, or the conduct of, the Company’s
clinical trials; and the Company’s critical accounting policies.
These and other risks and uncertainties that Albireo faces are
described in greater detail under the heading “Risk Factors” in
Albireo’s most recent Annual Report on Form 10-K or in subsequent
filings that it makes with the Securities and Exchange Commission.
As a result of risks and uncertainties that Albireo faces, the
results or events indicated by any forward-looking statement may
not occur. Albireo cautions you not to place undue reliance on any
forward-looking statement. In addition, any forward-looking
statement in this press release represents Albireo’s views only as
of the date of this press release and should not be relied upon as
representing its views as of any subsequent date. Albireo disclaims
any obligation to update any forward-looking statement except as
required by applicable law.
Media Contact:Colleen Alabiso,
857-356-3905, colleen.alabiso@albireopharma.comLance Buckley,
917-439-2241, lbuckley@lippetaylor.com
Investor Contact:Hans Vitzthum, LifeSci
Advisors, LLC., 617-430-7578
Albireo Pharma, Inc.
Consolidated Balance
Sheets(in thousands, except share and per share
data)(unaudited)
|
|
March 31, |
|
December 31, |
|
|
2022 |
|
|
2021 |
|
Assets |
|
|
|
|
|
|
Current assets: |
|
|
|
|
|
|
Cash and cash equivalents |
|
$ |
216,655 |
|
|
$ |
248,107 |
|
Accounts receivable, net |
|
|
1,275 |
|
|
|
3,272 |
|
Inventory |
|
|
1,821 |
|
|
|
194 |
|
Prepaid expenses |
|
|
6,486 |
|
|
|
5,261 |
|
Other current assets |
|
|
2,490 |
|
|
|
12,096 |
|
Total current assets |
|
|
228,727 |
|
|
|
268,930 |
|
Property and equipment, net |
|
|
837 |
|
|
|
668 |
|
Goodwill |
|
|
17,260 |
|
|
|
17,260 |
|
Other assets |
|
|
14,437 |
|
|
|
15,193 |
|
Total assets |
|
$ |
261,261 |
|
|
$ |
302,051 |
|
Liabilities and Stockholders' Equity |
|
|
|
|
|
|
Current liabilities: |
|
|
|
|
|
|
Accounts payable |
|
$ |
6,369 |
|
|
$ |
6,516 |
|
Accrued expenses |
|
|
21,103 |
|
|
|
35,951 |
|
Other current liabilities |
|
|
4,035 |
|
|
|
2,880 |
|
Total current liabilities |
|
|
31,507 |
|
|
|
45,347 |
|
Liability related to sale of future royalties |
|
|
60,899 |
|
|
|
60,132 |
|
Note payable, net of discount |
|
|
10,080 |
|
|
|
10,004 |
|
Other long-term liabilities |
|
|
10,589 |
|
|
|
10,960 |
|
Total liabilities |
|
|
113,075 |
|
|
|
126,443 |
|
Stockholders’ Equity: |
|
|
|
|
|
|
Preferred stock, $0.01 par value per share — 50,000,000 shares
authorized at March 31, 2022 and
December 31, 2021; 0 and 0 shares issued and outstanding
at March 31, 2022 and December 31, 2021,
respectively |
|
|
— |
|
|
|
— |
|
Common stock, $0.01 par value per share — 60,000,000 shares
authorized at March 31, 2022 and December 31, 2021; 19,527,920 and
19,520,160 shares issued and outstanding at March 31, 2022,
respectively, and 19,304,312 and 19,296,552 shares issued and
outstanding at December 31, 2021, respectively |
|
|
195 |
|
|
|
193 |
|
Additional paid-in capital |
|
|
483,276 |
|
|
|
475,390 |
|
Accumulated other comprehensive income |
|
|
8,229 |
|
|
|
1,105 |
|
Accumulated deficit |
|
|
(343,284 |
) |
|
|
(300,850 |
) |
Treasury stock at cost, 7,760 shares at March 31, 2022 and December
31 2021, respectively |
|
|
(230 |
) |
|
|
(230 |
) |
Total stockholders’ equity |
|
|
148,186 |
|
|
|
175,608 |
|
Total liabilities and stockholders’ equity |
|
$ |
261,261 |
|
|
$ |
302,051 |
|
|
|
|
|
|
|
|
Albireo Pharma, Inc.
Consolidated Statements of
Operations(in thousands, except share and per
share data)(unaudited)
|
|
Three Months Ended March 31, |
|
|
2022 |
|
|
2021 |
|
Revenue: |
|
|
|
|
|
|
Product revenue, net |
|
$ |
4,656 |
|
|
$ |
— |
|
Royalty revenue |
|
|
2,176 |
|
|
|
1,966 |
|
Total revenue |
|
|
6,832 |
|
|
|
1,966 |
|
Cost and operating expenses: |
|
|
|
|
|
|
Cost of product revenue |
|
|
234 |
|
|
|
— |
|
Research and development |
|
|
21,903 |
|
|
|
19,943 |
|
Selling, general and administrative |
|
|
16,855 |
|
|
|
15,273 |
|
Other operating expense, net |
|
|
7,398 |
|
|
|
6,528 |
|
Total cost and operating expenses |
|
|
46,390 |
|
|
|
41,744 |
|
Operating loss |
|
|
(39,558 |
) |
|
|
(39,778 |
) |
Other loss: |
|
|
|
|
|
|
Interest expense, net |
|
|
(2,876 |
) |
|
|
(3,955 |
) |
Net loss |
|
$ |
(42,434 |
) |
|
$ |
(43,733 |
) |
Net loss per share attributable to holders of common stock: |
|
|
|
|
|
|
Net loss per common share - basic and diluted |
|
$ |
(2.19 |
) |
|
$ |
(2.29 |
) |
Weighted-average common shares used to compute basic and diluted
net loss per common share |
|
|
19,379,586 |
|
|
|
19,131,557 |
|
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