Albireo Pharma, Inc. (Nasdaq: ALBO), a rare liver disease company
developing novel bile acid modulators to treat rare pediatric and
adult liver diseases, today provided a business update and reported
financial results for the quarter ended June 30, 2022.
"We continue to make meaningful progress with the global Bylvay
launch as we increase the number of patients on Bylvay, including
reimbursed patients and those covered through our managed access
programs. Compliance and persistency continue to be strong,
providing these patients an effective and well tolerated drug that
has potential to be their lifetime therapy, and we look forward to
our planned expansion beyond PFIC with the imminent ASSERT Phase 3
trial in Alagille syndrome readout this fall," said Ron Cooper,
President and Chief Executive Officer of Albireo.
Q2 and Upcoming Highlights
- Bylvay net product revenue was $5.9
million, with $3.5 million from the United States and $2.4 million
from international markets. As of the end of Q2 2022, there are 245
total Bylvay patients—which include reimbursed, non-reimbursed and
rollover patients—representing +18% growth compared to Q1. U.S.
prescriber base grew quarter over quarter with unique prescribers
increasing from 57 to 73.
- Bylvay reimbursement confirmed in
the U.S., Germany, United Kingdom, Italy and Belgium.
- Positively concluded the pricing
& reimbursement negotiation in Italy for Bylvay, thus enabling
access for Italian PFIC patients this fall. Bylvay was granted
“Full Therapeutic Innovation” status by the AIFA (Italian
Medicines Agency) which is a special designation applied to select
products where there is a high unmet need, added clinical benefit,
and robustness of the scientific evidence. This status will provide
access to dedicated national funding and direct inclusion into
regional formularies within 60 days from the official publication
of the Italian marketing authorization decree, expected next
month.
- Successfully completed negotiations
with RIZIV-INAMI in Belgium, with reimbursement granted from
October 1, 2022.
- Launched in Scotland following
Scottish Medicines Consortium (SMC) publication of the assessment
report for Bylvay in July. Bylvay is available for PFIC through the
ultra-orphan pathway assessment for medicines that treat very rare,
chronic and disabling conditions.
- In the U.S., nearly all patients,
across public and private insurers, have a path to coverage for
Bylvay.
- Presented and published data from
the global Phase 3 PEDFIC 1 & PEDFIC 2 studies, which were the
first and largest successful global gold standard pediatric Phase 3
studies ever conducted in cholestatic liver disease. The Lancet
Gastroenterology & Hepatology published the results of the
pivotal PEDFIC 1 trial of Bylvay in PFIC. Eleven abstracts were
also accepted and presented at the International Liver CongressTM
(EASL) and European Society for Paediatric Gastroenterology,
Hepatology and Nutrition (ESPGHAN).
- On track to report topline data
from the Phase 3 ASSERT study in Alagille syndrome (ALGS) this
fall. ASSERT is a gold standard, prospective intervention trial
with 35 sites across North America, Europe, Middle East
and Asia Pacific. Albireo currently has 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 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 America and China. The study remains on track
for full enrollment in 2022 and topline data in 2024.
Pipeline News:
- Plan to initiate a Phase 1 first-in-human study for A2342 by
the end of 2022. This will follow recently completed IND-enabling
toxicology studies. A2342 is the first oral sodium-taurocholate
co-transporting peptide (NTCP) inhibitor in development for
hepatitis B and D.
Corporate News:
- Completed an agreement with Medison
Canada, a subsidiary of Medison. Similar to the existing agreement
with Medison in Israel, this one is for the commercial
distributorship of Bylvay in Canada. Medison is recognized as a
leader in rare diseases with partnerships with top-tier biotech
companies in international markets.
- Appoints Habib J. Dable, Former
President and CEO of Acceleron Pharma, and Susan Alesina, Vice
President, National Business Development and Alliances at Boston
Children’s Hospital, as members of the Company’s Board of
Directors.
Second Quarter 2022 Financial Results
- Product revenue, net was $5.9 million for the second 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 second quarter of 2021.
- Royalty revenue was $2.3 million for the second quarter of
2022, compared with $2.4 million for the second quarter of 2021, a
decrease of $0.1 million. The decrease relates to estimated royalty
revenue which is passed on to HealthCare Royalty Partners.
- Cost of product revenue was $0.8 million for the second 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 second quarter of 2021.
- R&D expenses were $22.9 million for the second quarter of
2022 compared with $20.9 million for the second quarter of 2021, an
increase of $2.0 million. The increase in R&D expenses for the
second quarter of 2022 was principally due to expenses related to
clinical program activities, personnel expenses and other costs as
we continue to increase our headcount and program activities. The
increase in program activities related to ongoing phase 3 clinical
trials for biliary atresia and Alagille syndrome, as well as Bylvay
PFIC expenses and were partially offset by a decrease in
preclinical expenses and A3907 due to completion of the Phase 1
study.
- Selling, general and administrative expenses were $21.6 million
for the second quarter of 2022 compared with $16.9 million for the
second quarter of 2021, an increase of $4.7 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 second quarter of
2022 was $39.9 million, or $(2.04) per share, compared to $36.4
million, or $(1.90) per share for the second quarter of 2021.
- The Company had cash
and cash equivalents of $181.0 million as of June 30, 2022, versus
$216.7 million as of March 31, 2022. The Company expects to have
sufficient cash into 2024 based on current revenue and expense
projections. Bylvay 2022 sales are now expected to be $24
million.
Conference Call
To access the live conference call by phone, dial 1-855-327-6837
(domestic) or 1-631-891-4304 (international), and provide the
access code 10019926. 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. A potent,
once-daily, non-systemic ileal bile acid transporter inhibitor
(IBATi), Bylvay has minimal systemic exposure and 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 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 in Europe, Bylvay has been approved
for the treatment of PFIC. Bylvay is also being developed to treat
other rare pediatric cholestatic liver diseases with global Phase 3
trials in Alagille syndrome (ALGS) and biliary atresia, as well as
Open-label Extension (OLE) studies for PFIC and ALGS. 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. |
Condensed Consolidated Balance Sheets |
(in thousands, except share data) |
|
|
|
|
|
June 30, |
|
December 31, |
|
|
|
2022 |
|
|
|
2021 |
|
|
|
(Unaudited) |
|
|
|
Assets |
|
|
|
|
|
|
Current assets: |
|
|
|
|
|
|
Cash and cash equivalents |
|
$ |
180,971 |
|
|
$ |
248,107 |
|
Accounts receivable, net |
|
|
1,849 |
|
|
|
3,272 |
|
Inventory |
|
|
2,096 |
|
|
|
194 |
|
Prepaid expenses |
|
|
8,170 |
|
|
|
5,261 |
|
Other current assets |
|
|
2,666 |
|
|
|
12,096 |
|
Total current assets |
|
|
195,752 |
|
|
|
268,930 |
|
Property and equipment, net |
|
|
1,388 |
|
|
|
668 |
|
Goodwill |
|
|
17,260 |
|
|
|
17,260 |
|
Other assets |
|
|
14,614 |
|
|
|
15,193 |
|
Total assets |
|
$ |
229,014 |
|
|
$ |
302,051 |
|
Liabilities and Stockholders' Equity |
|
|
|
|
|
|
Current liabilities: |
|
|
|
|
|
|
Accounts payable |
|
$ |
5,394 |
|
|
$ |
6,516 |
|
Accrued expenses |
|
|
23,034 |
|
|
|
35,951 |
|
Current portion of note payable, net of discount |
|
|
10,158 |
|
|
|
— |
|
Other current liabilities |
|
|
5,005 |
|
|
|
2,880 |
|
Total current liabilities |
|
|
43,591 |
|
|
|
45,347 |
|
Liability related to sale of future royalties |
|
|
61,493 |
|
|
|
60,132 |
|
Note payable, net of discount |
|
|
— |
|
|
|
10,004 |
|
Other long-term liabilities |
|
|
10,253 |
|
|
|
10,960 |
|
Total liabilities |
|
|
115,337 |
|
|
|
126,443 |
|
Stockholders’ Equity: |
|
|
|
|
|
|
Preferred stock, $0.01 par value per share — 50,000,000 shares
authorized at June 30, 2022 and December 31, 2021; 0
and 0 shares issued and outstanding at June 30, 2022 and
December 31, 2021, respectively |
|
|
— |
|
|
|
— |
|
Common stock, $0.01 par value per share — 60,000,000 shares
authorized at June 30, 2022 and December 31, 2021; 19,610,205 and
19,602,445 shares issued and outstanding at June 30, 2022,
respectively, and 19,304,312 and 19,296,552 shares issued and
outstanding at December 31, 2021, respectively |
|
|
196 |
|
|
|
193 |
|
Additional paid-in capital |
|
|
488,692 |
|
|
|
475,390 |
|
Accumulated other comprehensive income |
|
|
8,252 |
|
|
|
1,105 |
|
Accumulated deficit |
|
|
(383,233 |
) |
|
|
(300,850 |
) |
Treasury stock at cost, 7,760 shares at June 30, 2022 and December
31 2021, respectively |
|
|
(230 |
) |
|
|
(230 |
) |
Total stockholders’ equity |
|
|
113,677 |
|
|
|
175,608 |
|
Total liabilities and stockholders’ equity |
|
$ |
229,014 |
|
|
$ |
302,051 |
|
|
|
|
|
|
|
|
Albireo Pharma, Inc. |
Condensed Consolidated Statements of
Operations |
(in thousands, except share and per share
data) |
(Unaudited) |
|
|
|
Three Months Ended June 30, |
|
Six Months Ended June 30, |
|
|
|
2022 |
|
|
|
2021 |
|
|
|
2022 |
|
|
|
2021 |
|
Revenue: |
|
|
|
|
|
|
|
|
|
|
|
|
Product revenue, net |
|
$ |
5,891 |
|
|
$ |
— |
|
|
$ |
10,547 |
|
|
$ |
— |
|
Royalty revenue |
|
|
2,315 |
|
|
|
2,428 |
|
|
|
4,491 |
|
|
|
4,394 |
|
Total revenue |
|
|
8,206 |
|
|
|
2,428 |
|
|
|
15,038 |
|
|
|
4,394 |
|
Cost and operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
Cost of product revenue |
|
|
776 |
|
|
|
— |
|
|
|
1,010 |
|
|
|
— |
|
Research and development |
|
|
22,888 |
|
|
|
20,894 |
|
|
|
44,791 |
|
|
|
40,837 |
|
Selling, general and administrative |
|
|
21,600 |
|
|
|
16,940 |
|
|
|
38,455 |
|
|
|
32,213 |
|
Other operating expense (income), net |
|
|
145 |
|
|
|
(2,374 |
) |
|
|
7,543 |
|
|
|
4,154 |
|
Total cost and operating expenses |
|
|
45,409 |
|
|
|
35,460 |
|
|
|
91,799 |
|
|
|
77,204 |
|
Operating loss |
|
|
(37,203 |
) |
|
|
(33,032 |
) |
|
|
(76,761 |
) |
|
|
(72,810 |
) |
Other loss: |
|
|
|
|
|
|
|
|
|
|
|
|
Interest expense, net |
|
|
(2,746 |
) |
|
|
(3,389 |
) |
|
|
(5,622 |
) |
|
|
(7,344 |
) |
Net loss |
|
$ |
(39,949 |
) |
|
$ |
(36,421 |
) |
|
$ |
(82,383 |
) |
|
$ |
(80,154 |
) |
Net loss per share attributable to holders of common stock: |
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per common share - basic and diluted |
|
$ |
(2.04 |
) |
|
$ |
(1.90 |
) |
|
$ |
(4.23 |
) |
|
$ |
(4.18 |
) |
Weighted-average common shares used to compute basic and diluted
net loss per common share |
|
|
19,585,164 |
|
|
|
19,200,747 |
|
|
|
19,482,943 |
|
|
|
19,196,798 |
|
Albireo Pharma (NASDAQ:ALBO)
Historical Stock Chart
From Mar 2024 to Apr 2024
Albireo Pharma (NASDAQ:ALBO)
Historical Stock Chart
From Apr 2023 to Apr 2024