Albireo Pharma, Inc. (Nasdaq: ALBO), a clinical-stage orphan
pediatric liver disease company developing novel bile acid
modulators, today provided a business update and reported financial
results for the fourth quarter and year ended December 31,
2019.
“In 2019, we took major steps forward in multiple development
programs, advanced commercial preparations and evolved as an
organization, laying the groundwork for Albireo to enter the next
stage,” said Ron Cooper, President and Chief Executive Officer of
Albireo. “We have always believed in odevixibat’s potential across
a range of cholestatic liver diseases, and 2020 will be a
transformational year, as we expect results from our Phase 3 trial
in PFIC, look forward to initiating a pivotal trial in biliary
atresia and plan to begin a pivotal trial in Alagille syndrome
following discussions with the FDA this quarter. Meanwhile, we
eagerly anticipate the results from the elobixibat NASH/NAFLD study
and are excited to have selected a lead candidate to advance from
our robust library of preclinical compounds.”
Recent Highlights
Odevixibat
- Completed full randomization of the PEDFIC 1 Phase 3 study in
progressive familial intrahepatic cholestasis (PFIC). The PEDFIC 1
trial is studying odevixibat in both PFIC type 1 and type 2
patients aged 6 months to 18 years. Patients randomized to
odevixibat are being treated with high- (120µg/kg) or low-
(40µg/kg) dose once-daily oral capsules or sprinkles. The trial
uses Albireo’s planned commercial formulation of odevixibat, which
does not require refrigeration. Albireo expects topline results
mid-2020, and anticipates a potential first regulatory approval and
launch in the second half of 2021.
- Continued enrolling patients in PEDFIC 2, the long-term,
open-label extension study of PEDFIC 1. Cohort 1 of PEDFIC 2 is
composed of patients who have rolled over from the PEDFIC 1 study.
Albireo now has patients who have been treated with odevixibat for
more than 1 year. Cohort 2 is expected to broaden the evidence base
for odevixibat by including PFIC patients who were ineligible for
PEDFIC 1. This includes patients with all types of PFIC, and
patients younger than 6 months or older than 18 years of
age.
- Received clearance from the U.S. Food and Drug Administration
(FDA) for the company’s Investigational New Drug (IND) application
to initiate a precedent-setting global pivotal trial in biliary
atresia following agreement on a study design. Biliary atresia is a
rare pediatric liver disease that is the leading cause of liver
transplants among children, and for which there is no approved
pharmacological therapy. The double-blind, placebo-controlled trial
is designed to enroll approximately 200 patients at 70 sites.
Patients will receive either placebo or high-dose (120µg/kg)
odevixibat once daily. The primary endpoint is survival with native
liver after 2 years of treatment. The FDA and European Commission
have granted orphan designations for odevixibat in the treatment of
biliary atresia. Albireo plans to initiate the trial in 1H
2020.
- Plans to commence an additional pivotal program in Alagille
syndrome by the end of 2020, following scheduled FDA interactions
in the first quarter. The FDA and the European Commission have
granted orphan designations for odevixibat in the treatment of
Alagille syndrome.
- Advanced preparations for commercialization across physician,
patient and payer communities. The company initiated activities to
develop a detailed understanding of treatment centers, continued to
build the PFIC Voices initiative in collaboration with patients and
families, and progressed the odevixibat value story and economic
models for reimbursement.
Pipeline
- Nearing completion of enrollment in the company’s Phase 2
multicenter, placebo-controlled clinical trial of elobixibat 5mg in
non-alcoholic fatty liver disease (NAFLD)/nonalcoholic
steatohepatitis (NASH). Albireo expects topline data by mid-2020.
In addition, the company expects data from a second study being
conducted in Japan with elobixibat 10mg and in combination with a
bile acid sequestrant later this year or early 2021.
- Selected lead preclinical product candidate, filed for
intellectual property protection and initiated IND-enabling
studies.
Fourth Quarter 2019 Financial Results
- Revenues were $6.4 million for the fourth quarter of 2019,
compared to $0.6 million for the fourth quarter of 2018. The
year-over-year increase was primarily the result of estimated
milestone royalty revenue received from EA Pharma for elobixibat
for the treatment of chronic constipation.
- R&D expense was $14.2 million for the fourth quarter of
2019, compared to $9.5 million for the fourth quarter of 2018. The
year-over-year change was primarily the result of program expenses
for odevixibat and elobixibat, as well as personnel costs, as we
continue to increase our program activities and
headcount.
- G&A expense was $6.2 million for the fourth quarter of
2019, compared to $5.8 million for the fourth quarter of 2018. The
year-over-year change was primarily the result of personnel and
related expenses, as we continue to increase our
headcount.
- Net loss for the fourth quarter of 2019 was $7.5 million, or
$(0.57) per share, compared to $15.9 million, or $(1.34) per share
for the fourth quarter of 2018.
Financial Results for the Year Ended December 31,
2019
- Revenues were $9.6 million for the year ended December 31,
2019, compared to $12.7 million for the year ended December 31,
2018. The year-over-year decline is primarily due to a milestone
payment received in 2018 from EA Pharma due to the approval by the
Japanese MHLW of elobixibat for the treatment of chronic
constipation offset by royalty revenue from EA Pharma.
- R&D expense was $45.6 million for 2019 up from $31.7
million for the same period in 2018. The year-over-year increase
was primarily the result of program expenses for odevixibat,
elobixibat and preclinical work, as well as personnel costs, as we
continue to increase our program activities and
headcount.
- G&A expense was $23.0 million for 2019, compared to $18.1
million for the previous year. The year-over-year increase was
primarily the result of personnel and stock-based compensation, as
we continue to increase our headcount.
- Net loss for the year ended December 31, 2019, was $62.7
million, or $(5.04) per share, compared to $46.1 million, or
$(3.94) per share for the year ended December 31, 2018.
- The company had cash and cash equivalents at December 31, 2019,
of $131.8 million, which compares to $163.9 million at December 31,
2018.
Financial GuidanceFor the full year 2020, the
company anticipates total expenses, including R&D and G&A
expenses, to be around $100M. Following a February 2020 common
stock offering of $43.2 million, Albireo now anticipates that its
current cash balance will be sufficient to meet its operating needs
into the second half 2021.
Conference CallAs previously announced, Albireo
will host a conference call and webcast today, March 2, 2020,
at 10:00 a.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 13697430. A live audio webcast will be
accessible from the Media & Investors page of Albireo’s
website, http://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 Media & Investors
page of Albireo’s website for 3 months following the event.
About Albireo Albireo Pharma is a
clinical-stage biopharmaceutical company focused on the development
of novel bile acid modulators to treat orphan pediatric liver
diseases, and other liver and gastrointestinal diseases and
disorders. Albireo’s lead product candidate, odevixibat, is being
developed to treat rare pediatric cholestatic liver diseases and is
in Phase 3 development in its initial target indication,
progressive familial intrahepatic cholestasis (PFIC). Albireo’s
clinical pipeline also includes two Phase 2 product candidates.
Elobixibat is in Phase 2 development in NAFLD and NASH. Approved in
Japan for the treatment of chronic constipation, elobixibat is the
first ileal bile acid transporter (IBAT) inhibitor approved
anywhere in the world.
Albireo was spun out from AstraZeneca in 2008. Albireo Pharma is
located in Boston, Mass., and its key operating subsidiary is
located in Gothenburg, Sweden. The Boston Business Journal named
Albireo one of the 2019 Best Places to Work in Massachusetts. For
more information on Albireo, please visit
www.albireopharma.com.
About OdevixibatOdevixibat is a product
candidate being developed to treat rare pediatric cholestatic liver
diseases and is in Phase 3 development in its initial target
indication, progressive familial intrahepatic cholestasis (PFIC). A
highly potent and selective inhibitor of the ileal bile acid
transporter (IBAT), odevixibat has minimal systemic exposure and
acts locally in the small intestine.
The odevixibat PFIC program has received fast track, rare
pediatric disease and orphan drug designations in the United
States. In addition, the U.S. Food and Drug Administration (FDA)
has granted orphan drug designation to odevixibat for the treatment
of Alagille syndrome, biliary atresia and primary biliary
cholangitis. The European Medicines Agency (EMA) has granted
odevixibat orphan designation, as well as access to the PRIority
MEdicines (PRIME) scheme for the treatment of PFIC. Its Pediatric
Committee has agreed to Albireo's odevixibat Pediatric
Investigation Plan for PFIC. EMA also has granted orphan
designation to odevixibat for the treatment of Alagille syndrome,
biliary atresia and primary biliary cholangitis.
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:
the plans for, or progress, scope, cost, initiation, duration,
enrollment, results or timing for availability of results of,
development of odevixibat, elobixibat or any other Albireo product
candidate or program, including regarding the Phase 3 clinical
program for odevixibat in patients with PFIC; the planned pivotal
trial for odevixibat in biliary atresia, the Phase 2 clinical trial
for elobixibat in NAFLD/NASH, the target indication(s) for
development, the size, design, population, location, conduct,
objective, enrollment, duration or endpoints of any clinical trial,
or the timing for initiation or completion of or reporting of
results from any clinical trial, including the double-blind Phase 3
PFIC trial for odevixibat, and the long-term open-label extension
study, the planned pivotal trials for odevixibat in biliary atresia
and Alagille syndrome, or the Phase 2 trial for elobixibat in
NAFLD/NASH; the potential approval and commercialization of
odevixibat; discussions with the FDA regarding our programs; 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 an orphan drug designation; the pricing of odevixibat
if approved; the period for which Albireo’s cash resources will be
sufficient to fund its operating requirements (runway); 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,” and 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: whether favorable findings
from clinical trials of odevixibat to date, including findings in
indications other than PFIC, will be predictive of results from the
trials comprising the Phase 3 PFIC program or any other clinical
trials of odevixibat; whether either or both of
the FDA and EMA will determine that the primary endpoint
for their respective evaluations and treatment duration of the
double-blind Phase 3 trial in patients with PFIC are sufficient,
even if the 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 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 odevixibat, including the trials
comprising the Phase 3 PFIC program, or the planned pivotal
programs in biliary atresia and Alagille syndrome, and the outcomes
of such trials; Albireo’s completion of discussions with the FDA
regarding the planned pivotal trial for odevixibat in Alagille
syndrome; 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, or the conduct of, the
double-blind Phase 3 trial or other pivotal trials; and Albireo’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.
Investor Contact: Hans Vitzthum, LifeSci
Advisors, LLC.,
212-915-2568
Media Contact: Heather Anderson, 6 Degrees,
980-938-0260, handerson@6degreespr.com
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Albireo Pharma, Inc.Condensed
Consolidated Balance Sheets(in thousands, except
share and per share data)(unaudited) |
|
|
|
|
|
December 31, |
|
December 31, |
|
|
2019 |
|
2018 |
Assets |
|
|
|
|
|
|
Current
assets: |
|
|
|
|
|
|
Cash and cash equivalents |
|
$ |
131,843 |
|
|
$ |
163,885 |
|
Prepaid
expenses and other current assets |
|
|
9,956 |
|
|
|
3,765 |
|
Total current assets |
|
|
141,799 |
|
|
|
167,650 |
|
Property and
equipment, net |
|
|
597 |
|
|
|
187 |
|
Goodwill |
|
|
17,260 |
|
|
|
17,260 |
|
Other
assets |
|
|
5,413 |
|
|
|
369 |
|
Total
assets |
|
$ |
165,069 |
|
|
$ |
185,466 |
|
Liabilities
and Stockholders' Equity |
|
|
|
|
|
|
Current
liabilities: |
|
|
|
|
|
|
Accounts
payable |
|
$ |
4,785 |
|
|
$ |
4,352 |
|
Accrued expenses |
|
|
13,486 |
|
|
|
8,165 |
|
Other
current liabilities |
|
|
653 |
|
|
|
308 |
|
Total
current liabilities |
|
|
18,924 |
|
|
|
12,825 |
|
Liability
related to sale of future royalties |
|
|
48,714 |
|
|
|
49,969 |
|
Other
long-term liabilities |
|
|
4,270 |
|
|
|
35 |
|
Total
liabilities |
|
|
71,908 |
|
|
|
62,829 |
|
Stockholders’ Equity: |
|
|
|
|
|
|
Common
stock, $0.01 par value per share — 30,000,000 authorized at
December 31, 2019 and December 31, 2018;
12,749,443 and 11,969,928 issued and outstanding at
December 31, 2019 and December 31, 2018,
respectively |
|
|
127 |
|
|
|
120 |
|
Additional paid-in capital |
|
|
245,769 |
|
|
|
214,694 |
|
Accumulated
other comprehensive income |
|
|
6,452 |
|
|
|
4,293 |
|
Accumulated deficit |
|
|
(159,187 |
) |
|
|
(96,470 |
) |
Total stockholders’ equity |
|
|
93,161 |
|
|
|
122,637 |
|
Total
liabilities and stockholders’ equity |
|
$ |
165,069 |
|
|
$ |
185,466 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
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Albireo Pharma, Inc.Condensed
Consolidated Statements of Operations(in
thousands, except share and per share
data)(unaudited) |
|
|
|
|
|
|
Three Months Ended December 31, |
|
Year Ended December 31, |
|
|
2019 |
|
2018 |
|
2019 |
|
2018 |
Revenue |
|
$ |
6,431 |
|
|
$ |
571 |
|
|
$ |
9,636 |
|
|
$ |
12,740 |
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
|
14,216 |
|
|
|
9,504 |
|
|
|
45,575 |
|
|
|
31,732 |
|
General and administrative |
|
|
6,175 |
|
|
|
5,845 |
|
|
|
22,963 |
|
|
|
18,061 |
|
Other operating expense (income), net |
|
|
(4,109 |
) |
|
|
(540 |
) |
|
|
2,210 |
|
|
|
837 |
|
Total operating expenses |
|
|
16,282 |
|
|
|
14,809 |
|
|
|
70,748 |
|
|
|
50,630 |
|
Operating
loss |
|
|
(9,851 |
) |
|
|
(14,238 |
) |
|
|
(61,112 |
) |
|
|
(37,890 |
) |
Interest
expense, net |
|
|
(1,362 |
) |
|
|
(789 |
) |
|
|
(5,296 |
) |
|
|
(4,838 |
) |
Non-operating income (expense), net |
|
|
3,691 |
|
|
|
(817 |
) |
|
|
3,691 |
|
|
|
(3,363 |
) |
Net loss
before income taxes |
|
|
(7,522 |
) |
|
|
(15,844 |
) |
|
|
(62,717 |
) |
|
|
(46,091 |
) |
Income
tax |
|
|
— |
|
|
|
20 |
|
|
|
— |
|
|
|
20 |
|
Net
loss |
|
$ |
(7,522 |
) |
|
$ |
(15,864 |
) |
|
$ |
(62,717 |
) |
|
$ |
(46,111 |
) |
Net loss per
share attributable to holders of common stock: |
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per common share - basic and diluted |
|
$ |
(0.57 |
) |
|
$ |
(1.34 |
) |
|
$ |
(5.04 |
) |
|
$ |
(3.94 |
) |
Weighted-average shares outstanding: |
|
|
12,698,492 |
|
|
|
11,969,928 |
|
|
|
12,437,742 |
|
|
|
11,702,785 |
|
|
|
|
|
|
|
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