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 second quarter ended June 30, 2020.
“We are approaching arguably the most important moment in the
history of our company having achieved last patient visits in our
odevixibat Phase 3 program in PFIC and elobixibat Phase 2 program
in NASH,” said Ron Cooper, President and Chief Executive Officer of
Albireo. “We eagerly await the topline data in those two trials,
while continuing to enroll patients in our precedent setting
odevixibat biliary atresia pivotal program (BOLD) and gearing up to
initiate a third odevixibat pivotal trial in Alagille syndrome by
end of year. With a strong financial foundation and cash
runway into the beginning of 2022, we have enabled our continued
growth past the planned approval and commercial launch of
odevixibat for PFIC, supported additional odevixibat pivotal
trials, and advanced development of both elobixibat in NASH and the
exciting preclinical programs rounding out our pipeline.”
Recent Highlights
Odevixibat
- Achieved last patient visit in the PEDFIC 1 Phase 3 trial with
odevixibat in progressive familial intrahepatic cholestasis (PFIC).
Expect topline data to include 62 out of a planned 60 enrolled
patients, with no patients lost to follow-up due to COVID-19.
Company continues to anticipate topline data in the coming weeks
aligned to our mid-2020 guidance, with regulatory approval,
issuance of a rare pediatric disease priority review voucher and
launch anticipated in H2 2021.
- Enrolled first patients in Company’s Phase 3 BOLD study in
biliary atresia, a rare pediatric liver disease that is the leading
cause of liver transplants among children, and for which there is
no approved pharmacological treatment. BOLD is a double-blind,
randomized, placebo-controlled trial designed to evaluate the
efficacy and safety of odevixibat in children who have biliary
atresia and have undergone a Kasai procedure before age three
months. The U.S. Food and Drug Administration (FDA) and European
Commission have granted orphan designations for odevixibat in the
treatment of biliary atresia.
- Preparing to initiate clinical trial in Alagille syndrome by
the end of 2020, following agreement on the protocol design by U.S.
and European regulatory authorities. Topline data expected to
be available between the announcement of PFIC and biliary atresia
topline results. The FDA and European Commission have granted
orphan designations for odevixibat in the treatment of Alagille
syndrome.
- Launched Expanded Access Program (EAP) for eligible patients
with PFIC in the U.S., Canada, Australia and Europe.
- Agreed to financial support of a genetic testing program for
all types of PFIC and Alagille syndrome at no cost to qualified
patients in the U.S. The program builds on Albireo’s commitment to
support patients in their journey to diagnosis and treatment of
rare pediatric cholestatic liver diseases.
Elobixibat
- Achieved last patient visit in the Phase 2 trial in
nonalcoholic steatohepatitis (NASH) and nonalcoholic fatty liver
disease (NAFLD) designed as a proof of concept to demonstrate a
combination of positive trends in liver markers, CV risk factors
and favorable GI tolerability. Data will be available for 43
out of 47 patients, as 4 patients were lost to follow-up primarily
due COVID-19. Company anticipates topline data in the coming
weeks ahead of the odevixibat PEDFIC 1 data.
- Company continues to anticipate topline data by the end of the
year or early next year in a second Phase 2 study of elobixibat in
100 patients with NASH and NAFLD, conducted through partner EA
Pharma in Japan.
Early Stage Pipeline
- Progressed preclinical development and expect to complete
IND-enabling studies in lead preclinical candidate this
year.
Corporate
- Announced two financing transactions: restructured royalty
monetization agreement with HealthCare Royalty Partners III, L.P.
to receive an additional $15 million in non-dilutive capital for
elobixibat in the treatment of chronic constipation in Japan, and
an agreement with Hercules Capital, Inc. on a debt facility to
provide up to $80 million of new capital with initial draw down of
$10 million, resulting in net cash of $24.3 million and extending
the cash runway into the beginning of 2022.
- Hosted a Key Opinion Leader call with Chad Gwaltney, Ph.D., who
discussed key considerations for the design and implementation of
clinical outcome assessments, including patient-reported and
observer-reported outcomes in PEDFIC 1 & 2. Dr. Gwaltney
provided further background on the PRUCISION measurement tools used
in the Phase 3 trials, which were developed rigorously with
patients, caregivers, and expert clinicians, as well as in close
consultation with the FDA.
- Presented at the Jefferies virtual investor conference.
Second Quarter 2020 Financial Results
- Revenue was $1.9 million for the second quarter of 2020,
compared with $1.3 million for the second quarter of 2019. The
higher revenue was due to the estimated royalty revenue received
from EA Pharma for elobixibat for the treatment of chronic
constipation. The royalty revenue is passed on to HealthCare
Royalty Partners.
- R&D expenses were $18.4 million for the second quarter of
2020, compared with $11.0 million for the second quarter of 2019.
The higher expenses were primarily due to program expenses for
odevixibat, as well as personnel costs, as the Company continues to
develop additional indications for its lead asset.
- G&A expenses were $8.5 million for the second quarter of
2020, compared with $5.5 million for the second quarter of 2019.
The increase was attributable to personnel and related expenses, as
the Company continues to increase its headcount and
commercialization readiness expenses.
- Net loss for the second quarter of 2020 was $20.6 million, or
$(1.38) per share, compared with $16.6 million, or $(1.35) per
share for the second quarter of 2019.
- Company had cash and cash equivalents at June 30, 2020 of
$152.0 million. During the second quarter of 2020, an additional
$24.3 million of net cash was received from recently completed
non-dilutive financings. As a result, cash and cash equivalents are
anticipated to be sufficient into the beginning of 2022.
Conference CallAs previously announced, Albireo
will host a conference call and webcast today, August 6, 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 13706144. 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 AlbireoAlbireo 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 progressive familial intrahepatic cholestasis (PFIC) and biliary
atresia, with a third Phase 3 trial being planned in Alagille
syndrome. 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 2020 Best Places to Work in Massachusetts for the second
consecutive year. 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:
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 expectations regarding
the impact of COVID-19 on our business and our ability to adapt our
approach as appropriate; the Phase 3 clinical program for
odevixibat in patients with PFIC, the potential availability of
odevixibat through the EAP, whether the FDA continues to allow
odevixibat to be administered through the EAP, the pivotal trial
for odevixibat in biliary atresia (BOLD), the planned pivotal trial
for odevixibat in Alagille syndrome, and the Phase 2 clinical trial
for elobixibat in NAFLD/NASH; 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 Phase 3 PFIC trial for odevixibat, and the long-term
open-label extension study, the pivotal trial for odevixibat in
biliary atresia, the planned pivotal trial for odevixibat in
Alagille syndrome, or the Phase 2 trial for elobixibat in
NAFLD/NASH; the potential approval and commercialization of
odevixibat; discussions with the FDA or EMA 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 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: 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 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, the pivotal program
in biliary atresia or the planned pivotal program in Alagille
syndrome, 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, 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,
617-430-7578
Media Contact: Claire LaCagnina, 6 Degrees,
315-765-1462, clacagnina@6degreespr.com
Source: Albireo Pharma, Inc.
Albireo Pharma, Inc.
Condensed Consolidated Balance
Sheets
(in thousands, except share
data)
(unaudited)
|
|
|
|
|
|
|
|
|
June 30, |
|
December 31, |
|
|
2020 |
|
2019 |
Assets |
|
|
|
|
|
|
Current assets: |
|
|
|
|
|
|
Cash and cash equivalents |
|
$ |
152,020 |
|
|
$ |
131,843 |
|
Prepaid expenses and other current assets |
|
|
7,967 |
|
|
|
9,956 |
|
Total current assets |
|
|
159,987 |
|
|
|
141,799 |
|
Property and equipment,
net |
|
|
597 |
|
|
|
597 |
|
Goodwill |
|
|
17,260 |
|
|
|
17,260 |
|
Other assets |
|
|
6,161 |
|
|
|
5,413 |
|
Total assets |
|
$ |
184,005 |
|
|
$ |
165,069 |
|
Liabilities and
Stockholders' Equity |
|
|
|
|
|
|
Current liabilities: |
|
|
|
|
|
|
Accounts payable |
|
$ |
4,734 |
|
|
$ |
4,785 |
|
Accrued expenses |
|
|
11,752 |
|
|
|
13,486 |
|
Other current liabilities |
|
|
732 |
|
|
|
653 |
|
Total current liabilities |
|
|
17,218 |
|
|
|
18,924 |
|
Liability related to sale of
future royalties |
|
|
64,351 |
|
|
|
48,714 |
|
Note payable, net of
discount |
|
|
9,400 |
|
|
|
— |
|
Other long-term
liabilities |
|
|
3,916 |
|
|
|
4,270 |
|
Total liabilities |
|
|
94,885 |
|
|
|
71,908 |
|
Stockholders’ Equity: |
|
|
|
|
|
|
Common stock, $0.01 par value per share — 30,000,000 authorized at
June 30, 2020 and December 31, 2019; 14,989,021
and 12,749,443 issued and outstanding at June 30, 2020
and December 31, 2019, respectively |
|
|
149 |
|
|
|
127 |
|
Additional paid-in capital |
|
|
294,075 |
|
|
|
245,769 |
|
Accumulated other comprehensive income |
|
|
6,174 |
|
|
|
6,452 |
|
Accumulated deficit |
|
|
(211,278 |
) |
|
|
(159,187 |
) |
Total stockholders’ equity |
|
|
89,120 |
|
|
|
93,161 |
|
Total liabilities and
stockholders’ equity |
|
$ |
184,005 |
|
|
$ |
165,069 |
|
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, |
|
|
2020 |
|
2019 |
|
2020 |
|
2019 |
Revenue |
|
$ |
1,912 |
|
|
$ |
1,250 |
|
|
$ |
3,461 |
|
|
$ |
1,820 |
|
Operating
expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
|
18,397 |
|
|
|
11,034 |
|
|
|
34,527 |
|
|
|
19,363 |
|
General and administrative |
|
|
8,474 |
|
|
|
5,485 |
|
|
|
16,627 |
|
|
|
10,778 |
|
Other operating (income) expense, net |
|
|
(6,744 |
) |
|
|
8 |
|
|
|
72 |
|
|
|
2,304 |
|
Total operating expenses |
|
|
20,127 |
|
|
|
16,527 |
|
|
|
51,226 |
|
|
|
32,445 |
|
Operating loss |
|
|
(18,215 |
) |
|
|
(15,277 |
) |
|
|
(47,765 |
) |
|
|
(30,625 |
) |
Interest expense, net |
|
|
(2,388 |
) |
|
|
(1,351 |
) |
|
|
(4,326 |
) |
|
|
(2,660 |
) |
Net loss |
|
$ |
(20,603 |
) |
|
$ |
(16,628 |
) |
|
$ |
(52,091 |
) |
|
$ |
(33,285 |
) |
Net loss per share
attributable to holders of common stock: |
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per common share - basic and diluted |
|
$ |
(1.38 |
) |
|
$ |
(1.35 |
) |
|
$ |
(3.58 |
) |
|
$ |
(2.73 |
) |
Weighted-average shares outstanding: |
|
|
|
|
|
|
|
|
|
|
|
|
Weighted-average common shares used to compute basic and diluted
net loss per common share |
|
|
14,981,756 |
|
|
|
12,355,969 |
|
|
|
14,556,986 |
|
|
|
12,178,376 |
|
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