Albireo Pharma, Inc. (Nasdaq: ALBO), a clinical-stage rare liver
disease company developing novel bile acid modulators, today
provided a business update and reported financial results for the
third quarter ended September 30, 2020.
“There is great excitement around our very positive data and
late breaker acceptance for PEDFIC 1, the largest global Phase 3
clinical trial in PFIC,” said Ron Cooper, President and Chief
Executive Officer of Albireo. “We are moving to a new stage for
Albireo, anticipating regulatory approval, priority review voucher
issuance and launch in the second half of next year, while
deepening our pipeline with Phase 3 trials in biliary atresia and
Alagille syndrome and advancing A3907 in adult liver diseases.”
Recent and Upcoming
Highlights
Odevixibat
- Achieved high statistical significance on both the U.S. and EU
primary endpoints for PEDFIC 1, the largest ever global Phase 3
clinical trial evaluating the efficacy and safety of odevixibat.
The study demonstrated sustained reductions in bile acids and
pruritus, improved growth parameters, and was well tolerated with
very low incidence of diarrhea for both PFIC1 and PFIC2 children.
Positive results from the trial were announced on September 8,
2020: News Release: Albireo Phase 3 Trial Meets Both Primary
Endpoints for Odevixibat in PFIC.
- On track to complete regulatory filings in the U.S. and EU no
later than early 2021, in anticipation of potential regulatory
approval, issuance of a rare pediatric disease priority review
voucher and launch of odevixibat in the second half of
2021.
- Announced late-breaker acceptances at the American Association
for the Study of Liver Diseases (AASLD) The Liver Meeting Digital
Experience™ (TLMdX), being held November 13-16. News Release:
Albireo’s Odevixibat PFIC Phase 3 Results Accepted for AASLD
Late-Breakers. The full data set from the PEDFIC 1 trial will be
presented. There will also be a late breaker poster session for
interim results from PEDFIC 2, the open-label Phase 3 extension
study of the long-term efficacy and safety of odevixibat in
children with PFIC.
- BOLD, the second Phase 3 trial with odevixibat, continues to
activate new sites and enroll patients despite COVID-19 challenges.
Biliary atresia is the most common pediatric cholestatic liver
disease, and the number one cause of liver transplantation in
children.
- On track to initiate third Phase 3 trial of odevixibat in
Alagille syndrome by the end of 2020.
Early Stage Pipeline
- Preclinical development programs are progressing in adult liver
disease and the Company expects to complete IND-enabling studies
for a new lead preclinical candidate A3907 this year. The Company
will present adult liver disease data at the upcoming AASLD meeting
on November 13-16, 2020. News Release: Albireo’s Odevixibat PFIC
Phase 3 Results Accepted for AASLD Late-Breakers.
Corporate
- Completed an underwritten public offering of 4,000,000 shares
of common stock at a price of $40.00 per share. The net proceeds
from this offering were approximately $150.4 million after
deducting underwriting discounts and commissions but before
offering expenses.
- Despite the global COVID-19 pandemic, Albireo continues to
progress clinical programs and commercial planning, while
monitoring the situation closely in anticipation of product
filings, approvals and launch in 2021.
- Following the AASLD meeting, Albireo will host an investor call
on November 17, 2020 to present highlights from the Company’s
odevixibat PEDFIC 1 and PEDFIC 2 data presentations. The Company
will also highlight data on novel bile acid modulator approaches
and A3907, a new product candidate for adult liver diseases.
Third Quarter
2020 Financial
Results
- Revenues were $2.1 million for the third quarter of 2020,
compared with $1.4 million for the third 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 $22.2 million for the third quarter of
2020, compared with $12.0 million for the third quarter of 2019.
The higher expenses were primarily due to personnel expenses as the
Company continues to increase headcount, as well as program
expenses related to odevixibat as the Company prepares for
regulatory filings and continues to develop additional indications
for its lead asset. Continued advancement of preclinical programs
also contributed to higher program expenses.
- G&A expenses were $11.7 million for the third quarter of
2020, compared with $6.0 million for the third 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 third quarter of 2020 was $30.7 million, or
$(1.96) per share, compared with $21.9 million, or $(1.73) per
share for the third quarter of 2019.
- The Company had cash and cash equivalents on September 30,
2020, of $278.7 million. During the third quarter of 2020, an
additional $150.4 million of net proceeds were received from
recently completed public financings after deducting underwriting
discounts and commissions but before offering expenses. As a
result, cash and cash equivalents are anticipated to be sufficient
to fully fund the launches of odevixibat and into the revenue
generating phase for Albireo.
Virtual Business Update
CallAlbireo will host a post-AASLD event on Tuesday,
November 17, 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 13712304. 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 Business Update webcast will
be available for replay on the Events & Presentations section
of the Media & Investors page of Albireo’s website for 2 months
following the event.
About OdevixibatOdevixibat is an
investigational product candidate being developed to treat rare
pediatric cholestatic liver diseases, including progressive
familial intrahepatic cholestasis (PFIC), biliary atresia and
Alagille syndrome. A highly potent, once-daily, non-systemic ileal
bile acid transport inhibitor (IBATi), odevixibat acts locally in
the small intestine. Odevixibat does not require refrigeration
and can be taken as a capsule for older children, or opened and
sprinkled onto food, which are factors of key importance for
adherence in a pediatric patient population. The Company announced
positive results from the Phase 3 PEDFIC 1 trial with odevixibat in
September. Odevixibat is currently being evaluated in the ongoing
PEDFIC 2 open-label trial (NCT03659916) in patients with PFIC and
the BOLD Phase 3 trial in patients with biliary atresia
(NCT04336722). Initiation of a pivotal Phase 3 trial of odevixibat
for Alagille syndrome is also anticipated by the end of 2020.
Odevixibat has received fast track, rare pediatric disease and
orphan drug designations in the United States. In
addition, the FDA has granted orphan drug designation to
odevixibat for the treatment of Alagille syndrome, biliary atresia
and primary biliary cholangitis. The 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 and biliary atresia. EMA has also
granted orphan designation to odevixibat for the treatment of
Alagille syndrome, biliary atresia and primary biliary cholangitis.
Odevixibat has the potential to become the first approved drug
treatment for patients with PFIC. The Company intends to complete
regulatory filings in the EU and U.S. for odevixibat in PFIC no
later than early 2021, in anticipation of potential regulatory
approval, issuance of a rare pediatric disease priority review
voucher and launch in the second half of 2021.
About AlbireoAlbireo Pharma is a clinical-stage
biopharmaceutical company focused on the development of novel bile
acid modulators to treat rare pediatric and adult liver diseases,
and other adult liver 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. The Company expects to complete IND-enabling studies for
new preclinical candidate A3907 this year. Albireo was spun out
from AstraZeneca in 2008 and is headquartered in Boston,
Massachusetts, with its key operating subsidiary
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 StatementsThis 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 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 pivotal trial for odevixibat in biliary
atresia (BOLD), and the planned pivotal trial for odevixibat in
Alagille syndrome; 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
long-term open-label extension study for odevixibat in PFIC, the
pivotal trial for odevixibat in biliary atresia, the planned
pivotal trial for odevixibat in Alagille syndrome; 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 effects of odevixibat of
the treatment of PFIC patients and its potential to improve the
current standard of care; the potential benefits of an orphan drug
designation; the potential issuance of a rare pediatric disease
priority review voucher; 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 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 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 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:Colleen Alabiso,
857-356-3905, colleen.alabiso@albireopharma.comLisa Rivero,
617-947-0899, lisa.rivero@syneoshealth.com
Albireo Pharma, Inc.
Condensed Consolidated Balance
Sheets
(in thousands, except share
data)
(unaudited)
|
September 30, |
|
December
31, |
|
2020 |
|
2019 |
Assets |
|
|
|
|
|
Current
assets: |
|
|
|
|
|
Cash and cash equivalents |
$ |
278,691 |
|
|
$ |
131,843 |
|
Prepaid expenses and other current assets |
|
8,199 |
|
|
|
9,956 |
|
Total current assets |
|
286,890 |
|
|
|
141,799 |
|
Property and
equipment, net |
|
551 |
|
|
|
597 |
|
Goodwill |
|
17,260 |
|
|
|
17,260 |
|
Other
assets |
|
6,401 |
|
|
|
5,413 |
|
Total
assets |
$ |
311,102 |
|
|
$ |
165,069 |
|
Liabilities and Stockholders' Equity |
|
|
|
|
|
Current
liabilities: |
|
|
|
|
|
Accounts payable |
$ |
3,601 |
|
|
$ |
4,785 |
|
Accrued expenses |
|
16,817 |
|
|
|
13,486 |
|
Other current liabilities |
|
810 |
|
|
|
653 |
|
Total
current liabilities |
|
21,228 |
|
|
|
18,924 |
|
Liability
related to sale of future royalties |
|
64,871 |
|
|
|
48,714 |
|
Note
payable, net of discount |
|
9,508 |
|
|
|
— |
|
Other
long-term liabilities |
|
3,735 |
|
|
|
4,270 |
|
Total
liabilities |
|
99,342 |
|
|
|
71,908 |
|
Stockholders’ Equity: |
|
|
|
|
|
Common stock, $0.01 par value per share — 30,000,000 authorized at
September 30, 2020 and December 31, 2019;
19,073,498 and 12,749,443 issued and outstanding at
September 30, 2020 and December 31, 2019,
respectively |
|
190 |
|
|
|
127 |
|
Additional paid-in capital |
|
451,448 |
|
|
|
245,769 |
|
Accumulated other comprehensive income |
|
2,143 |
|
|
|
6,452 |
|
Accumulated deficit |
|
(242,021 |
) |
|
|
(159,187 |
) |
Total stockholders’ equity |
|
211,760 |
|
|
|
93,161 |
|
Total
liabilities and stockholders’ equity |
$ |
311,102 |
|
|
$ |
165,069 |
|
|
|
|
|
|
|
Albireo Pharma, Inc.
Condensed Consolidated Statements of
Operations
(in thousands, except share and per share
data)
(unaudited)
|
Three Months Ended September 30, |
|
Nine Months Ended September 30, |
|
2020 |
|
2019 |
|
2020 |
|
2019 |
Revenue |
$ |
2,131 |
|
|
$ |
1,385 |
|
|
$ |
5,592 |
|
|
$ |
3,205 |
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
22,200 |
|
|
|
11,996 |
|
|
|
56,727 |
|
|
|
31,359 |
|
General and administrative |
|
11,663 |
|
|
|
6,010 |
|
|
|
28,290 |
|
|
|
16,788 |
|
Other operating (income) expense, net |
|
(4,628 |
) |
|
|
4,015 |
|
|
|
(4,556 |
) |
|
|
6,319 |
|
Total operating expenses |
|
29,235 |
|
|
|
22,021 |
|
|
|
80,461 |
|
|
|
54,466 |
|
Operating
loss |
|
(27,104 |
) |
|
|
(20,636 |
) |
|
|
(74,869 |
) |
|
|
(51,261 |
) |
Interest
expense, net |
|
(3,639 |
) |
|
|
(1,274 |
) |
|
|
(7,965 |
) |
|
|
(3,934 |
) |
Net
loss |
$ |
(30,743 |
) |
|
$ |
(21,910 |
) |
|
$ |
(82,834 |
) |
|
$ |
(55,195 |
) |
Net loss per common share - basic and diluted |
$ |
(1.96 |
) |
|
$ |
(1.73 |
) |
|
$ |
(5.54 |
) |
|
$ |
(4.47 |
) |
Weighted-average common shares used to compute basic and diluted
net loss per common share |
|
15,704,293 |
|
|
|
12,685,000 |
|
|
|
14,942,213 |
|
|
|
12,349,870 |
|
|
|
|
|
|
|
|
|
|
|
|
|
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