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 third quarter ended September 30, 2019.
“Randomization of patients in our Phase 3 trial of odevixibat in
PFIC is nearing completion, and we remain on track to report
topline results in mid-2020,” said Ron Cooper, President and Chief
Executive Officer of Albireo. “Odevixibat is being developed for a
range of cholestatic liver diseases, and Albireo is finalizing
discussions with regulators for a pivotal program in biliary
atresia, one of the most common rare pediatric liver diseases. We
expect to initiate the world’s first pivotal trial in this
indication during 2020.”
Recent Highlights
Odevixibat
- Over three-quarters of the patients needed to complete the
PEDFIC 1 Phase 3 study in progressive familial intrahepatic
cholestasis (PFIC) had been randomized as of end of October 2019.
Additionally, the patients in screening or entering screening
should be sufficient to meet the trial’s 60-patient target based on
the screening success rate to date. PEDFIC 1 is enrolling both PFIC
type 1 and type 2 patients that are 6 months to 18 years of age.
The trial is studying both high- and low-dose odevixibat using the
company’s planned commercial formulation. Patients randomized to
odevixibat are treated with once-daily oral capsules or sprinkles,
which do not require refrigeration. Albireo expects to report
topline results in mid-2020, and continues to project a potential
first regulatory approval and launch in 2021.
- Enrolled the first patients in the second cohort in PEDFIC 2,
the long-term, open-label extension study of PEDFIC 1. The expanded
cohort is expected to broaden the evidence base for odevixibat by
including PFIC patients who do not meet eligibility criteria for
PEDFIC 1, but have elevated serum bile acid levels and pruritus.
This includes patients with all types of PFIC, and patients younger
than 6 months or older than 18 years of age. Cohort 1 of PEDFIC 2
is composed of patients who have completed, and rolled over from,
the PEDFIC 1 study. We now have patients who are approaching a year
on odevixibat treatment.
- Presented data on odevixibat at the North American Society for
Pediatric Gastroenterology, Hepatology and Nutrition Annual Meeting
in Chicago. The presentations included data regarding the clinical
pharmacology of odevixibat, as well as information about the
development of the proprietary patient- and observer-reported
outcome (PRO and ObsRO) tools used in the PEDFIC
program.
- Completing dialogue with the U.S. Food and Drug Administration
(FDA) regarding key parameters for a planned pivotal clinical trial
of odevixibat in biliary atresia, one of the most common rare
pediatric liver diseases. Albireo plans to initiate this trial in
2020.
Elobixibat
- Randomized the first patients in the company’s Phase 2
multicenter, placebo-controlled clinical trial of elobixibat in
non-alcoholic fatty liver disease (NAFLD)/nonalcoholic
steatohepatitis (NASH). This trial, which has all 10 initial
clinical trial sites active, is designed to randomize 46 patients
with biopsy-confirmed NASH, or a diagnosis of suspected NAFLD or
NASH based on metabolic syndrome definitions.
Corporate
- Strengthened the company’s leadership team with the appointment
of Michelle Graham as Chief Human Resources Officer.
Third-Quarter Financial Results
- Revenues were $1.4 million for the third quarter of 2019,
compared to $0.2 million for the third quarter of 2018. The
year-over-year change was primarily the result of estimated royalty
revenue received from EA Pharma for elobixibat for the treatment of
chronic constipation.
- R&D expense was $12.0 million for the third quarter of
2019, compared to $9.7 million for the third quarter of 2018. The
year-over-year change was primarily the result of personnel, and
program expenses as we continue to increase our headcount, and
program activities, respectively.
- G&A expense was $6.0 million for the third quarter of 2019,
compared to $3.9 million for the third 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 third quarter of 2019 was $21.9 million, or
$(1.73) per share, compared to $14.0 million, or $(1.17) per share
for the third quarter of 2018.
- The company had cash and cash equivalents at September 30,
2019, of $142.7 million, which compares to $163.9 million at
December 31, 2018.
Financial
GuidanceFor the full year 2019, the company anticipates
total expenses, including R&D and G&A expenses, to be in
the range of $70M-$75M. Albireo continues to anticipate that its
current cash balance will be sufficient to meet its operating needs
into 2021.
Conference CallAs previously announced, Albireo
will host a conference call and webcast today, November 6,
2019, at 10 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 13694376. 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.
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, duration or 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 trial for odevixibat in biliary atresia 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, and the outcomes of
such trials; Albireo’s completion of discussions with the FDA
regarding the planned pivotal trial for odevixibat in biliary
atresia; 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; 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
Albireo Pharma, Inc.Condensed
Consolidated Balance Sheets(in thousands, except
share and per share data)(unaudited) |
|
|
|
September 30, |
|
December 31, |
|
|
2019 |
|
|
2018 |
|
Assets |
|
|
|
|
|
|
Current
assets: |
|
|
|
|
|
|
Cash and cash equivalents |
|
$ |
142,666 |
|
|
$ |
163,885 |
|
Prepaid expenses and other current assets |
|
|
5,353 |
|
|
|
3,765 |
|
Total current assets |
|
|
148,019 |
|
|
|
167,650 |
|
Property and
equipment, net |
|
|
633 |
|
|
|
187 |
|
Goodwill |
|
|
17,260 |
|
|
|
17,260 |
|
Other
assets |
|
|
5,578 |
|
|
|
369 |
|
Total
assets |
|
$ |
171,490 |
|
|
$ |
185,466 |
|
Liabilities and Stockholders' Equity |
|
|
|
|
|
|
Current
liabilities: |
|
|
|
|
|
|
Accounts payable |
|
$ |
3,185 |
|
|
$ |
4,352 |
|
Accrued expenses |
|
|
8,459 |
|
|
|
8,165 |
|
Other current liabilities |
|
|
683 |
|
|
|
308 |
|
Total
current liabilities |
|
|
12,327 |
|
|
|
12,825 |
|
Liability
related to sale of future royalties |
|
|
53,073 |
|
|
|
49,969 |
|
Other
long-term liabilities |
|
|
4,418 |
|
|
|
35 |
|
Total
liabilities |
|
|
69,818 |
|
|
|
62,829 |
|
Stockholders’ Equity: |
|
|
|
|
|
|
Common stock, $0.01 par value per share — 30,000,000 authorized at
September 30, 2019 and December 31, 2018;
12,685,326 and 11,969,928 issued and outstanding at
September 30, 2019 and December 31, 2018 |
|
|
126 |
|
|
|
120 |
|
Additional paid in capital |
|
|
242,638 |
|
|
|
214,694 |
|
Accumulated other comprehensive income |
|
|
10,573 |
|
|
|
4,293 |
|
Accumulated deficit |
|
|
(151,665 |
) |
|
|
(96,470 |
) |
Total stockholders’ equity |
|
|
101,672 |
|
|
|
122,637 |
|
Total
liabilities and stockholders’ equity |
|
$ |
171,490 |
|
|
$ |
185,466 |
|
|
|
|
|
|
|
|
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, |
|
|
|
2019 |
|
|
2018 |
|
|
2019 |
|
|
2018 |
|
|
Revenue |
|
$ |
1,385 |
|
|
$ |
237 |
|
|
$ |
3,205 |
|
|
$ |
12,169 |
|
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
|
11,996 |
|
|
|
9,666 |
|
|
|
31,359 |
|
|
|
22,228 |
|
|
General and administrative |
|
|
6,010 |
|
|
|
3,850 |
|
|
|
16,788 |
|
|
|
12,216 |
|
|
Other operating expense (income), net |
|
|
4,015 |
|
|
|
(614 |
) |
|
|
6,319 |
|
|
|
1,377 |
|
|
Total operating expenses |
|
|
22,021 |
|
|
|
12,902 |
|
|
|
54,466 |
|
|
|
35,821 |
|
|
Operating
loss |
|
|
(20,636 |
) |
|
|
(12,665 |
) |
|
|
(51,261 |
) |
|
|
(23,652 |
) |
|
Interest
expense, net |
|
|
(1,274 |
) |
|
|
(1,367 |
) |
|
|
(3,934 |
) |
|
|
(4,049 |
) |
|
Non-operating income (expense), net |
|
|
— |
|
|
|
7 |
|
|
|
— |
|
|
|
(2,546 |
) |
|
Net
loss |
|
$ |
(21,910 |
) |
|
$ |
(14,025 |
) |
|
$ |
(55,195 |
) |
|
$ |
(30,247 |
) |
|
Net loss per common share - basic and diluted |
|
$ |
(1.73 |
) |
|
$ |
(1.17 |
) |
|
$ |
(4.47 |
) |
|
$ |
(2.60 |
) |
|
Weighted-average common shares used to compute basic and diluted
net loss per common share |
|
|
12,685,000 |
|
|
|
11,969,791 |
|
|
|
12,349,870 |
|
|
|
11,612,760 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
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