Albireo Pharma, Inc. (Nasdaq: ALBO), a clinical-stage orphan
pediatric liver disease company developing novel bile acid
modulators, today announced financial results and corporate
highlights for the second quarter ended June 30, 2019.
“The momentum is building for Albireo, as we advance programs on
multiple fronts and ramp up commercial preparations,” said Ron
Cooper, President and Chief Executive Officer of Albireo. “While
the PEDFIC program moves toward full enrollment, and we plan for
potential approval and launch in 2021, we continue to strengthen
the body of evidence supporting the potential of odevixibat, and we
are preparing to initiate a pivotal study in biliary atresia. At
the same time, we are very excited to have initiated an elobixibat
Phase 2 trial in NAFLD/NASH, and we believe elobixibat has the
potential to find a significant place in the emerging NASH
treatment landscape.”
Recent Highlights
Odevixibat (A4250)
- Achieved full trial site activation in the PEDFIC 1 Phase 3
study in progressive familial intrahepatic cholestasis (PFIC), with
44 active sites globally, and enrollment progressing steadily.
Patients in screening or entering screening are potentially
sufficient to complete the trial based on the current screening
success rate. PEDFIC 1 is enrolling both PFIC1 and PFIC2 patients
both with and without diversion surgery. The trial is studying both
high- and low-dose odevixibat using the planned commercial
formulation. Patients randomized to odevixibat are treated with
once-daily oral capsules or sprinkles, which do not require
refrigeration. Albireo expects topline results in mid-2020, and
continues to project potential first regulatory approval and launch
in 2021.
- Activated first site and prepared for patient enrollment in
expanded second cohort in PEDFIC 2, the long-term, open-label
extension study of PEDFIC 1. Cohort 2 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.
- Presented data on odevixibat and rare cholestatic liver disease
that affirm the potential of Albireo’s development programs at the
52nd European Society for Paediatric Gastroenterology, Hepatology
and Nutrition (ESPGHAN) Annual Meeting in Glasgow, Scotland.
Results from the Phase 2 study in pediatric cholestasis were
presented on the effect of odevixibat on reducing serum bile acids,
and improving pruritus and sleep measures in a subset of patients
with Alagille syndrome, as well as the effect of odevixibat on
reducing serum bile acids and improving pruritus in a small subset
of patients with biliary atresia. A case study compared the effect
of odevixibat on serum bile acids and pruritus in a patient who
subsequently underwent biliary diversion surgery. Data on the
development of the proprietary patient- and observer-reported
outcome (PRO and ObsRO) tools used in the PEDFIC program also were
presented.
- Launched PFIC Voices, the first-ever PFIC disease awareness
initiative, in collaboration with parents, patients, the patient
advocacy community and physicians (pficvoices.com). PFIC Voices is
a global initiative to build awareness and understanding of PFIC by
sharing perspectives about the impact of PFIC, and the critical
need for education, treatment and support.
- Prepared to begin an odevixibat pivotal trial in biliary
atresia, estimated to be one of the most common rare pediatric
liver diseases. Productive discussions are underway with
regulators, and the Company plans to initiate the trial in
2020.
Elobixibat
Initiated a Phase 2 multicenter, placebo-controlled clinical
trial of elobixibat in non-alcoholic fatty liver disease
(NAFLD)/nonalcoholic steatohepatitis (NASH) designed to enroll 46
patients with biopsy-confirmed NASH, or a diagnosis of suspected
NAFLD or NASH based on metabolic syndrome definitions. The Company
expects to report topline data in mid-2020.
Corporate
- Presented at several conferences, including: the Jefferies 2019
Global Healthcare Conference in New York and the William Blair 39th
Annual Growth Stock Conference in Chicago.
Second-Quarter Financial Highlights
- Revenues were $1.3 million in the second quarter of 2019,
compared to $0.7 million in the second quarter of 2018.
- R&D expense was $11.0 million for the second quarter of
2019, up 72% from $6.4 million in the second quarter of
2018.
- G&A expense was $5.5 million for the second quarter of
2019, up 29%, compared to $4.2 million in the second quarter of
2018.
- Net Loss in the second quarter of 2019 was $16.6 million, or
$(1.35) per share, compared to $14.6 million, or $(1.22) per share
in the second quarter of 2018.
- The Company had cash and cash equivalents at June 30, 2019, of
$157.7 million.
Financial GuidanceFor the full year 2019, the
Company anticipates total expenses, including R&D and G&A
expenses, to be in the range of $75-$80 million. Albireo
anticipates its current cash balance to be sufficient to meet its
operating needs into 2021.
Conference CallAs previously announced, Albireo
will host a conference call and webcast today, August 8, 2019,
at 10 a.m. EDT. To access the live conference call by phone, dial
877-407-0792 (domestic) or 201-689-8263 (international), and
provide the access code 13691544. 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 through its
operating subsidiary 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 (A4250), 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. 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, 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; the size of
the PFIC population, the biliary atresia population, the NASH
population or any other disease population for indications that may
be targeted by Albireo; 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 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
Source: Albireo Pharma, Inc.
Albireo
Pharma, Inc. |
Condensed
Consolidated Balance Sheets |
(in
thousands, except share and per share data) |
(unaudited) |
|
|
|
|
|
|
|
|
|
June 30, |
|
December 31, |
|
|
2019 |
|
2018 |
Assets |
|
|
|
|
|
|
Current assets: |
|
|
|
|
|
|
Cash and cash equivalents |
|
$ |
157,722 |
|
|
$ |
163,885 |
|
Prepaid expenses and other current assets |
|
|
5,310 |
|
|
|
3,765 |
|
Total current assets |
|
|
163,032 |
|
|
|
167,650 |
|
Property and equipment,
net |
|
|
544 |
|
|
|
187 |
|
Goodwill |
|
|
17,260 |
|
|
|
17,260 |
|
Other assets |
|
|
1,115 |
|
|
|
369 |
|
Total assets |
|
$ |
181,951 |
|
|
$ |
185,466 |
|
Liabilities and
Stockholders' Equity |
|
|
|
|
|
|
Current liabilities: |
|
|
|
|
|
|
Accounts payables |
|
$ |
3,837 |
|
|
$ |
4,352 |
|
Accrued expenses |
|
|
7,555 |
|
|
|
8,165 |
|
Other current liabilities |
|
|
393 |
|
|
|
308 |
|
Total current liabilities |
|
|
11,785 |
|
|
|
12,825 |
|
Liability related to sale of
future royalties |
|
|
52,224 |
|
|
|
49,969 |
|
Other long-term
liabilities |
|
|
255 |
|
|
|
35 |
|
Total liabilities |
|
|
64,264 |
|
|
|
62,829 |
|
Stockholders’ Equity: |
|
|
|
|
|
|
Common stock, $0.01 par value per share — 30,000,000 authorized at
June 30, 2019 and December 31, 2018; 12,685,326
and 11,969,928 issued and outstanding at June 30, 2019
and December 31, 2018 |
|
|
126 |
|
|
|
120 |
|
Additional paid in capital |
|
|
240,734 |
|
|
|
214,694 |
|
Accumulated other comprehensive income |
|
|
6,582 |
|
|
|
4,293 |
|
Accumulated deficit |
|
|
(129,755 |
) |
|
|
(96,470 |
) |
Total stockholders’ equity |
|
|
117,687 |
|
|
|
122,637 |
|
Total liabilities and
stockholders’ equity |
|
$ |
181,951 |
|
|
$ |
185,466 |
|
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, |
|
|
2019 |
|
2018 |
|
2019 |
|
2018 |
Revenue |
|
$ |
1,250 |
|
|
$ |
730 |
|
|
$ |
1,820 |
|
|
$ |
11,932 |
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
|
11,034 |
|
|
|
6,411 |
|
|
|
19,363 |
|
|
|
12,562 |
|
General and administrative |
|
|
5,485 |
|
|
|
4,238 |
|
|
|
10,778 |
|
|
|
8,366 |
|
Other operating expense, net |
|
|
8 |
|
|
|
487 |
|
|
|
2,304 |
|
|
|
1,991 |
|
Total operating expenses |
|
|
16,527 |
|
|
|
11,136 |
|
|
|
32,445 |
|
|
|
22,919 |
|
Operating loss |
|
|
(15,277 |
) |
|
|
(10,406 |
) |
|
|
(30,625 |
) |
|
|
(10,987 |
) |
Interest expense, net |
|
|
(1,351 |
) |
|
|
(1,666 |
) |
|
|
(2,660 |
) |
|
|
(2,682 |
) |
Non-operating expense, net |
|
|
— |
|
|
|
(2,531 |
) |
|
|
— |
|
|
|
(2,553 |
) |
Net loss |
|
$ |
(16,628 |
) |
|
$ |
(14,603 |
) |
|
$ |
(33,285 |
) |
|
$ |
(16,222 |
) |
Net loss per share attributable to holders of common
stock: |
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per common share - basic and diluted |
|
$ |
(1.35 |
) |
|
$ |
(1.22 |
) |
|
$ |
(2.73 |
) |
|
$ |
(1.42 |
) |
Weighted-average common shares used to compute basic and
diluted net loss per common share |
|
|
12,355,969 |
|
|
|
11,938,357 |
|
|
|
12,178,376 |
|
|
|
11,417,463 |
|
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