Albireo Pharma, Inc. (Nasdaq: ALBO), a 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, 2021.
“With Bylvay now available, the positive responses from
prescribers, patients and payors have translated to early sales,”
said Ron Cooper, President and Chief Executive Officer of Albireo.
“Launching Bylvay successfully and delivering on expectations is
our focus while advancing our Phase 3 studies for additional
indications in Alagille syndrome and biliary atresia. Beyond
Bylvay, our two next generation bile acid modulators, A3907 and
A2342, are getting closer to value generating inflection
points.”
Recent and Upcoming Highlights
Bylvay (odevixibat)
- Bylvay was approved by the U.S. Food & Drug
Administration (FDA) on July 20, 2021 as the
first drug for the treatment of pruritus in all types
of progressive familial intrahepatic cholestasis (PFIC).
Bylvay was available days after approval and the U.S. commercial
launch is underway with prescriptions and sales being
generated.
- In Europe, Bylvay was authorized for the treatment of PFIC by
the European Commission (EC) and UK Medicines and Healthcare
Products Regulatory Agency (MHRA). Bylvay is currently available in
Germany and is expected to become available for sale in additional
European countries following country price listings and
reimbursement approvals.
- Albireo completed an exclusive distribution and supply
agreement with Swixx BioPharma AG in Central and Eastern Europe.
This follows agreements with Medison Pharma, Ltd. in Israel, GEN
İlaç in Turkey, Genpharm Services in Saudi Arabia and other Gulf
countries and Jadeite Medicines Inc. in Japan, all of which are
expected to drive availability of Bylvay in key markets
globally.
- The Company had five abstracts from the PEDFIC 1 and PEDFIC 2
studies of Bylvay accepted at the American Association for the
Study of Liver Diseases (AASLD) The Liver Meeting® to be held
November 12 – 15, 2021. The data being presented show evidence of
long-term safety and efficacy; evidence of disease modification;
improvements in hepatic health, growth and sleep that reduce the
disease burden as well as improvement in serum bile acids and
improvement in pruritus symptoms for all types of PFIC. These data
further contribute to the largest body of prospective data
collected in patients with PFIC.
- Five abstracts were accepted at the 2021 North American Society
for Pediatric Gastroenterology, Hepatology & Nutrition
(NASPGHAN) Annual Meeting to be held December 12 – 18, 2021.
Highlighted PFIC data from the Phase 3 PEDFIC 1 and PEDFIC 2
studies of Bylvay showed correlations in serum bile acid reductions
and long-term improvements in pruritus and sleep; treatment with
Bylvay had considerable impact on quality of life of patients and
their caregivers as well as efficacy and safety of Bylvay in PFIC
patients with prior partial external biliary diversion. There will
also be a poster presentation on the ASSERT study design for
Alagille syndrome.
- The Company continues to enroll and dose patients in the Phase
3 ASSERT Study, a pivotal trial of Bylvay in patients with Alagille
syndrome and remains on track to report topline data in 2022.
- The Phase 3 BOLD Study, which is the first and only pivotal
trial of an ileal bile acid transporter (IBAT) inhibitor in biliary
atresia, remains on track to report topline data in 2024. Biliary
atresia is the largest pediatric cholestatic liver disease.
- The Bylvay Phase 2 study in Alagille
syndrome, biliary atresia and PFIC patients was published in the
Clinics and Research in Hepatology and Gastroenterology October
issue. Study results from the open-label, multi-center study in
pediatric patients with cholestatic liver disease showed Bylvay was
well tolerated, reduced serum bile acids and improved pruritus and
sleep. Data also showed reduced pruritus correlated significantly
with reduced serum bile acids. No serious adverse events were
treatment related; most adverse events were transient. No diarrhea
was reported during the treatment period.
Pipeline: Next Generation Bile Acid
Modulators
- Clinical development continues for A3907, the first oral
systemic apical sodium-dependent bile acid transporter (ASBT)
inhibitor being developed for adult cholestatic liver diseases. A
Phase 1, first-in-human, double-blind, single and multiple
ascending dose study in healthy adult subjects to investigate the
safety, tolerability, pharmacokinetics and pharmacodynamics of an
A3907 oral formulation is underway with data readout expected by
end of year.
- Two posters on A2342 will be presented at AASLD The Liver
Meeting®, with data showing inhibition of hepatitis B viral entry
and replication in preclinical models as well as effects in serum
bile acids in organic anion transporting polypeptide (OATP) KO mice
and in cynomolgus monkeys, a species expressing human-like OATPs,
indicating target engagement. IND-enabling studies for A2342 are
currently being completed for the first potent oral systemic
sodium-taurocholate co-transporting peptide (NTCP) inhibitor for
development in viral and cholestatic liver diseases, demonstrating
Albireo’s leading position in the science of bile acid
modulation.
- A2342 composition of matter patent allowed in the U.S., with
expiration in 2040 upon grant.
Corporate
- In October, the Company entered into an exclusive licensing
agreement with Jadeite Medicines, Inc. for the development and
commercialization of Bylvay in Japan for PFIC, ALGS and biliary
atresia. Under the terms of the agreement, Jadeite Medicines, a
biopharmaceutical platform company focused on developing innovative
pharmaceutical products, will be responsible for clinical
development, regulatory approval and commercialization of Bylvay in
Japan, where there is significant market opportunity. In addition
to an upfront payment of $15 million, Albireo is entitled to
receive up to $120 million in milestone payments and double-digit
royalties.
- On September 7, the Company entered into a definitive agreement
to sell the rare pediatric disease Priority Review Voucher (PRV).
The PRV was granted to Albireo in July 2021 under an FDA provision
that encourages the development of treatments for rare pediatric
diseases. The non-dilutive capital from the sale further
strengthens the Company’s financial position.
- The Company presented a corporate overview at several investor
conferences during the quarter, including the 2021 Wedbush PacGrow
Healthcare Virtual Conference, the 2021 Virtual Wells Fargo
Healthcare Conference, the H.C. Wainwright 23rd Annual Global
Investment Conference and the Baird 2021 Global Healthcare
Conference.
Third Quarter 2021 Financial Results
- Bylvay net revenue was $1.1 million for the third quarter of
2021. Bylvay was approved during this quarter, therefore revenue is
from a partial quarter and there was no product revenue for the
third quarter of 2020.
- Royalty revenue was $2.6 million for the third quarter of 2021,
compared with $2.1 million for the third quarter of 2020, an
increase of $0.5 million. The increase relates to estimated royalty
revenue to be received from EA Pharma for elobixibat for the
treatment of chronic constipation which is passed on to HealthCare
Royalty partners.
- Cost of product revenue was $0.4 million for the third quarter
of 2021. Following Bylvay approval certain manufacturing and
quality headcount costs are now included in cost of product
revenue. There were no material costs, as materials related to
current product sold was expensed prior to approval. Bylvay was
approved during this quarter, therefore there was no cost of
product revenue for the third quarter of 2020.
- R&D expenses were $21.1 million for the third quarter of
2021 compared with $22.2 million for the third quarter of 2020, a
decrease of $1.1 million. The decrease in R&D expenses for the
2021 period were principally due to the completion of the PEDFIC1
study and the completion of the elobixibat Phase 2 trial in NASH in
the third quarter 2020 offset by increases in expenses related to
the Bylvay Phase 3 studies and early assets.
- Selling, general and administrative expenses were $17.6 million
for the third quarter of 2021 compared with $11.7 million for the
third quarter of 2020, an increase of $5.9 million. The increase is
attributable to personnel and related expenses as we continue to
increase our headcount, and commercial expenses to launch
Bylvay.
- Net income for the third quarter of 2021 was $57.1 million
compared to a net loss of $30.7 million for the third quarter of
2020 driven by the onetime sale of the PRV sales. Earnings per
share for the third quarter of 2021 were $2.90 on a fully diluted
basis, compared to ($1.96) for the third quarter of 2020.
- The Company had cash and cash equivalents of $262.6 million as
of September 30, 2021, compared to $186.3 million at June 30, 2021.
The 2021 operating cash burn guidance has been $130-$135 million
and it’s expected to be closer to $130 million. During the third
quarter of 2021, an additional $103.4 million of net proceeds were
received from the completed sale of the Priority Review Voucher. An
additional $15 million cash upfront fee from the recently announced
Japan licensing agreement due to be received in the fourth quarter
of 2021. As a result, cash and cash equivalents are anticipated to
be sufficient to fully fund the launches of Bylvay and the next
stages of the early asset portfolio. 2021 revenue from Bylvay is
anticipated to be $3-4 million.
November 4 Conference CallTo access the live
conference call by phone, please dial 877-407-0792 (domestic) or
201-689-8263 (international) and provide the access code 13723976.
Live audio webcast will be accessible from the Media &
Investors page of Albireo’s website. 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.
November 16 Post-AASLD Conference Call Albireo
will host a post-AASLD conference call and live audio webcast on
November 16 at 10:00am EST. Presenters will include Ron Cooper,
President and Chief Executive Officer; Patrick Horn, M.D., Ph.D.,
Chief Medical Officer and Jan Mattsson, Ph.D., Chief Scientific
Officer and Co-Founder. To access the live conference call by
phone, please dial 877-407-0792 (domestic) or 201-689-8263
(international) and provide the access code 13724001.The virtual
event will also be accessible from the Albireo Media and Investors
page. 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 Bylvay
(odevixibat)Bylvay is the first drug approved in
the U.S. for the treatment of pruritus in patients 3 months of age
and older in all types of progressive familial intrahepatic
cholestasis (PFIC). The European Commission (EC) and UK Medicines
and Healthcare Products Regulatory Agency (MHRA) have also granted
marketing authorization of Bylvay for the treatment of PFIC in
patients aged 6 months or older. Bylvay is available for sale in
Germany and will be available for sale in other European countries
following pricing and reimbursement approval. A potent, once-daily,
non-systemic ileal bile acid transport inhibitor, Bylvay acts
locally in the small intestine. Bylvay 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 medicine can only be obtained with a prescription
and treatment should be started and supervised by a doctor who has
experience in the management of PFIC. For more information about
using Bylvay, see the package leaflet or contact your doctor or
pharmacist. For full prescribing information, visit
www.bylvay.com.
In the U.S. and Europe, Bylvay has orphan exclusivity for its
approved PFIC indications, and orphan designations for the
treatment of Alagille syndrome, biliary atresia and primary biliary
cholangitis. Bylvay is being evaluated in the ongoing PEDFIC 2
open-label trial in patients with PFIC, in the BOLD Phase 3 study
for patients with biliary atresia and the ASSERT Phase 3 study for
Alagille syndrome.
About AlbireoAlbireo Pharma is a rare disease
company focused on the development of novel bile acid modulators to
treat rare pediatric and adult liver diseases. Albireo’s lead
product, Bylvay, was approved by the U.S. FDA as the first drug for
the treatment of pruritus in all types of progressive familial
intrahepatic cholestasis (PFIC), and it is also being developed to
treat other rare pediatric cholestatic liver diseases with Phase 3
trials in Alagille syndrome and biliary atresia, as well as an
Open-label Extension (OLE) study for PFIC. In Europe, Bylvay has
been approved for the treatment of PFIC and has been submitted for
pricing and reimbursement approval. The Company is progressing a
Phase 1 clinical trial for A3907 to advance development in adult
cholestatic liver disease, with IND-enabling studies moving ahead
with A2342 for viral and cholestatic liver disease. 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 2019
and 2020 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: Albireo’s commercialization
plans and expectations for commercializing Bylvay in the U.S. and
Europe; the plans for, or progress, scope, cost, initiation,
duration, enrollment, results or timing for availability of results
of, development of Bylvay, A3907, A2342 or any other Albireo
product candidate or program; the pivotal trial for Bylvay in
biliary atresia (BOLD); the pivotal trial for Bylvay in Alagille
syndrome (ASSERT); the Phase 1 trial for A3907; the IND-enabling
studies for A2342; 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 Bylvay in PFIC, the BOLD
and ASSERT trials, Phase 1 trial for A3907 and the IND-enabling
studies for A2342; expectations that biliary atresia is one of the
most common rare pediatric liver diseases and is the leading cause
of liver transplants in children; potential regulatory approval by
and discussions with the FDA or EMA regarding our programs;
expectations that the Company’s distribution and supply agreements
will drive availability of Bylvay in key markets globally, and
potential revenue that may be generated by such agreements;
potential regulatory approval and plans for potential
commercialization of Bylvay in countries outside of the U.S. and
Europe, including Japan; the potential benefits or competitive
position of Bylvay or any other Albireo product candidate or
program or the commercial opportunity in any target indication; the
potential effects of Bylvay 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 length of
time for which Albireo’s cash resources are expected to be
sufficient, and the milestones and activities to be funded with
those cash resources; 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,” or the negative of these terms or other
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: there are no guarantees that Bylvay will be
commercially successful; we may encounter issues, delays or other
challenges in launching or commercializing Bylvay; whether Bylvay
receives adequate reimbursement from third-party payors; the degree
to which Bylvay receives acceptance from patients and physicians
for its approved indication; challenges associated with execution
of our sales activities, which in each case could limit the
potential of our product; challenges associated with supply and
distribution activities, which in each case could limit our sales
and the availability of our product; results achieved in Bylvay in
the treatment of patients with PFIC may be different than observed
in clinical trials, and may vary among patients; other potential
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 Bylvay to date, including findings in
indications other than PFIC, will be predictive of results from
other clinical trials of Bylvay; there is no guarantee that Bylvay
will be approved in jurisdictions or for indications beyond the
jurisdictions in which or indications for which Bylyay is currently
approved; there is no guarantee that our other products candidates
will be approved; estimates of the addressable patient population
for target indications may prove to be incorrect; 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 Bylvay, including BOLD and ASSERT, and the
Phase 1 clinical trial of A3907, and the outcomes of such trials;
Albireo’s ability to obtain coverage, pricing or reimbursement for
approved products in the United States or Europe; delays or other
challenges in the recruitment of patients for, or the conduct of,
the Company’s clinical trials; and the Company’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.
Media Contact: Colleen
Alabiso, 857-356-3905, colleen.alabiso@albireopharma.comLauren
Sneider, 857-300-1737, lauren.sneider@albireopharma.com
Investor Contact: Hans Vitzthum, LifeSci
Advisors, LLC., 617-430-7578
Albireo Pharma,
Inc.Condensed Consolidated Balance
Sheets(in thousands, except share and per share
data)(unaudited)
|
|
|
|
|
|
|
|
|
September 30, |
|
December 31, |
|
|
2021 |
|
|
2020 |
|
Assets |
|
|
|
|
|
|
Current assets: |
|
|
|
|
|
|
Cash and cash equivalents |
|
$ |
262,612 |
|
|
$ |
251,272 |
|
Accounts receivable, net |
|
|
1,307 |
|
|
|
— |
|
Inventory |
|
|
196 |
|
|
|
— |
|
Prepaid expenses and other current assets |
|
|
7,779 |
|
|
|
10,593 |
|
Total current assets |
|
|
271,894 |
|
|
|
261,865 |
|
Property and equipment,
net |
|
|
775 |
|
|
|
478 |
|
Goodwill |
|
|
17,260 |
|
|
|
17,260 |
|
Other assets |
|
|
6,516 |
|
|
|
6,004 |
|
Total assets |
|
$ |
296,445 |
|
|
$ |
285,607 |
|
Liabilities and
Stockholders' Equity |
|
|
|
|
|
|
Current liabilities: |
|
|
|
|
|
|
Accounts payable |
|
$ |
7,222 |
|
|
$ |
5,283 |
|
Accrued expenses |
|
|
20,884 |
|
|
|
19,051 |
|
Other current liabilities |
|
|
5,043 |
|
|
|
948 |
|
Total current liabilities |
|
|
33,149 |
|
|
|
25,282 |
|
Liability related to sale of
future royalties |
|
|
68,513 |
|
|
|
65,894 |
|
Note payable, net of
discount |
|
|
9,929 |
|
|
|
9,621 |
|
Other long-term
liabilities |
|
|
3,138 |
|
|
|
3,579 |
|
Total liabilities |
|
|
114,729 |
|
|
|
104,376 |
|
Stockholders’ Equity: |
|
|
|
|
|
|
Preferred stock, $0.01 par value per share — 50,000,000 shares
authorized at September 30, 2021 and
December 31, 2020; 0 and 0 shares issued and outstanding
at September 30, 2021 and December 31, 2020,
respectively |
|
|
— |
|
|
|
— |
|
Common stock, $0.01 par value per share — 60,000,000 and 30,000,000
shares authorized at September 30, 2021 and
December 31, 2020, respectively; 19,283,269 and
19,275,509 shares issued and outstanding at
September 30, 2021, respectively, and 19,107,040 shares
issued and outstanding at December 31, 2020 |
|
|
193 |
|
|
|
191 |
|
Additional paid-in capital |
|
|
472,363 |
|
|
|
456,472 |
|
Accumulated other comprehensive loss |
|
|
(722 |
) |
|
|
(8,612 |
) |
Accumulated deficit |
|
|
(289,888 |
) |
|
|
(266,820 |
) |
Treasury stock at cost, 7,760 shares and 0 shares at September 30,
2021 and December 31, 2020, respectively |
|
|
(230 |
) |
|
|
— |
|
Total stockholders’ equity |
|
|
181,716 |
|
|
|
181,231 |
|
Total liabilities and
stockholders’ equity |
|
$ |
296,445 |
|
|
$ |
285,607 |
|
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, |
|
|
|
2021 |
|
|
2020 |
|
|
2021 |
|
|
2020 |
|
|
Revenue: |
|
|
|
|
|
|
|
|
|
|
|
|
|
Product revenue, net |
|
$ |
1,060 |
|
|
$ |
— |
|
|
$ |
1,060 |
|
|
$ |
— |
|
|
Royalty revenue |
|
|
2,604 |
|
|
|
2,131 |
|
|
|
6,998 |
|
|
|
5,592 |
|
|
Total revenue |
|
|
3,664 |
|
|
|
2,131 |
|
|
|
8,058 |
|
|
|
5,592 |
|
|
Cost of product revenue |
|
|
431 |
|
|
|
— |
|
|
|
431 |
|
|
|
— |
|
|
Gross profit |
|
|
3,233 |
|
|
|
2,131 |
|
|
|
7,627 |
|
|
|
5,592 |
|
|
Operating
expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
|
21,083 |
|
|
|
22,200 |
|
|
|
61,920 |
|
|
|
56,727 |
|
|
Selling, general and administrative |
|
|
17,612 |
|
|
|
11,663 |
|
|
|
49,825 |
|
|
|
28,290 |
|
|
Other operating expense (income), net |
|
|
3,719 |
|
|
|
(4,628 |
) |
|
|
7,873 |
|
|
|
(4,556 |
) |
|
Total operating expenses |
|
|
42,414 |
|
|
|
29,235 |
|
|
|
119,618 |
|
|
|
80,461 |
|
|
Operating loss |
|
|
(39,181 |
) |
|
|
(27,104 |
) |
|
|
(111,991 |
) |
|
|
(74,869 |
) |
|
Other income
(loss): |
|
|
|
|
|
|
|
|
|
|
|
|
|
Gain from sale of priority
review voucher, net of transaction costs |
|
|
103,387 |
|
|
|
— |
|
|
|
103,387 |
|
|
|
— |
|
|
Interest expense, net |
|
|
(3,331 |
) |
|
|
(3,639 |
) |
|
|
(10,675 |
) |
|
|
(7,965 |
) |
|
Net income (loss) before
income taxes |
|
|
60,875 |
|
|
|
(30,743 |
) |
|
|
(19,279 |
) |
|
|
(82,834 |
) |
|
Provision for income
taxes |
|
|
3,789 |
|
|
|
— |
|
|
|
3,789 |
|
|
|
— |
|
|
Net income (loss) |
|
$ |
57,086 |
|
|
$ |
(30,743 |
) |
|
$ |
(23,068 |
) |
|
$ |
(82,834 |
) |
|
Net income (loss) per share
attributable to holders of common stock: |
|
|
|
|
|
|
|
|
|
|
|
|
|
Basic |
|
$ |
2.96 |
|
|
$ |
(1.96 |
) |
|
$ |
(1.20 |
) |
|
$ |
(5.54 |
) |
|
Diluted |
|
$ |
2.90 |
|
|
$ |
(1.96 |
) |
|
$ |
(1.20 |
) |
|
$ |
(5.54 |
) |
|
Weighted-average common shares outstanding: |
|
|
|
|
|
|
|
|
|
|
|
|
|
Basic |
|
|
19,258,905 |
|
|
|
15,704,293 |
|
|
|
19,197,536 |
|
|
|
14,942,213 |
|
|
Diluted |
|
|
19,651,243 |
|
|
|
15,704,293 |
|
|
|
19,197,536 |
|
|
|
14,942,213 |
|
|
Albireo Pharma (NASDAQ:ALBO)
Historical Stock Chart
Von Mär 2024 bis Apr 2024
Albireo Pharma (NASDAQ:ALBO)
Historical Stock Chart
Von Apr 2023 bis Apr 2024