Albireo Pharma, Inc. (Nasdaq: ALBO), a clinical-stage rare
pediatric liver disease company developing novel bile acid
modulators, today provided a business update and reported financial
results for the first quarter ended March 31, 2021.
“It is estimated that there are approximately one hundred
thousand pediatric cholestatic patients in the world without an
approved drug therapy. We are focused on global commercial
readiness and pleased with the progress we have made towards
ensuring we reach families in need with the potential
first-in-class oral drug option for the treatment of PFIC
patients,” said Ron Cooper, President and Chief Executive Officer
of Albireo. “Beyond PFIC, we are rapidly advancing our Phase 3
programs in biliary atresia and Alagille syndrome while moving
A3907, a new bile acid modulator with novel MOA, into the
clinic.”
Recent and Upcoming Highlights
Bylvay (odevixibat)
- The New Drug Application (NDA) for
Bylvay is currently being reviewed by the U.S. Food and Drug
Administration (FDA) under Priority Review with a Prescription Drug
User Fee Act (PDUFA) goal date of July 20, 2021. The Company was
informed that there are no plans for an FDA advisory committee
meeting and continues to engage in ongoing discussions. Upon
approval, the Company expects issuance of a rare pediatric disease
Priority Review Voucher and commercial launch.
- In Europe, the Marketing
Authorization Application (MAA) for Bylvay is the only submission
for an ileal bile acid transport inhibitor (IBATi) granted
accelerated assessment by the EMA. Bylvay was granted Orphan
Designation as well as access to the PRIority MEdicines (PRIME)
scheme for the treatment of PFIC. The Company continues on the
accelerated assessment process and anticipates approval and launch
in the second half of 2021.
- Bylvay has been provisionally
accepted by both the FDA and EMA as the brand name for
odevixibat.
- Global commercial readiness
continues. Hiring, onboarding and training of specialized U.S. and
international teams is underway. In the U.S., sales representatives
have conducted onboarding and training activities, while also
surveying U.S. HCPs on their current management of PFIC patients in
preparation for launch. The team is actively engaging with Travere
Therapeutics to operationalize our combined efforts for the U.S.
launch. In Europe, Germany is an early launch country that has the
largest market potential, so the Company has prioritized and
completed hiring of the full in-country commercial
organization.
- Countries such as Turkey and Saudi Arabia have an increased
prevalence of PFIC, making them top 10 commercial market
opportunities. Albireo completed two ex-U.S. commercial
distributorships with GEN İlaç in Turkey, and Genpharm Services for
Saudi Arabia and other Gulf countries, which follows the agreement
with Medison Pharma, Ltd. ("Medison”) for Bylvay in Israel. Each
company is a rare disease market leader in its respective region
and these agreements advance the Company’s global strategy for the
commercialization of Bylvay.
- The BOLD Study, the first and only
pivotal Phase 3 trial of an IBAT inhibitor in biliary atresia, the
largest pediatric cholestatic liver disease, is on track with 48
global site activations, including 18 U.S. and 30 ex-U.S. global
sites.
- The Company continues to enroll and
dose patients in the ASSERT Study, a global Phase 3 pivotal trial
of Bylvay in patients with Alagille Syndrome (ALGS).
Early-Stage Pipeline
- Initiated Phase 1 study with A3907, the first oral systemic
apical sodium-dependent bile acid transporter (ASBT) inhibitor in
clinical studies, being developed for adult cholestatic liver
diseases such as primary sclerosing cholangitis (PSC) and primary
biliary cholangitis (PBC). Phase 1 study is a 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.
- The Company was issued a U.S.
composition of matter and method of use patent for novel compound
A3907. The patent covers the chemical composition of A3907 and
use in multiple adult liver and viral diseases and is one of a
series of patents that will expire between 2039 and 2040, not
including patent term extension. The Company continues to pursue
additional patents for A3907.
- Pre-clinical studies and modeling
for A2342, the first potent oral systemic sodium-taurocholate
co-transporting peptide (NTCP) inhibitor continue in viral and
cholestatic liver diseases.
Corporate
- Recently appointed Joan Connolly as
Chief Technology Officer and key member of the Albireo Enterprise
Team. In this new role, Connolly will be responsible for overseeing
drug substance and product development, clinical supply
distribution, commercial supply chain and quality. Leadership
addition strengthens the Company for commercial readiness, the
potential global Bylvay launch and the ongoing clinical development
programs.
First Quarter 2021 Financial Results
- Revenues were $2.0 million for the first quarter of 2021,
compared to $1.5 million for the first quarter of 2020. 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 $19.9 million for the first quarter of
2021, compared to $16.1 million for the first quarter of 2020. The
higher expenses were principally due to personnel expenses as we
continue to increase our headcount and program activities. The
increase in program activities were primarily related to Bylvay for
regulatory submissions in PFIC, the additional indications for
biliary atresia and Alagille syndrome, as well as A3907 and
partially offset by elobixibat and preclinical programs.
- G&A expenses were $15.3 million for the first quarter of
2021, compared to $8.2 million for the first quarter of 2020. The
increase is attributable to personnel and related expenses as the
Company continues to increase headcount and commercialization
readiness activities related to PFIC.
- Net loss for the first quarter of 2021 was $43.7 million, or
$(2.29) per share, compared to $31.5 million, or $(2.23) per share
for the first quarter of 2020.
- The Company had cash and cash equivalents at March 31, 2021, of
$217.1 million, which compares to $251.3 million at December 31,
2020. The Company has sufficient capital resources to fund the
planned launch and development programs. Cash runway into 2023 and
plans to monetize a Priority Review Voucher, if received upon
approval. The 2021 operating cash burn is anticipated to be in the
range of $130-$135 million. 2021 revenue from Bylvay is anticipated
to be in the low single digit U.S. $ millions.
Conference CallTo access the live conference
call by phone, please dial 888-599-8686 (domestic) or 323-994-2082
(international) and provide the access code 8407763. Live audio
webcast will be accessible from the Media & Investors page of
Albireo’s website 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 Bylvay (odevixibat)Bylvay is an
investigational product candidate being developed to treat rare
pediatric cholestatic liver diseases, including PFIC, biliary
atresia and ALGS. A potent, once-daily, non-systemic ileal bile
acid transport inhibitor (IBATi), Bylvay acts locally in the small
intestine. Bylvay 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 FDA has granted Priority Review
and set a PDUFA goal date of July 20, 2021. In Europe, the EMA
validated MAA. Bylvay is the only IBATi granted accelerated
assessment by the EMA.
Bylvay also been granted Orphan Designation, as well as access
to the PRIority MEdicines (PRIME) scheme for the treatment of PFIC.
The EMA’s Pediatric Committee has agreed to Albireo’s Bylvay
Pediatric Investigation Plans for PFIC and biliary atresia. In
addition to PFIC, Bylvay has Orphan Drug Designations for the
treatment of Alagille syndrome, biliary atresia and primary biliary
cholangitis. With FDA and EMA regulatory submissions complete,
Bylvay has the potential to become the first approved drug
treatment for patients with PFIC in the U.S and Europe. The Company
anticipates potential regulatory approvals, issuance of a rare
pediatric disease priority review voucher and launch in the second
half of 2021.
The MAA and NDA filings are supported by results from PEDFIC 1
and PEDFIC 2 Phase 3 studies. PEDFIC 1 was the first and largest,
global, pivotal Phase 3 study conducted in PFIC, which evaluated
the efficacy and tolerability of Bylvay in reducing pruritus and
serum bile acids in a randomized, double-blind, placebo-controlled
trial. In the PEDFIC 1 study, Bylvay met both primary endpoints and
was well tolerated with very low incidence of diarrhea/frequent
bowel movements (9.5% of Bylvay treated patients vs. 5.0% of
placebo patients).
ir.albireopharma.com/news-releases/news-release-details/albireo-phase-3-trial-meets-both-primary-endpoints-odevixibat.
PEDFIC 2 is a long-term, open-label Phase 3 extension study. The
Company also provides an Expanded Access Program (EAP) for eligible
patients with PFIC in the U.S., Europe, Canada and Australia.
Bylvay is also currently being evaluated in the BOLD Phase 3 trial
in patients with biliary atresia, and the global Phase 3 ASSERT
trial for ALGS.
About GEN
GEN is the leading specialty pharmaceutical company in Turkey
with more than 20 years of experience. GEN partners with global
pharmaceutical companies to bring innovative therapies and rare
solutions to the community. GEN works compliant with ethical and
scientific principles and strives to set the best standards for
quality, safety, and value in the manufacture and access to health
care products. With its GMP certificated production facility and
R&D center based in Ankara, GEN offers solutions around the
globe in the treatment of rare diseases and disorders. In addition
to its HQ and offices in Turkey, GEN has offices in Germany,
Russia, Kazakhstan, Uzbekistan, and Azerbaijan. For more
information please visit GEN website at https://en.genilac.com.tr/.
You can also follow GEN
on LinkedIn, Twitter, Instagram,
and Facebook.
About Genpharm ServicesGenpharm Services is a
privately held regional pharmaceutical company, focused on Rare
Diseases and Specialty therapeutics for the Middle East and North
Africa (MENA) region. It was founded in 2012 and has its regional
offices in Dubai, UAE. It is led by senior industry executives and
a dedicated team of experienced staff. It provides fast-track and
early market access, sustainable commercial solutions and strategic
advice on medical, regulatory, pricing and launch excellence. Our
current strategic partners are Novartis Gene Therapy, Orchard
Therapeutics, PTC Therapeutics, Sarepta Therapeutics, Ultragenyx
Pharmaceuticals, Biohaven Pharmaceuticals and others. Thanks to our
“patient first” approach and our Mantra “Bringing Cures to MENA
patients”, we are the first and only company to launch successfully
Gene therapy in the region. For more information visit
https://www.genpharmservices.com/ You can also follow us on
LinkedIn and Twitter.
About Albireo
Albireo Pharma is a clinical-stage biopharmaceutical company
focused on the development of novel bile acid modulators to treat
rare pediatric and adult liver diseases. Albireo’s lead product
candidate, Bylvay, is being developed to treat rare pediatric
cholestatic liver diseases with Phase 3 trials in PFIC, Alagille
syndrome and biliary atresia. For PFIC, the FDA recently granted
Priority Review and set a PDUFA goal date of July 20, 2021. In
Europe, the EMA validated MAA. Bylvay is the only IBATi granted
accelerated assessment by the EMA. The Company has also initiated 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 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 Bylvay or any other Albireo product candidate or
program; including expectations regarding the impact of the
COVID-19 pandemic on our business and our ability to adapt our
plans and activities as appropriate; the pivotal trial for Bylvay
in biliary atresia (BOLD), and the pivotal trial for Bylvay in
Alagille syndrome (ASSERT); 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, BOLD, ASSERT ; the Phase 1 clinical trial for A3907, the
potential approval and commercialization of Bylvay; the potential
for Bylvay to become the first approved drug for PFIC patients;
discussions with the FDA or EMA regarding our programs; 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 potential issuance of a rare pediatric disease
priority review voucher; 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: whether the NDA for Bylvay for the treatment of
pruritus in patients with PFIC will be approved by the FDA and
whether the MAA for Bylvay in PFIC will be approved by the EMA;
whether the FDA or EMA will complete their respective reviews
within the target timelines, including the FDA’s PDUFA goal date,
as a potential result of the impact of the COVID-19 pandemic or
otherwise; the risk that the NDA will not be approved despite the
FDA’s acceptance of the NDA for review; whether the FDA will
require additional information, whether we will be able to provide
in a timely manner any additional information that the FDA
requests, and whether such additional information will be
satisfactory to the FDA; 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; 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 Bylvay 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 Bylvay or A3907, including BOLD and
ASSERT, 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, company’s clinical 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.
Media Contact:Colleen Alabiso,
857-356-3905, colleen.alabiso@albireopharma.comLisa Rivero,
617-947-0899, lisa.rivero@syneoshealth.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)
|
|
March 31, |
|
December 31, |
|
|
2021 |
|
2020 |
Assets |
|
|
|
|
|
|
Current assets: |
|
|
|
|
|
|
Cash and cash equivalents |
|
$ |
217,081 |
|
$ |
251,272 |
Prepaid expenses and other current assets |
|
|
8,884 |
|
|
10,593 |
Total current assets |
|
|
225,965 |
|
|
261,865 |
Property and equipment, net |
|
|
440 |
|
|
478 |
Goodwill |
|
|
17,260 |
|
|
17,260 |
Other assets |
|
|
6,154 |
|
|
6,004 |
Total assets |
|
$ |
249,819 |
|
$ |
285,607 |
Liabilities and Stockholders' Equity |
|
|
|
|
|
|
Current liabilities: |
|
|
|
|
|
|
Accounts payable |
|
$ |
6,577 |
|
$ |
5,283 |
Accrued expenses |
|
|
13,628 |
|
|
19,051 |
Current portion of note payable, net of discount |
|
|
600 |
|
|
— |
Other current liabilities |
|
|
1,406 |
|
|
948 |
Total current liabilities |
|
|
22,211 |
|
|
25,282 |
Liability related to sale of future royalties |
|
|
67,113 |
|
|
65,894 |
Note payable, net of discount |
|
|
9,136 |
|
|
9,621 |
Other long-term liabilities |
|
|
3,441 |
|
|
3,579 |
Total liabilities |
|
|
101,901 |
|
|
104,376 |
Stockholders’ Equity: |
|
|
|
|
|
|
Preferred stock, $0.01 par value per share — 50,000,000 authorized
at March 31, 2021 and December 31, 2020; 0 and
0 issued and outstanding at March 31, 2021 and
December 31, 2020, respectively |
|
|
— |
|
|
— |
Common stock, $0.01 par value per share — 30,000,000 authorized at
March 31, 2021 and December 31, 2020;
19,192,805 and 19,107,040 issued and outstanding at
March 31, 2021 and December 31, 2020,
respectively |
|
|
192 |
|
|
191 |
Additional paid-in capital |
|
|
459,937 |
|
|
456,472 |
Accumulated other comprehensive loss |
|
|
(1,658) |
|
|
(8,612) |
Accumulated deficit |
|
|
(310,553) |
|
|
(266,820) |
Total stockholders’ equity |
|
|
147,918 |
|
|
181,231 |
Total liabilities and stockholders’ equity |
|
$ |
249,819 |
|
$ |
285,607 |
Albireo Pharma,
Inc.Condensed Consolidated Statements of
Operations(in thousands, except share and per
share data)(unaudited)
|
|
Three Months Ended March 31, |
|
|
2021 |
|
2020 |
Revenue |
|
$ |
1,966 |
|
$ |
1,549 |
Operating expenses: |
|
|
|
|
|
|
Research and development |
|
|
19,943 |
|
|
16,130 |
General and administrative |
|
|
15,273 |
|
|
8,153 |
Other operating expense, net |
|
|
6,528 |
|
|
6,816 |
Total operating expenses |
|
|
41,744 |
|
|
31,099 |
Operating loss |
|
|
(39,778) |
|
|
(29,550) |
Interest expense, net |
|
|
(3,955) |
|
|
(1,938) |
Net loss |
|
$ |
(43,733) |
|
$ |
(31,488) |
Net loss per common share - basic and diluted |
|
$ |
(2.29) |
|
$ |
(2.23) |
Weighted-average common shares used to compute basic and diluted
net loss per common share |
|
|
19,131,557 |
|
|
14,132,217 |
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