- A4250 Phase 3 PFIC pivotal program on target for end of ‘19/early ’20 topline readout -
- Plan to initiate second A4250 pivotal program in biliary atresia -
- Plan to initiate Phase 2 study with elobixibat in NASH -
- Management to host conference call and webcast today at
“In 2018, Albireo successfully delivered according to plan,” said
“As positive as last year was, we anticipate 2019 will be truly transformative, with the potential for Phase 3 results in PFIC, initiation of a second rare pediatric cholestatic liver disease pivotal program, and a move into nonalcoholic steatohepatitis (NASH) with an elobixibat Phase 2 trial initiation. Our team is energized for the year ahead.”
- Achieved trial site activation target for the A4250 PEDFIC 1 Phase 3 pivotal study. We currently have 37 trial sites active in the U.S.,
Europeand ROW as of February 28, 2019, and we continue to expect topline trial results end of 2019 or early 2020.
- Received multiple key regulatory designations that support our development program across indications, including fast track designation from the
U.S. Food and Drug Administration( FDA) for A4250 in the treatment of pruritus in PFIC, orphan drug designation from the FDAfor A4250 in Alagille syndrome, and orphan designation from both the FDAand the European Commission for A4250in biliary atresia.
- Announced the presentation of important findings in PFIC and other cholestatic liver diseases at NASPGHAN and AASLD, illustrating the unmet need in PFIC and the potential impact of A4250. Results presented at NASPGHAN demonstrate that lower serum bile acids and bilirubin after partial external biliary diversion (PEBD) surgery are associated with decreased aggregate need for liver transplant in PFIC patients. Findings presented by the NAPPED Consortium at AASLD The Liver Meeting demonstrate the association of native liver survival rates and serum bile acid levels.
- Presented at the William Blair Biotech Focus Day event “Bay Area Innovation” and the Jefferies 2018
London Healthcare Conference.
- Strengthened senior management team with the appointment of
Simon Harfordas Chief Financial Officer and Treasurer, Patrick Hornas Chief Medical Officer and Jason Duncanas General Counsel.
- Planning for PEDFIC 1 topline results end of 2019 or beginning of 2020.
- Planning a major step toward realizing the larger opportunity for A4250. Preparing to initiate a pivotal trial in biliary atresia with A4250 in the second half of 2019. Biliary atresia may be the largest of the rare pediatric liver diseases of primary initial interest for A4250. Have received orphan designations for A4250 in biliary atresia in U.S. and EU, and engaged regulators on trial design. We continue to see potential for A4250 beyond PFIC and biliary atresia, and are evaluating other indications.
- Planning a significant development effort in NASH. Preparing to initiate a Phase 2 trial with elobixibat in Q2 and continue to progress preclinical novel bile acid modulators for NASH, all of which are potential candidates for partnering in the future.
Financial Update for the Year Ended
- Revenues were
$12.7 millionfor the year ended December 31, 2018
- R&D expense was
$31.7 million, up 144.3% compared to $13.0 millionfor the year ended December 31, 2017.
- G&A expense was
$18.1 million, up 18.5% compared to $15.2 millionin the same period of 2017.
- Net Loss was
$46.1 million, or $(3.94)per share, compared to $24.4 million, or $(3.12)per share for the year ended December 31, 2017.
- The Company had cash and cash equivalents at
December 31, 2018of $163.9 million, compared to $53.2 millionat December 31, 2017.
For the full year 2019, we anticipate total expenses, including R&D and G&A expenses, to be in the range of
As previously announced, Albireo will host a conference call and webcast today, March 7, 2019, at
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, 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 (PFIC). Albireo’s clinical pipeline also includes two Phase 2 product candidates. Albireo’s elobixibat, approved in Japan for the treatment of chronic constipation, 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, Massachusetts, and its key operating subsidiary is located in Gothenburg, Sweden. For more information on Albireo, please visit www.albireopharma.com.
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 A4250 or any other Albireo product candidate or program, including regarding the Phase 3 clinical program for A4250 in patients with PFIC; the target indication(s) for development, the size, design, population, location, conduct, objective, 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 A4250; 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 A4250, or any other Albireo product candidate or program or the commercial opportunity in any target indication; the potential benefits of a rare pediatric disease designation, the potential benefits of an orphan drug designation, the potential benefits of a fast track designation, the pricing of A4250 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 A4250 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 A4250; whether either or both of the
Consolidated Balance Sheets
(in thousands, except share and per share data)
|December 31,||December 31,|
|Cash and cash equivalents||$||163,885||$||53,231|
|Prepaid expenses and other assets||850||1,054|
|Total current assets||167,650||55,011|
|Property and equipment, net||187||178|
|Other noncurrent assets||369||775|
|LIABILITIES AND STOCKHOLDERS’ EQUITY|
|Total current liabilities||12,825||7,929|
|Liability related to sale of future royalties||49,969||—|
|Common stock, $0.01 par value per share — 30,000,000 authorized at December 31, 2018 and December 31, 2017; 11,969,928 and 8,902,784 issued and outstanding at December 31, 2018 and December 31, 2017||120||89|
|Additional paid in capital||214,694||114,522|
|Accumulated other comprehensive income||4,293||1,001|
|Total stockholders’ equity||122,637||65,253|
|Total liabilities and stockholders’ equity||$||185,466||$||73,224|
Consolidated Statements of Operations
(in thousands, except share and per share data)
|Year Ended December 31,|
|Research and development||31,732||12,991|
|General and administrative||18,061||15,246|
|Other operating (income) expense, net||837||(3,659||)|
|Total operating expenses||50,630||24,578|
|Interest income (expense), net||(4,838||)||40|
|Other non-operating income (expense), net||(3,363||)||335|
|Net loss before income taxes||(46,091||)||(24,202||)|
|Net loss per share attributable to holders of common stock:|
|Net loss per share - basic and diluted||$||(3.94||)||$||(3.12||)|
|Weighted average shares outstanding - basic and diluted||11,702,785||7,819,302|
Source: Albireo Pharma, Inc.