Odevixibat Phase 3 PEDFIC 1 PFIC trial continues to enroll as planned
Initiation of additional cohort of PFIC patients in PEDFIC 2 extension trial announced
Odevixibat pivotal trial in biliary atresia on track to begin H2 2019
IND cleared to initiate elobixibat Phase 2 trial in NASH Q2 2019
Management to host conference call and webcast today at
“For Albireo, 2019 is focused on advancing toward potential approval and launch of odevixibat in its first indication, while further developing odevixibat into a pediatric cholestasis medicine that could benefit people across multiple rare diseases,” said
- Achieved 43 trial sites activated in the U.S.,
Europeand other territories for the PEDFIC 1 Phase 3 study in PFIC as of May 1. PEDFIC 1 is enrolling PFIC subtypes 1 and 2. Expect topline trial results in late 2019 or early 2020, and plan to refine guidance on full enrollment of the study.
- Data in a wider range of PFIC patient types will be generated to support odevixibat in a planned additional patient cohort of PEDFIC 2, the long-term, open-label extension study of PEDFIC 1. The additional cohort will include PFIC patients who are not eligible for PEDFIC 1, including additional PFIC types, adults and neonates. We anticipate initiation of this additional cohort in the second half of this year.
- Held scientific advice meeting with the
European Medicines Agency(EMA), following meeting with the Food and Drug Administration ( FDA), on design of odevixibat pivotal trial in biliary atresia. Plan to initiate trial in H2 2019.
- Granted orphan drug designation from the
FDAfor odevixibat for treatment of biliary atresia, estimated to be one of the larger rare pediatric liver diseases, with extremely high unmet need.
- Presented data from our Phase 2 trial in pediatric cholestasis that support the potential of odevixibat in biliary atresia and Alagille syndrome at the
European Association for the Study of the Liver(EASL) annual conference held in April in Vienna, Austria. The Alagille data were selected for inclusion in “The Best of ILC,” which EASL prepared to highlight the most noteworthy contributions to the scientific program at the conference this year.
- Also at EASL, the academic consortium NAPPED (NAtural course and Prognosis of PFIC and Effect of biliary Diversion), which has the world’s largest PFIC database, presented data on the natural history of PFIC. Notably, the NAPPED data showed that surgical biliary diversion improved survival in PFIC2 patients and that lowering serum bile acids to 118 μmol/L or a reduction of 70% was associated with favorable native liver survival outcomes. Albireo is one of the sponsors of NAPPED through an unrestricted grant.
- Odevixibat published in WHO Drug Information as the recommended international name for A4250.
- Submitted an IND and was cleared by
FDAto begin a Phase 2 multicenter, placebo-controlled trial of elobixibat in NAFLD/NASH.
- Presented at several conferences, including: the 39th Annual Cowen Health Care conference in
Boston, the Roth conferences in Californiaand New York, the H.C. Wainwrightconference in Londonand the Needham Healthcare conference in New York.
- Strengthened the Company’s commercial team with the appointment of
Pamela Stephensonas Chief Commercial Officer.
First-Quarter Financial Highlights
- Revenues were
$570 thousandin the first quarter of 2019, compared to $11.2 millionin the first quarter of 2018.
- R&D expense was
$8.3 millionfor the first quarter of 2019, up 35.4% from $6.2 millionin the first quarter of 2018.
- G&A expense was
$5.3 millionfor the first quarter of 2019, up 28.2% compared to $4.1 millionin the first quarter of 2018.
- Net Loss in the first quarter of 2019 was
$16.7 million, or $(1.39)per share, compared to $1.6 million, or $(0.15)per share in the first quarter of 2018.
- The Company had cash and cash equivalents at
March 31, 2019, of $150.3 million.
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, May 9, 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, 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 (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. The
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 planned 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 planned 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
LifeSci Advisors, LLC
Condensed Consolidated Balance Sheets
(in thousands, except share and per share data)
|March 31,||December 31,|
|Cash and cash equivalents||$||150,339||$||163,885|
|Prepaid expenses and other assets||1,194||850|
|Total current assets||154,336||167,650|
|Property and equipment, net||173||187|
|Other noncurrent assets||1,209||369|
|LIABILITIES AND STOCKHOLDERS’ EQUITY|
|Total current liabilities||9,941||12,825|
|Liability related to sale of future royalties||51,433||49,969|
|Common stock, $0.01 par value per share — 30,000,000 authorized at
March 31, 2019 and December 31, 2018; 12,038,836 and 11,969,928 issued
and outstanding at March 31, 2019 December 31, 2018
|Additional paid in capital||217,807||214,694|
|Accumulated other comprehensive income||6,591||4,293|
|Total stockholders’ equity||111,391||122,637|
|Total liabilities and stockholders’ equity||$||172,978||$||185,466|
Condensed Consolidated Statement of Operations
(in thousands, except share and per share data)
|Three Months Ended March 31,|
|Research and development||8,329||6,151|
|General and administrative||5,293||4,128|
|Other operating (income) expense, net||2,296||1,504|
|Total operating expenses||15,918||11,783|
|Interest income (expense), net||(1,309||)||(1,016||)|
|Non-operating income (expense), net||—||(22||)|
|Net loss before income taxes||(16,657||)||(1,619||)|
|Net loss per share attributable to holders of common stock:|
|Net loss per share - basic and diluted||$||(1.39||)||$||(0.15||)|
|Weighted-average shares outstanding:|
|Weighted average shares outstanding - basic and diluted||12,001,125||10,896,575|
Source: Albireo Pharma, Inc.