— Phase 3 PFIC trial randomization more than three-quarters complete. —
— Topline results expected mid-2020. —
— Pivotal trial in biliary atresia expected to be initiated in 2020. —
— Management to host conference call and webcast today at
“Randomization of patients in our Phase 3 trial of odevixibat in PFIC is nearing completion, and we remain on track to report topline results in mid-2020,” said
- Over three-quarters of the patients needed to complete the PEDFIC 1 Phase 3 study in progressive familial intrahepatic cholestasis (PFIC) had been randomized as of end of
October 2019. Additionally, the patients in screening or entering screening should be sufficient to meet the trial’s 60-patient target based on the screening success rate to date. PEDFIC 1 is enrolling both PFIC type 1 and type 2 patients that are 6 months to 18 years of age. The trial is studying both high- and low-dose odevixibat using the company’s planned commercial formulation. Patients randomized to odevixibat are treated with once-daily oral capsules or sprinkles, which do not require refrigeration. Albireo expects to report topline results in mid-2020, and continues to project a potential first regulatory approval and launch in 2021.
- Enrolled the first patients in the second cohort in PEDFIC 2, the long-term, open-label extension study of PEDFIC 1. The expanded cohort 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. Cohort 1 of PEDFIC 2 is composed of patients who have completed, and rolled over from, the PEDFIC 1 study. We now have patients who are approaching a year on odevixibat treatment.
- Presented data on odevixibat at the
North American Society for Pediatric Gastroenterology, Hepatology and Nutrition Annual Meeting in Chicago. The presentations included data regarding the clinical pharmacology of odevixibat, as well as information about the development of the proprietary patient- and observer-reported outcome (PRO and ObsRO) tools used in the PEDFIC program.
- Completing dialogue with the
U.S. Food and Drug Administration( FDA) regarding key parameters for a planned pivotal clinical trial of odevixibat in biliary atresia, one of the most common rare pediatric liver diseases. Albireo plans to initiate this trial in 2020.
- Randomized the first patients in the company’s Phase 2 multicenter, placebo-controlled clinical trial of elobixibat in non-alcoholic fatty liver disease (NAFLD)/nonalcoholic steatohepatitis (NASH). This trial, which has all 10 initial clinical trial sites active, is designed to randomize 46 patients with biopsy-confirmed NASH, or a diagnosis of suspected NAFLD or NASH based on metabolic syndrome definitions.
- Strengthened the company’s leadership team with the appointment of
Michelle Grahamas Chief Human Resources Officer.
Third-Quarter Financial Results
- Revenues were
$1.4 millionfor the third quarter of 2019, compared to $0.2 millionfor the third quarter of 2018. The year-over-year change was primarily the result of estimated royalty revenue received from EA Pharma for elobixibat for the treatment of chronic constipation.
- R&D expense was
$12.0 millionfor the third quarter of 2019, compared to $9.7 millionfor the third quarter of 2018. The year-over-year change was primarily the result of personnel, and program expenses as we continue to increase our headcount, and program activities, respectively.
- G&A expense was
$6.0 millionfor the third quarter of 2019, compared to $3.9 millionfor the third quarter of 2018. The year-over-year change was primarily the result of personnel and related expenses as we continue to increase our headcount.
- Net loss for the third quarter of 2019 was
$21.9 million, or $(1.73)per share, compared to $14.0 million, or $(1.17)per share for the third quarter of 2018.
- The company had cash and cash equivalents at
September 30, 2019, of $142.7 million, which compares to $163.9 millionat December 31, 2018.
For the full year 2019, the company anticipates total expenses, including R&D and G&A expenses, to be in the range of
As previously announced, Albireo will host a conference call and webcast today, November 6, 2019, at
Albireo was spun out from
This press release includes “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include statements, other than statements of historical fact, regarding, among other things: the plans for, or progress, scope, cost, duration or results or timing for availability of results of, development of odevixibat, elobixibat or any other Albireo product candidate or program, including regarding the Phase 3 clinical program for odevixibat in patients with PFIC; the planned pivotal trial for odevixibat in biliary atresia, the Phase 2 clinical trial for elobixibat in NAFLD/NASH, the target indication(s) for development, the size, design, population, location, conduct, objective, enrollment, duration or endpoints of any clinical trial, or the timing for initiation or completion of or reporting of results from any clinical trial, including the double-blind Phase 3 PFIC trial for odevixibat, and the long-term open-label extension study, the planned pivotal trial for odevixibat in biliary atresia or the Phase 2 trial for elobixibat in NAFLD/NASH; the potential approval and commercialization of odevixibat; discussions with the
|Albireo Pharma, Inc.
Condensed Consolidated Balance Sheets
(in thousands, except share and per share data)
|September 30,||December 31,|
|Cash and cash equivalents||$||142,666||$||163,885|
|Prepaid expenses and other current assets||5,353||3,765|
|Total current assets||148,019||167,650|
|Property and equipment, net||633||187|
|Liabilities and Stockholders' Equity|
|Other current liabilities||683||308|
|Total current liabilities||12,327||12,825|
|Liability related to sale of future royalties||53,073||49,969|
|Other long-term liabilities||4,418||35|
|Common stock, $0.01 par value per share — 30,000,000 authorized at
September 30, 2019 and December 31, 2018; 12,685,326 and 11,969,928 issued and
outstanding at September 30, 2019 and December 31, 2018
|Additional paid in capital||242,638||214,694|
|Accumulated other comprehensive income||10,573||4,293|
|Total stockholders’ equity||101,672||122,637|
|Total liabilities and stockholders’ equity||$||171,490||$||185,466|
|Albireo Pharma, Inc.
Condensed Consolidated Statements of Operations
(in thousands, except share and per share data)
|Three Months Ended September 30,||Nine Months Ended September 30,|
|Research and development||11,996||9,666||31,359||22,228|
|General and administrative||6,010||3,850||16,788||12,216|
|Other operating expense (income), net||4,015||(614||)||6,319||1,377|
|Total operating expenses||22,021||12,902||54,466||35,821|
|Interest expense, net||(1,274||)||(1,367||)||(3,934||)||(4,049||)|
|Non-operating income (expense), net||—||7||—||(2,546||)|
|Net loss per common share - basic and diluted||$||(1.73||)||$||(1.17||)||$||(4.47||)||$||(2.60||)|
|Weighted-average common shares used to
compute basic and diluted net loss per common
Source: Albireo Pharma, Inc.