Final PEDFIC 1 and interim PEDFIC 2 results accepted at AASLD’s The Liver Meeting 2020 -
- PEDFIC 1 and PEDFIC 2: largest global Phase 3 in children with PFIC types 1, 2 & 3 -
- Two abstracts to be presented on new pipeline compound -
BOSTON, Nov. 02, 2020 (GLOBE NEWSWIRE) -- Albireo Pharma, Inc. (Nasdaq: ALBO), a clinical-stage rare liver disease company developing novel bile acid modulators, today announced late-breaker acceptances by the American Association for the Study of Liver Diseases (AASLD) for the Phase 3 PEDFIC 1 trial and PEDFIC 2 long-term extension trial. There will be an oral presentation of the results from PEDFIC 1, the global Phase 3 clinical trial evaluating the efficacy and safety of odevixibat in children with progressive familial intrahepatic cholestasis (PFIC) in the late-breaker session. PEDFIC 2 is an open-label Phase 3 extension study of the long-term efficacy and safety of odevixibat in children with PFIC, and interim results will be presented as a late-breaker poster. In addition, data on novel bile acid modulator approaches and new product candidate A3907 have also been accepted for poster presentations. The AASLD The Liver Meeting Digital Experience™ (TLMdX) is being held November 13-16.
“PEDFIC 1 achieved very high statistical significance on both the U.S. and EU primary endpoints and we are eager to share the full results from the PEDFIC program to date. Odevixibat sustained reductions in bile acids and pruritus, improved growth parameters, and was well tolerated with very low incidence of diarrhea across a wide range of PFIC patients,” said Ron Cooper, President and Chief Executive Officer of Albireo. “Our scientific leadership and pipeline execution will also shine with exciting data introducing multiple new bile acid modulator approaches focusing on our novel compound A3907 in adult liver diseases.”
Abstracts are now available on the AASLD website. Details of the late-breaking abstracts will be presented in November and published in the December issue of HEPATOLOGY:
Presentation #L04: Efficacy and Safety of Odevixibat, an Ileal Bile Acid Transporter Inhibitor, in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2: Results from PEDFIC 1, a Randomized, Double-Blind, Placebo-Controlled Phase 3 Trial
Session: Late-breaking Oral Abstract Session 1 with presentation by lead author Richard J. Thompson, M.D., Ph.D., Professor of Molecular Hepatology at King's College London and principal investigator of the study
Date & Time: Sunday, Nov. 15, 5:30 p.m. EST
Poster #LP19: Long-term Efficacy and Safety of Odevixibat, an Ileal Bile Acid Transporter Inhibitor, in Children with Progressive Familial Intrahepatic Cholestasis: Interim Results from PEDFIC 2, an Open-label Phase 3 Trial
Session: Late-breaking Abstract Posters
Abstracts accepted earlier in the month were published in the October issue of HEPATOLOGY and included one abstract selected by TLMdX as a Poster of Distinction for being in the top 10 percent of scored poster abstracts.
Poster #348: Discovery and Characterization of Novel Bile Acid Transporter Inhibitors with Potential for Treatment of Liver Diseases
Session: Biliary Transport and Metabolism
Poster of Distinction #509: A3907, a Novel Inhibitor of Bile Acid Transport in the Intestine and Kidney, Improves Markers of Metabolic and Hepatic Pathology, and Reduces Nonalcoholic Fatty Liver Disease Activity Score and Fibrosis Stage in a Diet-induced and Biopsy-confirmed Mouse Model of Nonalcoholic Steatohepatitis
Session: Experimental NAFLD and NASH
About Odevixibat
Odevixibat is an investigational product candidate being developed to treat rare pediatric cholestatic liver diseases, including progressive familial intrahepatic cholestasis (PFIC), biliary atresia and Alagille syndrome. A highly potent, once-daily, non-systemic ileal bile acid transport inhibitor (IBATi), odevixibat acts locally in the small intestine. Odevixibat 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. Albireo conducted the largest ever global Phase 3 trial in PFIC1 and PFIC2. The PEDFIC 1 trial evaluated odevixibat 40 µg/kg/day or 120 µg/kg/day or placebo in 62 patients, ages 6 months to 15.9 years. Positive results from the trial were announced on September 8, 2020: News Release – Albireo Phase 3 Trial Meets Both Primary Endpoints for Odevixibat in PFIC. Odevixibat is currently being evaluated in the ongoing PEDFIC 2 open-label trial (NCT03659916) and the BOLD Phase 3 trial in patients with biliary atresia (NCT04336722). Initiation of a pivotal Phase 3 trial of odevixibat for Alagille syndrome is also anticipated by the end of 2020.
Odevixibat has received fast track, rare pediatric disease and orphan drug designations in the United States. In addition, the FDA has granted orphan drug designation to odevixibat for the treatment of Alagille syndrome, biliary atresia and primary biliary cholangitis. The EMA has granted odevixibat orphan designation, as well as access to the PRIority MEdicines (PRIME) scheme for the treatment of PFIC. Its Pediatric Committee has agreed to Albireo’s odevixibat Pediatric Investigation Plan for PFIC and biliary atresia. EMA has also granted orphan designation to odevixibat for the treatment of Alagille syndrome, biliary atresia and primary biliary cholangitis. Odevixibat has the potential to become the first approved drug treatment for patients with PFIC. The Company intends to complete regulatory filings in the EU and U.S. for odevixibat in PFIC no later than early 2021, in anticipation of potential regulatory approval, issuance of a rare pediatric disease priority review voucher and launch in the second half of 2021.
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, and other adult liver diseases and disorders. Albireo’s lead product candidate, odevixibat, is being developed to treat rare pediatric cholestatic liver diseases and is in Phase 3 development in progressive familial intrahepatic cholestasis (PFIC) and biliary atresia, with a third Phase 3 trial being planned in Alagille syndrome. The Company expects to complete IND-enabling studies for new preclinical candidate A3907 this year. 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.
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