- 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 -
“PEDFIC 1 achieved very high statistical significance on both the
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
Date & Time:
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
Poster of Distinction #509: A3907, a Novel Inhibitor of
Session: Experimental NAFLD and NASH
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
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
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 odevixibat or any other Albireo product candidate or program, including regarding expectations regarding the impact of COVID-19 on our business and our ability to adapt our approach as appropriate; the Phase 3 clinical program for odevixibat in patients with PFIC, the pivotal trial for odevixibat in biliary atresia (BOLD), and the planned pivotal trial for odevixibat in Alagille syndrome; 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 odevixibat in PFIC, the pivotal trial for odevixibat in biliary atresia, the planned pivotal trial for odevixibat in Alagille syndrome; the potential approval and commercialization of odevixibat; discussions with the FDA or EMA regarding our programs; 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 effects of odevixibat 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,” 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: 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 odevixibat to date, including findings in indications other than PFIC, will be predictive of results from other clinical trials of odevixibat; 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 odevixibat 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 odevixibat, including the pivotal program in biliary atresia or the planned pivotal program in Alagille syndrome, 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.
Source: Albireo Pharma, Inc.