Benzinga | Dec 13, 2021 08:35AM EST

Albireo To Present Posters At NASPGHAN 2021 Dec. 13 And 15

-- Data show significant correlations in serum bile acid reductions and long-term improvements in pruritus and sleep



-- Reductions in serum bile acids and improvements in pruritus with and without concomitant ursodeoxycholic acid and/or rifampicin use and Bylvay treatment

-- Improvements in serum bile acids, pruritus, quality of life, and sleep observed in some children with prior partial external biliary diversion surgery

-- Considerable impact on quality of life of patients and their caregivers

BOSTON, Dec. 13, 2021 (GLOBE NEWSWIRE) -- Albireo Pharma, Inc. (NASDAQ:ALBO), a rare liver disease company developing novel bile acid modulators, will be presenting five posters on Bylvay (odevixibat) at the North American Society for Pediatric Gastroenterology, Hepatology and Nutrition (NASPGHAN) meeting being held December 12--18. Data will be presented that show evidence of correlations in serum bile acid reductions and long-term improvements in pruritus and sleep in patients with progressive familial intrahepatic cholestasis (PFIC); reductions in serum bile acids and improvements in pruritus with and without concomitant ursodeoxycholic acid (UDCA) and/or rifampicin use; and the efficacy and safety of Bylvay in patients with PFIC and prior partial external biliary diversion (PEBD). Data on disease burden and natural history of PFIC will be presented to describe the clinical characteristics of the disease. There will also be a poster presentation on the Phase 3 double-blind, randomized, placebo-controlled ASSERT study of Bylvay in Alagille syndrome (ALGS). Bylvay is a potent, non-systemic ileal bile acid transport inhibitor (IBATi) that is approved in the U.S. for the treatment of pruritus in patients 3 months of age and older in all types of PFIC and in Europe for the treatment of all types of PFIC in patients aged 6 months or older.

"We are pleased to present additional data supporting Bylvay's potential to reduce disease burden and improve the standard of care in the treatment of pruritus in PFIC," said Jan Mattsson, Chief Scientific Officer at Albireo. "As we continue to see sustained correlations between the reduction of serum bile acids and long-term improvements in pruritus and quality of life measures, as well as certain results from the use of Bylvay with post-PEBD patients, we are encouraged that data could demonstrate Bylvay's impact on disease modification over time."

Bylvay PEDFIC 1 & 2 Treatment Data

PEDFIC 1 was the first and largest, global, pivotal Phase 3 study conducted in PFIC, which evaluated the efficacy and tolerability of Bylvay in reducing pruritus and serum bile acids (sBAs) in a randomized, double-blind, placebo-controlled trial, and PEDFIC 2 is a long-term, open-label Phase 3 extension study.

Poster of Distinction

Poster #69: Relationships Between Decreases in Serum Bile Acids, Pruritus, and Sleep Disturbance Scores With Up to 72 Weeks of Odevixibat Treatment in Patients With Progressive Familial Intrahepatic Cholestasis

Poster Session I: Monday, December 13, 1:30-2:15pm ET

* Significant correlations were observed between reductions in sBAs and reductions in pruritus and most sleep disturbance scores.

* Percentage change in sBAs from baseline to weeks 49?72 was significantly correlated with change in pruritus scores during that interval (r=0.58; P<0.001).

* Moderate correlations were also observed between percentage change in sBAs from baseline to weeks 49?72 and changes during that interval in caregiver-reported percentage of days where patients had bleeding associated with scratching, needed soothing or help falling asleep, and were sleeping with caregivers.

* No serious drug-related treatment emergent adverse events (TEAEs) or deaths occurred. The overall incidence of any event of diarrhea was 21% and all instances were mild to moderate in severity and resolved, most without intervention.

Poster of Distinction

Poster #78: Efficacy and Safety of Odevixibat Therapy With Concomitant UDCA or Rifampicin in Children With Progressive Familial Intrahepatic Cholestasis: Data From the PEDFIC 1 and PEDFIC 2 Trials

Poster Session I: Monday, December 13, 1:30-2:15pm ET

* Patients with PFIC receiving Bylvay treatment experienced reductions in sBAs and improvements in pruritus with and without concomitant ursodeoxycholic acid (UDCA) and/or rifampicin use.

* After 48 weeks of Bylvay treatment, percentages of patients meeting criteria for sBA response were similar among patients using vs not using UDCA and/or rifampicin (using vs not using, each 67%) 74% and 40% in patients using vs not using UDCA, respectively. 54% and 82% in patients using vs not using rifampicin.

* Mean proportions of positive pruritus assessments (PPAs) were similar in patients using vs not using UDCA (65% vs 72%, respectively) and in patients using vs not using UDCA and/or rifampicin (66% vs 69%); mean proportions of PPAs in patients using vs not using rifampicin (56% and 81%).

* Safety and tolerability were comparable in patients using vs not using UDCA and/or rifampicin with Bylvay. Incidence of TEAEs was similar in patients using vs not using UDCA and/or rifampicin (78% vs 90%, respectively), as well as in patients using vs not using UDCA (77% vs 87%) and rifampicin (75% vs 86%).

Poster #303: Efficacy And Safety Of Odevixibat In Children With Progressive Familial Intrahepatic Cholestasis With Prior Partial External Biliary Diversion

Poster Session II: Wednesday, December 15, 1:30-2:15pm ET

* Improvements were observed in sBA levels, pruritus, quality of life (QoL), and sleep parameters in some patients who had prior partial external biliary diversion, or PEBD surgery. Improvements were observed despite elevated sBAs and pruritus in these patients at baseline, suggesting that some patients with poor response to PEBD could potentially respond to Bylvay treatment.

* All of the 10 patients with prior PEBD had elevated sBAs and pruritus scores prior to the first dose of Bylvay, indicating that prior PEBD surgery was unsuccessful or only partially successful. 7 patients had reductions in pruritus score and 4 met criteria for pruritus response (i.e., ?1-point drop in pruritus score). 5 patients had reductions in sBA levels; 1 patient, who also met pruritus response criteria, met criteria for sBA response (i.e., sBAs reduced by ?70% or levels ?70 ?mol/L) at last assessment. Of the 9 patients with post-baseline QoL assessments, 6 had improved observer-reported PedsQL total scores, including all 4 pruritus and/or sBAs responders. Pruritus and/or sBA responders also had reductions from baseline to last assessment in percentage of days with bleeding associated with scratching, needing soothing or help falling asleep, and sleeping with caregivers.

* TEAEs, including mild abdominal pain, were observed in the subgroup of patients with prior PEBD; all TEAEs were mild to moderate in severity, and no patients discontinued due to a TEAE.

Poster #292: Disease Burden and Natural History of Progressive Familial Intrahepatic Cholestasis: Baseline Clinical Characteristics Among Odevixibat-Treated Patients in the Phase 3 PEDFIC Studies

Poster Session II: Wednesday, December 15, 1:30-2:15pm ET

* Baseline characteristics of the patient population studied reflect the broader population of patients with PFIC; specifically this is a pediatric population with cholestasis, impaired hepatic function, growth deficits, and impacted sleep parameters; and PFIC had a considerable impact on the QoL of patients and their caregivers.

* The complexity of PFIC was illustrated by the heterogeneity and severity of the clinical signs and symptoms in these patients with different types of PFIC.

Bylvay in ASSERT Study

Poster #281: The ASSERT Study: A Phase 3 Double-blind, Randomized, Placebo-controlled Study of the Safety and Efficacy of Odevixibat In Patients With Alagille Syndrome

Poster Session II: Wednesday, December 15, 1:30-2:15pm ET

* ASSERT is an on-going, global Phase 3 pivotal trial designed to evaluate the safety and efficacy of Bylvay for 24 weeks in relieving pruritus in patients with Alagille syndrome.

* ASSERT is expected to enroll approximately 63 patients, including approximately 45 patients aged <18 years and an additional exploratory cohort of up to 18 patients aged ?18 years.

Full scientific abstracts are available and can be viewed here: Journal of Pediatric Gastroenterology and Nutrition.