Albireo Pharma, Inc. (Nasdaq: ALBO), a clinical-stage rare liver disease company developing novel bile acid modulators, today presented clinical data from its Phase 3 PEDFIC 1 study and an interim data cut of the PEDFIC 2 long-term extension study of its lead product candidate, Bylvay (odevixibat). Data being shared at the 6th World Congress of Pediatric Gastroenterology, Hepatology, and Nutrition (WCPGHAN) Meeting on June 2 - 5 shows that long-term treatment (up to 48 weeks) was associated with clinically meaningful, positive effects on cholestasis, growth, and sleep parameters in patients with progressive familial intrahepatic cholestasis (PFIC). The totality of the data supports the potential of Bylvay to provide benefits to patients with PFIC. Bylvay is a potent, once-daily, non-systemic ileal bile acid transport inhibitor (IBATi) currently being developed for the treatment of PFIC, biliary atresia, and Alagille syndrome.
"The data being presented at WCPGHAN showed consistent long-term safety and tolerability across studies, treatment groups, and doses and long-term treatment benefits of Bylvay in children with PFIC," said Ron Cooper, President and Chief Executive Officer of Albireo. "These results not only give us confidence in the potential for Bylvay in patients with PFIC, but also in our global pivotal studies in biliary atresia and Alagille syndrome."
Long-Term Safety and Tolerability
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 in a randomized, double-blind, placebo-controlled trial, and PEDFIC 2 is a long-term, open-label Phase 3 extension study. Bylvay improved pruritus, cholestasis, and growth with durable effect.
The data being presented confirms the safety and tolerability of Bylvay in children with PFIC. The observed safety and tolerability profile of Bylvay was consistent across studies, treatment groups, and doses. The analyses discussed in this oral presentation include data for of the safety and tolerability of Bylvay in children with PFIC treated in both PEDFIC 1 and PEDFIC 2 treated for up to 48 weeks. No drug-related serious adverse events were reported in either PEDFIC 1 or PEDFIC 2. One patient in PEDFIC 1 and 3 patients in PEDFIC 2 treated with Bylvay withdrew due to an adverse event. There were low numbers of gastrointestinal adverse events; specifically, treatment-related diarrhea/frequent bowel movements was reported in 10% of Bylvay treated patients in PEDFIC 1 and 5% of placebo-treated patients.
Long-Term Benefits of Bylvay
The oral presentation shows data for secondary and exploratory efficacy outcomes from the PEDFIC 1 and PEDFIC 2 studies, including growth, hepatic chemistries, and sleep parameters. The PEDFIC 2 study observed two cohorts:
Cohort 1 consists of PFIC1 and PFIC2 patients from PEDFIC 1 who rolled into PEDFIC 2. This includes patients treated with Bylvay, as well as patients treated with placebo.
Cohort 2 consists of newly enrolled patients who did not participate in the PEDFIC 1 trial, including patients with PFIC1, PFIC2, PFIC3 and MYO5B deficiency.
At the PEDFIC 2 interim data cut, median duration of exposure to Bylvay was 43 weeks in patients treated with Bylvay, 36 weeks for cohort 1 patients previously treated with placebo in PEDFIC 1, and 19 weeks in cohort 2. Long-term treatment with Bylvay was associated with clinically meaningful, positive effects on cholestasis, growth, and sleep parameters in patients with PFIC. The totality of the data support the potential of Bylvay to provide benefits to patients with PFIC. Key findings include:
Significant improvement in height and weight: Mean height Z sco...
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