Albireo Completes Enrollment in Pivotal Phase 3 ASSERT Study

Albireo Pharma, Inc. recently announced the completion of patient enrollment in the ASSERT study, a Phase 3 pivotal trial of Bylvay (odevixibat) in patients with Alagille syndrome (ALGS). Topline results are expected to be available by the end of the year, consistent with guidance, and with the enrollment of 52 patients versus an original target of 45. ASSERT is a gold standard, global, double-blind, randomized, placebo-controlled trial designed to evaluate the safety and efficacy of Bylvay in patients with ALGS over 24 weeks. Both the US FDA and EMA have agreed on the Phase 3 study design and have indicated that this single study would be sufficient for regulatory filings. Bylvay is a potent, once-daily, non-systemic ileal bile acid transport inhibitor (IBATi) already approved in the US 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.

“Families coping with Alagille syndrome are deeply in need of more therapeutic options, as evidenced by the completed enrollment for our Phase 3 ASSERT study, consistent with guidance, while exceeding our original enrollment target,” said Ron Cooper, President and Chief Executive Officer of Albireo. “Beyond this important clinical milestone, we continue to deliver on our promise to increase access to Bylvay for all eligible patients and are pleased to announce the opening of an Expanded Access Program for Alagille syndrome patients.”

ALGS is a rare, multisystem genetic disorder that can affect the liver, heart, skeleton, eyes, central nervous system, kidneys, and facial features. Liver damage is caused by a paucity of bile ducts preventing bile flow from the liver to the small intestine. Approximately 95% of patients with the condition present with chronic cholestasis, usually within the first three months of life, and as many as 88% also present with severe, intractable pruritus.

Aligned to the company’s mission of providing hope for families, Albireo continues to prioritize access and continued scientific research for patients living with rare cholestatic liver diseases. Albireo opened an Expanded Access Program (EAP) with the first patient already enrolled. The program is available in the US and Europe, which aims to provide access to Bylvay for patients suffering from ALGS, prior to the product’s planned approval and reimbursement. Timing of availability in Europe will vary due to country-specific regulations and stock availability. This program is available for patients with a clinical diagnosis of ALGS who have no other therapeutic options. For full eligibility criteria and additional information, please contact odevixibat@tannerpharma.com.

“I am proud of the work and great progress we are making in our studies of Bylvay in rare cholestatic diseases,” said Jan Mattsson, Chief Scientific Officer and Head of R&D at Albireo. “Completing enrollment of the Phase 3 ASSERT study, while continuing to enroll biliary atresia patients in the Phase 3 BOLD study, are significant steps in our clinical development and scientific leadership in bile acid modulation for the treatment of liver diseases.”

The company continues to enroll and dose patients in the Phase 3 BOLD study, which is the first and only pivotal trial of an IBATi in biliary atresia, that passed the 50% enrollment milestone and remains on track for topline data in 2024. Biliary atresia is the most common pediatric cholestatic liver disease with no approved drug treatment.

ASSERT is a gold standard, prospective intervention trial with 35 sites across North America, Europe, Middle East and Asia Pacific. The double-blind, randomized, placebo-controlled trial is designed to evaluate the safety and efficacy of 120 µg /kg/day Bylvay (odevixibat) for 24 weeks in relieving pruritus in patients with ALGS. Secondary endpoints will measure serum bile acid levels and safety and tolerability. The trial enrolled patients aged 0 to 17 years of age with a genetically confirmed diagnosis of ALGS. The primary efficacy endpoint is a change from baseline in scratching to Month 6 (Weeks 21 to 24) as measured by the Albireo ObsRO caregiver instrument. The key secondary efficacy endpoint is a change in serum bile acid levels from baseline to the average of Week 20 and Week 24.

After completing the ASSERT trial, study participants have the option to enroll in an extension study to continue receiving access to Bylvay while it advances along the regulatory pathway. An EAP program is also available to provide eligible patients with ALGS with access to Bylvay (odevixibat) prior to the product’s approval and reimbursement, subject to authorization by the relevant competent authority. Learn more about Albireo’s commitment to access at www.albireopharma.com/responsibility/access/.

Albireo has partnered with Tanner Pharma Group for the ALGS EAP. Eligible patients with ALGS, that are not in screening in the ASSERT study, may receive Bylvay on a free-of-charge (FOC) basis, subject to authorization by the relevant country competent authority, and meeting of Albireo’s eligibility criteria. If you are a physician who would like to request ALGS EAP access for your patient, please send your enquiry to Tanner using odevixibat@tannerpharma.com, and you will receive a response within one working day with further information.

Bylvay is the first drug approved in the US for the treatment of pruritus in patients 3 months of age and older in all types of progressive familial intrahepatic cholestasis (PFIC). Limitation of Use: Bylvay may not be effective in PFIC type 2 patients with ABCB11 variants resulting in non-functional or complete absence of bile salt export pump protein (BSEP-3). The European Commission (EC) and UK Medicines and Healthcare Products Regulatory Agency (MHRA) have also granted marketing authorization of Bylvay for the treatment of PFIC in patients aged 6 months or older. Bylvay is available in Germany and the UK and will be available for sale in other European countries following pricing and reimbursement approval. A potent, once-daily, non-systemic ileal bile acid transport inhibitor, Bylvay acts locally in the small intestine. Bylvay can be taken as a capsule for patients that are able to swallow capsules, or opened and sprinkled onto food, which is a factor of key importance for adherence in a pediatric patient population. The most common adverse reactions for Bylvay are diarrhea, liver test abnormalities, vomiting, abdominal pain, and fat-soluble vitamin deficiency. The medicine can only be obtained with a prescription. For more information about using Bylvay, see the package leaflet or contact your doctor or pharmacist. For full prescribing information, visit www.bylvay.com.

In the U.S. and Europe, Bylvay has orphan exclusivity for its approved PFIC indications, and orphan designations for the treatment of ALGS, biliary atresia and primary biliary cholangitis. Bylvay is being evaluated in the ongoing PEDFIC 2 open-label trial in patients with PFIC, in the BOLD Phase 3 study for patients with biliary atresia and the ASSERT Phase 3 study for ALGS.

Albireo Pharma is a rare disease company focused on the development of novel bile acid modulators to treat rare pediatric and adult liver diseases. Albireo’s lead product, Bylvay, was approved by the US FDA as the first drug for the treatment of pruritus in all types of progressive familial intrahepatic cholestasis (PFIC), and it is also being developed to treat other rare pediatric cholestatic liver diseases with Phase 3 trials in Alagille syndrome (ALGS) and biliary atresia, as well as Open-label Extension (OLE) studies for PFIC and ALGS. In Europe, Bylvay has been approved for the treatment of PFIC with pricing listing in Germany and guidance from the National Institute for Health and Care Excellence (NICE) recommending Bylvay for use in the National Health Service in the England, Wales and Northern Ireland UK. The Company has also completed a Phase 1 clinical trial for A3907 to advance development in adult cholestatic liver disease, with IND-enabling studies progressing with A2342 for viral and cholestatic liver disease. 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 2019 and 2020 Best Places to Work in Massachusetts. For more information, visit www.albireopharma.com.