Albireo Completes Phase 3 BOLD Study Enrollment of Bylvay(R) in Biliary Atresia
– BOLD is the first, largest, and only global Phase 3 trial designed to meet regulatory agencies' requirements
– On track to report topline data by the end of 2024, reinforcing Bylvay's expected position as first IBAT inhibitor to market in biliary atresia
“Enrolling 205 children suffering from biliary atresia into the largest Phase 3 pediatric cholestasis trial ever is a significant achievement allowing us to evaluate Bylvay in an unprecedented number of patients across geographies,” said
The BOLD study is the first and only global Phase 3 study of an ileal bile acid transporter (IBAT) inhibitor in biliary atresia with 58 clinical trial sites in 19 countries. BOLD is a double-blind, randomized, placebo-controlled trial that measures native liver survival with Bylvay over 24 months in children with biliary atresia. Bylvay has received orphan drug designation for biliary atresia in the United States and
“The completion of enrollment for the BOLD trial is a major milestone for the children with biliary atresia and their families. I and my pediatric hepatology colleagues from around the world await completion of the trial in order to evaluate the results. As such, we expect it to provide important insights into the mechanisms of disease progression and potentially demonstrate that we can change the outcome of this disease with a drug for the first time,” said
Biliary atresia is a rare pediatric liver disease and yet the most common pediatric cholestatic liver disease and the leading cause of pediatric liver transplant across all diseases with symptoms typically developing about two to eight weeks after birth. Damaged or absent bile ducts result in bile and bile acids being trapped inside the liver, quickly resulting in cirrhosis and even liver failure. The disease impacts an estimated 45,000 people around the globe and is the leading cause of liver transplants among children. There are no approved pharmacological treatments for biliary atresia.
“It is terrifying to receive a diagnosis of biliary atresia for your newborn, and even worse, when you are informed that your child will likely need a liver transplant. We are very excited that the BOLD trial is fully enrolled, and we eagerly await the results, hoping that families will finally have an option that could help avoid a liver transplant,” said
Bylvay is a potent, oral, once-daily, non-systemic ileal bile acid transport inhibitor (IBATi), with minimal systemic absorption that acts locally in the small intestine. Bylvay is already approved in the
BOLD (NCT04336722) is a double-blind, randomized, placebo-controlled trial to evaluate the efficacy and safety of odevixibat in children who have biliary atresia and have undergone a Kasai procedure before the age of three months. Children in the treatment arm receive odevixibat (120 μg/kg) orally once daily for 24 months. The primary efficacy endpoint is improvement in the proportion of patients who are alive and have not undergone a liver transplant after two years of treatment compared to placebo, and secondary outcome measures include time to onset of any sentinel events, total bilirubin levels and serum bile acid levels. The trial enrolled 205 patients at 58 sites globally.
About Bylvay (odevixibat)
Bylvay is the first drug approved in the
Important Safety Information
- The most common adverse reactions for Bylvay are diarrhea, liver test abnormalities, vomiting, abdominal pain, and fat-soluble vitamin deficiency.
- Liver Test Abnormalities: Patients should obtain baseline liver tests and monitor during treatment. Dose reduction or treatment interruption may be required if abnormalities occur. For persistent or recurrent liver test abnormalities, consider treatment discontinuation.
- Diarrhea: Treat dehydration. Treatment interruption or discontinuation may be required for persistent diarrhea.
- Fat-Soluble Vitamin (FSV) Deficiency: Patient should obtain baseline vitamin levels and monitor during treatment. Supplement if deficiency is observed. If FSV deficiency persists or worsens despite FSV supplementation, discontinue treatment.
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: Albireo’s expected cash runway; Albireo’s commercialization plans; the plans for, or progress, scope, cost, initiation, duration, enrollment, results or timing for availability of results of, development of Bylvay, A3907, A2342 or any other Albireo product candidate or program; the target indication(s) for development or approval; the timing for anticipated regulatory filings; discussions with the FDA or EMA regarding our programs; potential regulatory approval and plans for potential commercialization of Bylvay in biliary atresia or ALGS or Albireo’s other product candidates; the potential benefits or competitive position of Bylvay or any other Albireo product candidate or program or the commercial opportunity in any target indication; 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,” or the negative of these terms or other 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 whether we will achieve a positive outcome in the BOLD trial on the timeline we expect, and if so, whether results from the BOLD study will be sufficient for approval by the FDA and EMA, or whether the FDA and EMA will require additional information, whether we will be able to provide in a timely manner any additional information that the FDA and EMA request, and whether such additional information will be satisfactory to the FDA and EMA; there are no guarantees that Bylvay will be commercially successful; we may encounter issues, delays or other challenges in commercializing Bylvay; whether Bylvay receives adequate reimbursement from third-party payors; the degree to which Bylvay receives acceptance from patients and physicians for its approved indication; challenges associated with execution of our sales activities, which in each case could limit the potential of our product; challenges associated with supply and distribution activities, which in each case could limit our sales and the availability of our product; results achieved in Bylvay in the treatment of patients with PFIC or other approved indications may be different than observed in clinical trials, and may vary among patients; potential 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 Bylvay to date, including findings in PFIC, ALGS and other indications, will be predictive of results from other clinical trials of Bylvay; there is no guarantee that Bylvay will be approved in jurisdictions or for indications (such as biliary atresia or ALGS) beyond the jurisdictions in which or indications for which Bylvay is currently approved; there is no guarantee that our other product candidates will be approved; estimates of the addressable patient population for target indications may prove to be incorrect; 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 Bylvay, including BOLD, and the Phase 2 clinical trial of A3907, and the outcomes of such trials; Albireo’s ability to obtain coverage, pricing or reimbursement for approved products in
Hans Vitzthum, LifeSci Advisors, LLC., 617-430-7578
Source: Albireo Pharma, Inc.