Albireo Reports Positive Topline Data from Phase 3 Trial of Bylvay® (odevixibat) in Alagille Syndrome
- Highly statistically significant improvement in pruritus primary endpoint (p=0.002)
- Significant reductions in key secondary endpoint of serum bile acid levels (p=0.001)
- Substantial improvements in multiple sleep parameters
- Well tolerated with low incidence of drug-related diarrhea, no discontinuations
- Plans to immediately file in the
U.S.and EU, adding to approved indication for PFIC
- Conference call to be held today at
“Our ASSERT Phase 3 study demonstrated early, rapid, and sustained effects on reducing pruritus and bile acids in Alagille syndrome, as it did in the Phase 3 PFIC study,” said
A potent, once-daily, non-systemic ileal bile acid transport inhibitor (IBATi), Bylvay has minimal systemic exposure and acts locally in the small intestine. Bylvay is already approved in the
“The robust results from the ASSERT Phase 3 trial are important because physicians urgently need more options in the treatment of Alagille syndrome. Bylvay reduced the devastating pruritus, which is so common among this patient population and critical to helping us improve sleep, among other burdens of the disease,” explained Nadia Ovchinsky, MD, Pediatric Gastroenterologist and Hepatologist, Children’s Hospital at Montefiore and ASSERT Principal Investigator. “The study also showed Bylvay reduced serum bile acid levels, which could indicate that Bylvay may diminish the severity of liver disease over time, an important consideration for me as a treating physician.”
Alagille syndrome, or ALGS, is a rare, multisystem genetic disorder that the Company estimates impacts 25,000 people globally. ALGS 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 percent also present with severe, intractable pruritus.
“Alagille syndrome is a devastating diagnosis and families of children with this syndrome need more treatment options,” said
ASSERT Phase 3 Clinical Trial Data
ASSERT is a gold standard, prospective intervention trial with 32 sites across
In the primary analysis, the study met the primary endpoint showing statistically significant reduction in pruritus as measured by the PRUCISION Observer-Reported Outcome scratching score (0-4 point scale), from baseline at month 6 (weeks 21 to 24), compared to the placebo arm (p=0.002). The study also met the key secondary endpoint showing a statistically significant reduction in serum bile acid concentration from baseline to the average of weeks 20 and 24 (compared to the placebo arm p=0.001). Statistically significant improvements in multiple sleep parameters were observed as early as week 1-4 compared to patients on placebo with continued improvement through week 24. In the study, there were no patient discontinuations. Bylvay was well tolerated, with an overall adverse event incidence similar to placebo and a low incidence of drug-related diarrhea (11.4% vs. 5.9% placebo). Full results from the Phase 3 clinical trial will be presented at a future scientific meeting.
|Mean change from baseline in scratching score at month 6 (weeks 21 to 24)||-0.80||-1.69||0.002|
|Mean change in serum bile acid levels from baseline to average of weeks 20 & 24||22.39||-90.35||0.001|
|Rate of drug-related diarrhea||5.9%||11.4%||-|
|Number of discontinuations||0||0||-|
The Company continues to enroll patients in the Phase 3 BOLD study, which is the first and only pivotal trial of an IBATi in biliary atresia (BA) and remains on track to fully enroll by end of year, with topline data planned for 2024. BA is the most common pediatric cholestatic liver disease with no approved drug treatment. With clinical programs in ALGS and BA, Bylvay has the potential to be approved for three pediatric cholestatic liver diseases.
Albireo will host a conference call and live audio webcast at 8:30 a.m. EDT. To access the live conference call by phone, dial 1-877-407-0792 (domestic) or 1-201-689-8263 (international), and provide the access code 13733685. A live audio webcast will be accessible from the Investors page at ir.albireopharma.com/. To ensure a timely connection to the webcast, it is recommended that participants register at least 15 minutes prior to the scheduled start time. An archived version of the webcast will be available for replay on the Events & Presentations section of the Investors page of Albireo’s website for 3 months following the event.
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.
About ALGS Expanded Access Program
Albireo continues to prioritize access and continued scientific research for patients living with rare cholestatic liver diseases, with the Expanded Access Program (EAP) for ALGS. Albireo has partnered with
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 impact of the Expanded Access Program; 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 the regulatory filings to be made for Bylvay in patients with ALGS will be made on the timelines we expect and be approved by the FDA and EMA; whether the FDA and EMA will complete their respective reviews within target timelines, once determined; 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.