– Company outlining large global rare cholestatic liver disease opportunity –
– Advancing plans for anticipated odevixibat
– Expansion opportunity beyond PFIC and beyond odevixibat –
– Commercial management to host conference call and webcast today at
“We are delighted that both the EMA and FDA confirmed acceptance of our odevixibat filings in the
Today’s presentation will describe efforts the Company is taking to advance its broad, wholly owned pipeline of rare pediatric and adult liver assets. Management will present commercial plans for lead candidate odevixibat, which has imminent regulatory milestones in the
Large Global Opportunity
Odevixibat is a potent, once-daily, non-systemic ileal bile acid transport inhibitor (IBATi) being developed to treat patients with rare pediatric cholestatic liver diseases, including PFIC, biliary atresia and Alagille syndrome (ALGS). Albireo conducted extensive research to understand the global market opportunity for PFIC, an ultra-rare pediatric liver disease for which there is no approved drug therapy today. Based on these efforts, the Company expects the global market opportunity in pediatric cholestatic liver diseases to be larger than anticipated. The top 25 global markets (excluding
Albireo is advancing its global commercialization strategy and launch readiness for odevixibat in anticipation of a potential launch in H2 2021. The Company has commenced educational efforts with payors to educate them on the disease progression and burden of PFIC. As part of Albireo’s commitment to ensuring patients living with PFIC have access to odevixibat, the Company is building AlbireoAssistTM, a fully owned and operated patient support program. This proprietary program will be staffed internally at Albireo by dedicated care coordinators helping to deliver on Albireo’s commitment to patient access.
“Children and families are urgently waiting for odevixibat, which, if approved, will be potentially the first non-surgical treatment for PFIC,” said
Until odevixibat is approved for commercial sale, the Company provides an Expanded Access Program (EAP) for eligible patients with PFIC in the
Albireo has all key leadership in place to support rapid launches in the
As part of the ex-
Expansion Beyond PFIC
The Company has been building infrastructure needed to launch odevixibat in PFIC globally, which will serve as a framework for future indications in ALGS and biliary atresia with minimal added cost. Odevixibat is also currently being evaluated in a Phase 3, long-term extension study and two pivotal Phase 3 trials: the ongoing PEDFIC 2 Phase 3 open-label trial in patients with PFIC, the BOLD Phase 3 trial in patients with biliary atresia and the ASSERT Phase 3 trial in ALGS.
The Company is exploring multiple methods for modulating bile acids to significantly change the bile acid transporter approach in adult liver. In developing therapies for adult liver conditions, the Company will initially focus on primary sclerosing cholangitis (PSC) and primary bilateral cholangitis (PBC) with two promising new candidates with two different mechanisms of action.
“Albireo maintains a global, wholly owned, unencumbered pipeline of products addressing multiple indications and commercial opportunities in multiple rare liver indications,” added Cooper. “The enterprise infrastructure we are building for the PFIC launch will be sufficient to effectively serve multiple rare liver diseases in the coming years.”
The Company has sufficient capital resources to fund the planned launch and development programs. Cash runway into 2023 and plans to monetize a Priority Review Voucher, if received upon approval. 2020 unaudited cash burn was
As previously announced, Albireo will host a conference call and webcast today. Details and dial-in information are listed below. 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 Media & Investors page of the Albireo website for 2 months following the event.
Dial-in: To access the live conference call by phone, dial 1-877-407-0792 (domestic) or 1-201-689-8263 (international). Provide access code: 13714756
Live webcast link: http://public.viavid.com/index.php?id=142919
Link is also accessible from the Media & Investors page of Albireo’s website http://ir.albireopharma.com/
Odevixibat is an investigational product candidate being developed to treat rare pediatric cholestatic liver diseases, including progressive familial intrahepatic cholestasis (PFIC), biliary atresia and Alagille syndrome. A potent, once-daily, non-systemic ileal bile acid transport inhibitor (IBATi), odevixibat acts locally in the small intestine. Odevixibat does not require refrigeration and can be taken as a capsule for older children, or opened and sprinkled onto food, which are factors of key importance for adherence in a pediatric patient population. The FDA has granted Priority Review and set a Prescription Drug User Fee Act (PDUFA) goal date of
The MAA and NDA filings are supported by results from PEDFIC 1 and PEDFIC 2 Phase 3 studies. PEDFIC 1 was the first and largest, global, pivotal Phase 3 study conducted in PFIC, which evaluated the efficacy and tolerability of odevixibat in reducing pruritus and serum bile acids in a randomized, double-blind, placebo-controlled trial. In the PEDFIC 1 study, odevixibat met both primary endpoints and was well tolerated with very low incidence of diarrhea/frequent bowel movements (9.5% of odevixibat treated patients vs. 5.0% of placebo patients). PEDFIC 2 is a long-term, open-label Phase 3 extension study. Odevixibat is also currently being evaluated in the BOLD Phase 3 trial in patients with biliary atresia, and the global Phase 3 ASSERT trial for ALGS.
Progressive familial intrahepatic cholestasis (PFIC) is a rare disorder that causes progressive, life-threatening liver disease. Patients have impaired bile flow, or cholestasis, caused by genetic mutations. The resulting bile build-up in liver cells causes liver disease and symptoms. The most prominent and problematic ongoing manifestation of the disease is pruritus, or intense itching, which often results in a severely diminished quality of life. Other symptoms include jaundice, poor weight gain and slowed growth. In many cases, PFIC leads to cirrhosis and liver failure within the first 10 years of life, and nearly all people with PFIC require treatment before age 30. There are no drugs currently approved for PFIC, only surgical options that include partial external biliary diversion (PEBD) and liver transplantation. Additional information on PFIC is available at https://www.pficvoices.com.
Alagille syndrome (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 ALGS present with chronic cholestasis, usually within the first three months of life, and up to 88% also present with severe, intractable pruritus. Currently, there are no approved drug treatments.
About Biliary Atresia
Biliary atresia is a rare pediatric liver disease with symptoms typically developing about two to eight weeks after birth. Damaged or absent bile ducts outside the liver result in bile and bile acids being trapped inside the liver, quickly resulting in cirrhosis and even liver failure. Children have clay-colored or no color in their stools and jaundice, among other things, and a few patients are pruritic. Biliary atresia is the most common pediatric cholestatic liver disease and is the leading cause of liver transplants among children as there are no approved drug treatments.
Medison is one of the world’s largest commercial partners of leading global biotech companies. Medison is uniquely qualified to provide the complete spectrum of integrated services for biotech companies looking to enter or expand their presence in
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: the plans for, or progress, scope, cost, initiation, duration, enrollment, results or timing for availability of results of, development of odevixibat or any other Albireo product candidate or program; including expectations regarding the impact of the COVID-19 pandemic on our business and our ability to adapt our plans and activities as appropriate; the pivotal trial for odevixibat in biliary atresia (BOLD), and the pivotal trial for odevixibat in Alagille syndrome (ASSERT); the target indication(s) for development or approval, the size, design, population, location, conduct, cost, objective, enrollment, duration or endpoints of any clinical trial, or the timing for initiation or completion of or availability or reporting of results from any clinical trial, including the long-term open-label extension study for odevixibat in PFIC, the pivotal trial for odevixibat in biliary atresia, the pivotal trial for odevixibat in Alagille syndrome; the potential approval and commercialization of odevixibat; the potential for odevixibat to become the first approved drug for PFIC patients; discussions with the FDA or EMA regarding our programs; the potential benefits or competitive position of odevixibat or any other Albireo product candidate or program or the commercial opportunity in any target indication; the potential effects of odevixibat of the treatment of PFIC patients and its potential to improve the current standard of care; the potential benefits of an orphan drug designation; the potential issuance of a rare pediatric disease priority review voucher; 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 NDA for odevixibat for the treatment of pruritus in patients with PFIC will be approved by the FDA and whether the MAA for odevixibat in PFIC will be approved by the EMA; whether the FDA or EMA will complete their respective reviews within the target timelines, including the FDA’s PDUFA goal date, as a potential result of the impact of the COVID-19 pandemic or otherwise; the risk that the NDA will not be approved despite the FDA’s acceptance of the NDA for review; whether the FDA will require additional information, whether we will be able to provide in a timely manner any additional information that the FDA requests, and whether such additional information will be satisfactory to the FDA; other 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 odevixibat to date, including findings in indications other than PFIC, will be predictive of results from other clinical trials of odevixibat; whether either or both of the FDA and EMA will determine that the primary endpoint for their respective evaluations and treatment duration of the double-blind Phase 3 trial in patients with PFIC are sufficient to support approval of odevixibat in the United States or the European Union, to treat PFIC, a symptom of PFIC, a specific PFIC subtype(s) or otherwise; 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 odevixibat, including the pivotal program in biliary atresia or the pivotal program in Alagille syndrome, and the outcomes of such trials; Albireo’s ability to obtain coverage, pricing or reimbursement for approved products in the United States or European Union; delays or other challenges in the recruitment of patients for, or the conduct of, company’s clinical trials; and Albireo’s critical accounting policies. These and other risks and uncertainties that Albireo faces are described in greater detail under the heading “Risk Factors” in Albireo’s most recent Annual Report on Form 10-K or in subsequent filings that it makes with the Securities and Exchange Commission. As a result of risks and uncertainties that Albireo faces, the results or events indicated by any forward-looking statement may not occur. Albireo cautions you not to place undue reliance on any forward-looking statement. In addition, any forward-looking statement in this press release represents Albireo’s views only as of the date of this press release and should not be relied upon as representing its views as of any subsequent date. Albireo disclaims any obligation to update any forward-looking statement except as required by applicable law.
Hans Vitzthum, LifeSci Advisors, LLC., 617-430-7578
Source: Albireo Pharma, Inc.