Benzinga | Feb 11, 2021 08:40AM EST

Albireo Pharma To Outline Commercial Plans For Odevixibat In Thursday Presentation At 11:30 a.m. EST

Albireo Pharma, Inc. (NASDAQ:ALBO), a clinical-stage rare liver disease company developing novel bile acid modulators, is hosting a commercial deep dive today at 11:30 a.m. EST. The presentation will detail the Company's corporate potential, market opportunity in rare pediatric and adult liver disease and the global commercialization and launch readiness plans for odevixibat in progressive familial intrahepatic cholestasis (PFIC), an ultra-rare pediatric cholestatic liver disease.

"We are delighted that both the EMA and FDA confirmed acceptance of our odevixibat filings in the U.S. and Europe with priority and accelerated reviews," said Ron Cooper, President and Chief Executive Officer of Albireo. "Odevixibat's demonstrated profile in the largest ever clinical program of PFIC, coupled with our strong commercial strategy and ongoing development programs, positions Albireo to seize the large global cholestatic liver disease market."

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 U.S. and EU. Odevixibat holds the promise to expand to multiple rare pediatric cholestatic indications to drive company growth. Albireo is in a strong financial position for commercialization, sufficient to fund odevixibat launches, advance clinical studies and support the Company into revenue generation.

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 China and India) have approximately 100,000 patients living with rare cholestatic liver diseases. Albireo has initiated or completed three gold standard Phase 3 studies in PFIC, biliary atresia and Alagille syndrome and these studies should be sufficient for approval in multiple geographies around the world.

Patient Access

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 Pamela Stephenson, Chief Commercial Officer of Albireo. "We are building a commercial team that is dedicated to delivering seamless access to patients and HCPs and delivering good value for payors and HCPs."

Until odevixibat is approved for commercial sale, the Company provides an Expanded Access Program (EAP) for eligible patients with PFIC in the U.S., Europe, Canada and Australia. Physicians and patients can learn more about Albireo's odevixibat EAP at albireopharma.com/patients-families/expanded-access-policy.

Launch Ready

Albireo has all key leadership in place to support rapid launches in the U.S. and abroad. The Company recently appointed Kevin Springman as the President of the Americas and Steve Arnold as the President of International. These leaders are finalizing specialized U.S. and international teams to have in place to execute the launch of odevixibat. Springman was appointed as the President of Americas after a successful track record in sales, marketing and market access roles at Sobi and before that at AstraZeneca. Arnold has extensive experience building and leading international teams, most recently at Intercept, and prior to that UCB and Gilead.

As part of the ex-U.S. commercial strategy, Albireo entered into its first of multiple ex-U.S. commercial distributorships with Medison Pharma, Ltd. ("Medison") for odevixibat in Israel. Medison is a leading international commercial partner for highly innovative therapies. Under the agreement, Medison will be responsible for approval and commercialization in Israel in close alignment and with oversight from Albireo.

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 $101 million, cash at the end of 2020 $251 million, and planned operating cash burn in 2021 $120-$130 million. 2021 revenue from odevixibat is anticipated to be in the low single digit US $ millions.

Conference Call

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.

When: Today, February 11, 2021, from 11:30am-1:00pm EST

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