Member Spotlight: Q&A with Albireo Pharma

Aug 01, 2019

Every month, MassBio spotlights a member company and the great work they’re doing to advance the life sciences industry and support the patients we serve. In August, we spoke with Ron Cooper, President and Chief Executive Officer of Albireo Pharma. Ron is a seasoned biopharma leader with a track record of growing businesses, brands and organizations in the U.S. and Europe. Before joining Albireo in 2015, Ron worked in five different countries and held positions of increasing responsibility in sales, marketing and general management with Bristol-Myers Squibb, culminating in his role as President of Europe.

Tell us about your organization, its mission, and current initiatives.

Albireo is focused on addressing unmet needs in the treatment of liver diseases. We do this by leveraging our bile acid modulation technology to develop ileal bile acid transporter (IBAT) inhibitors. The lead product candidate, odevixibat, is an IBAT inhibitor currently being evaluated in a Phase 3 program in its lead indication, progressive familial intrahepatic cholestasis (PFIC). Odevixibat could be the first medicine approved to treat this rare and life-threatening pediatric liver disease. We’re also working to realize odevixibat’s potential in other rare pediatric cholestatic liver diseases. We plan to initiate a trial in biliary atresia and explore other indications in 2020. At the same time, we’re expanding the potential of our IBAT platform to address a large unmet need in nonalcoholic steatohepatitis (NASH).

How does your organization’s activities help patients now and into the future?

Imagine your child scratching herself so incessantly throughout the night that she wakes with blood-stained sheets. She has a life-threatening liver disease, and it is getting worse. This is what the parents of children with PFIC experience. For them, Albireo represents hope. We are committed to advancing odevixibat, which has the potential to become the first approved drug therapy for this devastating disease for which there are no pharmacologic therapies. In addition to moving forward with the PEDFIC 1 clinical trial, which has over 40 sites recruiting worldwide, we have collaborated with patient organizations to support their work in community building and raising awareness through education. To this end, we launched “PFIC Voices,” a multimedia effort to increase understanding of PFIC together with the PFIC Resource & Advocacy Network (

What do you see as the biggest challenge facing the life sciences industry today?

For rare disease drug companies, a challenge—and an opportunity—exists in formalizing the patient voice in drug development. While many are already working closely with patient groups to better understand rare disease patient populations and address their unique needs, integrating these insights and context about the patient journey should be a key part of the process for identifying and evaluating a therapy’s ability to offer meaningful benefit to patients.

Another challenge is bridging the gap between innovation and the patient. While the pace of breakthroughs in our industry has accelerated, the unmet medical needs remain critical in rare disease and in other areas. Society demands more and better healthcare but must reconcile the cost with willingness to devote the resources needed to develop new treatment options and cures.

What’s next for your organization / what are you focused on in the coming year?

This is an exciting time to be at Albireo. From a clinical perspective, we are moving towards completion of PEDFIC 1, the Phase 3 trial of odevixibat. We’re actively discussing studying odevixibat in other rare pediatric liver diseases and expect to begin a trial in one of the largest rare pediatric cholestatic liver diseases, biliary atresia. We also have multiple programs underway to help realize the potential of our bile acid modulation science in NASH. Simultaneously, we’re preparing for growth and the potential commercial launch of odevixibat. We’re looking to grow our talented team in Boston and beyond. We are even relocating to a larger office space to accommodate this growth.

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