'No small feat': Albireo CEO talks biotech's commercial transition amid Bylvay launch

Established in Sweden as a small spinout of AstraZeneca in 2008, Albireo always had big ambitions. After all, it was named after the striking “double star” in the Cygnus constellation, which has defied astronomers who have never determined if its two stars are gravitationally bound.

Until recently, Albireo was a struggling biotech—twice on the verge of bankruptcy—and staffed by a “couple handfuls of individuals,” as described by its CEO Ron Cooper in a recent interview.

But that changed with Albireo’s approval of Bylvay, a drug that treats liver disease. The treatment was sanctioned in both the United States and Europe—four days apart last July—for progressive familial intrahepatic cholestasis (PFIC), forcing Albireo to make more than a few changes.

“For a small company that’s been an R&D company for a long time, to transition to a commercial company is no small feat,” Cooper said. “We have an approved product and the opportunity to take Bylvay into other indications. Now we’re at about 140 individuals.”

So far, the transition to a commercial company is going smoothly, Cooper said. Bylvay generated sales of $7 million last year—nearly double what the company initially projected—with $5.3 million coming in the U.S. and $1.7 million internationally. There are 93 patients currently prescribed and more than 100 are in long-term studies or early access programs.

Albireo is handling commercial activities in the U.S. and Europe. In other territories, it has hired third-party marketers such as Jadiete (Japan), Medison Pharma (Israel) and Swixx Biopharma (Central and Eastern Europe). Other third-party companies are handling the commercial end in Saudi Arabia, Turkey and several other countries in the Middle East.

Bylvay’s uptake should be quick and relatively uncomplicated, the company figures. It is the lone drug prescribed for PFIC, the diagnosis of the condition is straightforward and the network of specialists who treat children with cholestatic liver disease is small and firmly established in Europe and the U.S.

“We only have 10 sales reps in the U.S., so that makes it a pretty efficient and attractive model,” Cooper said. “These diseases were identified some 25 years ago. They’ve been using a patchwork to manage these patients, so this is a huge change in the standard of care for children with PFIC. Getting the word out to them and helping them understand that is kind of our job now.”

In addition to chief commercial officer, Pamela Stephenson, Albireo will have commercial presidents for the U.S. and Europe. U.S. chief Mary Ann Keyser, brings 22 years of experience from Sanofi.

Upon Bylvay’s approval, Evaluate projected its sales to reach $300 million by 2026. For its part, Albireo sees Bylvay as a billion dollar drug, eying two more indications.

A phase 3 study of Bylvay in another pediatric liver disease, Alagille syndrome, will read out sometime this year. An approval there would double its potential market. Albireo estimates there are 25,000 people around the world with Alagille syndrome and another 25,000 with PFIC.

Albireo also is enrolling patients in a phase 3 study of Bylvay against another pediatric liver disorder, biliary atresia. If all goes according to plan, that trial would read out in 2024. There are approximately 50,000 in the world with biliary atresia.

“The really strong results of Bylvay and PFIC translate really well to other diseases like Alagille and biliary atresia,” Cooper said. “We believe Bylvay could be a blockbuster product—over a billion dollars in the second half of the decade. We get there by making Bylvay, not into a PFIC drug, but into a pediatric cholestatic liver disease drug.”

While Bylvay is the lone drug that treats PFIC, there is competition on the horizon. Last September, the FDA blessed Mirum Pharmaceuticals’ Livmarli as the first treatment for Alagille syndrome. Mirum also is testing the drug in PFIC and biliary atresia.

Livmarli is from the same ileal bile acid transporter (iBAT) inhibitor drug class as Bylvay. Both treatments have been shown to control pruritis, the unrelenting itch that accompanies the two diseases. Yet to be determined is whether Livmarli and Bylvay are disease modifying.

With annual price tags of $391,000 and $386,000 respectively, Livmarli and Bylvay aren’t differentiated by cost. Mirum reported sales of $3.1 million for Livmarli in the final quarter of 2021.

As Albireo proceeds with its launch, Cooper said the company is in strong financial shape with the help of financial incentives provided through the Orphan Drug Act, including a priority review voucher which it sold for $105 million.

“That’s allowed us to invest in these other phase 3 studies but it has also allowed us to keep our balance sheet strong," Cooper said. "We have sufficient cash to execute, based on our current plans, until 2024.”