Each week, in an email to his team, Albireo Pharma CEO Ron Cooper sends out the story of a child or family affected by conditions such as PFIC or Alagille syndrome that the company has been focused upon. To Ron, this is a crucial part of embracing Albireo’s patient-centric attitude – and ensuring that care for the patient remains a mainstay of the company mission. He is particularly committed to taking this approach within the rare disease sphere, sharing:

 “Rare diseases are often lonely. A lot of research focuses on bigger diseases. But we believe that those with rare diseases should get the same level of care as high cholesterol or high blood pressure. Our goal is to make an impact and giving people hope is unbelievably important and so rewarding.”

Recently, Ron sat down with the Patient Worthy team to discuss new updates and data shared at the AASLD – The Liver Meeting, the challenges of rare disease drug development, and how Albireo upholds its patient-centricity in all that it does.

About Ron Cooper

Ron Cooper is a life sciences leader with over 25 years of experience in rejuvenating and growing brands, businesses, and organizations in both the United States and Europe. As Ron explains:

 “I grew up in big pharma – Bristol Myers Squibb – and worked in various roles around the world. I took part in country management, sales, marketing, and general management, where I was responsible for 30 different countries. Through my role, I also played a part in launching a number of different products such as Abilify, Orencia, and Yervoy.”

In June 2015, Ron took on his new role as the first President and CEO of Albireo Pharma, Inc., a clinical-stage company focused on developing therapeutics for orphan pediatric liver diseases as well as other gastrointestinal and liver conditions.

 “At Albireo, I saw tremendous potential. Albireo has really elegant, well-developed science and people who are passionate about making an impact on patients. To me, that’s what it takes to succeed in this realm: good science and good scientists. Staying true to the science and believing that we’d positively affect patients has been a north star for our company.”

Learn more about Ron Cooper here.

AASLD – The Liver Meeting

The American Association for the Study of Liver Diseases (AASLD)’s The Liver Meeting took place from November 4-8, 2022. This meeting brings stakeholders in the hepatology field – clinicians, scientists, and others with an interest in this realm – together to discuss new trends, research, and development.

During the AASLD The Liver Meeting, Albireo shared some new updates and exciting data from its studies into Bylvay (odevixibat), the first FDA-approved treatment for pruritus (intense itching) in patients with progressive familial intrahepatic cholestasis (PFIC). Ron describes Bylvay as:

 “An ileal bile acid transport inhibitor, which is a fancy way of saying that it puts bile acids out in your stool. Bile acids are important for many things, but primarily for helping you to absorb nutrients and fat. At the end of the small intestine, 95% are transported back to the liver and 5% are transported out. For these children with PFIC, we want to get rid of excess bile acids that end up in the wrong place. So Bylvay blocks the reuptake, and more is removed through stool, which relieves cholestasis.”

For 25 years, there was not necessarily a good therapeutic option for children with cholestatic liver diseases. These diseases also cause liver damage; many children eventually need a liver transplant. Bylvay offers a potential transformative therapeutic option to improve outcomes in this patient population.

The Study Findings

In the first AASLD presentation, Albireo shared insights into the study evaluating Bylvay for PFIC. The original study highlighted the benefits of Bylvay in terms of bile acids, safety, efficacy, and tolerability:

In the longer-term analysis, the data showed evidence that Bylvay could improve native liver survival inpatients. These children were able to keep their original liver for a longer period of time due to Bylvay actually normalizing the mix of bile acids.

Outside of PFIC, Albireo is also exploring Bylvay as a therapeutic option for individuals with Alagille syndrome, an inherited disorder that can affect multiple parts of the body and causes bile buildup in the liver. The Liver Meeting was the first time at a medical conference that Albireo shared data from the Phase 3 ASSERT trial. Findings from the study showed that:

•  Bylvay was relatively safe and well-tolerated for those with Alagille syndrome.
• The treatment significantly improved pruritus (p=0.002) and reduced serum bile acid levels. It also helped improve sleep for those affected.
• While some side effects did occur – such as diarrhea, vomiting, abdominal pain, and liver test abnormalities – these were mostly mild and led to no trial discontinuation.

Future Research

Looking to the future, Ron and the Albireo team hope to continue their research with patients in mind. They believe that Albireo is at the start of a very exciting time in drug development with their lead product, Bylvay, which was the first FDA approved drug for the treatment of pruritus in all types of PFIC. It is also being developed to treat other rare pediatric cholestatic liver diseases with a completed Phase 3 trial in Alagille syndrome (ALGS), an ongoing Phase 3 study in biliary atresia. Moving forward, they also hope for an approval for Alagille syndrome in 2023. Bylvay is also being evaluated in a Phase 3 clinical trial for biliary atresia, with the anticipated read-out coming sometime in 2024.

In terms of adult diseases, Ron shares:

 “The beauty of our company is that we have so much expertise in bile acid modulation. We can apply this to multiple indications to really serve the rare disease community. In our pipeline, we also have two other drugs that we are very excited about. One will be moving forward for rare adult cholestatic diseases and one for rare adult viral diseases. We’re going to try to work as hard and as fast as possible to bring solutions to patients.”

Overcoming the Challenges of Rare Disease Drug Development

Of course, bringing solutions to patients requires a drug development process that, while rewarding, can also be challenging at times. Ron explains:

 “The challenge of rare disease is that you very rarely have a defined path. There is not much in the way of natural history, regulatory paths, or clear endpoints. When you’re designing trials in rare diseases, you really have to forge that path. At Albireo, we feel that you can only forge that path through collaboration.”

Therefore, to overcome these challenges, Albireo takes a patient-centric approach. As Ron says:

 “We’ve enjoyed great input and support from patients and advocacy organizations who have given us insight into the burden of the disease, what type of studies they would or would not enroll in, and meaningful endpoints. We have shared this with regulators and used those to design studies. In fact, one of the reasons why we are so able to successfully recruit for these studies is because of the great insights from the patient community.”

These studies include:

• The first Phase 3 study on Bylvay for PFIC, which showed improvements in growth (height/weight), sleeping, and itchiness. Bylvay was well-tolerated with little discontinuation.
• A Phase 3 study on Alagille syndrome which just read out in October 2022, which showed improvements in pruritus, bile acids, and sleep over time.
• An ongoing biliary atresia study which is considered the largest study ever done with an IBAT inhibitor. Over 200 patients are enrolled. The primary endpoint is survival with the liver they were born with.

In terms of the biliary atresia trial, Ron shares:

 “We designed a study and powered it for babies that weren’t even conceived yet. Insights from patients and advocacy groups helped us not just to recruit this study, but to really understand the needs of parents. These parents find out that their child isn’t growing and the next thing they know, surgery is needed. They ask about treatment but there is no approved treatment. We’re hopeful that we can change those dynamics.”