Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) has published groundbreaking findings from the CAMEO study in the journal Hepatology Communications, providing new insights into the safety, tolerability, and efficacy of LIVMARLI® (maralixibat) oral solution in patients with primary sclerosing cholangitis (PSC). These findings show promise in the potential of this treatment to provide relief for PSC patients.
In a groundbreaking study, the CAMEO study evaluated the safety and tolerability of LIVMARLI, an ileal bile acid transporter (IBAT) inhibitor, in adults with primary sclerosing cholangitis (PSC) who suffered from pruritus.
The open-label study followed 27 participants for 14 weeks, measuring pruritus, serum bile acid levels, and liver function. This was the first study of its kind to assess the potential of IBAT inhibitor therapy for PSC-related pruritus, offering hope to those suffering from this debilitating condition.
In this promising Phase 2 pilot study, participants with elevated serum bile acid levels at baseline were able to reduce them by 40%, while those with an average pruritus score of 3 or more out of 10 saw a whopping 70% improvement in pruritus.
LIVMARLI showed to be an effective treatment, with the most common treatment emergent adverse events (TEAEs) being related to the gastrointestinal system (diarrhea, nausea, and abdominal pain). Despite these minor side effects, the results indicate that this IBAT inhibitor is well-tolerated and may provide a safe and effective solution to pruritus.
Patients with Primary Sclerosing Cholangitis (PSC) often suffer from severe, chronic pruritus, affecting their quality of life and for which the treatment options are limited. However, the early data from the CAMEO study suggests that IBAT inhibitors may provide a potential relief for these patients. Led by Christopher Bowlus, MD, Chief of Gastroenterology and Hepatology at University of California, Davis, this study offers hope to those dealing with PSC and its symptoms.
Mirum is excited to announce the publication of the first data on the potential of IBAT inhibition for the treatment of patients with PSC. The CAMEO study with LIVMARLI has laid the groundwork for the upcoming Phase 2b VISTAS study with volixibat, a minimally absorbed IBAT inhibitor currently recruiting patients. Mirum is thankful for the clinicians and patients who made this research possible through their participation in the CAMEO study.
About Primary Sclerosing Cholangitis
Sclerosing cholangitis (PSC) is a rare and serious chronic liver disease that affects around 79,000 people in the US and Europe. The cause is unknown, but it is believed to be a combination of genetic and environmental factors. It is most commonly diagnosed in those aged around 35, and around 70% of those with PSC also have inflammatory bowel disease.
Symptoms include pruritus, fatigue, jaundice, and abdominal discomfort. Bile duct injury and obstruction can lead to complications such as cholangitis, ductal strictures, and gallstones, which may require endoscopic or surgical interventions.
The long-term risks include liver failure and a 25% chance of the PSC recurring after a liver transplant. Malignancies, specifically cholangiocarcinoma, are also more likely to develop in those with PSC.
About Volixibat
Volixibat is an exciting new oral therapy that could potentially revolutionize the treatment of adult cholestatic diseases. By blocking the ileal bile acid transporter (IBAT), Volixibat reduces bile acids both systemically and in the liver, leading to decreases in LDL cholesterol and increases in 7αC4, markers of bile acid synthesis.
Clinical trials involving over 400 individuals have demonstrated the efficacy of Volixibat, with the most common side effects being mild to moderate gastrointestinal issues. With its innovative approach, Volixibat could be a game-changer in the treatment of cholestatic diseases.
Volixibat is making groundbreaking progress in the treatment of primary sclerosing cholangitis and primary biliary cholangitis through its Phase 2b clinical trials, dubbed VISTAS and VANTAGE. These studies are providing hope to patients suffering from these debilitating diseases and promise to revolutionize the medical field.
About LIVMARLI® (maralixibat) oral solution
LIVMARLI® (maralixibat) oral solution, the only approved medication for treating cholestatic pruritus related to Alagille syndrome, has been approved by both the U.S. Food and Drug Administration and the European Commission.
This safe and effective treatment can be administered orally on a daily basis, and is suitable for patients aged 3 months and older in the US and 2 months and older in Europe. Make sure to consider LIVMARLI® if your child is suffering from cholestatic pruritus associated with Alagille syndrome.
Mirum has submitted LIVMARLI for approval in the U.S. and Europe for the treatment of cholestatic pruritus in PFIC patients. In the U.S., the medication is approved for patients aged three months and older, while in Europe, it is approved for patients aged two months and older.
LIVMARLI is making waves in the medical field with its late-stage clinical studies evaluating its potential to treat rare cholestatic liver diseases, including biliary atresia. In recognition of its groundbreaking research, the therapy has been granted Breakthrough Therapy designation for ALGS and PFIC type 2 and orphan designation for ALGS, PFIC and biliary atresia. It’s no wonder LIVMARLI has become a beacon of hope for those suffering from these diseases.