Potential new options for those living with primary biliary cholangitis (PBC)

(BPT) - Do you have primary biliary cholangitis or know somebody who does? If so, you know that people living with PBC, a chronic, serious and potentially life-threatening liver disease, are frequently challenged by symptoms of their disease, including pruritus (itching) and fatigue,1 which may be debilitating for some patients.

PBC patients have an increased risk of liver cancer and liver-related mortality as the disease progresses, and may eventually need liver transplantation.2,3,4 Many people with PBC don’t reach their treatment goals or cannot tolerate their current treatment. Alternative treatment options are still needed.

A clinical research study called RESPONSE is evaluating the investigational drug seladelpar for people with PBC who have not achieved their recommended treatment goals or cannot tolerate their current treatment. Seladelpar has not been approved by the United States Food and Drug Administration and clinical trials are ongoing.

RESPONSE: A clinical research study of an investigational drug for PBC

RESPONSE is a phase 3 clinical research study of seladelpar, an investigational drug for people already diagnosed with PBC who have been taking ursodeoxycholic acid (also known as UDCA or ursodiol) and don’t have adequate results or cannot tolerate UDCA. In this study, the goal is to find out if seladelpar can improve PBC-related blood laboratory tests and can improve symptoms of PBC, such as itching.

You may be able to participate in RESPONSE if you meet the following requirements:

  • You are an adult 18 to 75 years old (inclusive)
  • You have been diagnosed with PBC
  • You have been on a stable and recommended dose of Ursodeoxycholic acid (also known as UDCA) for the past 12 months or don’t have adequate results or cannot tolerate UDCA
  • You have blood laboratory test results in the following range: alkaline phosphatase (ALP) levels at least 1.67 times the upper limit of normal (ULN) AND bilirubin less than two times the ULN

If you qualify for and participate in RESPONSE, you will receive seladelpar (the investigational drug being evaluated in this study), or placebo, and UDCA (if applicable). You will also receive study-related care from a study doctor with experience treating people with PBC, and study-related medical tests.

The RESPONSE study will also ask participants who enter the study to consider a biopsy at baseline and end of treatment. The biopsy is voluntary and performed only if it is considered safe by the study doctor. All study medication and study-related care will be at no cost to participants in RESPONSE.

Seladelpar: An investigational drug with the potential to address unmet need in PBC

Seladelpar is a novel drug candidate closely related to other FDA approved drugs such as fenofibrate (lipid lowering drug) and pioglitazone (type 2 diabetes drug) in development for the potential treatment of people with PBC. It controls metabolic and inflammatory pathways in the liver, muscle, fat, and intestinal tissues and the data supports the potential for seladelpar to improve bile production and secretion, the key mechanism involved in cholestastic pathology in PBC.

To date, more than 500 individuals have received seladelpar in clinical trials, with some being exposed to seladelpar beyond two years. Seladelpar has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) and Priority Medicine Designation from the European Medicines Agency (EMA) because it has the potential to serve key unmet needs for people with PBC. Data from Preclinical, Phase 2 and Phase 3 clinical studies provide support for the potential ability of seladelpar to decrease bile acid synthesis and inflammation, which are two of the central abnormalities observed in PBC.

For more information on the study, please visit PBCStudies.com.

1 National Institute of Diabetes and Digestive and Kidney Diseases. Symptoms & Causes of Primary Biliary Cholangitis. Updated March 2017. Available at National Institute of Diabetes and Digestive and Kidney Diseases. Definition & Facts of Primary Biliary Cholangitis. Updated March 2017. Available at https://www.niddk.nih.gov/health-information/liver-disease/primary-biliary-cholangitis/definition-facts. Accessed June 4, 2019.

2 Kaplan MM, Gershwin ME. Primary Biliary Cirrhosis. N Engl J Med. 2005;353(12):1261-1273.

3 Kumagi T, Heathcote EJ. Primary biliary cirrhosis. Orphanet J Rare Dis. 2008;3:1-17.

4 Lindor KD. Primary biliary cirrhosis. Hepatology. 2009;50(1):291-308.