First Patient Enrolls for Primary Sclerosing Cholangitis Proof-of-Concept Study

According to a story from BioSpace, the biopharmaceutical company Immunic, Inc. has announced that the first patient has signed up for its proof-of-concept clinical study. This study is intended to test the effect of the company’s investigational drug candidate IMU-838 in patients with primary sclerosing cholangitis, a rare disease that affects the liver. With no approved medications that can improve patient survival, there is serious need for more effective treatments for this disease. Immunic is focused on the development of orally available treatments for autoimmune and inflammatory diseases.

About Primary Sclerosing Cholangitis (PSC)

Primary sclerosing cholangitis is a progressive, long term rare disease which affects the function of the liver and gallbladder. It is most characterized by the scarring and inflammation of the bile ducts, which are essential for allowing bile to exit the gallbladder. It can ultimately lead to cirrhosis and liver failure. It also increases the risk of several types of cancer. Although it is regarded by many as an autoimmune disease, it does not respond to typical immune system suppressants, so the precise cause of primary sclerosing cholangitis is poorly understood. Most patients also have some form of inflammatory bowel disease. Symptoms include itching, jaundice, portal hypertension, abdominal pain, malabsorption, hepatic encephalopathy, and dark urine. Treatment of primary sclerosing cholangitis is symptomatic and supportive, and there are currently no approved treatments specifically for the disease. To learn more about primary sclerosing cholangitis, click here.

About The Proof-of-Concept Study

Only a highly risky liver transplant can improve survival times in patients with this rare disease. This exploratory, open-label clinical trial will include 30 patients from ages 18-75 that have the disease. For a treatment period lasting six months, the participants will receive IMU-838 in a 30mg dose to be taken once per day. The primary endpoint measure in the study will be changes to serum alkaline phosphatase (ALP), a biomarker that has been previously used in other trials involving this illness.

About IMU-838

IMU-838 is designed as a selective modulator of immune system activity. The drug is intended to intervene in the activity of T cells and B cells while leaving the rest of the immune system unaffected, thereby hopefully reducing the risk of infection or other complications that normally appear as a result of immune suppression. 

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