Investigational Compound for Primary Sclerosing Cholangitis; Mechanism Suggests Potential to Target Multiple Inflammatory and Fibrotic Diseases

Acorda has indirectly obtained global development and commercialization rights to BTT1023 (timolumab) as part of our acquisition of Biotie Therapies in 2016.

Although the mechanism of action is not fully elucidated, BTT1023 is a fully human monoclonal antibody that targets VAP-1 (vascular adhesion protein-1). Biotie conducted multiple preclinical and clinical studies suggesting VAP-1 may play a role in inflammatory and fibrotic diseases. Inflammatory diseases, such as rheumatoid arthritis, inflammatory bowel disease and psoriasis, are the result of the immune system mistakenly attacking the body’s own cells and tissue. Fibrotic diseases, such as primary sclerosing cholangitis (PSC) and cirrhosis of the liver, are characterized by the formation of scar tissue that prevents normal function of the body’s organs.

BTT1023 is currently in Phase 2 development as a treatment for PSC. PSC is a rare, chronic disease that damages the bile ducts over time. Bile fluid is an essential aid to digestion that is made in the liver. In people with PSC, the bile ducts become blocked due to inflammation and scarring (fibrosis). This causes bile to accumulate in the liver, where it gradually damages liver cells and eventually results in liver failure. There are no approved drug therapies for PSC and liver transplant is the only treatment.

The Phase 2 study is being funded through the Efficacy and Mechanism Evaluation (EME) Program, a Medical Research Council and National Institute for Health Research partnership in the United Kingdom. Interim data from this clinical trial are expected in the second half of 2017.

BTT1023 has been designated as an orphan drug for PSC by the United States Food and Drug Administration (FDA) and the European Medicines Agency (EMA).

The safety and efficacy of BTT1023 has also been explored in early-stage clinical trials in rheumatoid arthritis and psoriasis. 

Acorda is exploring strategic partnerships to further develop this therapy.