Ulcerative colitis (UC) is a chronic, idiopathic inflammatory disease that targets the colon. It is characterized by relapsing and remitting mucosal inflammation. UC inflammation is known to be associated with the presence of high levels of neutrophils and neutrophil biomarkers (e.g., calprotectin). The incidence and prevalence of UC has been increasing worldwide with a prevalence in the US estimated at >200 cases per 100,000 persons per year. Moderate to severe UC can be extremely debilitating to the affected patient, with a major impact on quality of life, overall health and predisposition to gastrointestinal cancer. Pharmacological treatments for moderate to severe UC include anti-inflammatory agents, immunosuppressants (e.g., azathioprine and 6-mercaptopurine), corticosteroids and immunomodulating biologics (e.g., Humira®, Entyvio®).. Although these treatments can be effective at inducing remission in patients, typical side-effects are significant, including increased risk of infection and malignancies, and tolerability. In addition, corticosteroids cannot be used long term, and newer biologics are only effective in about 20% of patients. Therefore, there is an unmet need for safer, more effective treatments.
Orally dosed BT051 is specifically targeted to modulate both neutrophil migration to the colonic lumen and their activation once there. The drug lacks general immunosuppressive activity and has been shown to have very low systemic exposure and a good safety profile in preclinical studies.