According to a story from ucb.com, the biopharmaceutical company UCB has recently released the final results from its phase 2 clinical trial which tested the company’s investigational monoclonal antibody rozanolixizumab as a treatment for primary immune thrombocytopenia (ITP). The results of the study support the continued development of the drug for this indication and was first presented that American Society of Hematology’s 61st Annual Meeting & Exposition, which took place in Orlando, FL.

About Immune Thrombocytopenia

Immune thrombocytopenia (ITP) describes a condition if abnormally low platelet count in bone marrow that is otherwise normal without any recognizable external cause. Platelets play an essential role in forming blood clots in order to halt wound bleeding. Immune thrombocytopenia is known as an autoimmune disease, in which the immune system mistakenly attacks parts of the body; antibodies that target surface antigens found on platelets are the telltale sign. Symptoms of the condition include purpura (small purplish skin rashes), excessive menstrual bleeding in women, bleeding from the gums and/or nose, small bruises called petechiae, and blood masses (hematomas) on mucosal surfaces. In severe cases of the condition, very low platelet counts can lead to potentially lethal internal bleeding. Treatment, which is usually only recommended when severe bleeding occurs, may include steroids, IVIg, surgical removal of the spleen, and platelet transfusion. To learn more about immune thrombocytopenia, click here.

Trial Findings

The trial included a total of 66 patients with the condition. Patients were either administered a single, larger dose of the drug per week (15 to 20 mg/kg) or multiple smaller doses that added up to the same overall number. The patients saw both reductions in signs of IgG and improved platelet counts. The best results were achieved in patients who took the larger, single doses. These patients responded at a rate of 55 to 67 percent. This group also responded to the drug more quickly than the other group. These results suggest that a single, larger dose is more likely to be effective.

Adverse effects in the study were consistent with previous observations, with headaches being the most commonly reported. Overall, immune thrombocytopenia patients should be encouraged by the findings from the trial. Rozanolixizumab is also being developed as a treatment for chronic inflammatory demyelinating polyneuropathy (CIDP) and myasthenia gravis (MG).