Experimental Treatment for Immune Thrombocytopenia Begins Trials in China

According to a story from uk.finance.yahoo.com, the company Hutchison China MediTech Limited (“Chi-Med”) has recently kicked off a phase 1 clinical trial. This trial is testing the company’s investigational drug candidate HMPL-523 as a treatment for immune thrombocytopenia (ITP), a rare condition that can cause unexpected bleeding. The first patient was dosed on August 12th and is taking place in China.

About Immune Thrombocytopenia (ITP)

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.

About HMPL-523 And The Trial

HMPL-523, which is classified as an inhibitor of spleen tyrosine kinase (Syk), will be tested in 50 to 60 immune thrombocytopenia patients. Syk plays a critical role in B-cell signaling and prior research has suggested that it could be a useful treatment target in a variety of illnesses involving B-cells, such as various blood cancers and autoimmune diseases. The primary endpoint is how many patients experience an adverse event; phase 1 trials are primarily meant to test safety and tolerability with therapeutic effect being secondary. All patients in this trial will be adults. 

This trial is occurring alongside phase 1b trial of HMPL-523 in China and Australia. These trials are testing the drug as a treatment for a number of rare blood cancers, such as acute myeloid leukemia, Waldenstrom’s macroglobulinemia, chronic lymphocytic leukemia, diffuse large B-cell lymphoma, mantle cell lymphoma, and more.


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