In a recent news release, commercial-stage biopharmaceutical company HUTCHMED shared that its investigational therapy HMPL-523 had received Breakthrough Therapy designation from the National Medical Products Administration (NMPA) in China. HMPL-523 was designed to treat chronic primary immune thrombocytopenia, or immune thrombocytopenic purpura (ITP), patients who have previously tried at least one treatment option. As many patients relapse following treatment, HMPL-523 has the potential to change the treatment realm and improve patient outcomes.
HMPL-523
Developed by HUTCHMED, HMPL-523 is an orally administered small molecule inhibitor. The therapy targets spleen tyrosine kinase (Syk), which has been found to play a role in various autoimmune diseases, B-cell cancers, and other conditions. Thus, by inhibiting Syk, researchers believe that HMPL-523 could confer benefits for those with ITP. Currently, HMPL-523 is being evaluated within a Phase 3 clinical trial. Throughout the trial, researchers seek to determine the treatment’s safety, efficacy, and tolerability.
HMPL-523 received Breakthrough Therapy designation following data from a Phase 1b clinical trial. If you are interested in learning more about this trial or its findings, take a look at the data shared by HUTCHMED at the ASH Annual Meeting. In China, Breakthrough Therapy designation is designed to expedite the development and review of investigational drugs and biologics. Breakthrough Therapy designation is granted to therapies designed for serious, rare, or life-threatening conditions. To receive this designation, the product must show advantages or benefits over the current standard(s)-of-care. Additionally, the status may be given to a therapeutic which fills an unmet need (i.e. there are no other treatments available).
About Immune Thrombocytopenic Purpura (ITP)
Immune thrombocytopenic purpura (ITP), or primary immune thrombocytopenia, is an autoimmune blood disorder characterized by low platelet counts. Normally, platelets play a role in blood vessel strength and function, as well as clotting. In a healthy individual, normal platelet counts are around 150,000-450,000. However, in ITP, the immune system mistakenly attacks platelets, causing widespread platelet destruction. As a result, the platelet count dramatically decreases (to under 100,000), causing an increased risk of abnormal bleeding. ITP may be acute (typically impacting children and resolving within weeks) or chronic (symptoms lasting 6 months to an entire lifetime). Symptoms and characteristics can include:
- Easy bruising and bleeding
- Debilitating fatigue
- Blood in urine, vomit, or stool
- Frequent or recurrent nosebleeds
- Blood blisters on the insides of the cheeks
- Petechiae (broken blood vessels that cause small, red dots under the skin)
- Heavy and prolonged menstruation
- Low energy
- Intracranial hemorrhage
