Fostamatinib – ITP


Oral SYK Inhibitor for ITP:

  • Immune Thrombocytopenic Purpura (ITP) is an autoimmune disease where the immune system attacks and destroys platelets in the blood. The result - abnormally low platelet counts.
  • There are two forms of ITP: acute thrombocytopenic purpura, which is most commonly seen in young children; and chronic thrombocytopenic purpura, which requires continual follow up care with a hematologist.
  • ~50,000 - 60,000 people suffer from chronic ITP - the majority are women.
  • Rigel is focusing its Fostamatinib ITP program on the chronic form of this disorder targeting the underlying autoimmune cause of the disease rather than stimulating platelet production.
  • Antibodies, usually of the IgG type, mediate platelet destruction in ITP. Fostamatinib has a novel mechanism of action, blocking IgG receptor signaling in both macrophages and B cells via SYK kinase.
  • Rigel reported data from the first of two Phase 3 trials in August 2016; the second Phase 3 trail results are expected in October/November 2016.

PUBLICATION - Of mice and men: an open-label pilot study for treatment of immune thrombocytopenic purpura by an inhibitor of Syk

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