Takeda Reports Positive Topline Results from Phase 2 Study in ITP

Rare Daily Staff

Takeda reported positive topline results from a phase 2, randomized, double-blind, placebo-controlled study evaluating the safety, tolerability and efficacy of mezagitamab in patients with persistent or chronic primary immune thrombocytopenia.

Primary immune thrombocytopenia (ITP) is a rare, IgG mediated autoimmune disease caused, in part, by the development of autoantibodies to platelets, which are blood cells responsible for preventing or stopping bleeding. ITP is characterized by the accelerated destruction of platelets, resulting in a decreased platelet count and an increased risk of bleeding, which can be debilitating, and in severe cases may be life-threatening.

Mezagitamab (TAK-079) is a fully human immunoglobulin IgG1 monoclonal antibody with high affinity for CD38 expressing cells resulting in their depletion and is designed to deliver rapid and sustained improvement in platelet response and generally rapidly restores platelet counts to functional levels.

The TAK-079-1004 trial evaluated three different doses of subcutaneous mezagitamab vs. placebo, given once weekly for eight weeks in patients with chronic or persistent primary ITP. An interim analysis of the ongoing phase 2 study demonstrated positive safety and efficacy results.

Mezagitamab has been generally safe and well tolerated across all three cohorts. All mezagitamab doses tested demonstrated a higher platelet response rate than placebo. The increases in platelet count were dose-dependent with the greatest platelet response observed at the highest dose tested. Platelet response in mezagitamab treated patients occurred rapidly and was maintained post-therapy.

Based on these positive results, and following consultation with global health authorities, Takeda plans to initiate a global phase 3 trial of mezagitamab in ITP in fiscal year 2024.

“These phase 2 results demonstrate mezagitamab’s compelling disease modifying mechanism of action, which has the potential to achieve disease remission for people with ITP,” said Chinwe Ukomadu, head of the Gastrointestinal & Inflammation Therapeutic Area Unit at Takeda.