Rigel announces publication summarizing results from Phase 2 study of REZLIDHIA

Rigel Pharmaceuticals announced a peer-reviewed publication of data in Blood Advances, which summarizes clinical results from the Phase 2 registrational study of REZLIDHIA, or olutasidenib, a potent, selective, oral, small-molecule inhibitor of mutant isocitrate dehydrogenase 1, or mIDH1, in patients with mIDH1 relapsed or refractory acute myeloid leukemia (R/R AML). The published data demonstrate that REZLIDHIA induced durable remissions and transfusion independence with a well-characterized safety profile. The observed efficacy is clinically meaningful and represents a therapeutic advance in this poor prognosis patient population with limited treatment options. The pivotal cohort of the Phase 2 registrational study enrolled 153 adult patients with mIDH1 R/R AML who received REZLIDHIA monotherapy 150 mg twice daily orally. The efficacy evaluable population included 147 patients with centrally confirmed mIDH1. The primary endpoint was a composite of complete remission, or CR, plus complete remission with partial hematologic recovery, or CRh. REZLIDHIA demonstrated a 35% CR+CRh rate in mIDH1 R/R AML patients. Of the patients who achieved the primary endpoint of CR+CRh, 92% were CR. Most patients who achieved CR or CRh responded early, with a median time to response of 1.9 months. The overall response rate was 48%. Response rates were similar in patients who had and who had not received prior venetoclax.