Rigel Pharmaceuticals, Inc. announced a peer-reviewed publication of data in Blood Advances, which summarizes clinical results from the Phase 2 registrational study of REZLIDHIA™, a potent, selective, oral, small-molecule inhibitor of mutant isocitrate dehydrogenase 1 , in patients with mIDH1 relapsed or refractory acute myeloid leukemia. 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.
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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 (CR) plus complete remission with partial hematologic recovery (CRh). CRh is defined as less than 5% blasts in the bone marrow, no evidence of disease, and partial recovery of peripheral blood counts (platelets >50,000/microliter and absolute neutrophil count >500/microliter).
Key findings from the trial are summarized below:
- REZLIDHIA demonstrated a 35% (51/147) CR+CRh rate in mIDH1 R/R AML patients. Of the patients who achieved the primary endpoint of CR+CRh, 92% (47/51) 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% (71/147). Response rates were similar in patients who had and who had not received prior venetoclax.
- In patients treated with REZLIDHIA, the median duration of CR+CRh was 25.9 months and the median duration of CR was 28.1 months. The median duration of overall response was 11.7 months.
- Median overall survival (OS) was 11.6 months in the overall population of 153 patients. In patients who achieved CR+CRh, median OS was not reached and the estimated 18-month survival was 78%.
- Conversion to transfusion independence (TI), another recognized indicator of clinical benefit, was achieved in 34% of patients receiving REZLIDHIA that had been classified as dependent on RBC and/or platelet transfusions at baseline. TI was observed in patients in all response groups.
SOURCE: PR Newswire