IMerge is a Phase 2/3 clinical trial evaluating imetelstat as a treatment for patients with lower risk myelodysplastic syndromes (MDS)that are non-del(5q), dependent on red blood cell transfusion, and are relapsed after or refractory to treatment with erythropoiesis stimulating agents.

The primary efficacy endpoint of IMerge is 8-week red blood cell transfusion independence (RBC-TI) rate, defined as the proportion of patients not receiving any RBC transfusion during any consecutive eight weeks since entry into the trial.

This presentation reports long-term efficacy and safety data from 38 patients in the IMerge phase 2 clinical trial, based on a February 4, 2020 cut-off date and a
median follow-up of 24 months:

• 16 patients (42%) achieved 8-week RBC-TI, and 12 of these responders (75%) showed a haemoglobin rise of > 3 g/dL compared to pretreatment during the transfusion-free interval

• 12 patients (32%) achieved a 24-week RBC-TI

• 11 patients (29%) were transfusion-free for more than one year; the longest transfusion-free
interval was 2.7 years

• Median RBC-TI duration was 88 weeks, the longest reported to date in non-del(5q) LR MDS

• Haematologic improvement-erythroid was achieved by 26 patients (68%) with a median
duration of 93 weeks

• Durability of TI, cytogenetic and mutational malignant clone reduction in some patients
indicates potential disease-modifying activity of imetelstat

• Most frequently reported adverse events were manageable and reversible grade > 3
cytopenias

The phase 3 double-blind, placebo-controlled stage of IMerge is currently recruiting patients and is ongoing.

Source: EHA