Geron reports ‘positive’ results from IMerge Phase 3 trial of imetelstat

Geron announced positive top-line results from its IMerge Phase 3 clinical trial evaluating the company’s first-in-class telomerase inhibitor, imetelstat, in lower risk myelodysplastic syndromes patients who are relapsed, refractory or ineligible for erythropoiesis stimulating agents. The trial met its primary efficacy endpoint of 8-week TI and a key secondary endpoint of 24-week TI, demonstrating highly statistically significant and clinically meaningful benefit of imetelstat versus placebo with no new safety signals and safety results consistent with prior imetelstat clinical trials. In light of the positive top-line results from IMerge Phase 3, combined with data from earlier clinical trials, the company plans to submit an NDA in the U.S. in mid-2023 and a Marketing Authorization Application in the EU in the second half of 2023. With Fast Track designation for imetelstat from the U.S. FDA for the treatment of adult patients with transfusion dependent anemia due to Low or Intermediate-1 risk MDS that is not associated with del(5q) who are refractory or resistant to an ESA, a request for rolling submission of the NDA was submitted and has been granted. Geron also plans to present additional data from IMerge Phase 3 at medical meetings later this year, including data relating to potential correlations of decreases in mutation burden and abnormal cytogenetic clones with clinical responses, patient reported outcomes, hTERT and telomerase activity biomarker data and continued follow-up of durability of transfusion independence, that may be indicative of the potential for disease modification with imetelstat. Geron is preparing for an anticipated commercial launch of imetelstat in lower risk MDS in the first half of 2024 in the U.S. and by the end of 2024 in the EU, assuming regulatory approvals are granted.