EMA validates MAA for novel telomerase inhibitor

The European Medicines Agency (EMA) has validated the Marketing Authorisation Application (MAA) for imetelstat, a first-in-class telomerase inhibitor as a treatment for transfusion-dependent anaemia in lower risk myelodysplastic syndromes (MDS).

Commenting on the validation of the MAA, Dr John Scarlett, Geron’s Chairman and Chief Executive Officer declared: “Based on the clinical profile of imetelstat to date, we are optimistic about its potential to become a standard of care and address longstanding unmet needs of lower risk MDS patients.”

Clinical evidence of efficacy

The MAA is based on results from IMerge Phase III. The trial’s primary endpoint of 8-week transfusion independence (TI) was significantly higher with imetelstat versus placebo, with median TI duration approaching twelve months for imetelstat 8-week TI responders, according to Geron.

Additionally, mean haemoglobin levels in imetelstat-treated patients increased significantly over time compared to placebo patients.

Data from non-clinical studies and clinical trials of imetelstat provide strong evidence that imetelstat targets telomerase to inhibit the uncontrolled proliferation of malignant stem and progenitor cells in myeloid hematologic malignancies resulting in malignant cell apoptosis and potential disease-modifying activity, the company stated.

In the Phase III portion of the IMerge Phase II/III study, the primary efficacy endpoint of IMerge Phase III is the rate of red blood cell transfusion independence (RBC-TI) lasting at least eight weeks. This is defined as the proportion of patients without any RBC transfusion for at least eight consecutive weeks since entry to the trial (8-week TI).

Imetelstat’s regulatory pathway to an MAA

Imetelstat has been granted Fast Track designation by the US Food and Drug Administration (FDA) for both adults with transfusion dependent anaemia due to Low or Intermediate-1 risk MDS that is not associated with del(5q) who are refractory or resistant to an erythropoiesis stimulating agent, and for adult patients with Intermediate-2 or High-risk myelofibrosis (MF) whose disease has relapsed after or is refractory to janus associated kinase (JAK) inhibitor treatment.

Review of the MAA for imetelstat is anticipated to be approximately 14 months.

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