Compelling and Differentiating Phase 2 Data Provide Strong Evidence of Disease Modification
Over 500 patients have been enrolled and treated in Phase 1 and 2 clinical trials of imetelstat. We believe that data from two prior Phase 2 clinical trials provide strong evidence that imetelstat targets telomerase to inhibit the uncontrolled proliferation of malignant stem and progenitor cells in myeloid hematologic malignancies, potentially resulting in meaningful clinical benefits for patients.
Data reported from our Phase 2 clinical trial in lower risk myelodysplastic syndromes (MDS) demonstrated that imetelstat has the potential to become a beneficial therapy for patients by providing meaningful and durable transfusion independence and increases in hemoglobin levels. Similarly, data reported from our Phase 2 clinical trial in myelofibrosis (MF) suggest imetelstat potentially improves overall survival (OS) for MF patients who have relapsed after or are refractory to prior treatment with a janus associated kinase (JAK) inhibitor, also known as relapsed/refractory MF.
We believe the observed disease-modifying activity of imetelstat has the potential to differentiate it from other currently approved and investigational treatments for MDS and MF. Importantly in these Phase 2 clinical trials, we observed depletion of cytogenetic abnormalities and reductions in key driver mutations of the underlying diseases in both lower risk MDS and MF patients, as well as improvement in bone marrow fibrosis in MF patients. Also, the molecular and histology data are correlated with the clinical benefits of transfusion independence in lower risk MDS and improved OS in relapsed/refractory MF. These data highlight the magnitude of imetelstat’s unique mechanism of action of telomerase inhibition, and provide strong evidence that imetelstat may alter the course of MDS and MF.