Compelling and Differentiating Phase 2 Data Provide Strong Evidence of Disease Modification
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 hematologic myeloid malignancies, potentially resulting in meaningful clinical benefits for patients. Data reported from our Phase 2 clinical trial in lower risk MDS provide evidence that imetelstat may achieve meaningful and durable transfusion independence and increase in hemoglobin levels, suggesting potential recovery of normal blood cells. Similarly, data reported from our Phase 2 clinical trial in myelofibrosis, or MF, suggest imetelstat potentially improves overall survival, or OS, for MF patients who have relapsed after or are refractory to prior treatment with a janus kinase, or JAK, inhibitor, or relapsed/refractory MF. Additionally, from these Phase 2 clinical trials, we have 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, all of which we believe provides evidence of disease-modifying activity. Furthermore, these molecular and histology data have been correlated with the clinical benefits of transfusion independence in lower risk MDS and improved OS in relapsed/refractory MF. We believe the clinical benefits, molecular observations and correlations from these two Phase 2 trials 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. We believe this disease-modifying activity has the potential to differentiate imetelstat from other currently approved and investigational treatments for MDS and MF.