Geron is a late-stage clinical biopharmaceutical company focused on the development and potential commercialization of a first-in-class telomerase inhibitor, imetelstat, in hematologic myeloid malignancies.
Imetelstat is a novel, first-in-class telomerase inhibitor exclusively owned by Geron and being developed in hematologic myeloid malignancies. Early clinical data suggest imetelstat may have disease-modifying activity through the suppression of malignant progenitor cell clone proliferation, which allows potential recovery of normal hematopoiesis. Ongoing clinical studies of imetelstat include a Phase 2/3 trial called IMerge™ in lower risk myelodysplastic syndromes (MDS) and a Phase 2 trial called IMbark™ in Intermediate-2 to High-risk myelofibrosis. Imetelstat received Fast Track designation from the United States Food and Drug Administration for the treatment of patients with transfusion-dependent anemia due to lower risk non-del(5q) MDS who are refractory or resistant to an erythroid stimulating agent.
The role of telomerase and the potential implications for cancer have been a focus of Geron since our founding. The discovery and early development of imetelstat, our sole product candidate, was based on our core expertise in telomerase and telomere biology. Telomerase is an enzyme that enables cancer cells, including malignant progenitor cells, to maintain telomere length, which provides them with the capacity for limitless, uncontrolled proliferation. Using our proprietary nucleic acid chemistry, we designed imetelstat to be an oligonucleotide that targets and binds with high affinity to the active site of telomerase, thereby directly inhibiting telomerase activity and impeding malignant cell proliferation. Early clinical data, including molecular responses in essential thrombocythemia (ET) and remissions, including reversal of bone marrow fibrosis in myelofibrosis (MF), suggest imetelstat may have disease-modifying activity by inhibiting the proliferation of malignant progenitor cell clones for the underlying diseases.