Imetelstat sodium by Geron for Refractory Acute Myeloid Leukemia: Likelihood of Approval

GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.

Imetelstat sodium overview

Imetelstat (JNJ-63935937) is under development for the treatment of intermediate-2-risk or high-risk myelofibrosis, intermediate-1 or intermediate-2 risk myelodysplastic syndrome, acute myeloid leukemia multiple myeloma, myeloproliferative neoplasms and lymphoid hematologic malignancy. The drug candidate is administered by intravenous infusion. It is a new molecular entity (NME). It is a 13-mer oligonucleotide N3’–P5′ thio-phosphoramidate (NPS oligonucleotide) that is covalently attached to a C16 (palmitoyl) lipid moiety. GRN163L binds directly with high affinity to the template region of the RNA component of human telomerase (hTR), which lies in the active or catalytic site of hTERT, the telomerase reverse transcriptase. GRN163L binding to hTR results in direct, competitive inhibition of telomerase enzymatic activity. It is a new molecular entity.    

Imetelstat (GRN163L) was under development for the treatment of chronic lymphoproliferative disease including, small lymphocytic lymphoma (SLL), T cell prolymophocytic leukemia (T-PLL), B cell prolymphocytic leukemia (B-PLL), mantle cell lymphoma or other non-Hodgkins lymphoma in leukemic phase, Waldenstrom’s macroglobulinemia, multiple myeloma, post-essential thrombocythemia myelofibrosis, post-polycythemia vera myelofibrosis, non-small cell lung cancer, solid tumors, neuroblastoma, polycythemia vera and breast cancer.

Geron overview

Geron is a late-stage clinical biopharmaceutical company that focuses on the development and commercialization of imetelstat, a first-in-class telomerase inhibitor in hematologic myeloid malignancies. It’s clinical studies include IMerge, a Phase 2 trial in lower risk myelodysplastic syndromes; and IMbark, a Phase 2 clinical trial in intermediate or high-risk myelofibrosis. Imetelstat received Fast Track designation from the US FDA to treat patients with transfusion-dependent anemia due to lower or intermediate-risk myelodysplastic syndromes (MDS) and patients with intermediate or high-risk myelofibrosis (MF). The company utilizes core expertise in telomerase and telomere biology and its proprietary nucleic acid chemistry for the development of imetelstat. Geron is headquartered in Menlo Park, California, the US.

For a complete picture of Imetelstat sodium’s drug-specific PTSR and LoA scores, buy the report here.