CR1-02 / Curamir Therapeutics - LARVOL DELTA

A phase I, open-label, multi-center dose-finding and expansion study to investigate the safety, tolerability, and preliminary efficacy of CR1-02 (5-FU-miR-15a) in patients with acute myeloid leukemia (AACR 2025) - "By modifying miR-15a with the cytotoxic pyrimidine 5-fluorouracil (5-FU), the 5-FU-miR-15a (CR1-02) miRNA has exhibited enhanced therapeutic efficacy, stability, as well as deliverability in multiple tumor types in pre-clinical testing.Aim: CR1-02 (EudraCT number: 2021-006332-46) is a phase I dose-escalation study evaluating the safety and tolerability of CR-001 in adults with relapsed/refractory (R/R) AML. A 3+3 study design was used with patients receiving dose levels of the synthetic double-stranded mimic of miR-15a (miRNA) (CR1-02) of 7.5 mg, 11.25 mg, 15 mg, and 18.75 mg/administration, weekly dosing...One patient (NRAS VAF 48%, ASXL1 41%, TP53 23%) responded with elimination of extramedullary pericardial AML disease after 4 doses of CR1-02 but progressed in bone marrow after 8+8 doses of CR-001 (eight study doses and an additional eight doses given under compassionate use approval from the Norwegian Medicines Agency) followed by trametinib-treatment as part of..."

Clinical • IO biomarker • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • ASXL1 • BCL2 • BMI1 • CHEK1 • MIR15A • NRAS • TP53