Treatment will be given for 2 cycles of 28 days, for a total treatment of 2 months. At the end of each treatment cycle, there is an evaluation to see how the leukemia is responding to therapy. After completing two cycles, patients may enter into the Optional Treatment Continuation Period if the doctor believes that there is clinical benefit.~This is a Dose Escalation Safety Study to identify the maximum tolerated dose (MTD) and/or the recommended phase 2 dose (RP2D). This study will also evaluate the tolerability of ELU001. The drug is given by an infusion through a vein over a period of about one hour.~ELU001 is not a drug approved by the FDA (Food and Drug Administration) yet. But it is believed that ELU001 could play a role in stopping this type of leukemia from getting worse and offer a treatment option that may have less side effects. This is because it focuses on leukemic cells that have increased levels of FRα more than it targets healthy cells which express normal or no FRα.
This research study focuses on a rare type of acute myeloid leukemia (with the subtype CBFA2T3::GLIS2 that overexpresses folate receptor alpha (FRα) (a protein on the surface of leukemia cells)) that has relapsed or is refractory. Relapse means the cancer has come back after treatment. Refractory means the cancer does not respond to treatment.~ELU001 is a new chemical entity described as a C'Dot drug conjugate (CDC), consisting of payloads (exatecans) and targeting moieties (folic acid analogs) covalently bound by linkers to the C'Dot particle carrier. ELU001 will be the first drug-conjugate of its kind to be introduced into the clinic, a first in class, and a novel molecular entity.