ELU001

ELU001 is a first-in-class, ultra-small nanoparticle drug conjugate (C’Dot Drug Conjugate or CDC) designed to target and penetrate solid tumors that overexpress folate receptor alpha (FRα). The CDC platform consists of a silica core surrounded by short polyethylene glycol chains, with approximately 13 folic acid molecules for targeting and about 20–22 cathepsin-B cleavable exatecan topoisomerase-1 inhibitor payloads covalently bound to the surface. Upon binding to FRα-expressing cancer cells, ELU001 is internalized into lysosomes where it releases its cytotoxic exatecan payload. Its small size (~6 nm) allows for deep tumor penetration and rapid renal elimination, which may reduce off-target toxicities compared to traditional antibody-drug conjugates (ADCs). Preclinical and early clinical data show activity in tumors with varying levels of FRα expression—including ovarian and endometrial cancers—and suggest a differentiated safety profile with minimal normal tissue toxicity. Developed by Elucida Oncology, ELU001 is currently being evaluated in Phase 1/2 trials for multiple solid tumor indications[1][2][3][4][5].

Other names

pasifolate exatecan