Drug intelligence / Profile preview

abequolixron

Development stage
Phase 1
Lead developer
Inspirna
Modality
Small Molecules, Vaccines & Immunotherapeutics
Administration
Oral
01

Overview

Abequolixron is an orally bioavailable, first-in-class small molecule agonist of the nuclear receptor liver X receptor beta (LXRβ; NR1H2). It is designed to activate expression of the APOE tumor suppressor protein, which becomes dysregulated in certain solid tumors. By targeting LXRβ, abequolixron enhances anti-tumor immunity through several mechanisms: it inhibits tumor angiogenesis, depletes myeloid-derived suppressor cells (MDSCs), stimulates dendritic cells (DCs), and activates cytotoxic T lymphocytes (CTLs). These effects collectively counteract immune suppression within the tumor microenvironment and promote anti-tumor responses. Abequolixron is being developed primarily for advanced solid tumors including non-small cell lung cancer (NSCLC), small cell lung cancer (SCLC), lymphoma, neuroendocrine tumors, endometrial carcinoma, and other malignancies. The drug has orphan designation for malignant melanoma, glioblastoma, and ovarian cancer[1][3][4][5][6][7].

Brand names
RGX-104RGX104RGX 104
Other names
abequolixron zincRGX 104RGX104RGX-104RGX-104 ZincRGX104 ZincRGX 104 Zinc
02

Targets

LXRB (Liver X receptor beta)

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