The USA-based company identifies novel cancer targets using its microRNA-based target discovery platform, RNA-DRIVEr.

Inspirna’s lead drug candidate, ompenaclid (RGX-202), is an orally-administered small molecule that targets the CKB/SLC6A8 pathway. Ompenaclid is being tested in multiple mid stage clinical trials in patients with advanced or metastatic RAS mutant colorectal cancer as of Q1 2024.

Inspirna's second clinical stage drug candidate, abequolixron (RGX-104), is an oral small molecule activator of LXR/APOE that inhibits angiogenesis and tumor myeloid derived suppressor cells to enhance the immune response against tumors. As of Q1 2024, abequolixron is being tested in a Phase Ib/II clinical trial in patients with advanced or metastatic lung cancer in combination with docetaxel and endometrial cancer in combination with ipilimumab through a clinical collaboration with Bristol Myers Squibb.