First-in-class Small Molecule Agonists of the Insulin-like (INSL3) Peptide Receptor RXFP2 and Uses in Bone Disorders and Fertility

Recent studies have identified the G-protein-coupled receptor (GPCR) for insulin-like 3 peptide (INSL3), relaxin family peptide receptor 2 (RXFP2), as an attractive target for the treatment of bone diseases such as osteoporosis and rare bone diseases such as osteogenesis imperfecta. Currently, the most effective available treatment for osteoporosis is an expensive hormone therapy that requires daily injections. A stable, orally deliverable drug is a much more desirable alternative. Our RXFP2 agonists perform as well as the natural ligand INSL3 in cellular assays.

NCATS in collaboration with Florida International University (FIU) has identified low molecular weight, highly potent, and efficient full RXFP2 agonists with low cytotoxicity. The identification and characterization of these compounds may lead to the development of a new class of cost-effective drugs for the treatment of osteoporosis and other diseases associated with bone loss.

NCATS is actively seeking licensing and/or co-development research collaborations for the treatment of osteoporosis and other diseases associated with bone loss as well as bone development disorders such as rickets and osteomalacia.