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CAN1405 is a low-molecular-weight, small molecule allosteric agonist of the follicle-stimulating hormone receptor (FSHR). Developed primarily by TocopheRx, it is designed as an oral alternative to injectable recombinant follicle-stimulating hormone (FSH) for the treatment of infertility and premature ovarian insufficiency (POI). CAN1405 binds to an allosteric site within the transmembrane domain of the FSHR, where it acts as a positive allosteric modulator and agonist, triggering the G protein-coupled signaling pathway (primarily cAMP production). Research indicates that CAN1405 can rescue the signaling function of certain misfolded or mutated FSHR variants associated with POI, suggesting its potential as a targeted therapy for patients with specific genetic receptor defects.
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