REC-102

REC-102 is an orally available, small molecule ENPP1 inhibitor, being developed for the treatment of hypophosphatasia (HPP). HPP is a rare, potentially life-threatening genetic disease, characterized by impaired mineralization of bones and teeth. Inhibiting ENPP1 reduces inorganic pyrophosphate (PPi) levels which may restore the PPi and phosphate balance needed to promote bone mineralization. With our collaborators, we have shown that ENPP1 inhibition is safe and well-tolerated by preclinical models, and for the first time demonstrated that ENPP1 is a druggable target for later-onset HPP.