DPM-1003

DPM-1003 is an orally bioavailable small molecule inhibitor developed by DepYmed that targets protein tyrosine phosphatase 1B (PTP1B), a key enzyme involved in the regulation of cell signaling pathways. By inhibiting PTP1B, DPM-1003 aims to modulate disrupted signaling associated with several diseases. The drug has shown promising efficacy in preclinical models for Rett syndrome and is being developed as a potential once-daily oral therapy for this condition. In March 2024, the U.S. FDA cleared DepYmed to initiate a phase 1 clinical trial of DPM-1003 for Rett syndrome, and it has been granted Orphan Drug designation as well as conditional designation under the FDA’s Rare Pediatric Disease program[1][4][5]. Beyond Rett syndrome, targeting PTP enzymes like PTP1B may have therapeutic potential in cancer, metabolic disease and obesity, and Alzheimer’s disease[1].