FDA Approves DepYmed's Phase 1 Trial for Rett Syndrome Drug DPM-1003

DepYmed Inc., a pharmaceutical firm specializing in the development of innovative treatments for a variety of diseases, has announced that it has obtained approval from the U.S. Food and Drug Administration (FDA) to begin phase 1 clinical trials for DPM-1003, a drug intended for the treatment of Rett Syndrome. The company, which is focused on targeting the protein tyrosine phosphatase (PTP) enzyme family, has achieved a significant milestone with this clearance, marking its transition to a clinical-stage entity.
Andreas Grill, CEO of DepYmed, highlighted the importance of this development, stating that it could pave the way for the creation of new treatments for a spectrum of diseases where PTP inhibition could be beneficial. These include Rett Syndrome, cancer, metabolic disorders, obesity, and Alzheimer's Disease. The company is eager to evaluate the safety and efficacy of DPM-1003 and to advance its development for Rett Syndrome and other ongoing projects.
DPM-1003 is a novel drug candidate that targets PTP1B, a key enzyme involved in the regulation of cell signaling pathways disrupted in numerous diseases. DepYmed has pioneered the development of orally bioavailable drugs that inhibit PTP1B, with one of its compounds demonstrating potential in preclinical Rett Syndrome models. The drug has received Orphan Drug status and conditional designation under the FDA's Rare Pediatric Disease program.
Rett Syndrome is a neurodevelopmental disorder predominantly affecting females, characterized by a regression in developmental skills and the onset of various symptoms such as autistic behaviors, breathing issues, and seizures. The syndrome is often linked to mutations in the MECP2 gene, which regulates PTP1B expression.
DepYmed, Inc., headquartered in New York, was established to leverage the scientific insights from the Tonks lab at Cold Spring Harbor Laboratory regarding PTP1B's role in human diseases. The company is developing a range of small molecule inhibitors targeting PTP1B for therapeutic use in Rett Syndrome, cancer, metabolic diseases, obesity, and Alzheimer's Disease. Additionally, DepYmed is working on a new class of copper-chelating molecules for diseases like Wilson disease and certain cancers, in collaboration with Cold Spring Harbor Laboratory. 

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