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DepYmed Granted FDA Clearance to Commence Phase 1 Clinical Trial of DPM-1003 for Rett Syndrome Treatment

Wednesday, March 20, 2024

DepYmed, Inc. ("DepYmed") is a pharmaceutical company dedicated to developing medications for various diseases, with a focus on targeting the protein tyrosine phosphatase (PTP) family of enzymes. Today, DepYmed announces that the U.S. Food and Drug Administration has cleared its phase 1 clinical trial for DPM-1003, intended for treating Rett Syndrome.

Andreas Grill, President and CEO of DepYmed, stated, "This achievement is pivotal for DepYmed, marking our transition into a clinical-stage company with a potentially transformative drug candidate poised to offer substantial benefits to patients. As the first of its kind, this Investigational New Drug (IND) clearance paves the way for the development of crucial new therapies across a spectrum of diseases where targeting PTP enzymes shows promise, including Rett Syndrome, cancer, metabolic disorders, obesity, and Alzheimer's Disease. We eagerly anticipate assessing the safety and tolerability of DPM-1003 and advancing its development for Rett Syndrome and our other ongoing initiatives."

Protein tyrosine phosphatases play crucial roles in regulating cell signaling pathways implicated in various diseases. However, no successful drug modulators targeting these enzymes have been developed to date. DepYmed is pioneering a new class of orally bioavailable drug candidates that inhibit PTP1B, a significant PTP drug target. Encouragingly, one of its lead compounds has demonstrated promising efficacy in preclinical models of Rett Syndrome, paving the way for a phase 1 clinical trial slated for 2024. DPM-1003 has received Orphan Drug designation from the FDA and is conditionally designated under the Rare Pediatric Disease program.

Rett Syndrome is a progressive neurodevelopmental disorder predominantly affecting females. Typically, infants with Rett Syndrome exhibit normal development for the first 6 to 18 months after birth, followed by a regression in acquired skills such as purposeful hand movements and communication abilities. Affected children often display autistic-like behaviors, breathing irregularities, feeding difficulties, growth delays, and seizures. The majority of Rett Syndrome cases stem from mutations in the MECP2 gene on the X chromosome, resulting in a wide spectrum of disability severity. Notably, PTP1B, the target of DepYmed in Rett Syndrome, is regulated by MECP2, thereby controlling critical cellular functions disrupted in the disease.

 

Source: globenewswire.com

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