DepYmed Presents Development Update for its Small Molecule PTP1B Inhibitor Program, DPM-1003, at the 2023 IRSF Rett Syndrome Scientific Meeting

Data were presented to demonstrate that DPM-1003 provides a mechanism-based approach to the treatment of Rett Syndrome and is poised to enter clinical development in 2024

FARMINGDALE, N.Y., June 15, 2023 (GLOBE NEWSWIRE) — DepYmed, Inc. (“DepYmed” or the “Company”) is a preclinical stage pharmaceutical company focused on developing transformative treatments for rare diseases and oncology that target the protein tyrosine phosphatase (PTP) family of enzymes. Today DepYmed announces the presentation of a development update for its small molecule PTP1B inhibitor program and its lead clinical candidate, DPM-1003, at the 2023 International Rett Syndrome Foundation (IRSF) Rett Syndrome Scientific Meeting.

The Company presented a poster outlining the development of DPM-1003 and Dr. Nicholas Tonks, DepYmed’s founding scientist, delivered a podium presentation demonstrating the potential of this new treatment of Rett Syndrome. The conference was held June 5-7, 2023 at the Nashville Marriott at Vanderbilt University in Nashville, Tenn.

The data illustrated the Company’s mechanism-based approach and provided an overview of DPM-1003 preclinical efficacy and safety moving the compound through IND-enabling studies to initiate clinical studies in 2024. The data demonstrate that DPM-1003 is a potent, specific inhibitor of PTP1B and a potential once-daily orally administered drug to treat Rett Syndrome.

“We believe these data, which were presented for the first time in a public forum, validate our continued development of DPM-1003 toward treating Rett Syndrome,” said Andreas Grill, DepYmed’s President and CEO. “We are diligently continuing our IND-enabling studies and intend to enter clinical development under an IND submission to the FDA this year. We will continue to provide relevant updates on our progress.”

About IRSF

In 1983, a small, dedicated group of parents whose children had Rett syndrome formed the first non-profit to focus exclusively on Rett syndrome – the International Rett Syndrome Association (IRSA). In 2007, IRSA and Rett Syndrome Research Foundation (RSRF) consolidated resources to better serve families and maximize research investments toward a cure. The International Rett Syndrome Foundation (IRSF) emerged with a mission to accelerate research and empower families that builds upon these foundations’ pioneering work toward care and cure. empower families with information, knowledge, and connectivity. Today, the International Rett Syndrome Foundation continues its almost 40-year commitment to creating a world without Rett. It remains focused on accelerating research to treat and cure Rett syndrome as it seeks to empower families with information, knowledge, and connectivity.

About DPM-1003

Protein tyrosine phosphatases are major players in the control of cell signaling pathways that are disrupted in many diseases, yet no drug modulators of these enzymes have been successfully developed to date. DepYmed is the first company to develop a new class of orally bioavailable drug candidates that act by inhibiting PTP1B, one of the most important PTP drug targets. One of its lead compounds has shown promising efficacy in preclinical models of Rett syndrome, and the Company hopes to initiate a Phase 1 clinical trial in 2024. DPM-1003 has been granted Orphan Drug designation by the U.S. Food and Drug Administration and conditionally designated under the FDA’s Rare Pediatric Disease program.

About Rett Syndrome

Rett syndrome is a progressive neurodevelopmental disorder that almost exclusively affects females. Infants with Rett syndrome generally develop normally for 6 to 18 months after birth. At this point, they lose previously acquired skills (developmental regression) such as purposeful hand movements and the ability to communicate. Affected children often develop autistic-like behaviors, breathing irregularities, feeding and swallowing difficulties, growth retardation, and seizures. Most Rett syndrome cases are caused by mutations of the MECP2 gene on the X chromosome and can present with a wide range of disability ranging from mild to severe. The expression of PTP1B, DepYmed’s target in Rett syndrome, is regulated by MECP2; in turn, PTP1B controls important cell functions that are disrupted in the disease.

About DepYmed, Inc.

DepYmed Inc., is a New York based rare disease and cancer therapeutic development company that was founded to capitalize on the scientific discoveries of the Tonks lab at Cold Spring Harbor Laboratory in the physiological function of PTP1B and ways to modulate its role in various human diseases. DepYmed is currently developing a new class of potent, orally bioavailable small molecule inhibitors of the enzyme PTP1B as novel therapeutics for Rett Syndrome and different types of cancer. In addition, DepYmed has also discovered a novel class of small molecules with copper chelating properties that it is developing as potential therapeutic agents for such diseases as Wilson disease and various cancers. The company is actively developing a deep pipeline of new compounds with broad therapeutic potential in these emerging drug classes in collaboration with Cold Spring Harbor Laboratory. For more information, please visit the Company’s website at:

For Media Inquiries please contact:

Jules Abraham
JQA Partners, Inc.


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