DepYmed Receives European Medicines Agency Orphan Drug Designation for Its Lead Clinical Candidate for the Treatment of Rett Syndrome

FARMINGDALE, N.Y., Nov. 16, 2022 (GLOBE NEWSWIRE) — DepYmed, Inc. (“DepYmed” or the “Company”) is a preclinical stage pharmaceutical company focused on developing transformative treatments for rare diseases and oncology based upon targeting the protein tyrosine phosphatase (PTP) family of enzymes. Today, DepYmed announces that the European Medicines Agency (EMA) has granted Orphan Drug Designation for its lead clinical candidate, a small molecule PTP1B inhibitor for the treatment of patients with Rett Syndrome (RTT).

“EMA’s decision to grant Orphan Drug Designation for our lead clinical candidate for the treatment of Rett Syndrome is another significant achievement for DepYmed and further validation among the world’s medical regulatory bodies of the potential for our compound,” said Andreas Grill, DepYmed’s President & CEO. “In addition, it continues to align with our goal to provide treatments worldwide for patients with conditions where few if any options exist. There continues to be an urgent need to develop a treatment for patients with Rett Syndrome, a devastating rare genetic neurological disorder that occurs primarily in girls, and we are pleased to be working closely with EMA, as well as the FDA in the U.S. to potentially address this need.”

Protein tyrosine phosphatases are major players in the control of cell signaling pathways that are disrupted in many diseases; however, to date, development of drug modulators of these enzymes has not been successful. 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 the second half of 2023.

About Orphan Drug Designation

Orphan Drug status is given to drugs and biologics in the European Union for conditions defined as rare diseases, which affects not more than 5 in 10,000 people in the EU, with about 60% of the drugs awarded this status in the EU being for pediatric use. Sponsors who obtain orphan designation benefit from protocol assistance, a type of scientific advice specific for designated orphan medicines, and market exclusivity once the medicine is on the market. Fee reductions are also available depending on the status of the sponsor and the type of service required.1 As previously announced, DepYmed has also been awarded Orphan Drug status in the US by the FDA, as well as being approved for consideration under the FDA’s Rare Pediatric Disease program if it successfully completes the development of its product candidate for Rett Syndrome.

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 PTP1B enzyme as potential 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 in these emerging drug classes, in collaboration with Cold Spring Harbor Laboratory, to exploit their broad therapeutic potential. For more information, please visit the Company’s website at:

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Jules Abraham
JQA Partners, Inc.



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