DepYmed Receives FDA Orphan Drug Designation for Clinical Candidate DPM-1001 for the Treatment of Wilson Disease
FARMINGDALE, N.Y., May 23, 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, the Company announces that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation for its clinical candidate DPM-1001, a small molecule PTP1B inhibitor that also has high specificity and high affinity for binding copper and is therefore being studied in Wilson Disease.
Wilson Disease is a rare genetic disorder that prevents the body from removing copper, causing the metal to build up in the liver, brain, and corneas. Without treatment, high copper levels can cause life-threatening organ damage. Symptoms vary, but are often liver-related, including acute liver failure and/or cirrhosis, as well as symptoms related to the nervous system and mental health, and the formation of Kayser-Fleischer rings in the eyes. In early pre-clinical studies, orally available DPM-1001 has been shown to remove excess copper from the organs of mouse models with Wilson Disease, including the liver and the brain, as it also safely passes the blood/brain barrier in these preclinical models.
“The Orphan Drug designation for DPM-1001 is another important milestone for DepYmed in the development of its PTP1B inhibitor class of drugs designed to treat rare diseases. With an estimated occurrence of 1 in 30,000 people in the U.S., we see this as a significant opportunity to bring a powerful new approach to reducing copper overload in these patients to alleviate their symptoms and extend their lives. We look forward to discussing further progress in the development of DPM-1001,” said Andreas Grill, DepYmed’s President & CEO.
Protein tyrosine phosphatases are major players in the control of cell signaling pathways that are disrupted in many diseases; however, to date, success in development of drug modulators of these enzymes has been limited. 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.
About Orphan Drug Designation
Orphan Drug status is given to drugs and biologics defined as "those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of development.” These definitions, and the benefits that come with it, including significant tax credits for qualified clinical trials, exemption from user fees, and a potential of seven years of marketing exclusivity following FDA approval, were all part of the Orphan Drug Act of 1983.
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 these small molecule PTP1B inhibitors 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 the Tonks lab and Cold Spring Harbor Laboratory, to exploit their broad therapeutic potential. For more information, please visit the Company’s website at: www.depymed.com.
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