Reported by Researchers at Cold Spring Harbor Laboratory

June 29, 2018 (Farmingdale, NY) — DepYmed, Inc. in collaboration with Cold Spring Harbor Laboratory (CSHL), has reported promising preclinical experiments on a compound, DPM-1001, currently being developed by DepYmed, Inc. for the potential treatment of patients with Wilson’s Disease as well as other disorders—including certain types of cancer. The new research, performed at CSHL, was published June 26, 2018 in the journal, Genes & Development.

DepYmed continues to conduct the preclinical development requirements that are necessary to bring DPM-1001 to clinical trials and hopes have this completed in the near future.

“We look forward to bringing our portfolio of novel molecules into clinical development with the goal of improving patient lives,” says Andreas Grill, President & CEO of DepYmed, Inc. “DepYmed seeks to develop novel compounds for the treatment of cancers and rare diseases including Wilson’s Disease with the continued collaboration with Cold Spring Harbor Laboratory.”


Wilson’s disease is a genetic disorder in which copper builds up in the body. Symptoms are typically related to the brain and liver. Liver-related symptoms include vomiting, weakness, fluid build-up in the abdomen, swelling of the legs, yellowish skin, and itchiness. Brain-related symptoms include tremors, muscle stiffness, trouble speaking, personality changes, anxiety, and seeing or hearing things that others do not.

Wilson’s disease is an autosomal recessive condition due to a mutation in the Wilson disease protein (ATP7B) gene. For a person to be affected, they must inherit an affected copy of the gene from each parent. Diagnosis may be difficult and often involves a combination of blood tests, urine tests, and a liver biopsy. Genetic testing may be used to screen family members of those affected.

Current medications used to treat Wilson’s Disease include chelating agents such as trientine and d-penicillamine and zinc supplements. Chelating agents enable the body to absorb and naturally excrete copper, which is toxic in excess.  Complications of Wilson’s disease can include liver failure, liver cancer, and kidney problems. A liver transplant may be helpful in those in whom other treatments are not effective or if liver failure occurs.

Wilson’s disease occurs in about 1 in 30,000 people. Symptoms usually begin between the ages of 5 and 35 years. Males and females are equally affected. It was first described in 1854 by Friedrich Theodor von Frerichs and is named after Samuel Wilson.


Krishnan, N et al, “DPM-1001 decreased copper levels and ameliorated deficits in a mouse model of Wilson’s disease” is published June 26, 2018 in Genes & Development.


DepYmed Inc., is a New York-based cancer and rare disease therapeutic development company. DepYmed is developing potent inhibitors of the enzyme PTP1B (protein tyrosine phosphatase 1B) with its current lead compound, MSI-1436C, in clinical development as a therapeutic candidate for HER2-positive breast cancer. In addition to PTP1B inhibitors, DepYmed has developed a portfolio of small molecules with copper chelating properties which can be applied as potential therapeutic agents for multiple diseases. DepYmed continues to develop the next generation of more potent PTP1B inhibitors / analogs as well as selective copper chelators which are currently in preclinical development in collaboration with Cold Spring Harbor Laboratory and continues to explore additional indications that may benefit from using PTP1B inhibitors and selective copper chelators as therapeutic agents.


Founded in 1890, Cold Spring Harbor Laboratory has shaped contemporary biomedical research and education with programs in cancer, neuroscience, plant biology and quantitative biology. Home to eight Nobel Prize winners, the private, not-for-profit Laboratory employs 1,100 people including 600 scientists, students and technicians. The Meetings & Courses Program annually hosts more than 12,000 scientists. The Laboratory’s education arm also includes an academic publishing house, a graduate school and the DNA Learning Center with programs for middle and high school students and teachers. For more information, visit

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