DepYmed Announces Validation of Trodusquemine as a Therapeutic Candidate for HER2-positive Breast Cancer

NEW YORK, May 27, 2014 (GLOBE NEWSWIRE) -- DepYmed Inc., a joint venture of Ohr Pharmaceutical, Inc. (Nasdaq:OHRP) and Cold Spring Harbor Laboratory, today announced the validation of Trodusquemine (MSI-1436) as a therapeutic candidate for HER2-positive breast cancer. Trodusquemine is the Company's inhibitor of the enzyme PTP1B (protein tyrosine phosphatase 1B). The results were published online on May 20, 2014 in Nature Chemical Biology.

HER2-positive breast cancer is a breast cancer that tests positive for a protein called human epidermal growth factor receptor 2 (HER2), which promotes the growth of cancer cells. In approximately 1 out of every 4 breast cancers, tumor cells make an excess of HER2 due to a gene amplification. HER2-positive breast cancers tend to be aggressive and the prognosis for patients is poor. The drug Herceptin® (trastuzumab) is a first-line treatment for many women with HER2-positive breast cancer, but in most cases resistance develops within a year. It is anticipated that alternative therapies which act either alone or in combination with Herceptin may enhance patient outcomes.

In a paper entitled "Targeting the Disordered C Terminus of PTP1B With an Allosteric Inhibitor" a multi-institution team led by Professor Nicholas Tonks of Cold Spring Harbor Laboratory reports that it has found a means of inhibiting PTP1B, expression of which is upregulated in HER2-positive breast cancer. They provide compelling evidence of PTB1B as a therapeutic target in HER2-positive breast cancer.

PTB1B is a well-characterized target for several major diseases, including diabetes and obesity. However, it has been a challenging target for therapeutic development due to the chemical properties of the enzyme at its active site. "Novel approaches are required to exploit this important target fully," Dr. Tonks observes. In the approach reported in the newly published paper, Trodusquemine has been identified as a selective, allosteric inhibitor of PTP1B that exerts its effects outside the active site of the enzyme.

Dr. Tonks and his team tested Trodusquemine in several mouse models of HER2-dependent breast cancer. They showed that in animals treated with Trodusquemine, there was extensive inhibition of tumor burden and prevention of metastasis to the lung. They also demonstrated that it was inhibition of PTP1B that antagonized signaling by HER2 proteins in the treated animals.

"We are pleased with the publication of this important paper on Trodusquemine in breast cancer," said Dr. Irach Taraporewala, President and Chief Executive Officer of Ohr Pharmaceutical. "These results build on data from earlier studies showing PTP1B to be a tumor promoter and suggest it may be a viable therapeutic target in HER2-positive breast cancer. This form of breast cancer is very aggressive and difficult to treat. Many women ultimately build up a resistance to current treatments, so it is important to identify additional therapeutic agents for intervention."

In other studies, Trodusquemine has been shown to cross the blood-brain barrier, which enhances its potential applications. It has been well tolerated in dose-escalation clinical studies completed to date in over 65 patients. Novel analogs of Trodusquemine have also been identified that are potent inhibitors of PTP1B and have potential delivery route advantages.

A Phase I clinical trial evaluating Trodusquemine in HER2-positive breast cancer patients is planned for later this year and will be conducted at North Shore-Long Island Jewish Hospital.

About DepYmed, Inc.

DepYmed is a privately owned joint venture between Ohr Pharmaceutical, Inc. and Cold Spring Harbor Laboratory, established to conduct research and development on Trodusquemine and its analogs. DepYmed plans to take Trodusquemine into the clinic and demonstrate proof of concept. The continued research is being conducted under the direction of Dr. Nicholas Tonks at CSHL. Dr. Tonks discovered PTP1B, the primary target for Trodusquemine.

About Ohr Pharmaceutical, Inc.

Ohr Pharmaceutical Inc. (Nasdaq:OHRP) is a research and development company with a primary focus in ophthalmology. The Company's lead product, Squalamine, is currently being studied as an eye drop formulation in several company sponsored and investigator sponsored Phase 2 clinical trials for various back-of-the-eye diseases, including the wet form of age related macular degeneration, retinal vein occlusion, diabetic macular edema, and proliferative diabetic retinopathy. Additional information on the Company can be found at found at

About Cold Spring Harbor Laboratory

Cold Spring Harbor Laboratory (CSHL) is a private, not-for-profit research and education institution at the forefront of molecular biology and genetics. Founded in 1890, CSHL has shaped contemporary biomedical research and education with programs in cancer, neuroscience, plant biology and quantitative biology. CSHL is ranked number one in the world by Thomson Reuters for the impact of its research in molecular biology and genetics. The Laboratory has been home to eight Nobel Prize winners. Today, CSHL's multidisciplinary scientific community is more than 600 researchers and technicians strong and its Meetings & Courses Program hosts more than 12,000 scientists from around the world each year to its Long Island campus and its China center. For more information, visit

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CONTACT: Ohr Pharmaceutical Inc. Investor Relations (877) 215-4813 LifeSci Advisors, LLC Andrew McDonald 646-597-6987 andrew@lifesciadvisors.comSource:Ohr Pharmaceutical, Inc.