Date: Feb 19, 2013 Author: Synergene Therapeutics, Inc. Source: Company Data (
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Potomac, Md., Feb. 19, 2013 -- SynerGene Therapeutics, Inc. (SGT) today announced results of a Phase Ia clinical trial evaluating the safety profile of SGT-53, which are published in Molecular Therapy. SGT-53 is an investigational intravenously administered, tumor-targeting nanoparticle which delivers the human tumor suppressor gene p53. P53, known as "Guardian of the Genome", is missing or non-functional in almost all human tumors. The Phase Ia trial of SGT-53 was designed to reactivate the tumor suppressor gene in cancer patients.
The trial was conducted through collaboration with John Nemunaitis, M.D., and Neil Senzer, M.D., at Mary Crowley Cancer Research Centers (MCCRC) in Dallas, TX. The first 11 patients with various types of advanced solid tumors were treated beginning in 2008. Minimal drug-related side effects were observed and, at the end of treatment, eight of the eleven patients showed stable disease, with a median survival time of 340 days. Results showed not only that the systemically delivered SGT-53 was well tolerated and exhibited anti-cancer activity, but also supplied evidence of targeted delivery of SGT-53 to metastatic cancer and not to normal cells.
"Results of this trial are a remarkable first step towards solving our issues of targeted drug delivery", Dr. Nemunaitis, the Principal Investigator of the trial, said. "Clinical and molecular evidence of activity was demonstrated justifying further clinical investigation."
"Tumor specificity is very crucial to a cancer therapy's efficacy", said Esther H. Chang, Ph.D., senior consultant for SynerGene and professor at Georgetown University Medical Center (GUMC). "Because we have a targeting moiety, the nanocomplex will travel through the bloodstream and whenever it encounters a tumor cell -- whether a primary tumor or metastasis -- the nanoparticle will bind to it, enter the tumor cell and destroy it." These agents are intended to work alongside and augment standard therapies. Preclinical data has shown significant tumor reduction when SGT-53 is used in combination with standard treatments such as chemotherapy, as the newly restored p53 tumor suppressor sensitizes the tumor cells to the killing effects of standard therapies, resulting in enhanced survival due to tumor growth inhibition and tumor regression.
SGT-53 is the prototype of SynerGene's delivery platform technology, which was developed by a research team led by Esther H. Chang, Ph.D. and Kathleen F. Pirollo, Ph.D., a research professor at GUMC. This tiny, simple structure, measuring a millionth of an inch across, has only three components (a protein as the targeting moiety, a liposome formulation which functions as an envelope for the therapeutic gene, and the therapeutic gene, which in the case of SGT-53 is the tumor suppressor gene p53), which allow it to penetrate into the tumor and deliver therapeutics specifically into cancer cells.
About SynerGene Therapeutics, Inc.
SynerGene Therapeutics, Inc. is a biopharmaceutical company that is developing targeted diagnostics and therapeutics in the areas of cancer, vaccine delivery, and neurological disorders. SynerGene's proprietary nanocomplex actively targets receptors present on diseased cells, delivering therapeutic drugs specifically to targeted sites while avoiding healthy cells. The company's nanocomplex is able to deliver numerous molecular medicines, including plasmid DNA, siRNA/miRNA, AS ODN, small molecules, chemotherapy drugs, and imaging agents. SynerGene has two products in human clinical trials. SGT-53 is currently being tested in a Phase Ib trial at MCCRC in combination with chemotherapeutic agent docetaxel in patients with various solid tumors. A new Phase Ib/II trial testing SGT-53 in combination with gemcitabine in pancreatic cancer is expected to begin shortly. SGT-94 is testing the delivery of another tumor suppressor gene, RB94, in a Phase I trial in patients with genitourinary tumors.
