Sangamo BioSciences initiates two new clinical trials of ZFP Therapeutics

Jan 11 2010

Sangamo BioSciences, Inc. (Nasdaq: SGMO) announced the initiation of two new clinical trials of ZFP Therapeutics, a Phase 2b study in diabetic neuropathy (DN) and a Phase 1 trial in glioblastoma, as well as the renewal of $3.0 million in funding for the Phase 2b trial by the Juvenile Diabetes Research Foundation International (JDRF). Edward Lanphier, Sangamo's president and CEO, will provide an update on the company's ZFP Therapeutic(TM) pipeline and an overview of the company's business strategy and objectives for 2010 during his presentation at the 28th Annual J.P. Morgan Healthcare Conference at 2:30 pm PT, on Wednesday, January 13, 2010.

"We enter 2010 with a strong balance sheet and a robust clinical pipeline," said Mr. Lanphier. "With the initiation of these two new clinical trials, we continue to increase the value, maturity and visibility of our novel drug development platform."

"We are developing our lead therapeutic, SB-509, for neurological indications including DN and ALS. The data generated to date in DN, our most advanced program, provide direct histologic evidence of nerve regrowth with SB-509 treatment and a mechanistic proof of concept for its neuroregenerative effects. Our enthusiasm for the potential and importance of this drug is shared by JDRF who, after extensive review of the clinical data, has committed to fund a further $3.0 million of development costs for this new Phase 2b trial. While we continue active discussions with potential corporate partners, timely initiation of this trial enables us to maximize the future value of this program. Finally, the Phase 1 glioblastoma trial is our third ZFP Therapeutic and the second zinc finger nuclease (ZFN)-based program to enter the clinic and we are pleased to have achieved this significant milestone."

"Neuropathy is a significant complication of diabetes and represents a major unmet medical need, so developing better treatments would be a tremendous near-term benefit for people with diabetes," stated Alan Lewis, Ph.D., President and Chief Executive Officer of JDRF. "JDRF is excited to move this partnership forward, particularly as it involves a drug candidate designed to harness the body's own regenerative potential to address the nerve loss characteristic of neuropathy."

The Phase 1 glioblastoma trial will be initiated by Sangamo's collaborators at City of Hope and is designed to evaluate the safety and tolerability of a modified CD8+ cytotoxic T lymphocyte (CTL) product that has been made resistant to glucocorticoid steroids using Sangamo's ZFP nuclease ZFN-based technology. The study will accrue subjects with recurrent/refractory malignant glioblastoma multiforme (GBM). The US Food and Drug Administration (FDA) has reviewed and accepted an Investigational New Drug (IND) application to initiate this open-label, multi-dosing Phase 1 clinical trial.

"We look forward to initiating this study to evaluate a promising therapeutic approach for the treatment of malignant brain tumors for which there are currently few effective therapeutic options after surgery," said Michael C.V. Jensen, M.D., Associate Chair, Department of Cancer Immunotherapeutics and Tumor Immunology, City of Hope. "In collaboration with Sangamo, we have successfully generated engineered T-cells that can destroy glioblastoma tumor cells in animals in the presence of glucocorticoids. Treatment of recurrent GBM with modified CTLs, an otherwise promising approach for this cancer, is rendered ineffective by the use of glucocorticoids to control inflammation of the brain due to the tumor and surgery. The ZFN-modification of the glucocorticoid receptor (GR) in our engineered CTLs protects the cells from the effects of steroids, which would normally inhibit T-cell function, but does not alter their cytolytic or tumor-killing properties."

"Our significant clinical, commercial and financial progress in 2009 provides us with a solid foundation upon which to build in 2010," commented Mr. Lanphier. "In the next twelve months, we expect to have final data from two Phase 2 clinical trials of SB-509, one in severe DN (SB-509-701) and one in ALS (SB-509-801). In addition, we expect to have preliminary data from our two ongoing Phase 1 trials of our ZFN-based Therapeutic, SB-728-T for the treatment of HIV/AIDS. We look forward to advancing our clinical pipeline as well as participating in the successful commercial expansion of our ZFP technology through our partnerships in plant agriculture and in the growing area of cell line engineering and transgenic animal model production."