RICHMOND, Calif., Nov. 30 /PRNewswire-FirstCall/ -- Sangamo BioSciences, Inc. today announced that it has completed subject enrollment and treatment in its Phase 1 study of SB-509 in subjects with mild to moderate diabetic neuropathy. SB-509 is a novel ZFP Therapeutic(TM) designed to upregulate the expression of the patient’s own vascular endothelial growth factor (VEGF) gene to protect and stimulate the regeneration of peripheral nerve function in diabetics suffering from peripheral neuropathy. Sangamo expects to announce results from this study in the first half of 2006 and to initiate a Phase 2 clinical study in the second half of 2006.
This single blind, placebo-controlled, single treatment, dose-escalation trial is designed to evaluate clinical safety of SB-509 in diabetics with mild to moderate diabetic peripheral sensory motor neuropathy in both legs. 12 subjects have been treated in the trial all of whom received treatment in one leg and placebo in the other. Either SB-509 or the placebo was administered by injection in a distribution that targets the major peripheral nerves in the legs and feet. Four dose levels of drug were tested. Safety will be monitored throughout the study, and visits at one, two, three and six months include neurological examination and electrophysiological testing.
“This is the first demonstration of the tolerability of ZFP Therapeutics in man. We are pleased to report that we did not observe any serious adverse events (SAEs) associated with treatment in this Phase 1 study,” said Dale Ando, M.D. Sangamo’s vice president, therapeutic development and chief medical officer. “The six month follow-up period of the study is ongoing and we intend to present the data from this trial in the first half of 2006. While the study is designed to evaluate safety and the maximal tolerated dose, its design enables us to compare endpoints in the treated and untreated leg and to compare these to baseline measurements. This may allow us to gain some preliminary data on the therapy’s effectiveness in improving subjects’ neurological functions and electrophysiological parameters. We have been able to recruit subjects to this study at a faster rate than anticipated and believe that this is a good indication of the willingness of physicians and patients to readily accept a new treatment option for this debilitating complication of diabetes. The current standard of care for these patients addresses only their symptoms with pain medications and antidepressants. In contrast, our ZFP Therapeutic has the potential to protect and stimulate the regeneration of peripheral nerves damaged by the build up of toxic metabolites in diabetes.”
“The completion of recruitment and treatment in our first clinical trial of a ZFP Therapeutic is a significant clinical development milestone for Sangamo,” said Edward Lanphier, Sangamo’s president and CEO. “Upon successful completion of the Phase 1 study follow-up, we will establish the tolerability of this product. Our goal is then to rapidly move this program into a randomized, placebo-controlled, Phase 2 study in the second half of 2006.”
About SB-509
SB-509 is administered as an injectable formulation of plasmid DNA that encodes a zinc finger DNA-binding protein transcription factor (ZFP TF(TM)), designed to upregulate the VEGF-A gene. VEGF-A has been demonstrated to have direct neurotrophic and neuroprotective properties. In preclinical animal efficacy studies in a diabetic rat model, SB-509 has proven effective in protecting motor and sensory nerve function from disease-induced nerve damage.
About Diabetic Neuropathy
Diabetic peripheral sensory motor neuropathy is one of the most frequent complications of diabetes. Symptoms include numbness, tingling sensations and pain particularly in the toes or feet. This is gradually replaced by loss of sensation and motor function as nerve damage progresses. Ulcers and sores may appear on numb areas of the foot because pressure or injury goes unnoticed. Despite adequate treatment, these areas of trauma frequently become infected and this infection may spread to the bone, necessitating amputation of the leg or foot. More than 60% of non-traumatic lower-limb amputations in the United States occur among people with diabetes. In the period from 2000 to 2001 this translated to approximately 82,000 amputations. The American Diabetes Association estimates that there are approximately 18.3 million people with diabetes in the United States and that of those about 60% to 70% have mild to severe forms of neuropathy. According to the CDC, diabetes is becoming more common in the United States. From 1980 through 2002, the number of Americans with diabetes more than doubled.
About Sangamo
Sangamo BioSciences, Inc. is focused on the research and development of novel DNA-binding proteins for therapeutic gene regulation and modification. The most advanced ZFP Therapeutic(TM) development programs are currently in Phase I clinical trials for evaluation of safety in patients with diabetic neuropathy and peripheral artery disease. Other therapeutic development programs are focused on macular degeneration, ischemic heart disease, congestive heart failure, neuropathic pain, and infectious and monogenic diseases. Sangamo’s core competencies enable the engineering of a class of DNA-binding proteins known as zinc finger DNA-binding proteins (ZFPs). By engineering ZFPs that recognize a specific DNA sequence Sangamo has created ZFP transcription factors (ZFP TF(TM)) that can control gene expression and, consequently, cell function. Sangamo is also developing sequence-specific ZFP Nucleases (ZFN(TM)) for therapeutic gene modification as a treatment for a variety of monogenic diseases, such as sickle cell anemia, and for infectious diseases, such as HIV. Sangamo has established several Enabling Technology Agreements with companies to apply its ZFP Technology to enhance the production of protein pharmaceuticals. For more information about Sangamo, visit the company’s web site at www.sangamo.com.
This press release may contain forward-looking statements based on Sangamo’s current expectations. These forward-looking statements include, without limitation, references to the clinical trials of SB-509, research and development of novel ZFP TFs and ZFNs and therapeutic applications of Sangamo’s ZFP technology platform. Actual results may differ materially from these forward-looking statements due to a number of factors, including uncertainties relating to the initiation and completion of stages of the SB-509 clinical trial, whether the SB-509 clinical trial will validate and support tolerability and efficacy of SB-509, technological challenges, Sangamo’s ability to develop commercially viable products and technological developments by our competitors. See the company’s SEC filings, and in particular, the risk factors described in the company’s Annual Report on Form 10-K and its most recent 10-Q. Sangamo BioSciences, Inc. assumes no obligation to update the forward-looking information contained in this press release.
Sangamo BioSciences, Inc.
CONTACT: Elizabeth Wolffe, Ph.D. of Sangamo BioSciences, Inc.,+1-510-970-6000, ext. 271, or ewolffe@sangamo.com; or media, Justin Jacksonof Burns McClellan, Inc., +1-212-213-0006, or investors, John Cummings,+1-415-352-6262, for Sangamo BioSciences, Inc.
Web site: http://www.sangamo.com//
