Sangamo Announces Completion of Accrual and Expansion Plans for SB-509-701
Phase 2 Clinical Trial for Treatment of Moderate to Severe Diabetic
SAN FRANCISCO, June 6 /PRNewswire-FirstCall/ -- Sangamo BioSciences, Inc. (Nasdaq: SGMO) announced today the completion of patient enrollment into its Phase 2 clinical trial (SB-509-701) in subjects with moderate to severe diabetic neuropathy. Additionally, based upon positive initial data, the company plans to expand this study and enroll additional subjects to obtain more information for the design of a potential Phase 3 trial.
"Our initial review of data from SB-509-701, while preliminary, was quite encouraging," stated Dale Ando, M.D., Sangamo's vice president of therapeutic development and chief medical officer. "As such, we plan to double the number of subjects in this study and evaluate an additional treatment schedule. Because of the definitive nature of the endpoint in this trial -- recovery of nerve conduction velocity or NCV -- these additional data may provide important information which could potentially expedite a Phase 3 study."
SB-509-701 is a randomized, single-blind, placebo-controlled, repeat-dosing, multi-center Phase 2 clinical trial of SB-509 in subjects with moderate to severe diabetic peripheral sensory motor neuropathy (DN). The study is designed to evaluate the clinical safety and clinical effects of repeat administration of SB-509 in diabetic subjects that have unmeasurable nerve conduction velocity (NCV) in at least one of the nerves in the leg.
In the initial trial, 45 subjects have completed enrollment. Subjects were randomized to one of two groups in a 2:1 ratio. The larger group (approximately 30 subjects) was treated by intramuscular injection of 60 mg of SB-509 (30 mg of SB-509 per leg) into the lower limb and will be treated every 3 months. The remaining group (approximately 15 subjects) received an equal volume of placebo on the same schedule. Each subject will receive a total of two treatments (Day 0 and 90). Subjects receive injections in a distribution pattern that targets the major peripheral nerves in the legs and feet.
The expansion protocol will enroll approximately 45 additional subjects in the trial. Subjects will again be randomized to one of two groups in a 2:1 ratio. The larger group (approximately 30 subjects) will be treated by intramuscular injection of 60 mg of SB-509 (30 mg of SB-509 per leg) into the lower limb every 2 months. The remaining group (approximately 15 subjects) will receive an equal volume of placebo on the same schedule. Each subject will receive a total of three treatments (Day 0, 60 and 120). Subjects will receive injections in a similar pattern to that used in the initial part of the trial.
"In our Phase 1 studies we noted interesting clinical improvements in a number of subjects," said Edward Lanphier, Sangamo's president and CEO. "In addition to improvement in quantitative sensory testing (QST), three subjects with so-called "blocked nerves" or unmeasurable nerve conduction velocity measurements in the leg showed recovered and improved NCV after a single treatment with SB-509 and two of the three subjects showed recovery and improvement past a six-month follow-up period. As these observations were made in the Phase 1 trial, which studied only a small number of subjects, we initiated a Phase 2 trial, SB-509-701, to further investigate this finding. Our primary review of data from the SB-509-701 study continued to show a marked neurophysiologic improvement in severe diabetic neuropathy patients with unmeasurable nerve conduction velocities treated with SB-509. Based upon these observations we plan to expand this trial to provide additional data that could help support a future pivotal study."
Data from SB-509-701 will be presented at appropriate clinical meetings.
SB-509 is an injectable formulation of a zinc finger DNA-binding protein transcription factor (ZFP TF(TM)) designed to upregulate the expression of the gene encoding vascular endothelial growth factor (VEGF-A). VEGF-A has been demonstrated to have direct neurotrophic and neuroprotective properties. In preclinical animal efficacy studies in a diabetic rat model (Diabetes, June 1, 2006; 55(6): 1847-1854), SB-509 has proven effective in protecting motor and sensory nerve function from disease-induced nerve damage. Sangamo is currently evaluating SB-509 in three ongoing Phase 2 clinical trials for the treatment of diabetic neuropathy.
About Diabetic Neuropathy
Diabetic peripheral neuropathy is one of the most frequent complications of diabetes. Symptoms include numbness, tingling sensations and pain particularly in the toes or feet. This gradually evolves to 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 percent 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 20.8 million people with diabetes in the United States and that of those about 60 percent to 70 percent have mild to severe forms of neuropathy. According to the Centers for Disease Control, diabetes is becoming more common in the United States. From 1980 through 2002, the number of Americans with diabetes more than doubled.
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 program is currently in Phase 2 clinical trials for evaluation of safety and clinical effect in patients with diabetic neuropathy. Phase 1 clinical trials are ongoing to evaluate a ZFP Therapeutic for peripheral artery disease. Other therapeutic development programs are focused on ALS, cancer, HIV/AIDS, neuropathic pain, nerve regeneration, Parkinson's disease 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 gene modification. Sangamo has established strategic partnerships with companies outside of the human therapeutic space including Dow AgroSciences, Sigma-Aldrich Corporation and several companies applying its ZFP Technology to enhance the production of protein pharmaceuticals. For more information about Sangamo, visit the company's web site at http://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 clinical trials of SB-509 and the potential for further study and development 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 trials, whether the SB-509 clinical trials 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 Sangamo's SEC filings, and in particular, the risk factors described in its Annual Report on Form 10-K and its most recent Quarterly Report on Form 10-Q. Sangamo BioSciences, Inc. assumes no obligation to update the forward-looking information contained in this press release.
|SOURCE Sangamo BioSciences, Inc.|
Copyright©2008 PR Newswire.
All rights reserved