Sangamo BioSciences,
Inc. (Nasdaq: SGMO) announced that the company has completed
enrollment of its randomized, double-blind, repeat-dosing,
placebo-controlled, multi-center Phase 2 clinical trial evaluating SB-509,
a ZFP Therapeutic(TM) for the treatment of mild to moderate diabetic
neuropathy (DN). The company expects to have data from this trial in the
second half of 2008.
"The completion of enrollment of our first Phase 2 clinical trial
represents a significant milestone for Sangamo and I am pleased that we
achieved this major clinical goal as planned, before the end of 2007,"
commented Edward Lanphier, Sangamo's president and CEO. "We have been very
encouraged by the disease-altering improvements that we observed in our
preclinical studies and in the Phase 1b trial and look forward to
presenting the data from this current study by the end of 2008."
This Phase 2 trial followed a positive Phase 1 study that demonstrated
clinical safety of a single treatment with SB-509 as well as statistically
significant improvements in Quantitative Sensory Testing (QST), clinically
relevant improvements in motor and sensory nerve conduction velocities
(NCV) and a trend towards improvement in Total Neuropathy Score (TNS),
suggesting an alteration of disease progression in subjects with DN. The
Phase 2 study was designed to confirm and expand these findings and to
evaluate repeat dosing with SB-509.
The trial has been partially funded by a $3.0 million commitment from
the Juvenile Diabetes Research Foundation International (JDRF) pursuant to
a research agreement between Sangamo and JDRF. Completion of enrollment of
the trial triggers a milestone payment from JDRF under this agreement.
About SB-509
SB-509 is a formulation of a zinc finger DNA-binding protein
transcription factor (ZFP TF) designed to upregulate the expression of the
gene encoding vascular endothelial growth factor (VEGF-A). Sangamo is
currently evaluating this ZFP Therapeutic in a second ongoing Phase 2
clinical trial for the treatment of moderate to severe DN; plans to
initiate a third Phase 2 trial to evaluate the effect of SB-509 on the
mobilization of stem cells in subjects with DN; and has a preclinical
program targeting spinal cord injury. Sangamo has also announced a new
clinical program, the initiation of a Phase 2 clinical trial in Amyotrophic
Lateral Sclerosis (Lou Gehrig's disease) in the first half of 2008.
Phase 2 study of SB-509 for mild to moderate DN
The clinical trial is a double-blind, placebo-controlled, repeat-dosing
study designed to evaluate the clinical safety and clinical effects of
repeat administration of SB-509 in diabetics with mild to moderate diabetic
peripheral sensory motor neuropathy in the legs.
A total of 102 subjects have been enrolled into the trial. Subjects
were randomized to one of two groups in a 2:1 ratio. The larger group is
being 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 is
receiving 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 distribution pattern that targets the skeletal
muscle adjacent to the major peripheral nerves in the legs and feet.
The symptoms of diabetic peripheral neuropathy and any changes that
occur during the trial will be evaluated based on neurological examination
data, electrophysiological testing data, subject neurological
questionnaire, and subject pain assessment. Investigators will use
quantitative sensory testing (QST) with the Vibratron II instrument to
assess the threshold of detection of vibration, electrophysiological
testing using nerve conduction velocity (NCV) to assess the rate at which a
nerve can conduct an electrical signal, and a composite scoring system, the
total neuropathy score (TNS) to assess signs and symptoms of the condition.
In addition, skin biopsies will be taken to evaluate the direct therapeutic
effect of SB-509 on nerve regrowth. This test may provide an important
mechanistic marker for efficacy. Subjects will be assessed for seven months
following the last dosing.
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.
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 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 Amyotrophic Lateral
Sclerosis (ALS), cancer and 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
sangamo.
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, therapeutic applications of
Sangamo's ZFP technology platform and payments upon achievement of research
milestones. 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 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 Quarterly Reports on Form 10-Q. Sangamo BioSciences, Inc. assumes no
obligation to update the forward-looking information contained in this
press release.
Sangamo BioSciences, Inc
sangamo
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