The goal of the randomized, placebo-controlled SB-509-901 Phase 2b trial was to evaluate if the drug has clinical benefit in improving the regrowth of nerve and blood vessels in people suffering from diabetic neuropathy, a progressive degenerative disease that is one of the most frequent complications of diabetes.
The primary and secondary endpoints were sural nerve conduction velocity (sNCV), which measures how fast nerve impulses and signals pass along a particular nerve, and neuropathy impairment score in the lower limb (NIS-LL) which looks at different measures of nerve function and gives an overall score to determine the severity of neuropathy. However, the trial did show improvements in exploratory endpoints, such as pinprick and touch pressure sensation, and the drug was well tolerated in all subjects without any increased adverse side effect compared to placebo.
Based on lack of efficacy, Sangamo announced that it will discontinue all development of SB-509 and will focus its attention and resources on their pipeline of therapeutics for HIV and monogenic diseases. However, the trial has generated extensive data on the natural history and heterogeneity of diabetic neuropathy and on potential new biomarkers (for assessing progression of diabetic neuropathy. This information will prove invaluable for the design of future diabetic neuropathy trials.
Full data of the trial’s results will be presented or published at an upcoming medical meeting. To access the announcement from Sangamo, please click here.
JDRF will continue to report on ongoing developments as information becomes available.