sNN0031 is a novel drug candidate for the treatment of moderate to severe stage Parkinson’s disease that is designed to act on neural stem and progenitor cells in the brain. In animal models of Parkinson’s disease, treatment with sNN0031 restores motor function and improves neurochemical deficits. The product is comprised of the naturally occurring protein PDGF-BB (platelet-derived growth factor BB) formulated for intracerebroventricular (ICV) delivery. The intended therapy involves short-term continuous infusion of sNN0031 into the ventricular lumen, in order to optimize PDGF-BB access to the lateral ventricular walls of the brain where the targeted stem and progenitor cells reside. sNN0031 holds the potential to halt and even reverse disease progression, a much-needed improvement over currently available treatments, which only address the symptoms of Parkinson’s disease.
Neuronova has demonstrated in two different industry-standard preclinical models of Parkinson’s disease that PDGF-BB treatment for two weeks results in behavioral recovery and nerve cell restoration. The studies showed that the therapeutic benefits lasted for the duration of the study (three months), a result unmatched by any currently available dopamine-related drug. It is thought that PDGF-BB exerts its therapeutic effect through the stimulation of stem and progenitor cells present in the ventricular wall of the brain in proximity to the dopamine fibers involved in controlling motor function.
Preclinical safety assessment and dose-finding studies formed the basis for a formal regulatory submission (IND) required for the start of clinical trials in 2009. The initial phase I/II study investigates safety parameters in Parkinson’s disease patients. sNN0031 is administered for two weeks followed by a 10 week post-treatment period. Pivotal phase II clinical trials will evaluate the efficacy of sNN0031 over placebo to improve disabling motor symptoms. sNN0031 will be administered by means of an FDA-approved and CE-marked pump and a specialized catheter for protein drug delivery directly into the brain by ICV infusion. The intended device is very similar in design to a device currently used in thousands of patients.
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