sNN0029 is a novel drug candidate for the treatment of amyotrophic lateral sclerosis (ALS) that has demonstrated in preclinical in vivo studies the ability to slow disease progression and increase life span. The pharmaceutical formulation contains vascular endothelial growth factor (VEGF) protein, a naturally occurring protein scientifically proven to be an important survival factor for motor neurons. The intended therapy involves direct infusion of sNN0029 into the cerebrospinal fluid (CSF) that circulates around the brain and spinal cord by intracerebroventricular (ICV) administration.
Research has shown that mice with targeted deletions of the hypoxia-response element in the promoter of the VEGF gene display reduced brain and spinal cord VEGF protein levels and ALS-like symptomatology. In industry-standard animal models of the disease, treatment with VEGF results in neuroprotection, improved motor function and extended life span. Additional evidence for a role of VEGF in ALS has come from multiple scientific studies including the demonstration that patients with ALS have impaired production of VEGF in their nervous system. VEGF has also been shown to stimulate neurogenesis and therefore offers the potential for value at multiple levels.
Preclinical safety assessment and dose-finding studies formed the basis for a formal regulatory submission (IND) required for the start of clinical trials in December 2008. The initial clinical phase I/II study investigates safety parameters in ALS patients. Pivotal phase II/III clinical trials will evaluate the efficacy of sNN0029 over placebo to increase time of survival. sNN0029 is 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. This approach enables the administered dose of the VEGF protein to best reach vulnerable motor neurons, and minimizes the risk for side-effects since only the central compartments of the nervous system, and not the entire body, are exposed to the drug.
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