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Neuron Biopharma S.A.

Javier Burgos, PhD |

Neuron Biopharma S.A.

Javier Burgos, PhD |

PRECLINICAL EVALUATION OF A NEW GENERATION OF NON-CANONICAL STATIN DERIVATIVES AS NEUROPROTECTIVE MOLECULES TO MODIFY ALZHEIMER'S DISEASE

(300 WORDS MAX.) Current drug development strategies for Alzheimer's disease (AD) have not yielded effective disease-modifying treatments. The identification of novel compounds and mechanisms of neuroprotection is thus urgently needed. AD is characterized by a progressive decline in cognitive function and its major pathological hallmarks include extensive neuronal loss, formation of intracellular neurofibrillary tangles (NFT) and extracellular deposition of β-amyloid peptides (Aβ). However, a number of drugs designed to prevent or remove the accumulation of amyloid have failed in late stage clinical trials. This fact has fostered the search for alternative targets and therapeutic approaches. In this context, Neuron Biolabs (www.neuronbio.com) main mission is the search for this kind of neuroprotective molecules to modify AD progression, as well as the development of biomarker tools for the early diagnose of the disease. Over the last decade, statins have been proposed to be useful in treating or preventing neurodegenerative disorders such as AD. In this scenario, Neuron Biolabs has synthesized and patented a set of novel simvastatin-related derivatives aimed at preserving the neuroprotective activity of statins and enhancing its CNS-penetrating properties while avoiding potentially harmful cholesterol-lowering effects. The aim of this Neuron BiolabsĀ“ proposal is to gain further insight in the pharmacokinetics, mechanisms of action and in vivo neuroprotection effects of novel derivative NST0076. Moreover, Neuron Biolabs will lead collaborations with the expert academic groups of Prof. Pahan and Prof. Mayor, in order to clearly establish the novel mechanisms of action, what in turn will be useful to design new strategies to prevent and treat this devastating disorder. We postulate that the simultaneous ability of this compound to target key pathways related to neurotrophin synthesis and the control of neuronal survival and inflammation, while avoiding potentially deleterious effects of CNS cholesterol lowering, could be very relevant for preventing and modifying AD.