Newron Pharmaceuticals SpA, a clinical-stage company focused on developing novel CNS therapies, announced positive initial results from a Phase II study of safinamide in patients with Parkinson's disease. The data showed that patients administered safinamide concurrent with dopamine agonist therapy exhibited a 30% reduction in motor scores.

"These data provide strong evidence that safinamide has a role in improving symptoms in patients with Parkinson's disease, with a very good safety margin. Of course these preliminary data must now be confirmed in larger studies," Prof. Ruggero Fariello, CSO of Newron Pharmaceuticals stated. "The greater potential of safinamide would be if it acts as a neuroprotectant, slowing down the rate of death of the cells in the Substantia Nigra, the area in the brain targeted by the disease process in Parkinson disease. We infer this activity based on preclinical studies and safinamide's unique multifaceted mechanisms of action that all provide potential for improving neuronal survival."

In a multinational (Italy, France, Germany, Belgium and Poland), double blind, parallel study, a total of 151 patients were randomized to receive safinamide at 0.5 or 1.0 mg/kg in a once a day schedule or placebo for three months. Patients were either treatment naive (de novo patients) or safinamide was added to a pre-existing stable treatment with a dopamine agonist, currently the recommended way of starting treatment in Parkinson disease.

In all patients, the safety of safinamide was found to be very favorable. The primary efficacy end point was also achieved. In fact the data showed that the addition of safinamide (1.0 mg/kg/die) resulted in a statistically significant improvement, defined as at least 30% reduction of the UPDRS III motor scores, in those patients who were treated with a steady dose of a dopamine agonist. In the "de novo" patients safinamide provided a motor improvement which did not reach statistical significance most likely because of a strong placebo response that it is known to be still present after 3 months.

Parkinson's disease is one of the major neurological disorders, affecting approximately 4 million worldwide, according to the World Health Organisation. It is characterized as a chronic, progressive degeneration of nerve cells that use the neurotransmitter dopamine in a special area of the brain, called the Substantia Nigra (black substance) that controls muscle tone, initiation and smoothness of movements. Symptoms include limb tremors, muscle rigidity, slowness of motion and postural instability. Current treatments aim at replenishing the lost stores of dopamine or stimulating those dopamine receptors that carry forward dopamine motor messages.

Safinamide is an investigational new drug that is being studied in Phase II clinical trials for the treatment of Parkinson's disease and epilepsy. Safinamide is a unique molecule with multiple mechanisms of action, including potent, selective and reversible inhibition of monoamine oxidase (MAO-B), without a MAO-A effect, dopamine re-uptake inhibition, sodium channel blocking activity and calcium channel modulation. The development of safinamide in Parkinson's disease is supported by a euro 2.7 million grant from the Italian Ministry of Productive Development's Innovation Technology Fund.