Nicox Receives Positive Opinion for European Orphan Drug Designation for Naproxcinod for the Treatment of Duchenne Muscular Dystrophy

September 10, 2013

September 11, 2013.
Sophia Antipolis, France.

Nicox S.A. (NYSE Euronext Paris: COX) today announced that it has received a positive opinion from the European Union Committee for Orphan Medicinal Products (COMP) recommending orphan drug designation for naproxcinod, a CINOD (Cyclooxygenase-Inhibiting Nitric Oxide-Donating) anti-inflammatory candidate, for the treatment of Duchenne Muscular Dystrophy (DMD). The orphan drug designation allows companies to benefit from a number of incentives, including a 10-year market exclusivity post-approval, scientific advice and fee reductions. Final approval of the opinion on orphan designation is expected from the European Commission in the coming months. Naproxcinod has shown promising preclinical results in models of muscular dystrophy.

“Naproxcinod may have the potential to address the important unmet needs of the patients suffering from Duchenne Muscular Dystrophy, who currently have no approved therapies available.” said Michele Garufi, Chairman and CEO of Nicox. “Receiving the orphan drug designation for naproxcinod in Europe will be a key milestone for Nicox and will support our strategy of partnering the development of naproxcinod in this debilitating disease.”

Nicox is evaluating the options for developing naproxcinod through a partner as an adjuvant for the treatment of muscular dystrophy or for the treatment of the signs and symptoms of osteoarthritis of the knee. This approach is aimed at maximising the opportunities to progress the development of naproxcinod in one of these indications.

About naproxcinod in DMD

In DMD, muscle damage is caused by the inability to synthesize the dystrophin protein, which is associated with neuronal NO-synthase. Naproxcinod, through both its anti-inflammatory and NO-donating properties, may have the potential to bring valuable therapeutic activity to the dystrophic muscles of Duchenne patients.

In a long-term preclinical study, naproxcinod was shown to improve skeletal and cardiac muscle function and to reduce skeletal muscle inflammation in mdx mice. The data was presented at the Muscular Dystrophy Association (MDA) Scientific Conference in Washington, DC in April 2013.

A patent covering nitric oxide releasing compounds, including naproxcinod, for the treatment of muscular dystrophies is pending in Europe and has been granted in the United States in July 2013.

About Duchenne Muscular Dystrophy (DMD)

DMD is a chronically debilitating and life-threatening disease, characterized by rapidly progressive muscle weakness and wasting due to degeneration of skeletal, smooth and cardiac muscles. DMD is the most common and serious form of muscular dystrophy, with the onset of symptoms occurring in early childhood (usually between three and five years of age), and affects about 3 in 10,000 live-born males. This condition worsens throughout childhood, with patients becoming wheelchair-bound between the ages of seven and thirteen. Most DMD patients die by the age of twenty, most frequently as a direct result of respiratory/cardiac failure.

About Orphan Drug Designation

The Orphan Medicinal Product Designation is given to products to be developed for the diagnosis, prevention or treatment of life-threatening or very serious conditions that affect not more than 5 in 10,000 persons in the European Union.

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