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Nicox Receives Positive Opinion For European Orphan Drug Designation For Naproxcinod For The Treatment Of Duchenne Muscular Dystrophy

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

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

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.


About Nicox

Nicox  (Bloomberg:  COX:FP,  Reuters:  NCOX.PA)  is creating a new international
player  in  the  ophthalmic  market  by  building  a  diversified  portfolio  of
innovative  therapies  and  diagnostic  tools.  With  a  heritage of scientific,
business  development and  commercial expertise,  the Nicox  team is  focused on
developing  and marketing novel pharmaceuticals  and diagnostic devices that can
help  people  to  enhance  their  sight.  In  the  United  States, Nicox markets
AdenoPlus(®),  a test for the differential diagnosis of acute conjunctivitis in-
                                                             licensed from RPS®.

The  Company's pipeline  includes latanoprostene  bunod, a  novel drug-candidate
based  on Nicox's proprietary nitric oxide (NO)-donating R&D platform, developed
in  collaboration with Bausch + Lomb for the potential treatment of glaucoma and
ocular hypertension. Further NO-donating compounds are under development in non-
ophthalmic  indications, notably through partners, including Merck (known as MSD
                                          outside the United States and Canada).

Nicox  S.A.  is  headquartered  in  France  and  is  listed  on  Euronext  Paris
   (Compartment B: Small Caps). For more information please visit


This press release contains certain forward-looking statements. Although the
Company believes its expectations are based on reasonable assumptions, these
forward-looking statements are subject to numerous risks and uncertainties,
which could cause actual results to differ materially from those anticipated in
the forward-looking statements.

Risks factors which are likely to have a material effect on Nicox's business are
presented in the 4th chapter of the « Document de référence, rapport financier
annuel et rapport de gestion 2012 » filed with the French Autorité des Marchés
Financiers (AMF) on March 22, 2013 and available on Nicox's website
( and on the AMF's website (


 Nicox Contacts

 Nicox           Gavin Spencer | Executive Vice President Corporate Development

                 Tel +33 (0)4 97 24 53 00 |

 Media Relations FTI Consulting

 Europe          Julia Phillips | D+44 (0)20 7269 7187 | M +44 (0) 7770 827 263


                 Stephanie Cuthbert | D +44 (0)20 3077 0458 | M +44 (0)
                 7843 080947


                 Mo Noonan | D +44 (0)20 7269 7116 | M +44 (0)7876 444 977


 Positive Opinion for European Orphan Drug Designation:


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