Mapreg SAS Granted Orphan Status in Europe for its Lead Drug Candidate Under Development for Treatment of Spinal Cord Injury

Published: Mar 21, 2008

Paris - March 18, 2008 - Mapreg, a biotechnology company specialized in the development of novel drugs for the treatment of central nervous system diseases, announces today that the European Commission has granted its lead compound, MAP4343, orphan drug status for the treatment of spinal cord injury and has entered the compound in the European Community register of orphan drugs. This decision follows a favorable opinion from the Orphan Drugs Committee of the European Medicines Evaluation Agency (EMEA).

MAP4343 is patent-protected and has an identified and validated molecular target, namely Microtubule Associated Protein type 2 (MAP2). The product's main advantage resides in its efficiency when administered by peripheral injection up to 24 hours after the trauma. The results of studies on animal models yielded so far are unmatched elsewhere. Regulatory preclinical studies will be completed shortly (having shown no significant signs of toxicity so far) and a Phase I clinical trial could be initiated towards the end of 2008.

"The granting of orphan drug status in the European Union is an important step for Mapreg and for the future benefit of patients having suffered a spinal cord injury," observed Etienne-Emile Baulieu, President of Mapreg. "I am particularly pleased about this recognition at the European level, which will enable us to exploit neurosteroid derivatives discovered in my laboratory. The results on animal models are without parallel and we want to move forward to initiate the first clinical trials."

The orphan drug status granted by the EMEA facilitates the development of products designed to treat rare diseases, which would not otherwise be profitable enough because of the small number of patients involved. The official definition of an orphan drug in the European Union is a product designed to treat a disease that affects less than five people in 10,000. Orphan status gives a product 10 years' exclusivity in a particular therapeutic indication in the European market, including France, Germany, Italy, Spain and the United Kingdom. Orphan drug status also enables a company to obtain scientific assistance and advice from the EMEA for demonstrating the product's quality, safety and efficacy during the entire development cycle. It also ensures the company easy access to the EU's Mutual Recognition Procedure, grants it total or partial exemption from fees associated with the regulatory approval of the product, and gives it the possibility of obtaining funding under the European Union's program for supporting research into rare diseases.

There are more than 20,000 new cases of spinal cord injury every year in the western world, and no drug exists at present to treat them. The only therapeutic option is surgery, involving one or more operations. The consequences of such traumas are paraplegia or tetraplegia, and the total cost of caring for a patient is estimated at between 1 and 2 million euros.

About Mapreg Mapreg SAS is a biotechnology company specialized in the discovery and development of new drugs for the treatment of diseases of the central nervous system.

These drugs, neurosteroid derivatives, exploit a new mechanism for the protection and repair of nerve fibers. Neurosteroids are steroids which are synthesized in the central nervous system and which were discovered in the laboratory of Etienne-Emile Baulieu, Mapreg's president.

The first products developed by Mapreg are focused on acute pathologies of the central nervous system such as spinal cord injury, traumatic brain injury and stroke. There is no drug treatment currently available for these pathologies. Subsequently, Mapreg will develop products to treat age-related or neurodegenerative diseases such as Alzheimer's.

Mapreg was founded in 2000 to build on research carried out by Prof. Baulieu and his team. The company has already raised EUR 2 million (USD 3M). It is based in the Kremlin-BicĂȘtre Hospital near Paris.

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