HEIDELBERG, Germany, February 14 /PRNewswire/ -- SYGNIS Pharma AG (Frankfurt: LIO; ISIN DE0005043509; Prime Standard), today announced that it has received a positive recommendation from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMEA) for AX200 for the treatment of Amyotrophic Lateral Sclerosis (ALS).

ALS is a progressive neurodegenerative disease caused by the degeneration of nerve cells innervating skeletal muscles and affects an estimated 50,000 to 100,000 people in the industrialized nations. Patients with ALS suffer from progressive muscle wasting throughout their bodies resulting in increasing disabilities and their lifespan is cut short. Death in ALS patients normally occurs within only a few years after diagnosis and there are currently no adequate therapies available.

The SYGNIS research team has demonstrated in pre-clinical trials that AX200 counteracts the neuronal cell death and thereby decelerates the development of the disease. In the laboratory, protection of the motor neurons led to a reduced degeneration of muscles whilst maintaining muscular strength for a longer period of time. Moreover, AX200 advances regenerative processes by stimulating neuronal stem cells. This dual mechanism of AX200 is a unique approach in the drug development for ALS.

Dr. Alfred Bach, CEO of SYGNIS Pharma AG, said: "Our pre-clinical data has convinced the EMEA experts that AX200 is a promising drug candidate for the treatment of ALS. The EMEA's positive opinion validates the great potential of AX200 for the treatment of such a difficult indication and supports us in our efforts to develop an effective therapy for ALS, which is one of the most devastating neurological diseases. We will plan further steps in close cooperation with EMEA and hope to initiate clinical trials within the next twelve months. Moreover, this is certainly an important signal for the future development of AX200 as a whole."

About orphan drug designation

The EMEA orphan drug designation programme is dedicated to stimulate and support the development of pharmaceuticals to treat life-threatening or very serious and rare diseases, which cannot be treated or can only be treated insufficiently. For companies which face the challenges of research for such disorders, EMEA offers support regarding the conception of development plans (scientific advice) and gives reductions on approval fees. After the approval of the products, these so called "orphan drugs" (the orphans amongst the pharmaceuticals) are assigned market exclusivity for 10 years.

About AX200

AX200 is a pharmaceutical protein, developed by SYGNIS for the treatment of neurodegenerative diseases. In the indication stroke it is the most advanced drug candidate in SYGNIS' product pipeline. In Summer 2007 the Phase IIa of the clinical development was successfully completed. AX200 is an endogenous protein. As part of the body's own protective action the production of AX200 is boosted after brain damage. If the molecule is given as a medication it increases the existing endogeneous response to the damage. SYGNIS pursues a dual neurotherapeutic approach in the development of AX200 for the treatment of neurodegenerative diseases: AX200 stops neuronal cell death in the acute phase of the disease while at the same time stimulating the regeneration of the already damaged CNS through the stimulation of neurogenesis as well as arteriogenesis and the reorganisation of neuronal networks. Besides stroke, AX200 is currently in preclinical testing for further neurodegenerative indications such as Amyotrophic Lateral Sclerosis (ALS) and Spinal Cord Injuries (SCI).

About SYGNIS Pharma

SYGNIS Pharma AG, with head offices in Heidelberg, is a specialty pharmaceutical Company listed on the Prime Standard of the German stock exchange. The Company is focused on the research, development and marketing of innovative therapies for the treatment of neurodegenerative diseases. These include stroke, Amyotrophic Lateral Sclerosis (ALS), Huntington's Disease and neurological disorders resulting from injuries of the brain or spinal cord. All these disorders are characterized by the fact that, as the disease progresses, nerve cells are damaged and die. Although there is great medical demand, there are currently no or only inadequate treatment options available.


Some statements included in this press release, relating neither to proven financial results nor other historical data, should be viewed as forward-looking, i.e. not definite. Such statements are mainly predictions of future results, trends, plans or goals. These statements should not be considered to be total guarantees since given their very nature they are subject to known and unknown risks and imponderability and can be affected by other factors as a consequence of which the actual results, plans and goals of SYGNIS Pharma AG may deviate greatly from the established conclusions or implied predictions contained in such statements. SYGNIS does not undertake to publicly update or revise these statements in the light of new information or future results or for any other reason.

For further information please contact: SYGNIS Pharma AG: Dr. Franz-Werner Haas Vice President Operations Tel.: +49(0)6221-454-812 franz-werner.haas@sygnis.de Media-Contact: Julia Phillips Financial Dynamics Tel.: +44(0)20-7269-7187