A spinal cord injury (SCI) is damage or trauma to the spinal cord which interrupts communication of the brain with body regions below the site of injury. Spinal cord injuries are predominantly caused by accidents and, in the majority of cases, result in life-long loss of control of motor functions and sensation. After the primary injury to the spinal cord, a cascade of events leads to progressive loss of tissue which may further deteriorate the patient’s prognosis.
Spinal cord injuries most commonly affect young people in the fourth decade of life. Patients have an average life expectancy of more than 30 years after injury in which, alongside paralysis, they suffer from a large number of secondary conditions including chronic pain, circulatory problems or bladder dysfunction. There is a tremendous demand for novel therapies in view of the poor treatment options currently available.
Pre-clinical studies have shown that AX200 can counteract the processes leading to neuronal cell death. In addition, the compound has demonstrated a profound ability to foster neuronal regeneration and to support neuronal plasticity which facilitates functional recovery after SCI.
In April 2008, SYGNIS received Orphan Drug Designation from the European Commission for AX200 in the treatment of Amyotrophic Lateral Sclerosis.
Dr. Alfred Bach, CEO of SYGNIS Pharma AG, said: “We are pleased to announce the positive opinion of the EMEA which further validates our lead product AX200 as a promising neuroprotectant and recovery enhancer for the treatment of devastating CNS disorders. We continue to progress the development of AX200 in acute stroke and look forward to collaborating with the EMEA as we plan the initiation of clinical trials in ALS and SCI.”
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, EMEA offers support regarding the conception of control 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 biological molecule, 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 or 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 pre-clinical testing for further neurodegenerative indications such as Amyotrophic Lateral Sclerosis and Spinal Cord Injuries.
About SYGNIS Pharma
SYGNIS Pharma AG, headquartered 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 disorders of the Central Nervous System. These include Stroke, Amyotrophic Lateral Sclerosis and neurological disorders resulting from injuries to 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.
One of the central elements in this value-creation chain is the continued development of the existing product pipeline. This is achieved by testing the Company’s proprietary compounds which are already under development in a number of further CNS indications (“line extension”). By means of specific R&D programs at SYGNIS, new pre-clinical drug candidates are identified and evaluated and suitable CNS product candidates are also systematically tested for the purpose of acquisition or in-licensing.
For further information please contact:
SYGNIS Pharma AG: Dr. Franz-Werner Haas Vice President Operations +49 (0) 6221 454 812 franz-werner.haas@sygnis.de
