Heidelberg, November 14, 2008 – SYGNIS Pharma AG (Frankfurt: LIO; ISIN DE0005043509; Prime Standard), today announced that it has received Orphan Drug designation from the European Commission for AX200 in the treatment of Spinal Cord Injury (SCI). This follows the positive recommendation that SYGNIS received from the EMEA in September.
Orphan Drug designation can be granted for a product that is intended for the diagnosis, prevention or treatment of life-threatening or chronically debilitating conditions that affect no more than five in 10,000 people in the European Union. During the development phase certain incentives are given to the developing company to facilitate the product’s registration and market authorization. These incentives include reductions in fees, an accelerated registration procedure. Furthermore, for a period of up to 10 years market exclusivity is granted once the product is approved.
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. There is a large demand for novel therapies in view of the poor treatment options currently available.
The SYGNIS research team has shown in pre-clinical trials that AX200 counteracts neuronal cell death. Furthermore, the compound has demonstrated a profound ability to support neuronal regeneration and 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 (ALS).
Dr. Alfred Bach, CEO of SYGNIS Pharma AG, said: “We are very pleased to have also obtained the Orphan Drug status for AX200 for spinal cord injury. It is further validation of the potential of AX200 as a promising drug candidate in the treatment of diseases of the central nervous system. We look forward to exploiting this potential as we currently prepare a further Phase II efficacy study of AX200 in acute stroke.”
About AX200
AX200 is a biological molecule, developed by SYGNIS for the treatment of neurodegenerative diseases. In the indication of 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 preclinical testing for further neurodegenerative indications such as Amyotrophic Lateral Sclerosis (ALS) and Spinal Cord Injuries (SCI).
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 inlicensing.
