Orphan drug designation for AX200 for the treatment of Amyotrophic
Lateral Sclerosis
Feb. 14, 2008
HEIDELBERG, Germany - 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.
Disclaimer
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.
Contact:
Julia Phillips
+44 (0) 20-7269-7187
http://www.checkbio
infoId=2504
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StemCells subscribers may also be interested in these sites:
Children's Neurobiological Solutions
http://www.CNSfoundation.org/
Cord Blood Registry
http://www.CordBlood.com/at.cgi?a=150123
The CNS Healing Group
http://groups.yahoo.com/group/CNS_Healing
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