Amorfix confirms presence of misfolded SOD1 in sporadic ALS
TORONTO, Oct. 24, 2013 /CNW/ - Amorfix Life Sciences, a product development company focused on diagnostics and therapeutics for misfolded protein diseases, today announced that it has confirmed the presence of misfolded superoxide dismutase 1 (SOD1) in sporadic ALS. In the breakthrough study conducted at Amorfix, using the Company's proprietary antibodies that bind specifically to misfolded SOD1, the company detected misfolded SOD1 in spinal cord samples obtained from subjects with sporadic ALS. Previous studies have shown the presence of misfolded SOD1 in familial ALS, which represents about 10% of all diagnoses. This current finding suggests that ALS patients with either familial or sporadic disease can be potentially identified by detection of misfolded SOD1 and subsequently treated with therapies that target misfolded SOD1.
"This has major implications for the treatment of ALS, as therapeutic drugs that target misfolded SOD1 will be helpful for patients with either the familial or the sporadic forms of the disease" said Dr. Neil Cashman, Amorfix Chief Scientific Officer and Chairman of the Board. "This greatly expands the commercial market opportunity for our therapeutic antibody product in development as well as our blood test diagnostic as patients with the sporadic form of the disease represent approximately 90% of the entire ALS patient population".
SOD1 is called the Jekyll and Hyde protein. In its native configuration it serves as an anti-oxidant that protects nerves throughout the body from damage and death. However, in its misfolded form, SOD1 has the exact opposite effect and causes damage and death of neurons. SOD1 is known to be misfolded in familial ALS, which is the genetic form of the disease; however, until now it has been unclear whether SOD1 misfolds in the non-genetic, or sporadic form of the disease. As the vast majority of ALS cases are sporadic in origin, it is critical that new drugs are able to treat this common form of the disease.
Amorfix, through a license agreement with Biogen Idec, is developing therapeutics for the treatment of ALS. The Company is developing a vaccine treatment and also a simple blood test diagnostic based on the detection of misfolded SOD1 in the plasma of ALS patients which could serve as a much needed test that identifies the disease in its very early stages. "Together, these programs represent our disease management strategic approach with the potential for early diagnosis, treatment and prevention of ALS", said Dr. Robert Gundel, Amorfix President and Chief Executive Officer.
ALS is a common neuromuscular disease, affecting an estimated 120,000 people of all races and ethnic backgrounds worldwide. According to the ALS Association, more than 5,600 people in the U.S. are diagnosed with ALS annually and an estimated 30,000 Americans have ALS at any given time.
Amorfix Life Sciences Ltd. (TSX:AMF) is an early-stage product development company developing therapeutic antibodies and diagnostics targeting misfolded protein diseases. Amorfix utilizes its computational discovery platform, ProMIS™, to predict novel Disease Specific Epitopes (DSEs) on the molecular surface of misfolded proteins. Using this technology, Amorfix is developing novel antibody therapeutics and companion diagnostics for cancer and amyotrophic lateral sclerosis (ALS). In addition, Amorfix has developed two proprietary technologies to specifically identify very low levels of misfolded proteins in a biological sample: Epitope Protection™ and AMFIA™, an ultra-sensitive dual-bead immunoassay. Use of these technologies has generated a cerebrospinal fluid (CSF) screening test for both Alzheimer's disease (AD) and mild cognitive impairment (MCI), and an ultrasensitive method for detecting the hallmark of AD, aggregated beta-Amyloid, in brain tissue, CSF and blood from animal models of AD. For more information about Amorfix, visit www.amorfix.com.
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SOURCE Amorfix Life Sciences Ltd.For further information:
Dr. Robert Gundel
President and Chief Executive Officer
Amorfix Life Sciences Ltd.
Tel: (416) 847-6957
Fax: (416) 847-6899
Chief Financial Officer
Amorfix Life Sciences Ltd.
Tel: (416) 644-7358
Fax: (416) 847-6899