November 6, 2007 

The ALS Association Announces $ 3.5 Million Partnership with Cambria Biosciences to Accelerate Development of New Drugs for ALS

 Initial data from project presented at the Society for Neuroscience meeting

The ALS Association has awarded Cambria Biosciences a $3.5 million contract to develop new drug compounds for treatment of Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s disease. This partnership builds on previously funded efforts with Cambria to develop unique neuroprotective drug compounds that prevent cell death caused by misfolded proteins that aggregate together within the cell. Mutant copper-zinc superoxide dismutase, or SOD1, is a protein that is misfolded to form aggregates in the motor neurons of some people with inherited forms of ALS. 

“This academic-industry partnership is an excellent step forward to achieving the goals of TREAT ALS to ensure accelerated drug discovery and the development of new compounds as clinical candidates for ALS,” noted Dr. Lucie Bruijn, science director and vice president for The ALS Association.

The study is a unique collaboration between Dr. Donald Kirsch, senior vice president for drug discovery at Cambria Biosciences, Dr. Richard Morimoto, an international expert in protein misfolding (which can cause cellular damage associated with neurodegenerative diseases) from Northwestern University and Dr. Richard Silverman, a leading medicinal chemist from Northwestern University.

“With this award we will be able to set up a virtual mini-pharmaceutical company to pursue new ALS therapies.  It is a real privilege to have the opportunity to collaborate on this project with scientists of the stature of Drs. Morimoto and Silverman,” said Dr. Kirsch.  The project team presented their results to-date at the international meeting of the Society for Neuroscience in San Diego on November 6 in a poster entitled, “Protein Aggregation and the Development of ALS Therapeutics,” by R. Benmohamed and other researchers.

The compounds will be optimized to produce the lowest toxicity, maximum potency and efficacy and favorable pharmacological properties, such as oral activity, to develop an investigational new drug to enter clinical testing.  Cambria will retain intellectual property and commercialization rights on resulting drug candidates, and The ALS Association will be eligible to receive royalties from Cambria on net sales of any approved products.

Dr. Leo Liu, CEO of Cambria Biosciences, added: “This partnership further leverages Cambria’s significant and ongoing commitment to develop medicines for underserved neurodegenerative diseases.  We are very pleased to work with The ALS Association and appreciate its support of our neurodegenerative disease drug discovery programs.”

Currently there is only one Food and Drug Administration (FDA) approved compound on the market for ALS, Riluzole, with modest effects on disease progression.  There is therefore an urgent need for the development of new compounds with properties more suitable for the treatment of neurodegenerative diseases. The compounds identified through this consortium effort act on a mechanism thought to be involved not only in ALS but other neurodegenerative disorders such as Parkinson’s and Huntington’s disease. Therefore the successful development of such compounds will have a major impact on currently untreatable neurodegenerative disorders.

About ALS
Amyotrophic lateral sclerosis (ALS) is a motor neuron disease, first identified in 1869 by the noted French neurologist Jean-Martin Charcot. Although the cause of ALS is not completely understood, the 1990's have brought a wealth of new scientific understanding about the physiology of this disease. ALS is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. Motor neurons reach from the brain to the spinal cord and from the spinal cord to the muscles throughout the body. The progressive degeneration of the motor neurons in ALS eventually leads to their death. When the motor neurons die, the ability of the brain to initiate and control muscle movement is lost. With voluntary muscle action progressively affected, patients in the later stages of the disease may become totally paralyzed. Yet, through it all, for the vast majority of people, their minds remain unaffected.

Lou Gehrig, with whom ALS is most commonly associated, first brought national and international attention to the disease in 1939 when he abruptly retired from baseball after being diagnosed with ALS. However, ALS is not just Lou Gehrig's disease and it knows no boundaries.

About The ALS Association
The ALS Association is the only national, not-for-profit voluntary health organization devoted solely to fighting ALS through research, patient services, advocacy and public education and information.

The ALS Association’s TREAT ALS (Translational Research Advancing Therapy for ALS) program combines efficient new drug discovery with priorities set for existing drug candidates, to accelerate clinical testing of compounds with promise for the disease. For more information on TREAT ALS, please see The ALS Association's web site under the research tab: http://www.alsa.org/research/article.cfm?id=1048  and Laboratory Models in ALS http://www.alsa.org/research/article.cfm?id=812.

About Cambria Biosciences
Cambria Biosciences is an innovative biotechnology company building a product pipeline for serious neurological conditions such as Lou Gehrig’s disease and epilepsy. Cambria employs its chemical genetics platform to discover and elucidate novel drug candidates with unprecedented mechanisms of action, internally and in partnership with other companies.  Further information is available at http://www.cambriabio.com/. For more information about Cambria Biosciences, contact Doug MacDougall or Jennifer Greenleaf, MacDougall Biomedical Communications, at 508-647-0209.