PRESS RELEASE: Cambria Biosciences Receives $5.0 Million in Grants and Awards

Cambria Biosciences Receives $5.0 Million in Grants and Awards for Neurodegenerative Disease Drug Discovery and Preclinical Development Programs

WOBURN, Mass. -- Cambria Biosciences, a privately held biopharmaceutical company focused on the discovery and development of innovative proprietary small molecule drugs for neurological conditions, today announced that it has been awarded five grants from multiple organizations totaling $5.0 million. The grant funding will support the company’s drug discovery programs for neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease), muscular dystrophy and Parkinson’s disease.

“We are very grateful to receive these awards from leading organizations involved in the search for new treatments for serious neurological disorders,” said Donald Kirsch, PhD, Senior Vice President for Drug Discovery at Cambria Biosciences. “Because of the rigorous peer-review process employed by these organizations, we are also very pleased with the scientific recognition and support these awards bring to Cambria’s approach of developing new disease models to identify novel small molecules that suppress disease pathology in innovative ways.”

Protein misfolding and aggregation are common features of many neurodegenerative diseases, including ALS, Parkinson’s disease, Alzheimer’s disease, and others. Familial ALS caused by mutations in the SOD1 gene is a particularly well-characterized example of a neurodegenerative protein misfolding disease. Cambria has received three grants to support the company’s ALS research and development programs, led by Dr. Kirsch:

The National Institute of Neurological Disorders and Stroke (NINDS) granted Cambria a Phase 1 SBIR award to identify small molecule compounds that protect against cellular damage caused by the mutant SOD1 protein.
The ALS Association (ALSA) awarded Cambria a multi-year, multi-million dollar grant as part of a milestone-driven program that will include lead optimization, pharmacology, and preclinical transition to an Investigational New Drug Application for novel compounds that block protein aggregation and resulting cellular damage.
The ALS Therapy Alliance will fund a project to test orally available small molecule compounds discovered by Cambria researchers for efficacy in preclinical animal models of ALS.
Cambria’s ALS research program will be conducted in collaboration with Dr. Richard Morimoto and Dr. Richard Silverman at Northwestern University, Dr. Robert Ferrante at the Boston University Medical Center and Bedford VA Hospital, and other investigators.

ALS and muscular dystrophy are representative of a broad group of neuromuscular diseases, many of which have a defined underlying genetic basis that may offer clues for pharmacological treatments that may improve neuromuscular function. The Jain Foundation has awarded Cambria a grant to establish a drug discovery program for type 2A limb girdle muscular dystrophy (LGMD2A). Dr. Mohan Viswanathan, a senior scientist at Cambria, will establish and implement a novel whole-organism screen to discover small molecule compounds that may correct the cellular dysfunction caused by mutations in the dysferlin gene that are responsible for human LGMD2A and Miyoshi Myopathy.

Genetic mutations have also been found to underlie many forms of familial Parkinson’s disease. One form of early-onset parkinsonism is caused by loss of function of the human DJ-1 gene, and there is evidence to suggest that the DJ-1 protein may play a neuroprotective role by enhancing the cell’s ability to handle oxidative stress and/or misfolded proteins. The NINDS granted Cambria a Phase 1 SBIR Award to support a collaboration between Dr. Bethany Westlund, a senior scientist at Cambria, and Dr. Jin Xu at Tufts-St. Elizabeth Medical Center to discover therapeutics that target DJ-1 in Parkinson’s disease.

“Neurodegenerative diseases are among the greatest unmet needs in medicine,” said Leo Liu, MD, President and CEO of Cambria Biosciences. “We are therefore delighted to be part of a new paradigm of cooperation between biotechnology companies and non-profit organizations to attack these debilitating and life-threatening disorders.”

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 www.cambriabio.com.

For more information on the neurological diseases organizations cited in this announcement, please see:

www.alsa.org

www.massgeneral.org/als/ALS_Allied.htm

www.jain-foundation.org

www.ninds-nih.gov

This release contains certain forward-looking statements which involve known and unknown risks, delays, uncertainties and other factors not under the respective company's control which may cause actual results, performance or achievements of that company to be materially different from the results, performance or other expectations implied by these forward-looking statements. These factors include results of current or pending research and development activities, actions by regulatory authorities, and other activities.

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