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Knopp Biosciences Broadens Collaboration with NIH to Investigate Eosinophil-Lowering Effects of Dexpramipexole

March 13, 2014

PITTSBURGH, PA, MARCH 13, 2014-Knopp Biosciences LLC today announced a second collaboration with the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH), to investigate the eosinophil-lowering effects of the investigational drug dexpramipexole.

Execution of the latest Cooperative Research and Development Agreement (CRADA) between Knopp and NIAID follows an agreement announced in January under which NIAID will sponsor a Phase 2 proof-of-concept clinical trial of dexpramipexole in hypereosinophilic syndrome (HES).

Taken together, the agreements span the preclinical and clinical evaluation of dexpramipexole as a potential treatment for diseases characterized by elevated eosinophils, a type of white blood cell. Multiple clinical studies have shown that dexpramipexole reduces blood eosinophils in patients with amyotrophic lateral sclerosis (ALS). In addition, a reduction of blood eosinophils has been demonstrated in preclinical studies of dexpramipexole. These results are the basis for the hypothesis that dexpramipexole may be a potential treatment for diseases characterized by elevated eosinophils, for which off-label corticosteroid administration generally remains first-line treatment.

The principal goals of the latest CRADA are to further elucidate the mechanistic effects of dexpramipexole administration in hypereosinophilic mice in vivo and to evaluate the drug's interactions with eosinophil progenitors in tissue culture. The manner in which dexpramipexole reduces eosinophil counts in animals may help guide translational studies in eosinophil-related human disease.

The principal investigator under the preclinical CRADA is Helene F. Rosenberg, M.D., Ph.D., Chief of the Inflammation Immunobiology Section of the Laboratory of Allergic Diseases at NIAID. Among the foremost leaders in eosinophil biology, her laboratory explores the complex links between inflammation and infectious disease.

"This collaboration represents an important step in determining both the mechanism and the potential utility of dexpramipexole as a treatment in areas of high unmet medical need," said Steven Dworetzky, Ph.D., Vice President of Discovery Research at Knopp. "We are very pleased to secure a collaboration with an investigator of Dr. Rosenberg's experience and leadership in eosinophil biology."

About Knopp Biosciences

Knopp Biosciences, based in Pittsburgh, PA, USA, is a drug discovery and development company focused on delivering breakthrough treatments for unmet medical needs through innovation, experience, and partnership. Knopp's lead product candidate is dexpramipexole, an orally bioavailable small molecule in research and development for the treatment of eosinophil-associated disorders and amyotrophic lateral sclerosis (ALS). Knopp's discovery platform is directed to next-generation mitochondrial modulators for the treatment of neurodegenerative disorders, leukocyte-lowering agents for immunological disorders, and ion channel modulators for epilepsy and neuropathic pain.

Media Contact:
Thomas Petzinger Jr.