[January 08, 2014]

Knopp Biosciences and NIH to Collaborate in Evaluating Dexpramipexole in Patients with Hypereosinophilic Syndrome

Knopp Biosciences LLC today announced a collaboration agreement with the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH), to conduct a clinical trial of dexpramipexole in hypereosinophilic syndrome (HES).

Under the Cooperative Research and Development Agreement (CRADA), NIAID will sponsor the Phase 2 proof-of-concept trial, while Knopp will provide the dexpramipexole study drug and will collaborate on trial design and study oversight.

The 24-week, open-label clinical trial in HES subjects receiving corticosteroid therapy is expected to initiate enrollment in the first quarter of this year at the NIH Clinical Center in Bethesda, MD. The principal investigator is Amy Klion, M.D., immediate past president of the International Eosinophil Society and chief of the Eosinophil Pathology Unit of NIAID's Laboratory of Parasitic Diseases.

The principal objective of the trial is to evaluate the potential of dexpramipexole to reduce long-term and acute treatment with corticosteroids in people with HES while maintaining or reducing eosinophil count. Data presented at the July 2013 meeting of the International Eosinophilia Society in Oxford, England, demonstrated that dexpramipexole safely and persistently reduced blood eosinophil counts by approximately 70% (p < 0.0001) in people with amyotrophic lateral sclerosis (ALS) enrolled in a large Phase 3 trial (n=943).

Evidence from multiple preclinical and ALS clinical studies showing that dexpramipexole reduces blood eosinophils is the basis for the hypothesis that the drug may be a potential treatment for HES, for which off-label corticosteroid administration remains first-line treatment. HES is a colection of rare disorders characterized by excess levels of circulating eosinophils, a type of white blood cell, eventually leading to tissue damage in end organs. Dr. Klion will evaluate the primary endpoint of minimally effective corticosteroid dose reduction after treatment with dexpramipexole.

"Knopp is very pleased to collaborate with a researcher of Dr. Klion's experience and leadership in evaluating investigational treatments for HES," said Michael Bozik, M.D., president and CEO of Knopp. "HES is a very serious orphan disease with limited treatment options, and we expect this collaboration to provide an important assessment of the drug's potential for benefiting patients with this aggressive immunological disease."

In addition to assessing the potential of dexpramipexole as a corticosteroid-sparing treatment for HES, the planned Phase 2 study will examine drug effects on bone marrow eosinophils and myeloid precursors prior to steroid taper; changes in tissue eosinophils; and measures of eosinophil activation, cytokine/chemokine profile, and other immunologic parameters.

Study objectives also include evaluating the safety of 150 mg of dexpramipexole administered twice daily in HES patients currently treated with corticosteroids. The selection of dose and regimen is based on the safety and tolerability of dexpramipexole in earlier clinical studies of healthy volunteers and ALS subjects receiving 50 mg, 150 mg, and 300 mg total daily doses and in a healthy volunteer study of 600 mg total daily dose.

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 neuropathic pain and epilepsy.

This announcement contains "forward-looking statements," including statements relating to Knopp's plans regarding regulatory filings and clinical development programs for KNS-760704 (dexpramipexole). All forward-looking statements are based on management's current assumptions and expectations and involve risks, uncertainties, and other important factors, specifically including the uncertainties inherent in clinical trials and product development programs, the availability of funding to support continued research and studies, the availability or potential availability of potential alternative therapies or treatments, the availability of patent protection for the discoveries and strategic alliances, as well as additional factors that may cause Knopp's actual results to differ from our expectations. There can be no assurance that KNS-760704 will be successfully developed or manufactured or that final results of clinical studies will be supportive of regulatory approvals required to market the products. Knopp undertakes no obligation to update or revise any such forward-looking statements, whether as a result of new information, future events, or otherwise.

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