Knopp Biosciences LLC engages in the discovery and development of drugs for the treatments of neurological disorders. It offers KNS-760704, a drug candidate for the treatment of amyotrophic lateral sclerosis. Knopp Biosciences LLC was founded in 2010 and is based in Pittsburgh, Pennsylvania.
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Founded in 2010
Knopp Biosciences LLC Collaborate with National Institute of Allergy and Infectious Diseases to Investigate on Hypereosinophilic Syndrome Studies
Mar 20 14
Knopp Biosciences LLC has announced a second collaboration with the National Institute of Allergy and Infectious Diseases, (NIAID), to investigate the eosinophil-lowering effects of the investigational drug dexpramipexole. Execution of the latest Cooperative R&D Agreement (CRADA) between Knopp and NIAID follows an agreement announced in January under which NIAID will sponsor a Phase II 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.
Knopp Biosciences LLC Announces Collaboration Agreement with the National Institute of Allergy and Infectious Diseases to Conduct a Clinical Trial of Dexpramipexole in Hypereosinophilic Syndrome
Jan 8 14
Knopp Biosciences LLC announced a collaboration agreement with the National Institute of Allergy and Infectious Diseases (NIAID) 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 2014 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 collection 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. 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.