Clinical Programs: Immunology and Hematology
Knopp is advancing dexpramipexole as a small molecule immunological therapeutic based on its preclinical and clinical activity in modulating white blood cells known as eosinophils. Clinical experience in dexpramipexole-treated patients demonstrates that the drug produces a slowly-developing, significant and persistent reduction of eosinophil levels in blood and inflammed tissues.
Although essential to the response against certain infections, elevated eosinophils are also associated with allergic and other inflammatory disorders, including asthma, certain chronic gastrointestinal diseases, and hypereosinophilic syndromes (HES), a rare and often fatal disorder with limited treatment options. Dexpramipexole's activity in reducing peripheral blood eosinophils has been observed in preclinical studies, in Phase 2 and Phase 3 clinical trials of dexpramipexole in amyotrophic lateral sclerosis, and in pilot studies of eosinophilic subjects with chronic rhinosinusitis with nasal polyps and hypereosinophilic syndromes. Results of a Phase 2 study of dexpramipexole in HES have been presented to the American Society of Hematology and published in the journal Blood. An accompanying editorial in Blood termed dexpramipexole "very promising," noting that "the results in patients with durable and sustained responses are impressive and suggest that dexpramipexole could be an effective treatment of patients with eosinophil-related diseases."
Knopp has entered into a collaborative research and development agreement with the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH), under which NIAID recently completed a Phase 2 clinical trial of dexpramipexole in HES. Further clinical development of dexpramipexole in hypereosinophilic syndrome is expected to begin in late 2017. A Knopp-sponsored Phase 2 study of dexpramipexole in chronic rhinosinusitis with nasal polyps has also been completed. Knopp has also established a preclinical discovery effort to better understand the eosinophil-lowering effects of dexpramipexole and to explore additional possible indications in hematology and immunology.
Knopp's pipeline consists of investigational drug products that have not been approved by the U.S. Food and Drug Administration. These investigational drug products are still undergoing clinical study to verify their safety and effectiveness.