Publication Date:
2016-12-02
Description:
Hypereosinophilic syndromes (HES) are a heterogeneous group of disorders characterized by peripheral eosinophilia and eosinophil-related end organ damage. Whereas most patients respond to steroid therapy, high doses are often necessary and serious side effects are common. Dexpramipexole (KNS-760704) is an orally bioavailable synthetic amino-benzothiazole that was in development for the treatment of amyotrophic lateral sclerosis (ALS). Despite failure to meet the primary efficacy endpoint in a phase 3 trial in ALS patients, dexpramipexole showed an excellent safety profile and was found to significantly decrease absolute eosinophil counts (AEC) in 〉70% of the study participants. Consequently, a proof-of-concept study was designed to evaluate the safety and efficacy of dexpramipexole as a steroid-sparing agent in 10 HES subjects. Subjects with HES on steroid monotherapy were eligible for the study if they required ≥10 mg prednisone or equivalent for control of symptoms and eosinophilia. Subjects with AEC
Print ISSN:
0006-4971
Electronic ISSN:
1528-0020
Topics:
Biology
,
Medicine
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