Knopp Biosciences reports positive data from pilot study of dexpramipexole to treat HES


US-based drug discovery and development company Knopp Biosciences has reported positive data from its pilot study of dexpramipexole to treat patients with hypereosinophilic syndromes (HES).

Dexpramipexole has been developed as a small molecule immunological therapeutic to modulate white blood cells known as eosinophils.

It results in significant and persistent reduction of eosinophil levels within blood and inflammed tissues.

An increased level of eosinophils is attributive to allergic and other inflammatory disorders, including asthma, certain chronic gastrointestinal diseases and HES.

The 24-week, open-label clinical trial was held as a part of an agreement between Knopp Biosciences and the National Institute of Allergy and Infectious Diseases (NIAID).

It involved HES subjects receiving corticosteroid therapy who were administered with dexpramipexole 150mg on a twice-daily basis, after which a standardised corticosteroid taper was started and eosinophil counts and symptoms were monitored weekly.

"Given the deleterious effects of chronic steroid use, the need for well-tolerated alternate treatments in this rare disease is clear."

The study was primarily focused on achieving reduction in minimally effective corticosteroid dose (MECD) to maintain their eosinophil count at or below baseline levels and control clinical symptoms.

Findings from the study suggested efficacy of dexpramipexole in inducing hematological responses, symptom improvement, and steroid sparing in a subset of subjects afflicted with steroid-dependent HES.

Knopp Biosciences president and CEO Michael Bozik said: “We are very encouraged to see evidence of steroid-sparing and clinical improvement in a subset of HES subjects receiving dexpramipexole in this pilot study.

“Given the deleterious effects of chronic steroid use, the need for well-tolerated alternate treatments in this rare disease is clear.

“We look forward to completing this study and to discussing the potential for further evaluations of dexpramipexole in HES with our colleagues at NIAID.”

Dexpramipexole has also demonstrated efficacy in a resulting significant and targeted reduction of peripheral blood eosinophils within previous clinical trials, which treated patients with amyotrophic lateral sclerosis.