Clinical Programs: Immunology and Hematology
During the development of dexpramipexole for ALS, we extensively studied the safety and tolerability of the drug in both animals and humans. These tests included the collection of blood at regular intervals (typically every month) for standard laboratory safety assessments, including differential counts of circulating white blood cells, which play a primary role in the protection against infection and allergic insult.
The rationale for advancing dexpramipexole in HES is based on the repeated observation in controlled preclinical studies and clinical trials that dexpramipexole markedly reduces the levels of peripheral blood eosinophils. The eosinophil-lowering effects of dexpramipexole have also been demonstrated in Phase 2 studies in hypereosinophilic syndromes and chronic rhinosinusitis with nasal polyps. This rationale is strengthened by studies encompassing more than 1,000 human subjects showing that dexpramipexole is generally well tolerated, without increased incidence of infection, at doses relevant for testing in HES.
Current preclinical research by Knopp is assessing the effect of dexpramipexole on eosinophil levels in bone marrow and target tissues, its role in modulating eosinophil maturation, and its potential relevance in pathological conditions in which high levels of eosinophils are correlated with disease severity.
Publications of Interest include:
- The targeted eosinophil-lowering effects of dexpramipexole in clinical studies
- Effects of Dexpramipexole on White Blood Cells in a Minipig Toxicology Studyand from Two Clinical Trials in Patients with ALS
- Safety of Dexpramipexole for the Treatment of ALS: Results from the Randomized, Double-blind, Placebo-Controlled study EMPOWER
- Dexpramipexole As a Steroid-Sparing Agent in Hypereosinophilic Syndromes (HES): An Open-Label Proof-of-Concept Study
- Dexpramipexole Effectively Lowers Blood and Tissue Eosinophils in Subjects with Chronic Rhinosinusitis with Nasal Polyps
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.