Kopp Biosciences will study dexpramipexole in a Phase II clinical trial involving patients with severe eosinophilic asthma after partnering with BEAT Severe Asthma consortium in the UK. Dexpramipexole is an oral, small molecule that demonstrated ability to selectively lower eosinophil levels in various clinical trials, including Phase II studies for hypereosinophilic syndrome (HES) and chronic rhinosinusitis with nasal polyps. The company will advance the therapeutic into Phase II development for severe asthma and Phase III development in HES.
Dexpramipexole (KNS-760704) is a drug that was investigated by Knopp Biosciences and Biogen Idec for the treatment of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease. The drug failed to show efficacy in terms of function or survival in a Phase III study of patients with ALS.
More recently, dexpramipexole was shown in a Phase 2 open-label trial to reduce eosinophil counts and eliminate the need for corticosteroid treatment in a subset of patients with steroid-responsive hypereosinophilic syndrome. Knopp Biosciences is advancing the drug in both HES and eosinophilic asthma.
In September 2009, dexpramipexole received Fast Track designation for ALS from the U.S. Food and Drug Administration (FDA). It has received Drug designation by both the FDA and the European Medicines Agency. Dexpramipexole is a low molecular weight, water-soluble, orally bioavailable, renally excreted compound with linear pharmacokinetics, and has been shown to be well tolerated in human subject testing.
KNS-760704 is the enantiomer of pramipexole, and has been shown to improve mitochondrial function and to confer significant cellular protection in neurons under stress. KNS-760704 was originally identified as a candidate therapy for ALS by James Bennett, M.D., Ph.D., then of the University of Virginia.
In January 2013, Biogen Idec announced that it was discontinuing its development of dexpramipexole in ALS due to lack of efficacy in a Phase III study.
Knopp Biosciences Background
Founded in 2005, Knopp Biosciences (fka: Knopp Neurosciences) has raised $106 million. They are a drug discovery and development company focused on delivering breakthrough treatments in neurological disorders through innovation, experience and partnership. Knopp’s lead compound, KNS-760704, is currently in clinical development for amyotrophic lateral sclerosis, with additional indications under investigation.