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Steroid-Sparing Effects of Dexpramipexole in Hypereosinophilic Syndromes (HES) Reported at American Society of Hematology 2016 Annual Meeting

December 14, 2016

PITTSBURGH, PA, USA, December 14, 2016—Knopp Biosciences LLC today announced the presentation of data from an ongoing, open-label pilot-study of dexpramipexole in hypereosinophilic syndromes (HES) demonstrating hematological responses, symptom improvement, and steroid sparing in a subset of subjects with steroid-dependent HES.

These data (abstract #1327) were presented at a poster session at the 58th Annual Meeting of the American Society of Hematology (ASH) in San Diego, CA, by investigators from the National Institute of Allergy and Infectious Diseases (NIAID), a branch of the National Institutes of Health. A key goal of treatment for patients with steroid-responsive HES is to reduce the use of corticosteroids while maintaining hematological and symptom control. The study results support the potential of dexpramipexole as a steroid-sparing agent in HES, a heterogeneous group of rare disorders characterized by peripheral eosinophilia and eosinophil-related end organ damage. Although most HES patients respond to steroid therapy, chronic treatment is often necessary, frequently resulting in serious side effects.

Michael Bozik, M.D., President and CEO of Knopp Biosciences, 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."

A team of investigators led by Dr. Amy Klion at NIAID undertook the proof-of-concept study of dexpramipexole as a steroid-sparing agent in subjects with HES. Dexpramipexole had been observed to produce a significant, targeted reduction of peripheral blood eosinophils in earlier clinical trials in amyotrophic lateral sclerosis.

Subjects with HES on steroid monotherapy at a minimally effective corticosteroid dose (MECD) were eligible for the study if they required ≥10 mg prednisone or equivalent for control of symptoms and eosinophilia. Eligible subjects began treatment with dexpramipexole 150 mg twice daily for 12 weeks, after which a standardized corticosteroid taper was initiated and eosinophil counts and symptoms were monitored weekly. The primary efficacy endpoint was defined as a ≥50 percent reduction in MECD to maintain their eosinophil count at or below baseline levels and control clinical symptoms.

Study enrollment is complete. To date, dexpramipexole has been well tolerated and no drug-related serious adverse events or adverse events leading to discontinuation have been observed in the study.

In the abstract, the NIAID investigators noted, "In this pilot study, dexpramipexole showed remarkable efficacy as a steroid-sparing agent without apparent toxicity in a subset of subjects with steroid-responsive HES." The abstract of the ASH presentation is available on line at http://www.bloodjournal.org/content/128/22/1327  Full results from the completed study, including data from the secondary endpoints and bone marrow studies, will be submitted for publication in a peer-reviewed journal.

The study was conducted as part of an agreement between Knopp Biosciences and NIAID, demonstrating an example of industry – government collaboration in the field of rare disease drug development.

Knopp Biosciences, based in Pittsburgh, PA, USA, is a privately held drug discovery and development company focused on delivering breakthrough treatments for inflammatory and neurological diseases of high unmet need in clearly defined patient populations. Our clinical-stage small molecule, dexpramipexole, will be entering Phase 2 clinical studies in eosinophilic asthma. Our preclinical platform is directed to small molecule treatments for a devastating brain disorder of infants, epileptic encephalopathy, caused by a rare mutation in the KCNQ2 gene.  For more information, see www.knoppbio.com.

About Knopp Biosciences LLC

Knopp Biosciences LLC, based in Pittsburgh, PA, USA, is a drug discovery and development company focused on delivering breakthrough treatments for unmet needs in neurology through innovation, experience, and partnership. The company's lead product candidate is dexpramipexole, an orally bioavailable small molecule in Phase 3 development for the treatment of ALS. Biogen Idec holds an exclusive worldwide license from Knopp to develop and commercialize dexpramipexole, with Knopp providing development support and conducting certain U.S. commercialization activities. Knopp's discovery platform is directed to identifying next generation mitochondrial bioenergetic modulators for the treatment of Parkinson's disease and other neurodegenerative disorders.

This press release contains "forward-looking statements," including statements relating to planned regulatory filings and clinical development programs for dexpramipexole. All forward-looking statements are based on management's current assumptions and expectations and involve risks, uncertainties and other important factors, specifically including the uncertainties inherent in clinical trials and product development programs, the availability of funding to support continued research and studies, the availability or potential availability of alternative therapies or treatments, the availability of patent protection for the discoveries and strategic alliances, as well as additional factors that may cause Knopp's actual results to differ from our expectations. There can be no assurance that dexpramipexole will be successfully developed or manufactured or that final results of clinical studies will be supportive of regulatory approvals required to market the product. Knopp undertakes no obligation to update or revise any such forward-looking statements, whether as a result of new information, future events or otherwise.

Media Contact:
Thomas Petzinger Jr.