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Knopp Biosciences Receives Key Patent Directed to Treatment for Hypereosinophilic Syndromes, Asthma, and Other Eosinophilic Diseases


Knopp Biosciences LLC today announced it has received United States Patent No. 9,468,630 entitled “Compositions and Methods for Treating Conditions Related to Increased Eosinophils.”

The patent relates to the company’s clinical-stage small molecule dexpramipexole, which Knopp expects to advance into pivotal trials in hypereosinophilic syndromes (HES) and Phase 2 trials in eosinophilic asthma in 2017. U.S. Patent No. 9,468,630 specifically claims methods of treating HES and asthma by administering therapeutically effective amounts of dexpramipexole. Issued claims are also directed to methods of treating nasal polyposis, eosinophilic gastroenteritis, eosinophilic esophagitis, and atopic dermatitis, by administering therapeutically effective amounts of dexpramipexole. Eosinophils are white blood cells associated with inflammation.

“Issuance of this patent further strengthens our efforts to advance dexpramipexole as a small molecule treatment for HES, eosinophilic asthma, and other eosinophil-associated diseases,” said Michael Bozik, M.D., President and CEO of Knopp. “Recent drug approvals have confirmed the benefit of lowering eosinophil levels to reduce the risk of asthma exacerbations through the infusion or injection of monoclonal antibodies. We are developing dexpramipexole to provide the benefits of an oral, small molecule alternative for lowering eosinophils in HES and asthma.”

Two ongoing, proof-of-concept Phase 2 trials are currently characterizing the eosinophil-lowering effects of dexpramipexole in eosinophil-associated diseases. A 20-subject, multi-center, pilot study of dexpramipexole is nearing completion in subjects with chronic rhinosinusitis and nasal polyps with eosinophilia. A separate pilot study under the sponsorship of the National Institutes of Health is evaluating dexpramipexole as a steroid-sparing agent in hypereosinophilic syndromes (HES).

The targeted eosinophil-lowering effects of dexpramipexole were previously observed at doses of dexpramipexole well tolerated in earlier clinical studies of more than 1,000 subjects with amyotrophic lateral sclerosis (ALS).

Prosecution of the patent was led by Raymond A. Miller and N. Nicole Stakleff of the Intellectual Property Department of Pepper Hamilton LLP.


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, is entering Phase 2 clinical studies in eosinophilic asthma. Our preclinical platform is directed to small molecule treatments for neonatal epileptic encephalopathy, a devastating brain disorder of infants caused by a rare mutation in the KCNQ2 gene. For more information, see


Pepper Hamilton LLP is a multi-practice law firm with more than 450 lawyers nationally. The firm provides corporate, litigation, and regulatory legal services to leading businesses, governmental entities, nonprofit organizations, and individuals throughout the nation and the world. The firm was founded in 1890. For more information, see

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.

This release contains “forward-looking statements,” including statements that may relate to Knopp’s plans regarding regulatory filings and clinical development programs for dexpramipexole and other compounds and future financing and strategic alternatives. 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 potential alternative therapies or treatments, the availability of patent protection for the discoveries and the availability of 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 or any other compound will be successfully developed or manufactured or that final results of clinical studies will be supportive of regulatory approvals required to market the products. Knopp undertakes no obligation to update or revise any such forward-looking statements, whether as a result of new information, future events, or otherwise.

Contact information

Knopp Biosciences LLC
Tom Petzinger

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