PA-KNOPP-BIOSCIENCES

Knopp Biosciences LLC, a privately held drug discovery and development company focused on delivering breakthrough treatments for inflammatory and neurological diseases with a high unmet need, today announced the expansion of its research collaboration with Cincinnati Children’s Hospital Medical Center to further elucidate the eosinophil-depleting mechanism of action of Knopp’s lead investigational drug, dexpramipexole.

Eosinophils are white-blood cells that play a central role in several debilitating conditions, including asthma, hypereosinophilic syndrome (HES), and other inflammatory diseases. Dexpramipexole has been shown to selectively reduce eosinophil levels in multiple clinical trials, including a Phase 2 study in HES and a Phase 2 trial in chronic rhinosinusitis with nasal polyps . Knopp is advancing dexpramipexole into Phase 2 development in severe asthma and Phase 3 development in HES.

The research at Cincinnati Children’s Hospital Medical Center is being led by Patricia C. Fulkerson, M.D., Ph.D., Associate Professor of Pediatrics, University of Cincinnati College of Medicine, and Cincinnati Children’s Hospital Medical Center, and a leading authority on eosinophil-mediated diseases. Dr. Fulkerson’s lab has developed a number of innovative methods to study the differentiation and maturation of eosinophils. Through a previous collaboration with Knopp, she and her team employed an induced pluripotent stem cell (iPSC) culture system to study the ability of dexpramipexole to inhibit the maturation of eosinophil progenitor cells. Looking forward, Dr. Fulkerson and her team will investigate molecular pathways in eosinophil development and attempt to further elucidate the effect of dexpramipexole on those pathways.

“We are pleased to expand our research collaboration with Dr. Fulkerson to more fully characterize the eosinophil-lowering mechanism of dexpramipexole,” said Michael Bozik, M.D., Chief Executive Officer of Knopp. “Dr. Fulkerson’s expert knowledge of eosinophil biology and her extensive experience in treating children with eosinophil-associated diseases uniquely position her to lead this collaboration that will inform and support the clinical development of dexpramipexole for the treatment of asthma and HES.”

ABOUT KNOPP BIOSCIENCES LLC

Knopp Biosciences, based in Pittsburgh, PA, is a privately held drug discovery and development company focused on delivering breakthrough treatments for inflammatory and neurological diseases with a high unmet need. Knopp’s clinical-stage small molecule, dexpramipexole, is entering Phase 2 development in severe asthma and Phase 3 development in HES. Knopp’s preclinical Kv7 platform is directed to small molecule treatments for neonatal epileptic encephalopathy, other rare epilepsies, tinnitus, and neuropathic pain. Please visit www.knoppbio.com .

Forward Looking Statement

This press release contains "forward-looking statements," including statements relating to planned regulatory filings and clinical development programs. 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 any investigational drug product will be successfully developed or manufactured or that final results of clinical studies will be supportive of regulatory approvals required to market a 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.

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.

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