PA-KNOPP-BIOSCIENCES

Knopp Biosciences LLC, a privately held drug discovery and development company focused on delivering breakthrough treatments for inflammatory and neurological diseases of high unmet need, today announced poster and oral presentations on data for its small molecule immunological therapy, dexpramipexole, at the 11^th Biennial Symposium of the International Eosinophil Society to be held in Portland, Oregon, on July 9-13.

Poster Presentation Title: Dexpramipexole Responsiveness is Increased in Eosinophilic Patients
Date & Time: July 10, 5:00 pm PDT

Oral Presentation Title: Developing Dexpramipexole for the Treatment of Eosinophilic Disorders
Speaker: Calman Prussin, M.D., Vice President, Clinical and Translational Medicine, Knopp Biosciences
Date & Time: July 13, 8:30 am PDT

Dr. Prussin will present an analysis of the eosinophil-lowering effects of dexpramipexole seen in a Phase 3 randomized, double-blind, placebo-controlled study of dexpramipexole in amyotrophic lateral sclerosis (ALS) conducted at 81 centers in 11 countries. The study included 419 dexpramipexole-treated patients. The analysis showed that the magnitude of eosinophil lowering by dexpramipexole increased in a stepwise manner in subjects with greater baseline eosinophil counts, with the greatest effect seen in subjects with baseline eosinophilia.

The findings help explain the greater eosinophil lowering seen in previous trials of dexpramipexole in eosinophil-associated diseases relative to ALS. These results indicate that dexpramipexole is most effective in a milieu of greater pro-eosinophilia signaling, further suggesting that dexpramipexole acts on a pathway driven by inflammatory signaling molecules such as IL-5. Irrespective of baseline eosinophil count, absolute eosinophil count was lowered to the 0.02-0.03x10⁹ /L range, suggesting that dexpramipexole may have minimal effects on non-IL-5-driven homeostatic eosinophil populations.

In his oral presentation, Dr. Prussin will present an overview of data on the eosinophil-lowering effects of dexpramipexole observed in clinical studies for hypereosinophilic syndrome (HES) and chronic rhinosinusitis with nasal polyps (CRSwNP).

Eosinophils are white-blood cells that play a central role in a number debilitating conditions beyond ALS, including asthma, HES and other inflammatory diseases. Knopp has reported Phase 2 clinical trial results in HES and CRSwNP and is initiating a Phase 2 trial in severe eosinophilic asthma.

ABOUT KNOPP BIOSCIENCES LLC

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

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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