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Knopp Neurosciences Reports Presentation of Encouraging Clinical Trends in a Phase 2 Study of KNS-760704 in ALS

December 9, 2009

BERLIN, December 9, 2009--Knopp Neurosciences Inc. ("Knopp") announced the presentation today of encouraging clinical results in a Phase 2 safety and tolerability study of KNS-760704 in amyotrophic lateral sclerosis ("ALS").  The results were presented at the 20th International Symposium on ALS/MND in Berlin, Germany, by Merit Cudkowicz, M.D., Associate Professor of Neurology at the Massachusetts General Hospital of Harvard Medical School.

The two-part Phase 2 study found that KNS-760704 was safe and well-tolerated in ALS patients for up to nine months.  The study results also showed trends suggesting the potential for improved outcomes in function and survival. Knopp emphasized that the compound remains early in its development and that further testing in large, long-term, well-controlled Phase 3 studies is required to establish the necessary evidence that the drug is both safe and effective for patients with ALS.

"Knopp is very encouraged by the results presented today in Berlin and, at the same time, we're acutely aware of the work that remains to be done," said Michael Bozik, M.D., the President and CEO of Knopp. "We look forward to confirming and extending these results in Phase 3 studies, which we hope to initiate in 2010." Knopp has formally engaged regulators in the U.S. and European Union to obtain scientific input on its planned Phase 3 program, including a scheduled meeting with the U.S. FDA in January 2010.

Added Dr. Cudkowicz:  "The safety results and the trends in improved functional and survival outcomes observed in this study provide preliminary evidence supporting the ongoing evaluation of KNS-760704 in Phase 3 clinical trials."

The primary objective of the Phase 2 study was to assess the safety and tolerability of KNS-760704 in ALS subjects for up to nine months. Secondary objectives included measuring the clinical effects of KNS-760704 on functional decline and mortality. The two-part design of the study provided the opportunity to assess the effects of KNS-760704 in the same sample of ALS subjects in two randomized, double-blind treatment periods separated by a one-month placebo washout.

In Part 1 of the study, 102 subjects received daily doses of 50 mg, 150 mg, or 300 mg of KNS-760704 or placebo for 12 weeks. KNS-760704 showed a dose-dependent trend in slowing the rate of disease progression as measured by the difference in slopes of ALS Functional Rating Scale-Revised (ALSFRS-R) across treatment groups, with the greatest benefit observed in the 300 mg dose group.

In Part 2 of the study, 92 subjects were re-randomized to receive daily doses of 50 mg or 300 mg of KNS-760704 for 24 weeks.  In addition to results again suggesting a dose-dependent trend in slowing the rate of disease progression as measured by the ALSFRS-R, there was also a trend toward a survival benefit in the 300 mg group compared with the 50 mg group. In an exploratory test combining mortality and functional outcomes, subjects in the 300 mg group had a significantly improved outcome compared with the 50 mg group.

About KNS-760704

KNS-760704 is a low molecular weight benzothiazole shown to improve mitochondrial function and to confer significant cellular protection in neurons under stress. The chirally pure form of the synthetic benzothiazole (6R)-2-amino-4,5,6,7-tetrahydro-6-(propylamino) benzothiazole, KNS-760704 is highly orally bioavailable, water soluble, renally excreted, and only moderately protein bound. The compound has received orphan drug designation from the U.S. Food and Drug Administration and the European Commission for the treatment of patients with ALS, as well as Fast Track designation from the FDA.

About ALS

Amyotrophic lateral sclerosis, also known as Lou Gehrig's disease and Charcot's sclerosis, is a rapid, universally fatal neurodegenerative disorder characterized by progressive muscle weakness and wasting. ALS affects adults in the prime of life and creates a substantial burden for caregivers. U.S. prevalence is approximately 20,000 and the global incidence is approximately two per 100,000. Only one drug has been approved for the treatment of ALS. Life expectancy after symptom onset is usually three to five years.

About Knopp Neurosciences Inc.

Knopp Neurosciences, based in Pittsburgh, PA, USA, is a drug discovery and development company focused on delivering breakthrough treatments for neurological disorders through innovation, experience, and partnership. The company's lead product candidate is KNS-760704, an orally bioavailable small molecule in development for the treatment of ALS. Knopp's leadership includes experienced neuroscience drug development and discovery executives formerly associated with major pharmaceutical companies. Knopp's financing has been led by Saturn Capital Inc. of Boston as placement agent and Saturn Partners II as lead funder.

About the 20th International Symposium on ALS/MND (Berlin, Germany)

The International Symposium on ALS/MND is a unique annual event that brings together leading international researchers and health and social care professionals to present and debate key innovations in their respective fields. The International Symposium is firmly established as the premier forum for those interested in improving care, understanding disease pathogenesis and developing novel treatments for ALS/MND. The international exchange of knowledge and strategies is essential to provide the best possible care for people with ALS/MND and to bring us closer to a day when no one has to live with the devastating impact of ALS/MND. The Motor Neurone Disease (MND) Association in Northampton, United Kingdom, organises the International Symposium on ALS/MND. The 2009 event is being hosted by the Deutsche Gesellschaft für Muskelkranke e.V. (DGM) (German neuromuscular diseases Association).

This press release contains "forward-looking statements," including statements relating to Knopp's planned regulatory filings and clinical development programs for KNS-760704. 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 KNS-760704 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.

Media Contact:
Thomas Petzinger Jr.