Clinical results from Phase 2 study of KNS-760704 presented by Knopp Neurosciences

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 20^th 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.