Knopp Neurosciences Inc. said the results of a Phase 2 safety and tolerability study of KNS-760704 in amyotrophic lateral sclerosis (ALS) will be presented at the 20th International Symposium on ALS/MND in Berlin on December 9, 2009.
The Phase 2 study was a randomized, placebo-controlled, double-blind trial to evaluate the safety, tolerability, and clinical effects of three dosage levels of KNS-760704 versus placebo for 12 weeks. Enrollment totaled 102 subjects across 20 U.S. study centers.
The results will be presented in an oral session by Merit Cudkowicz, M.D., Associate Professor of Neurology at Harvard Medical School and a principal investigator in the study. Dr. Cudkowicz co-directs the ALS clinic and the Neurology Clinical Trials Unit at Massachusetts General Hospital and Partners Healthcare System.
Knopp Neurosciences believes the Phase 2 study demonstrated encouraging trends that support evaluation of KNS-760704 in a large, well-powered Phase 3 study, which the company expects to initiate in North America and Europe in 2010. As noted by Dr. Cudkowicz, “Further testing in a large, definitive trial is warranted to extend the results shown in Phase 2 and to establish that KNS-760704 is an effective treatment for ALS.” Knopp Neurosciences expects to review the Phase 2 results with the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMEA) prior to initiating Phase 3 studies.
The 98 subjects who completed the study were eligible for re-randomization in a double-blind extension phase to assess the safety, tolerability, and clinical effects of two dosage levels of KNS-760704 for six months, without placebo control. Following their completion of the six-month extension, which remains ongoing, subjects are eligible to participate in an open-label, 48-week extension phase in which all participants will receive the highest dose of KNS-760704 tested.