Knopp Neurosciences to present phase 2 results of drug candidate in amyotrophic lateral sclerosis

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.

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. In Phase 1 studies, the compound was shown to be safe and well tolerated in healthy human subjects. KNS-760704 has received orphan drug designation from the U.S. Food and Drug Administration and the European Medicines Agency for the treatment of patients with ALS.

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. Worldwide prevalence estimates of ALS range from 2 to 9 people 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.


  1. ivette rivera ivette rivera Puerto Rico says:

    For 2 years I've been living with amyotrophic lateral sclerosis.  I've seen my body degenerate and I've lost my independence.  I'm only 41 years old.

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