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Knopp Neurosciences to Present Phase 2 Results of KNS-760704 in ALS

August 11, 2009

PITTSBURGH, PA, USA, AUGUST 11, 2009—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.

About Knopp Neurosciences Inc.

Knopp Neurosciences 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 (December 8-10, 2009, 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).


Louise Coxon
Communications Manager
Motor Neurone Disease Association

Anja Alexandersson
Assistant General Manager
Deutsche Gesellschaft für Muskelkranke e.V.
(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.