Knopp Neurosciences Initiates Phase 2 Study Of KNS-760704 In Amyotrophic Lateral Sclerosis

Main Category: Muscular Dystrophy / ALS
Also Included In: Clinical Trials / Drug Trials
Article Date: 10 Apr 2008 - 1:00 PDT

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Knopp Neurosciences Inc. ("Knopp") said it initiated the first Phase 2 study of KNS-760704 in patients with amyotrophic lateral sclerosis (ALS).

The study is designed as a two-part, randomized, double-blind, placebo-controlled study to evaluate the safety and tolerability of KNS-760704 oral tablets. It will be conducted at approximately 20 centers in the U.S.

In Part 1 of the study, approximately 80 ALS patients from ages 21 to 80 whose symptoms started within the past two years will receive 12 weeks of treatment with low-dose, mid-dose, or high-dose KNS-760704 or matching placebo. Participants who complete Part 1 will be eligible for a Part 2 extension in which they will be re-randomized and will receive up to 28 weeks of treatment with either low-dose or high-dose KNS-760704.

Additional study details are available at http://www.clinicaltrials.gov.

Knopp said it plans to initiate a larger proof-of-concept study in 2009 if KNS-760704 is shown to be safe and well-tolerated in this Phase 2a study.

"Following the successful completion of our Phase 1 healthy volunteer studies in late 2007, we're pleased to be advancing KNS-760704 into clinical trials for ALS patients," said Michael Bozik, M.D., president and chief executive officer of Knopp. "This step represents another important milestone for Knopp in our efforts to develop an effective treatment for this relentless disease."

About KNS-760704

KNS-760704 is a chirally pure oral formulation of the synthetic amino-benzothiazole (6R)-4,5,6,7-tetrahydro-N6-propyl-2,6-benzothiazolediamine dihydrochloride monohydrate in clinical development for the treatment of amyotrophic lateral sclerosis (ALS). Like its S(-) optical enantiomer, pramipexole dihydrochloride (Mirapex®), KNS-760704 has been shown to be effective in a range of neuroprotective assays. Unlike Mirapex®, a selective, high affinity dopamine agonist approved for the treatment of Parkinson's disease, KNS-760704 exhibits very limited dopamine receptor affinity, making it possible to clinically evaluate its potential neuroprotective activity over a broad dose range. The compound's use in ALS has received orphan drug designation from the U.S. Food and Drug Administration. Phase 1 studies in healthy volunteers have been completed.

About ALS

Amyotrophic lateral sclerosis, often called Lou Gehrig's disease, 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 30,000 and the incidence is nearly 2 per 100,000. Only a single 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.

Mirapex® is a registered trademark of Boehringer Ingelheim Pharma KG Corporation.

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.

Knopp Neurosciences Inc.