Knopp Neurosciences Receives Orphan Designation For KNS-760704 For Treatment Of ALS
Main Category: Muscular Dystrophy / ALS News
Article Date: 16 Oct 2007 - 0:00 PDT


Knopp Neurosciences Inc. ("Knopp") announced that the U.S. Food and Drug Administration's Office of Orphan Products Development has granted orphan drug designation to KNS-760704 for the treatment of amyotrophic lateral sclerosis (ALS).

KNS-760704 is now completing Phase 1 studies to evaluate the safety, tolerability and pharmacokinetics of the compound in healthy human subjects. Knopp expects to initiate Phase 2 studies in ALS patients in the first quarter of 2008.

"Congress passed the Orphan Drug Act because it recognized that adequate drugs for many rare diseases have not been developed," said Michael Bozik, M.D., president and CEO of Knopp. "The designation of KNS-760704 as an orphan drug underscores the importance of developing effective treatments for patients with ALS."

About KNS-760704

KNS-760704 is an orally administered small molecule in clinical development by Knopp for the treatment of amyotrophic lateral sclerosis (ALS). The drug is an optical enantiomer of a selective, high affinity dopamine agonist marketed in other neurological indications. Both KNS-760704 and the marketed agonist demonstrate neuroprotective properties independent of dopamine receptor activity, but KNS-760704 exhibits greatly reduced dopamine receptor affinity. This makes it possible to clinically evaluate the potential neuroprotective activity of KNS-760704 over a broad dose range.

About ALS

Amyotrophic lateral sclerosis (ALS), 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 nearly 30,000 and the incidence is 1.2 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.

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.
http://www.knoppneurosciences.com