Knopp Neurosciences Presents Results of Phase 1 Ascending Single-Dose Study of KNS-760704
December 3, 2007
TORONTO, Ontario, December 3, 2007--Knopp Neurosciences Inc. ("Knopp") presented results of its ascending single-dose study of KNS-760704, an orally bioavailable small molecule therapy in development for amyotrophic lateral sclerosis (ALS), at the 18th International Symposium on ALS/MND in Toronto.
The Phase 1 study was a single-center, randomized, double-blind, placebo-controlled study to determine the safety and tolerability of ascending single doses of KNS-760704. Results of the study demonstrate that single oral doses up to 300 mg of KNS-760704 are safe and well-tolerated in healthy adult volunteers.
Secondary objectives involved assessment of single-dose pharmacokinetics and the effect of food on drug absorption and elimination. Pharmacokinetic results indicate that KNS-760704 is rapidly absorbed, with concentrations increasing in proportion to dose. The elimination half-life is independent of dose. A high-fat meal had no effect on the absorption and elimination of KNS-760704.
A single 300 mg dose of KNS-760704 is 2,400 times higher than the recommended starting dose of Mirapex® (pramipexole), which is the (S)-optical enantiomer of KNS-760404. Mirapex® is a selective, high affinity dopamine agonist approved for the treatment of Parkinson's disease and restless legs syndrome. Both KNS 760704 and Mirapex® demonstrate neuroprotective properties independent of dopamine receptor activity, but KNS 760704 exhibits greatly reduced dopamine receptor affinity.
"We are encouraged that based on this study, KNS-760704 may enable neuroprotective dose levels to be rapidly achieved without the need for titration," said Michael Bozik, M.D., president and CEO of Knopp Neurosciences.
Knopp's ascending multiple-dose study of KNS-760704 in healthy volunteers is ongoing.
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. The compound's use in ALS has received orphan drug designation from the U.S. Food and Drug Administration.
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 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.
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.
Thomas Petzinger Jr.