Knopp Neurosciences Receives Orphan Designation For KNS-760704 For Treatment Of ALS
FDA Office of Orphan Products Development has granted orphan drug designation to Knopp Neurosciences's 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."
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.
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.