Dexpramipexole

Dexpramipexole
Dexpramipexole
IUPAC name
(R)-N6-propyl-4,5,6,7-tetrahydrobenzo[d]thiazole-2,6-diamine
Identifiers
CAS number 104632-28-2, 
104632-27-1 (Dihydrochloride)
Jmol-3D images Image 1
Properties
Molecular formula C10H17N3S
Molar mass 211.33 g mol−1
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Except where noted otherwise, data are given for materials in their standard state (at 25 °C, 100 kPa)
Infobox references

Dexpramipexole (KNS-760704) is an experimental drug being investigated by Knopp Biosciences and Biogen Idec for the treatment of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease. In September 2009, it received Fast Track designation for ALS from the U.S. Food and Drug Administration (FDA).[1] It has received Orphan Drug designation by both the FDA and the European Medicines Agency. Dexpramipexole is a low molecular weight, water-soluble, orally bioavailable, renally excreted compound with linear pharmacokinetics, and has been shown to be well tolerated in human subject testing.[2]

KNS-760704 is the chirally pure form of dexpramipexole, which has been shown to improve mitochondrial function and to confer significant cellular protection in neurons under stress.[3] KNS-760704 was originally identified as a candidate therapy for ALS by James Bennett, M.D., Ph.D., then of the University of Virginia.

A 2010 Phase II clinical trial involving 102 patients showed a slowing of ALS disease progression.[4] Phase III clinical trials have been initiated.

See also

References

  1. ^ FDA Fast Track KNS-760704 for ALS, September 2, 2009
  2. ^ Safety, Tolerability, and Pharmacokinetics of KNS-760704 (Dexpramipexole) in Healthy Adult Subjects. Michael E. Bozik, James L. Mather, William G. Kramer, Valentin K. Gribkoff and Evan W. Ingersoll. J Clin Pharmacol. Published online 19 October 2010.
  3. ^ KNS-760704 [(6R)-4,5,6,7-tetrahydro-N6-propyl-2, 6-benzothiazole-diamine dihydrochloride monohydrate] for the Treatment of Amyotrophic Lateral Sclerosis. Valentin K. Gribkoff and Michael E. Bozik. CNS Neuroscience & Therapeutics 14 (2008) 215–226.
  4. ^ Robinson, Richard (2010). "New Als Drug Shows Dose-Dependent Efficacy in Phase 2 Trial". Neurology Today 10 (13): 1. doi:10.1097/01.NT.0000384108.10957.21. http://journals.lww.com/neurotodayonline/Fulltext/2010/07010/New_Als_Drug_Shows_Dose_Dependent_Efficacy_in.2.aspx. 

External links


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