Arimoclomol (Orph-001)

Drug Name: Arimoclomol (Orph-001)
Company: Orphazyme
Location: Denmark
Website: http://www.orphazyme.com
Drug Type: Small molecule
Conditions: ALS
Mechanism Type: Proteostasis, Protein misfolding modulator
Mechanism: Arimoclomol is a small molecule that increases expression of 70-kDa heat shock proteins (Hsp70) and Hsp90s, chaperone proteins that play a role in folding of newly translated proteins and refolding or clearance of misfolded proteins.
U.S. Status for ALS: Phase III

References:
[1] Randomized, double-blind, placebo-controlled trial of arimoclomol in rapidly progressive SOD1 ALS. Benatar, M et al. Neurology. 2018 Jan 24. pii: 10.1212/WNL.0000000000004960.
[2] Arimoclomol at dosages up to 300 mg/day is well tolerated and safe in amyotrophic lateral sclerosis. Cudkowicz, ME et al. Muscle Nerve. 2008 Jul;38(1):837-44.
[3] Late stage treatment with arimoclomol delays disease progression and prevents protein aggregation in the SOD1 mouse model of ALS. Kalmar, B et al. J Neurochem. 2008 Oct;107(2):339-50.
[4] Treatment with arimoclomol, a coinducer of heat shock proteins, delays disease progression in ALS mice. Kieran, D et al. Nat Med. 2004 Apr;10(4):402-5.
[5] Arimoclomol in Amyotrophic Lateral Sclerosis. ClinicalTrials.gov, 9 Apr 2018. Accessed 9 Aug 2018 from https://www.clinicaltrials.gov/ct2/show/NCT03491462.

Last updated August 9th, 2018

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