Synonyms: BRX-220 | BRX220
Compound class:
Synthetic organic
Comment: Arimoclomol is an oral therapeutic candidate for the treatment of amyotrophic lateral sclerosis (ALS). Arimoclomol may be delivered as the citrate salt (BRX-345 with PubChem CID 72710735)
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No information available. |
Summary of Clinical Use |
The EMA and FDA have granted arimoclomol orphan drug status for the treatment of the neurodegenerative disease amyotrophic lateral sclerosis (ALS, aka Lou Gehrig's disease). A Phase 2/III clinical trial assessing arimoclomol in ALS (NCT00706147) is ongoing. |
Mechanism Of Action and Pharmacodynamic Effects |
Arimoclomol acts as a coinducer of heat shock proteins (Hsps) [2] and this action has been reported to slow the process of motor neuron death and extend life in cellular and whole animal models of ALS [1,3-5,7]. Arimoclomol is reported to prolong the activation of heat shock transcription factor 1 (HSF1; Q00613), the master regulator of stress-inducible Hsps, including Hsp70 and Hsp90 [2]. Increased Hsp70 expression is believed to be responsible for the neuroprotective effect of arimoclomol in ALS mice [6]. |
Clinical Trials | |||||
Clinical Trial ID | Title | Type | Source | Comment | References |
NCT00706147 | Phase II/III Randomized, Placebo-controlled Trial of Arimoclomol in SOD1 Positive Familial Amyotrophic Lateral Sclerosis | Phase 2/Phase 3 Interventional | University of Miami |