arimoclomol

Ligand id: 8271

Name: arimoclomol

Structure and Physico-chemical Properties

2D Structure
Calculated Physico-chemical Properties
Hydrogen bond acceptors 2
Hydrogen bond donors 1
Rotatable bonds 6
Topological polar surface area 72
Molecular weight 313.12
XLogP 1.42
No. Lipinski's rules broken 0

Molecular properties generated using the CDK

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 II/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].