Company: ProCypra Therapeutics
Drug Type: Small Molecule
Conditions: ALS, Parkinson’s disease
Mechanism Type: SOD1 Metalation
Mechanism: Cu(II)ATSM is a bisthiosemicarbazone which selectively delivers copper to cells with impaired mitochondrial electron transport chains, a characteristic of ALS and many other neurodegenerative diseases. In both SOD1 mouse models of ALS and in ALS patients, scientists have found alterations in copper homeostasis, providing a rationale for testing this candidate in ALS. In a series of publications in mouse models of ALS, Cu(II)ATSM improved motor function and prolonged survival.
U.S. Status for ALS: Phase I
 Phase 1 Dose Escalation and PK Study of Cu(II)ATSM in ALS/MND ClinicalTrials.gov, 8 Mar 2017. Accessed 8 Feb 2018 from https://clinicaltrials.gov/ct2/show/NCT02870634?term=ATSM&rank=2.
 CuATSM protects against the in vitro cytotoxicity of wild type-like SOD1 mutants but not mutants that disrupt metal binding. Farrawell, NE et al. ACS Chem Neurosci. 2018 Nov 21.
 CuATSM efficacy is independently replicated in a SOD1 mouse model of ALS while unmetallated ATSM therapy fails to reveal benefits. Vieira, FG et al. IBRO Reports 2017 Jun; 2:47-53
 Oral treatment with Cu(II)(atsm) increases mutant SOD1 in vivo but protects motor neurons and improves the phenotype of a transgenic mouse model of amyotrophic lateral sclerosis. Roberts, BR et al. J Neurosci. 2014 Jun 4;34(23):8021-31.
Last updated June 29th, 2017