U.S. Department of Health & Human Services Divider Arrow National Institutes of Health Divider Arrow NCATS
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Showing 1 - 10 of 39 results

Status:
US Approved Rx (2024)
First approved in 2024

Class (Stereo):
CHEMICAL (ABSOLUTE)



Arimoclomol citrate is an experimental drug developed by a biopharmaceutical company CytRx Corporation. In 2011 the worldwide rights to arimoclomol were bought by Danish biotech company Orphazyme ApS. The European Medicines Agency (EMA) and U.S. Food...
Status:
US Approved Rx (2024)
First approved in 2017

Class (Stereo):
CHEMICAL (ACHIRAL)



Edaravone is a free radical scavenger developed for the treatment of amyotrophic lateral sclerosis.
Status:
US Approved Rx (2016)
First approved in 1995

Class (Stereo):
CHEMICAL (ACHIRAL)



Riluzole, a member of the benzothiazole class, is indicated for the treatment of patients with amyotrophic lateral sclerosis. Its pharmacological properties include the following, some of which may be related to its effect: 1) an inhibitory effect on...
Status:
Investigational

Class (Stereo):
CHEMICAL (ABSOLUTE)


PYM50018 (also known as Myogane or SARSAGENIN) has demonstrated neuroprotective effects in several preclinical models. It was observed that PYM50018 protects against neuronal damage, increases neurite outgrowth, reverses oxidative damage and reversed...
Status:
Investigational

Class (Stereo):
CHEMICAL (ABSOLUTE)



Dexpramipexole (also known as KNS-760704/R-pramipexole) was originally developed by University of Virginia researchers to treat Amyotrophic Lateral Sclerosis and then was licensed to global biotechnology company Biogen Idec for further development. H...
Status:
Investigational

Class (Stereo):
CHEMICAL (ACHIRAL)



Cutamesine, an agonsit of brain sigma 1 receptors, was developed by Santen Pharmaceutical for the treatment of cognitive diseases. The drug was tested in phase II in patients with major depressive disorders and for recovery of patients with stroke, h...
Status:
Investigational

Class (Stereo):
CHEMICAL (ABSOLUTE)



Sodium taurodeoxycholate is a bile salt-related, anionic detergent used for isolation of membrane proteins including inner mitochondrial membrane proteins. It is formed by the conjugation of ursodeoxycholic acid (UDCA) with taurine. Sodium taurodeoxy...
Status:
Investigational

Class (Stereo):
CHEMICAL (ACHIRAL)



Tirasemtiv is a fast skeletal troponin activator and is a candidate amyotrophic lateral sclerosis (AML) therapeutic. It is a small molecule that enhances the signals between motor neurons and neuromuscular junctions. Tirasemtiv increases muscle stren...
Status:
Investigational

Class (Stereo):
CHEMICAL (ABSOLUTE)



Talampanel (TLP) was developed as a noncompetitive (allosteric) antagonist of the AMPA receptor. Talampanel does not act directly on the AMPA receptor, but at an allosteric site referred to as the GYKI receptor. Talampanel is being studied in the tre...
Status:
Investigational

Class (Stereo):
CHEMICAL (ACHIRAL)



Y-700 (Niraxostat or 1-[3-Cyano-4-(2,2-dimethylpropoxy)phenyl]-1H-pyrazole-4-carboxylic acid) is an inhibitor of xanthine oxidoreductase. Y-700 demonstrated high oral bioavailability being predominantly eliminated via the liver. It potently reduces s...