We are happy to continue our series of posts which are inspired by Drug Hunter’s “Molecules of the Month”!
Here are two molecules that were recently highlighted by Drug Hunter!

1. Ervogastat (PF-06865571) is a potent and selective inhibitor of diacylglycerol acyltransferase (DGAT2). DGAT enzymes catalyze the terminal step in triglyceride biosynthesis, the accumulation of which in the liver can lead to non-alcoholic fatty liver disease (NAFLD), and further to non-alcoholic steatohepatitis (NASH). Currently, NASH is a disease with unmet needs, which is characterized by hepatocyte damage, inflammation, and fibrosis. Ervogastat works systematically and reduces steatosis and hepatic triglyceride levels. It has emerged from improving the pharmacokinetic properties of a previous clinical candidate by scaffold hopping in a ring system and substituent replacing near amide group. Now, ervogastat is in Phase 2 of clinical trials as a first-in-class DGAT2 inhibitor in the treatment of NASH.

You can find the analogs to this molecule here!

2. PTC258 is a novel, orally available, and blood-brain barrier penetrating compound that corrects splicing defects in the Elongator complex protein 1 (ELP1) gene. Given mutation leads to the exon 20 skipping and reduction of ELP1 protein in the nervous system and causes Familial Dysautonomia (FD). This rare neurodegenerative disease is associated with severe gait ataxia, and retinal degeneration and can lead to unexplained death. PTC258 is an example of the second-generation splicing modulator compounds (SMCs), the result of kinetin derivatives optimization. It shows the restoration of proper ELP1 splicing in a phenotypic mouse model, recovers protein levels in the central and peripheral nervous systems, and prevents progressive neuronal degeneration.

You can find the analogs to this molecule here!

Use chem-space.com/search to find off-the-shelf and Make-on-Demand molecules of your interest!