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Compound 20

Context. “Compound 20” (Gedeon Richter) is an oral α5-GABAA receptor negative allosteric modulator (NAM) being developed for cognitive disorders. GABA receptor NAMs are known to exert effects opposite to the anxiolytic and anticonvulsant effects of positive allosteric modulators. However, NAM agents targeting α5-GABAA receptors, which have been implicated in learning and memory, have been heavily…

INCB13739

Context. INCB13739 (Incyte) is an oral 11β-hydroxysteroid dehydrogenase 1 (11β-HSD1) antagonist. Inhibition of the tissue-specific generation of cortisol using agents that antagonize 11β-HSD1, the key metabolic enzyme involved in the conversion of inactive glucocorticoids to active ones, has been considered a desirable therapeutic strategy for type 2 diabetes and metabolic syndrome. Indeed, at least 25…

Sudapyridine (WX-081)

Context. Sudapyridine (WuXi AppTec) is an oral mycobacterial ATP synthase inhibitor being developed for tuberculosis (TB). Despite the significant success achieved with the discovery of antibiotic drugs in the last several decades, drug resistance, particularly related to agents targeting organisms such as M. tuberculosis, remains a predominant challenge. To make matters worse, potent agents such…

ABBV-318

Context. ABBV-318 (AbbVie) is a CNS-penetrating, oral inhibitor of Nav1.7/Nav1.8 being developed for pain treatment. Despite being a highly validated pain target due to its important role in excitable cells, Nav1.7 inhibition has not lived up to its potential, and translation of the promising preclinical observations to human studies has been elusive. A close isoform,…

INE963

Context. INE963 (Novartis Institute for Tropical Diseases) is an oral, single dose, fast-acting blood-stage antimalarial candidate. The molecule was identified using a phenotypic high-throughput screening approach which has been successfully used to identify new antimalarial chemotypes. Phenotypic screens can be preferable to target-based screens in antimalarial drug discovery because the emergence of antimalarial drug resistance…

branaplam

Context. Branaplam (Novartis Institutes for Biomedical Research) is an oral brain-penetrant splicing modulator, originally developed as a small molecule splicing modulator for spinal muscular atrophy (SMA). It turns out to also lower huntingtin levels and is being developed (with Fast Track Designation) for patients with Huntington’s Disease which is driven by mutant huntingtin prion. It…