Search Results

sebetralstat

Context. Sebetralstat (KalVista Pharmaceuticals) is an oral, on-demand plasma kallikrein (PKa) inhibitor intended for hereditary angioedema (HAE). As an on-demand medication, it can be administered to treat acute attacks rather than chronically to prevent attacks. Its structure was first disclosed at the ACS Spring Meeting earlier this year. HAE is a rare autosomal dominant disease…

GDC-2394

Context. GDC-2394 (Genentech) is an oral NLRP3 inflammasome inhibitor. The NLRP3 inflammasome is the best studied and most characterized inflammasome, with the protein playing a key role in the detection of inflammatory danger signals which are a hallmark of many inflammatory diseases. Hyperactivation of the NLRP3 has been implicated in the pathogenesis of several diseases…

AZD1656

Context. AZD1656 (AstraZeneca)is an oral glucokinase (GK) activator (GKA) for hyperglycemia in type 2 diabetes mellitus. Despite GK being first reported more than 50 years ago and subsequent extensive research efforts, a GKA is yet to be approved. Most GKAs have failed in clinical development owing to an increased risk of hypoglycemia and gradual loss…

GB1211

Context. GB1211 (Galecto Biotech) is an oral galectin-3 inhibitor under development for fibrotic disease. First described in 1971, galectin-3, a unique subtype of a 15-member family of carbohydrate-binding proteins, has been implicated in a host of different diseases, such as cancer, liver cirrhosis, and atherosclerosis. Incredibly, over 8000 papers featuring the protein have since been…

compound 7

Context. “Compound 7” (NIBR) is an oral Trypanosoma brucei 20S proteasome inhibitor being developed for Human African Trypanosomiasis (HAT). African sleeping sickness, or HAT, is caused by a parasite in the genus Trypanosoma.  The parasite is spread by the Tsetse fly, and the lifecycle of the parasite involves a phase in the blood of the…

ACT-660602

Context. ACT-660602 (Idorsia Pharmaceuticals) is an oral chemokine receptor CXCR3 antagonist being developed for autoimmune diseases. Chemokines and their receptors make up the signaling system that is responsible for immune cell migration. This is important for the innate immune system’s ability for fighting infection, but dysregulation of these pathways can lead to autoimmune disorders, especially…