Congruence Therapeutics Inc. has closed a $39.5 million financing to advance into its portfolio of small-molecule correctors for diseases of protein misfolding into the clinic.

Kalohexis LLC has launched as a spin-out from Endevica Bio Inc. with the goal of advancing the clinical development of a portfolio of drug candidates harnessing the melanocortin system for the treatment of metabolic disorders such as obesity and cancer cachexia.

Neurodegenerative disease and cognitive decline cannot be explained by a single process. Beta-amyloid plaques, hyperphosphorylated tau, alpha-synuclein, activated microglia and astrocytes, altered receptors such as TREM2, mitochondrial dysfunction, epigenetic changes and cerebrovascular alterations all seem to contribute to the development of dementia in Alzheimer’s disease (AD). While scientists attempt to address each of these elements, prevention is growing as a primary goal.

Ascletis Pharma Inc. has selected ASC-39, an amylin-selective oral small-molecule amylin receptor agonist, as a clinical development candidate.

Drugs that mimic GLP-1 are widely used to treat diabetes and obesity, but it is not fully understood exactly how they produce many of their beneficial effects. Investigators from The Salk Institute for Biological Studies published a paper in Proceedings of the National Academy of Sciences shedding some light on the mechanisms. “Understanding this process more clearly could help researchers design the next generation of GLP-1-based treatments that are even more precise and effective,” first author Sam Van de Velde told BioWorld.

GM1 gangliosidosis is a lysosomal storage disease caused by mutations in the human GLB1 gene, encoding the ubiquitous lysosomal β-galactosidase. GM1 causes a rapidly progressing neurodegeneration, which can be lethal in the first 2 years of life in the most severe cases.