Nico Therapeutics Inc. has described NLRP3 inflammasome inhibitors reported to be useful for the treatment of Alzheimer’s disease and nonalcoholic fatty liver disease (metabolic dysfunction-associated steatotic liver disease [MASLD]).
Deficiencies in the SLC6A1 gene, encoding the γ-aminobutyric acid transporter GAT-1, are associated with infantile encephalopathy with intellectual disability. Like other neurodevelopmental disorders, SLC6A1-related disorders lack effective treatments; therefore, gene therapy using viral vectors has been proposed as a potential therapeutic strategy.
Spiromacrocyclic compounds acting as orexin OX2 receptor agonists have been described in an H. Lundbeck A/S patent and reported to be useful for the treatment of type 1 and type 2 narcolepsy.
Among the most profound results presented at the 2024 European Society for Medical Oncology (ESMO) Congress were the 10-year data from the Checkmate-067 and Keynote-006 trials of Opdivo and Keytruda as first-line agents in advanced or metastatic melanoma in which 10-year overall survival topped 40%. The success of checkpoint blockade, however, has not extended to all tumor types, but in 2024, molecular studies have led to advances in gene therapies and a multitude of approaches that have opened the door to hope.
Gait instability and somnolence are the main hindbrain-related adverse effects associated with the inhibition of AMPA receptors (AMPARs). Transmembrane AMPAR regulatory proteins (TARPs) modulate AMPAR function, with the specific member TARPγ8 being highly expressed in brain regions associated with seizures and expressed at low or negligible levels in the hindbrain.
2024 was another banner year for GLP-1 receptor agonists (GLP-1RAs) on multiple fronts. They continued to expand into new indications, and provide their developers with both rich remuneration and scientific acclamation. There are now seven approved GLP-1RAs. Commercially, the most successful one so far is semaglutide, sold under the brand name Wegovy or Ozempic depending on the indication.
Pimavanserin is a 5-HT2A inverse agonist that is FDA approved for treating Parkinson’s disease psychosis. Acadia Pharmaceuticals Inc. searched for a compound that relies on pimavanserin but with an improved profile, including shorter half-life and reduced QT prolongation risk, which led to the identification of ACP-204.
In a deal worth $100 million up front and up to $1.25 billion in milestone payments, Bioarctic AB licensed its pyroglutamate-amyloid-β (pyroglutamate-Aβ) antibody program to Bristol Myers Squibb Co. to advance treatments for Alzheimer’s disease.
DM Intelligence Medicine Ltd. has disclosed leucine-rich repeat kinase 2 (LRRK2; dardarin), LRRK2 (G2019S mutant) and/or EGFR (HER1; erbB1 mutant) inhibitors reported to be useful for the treatment of cancer, neurological and immunological disorders.
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