Fatty acid synthase (FASN), an enzyme that drives de novo lipogenesis, plays a relevant role in metabolic disorders such as obesity, non-alcoholic fatty liver disease (NAFLD) or type 2 diabetes. Denifanstat (TVB-2640), a FASN inhibitor, has recently shown positive results in a phase II study in patients with metabolic dysfunction-associated steatohepatitis (MASH).

C-C chemokine receptor-like 2 (CCRL2) is a chemokine receptor involved in inflammation activation and is usually expressed in differentiated myeloid cells. CCRL2 is overexpressed in acute erythroid leukemia (AEL) cells compared to healthy cells. Antibody-drug conjugates (ADCs) against CCRL2 in AEL were developed and tested in the preclinical setting.

The protease mucosa-associated lymphoid tissue lymphoma translocation 1 (MALT1) is a signaling protein with both molecular scaffolding and protease activity involved in lymphocyte activation. MALT1 is considered a therapeutic target for chronic lymphocytic leukemia (CLL) in patients who develop resistance to Bruton tyrosine kinase (BTK) inhibitors.

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.

Io Therapeutics Inc. has presented new preclinical data on their RARγ agonist IRX-5010 for the treatment of triple-negative breast cancer (TNBC).

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.