Several neurodegenerative disorders have TAR DNA-binding protein 43 (TDP-43) inclusions as a pathological hallmark; thus, the development of PET tracers able to detect TDP-43 aggregates is essential to advance the diagnosis and treatment monitoring in diseases such as frontotemporal dementia, amyotrophic lateral sclerosis and others.
Researchers from Prothena Biosciences Ltd. recently presented preclinical data for the full effector-function IgG1 monoclonal antibody PRX-012, which binds with high affinity to both soluble and insoluble forms of toxic amyloid-β (Aβ) aggregates.
The picture keeps getting brighter for Argenx SE which, having targeted $750 million in a financing after positive phase II data, priced a global public offering of $1.1 billion, one of history’s larger rounds. Amsterdam-based Argenx capitalized on positive top-line data from the phase II Adhere study with Vyvgart Hytrulo (efgartigimod alfa plus hyaluronidase-qvfc) in chronic inflammatory demyelinating polyneuropathy.
Sigma nonopioid intracellular receptor 1 (SIGMAR1) is a protein enriched in motor neurons (MNs), and mutations in its gene have been previously linked to various motor neuron diseases (MNDs). Researchers from Welab Barcelona and affiliated organizations recently presented preclinical data for two novel SIGMAR ligands, EST-79232 and EST-79376, being evaluated as potential candidates for the prevention of MN degeneration.
Argenx SE’s top-line results from the phase II Adhere study with Vyvgart Hytrulo (efgartigimod alfa plus hyaluronidase-qvfc) in chronic inflammatory demyelinating polyneuropathy (CIDP) sparked buzz about other developers in the space with similar mechanisms of action to Vyvgart Hytrulo, such as Immunovant Inc.
Strong and complete phase III results for Eli Lilly and Co.’s donanemab for treating early Alzheimer's disease (AD) will no doubt inspire more comparisons with recently approved Leqembi (lecanemab). The newly released data for donanemab show it significantly slowed cognitive and functional decline for those with amyloid-positive early symptomatic AD, which lowered the disease-progression risk.
Questionable efficacy, high priced and risky side effects are some words to describe Leqembi (lecanemab), the latest amyloid beta-targeting antibody approved by the U.S. FDA, Korean experts said, but none of that diminishes the profound significance of the drug for Alzheimer’s disease.
Neuren Pharmaceuticals Ltd. and Acadia Pharmaceuticals Inc. have expanded their development and commercialization partnership for Daybue (trofinetide) beyond the U.S. to take the Rett syndrome treatment to the world in a deal valued at $527 million plus royalties.
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