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.
New and updated clinical data presented by biopharma firms at the Alzheimer's Association International Conference, including: Acumen, Alnylam, Athira, Biovie, Coya, Nkgen, Prothena, Taurx.
Responding to medical advances and new standards of care in Alzheimer’s, the U.S. Centers for Medicare & Medicaid Services (CMS) is proposing to end its 10-year-old coverage with evidence development policy that has limited Medicare reimbursement of amyloid PET scans to once in a lifetime for beneficiaries – and then only when they’re used in a CMS-approved trial.
Novartis AG just added muscle to its siRNA development effort by buying Dtx Pharma Inc. in an M&A deal that could total $1 billion. Novartis is getting a preclinical asset, DTx-1252, which just received the U.S. FDA’s orphan drug designation on June 8. Dtx is developing the siRNA candidate for treating the neuromuscular disease Charcot-Marie-Tooth disease type 1A (CMT1A). Basel, Switzerland-based Novartis will pay Dtx $500 million up front and make payments of up to $500 million in milestones completions.
Dealmaking proved alive and well, with Sangamo Biosciences Inc. disclosing a tie-up worth as much as $1.19 billion with a subsidiary of Eli Lilly and Co., while Mirum Pharmaceuticals Inc. pledged to acquire for $445 million the bile-acid product portfolio owned by Travere Therapeutics Inc.
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.
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.
In brain research, be it basic or clinical, neurons have long hogged the limelight. But at the 2023 European Meeting on Glial Cells in Health and Disease, neurons take a back seat to glia – cell types that have often been described as support cells and treated as an afterthought, but that play critical roles in all aspects of brain function, including information processing.
Nodthera Ltd. claims to be first to demonstrate it is possible to modulate the NLRP3 inflammasome in the brain, after showing there were reductions in inflammatory and disease-specific biomarkers in blood and cerebrospinal fluid after seven days of daily administration of its lead product, NT-0796, an oral NLRP3 inhibitor.
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