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.
Institutional investors are not in the market for now, but a survey of 104 firms on both sides of the Atlantic shows all but 6% have an interest in psychedelic medicines. However, only 23% say they are “very likely” to invest in the sector in the future, whilst the majority are sitting on the fence.
The British Museum in London, steeped in history and tradition, sits alongside world class biomedical and clinical research centers, where numerous technological advances and scientific breakthroughs have originated. “This unique combination of tradition and innovation […] also defines our industry today,” said Rivki Stern, co-founder and CEO of Shortwave Pharma Inc., which is working to develop psychedelic drugs as approved medicines that meet unmet medical needs of patients with treatment-resistant depression, addictions and eating disorders.
At the 2023 Annual Congress of the European Academy of Neurology, Mary Reilly described the relationship between bench and bedside as “a continuous circle of translation,” with each cycle beginning with patients and their needs.
Artificial intelligence (AI) continues to entice. On the exhibition floor at the 2023 Congress of the European Academy of Neurology, one company’s booth featured “Mindart” technology. A passersby could answer a short series of prompts, and get a unique image based on the input made by generative AI. Entertainment aside, medically speaking, AI applications “are still research,” Riccardo Soffietti told his audience at one of several sessions devoted to AI. “But obviously, research is the future.”
Although the highest dose of Bioxcel Therapeutics Inc.’s BXCL-501 hit the primary endpoint in the phase III Tranquility II trial for acute agitation in Alzheimer’s disease patients, shares fell dramatically on June 29 by 64% when investors learned the company received an FDA warning letter over infractions at a trial site.