Europe may still await its first disease-modifying Alzheimer’s drug after the EMA postponed its decision on Leqembi (lecanemab, Biogen Inc./Eisai Co. Ltd) on March 22, but leading members of the World Dementia Council were in an optimistic mood when they convened in London four days later. “We are working to make the inevitable happen earlier,” said Lenny Shallcross, executive director of WDC. “The inevitable will be rollout of medicines, rollout of better diagnostics and the improvement of care. All of those things over the next 10 years are inevitably going to happen.”
Europe may still await its first disease-modifying Alzheimer’s drug after the EMA postponed its decision on Leqembi (lecanemab, Biogen Inc./Eisai Co. Ltd) on March 22, but leading members of the World Dementia Council were in an optimistic mood when they convened in London four days later. “We are working to make the inevitable happen earlier,” said Lenny Shallcross, executive director of WDC. “The inevitable will be rollout of medicines, rollout of better diagnostics and the improvement of care. All of those things over the next 10 years are inevitably going to happen.”
Researchers from Merck & Co have presented the discovery of inhibitors of the YAP/TAZ-TEAD complex as potential anticancer agents. YAP and its paralogue TAZ act as terminal effectors of the Hippo signaling pathway by regulating the transcriptional activity of the different TEAD isoforms.
Mucopolysaccharidosis type III (MPS III), also known as Sanfilippo syndrome, is a congenital metabolic disorder that leads to the accumulation of partially degraded heparan sulfate, which triggers neurodegeneration.
Investigators at Incyte Corp. have reported details on the discovery and preclinical characterization of new potent and selective inhibitors of cyclin-dependent kinase 2 (CDK2) as potential anticancer candidates.
Plasma pharmacodynamic biomarkers may be a reliable tool for biosimilarity assessment without having to rely on clinical trials, which are costly and time consuming.
Japan Tobacco Inc. has disclosed pyrazolopyrimidine compounds acting as NLRP3 inflammasome inhibitors reported to be useful for the treatment of traumatic brain injury, inflammatory bowel disease and more.
Volastra Therapeutics Inc. has entered into partnerships with Microsoft Corp., Function Oncology Inc. and Tailor Bio Ltd. with the goal of expanding the potential use of its KIF18A inhibitors.
Acurex Biosciences Corp. has synthesized phenothiazines acting as ferroptosis inhibitors reported to be useful for the treatment of mitochondrial disease.
Mucosa-associated lymphoid tissue lymphoma translocation protein 1 (MALT1) is a key regulator of physiological antigen receptor signaling in B cells and T cells, as it is the only component of the MALT1-BCL10-CARD11 (CBM) signalosome with proteolytic activity.
Previous research has shown that adenosine impairs antitumor immunity, and high levels of adenosine and CD73 have been associated with poor prognosis in cancer.
Scientists at the Karolinska Institutet in Sweden have found a new region in the c-Myc oncogene that would allow the development of a binding compound to target it. Their discovery is based on a structural switch that leads to open and closed conformations of the domain, allowing or not its...
While there has been progress in the development and availability of treatments and vaccines for Ebola virus (EBOV), there are still no treatments for other filoviruses such as Sudan virus (SUDV, species Sudan ebolavirus), which caused the 2022-23 outbreak in Uganda.
The US Department of Veterans Affairs has synthesized substituted aminopiperazine compounds reported to be useful for the treatment of bacterial and fungal infections.
Scientists at Fujifilm Corp. and Osaka University have described piperazine derivatives acting as drug efflux pump inhibitors reported to be useful for the treatment of bacterial infections.
Atamyo Therapeutics SAS has received clinical trial application (CTA) authorizations in Italy and France for ATA-200, its gene therapy for the treatment of γ-sarcoglycan related limb-girdle muscular dystrophy type 2C/R5 (LGMD2C/R5).
Pannex Therapeutics Inc. has divulged pannexin-1 (PANX1; MRS1) inhibitors reported to be useful for the treatment of neuropathic pain and opioid dependency.
Brise Pharmaceutical (Shanghai) Co. Ltd. has identified calcitonin gene-related peptide (CGRP) receptor antagonists reported to be useful for treatment of cerebrovascular and vascular disorders.
Nurexone Biologic Inc. has signed a strategic service agreement with Vivox Ltd. for large-scale animal testing of Exopten therapy for spinal cord injury (SCI).
To address the need for novel therapeutic candidates against Duchenne muscular dystrophy (DMD), investigators at Mitorx Therapeutics Ltd. developed a library of novel small-molecule mitochondriotropic agents.
Beam Therapeutics Inc. has received clearance of its clinical trial authorization (CTA) application by the U.K.’s Medicines and Healthcare Products Regulatory Agency for BEAM-302, an in vivo base editor, as a potential treatment for patients with α1-antitrypsin deficiency (AATD).
Amyotrophic lateral sclerosis (ALS) is a devastating neurodegenerative disease for which there is a 10% rate of familial cases, with the rest being sporadic cases. Both genetic and environmental factors contribute to the etiology of ALS, and more than 120 genes have been reported to be tied to the...
Patients with amyotrophic lateral sclerosis (ALS) have a median survival of 2 to 5 years. There are 3 FDA-approved drugs for ALS (riluzole, edaravone and Relyvrio [phenylbuturate/taurursodiol]), but they only lead to modest benefit. There are several pathways involved in the disease, but all of...
Parvus Therapeutics Inc. has entered into an exclusive worldwide collaboration and option agreement with Abbvie Inc. for the development and commercialization of novel treatments for inflammatory bowel disease (IBD), utilizing Parvus’ Navacim...
Entera Bio Ltd. has announced promising pharmacokinetic (PK) results from its collaborative research combining a proprietary long-acting GLP-2 agonist developed by Opko Health Inc. with Entera’s proprietary N-Tab technology.
Nuclear factor erythroid 2-related factor 2 (NRF2) is a master regulator of cytoprotective mechanisms, and the cellular levels of this transcription factor are maintained low under normal conditions via actions of a CUL3-based E3 ligase,...
Asialoglycoprotein receptor 1 (ASGR1) is a transmembrane protein specifically expressed in hepatocytes that plays a key role in maintaining circulating glycoprotein homeostasis.
In a recent study published in PNAS, researchers from the University of Texas Southwestern Medical Center investigated cholesterol-mimetic compounds specifically binding and inhibiting Scap. Their final goal was to understand cholesterol...
Everzom SAS, a CNRS/Université Paris Cité spin-off, has signed a second exclusive license agreement with Erganeo SAS for the development of Evergel, an exosome drug candidate for fistulas and fibrosis of the digestive tract.
Breakthrough medicines, billion-dollar deals, spectacular clinical successes and crushing failures all play a part in biopharma’s dynamic story. Developers make scientific advancements with the potential to change everything, only to face regulatory conundrums and ever-fluctuating markets. BioWorld tracks key events in the fast-moving sector every business day. Now, the BioWorld Insider podcast lets you hear directly from the movers and shakers whose collective work is changing how we all live. Join us each week for a new conversation.