South Korean biopharmaceutical firm GNT Pharma Co. Ltd. on Feb. 13 reported positive findings from a domestic phase III trial of its neuroprotectant therapy, nelonemdaz (NEU-2000), for patients with acute ischemic stroke.
Jazz Pharmaceuticals plc has identified fatty acid amide hydrolase (FAAH) inhibitors reported to be useful for the treatment of anxiety, autism spectrum disorders, depression, schizophrenia, substance abuse and dependence, psychosis, parasomnia and trauma and stress related disorders.
Oligomeric amyloid-β (Aβ) peptide causes synaptic dysfunction, accumulates within synapses, and has been associated with synapse loss around plaques in Alzheimer’s disease (AD). However, there is a need to identify synaptic binding partners of Aβ that mediate synaptotoxicity in the brain. A team of investigators from the University of Edinburgh and affiliated organizations aimed to identify synaptic receptors that bind Aβ in human AD.
It has been shown that vascular endothelial growth factor A (VEGF-A) induces blood-brain barrier disruption and vasogenic edema and it is up-regulated in stroke. When bound to its receptor, VEGF promotes angiogenesis and neuroprotection, in addition to inducing vasogenic edema. VST Bio Ltd. and Yale University have presented data on their monoclonal antibody against syndecan-2, named VST-002, that completely blocks VEGF-driven vasogenic edema while preserving neuroprotective effects.
In repeated concussions, removing damaged mitochondria could prevent the neurodegeneration that occurs when pathology progresses in some patients. The key would be in the role of the p17 protein in restoring mitophagy, according to scientists from the Medical University of South Carolina (MUSC). “Brain injury is an extrinsic disease. It is not idiopathic. When the primary injury occurs, the secure mechanism only relies on an endogenous protection of the brain. If you have a good neuroprotective mechanism, then after the primary injury, basically you don’t see any symptomatic effect,” Onder Albayram told BioWorld.
Otsuka Pharmaceutical Co. Ltd.’s AVP-786 missed the primary endpoint for a third time in a phase III trial for agitation associated with dementia due to Alzheimer’s disease (AD). Two previous phase III trials also failed to show statistical significance for AVP-786.
The European Commission approved two therapies for progressive, genetic diseases: Biogen Inc.’s Friedreich’s ataxia drug, Skyclarys (omaveloxolone), and Crispr Therapeutics AG’s CRISPR/Cas9 gene therapy for sickle cell disease and transfusion-dependent beta-thalassemia, Casgevy (exagamglogene autotemcel, exa-cel).
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