Burke Neurological Institute’s Post

Alzheimer’s disease (AD) is the most common form of dementia, marked by progressive cognitive decline, amyloid plaques, and neurofibrillary tangles. A key factor in cognitive dysfunction in AD is synaptic loss. Plant-derived multitarget compounds, such as artemisinin and its derivatives, show promise as therapeutic agents for AD. These approved antimalarial drugs have been found to positively influence various aspects of AD pathology in animal models by regulating processes like energy homeostasis, apoptosis, proliferation, and inflammation. This review highlights the neuroprotective activities of artemisinins, advocating for their potential use in treating AD and recommending further clinical trials. For a deeper insight, click on the link and read Eva Fenyvesi's blog post on Cyclodextrin News: https://lnkd.in/d98d3An5 #cyclolab #cyclodextrin #dementia #AlzheimersDisease #alzheimers #cognitivedecline #therapy #treatment #pharma #pharmaceutical #pharmaindustry #science #chemistry #research

After spending nearly 9 Billion USD to acquire Cerevel, AbbVie’s Parkinson’s drug looks like its paying off!! ✴️ Tavapadon’s trial success AbbVie’s investigational drug, tavapadon, gives significant improvements in motor and non-motor symptoms for Parkinson’s disease patients. Phase III results show improvement in movement metrics for affected individuals. #ParkinsonsTreatment #ClinicalTrials 🎯 Targeted dopamine receptor treatment Tavapadon works by targeting D1/D5 dopamine receptors, specifically regulating motor functions, while minimising side effects like hallucinations and sleepiness. #Neurology #DopamineReceptors 🏁 Study and next steps AbbVie is to proceed with regulatory filing and show the results in conferences. #DrugDevelopment #Pharma ❓ How will the other drugs in the pipeline of Cerevel come to fruition? Its looking good. Source: https://lnkd.in/erBS7UQy Image credits: https://lnkd.in/ek4DpPpj Did you like this content? Network/follow me for more on the latest in Biotech/Neuro/AI&CompBio 📝

1. Insilico Medicine reported positive results from two Phase I trials of ISM5411, an AI-designed therapy for inflammatory bowel disease (IBD). 2. The trials evaluated the safety, tolerability, pharmacokinetics (PK), and food effects of ISM5411, which targets prolyl hydroxylase domain (PHD) enzymes. 3. ISM5411 demonstrated a favorable PK profile and was well-tolerated by participants, with low occurrences of treatment-emergent adverse events (TEAEs). 4. The therapy aims to alleviate inflammation while also promoting epithelial repair, providing a dual mechanism for improved outcomes. 5. Insilico plans to initiate a Phase 2 proof-of-concept study for ISM5411 by late 2025, focusing on active ulcerative colitis populations. 6. The company has submitted 22 preclinical candidates since 2019, highlighting its commitment to AI-driven drug development. 7. Insilico's generative AI platform, Chemistry42, significantly accelerates the drug development process and enhances treatment tailoring. 8. ISM5411 represents a significant advancement in chronic disease management, showcasing the potential of AI in transforming therapeutic possibilities.

Have you ever considered how drugs cross the blood-brain barrier (#BBB)? With our J-Brain Cargo® technology, JCR Pharmaceuticals is making promising strides to overcome this challenge. Much like the name implies, we attach “cargo” therapeutics to the molecule that can deliver the drug across the BBB. There is great potential for neurodegenerative and other devastating neurological diseases, and you can learn more about the mechanism of action of J-Brain Cargo® in this @Biopharma Dealmakers article here: https://bit.ly/3xhmCW8.    #RareDisease #BloodBrainBarrier #Neurodegeneration #Neurological #CNS #LysosomalStorageDisorders #JCRPharma #BPDealmakers

Applied Therapeutics announced FDA acceptance and priority review of new drug application for Govorestat for the treatment of classic galactosemia. Govorestat is a central nervous system (CNS) penetrant Aldose Reductase Inhibitor (ARI) in development for the treatment of several rare neurological diseases, including Galactosemia, SORD Deficiency, and PMM2-CDG. Chemenu team is always committed to developing more compounds for drug discovery. Here come the related building blocks we can provide: https://lnkd.in/g7GT8Gkf 👇   #Chemenu #AppliedTherapeutics #FDA #Govorestat #galactosemia #CNS #ARI #neurologicaldiseases #SORDDeficiency #PMM2-CDG #drugdiscovery #buildingblocks

The triazine core has gained significant interest among medicinal chemists as a strategy to address DMPK and toxicity issues in molecules. In our previous post, we discussed compound 20, which was modified from revumenib to mitigate hERG-related concerns. Revumenib received FDA approval on November 15, 2024, for the treatment of adult and pediatric patients aged 1 year and older with relapsed or refractory acute leukemia harboring a KMT2A translocation. During the optimization process, the pyrimidine core was replaced with a triazine core, which led to notable improvements in vitro clearance (CL) and a reduction in hERG inhibition. Explore our triazine building blocks today. (Author & Editor: Lisha Wang) salesusa@pharmablock.com; product.pharmablock.com PharmaBlock Group; PharmaBlock (USA), Inc. #DrugDiscovery #Innovation #Biotech

Minjun Liao, Xueke He, Yangyang Zhou, Weiqiang Peng, Xiao-Mei Zhao, Miao Jiang,Coenzyme Q10 in atherosclerosis,European Journal of Pharmacology, Volume 970,2024,176481,ISSN 0014-2999, Atherosclerotic disease, a leading cause of cardiovascular death, predominantly affects the elderly and involves abnormal lipid transport, impaired endothelial function, inflammation, and oxidative stress. Coenzyme Q10 (CoQ10) is crucial in the electron transport chain and acts as an antioxidant. It may help decelerate atherosclerosis by reducing lipid transportation, endothelial inflammation, and metabolic abnormalities. Evidence from molecular mechanisms, animal studies, and clinical trials indicates CoQ10's beneficial effects on atherosclerosis. Additionally, combining CoQ10 with other drugs can enhance therapeutic outcomes, suggesting its potential role in treating atherosclerotic cardiovascular diseases, warranting further research. https://lnkd.in/d2DHx4_W

According to our latest research, the gastrointestinal (GI) therapeutics and #diagnostics #market is projected to reach $122.5 billion by 2028. Read our latest #blogpost to discover the emerging #opportunities, #marketdynamics, and leading #players in this #industry. Tap the link to read more! https://bit.ly/4bjtP7h #GItherapy #DigestiveHealth #GIhealthcare

Clinical pharmacology considerations for first-in-human clinical trials for enzyme replacement therapy Sydney Stern, Jie Wang, Ruo-Jing Li, Yuen Yi Hon, Shawna Weis, Yow-Ming Wang, Robert Schuck, and Michael Pacanowski https://lnkd.in/eiTRezFN Enzyme replacement therapy has been transformational in the development of treatments for inherited metabolic disorders. Stern et al offer considerations for the design of first in-human studies.

Travere Therapeutics has announced that the FDA has granted full approval for FILSPARI (sparsentan) as the only non-immunosuppressive agent to slow kidney function decline in adults with primary IgAN who are at risk of disease progression. Initially, FILSPARI received accelerated approval in February 2023 based on its efficacy in lowering proteinuria.   The full approval is based on long-term confirmatory results from the PROTECT study, which showed that FILSPARI significantly slowed kidney function decline over a two-year period compared to irbesartan. Read More: https://lnkd.in/esZctyMQ #Nephrology #FDAApproval #FILSPARI #Travere