Neurological Disease

On Monday, Novo Nordisk announced that the results from its Phase 3 trial investigating semaglutide as a treatment for Alzheimer’s “did not confirm the superiority of semaglutide versus placebo in the reduction of progression,” of the disease, according to the press release.  Monday’s announcement had a silver lining. That is, the treatment did result in…

Alzheimer’s disease remains one of the most complex and devastating neurodegenerative disorders, affecting over 55 million people globally. With projections estimating this number will rise to 139 million by 2050, the urgency to develop effective treatments has never been greater.  Historically, AD drug development has been marked by setbacks and limited therapeutic success due to…

Biomarkers have moved from “nice-to-have” to the backbone of drug development, says Quanterix’s Jorge Marques Signes, who argues they now shape feasibility, timelines and even payer expectations before sponsors commit thousands of patients or years of spend. “Biomarkers are not supplemental anymore,” said Marques Signes, VP of Accelerator & Clinical Services at Quanterix. “They are…

Ken Paxton, the Republican attorney general of Texas, sued Kenvue, the manufacturer of Tylenol, on Tuesday, for “deceptively marketing Tylenol to pregnant mothers despite knowing that early exposure to acetaminophen leads to a significantly increased risk of autism and other disorders,” according to the press release.  J&J illegally offloaded liability, Paxton claims Paxton also claimed…

Following a White House press conference on Sept. 22, during which Donald Trump alleged a link between acetaminophen use during pregnancy and autism, the Informed Consent Network (ICAN) filed a petition with the FDA to add a warning to all drugs that contain acetaminophen. ICAN is a nonprofit organization that aims to provide “every person…

Advanced Parkinson’s patients can spend hours each day locked in ‘OFF’ states, periods when tremors, rigidity and immobility return as oral medications wear off. The first multi-country real-world data on continuous 24-hour foslevodopa/foscarbidopa infusion shows the approach cut that OFF time by 2.7 hours daily at six months, with improvements across motor symptoms, sleep, pain,…

Novartis has signed a global licensing agreement with Arrowhead Pharmaceuticals for the preclinical RNA interference (RNAi) therapy ARO-SNCA, targeting alpha-synuclein in Parkinson’s disease and related disorders. The deal, announced Tuesday and reported by Reuters, includes a $200 million upfront payment to Arrowhead, with up to $2 billion in potential milestones, plus tiered royalties (capped in…

GE HealthCare’s amyloid PET imaging agent Vizamyl (flutemetamol) has won an FDA nod for expanded indications on June 24, 2025. The specific update adds quantification and therapy monitoring capabilities that competing products currently lack, positioning the diagnostic as an enabler for the rapidly growing anti-amyloid therapy market. At present, there are two disease-modifying anti-amyloid therapies…

For the more than one billion people worldwide living with migraine, the path to effective prevention can feel like an obstacle course. While some migraine patients struggle to find a formal diagnosis, others must navigate a lengthy trial-and-error process with outdated therapies before they can access treatments that provide real relief. Despite migraine ranking as…

Applied Cognition, a clinical-stage platform therapeutic company, and academic partners have published the first continuous, non-invasive measurements of the human glymphatic system in Nature Biomedical Engineering. The glymphatic system is a brain-wide waste-clearance network that removes toxic proteins including amyloid-β and tau during sleep. Dysfunction of the system is implicated in Alzheimer’s disease and other neurodegenerative…

N-acetyl-L-leucine (NALL), an FDA-approved drug for Niemann-Pick disease Type C from IntraBio may hold promise for Parkinson’s disease (PD). A pre-print manuscript posted on Research Square (currently under review at a Nature Portfolio journal) details the relevant preclinical research of the mode of action. It indicates that NALL directly reduces pathological alpha-synuclein, upregulates protective proteins like…

Researchers at the University of California San Diego believe they have found an early pinch point in the biology of Alzheimer’s disease, one that can be blocked without disturbing the brain’s normal chemistry. The scientists credit AI for guiding them there. The study, published April 23 in Cell, focused on phosphoglycerate dehydrogenase, or PHGDH, an…

Targeted Protein Degradation (TPD) has  emerged as a promising modality in drug discovery, hijacking the body’s natural disposal systems to eliminate disease-causing proteins. While bivalent degraders like PROTACs have pioneered the field, demonstrating clinical promise, attention is increasingly turning toward a new upstart — Molecular Glue Degraders (MGDs). These smaller, simpler molecules offer advantages but…

Gantenerumab—an experimental monoclonal antibody discontinued that Roche/Genentech discontinued in 2022, has shown significant effectiveness in delaying symptoms when administered long-term to patients with a rare genetic form of the disease. A recent study from Washington University School of Medicine reveals that among patients who received the drug for an average of eight years, the risk…

The humble vitamin biotin (vitamin B7) has emerged as a potential neuroprotective agent against manganese-induced neurotoxicity linked to Parkinson’s disease (PD). A recent study published in Science Signaling used fruit fly models and human dopaminergic neurons to show that biotin supplementation can reverse neurologic damage caused by excessive manganese exposure. Manganese, though essential at low…

Capsida Biotherapeutics, a gene therapy startup that has quietly amassed a network of Big Pharma partnerships, announced in January that its partner AbbVie will exercise its option on their first neurodegenerative disease program, triggering a $40 million license payment. The five-year-old company, which has built its war chest primarily through pharma collaborations rather than traditional…

Migraines, like many diseases, are not monolithic. In that regard, they are something like weather — they can shift suddenly, and predicting when they might arise can be elusive. Clinical trials traditionally capture a snapshot of the migraine storm, often failing to reflect the dynamic, lived experience of patients. Yet recent studies, including the phase…

Novartis has released positive topline results from the phase 3 STEER trial evaluating OAV101 IT (intrathecal onasemnogene abeparvovec) in treatment-naïve patients with spinal muscular atrophy (SMA) Type 2, aged two to under 18 years. The STEER trial is a pivotal, sham-controlled study designed to evaluate OAV101 IT against a procedure that mimics drug administration without…

MIRA Pharmaceuticals has announced that its oral ketamine analog, Ketamir-2, has outperformed current FDA-approved neuropathic pain treatments in preclinical studies. In trials using a nerve ligation model, Ketamir-2 demonstrated 112% more effectiveness than pregabalin and 70% greater relief than gabapentin by Day 22, with significant pain relief appearing as early as Day 15. The studies,…

Alzheimer’s clinical trials are among the most challenging and expensive studies to conduct with a 2021 study pegging the total costs since 1995 at $42.5 billion. Phase 3 trials are the biggest cost drivers, involving hundreds or thousands of participants. “We’re talking about studies with sometimes two thousand people per phase three clinical trial in…

Liraglutide, a GLP-1 receptor agonist medication that first won FDA approval in 2010, may reduce cognitive impairment in Alzheimer’s patients. At this year’s Alzheimer’s Association International Conference, data from a Phase 2b clinical trial with 204 mild AD patients serving as participants was revealed, showing that the drug lowered decline in cognition by up to…

The recent FDA approval of Lilly’s Kisunla (donanemab) represents a significant win in Alzheimer’s drug development, a field that has seen a  decades of false starts and fizzled hopes. This approval, along with that of lecanemab in 2023, could mark a turning point in AD treatment. Alzheimer’s disease is reaching epidemic proportions with an estimated…

Friso Postma, vice president of AI for drug discovery at BioXcel Therapeutics, prefers the term “augmented intelligence” to “artificial intelligence.” His company uses AI tools to support human experts in drug repurposing efforts. Postma, who holds a PhD in signal transduction from the Netherlands Cancer Institute, transitioned to AI from wearable digital health devices. “I…

Which major pharmaceutical company leads the way in research and development? Data reveals that the top R&D spender in 2023 was Merck & Co., by a wide margin. While Pfizer had almost as much revenue as Merck ($60.1 billion vs. $58.5 billion), its R&D ratio was on the lower end at 18.29%, or $10.7 billion.…

The FDA continues to signal openness to psychedelic-based therapies. Following its recent Breakthrough Therapy Designation (BTD) for a potential LSD-based anxiety treatment, the agency has extended the same status to Cybin Inc.‘s CYB003 for major depressive disorder (MDD). The move marks the first FDA Breakthrough Therapy Designation for an adjunctive, psychedelic-based Major Depressive Disorder treatment.…