Neurological Disease

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some migraine patients struggle to find a formal diagnosis, others must navigate a lengthy trial-and-error process with outdated therapies before they can access treatments that provide real relief.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.…

The Bethesda biotech Gain Therapeutics, armed with supercomputer firepower, aims to disrupt Parkinson’s disease treatment. Their GCase regulator, named GT-02287, completely restored motor function in a recent animal study. GCase, short for glucocerebrosidase, targets both lysosomal and mitochondrial dysfunction. In Parkinson’s, the failure of cellular powerhouses (mitochondria) and recycling centers (lysosomes) disrupts essential functions. This…

The story of Aduhelm has been rocky now for years. Biogen turned heads when its controversial Alzheimer’s therapy won accelerated approval from the FDA against the advice of its own advisory panel in mid-2021. The company had high hopes for the antibody at that point, pricing it at an average of $56,000 per year. Aduhelm…

2023 was a big year for hematology, neurology and oncology, with the medical specialties seeing the most FDA approvals. In terms of sponsors, Pfizer had the most approvals with six total, followed by UCB and Chiesi, each with three apiece. When looking at commercial prospects, AstraZeneca’s respiratory syncytial virus antibody Beyfortus could be the biggest…

Zai Lab is a global biopharmaceutical company founded in 2014 and based in China and the U.S.. Led by Chairperson and CEO Dr. Samantha Du, the company now has more than 2,000 employees internationally. Focusing on developing novel therapies for oncology, autoimmune disorders, infectious diseases, and neurological disorders, it has built a broad pipeline of…

Genetic tools are growing more powerful by the day and hold immense medical promise. Kristofer Mussar, managing director of VectorBuilder GmbH who holds a Ph.D. in molecular genetics and epigenetics, noted that in the wake of the pandemic, genetic research requires increased awareness and ethical stewardship to responsibly tap into its power. While the current…

The treatment landscape for major depressive disorder (MDD) continues to diversify, and in the coming years, psychedelic options may be available, including COMPASS Pathways COMP360 and the deuterated psilocybin analog CYB003 from Cybin. CYB003 demonstrated a significant -14.08 point reduction in MADRS score, a 53.3% response rate, and a 20% remission rate at a 12mg…

Sage Therapeutics has pegged the wholesale acquisition price of the oral postpartum depression (PPD) drug Zurzuvae (zuranolone) at $15,900 for a 14-day course of the therapy. The drug, co-developed by Biogen, won the FDA green light in August. The companies plan on launching the drug in December. The DEA has classified zuranolone as Schedule IV,…