Neurological Disease

The explosive sales growth of GLP-1 drugs has analysts projecting that the antiobesity drugs could be a $44 billion market by 2030. Some observers are more upbeat, projecting that the sector could be worth more than $100 billion in the coming years. Pfizer CEO Albert Bourla projects that the market will reach $90 billion by…

Alzheimer’s disease research appears to be hitting its stride, thanks to recent therapeutic advances in drug development and the emergence of biomarkers to detect the condition. “All the pieces of the puzzle of precision medicine, which is already quite common in oncology, are now in place,” said Hartmuth Kolb, vice president, neuroscience biomarkers and R&D…

Vision, one of our most valued senses, is a complex orchestration of light-sensing photoreceptors, neurons, and intricate pathways leading to the brain. Central to this native biological system are the photoreceptors – the rods and cones – cells that are responsible for capturing light and initiating visual perception. For many with inherited retinal diseases (IRDs),…

Vraylar (cariprazine), which recently scored FDA approval as an adjunctive treatment for Major Depressive Disorder (MDD), could have positive implications for healthcare resource utilization. A new analysis suggests that this atypical antipsychotic, initially developed by Allergan (now AbbVie), could curb healthcare spending of MDD treatment. In the economic analysis, patients who received cariprazine as their…

The dopamine D3 receptor-targeting cariprazine could potentially be a new tool for managing anhedonia, one of the most stubborn symptoms of major depressive disorder (MDD). Characterized by a lack of enjoyment in previously pleasurable activities, anhedonia has been “difficult to address with traditional antidepressants,” said Dr. Vladimir Maletic, coauthor of a poster presented at the…

Mixed-bag approval hit Sage hard but spared Biogen Sage Therapeutics recently notched an FDA win for the neuroactive steroid zuranolone (Zurzavae) for postpartum depression (PPD). For major depressive disorder (MDD), however, the agency handed the drug a complete response letter. In the wake of the news, Sage’s stock is down 46% to $19.52, while BIIB,…

Recent research has indicated a link between Epstein-Barr virus (EBV) and multiple sclerosis (MS), with some researchers going as far as to say that EBV might be a potential trigger of MS. However, it remains unclear whether the virus also drives the progression of the disease. Current treatments focus largely on moderating inflammation. In this…

FDA’s approval of Zurzuvae for PPD could mark a significant shift in the fight against postpartum depression (PPD). On August 4, the agency gave the green light for the oral medication, although it declined its use for major depressive disorder (MDD), citing insufficient evidence of effectiveness. The former approval represents a milestone as the first ever…

Cambridge-based biotech Biogen has unveiled plans to cut 1,000 jobs, or 11% of its workforce, as it prepares for the launch of its newly approved Alzheimer’s disease drug, Leqembi (lecanemab) that it developed with Eisai.  This move follows a pattern of significant layoffs, with the company having cut 885 jobs last year after the troubled…

According to results from the Phase 3 TRAILBLAZER-ALZ 2 study published in JAMA, the monoclonal antibody donanemab significantly slowed cognitive and functional decline in patients with early symptomatic Alzheimer’s disease by approximately 35% at one year compared to placebo. The trial enrolled 1736 patients across 277 research centers in 8 countries. Another monoclonal antibody, Leqembi…

Cambridge, Massachusetts–based biopharma Scholar Rock has been making encouraging progress in the development of apitegromab, a potential new therapy for spinal muscular atrophy (SMA), a genetic disease characterized by progressive muscle weakness and atrophy. Apitegromab, a novel monoclonal antibody, works through a unique mechanism of action — it binds to and inhibits the precursor form…

Today, the FDA granted traditional approved to lecanemab (branded as Leqembi), a monoclonal antibody from Eisai and Biogen for adult patients with Alzheimer’s disease. The agency made the decision on the basis of a confirmatory trial that showed its clinical benefit. The drug, which reduces the formation of amyloid plaques in the brain, is the…

In the realm of drug discovery, the Harvard-educated chemist Nick Paras, Ph.D. compares the early stages of identifying new compounds to a game of Zelda, in which a player at times must navigate through dark rooms and solve puzzles to progress. Similarly, medicinal chemists and biochemists too must traverse the vast, often obscure landscape of…

High-resolution mapping of the human brain involves managing and interpreting a colossal amount of data. Shoaib Mufti, the head of data and technology at the Allen Institute for Brain Science, described the organization’s approach to these challenges in a recent interview. The project uses artificial intelligence to analyze millions of data points from brain imaging…

Despite substantial progress in brain research, our understanding of the human brain remains limited. “We really don’t understand the fine circuitry of the brain — even in a relatively simple organism like a mouse,” confessed Ed Lein, a senior investigator at the Allen Institute for Brain Science. But AI in neuroscience research promises to change…

Interest in ketamine and psilocybin as potential therapies for mood disorders has surged since around 2010. A groundbreaking 2000 study at Yale revealed the powerful antidepressant effects of ketamine, a dissociative anesthetic. Unlike traditional antidepressants which can take weeks or months to have an impact, a single dose of ketamine led to significant improvements in…

In an era of rapid AI progress, the quest to pioneer the first AI-developed drug candidates has led to an increasing number of these drug candidates entering clinical trials. One contender is ulotaront, an antipsychotic drug, that fared well in a phase 3 schizophrenia study published in NEJM in 2020. Sunovion discovered the drug in…

A recent exposé in Science magazine concluded that “Fake scientific papers are alarmingly common.” The article quoted a preprint from the German neuropsychologist Bernhard Sabel, which estimated that in 2020, up to one-third of neuroscience papers and nearly a quarter of medicine papers were likely falsified or plagiarized, highlighting a concerning prevalence of suspicious medical…

Biogen’s Tysabri (natalizumab), the first humanized monoclonal antibody for multiple sclerosis (MS), sparked optimism among MS patients following its FDA approval in 2004. The drug offered significant benefits, reducing relapses for patients resistant to other treatments. This was a significant milestone in the treatment of MS, but the journey of natalizumab and PML soon took…

The gene therapy platform company Capsida Biotherapeutics (Thousand Oaks, California) is gearing up to unveil initial data on its engineered adeno-associated virus (AAV) capsids at the upcoming American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting, held May 16–20 in Los Angeles. In developing these capsids, Capsida Chief Scientific Officer Susan Catalano explained…

Lilly’s (NYSE:LLY) shares were up more than 5.36% to $425.88 after the company announced upbeat results from the phase 3 TRAILBLAZER-ALZ 2 study, which revealed that the experimental Alzheimer’s drug donanemab significantly slowed cognitive decline in patients with early symptomatic Alzheimer’s disease at 18 months. Almost half of patients, 47%, who received donanemab had no…

In mid-April, San Diego–based Pipeline Therapeutics announced a global license and development agreement with Janssen Pharmaceutica NV (NYSE:JNJ), focusing on PIPE-307, an investigational compound targeting nervous system disorders. As a pioneer in precision neuroscience, Pipeline Therapeutics aims to create targeted treatments for patients. PIPE-307, an oral, highly selective antagonist of the muscarinic M1 receptor, embodies…

Neurodegenerative disease research is witnessing significant advances. To that end, Hong Kong-headquartered Aprinoia Therapeutics is embracing a ‘precision neuroscience’ approach to neurodegeneration diagnostics. The company’s lead program, APN-1607, represents a new generation of advanced tau positron emission tomography (PET) tracers, which play a crucial role in effective and efficient diagnosis of patients suffering from neurodegenerative…

The COVID-19 pandemic has had a profound impact on the best-selling pharmaceuticals, leading to shifts in the list with Pfizer and BioNTech’s Comirnaty surpassing AbbVie’s Humira for the No. 1 spot in 2021. That momentum continued in 2022, with Pfizer and BioNTech jointly raking in $59.1 billion in revenue from the sales of the COVID-19…

Cambridge, Massachusetts–based Amylyx has dosed the first participant in its phase 2 HELIOS clinical trial of AMX0035 focusing on Wolfram syndrome. The condition is an ultra-rare genetic disorder involving the endocrine system. Symptoms of Wolfram syndrome can include diabetes insipidus, diabetes mellitus, optic atrophy and hearing loss. In September, Amylyx won FDA approval for AMX0035…