A new study led by the Medical Research Council’s (MRC) Laboratory of Molecular Biology (LMB) has identified a new protein as a potential therapeutic target for frontotemporal dementia, a type of early-onset dementia. Dementia is a progressive neurodegenerative disorder that affects more than 944,000 people in the UK. Frontotemporal dementia occurs from the degeneration of the frontal and temporal lobes of the brain, which control emotions, personality and behaviour, along with speech. Most neurodegenerative diseases involve proteins aggregating into filaments known as amyloids. When identified, researchers can target these proteins for diagnostic tests and treatments. However, in around 10% of frontotemporal dementia cases, researchers had to identify the TAF15 protein. Published in Nature, MRC LMB researchers successfully pinpointed the aggregated structures of the protein using cutting-edge cryo-electron microscopy to study the brains of four patients who had this type of early-onset dementia. The FUS protein is already known to be ...
Americans can’t afford their medications. About 25% leave prescriptions unfilled, split pills, or skip doses. Seniors face challenges covering non-Medicare services, including prescriptions. Even those with commercial health insurance are not immune. One-third of insured adults worry about affording their premiums, and 44% must meet increasingly high deductibles before insurance even kicks in. As a result, 41% of adults carry substantial medical debt. At the same time, FDA approvals for therapeutics targeting rare diseases are on the rise, including those for cancer, Alzheimer’s, and many previously untreatable diseases. In fact, 54% of 2022’s novel drug approvals targeted rare diseases. They encompass groundbreaking therapies like the first acid sphingomyelinase deficiency treatment, a prurigo nodularis remedy, and an obstructive hypertrophic cardiomyopathy therapy. In 2023, the first enzyme replacement therapy for non-neurological alpha-mannosidosis effects gained early approval. Breakthroughs, though welcome, are staggeringly expensive. In fact, Hemgenix, a new treatment for hemophilia, is officially ...
Pharmaceutical Executive Editorial Staff KarXT (xanomeline-trospium) is currently in development to treat schizophrenia and psychosis related to Alzheimer disease. The FDA has accepted a New Drug Application (NDA) for Karuna Therapeutics, Inc’s KarXT (xanomeline-trospium) treatment for schizophrenia in adult patients. The novel muscarinic antipsychotic is currently in development to treat schizophrenia and psychosis related to Alzheimer disease. “There is a significant need for new treatment options for serious mental illness. If approved, KarXT could be one of the more important new product introductions in neuropsychiatry by providing a novel pharmacological approach for the treatment of schizophrenia,” said Bill Meury, Karuna Therapeutics president and chief executive officer, said in a press release.1 Schizophrenia can be a disabling condition, potentially causing a combination of hallucinations, delusions, and extremely disordered thinking and behavior that has a negative impact on the ability to function in daily life. As such, patients with schizophrenia require lifelong ...
For more than a year, Biogen has been working to turn the page from its Aduhelm fiasco and focus on its newer Eisai-partnered Alzheimer’s disease medicine Leqembi. But a new ruling from the U.S. Court of Appeals for the First Circuit will make that effort a little tougher. After Biogen last year won the dismissal of an investor lawsuit surrounding disclosures into its Aduhelm research, the appeals court has reversed the ruling—in part—by focusing on one statement made by Biogen’s former chief medical officer Al Sandrock. Specifically, the appeals court flagged Sandrock’s statement from the company’s second-quarter earnings call in 2020. During the call, Sandrock stated that “[Y]ou really need to get to the higher dose,” adding, “I think our data are all consistent with that.” The court dubbed this the “all data” statement and painstakingly broke down the context behind the remark. To refresh readers’ memory, Biogen in March ...
Takeda Pharmaceutical drug Exkivity failed the confirmatory study required of its 2021 accelerated approval. Our recap of other recent regulatory developments includes a partial clinical hold on a cancer drug, a Covid-19 vaccine authorization, and several drug approvals in the U.S. and beyond. By FRANK VINLUAN Accelerated approval offers a way to more quickly bring patients drugs for diseases that have few treatment options. But that speedy path to the market comes with the understanding that the FDA can take its regulatory blessing away—unless the company takes its drug away first. That is what Takeda Pharmaceutical has decided to do with its cancer drug, Exkivity. Following discussions with the FDA, the Japanese pharmaceutical giant is voluntarily withdrawing Exkivity from the market. Exkivity treats non-small cell lung cancer by targeting epidermal growth factor (EGFR). That cancer protein must have exon 20 mutations—the same genetic signature addressed by Johnson & Johnson’s Rybrevant. ...
First Wave BioPharma has acquired Sanofi’s capeserod with plans to repurpose it as a gastrointestinal (GI) disease therapy. Following the news, First Wave’s stock skyrocketed by over 80% in pre-market trading. The US-based company has a market cap of $3.112m. The agreement includes a $500,000 upfront payment, with developmental and commercial milestone payments of up to $46m and $235m, respectively. Additionally, Sanofi would have the right of first refusal on any capeserod sublicensing agreement and would be entitled to a percentage of any licensing payments. Sanofi also reserves the first refusal right to reacquire capeserod during development. Capeserod is a selective partial agonist of the serotonin type 4 (5-HT4) receptor. In the GI tract, these receptors perform various functions, including gastric emptying, decreasing oesophageal reflux, and promoting intestinal peristalsis. Sanofi had previously evaluated capeserod in Alzheimer’s disease and urinary incontinence but had since terminated the drug development. However, Fist Wave ...
By Tristan Manalac AstraZeneca, through its rare disease division Alexion, has entered into a multi-target agreement with Verge Genomics to discover and develop drugs against novel targets in rare neurodegenerative and neuromuscular diseases, the companies announced Friday. Under the agreement, Alexion will make a $42 million payment in upfront fees, as well as equity and near-term investments. Counting milestones, the four-year collaboration can yield up to $840 million for Verge, plus potential royalties down the line. Alongside the drug discovery pact, AstraZeneca will also take an equity position in Verge. In return, Alexion will gain access to the California biotech’s proprietary full-stack CONVERGE platform, which applies machine learning to human tissue data in order to identify novel disease targets that have a high probability of yielding clinical success. Friday’s partnership will apply the AI-driven approach to several yet-undisclosed rare neurodegenerative and neuromuscular conditions. Alexion will have the option to select ...
IBM has announced a data breach of Janssen’s CarePath platform and has started informing affected customers. The Janssen CarePath portal provides patients and healthcare professionals in the US with information regarding insurance coverage, out-of-pocket costs, and prescribing information at no cost to the users. The number of highest grossing Janssen drugs are included in the platform such as Darzalex (daratumumab) and Stelara (ustekinumab) which generated $2.5bn and $3.2bn in US sales in H1 2023, respectively, as per the company’s Q2 2023 financial report. Data breaches at the top pharma companies are nothing new, as companies such as Merck & Co (MSD), Roche, and AstraZeneca have all been hacked in the past. Data breaches cost the companies an average of $5m to remediate, as per IBM’s 2020 data breach report. The Janssen CarePath systems provider, IBM, indicated that the stolen data may have contained information provided as part of the Janssen ...
A new study has revealed that a new dementia risk score ‘calculator’ can strongly predict the chances of people developing dementia within the next 14 years. Based on 11 mostly modifiable risk factors in people in the middle stages of life and onwards, the UK Biobank Dementia Risk Score (UKBDRS) outperformed three additional widely used risk sources, according to the results from the study published in the British Medical Journal. Around 50 million people are thought to be living with dementia worldwide, with numbers expected to triple by 2050. Targeting key risk factors could potentially avert around 40% of dementia cases, and various risk scores have been devised to predict a person’s chances of developing the disease while preventive measures are still possible. However, these scores have been shown to be unreliable across different age groups and geographies, and some are reliant on expensive and invasive tests. After analysing two ...
New research published in the journal Gut suggests that certain gastrointestinal symptoms could be an early warning sign of Parkinson’s disease. Symptoms including constipation, dysphagia (difficulty swallowing) and irritable bowel syndrome (IBS) are suspected to drive the development of Parkinson’s disease, along with cerebrovascular disease (CD), such as a stroke or brain aneurysm, and Alzheimer’s disease (AD). Parkinson’s is one of the fastest-growing neurodegenerative diseases in the world and affects around 145,000 people in the UK. There is currently no cure for the condition. Researchers analysed the US medical records of 24,624 people with Parkinson’s and compared them to 19,046 people with AD, 23,942 with CD, and 24,624 with healthy brains to determine whether patients with Parkinson’s experienced gut issues six years prior to diagnosis and whether they had a higher chance of developing the condition. The gastrointestinal tract possesses millions of nerve cells that communicate with the brain. Researchers ...
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