Seven months after Travere Therapeutics’ flagship drug received accelerated FDA approval in a rare kidney disease, the molecule has fallen short of the key goal of its confirmatory test. It’s the small molecule’s second Phase 3 failure this year. By FRANK VINLUAN A Travere Therapeutics drug that won speedy approval early this year for a rare autoimmune kidney disorder has failed to meet the key goal of its confirmatory study. Still, the biotech points to data trends favoring patients treated with its drug, Filspari, and it aims to seek full approval. But even if the FDA permits the drug to remain on the market, the disappointing trial results reported Thursday call into question the commercial prospects of a product once projected to become a blockbuster seller. Filspari was developed to treat immunoglobulin A nephropathy (IgAN), in which the buildup of antibodies in the kidneys damages these organs. The FDA awarded ...
The investment will support research into cancer treatments and diagnosis Cancer Research UK (CRUK) has announced its largest ever investment of £123m in Scotland as part of a seven-year commitment to the CRUK Scotland Institute. The institute, which is facilitated in partnership with the University of Glasgow, will use the investment to support research into new and improved cancer treatments and diagnosis, as well as bolster Scotland as a major global hub for cancer research. It will also aid in attracting research talent to the Scotland Institute and will support major studies into specific types of cancer that largely affect Scotland’s population, including liver, pancreatic, bowel and lung. Despite mortality rates falling by 8% in the past decade, more than 34,000 people are diagnosed with cancer every year in Scotland. Previously known as the CRUK Beatson Institute, the CRUK Scotland Institute studies the roots of how cancer begins, the genetics ...
A new Massachusetts Institute of Technology (MIT) study has revealed a potential explanation as to why certain immunotherapies for cancerous tumours do not always work as predicted. If validated in clinical trials, the findings could help doctors identify cancer patients who would benefit the most from drugs known as checkpoint blockade inhibitors. Checkpoint blockade inhibitors work by stimulating immune cells to destroy tumours in the body’s T cell response. In previous studies, findings have shown that these drugs work effectively in patients whose tumours have a large number of mutated proteins. However, 50% of patients who received the US Food and Drug Administration-approved checkpoint blockade inhibitor, pembrolizumab, did not respond well or only showed short-lived responses, despite their tumours showing high mutational burden. In a study of mice, the researchers revealed that measuring the diversity of mutations within a tumour generated a more accurate prediction as to whether immunotherapy treatment ...
The Danish artificial intelligence (AI) biotech Evaxion has announced plans to develop an mRNA vaccine against gonorrhea in partnership with Afrigen Biologics. Gonorrhea antigens identified through Evaxion’s EDEN platform have shown strong protective effects in preclinical trials, as per the company. The partnership will investigate the function of these antigens when presented in the mRNA form. After the validation phase, the partners will discuss an agreement for clinical development and commercialisation, with the opportunity to involve additional collaborators. Cape Town, South Africa-based Afrigen Biologics is set up as a specialised centre to support mRNA vaccine development and technology transfer, will take charge of developing the mRNA vaccine in low and middle-income countries and African territories. Gonorrhea is a sexually transmitted infection, caused by the bacteria Neisseria gonorrhoeae. It heightens the vulnerability to HIV, a prevalent health concern in many low and middle-income countries (LMICs). Birgitte Rønø, Evaxion’s Chief Scientific Officer ...
The universal vaccine could protect people against more strains of flu The National Institutes of Health (NIH) has announced that it has begun dosing patients in a phase 1 clinical trial of a new influenza (flu) vaccine candidate, FluMos-v2, to evaluate the vaccine for safety and its ability to elicit an immune response. Designed by researchers at the National Institute of Allergy and Infectious Diseases’s (NIAID) Vaccine Research Center (VRC), FluMos-v2 works to induce antibodies against various influenza strains by displaying part of the influenza virus haemagglutinin (HA) protein in repeating patterns on self-assembling nanoparticle scaffolds, preparing the immune system to recognise and fight the actual virus. Sponsored by NIAID, the trial is expected to enrol 24 healthy individuals, aged 18 to 50 years, to receive two intramuscular injections of the vaccine candidate, 16 weeks apart. The researchers aim to enrol 12 participants in a lower dose group (60mcg per ...
A study conducted by researchers at University College London and published in Cell revealed that if the protein mechanisms of COVID-19 variants are similar to those of other coronaviruses, it could potentially make identifying coronaviruses viruses with pandemic potential easier. The researchers looked at both the innate and adaptive immune systems – the two parts that form the human immune system. Innate immunity acts as the first line of defence. It is present from birth and tries to prevent infection spreading through a rapid response. Adaptive immunity, developing throughout our lives, enables us to recognise future pathogens by learning from past infections. The study, which focused on the ability of the most infectious COVID-19 variants to overcome our innate immune systems, found that variants could increase the production of proteins called Orf6, Nucleocapsid (N) and Orf96, to defeat our first line of defence. As a result, the virus has evolved ...
CSL Seqirus has unveiled new data highlighting the potential impact of influenza vaccines on reducing the burden seasonal flu has on hospitals and broader healthcare systems. The analyses were presented at this year’s European Scientific Working Group on Influenza (ESWI) Conference. Seasonal flu is an acute respiratory infection caused by influenza viruses, which circulate in all parts of the world. Although most people can recover within a week without requiring medical attention, the flu can cause severe illness among high-risk groups such as young children, older adults, pregnant individuals and those with serious medical conditions. In France, approximately 2 to 6 million people are affected by influenza every year, leading to over 20,000 hospitalisations and 10,000 excess deaths, 90% of which occur in older adults. According to findings from a dynamic transmission model, standard-dose quadrivalent flu vaccines for older adults could reduce hospitalisations by 11.1% to 30.0% and cut ICU ...
By Tristan Manalac Pictured: Novartis’ head office in Canada/iStock, JHVEPhoto Novartis on Tuesday gave back the investigational monoclonal antibody tislelizumab to BeiGene, returning worldwide rights to develop, manufacture and commercialize the anti-PD-1 candidate to the cancer-focused biotech. Also on Tuesday, BeiGene announced that the European Commission had approved tislelizumab for patients with unresectable, locally advanced or metastatic esophageal squamous cell carcinoma (ESCC) who had previously failed platinum-based chemotherapy. The treatment will be marketed under the brand name Tevimbra. After more than a year of regulatory delay, the FDA has also accepted BeiGene’s Biologics License Application (BLA) for tislelizumab as a first-line treatment option in this indication, according to Tuesday’s announcement. Novartis and BeiGene first entered into a collaboration and license agreement over tislelizumab in January 2021 to develop tislelizumab in ESCC, as well as other malignant diseases such as non-small cell lung cancer and hepatocellular carcinoma. In July 2022, however, ...
Sanofi and Ad Scientiam have partnered to launch an international, multicentre, longitudinal study to evaluate the ability of medical software, MSCopilot, to assess disability progression in people with multiple sclerosis (MS). The main objective of the MS-DETECT study, which explores digital biomarkers of MS, is to determine whether the MSCopilot software can effectively identify subtle and early signs of disability worsening. MS is a chronic immune-mediated, neurodegenerative disease of the central nervous system that currently affects 2.8 million people globally. The study will include 314 people with MS and will be conducted in the US, Canada, Germany, Italy, Spain, Denmark and France. Coordinating investigator and chairman of the study steering committee, professor Patrick Vermersch, believes that “these novel digital biomarkers are key to helping clinicians make appropriate treatment decisions and, ultimately, improve patients’ care.” As part of the agreement, the study will draw upon Ad Scientiam’s expertise in MS and ...
By Kate Goodwin Pictured: Human lung anatomy concept/iStock, magicmine Oxford-based biotech AlveoGene launched Thursday with plans to leverage a proprietary platform from the U.K.’s Gene Therapy Consortium to develop inhaled gene therapies for rare respiratory disorders. With seed funding from Oxford Science Enterprises, alongside other academia venture capital funds, AlveoGene has exclusively licensed the Gene Therapy Consortium’s next-gen lentiviral delivery platform—called InGenuiTy—for the treatment of respiratory diseases with high unmet need. However, use of the CTFR gene is excluded from the deal as Boehringer Ingelheim exercised its option in 2021 to develop the inhaled formulation, which is in the works for cystic fibrosis. AlveoGene’s first target will be treating Alpha-1 Antitrypsin Deficiency (AATD), one of the most common rare disorders. AATD is inherited and leads to reduced levels of a plasma protein that protects the lungs, leading to increased risk for lung and other diseases. It’s a major genetic risk ...
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