Breakthroughs Blog: Medicines

Last week, Moderna announced that it has received an award from the Coalition for Epidemic Preparedness Innovations (CEPI) for up to $54.3 million to support ongoing development of its bird flu mRNA vaccine candidate, after US government backing was cancelled earlier this year. There are currently no vaccines available specifically for bird flu, as the virus continues its worrying spread among animal populations in the United States and globally. A vaccine, especially one using the mRNA platform known for its speed and adaptability, could play a crucial role in curbing a future outbreak in humans with pandemic potential.  A research team led by the Australian Peter Doherty Institute for Infection and Immunity has identified a tool that can detect emerging hotspots for Buruli ulcer years before human infections occur, as the serious bacterial skin infection continues to spread across Australia and endures in other regions around the world. The infection has a long and variable incubation period, making it hard to identify when and where patients were exposed. The researchers tracked the excreta of possums, who have small ranges, making the presence of the bacterium that cause the infection in their excreta a likely sign that it is present locally. Then, they used DNA sequencing to compare the bacterial genomes of possum-derived and human-derived infections, finding where they overlapped to identify areas where human infections could occur. The method aims to allow public health authorities to implement targeted interventions early on, far before human cases occur. Two new studies looking at preventive RSV vaccines and drugs found that that their effectiveness waned over time, suggesting that boosters may be needed to ensure long-lasting protection, as RSV continues to pose a growing seasonal challenge, especially for older adults and infants. The first study found that one dose of the Abrysvo RSV vaccine was highly effective at preventing infections and reducing hospital, emergency department, and intensive care unit admissions in US veterans 60 and older, but this protection decreased over time, with the reductions most notable for immunocompromised individuals. A second study found that the long-acting monoclonal antibody nirsevimab was highly effective in hospitalized European children two years or younger for one season, but after that, protection waned. Both studies demonstrate the real-world impact of these crucial tools and suggest that boosters could be implemented to ensure long-lasting protection, especially for higher-risk individuals. 

A study, led by the Global Antibiotic Research and Development Partnership, testing zoliflodacin, found that the antibiotic was as effective and tolerable as the standard treatment for gonorrhea and was effective against drug-resistant strains of the bacteria, as resistance continues to rise globally. Injectable ceftriaxone is our last remaining antibiotic that works against most strains of Neisseria gonorrhoeae; however, we are already seeing evidence of evolving resistance, underscoring the urgent need for new antibiotic options to treat drug-resistant cases. If approved by the US Food and Drug Administration, which is expected to make a decision soon, zoliflodacin could be used as both a first-line treatment and a last-resort option after standard treatments fail due to drug resistance.An international team of researchers has made the first major step toward a new and improved vaccine or antibody therapy to address mpox, a dangerous virus that has been behind worrying outbreaks that have emerged around the world. Using the blood of patients with previous infection or vaccination, researchers identified 12 antibodies that could clear the virus. Then, another research team used artificial intelligence (AI) to pinpoint what part of the virus acted as an antigen for those antibodies—a process that could have taken years without the use of AI—and then engineered an antigen to elicit similar antibodies in mice, which produced antibodies that neutralized the virus when injected with it. The researchers are now working to develop versions of the vaccine antigen and antibodies that are more effective at fighting disease and also cheaper and easier to produce than existing products that use a weakened version of the related poxvirus. The Korean Disease Control Agency, the International Vaccine Institute (IVI), ST Pharm, and Seoul National University are partnering with the Coalition for Epidemic Preparedness Innovations (CEPI) to advance a new mRNA vaccine against a deadly emerging virus, which was designed with the use of AI. Cases of the tick-borne thrombocytopaenia syndrome, or SFTS, have been reported in five Asian countries and cause severe symptoms and, in several cases, death. IVI will advance the vaccine candidate into preclinical and Phase 1 and 2 clinical trials in Korea, the first time a candidate for the virus would be tested in humans. STFS virus is also part of the Phenuivirus family; a successful vaccine against this virus could advance work to develop vaccines against other closely related viruses with pandemic potential. 

Newly published data shows that the antiviral compound mosnodenvir, developed by Johnson & Johnson, is effective at preventing dengue infection in people deliberately exposed to the virus, which affects millions around the world every year. There are no approved treatments for dengue, and available vaccines face production and efficacy issues. Despite its promise, the future development of the drug is in flux after Johnson & Johnson halted all its infectious disease work last year. Negotiations are underway to find another company to advance the drug, which, if it continues to demonstrate potential, could be used to protect large populations during epidemics, as well as to protect people traveling to endemic areas. Last week, Brazil’s regulatory agency approved the Butantan Institute’s Butantan-DV vaccine for dengue for people aged 12 to 59. The vaccine demonstrated high efficacy in clinical trials against dengue, which causes millions of cases globally, with a substantial burden faced by Brazil. The single-dose vaccine could offer faster and simpler vaccination campaigns compared to existing vaccines. Brazil signed an agreement with WuXi Biologics to deliver approximately 30 million doses of the vaccine in the second half of next year. The results of a human-challenge Phase 2a trial published last week found that an mRNA vaccine was more effective against symptomatic and febrile flu compared to the conventional vaccine. Another trial published last week also found that mRNA flu vaccines were more effective than conventional vaccines. Beyond effectiveness, mRNA vaccines can also be made more quickly, helping ensure that vaccines match circulating flu strains. Additional studies that include older people, those with coexisting illnesses, and school-age children, who face elevated risks, will be required to ensure broad efficacy across populations. 

At the recent Union World Conference on Lung Health, TB Alliance presented the results of a Phase 2 clinical trial showing that the novel antibiotic candidate sorfequiline has the potential to improve tuberculosis (TB) treatment when combined with pretomanid and linezolid in people with drug-sensitive TB. The regimen was more effective than the current standard of care, and sorfequiline also demonstrated greater activity than bedaquiline, a key component of the BPaL regimen, a previous breakthrough that drastically reduced treatment time for most drug-resistant TB. This new treatment could shorten TB therapy even further. TB Alliance is working with partners ahead of the launch of a Phase 3 trial next year. Researchers at the University of California San Diego and partners have developed a new method to combat antibiotic-resistant bacteria, which could pave the way for developing alternatives to traditional antibiotics for treating a wide range of deadly infections, as the global threat of antimicrobial resistance continues to grow. The researchers used bacteriophages (or phages), which are viruses that infect and kill bacteria. Phages have been used to treat bacterial infections for more than 100 years but are usually limited in the bacterial strains they can successfully target. Researchers overcame this by allowing the pages to evolve alongside bacteria in a controlled laboratory setting for 30 days, enabling the phages to adapt their bacterial defenses, becoming more effective at killing a wide variety of bacterial strains, including drug-resistant ones, and better at suppressing bacterial growth for extended periods. Researchers at Indiana University have developed a molecular test that can simultaneously detect three dangerous fungi much faster than traditional methods, helping patients receive accurate, timely treatment against this growing global challenge. The test works by targeting genetic regions unique to each fungus, and it was much faster and more accurate than standard methods, a key advance as fungal infections are often mistaken for other respiratory illnesses. While the test still needs to be clinically validated in further studies, the research team believe it could eventually fill a major gap in fungal diagnostics.