Breakthroughs Blog: BARDA

Recently published results from a large multi-country study found that pooled tuberculosis (TB) testing could greatly reduce costs and boost access to diagnosis. Access to the gold standard of rapid molecular testing is limited—only just over half of people diagnosed with TB in 2024 were first tested using the technology. Instead, they were diagnosed using older technology, which can take weeks to produce results, during which the infection could progress or be transmitted to others. Millions of additional individuals with TB go completely undiagnosed. A major contributing challenge is that rapid molecular testing requires an expensive diagnostic cartridge for each sample. Pooled testing involves testing samples from multiple individuals with one cartridge. Patients are only retested individually if the result is positive. Unlike new diagnostic technologies, like AI-enabled portable X-ray machines, that are promising but would require additional funding to implement, pooled testing could be scaled up rapidly without new funding because it utilizes technologies that many labs already have.Medicines for Malaria Venture (MMV) is advancing the novel, first-in-class antimalarial compound, cabamiquine, into the next stage of clinical development, with the hope that the compound could be part of a single-dose, long-acting treatment for the prevention and treatment of malaria, which could greatly simplify care compared to current options. Cabamiquine was discovered by researchers at the University of Dundee, with financial and scientific support from MMV, and then advanced by Merck to proof of concept. Recently published results from two Phase 2a trials showed that a regimen of cabamiquine and pyronaridine was able to clear uncomplicated Plasmodium falciparum malaria in more than 90 percent of participants and demonstrated a strong safety profile. MMV will now lead Phase 2b clinical development of the compound. The Biomedical Advanced Research and Development Partnership (BARDA) recently announced that it has awarded Scynexis a $214 million contract for the development of an antifungal to treat and prevent invasive fungal infections, the less common but more serious form of fungal infection that penetrates deep into the tissues of the body. Fungal infections are a growing global challenge, but there is a significant gap in the pipeline of new tools to address them. Last year, the company reported positive findings from a Phase 1 trial of an oral dose of the candidate and is currently conducting a Phase 1 trial of an intravenous formulation. The award includes initial funding to advance the company’s antifungal candidate into Phase 2 trial, with an option for additional, longer-term funding. 

Amid the ongoing outbreak of the Bundibugyo strain of Ebola—for which there are no specific approved vaccines or therapies—in the Democratic Republic of the Congo and Uganda, the World Health Organization (WHO) has collaborated with outside experts to identify priority Ebola treatments and vaccines for clinical trials. On the treatment side, WHO recommended prioritizing Mapp Biopharmaceutical’s MBP134, Regeneron’s maftivimab, and Gilead Sciences’ antiviral remdesivir. For prevention, Gilead's experimental oral antiviral obeldesivir was highlighted as a priority for post-exposure use in contacts of confirmed cases. IAVI’s rVSV Bundibugyo was identified as the most promising vaccine candidate, but Oxford University and the Serum Institute of India’s ChAdOx1 Bundibugyo vaccine candidate could be available for testing sooner following additional animal testing. WHO is now working with authorities in Congo and Uganda and other partners to design and implement ethical clinical trials during the outbreak. An experimental drug for chronic hepatitis B could potentially serve as a functional cure for the virus, as demonstrated in recently published results from two international studies. For many, hepatitis B only causes short-term illness, but for some, it can become a chronic infection, gradually damaging the liver and, without treatment, leading to liver cancer, liver failure, or even death—it causes around 1.1 million deaths globally each year. Chronic infection requires lifelong therapy in the form of daily pills to reduce viral levels and prevent liver damage, which can be hard to adhere to or access in some countries. Bepirovirsen, developed by GSK and Ionis Pharmaceuticals, was able to reduce the virus in around one in five patients to levels low enough for the immune system to keep it under control without treatment. The drug is under fast-track review by the US Food and Drug Administration, which is expected to make a decision later this year, and is also being considered by international regulators in Japan, China, and Europe. A research team at Georgia State University has developed a promising antiviral drug candidate that could help treat measles, croup, and related viral diseases that cause contagious and life-threatening respiratory infections, as falling coverage of the vaccine for measles, mumps, and rubella raises the risk of infection. The drug, GHP-88310, was designed to be a once-a-day oral pill for vulnerable patent groups, like children and immunocompromised patients, which make up the majority of croup and measles cases. GHP-88310 has been shown to be well-tolerated in rats, mice, ferrets, and dogs at higher concentrations. 

Last week, the Global Health Investment Corporation announced the launch of the Global Health Security Fund, which will strategically invest in companies to accelerate the development of technologies that strengthen global health security. The investments will focus on companies developing vaccines, therapeutics, diagnostics, and enabling technologies with impact and commercial potential, addressing critical gaps in global health preparedness and response. The fund has received support from the US Biomedical Advanced Research and Development Authority (BARDA), as well as investors including the Coalition for Epidemic Preparedness Innovations (CEPI), Flu Lab, Eli Lilly and Company, Meiji Seika Pharma Co., The MITRE Corporation, and the Wisconsin Alumni Research Foundation. A research team at the La Jolla Institute for Immunology has shared promising research that could inform the development of a vaccine that provides protection against a host of viruses in the arenavirus family. Arenaviruses, which are most common in West Africa, are considered to be a viral family with significant pandemic potential and are very similar to hantaviruses, the virus family behind a current worrying outbreak, in how they spread and function. The researchers demonstrated how harnessing T cells could be critical to designing vaccines that can combat many different species of virus at once, an approach that could be applied to other viral families as well. BARDA is working with US-based company Mapp Biopharmaceutical to supply its experimental Ebola treatment for potential use in people who may have been exposed in the current outbreak of Bundibugyo ebolavirus, a strain for which there are no specific vaccines or treatments available. Mapp’s investigational monoclonal antibody treatment was developed through a longstanding public-private partnership supported by BARDA to address Ebola Sudan, which is closely related to other ebolaviruses, Laboratory studies suggest the treatment has the potential to be effective against the Bundibugyo strain. 

Last week, Shionogi announced that its US subsidiary has received a $119 million contract from the Biomedical Advanced Research and Development Authority (BARDA) to boost production and advance development of a key antibiotic, helping improve US preparedness for future outbreaks of drug-resistant bacterial pathogens. Specifically, the funding will go toward the establishment of a US manufacturing site and procurement of cefiderocol, an antibiotic that has the ability to target difficult-to-treat, multidrug-resistant infections, which is already approved for several indications. The award will also fund the development of cefiderocol to respond to high-priority bacterial threats. In a newly published study, a Cornell University research team shared the results of a long-term study that could be a first step toward developing the first safe, reversible, long-acting, and 100 percent effective nonhormonal male contraceptive. In a proof-of-principle study in mice, the researchers targeted a natural checkpoint in meiosis, the reproduction process for sex cells, and were able to safely stop sperm production and, importantly, then recover normal meiosis and complete sperm function, with no impacts on other functions or on offspring. If this approach continues to demonstrate success in further studies, it could eventually be utilized in an every-three-month injection or patch contraceptive product. IAVI recently announced that the first participants have been vaccinated in a Phase 1 clinical trial evaluating the safety and immunogenicity of its investigational vaccine for Marburg virus disease, a highly fatal, high-priority disease, which has been behind a growing number of outbreaks in sub-Saharan Africa in recent years and for which there are no specific approved vaccines. In nonclinical studies, the candidate demonstrated consistently strong efficacy results: a single intramuscular vaccination protected 100 percent against lethal challenge with the virus. IAVI is simultaneously continuing to work with partners in sub-Saharan Africa to ensure the availability of candidate doses for evaluation during future Marburg outbreaks.