Breakthroughs Blog: Research Roundup

An international clinical trial led by the Global Antibiotic Research and Development Partnership (GARDP) seeking improved antibiotic treatments for newborn sepsis has expanded to India. Neonatal sepsis is one of the leading causes of newborn deaths worldwide and is becoming harder to treat due to rising antibiotic drug resistance. Newborns have long been underrepresented in antibiotic research, leaving health care providers without an evidence-based standard-of-care treatment. The lack of a standard treatment has in turn complicated neonatal sepsis research because there is no obvious treatment to use as a comparison in trials. The trial aims to test and rank multiple potential antibiotic combinations for neonatal sepsis.Last week, the World Health Organization announced that a clinical trial evaluating two potential antiviral therapeutics for the Bundibugyo strain of Ebola will be launched in the Democratic Republic of the Congo next week amid the ongoing outbreak. There are currently no approved vaccines or therapeutics for the Bundibugyo strain, which is only known to have caused three outbreaks in history. The trial will evaluate the ability of Mapp Biopharmaceutical’s MBP134 and Gilead Sciences’ remdesivir to reduce mortality in patients, alone and in combination. The United States and Gilead Sciences are donating all the doses that will be used in the trial.The US Food and Drug Administration (FDA) has approved the first generic version of rifapentine as part of combination treatment regimens for active and latent pulmonary tuberculosis (TB). Rifapentine was first approved by FDA in 1998 to treat pulmonary TB. For active pulmonary TB cases in patients 12 or older, the generic antibiotic is approved only in combination with one or more anti-TB drugs, while for latent TB cases, the generic is approved in combination with isoniazid to treat patients 2 or older at high risk of progressing to TB disease.

Last week, the Food and Drug Administration’s (FDA’s) Vaccines and Related Biological Products Advisory Committee voted unanimously to recommend approval of Moderna’s mRNA influenza vaccine for older adults. In a Phase 3 trial in more than 40,000 adults in 11 countries during the 2024-2025 flu season, the vaccine demonstrated significantly better protection than standard-dose shots. Moderna's vaccine also uses mRNA technology, meaning it can be updated more quickly to match circulating flu strains compared to traditional vaccines that use egg-based methods. After initially agreeing to review the vaccine, the FDA had previously raised concerns about the Phase 3 trial for the vaccine, prompting backlash from vaccine and public health experts, before deciding to go ahead with its review. Following the recommendation, the FDA will make a final decision on approval, which is expected by early August.The Global Health Innovative Technology (GHIT) Fund has started an emergency framework to surge support and funding for the development of rapid and point-of-care diagnostic technologies in response to the Ebola outbreak caused by the Bundibugyo virus in the Democratic Republic of the Congo and Uganda. There are currently no approved vaccines, therapeutics, or point-of-care diagnostics for Bundibugyo virus. GHIT Fund is prepared to support fast-track funding for infections caused by filoviruses, which include Ebola, with the aim of advancing a diagnostic test, particularly one that is suited to low- and middle-income countries with limited infrastructure, that can improve control capacity for this and future outbreaks.  Inflammatix is collaborating with the University of Nebraska Medical Center to explore the use of the company’s diagnostic test platform for hantaviruses after the recent hantavirus outbreak exposed gaps in testing capacity due to limited technology and testing standards. Inflamattrix’s TriVerity test is a blood-based test that used machine learning to detect infection. It previously received clearance from the US Food and Drug Administration as a sepsis test in 2025. The partners are aiming to develop a tool that can assess the early signatures of infection in people with high-risk exposure to the virus, which could help improve early response capacity in future outbreaks.  

The Democratic Republic of the Congo has approved the use of a new single-dose oral treatment for sleeping sickness, a potentially fatal disease which is endemic in several countries in Central and West Africa. The approval of Acoziborole Winthrop, codeveloped by the Drugs for Neglected Diseases initiative (DNDi) and Sanofi, follows successful Phase 2 and 3 clinical trials conducted in the DRC and Guinea by DNDi in partnership with national authorities, which demonstrated that the drug is safe and effective. Existing treatments require a ten-day oral treatment regimen or a combination of injection and oral therapy that requires several days of hospitalization for patients in the most advanced stages. The DRC approval, which will make the drug available free of cost to patients in the country, is expected to be followed by endorsements in other endemic countries soon.A research team from the University of Maryland School of Medicine’s Center for Vaccine Development and Global Health has shared promising early results from a first-in-human clinical trial comparing a new investigational vaccine against Lassa fever and rabies to a licensed rabies vaccine, finding that the vaccine candidate was safe and induced rapid and robust immune responses against both viruses. There are currently no available vaccines for Lassa fever, a dangerous disease considered a priority threat with epidemic and pandemic potential that is common in parts of Western and sub-Saharan Africa, which also have a high burden of another severe disease: rabies. A dual vaccine could help streamline health care delivery in settings with limited health care infrastructure; the vaccine candidate can also be freeze-dried for storage, making it well suited to low-resource settings where it can be difficult to maintain the cold chain. Last week, DNDi, the Medicines for Malaria Venture (MMV), and the Global Antibiotic Research and Development Partnership (GARDP) announced a new cooperative agreement to explore how the three organizations can work together to advance global health research and development (R&D) amid global aid cuts and calls to reshape the global health architecture. The organizations plan to pool their expertise and resources to advance the R&D of effective and affordable medicines, while centering patient needs and equity. In addition, the partners will collaborate on policy advocacy and communications activities to raise awareness of the impact and promise of the nonprofit product development partnership model in advancing innovative, lifesaving medicines for critical, but often overlooked, diseases.  

A Phase 1 clinical trial testing a candidate that could become the first vaccine for leishmaniasis, a disfiguring skin disease, is set to begin soon in Kenya and Brazil, where the disease in endemic, as well as in Cincinnati, Ohio, as the disease risk grows in the United States. The Global Health Innovative Technology (GHIT) Fund is supporting the trial, which will test the safety and immune response of the candidate in healthy people. The vaccine, which was developed using CRISPR gene-editing technology to mutate the parasite that causes the disease, previously demonstrated strong results in mice and has received US Food and Drug Administration approval as an investigational new drug, which allows the product to be tested in humans. The Coalition for Epidemic Preparedness Innovations (CEPI) has announced that it is providing funding to fast-track the development of three Ebola Bundibugyo virus vaccine candidates, as the concerning outbreak of the Bundibugyo strain, for which there are no approved vaccines, continues to grow in the Democratic Republic of the Congo and Uganda. Specifically, the funding will support preclinical development and Phase 1 clinical testing of Moderna’s mRNA vaccine candidate; help accomplish a key early step in vaccine manufacturing for IAVI’s vaccine, which is based on the organization’s rVSV vaccine platform; and help the University of Oxford prepare for a Phase 1 trial of its vaccine, which is based on the same platform as its Oxford/AstraZeneca COVID-19 vaccine.  Last week, Zendal announced the launch of the international MTBVAC AVANTE consortium to accelerate clinical development and prepare regulatory and policy pathways to ensure broad and equitable access of the MTBVAC tuberculosis (TB) vaccine candidate over the next four years. The current TB vaccine is more than 100 years old and offers inconsistent protection in adults against pulmonary TB, so a new and improved vaccine that is safe and effective across diverse populations who face TB risk will be essential to ending the pandemic. With funding from the Global Health EDCTP3, the consortium of 12 European and African partners will initially support two major ongoing trials in high-TB regions in sub-Saharan Africa, one evaluating the vaccine in adolescents and adults and one comparing the candidate’s protection in newborns to the existing vaccine.