Breakthroughs Blog: Access

Newly shared results from a Phase 3 clinical trial found that a single-dose dengue vaccine was highly effective at preventing severe dengue, as the mosquito-borne disease continues to spread into new areas around the world. The vaccine, developed by the Butantan Institute in Brazil, was recently approved in Brazil for use in people aged 12 to 49 years old. The new trial found that among thousands of participants aged 2 to 59 years old, the vaccine was effective at preventing symptomatic dengue but especially effective—80.5 percent— at preventing hospitalization for at least five years. The World Health Organization (WHO) has shared new guidance highlighting critical targets for new antibiotics, as the global threat of antimicrobial resistance (AMR) continues to grow and outpace the development of new and innovative antimicrobials. The three areas outlined are the development of new drugs to fight Gram-negative bacteria that cause serious infections that are especially difficult to fight; new drugs for patients with serious infections that can no longer be treated with vancomycin, a powerful last-resort antibiotic; and new treatments for bacterial meningitis, which is getting harder to treat due to growing antibiotic resistance. While the research and development of a new antibiotics can be difficult, expensive, and not as profitable as other products, WHO aims to highlight key areas for investors and developers to focus their efforts to grow the pipeline of new, urgently needed tools. Last week, the Sabin Vaccine Institute announced that it has completed enrollment in six Phase 2 clinical trials for its Marburg and Sudan Ebolavirus vaccines in Kenya, Uganda, and the United States, as increasing outbreaks of both viruses in recent years have highlighted the urgent need for vaccines. There are currently no licensed vaccines for protection against the highly contagious, life-threatening diseases caused by these viruses. The Marburg vaccine has also been evaluated during recent outbreaks in Rwanda in Ethiopia. Both candidates have so far demonstrated strong safety and efficacy profiles. The Sabin Vaccine Institute has also begun planning larger Phase 3 trials for each of the vaccines. 

The European Medicines Agency’s Committee for Medicinal Products for Human Use has granted a positive opinion to a new and simpler treatment for sleeping sickness, a serious vector-borne disease found in rural sub-Saharan Africa, for which the current treatment can be long and challenging. The Drugs for Neglected Diseases initiative (DNDi) and Sanofi co-developed a single-dose oral treatment, acoziborole, for both early- and advanced-stage infections in people 12 and older, which was found to be highly effective in a pivotal clinical trial. The endorsement will support further regulatory approval and rollout. Sanofi has pledged to donate doses to the World Health Organization, helping improve access to the new drug in the places where it’s most needed. Another study underway is testing the drug in children between 1 and 14 years old. The Global Antibiotic Research and Development Partnership (GARDP) recently announced that it has acquired all data and licensing rights for apramycin, an antibiotic introduced in the early 1980s to treat serious bacterial infections in animals, to evaluate its potential as a promising antibiotic against drug-resistant infections in humans. The drug has shown potential for human use in preclinical trials, but early-stage development stalled after the company developing it closed. The organization has planned a Phase 1 study set for 2027 to assess safety and pharmacokinetics. Last week, the Combating Antibiotic-Resistant Bacteria Biopharmaceutical Accelerator (CARB-X) announced that it’s awarding $1.2 million to Harvard University’s Andrew G. Myers Research Group to develop novel antibiotics for multidrug-resistant bacteria like Escherichia coli and Klebsiella pneumoniae, which cause serious infections around the world. Despite their significant global threat, the pipeline for novel antibiotics against these priority pathogens is lacking. The Harvard team will use the funding to further preclinical development of a new class of antibiotics that target a critical part of multidrug-resistant bacterial pathogens that plays a key role in the pathogens’ ability to cause infection and evolve drug resistance, which the researchers hope will be able to overcome drug-resistant infections without contributing to the emergence of broader cross-resistance.  

Sino Biological, Inc. recently announced an effort to accelerate the development of critical research tools to support the global scientific community in advancing Nipah virus research, as new cases of the deadly pathogen have raised international concerns. Sino Biological has launched a series of proteins, which researchers can use in the lab to better understand how the virus infects cells and how to design antibodies that might successfully target the virus. These proteins are based on the currently circulating Nipah virus strains, so could help boost the accuracy and relevance of research efforts. There are currently no approved vaccines or specific antiviral treatments for Nipah, underscoring the importance and urgency of research and development efforts to advance vaccines and therapies. Newly shared results from the first blinded, randomized non-inferiority trial comparing one versus two doses of HPV vaccine show that a single dose provided similar protection to the two-dose schedule. These results verify a previous study, which undergirded the World Health Organization’s 2022 recommendation of the lower-dose regimen, which has since led to its adoption by almost 90 countries. The lower-dose regimen can help boost access and uptake of the HPV vaccine by reducing the cost and complexity of vaccination for public vaccine procurers, health care providers, and patients. Specifically, the researchers found that a single dose was 97 percent effective against HPV infections in a large study with 20,000 participants, who were studied for up to five years post-vaccination. Last week, the Drugs for Neglected Diseases initiative (DNDi) announced a new memorandum of understanding with the African Population and Health Research Center aimed at helping translate African-led research into transformative innovations and health policies. Specifically, the agreement involves coordination to promote joint research, advance evidence-based programming and policymaking, and facilitate capacity-building. The memorandum also outlines collaborative commitments to ethical, value-enhancing data-sharing activities, including generating high-quality evidence and creating a robust system to preserve and share findings. 

The Coalition for Epidemic Preparedness Innovations (CEPI) is providing $30 million to support the collaborative development of an updated Ebola vaccine, enhancing global preparedness against this deadly health threat. The updated vaccine will build on Merck’s vaccine, which has a complex and expensive manufacturing process that is vulnerable to supply chain disruptions, as well as logistical challenges, including an ultra-cold storage requirement, which is unsustainable in remote, low-resource settings that have a higher likelihood of outbreaks. The partners will update the current manufacturing processes, hopefully lowering costs and making the vaccine easier to manufacture and store. The reduced production costs are expected to enable Merck to make the vaccine available in low- and middle-income countries at a more affordable price. The updated vaccine will be compared against the current vaccine in a Phase 3 trial, also funded by CEPI. A new study led by the University of Cambridge suggests that mpox may be capable of spreading asymptomatically, meaning that transmission may be more widespread in Africa than previously thought. The researchers analyzed blood samples from 176 healthy adults with no known exposure or history of infection in Nigeria, which has experienced intermittent outbreaks of the virus for decades. At least five participants had mpox antibodies in their blood, suggesting recent infection, but none of them reported any symptoms. These findings will inform how public health authorities track the spread of the virus, which has been behind several major outbreaks in recent years. This research also underscores the urgent need for improved vaccines and other tools for mpox. ENABLE-2, an antibiotic drug development engine backed by the Swedish Research Council and the National Research Programme on Antibiotic Resistance, is supporting the Global Antibiotic Research & Development Partnership (GARDP) to develop a novel class of antibiotics. The researchers plan to use an innovative approach by targeting a specific protein complex that bacteria use to strengthen their outer membrane. The ultimate goal of the work is to develop a new treatment for priority antibiotic-resistant bacterial pathogens. GARDP also aims to ensure the new treatment can be made accessible globally, including in low- and middle-income countries, which face a particular threat from antibiotic resistance.