Breakthroughs Blog: Medicines

Interested in more global health innovation news? Every week GHTC scours media reports worldwide to deliver essential global health R&D news and content to your inbox. Sign up now to receive our weekly R&D News Roundup email. A six-month oral treatment regimen for rifampicin-resistant tuberculosis (TB) is substantially more effective than the current standard of care, according to preliminary trial results presented at the 52nd Union World Conference on Lung Health on Wednesday. The TB-Practecal trial tested a six-month regimen of bedaquiline, pretomanid, linezolid, and moxifloxacin (BPaLM) among 552 patients against the locally accepted standard of care at seven trial sites across Belarus, South Africa, and Uzbekistan. Findings showed around 89 percent of patients in the BPaLM group were cured, compared to 52 percent in the standard of care group. Current standard care for rifampicin-resistant TB can require up to 20 pills daily, along with injections, for up to 20 months.A new fingerstick blood test could be a promising tool for screening TB patients, interim study results indicate. The test, developed by Cepheid, gives results in under one hour using a few drops of blood. The new results, from a study of 195 participants in Gambia, Uganda, South Africa, and Vietnam, show an overall sensitivity of 91 percent and specificity of 86 percent, regardless of HIV status or geographical location, which meets the World Health Organization’s minimum specifications for a TB triage test. Researchers plan to expand trials to further test the technology’s accuracy, including among people who have other risk factors for TB, as well for people who have both TB and COVID-19.On Friday, the US Food and Drug Administration (FDA) released a briefing document indicating the Pfizer-BioNTech COVID-19 vaccine is more than 90 percent effective against symptomatic COVID-19 among children ages 5 to 11. The data is based on a 10-microgram vaccine dose, a third of the dose used for adults and older children, which minimizes side effects but still induces a strong immune response. The antibody response was comparable to the one seen in individuals 16 to 25 years old, and no new safety problems were identified in the companies' studies. The FDA also released an analysis later Friday showing the protection “would clearly outweigh” the risks of the shot. FDA advisers will meet Tuesday to discuss expanding authorization of the vaccine, currently greenlit for people over 12, to those ages 5 to 11.

Interested in more global health innovation news? Every week GHTC scours media reports worldwide to deliver essential global health R&D news and content to your inbox. Sign up now to receive our weekly R&D News Roundup email. On Wednesday, the World Health Organization recommended broad rollout of the world’s first malaria vaccine, known as RTS,S, among children in sub-Saharan Africa and in other regions with moderate to high P. falciparum malaria transmission. The recommendation is based on results from an ongoing pilot program—which has reached more than 800,000 children in Ghana, Kenya, and Malawi since 2019—showing the vaccine’s safety and effectiveness in remote and rural settings. Clinical trials of RTS,S, which was developed by GSK and PATH with funding from the Bill & Melinda Gates Foundation, indicated it reduces the risk of contracting malaria by 40 percent and risk of hospitalization for severe malaria by 30 percent. The vaccine will now face funding decisions from the global health community for broader rollout and country decision-making on whether to adopt the vaccine as part of national malaria control strategies.A new study has identified a compound able to block dengue virus replication in test tube experiments and in mice, researchers reported in Nature last week. There are currently no specific medicines to treat dengue, which infects more than 400 million people annually and can lead to fevers, rashes, and muscle and joint aches. Researchers behind the new study screened tens of thousands of small molecules for anti-dengue activity using an automated high-throughput testing process, then tweaked several molecules to produce more than 2,000 compounds for further testing. One of those drugs, JNJ-A07, proved equally potent against all four dengue serotypes in test tube experiments. In subsequent testing in mice, the compound was highly effective in reducing viral loads and viral-induced disease. The drug will be further tested in clinical trials for safety and efficacy in humans, according to developer Janssen Pharmaceutical Companies of Johnson & Johnson.The African Medicines Agency (AMA) treaty was ratified by its 15th African Union (AU) member country last week, meeting the threshold for the agency to be officially set up. The treaty was adopted by the AU in February 2019, though it required ratification and formal notification from 15 countries to launch implementation. Following Cameroon’s ratification on Tuesday, the AU now has 30 days, or until November 5, to begin setting up the agency, which is intended to support African countries in improving and harmonizing the regulation of medicines across the continent in order to improve access to quality, safe, and efficacious products. The AMA will be Africa’s second continental health agency, joining the Africa Centers for Disease Control and Prevention.

Interested in more global health innovation news? Every week GHTC scours media reports worldwide to deliver essential global health R&D news and content to your inbox. Sign up now to receive our weekly R&D News Roundup email. Merck plans to seek authorization for its COVID-19 antiviral pill after clinical trial results showed the drug, molnupiravir, cut the risk of hospitalization or death in half among high-risk individuals early in their infections. An independent board of experts monitoring the study data had recommended that the trial be stopped early because the drug’s benefit to patients had proved so convincing, and the US Food and Drug Administration agreed, according to Merck. The drug, designed to stop the virus from replicating, is taken as four capsules twice a day for five days. As an oral antiviral treatment, the pill could be a crucial new tool in combating COVID-19 by reaching more people than the antibody treatments being widely used in the United States.COVID Moonshot, an open-source effort to design a COVID-19 pill, has won US$11 million, or GBP£8 million, from the Wellcome Trust to develop a low-cost, broadly available antiviral to fight the pandemic. The project, coordinated by the Drugs for Neglected Diseases initiative, is a nonprofit consortium of scientists that began in March 2020 and led to around 250 people submitting more than 4,500 potential molecular designs intended to block the key protein that helps the virus replicate. The new funding, through Wellcome on behalf of the COVID-19 Therapeutics Accelerator, will help pay for the expensive last step of research needed to bring the project into human clinical trials. Moonshot hopes to have a compound able to be tested in humans by next year.Sanofi’s mRNA-based COVID-19 vaccine showed positive results in a phase 1/2 clinical trial, the company announced last week, providing confidence in its mRNA program that will now be used to pursue vaccines against other pathogens, including influenza. Sanofi will not be continuing to develop its mRNA COVID-19 vaccine, as the market will have been saturated by the time it would be authorized, the company said. In the preliminary phase 1/2 trial results, 91 percent of people who received the lowest tested dose saw their neutralizing antibody levels increase four-fold, a measure thought to equate to a protective level of antibodies, confirming that the mRNA platform is potent, can generate a strong immune response, and has no safety signal. Sanofi will now turn to accelerating its work on a vaccines “center of excellence” intended to be ready to produce vaccines when the next pandemic hits.

Interested in more global health innovation news? Every week GHTC scours media reports worldwide to deliver essential global health R&D news and content to your inbox. Sign up now to receive our weekly R&D News Roundup email. New study results show that combining antimalarial drugs with a vaccine, given just before peak transmission period of rainy season, could reduce malaria deaths in children by more than 70 percent. The trial—conducted over three years among 6,000 young children in Burkina Faso and Mali—tested the RTS,S malaria vaccine, the world’s first-developed shot for the disease, along with seasonal malaria chemoprevention (SMC), an existing preventative drug regimen. Results indicated the RTS,S vaccine was as effective as SMC when administered to children ahead of rainy season in countries with seasonal transmission—and that combining the two methods reduced cases by around 60 percent, hospitalizations by around 70 percent, and deaths by around 73 percent, when compared to SMC alone. The World Health Organization (WHO) is set to review evidence from the study in October.The International Vaccine Institute (IVI) and Bharat Biotech have launched a phase 2/3 trial for their chikungunya vaccine candidate. The two-dose vaccine, BBV87, is an inactivated whole virion vaccine, which showed “optimum immune response” in phase 1 studies in India. The phase 2/3 trial, led by IVI through the Global Chikungunya vaccine Clinical Development Program (GCCDP) in partnership with Bharat Biotech and supported by the Coalition for Epidemic Preparedness Innovations and the government of India’s epidemic preparedness initiative, Ind-CEPI, will test BBV87 for safety and immunogenicity in healthy adults. GCCDP aims to develop and manufacture an affordable chikungunya vaccine with the goal of achieving WHO prequalification to enable distribution in low- and middle-income countries. The trial has begun in Costa Rica and is set to expand to Panama, Colombia, Thailand, and Guatemala in the coming months.On Tuesday, the US Food and Drug Administration granted its first emergency use authorization for an at-home COVID-19 diagnostic that delivers results through the user’s smartphone. The Veritor rapid antigen test, developed by BD and Scanwell Health, involves swishing a nasal swab in a tube of liquid reagents, after which drops on a testing strip trigger colored lines, which are then read and interpreted by the smartphone. The diagnostic is designed for use by individuals ages 14 and up and can be used for children as young as two with samples collected by an adult. The digital test, which does not require a prescription, will initially be made available to employers, schools, and public health organizations looking to support routine screening programs.