Research Roundup: New financing scheme to boost access, mRNA tech to be applied to malaria vaccine research, and genetic engineering could help eliminate malaria

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.Solidarity, a global clinical trial led by the World Health Organization, will resume its efforts, testing three new treatments among hospitalized COVID-19 patients: artesunate, an antimalaria; infliximab, an antibody used to treat autoimmune disease; and imatinib, a cancer drug. The original Solidarity trial, which took place across dozens of countries simultaneously in 2020, found that none of the four treatments it tested showed benefit. While the original trial included antivirals, this new phase will focus on drugs that target the body’s immune response, rather than the virus itself, given the two drugs that have shown success in reducing mortality in hospitalized patients, dexamethasone and tocilizumbab, work by dampening patients’ immune reactions. Medicines have shipped to Finland as the first country to begin the trial with 40 more countries in the process of seeking ethical and regulatory approvals to join.Use of the monoclonal antibody therapy REGEN-COV reduced the risk of developing symptomatic COVID-19 infection by 81 percent in those exposed to an infected patient, according to a study published last week in the New England Journal of Medicine. The study, which included individuals over 12 who had a household member test positive within the prior 96 hours, found that by day 28, 1.5 percent of participants who received the therapy developed symptomatic infection compared to 7.8 percent who received the placebo. When including asymptomatic infections, risk reduction from the treatment was 66.4 percent. The study also found that patients who did become infected after receiving the therapy were more likely to have milder disease. These findings were used to support a decision late last month by the US Food and Drug Administration (FDA) to expand emergency use authorization for REGEN-COV to be used as a post-exposure prophylaxis among high-risk populations.Valneva shared positive results last week from the phase 3 trial of its single-dose vaccine against chikungunya, which found that 98.5 percent of participants saw protective antibody concentrations 28 days after receiving the shot. This protection rate exceeds the 70 percent threshold set by the FDA. The trial also found that the VLA1553 vaccine was generally well-tolerated and induced a high immune response among elderly trial participants. The trial will continue with outcomes evaluated until month six. VLA153 is currently the only chikungunya vaccine in phase 3 trials that targets long-term protection from a single-dose, according to its developer. The company signed an agreement earlier this year with the Instituto Butantan in Brazil for development, manufacturing, and marketing, with the goal of making the product more accessible in low- and middle-income countries.

The Biden administration has proposed a new, cutting-edge research entity that has the potential to benefit global health the way the Defense Advanced Research Projects Agency (DARPA)—the research agency behind the internet, GPS, and self-driving cars—benefited the rest of society. This proposal, the Advanced Research Projects Agency for Health (ARPA-H), could lead to transformative health innovations for neglected diseases and address gaps caused by market failures, ultimately bolstering global health research and development (R&D). What is ARPA-H? Initial proposals from the Biden administration and allies in Congress have described ARPA-H as a driver of scientific breakthroughs for innovations to prevent, detect, and treat diseases that will be housed within the National Institutes of Health (NIH). The idea for ARPA-H was originally conceived by the Suzanne Wright Foundation, a pancreatic cancer advocacy organization, which outlined a Health Advanced Research Projects Agency that would focus on overcoming market failures by de-risking investments from the private sector. Now that the proposal has been taken into the mainstream by the Biden administration and biomedical R&D champions like Representatives Diana DeGette (D-CO) and Fred Upton (R-MI), advocates hope this mandate of addressing market failures might also encompass global health issues including HIV/AIDS, malaria, tuberculosis, and neglected tropical diseases. The scope of research to be conducted under ARPA-H has not yet been set in stone, even though the president’s fiscal year 2022 (FY22) budget request lists as initial focus areas cancer, diabetes, and Alzheimer’s in its $6.5 billion request for the agency. NIH has organized listening sessions, including one for allergies, infectious diseases, and global health, to further discuss the potential research areas for ARPA-H.  To promote high-risk, high-reward research, ARPA-H will likely be modeled after DARPA, which has a flat and nimble organizational structure and a large budget with limited oversight. In contrast to NIH’s sometimes slow-moving peer review process and multiyear grants, DARPA utilizes program managers who make unilateral funding decisions based on research milestones achieved. If the agency is stood up, GHTC supports an ARPA-H funding framework that is similar to DARPA, as it could enable easy, flexible collaborations with product development partnerships—nonprofit partnerships that address public health goals.The Biden administration’s proposal to house ARPA-H within NIH has raised concerns among some advocates and skeptics that this could limit its flexibility and independence, two critical aspects of the ambitious high-risk, high-reward ARPA-H agenda. An additional concern is that placement within NIH could eventually limit growth of top-line funding available for other NIH institutes and centers, and the lack of clarity regarding the scope of focus areas for research has created hesitancy among congressional appropriators to fund the full $6.5 billion proposed. However, if ARPA-H is created with the flexibility and independence of DARPA, and its funding is additive so as not to diminish work on any other NIH program, the agency holds great promise for global health innovations. Global health potential With the right influence from advocates, ARPA-H has the potential to advance research that could benefit the lives of people living in impoverished and underprivileged communities worldwide. Poverty-related and neglected diseases (PRNDs) affecting these communities remain inadequately addressed by the market, as they offer little commercial incentive for the private sector to invest in developing medical products to diagnose, treat, or prevent them. The lessons of the COVID-19 pandemic highlight the need to address gaps in existing PRNDs as their impacts are exacerbated during health emergencies. With a focus on PRNDs, ARPA-H could make a substantial impact toward developing innovations to address the world’s most overlooked diseases.GHTC is not the only stakeholder that recognizes the global health potential for ARPA-H. During a Senate budget hearing for FY22 on NIH, Dr. Francis Collins, NIH’s director, said that one focus for ARPA-H could be the development of a universal vaccine that protects against the ten most common infectious diseases in a single shot. The opportunity for further influencing the scope and mission of ARPA-H remains open. Just recently, Representatives DeGette and Upton, lead sponsors of the landmark 21st Century Cures Act of 2016 who are now developing a follow-up bill, issued a request for information on the possible authorization of ARPA-H in their bill, with questions including, “To ensure it has the biggest impact, on what activities or areas should ARPA-H focus?” In response, GHTC submitted feedback advocating for a focus on PRNDs, including HIV/AIDS, tuberculosis, malaria, and neglected tropical diseases, as well as antimicrobial resistance, arguing that a focus on these areas of historic market failures holds the greatest potential for ARPA-H’s societal impact. ARPA-H could produce transformative health innovations in chronically underfunded areas. If the agency is authorized, GHTC and other advocates should encourage lawmakers to ensure R&D for PRNDs is prioritized. The innovations that result could yield additional advancements across the entire field of biomedical R&D, improving health for all.