Single Centre Open-label, Non-randomised, 3-treatment, 2-period, Pharmacokinetic Drug Interaction Study of Single Oral Dose of Acoziborole With Sequential Co-administration of Midazolam and Dextromethorphan in Healthy Male Participants

To assess Drug drug interactions between Acoziborole and Dextromethorphan and Midazolam in healthy male volunteers.

Start Date09 Feb 2023

Sponsor / Collaborator

Drugs for Neglected Diseases initiative

NCT05433350

/ CompletedPhase 2/3IIT

Pharmacokinetic, Efficacy, Safety, and Tolerability Study of a Single Dose of Acoziborole Under Fasting Conditions in Paediatric Patients From 1 to 14 Years of Age and With g-HAT: a Multicentre, Open-label Study

Acoziborole has been studied in an open-label pivotal Phase II/III trial (DNDi-OXA-02-HAT) in the DRC and Guinea. As the numbers of reported cases diminish, resources for surveillance and specialised screening will also taper. This decrease, coupled with the loss of diagnostic skills and disease management expertise, will lead to a weak and less specialised HAT technical environment. The history of g-HAT has shown that outbreaks or re-emergence of the disease have already happened under different circumstances when surveillance was relaxed or simply because the populations at risk live in areas of political instability, limiting access to specialised care. Even with a steady decrease of reported incidence, no model can currently predict that HAT could not re-emerge.
Although g-HAT is predominantly a disease of adults, children are also affected at diverse rates depending on the geographical and behavioural characteristics in the different areas of disease transmission. Hence efforts are needed to develop a paediatric formulation from a new generation of oral HAT treatments.

Start Date09 Jul 2022

Sponsor / Collaborator

Drugs for Neglected Diseases initiative

NCT05256017

/ CompletedPhase 2/3IIT

Safety and Tolerability Study of Acoziborole in g-HAT Seropositive Non-parasitologically Confirmed Subjects: a Multicentre Randomised Double-blind Placebo-controlled Study

Human African trypanosomiasis (HAT) or sleeping sickness is a tropical disease which is endemic in sub-Saharan Africa. Most cases of HAT are due to the parasite Trypanosoma brucei gambiense (T.b. gambiense), which is transmitted by the bite of the tsetse fly. HAT can be fatal without diagnosis and treatment. Several treatment options are currently available to treat HAT caused by the T.b. gambiense parasite (g-HAT), but these treatments can be administered only after demonstrating via microscopy the presence of the parasite in a body fluid. However, there are factors such as low parasitaemia and the complexity and low sensitivity of parasitological methods that make such demonstration difficult. It has been demonstrated that a variable proportion (mainly depending on the prevalence) of such g-HAT "sero-suspects" are confirmed cases and, therefore, remain potential reservoirs of the parasite and a source of new infections hindering the efforts to eliminate the disease.
The drug acoziborole was evaluated in a study called "DNDi-OXA-02-HAT". During this study, patients with g-HAT from the DRC and Guinea took a single dose of acoziborole. This study showed that acoziborole has a high efficacy and is safe for treating patients with confirmed g-HAT .
The present study is called "DNDi-OXA-04-HAT". It included seropositive participants from the DRC and Guinea who did not have parasites detected via microscopy in a body fluid. Its objective was to collect data on the safety and tolerability of a single dose of acoziborole compared to a placebo (i.e. a dummy treatment). The results of this study would help decide if acoziborole can be used in the population of g-HAT seropositive individuals and help eliminate the HAT disease.

Start Date30 Dec 2021

Sponsor / Collaborator

Drugs for Neglected Diseases initiative

100 Clinical Results associated with Acoziborole

100 Translational Medicine associated with Acoziborole

100 Patents (Medical) associated with Acoziborole

Literatures (Medical) associated with Acoziborole

05 Nov 2025·ANTIMICROBIAL AGENTS AND CHEMOTHERAPY

Mass balance, pharmacokinetics, metabolism, and excretion of radiolabeled acoziborole, a potential novel treatment for human African trypanosomiasis, following single microtracer oral dose to humans

Article

Author: Launay, Delphine ; Pelay-Gimeno, Marta ; Wauthier, Valérie ; Tarral, Antoine ; Gillon, Jean-Yves ; Loyau, Sabrina ; Andel, Lotte van ; Sidhu, Sharan ; Rembry, Sandra ; Louis, Mathieu ; Weinling, Estelle ; Simon, François

ABSTRACT:

Acoziborole is an oxaborole 6-carboxamide active against Trypanosoma brucei gambiense and rhodesiense , the parasites responsible for human African trypanosomiasis. This open-label, phase I study in six healthy male participants evaluated the mass balance, pharmacokinetics, metabolism pathways, and excretion of a single oral 960 mg dose of [ 14 C]-acoziborole. Blood, plasma, and feces were collected for 120 days and urine for 16 days. The excretion balance and systemic exposure of total circulating radioactivity were determined using accelerator mass spectrometry. Metabolism profiling was performed in pools of plasma, urine, and feces. Liquid chromatography coupled with tandem mass spectrometry quantified acoziborole and its metabolite SCYX-3109 in plasma and urine. By Day 60, 87.3% of the total radioactivity had been recovered (10.9% and 74.2% of the total dose in urine and in feces, respectively), with an excretion increment of <1% between Days 59 and 61. Mean ratios of total radioactivity indicated an equivalent distribution between blood cells and plasma. The concentration-time profiles of total radioactivity and acoziborole in plasma were similar. In plasma, acoziborole accounted for 95.1%, an oxidized form of acoziborole for 2.3% of the total radioactivity, while SCYX-3109 was not detected. Seven metabolites (oxidative deboronation, glucuronidation, and mono-oxidation) were detected in urine with individual abundances relative to the dose of 0.1–2.1%; unchanged acoziborole accounted for 0.6%. In feces, acoziborole, SCYX-3109, and two other oxidized or deboronated forms of acoziborole represented 33.6%, 12.3%, and 2.3% of the dose, respectively. Acoziborole showed good absorption, limited metabolism, minimal urinary elimination, and predominant but slow biliary-fecal elimination.

01 Jun 2025·COMBINATORIAL CHEMISTRY & HIGH THROUGHPUT SCREENING

To Explore Potential Inhibitors against Various Enzymatic Targets of Human African Trypanosomiasis

Review

Author: Bagchi, Soumya Ranjan ; Brahma, Sayandeep ; Samadder, Asmita ; Moitra, Susmita ; Nandi, Sisir

Synthetic drugs currently prescribed for the treatment of Human African Trypanosomiasis (HAT) are non-specific, toxic, demand extended therapeutic regimes and are of varying efficacy. Along with the challenging demographic and socio-economic hurdles, the everincreasing risk of drug resistance is another major problem to be addressed. Cysteine protease, Heat shock proteins (HSP-90), Trypanothione reductase (TR), Farnesyl diphosphate synthase, Glucose-6-phosphate dehydrogenase, UP-4-galactose epimerase, and Cytidine triphosphate synthetase are potential enzymatic targets for the development of novel inhibitors against HAT which are the main focus of this review. The potential enzymatic targets of Trypanosoma brucei, especially small molecules like cysteine proteases and heat shock proteins are identified as major candidates for the sustenance of the parasite, their proliferation, infection, and spread of the disease. The development of new compounds to combat the disease by thorough ligand modification has been explored in the current review. Extracting these compounds and studying their efficacy, toxicity, and target mechanism extensively, this review has proposed a list of different compounds, including some synthetic and natural compounds along with multi-target inhibitors such as acoziborole, fexinidazole, etc. Potential inhibitors against these enzymatic targets of the T. brucei are important candidates for designing novel therapeutics against HAT. Multi-target inhibitors have also been identified as crucial molecules because of their potential advantage against the development of drug resistance.

13 Mar 2025·CLINICAL MICROBIOLOGY REVIEWS

Transforming the chemotherapy of human African trypanosomiasis

Review

Author: Barrett, Michael P.

SUMMARY:

Prior to 2019, when the orally available drug fexinidazole began its clinical use, the treatment of human African trypanosomiasis (HAT) was complex and unsatisfactory for many reasons. Two sub-species of the Trypanosoma brucei parasite are responsible for HAT, namely the rhodesiense form found in East and Southern Africa and the gambiense form found in Central and West Africa. Diseases caused by both forms manifest in two stages: stage 1 before and stage 2 after central nervous system involvement. Prior to 2019, different drugs were required for each of the two parasite sub-species at each stage. Gambiense disease required pentamidine or nifurtimox-eflornithine combination therapy, while for rhodesiense disease, suramin or melarsoprol was given for stages 1 and 2, respectively. These drugs all suffered complications including protracted administration regimens involving multiple injections with drug-induced adverse effects common. Today, a single drug, fexinidazole, can be given orally in most cases for both diseases at either stage. Another compound, acoziborole, effective in both stages 1 and 2 gambiense disease with a single dosing is anticipated to become available within a few years. Moreover, the recent engagement of multilateral organizations in seeking other compounds that could be used in HAT therapy has also been successful, and a rich vein of new trypanocides has been discovered. Here, the clinical use, modes of action, and resistance risks for drugs used against HAT are discussed.

News (Medical) associated with Acoziborole

02 Mar 2026

·www.drugs.com

New Drug, Acoziborole, Could Boost Efforts to Wipe Out Sleeping Sickness

MONDAY, March 2, 2026 — A new treatment for sleeping sickness could make it much easier to treat and possibly eliminate the deadly disease. On Friday, a committee of the European Medicines Agency (EMA) recommended approval of acoziborole, a drug made by Sanofi . The decision is a key step to making the medicine available in Congo, where most cases occur, and in other African countries. Unlike current treatments, which can take up to 10 days and require difficult trips from remote areas to the hospital, acoziborole is taken as three pills swallowed in a single dose. Supporters say that could make a major difference. “This disease is on the brink of elimination," Dr. Junior Matangila of the Drugs for Neglected Diseases initiative told The Associated Press . He said the new drug could help “accelerate progress toward finishing the job.” Sleeping sickness, also called human African trypanosomiasis (HAT), is spread by tsetse flies in rural parts of sub-Saharan Africa. The flies carry a parasite that infects people through their bite, according to the U.S. Centers for Disease Control and Prevention (CDC). Early symptoms can look like the flu. But as the infection spreads, the parasite can reach the brain, according to the CDC . People may develop confusion, personality changes and a reverse sleep pattern: Awake at night and sleepy during the day. Without treatment, the disease can lead to coma and death. “It’s a disease of poverty,” Matangila said, who is based in Congo. Older medicines were painful and sometimes toxic. While treatments improved in the early 2000s, they still involve hospital stays and spinal taps to determine how far along the infection is. Acoziborole could change that. The new drug works for both early and advanced stages of the most common form of sleeping sickness. It can be used in patients ages 12 and older, and it removes the need for spinal taps. In a 2023 study of about 200 patients in Congo and Guinea, more than 95% were considered cured 18 months after treatment. Sanofi has pledged to donate the drug to the World Health Organization (WHO), meaning it would be free for patients. Sleeping sickness surged in the 1970s and 1990s during times of political unrest in Africa. But better treatments and efforts to control tsetse flies have helped bring cases down. Reported infections dropped below 10,000 in 2009 for the first time in 50 years. In 2024, fewer than 600 cases of the most common form were reported. The WHO aims to stop the spread of this form of sleeping sickness by 2030. “This isn’t solved yet,” Monica Mugnier , a sleeping sickness researcher at Johns Hopkins University, told The AP . She noted that scientists still do not fully understand where the parasite may be hiding. If eliminated, sleeping sickness could become the first infectious disease controlled without a vaccine, Sanofi officials said. Sources The Associated Press, Feb. 27, 2026

Drug Approval

27 Feb 2026

·www.fiercepharma.com

After CHMP nod, Moderna CEO applauds EU's 'rigorous scientific review'

After Europe's CHMP signed off on Moderna's combination COVID-19 and flu vaccine, CEO Stéphane Bancel praised the "EMA’s rigorous scientific review." While the FDA has flip-flopped on whether to review Moderna’s combination influenza and COVID-19 vaccine, it’s full speed ahead for the mRNA shot in Europe.The Committee for Medicinal Products for Human Use (CHMP) has recommended granting marketing authorization to mCombriax for people aged 50 and older. The committee's thumbs up is the first for a combo vaccine that protects against the two respiratory infections. Earlier this month, the FDA sent a refusal-to-file (RTF) letter to Moderna, explaining that the comparator arm of the successful trial backing the submission did not “reflect the best-available standard of care.” Eight days later, after meeting with Moderna, the U.S. regulator accepted the company’s application and established an Aug. 5 action date for its decision on whether to approve the shot. The hesitance of the FDA and the quick sign off from the European Medicines Agency (EMA) highlights the differing regulatory environments vaccine makers are facing in their two largest markets. The regulators were largely aligned until the installation last year of vaccine skeptic Robert F. Kennedy Jr. as the chief of Health and Human Services (HHS).“We appreciate the EMA’s rigorous scientific review,” Moderna CEO Stéphane Bancel said in a release on Friday.It’s a much-needed win for Moderna, which has slumped since its overwhelming success with mRNA vaccine Spikevax, which generated $37 billion in revenue for the company in 2021 and 2022. “The CHMP’s positive opinion represents an important milestone for respiratory virus vaccination and for Moderna, with the introduction of the world’s first flu plus COVID combination vaccine,” Bancel added. “If approved, this would be Moderna’s fourth marketed product in Europe.” In addition to the growing anti-vaccine sentiment in the U.S.—much of it fueled by misinformation—there is particular distrust from regulators in the U.S. about mRNA products, despite evidence of their safety.In making its decision, the CHMP cited World Health Organization data, which showed there have been more than 281 million cases of COVID-19 reported in Europe up until Feb. 1 of this year. Additionally, there are up to 50 million cases of seasonal flu each year in Europe. Sleeping sickness nod The CHMP has also recommended Acoziborole Winthrop as a treatment for gambiense sleeping sickness, a parasitic disease which is transmitted by the bite of an infected tsetse fly and has killed millions in central and west Africa over the past century. The medicine was co-developed by Sanofi and the Drugs for Neglected Diseases initiative (DNDi).The single-dose oral treatment would provide a significant advance over current therapies, which require either a 10-day course of oral medicine or a combination of injections and oral therapy for advanced cases. The recommendation applies to early and advanced-stage gambiense in those ages 12 and older.“In just 20 years, we have gone from complicated treatments including arsenic derivatives with serious side effects, to today, when a single-dose, one-day therapy could safely cure patients,” Luis Pizarro, M.D., the executive director of DNDi, said in a release. “This progress is [a] testament to the transformative power of collaborative science.” Two CHMP recommendations for hives condition Meanwhile, Novartis has secured a positive opinion from the CHMP on remibrutinib as an oral treatment for adults with chronic spontaneous urticaria (CSU) who have an inadequate response to antihistamines. In September of last year, the FDA approved the Bruton’s tyrosine kinase inhibitor (BTKi) treatment for the same indication, also known as chronic hives. The medicine’s commercial moniker in the U.S. is Rhapsido.“The introduction of remibrutinib represents a major advancement … with improvements in symptoms observed as early as Week 1,” Martin Metz, M.D., of the Institute of Allergology, Charité Universitätsmedizin in Berlin, said in a release.Sanofi also gained a CHMP positive opinion for its Regeneron-partnered blockbuster, Dupixent, as a treatment for children ages two through 11 years with CSU. This is the first biologic treatment for those under age 12 with the disorder. The FDA, which approved Dupixent for CSU in April of last year, has an April decision date on whether to expand its use to those ages of two through 11.Earlier this week, the FDA signed off on Dupixent in its ninth indication, allergic fungal rhinosinusitis (AFRS). Other CHMP recommendations Ipsen has scored a CHMP positive recommendation for its Day One Biopharmaceuticals-partnered Ojemda (tovorafenib) for patients six months of age and older with relapsed or refractory pediatric low-grade glioma (pLGG), a type of cancerous brain tumor.The FDA endorsed Ojemda, a once-weekly oral therapy, in 2024. It competes with Novartis’ Tafinlar-Mekinist combo, which has a narrower label. The treatments provide an option to surgery and chemotherapy.Also scoring a CHMP nod is Mitsubishi Tanabe’s Onerji, a subcutaneous infusion of liquid levodopa and carbidopa, for adults with Parkinson’s disease. In 2015, AbbVie gained FDA approval for the combination treatment, known commercially as Duopa. The Japanese company gained Onerji in a $1.1-billion acquisition of NeuroDerm in 2017. Crinetics has won a CHMP endorsement for Palsonify for the treatment of acromegaly, a rare hormonal disorder that usually occurs in middle-aged adults and is caused by the pituitary gland producing excess growth hormone. The FDA signed off on the selectively targeted somatostatin receptor type 2 agonist in September of last year.Also making the grade with the CHMP is X4 Pharmaceuticals’ Xolremdi for WHIM syndrome, an ultra-rare hereditary condition in which the immune system does not work properly, making patients more susceptible to bacterial and viral infections. The FDA approved the first-of-its-kind treatment in 2024. 2 CHMP rejections Vanda Pharmaceuticals has been on a roll, gaining FDA approvals for two new drugs in the last two months. But the Washington, D.C.-based drugmaker has failed to make the grade with its atypical antipsychotic drug iloperidone. The CHMP has questioned its efficacy and safety, citing that iloperidone can cause a change in the heart’s electrical activity, which can lead to a “life-threatening heart rhythm abnormality.” The risk of using iloperidone outweighs the benefits of treatment, the CHMP said.Vanda was seeking approval for iloperidone as a treatment for schizophrenia and bipolar I disorder. It has been on the market in the U.S. as Fanapt since 2009. The CHMP also swatted back Acadia Pharmaceuticals’ Rett syndrome drug Daybu, questioning its efficacy. The FDA approved the treatment in 2023.

Drug ApprovalAcquisitionVaccinemRNA

27 Feb 2026

·www.biospace.com

Press Release: Acoziborole Winthrop, developed by DNDi and Sanofi, receives CHMP positive opinion as three-tablet, single-dose treatment for most common form of sleeping sickness

Acoziborole Winthrop, developed by DNDi and Sanofi, receives CHMP positive opinion as three-tablet, single-dose treatment for most common form of sleeping sickness Recommendation based on phase 2/3 study demonstrating up to 96 percent success rates at 18 months across both early and advanced stages of T.b. gambiense , the most common form of sleeping sickness The therapy, given as a single dose of three tablets, could offer a simpler alternative to longer, more complex regimens and help support the World Health Organization’s (WHO) goal of eliminating the disease by 2030 Sanofi will donate the medicine to WHO through its philanthropic arm Foundation S Kinshasa / Paris / Geneva / Amsterdam – February 27, 2026 . The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has granted a positive opinion to Acoziborole Winthrop (acoziborole) as a single-dose oral treatment for both early and advanced-stage gambiense sleeping sickness in adults as well as in adolescents 12 years and older weighing at least 40 kilograms. The CHMP positive opinion is a critical step. It is granted through accelerated assessment under a specific procedure intended for countries outside of the European Union and used for high-priority medicines for diseases with unmet needs. This will facilitate approval of the medicine in the Democratic Republic of Congo (DRC) and pave the way for an update of WHO’s sleeping sickness treatment guidelines, a move that would eventually expand access to other countries in Central and West Africa, where the disease is endemic. Once approved in endemic countries, the medicine, co-developed by the Drugs for Neglected Diseases initiative (DNDi) and Sanofi, could provide a significant advance over current therapies. Existing treatments require either a 10-day course of oral medicine or a combination of injections and oral therapy for advanced cases. Transmitted by the bite of an infected tsetse fly, human African trypanosomiasis, commonly known as sleeping sickness, is almost always fatal without treatment. In the early stage of the disease, people experience headaches or fever. In the late stage, the parasite crosses the blood-brain barrier and invades the central nervous system, causing behavioral, cognitive and neurological symptoms, including seizures, sleep disturbances, aggression, confusion, lethargy, convulsions, and, ultimately, death. “ In just 20 years, we have gone from complicated treatments including arsenic derivatives with serious side effects, to today, when a single-dose, one-day therapy could safely cure patients,” said Luis Pizarro, MD, Executive Director at DNDi . “ This progress is testament to the transformative power of collaborative science and will bring us closer to finally eliminating sleeping sickness, a disease that has killed millions on the African continent in the past century.” DNDi conducted a pivotal phase 2/3 study in the DRC and Guinea in partnership with National Sleeping Sickness Control Programs, while Sanofi carried out the regulatory approval process. The CHMP positive opinion is based on clinical and non-clinical data provided by the partners, with efficacy and safety supported by the phase 2/3 study, published in The Lancet Infectious Diseases medical journal, which demonstrated success rates at 18 months of up to 96 percent across both stages of the disease with a good safety profile. “ The development of acoziborole and today’s positive scientific opinion is a victory for Africa-led science, made possible thanks to African doctors and researchers who conducted cutting-edge pharmaceutical research in some of the most remote and difficult-to-reach areas on the continent,” said Erick Miaka, MD, Director of the DRC’s national sleeping sickness control program. In 1998, nearly 40,000 cases of gambiense sleeping sickness were reported, with an estimated 300,000 undiagnosed. At the time, the only available treatment for those with the late stage of the disease was an injectable arsenic derivative with serious side effects. More than two decades of investment in new therapeutic tools resulted in increasingly improved treatments, including nifurtimox-eflornithine combination therapy in 2009 and the first oral treatment fexinidazole in 2018. In 2024, fewer than 600 cases of the disease were reported. “ For decades, Sanofi has maintained an unwavering commitment to the fight against sleeping sickness, standing alongside DNDi, the World Health Organization, and other partners in one of the most enduring and successful public-private health collaborations ,” said Audrey Duval, Executive Vice President, Corporate Affairs at Sanofi. “ Together, we have helped drive cases to historic lows—achieving a remarkable 98% reduction since 2001—by putting patients first and investing in innovation where it is needed most. Acoziborole builds on this legacy and represents a decisive step forward in eliminating gambiense sleeping sickness by 2030.” Sanofi will donate Acoziborole Winthrop to the WHO through its philanthropic organization, Foundation S – The Sanofi Collective. The medicine will be available free of charge to patients. Another study underway in the DRC and Guinea is investigating Acoziborole Winthrop for the treatment of children ages one to 14. About the DNDi programme for the development of acoziborole The DNDi programme for the development of acoziborole was supported by grants from the Federal Ministry of Research, Technology and Space (BMFTR) through KfW, Germany; the BBVA Foundation (through the ‘Frontiers of Knowledge Award in Development Cooperation’); Dutch Ministry of Foreign Affairs (DGIS), the Netherlands; European and Developing Countries Clinical Trials Partnership Association (EDCTP2 programme) supported by the European Union; Global Health EDCTP3 and its members; Gates Foundation; Médecins Sans Frontières International; Norwegian Agency for Development Cooperation (Norad), Norwegian Ministry of Foreign Affairs, as part of Norway’s in-kind contribution to EDCTP2; Swiss Agency for Development and Cooperation (SDC); Swiss State Secretariat for Education, Research and Innovation (SERI); Stavros Niarchos Foundation; Spanish Agency for International Development Cooperation (AECID); UK International Development; and other private foundations and individuals. About DNDi’s sleeping sickness programme Acoziborole Winthrop is the first oral single-dose new chemical entity to be issued from DNDi’s lead optimization programme for sleeping sickness. It started with an initial hit identified in the chemical library of Anacor Pharmaceuticals, which was acquired by Pfizer in 2016. The initial structure was then optimized with Scynexis and Pace University and was then selected as a candidate for development and Phase I safety studies conducted successfully in France, the UK, and Malaysia. Acoziborole Winthrop is the latest innovation brought by more than two decades of innovation efforts by DNDi, Sanofi, and partners. In 2009, they developed a combination of existing drugs known as NECT, which was extremely effective and with a good safety profile. A donation programme was set up by Sanofi and Bayer to provide NECT free of charge to endemic countries through the World Health Organization (WHO), radically improving treatment options for patients. DNDi, Sanofi, and partners developed fexinidazole, which in 2018 became the first all-oral treatment available for gambiense sleeping sickness. This ten-day treatment is now available in all sleeping sickness-endemic countries. Acoziborole Winthrop can be administered as a single oral dose, potentially without the need of hospitalization or supervision of the treatment at home. This means it could become a major tool to facilitate efforts to finally eliminate sleeping sickness. About DNDi The Drugs for Neglected Diseases initiative (DNDi) is a not-for-profit medical research organization that discovers, develops, and delivers safe, effective, and affordable treatments for neglected populations. DNDi is developing medicines for sleeping sickness, leishmaniasis, Chagas disease, river blindness, mycetoma, dengue, paediatric HIV, cryptococcal meningitis, and hepatitis C. Its research priorities include children’s health; gender equity and gender-responsive R&D; and diseases impacted by climate change. Since its creation in 2003, DNDi has collaborated with public and private partners worldwide to deliver new treatments for six deadly diseases, saving millions of lives. Acoziborole is the 14 th treatment delivered by DNDi. dndi.org About Sanofi Sanofi is an R&D driven, AI-powered biopharma company committed to improving people’s lives and delivering compelling growth. We apply our deep understanding of the immune system to invent medicines and vaccines that treat and protect millions of people around the world, with an innovative pipeline that could benefit millions more. Our team is guided by one purpose: we chase the miracles of science to improve people’s lives; this inspires us to drive progress and deliver positive impact for our people and the communities we serve, by addressing the most urgent healthcare, environmental, and societal challenges of our time. Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY Sanofi Media Relations Sandrine Guendoul | +33 6 25 09 14 25 | sandrine.guendoul@sanofi.com Evan Berland | +1 215 432 0234 | evan.berland@sanofi.com Timothy Gilbert | +1 516 521 2929 | timothy.gilbert@sanofi.com Léo Le Bourhis | +33 6 75 06 43 81 | leo.lebourhis@sanofi.com Victor Rouault | +33 6 70 93 71 40 | victor.rouault@sanofi.com Léa Ubaldi | +33 6 30 19 66 46 | lea.ubaldi@sanofi.com Ekaterina Pesheva | +1 410 926 6780 | ekaterina.pesheva@sanofi.com Sanofi Investor Relations Thomas Kudsk Larsen | + 44 7545 513 693 | thomas.larsen@sanofi.com Alizé Kaisserian | + 33 6 47 04 12 11 | alize.kaisserian@sanofi.com Keita Browne | + 1 781 249 1766 | keita.browne@sanofi.com Nathalie Pham | + 33 7 85 93 30 17 | nathalie.pham@sanofi.com Nina Goworek | +1 908 569 7086 nina.goworek@sanofi.com Thibaud Châtelet | + 33 6 80 80 89 90 | thibaud.chatelet@sanofi.com Yun Li | +33 6 84 00 90 72 | yun.li3@sanofi.com DNDi Media Relations Frédéric Ojardias (Geneva) fojardias@dndi.org +41 79 431 62 16 Linet Atieno Otieno (Nairobi) latieno@dndi.org +254 733 624 206 Ilan Moss (New York) imoss@dndi.org +1 646 266 5216 Sanofi forward-looking statements This press release contains forward-looking statements within the meaning of applicable securities laws, including the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These statements include projections and estimates and their underlying assumptions, statements regarding plans, objectives, intentions, and expectations with respect to future financial results, events, operations, services, product development and potential, and statements regarding future events and economic performance. Words such as “expect,” “anticipate,” “believe,” “intend,” “estimate,” “plan,” “can,” “contemplate,” “could,” “is designed to,” “may,” “might,” “potential,” “objective,” "attempt," “target,” “project,” "strategy," "strive," "desire," “predict,” “forecast,” “ambition,” “guideline,” "seek," “should,” “will,” "goal," or the negative of these, and similar expressions are intended to identify forward-looking statements. Although Sanofi’s management believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Sanofi, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include among other things, the uncertainties inherent in research and development, future clinical data and analysis, including post marketing, decisions by regulatory authorities, such as the U.S Food and Drug Administration or the European Medicines Agency, regarding whether and when to approve any drug, device or biological application that may be filed for any such product candidates as well as their decisions regarding labelling and other matters that could affect the availability or commercial potential of such product candidates; the fact that product candidates if approved may not be commercially successful; unexpected regulatory actions or delays, or government regulation generally; authorities’ decisions regarding whether and when to approve a product candidate; political pressure in the United States to mandate lower drug prices including “most favored nation” pricing for State Medicaid programs; the future approval and commercial success of therapeutic alternatives; Sanofi’s ability to benefit from external growth opportunities, to complete related transactions and/or obtain regulatory clearances, including future clinical data and analysis of existing clinical data relating to the product, including post marketing, unexpected safety, quality or manufacturing issues, competition in general; risks associated with intellectual property and any related pending or future litigation and the ultimate outcome of such litigation; trends in exchange rates and prevailing interest rates, volatile economic and market conditions, cost containment initiatives and subsequent changes thereto, and the impact that global crises may have on us, our customers, suppliers, vendors, and other business partners, and the financial condition of any one of them, as well as on our employees and on the global economy as a whole. The risks and uncertainties also include the uncertainties discussed or identified in the public filings with the SEC and the French Markest Authority (AMF) made by Sanofi, including those listed under “Risk Factors” and “Cautionary Statement Regarding Forward-Looking Statements” in Sanofi’s annual report on Form 20-F for the year ended December 31, 2025 or contained in our periodic reports on Form 6-K. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any forward-looking information or statements. In light of these risks, uncertainties and assumptions, you should not place undue reliance on any forward-looking statements contained herein. Attachment Press_Release

AcquisitionPhase 1Clinical ResultDrug Approval

100 Deals associated with Acoziborole

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R&D Status

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Indication	Highest Phase	Country/Location	Organization	Date
Infection by Trypanosoma brucei brucei	NDA/BLA	European Union	DNDi Japan	27 Feb 2026
Infection by Trypanosoma brucei brucei	NDA/BLA	European Union	Sanofi	27 Feb 2026
Heparin-induced thrombocytopenia	Phase 3	Guinea	Drugs for Neglected Diseases initiative	30 Dec 2021
Heparin-induced thrombocytopenia	Phase 3	Republic of the Congo	Drugs for Neglected Diseases initiative	30 Dec 2021
Trypanosomiasis, African	Phase 3	Democratic Republic of the Congo	Drugs for Neglected Diseases initiative	11 Oct 2016
Trypanosomiasis, African	Phase 3	Guinea	Drugs for Neglected Diseases initiative	11 Oct 2016

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03087955 (OXA002, CTgov)	Phase 2/3	Trypanosomiasis, African	208	Acoziborole (Late-stage HAT)	xtyoiucpuu = nheyzuntfj mmkkrmoqir (pkykjkzmtq, cdvbxddyev - zlqihdrjya) View more	-	17 Sep 2025
NCT03087955 (OXA002, CTgov)	Phase 2/3	Trypanosomiasis, African	208	Acoziborole (Early- and Intermediate-stage HAT)	dfpmxyogxq = fdgmhookzu kxkvxtvwht (txdosvgnwo, yiwfuynpht - lguoshevey) View more	-	17 Sep 2025
NCT05256017 (OXA004, CTgov)	Phase 2/3	Infection by Trypanosoma brucei brucei \| Trypanosomiasis, African \| Heparin-induced thrombocytopenia	1,208	Acoziborole (Acoziborole)	fxtonuenvc = ogsnkikqnt ejrfwmlrqv (teixzubber, rbafveducv - bktdrzmfzf) View more	-	20 Mar 2025
NCT05256017 (OXA004, CTgov)	Phase 2/3		1,208	Placebo (Placebo)	fxtonuenvc = aqgtuyglhz ejrfwmlrqv (teixzubber, mpgjempgqj - qpamcavlvh) View more	-	20 Mar 2025
NCT01533961 (Pubmed \| Clin Pharmacokinet)	Phase 1	Trypanosomiasis, African	128	Acoziborole 960mg	nelovkvfxe(ypcinbcnax) = appeared rapidly in plasma (at 1 h), reached tmax between 24 and 72 h, and remained stable for up to 96 h, after which a slow decrease was quantifiable until 14 weeks after dosing qnhyjthdag (gcrdpxytdi ) View more	Positive	01 Mar 2023
NCT03087955 (Pubmed) Manual	Phase 2/3	Trypanosomiasis, African	208	Acoziborole	qieqzubxmn(imcbhruant) = ygirrhcrbt gxnvfwhjth (tmsazdindj, 91.2 - 97.7) View more	Positive	29 Nov 2022

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