Eflornithine Hydrochloride

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. 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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. 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本期看点： 1. GT Biopharma公司宣布，其新型B7-H3靶向三特异性自然杀伤（NK）细胞衔接器GTB-5550的IND申请已获得美国FDA批准，将在7种不同的转移性癌症队列中开展研究。 2. Prelude Therapeutics公司宣布，其新型JAK2V617F突变选择性抑制剂PRT12396的IND申请已获美国FDA批准，1期临床试验将在高危真红细胞增多症（PV）以及中高危骨髓纤维化（MF）患者中开展。 GTB-5550：IND申请获得FDA许可 GT Biopharma公司宣布，其新型候选药物GTB-5550的IND申请已获得美国FDA批准。GTB-5550是一种靶向B7-H3的三特异性NK细胞衔接器，用于治疗表达B7-H3抗原的实体肿瘤。该分子由三种组分组成，通过柔性接头连接：1）靶向NK细胞表面CD16激活受体的纳米体臂；2）野生型IL-15（WT IL-15）连接臂，用于促进NK细胞的增殖、活化和存活；3）靶向肿瘤细胞上B7-H3抗原的纳米体臂，从而将NK细胞精准引导至肿瘤部位。 计划开展的1期临床试验共分为两个部分：1a期为剂量递增阶段，将评估最多6个剂量水平以确定最大耐受剂量（MTD）；1b期为剂量扩展阶段，将在7种不同的转移性癌症队列中（包括去势抵抗性前列腺癌、卵巢癌、乳腺癌、头颈癌、非小细胞肺癌、胰腺癌和膀胱癌）验证1a期研究确定的MTD，并进一步评估药物的耐受性。 PRT12396：IND申请获得FDA许可 Prelude Therapeutics公司宣布，其新型JAK2V617F突变选择性抑制剂PRT12396的IND申请已获美国FDA批准，将启动一项1期临床研究。该研究为开放标签、多中心试验，旨在评估PRT12396在高危真红细胞增多症以及中高危骨髓纤维化患者中的安全性、初步疗效和药代动力学特征。公司预计将于2026年第二季度完成首例患者给药。 PRT12396是Prelude公司设计并验证的新型变构抑制剂，能够结合到V617F突变所在的JAK2 JH2“深口袋”。该突变是骨髓增殖性肿瘤（MPNs）的主要驱动突变，在约95%的PV、60%的原发性血小板增多症（ET）和55%的MF患者中存在。Prelude公司开发的这类变构抑制剂在MPN临床前模型中展现了突变特异性抑制作用，有望降低突变等位基因负荷、延缓甚至逆转疾病进展，从而改善MPN患者的治疗结果。 AMXT 1501：启动1b/2期联合治疗试验 Aminex Therapeutics公司宣布启动一项AMXT 1501联合二氟甲基鸟氨酸（DFMO）的1b/2期临床试验。这项多中心、开放标签研究将评估口服AMXT 1501与口服DFMO联合标准治疗在两类人群中的安全性、耐受性和初步疗效，包括既往治疗失败、绝经前/后、雌激素受体（ER）阳性、人表皮生长因子受体2（HER2）阴性乳腺癌和转移性黑色素瘤患者。 AMXT 1501是一种新型多胺转运抑制剂，旨在阻断肿瘤细胞摄取外源性多胺——这些分子对肿瘤生长和存活至关重要。DFMO是一种已知的多胺生物合成抑制剂。两者联合旨在全面抑制多胺代谢，从而抑制肿瘤生长。 参考资料： [1] Onchilles Pharma Announces IND Clearance for N17350, Advancing the First Next-Generation Cytotoxic Therapeutic Leveraging the ELANE Pathway into the Clinic. Retrieved February 6, 2026, from https://www.onchillespharma.com/post/onchilles-pharma-announces-ind-clearance-for-n17350 [2] GT Biopharma Announces FDA Clearance of Investigational New Drug (IND) Application for GTB-5550 TriKE®, a B7-H3-Targeted Natural Killer (NK) Cell Engager for Solid Tumors Expressing B7-H3. Retrieved February 6, 2026, from https://www.globenewswire.com/news-release/2026/02/03/3231077/0/en/GT-Biopharma-Announces-FDA-Clearance-of-Investigational-New-Drug-IND-Application-for-GTB-5550-TriKE-a-B7-H3-Targeted-Natural-Killer-NK-Cell-Engager-for-Solid-Tumors-Expressing-B7-H.html [3] Prelude Therapeutics Receives FDA Clearance of Investigational New Drug Application (IND) for PRT12396, a Mutant-selective JAK2V617F Inhibitor. Retrieved February 6, 2026, from https://www.globenewswire.com/news-release/2026/02/03/3231175/0/en/Prelude-Therapeutics-Receives-FDA-Clearance-of-Investigational-New-Drug-Application-IND-for-PRT12396-a-Mutant-selective-JAK2V617F-Inhibitor.html [4] Affinia Therapeutics Announces FDA Acceptance of IND Application to Advance AFTX-201 to a Phase 1/2 Trial for the Treatment of BAG3-Associated Dilated Cardiomyopathy (DCM). Retrieved February 6, 2026, from https://affiniatx.com/affinia-therapeutics-announces-fda-acceptance-of-ind-application-to-advance-aftx-201-to-a-phase-1-2-trial-for-the-treatment-of-bag3-associated-dilated-cardiomyopathy-dcm/ [5] PharmaResearch Receives U.S. FDA Clearance to Initiate Phase 1 Clinical Trial of Nano Anticancer Drug, PRD-101. Retrieved February 6, 2026, from https://www.prnewswire.com/news-releases/pharmaresearch-receives-us-fda-clearance-to-initiate-phase-1-clinical-trial-of-nano-anticancer-drug-prd-101-302681109.html [6] Spur Therapeutics Presents New Phase 1/2 Data on Its Gene Therapy Candidate in Gaucher Disease at 22nd Annual WORLDSymposium™. Retrieved February 6, 2026, from https://www.globenewswire.com/news-release/2026/02/04/3231939/0/en/Spur-Therapeutics-Presents-New-Phase-1-2-Data-on-Its-Gene-Therapy-Candidate-in-Gaucher-Disease-at-22nd-Annual-WORLDSymposium.html [7] Aminex Therapeutics Announces Multiple Sites Activated for Phase 1b/2 Clinical Trial of Novel Investigational Cancer Treatment AMXT 1501 and DFMO in Patients with Breast Cancer or Melanoma. Retrieved February 6, 2026, from https://www.prnewswire.com/news-releases/aminex-therapeutics-announces-multiple-sites-activated-for-phase-1b2-clinical-trial-of-novel-investigational-cancer-treatment-amxt-1501-and-dfmo-in-patients-with-breast-cancer-or-melanoma-302678969.html [8] Neomorph Announces First Patient Dosed in Phase 1/2 Trial Evaluating NEO-811 For the Treatment of Locally Advanced or Metastatic Non-Resectable Clear Cell Renal Cell Carcinoma. Retrieved February 6, 2026, from https://www.globenewswire.com/news-release/2026/02/03/3231475/0/en/Neomorph-Announces-First-Patient-Dosed-in-Phase-1-2-Trial-Evaluating-NEO-811-For-the-Treatment-of-Locally-Advanced-or-Metastatic-Non-Resectable-Clear-Cell-Renal-Cell-Carcinoma.html [9] Ultragenyx Announces Positive Longer-Term Data Demonstrating Treatment with UX111 Gene Therapy Results in Sustained, Significant Reductions in CSF-HS and Continued Meaningful Improvements in Clinical Function Across Multiple Developmental Domains in Children with Sanfilippo Syndrome (MPS IIIA). Retrieved February 6, 2026, from https://ir.ultragenyx.com/news-releases/news-release-details/ultragenyx-announces-positive-longer-term-data-demonstrating [10] uniQure Announces Updated Preliminary AMT-191 Phase I/IIa Data Showing Sustained Increases in α-Gal A Enzyme Activity in Patients with Fabry Disease. Retrieved February 6, 2026, from https://www.globenewswire.com/news-release/2026/02/06/3233740/0/en/uniQure-Announces-Updated-Preliminary-AMT-191-Phase-I-IIa-Data-Showing-Sustained-Increases-in-%CE%B1-Gal-A-Enzyme-Activity-in-Patients-with-Fabry-Disease.html 免责声明：本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。 版权说明：欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。 分享，点赞，在看，聚焦全球生物医药健康创新