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.

开始日期2023-02-09

申办/合作机构

Drugs for Neglected Diseases initiative

NCT05433350 / Recruiting临床2/3期IIT

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.

开始日期2022-07-15

申办/合作机构

Drugs for Neglected Diseases initiative

NCT05256017 / Completed临床2/3期IIT

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

Acoziborole as an oral, single-dose treatment was studied in an open-label pivotal Phase II/III trial (DNDi-OXA-02-HAT) in DRC and Guinea. The safety and efficacy results on g-HAT confirmed cases (all disease stages) from the pivotal study provided data, that allows to envision the treatment of confirmed g-HAT cases but there is still a gap in the management of g-HAT seropositive non-parasitologically confirmed individuals. Indeed, the standard g-HAT case definition implies the demonstration of the parasite in any body fluid via microscopy. 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, remaining as potential reservoirs of the parasite and a source of new infections hindering the efforts to eliminate the disease.
The present clinical trial intends to expand the safety data of acoziborole and complement the safety profile obtained from the pivotal trial by assessing the safety and tolerability of a single dose of acoziborole compared with placebo in seropositive individuals who are not confirmed parasitologically.
In addition to this study, an exploratory sub-study named 'TrypSkin' is planned to assess the presence of extravascular dermal T.b. gambiense in the population enrolled.

开始日期2021-12-30

申办/合作机构

Drugs for Neglected Diseases initiative

100 项与 Acoziborole 相关的临床结果

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100 项与 Acoziborole 相关的转化医学

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100 项与 Acoziborole 相关的专利（医药）

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项与 Acoziborole 相关的文献（医药）

2024-09-01·Trends in Parasitology

Pass the boron: benzoxaboroles as antiparasite drugs

Review

作者: Horn, David ; Zoltner, Martin ; Field, Mark C

The development of new drug modalities has been facilitated recently by the introduction of boron as a component of organic compounds, and specifically within a benzoxaborale scaffold. This has enabled exploration of new chemical space and the development of effective compounds targeting a broad range of morbidities, including infections by protozoa, fungi, worms, and bacteria. Most notable is the recent demonstration of a single oral dose cure using acoziborole against African trypanosomiasis. Common and species-/structure-specific interactions between benzoxaboroles and parasite species have emerged and provide vital insights into the mechanisms of cidality, as well as potential challenges in terms of resistance and/or side effects. Here, we discuss the literature specific to benzoxaborole studies in parasitic protists and consider unanswered questions concerning this important new drug class.

2023-11-01·European Journal of Medicinal Chemistry

Elemental exchange: Bioisosteric replacement of phosphorus by boron in drug design

Review

作者: Degani, Mariam S ; Mehta, Namrashee V ; Abhyankar, Arundhati

Continuous efforts are being directed toward the employment of boron in drug design due to its advantages and unique characteristics including a plethora of target engagement modes, lower metabolism, and synthetic accessibility, among others. Phosphates are components of multiple drug molecules as well as clinical candidates, since they play a vital role in various biochemical functions, being components of nucleotides, energy currency- ATP as well as several enzyme cofactors. This review discusses the unique chemistry of boron functionalities as phosphate bioisosteres - "the boron-phosphorus elemental exchange strategy" as well as the superiority of boron groups over other commonly employed phosphate bioisosteres. Boron phosphate-mimetics have been utilized for the development of enzyme inhibitors as well as novel borononucleotides. Both the boron functionalities described in this review-boronic acids and benzoxaboroles-contain a boron connected to two oxygens and one carbon atom. The boron atom of these functional groups coordinates with a water molecule in the enzyme site forming a tetrahedral molecule which mimics the phosphate structure. Although boron phosphate-mimetic molecules - FDA-approved Crisaborole and phase II/III clinical candidate Acoziborole are products of the boron-phosphorus bioisosteric elemental exchange strategy, this technique is still in its infancy. The review aims to promote the use of this strategy in future medicinal chemistry projects.

2023-04-01·The Lancet Infectious Diseases

Efficacy and safety of acoziborole in patients with human African trypanosomiasis caused by Trypanosoma brucei gambiense: a multicentre, open-label, single-arm, phase 2/3 trial

Article

作者: Embana Mankiara, Hugues ; Betu Kumeso, Victor Kande ; Delhomme, Sophie ; Rembry, Sandra ; Mahenzi Mbembo, Hélène ; Catusse, Julie ; Ngolo Tete, Digas ; Prêtre, Adeline ; Mulenge Nasandhel, Ernest ; Akwaso Massa, Félix ; Mutanda Kalonji, Sylvain ; Tarral, Antoine ; Makaya Mayawula, Joseph ; Layba Camara, Mariame ; Scherrer, Bruno ; Kavunga Lukula, Papy ; Ilunga Wa Kyhi, Médard ; Kasongo Bonama, Augustin ; Schneitter, Stefan ; Strub-Wourgaft, Nathalie ; Kobo Muanza, Vincent ; Mariero Philemon, Phyll ; Fifi Nzeza Bambuwu, Aimée ; Mwamba Miaka, Erick ; Mokilifi Nganyonyi, Ricardo ; Kaninda Badibabi, Lewis ; Camara, Mamadou ; Asuka Akongo Nguba, André ; Valverde Mordt, Olaf ; Nusbaumer, Morgane ; Kalonji, Wilfried Mutombo

BACKGROUNDHuman African trypanosomiasis caused by Trypanosoma brucei gambiense (gambiense HAT) in patients with late-stage disease requires hospital admission to receive nifurtimox-eflornithine combination therapy (NECT). Fexinidazole, the latest treatment that has been recommended by WHO, also requires systematic admission to hospital, which is problematic in areas with few health-care resources. We aim to assess the safety and efficacy of acoziborole in adult and adolescent patients with gambiense HAT.METHODSThis multicentre, prospective, open-label, single-arm, phase 2/3 study recruited patients aged 15 years or older with confirmed gambiense HAT infection from ten hospitals in the Democratic Republic of the Congo and Guinea. Inclusion criteria included a Karnofsky score greater than 50, ability to swallow tablets, a permanent address or traceability, ability to comply with follow-up visits and study requirements, and agreement to hospital admission during treatment. Oral acoziborole was administered as a single 960 mg dose (3 × 320 mg tablets) to fasted patients. Patients were observed in hospital until day 15 after treatment administration then for 18 months as outpatients with visits at 3, 6, 12, and 18 months. The primary efficacy endpoint was the success rate of acoziborole treatment at 18 months in patients with late-stage gambiense HAT (modified intention-to-treat [mITT] population), based on modified WHO criteria. A complementary post-hoc analysis comparing the 18-month success rates for acoziborole and NECT (using historical data) was performed. This study is registered at ClinicalTrials.gov, NCT03087955.FINDINGSBetween Oct 11, 2016, and March 25, 2019, 260 patients were screened, of whom 52 were ineligible and 208 were enrolled (167 with late-stage and 41 with early-stage or intermediate-stage gambiense HAT; primary efficacy analysis set). All 41 (100%) patients with early-stage or intermediate-stage and 160 (96%) of 167 with late-stage disease completed the last 18-month follow-up visit. The mean age of participants was 34·0 years (SD 12·4), including 117 (56%) men and 91 (44%) women. Treatment success rate at 18 months was 95·2% (95% CI 91·2-97·7) reached in 159 of 167 patients with late-stage gambiense HAT (mITT population) and 98·1% (95·1-99·5) reached in 159 of 162 patients (evaluable population). Overall, 155 (75%) of 208 patients had 600 treatment-emergent adverse events. A total of 38 drug-related treatment-emergent adverse events occurred in 29 (14%) patients; all were mild or moderate and most common were pyrexia and asthenia. Four deaths occurred during the study; none were considered treatment related. The post-hoc analysis showed similar results to the estimated historical success rate for NECT of 94%.INTERPRETATIONGiven the high efficacy and favourable safety profile, acoziborole holds promise in the efforts to reach the WHO goal of interrupting HAT transmission by 2030.FUNDINGBill & Melinda Gates Foundation, UK Aid, Federal Ministry of Education and Research, Swiss Agency for Development and Cooperation, Médecins Sans Frontières, Dutch Ministry of Foreign Affairs, Norwegian Agency for Development Cooperation, Norwegian Ministry of Foreign Affairs, the Stavros Niarchos Foundation, Spanish Agency for International Development Cooperation, and the Banco Bilbao Vizcaya Argentaria Foundation.TRANSLATIONFor the French translation of the abstract see Supplementary Materials section.

项与 Acoziborole 相关的新闻（医药）

2023-02-02

·Sanofi

Strong sales performance and double digit EPS growth marking the achievement of the 2022 profitability milestone

Strong sales performance and double digit EPS growth marking the achievement of the 2022 profitability milestone Paris, February 3, 2023 Q4 2022 sales growth of 2.6% at CER and business EPS (1) growth of 17.4% at CER Specialty Care grew 18.1% driven by Dupixent ® (€2,402 million, +42.1%) and new product launches Vaccines sales (-16.3%) reflecting influenza and PPH sales phasing (Q3 influenza sales: up 32.4%) as well as ramp up of non-consolidated Vaxelis ® sales General Medicines core assets up 8.0% while GBU sales were lower (-3.7%) mainly due to Lantus ® and spin-off of EUROAPI CHC sales increased 6.6% driven by double-digit growth of Digestive Wellness, Cough & Cold and Allergy categories Full-year 2022 delivered 7.0% sales growth and 17.1% business EPS growth at CER Sales grew to €42,997 million driven by Dupixent ® (€8,293 million, +43.8%), adding €3 billion of incremental sales, Vaccines up 6.3% in line with the mid-term growth objective as well as CHC strategy execution (+8.6%) Mid-term BOI margin target of 30% and cost savings objective of €2.5 billion achieved Business EPS (1) of €8.26 up 25.9% on a reported basis and 17.1% at CER IFRS EPS of €5.37 (up 8.0%) Board held on February 2, proposes annual dividend of €3.56, an increase of 6.9% Progress on Corporate Social Responsibility strategy in Q4 Positive phase 2/3 results of acoziborole with the potential to further transform the treatment of sleeping sickness Accelerating our ambition towards net zero emissions by 5 years, now targeting 2045 Key R&D milestones and regulatory achievements in Q4 Dupixent ® approved in Europe for prurigo nodularis and CHMP positive opinion for eosinophilic esophagitis Beyfortus ® ( nirsevimab) approved in Europe for the prevention of RSV disease in all infants VidPrevtyn ® Beta approved in Europe as a booster for the prevention of COVID-19 in adults Enjaymo ® approved in Europe in adult patients with cold agglutinin disease (CAD) Full-year 2023 business EPS guidance Sanofi expects 2023 business EPS (1) to grow low single digit (2) at CER, barring unforeseen major adverse events. Applying average January 2023 exchange rates, the currency impact on 2023 business EPS is estimated between -3.5% to -4.5% Sanofi Chief Executive Officer, Paul Hudson, commented: “We closed 2022, marking the successful execution of the first chapter of our 6-year ‘Play to Win’ strategy. Specialty Care delivered the highest sales among our businesses. Dupixent ® and Vaccines continue to be our leading growth drivers. We are particularly proud of the progress we made in R&D transformation with multiple approvals of transformative medicines and new product launches across Specialty Care. At the same time, we keep delivering strong proof points of our improved financial performance underpinned by the achievement of the 30% BOI margin. Moving to the next chapter of our strategy, we are looking forward to the planned launches of Altuviiio ® and Beyfortus ® as well as key pivotal readouts, including the COPD indication for Dupixent ® . With the view on the expected entrants of generic competition for Aubagio ® in the coming months, we remain confident in our outstanding commercial capabilities, including the ambition to reach sales of 10 billion euros for Dupixent ® in 2023, enabling us to guide to low single-digit EPS growth for the year.” Q4 2022 Change Change at CER 2022 Change Change at CER IFRS net sales reported €10,725m +7.3% +2.6% €42,997m +13.9% +7.0% IFRS net income reported €1,460m +29.1% _ €6,720m +8.0% — IFRS EPS reported €1.16 +28.9% _ €5.37 +8.0% — Free cash flow (3) €2,546m +0.2% _ €8,483m +4.8% — Business operating income €2,724m +20.7% +15.0% €13,040m +21.7% +13.3% Business net income (1) €2,141m +23.8% +17.6% €10,341m +25.9% +17.0% Business EPS (1) €1.71 +23.9% +17.4% €8.26 +25.9% +17.1% Changes in net sales are expressed at constant exchange rates (CER) unless otherwise indicated (definition in Appendix 9). (1) In order to facilitate an understanding of operational performance, Sanofi comments on the business net income statement. Business net income is a non-GAAP financial measure (definition in Appendix 9). The consolidated income statement for Q4 2022 is provided in Appendix 3 and a reconciliation of reported IFRS net income to business net income is set forth in Appendix 4; (2) 2022 business EPS was €8.26; (3) Free cash flow is a non-GAAP financial measure (definition in Appendix 9). Attachment Press Release

上市批准生物类似药

2022-12-04

·药研发

【药研发1205】先声脑梗死复方舌下片III期临床积极 | 勤浩抗耐药ERK1/2抑制剂获批临床...

「本文共：17条资讯，阅读时长约：3分钟」今日头条先声脑梗死复方舌下片III期临床积极。先声药业与宁丹新药合作开发的先必新舌下片治疗急性缺血性脑卒中（AIS）的III期临床达到疗效终点。与安慰剂相比，先必新舌下片显著改善AIS患者治疗后神经功能恢复及独立生活能力，且耐受性良好。先必新舌下片（Y-2舌下片）是一种含有依达拉奉和右莰醇两种活性成分的口服固体制剂。此前，先必新（依达拉奉右莰醇注射用浓溶液）已获NMPA批准上市，用于改善急性脑梗塞所致的神经症状。国内药讯1.翰森新一代EGFR-TKI在欧洲报产。翰森制药与EQRx公司合作开发的第三代EGFR-TKI甲磺酸阿美替尼片的上市申请获欧洲药品管理局受理，用于一线治疗具有EGFR敏感突变的局部晚期或转移性非小细胞肺癌（NSCLC）成人患者，及治疗存在EGFR T790M突变阳性的局部晚期或转移性NSCLC成人患者。在Ⅲ期AENEAS研究中，与吉非替尼相比，阿美替尼可显著延长患者无进展生存期（中位PFS：19.3个月vs9.9个月）。2.齐鲁PD-1/CTLA-4双抗Ⅱ期临床积极。齐鲁制药在ESMO2022亚洲大会上公布PD-1/CTLA-4双抗QL1706联合化疗+/-贝伐珠单抗治疗晚期非小细胞肺癌（NSCLC）的Ⅱ期临床积极结果。在EGFR野生型患者中，中位随访为9.17个月时，患者的客观缓解率（ORR）为58.6%，其中鳞状NSCLC患者ORR为70.6%，非鳞状NSCLC患者ORR为41.7%；患者的疾病控制率（DCR）为93.1%（27/29）。在EGFR突变型的晚期NSCLC患者中，中位随访为5.75个月时，ORR为64.5%（20/31），DCR为93.5%（29/31）。3.翰宇鼻喷型新冠药启动II期临床。翰宇药业1类创新药HY300鼻喷雾II期临床研究方案获得组长单位南方医科大学珠江医院学伦理委员会伦理审查批件，用于严重急性呼吸系统综合征冠状病毒2（SARS-CoV-2）感染的暴露前预防。HY3000是一种膜融合抑制剂多肽，作用于新冠病毒刺突蛋白的HR1区域，通过HR1区域结合，阻止病毒六螺旋束结构形成，阻断病毒侵染细胞的路径。4.迈威CD47/PD-L1双抗临床前研究见刊。迈威生物CD47/PD-L1双抗6MW3211的临床前研究结果在线发表在国际期刊Theranostics上。6MW3211在转基因小鼠模型上显示出显著抑制肿瘤生长，延长小鼠生存期。在PD-L表达肿瘤细胞中，6MW3211中CD47抗体臂的抗肿瘤效果显著提高。在恒河猴的毒理试验中，6MW3211在200mg/kg剂量下未观察到任何红细胞毒性。6MW3211目前正在多项Ⅱ期临床中评估用于治疗透明细胞肾细胞癌、淋巴瘤和肺癌的潜力。5.勤浩抗耐药ERK1/2抑制剂获批临床。勤浩医药双机制ERK1/2抑制剂新药GH55获FDA临床许可。GH55在抑制ERK1/2激酶活性的同时，能够抑制MEK对于ERK1/2的激活，防止由于负反馈调节造成的耐药。今年9月，GH55胶囊的国内IND申请已获得NMPA批准，拟开发用于MAPK信号通路突变的晚期实体瘤。目前全球范围内尚无ERK1/2抑制剂获批上市。‍‍‍‍国‍‍‍‍‍‍‍‍‍‍际‍‍‍‍药‍讯‍‍1.创新IDH1抑制剂治疗AML获批上市。Rigel公司与Forma公司开发的IDH1选择性抑制剂Rezlidhia（olutasidenib）获FDA批准上市，用于治疗IDH1突变、复发或难治性急性髓系白血病（AML）患者。Olutasidenib可通过抑制突变IDH1降低2-羟基戊二酸（2-HG）水平和恢复正常髓系细胞的分化。在Ⅱ期临床中，Rezlidhia在这类患者中达到35%缓解率，中位缓解持续时间为25.9个月。2.GSK开发PD-1抗体子宫内膜癌Ⅲ期临床成功。葛兰素史克（GSK）PD-1抗体Jemperli（dostarlimab）一线治疗原位晚期或复发性子宫内膜癌的Ⅲ期RUBY试验达到主要终点。无论是在总患者群体或是dMMR/MSI-H亚群中，使用Jemperli与标准化疗的组合疗法再以Jemperli治疗的患者，研究人员评估的PFS均显著优于安慰剂组。在MMRp/MSS群体中，亦可见到在PFS上的临床益处。预计该公司将在2023年上半年递交补充监管申请。3.罗氏皮下注射PD-L1抗体Ⅲ期临床成功。罗氏PD-L1抗体Tecentriq（atezolizumab）皮下制剂治疗局部晚期或转移性非小细胞肺癌（NSCLC）的Ⅰb/Ⅲ期试验（IMscin001）获积极结果。该试验此前已达到主要终点，与需要30-60分钟的静脉（IV）输注相比，只需3-8分钟的皮下注射Tecentriq在血液中的水平（药代动力学）显示出非劣效性。最新数据显示，接受Tecentriq皮下制剂与静脉输注患者的总缓解率和无进展生存期相当。两者在安全性方面也展现一致性。4.赛诺菲昏睡病口服新药Ⅱ/Ⅲ期临床积极。非营利性药物研发组织“被忽视疾病药物倡议”（DNDi）与赛诺菲开发的单次口服给药的新化学药物acoziborole，在刚果和几内亚针对昏睡病开展的Ⅱ/Ⅲ期临床结果积极。208例确诊患者服用一剂acoziborole后达到95%有效率，详细结果已发表在《柳叶刀-传染病上》。昏睡病是一种通过采采蝇叮咬传播的寄生虫病，也被称为非洲人类锥虫病（HAT）。5.Sigma-1受体激活剂治疗AD IIb/III期临床成功。Anavex Life Sciences公司开发的口服小分子sigma-1（σ-1）受体激活剂ANAVEX®2-73 (blarcamesine)，在治疗阿尔茨海默病(AD)引起的轻度认知障碍(MCI)的IIb/III研究ANAVEX®2-73-AD-004达到主要终点及关键次要终点。与安慰剂相比，ANAVEX®2-73治疗组患者AD评定量表（ADAS-Cog)评分平均下降4.03分，下降≥0.50分(p=0.015)的患者比例超过84%；该组日常生活活动量表(ADCS-ADL)评分增加≥3.5分 (p=0.0255)，改善功能的可能性提高167%。药物的总体耐受性良好。6.阿斯利康FIC淀粉样蛋白单抗II期研究积极。阿斯利康旗下Alexion公司将在ASH2022年会上公布潜在first-in-class淀粉样蛋白单抗CAEL-101针对轻链淀粉样变性（AL）患者的II期研究（NCT04304144）积极结果。最新数据显示，22例心脏可评估患者接受CAEL-101治疗1年后，有46%的患者达到临床缓解，18%的患者病情稳定。在9例肾脏可评估患者中，有89%患者的蛋白尿较基线减少≥30%。所有25例患者均经历不良反应，多为轻中度。60%患者经历3级及以上的不良反应。7.溶瘤病毒联合疗法获FDA快速通道认定。FDA授予Oncolytics Biotech公司溶瘤病毒Pelareorep与罗氏PD-L1阿替利珠单抗、吉西他滨和白蛋白紫杉醇的联合方案快速通道资格，用于一线治疗晚期/转移性胰腺导管腺癌（PDAC）。在I/II期GOBLET研究中，Pelareorep组合疗法达到69%的客观缓解率，包括1例完全缓解和8例部分缓解，是历史对照试验（吉西他滨联合白蛋白紫杉醇）中平均ORR的3倍。‍‍‍‍‍‍‍医‍‍‍‍药‍‍‍‍热‍‍‍‍点‍‍‍‍‍‍‍1.上海：公交和室外公共场所不再查验核酸证明。12月4日，上海市疫情防控工作领导小组办公室发布优化调整疫情防控的相关措施，具体为：乘坐轨道交通、地面公交、轮渡等市内公共交通工具，不再查验核酸检测阴性证明；全市公园、景区等室外公共场所，不再查验核酸检测阴性证明。以上优化调整措施自12月5日零时起实施。2.北京新冠肺炎线上咨询平台再次上线运行。根据北京地区新冠肺炎疫情形势，北京医学会再次启动北京市新冠肺炎线上医生咨询平台，方便市民足不出户即可获得疫情防治相关知识和医学方面的专业指导。北京医学会此次安排了呼吸病学、感染病学、老年医学、儿科学、围产医学、心理学、急诊医学、全科医学等8个专科分会的专家参与平台工作，每日从8时至22时，由专家在线回答群众提出的咨询问题。3.广州：新冠症状同感冒，毒力不如流感严重。12月2日晚8点，多位医学专家联合发布重磅判断，广州这波新冠疫情当中，奥密克戎变异株引起的症状大部分都很轻，症状同季节性感冒类似；超过90%都是无症状感染者，无需太多的治疗干预；轻症患者的治疗按照普通感冒的治疗手段处理，平均住院时间一般10天左右。专家呼吁市民无需恐慌。4.冠脉支架接续集采拟中结果出炉。11月29日，国家组织冠脉支架集采协议期满后接续采购在江苏常州开标，产生拟中选结果。与上一轮集采相比，本次接续采购参加的医疗机构增加40%，支架采购量增加30%。共有10家企业的14个产品获得拟中选资格，平均中选支架价格770元左右，加上伴随服务费，终端价格区间在730元至848元，预计2023年1月患者将用上接续采购的中选产品。‍‍‍‍‍‍评‍‍‍审‍‍动态‍‍‍‍‍‍ 1. CDE新药受理情况（11月23日) 2. FDA新药获批情况（北美12月01日）股市资讯上个交易日 A 股医药板块 -0.16%涨幅前三跌幅前三易瑞生物 +20.00% 特一药业 -8.43%步长制药 +10.01% 众生药业 -7.94%贵州三力 +10.01% 神奇B股 -3.65% 【乐心医疗】由报的电子血压计医疗器械注册证获得FDA受理。【翰宇药业】HY3000预防新冠多肽鼻喷药物获得II期临床试验组长单位伦理批件。【神州细胞】控股子公司新冠疫苗SCTV01C经国家有关部门论证被纳入紧急使用。【普利制药】注射用更昔洛韦获得奥地利联邦卫生安全办公室上市许可。【桂林三金】拟与上海赛金实控人工邦及其团队持股平台签订《投资合作框架协议》，交易内容为:公司拟以现金及全资孙公司宝船生物全部股权支付的方式成为上海赛金的第一大股东并相对控股。- The End -戳“阅读原文”，了解更多医药研发及股市资讯。

临床结果临床3期上市批准临床2期

2022-12-03

·药明康德

速递 | 晚期患者95%治疗成功率！赛诺菲在研新药有望改变致命疾病的治疗格局

▎药明康德内容团队编辑近日，非营利性药物研发组织“被忽视疾病药物倡议”（DNDi）和赛诺菲（Sanofi）共同宣布，其针对昏睡病的潜在疗法acoziborole在一项2/3期临床试验中取得了喜人的疗效，治疗成功率高达95%。昏睡病是一种通过采采蝇叮咬传播的寄生虫病，也被称为非洲人类锥虫病（HAT）。其中，非州锥虫病冈比亚菌株（g-HAT）感染占所有昏睡病病例的93%。该病早期阶段的症状为头痛或发烧。到晚期阶段，寄生虫穿过血脑屏障并侵入中枢神经系统，进而引起神经精神相关的症状，例如睡眠混乱、意识模糊、嗜睡和抽搐，最终可能导致死亡。在过去20年中，昏睡病的报告病例数量急剧下降，从1998年的近4万例报告病例（未确诊病例估计超过30万例）下降到2020年的不到1000例。这都得益于DNDi、赛诺菲、刚果民主共和国和几内亚的国家昏睡病控制项目、世界卫生组织（WHO）、无国界医生组织（MSF）和其他合作伙伴之间的成功合作，使昏睡病的治疗有了根本性的改善。这虽是令人鼓舞的趋势，但我们依然要警惕这种能够广泛传播的寄生虫病的再次爆发。▲血液涂片中弯曲扭动的锥虫（图片来源：Photo Credit:Content Providers: CDC/Dr. Myron G. Schultz [Public domain]）2009年，赛诺菲和合作伙伴开发了一种称为NECT的联合疗法，疗效很好，安全性相比于此前用于治疗锥虫感染的毒性强烈的砷衍生物有了极大的提高，但该疗法在临床中的使用有诸多不便。为此，DNDi、赛诺菲及其合作伙伴于2018年开发了首个用于昏睡病的全口服疗法非昔硝唑，这种为期10天的疗法现已在所有昏睡病流行的国家供应。Acoziborole是DNDi，赛诺菲和合作伙伴开发的新化学药物，其能够单次口服给药的特点意味着该疗法有望成为促进消灭昏睡病的主要工具。流动团队的医护人员一旦确诊患者，就可以立即给患者服用一剂acoziborole，并且无需住院或在家中监督治疗。该2/3期研究由DNDi及其在刚果民主共和国（DRC）和几内亚的合作伙伴主导，结果已发表在《柳叶刀-传染病》医学杂志上。研究人员在2016年至2019年间从刚果民主共和国和几内亚的10家医院招募了208名患者，以评估acoziborole在早期和晚期g-HAT感染患者中的安全性和有效性。在晚期g-HAT感染患者中，acoziborole在18个月时的治疗成功率为95%（现有治疗研究的最佳结果为94%）。此外，41例早期g-HAT感染患者在所有时间点的治疗成功率都为100%。该研究表明，acoziborole具有良好的安全性，没有报告与药物相关的显著安全信号。这些关键结果将成为赛诺菲向欧洲药品管理局（EMA）提交申请的基础，这是消灭昏睡病的又一个里程碑。如果获得批准，赛诺菲将通过其慈善组织向WHO捐赠acoziborole。Acoziborole目前仍处于临床研究阶段，其安全性和有效性尚未得到任何监管机构的评估。WHO的被忽视热带疾病路线图战略设置了一个目标，要在2030年之前阻断g-HAT传播。基于这个目标，acoziborole的出现为开辟一种简单的“筛查和治疗”方法提供了可能——通过快速针刺血液测试筛查可能被感染的患者，然后给予acoziborole治疗。这种方法既不需要更复杂的寄生虫学确认，也不需要住院治疗。该试验的首席研究员兼《柳叶刀》子刊文章的主要作者Victor Kande博士表示：“昏睡病是一种噩梦般的疾病，它影响着西非和中非一些最偏远地区的患者，这些地区离医院的距离通常要用天来衡量。我们现在正处于一种潜在疗法的前沿，只需一次服用三粒药，一天就能完成所有的治疗——这将是医生和社区的一场革命。”▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放参考资料：[1] Acoziborole: Investigational single-dose oral treatment raises hope for elimination of sleeping sickness in Africa. Retrieved November 30, 2022, from https://www.sanofi.com/en/media-room/press-releases/2022/2022-11-30-07-00-00-2564640免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。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：本文来自药明康德内容团队，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

临床研究

100 项与 Acoziborole 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	Acoziborole	-	DB13086

研发状态

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
布氏锥虫感染	临床3期	刚果共和国	Drugs for Neglected Diseases initiative	2021-12-30
布氏锥虫感染	临床3期	几内亚	Drugs for Neglected Diseases initiative	2021-12-30
肝素诱导血小板减少	临床3期	刚果共和国	Drugs for Neglected Diseases initiative	2016-10-11
肝素诱导血小板减少	临床3期	几内亚	Drugs for Neglected Diseases initiative	2016-10-11
非洲锥虫病	临床3期	-	Anacor Pharmaceuticals LLC	-

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临床结果

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT03087955 (Pubmed) 人工标引	临床2/3期	非洲锥虫病	208	Acoziborole	構範鬱顧鹹鏇簾窪壓襯(構鬱齋廠鹹簾積窪願顧) = 簾選憲構選鹽膚膚獵淵壓顧夢獵廠齋壓艱積構 (廠膚艱夢繭積選窪積鏇, 91.2 ~ 97.7) 更多	积极	2022-11-29