This phase II trial tests how well fezolinetant works in improving vasomotor symptoms (VMS) in breast cancer patients taking endocrine therapy (ET). Anti-hormone treatments are effective for lowering the risk of breast cancer but can cause bothersome VMS, such as hot flashes and night sweats. Fezolinetant inhibits the activity of the neurokinin type 3 receptor and has shown activity against VMS in postmenopausal women. Taking fezolinetant may work well at improving VMS in breast cancer patients taking ET.

开始日期2024-12-01

申办/合作机构

Rogel Cancer Center: University of Michigan

[+1]

NCT06628388 / Not yet recruitingN/AIIT

Nurse-led Primary Healthcare Intervention Model in Women's Health Management in Hong Kong

The goal of this randomized clinical trial is to learn if a nurse-led primary healthcare intervention can help improve health management in women adults aged 45 to 64, and to develop an evidence-based primary healthcare model for women. The questions it aims to answer are:
if the intervention improves medical resource use, especially at 3-month from the beginning of intervention? if the intervention improves health confidence, self-management, and quality-of-life? if the intervention improves health conditions as reflected by risk levels?
Researchers will compare the intervention with the control group over time to see if there are better medical resource use, health confidence, self-management, and quality-of-life, and health conditions risk levels.
All participants will be asked to answer a set of questionnaires assessing their social demographics, health-related status, and risk levels of the targeted health conditions. All participants will receive an education booklet containing essential knowledge and available medical resources. Participants in the intervention group will receive nurse-led 5As (ask, advice, assess, assist, arrange) intervention which offers risk-specific health services. Participants will be followed at 3-, 6-, 12-month; with high risk participants will be followed additionally at 1-month after the beginning of intervention. Participants in the waitlist control group will be given general health advice as minimal intervention, followed-up at 3- and 6-month, provided with intervention of the same content with intervention group at 6-month, followed-up at 7-month and 12-month.

开始日期2024-11-01

申办/合作机构

香港大学

[+2]

NCT06497894 / Not yet recruiting临床4期

No More Sleepless Nights in Perimenopause - an Open Label, Randomized, Parallel-group, Active Controlled Intervention Study in Perimenopausal Women With Vasomotor Symptoms and Insomnia to Investigate the Efficacy of Hormone Replacement Therapy and Cognitive Behavioral Therapy

The goal of the clinical trial is to learn if Hormone Replacement Therapy (HRT) and Cognitive Behavioral Therapy (CBT-I) can treat insomnia in menopausal women. The main questions it aims to answer are:
* Are HRT and CBT-I effective in reducing insomnia in menopausal women?
* Are HRT and CBT-I comparable in terms of reducing insomnia severity in this population?
Researchers will compare HRT and CBT-I to an active control group, receiving sleep hygiene instructions.
Participants will:
* Complete a screening and baseline assessment
* Receive Hormone Replacement Therapy (as prescribed), Cognitive Behavioral Therapy (1/week), or sleep hygiene instructions (1/week) for 8 weeks.
* Keep a daily diary (sleep e-diary), to assess sleep-quality.
* Wear an electroencephalogram (EEG) during 6 nights (3 at baseline & 3 post-intervention), to assess deep sleep cycles and waking episodes.
* Receive a phone call for intervention compliance
* Complete a post-intervention assessment.

开始日期2024-10-01

申办/合作机构

Insel Gruppe AG

100 项与血管舒缩症相关的临床结果

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100 项与血管舒缩症相关的转化医学

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0 项与血管舒缩症相关的专利（医药）

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2,781

项与血管舒缩症相关的文献（医药）

2024-12-31·Gynecological Endocrinology

Transfer of preclinical study data on the influence of cimicifuga racemosa on functional changes in the hippocampus during menopause

Review

作者: Henneicke-von Zepelin, Hans-Heinrich ; Stute, Petra ; Nicken, Petra

Menopausal transition in women involves complex neurobiochemical changes linked to ovarian dysfunction, resulting in symptoms like vasomotor symptoms (VMS), sleep disturbances, anxiety, and cognitive impairments. Hormone replacement therapy is the first-line treatment. However, many women are reluctant to use HRT or have contraindications toward HRT and seek for alternatives. Non-hormonal therapies with extracts of Cimicifuga racemosa rhizomes like the isopropanolic extract (iCR, black cohosh) offer a promising alternative. A preclinical pilot study exploring iCR's effects on gene expression in the hippocampus and hypothalamus of ovarectomized (OVX) rats mimicking menopausal conditions identified important signaling pathways and CNS-based contributions to the multitargeted modes of action of iCR. Especially in the hippocampus, iCR compensated effects of OVX on gene expression profiles. These changes are reflected by the genes AVPR1A, GAL, CALCA, HCRT, PNOC, ESR1, ESR2 and TAC3 contributing to the formation of hot flushes or thermoregulation as well as to secondary effects such as blood pressure, metabolism, hormonal regulation, homeostasis, mood regulation, neuroendocrine modulation, regulation of sleep and arousal, and in learning, memory and cognition. To understand the mechanisms in the brain of estrogen-depressed animals (OVX) and subsequent iCR treatment we combined the results of the pilot study with those of up-to-date literature and tried to transfer the current knowledge to humans during menopausal transition and adaptation. Focus was laid on changes in the hippocampal function, that is disturbed by hormonal fluctuations, but can also be brought back into balance by iCR.

2024-12-01·European Journal of Medicinal Chemistry

Nitrogen-containing heterocyclic drug products approved by the FDA in 2023: Synthesis and biological activity

Review

作者: Wang, Suqi ; Zhao, Zeyan ; Liu, Yiqi ; Tang, Shengsong ; Qin, Hui ; Luo, Weijiang ; He, Weimin ; Peng, Junmei

This article profiles 13 newly approved nitrogen-containing heterocyclic drugs by the U.S. Food and Drug Administration (FDA) in 2023. These drugs target a variety of therapeutic areas including proteinuria in patients with IgA nephropathy, migraine in adults, Rett syndrome, PI3Kδ syndrome, vasomotor symptoms, alopecia areata, acute myeloid leukemia, postpartum depression, myelofibrosis, and various cancer and tumor types. The molecular structures of these approved drugs feature common aromatic heterocyclic compounds such as pyrrole, imidazole, pyrazole, isoxazole, pyridine, and pyrimidine, as well as aliphatic heterocyclic compounds like caprolactam, piperazine, and piperidine. Some compounds also contain multiple heteroatoms like 1,2,4-thiadiazole and 1,2,4-triazole. The article provides a comprehensive overview of the bioactivity spectrum, medicinal chemistry discovery, and synthetic methods for each compound.

2024-12-01·Maturitas

Ultra-low dose estradiol and dydrogesterone for the treatment of menopausal symptoms in a pooled, multi-ethnic population

Article

作者: Karpova, Viktoriya ; Simoncini, Tommaso ; Nappi, Rossella Elena ; Kahler, Elke ; Yu, Qi ; Stevenson, John C. ; Tatarchuk, Tetiana ; Ren, Mulan ; Custodio, Marcelo Graziano ; Stevenson, John C

OBJECTIVESEvidence suggests ethnicity-specific differences in postmenopausal symptoms, highlighting the need for therapies that are efficacious across different ethnicities. We evaluated the efficacy of an ultra-low dose combination of 0.5 mg estradiol and 0.25 mg dydrogesterone (E 0.5 mg/D 2.5 mg) in alleviating vasomotor symptoms across a multi-ethnic population.STUDY DESIGNData from two controlled trials were pooled to form a dataset of 583 postmenopausal women from across Europe and China. Participants were randomized to receive treatment with E 0.5 mg/D 2.5 mg or placebo for 12 weeks.MAIN OUTCOME MEASURESThe main efficacy variable was absolute change in the number of hot flushes from baseline to end of treatment. Health-related quality of life and safety were also assessed.RESULTSChange in the number of hot flushes per day was greater with E 0.5 mg/D 2.5 mg versus placebo (mean difference - 1.5, 95 % confidence interval - 2.1, -1.0; p < 0.001). Participants treated with E 0.5 mg/D 2.5 mg reported improvement in health-related quality of life (including psychological symptoms, vaginal dryness), and high amenorrhea rates. Combined E 0.5 mg/D 2.5 mg was well tolerated: there were no differences between groups in the percentage of participants with at least one serious adverse event or treatment-emergent serious adverse events. Analysis of change in body weight indicated no differences between groups.CONCLUSIONSThis pooled analysis demonstrates the consistent efficacy of E 0.5 mg/D 2.5 mg in the treatment of menopause-related symptoms across a multi-ethnic population of postmenopausal women.

210

项与血管舒缩症相关的新闻（医药）

2024-10-30

·GlobeNewswire-Health Care

Noema Pharma Announces First Patients Dosed in Phase 2b Study with Gemlapodect (NOE-105), a First-In-Class Investigational Therapy for Tourette Syndrome

Phase 2b study will evaluate the safety and efficacy of gemlapodect (NOE-105) in patients with Tourette syndromeBASEL, Switzerland, Oct. 30, 2024 (GLOBE NEWSWIRE) -- Noema Pharma AG, a clinical-stage neuroscience-based company, today announced the first patients have been dosed in a global Phase 2b study evaluating the safety and efficacy of gemlapodect (NOE-105) in Tourette syndrome. NOE-TTS-201 (NCT06315751) is a Phase 2b, 12-week, randomized, double-blind, placebo-controlled study that is designed to examine the safety and efficacy of gemlapodect. The trial will enroll 140 patients with Tourette syndrome in multiple centers in the US and Europe. Patients will be treated with up to 15 mg of gemlapodect once daily, or a matching placebo. The primary endpoint is the Yale Global Tic Severity Scale (YGTSS) Total Tic Score. “Patients with Tourette syndrome carry an incredible burden related to the expression of motor and vocal tics with limited treatment options,” said Ilise Lombardo, M.D., Chief Executive Officer of Noema Pharma. “I am happy to announce the progression of the development of gemlapodect in Tourette syndrome with the dosing of the first patients in our Phase 2b trial.” About Tourette Syndrome Tourette syndrome is a neurodevelopmental disorder in the wider spectrum of tic disorders, consisting of motor and vocal abrupt and brief movement or sound which is preceded by a local or generalized urge/premonitory sensation. TS impacts over a half-million individuals and families in the US alone, as well as millions of individuals worldwide. About NOE-105/gemlapodect NOE-105/gemlapodect is a potential first-in-class phosphodiesterase-10A (PDE10A) inhibitor in mid-stage clinical development as a dopamine modulator for Tourette syndrome. NOE-105 has been well-tolerated in studies with healthy volunteers and in patients with Tourette syndrome. NOE-105 has the potential to achieve a more targeted effect by modulating dopamine signaling exclusively in the medium spiny neurons of the striatum in the brain, while limiting off-target effects compared to other dopamine antagonists. About Noema Pharma Noema Pharma is a clinical-stage biotech company advancing a portfolio of transformative therapeutics utilizing first-in-disease approaches targeting neuroscience-based conditions with high unmet need. Noema has four programs currently in active Phase 2 clinical trials evaluating pain in Trigeminal Neuralgia, seizures in Tuberous Sclerosis Complex, Tourette syndrome and vasomotor symptoms plus associated symptoms of menopause with readouts expected in 2025. Noema was founded by leading venture capital firm Sofinnova Partners and is supported by current investors including Forbion, Gilde Healthcare, Invus, Jeito Capital, Polaris Partners and UPMC Enterprises. Learn more at www.noemapharma.com. Investor Contact: Precision AQAlex Lobo212-698-8802alex.lobo@precisionaq.com

临床2期临床结果

2024-10-29

·安斯泰来中国

安斯泰来在IMS世界更年期大会上展示VEOZA™️（fezolinetant）数据

在10月19日至22日于澳大利亚墨尔本举行的国际绝经协会（IMS）第19届世界绝经大会上，安斯泰来治疗绝经相关中重度血管舒缩症状（VMS）的同类首创药物VEOZA™（fezolinetant）*在四场口头报告和两张壁报展示。VMS，也称为潮热和/或盗汗，是绝经的常见症状。1,2 安斯泰来副总裁兼生物制药开发负责人 Marci English “Astellas致力于推进创新科学，世界绝经大会上的数据进一步加深了我们对VMS如何影响个人和女性治疗偏好的理解。此外，我们很高兴分享我们最近启动的HIGHLIGHT研究的试验设计，该研究旨在评估fezolinetant用于治疗接受辅助内分泌治疗的乳腺癌症患者中度至重度VMS。” 医学博士、安斯泰来副总裁兼医学事务特药治疗领域负责人 Emad Siddiqui “我们致力于在关键试验之外继续扩大对fezolinetant的了解。我们在世界绝经大会上的演讲包括广泛的临床和健康相关的生活质量分析，进一步强调了fezolinetant的安全性和有效性，以及它对患者报告结局（包括睡眠和工作效率）的积极影响。” Fezolineant数据在四场口头报告和两张壁报中呈现： •SKYLIGHT 1和2研究的两场口头报告侧重于患者报告的睡眠结果以及Fezolinetant对工作效率的影响。 •DAYLIGHT研究的两场口头报告侧重在Fezolinetant用于不适合激素治疗的女性，包括药物应答和生活质量。 •壁报强调了对临床和非临床数据的分析，这些数据表明Fezolinetant治疗与恶性肿瘤发病率之间没有关联。 •壁报还包括了最近启动的3期临床研究-HIGHLIGHT 1的研究设计，旨在评估Fezolinetant用于正接受辅助内分泌治疗的0至3期激素受体阳性的乳腺癌患者中度至重度VMS的疗效和安全性。另外两场口头报告强调了澳大利亚女性对与绝经相关的VMS治疗的偏好，以及一项文献综述的结果，该文献综述旨在确定和描述与个体VMS经历相关的概念，目的是为开发一种文化敏感的VMS自我评估工具提供信息。 *VEOZA：在美国被批准为“VEOZAH™”。向下滑动查看更多▼ 关于BRIGHT SKY™第三阶段项目 BRIGHT SKY关键试验SKYLIGHT 1™（NCT04003155）和SKYLIGHT 2™（NCT04003142）招募了1000多名患有中度至重度VMS的绝经女性。试验在前12周采用双盲、安慰剂对照，然后接受40周的扩展期治疗。入组患者来自美国、加拿大和欧洲的180多个研究中心。SKYLIGHT 4™（NCT04003389）是一项为期52周的双盲安慰剂对照研究，旨在研究Fezolinetant的长期安全性。对于SKYLIGHT 4，在美国、加拿大和欧洲的180多个研究中心招募了超过1800多名患有VMS的绝经妇女。关于DAYLIGHT DAYLIGHT（NCT05033886）是一项临床3b研究，为期24周的随机、双盲、安慰剂对照研究，旨在评估Fezolinetant对40-65岁患有中度至重度VMS且被认为不适合激素治疗的绝经女性的疗效和安全性。在加拿大、欧洲和土耳其的69个研究中心共入组453名女性。关于HIGHLIGHT 1TM HIGHLIGHT 1（NCT06440967）是一项随机、安慰剂对照、双盲、3期临床研究，旨在评估在接受辅助内分泌治疗的0至3期激素受体阳性乳腺癌症患者中， fezolinetant用于治疗中度至重度VMS的有效性和安全性。计划在欧洲和加拿大的100多个研究中心，约有540名患者入组，1:1随机分配到fezolinetant治疗组或安慰剂组。四个共同的主要终点是从基线至第4周和第12周，中重度VMS发生频率和严重程度的变化。患者将接受52周的治疗，并在55周时进行最终评估。关于VEOZATM（fezolinetant） VEOZA（fezolinetant）是一款非激素神经激肽3（NK3）受体拮抗剂，在澳大利亚用于治疗与绝经相关的中重度血管舒缩症状（潮热和盗汗）。VEOZA通过阻断神经激肽B（NKB）与kisspeptin/神经激肽/强啡肽（KNDy）神经元的结合来调节大脑温度控制中心（下丘脑）的神经元活动，从而减少潮热和盗汗的频率和严重程度。3,4,5 重要安全信息关于fezolinetant 的完整澳大利亚产品信息和澳大利亚公众评估报告（AusPAR）可从澳大利亚政府治疗用品管理局获得。澳大利亚产品信息： https://www.tga.gov.au/sites/default/files/2024-05/auspar-veoza-240503-pi.pdf 澳大利亚公众评估报告： https://www.tga.gov.au/resources/auspar/auspar-veoza 关于安斯泰来安斯泰来制药集团是一家制药企业，业务遍及全球70多个国家和地区。目前，我们正在推进“焦点领域的研究策略(Focus Area Approach)”，旨在通过聚焦生理机制和治疗手段，确定持续研发新药的机会，解决尚未被满足的医疗需求。与此同时，我们正在将目光投向处方药以外的业务领域，将我们的专业技能和知识与不同领域外部合作伙伴的尖端技术相结合，打造Rx+®医疗解决方案。通过这些努力，安斯泰来立志处于不断变化的医疗行业的最前沿，将科学的进步转变为患者的价值。更多信息，请访问我们的网站https://www.astellas.com/en。安斯泰来警戒声明本新闻稿中，有关当前计划、估计、战略和信念以及其他非历史事实的陈述，均为关于安斯泰来未来表现的前瞻性陈述。这些陈述是根据管理层结合当前可获得的信息而形成的当前假设和信念得出的，并涉及已知和未知的风险与不确定性。许多因素可能导致实际结果与前瞻性陈述中讨论的结果产生重大差异。这些因素包括但不限于：(i)与制药市场有关的一般经济条件和法律法规的变化，(ii)货币汇率波动，(iii)新产品上市的延迟，(iv)安斯泰来无法有效地销售现有产品和新产品，(v)安斯泰来无法继续有效地研究和开发在竞争激烈的市场中被客户接受的产品，以及(vi)第三方侵犯安斯泰来的知识产权。本新闻稿中包含的有关药品(包括当前正在开发的产品)的信息并不构成广告或医疗建议。参考文献 Utian WH. Psychosocial and socioeconomic burden of vasomotor symptoms in menopause: a comprehensive review. Health Qual Life Outcomes. 2005;3:47. Jones RE, Lopez KH, eds. Human Reproductive Biology. 4th ed. Waltham, MA: Elsevier, 2014:120. Depypere H, Timmerman D, Donders G, et al. Treatment of menopausal vasomotor symptoms with fezolinetant, a neurokinin 3 receptor antagonist: a phase 2a trial. J Clin Endocrinol Metab. 2019;104:5893-5905. Fraser GL, Lederman S, Waldbaum A, et al. A phase 2b, randomized, placebo-controlled, double-blind, dose-ranging study of the neurokinin 3 receptor antagonist fezolinetant for vasomotor symptoms associated with menopause. Menopause. 2020;27:382-392. Fraser GL, Hoveyda HR, Clarke IJ, et al. The NK3 receptor antagonist ESN364 interrupts pulsatile LH secretion and moderate levels of ovarian hormones throughout the menstrual cycle. Endocrinology. 2015;156:4214-4225.

临床3期临床成功

2024-10-19

·药渡Daily

【药渡药闻报告-创新药篇】-2024年第40期/总第161期

全球药物批准/研发动态 01 全球新药批准情况根据药渡数据统计分析，本次统计周期（2024.10.05-10.11）全球（不含中国）共有4个新药获批上市。其中，NDA批准2个，BLA批准1个，新适应症批准1个。与上个统计周期相比，本次减少1个批准新药。 10月3日，FDA批准纳武利尤单抗（Opdivo）联合铂类双药化疗作为新辅助治疗，随后在手术后使用纳武利尤单抗单药作为辅助治疗，用于可切除（肿瘤≥4cm和/或淋巴结阳性）非小细胞肺癌（NSCLC）且无已知EGFR突变或ALK重排的成人。纳武利尤单抗组未达到中位无事件生存期，化疗组未达到18.4个月，不良反应与Nivolumab联合化疗的其他临床试验中发生的不良反应相似。推荐的纳武利尤单抗剂量为每3周360毫克（新辅助治疗）和每4周480毫克（辅助治疗）。 10月8日，卫材宣布“URECE片剂”（Dotinurad）已在泰国获得批准，用于治疗痛风和高尿酸血症者。URECE通过选择性抑制与肾脏尿酸重吸收相关的尿酸盐转运蛋白来抑制尿酸重吸收并降低血尿酸水平。已知高尿酸血症与多种疾病有关，包括泌尿系统、内分泌系统、代谢系统和心脑血管系统的疾病，以及痛风，由于生活方式和饮食偏好的变化，患者人数将进一步增加。全球（不含中国）新药批准情况（部分） 02 全球新药申报进展根据药渡数据统计分析，本次统计周期（2024.10.05-10.11）全球（不含中国）共有6个新药申报上市。其中，NDA申报4个，BLA申报1个，细胞疗法1个。与上次统计周期相比，本次减少2个NDA/BLA申报。 10月9日，拜耳宣布FDA已受理Elinzanetant的新药申请，用于治疗与更年期相关的中度至重度血管舒缩症状，其新药申请基于三项III期研究的积极数据OASIS 1、2和3。Elinzanetant是第一个双重神经激肽-1和3受体拮抗剂，处于后期临床开发阶段，每天口服一次。 NDA/BLA申报（部分）根据药渡数据统计分析，本次统计周期（2024.10.05-10.11）全球（不含中国）共有6个药物获监管机构特殊资格认定。其中，化学药4个，生物药2个。与上次统计周期相比，本次增加1个获监管机构特殊资格认定的药物。 10月9日，索元生物宣布DB104（Liafensine）用于难治性抑郁症（TRD）获FDA授予快速通道资格。Liafensine是三重再摄取抑制剂，可同时抑制在抑郁症中起重要作用的5-羟色胺、去甲肾上腺素和多巴胺的再摄取。今年4月，索元生物宣布ENLIGHTEN研究顺利完成并达到了主要终点及所有次要终点。DGM4阳性TRD受试者的蒙哥马利-阿斯伯格抑郁量表评分相比对照组改善了近4.4分，其安全性及耐受性表现特别优异。 10月10日，FDA授予Coherent Biopharma的注射用CBP-1008快速通道资格，用于治疗铂类耐药卵巢透明细胞癌（OCCC）。超过315名恶性实体瘤患者已被纳入CBP-1008试验，CBP-1008耐受性良好，不良事件多为轻度至中度，ORR达到31.3%，mDOR达到7.2个月。此外，CBP-1008在RP3D为0.15 mg/kg的先前1-3L治疗中显示出优异的PROC疗效。在高级别浆液性卵巢癌（HGSOC）目标人群中，ORR和DCR分别达到50.0%和85.7%。特殊资格认定情况（部分） 03 全球新药研发进展根据药渡数据统计分析，本次统计周期（2024.10.05-10.11）全球（不含中国）新药临床研发状态更新共计33条，涉及肿瘤、血液和淋巴疾病、遗传代谢病、神经疾病以及免疫系统疾病等共计10个领域。其中，肿瘤领域临床进展更新居各领域之首，为12条：涉及化学药5条，生物药5条，细胞疗法2条。 10月8日，Boehringer宣布FDA已授予胰高血糖素/GLP-1受体双重激动剂Survodutide突破性疗法认定，用于治疗患有非肝硬化代谢功能障碍相关脂肪性肝炎和中度或晚期纤维化（2期或3期）的成人。Boehringer启动了两项关于Survodutide的III期研究，分别是LIVERAGE治疗MASH和中度或晚期纤维化患者的LIVERAGE-Cirrhosis。 10月9日，多域宣布其FLT3/IRAK4双靶点抑制剂HPB-092的临床研究申请获FDA批准，即将开展用于治疗急性髓性白血病（AML）的I期临床研究。在临床前研究中，其FLT3/IRAK4双靶点抑制剂HPB-092活性指标与安全性显著优于目前已获批的同靶点药物。已有数据证明抑制IRAK4活性可以治疗RNA剪切体突变的AML和骨髓增生异常综合征。全球新药研发进展详情（部分） 04 全球医药交易事件本次统计周期（2024.10.05-10.11）全球（含中国）医药交易时间共计37起，涉及药物权益转让、公司并购等多起交易事件。全球医药交易时间汇总表（部分）国内药物批准/研发动态 01 国内新药批准情况根据药渡数据统计分析，本次统计周期（2024.10.05-10.11）国内共有8个新药获NMPA批准上市。其中，NDA批准1个，BLA批准3个，新适应症批准4个。与上次统计周期相比，本次增加5个NMPA批准新药。 10月8日，NMPA官网显示，丽珠医药注射用醋酸曲普瑞林微球的新适应症上市申请已获得批准。该产品是每月一次肌肉注射的一种促性腺激素释放激素（GnRH）激动剂，本次获批的适应症为治疗子宫内膜异位症。GnRH可显著降低血雌激素水平，强效减轻内异症相关疼痛症状，一定程度缩小内异症病灶。此款曲普瑞林微球具有起效时间长、减少用药次数等特点，可减轻患者痛苦和用药负担，提高用药耐受性和可及性。 10月11日，君实生物宣布昂戈瑞西单抗注射液（重组人源化抗PCSK9单克隆抗体注射液）的上市许可申请获得NMPA批准，用于治疗原发性高胆固醇血症（非家族性）和混合型血脂异常的成人患者。此次获批包括2种规格，分别为150mg（1ml）/支（预充式注射器）、150mg（1ml）/支（预充式自动注射器）。JS002-003研究结果显示，昂戈瑞西单抗150mg Q2W或300mg Q4W皮下注射，可显著降低LDL-C水平达60%以上，且在52周的治疗期间始终维持稳定的降幅。国内新药批准情况（部分） 02 国内新药临床默示许可进展根据药渡数据统计分析，本次统计周期（2024.10.05-10.11）国内共有24个新药获临床默示许可，涉及34个受理号。其中，化学药6个，治疗用生物制品17个，预防用生物制品1个。与上次统计周期相比，本次增加14个临床默示许可获批受理号。本周国内新药临床默示许可进展（部分） 03 国内新药申报进展根据药渡数据统计分析，本次统计周期（2024.10.05-10.11）国内共有9个新药申报上市，涉及17个受理号。其中，化药4个，治疗用生物制品5个。与上个统计周期相比，本次增加9个新药申报上市受理号。国内新药申报上市情况（部分）根据药渡数据统计分析，本次统计周期（2024.10.05-10.11）国内共有23个新药申报临床，涉及31个受理号。其中，化学药13个，治疗用生物制品8个，预防用生物制品2个。与上次统计周期相比，本次增加6个临床申报受理号。国内新药临床申报情况（部分）根据药渡数据统计分析，本次统计周期（2024.10.05-10.11）国内共有1个药物获NMPA特殊资格认定。其中，化学药1个。与上次统计周期相比，本次减少1个获监管机构特殊资格认定的药物。 10月13日，恒瑞医药收到国家药品监督管理局下发的《受理通知书》，公司1类创新药、EZH2抑制剂SHR2554片的药品上市许可申请获国家药监局受理，且被纳入拟优先审评品种公示，拟定适应症为：用于既往接受过至少1线系统性治疗的复发或难治外周T细胞淋巴瘤（PTCL）。SHR2554-I-101研究前期数据显示：每天2次350mg给药SHR2554安全性良好，对包括复发或难治PTCL和滤泡性淋巴瘤（FL）在内的多种淋巴瘤中显示出良好的抗肿瘤活性；II期推荐剂量下，61%的复发或难治PTCL获得缓解，预估中位PFS为11.1个月。国内监管机构特殊资格认定药物 04 国内新药研发进展根据药渡数据统计分析，本次统计周期（2024.10.05-10.11）国内新药临床研发状态更新共计6条，涉及肿瘤、遗传和代谢疾病以及皮肤病共计3个领域。其中，化学药4条，生物药2条。与上次统计周期相比，本次增加1条国内新药临床研发状态更新。 10月9日，诺诚健华宣布其新型酪氨酸激酶2（Tyrosine Kinase 2, TYK2）抑制剂ICP-488治疗中重度斑块状银屑病II期临床研究达到主要终点。在接受治疗12周的患者中，ICP-488展示了卓越的有效性和安全性，在每日一次6毫克和9毫克两个剂量组中，PASI 75的应答率分别达到77.3%和78.6%，PASI 90的应答率分别达到36.4%和50.0%，PASI 100的应答率分别达到11.4%和11.9%，sPGA 0/1的应答率分别达到70.5%和71.4%。 10月10日，中国药物临床试验登记与信息公示平台官网近日公示，辉瑞已经启动了一项国际多中心（含中国）III期临床研究，该研究旨在评价PF-06821497联合恩扎卢胺对比恩扎卢胺或多西他赛对既往接受过醋酸阿比特龙治疗的转移性去势抵抗性前列腺癌患者的疗效和安全性。研究表明，EZH2的过表达可通过多种作用机制来促进癌症的发生和进展，包括沉默肿瘤抑制基因的表达、促进参与细胞增殖和存活的基因表达等。国内新药研发进展（部分） 05 国内新药研发领域政策法规动态国家药监局药审中心关于发布《抗肿瘤药物临床试验中SUSAR分析与处理技术指导原则》的通告（2024年第42号）为加强临床试验可疑且非预期严重不良反应（SUSAR）信息的风险评估，提升SUSAR信息的发现、识别和风险处理，药审中心组织制定了《抗肿瘤药物临床试验中SUSAR分析与处理技术指导原则》（见附件）。根据《国家药监局综合司关于印发药品技术指导原则发布程序的通知》（药监综药管〔2020〕9号）要求，经国家药品监督管理局审查同意，现予发布，自发布之日起施行。特此通告。 06 国内新药研发领域热点新闻站在十字路口上的“ADC一哥“ 如果要问中国创新药最拿得出手的是什么，ADC药物绝对拥有一战之力。2022年科伦博泰与默沙东的百亿美元合作，以及2023年百利天恒与BMS的84亿美元交易打破了中国创新药单个项目出海记录，让外界意识到中国已经成为全球ADC药物研发的中心。然而，在国产ADC大放异彩的同时，曾经的国产“ADC一哥”荣昌生物正在面临“生死抉择”，核心产品商业化加速的同时，机会与挑战共存。更多资讯，请阅读原文罗氏，挺进CVRM领域近期，罗氏在2024年医药日活动中阐述了最新药物研发进展以及未来研发的战略方向。值得注意的是，除了肿瘤、自身免疫、神经系统、眼科等传统优势业务，心血管、肾脏及代谢领域（CVRM）成为罗氏新押注的疾病领域，罗氏认为，在下一个增长周期中，CVRM将成为业绩增长的重要引擎。更多资讯，请阅读原文咨询、合作请联系作者小D有话说为方便读者阅读及保存，我们将周报原文整理成了PDF版本，如需获取全文，可点击最上方蓝字，在药渡Daily公众号后台回复“1018创新药周报”，即可下载。

上市批准申请上市临床3期临床结果