更新于：2024-05-01

Smith-Magenis Syndrome

Smith-Magenis综合征

更新于：2024-05-01

基本信息

别名

17p deletion syndrome、17p- syndrome、17p11.2 microdeletion syndrome

+ [26]

简介

Complex neurobehavioral disorder characterized by distinctive facial features (FACIES), developmental delay and INTELLECTUAL DISABILITY. Behavioral phenotypes include sleep disturbance, maladaptive, self-injurious and attention-seeking behaviors. The sleep disturbance is linked to an abnormal circadian secretion pattern of MELATONIN. The syndrome is associated with de novo deletion or mutation and HAPLOINSUFFICIENCY of the retinoic acid-induced 1 protein on chromosome 17p11.2.

关联

项与 Smith-Magenis综合征相关的药物

Setmelanotide Acetate

靶点

MC4R

作用机制

MC4R激动剂

在研机构

原研机构

在研适应症

非在研适应症

最高研发阶段批准上市

首次获批国家/地区

美国

首次获批日期2020-11-25

Tasimelteon

他司美琼

靶点

Melatonin receptor

作用机制

褪黑激素受体家族激动剂

在研机构

Vanda Pharmaceuticals Netherlands BV [+1]

原研机构

Vanda Pharmaceuticals, Inc.

在研适应症

入睡和睡眠障碍 [+6]

非在研适应症

重度抑郁症 [+5]

最高研发阶段批准上市

首次获批国家/地区

美国

首次获批日期2014-01-31

Irradiated Allogeneic Cells(NCI)

靶点-

作用机制

细胞替代物

在研机构-

原研机构

National Cancer Institute

在研适应症-

非在研适应症

急性髓性白血病 [+11]

最高研发阶段无进展

首次获批国家/地区-

首次获批日期1800-01-20

项与 Smith-Magenis综合征相关的临床试验

DRKS00033193 / RecruitingN/A

MySyndrome - Rare Disease Patient Registry for Treatability, Natural History, Phenotype and Psychosozial Aspects of Rare Genetic Developmental Diseases - MySyndrome

开始日期2024-01-01

申办/合作机构-

NCT06247852 / RecruitingN/AIIT

Persistent Pain After Cesarean Delivery - A Danish Multicenter Cohort Study

In Denmark, around 12,700 patients undergo Cesarean delivery (CD) annually, constituting approximately 20% of all childbirths in the country. Worldwide, the number is increasing each year, with more than 20 million CD performed annually.
Postoperative pain after CD is typically moderate to severe in intensity and still constitutes a significant challenge, balancing effective pain relief and potential side effects. Correlations between moderate to severe acute pain and the development of persistent pain after CD is still unclear.
Postsurgical persistent pain is a significant, often unrecognized clinical problem that causes distress and diminishes the quality of life for patients. Despite advances in understanding the factors contributing to persistent postsurgical pain and an increased focus on identifying patients at risk, the management and prevention of postsurgical persistent pain are still inadequate.
It is important to gain further insights into this population, and we have a unique opportunity to do so by following the national cohort from the ongoing Danish multicenter cohort study on pain after cesarean section (ClinicalTrials.gov Identifier: NCT06012747) over an extended follow-up period.
This involves continued prospective registration of Patient-Reported Outcome Measures (PROM) collected by SMS-based questionnaires in the months and years after the CD, thereby investigating the occurrence of both acute and persistent pain after CD.
The outcomes are focused on pain levels, the impact of pain on physical function and neuropathic pain characteristics in the months and years following the CD. The study also aims to explore the relationship between persistent and acute pain.

开始日期2023-09-01

申办/合作机构-

NL-OMON52697 / RecruitingN/A

Effectiveness of methylphenidate in children and adults with Smith Magenis syndrome and attention-deficit/hyperactivity disorder: An N-of-1 series - Methylphenidate for ADHD in Smith Magenis syndrome

开始日期2022-04-01

申办/合作机构-

100 项与 Smith-Magenis综合征相关的临床结果

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100 项与 Smith-Magenis综合征相关的转化医学

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0 项与 Smith-Magenis综合征相关的专利（医药）

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489

项与 Smith-Magenis综合征相关的文献（医药）

2024-02-22·American journal of medical genetics. Part B, Neuropsychiatric genetics : the official publication of the International Society of Psychiatric Genetics

Low-grade parental gonosomal mosaicism in CHD2 siblings with Smith-Magenis-like syndrome.

Article

作者: Francesca Cogliati ; Letizia Straniero ; Valeria Rimoldi ; Maura Masciadri ; Sara Perego ; Berardo Rinaldi ; Donatella Milani ; Davide Gentilini ; Lidia Larizza ; Rosanna Asselta ; Silvia Russo ; Maria Francesca Bedeschi

Loss-of-function CHD2 (chromodomain helicase DNA-binding protein 2) mutations are associated with a spectrum of neurodevelopmental disorders often including early-onset generalized seizures, photosensitivity, and epileptic encephalopathies. Patients show psychomotor delay/intellectual disability (ID), autistic features, and behavior disorders, such as aggression and impulsivity. Most reported cases are sporadic with description of germline mosaicism only in two families. We detect the first case of parental gonosomal CHD2 mosaicism disclosed by two brothers showing mild ID, born to healthy parents. The eldest brother has a history of drug-controlled generalized tonic-clonic seizures and displays sleep disorder and aggressive behavior suggestive of Smith-Magenis syndrome (SMS). Analysis of brothers' DNAs by next-generation sequencing (NGS) custom gene panel for pediatric epilepsy and/or ID disclosed in both the same pathogenic CHD2 variant. Additional NGS experiment on genomic DNA from parents' peripheral blood and from buccal swab raised the suspicion of low-grade gonosomal mosaicism in the unaffected mother subsequently confirmed by digital polymerase chain reaction (dPCR). This report underlines as worthwhile CHD2 screening in individuals presenting ID/developmental delay, with/without epilepsy, and behavior and sleep disorders suggestive of SMS. Detecting a CHD2 variant should prime testing probands' parents by NGS coupled to dPCR on different tissues to exclude/confirm gonosomal mosaicism and define the recurrence risk.

2024-02-22·Neurogenetics

Expanding the clinical and genetic landscape of (developmental) epileptic encephalopathy with spike-and-wave activation in sleep: results from studies of a Turkish cohort.

Article

作者: Ayberk Türkyılmaz ; Safiye Güneş Sağer ; Emine Tekin ; Kerem Teralı ; Hanife Düzkalır ; Metin Eser ; Yasemin Akın

The terms developmental epileptic encephalopathy with spike-and-wave activation in sleep (DEE-SWAS) and epileptic encephalopathy with spike-and-wave activation in sleep (EE-SWAS) designate a spectrum of conditions that are typified by different combinations of motor, cognitive, language, and behavioral regression linked to robust spike-and-wave activity during sleep. In this study, we aimed at describing the clinical and molecular findings in "(developmental) epileptic encephalopathy with spike-and-wave activation in sleep" (D)EE-SWAS) patients as well as at contributing to the genetic etiologic spectrum of (D)EE-SWAS. Single nucleotide polymorphism (SNP) array and whole-exome sequencing (WES) techniques were used to determine the underlying genetic etiologies. Of the 24 patients included in the study, 8 (33%) were female and 16 (67%) were male. The median age at onset of the first seizure was 4 years and the median age at diagnosis of (D)EE-SWAS was 5 years. Of the 24 cases included in the study, 13 were compatible with the clinical diagnosis of DEE-SWAS and 11 were compatible with the clinical diagnosis of EE-SWAS. Abnormal perinatal history was present in four cases (17%), and two cases (8%) had a family history of epilepsy. Approximately two-thirds (63%) of all patients had abnormalities detected on brain computerized tomography/magnetic resonance (CT/MR) imaging. After SNP array and WES analysis, the genetic etiology was revealed in 7 out of 24 (29%) cases. Three of the variants detected were novel (SLC12A5, DLG4, SLC9A6). This study revealed for the first time that Smith-Magenis syndrome, SCN8A-related DEE type 13 and SLC12A5 gene variation are involved in the genetic etiology of (D)EE-SWAS. (D)EE-SWAS is a genetically diverse disorder with underlying copy number variations and single-gene abnormalities. In the current investigation, rare novel variations in genes known to be related to (D)EE-SWAS and not previously reported genes to be related to (D)EE-SWAS were discovered, adding to the molecular genetic spectrum. Molecular etiology enables the patient and family to receive thorough and accurate genetic counseling as well as a personalized medicine approach.

2024-02-09·Pathophysiology : the official journal of the International Society for Pathophysiology

Rab11a Controls Cell Shape via C9orf72 Protein: Possible Relationships to Frontotemporal Dementia/Amyotrophic Lateral Sclerosis (FTDALS) Type 1.

Article

作者: Shoya Fukatsu ; Hinami Sashi ; Remina Shirai ; Norio Takagi ; Hiroaki Oizumi ; Masahiro Yamamoto ; Katsuya Ohbuchi ; Yuki Miyamoto ; Junji Yamauchi

Abnormal nucleotide insertions of C9orf72, which forms a complex with Smith-Magenis syndrome chromosomal region candidate gene 8 (SMCR8) protein and WD repeat-containing protein 41 (WDR41) protein, are associated with an autosomal-dominant neurodegenerative frontotemporal dementia and/or amyotrophic lateral sclerosis type 1 (FTDALS1). The differentially expressed in normal and neoplastic cells (DENN) domain-containing C9orf72 and its complex with SMCR8 and WDR41 function as a guanine-nucleotide exchange factor for Rab GTP/GDP-binding proteins (Rab GEF, also called Rab activator). Among Rab proteins serving as major effectors, there exists Rab11a. However, it remains to be established which Rab protein is related to promoting or sustaining neuronal morphogenesis or homeostasis. In this study, we describe that the knockdown of Rab11a decreases the expression levels of neuronal differentiation marker proteins, as well as the elongation of neurite-like processes, using N1E-115 cells, a well-utilized neuronal differentiation model. Similar results were obtained in primary cortical neurons. In contrast, the knockdown of Rab11b, a Rab11a homolog, did not significantly affect their cell morphological changes. It is of note that treatment with hesperetin, a citrus flavonoid (also known as Vitamin P), recovered the neuronal morphological phenotypes induced by Rab11a knockdown. Also, the knockdown of Rab11a or Rab11b led to a decrease in glial marker expression levels and in morphological changes in FBD-102b cells, which serve as the oligodendroglial differentiation model. Rab11a is specifically involved in the regulation of neuronal morphological differentiation. The knockdown effect mimicking the loss of function of C9orf72 is reversed by treatment with hesperetin. These findings may reveal a clue for identifying one of the potential molecular and cellular phenotypes underlying FTDALS1.

项与 Smith-Magenis综合征相关的新闻（医药）

2024-04-03

·Firstwordpharma

Vanda’s Fanapt clinches second FDA nod, this time for bipolar I disorder

The FDA cleared Vanda Pharmaceuticals’ Fanapt (iloperidone) for the acute treatment of manic or mixed episodes associated with bipolar I disorder in adults, marking the oral atypical antipsychotic’s second US approval following authorisation for schizophrenia in 2009."Manic or mixed episodes associated with bipolar I disorder are highly complex conditions, which require a host of trusted options to meet individual patient needs,” said Vanda CEO Mihael Polymeropoulos, adding that Fanapt is a “therapeutic agent that offers flexible dosing with a well-known safety profile.” The expanded label approval was based on findings from a Phase III study, in which 400 participants with bipolar I disorder experiencing a current episode of mania were randomly assigned to either Fanapt or placebo. At the four-week assessment, Fanapt demonstrated a significantly greater improvement than placebo on the primary endpoint of mania severity, as measured by the Young Mania Rating Scale (YMRS). Moreover, clinical benefits were evident in the Fanapt group as early as week two.Vanda noted that Fanapt’s safety profile was consistent with its previous studies in schizophrenia. The drug’s label carries a boxed warning highlighting an increased risk of death in elderly patients with dementia-related psychosis.Within its neuropsychiatric portfolio, Vanda is also looking to broaden the label for Hetlioz (tasimelteon), which was approved for non-24-hour sleep-wake disorder in 2014 and nighttime sleep disturbances associated with Smith-Magenis Syndrome in 2020. However, the melatonin receptor agonist was slapped with a complete response letter by the FDA for its label expansion in jet lag disorder in 2019 and more recently in insomnia.

上市批准临床3期临床结果

2024-04-02

·药融圈

FDA拒批9款药物 | 2024 Q1

▲5月30-31日第八届广州生物医药创新者峰会扫码立即报名注：本文不构成任何投资意见和建议，以官方/公司公告为准；本文仅作医疗健康相关药物介绍，非治疗方案推荐（若涉及），不代表平台立场。任何文章转载需得到授权。FDA是全世界最权威、审核及管理最严格的食品及药品管理机构，FDA审评的考量因素纷繁复杂，从FDA这里拿到获批上市的通行证门槛极高。2024年第一季度又有很多创新药被FDA拒之门外，小编整理如下。Regeneron：Odronextamab3月25日，再生元Regeneron宣布，FDA拒绝其CD3/CD20双抗Odronextamab治疗复发性或转移性滤泡淋巴瘤（FL）和弥漫大B细胞淋巴瘤的上市申请，再生元表示唯一原因是确证临床的入组状态问题，FDA在完整的回复函（CRL）中没有发现任何与有效性、安全性、试验设计、标签或制造有关的问题。这款CD3/CD20双抗已在复发/难治性滤泡性淋巴瘤以及复发／难治弥漫大B细胞淋巴瘤( DLBCL )中获得批准，均经过2线或2线以上的系统治疗。在12月的ASH上，Regeneron报告了DLBCL的2期数据，显示52%的反应率。如果上市成功，Odronextamab将是继基因泰克(Genentech)的Lunsumio和Genmab/艾伯维(AbbVie)的Epkinly之后，市场上第三种CD20xCD3双特异性抗体。Viatris/Mapi Pharma：GA Depot3月11日，晖致（Viatris）和Mapi Pharma共同宣布收到FDA就GA Depot（醋酸格拉替雷，40mg）用于治疗复发型多发性硬化症的新药申请（NDA）发出的完整回复函（CRL）。关于FDA拒绝的具体细节很少，这两家公司仅表示，他们目前正在审查完整回复函，以更好地确定GA Depot 40的“适当下一步”。GA Depot 40的活性成分醋酸格拉替雷是一种免疫调节剂，1996年在美国获批用于治疗多发性硬化症（MS），商品名为Copaxone。2023年6月，格拉替雷在中国获批上市。GA Depot的NDA主要是基于一项III期研究的积极结果。该研究评估了GA Depot对比安慰剂治疗RMS患者的有效性、安全性和耐受性。结果显示，GA Depot组患者的年复发率相比安慰剂组降低了30.1%（P=0.0066）。Venatorx/Melinta：cefepime-taniborbactam2月23日，Venatorx Pharmaceuticals和Melinta Therapeutics宣布，FDA发布了关于头孢吡肟-他尼波巴坦（cefepime-taniborbactam）新药申请(NDA)的完整回应函(CRL)，头孢吡肟-塔尼波巴坦是一种β-内酰胺/ β -内酰胺酶抑制剂(BL/BLI)联合抗生素，正在审查中，作为成人复杂尿路感染(cUTI)的潜在治疗方法，包括由敏感的革兰氏阴性微生物引起的急性肾盂肾炎。头孢吡肟-他尼波巴坦是一种实验性静脉注射(IV) β-内酰胺/ β-内酰胺酶抑制剂(BL/BLI)抗生素组合，用于治疗成人复杂尿路感染(cUTIs)，包括肾盂肾炎、医院获得性细菌性肺炎和呼吸机相关细菌性肺炎(HABP/VABP)。CRL没有发现NDA中的临床安全性或有效性问题，FDA也没有要求任何新的临床试验来支持头孢吡肟-他尼波巴坦的批准。FDA要求提供额外的化学、生产和控制(CMC)以及有关药物、测试方法和生产工艺的相关数据。Vanda Pharmaceuticals：Hetlioz3月6日，Vanda Pharmaceuticals宣布，已经收到FDA就Hetlioz（他司美琼）治疗失眠补充新药申请（sNDA）发出的完整回应函。FDA表示发现了标签和上市后要求/保证之外的缺陷，不能批准目前的sNDA。Vanda正在对其进行审查，并评估下一步行动。Hetlioz是一款褪黑素受体激动剂，2014年1月在美国获批治疗非24小时睡眠觉醒障碍。2020年12月，该产品拓展新适应症，获FDA批准治疗史密斯-马吉利综合征 (SMS) 夜间睡眠障碍。Minerva Neurosciences：Roluperidone2月27日，Minerva Neurosciences宣布收到FDA就Roluperidone用于治疗精神分裂症阴性症状的新药申请（NDA）发出的完整回复函（CRL），表示拒批其精神分裂症新药。受此消息影响，Minerva股价大跌59%。在CRL中，FDA 指出了以下四点拒批理由：虽然MIN-101C03研究在主要疗效终点上显示出统计学意义，但其本身不足以确立实质性的有效性证据；提交的NDA缺乏有关同时服用抗精神病药物的数据；NDA材料缺乏证明Roluperidone对改善精神分裂症阴性症状具有临床意义的必要数据；在已提交的安全性数据中，至少服用12个月拟议剂量（64mg）Roluperidone的患者数量不足。为了解决这些缺陷，FDA规定Minerva必须至少再提交一项积极、充分和对照良好的研究，以支持Roluperidone治疗阴性症状的安全性和有效性。Minerva还必须提供额外的数据来证明Roluperidone与其它抗精神病药物联合用药的安全性和有效性，以进一步支持Roluperidone的有效性并证明拟议剂量的长期安全性。总结尽管这些新药遭遇挫折，但FDA一次的拒批并不代表这些药物的研发不成功，FDA拒批的部分药物在欧洲或其他医药市场获批，比如安斯泰来Claudin18.2抗体Zolbetuximab在日本获批上市，默克公司口服、选择性的P2X3受体拮抗剂吉非匹生( gefapixant)在日本和欧盟获批上市。研发者也会吸取经验教训，会不断改进和优化药物的性能，以期在未来成功闯关FDA。参考资料：各公司官网往期文章：FDA拒批5款新药|1-2月汇总版权声明：本文转自生物药大时代，如不希望被转载的媒体或个人可与我们联系，我们将立即删除活动推荐 5月 • 第八届大湾区生物医药创新者峰会关键词：小分子，XDC，多肽及GLP-1热点话题，80+行业专家（点击下方图片查看详情）▼【关于药融圈】药融圈PRHub旨在帮助生物医药科技型企业进行品牌推广及商务拓展服务，针对客户的真实需求制定系统化解决方案，通过“翻译-降维-场景化”将客户的品牌信息以直白易懂的方式被公众知悉，同时在流量渠道覆盖100万+垂直用户基础上实现合作目的，帮助合作伙伴完成从品牌开始到商务为终的闭环营销服务。我们已经完成了数十场线下1000人规模的生物医药研发类会议，涵盖小分子新药，大分子新药，改良型新药，BD跨境交易等多个领域，服务了百余家上市/独角兽/生物技术/制药企业。

临床3期申请上市临床2期上市批准

2024-03-07

·药研发

【药研发0308】 BI银屑病改良制剂获批上市 | 苏州复融中美双报激动剂获批IND...

「本文共：16条资讯，阅读时长约：3分钟」今日头条BI银屑病改良制剂获批上市。勃林格殷格翰佩索利单抗（商品名：圣利卓，spesolimab）皮下注射制剂获国家药监局批准上市，这是该产品的全球首批，用于预防泛发性脓疱型银屑病（GPP）发作。spesolimab是一款靶向IL-36R抗体药物，其静脉注射液已在中国获批用于治疗GPP发作。在国际Ⅱ期EFFISAYIL研究中，与安慰剂相比，spesolimab皮下注射能显著降低GPP发作风险84%。国内药讯1.复宏汉霖实体瘤单抗东南亚上市。复宏汉霖宣布，该公司自主研发的曲妥珠单抗生物类似药汉曲优®分别获得泰国和菲律宾药监部门批准上市，用于HER2阳性乳腺癌和胃癌的治疗。2020年7月和8月，汉曲优先后获得欧盟委员会与中国药监局批准上市，为首个中国自主研发的中欧双批单抗药物。目前，汉曲优的美国上市许可申请已获得FDA受理，预计将于2024年获批。2.天境CD73单抗启动肺癌Ⅲ期临床。天境生物CD73抗体尤莱利单抗（TJD5，TJ004309）登记启动一项Ⅱ/Ⅲ期临床，在既往未接受过治疗的局部晚期且不可切除或转移性PD-L1和CD73选择性非小细胞肺癌患者中比较尤莱利单抗联合特瑞普利单抗、特瑞普利单抗单药与帕博利珠单抗单药治疗的效果。该项研究由广东省人民医院吴一龙教授牵头开展。在Ⅰb/Ⅱ期临床中，TJ004309联合特瑞普利单抗一线治疗达到31.3%的客观缓解率。3.CASI公司FIC抗体I期临床积极。CASI公司与BioInven公司联合开发的FcγRIIB单抗BI-1206与利妥昔单抗的组合方案，在中国评估用于治疗复发或难治性(R/R)惰性非霍奇金淋巴瘤(iNHL)的I期临床获积极结果。在8例可评估患者中，4例达到部分缓解(PR)， 1例达到完全缓解(CR)；其中1例复发性边缘区淋巴瘤（MZL）患者在达到CR后，缓解持续时间超过20周。此外，药物安全性良好。4.瑞顺现货通用型细胞疗法获批IND。广东瑞顺生物CD19-CAR-DNT细胞药“RJMty19注射液”获国家药监局批准开展新药研究，适应症为CD19阳性的成人复发/难治性B细胞急性淋巴细胞白血病。RJMty19是该公司基于人源化CD19-CAR全球专利开发的一款创新现货通用型第二代DNT细胞治疗产品。此前，NMPA已批准RJMty19两项IND申请，适应症分别为难治性系统性红斑狼疮，以及复发/难治性B细胞非霍奇金巴瘤。5.苏州复融中美双报激动剂获批IND。苏州复融生物自研1类生物药FL115注射液获国家药监局临床许可，即将开展用于治疗晚期/转移性实体瘤的新药研究。FL115是一款白介素15（IL-15）激动剂，已在临床前研究中显示出促进NK样细胞在肿瘤局部显著富集的潜力。去年5月，该新药已获FDA批准IND，目前正在美国开展针对晚期实体瘤的临床试验。6.恒瑞口服减肥药报新IND。恒瑞医药旗下盛迪医药1类化药HRS9531片的临床试验申请获CDE受理。HRS9531是一款靶向GLP-1和GIP双重受体激动剂，可通过同时激动GLP-1和GIP受体，发挥葡萄糖依赖性的促胰岛素分泌、抑制食欲和改善胰岛素敏感性等作用，帮助降低血糖和减轻体重。此前，HRS9531用于治疗2型糖尿病和减肥适应症已分别获得国家药监局临床批件。国际药讯1.长效HIV口服组合Ⅱ期临床积极。吉利德与默沙东联合开发的长效核苷逆转录酶易位抑制剂islatravir (ISL，MK-8591)与HIV衣壳抑制剂Lenacapavir（Sunlenca）组合方案，在治疗多药耐药HIV感染患者的Ⅱ期临床获积极结果。结果显示转换至每周一次islatravir和lenacapavir治疗或继续Biktarvy治疗的患者在第24周均保持较高的HIV抑制率（94.2%vs94.2%）；药物安全性良好。长期数据将在未来的科学会议上发布。2.Telios公司干眼症新药Ⅱ期临床积极。Telios Pharma公司布鲁顿氏酪氨酸激酶（BTK）抑制剂眼药水配方TL-925在治疗干眼症（DED）患者的Ⅱ期临床获积极数据。与溶剂对照相比，TL-925每天给药两次持续28天后，患者DED多种体征和症状显著改善，且药物耐受性良好。此外，在评估TL-925用于治疗过敏性结膜炎（AC）的另一项Ⅱ期临床中，TL-925也显示出显著改善AC相关瘙痒和发红的潜力。3.Vanda公司失眠新药上市失败。Vanda公司褪黑素受体激动剂Hetlioz（他司美琼）治疗失眠的补充新药申请（sNDA）收到FDA完整回应函。FDA表示发现了标签和上市后要求/保证之外的缺陷。褪黑素是由脑松果体分泌的激素之一，分泌具有明显的昼夜节律，白天分泌受抑制，晚上分泌活跃，具有改善睡眠作用。此前，FDA已批准该药物用于治疗非24小时睡眠觉醒障碍、史密斯-马吉利综合征 (SMS) 夜间睡眠障碍。4.Alumis公司融资推进银屑病新药开发。Alumis公司宣布完成2.59亿美元C轮融资，以用于推进其潜在“best-in-class”的酪氨酸激酶2（TYK2）别构抑制剂ESK-001的开发，开展评估用于治疗中重度斑块型银屑病患者的关键Ⅲ期临床，评估用于治疗系统性红斑狼疮（SLE）和非感染性葡萄膜炎患者的Ⅱ期临床，以及探索ESK-001在其他自身免疫性疾病中的潜在应用。此外，Alumis还将开展新型TYK2别构抑制剂A-005治疗神经炎症和神经退行性疾病的Ⅰ期临床。5.吉利德超15亿美元布局三抗肿瘤药。吉利德与Merus公司将利用后者专有的Triclonics平台，合作开发靶向新型肿瘤相关抗原（TAA）的三特异性抗体候选药物。根据协议，Merus将获得5600万美元预付款和2500万美元投资，以及高达15亿美元的开发和商业化里程碑潜在付款。Merus将负责两个项目的临床前研究，并可选择开展第三个项目。吉利德将拥有候选药物的选择权，将负责产品的临床开发和商业化。6.Sionna公司融资开发囊性纤维化小分子。Sionna公司宣布完成1.82亿美元C轮融资，以用于推进核苷酸结合域1（NBD1）稳定剂（SION-638、SION-451和SION-719）的临床开发，应用于CFTR基因突变导致囊性纤维化（CF）的治疗。临床前数据显示，NBD1稳定剂在与互补调节剂联合使用时，能恢复ΔF508-CFTR的成熟、运输和功能至野生型水平。目前，该公司的主打项目SION-63已在Ⅰ期临床中获得安全性结果以及初步疗效数据。医药热点1.人大代表建议将更多国产创新药纳入基药目录。全国人大代表、恒瑞医药董事长孙飘扬建议，在基药遴选过程中，对于独家药品，要优先考虑纳入创新价值高、临床必需、安全性优的创新药品。孙飘扬认为，推动紧密型县域医共体建设，将更多国产创新药纳入《国家基本药物目录》，对加强创新药在基层医疗机构的应用，提高基层防病治病和健康管理能力，具有十分重大的意义。2.天津社区卫生服务中心为老人免费查体。3月1日，天津市启动2024年老年人免费查体工作，全市268家社区卫生服务中心预计将为180万老年人进行免费查体。体检对象为辖区内60岁及以上常住居民(身份证出生日期为1964年12月31日前)。本次免费查体除常规项目外，还为65岁以上的老年人免费增加了中医体质辨识和腹部B超(肝胆胰脾)项目。3.德国男子私自接种217次新冠疫苗。国际医学期刊《柳叶刀·传染病》刊登的一项研究报告显示，1名在29个月内私自接种了217次新冠疫苗的德国男子从未感染过新冠病毒，也没有出现任何与疫苗相关的副作用。不过，研究人员称，单独案例不足以得出准确结论，没有证据表明接种更多疫苗对抵抗新冠病毒更有效，因此不建议将此作为预防手段。评审动态 1. CDE新药受理情况（03月07) 2. FDA新药获批情况（北美03月05日）股市资讯上个交易日 A 股医药板块 -1.79%涨幅前三跌幅前三锦波生物+5.07% 常山药业-7.85%景峰医药+4.64% 共同药业-7.41%冠福股份+3.62% 凯莱英-7.20%【复星医药】控股子公司星盛新辉新药XS-02胶囊获得用于治疗晚期实体瘤的临床试验批准。【复星医药】控股子公司精缮科技新药GCK-01细胞注射液获得用于治疗复发或化疗耐药的滤泡性淋巴瘤的临床试验批准。【药明康德】公司关于澄清事项的进一步公告，公司亦再次重申，药明康德既没有人类基因组学业务，公司现有各类业务也不会收集人类基因组数据。在未来数月中，S.3558草案将继续经历在参议院的立法过程。公司将与咨询顾问协力，继续与参与草案以及正在进行的美国众议院相应法案立法过程的相关方进行交流和对话，草案的内容仍有待进一步审议并可能变更。 - The End -戳“阅读原文”，了解更多医药研发及股市资讯。

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