An Open-label, Randomized, Three-arm Study Evaluating the Potential of BMS-984923 to Alter the Systemic Exposure of Three Orally Administered Probe Substrates in Healthy Adult Participants

The goal of this study is to learn if the investigational drug, BMS-984923 will impact the exposure and clearance of other medications when given together. Most drugs are broken down and cleared in the body via cytochrome P450 enzymes in the liver. The metabolism and clearance of certain drugs can be affected by other drugs when dosed together. To evaluate the impact of BMS-984923 on the clearance of other medications, we will investigate three known pathways which may be impacted by BMS-984923.
In this study participants will take one dose of the three known medications, midazolam, caffeine and dextromethorphan together. These drugs are known to be cleared by different cytochrome P450 enzymes. Blood will be collected to evaluate the exposure and clearance of these medications and their primary metabolites. Study participants will then be administered BMS-984923 for 18-days. On the 18th Day of BMS-984923 dosing, participants will again be co-administering midazolam, caffeine and dextromethorphan. Blood will be collected again, and the concentration of each drug will be measured. We will learn in this study if BMS-984923 changes the exposure and metabolism of the known drugs. These results will inform how BMS-984923 can be administered to patients who take multiple medications.

开始日期2024-11-01

申办/合作机构

Allyx Therapeutics, Inc.初创企业

[+1]

NCT06309147 / Recruiting临床1期

A Double-Blind, Single Center, Randomized, Placebo-Controlled Study of BMS-984923 in Participants With Parkinson's Disease

A Phase 1, randomized, double-blind, placebo-controlled study of BMS-984923 administered orally twice daily (BID) for 28 days in participants with Parkinson's disease.

开始日期2024-03-15

申办/合作机构

Allyx Therapeutics, Inc.初创企业

[+1]

NCT05804383 / Recruiting临床1期

A Phase 1B Multiple Ascending Dose Study of The Safety and Tolerability of BMS-984923 in Healthy Older Adults and Patients With Alzheimer's Disease

A Phase 1b Multiple Ascending Dose Study of the Safety and Tolerability of BMS-984923 in Healthy Older Adults and Patients with Alzheimer's Disease

开始日期2023-03-28

申办/合作机构

Allyx Therapeutics, Inc.初创企业

[+2]

100 项与 BMS-984923 相关的临床结果

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100 项与 BMS-984923 相关的转化医学

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100 项与 BMS-984923 相关的专利（医药）

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项与 BMS-984923 相关的文献（医药）

2024-10-18·Molecular psychiatry

Aging-dependent loss of functional connectivity in a mouse model of Alzheimer's disease and reversal by mGluR5 modulator.

Article

作者: Onofrey, John ; O'Connor, David ; Desrosiers-Grégoire, Gabriel ; Mandino, Francesca ; Owens, Ashley ; Lake, Evelyn M R ; Qu, An ; Chakravarty, M Mallar ; Mukherjee, Bandhan ; Shen, Xilin ; Strittmatter, Stephen M ; Papademetris, Xenophon

Amyloid accumulation in Alzheimer's disease (AD) is associated with synaptic damage and altered connectivity in brain networks. While measures of amyloid accumulation and biochemical changes in mouse models have utility for translational studies of certain therapeutics, preclinical analysis of altered brain connectivity using clinically relevant fMRI measures has not been well developed for agents intended to improve neural networks. Here, we conduct a longitudinal study in a double knock-in mouse model for AD (App^NL-G-F/hMapt), monitoring brain connectivity by means of resting-state fMRI. While the 4-month-old AD mice are indistinguishable from wild-type controls (WT), decreased connectivity in the default-mode network is significant for the AD mice relative to WT mice by 6 months of age and is pronounced by 9 months of age. In a second cohort of 20-month-old mice with persistent functional connectivity deficits for AD relative to WT, we assess the impact of two-months of oral treatment with a silent allosteric modulator of mGluR5 (BMS-984923/ALX001) known to rescue synaptic density. Functional connectivity deficits in the aged AD mice are reversed by the mGluR5-directed treatment. The longitudinal application of fMRI has enabled us to define the preclinical time trajectory of AD-related changes in functional connectivity, and to demonstrate a translatable metric for monitoring disease emergence, progression, and response to synapse-rescuing treatment.

2022-06-01·Science Translational Medicine1区 · 医学

Reversal of synapse loss in Alzheimer mouse models by targeting mGluR5 to prevent synaptic tagging by C1Q

1区 · 医学

Article

作者: Nicholson, LaShae ; Strittmatter, Stephen M. ; Siegert, Timothy R. ; Stoner, Austin J. ; Cai, Zhengxin ; Chiasseu, Marius ; Sadasivam, Pragalath ; Zhang, Le ; Spurrier, Joshua ; Takahashi, Hideyuki ; Nies, Sarah Helena ; Lee, Supum ; Pérez-Cañamás, Azucena ; Carson, Richard E. ; Huang, Yiyun H. ; Allnutt, Mary Alice ; Fang, Xiaotian T. ; Laird, William ; Toyonaga, Takuya ; Brody, A. Harrison ; Li, Songye ; Holden, Daniel

Microglia-mediated synaptic loss contributes to the development of cognitive impairments in Alzheimer’s disease (AD). However, the basis for this immune-mediated attack on synapses remains to be elucidated. Treatment with the metabotropic glutamate receptor 5 (mGluR5) silent allosteric modulator (SAM), BMS-984923, prevents β-amyloid oligomer–induced aberrant synaptic signaling while preserving physiological glutamate response. Here, we show that oral BMS-984923 effectively occupies brain mGluR5 sites visualized by [ 18 F]FPEB positron emission tomography (PET) at doses shown to be safe in rodents and nonhuman primates. In aged mouse models of AD ( APPswe/PS1 Δ E9 overexpressing transgenic and App NL-G-F / hMapt double knock-in), SAM treatment fully restored synaptic density as measured by [ 18 F]SynVesT-1 PET for SV2A and by histology, and the therapeutic benefit persisted after drug washout. Phospho-TAU accumulation in double knock-in mice was also reduced by SAM treatment. Single-nuclei transcriptomics demonstrated that SAM treatment in both models normalized expression patterns to a far greater extent in neurons than glia. Last, treatment prevented synaptic localization of the complement component C1Q and synaptic engulfment in AD mice. Thus, selective modulation of mGluR5 reversed neuronal gene expression changes to protect synapses from damage by microglial mediators in rodents.

2017-07-01·Cell Reports1区 · 生物学

Silent Allosteric Modulation of mGluR5 Maintains Glutamate Signaling while Rescuing Alzheimer’s Mouse Phenotypes

1区 · 生物学

ArticleOA

作者: Balakrishnan, Anand ; Smith, Levi M ; Herber, Charlotte S ; Macor, John E ; Albright, Charles F ; Degnan, Andrew P ; Salazar, Santiago V ; Haas, Laura T ; Cox, Timothy O ; Strittmatter, Stephen M ; Zhao, Helen R

Metabotropic glutamate receptor 5 (mGluR5) has been implicated in Alzheimer's disease (AD) pathology. We sought to understand whether mGluR5's role in AD requires glutamate signaling. We used a potent mGluR5 silent allosteric modulator (SAM, BMS-984923) to separate its well-known physiological role in glutamate signaling from a pathological role in mediating amyloid-β oligomer (Aβo) action. Binding of the SAM to mGluR5 does not change glutamate signaling but strongly reduces mGluR5 interaction with cellular prion protein (PrP^C) bound to Aβo. The SAM compound prevents Aβo-induced signal transduction in brain slices and in an AD transgenic mouse model, the APPswe/PS1ΔE9 strain. Critically, 4 weeks of SAM treatment rescues memory deficits and synaptic depletion in the APPswe/PS1ΔE9 transgenic mouse brain. Our data show that mGluR5's role in Aβo-dependent AD phenotypes is separate from its role in glutamate signaling and silent allosteric modulation of mGluR5 has promise as a disease-modifying AD intervention with a broad therapeutic window.

项与 BMS-984923 相关的新闻（医药）

2024-10-15

·GlobeNewswire-Health Care

Allyx Therapeutics Awarded $3.3 Million NIH Grant to Support Further Clinical Studies of Lead Candidate ALX-001

Funding will support metabolism studies of ALX-001, a first-in-class oral therapy in clinical development for Alzheimer’s disease and Parkinson’s diseaseNEW HAVEN, Conn., Oct. 15, 2024 (GLOBE NEWSWIRE) -- Allyx Therapeutics announced today that it has been awarded a $3.3 million grant from the National Institutes of Health as part of its Small Business Innovation Research Commercial Readiness Pilot program. This funding will support further investigation of Allyx’s lead clinical asset, ALX-001, a highly-selective, first-in-class, synapse-targeted, disease-modifying oral therapy in development for the treatment of Alzheimer’s disease and Parkinson’s disease. Allyx intends to utilize the Commercial Readiness Pilot funding to support a Phase I clinical pharmacokinetic drug-drug interaction study (NCT06632990) to determine the impact of multiple doses of ALX-001 on various cytochrome P450 enzymes. The first doses of ALX-001 were recently administered to people living with Alzheimer’s disease and Parkinson’s disease as part of two Phase Ib clinical studies (NCT05804383 and NCT06309147) currently underway in these patient populations. ALX-001 is ready to proceed to Phase 2 clinical development and is not restricted by completion of ongoing or planned studies. “We are honored to receive this grant from the National Institutes of Health Small Business Innovation Research Program as we continue to advance toward commercialization of ALX-001 as a potential first-in-class disease-modifying oral therapy in Alzheimer’s disease and Parkinson’s disease,” stated Tim Siegert, Co-Founder and COO at Allyx Therapeutics. “The metabolism study funded by this grant will help us to better understand the metabolic profile of ALX-001 which will be important for patient populations with a high likelihood of complex polypharmacy and comorbidities beyond neurodegenerative diseases.” With this latest grant, the ALX-001 program has been awarded more than $23 million in funding following previous grants from the National Institutes of Health, the U.S. Government’s highly competitive Small Business Innovation Research (SBIR) program, the Alzheimer’s Association and The Michael J. Fox Foundation for Parkinson’s Research, among others. About ALX-001 ALX-001 (previously BMS-984923) is a silent allosteric modulator of mGluR5, and is a first-in-class compound that selectively blocks the pathogenic activation of the receptor while preserving the normal physiological glutamate signaling that is required for cognition. As such, ALX-001 has a wide therapeutic window that can saturate receptors while avoiding on-target toxicity observed with negative allosteric modulators. mGluR5 has been shown to be essential for mediating synaptic dysfunction and loss caused by multiple misfolded extracellular protein species, and as such, presents a novel approach for treating Alzheimer's and Parkinson’s disease. Importantly, ALX-001 is an orally bioavailable and brain penetrant small molecule with demonstrated mGluR5 selective engagement. The molecule was originally identified by Bristol Myers Squibb, but the mechanism of action for neurodegenerative diseases and the identification of ALX-001 as disease-modifying for Alzheimer’s disease was discovered by Allyx scientific founder Stephen Strittmatter at Yale School of Medicine. Allyx Therapeutics obtained an exclusive worldwide license for ALX-001 from Bristol Myers Squibb and Yale School of Medicine. About Allyx Therapeutics Allyx Therapeutics was founded in 2019 by a group of seasoned biopharma industry executives, venture capitalists, and scientific experts. The company aims to deliver a novel approach to preserve and protect synapses for people living with neurodegenerative diseases. Its lead compound, ALX-001, is a first-in-class oral therapy with a unique mechanism of action at mGluR5 in clinical development for Alzheimer’s disease and Parkinson’s disease. Learn more at allyxthera.com. Contact Media: Eliza Schleifstein917-763-8106Eliza@schleifsteinpr.com Investors: Tim Siegert203-691-6543tsiegert@allyxthera.com

引进/卖出临床1期

2024-09-08

·药明康德

改善视力指标100倍的基因疗法；达完全缓解的CAR-T疗法…… | 一周盘点

▎药明康德内容团队编辑本期看点 1. 现货型同种异体嵌合抗原受体（CAR）-T细胞疗法azer-cel在一项1b期临床试验中使3名复发/难治性弥漫性大B细胞淋巴瘤（DLBCL）患者获得完全缓解（CR），CR率为33%。 2. 治疗因GUCY2D双等位基因突变引起的Leber先天性黑蒙（LCA1）患者的在研基因疗法ATSN-101的早期临床试验结果积极，高剂量组患者的一项视力指标改善了100倍。 3. 治疗杜氏肌营养不良（DMD）的在研抗体偶联寡核苷酸疗法DYNE-251在一项早期临床试验中使多个队列患者的抗肌萎缩蛋白表达达到前所未有的水平，且患者的临床功能获得改善。药明康德内容团队整理 Azer-cel：公布1b期临床试验数据 Imugene公司宣布，其现货型同种异体CD19靶向CAR-T细胞疗法azercabtagene zapreleucel（azer-cel）治疗复发/难治性弥漫性大B细胞淋巴瘤（一种非霍奇金淋巴瘤）的1b期临床试验取得了令人鼓舞的初步结果。该研究针对的是接受自体CAR-T细胞疗法治疗后复发的DLBCL患者，所有患者在试验前均接受过4-5线治疗。截至目前，已有10名患者接受了azer-cel治疗，A队列的6名患者接受了azer-cel+清淋化疗治疗，B队列的4名患者接受了azer-cel+清淋化疗+白介素-2（IL-2）治疗。研究结果显示，所有接受azer-cel和清淋化疗的患者都显示出可接受的安全性。此外，azer-cel+清淋化疗+IL-2的治疗在DLBCL患者中显示出了临床上有意义的疗效和持续性。在所有接受治疗的患者中，总缓解率为44%，其中3名患者达到了CR，1名患者达到了部分缓解（PR）。队列B中获得CR的两名患者已分别持续缓解超过90天和120天。目前，B队列的4名患者均仍在试验中，1名患者在等待评估。 ▲Azer-cel的1b期研究结果（图片来源：参考资料[1]）除了研究azer-cel在血液癌症中的疗效外，Imugene公司还计划将azer-cel与其新颖的onCARlytics结合，用于治疗实体肿瘤患者。 ATSN-101：公布1/2期临床试验数据 Atsena Therapeutics公司公布了其在研基因疗法ATSN-101治疗因GUCY2D双等位基因突变引起的Leber先天性黑蒙患者的1/2期临床试验数据。ATSN-101是一种以腺相关病毒5（AAV5）为载体的基因治疗药物，通过视网膜下注射将功能性的人类GUCY2D基因引入光感受器。ATSN-101已获得美国FDA授予的罕见儿科疾病认定、再生医学先进疗法认定和孤儿药资格，用于治疗GUCY2D相关LCA1。该研究中，15名经过确认的LCA1患者接受了单侧视网膜下注射，以确定ATSN-101的安全性和初步有效性。结果显示，在高剂量组中，暗适应全视场刺激测试（FST）的平均变化为20.3 dB，这意味着接受治疗眼睛的暗适应FST改善了100倍。这种改善在第28天时首次被观察到，并持续了12个月（p=0.012）。在最佳矫正视力（BCVA）方面也观察到轻微的改善，治疗后第12个月时高剂量组的平均改善为0.16 logMAR，或相当于视力表上的8个字母（p=0.10）。安全性方面，所有不良事件的严重程度均为轻度（91%）或中度（9%）。没有报告与药物相关的严重不良事件，且大多数治疗伴发不良事件与手术过程相关。眼部炎症为轻度，并可通过类固醇治疗逆转。 DYNE-251：公布1/2期临床试验的新数据 Dyne Therapeutics公司公布了其进行中的1/2期试验DELIVER的最新临床数据。该试验旨在评估其在研抗体偶联寡核苷酸疗法DYNE-251在适合进行外显子51跳跃治疗的杜氏肌营养不良患者中的疗效与安全性。DYNE-251由磷酸二氨基甲酰吗啉寡核苷酸（PMO）与靶向转铁蛋白受体1（TfR1）的抗体片段（Fab）偶联而成，TfR1在肌肉组织中高度表达。该疗法旨在实现靶向肌肉组织递送，促进核内外显子跳跃，从而使肌肉细胞生成截短但功能正常的抗肌萎缩蛋白，目标是停止或逆转疾病的进展。此次公布的结果显示，在接受DYNE-251治疗后，多个队列患者的抗肌萎缩蛋白表达达到前所未有的水平，且患者的临床功能获得改善。接受20 mg/kg剂量治疗患者的平均绝对抗肌萎缩蛋白表达为正常值的3.71%（未根据肌肉含量进行调整），比接受每周一次标准治疗的临床试验中所报告的0.3%高出10倍以上。接受20 mg/kg和10 mg/kg剂量治疗的患者在多个临床功能终点均有显著改善，包括北极星步行评估（NSAA）、步速95百分位数（SV95C）、10米步行/跑步时间（10-MWR）、从地面起身的时间。在试验6个月到12个月期间，10 mg/kg组患者在所有的指标中均显示出持续改善。基于这些积极数据和与监管单位讨论的结果，Dyne正在准备启动DELIVER试验中的注册性队列。 VAX-31：公布1/2期临床试验数据 Vaxcyte公司宣布，其候选31价肺炎球菌结合疫苗VAX-31在一项针对1015名50岁及以上健康成人的1/2期临床试验中取得了积极的顶线结果。基于这一积极结果，该公司已决定将VAX-31推进至成人3期临床试验。在这项1/2期研究中，VAX-31在所有研究剂量中均显示出良好的耐受性，并在整个六个月的评估期间展现了与获批20价肺炎球菌结合疫苗相似的安全性。VAX-31在所有研究剂量中对所有31种血清型均表现出强劲的调理吞噬作用（OPA）免疫反应。在中剂量和高剂量下，VAX-31与获批疫苗相比，针对两种疫苗共享的20种血清型，均达到或超过了OPA反应非劣效性标准。高剂量的VAX-31与获批20价疫苗相比，20种血清型中18种的平均OPA免疫反应更强，其中7种血清型的免疫反应显著高于活性对照。对于VAX-31独有的11种血清型，所有三种剂量与获批20价疫苗相比均达到了优效性标准。 JNT-517：公布1/2期临床试验的新数据 Jnana Therapeutics公司公布了其在研疗法JNT-517治疗苯丙酮尿症（PKU）成人患者的1/2期临床试验中的第二剂量组（150 mg，每日两次）的试验结果。JNT-517是一种针对苯丙氨酸（Phe）转运蛋白SLC6A19的小分子抑制剂，为潜在“first-in-class”口服疗法，适用于所有年龄段和基因型的PKU患者。此次公布的150 mg组数据基于18名受试者，其中11人服用JNT-517，7人服用安慰剂，历时28天。结果显示，JNT-517在第14、21、28天将患者平均血液Phe水平较基线降低了60%（p<0.0001）。相比之下，之前研究的75 mg剂量组在相同时间段内的平均血液Phe水平降低了44%。患者在用药开始后7天内观察到快速的疗效，血液Phe水平显著降低，并在28天的用药期间持续维持。无论基线Phe水平或之前的PKU治疗史如何，所有参与者均表现出显著的疗效。安全性方面，JNT-517耐受性良好，无严重不良事件，无临床显著的实验室参数变化，除Phe外，未观察到血液氨基酸的临床显著变化，与75 mg剂量组和1a期健康志愿者研究的安全性一致。基于这些积极结果，该公司计划在2025年初启动关键性3期临床试验，评估JNT-517治疗PKU的效果。 BX004：公布1b/2a期临床试验的新数据 BiomX公司公布了其用于治疗铜绿假单胞菌（PsA）引起慢性肺部感染的囊性纤维化（CF）患者的固定多噬菌体组合疗法BX004的1b/2a期研究的新数据。PsA是引起CF患者肺部感染和死亡的主要因素。BX004旨在靶向和破坏PsA。此次公布的结果显示，在研究的第一部分，接受BX004-A鸡尾酒疗法的受试者在第15天时，其痰液中的PsA菌落形成单位（CFU）/g的减少幅度较基线相比更大，而安慰剂组没有这种效果。在试验的第二部分，14%的BX004-A治疗组受试者在第10天（治疗结束）时痰液中的PsA培养结果为阴性，而安慰剂组的这一比例为0%。此外，在持续使用相同吸入抗生素且未更换或交替使用抗生素、正在接受elexacaftor/tezacaftor/ivacaftor（ETI）标准治疗、1秒用力呼气量（FEV1）＜70%的肺功能较差的患者亚组中，BX004-A治疗组患者的肺功能（以FEV1衡量）增加了5.66%，而安慰剂组则下降了3.23%。此外，所有接受BX004-A治疗的受试者痰液中均检测到了噬菌体，治疗过程中未发现与治疗相关的噬菌体耐药性，也没有严重的不良事件。 APTN-101：IND申请获得FDA许可 Adaptin Bio公司宣布其主打项目APTN-101的IND申请已获美国FDA批准，该公司即将推动该候选药物的“first-in-human”临床1期试验，以评估其用于治疗胶质母细胞瘤（GBM）的疗效与安全性。APTN-101是该公司专有的BRiTE（大脑双特异性T细胞接合剂）疗法，设计靶向EGFRvIII分子，这是一种与侵袭性脑肿瘤相关的蛋白。在临床前研究中，APTN-101具有超过对照药物7倍的大脑分布增强的效果，并在消除恶性胶质瘤肿瘤方面表现强劲，具有成为“best-in-class”药物的潜力。 MDG1015：IND申请获得FDA许可 Medigene公司宣布，其主打项目MDG1015的IND申请已获得美国FDA的批准，可启动1期临床试验，针对晚期胃癌、卵巢癌、粘液样/圆细胞脂肪肉瘤和滑膜肉瘤患者。MDG1015是一种潜在“first-in-class”的第三代T细胞受体（TCR）工程化T细胞疗法，靶向NY-ESO-1/LAGE-1a，其TCR能够特异性识别靶点，同时具有较高的安全性和敏感性。这些TCR-T细胞通过添加专有的PD1-41BB共刺激开关蛋白（CSP）技术得到进一步增强，对表达不同水平PD-L1的肿瘤细胞均具有显著增强的抗肿瘤活性。与第一代TCR-T疗法相比，MDG1015的生产过程更快，只需要6天就可以扩增出治疗所需的细胞。这些细胞更年轻、更健康，在治疗时需要的细胞数量可能更少。整个治疗过程从采集患者的细胞到输注治疗的时间缩短到大约20天。此外，生成的MDG1015细胞几乎都是CD8+细胞，其中大约95%的细胞都具有类似干细胞的特质，这意味着它们可以在体内存活更长时间，提供更持续的治疗效果，同时可能减少不良事件。 OpCT-001：IND申请获得FDA许可 BlueRock Therapeutics公司宣布OpCT-001的IND申请已获得美国FDA的批准。OpCT-001是一种在研诱导多能干细胞（iPSC）衍生的细胞疗法，旨在治疗原发性光感受器疾病。原发性光感受器疾病是遗传性视网膜病变的一个亚组，影响视网膜中光感受器细胞的结构和功能，导致儿童和成人视力的不可逆丧失。新闻稿指出，OpCT-001是首个针对原发性光感受器疾病进行临床测试的iPSC衍生细胞疗法。 LA-GLA：IND申请获得FDA许可 Hanmi Pharmaceutical公司宣布，其用于治疗法布里病的候选药物LA-GLA的IND申请已获得美国FDA的批准。目前，大多数法布里病患者通过使用基因重组技术开发的酶替代疗法进行治疗，患者每两周需要到医院接受长时间的静脉注射。LA-GLA是一种每月一次皮下注射的下一代长效酶替代疗法，使用更具便利性。在非临床研究中，LA-GLA在改善肾功能、血管疾病和外周神经障碍方面显示出优于现有治疗药物的效果。基于这些成果，该药物于2024年5月获得了美国FDA授予的孤儿药资格。 ▲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（可上下滑动查看） [1] Three Complete Responses in Azer-Cel Allogeneic CD19 CAR T Phase 1b Trial in Blood Cancer (Diffuse Large B-Cell Lymphoma). Retrieved September 6, 2024, from https://app.sharelinktechnologies.com/announcement/asx/9ec9d95529ab3adb318296ee5b344164 [2] Cartesian Therapeutics Announces First Patient Dosed in First-In-Human Phase 1 Trial of Next-Generation mRNA CAR-T Cell Therapy Descartes-15. Retrieved September 6, 2024, from https://ir.cartesiantherapeutics.com/news-releases/news-release-details/cartesian-therapeutics-announces-first-patient-dosed-first-human [3] BlueRock Therapeutics announces FDA clearance of IND application for investigational iPSC-derived cell therapy OpCT-001 for the treatment of primary photoreceptor diseases. Retrieved September 6, 2024, from https://www.globenewswire.com/news-release/2024/09/03/2939564/0/en/BlueRock-Therapeutics-announces-FDA-clearance-of-IND-application-for-investigational-iPSC-derived-cell-therapy-OpCT-001-for-the-treatment-of-primary-photoreceptor-diseases.html [4] GC녹십자-한미, ‘파브리병 치료제’ 1/2상 “美 IND 승인” . Retrieved September 6, 2024, from GC녹십자-한미, ‘파브리병 치료제’ 1/2상 “美 IND 승인” [5] Dyne Therapeutics Announces New Clinical Data from Phase 1/2 DELIVER Trial of DYNE-251 in Duchenne Muscular Dystrophy Demonstrating Unprecedented Dystrophin Expression and Functional Improvement in Multiple Cohorts. Retrieved September 3, 2024 from https://investors.dyne-tx.com/news-releases/news-release-details/dyne-therapeutics-announces-new-clinical-data-phase-12-deliver [6] BiomX to Present Positive Phase 1b/2a Clinical Trial Data for BX004 at the European Respiratory Society (ERS) Congress 2024. Retrieved September 3, 2024 from https://ir.biomx.com/news-events/press-releases/detail/120/biomx-to-present-positive-phase-1b2a-clinical-trial-data [7] Vaxcyte Reports Positive Topline Data from Phase 1/2 Study of VAX-31, its 31-Valent Pneumococcal Conjugate Vaccine Candidate, in Adults Aged 50 and Older. Retrieved September 3, 2024 from https://www.globenewswire.com/news-release/2024/09/03/2939548/0/en/Vaxcyte-Reports-Positive-Topline-Data-from-Phase-1-2-Study-of-VAX-31-its-31-Valent-Pneumococcal-Conjugate-Vaccine-Candidate-in-Adults-Aged-50-and-Older.html [8] Jnana Therapeutics Presents New Positive Data from Phase 1/2 Study of JNT-517 in Individuals with Phenylketonuria – Results Support Planned Pivotal Study in Early 2025. Retrieved September 4, 2024, from https://www.globenewswire.com/news-release/2024/09/04/2940409/0/en/Jnana-Therapeutics-Presents-New-Positive-Data-from-Phase-1-2-Study-of-JNT-517-in-Individuals-with-Phenylketonuria-Results-Support-Planned-Pivotal-Study-in-Early-2025.html [9] 7 Hills Pharma Safely Doses First Patient in Phase 1b/2a Clinical Trial Testing Alintegimod Against aPD-1-resistant Solid Tumors. Retrieved September 3, 2024 from https://www.globenewswire.com/news-release/2024/09/03/2940018/0/en/7-Hills-Pharma-Safely-Doses-First-Patient-in-Phase-1b-2a-Clinical-Trial-Testing-Alintegimod-Against-aPD-1-resistant-Solid-Tumors.html [10] Allyx Therapeutics Announces First Parkinson’s Disease Patient Treated with ALX-001. Retrieved September 3, 2024 from https://www.globenewswire.com/news-release/2024/09/04/2940463/0/en/Allyx-Therapeutics-Announces-First-Parkinson-s-Disease-Patient-Treated-with-ALX-001.html#:~:text=NEW%20HAVEN%2C%20Conn.%2C%20Sept.%2004%2C%202024%20%28GLOBE%20NEWSWIRE%29,potential%20therapeutic%20response%20in%20patients%20with%20Parkinson%E2%80%99s%20disease. [11] Boehringer Ingelheim announces plans to advance potential new treatment for Geographic Atrophy, following positive Phase I results. Retrieved September 6, 2024 from https://www.globenewswire.com/news-release/2024/09/05/2941540/0/en/Boehringer-Ingelheim-announces-plans-to-advance-potential-new-treatment-for-Geographic-Atrophy-following-positive-Phase-I-results.html [12] Atsena Therapeutics Announces 12-Month Safety and Efficacy Data from Phase I/II Clinical Trial of ATSN-101 in LCA1 Published in The Lancet. Retrieved September 6, 2024 from https://www.globenewswire.com/news-release/2024/09/05/2941896/0/en/Atsena-Therapeutics-Announces-12-Month-Safety-and-Efficacy-Data-from-Phase-I-II-Clinical-Trial-of-ATSN-101-in-LCA1-Published-in-The-Lancet.html [13] Medigene Announces U.S. FDA Clearance of IND Application for MDG1015, a First-in-Class, Third Generation T Cell Receptor Engineered T Cell (TCR-T) Therapy for Multiple Solid Tumor Indications. Retrieved September 6, 2024 from https://www.globenewswire.com/news-release/2024/09/05/2941272/0/en/Medigene-Announces-U-S-FDA-Clearance-of-IND-Application-for-MDG1015-a-First-in-Class-Third-Generation-T-Cell-Receptor-Engineered-T-Cell-TCR-T-Therapy-for-Multiple-Solid-Tumor-Indic.html [14] ALX Oncology Announces First Patients Dosed with Evorpacept and Sarclisa As Part of Randomized Phase 1/2 UMBRELLA Study with Sanofi. Retrieved September 6, 2024 from https://ir.alxoncology.com/news-releases/news-release-details/alx-oncology-announces-first-patients-dosed-evorpacept-and [15] OncoC4 Announces First Patient Dosed in Phase 1 Trial for Novel SIGLEC10 Immune Checkpoint Inhibitor ONC-841 for Solid Tumors. Retrieved September 6, 2024 from https://www.oncoc4.com/index.php/blog/news-releases/item/23-oncoc4-announces-first-patient-dosed-in-phase-1-trial-for-novel-siglec10-immune-checkpoint-inhibitor-onc-841-for-solid-tumors [16] Adaptin Bio Announces FDA Clearance of IND Application for APTN-101 in Glioblastoma. Retrieved September 5, 2024 from https://www.businesswire.com/news/home/20240905978578/en/Adaptin-Bio-Announces-FDA-Clearance-of-IND-Application-for-APTN-101-in-Glioblastoma 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

细胞疗法基因疗法临床结果孤儿药免疫疗法

2024-09-04

·GlobeNewswire-Health Care

Allyx Therapeutics Announces First Parkinson’s Disease Patient Treated with ALX-001

Allyx continues to advance clinical research of its first-in-class oral compound for neurodegenerative diseasesNEW HAVEN, Conn., Sept. 04, 2024 (GLOBE NEWSWIRE) -- Allyx Therapeutics has announced that the first patient has been dosed with its lead compound, ALX-001, in a new clinical study assessing safety, pharmacokinetics and potential therapeutic response in patients with Parkinson’s disease. ALX-001 is a highly selective, first-in-class, synapse-targeted, disease-modifying oral therapy. Allyx Therapeutics is a clinical-stage biotechnology company working to deliver a novel approach to preserve and protect synapses for people living with neurodegenerative diseases. “Allyx Therapeutics is moving forward with strong momentum to develop ALX-001 as the first-ever disease-modifying small molecule for neurodegenerative diseases, with two concurrent safety studies in patients with Parkinson’s disease and with Alzheimer’s disease, adding important information to the body of knowledge for this novel therapeutic approach,” said Tim Siegert, Ph.D. chief operating officer and co-founder of Allyx Therapeutics. The 28-day Parkinson’s disease study (NCT06309147) is assessing the safety of ALX-001 dosed twice daily at either 50 mg or 100 mg versus placebo in adults between 21 and 80 years of age, and will also investigate dopamine transporter levels in the brain measured with single photon emission computed tomography as an early marker of therapeutic response to a treatment that targets synapse restoration. The study is being conducted by the Duke Clinical Research Institute (DCRI) and is supported with grant funding awarded to Allyx Therapeutics from The Michael J. Fox Foundation for Parkinson’s Research. “I’m excited to collaborate with the Allyx team as we advance understanding of how Parkinson’s disease affects the brain and what more we might be able to do to provide a real impact for patients. Investigating the safety of this disease-modifying small molecule is a crucial first step, and one I’m proud to be a part of,” said Laurie H. Sanders, Ph.D., associate professor of Neurology at Duke University School of Medicine and DCRI. Building on twelve years of clinical research, ALX-001 continues to demonstrate promise in ongoing studies. The ALX-001 program has received more than $20 million in grant funding from the National Institutes of Health, the U.S. Government’s highly competitive Small Business Innovation Research (SBIR) programs, the Alzheimer’s Association, and The Michael J. Fox Foundation for Parkinson’s Research, among others. About ALX-001 ALX-001 (previously BMS-984923) is a silent allosteric modulator of mGluR5, and is a first-in-class compound that selectively blocks the pathogenic activation of the receptor while preserving the normal physiological glutamate signaling that is required for cognition. As such, ALX-001 has a wide therapeutic window that can saturate receptors while avoiding on-target toxicity observed with negative allosteric modulators. mGluR5 has been shown to be essential for mediating synaptic dysfunction and loss caused by multiple misfolded extracellular protein species, and as such, presents a novel approach for treating Alzheimer's and Parkinson’s disease. Importantly, ALX-001 is an orally bioavailable and brain penetrant small molecule with demonstrated mGluR5 selective engagement. The molecule was originally identified by Bristol Myers Squibb, but the mechanism of action for neurodegenerative diseases and the identification of ALX-001 as disease-modifying for Alzheimer’s disease was discovered by Allyx scientific founder Stephen Strittmatter at Yale School of Medicine. Allyx Therapeutics obtained an exclusive worldwide license for ALX-001 from Bristol Myers Squibb and Yale School of Medicine. About Allyx Therapeutics Allyx Therapeutics was founded in 2019 by a group of seasoned biopharma industry executives, venture capitalists, and scientific experts. The company aims to deliver a novel approach to preserve and protect synapses for people living with neurodegenerative diseases. Its lead compound, ALX-001, is a first-in-class oral therapy with a unique mechanism of action at mGluR5 in clinical development for Alzheimer’s disease and Parkinson’s disease. Learn more at allyxthera.com. About the Duke Clinical Research Institute The DCRI, part of the Duke University School of Medicine, is the largest academic clinical research organization in the world. Our mission is to develop, share, and implement knowledge that improves global health through innovative clinical research. The institute conducts multinational clinical trials, manages major national patient registries, and performs landmark outcomes research. The DCRI is a pioneer in cardiovascular and pediatric clinical research and conducts groundbreaking clinical research across multiple other therapeutic areas, including infectious disease, neuroscience, respiratory medicine, and nephrology. ContactMedia: Eliza Schleifstein917.763.8106Eliza@schleifsteinpr.com Investors: Tim Siegert203-691-6543tsiegert@allyxthera.com

引进/卖出临床研究

100 项与 BMS-984923 相关的药物交易

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适应症	最高研发状态	国家/地区	公司	日期
帕金森病	临床1期	美国	Allyx Therapeutics, Inc.初创企业	2024-03-15
阿尔茨海默症	临床前	美国	Bristol Myers Squibb Co.	2021-03-18

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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


NCT04805983 (CTgov)	临床1期	阿尔茨海默症	36	BMS-984923 (10 mg BMS-984923)	選襯襯願壓廠衊衊鏇築(鏇膚憲壓餘鏇廠夢艱鑰) = 淵窪蓋構網艱鏇蓋觸鹽獵鏇製壓獵範淵衊鑰製 (選遞選鏇糧憲艱顧獵夢, 製簾鏇積鏇淵餘鏇衊顧 ~ 鏇鹹壓齋壓構築衊齋鹹) 更多	-	2023-04-13
				BMS-984923 (40 mg BMS-984923)	選襯襯願壓廠衊衊鏇築(鏇膚憲壓餘鏇廠夢艱鑰) = 夢齋繭廠積範顧積淵鹹獵鏇製壓獵範淵衊鑰製 (選遞選鏇糧憲艱顧獵夢, 簾蓋鹽繭鏇觸遞餘鏇淵 ~ 觸膚鏇築憲觸艱積築餘) 更多