更新于：2024-06-30

Edasalonexent

更新于：2024-06-30

概要

研发状态

概要

基本信息

药物类型

小分子化药

别名

CAT-1004

靶点

NF-κB

作用机制

NF-κB抑制剂(核因子NF-κB复合体抑制剂)

治疗领域

神经系统疾病遗传病与畸形皮肤和肌肉骨骼疾病+ [2]

在研适应症-

非在研适应症

2型糖尿病杜氏肌营养不良症

原研机构

Astria Therapeutics, Inc.

在研机构-

非在研机构

Astria Therapeutics, Inc.

最高研发阶段终止临床3期

首次获批日期-

最高研发阶段(中国)-

特殊审评孤儿药 (美国)

结构

分子式C31H42N2O3

InChIKeyJQLBBYLGWHUHRW-KUBAVDMBSA-N

CAS号1204317-86-1

关联

项与 Edasalonexent 相关的临床试验

EUCTR2019-003563-22-IE / Not yet recruiting临床3期

An Open-Label Extension Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy

开始日期2020-06-23

申办/合作机构-

EUCTR2019-003563-22-DE / Not yet recruiting临床3期

An Open-Label Extension Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy

开始日期2020-04-16

申办/合作机构-

EUCTR2019-003563-22-SE / Not yet recruiting临床3期

An Open-Label Extension Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy

开始日期2020-04-15

申办/合作机构-

100 项与 Edasalonexent 相关的临床结果

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100 项与 Edasalonexent 相关的转化医学

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100 项与 Edasalonexent 相关的专利（医药）

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项与 Edasalonexent 相关的文献（医药）

2021-09-14·Journal of neuromuscular diseases

A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy: Results of the PolarisDMD Trial

Article

作者: Finanger, Erika ; Burnette, W. Bryan ; Nance, Jessica ; Müller-Felber, Wolfgang ; O’Rourke, Declan ; Mancini, Maria ; Ryan, Monique M. ; Mathews, Katherine D. ; Statland, Jeffrey ; Neil Knierbein, Erin ; Johannsen, Jessika ; Wagner, Kathryn R. ; McDonald, Craig M. ; Tian, Cuixia ; Finkel, Richard S. ; Nguyen, Cam-Tu ; Donovan, Joanne M. ; Marks, Warren ; Eagle, Michelle ; Lee Sweeney, H. ; McCullagh, Gary ; Tulinius, Mar ; Vijayakumar, Kayal ; McMillan, Hugh J. ; Phan, Han C. ; MacDougall, James

Background: Edasalonexent (CAT-1004) is an orally-administered novel small molecule drug designed to inhibit NF-κB and potentially reduce inflammation and fibrosis to improve muscle function and thereby slow disease progression and muscle decline in Duchenne muscular dystrophy (DMD). Objective: This international, randomized 2 : 1, placebo-controlled, phase 3 study in patients ≥4 – < 8 years old with DMD due to any dystrophin mutation examined the effect of edasalonexent (100 mg/kg/day) compared to placebo over 52 weeks. Methods: Endpoints were changes in the North Star Ambulatory Assessment (NSAA; primary) and timed function tests (TFTs; secondary). Assessment of health-related function used the Pediatric Outcomes Data Collection tool (PODCI). Results: One hundred thirty one patients received edasalonexent (n = 88) and placebo (n = 43). At week 52, differences between edasalonexent and placebo for NSAA total score and TFTs were not statistically significant, although there were consistently less functional declines in the edasalonexent group. A pre-specified analysis by age demonstrated that younger patients (≤6.0 years) showed more robust and statistically significant differences between edasalonexent and placebo for some assessments. Treatment was well-tolerated and the majority of adverse events were mild, and most commonly involved the gastrointestinal system (primarily diarrhea). Conclusions: Edasalonexent was generally well-tolerated with a manageable safety profile at the dose of 100 mg/kg/day. Although edasalonexent did not achieve statistical significance for improvement in primary and secondary functional endpoints for assessment of DMD, subgroup analysis suggested that edasalonexent may slow disease progression if initiated before 6 years of age. (NCT03703882)

2021-05-01·Neuromuscular disorders : NMD4区 · 医学

Disease-modifying effects of edasalonexent, an NF-κB inhibitor, in young boys with Duchenne muscular dystrophy: Results of the MoveDMD phase 2 and open label extension trial

4区 · 医学

Article

作者: Finanger, Erika ; Rooney, William D ; Walter, Glenn ; Finkel, Richard S ; Triplett, William T ; Sweeney, H Lee ; Mancini, Maria ; Bista, Pradeep ; Forbes, Sean C ; Vandenborne, Krista ; Yum, Sabrina W ; Nichols, Andrew ; Willcocks, Rebecca ; Fretzen, Angelika ; Tennekoon, Gihan ; Donovan, Joanne M ; Shieh, Perry B ; MacDougall, James

Chronic activation of NF-κB is a key driver of muscle degeneration and suppression of muscle regeneration in Duchenne muscular dystrophy. Edasalonexent (CAT-1004) is an orally-administered novel small molecule that covalently links two bioactive compounds (salicylic acid and docosahexaenoic acid) that inhibit NF-κB. This placebo-controlled, proof-of-concept phase 2 study with open-label extension in boys ≥4-<8 years old with any dystrophin mutation examined the effect of edasalonexent (67 or 100 mg/kg/day) compared to placebo or off-treatment control. Endpoints were safety/tolerability, change from baseline in MRI T₂ relaxation time of lower leg muscles and functional assessment, as well as pharmacodynamics and biomarkers. Treatment was well-tolerated and the majority of adverse events were mild, and most commonly of the gastrointestinal system (primarily diarrhea). There were no serious adverse events in the edasalonexent groups. Edasalonexent 100 mg/kg was associated with slowing of disease progression and preservation of muscle function compared to an off-treatment control period, with decrease in levels of NF-κB-regulated genes and improvements in biomarkers of muscle health and inflammation. These results support investigating edasalonexent in future trials and have informed the design of the edasalonexent phase 3 clinical trial in boys with Duchenne.

2019-01-30·Journal of neuromuscular diseases

Phase 1 Study of Edasalonexent (CAT-1004), an Oral NF-κB Inhibitor, in Pediatric Patients with Duchenne Muscular Dystrophy

Article

作者: Vandenborne, Krista ; Yum, Sabrina ; Finkel, Richard S. ; Mancini, Maria ; Fretzen, Angelika ; Donovan, Joanne M. ; Liu, Hanlan ; Tennekoon, Gihan ; Nichols, Andrew ; Lee Sweeney, H. ; Bista, Pradeep ; Finanger, Erika

BACKGROUND:

Edasalonexent is an orally administered small molecule designed to inhibit NF-κB, which is activated from infancy in Duchenne muscular dystrophy and is central to causing muscle damage and preventing muscle regeneration.

OBJECTIVE:

Evaluate the safety, tolerability, pharmacokinetics and exploratory pharmacodynamics of three doses of edasalonexent in ambulatory males ≥4 to <8 years of age with genetically confirmed Duchenne muscular dystrophy.

METHODS:

This was a 1-week, open-label, multiple-dose study with 3 sequential ascending doses (33, 67 and 100 mg/kg/day) of edasalonexent administered under different dietary conditions to 17 males with a mean age of 5.5 years.

RESULTS:

All doses of edasalonexent were well tolerated, with no serious adverse events, no drug discontinuations and no dose reductions. The majority of adverse events were mild, and the most common adverse events were gastrointestinal (primarily diarrhea). Edasalonexent was rapidly absorbed with peak levels observed 2-6 hours after dosing and exposures appeared to increase nearly proportionally to dose for the 2 lower and all 3 doses under low-fat and high-fat meal conditions, respectively. Only minor plasma accumulation of edasalonexent was observed with 7 days of dosing. After treatment with edasalonexent for 7 days, levels of NF-κB-regulated genes and serum proteins were decreased.

CONCLUSIONS:

This first report of edasalonexent oral administration for one week in male pediatric patients with Duchenne muscular dystrophy showed that treatment was well tolerated and inhibited NF-kB pathways.

项与 Edasalonexent 相关的新闻（医药）

2024-03-25

·FierceBiotech

Astria's pipeline star reduces hereditary angioedema attacks in early phase 1b/2 data

Patients receiving STAR-0215 once or twice over six months saw monthly HAE attack rates decline by 90% to 96%, according to Astria. Astria Therapeutics is planning a phase 3 launch for its star monoclonal antibody after early data demonstrated a favorable safety profile and reduction in attack rates of a rare immune disorder. The initial phase 1b/2 results, shared Monday morning, assessed Astria’s STAR-0215, a monoclonal antibody inhibitor of plasma kallikrein, among 16 patients with hereditary angioedema (HAE). The trial, dubbed ALPHA-STAR, is measuring the number of adverse events reported, along with other secondary endpoints like duration and severity of HAE attack. The initial data found STAR-0215 to be generally well tolerated with no serious treatment-emergent adverse events (TEAEs) occurring. No discontinuations were recorded and the two TEAEs reported were both mild: one case of dizziness and one rash at injection site. The study includes three dosing cohorts. Participants in the first arm received one 450-mg dose of STAR-0215, while participants in the second group received a 600-mg dose followed by a 300-mg dose three months later, and patients in the final cohort were given a 600-mg dose and then another 600-mg dose a month later. When evaluating monthly HAE attack rate, the first cohort saw a 92% reduction at a six month follow-up; group two recorded a 96% reduction at 84 days; and cohort three demonstrated a 90% decrease at Day 28. Astria said these findings support chronic dosing two or four times per year. The phase 1b/2 trial has a primary completion date of September 2025, according to ClinicalTrials.gov. “The initial results of the ALPHA-STAR trial represent a very exciting step forward in the HAE treatment landscape,” Marcus Maurer, M.D., executive director of the Institute of Allergology at the Universitatsmedizin Berlin, said in a March 25 release. “STAR-0215 has the potential to help patients manage their disease with a mechanism and modality that they trust, but with a substantially improved dosing regimen and the ability to administer without pain,” Maurer added. “Based on this profile, STAR-0215 has the potential to normalize the lives of people living with HAE.” With the early results in hand, Astria intends to launch STAR-0215 into phase 3 development by the first quarter of 2025, pending discussions with regulatory agencies. To get STAR-0215 to market as fast as possible, Astria wants to focus the potential late-stage program on dosing every three months, immediately followed by a second trial to support label expansion with dosing every six months. Astrai has its origins in Catabasis Pharmaceuticals. When Catabasis' doomed Duchenne muscular dystrophy drug edasalonexent flamed out in phase 3, the company rebranded in 2021 and shifted focus to STAR-0215.

临床结果临床3期

2023-11-06

·药渡Daily

浅谈杜氏肌营养不良治疗进展

关键词杜氏肌营养不良；研究进展；Sarepta Therapeutics杜氏肌营养不良（DMD），又称假肥大型进行性肌营养不良，是临床上常见的进行性肌营养不良症。DMD是由于X染色体上编码抗肌萎缩蛋白的基因发生突变引起的遗传性疾病，以进行性肌肉无力为特征，患者初期多表现为走路不稳，易跌倒，最终可累及呼吸肌、心肌导致死亡。DMD主要影响男性，全球大约每3500-5000名新生男性中就有1人发病。Part 1已获批的DMD药物目前，DMD还无法治愈，治疗药物非常有限，DMD获批药物有八款，多为反义寡核苷酸药物。详见下表。全球已获批的DMD疗法资料来源：药渡数据库AtalurenAtaluren是一种蛋白质修复疗法，能够与核糖体相互作用，使核糖体能够通过mRNA上的过早无义停止信号，允许细胞产生全长的功能性蛋白质。2014年7月，该药在欧盟被批准用于治疗5岁及以上、非卧床、无义突变型DMD（nmDMD）患者。EmflazaEmflaza是皮质类固醇药物。Emflaza是FDA批准的首款用于治疗DMD的皮质类固醇类药物，通过减少炎症和降低免疫系统的活性起作用，2017年2月其片剂和口服混悬剂被批准用于治疗5岁及以上DMD，2019年又被FDA批准扩大用于2岁至5岁DMD儿童患者。然而，Emflaza存在一些与皮质类固醇相似的副作用，如面部浮肿、体重增加、食欲增强、上呼吸道感染、咳嗽、尿频、多毛症和向心性肥胖。AgamreeAgamree也是皮质类固醇药物，然而，与普通类固醇药物不同，可以有选择性地激活类固醇的某些信号通路，能够在保持类固醇对DMD疗效的同时减少副作用的产生。今年10月，该药被FDA批准用于治疗2岁及以上DMD患者。反义寡核苷酸药物Exondys 51、Viltepso、Vyondys 53、Amondys 45均是反义寡核苷酸药物，通过改变表达抗肌萎缩蛋白的mRNA的剪接过程，跳过让蛋白合成过早中断的基因变异发挥作用。其中Exondys 51于2016年9月被FDA批准用于治疗发生51外显子跳跃突变的DMD患者。Vyondys 53于2019年12月在美国被批准用于治疗携带53外显子跳跃突变的DMD患者。Viltepso于2020年5月在日本被批准用于治疗发生携带53外显子跳跃突变的DMD患者。Amondys 45于2021年2月在美国被批准用于治疗发生携带45外显子跳跃突变的DMD患者。Elevidys是全球批准的首款治疗DMD的基因疗法。该药是一款AAV基因疗法，通过将目的基因递送到肌肉组织，针对性地产生抗肌萎缩蛋白的有效成分，从而减缓或阻止肌肉退化。今年6月，Elevidys被FDA批准用于治疗4-5岁携带抗肌萎缩蛋白基因突变的非卧床儿科DMD患者。市场表现上，Exondys 51表现最好，2022年销售额高达511.75百万美元Amondys 45次之，2022年销售额达214.58亿美元。Vyondys 53 排名第三，2022年销售额为117.44亿美元。Part 2在研DMD新药DMD市场即将进入收获期，根据药渡数据调研，目前全球还有多款在研新药被开发用于治疗DMD，多款处于后期临床，详见下表。全球部分在研DMD新药资料来源：药渡数据库GivinostatGivinostat进展相对较快，目前正在美国接受优先审查，PDUFA日期为2023年12月21日。Givinostat是一种具有创新作用机制的口服组蛋白去乙酰化酶（HDAC）小分子抑制剂。DMD患者肌肉组织中HDAC活性异常升高，这种升高的HDAC活性可能促使肌肉再生受损，并妨碍肌肉超细纤维适应收缩的能力。通过抑制HDAC活性，Givinostat可以缓解DMD患者肌肉组织的损害，保护肌肉超细纤维的功能。3期临床在研药物CAP-1002、TAS-205、Pamrevlumab、Edasalonexent等几款药物处于3期临床。CAP-1002是一种用于治疗晚期DMD的同种异体细胞疗法，由心脏来源细胞（CDCs）组成，已在临床前和临床研究中显示出有效的免疫调节活性。在为期18个月的临床试验HOPE-2中，与安慰剂相比，CAP-1002受试者的上肢性能观察到明显差异。今年6月，Capricor Therapeutics宣布将在今年三季度与FDA召开Type-B会议，讨论CAP-1002的BLA计划以及当前的临床3期试验。Pamrevlumab是一款靶向结缔组织生长因子（CTGF）的新型抗体，被开发用于治疗特发性肺纤维化（IPF）、胰腺癌（LAPC）和DMD等，其中针对DMD适应症曾被FDA授予孤儿药资格认定、快速通道认定、罕见儿科疾病认定。遗憾的是，Pamrevlumab用于治疗非卧床DMD患者的3期LELANTOS-1在第52周没有达到上肢表现2.0（PUL 2.0）得分这一主要终点。PF-06939926是辉瑞从Bamboo Therapeutics引进的DMD基因疗法，由人肌肉特异性启动子驱动，利用rAAV9作为载体递送微型肌营养不良蛋白基因（rAAV9具有靶向定位肌肉组织的潜力）。2期临床在研药物在研DMD药物大多处于2期临床。Brogidirsen是一种反义核酸药物（ASO），今年7月被FDA授予罕见儿科疾病资格认定（RPDD），用于接受外显子44跳跃治疗的DMD。PGN-EDO51是基于PepGen增强递送寡核苷酸（EDO）技术平台开发的一种多肽-寡核苷酸偶联物，主要由EDO肽和寡核苷酸组成。其中EDO肽实现对寡核苷酸的有效递送，寡核苷酸通过跳跃外显子51来恢复开放阅读框并产生一个较小的功能性抗萎缩蛋白以治疗DMD。该药在健康志愿者中进行的单剂量递增1期临床试验达主要终点，安全性和耐受性良好。而且，在所有获批或已知在研DMD治疗药物中，PGN-EDO51的外显子跳跃水平最高，有助于产生更高水平的抗肌萎缩蛋白。RGX-202是一款使用AAV8载体的DMD基因治疗药物，递送一种含有天然肌营养不良蛋白C端功能部分的创新型微型肌营养不良蛋白转基因。相关临床前研究中显示，含有功能性C端的RGX-202可以将多种关键蛋白募集到肌肉细胞膜表面，从而在DMD小鼠模型中提高肌肉细胞对骨骼肌收缩活动诱导损伤的抗性。同时，通过密码子优化与CpG部分的减少，RGX-202还具备提高基因表达，提高翻译效率，降低免疫原性的能力。此外，RGX-202所使用的肌肉细胞特异性启动子Spc5-12在相关研究中也显示出了很好的效果。2023年5月，该药被FDA授予治疗DMD的快速通道资格认定。DYNE-251是针对携带51号外显子跳跃突变DMD患者开发的抗体偶联寡核苷酸药物，由磷酸二酰胺吗啉寡聚物（PMO）与Fab片段结合组成。该药可以靶向结合目标肌肉组织中高度表达的转铁蛋白受体1（TfR1），通过促进外显子的跳跃，使肌肉细胞产生短且具有功能的肌肉营养不良蛋白，从而阻止或逆转疾病的进程。临床前研究表明，DYNE-251治疗可使mxd模型小鼠基因发生跳跃的能力强劲持久，诱导骨骼肌和心肌中的肌肉营养不良蛋白表达从而减少肌肉损伤、增加肌肉功能。2022年10月，该药被FDA授予快速通道资格认定，用于治疗51号外显子跳跃突变的DMD。Part 3总结整体来看，近十年来DMD治疗领域取得了很大的突破，多款新疗法先后获批，其中涉及2款皮质类固醇药物、1款基因疗法、1款蛋白质修复疗法和4款反义寡核苷酸药物。此外，目前还有多款在研DMD新疗法进入后期临床，而且在研DMD疗法药物类型愈发多样，涉及单抗、细胞疗法、多肽寡核苷酸偶联物、抗体寡核苷酸偶联物。期待未来有更多的DMD新药获批上市，给DMD患者提供更多的选择。

寡核苷酸信使RNA基因疗法临床研究核酸药物

2021-01-29

·Endpoints

Catabasis Pharmaceuticals snaps up HAE antibody in buyout; Gladstone, UCSF scientist spearhead launch of neuro research center

A few months after finally admitting defeat on their failed Duchenne MD program, Catabasis Pharmaceuticals is heading in a new direction with the purchase of Quellis Biosciences and their lead antibody for hereditary angioedema. Catabasis shared the news on Friday that they’re buying out Quellis, a rare disease-focused biotech that was unveiled over a year ago by Chris Garabedian and Perceptive Advisors’ PXV Fund. With it, Catabasis is snapping up QLS-215, a monoclonal antibody inhibitor of plasma kallikrein. The candidate is currently in preclinical development — but won’t be for long, according to Catabasis. In addition to the buyout, the Boston-based biotech closed a $110 million private placement to fund the completion of IND-enabling, Phase Ia and Phase Ib/II studies. Catabasis and Quellis are keeping the terms of their deal under wraps. But after the acquisition and private placement, Catabasis expects to have a cash runway through 2023. The company plans on filing an IND application for QLS-215 in the first half of 2022, and reading out initial Phase I results by the end of 2022. If all goes well, they hope to launch a Phase Ib/II trial in 2023, with initial results by the end of that year. Back in October, Catabasis acknowledged a final failure for its Duchenne MD drug edasalonexent. Three years earlier, CEO Jill Milne and the crew insisted they found reason for hope in data from a failed study, and that it justified a try in Phase III. After the primary, change in baseline on the North Star Ambulatory Assessment, and the secondary on timed function tests both came up short of statistical significance, Catabasis killed the effort — leaving them with one other drug in the pipeline, a preclinical program on autophagy. Drug hunters and developers working in neurological diseases will now have a new place to turn to for insights. The Gladstone-UCSF Center for Neurovascular Brain Immunology is launching with a $2.5 million donation and neuroimmunologist Katerina Akassoglou as the founding director. “Historically, neurological diseases have been classified as being only degenerative, inflammatory, or vascular,” she said in a statement . “But we now know, given recent insights from clinical research and the failure of many clinical trials, that this classification cannot explain how diseases start and progress, nor has it been able to identify the best potential drug candidates.” By integrating basic and clinical research on vascular biology, immunology and neuroscience, scientists at the new center aim to shed light on overlapping mechanisms behind diseases like multiple sclerosis, Alzheimer’s disease and traumatic brain injury — and have implications extending as far as neonatal brain injury, epilepsy, infectious diseases and psychiatric disorders. Add it all together, Akassoglou said, and their approaches in imaging and drug discovery may just “transform biomedicine.” To uncover the role of vascular abnormalities in the brain, they will bring together basic and clinical experts and develop new molecular diagnostics, while testing new therapies and biomarkers.

抗体并购

100 项与 Edasalonexent 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	-	-	DB15010

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
杜氏肌营养不良症	临床3期	美国	Astria Therapeutics, Inc.	2018-10-02
杜氏肌营养不良症	临床3期	澳大利亚	Astria Therapeutics, Inc.	2018-10-02
杜氏肌营养不良症	临床3期	加拿大	Astria Therapeutics, Inc.	2018-10-02
杜氏肌营养不良症	临床3期	德国	Astria Therapeutics, Inc.	2018-10-02
杜氏肌营养不良症	临床3期	爱尔兰	Astria Therapeutics, Inc.	2018-10-02
杜氏肌营养不良症	临床3期	以色列	Astria Therapeutics, Inc.	2018-10-02
杜氏肌营养不良症	临床3期	瑞典	Astria Therapeutics, Inc.	2018-10-02
杜氏肌营养不良症	临床3期	英国	Astria Therapeutics, Inc.	2018-10-02
2型糖尿病	临床1期	美国	Astria Therapeutics, Inc.	2011-12-01

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适应症

分期

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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


NCT02439216 (CTgov)	临床1/2期	杜氏肌营养不良症	31	CAT-1004 (Part B - CAT-1004 67 mg/kg/Day)	選顧壓選壓衊醖製繭鏇(簾鬱繭壓蓋餘蓋夢餘獵) = 憲願壓衊顧襯願觸夢鬱鬱鹹選鏇鏇獵選淵顧艱 (築蓋夢艱淵構齋艱壓憲, 壓願鹹壓窪選餘範衊鹽 ~ 蓋壓簾網鹽觸鬱齋餘襯) 更多	-	2022-09-23
				CAT-1004 (Part B - CAT-1004 100 mg/kg/Day)	選顧壓選壓衊醖製繭鏇(簾鬱繭壓蓋餘蓋夢餘獵) = 範壓齋願憲醖鬱廠醖鏇鬱鹹選鏇鏇獵選淵顧艱 (築蓋夢艱淵構齋艱壓憲, 簾鬱壓夢餘製淵觸蓋獵 ~ 餘艱鏇構簾壓積壓製衊) 更多
NCT03703882 (PolarisDMD, CTgov)	临床3期	杜氏肌营养不良症	131	Edasalonexent (Dose 1)	網餘夢夢糧願糧築網鏇(齋繭鑰壓窪觸鹽構觸齋) = 膚製襯網遞構鏇鬱齋遞鹽餘鑰積壓鹹簾蓋糧構 (願憲襯鏇繭構鹹餘糧淵, 膚願齋蓋糧範襯壓繭選 ~ 蓋網觸簾壓鹹簾窪鏇製) 更多	-	2022-06-21
				Placebo (Placebo)	網餘夢夢糧願糧築網鏇(齋繭鑰壓窪觸鹽構觸齋) = 鬱觸鑰憲鏇選餘艱願顧鹽餘鑰積壓鹹簾蓋糧構 (願憲襯鏇繭構鹹餘糧淵, 鏇糧築鬱鬱鹽蓋顧齋鹽 ~ 顧餘鏇選鹽憲範鏇構憲) 更多
Phase 3 Trial of Edasalonexent for Duchenne Muscular Dystrophy (MDA2021)	临床3期	杜氏肌营养不良症	131	Edasalonexent	鹽衊鏇憲廠憲製鹹蓋衊(衊構醖鹹膚鹹鬱壓簾憲) = 觸憲鏇鹹鹹鏇顧夢壓齋衊壓鏇顧糧願襯憲憲鹹 (繭夢憲餘蓋膚艱醖繭網 ) 更多	不佳	2021-03-01
NCT03703882 (PolarisDMD, Pubmed) 人工标引	临床3期	杜氏肌营养不良症	131	Edasalonexent 100 mg/kg/day	繭遞膚衊淵鬱襯窪遞餘(蓋獵糧鹹範憲齋選齋觸) = not statistically significant 觸鏇蓋製醖鏇築艱遞鏇 (襯憲膚衊糧鏇壓壓選製 ) 更多	不佳	2021-01-01
				Placebo