A pivotal, randomized, double-blind, placebo-controlled, multi-center therapeutic study for patients age 4 and older with a confirmed diagnosis of CACNA1A. The objective of this study is to evaluate the safety, tolerability and efficacy of N-acetyl-L-leucine (IB1001) compared to standard of care.

开始日期2026-08-17

申办/合作机构

IntraBio Ltd.

NCT06673056

/ Active, not recruiting临床3期

Effects of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T: A Phase III, Randomized, Placebo-controlled, Double-blind, Crossover Study

A pivotal, randomized, double-blind, placebo-controlled, multi-center therapeutic study for patients age 4 and older with a confirmed diagnosis of Ataxia-Telangiectasia (A-T). The objective of this study is to evaluate the safety, tolerability and efficacy of N-acetyl-L-leucine (IB1001) compared to standard of care.

开始日期2025-03-18

申办/合作机构

IntraBio Ltd.

NCT05163288

/ Recruiting临床3期

Effects of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C (NPC): A Phase III, Randomized, Placebo-controlled, Double-blind, Crossover Study

A pivotal, randomized, double-blind, placebo controlled, multi-center therapeutic study for patients age 4 and older with a confirmed diagnosis of Niemann Pick disease type C (NPC). The objective of this study is to evaluate the safety, tolerability and efficacy of N-acetyl-L-leucine (IB1001) compared to standard of care.
Following this randomized, double-blind, placebo-controlled "Parent Study", an extension phase is conducted for (1) patients who completed the "Parent Study" and (2) patients who are enrolled directly into the Extension Phase. Currently, the Extension Phase provides patients with 3 years of open-label treatment.

开始日期2022-06-30

申办/合作机构

IntraBio Ltd.

100 项与 N-乙酰-L-亮氨酸相关的临床结果

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100 项与 N-乙酰-L-亮氨酸相关的转化医学

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100 项与 N-乙酰-L-亮氨酸相关的专利（医药）

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690

项与 N-乙酰-L-亮氨酸相关的文献（医药）

2026-08-01·JOURNAL OF NEUROLOGY

Neurofilament light chain (NfL) as a surrogate outcome measure for GM2 gangliosidoses

Article

作者: Baker Ii, Peter R ; Factor, Mallory ; Bremova-Ert, Tatiana ; Abreu, Nicolas J ; Burrow, T Andrew ; Billington, Ian ; Gannon, Jennifer L ; Fields, Taylor ; Shayota, Brian J ; Strupp, Michael ; Kerthi, Jorgji ; Patterson, Marc C ; Fields, Cassandra ; Grosso, Megan ; Martakis, Kyriakos ; Strupp, Lennard ; Baker, Joshua J

Abstract:

Background:

The GM2 gangliosidoses (GM2) are ultra-rare neurodegenerative disorders caused by deficient hexosaminidase A and/or B activity, leading to lysosomal GM2 ganglioside accumulation. Disease onset ranges from infancy to adulthood, with earlier onset associated with more rapid progression. Neurofilament light chain (NfL), a sensitive marker of axonal injury, has been extensively investigated as a biomarker for neurodegenerative disorders, including GM2.

Methods:

To evaluate its clinical utility as a biomarker for GM2, NfL was measured in patients with GM2 enrolled in a Phase 2b, multinational, rater-blinded study of levacetylleucine [NCT03759665], and in its open-label Extension Phase (EP).

Results:

Nineteen participants had viable samples for NfL analysis at baseline, after six weeks of treatment, and after a six-week washout; 10 had samples in the long-term EP. After the initial 6-week treatment phase, NfL concentration declined a mean − 8.9% (SD 13%; p < 0.008), followed by a rebound of + 9.2% (SD 16.1%; p = 0.022) during the post-treatment 6-week washout. Changes in NfL correlated with the statistically significant and clinically meaningful changes captured on the primary Clinical Impression of Change in Severity (CI-CS), and secondary Scale for the Assessment and Rating of Ataxia (SARA) and Modified Disability Rating Scale (mDRS). In the EP, patients showed a mean NfL reduction of − 16.9% after 1 year (SD 15.0; p = 0.010) and − 33.5% after 2 years (SD 12.8; p < 0.001) of levacetylleucine treatment.

Conclusions:

These findings support NfL as a promising surrogate outcome candidate for GM2 and link biochemical improvement with functional benefit, which is reasonably likely to predict both disease activity and treatment response/clinical benefit.

2026-07-03·JOURNAL OF BIOMOLECULAR STRUCTURE & DYNAMICS

Amino acid osmolytes disrupting intradomain interactions reduce the amyloidogenicity of α-synuclein: studies with charged l -amino acids and their derivatives

Article

作者: Prakash, Jogadhenu S. S. ; Prabhu, N. Prakash ; Saurabh, Archi ; Chauhan, Suraj

Aggregation of α-synuclein (α-syn) is a hallmark of Parkinson's and dementia with Lewy bodies pathogenesis. The high plasticity and lack of stable tertiary structure make α-syn more susceptible to its surrounding environment. Under stress conditions, small organic molecules known as osmolytes accumulate inside the cells. They affect the conformational states and fibrillation pathways of proteins. Here, the effects of eight different amino acid osmolytes (charged: l-glutamate, l-aspartate, l-lysine, l-arginine; amide side chains: l-glutamine, l-asparagine; and N-acetylated: N-acetyl-l-glutamic acid, N-acetyl-l-lysine) on the fibrillation of human α-synuclein were examined. Arginine and N-acetyl-l-lysine inhibited the fibrillation at concentrations above 0.2 and 0.4 M, respectively. Lysine, asparagine, and glutamate accelerated the fibrillation by reducing lag time. N-acetyl-l-glutamic acid induced lag-independent fibrillation, whereas glutamine and asparagine showed concentration-dependent effects on the fibrillation with reduced lag time at higher concentrations. Molecular dynamics simulations revealed that interdomain contacts facilitated the fibrillation. The amino acids interacting predominantly through van der Waals interactions reduced the lag time of α-syn. However, the amino acids having strong electrostatic interactions with the protein disrupted intradomain contacts, favored extended conformation, and inhibited the fibril formation.

2026-07-01·LANCET NEUROLOGY

Safety and efficacy of levacetylleucine in ataxia-telangiectasia: a phase 3, randomised, double-blind, placebo-controlled crossover trial

Article

作者: Skorvanek, Matej ; Foltan, Tomas ; Bremova-Ertl, Tatiana ; Thakur, Nivedita ; Del Mar Garcia Romero, Maria ; Bolton, Clare ; Gautschi, Matthias ; Schmahmann, Jeremey ; Hoche, Franziska ; Kolnikova, Miriam ; Hahn, Andreas ; Han, Vladimir ; Martakis, Kyriakos ; Prasad, Manish ; Perlman, Susan ; Thiel, Moritz ; Panagioti, Ouliana

BACKGROUND:

Ataxia-telangiectasia is a rare, autosomal recessive neurodegenerative disorder. Levacetylleucine (N-acetyl-L-leucine) has been shown to be efficacious for the treatment of neurological manifestations and to have a disease-modifying effect in lysosomal storage disorders such as Niemann-Pick disease type C. We aimed to assess the safety and efficacy of levacetylleucine for paediatric and adult patients with ataxia-telangiectasia.

METHODS:

In this phase 3, randomised, double-blind, placebo-controlled crossover trial, participants were enrolled across ten research hospitals in Germany, Slovakia, Spain, Switzerland, the UK, and the USA. Eligible patients aged 4 years or older with genetically confirmed ataxia-telangiectasia were randomly assigned (1:1) using interactive response technology to receive two or three times daily orally administered levacetylleucine or a matching placebo over two consecutive 12-week treatment periods (patients weighing 35 kg or more received 4 g per day of orally administered levacetylleucine or a matching placebo three times per day and patients weighing less than 35 kg received weight-tiered doses two or three times per day based on approximately 0·1 g/kg per day). All participants, investigators, and assessors were blinded to group assignment. The primary outcome was the mean change on the Scale for the Assessment and Rating of Ataxia (SARA), assessed at baseline and at the end of each 12-week treatment period of levacetylleucine or placebo. Safety and efficacy analyses were done in all randomly assigned patients who received at least one dose of study medication, and a linear mixed-effects model was used to account for data missing at random. The trial is registered with ClinicalTrials.gov, NCT06673056, and CTIS, 2024-517706-29; the open-label extension phase is ongoing.

FINDINGS:

Between March 18, 2025 and June 30, 2025, 77 participants with a genetically confirmed diagnosis of ataxia-telangiectasia were screened for inclusion. Four patients were excluded (not meeting inclusion criteria) and 73 were enrolled and randomly assigned (36 to levacetylleucine followed by placebo and 37 to placebo followed by levacetylleucine. 73 patients were included in the primary analysis and safety sets. 38 (52%) of 73 patients were female and 35 (48%) were male; 55 (75%) of 73 patients were White. 47 (64%) of 73 were younger than 18 years and 26 (36%) were aged 18 years or older. The mean change in the SARA total score with levacetylleucine was -1·92 (SD 2·81) versus -0·14 (2·38) with placebo (linear mixed model treatment effect -1·88 [SD 0·41], 95% CI -2·70 to -1·06; p<0·0001). 54 adverse events occurred in 29 patients receiving levacetylleucine versus 75 events in 25 patients receiving placebo. No treatment-related serious adverse events or deaths occurred.

INTERPRETATION:

Levacetylleucine showed a significant and clinically meaningful improvement in functioning and was safe and well-tolerated, providing a favourable benefit-risk profile for the treatment of ataxia-telangiectasia. An ongoing open-label extension phase of this trial will investigate potential long-term, neuroprotective and disease-modifying effects.

FUNDING:

IntraBio.

110

项与 N-乙酰-L-亮氨酸相关的新闻（医药）

2026-10-08

·PMLiVE

Shionogi to acquire IntraBio in deal worth $2bn

Shionogi & Co, headquartered in Osaka, Japan, has agreed to acquire IntraBio, a biopharmaceutical company developing and commercialising therapies for neurodegenerative diseases. IntraBio would become a wholly owned subsidiary of Shionogi Inc, a New Jersey, US-based subsidiary of Shionogi. The deal would add Aqneursa (levacetylleucine) and IntraBio’s capabilities in the rare disease area to Shionogi’s growing portfolio. Aqneursa was approved by the FDA in September 2024 for neurological manifestations of Niemann-Pick disease Type C (NPC) in adults and paediatric patients and by the European Medicines Agency (EMA) in January 2026. It was approved by the FDA to treat Ataxia in adults and paediatric patients diagnosed with Ataxia-Telangiectasia (A-T) in September 2026. Aqneursa is currently under review by the EMA for adult and paediatric patients diagnosed with A-T. Isao Teshirogi, CEO of Shionogi, said: “Bringing Aqneursa to Shionogi after our acquisition of Radicava will deepen our commitment to rare disease communities, expand our capabilities and strengthen our portfolio as we advance future innovation for patients with significant unmet needs.” “This milestone gives us the opportunity to build on the meaningful foundation IntraBio has established with the Niemann-Pick Type C community and to deliver the first and only therapy for Ataxia-Telangiectasia to a community that has lived for many years without options to treat the diverse and debilitating symptoms caused by this disorder,” said Nathan McCutcheon, President and CEO of US-based Shionogi Inc. “In addition to Aqneursa, the capabilities and resources gained through this acquisition would help to accelerate the development of new treatment options.” Shionogi would acquire global rights, including intellectual property and commercialisation rights, for Aqneursa, which is currently under review by the EMA for adult and paediatric patients diagnosed with A-T. In addition, IntraBio’s rare disease expertise would strengthen Shionogi’s capabilities across its rare disease pipeline, including clinical programmes in Pompe disease, Fragile X syndrome and Jordan’s syndrome, as well as additional early-stage rare neurodegenerative disease programmes recently added to the pipeline. The content above comes from the network. if any infringement, please contact us to modify.

上市批准并购

2026-10-07

·Newleader

全球生物医药圈动态速览 2026.10.07 | 监管审批 / BD 并购 / 临床数据 / 中国市场

WENDY'S RESEARCH · 生物医药动态全球生物医药圈动态速览 2026 年 10 月 7 日星期三 · 监管审批 / BD 并购 / 临床数据 / 中国市场今日主线：罕见病与自免的密集兑现。FDA 一天批出胃癌一线双抗与首个温抗体型溶血性贫血疗法；本周 MNC 交易总额（诺华 78 亿美元 mRNA、盐野义 20 亿美元收购、赛诺菲/Regeneron 最高 80 亿美元续约）显示大药企正向 RNA 药物与下一代自免重仓转移；中国资产端借 ESMO 数据窗口与 BD 落地行情持续修复。一、监管审批｜FDA 两项批准 + 一项罕见病认定 Zymeworks 的 Ziihera（zanidatamab）获 FDA 批准一线 HER2 阳性胃食管腺癌覆盖两种方案：Ziihera + 替雷利珠单抗 + 化疗（不限 PD-L1 状态）、Ziihera + 化疗（HER2 IHC 3+）。基于 III 期 HERIZON-GEA-01：联合方案较曲妥珠单抗 + 化疗降低 28% 死亡风险，中位总生存 26.4 个月。该批准触发 Jazz Pharmaceuticals 向 Zymeworks 支付2.5 亿美元里程碑款。来源：BioPharm International「The BioPharm Brief」（2026-10-07）强生 IMAAVY（nipocalimab）：全球首个获批用于温抗体型自身免疫性溶血性贫血（wAIHA）的疗法 FcRn 拮抗剂，适用于 12 岁及以上、正在或曾接受皮质类固醇治疗的患者；通过减少循环致病性 IgG 自身抗体、针对疾病关键驱动机制起效，而非广谱免疫抑制。基于 II/III 期 ENERGY 数据（持续血红蛋白应答、疲劳改善）。来源：BioPharm International「The BioPharm Brief」（2026-10-07） Mahzi Therapeutics 的 MZ-1866 获 FDA 罕见儿科疾病认定 AAV9 基因替代疗法，递送功能性 TCF4 基因治疗 Pitt Hopkins 综合征（罕见神经发育障碍）；I/II 期 UNITE 研究入组已过半（7/12），预计 2026 年底完成入组。若最终获批，有资格获得优先审评券（PRV）。来源：BioPharm International「The BioPharm Brief」（2026-10-07）二、BD 与并购｜本周交易密集，MNC 持续下注 RNA 与双抗 01 诺华 × 中国 Abogen（应诺生物）｜最高78 亿美元。押注 mRNA 体内 T 细胞衔接器（设计用于清除致病 B 细胞）；诺华看重其差异化 RNA 平台与 mRNA 编码 T 细胞衔接器的开创性工作。（BioSpace，2026-10-06） 02 盐野义 × IntraBio ｜ 20 亿美元整体收购。标的罕见病药 Aqneursa 已获 FDA 批准用于 Niemann-Pick 病 C 型，并成为首个共济失调毛细血管扩张症疗法；Jefferies 预估全球峰值销售 10 亿美元。（BioSpace） 03 赛诺菲 × Regeneron ｜首付 10 亿美元，里程碑最高再 +70 亿美元。续约长期合作，开发 4 个下一代 Regeneron 自免抗体，目标打造重磅药 Dupixent 的继任者。（BioSpace） 04 礼来 × Gate Bioscience ｜追加最高8.7 亿美元。在去年总额最高 8.56 亿美元合作基础上的扩展。（BioSpace） 05 CSL × Alentis Therapeutics ｜最高约 16 亿美元。合作开发针对肾脏与肝脏罕见进展性疾病的药物。（BioSpace） 06 Alector × Genentech ｜首付 1 亿美元，里程碑至 11.7 亿美元。授权脑渗透性 GCase 替代疗法 AL050；非稀释性授权将 Alector 现金跑道延至 2029 年，其 Alector Brain Carrier 平台保留自研。（BioPharmCatalyst，10-05）三、临床数据｜两条关键读出 + 两家波折 01 Genmab/AbbVie 双抗 Epkinly（epcoritamab）III 期一线 DLBCL 数据强劲：联合 R-CHOP 使疾病进展或死亡风险降低 51% ，分析师评价「高度差异化」，有望改写弥漫大 B 细胞淋巴瘤一线标准治疗（对比罗氏 Polivy、Incyte Monjuvi）。（BioSpace） 02 Vaxcyte（PCVX）III 期 OPUS-1 达主要终点：31 价肺炎球菌结合疫苗 VAX-31 在成人中达到安全性、免疫原性与免疫桥接目标，对 3 个新血清型及交叉反应血清型 20B 显示优效；2027 上半年再读出更多 III 期数据，计划 2028 上半年提交 BLA。（BioPharmCatalyst，10-05） 03 艾力斯（688578）伏美替尼 III 期 FURVENT 未达主要终点：单药一线治疗 EGFR 20 外显子插入突变 NSCLC 的 PFS 未达标；公司正与 ArriVent 评估完整数据集以确定后续方案。截至 2026-09-30 该关键试验累计投入约 7,082 万元。（公司公告，10-07） 04 Sarepta 出现转机信号：Elevidys 在年长 DMD 患者中显示持续且有临床意义的获益（世界肌肉学会年会数据）；叠加强直性肌营养不良 1 型与 FSHD 中期数据读出、FDA 对外显子跳跃疗法 Amondys 45 与 Vyondys 53 的传统批准决定等催化剂，Jefferies 转向乐观。（BioSpace）四、中国市场｜港股医药假期大涨，ESMO 前资金抢跑 01 10 月 6 日港股医药领涨大市：恒生生物科技指数涨 3.21% 逼近年内高点；维亚生物 +23.31%、康希诺 +14.04%、歌礼近 +12%，药明生物、再鼎医药、亚盛医药等跟涨。（新浪财经 / 中国基金报） 02 四因素共振： ① ESMO 年会（10/23–27，马德里）96 项突破摘要中 31 项来自中国公司； ② BD 密集落地——恒瑞 HRS-1596 授权诺和诺德（最高 26 亿美元）、阿斯利康 20 亿美元战投康方生物海外伙伴 Summit、中国生物制药 × STADA 生物类似药合作； ③ 南向资金近三个月净买入超 1,200 亿港元； ④ 「十五五」规划强化生物医药支柱产业定位。（同上） 03 复星医药 FXS5626 片（AC-201）获批 II/III 期临床：口服 TYK2/JAK1 双靶点抑制剂，用于非节段型白癜风，国内暂无同类获批药物。（公司公告，10-07） 04 华海药业业绩预告：预计前三季度归母净利润 10.28–11.03 亿元，同比 +170%~190%，主因原料药、国内制剂及美国制剂业务增长等。（公司公告，10-07） 05 明日（10/8）A 股及港股通恢复交易：假期港股医药大涨行情能否向 A 股传导，是短期关注点。（同上）小结一句话总结今日主线是「罕见病与自免的密集兑现」——FDA 一天批出胃癌一线双抗与首个 wAIHA 疗法，本周 MNC 交易显示大药企正加速向 RNA 药物与下一代自免重仓转移；中国资产端则借 ESMO 数据窗口与 BD 落地行情持续修复，10 月 8 日 A 股恢复交易后关注行情传导。说明：以上内容全部来自公开披露信息，已逐条标注来源；个别条目（Vaxcyte、Alector）为 10 月 5–6 日周内动态，已在文中注明日期。数据口径以原始报道为准，本简报仅为资讯整理，不构成任何投资建议。 Wendy's Research BIOMED DAILY BRIEF 本简报为公开资讯的整理与归纳，仅供学习与参考，不构成任何投资、经营或决策建议。数据与事实以各原始来源为准 · Wendy's Research 出品

信使RNA并购基因疗法临床3期优先审批

2026-10-07

·BioSpace

Genmab and AbbVie’s Epkinly win, a rush of deals, Sarepta’s turnaround hopes, more

New data for Genmab and AbbVie’s bispecific antibody Epkinly wowed analysts and could set the partners up for an expanded approval in diffuse large B cell lymphoma; deals have flowed steadily this week, including Novartis’ nearly $8 billion bet on mRNA drugs with China’s Abogen Biosciences; and could Sarepta Therapeutics turn things around from last year’s annus horribilis? > Listen on Spotify > Listen on Apple Products > Listen on Amazon Music > Listen on iHeart > Watch on YouTube Genmab and AbbVie’s bispecific antibody Epkinly could set a new standard of care in diffuse large B cell lymphoma. Approved in May 2023 for patients who have undergone at least two prior lines of systemic treatment, new late-stage data speak to the drug’s use in frontline treatment. In the Phase 3 study, Epkinly plus R-CHOP— a five-drug regimen based on Roche’s rituximab—generated a 51% reduction in risk of disease progression or death, leading to effusive analyst commentary about the “highly differentiated” results, comparing the drug to Roche’s Polivy and Incyte’s Monjuvi. On the deals side of biopharma, this past week has seen considerable action. Just weeks after securing expanded approval for the rare disease drug Aqneursa, IntraBio has exited in a $2 billion buyout by Japan’s Shionogi. First cleared by the FDA in September 2024 for Niemann-Pick disease type C, Aqneursa is now also approved as the first ever treatment for ataxia-telangiectasia, a fatal disease that involves the progressive degeneration of the cerebellum. Shionogi saw the value in the drug, for which Jefferies analysts project global peak sales of $1 billion. Meanwhile, Novartis has bet up to $7.8 billion on Abogen Biosciences and an mRNA-based in vivo T cell engager designed to deplete disease-causing B cells. Novartis was attracted to Abogen because of its differentiated proprietary RNA platform and pioneering work in mRNA-encoded T cell engagers and other RNA-encoded therapeutic modalities, a spokesperson told BioSpace on Monday. In addition, Eli Lilly is expanding its relationship with Gate Bioscience, putting some $870 million more on the line after first striking an alliance last year worth up to $856 million, and CSL is risking up to around $1.6 billion to partner with Alentis Therapeutics on drugs for rare and progressive diseases affecting the kidneys and liver. Finally, Sanofi and Regeneron have renewed their longstanding collaboration with a $1 billion upfront payment in an effort to develop a successor to their blockbuster Dupixent. Regeneron will have the chance to collect up to $7 billion more in biobucks as the two work on four next-gen Regeneron antibodies for immune-mediated conditions. Over at Sarepta Therapeutics, things are looking up —at least according to Jefferies analysts. Last year, Sarepta faced a very public saga over its Duchenne muscular dystrophy gene therapy Elevidys after multiple deaths were associated with the vector technology that underlies the treatment. Last week, the biotech shared new data at the World Muscle Society’s annual meeting in Japan, highlighting Elevidys’ durable and clinically meaningful benefits in older patients with Duchenne muscular dystrophy. Jefferies also pointed to upcoming catalysts in the form of mid-stage readouts in myotonic dystrophy type 1 and facioscapulohumeral muscular dystrophy and an anticipated FDA decision on traditional approval of Sarepta’s exon-skippers, Amondys 45 and Vyondys 53. Finally, check out BioSpace ’s Q3 recaps on M&A and VC activity, and read about who’s hot and who’s not in biotech and pharma , as well as our annual 40 under 40 list of young biopharma leaders who demonstrate the industry’s values of innovation, impact and commitment to improving outcomes for patients.

上市批准临床3期基因疗法临床结果

100 项与 N-乙酰-L-亮氨酸相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D12967	-	-	-

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
共济失调毛细血管扩张	美国	IntraBio, Inc.	2026-09-18
神经系统表现	欧盟	Intrabio Ireland Ltd.	2026-01-19
神经系统表现	冰岛	Intrabio Ireland Ltd.	2026-01-19
神经系统表现	列支敦士登	Intrabio Ireland Ltd.	2026-01-19
神经系统表现	挪威	Intrabio Ireland Ltd.	2026-01-19
C型尼曼匹克病	美国	IntraBio, Inc.	2024-09-24

未上市

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
阵发性共济失调2型	临床3期	美国	IntraBio Ltd.	2026-08-17
阵发性共济失调2型	临床3期	奥地利	IntraBio Ltd.	2026-08-17
阵发性共济失调2型	临床3期	德国	IntraBio Ltd.	2026-08-17
阵发性共济失调2型	临床3期	希腊	IntraBio Ltd.	2026-08-17
阵发性共济失调2型	临床3期	意大利	IntraBio Ltd.	2026-08-17
阵发性共济失调2型	临床3期	瑞士	IntraBio Ltd.	2026-08-17
阵发性共济失调2型	临床3期	英国	IntraBio Ltd.	2026-08-17
家族性1型偏瘫性偏头痛	临床3期	美国	IntraBio Ltd.	2026-08-17
家族性1型偏瘫性偏头痛	临床3期	奥地利	IntraBio Ltd.	2026-08-17
家族性1型偏瘫性偏头痛	临床3期	德国	IntraBio Ltd.	2026-08-17

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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


NCT03759665 (Pubmed \| J Neurol)	临床2期	GM2神经节苷脂贮积症 Neurofilament light chain (NfL)	19	levacetylleucine	繭壓網觸鑰範窪糧餘觸(繭夢膚襯觸製艱遞夢構) = 壓壓願觸築網淵製糧餘淵糧膚簾築糧蓋膚憲憲 (膚願製範願襯簾壓鹽憲 )	积极	2026-08-01
IB1001-301 (EAN2026)	临床3期	C型尼曼匹克病	40	N-acetyl-L-leucine (NALL) (Extension Phase (open-label))	觸積鬱餘艱獵簾顧衊艱(鹽觸艱夢遞製蓋鬱網衊) = 積齋願壓衊鑰鏇顧艱鬱蓋鹹築壓齋鑰夢顧鹽廠 (積襯製齋窪獵餘積範簾, 4.0)	积极	2026-06-26
				Placebo	範蓋製遞鏇繭膚繭鑰選(鬱窪餘繭襯壓餘鏇鏇範) = 繭願餘夢膚積構壓網襯製餘鏇蓋築遞衊製遞壓 (築鏇窪範淵艱鑰餘糧鏇, 7.9) 更多
S31.004 (AAN2025)	临床3期	C型尼曼匹克病	60	Levacetylleucine	製艱醖淵積築糧醖膚鹽(壓簾蓋齋鏇簾鬱壓簾積) = On each mDRS domain there was improvement from baseline when patients received levacetylleucine and a greater improvement in each domain when patients were treated with levacetylleucine versus placebo 鏇襯製遞鏇選鏇製鑰廠 (餘淵積蓋糧憲艱構餘蓋 ) 更多	积极	2025-04-07
				Placebo
NCT05163288 (FDA_CDER) 人工标引	临床3期	C型尼曼匹克病	60	AQNEURSA	夢艱鏇簾顧鹽憲觸繭壓(繭繭構鏇襯鏇鹽壓鬱鬱) = 範簾窪艱餘願築壓製廠壓糧衊膚選艱齋鑰鬱鑰 (繭遞膚窪積積鑰淵網鹹, 0.1)	积极	2024-09-24
				Placebo	夢艱鏇簾顧鹽憲觸繭壓(繭繭構鏇襯鏇鹽壓鬱鬱) = 築夢網鹽顧積憲遞顧艱壓糧衊膚選艱齋鑰鬱鑰 (繭遞膚窪積積鑰淵網鹹, 0.1)
NCT03759665 (Accesswire) 人工标引	临床2期	GM2神经节苷脂贮积症	30	IB1001	憲憲網鬱廠積構鹽壓簾(簾鏇齋齋糧淵鏇蓋顧鬱) = statistically significant change. 90% CI: 0.00, 1.50, p-value = 0.039 壓憲構夢鬱憲廠鬱襯衊 (鹽獵構願製鑰壓鑰繭鑰 ) 更多	积极	2021-09-14
NCT03759639 (Pubmed \| J Neurol) 人工标引	临床2期	C型尼曼匹克病	32	N-acetyl-L-leucine	衊蓋壓遞廠廠鹽廠網願(鹹選齋衊艱醖網艱鹽鹽) = NALL met the CI-CS primary endpoint (mean difference 0.86, SD = 2.52, 90% CI 0.25, 1.75, p = 0.029) 選鬱淵鑰鹹網選襯鹽顧 (簾膚淵築積簾觸願鑰餘 )	积极	2021-08-13
NCT00768287 (CTgov)	临床2/3期	血友病B	77	IB1001 (IB1001)	範簾範夢鹽廠糧鏇積醖(艱襯繭鬱膚艱蓋艱築膚) = 簾衊襯夢壓範壓糧齋獵築鏇齋憲憲齋蓋憲繭蓋 (壓夢願鏇觸獵構窪繭淵, 451.5) 更多	-	2018-09-10
				nonacog alfa (Nonacog Alfa)	範簾範夢鹽廠糧鏇積醖(艱襯繭鬱膚艱蓋艱築膚) = 顧衊鹹壓糧衊蓋築鹹鹹築鏇齋憲憲齋蓋憲繭蓋 (壓夢願鏇觸獵構窪繭淵, 468.6) 更多