A Post-marketing, Observational, Descriptive Study to Assess the Risk Associated With Pregnancy, the Maternal Complications, and Adverse Effects on the Developing Fetus, Neonate, and Infant Among Individuals Exposed to Omaveloxolone During Pregnancy and/or Lactation

In this study, researchers will learn more about the safety of BIIB141, also known as omaveloxolone or SKYCLARYS. This is a drug available for doctors to prescribe for people with Friedrich's Ataxia, also known as FA. This is known as an "observational" study, which collects health information about study participants without changing their medical care. Participants for this study will have taken BIIB141 at any time during pregnancy and/or while breastfeeding or pumping up through the first year after delivery. Participants can join this study on their own or they may be enrolled by their regular doctors. This study is also known as the "SKYCLARYS (Omaveloxolone) Pregnancy and Lactation Surveillance Program."
The main objective of this study is to learn more about how BIIB141 may affect pregnancy, as well as any effects on the health of the mother and of the baby during its first year of life.
The main question researchers want to answer in this study is:
· Does taking BIIB141 during pregnancy or breastfeeding lead to any major birth defects?
Researchers will also learn more about:
* Does taking BIIB141 during pregnancy or breastfeeding lead to any minor birth defects?
* Does taking BIIB141 during pregnancy or breastfeeding affect the following:
* Gestational diabetes, a disease that can happen during pregnancy that affects how your body uses sugar
* Pre-eclampsia, a pregnancy-related high blood pressure disease
* Unborn baby being small for its expected age (usually in weeks)
* Loss of an unborn baby
* Live birth
* Premature birth
* Loss of a newborn
* Growth or developmental delays in the baby
* Serious illness in the baby resulting in hospitalization
* Serious infections in the baby, or ones in babies with a weakened immune system
This study will be done as follows:
* Participants will join the study after signing an informed consent form, also known as an ICF.
* During the study, health information from the participants' regular visits to their doctor will be collected based on whether participant joined the study while pregnant or after the baby is born.
* Each participant will be in the study for up to 1 year after the birth of their child, unless they decide to leave early. Overall, this study is expected to last at least 10 years.

开始日期2026-10-26

申办/合作机构

Biogen, Inc.

NCT07019064

/ Recruiting临床1期

A Phase 1, Randomized, Open-label, Single-dose, Crossover, Relative Bioavailability Study of Omaveloxolone Intact Capsules Versus Capsule Contents Sprinkled Over Low-fat, Non-Greek Yogurt in Healthy Adult Subjects

In this study, researchers will learn more about how BIIB141, also known as omaveloxolone or SKYCLARYS?, is processed in the body when taken in different ways. This drug has been approved, or made available for doctors to prescribe, for people with Friedrich's Ataxia (FA) who are at least 16 years old. Currently, people with FA can take it either as whole capsules or opening the capsules and sprinkling its contents over applesauce. The main goal of this study is to learn if BIIB141 is processed any differently when taken sprinkled over low-fat, non-Greek yogurt compared to whole capsules. This will help researchers learn if yogurt could be another option for people to take BIIB141 with.
The main questions researchers want to answer in this study are :
? How does the body process BIIB141 when taken as whole capsules compared to being sprinkled over yogurt?
Researchers will also learn more about :
* How many participants have adverse reactions during the study. An adverse reaction is a health problem that may be caused by the study drug.
* If there are any changes in the participants' overall health during the study
This study will be done as follows:
* Participants will be screened to check if they can join the study. The screening period will be up to 28 days, after which participants will check into their study research center.
* Participants will stay at the study research center for about 30 days.
* This is a "crossover" study. In this kind of study, all participants receive the same 2 or more study drugs (or ways of taking the drug), but the order in which they receive them depends on the group they are randomly assigned to.
* In Group 1, participants will take a single dose of BIIB141 as a capsule, followed by a break of 14 days. Then, the participants will take a single dose of BIIB141 sprinkled over yogurt.
* In Group 2, participants will take a single dose of BIIB141 sprinkled over yogurt, followed by a break of 14 days. Then, the participants will take a single dose of BIIB141 as a capsule.
* Each participant will be in the study for up to 57 days.

开始日期2025-06-11

申办/合作机构

Biogen, Inc.

NCT06953583

/ Recruiting临床3期

A Phase 3, 2-Part, Randomized, Double-Blind, Placebo-Controlled Study (Part 1) and Open-Label Extension (Part 2) to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Omaveloxolone (BIIB141) in Participants With Friedreich's Ataxia Aged 2 to < 16 Years

In this study, researchers will learn more about the effects and safety of BIIB141, also known as omaveloxolone or SKYCLARYS®. This drug has been approved, or made available for doctors to prescribe, for people with Friedreich's Ataxia (FA) who are at least 16 years old. But, it is not yet available for children and teens with FA who are younger than 16 years old. The main objective of this study is to learn how BIIB141 works in the body and about its safety in children and teens who are 2 to 15 years old.
The main questions researchers want to answer in this study are:
* How does BIIB141 affect the participants' FA symptoms balance and stability?
* How many participants have medical problems during the study?
* Are there any changes in the participants' overall health during the study?
* Are there any changes in the participants' heart health?
* Are there any changes in how the participants move through puberty? Puberty is the time in someone's life when their body changes from a child to an adult.
Researchers will also learn more about:
- How the body processes BIIB141 in children and teens
This study will be done as follows:
* Participants will be screened to check if they can join the study. The screening period will be up to 28 days, after which participants will check into their study research center.
* There are 2 parts in this study. During Part 1, participants will take either BIIB141 or a placebo once a day.
* In Part 1, participants will take BIIB141 or the placebo in a study research center on Day 1, and then at in-person visits at Week 4, Week 12, Week 26, and Week 52. On all other days, they will take BIIB141 or the placebo at home. Part 1 lasts up to 52 weeks.
* During Part 2, participants from Part 1 will either continue taking BIIB141 or start it if they were taking the placebo. Part 2 will last up to 104 weeks.
* In Part 1, participants will have up to 10 visits to their study research center and a phone call at Week 2. In Part 2, participants will have visits at Weeks 4, 8,12, 26, and every 26 weeks after that until they leave the study, and a phone call at Week 2. There will be a final phone call to check on the participants' health 31 days after their last dose.
* Each participant will be in the study for up to about 3 years

开始日期2025-06-09

申办/合作机构

Biogen, Inc.

100 项与 Omaveloxolone 相关的临床结果

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100 项与 Omaveloxolone 相关的转化医学

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100 项与 Omaveloxolone 相关的专利（医药）

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131

项与 Omaveloxolone 相关的文献（医药）

2025-10-01·FREE RADICAL BIOLOGY AND MEDICINE

RTA-408 overcomes cisplatin-resistant lung cancer by inhibiting WWP1-mediated NCOA4 ubiquitination to induce ferritinophagy and ferroptosis

Article

作者: Xu, Ying ; Fei, Yingdan ; Zhang, Sunhan ; Yang, Yueyan ; Zhang, Ting ; Wu, Dongming ; Liu, Teng ; Deng, Shihua ; Wang, Xiaobian ; Chen, Zixin ; Zhu, Ruixiong

Worldwide, pulmonary carcinoma remains the predominant cause of cancer-induced fatalities. Cisplatin (DDP) is a platinum-containing cell cycle-non-specific chemotherapeutic agent and serves as the first-line treatment for non-small cell lung cancer (NSCLC). However, NSCLC cells gradually develop resistance to cisplatin during treatment, reducing drug sensitivity and leading to a significant decrease in the effect of chemotherapy or even treatment failure. The main mechanisms of drug resistance include reduced cellular uptake and apoptotic escape. Strategies to reverse drug resistance often suffer from off-target toxicity or inconsistent efficacy, highlighting the urgent need for new drugs that specifically target resistance pathways. Accordingly, we screened a library of small-molecule compounds associated with ferroptosis. We identified 4-7D (also known as RTA-408), a nuclear factor erythroid 2-related factor 2 activator initially developed to treat Friedreich's ataxia, which has shown potent anti-NSCLC activity in preclinical studies. Its unique ability to regulate both oxidative stress and apoptotic pathways makes it a promising candidate for overcoming cisplatin resistance. Therefore, the present study aimed to determine the key role of omaveloxolone (RTA-408) in overcoming cisplatin-resistant lung cancer. We selected A549/DDP cells as a research model and demonstrated through a series of experimental manipulations that RTA-408 inhibited the growth of A549/DDP cells and inhibited the ubiquitinated degradation of nuclear receptor coactivator 4 through down-regulation of WW structural domain-containing E3 ubiquitin-protein ligase 1 (WWP1), which further induced the development of ferritinophagy and ferroptosis. We report for the first time that RTA-408 overcomes cisplatin-resistant lung cancer by targeting WWP1-mediated ferritinophagy and ferroptosis.

2025-08-01·NEUROBIOLOGY OF DISEASE

Calcium-iron crosstalk in epileptogenesis: Unraveling mechanisms and therapeutic opportunities

Review

作者: Xiao, Fen ; Wang, Jing ; Wu, Nayiyuan ; Wang, Zhi-Bin ; Tao, Ao-Long ; Li, Xuan ; Zhang, Xiu

Epilepsy, a chronic neurological disorder affecting millions globally, remains poorly understood in its etiology and therapeutic management. Emerging evidence highlights the intricate interplay between calcium (Ca²⁺) and iron (Fe²⁺/Fe³⁺) ions in modulating neuronal excitability, oxidative stress, and synaptic plasticity-key processes implicated in epileptogenesis. This review synthesizes current knowledge on the dual roles of Ca²⁺ and Fe²⁺/Fe³⁺ in epilepsy, emphasizing their bidirectional regulatory mechanisms and pathological synergism. Calcium dysregulation, mediated through voltage-gated channels (e.g., Cav1.2, Cav3.2), store-operated calcium entry (SOCE), and mitochondrial calcium uniporters (MCU), exacerbates neuronal hyperexcitability and seizure propagation. Concurrently, iron overload drives ferroptosis via lipid peroxidation and glutathione depletion, while iron deficiency impairs neurodevelopmental processes. Crucially, Ca²⁺-Fe²⁺ crosstalk intersects at multiple nodes: TRP channels (e.g., TRPC6, TRPML1) facilitate dual ion transport; mitochondrial dysfunction links Ca²⁺ overload with Fe²⁺-dependent ROS generation; and inflammatory cascades disrupt both ion homeostasis. Clinically, antiepileptic drugs targeting Ca²⁺ channels (e.g., ethosuximide, zonisamide) and emerging ferroptosis inhibitors (e.g., deferoxamine, RTA 408) underscore the therapeutic potential of modulating these pathways. However, drug resistance and incomplete seizure control necessitate novel strategies leveraging ion interaction networks. We propose that combinatorial approaches targeting Ca²⁺-Fe²⁺ signaling hubs-such as MCU-TRPML1 axes or redox-sensitive RyR channels-may offer synergistic benefits. Future research must prioritize cross-model validation, advanced neuroimaging biomarkers, and multidisciplinary frameworks to translate mechanistic insights into precision therapies. This comprehensive analysis positions Ca²⁺-Fe²⁺ crosstalk as a pivotal frontier in epilepsy research, bridging molecular pathophysiology with innovative treatment paradigms.

2025-08-01·ANTIOXIDANTS & REDOX SIGNALING

Sulforaphane Targets Multiple Pathological Processes in Friedreich Ataxia Patient-Induced Pluripotent Stem Cell-Derived Sensory Neurons

Article

作者: Dottori, Mirella ; Yang, Wenyao ; Thompson, Bruce ; Maddock, Marnie ; Kwa, Faith ; Napierala, Marek ; Miellet, Sara

Aims: In Friedreich ataxia (FRDA), early motor discoordination stems from dysfunctional sensory neurons in the spinal cord driven by epigenetic dysregulation, frataxin (FXN) deficiency, oxidative stress, and inflammation. Omaveloxolone, a nuclear factor erythroid 2-related factor-2 (NRF2) inducer, is the only treatment available. In various chronic disease models, sulforaphane (SF) can target NRF2 and the above processes. This study compared the effects of SF with omaveloxolone and dimethyl fumarate (DMF) in sensory neurons generated from FRDA patient-induced pluripotent stem cells and their isogenic control. Results: The successful generation of the FRDA and isogenic control sensory neurons was confirmed by the positive expression of β-III TUBULIN, BRN3A, ISLET1, PERIPHERIN, and tropomyosin receptor kinase C. In comparison with the isogenic control, FRDA sensory neurons displayed an aberrant gene expression profile alike to that reported in patients. None of the drugs affected the viability of the isogenic control sensory neurons. SF treatment improved the viability of FRDA sensory neurons by up to 61% versus the untreated control. DMF treatment showed a modest 35% increase, while omaveloxolone lacked an effect. SF-treated FRDA sensory neurons demonstrated increased reduced glutathione/oxidized glutathione ratio and expression of FXN and redox markers, and a reduced expression of selected epigenetic enzymes and inflammatory cytokines, at the respective gene and protein levels. DMF and omaveloxolone treatments only modulated some of these biomarkers. Innovation: We revealed the therapeutic potential of SF and how it performs in comparison with omaveloxolone and DMF, in a physiologically and genetically relevant in vitro FRDA model. Conclusion: SF offers a multipronged approach to alleviating the different cellular events underlying FRDA. Antioxid. Redox Signal. 43, 308-327.

146

项与 Omaveloxolone 相关的新闻（医药）

2025-08-20

·渤健生物

2025年度“医药界诺贝尔奖-盖伦奖”提名揭晓，渤健四款产品入围！

2025年8月11日，美国盖伦基金会（The Galien Foundation）公布2025年度盖伦奖（Prix Galien USA Awards）候选名单。渤健旗下四款创新产品，Qalsody™（凯盛迪™）、Leqembi®（乐意保®）、Skyclarys®及Zurzuvae®入围。盖伦奖被誉为“医药界的诺贝尔奖”，是医药和生物医疗行业的最高荣誉，旨在表彰为改善人类健康做出的杰出科学创新1。盖伦奖的候选药品均为在近5年内获得美国食品药品监督管理局（FDA）批准上市的，并且展现出对改善人类健康产生重大影响的产品。 Qalsody（中文名：凯盛迪™）是全球首款获批上市，用于治疗携带超氧化物歧化酶1（SOD1）基因突变的肌萎缩侧索硬化（ALS）成人患者的疾病修正治疗药物2,3。该产品已于2025年6月在中国商业上市4。 ALS是一种进行性神经退行性疾病，患者大脑和脊髓的上下运动神经元受累，导致肌肉无力、萎缩，最终因吞咽困难、呼吸无力而死亡，患者确诊后的平均寿命为3-5年。ALS的发病原因尚未完全明确，目前已知有多个致病基因被证实与ALS的发病有关。其中，SOD1是首个被发现的ALS致病基因，也是中国ALS人群中最常见的致病基因。SOD1-ALS患者的平均发病年龄约为50岁，患者多为脊髓起病，主要特点为下肢起病，典型表现为上下神经元同时受损5。由于疾病进程不可逆，因此“早发现、早诊断、早治疗”对于延缓疾病进展，维持生活质量至关重要。 Leqembi（中文名：乐意保®）是由渤健和卫材共同开发的，20年来首款被FDA完全批准用于治疗阿尔茨海默病（AD）的疾病修正治疗药物6。该产品已于2024年在中国商业上市7。FDA曾授予其加速审评资格及突破性疗法认定。阿尔茨海默病（AD）是老年人中最常见的神经退行性疾病8，已经成为日益严重的健康危机。全球现存5,000万痴呆患者，其中60-70%是AD源性的痴呆患者9。目前，我国阿尔茨海默病的早期就诊率为28.6%，轻度认知障碍就诊率仅为2.8%。大多数患者确诊时已经是疾病的中晚期，错失了获得早期诊疗的机会。早发现、早诊断、早治疗是最迫切也是最可行的第一步10。 Zurzuvae是FDA批准的，首个用于治疗产后抑郁症（PPD）成人患者的口服疗法11。在此之前，FDA批准的PPD治疗方式仅为静脉注射12。 PPD是一种常见的在女性分娩后出现的精神疾病，其症状可能包括情绪低落、兴趣和愉悦感丧失、睡眠和食欲问题、注意力难以集中、精神运动迟缓或激进、持续的疲劳感、强烈的内疚感以及自我价值感缺失。在极端情况下，患有PDD的产妇甚至可能产生导致自杀的念头或行为13。 *该药品尚未在中国大陆获批上市。 Skyclarys是首款获得FDA批准的，用于治疗16岁以上弗里德赖希共济失调症（Friedreich’sAtaxia，FA）患者的药物14。Skyclarys在2017年6月获得孤儿药认定，并在2021年11月被FDA授予快速通道资格。 FA是一种遗传性的、并会寿命缩短的神经肌肉疾病。大多数患者在青少年时期确诊。患者会出现下肢反射消失，构音障碍，视觉和听觉障碍等症状，并可能在确诊后的10到20年内失去行走能力，平均预期寿命为37岁15。 *该药品尚未在中国大陆获批上市。 Reference： 1. 盖伦奖百度百科：https://baike.baidu.com/item/%E7%9B%96%E4%BC%A6%E5%A5%96/20276181?fr=ge_ala 2. Ionis announced FDA advisory committee voted unanimously for a potential accelerated approval of tofersen for SOD1-ALS. Retrieved April 25, 2023, from https://ir.ionispharma.com/news-releases/news-release-details/ionis-accounces-fda-advisory-committee-voted-unanimously 3. 托夫生注射液说明书（NMPA批准版本：2024-09-26）[EB/OL]. https://www.biogen.cn/content/dam/corporate/apac/china/zh-cn/disease-areas/als/%E6%89%98%E5%A4%AB%E7%94%9F%E6%B3%A8%E5%B0%84%E6%B6%B2%E8%AF%B4%E6%98%8E%E4%B9%A6_202409.pdf 4. 新华网：《渐冻症精准治疗药物凯盛迪在中国商业上市开启渐冻症“对因治疗”新纪元》https://app.xinhuanet.com/news/article.html?articleId=f1ca049e083c14c989648171694c4099×tamp=1205 5. 王诗婵，商慧芳. 肌萎缩侧索硬化基因型和表型研究进展. Chin J Nerv Ment Dis. 2023:49(5):296-304 6. 湖南日报：《20年来首款获得FDA完全批准的阿尔茨海默病新药上市》https://baijiahao.baidu.com/s?id=1770724150205951078&wfr=spider&for=pc 7. 新浪财经：《中国紧随美国和日本之后，成为第三个"乐意保"上市国家》https://cj.sina.com.cn/articles/view/7243395680/1afbd726002001fzu2 8. https://www-niehs-nih-gov.libproxy1.nus.edu.sg/research/supported/health/neurodegenerative 9. 丁香园：《历时五年余，华山医院郁金泰教授牵头制定阿尔茨海默病循证预防国际指南》 https://neuro.dxy.cn/article/718681 10. 中国新闻网：《专家呼吁：多方聚力共促阿尔茨海默病早诊早治》http://m.chinanews.com/wap/detail/zw/jk/2025/03-31/10392193.shtml 11. FDA Approves First Oral Treatment for Postpartum Depression. Retrieved August 4, 2023 from https://www.fda.gov/news-events/press-announcements/fda-approves-first-oral-treatment-postpartum-depression 12. 中国医药创新促进会：《重磅！口服抗产后抑郁疗法获FDA批准上市》 https://www.phirda.com/artilce_32390.html?module=trackingCodeGenerator 13. 王雅倩，马占强. 产后抑郁症的病理机制与治疗药物. Pharmacy Information 药物资讯, 2025, 14(2), 97-105 14. Reata Pharmaceuticals Announces FDA Approval of SKYCLARYS™ (Omavaloxolone), the First and Only Drug Indicated for Patients with Friedreich’s Ataxia. Retrieved February 28, 2023, from https://www.businesswire.com/news/home/20230228006450/en/Reata-Pharmaceuticals-Announces-FDA-Approval-of-SKYCLARYS%E2%84%A2-Omavaloxolone-the-First-and-Only-Drug-Indicated-for-Patients-with-Friedreich%E2%80%99s-Ataxia 15. Corben LA, et al., Clinical Management Guidelines Writing Group. Clinical management guidelines for Friedreich ataxia: best practice in rare diseases. Orphanet J Rare Dis. 2022;17(1):415. Biogen-270863-CHN-08/2025

突破性疗法上市批准加速审批

2025-08-20

·Pharmaceutical Technology

PTC’s vatiquinone approval hopes dashed in Friedreich’s Ataxia

The US Food and Drug Administration (FDA) has rejected PTC Therapeutics ’ new drug application (NDA) for vatiquinone in Friedreich’s Ataxia . This follows the biotech’s decision to submit the 15-lipoxygenase (15-LO) inhibitor for approval in the indication, despite it failing to meet its primary endpoint in the Phase III MOVE-FA trial (NCT04577352). PTC Therapeutics’ vatiquinone for Friedrich ataxia has been rejected by the FDA. Image credit: Piotr Swat / ShutterStock.com. Hedging its bets, PTC applied for FDA approval based on statistically significant improvements observed in two subscales of the modified Friedreich Ataxia Rating Scale (mFARS) in the Phase III trial. These related to bulbar and upright stability improvements, which the company claimed were “reflective of key aspects of disease morbidity” in a December 2024 statement. However, the FDA issued PTC with a complete response letter, stating that the application did not provide substantial evidence of vatiquinone’s efficacy, and that “an additional adequate and well-controlled study would be needed to support NDA resubmission”. Despite the FDA’s decision, PTC Therapeutics’ CEO Matthew Klein said: “We believe the data collected to date demonstrate that vatiquinone could provide a safe and effective therapy for both children and adults living with Friedreich’s Ataxia.” GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence The company plans to meet with the FDA to discuss potential next steps for the drug in this indication. Friedreich’s Ataxia is a rare, inherited disorder that causes progressive damage to the nervous system. This can cause movement and sensory symptoms, as well as trouble with walking and gait. If the FDA were to be convinced of vatiquinone’s efficacy further down the line, it could become the second drug on the market for Friedreich’s Ataxia – joining Biogen’s Skyclarys (omaveloxolone), which was approved in February 2023 . It could also be the first to be authorised for use in a paediatric population, as Skyclarys is only permitted for use in patients aged 16 years or older. According to GlobalData’s Intelligence Center, Skyclarys made $383m in 2024, with analysts predicting that the drug will become a blockbuster in 2029, raking in $1bn for Biogen. GlobalData is the parent company of Pharmaceutical Technology . PTC’s rare disease woes persist Vatiquinone’s FDA rejection is the most recent event in a swathe of disappointing turnouts for the company, as the conditional approval for its nonsense mutation Duchenne muscular dystrophy (DMD) drug Translarna (ataluren) was pulled by the European Medicines Agency (EMA) in January 2024. This follows a back-and-forth between the two between 2023 and 2024, with the regulator’s committee doubling down on its negative stance after PTC submitted further documentation to the EMA, stating that it “could not draw conclusions on the benefits of Translana” from the new data provided. The EMA also cited “differences between the registries and biases making study comparisons inconclusive” as a key reason for the drug’s recent rejection. PTC has had even less luck in its attempts to get Translarna approved in the US, having faced rejections in 2016 and 2017. The first was related to the insufficient completion of the new drug application (NDA) while the FDA cited a lack of clinical evidence as the primary reason for the second application’s rejection. The company has now filed for approval for the third time in the US, with the FDA accepting its application in October 2024. An action date has not yet been provided by the agency. The drug remains available in the UK through the Medicines and Healthcare products Regulatory Agency (MHRA). Pharmaceutical Technology Excellence Awards - The Benefits of Entering Gain the recognition you deserve! The Pharmaceutical Technology Excellence Awards celebrate innovation, leadership, and impact. By entering, you showcase your achievements, elevate your industry profile, and position yourself among top leaders driving pharmaceutical advancements. Don’t miss your chance to stand out—submit your entry today! Nominate Now

临床3期申请上市上市批准

2025-08-01

·抗体圈

渤健 Leqembi 推广现转机，CEO 着眼早期管线储备

摘要：渤健首席执行官克里斯・维赫巴赫在第二季度财报电话会议上表示，与卫材在阿尔茨海默病药物 Leqembi 推广上的合作良好，此前双方不和传言被夸大。本季度 Leqembi 销售额攀升，全球达 1.6 亿美元，美国市场增长 20% 至 6300 万美元。同时，维赫巴赫透露公司将重点关注早期研发项目，通过合作和收购充实管线，为长期发展奠定基础。一、Leqembi 销售增长，市场份额稳固渤健与合作伙伴卫材在阿尔茨海默病药物 Leqembi 的推广上，努力开始收获成果。根据渤健周四发布的第二季度财报，Leqembi 全球销售额达到 1.6 亿美元，其中美国市场销售额增长 20%，达 6300 万美元。这一数据让分析师感到惊喜，尽管他们仍保持谨慎态度。本季度渤健营收增长 7%，达到 26 亿美元。Leqembi 在市场上虽面临礼来 Kisunla 的竞争压力，但据北美业务总裁兼负责人阿丽莎・阿拉伊莫在财报电话会议上称，Leqembi 仍占据 70% 的市场份额。此前，由于医生需适应诊断测试要求及其他挑战，该药物市场推广速度缓慢。不过，阿拉伊莫指出，随着更快、更便捷的生物标志物检测获得批准，生物标志物检测显著增加，更多患者得以诊断并进入治疗流程。她表示：“这远超我们在此时点的预期，我们相信这是一个关键时刻。”首席执行官克里斯・维赫巴赫也提到，基于血液的生物标志物检测有助于 “消除系统中的一些瓶颈”。二、合作关系稳固，回应不和传言渤健与卫材在阿尔茨海默病领域的合作已持续十年。针对因渤健就欧洲商业分配问题提起仲裁，而引发的双方合作关系恶化传言，维赫巴赫在电话会议上进行了回应。他承认多年来双方存在分歧，但强调目前合作关系 “比以往任何时候都要好”。自 2014 年起，双方就联合开发和商业化阿尔茨海默病治疗药物展开合作，卫材主导 Leqembi 全球研发和监管申报，双方共同商业化和推广该产品，且卫材拥有最终决策权。这种合作模式在过去十年中不断磨合，尽管面临挑战，但目前看来仍在稳步推进。三、着眼早期研发，充实管线布局维赫巴赫在电话会议上一如既往地谈到了渤健的业务发展努力。他明确表示：“早期研发项目绝对在计划之中。” 未来，公司将达成更多类似与 City Therapeutics 的研究合作。今年 5 月，渤健与 City Therapeutics 达成合作，支付 4600 万美元预付款，里程碑付款最高可达 10 亿美元，用于开发中枢神经系统疾病的新型 RNAi 疗法。维赫巴赫将早期项目搜寻比作以 18 亿美元收购 Hi - Bio 的行动。他解释称，收购 Hi - Bio 后，渤健在整合的同时保留其完整性，这使得该生物技术公司能够在渤健全球临床试验网络的支持下发挥其专长。他补充道：“这就是我们希望在早期阶段所做的事情。” 通过此类合作与收购，渤健旨在充实早期研发管线，为公司未来发展注入新动力，尤其是在竞争激烈的神经科学领域。四、全年营收预期调整，发展前景可期尽管 Leqembi 销售表现良好，但渤健预计下半年营收增长将放缓。基于此，公司上调了全年业绩指引，预计全年营收将保持平稳增长，营收指引从之前的 145 - 155 亿美元上调至 155 - 160 亿美元。这一调整反映了公司对当前业务状况的评估以及对未来发展的信心。在多发性硬化症（MS）业务方面，由于 Tecfidera 在全球面临仿制药竞争，Tysabri 在欧洲遭遇生物类似药竞争，MS 业务营收下降 4%，至 11 亿美元。不过，罕见病药物业务，如遗传性疾病药物 Skyclarys，营收增长近 2%，达 5.43 亿美元，在一定程度上抵消了 MS 业务的下滑。随着 Leqembi 市场份额的巩固和早期研发管线的逐步充实，渤健在神经科学领域有望迎来新的发展阶段。未来，公司如何平衡现有产品推广与早期研发投入，将持续受到行业和投资者的关注。但就目前而言，Leqembi 销售增长带来的积极信号，以及在早期研发布局上的明确规划，为渤健的未来发展奠定了较为坚实的基础。参考来源：https://www.biospace.com/business/biogens-leqembi-push-getting-easier-as-ceo-eyes-early-stage-pipeline-restock识别微信二维码，添加抗体圈小编，符合条件者即可加入抗体圈微信群！请注明：姓名+研究方向！本公众号所有转载文章系出于传递更多信息之目的，且明确注明来源和作者，不希望被转载的媒体或个人可与我们联系(cbplib@163.com)，我们将立即进行删除处理。所有文章仅代表作者观点，不代表本站立场。

并购财报临床2期

100 项与 Omaveloxolone 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D10964	Omaveloxolone	-	DB12513

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
Friedreich共济失调	美国	Biogen U.S. Corp.	2023-02-28

未上市

10 条进展最快的记录,

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
线粒体肌病	临床2期	美国	Biogen, Inc.	2015-05-05
线粒体肌病	临床2期	丹麦	Biogen, Inc.	2015-05-05
转移性黑色素瘤	临床2期	美国	Biogen, Inc.	2014-10-31
不可切除的黑色素瘤	临床2期	美国	Biogen, Inc.	2014-10-31
乳腺癌	临床2期	美国	Biogen, Inc.	2014-06-30
放射性皮炎	临床2期	美国	Biogen, Inc.	2014-06-30
白内障	临床2期	美国	Biogen, Inc.	2014-05-31
角膜内皮细胞损失	临床2期	美国	Biogen, Inc.	2014-05-31
核性白内障	临床2期	美国	Biogen, Inc.	2014-05-31
眼科外科手术	临床2期	美国	Biogen, Inc.	2014-05-31

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临床结果

适应症

分期

评价

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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


MDA2024	临床1期	-	32	Omaveloxolone Intact Capsules	壓餘網願鬱鹽襯獵艱選(憲廠鹹製夢廠鑰網鑰獵) = 積遞窪顧夢糧廠選網繭艱願壓鬱艱襯顧鏇製廠 (鑰膚繭齋糧遞醖餘構壓, 17.2) 更多	积极	2024-03-03
				Omaveloxolone Sprinkled on Applesauce	壓餘網願鬱鹽襯獵艱選(憲廠鹹製夢廠鑰網鑰獵) = 選鏇觸窪廠鬱憲鹹製膚艱願壓鬱艱襯顧鏇製廠 (鑰膚繭齋糧遞醖餘構壓, 19.7) 更多
NCT02128113 (CTgov)	临床2期	白内障 \| 眼睛疼痛 \| 角膜内皮细胞损失 ... 更多	307	Vehicle Ophthalmic Solution (Vehicle Ophthalmic Solution)	憲醖顧鹹廠襯顧獵簾衊(獵壓醖積壓築齋積繭蓋) = 蓋鏇觸簾夢鹽鑰構淵衊鬱範築壓繭獵艱鹹窪膚 (簾鬱繭壓繭艱醖遞選鹹, 340.58) 更多	-	2023-05-26
				Omaveloxolone Ophthalmic Suspension 0.5% (Omaveloxolone Opthalmic Suspension 0.5%)	憲醖顧鹹廠襯顧獵簾衊(獵壓醖積壓築齋積繭蓋) = 鹽蓋構製壓艱鹽願夢襯鬱範築壓繭獵艱鹹窪膚 (簾鬱繭壓繭艱醖遞選鹹, 237.00) 更多
NCT02142959 (CTgov)	临床2期	乳腺癌 \| 放射性皮炎	187	3D conformal radiation therapy+Omaveloxolone Lotion 0.5% (Omaveloxolone (RTA 408) Lotion 0.5%)	餘壓範繭築鏇襯夢餘鬱(壓蓋顧窪顧艱齋艱夢願) = 積選積壓壓齋齋襯淵獵網醖顧鬱醖願襯鑰餘選 (襯鑰製鹽襯選鬱齋蓋築, 選鬱選簾鹽選鑰廠鏇壓 ~ 構獵窪築遞鏇鹹蓋鑰壓) 更多	-	2023-05-06
				3D conformal radiation therapy+Omaveloxolone Lotion 3% (Omaveloxolone (RTA 408) Lotion 3%)	餘壓範繭築鏇襯夢餘鬱(壓蓋顧窪顧艱齋艱夢願) = 餘衊廠淵廠糧鹹艱襯鬱網醖顧鬱醖願襯鑰餘選 (襯鑰製鹽襯選鬱齋蓋築, 餘構鏇獵鏇選製積鹹觸 ~ 廠窪構壓願鹽築鏇願艱) 更多
NCT02255435 (MOXIe, MDA2023) 人工标引	临床2期	Friedreich共济失调	103	Omaveloxolone 150 mg	鹹簾鹽糧鑰顧醖網鹹廠(襯夢艱顧鑰鹽窪蓋餘壓) = 選積構鬱積餘鑰鏇鬱築範蓋膚淵憲鏇夢憲製鬱 (鹹鹽夢遞選簾願壓願廠 ) 更多	积极	2023-03-19
				Placebo	鹹簾鹽糧鑰顧醖網鹹廠(襯夢艱顧鑰鹽窪蓋餘壓) = 獵憲積鑰膚襯築衊築選範蓋膚淵憲鏇夢憲製鬱 (鹹鹽夢遞選簾願壓願廠 ) 更多
NCT02255435 (MOXIe, MDA2023)	临床2期	Friedreich共济失调 Nrf2	-	Omaveloxolone	夢窪衊積網鹹餘觸壓夢(憲積構壓願衊餘淵憲遞) = 鏇襯鬱鹹艱鏇願窪製糧鬱鑰鹽憲醖餘廠廠窪範 (餘夢網壓夢憲憲艱築蓋, 1.09) 更多	积极	2023-03-19
				Placebo	夢窪衊積網鹹餘觸壓夢(憲積構壓願衊餘淵憲遞) = 觸顧範範鏇範鹹衊蓋願鬱鑰鹽憲醖餘廠廠窪範 (餘夢網壓夢憲憲艱築蓋, 1.44) 更多
NCT02255435 (FDA) 人工标引	临床2期	Friedreich共济失调	103	SKYCLARYS 150 mg	壓簾觸憲廠獵壓遞積壓(襯壓淵構窪構鬱淵齋窪) = 製鏇簾淵網餘艱壓構廠鬱齋糧鹽範醖糧遞鬱構 (壓顧構衊積鹹廠獵鹹繭 )	积极	2023-02-28
				placebo	壓簾觸憲廠獵壓遞積壓(襯壓淵構窪構鬱淵齋窪) = 繭選憲願鬱製鬱糧艱鹽鬱齋糧鹽範醖糧遞鬱構 (壓顧構衊積鹹廠獵鹹繭 )
NCT02255435 (MOXIe, Corporate Publications) 人工标引	临床2期	Friedreich共济失调	-	Omaveloxolone	鬱觸淵憲鏇築鏇鏇餘衊(糧膚顧齋選壓顧衊蓋淵): difference = -2.17±1.09 更多	积极	2022-08-08
				Placebo
NCT02255435 (MOXIe, MDA2022)	临床2期	Friedreich共济失调 Nrf2	-	Omaveloxolone	願餘願積窪鏇簾壓餘簾(艱鏇獵鏇網構壓襯鑰構) = 鹽顧構築膚積遞餘鑰夢齋觸淵壓鑰選鏇鏇窪衊 (夢襯衊壓簾顧網鹽鹹夢, 1.45) 更多	积极	2022-03-13
				Placebo	願餘願積窪鏇簾壓餘簾(艱鏇獵鏇網構壓襯鑰構) = 餘顧製夢繭製網齋壓築齋觸淵壓鑰選鏇鏇窪衊 (夢襯衊壓簾顧網鹽鹹夢, 1.07) 更多
NCT02259231 (CTgov)	临床1/2期	转移性黑色素瘤 \| 不可切除的黑色素瘤	41	Ipilimumab (3 mg/kg)+Omaveloxolone Capsules (2.5 mg/capsule) (Omaveloxolone 5 mg & Ipilimumab)	網餘鹹膚襯構衊壓網網 = 壓淵鬱鏇鹽夢壓鹽膚築廠鹽鹽糧鏇網糧壓衊鏇 (鏇簾積窪窪憲廠艱積選, 選艱憲蓋獵憲襯選觸餘 ~ 鏇鏇願鑰憲醖鬱構艱蓋) 更多	-	2021-06-24
				Ipilimumab (3 mg/kg)+Omaveloxolone Capsules (10 mg/capsule) (Omaveloxolone 10 mg & Ipilimumab)	網餘鹹膚襯構衊壓網網 = 齋醖範簾醖壓艱鹽顧餘廠鹽鹽糧鏇網糧壓衊鏇 (鏇簾積窪窪憲廠艱積選, 餘醖鏇鹽願鏇選齋繭繭 ~ 夢製鹽糧窪廠遞糧窪餘) 更多
NCT02255435 (MOXIe, Pubmed \| Ann Neurol) 人工标引	临床2期	Friedreich共济失调	103	Omaveloxolone	簾獵壓築憲憲構夢構築(鹹願壓積鹹壓築鬱構構) = 築獵鏇糧鬱網積製觸憲窪淵憲遞積壓餘醖獵艱 (餘餘顧鏇鬱範簾製鏇範, 0.69)	积极	2021-02-01
				Placebo	簾獵壓築憲憲構夢構築(鹹願壓積鹹壓築鬱構構) = 鑰願鹽簾壓蓋鬱壓憲憲窪淵憲遞積壓餘醖獵艱 (餘餘顧鏇鬱範簾製鏇範, 0.64)