A Phase 3 Study With an Open-Label Extension in Pediatric Participants to Evaluate the Safety, Efficacy, and Pharmacokinetics of Diroximel Fumarate and Dimethyl Fumarate for the Treatment of Relapsing Forms of Multiple Sclerosis

In this study, researchers will learn more about the study drugs diroximel fumarate (DRF) and dimethyl fumarate (DMF) in children with MS who may be experiencing relapses.
Participants will be divided into 2 groups based on their weight:
* Group A will include children who weigh 40 kilograms (kg) or less. They will receive DRF in both Part 1 and Part 2 of the study.
* Group B will include children who weigh more than 40 kg. They will be randomly assigned to receive DRF, DMF, or fingolimod. Fingolimod is a drug already used to treat MS in adults. In Part 2, they will receive DRF.
This is a 2-part study. Part 1 treatment will last 96 weeks. Participants who complete Part 1 may move to Part 2. Part 2 is an extension period. Treatment will last another 96 weeks and will help researchers learn about the long-term safety and effects of treatment.
The main goal of the study is to learn about the safety of DRF and DMF and compare their effect on relapses and brain lesions with fingolimod.
The main questions researchers want to answer are:
* How many participants have adverse events and serious adverse events?
* How often do participants relapse after treatment with DRF and DMF compared to fingolimod?
Researchers will take brain imaging scans to check for any new areas of brain inflammation and compare the brain lesions before and after treatment.
Researchers will also measure the amount of drug in the blood to understand how the body processes it. To check safety, they will monitor participants' growth and development, and compare changes in heart tests, vital signs, and lab tests. They will also use rating scales to monitor depression symptoms.
The study will be done as follows:
Part 1 (Treatment Period)
* After screening, participants will join Part 1 and be divided into 2 groups based on their weight.
* Participants in Group A will receive DRF.
* Participants in Group B will be randomly assigned to receive either DRF, DMF, or fingolimod.
* Neither the researchers nor the participants will know which drug or dose the participants will receive in Group B. Participants in Group B will also receive a placebo so they do not know which drug is being given. A placebo looks like a study drug but contains no real medicine.
* All study drugs will be taken by mouth, once or twice a day. Participants who take DRF or DMF will start with a lower dose during the 1st week, then move to a standard dose.
* Treatment in Part 1 will last for 96 weeks. Participants will have up to 11 study visits and 7 phone calls.
* Participants who do not move onto Part 2 will also have a safety follow-up period of 4 to 8 weeks. This will include 1 study visit and 1 phone call.
* The total length of Part 1, including the screening, treatment, and follow-up, will be up to 108 weeks.
Part 2 (Extension Period)
* Participants who complete Part 1 can move on to Part 2 of the study.
* All participants in Part 2 will receive DRF by mouth for 96 weeks.
* Participants will have up to 10 more study visits and 1 telephone call during treatment.
* They will also have a 4-week safety follow-up, including 1 study visit and 1 phone call.
* The total length of Part 2 will be up to 100 weeks.

开始日期2026-11-16

申办/合作机构

Biogen, Inc.

NCT07548996

/ Recruiting临床2/3期IIT

Non-Randomized, Parallel-Controlled, Single-Center, Open-Label Clinical Trial Evaluating the Efficacy and Safety of Dimethyl Fumarate in Preserving Pancreatic β-Cell Function in Adults With Type 1 Diabetes

This is a non-randomized, parallel-controlled, single-center, open-label clinical trial designed to evaluate the efficacy of dimethyl fumarate in preserving pancreatic beta-cell function in adults with type 1 diabetes, as well as its safety and tolerability in this population.
Eligible participants are adults aged 18 to 65 years who meet the ADA 2024 diagnostic criteria for type 1 diabetes, have at least 2 positive islet autoantibodies, and have residual beta-cell function as evidenced by a random C-peptide level of at least 200 pmol/L. A total of 96 participants are planned for enrollment, including 32 in the dimethyl fumarate treatment group and 64 in the standard-treatment control group.
Participants in the treatment group will receive dimethyl fumarate enteric-coated capsules in addition to standard insulin therapy for type 1 diabetes. Dimethyl fumarate will be initiated at 120 mg twice daily and increased after 7 days to a maintenance dose of 240 mg twice daily. Participants in the control group will receive standard insulin therapy alone. The intervention period will be 24 weeks, followed by 52 weeks of follow-up.
The primary efficacy endpoint is the baseline-adjusted geometric mean area under the serum C-peptide curve during a 2-hour mixed-meal tolerance test at Week 24. Secondary endpoints include measures of beta-cell function at multiple time points, changes in glycated hemoglobin, proportions of participants with good or poor glycemic control, insulin dose requirements, and immunologic markers including lymphocyte subsets, cytokine profiles, and islet autoantibody characteristics. Safety assessments will include the incidence of flushing, gastrointestinal adverse events, allergic reactions, opportunistic infections, liver function abnormalities, lymphopenia, renal abnormalities, hypoglycemia, severe hypoglycemia, ketosis, and ketoacidosis.
The total study duration is 36 months, from January 2026 to December 2028.

开始日期2026-04-27

申办/合作机构

南京医科大学

NCT07258394

/ Recruiting临床3期IIT

A Single-Center, Randomized, Double-Blind, Placebo-Controlled Clinical Trial Evaluating the Efficacy and Safety of Dimethyl Fumarate in Preserving Islet β-Cell Function in Patients With Type 1 Diabetes Mellitus

Purpose of the Clinical Trial:
This clinical trial aims to investigate whether dimethyl fumarate can treat adults with newly diagnosed type 1 diabetes and to evaluate the safety profile of dimethyl fumarate.
Primary Research Questions:
Does dimethyl fumarate protect pancreatic beta-cell function in adults with newly diagnosed type 1 diabetes? What medical issues may arise in individuals taking dimethyl fumarate?
Study Design:
Researchers will compare dimethyl fumarate with a placebo (an identical substance without active ingredients) to determine whether Dimethyl fumarate can effectively treat type 1 diabetes.
Participant Activities:
Take dimethyl fumarate or placebo orally twice daily for 24 weeks. Attend on-site visits every 4 weeks during the intervention period and every 12 weeks after the intervention for examinations and assessments.
Record symptoms, blood glucose control, islet function, and insulin usage throughout the trial.

开始日期2026-01-27

申办/合作机构

南京医科大学

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100 项与富马酸二甲酯相关的临床结果

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100 项与富马酸二甲酯相关的转化医学

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100 项与富马酸二甲酯相关的专利（医药）

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项与富马酸二甲酯相关的文献（医药）

2026-07-01·CELLULAR SIGNALLING

Dimethyl fumarate alleviated DSS-induced colitis by regulating Th17/Treg balance via suppressing JAK2/STAT3 and NF-κB signaling

Article

作者: Liang, Jinmeiqi ; Dai, Ao ; Du, Boyu ; Wang, Xiaodong ; Lin, Disi ; Jin, Lan ; Guo, Fuxin ; Guo, Yang ; Xi, Xueyan ; Yang, Peiqiong ; Wang, Yajie

Ulcerative colitis (UC) is a chronic inflammatory bowel disease characterized by immune dysregulation. Restoring Th17/Treg balance represents a promising therapeutic strategy for UC. Gut metabolism has been reported to critically regulates Th17 and Treg cell homeostasis. However, the role of fumarate in the pathogenesis of UC and the immunoregulatory effect of Dimethyl fumarate (DMF) in the treatment of UC remain unclear. Herein, results demonstrated decreased fumarate levels in colonic tissues of DSS-induced colitis mice, whereas DMF administration elevated colonic fumarate concentrations and relieved DSS-induced colitis by suppressing the inflammatory response and pro-inflammatory cytokines in vivo. Moreover, DMF treatment significantly decreased the frequency of Th17 cells, increased the frequency of Treg cells, thus improved Th17/Treg balance in vitro. Mechanistically, DMF significantly inhibited Th17 cell differentiation via interrupting JAK2/STAT3 and NF-κB signaling. Taken together, these results indicated that DMF alleviated DSS-induced UC by suppressing JAK2/STAT3 and NF-κB signaling to restore Th17/Treg balance. Furthermore, these findings preliminary demonstrate a role for fumarate in the pathogenesis of UC and suggest that DMF treatment leading to fumarate accumulation is a potential metabolic mechanism for colitis mitigation. Collectively, DMF-mediated immunometabolic reprogramming constitutes a novel strategy for UC immunotherapy.

2026-07-01·EXPERIMENTAL NEUROLOGY

Nrf2 modulates the IRF7–SLC31A1 axis to suppress neuronal cuproptosis after traumatic brain injury

Article

作者: Sun, Boyu ; Sun, Guozhu ; Feng, Shiyao ; Tian, Longlong ; Li, Mingkang

Traumatic brain injury (TBI) is one of the most prevalent neurological disorders and a major cause of death and disability worldwide. Cuproptosis is a recently identified form of cell death. Nuclear factor erythroid 2-related factor 2 (Nrf2), a key transcription factor that has been widely investigated in TBI, may contribute to the regulation of cuproptosis. In this study, we investigated the role of cuproptosis in TBI and the regulatory mechanisms of Nrf2. We found that cuproptosis was induced following TBI in both patients and mouse models. In TBI mice, cuproptosis peaked at 3 days post-injury and predominantly occurred in neurons. Nrf2 deficiency aggravates neuronal cuproptosis after TBI, primarily through IRF7-mediated upregulation of SLC31A1. The Nrf2 activator dimethyl fumarate (DMF) suppresses neuronal injury and improves neurological function in TBI mice by inhibiting cuproptosis. Our results establish that cuproptosis is an important mechanism in TBI and that activation of Nrf2 mitigates TBI-induced cuproptosis by regulating the IRF7-SLC31A1 axis.

2026-06-01·PHYTOMEDICINE

Effects of a combination of olive leaf extract and potassium on blood pressure in participants with mild to moderate hypertension: A double blind, randomized, placebo-controlled trial

Article

作者: Schön, Christiane ; Menzel, Daniel ; Fladerer-Grollitsch, Johannes-Paul ; Kompek, Albert ; Klein, Thomas ; Bucar, Franz

BACKGROUND:

Hypertension is a key modifiable driver of cardiovascular risk and often clusters with dyslipidemia and insulin resistance within the metabolic syndrome. Olive leaf extract (OLE) and potassium have traditionally been used to support blood pressure (BP) management and cardiometabolic health.

PURPOSE:

To investigate the effect of this OLE - potassium combination on BP and key cardiometabolic markers.

STUDY DESIGN:

In this randomized, double-blind, placebo-controlled study, 70 adults with untreated mildly to moderately elevated BP received an OLE - potassium supplement (1000 mg OLE, ≥160 mg oleuropein, 300 mg potassium/day) or placebo for 12 weeks.

RESULTS:

While office BP fell significantly over time in both groups, 7-day home-based BP monitoring showed a significant greater response, with morning systolic BP decreasing by 5.4 mmHg, in the verum group. Supplementation also reduced total cholesterol by 11.1 mg/dL, LDL-cholesterol by 6.9 mg/dL, and triglycerides by approximately 22 mg/dL at Week 6, while HDL-cholesterol remained stable. Fasting glucose and HbA1c were unchanged, but fasting insulin and HOMA-Index declined in the verum group, indicating a modest improvement in insulin sensitivity. Additionally, oxidized LDL decreased by ∼23 %, without changes in hs-CRP. Quality-of-life scores remained stable, although global self-ratings favoured the combination. The intervention was well tolerated, with no product-related serious adverse events.

CONCLUSION:

The combination of OLE and potassium is a safe adjunctive strategy for individuals with mildly to moderately elevated BP, with favourable effects on lipid profile, oxidative status, and insulin sensitivity that may help reduce cardiometabolic risk in the context of metabolic syndrome.

393

项与富马酸二甲酯相关的新闻（医药）

2026-05-02

·生物通

一种共价变构分子胶降解剂抑制NRF2依赖性肿瘤生长

NRF2转录因子在癌症中组成型激活，其功能是维持氧化稳态并重编程细胞代谢。NRF2活性肿瘤表现出NRF2依赖性和对化疗/放疗（RT）的抵抗。本研究表征了VVD-065，这是一种首创（first-in-class）NRF2抑制剂，通过前所未有的变构分子胶机制发挥作用。在无应激或突变的情况下，NRF2被Kelch样ECH关联蛋白1（KEAP1）–cullin3（CUL3）泛素连接酶复合物快速降解。VVD-065特异性且共价地结合KEAP1上的Cys151，进而促进KEAP1–CUL3复合物的形成，导致NRF2降解增强。先前报道的Cys151定向化合物会减少KEAP1–CUL3相互作用并稳定NRF2，从而确立KEAP1C151作为KEAP1–CUL3复合物和NRF2稳定性的可调节调节器。VVD-065抑制了NRF2依赖性肿瘤生长并使癌症对化疗/放疗增敏，支持了正在进行的I期临床试验（NCT05954312）。研究背景与立论依据 NRF2（由NFE2L2基因编码）作为细胞应对氧化和亲电应激的主要防御转录因子，在正常生理状态下受KEAP1-CUL3 E3泛素连接酶复合物的严格调控。然而，在肺癌、食管癌等多种实体瘤中，NRF2通路频繁发生遗传性或非遗传性异常激活，赋予肿瘤细胞显著的氧化还原稳态、代谢重编程及免疫抑制能力，同时导致肿瘤对化疗和放疗产生强烈抵抗。尽管NRF2作为药物靶点的潜力已被广泛认可，但由于其属于转录因子，缺乏传统的酶活性口袋，长期以来被视为“难成药”靶点。此外，现有的KEAP1抑制剂（如富马酸二甲酯、巴多索隆甲酯）均通过修饰KEAP1的Cys151来抑制其功能，反而导致NRF2的稳定和激活，这与治疗癌症所需的NRF2抑制策略背道而驰。因此，开发能够直接降解NRF2或增强其生理性降解机制的新型疗法成为亟待解决的临床需求。关键技术方法研究人员采用了基于质谱（MS）的化学蛋白质组学平台筛选共价片段库，利用工程化的转录报告基因（HEK293–ARE-Luc）评估功能影响，并通过晶体学解析了KEAP1 BTB结构域与化合物的复合物结构（PDB ID: 9DU7）。在机制验证层面，运用了均相时间分辨荧光（HTRF）检测蛋白互作、表面等离子共振（SPR）测定动力学参数、miniTurbo邻近标记技术以及泛素化分析。体内外药效评价涵盖了异种移植瘤模型（CDX）、患者来源异种移植瘤模型（PDX）以及同基因原位模型，并结合药代动力学（PK）和药效学（PD）分析进行了综合评估。研究结果 KEAP1_Cys151配体的鉴定与功能反转研究人员通过工业化的靶向质谱化学蛋白质组学筛选，发现了一系列共价配体。其中，VVD-065（VVD-325的对映异构体）表现出独特的生物学特性。不同于已知的KEAP1抑制剂（如PSTC、巴多索隆甲基）通过结合Cys151来抑制KEAP1功能，VVD-065虽然同样靶向KEAP1_Cys151，却导致了NRF2的降解而非积累。在KYSE70食管鳞癌细胞中，VVD-065能有效降低NRF2蛋白水平及其靶基因转录活性，且该效应依赖于CUL3和KEAP1_Cys151，显示出极高的选择性。 VVD-065的结构与机制评估为阐明VVD-065的作用机理，研究人员解析了其结合的KEAP1 BTB结构域晶体结构。结果显示，VVD-065的丙烯酰胺端与Cys151形成共价键，并诱导该残基发生构象翻转（从m-rotamer转变为p-rotamer），进而引发BTB结构域内M147、F52等疏水残基的重排以及螺旋6的位移。这种构象变化使得KEAP1呈现出与CUL3结合时的特征性构象。生化实验证实，VVD-065显著增强了KEAP1与CUL3的结合亲和力（KD从783 nmol/L提升至65 nmol/L），降低了复合物的解离速率，其作用机制被定义为一种变构“分子胶”，通过稳定KEAP1–CUL3相互作用来促进NRF2的泛素化降解。 VVD-065活性依赖于KEAP1–NRF2相互作用研究发现VVD-065的抑癌活性严格依赖于KEAP1与NRF2之间的残余相互作用。在携带NFE2L2突变（如W24C、G31A）或KEAP1锚定区突变的细胞中，VVD-065能有效诱导NRF2降解并抑制转录活性；而在破坏KEAP1–NRF2结合界面的突变体（如NRF2E79K、KEAP1G333C）中，VVD-065则无效。这表明只有保留一定结合能力的“机制敏感”型肿瘤才能响应VVD-065的治疗。 VVD-065抑制癌细胞生长在非粘附性的3D球体培养条件下，VVD-065显著抑制了KYSE70细胞的增殖，这与NRF2在三维微环境中的依赖性相符。在一系列细胞系中，VVD-065的抗增殖效应与其诱导NRF2降解的能力呈正相关。将KEAP1_Cys151突变为丝氨酸则完全消除了VVD-065的抑制作用，证实了其为靶向依赖性效应。 VVD-065的体内抗肿瘤效应在KYSE70和HCC95异种移植瘤模型中，口服VVD-065表现出良好的药代动力学性质和剂量依赖性的肿瘤生长抑制（TGI）。药效学分析显示，给药后肿瘤内KEAP1_Cys151被显著占据，伴随NRF2蛋白及其靶基因的持续下调。更为广泛的患者来源异种移植瘤（PDX）模型筛选表明，VVD-065在鳞状非小细胞肺癌（sqNSCLC）、食管鳞癌（ESCC）及头颈鳞癌（HNSCC）等多种组织学类型中均诱导了不同程度的TGI，且疗效与NRF2通路的突变状态密切相关。 VVD-065在同基因原位模型中的强效抗肿瘤作用考虑到免疫微环境对NRF2活性的潜在影响，研究人员在免疫功能完整的同基因原位模型中进行了验证。尽管KLN-205细胞在体外3D球体中对抗NRF2降解，但在DBA/2小鼠的肺原位移植模型中，VVD-065治疗显著延长了荷瘤动物的中位总生存期（OS），并在异体皮下移植模型中显示出显著的抑瘤效果。 VVD-065与化疗和放疗的联合获益鉴于NRF2上调与放化疗抵抗相关，研究人员探索了联合治疗策略。结果显示，VVD-065与顺铂联用在KYSE70和VMRC-LCP模型中产生了显著的协同抑瘤效果。此外，VVD-065还能有效增敏nab-紫杉醇、5-氟尿嘧啶及吉西他滨的疗效。在MOC1头颈癌细胞同基因模型中，VVD-065联合放射治疗（RT）几乎完全抑制了肿瘤生长，证实了该分子胶在克服肿瘤治疗抵抗方面的巨大潜力。结论与意义综上所述，本研究报道了一种首创的KEAP1依赖性变构分子胶降解剂VVD-065。该分子通过共价结合KEAP1_Cys151，诱导KEAP1构象重排，以分子胶机制稳定KEAP1–CUL3复合物，从而恢复NRF2的生理性降解。VVD-065在多种NRF2活化肿瘤模型中表现出单药抗肿瘤活性，并能有效逆转肿瘤对化疗和放疗的耐药性。该研究不仅为NRF2这一“难成药”靶点提供了全新的药物研发范式，也为携带NRF2通路异常激活的癌症患者带来了新的治疗希望。基于这些坚实的临床前数据，VVD-065已进入I期临床试验（NCT05954312），标志着转化医学的重要突破。

临床1期蛋白降解靶向嵌合体引进/卖出

2026-04-28

·PRNewswire

Immunic Appoints Accomplished Biopharmaceutical Executive Michael A. Panzara, M.D., M.P.H., as Chief Medical Officer

– Proven Leader in Neurology Drug Development, Including for Several Approved Multiple Sclerosis Therapies – – Strong Experience in Advancing Late-Stage Clinical Programs and Obtaining Global Regulatory Approvals, Key to Supporting Vidofludimus Calcium Through Pivotal Development – NEW YORK, April 28, 2026 /PRNewswire/ -- Immunic, Inc. (Nasdaq: IMUX), a late-stage biotechnology company pioneering the development of novel oral therapies for neurologic diseases, today announced the appointment of Michael A. Panzara, M.D., M.P.H., as Chief Medical Officer, effective April 24, 2026. Dr. Panzara will lead the company's development organization, including clinical development, medical affairs, and regulatory affairs, and will be a critical partner to the Chief Executive Officer and the Board of Directors in defining and driving the overall company strategy. Dr. Panzara succeeds Andreas Muehler, M.D., M.B.A.. "Mike is a globally renowned biopharmaceutical executive and drug developer with deep expertise in neurology and proven leadership in advancing transformational therapies through development and the regulatory approval process," stated Daniel Vitt, Ph.D., Chief Executive Officer of Immunic. "Notably, during his tenure at Sanofi Genzyme, Mike oversaw the global regulatory approvals of the multiple sclerosis (MS) drugs LEMTRADA® and AUBAGIO®. During his time at Biogen, he served as global clinical lead for the development of TYSABRI® and managed their late-stage portfolio of MS products. His depth of experience will be invaluable as we advance vidofludimus calcium through the pivotal clinical readout of our phase 3 ENSURE trials in relapsing MS, and toward potential regulatory approval and commercialization in this indication. His leadership will support the company strategy as we continue our transition into a fully integrated commercial-stage company." "This is a critically important time in Immunic's evolution and I am excited to be joining the company at such a pivotal inflection point," said Dr. Panzara. "Vidofludimus calcium's potential to treat MS by targeting both immunological and neuroprotective pathways holds promise to redefine the treatment landscape and meaningfully impact the lives of so many people who continue to struggle with their MS. I look forward to building upon the team's successes to further advance Immunic's clinical programs, including the phase 3 ENSURE trials and the planned phase 3 trial of vidofludimus calcium in primary progressive MS." Dr. Panzara brings over 25 years of global neurology experience to Immunic. He most recently served as Chief Medical Officer of Neurvati Neurosciences, Inc., where he built a Development Organization to support the creation of sub-companies focused on advancing therapeutic candidates in neurological and psychiatric disorders. This included serving as Chief Medical Officer of Neurvati's first sub-company, GRIN Therapeutics, overseeing development of radiprodil, a negative allosteric modulator targeting the NMDA receptor as a potential treatment for rare genetically defined neurodevelopmental disorders. Prior to Neurvati, Dr. Panzara served as Chief Medical Officer and Head of Therapeutics Discovery and Development at Wave Life Sciences Ltd., after initially joining the company as Franchise Lead, Neurology, where he oversaw Wave's therapeutic research and development portfolio with a focus on genetically-defined neurological diseases. Earlier, he served as the Head of the Multiple Sclerosis, Neurology and Ophthalmology Therapeutic Area for Global Development at Sanofi Genzyme, where he oversaw global regulatory approvals of the MS drugs LEMTRADA® (alemtuzumab) and AUBAGIO® (teriflunomide) and was responsible for development strategy and oversight within these therapeutic areas. Prior to joining Sanofi Genzyme, Dr. Panzara was Vice President and Chief Medical Officer of Neurology at Biogen. During his time there, he served as the global clinical lead for the development of TYSABRI® (natalizumab) for MS, overseeing its clinical program and global approvals, and managed clinical development activities for all late-stage MS products, including AVONEX® (interferon beta-1a), PLEGRIDY® (PEG-interferon beta-1a), and TECFIDERA® (dimethyl fumarate). He currently serves on the Boards of Directors of LeonaBio, Inc. and Cadenza Bio, Inc.. Dr. Panzara received his undergraduate degree in biology from the University of Pennsylvania and his medical degree from Stanford University School of Medicine. He completed his neurology training at Massachusetts General Hospital and conducted postdoctoral training in immunology and rheumatology at Brigham and Women's Hospital. He also holds a Master of Public Health from the Harvard School of Public Health. Dr. Vitt added, "On behalf of the entire team, I would like to sincerely thank Andreas for his leadership and invaluable contributions as Co-Founder and Chief Medical Officer of Immunic. It is due in large part to his expertise and stewardship that vidofludimus calcium has advanced successfully into late-stage clinical development. That said, we are delighted that he will continue to support the company as a consultant during this key phase for Immunic and our evolution into a fully-fledged commercial company." "It has been a true privilege to serve as Chief Medical Officer of Immunic for the past 10 years," said Dr. Muehler. "During this time, I had the opportunity to build and grow the clinical development organization and advance the company's development pipeline. Most notably, we progressed vidofludimus calcium from the preclinical stage into late-stage clinical development, achieving clinical proof-of-concept in both relapsing and progressive MS, and are now just months away from the pivotal phase 3 readout in relapsing MS. This journey has truly been a team effort. I have been fortunate to work alongside an exceptional group of colleagues, whose dedication and expertise made these achievements possible. I am delighted to pass the baton to Dr. Panzara, whose extensive medical, clinical and regulatory background makes him ideally suited to guide vidofludimus calcium into its next chapter. I am immensely proud of what our team has accomplished and remain highly confident in Immunic's potential to make a meaningful impact in the MS space by delivering a unique and innovative new therapeutic option for patients living with this devastating disease." The Compensation Committee of Immunic's Board of Directors granted Dr. Panzara an initial equity option to purchase 300,000 shares of common stock of the company under the Immunic, Inc. 2026 Inducement Equity Compensation Plan (the "Options"). The Options were granted as an inducement material to Dr. Panzara's commencement of employment pursuant to NASDAQ Listing Rule 5635(c)(4). The Options will be time vested, with one half vesting on the one-year anniversary of April 24, 2026 and one half vesting in equal monthly installments over a period of twenty-four (24) months following the first anniversary of April 24, 2026. About Immunic, Inc. Immunic, Inc. (Nasdaq: IMUX) is a late-stage biotechnology company pioneering the development of novel oral therapies for neurologic diseases. The company's lead development program, vidofludimus calcium (IMU-838), is currently in phase 3 clinical trials for the treatment of relapsing multiple sclerosis, for which top-line data is expected to be available by the end of 2026. It has already shown therapeutic activity in phase 2 clinical trials in relapsing-remitting multiple sclerosis, progressive multiple sclerosis and other diseases. Vidofludimus calcium combines neuroprotective effects, through its mechanism as a first-in-class nuclear receptor-related 1 (Nurr1) activator, with additional anti-inflammatory and anti-viral effects, by selectively inhibiting the enzyme dihydroorotate dehydrogenase (DHODH). The company's development pipeline also includes earlier-stage programs, including IMU-856 and IMU-381, aimed at building a broader therapeutics platform addressing neurodegenerative, chronic inflammatory, and autoimmune-related diseases. For further information, please visit: . Cautionary Statement Regarding Forward-Looking Statements This press release contains "forward-looking statements" that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in this press release regarding strategy, future operations, future financial position, future revenue, projected expenses, sufficiency of cash and cash runway, expected timing, development and results of clinical trials, prospects, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to expectations regarding the appointment of Dr. Panzara and his integration into the company; Immunic's development programs and the targeted diseases; the potential for Immunic's development programs to safely and effectively target diseases; preclinical and clinical data for Immunic's development programs; the timing of current and future clinical trials, anticipated clinical milestones and regulatory approvals; the nature, strategy and focus of the company and further updates with respect thereto; the development and commercial potential of any product candidates of the company; expectations regarding the capitalization, resources and ownership structure of the company; and the executive and board structure of the company. Immunic may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Such statements are based on management's current expectations and involve substantial risks and uncertainties. Actual results and performance could differ materially from those projected in the forward-looking statements as a result of many factors, including, without limitation, increasing inflation, tariffs and macroeconomics trends, impacts of the Ukraine – Russia conflict and the conflict in the Middle East on planned and ongoing clinical trials, risks and uncertainties associated with the ability to project future cash utilization and reserves needed for contingent future liabilities and business operations, the availability of sufficient financial and other resources to meet business objectives and operational requirements, and the ability to raise sufficient capital to continue as a going concern, the fact that the results of earlier preclinical studies and clinical trials may not be predictive of future clinical trial results, any changes to the size of the target markets for the company's products or product candidates, the protection and market exclusivity provided by Immunic's intellectual property, risks related to the drug development and the regulatory approval process and the impact of competitive products and technological changes. A further list and descriptions of these risks, uncertainties and other factors can be found in the section captioned "Risk Factors," in the company's Annual Report on Form 10-K for the fiscal year ended December 31, 2025, filed with the SEC on February 26, 2026, and in the company's subsequent filings with the SEC. Copies of these filings are available online at or ir.imux.com/sec-filings. Any forward-looking statement made in this release speaks only as of the date of this release. Immunic disclaims any intent or obligation to update these forward-looking statements to reflect events or circumstances that exist after the date on which they were made. Immunic expressly disclaims all liability in respect to actions taken or not taken based on any or all of the contents of this press release. Contact Information Immunic, Inc. Jessica Breu Vice President Investor Relations and Communications +49 89 2080 477 09 [email protected] US IR Contact Rx Communications Group Paula Schwartz +1 917 633 7790 [email protected] US Media Contact KCSA Strategic Communications Caitlin Kasunich +1 212 896 1241 [email protected] SOURCE Immunic, Inc. 21% more press release views with Request a Demo

临床2期临床3期高管变更临床结果

2026-04-28

·证券之星

宝济药业-B（02659.HK）2025年度管理层讨论与分析

证券之星消息，近期宝济药业-B（02659.HK）发布2025年年度财务报告，报告中的管理层讨论与分析如下：业务回顾：我们是一家于2019年成立的生物技术公司,战略性聚焦四大领域:(i)大容量皮下给药,(ii)抗体介导的自身免疫性疾病,(iii)辅助生殖,及(iv)重组生物制药。我们的管线主要包括12款自主开发的候选产品,包括三款核心产品(KJ017、KJ103及SJ02(晟诺娃))、五款其他临床阶段候选药物(KJ101、KJ015、BJ007、BJ009及SJ04)及四款临床前资产(BJ044、BJ045、BJ047及BJ008)。我们的核心产品包括:(i)SJ02(晟诺娃),是一种长效重组人卵泡刺激素羧基末端肽融合蛋白(FSH-CTP),作为辅助生殖,用于进行控制性卵巢刺激、促进多囊泡发育及诱发排卵,于2025年8月获得国家药监局的NDA批准;(ii)KJ017,是一种处于NDA阶段的重组人透明质酸酶,用于大容量皮下给药(联合治疗)、由于各种原因导致的体液流失(单药治疗)、辅助皮下补液(联合治疗);及(iii)KJ103,是一种处于III期开发阶段的创新重组免疫球蛋白G(IgG)降解酶,其临床开发进展最快的适应症已完成III期临床试验,该产品用于治疗肾脏移植前脱敏及病理性IgG介导自身免疫性疾病,如抗GBM病及GBS等。附注: (1)我们一直担任唯一申办者的角色,负责为KJ017在中国的各个临床开发阶段提供资金,并预期就KJ017在欧洲及美国的未来临床开发继续担任唯一申办者的角色。 (2)我们已在中国完成药用辅料注册,并正推进全球注册进程。KJ017的DMF已于2025年5月成功向FDA备案。 (3)抗生素皮下制剂是基于化学药物改良(2.2类)新给药途径研发,后续研究放在药物曲线下面积(AUC)等效性及PK/PD。 (4)我们一直担任唯一申办者的角色,负责为KJ103在中国的各个临床开发阶段提供资金,并预期就KJ103在美国的未来临床开发继续担任唯一申办者的角色。 (5)我们一直担任唯一申办者的角色,负责为SJ02在中国的各个临床开发阶段提供资金,并预期就SJ02在欧洲的未来临床开发继续担任唯一申办者的角色。 (6)病理性IgG介导的自身免疫性疾病是指免疫系统产生针对人体自身细胞、组织或器官的异常IgG抗体而引发的一组失调疾病。 (7)我们于2025年7月与安科生物订立独家销售代理协议,据此,安科生物将作为独家CSO,负责SJ02在大中华区的商业化。在此我们的候选产品于报告期间及直至本公告日期,我们持续推进管线的开发进程。截至本公告日期,我们的主要成就及后续计划包括: –KJ017(重组人透明质酸酶) KJ017是中国临床研发最为先进的重组人透明质酸酶。我们正推进KJ017作为单药在中国的商业化推广,作为静脉输注的替代方式用于晶体溶液的大容量皮下给药、治疗由于各种原因导致的体液流失及辅助皮下补液。于报告期间及直至本公告日期,我们已取得以下进展及里程碑,并就下步计划开展的工作规划如下: o我们于2024年向国家药监局提交KJ017的NDA申请。我们已于2025年1月通过国家药监局对KJ017临床试验所在医院的GCP检查。于2025年2月,我们通过国家药监局对KJ017的上市前GMP合规检查。于2025年3月,参与试验的生物分析实验室亦通过国家药监局的GCP检查。我们预计将于2026年上半年获得NDA批准。 o截至本公告日期,我们已与多家制药及生物科技公司(包括药明生物(2269.HK)、荃信生物(2509.HK)、上海莱士(002252.SZ)及尚健等)建立正式合作伙伴关系,共同开发皮下制剂。我们持续积极拓展合作生态系统,并已与处于不同磋商阶段的十余家潜在合作伙伴制定业务发展计划。我们的典型合作模式是,我们持续提供我们的重组人透明质酸酶产品作为辅料及提供相关技术服务,而合作伙伴则自行出资推进与其候选药物联用的皮下制剂的开发。 –于2026年1月,我们与上海莱士(002252.SZ)下属全资子公司广西莱士生物制药有限公司共同宣布,双方已就基于透明质酸酶皮下给药技术的血液制品新剂型开发达成战略合作。根据协议,双方将结合本公司的皮下给药技术平台与上海莱士在血液制品领域的领先优势,共同开发更具便捷性和患者友好度的新型血液制品治疗方案,旨在提升患者依从性并优化医疗资源利用。 –于2025年1月,我们与药明生物(2269.HK)下属全资子公司上海药明生物技术有限公司就重组人透明质酸酶供应、生产和授权许可达成战略合作协议。根据协议,本公司与药明生物旨在就重组人透明质酸酶皮下给药应用领域实现合作共赢,共同深度开发新业务、拓展新客户。 –于2024年8月,我们与荃信生物订立技术服务与供应协议,以共同开发由荃信生物拥有、正在开发或将开发的与我们的重组人透明质酸酶联合使用的选定原研生物产品的创新皮下制剂。荃信生物对我们而言属独立第三方,是一家领先的生物技术公司,专注于自身免疫性疾病及过敏性疾病的生物疗法。根据该协议,荃信生物将作为根据该协议开发的皮下制剂的上市许可持有人,并享有其独家开发、制造及商业化的权利,及承担所有相关成本。我们同意为产品开发提供重组人透明质酸酶,提供必要的技术支持及协助进行监管备案。 –于2022年3月,我们与尚健订立技术服务与供应协议,以共同开发与我们的重组人透明质酸酶联合使用的抗CD38单克隆抗体的皮下制剂。尚健对我们而言属独立第三方,是一家领先的生物技术公司,致力于推进抗体药物治疗领域的科学创新。根据该协议,尚健将作为上市许可持有人,并牵头根据该协议进行皮下制剂的开发、监管备案、生产及商业化。我们同意为产品开发提供重组人透明质酸酶,提供必要的技术支持及协助进行监管备案。 o于2025年5月,我们成功向FDA备案KJ017的DMF。我们计划向欧洲的欧洲药品管理局(EMA)和FDA提交KJ017的IND申请,同时亦在准备EMA和FDA的IND备案。我们预计于2026年提交其中一项申请,并随后完成另一地区的IND申请。我们的KJ017在多种治疗方式(包括抗体药物及化学药品(尤其是抗生素))上展现出广泛的应用,使皮下给药成为可能,并具有提高药物安全性、患者便利性和疗效的潜力。根据上市规则第18A.08(3)条发出的警告:概不保证本公司最终将能够成功开发及销售KJ017。 –KJ103(重组IgG降解酶) KJ103是全球首款也是唯一一款达到注册临床阶段的低免疫原性IgG降解酶,其作为肾脏移植前脱敏及抗肾小球基底膜(GBM)病的适应症已获得国家药监局的突破性疗法认定(「BTD」)。KJ103以血液和组织中的IgG抗体为靶向,使之降解,从而抑制导致各种免疫疾病的病理性IgG介导的免疫反应。于报告期间及直至本公告日期,我们已取得以下进展及里程碑,并就下步计划开展的工作规划如下: o对于肾脏移植前脱敏适应症,我们于2025年8月开展III期试验,于2025年12月完成III期肾脏移植研究的患者入组,并于2026年3月完成III期临床试验。我们预计于2026年上半年向国家药监局提交NDA申请。 o对于抗GBM病适应症,我们于2026年1月召开III期研究启动会议,并预计于2026年上半年开展III期试验。在KJ103用于抗GBM病的II期试验中,KJ103给药3个月后的整体存活率为100.0%,其中66.7%的患者无需透析并可保留肾功能。在使用KJ103治疗6.0个月后,整体存活率为100.0%,其中75.0%的患者无需透析并可保留肾功能。与历史数据的疗效对比分析表明,KJ103显示出明显的临床优势。接受当前标准强化治疗的病患的历史数据显示,给药3.0个月后的整体存活率为81.2%,其中仅30.6%的患者无需透析并可保留肾功能。KJ103具有良好的安全性,并无药物相关的严重不良事件报告。 o对于GBS适应症,我们于2025年11月开展II期试验,并预计于2026年完成患者入组。 o就KJ103靶向致病性IgG介导自身免疫疾病的海外项目,我们计划于2026年下半年向FDA提交GBS适应症的孤儿药资格认定申请以及III期前IND申请。根据上市规则第18A.08(3)条发出的警告:概不保证本公司最终将能够成功开发及销售KJ103。 –SJ02(晟诺娃)(长效重组人卵泡刺激素) SJ02是中国获批的长效重组人卵泡刺激素羧基末端肽融合蛋白(FSH-CTP),旨在与促性腺激素释放激素拮抗剂联合使用,进行控制性卵巢刺激。SJ02是中国首款获批的长效FSH-CTP产品。 SJ02治疗方案可有效促进接受促排卵或辅助生殖技术手术的女性的多个卵泡发育。SJ02以传统的短效FSH为基础,通过将人绒毛膜促性腺激素β亚基的CTP序列与FSHβ亚基的C端融合而在结构上加强。此改构可将FSH的体内半衰期显着延长两至三倍,而不会影响其功能性。SJ02的长效特性使单次注射可以替代短效FSH连续七天注射。通过将给药间隔从每天延长至每周,SJ02可提供更大的便利性,最大限度地减少注射带来的不适,提升患者的整体治疗体验及生活质量。于报告期间及直至本公告日期,我们已取得以下进展及里程碑,并就下步计划开展的工作规划如下: o在中国,我们于2025年8月获得SJ02的NDA批准,并于2025年11月完成交付首批订单。 o于2025年7月,我们与独立第三方安科生物订立独家销售代理协议,据此,我们向安科生物授予在大中华区营销、销售、分销及推广SJ02的独家权利,因此,安科生物将作为独家CSO,负责SJ02在该等地区的商业化。截至本公告日期,安科生物已开展SJ02的实际商业销售,并持续推进该产品进入医院及纳入处方的程序。 o在欧洲,我们计划于2026年向EMA提交SJ02的IND申请。根据上市规则第18A.08(3)条发出的警告:概不保证本公司最终将能够成功开发及销售SJ02。 –KJ101(重组人糜蛋白酶) KJ101是通过合成生物学开发的国内领先的重组人糜蛋白酶。糜蛋白酶已展示出广泛的临床应用,特别是烧伤、创伤、手术切口、压疮及糖尿病足溃疡等伤口愈合。糜蛋白酶为一种蛋白水解酶,历来均提取自牛胰腺组织,存在着产量低、潜在污染及宗教或道德等方面的顾虑。基于我们专有的绿色重组酵母发酵技术,KJ101提供了一种纯净、更安全且更具扩展性的高表达替代方案。此外,KJ101具有卓越的生物安全性,有效地解决了生化提取物中固有的病毒污染问题。我们已于2025年2月获得国家药监局对KJ101就烧伤、创伤、手术切口、压疮及糖尿病足溃疡等伤口愈合的适应症的IND批准,并于2025年7月开展II期临床试验。我们预计于2026年上半年完成II期试验。我们于2025年12月就新增KJ101用于胃镜检查时溶解去除胃内黏液的适应症向国家药监局提交IND申请,并已于2026年3月获得IND批准。我们预计于2026年下半年开展II期试验。 –BJ044(重组乌司他丁) BJ044是一种小的循环蛋白多糖,存在于尿液中(即尿胰蛋白酶抑制剂),亦存在于羊水中(即丝氨酸蛋白酶抑制剂)。BJ044经工程化改造,旨在模拟尿液中乌司他丁的作用,其于间-α-胰蛋白酶抑制剂被嗜中性球弹性蛋白酶降解时分泌。我们预计于2026年上半年向国家药监局提交IND申请。 –KJ015(双特异性抗HER2抗体(皮下制剂)) KJ015是一种源于通用轻链技术的创新双特异性抗HER2抗体的皮下给药制剂,其被设计为拥有两条带有通用轻链的Fab臂,形成接近天然的IgG1结构。我们已于2024年12月获得国家药监局对KJ015的IND批准,并于2025年6月开展I期临床试验。我们预计于2026年下半年完成I期临床试验。我们计划于2026年上半年向FDA提交KJ015的IND申请。 –BJ045(抗酶切的抗CD20抗体(皮下制剂)) BJ045是一款皮下给药的抗KJ103酶降解的抗CD20抗体,具有与KJ103联用治疗中重度自身免疫性疾病的潜力。其与KJ103联用将切割现存IgG抗体库(如抗乙酰胆碱受体IgG),将在降低重症肌无力危象中病理性抗体的来源及影响方面进一步产生互补益处。此外,凭藉我们在皮下给药候选药物方面的竞争优势,BJ045的皮下注射给药方式有望改善治疗体验及患者依从性。我们预计于2026年向国家药监局提交IND申请。 –BJ047(抗酶切的抗CD154抗体(皮下制剂)) BJ047是一款皮下给药的抗KJ103酶降解的抗CD154抗体,专为实体器官移植、异种移植、自身免疫性疾病(包括狼疮肾炎及多发性硬化症)而开发。BJ047的抗酶降解能力进一步提高其抵抗体内酶分解的稳定性,从而确保持续的免疫抑制并促进异种移植物的长期存活。这有助于在目标适应症中产生协同效应。例如,其与KJ103联合使用以有效降解抗异种移植抗体时,将进一步促进减少异种移植中病理性抗体的来源和影响。此外,凭藉优越的便利性及治疗的灵活性,BJ047作为简单好用的皮下给药方案有潜力在市场中脱颖而出。我们预计于2026年向国家药监局提交IND申请。 –BJ007(头孢曲松钠(皮下制剂)) BJ007是用于治疗细菌感染的皮下给药头孢曲松钠产品。迄今,全球尚无已获批准的皮下给药头孢曲松钠,而BJ007为该类别首款也是唯一一款进入临床阶段的候选药物。这一创新减少了血管通路和使用长期静脉导管的需求,提供了更方便、更安全且成本更低的给药选择。因此,BJ007可提供非劣效的治疗益处,而且不会产生与头孢曲松的较长疗程通常所需的输液管路相关的风险、不适和成本,同时克服了DIVA患者在治疗中面临的主要难题。于2025年2月,我们获得国家药监局的IND批准。获批后,我们于2025年8月开展BJ007的I期临床试验(CTR20253085),并已于2026年1月完成该试验,以及预计于2026年上半年进入关键性临床试验。 –BJ008(头孢哌酮钠舒巴坦钠(皮下制剂)) BJ008是一款头孢哌酮钠舒巴坦钠的创新皮下制剂。头孢哌酮钠舒巴坦钠是一种常用的复方制剂,用于治疗细菌感染,涵盖呼吸道感染、尿路感染、腹腔内感染、妇科感染、皮肤和软组织感染、骨骼和关节感染、细菌性败血症、脑膜炎、心内膜炎,以及外科预防性用药。头孢哌酮是一种第三代头孢菌素类抗生素,与舒巴坦钠(一种不可逆的β-内酰胺酶抑制剂)联用时,对革兰氏阴性菌具有很强的协同抗菌活性,且稳定性良好。通过利用我们的大容量皮下给药系统,BJ008具备以皮下注射取代现有头孢哌酮钠舒巴坦钠静脉输注的潜力,可同时降低并发症的风险并提高患者依从性。我们预计于2026年向国家药监局提交IND申请。 –BJ009(头孢唑林钠(皮下制剂)) BJ009被设计为头孢唑林钠(第一代头孢菌素类抗生素)的创新皮下制剂,其透过抑制细菌细胞壁的合成而发挥作用,最终致使细胞裂解。与静脉注射用头孢唑林钠相似,BJ009有潜力治疗由细菌引发的多种感染,包括皮肤、骨骼、关节、生殖器、血液、心脏瓣膜、呼吸道、胆道及尿路感染。再者,BJ009采用皮下给药的方式,能带来增强的治疗体验,降低并发症风险,同时减少治疗成本,这显示出其市场潜力。我们已于2025年5月提交BJ009的IND申请,并于2025年9月获得国家药监局的IND批准。获批后,我们于2025年12月开展BJ009的I期临床试验(CTR20255246),并预计于2026年完成I期试验。 –SJ04(重组人绒促性素) SJ04是一种重组人绒促性素(hCG),可用于辅助生殖手术,以加速卵泡成熟及促排卵。此外,SJ04适用于治疗青春期前隐睾症、男性低促性腺激素性性腺功能减退症、黄体功能不足及功能性子宫出血。对于女性,SJ04可促进卵泡成熟及激发排卵,同时促使卵泡破裂形成功能性黄体,以促进黄体酮的分泌。因此,SJ04可促进黄体功能不足人士的子宫内膜发育,改善生殖预后,并通过使功能性子宫出血人士的激素模式正常化,有助建立规律的月经周期。于2024年5月,我们获得国家药监局对SJ04的IND批准。随后,我们于2024年8月在中国开展SJ04的I期临床试验,并已于2025年8月完成患者入组。我们已于2025年9月完成I期临床试验。根据上市规则第18A.08(3)条发出的警告:概不保证本公司最终将能够成功开发及销售KJ101、BJ044、KJ015、BJ045、BJ047、BJ007、BJ008、BJ009及SJ04。我们的专有技术平台凭藉我们在合成生物技术方面的优势,我们有预见性地建立全面一体化内部研发及生产能力。迄今为止,我们运营三个技术平台,涵盖药物设计、底盘细胞打造及完整的生物制造,使我们能够驾驭复杂的过程,将我们的转化型重组蛋白药物从实验室推向临床。具体而言,我们的三个技术平台包括: –药物设计平台:我们的药物设计方法聚焦于开发符合药物独特性及目标患者群体特定需求的定制化给药系统及制剂。我们在药物开发中优先考虑免疫原性、分子稳定性和具成本效益的生产。我们利用AI驱动的模型,将先进的计算模拟与严格的实验验证相结合,以实现精确的蛋白质工程和功能优化。湿法实验产生的数据不断反馈到我们的模型中,以细化和增强其性能,从而促成一个迭代和适应性的设计过程。因此,作为我们核心产品之一的KJ103从我们的药物设计平台中诞生,为一种由复杂酶组成、具备卓越稳定性及功能性的候选药物,充分展现该平台的转化能力。 –底盘细胞打造平台:我们的底盘细胞打造平台专注于糖基化修饰及先进的表达技术。凭藉在酶工程、糖工程及合成生物学方面的广泛专业知识,我们已在多个领域取得关键突破,如重组人透明质酸酶的蛋白质翻译调控及翻译后修饰、中国仓鼠卵巢(CHO)细胞糖基化工程及蛋白质高表达技术。我们在三种主要生物制药宿主系统(包括大肠杆菌、毕赤酵母及CHO细胞系统)中采用多学科方法,以设计生物元件、工程化代谢途径及筛选来自修饰宿主的药物蛋白。此方法使我们能够根据特定药物蛋白的结构及功能需求,在最合适的宿主中表达蛋白质,从而大大缩短新疗法的开发周期。尤其是,我们已开发出一个含有工程化糖基转移酶的CHO细胞库,以生成结构均一性更佳的人源化糖蛋白。这显着降低免疫原性,延长半衰期,并提高疗效。此外,我们的毕赤酵母细胞库具有表达伴侣及经优化的宿主,可立即用于新项目工艺研究,从而简化我们的药物生产及加快项目时间表。 –完整的生物制造平台:我们的完整的生物制造平台整合了哺乳动物、酵母及大肠杆菌表达系统,以支持我们重组蛋白药物的大规模、高效及可持续生产。我们优化生产流程及设备,注重环境可持续性。透过整合高产量的菌株或细胞、优化的培养工艺及先进的纯化技术,我们实现了具有绿色制造优势的规模化制造能力。此平台可应对关键技术挑战,包括但不限于:(i)通过合成生物学及基因工程增强重组蛋白表达及解决发酵过程中的蛋白降解,从而为高效重组蛋白生产提供上游解决方案;(ii)采用不同的发酵策略来克服毒性副产物积累、蛋白质错误折叠及细胞快速生长过程中活性低等问题,从而使用高密度合成生物学技术实现靶蛋白的稳定、高效表达;(iii)结合不同的色谱分离技术,利用定制树脂开发可扩展且具有成本效益的高纯度重组蛋白制备工艺;及(iv)提高体积生产力并开发资源节约型、低能耗的绿色制造解决方案,以满足商业规模生产重组蛋白的需求。除我们的专有平台技术外,我们还拥有商业规模的制造能力以及严格的质量控制及保证体系,使我们能够有效地扩大生产,以满足候选药物商业化后不断增长的需求,同时确保卓越的质量及成本效益。业务展望：于2025年,我们稳步推进核心战略,利用合成生物技术研发底盘细胞,并开发及生产传统基因工程难以表达的重组生物药物,在研发创新、临床推进及产业布局方面实现多项重要里程碑。于2026年,我们将进一步深化合成生物学研发运作机制改革,聚焦重组生物制品的核心优势,持续提升研发效率,加强外部战略合作,加强科学决策,并在大容量皮下给药、抗体介导的自身免疫性疾病、辅助生殖及重组生物制品四大战略领域巩固和扩大我们的领先地位。我们将继续以市场及临床价值为导向,围绕高需求医疗领域持续开发具明确差异化及全球潜力的创新药物。以底盘细胞构建平台为核心并整合药物设计平台及端到端生物制造平台,我们将持续强化核心技术能力,提升创新药物研发的成功率。同时,我们将积极拓展全球合作,加快培育新的竞争优势,并全面释放我们产品管线的全球价值。具体而言,我们计划推行以下策略:(i)加快核心治疗领域管线的开发,全面释放临床及商业价值;(ii)提升商业规模制造能力及质量管理体系,稳步推进商业化;(iii)拓展全球布局,深化战略合作伙伴关系,实现产品管线的全球价值;及(iv)吸引、发展及保留高素质人才,优化营运体系,致力成为运用合成生物学开发重组生物制剂的全球领导者。加快核心治疗领域管线的开发,全面释放临床及商业价值我们将加速推进我们的产品管线,旨在获得监管批准、扩大适应症及拓宽应用场景。于2026年,我们的主要目标是继续推进多元化的临床产品组合,并在获批准后实现KJ017的计划中商业化上市。同时,我们将加快开发及注册四大战略领域的核心资产和其他临床及临床前候选药物。具体而言: –大容量皮下给药:我们专注于以重组人透明质酸酶作为皮下给药平台的核心支柱。我们的核心产品KJ017预期将成为中国首款获批的重组人透明质酸酶,预计将于2026年上半年获得NDA批准,并随后在中国进行商业化上市。与此同时,我们正推进「双抗」战略,开发抗体药物及化学药物(尤其是抗生素)的皮下制剂。我们自主研发的创新抗HER2双特异性抗体KJ015预计将于2026年下半年完成I期试验,并计划于2026年上半年向FDA提交IND。除深化与抗体开发商合作,推动更多皮下抗体项目进入后期临床开发外,我们亦将加快皮下抗生素的临床开发。 –抗体介导的自身免疫性疾病:为应对多种抗体介导的自身免疫性疾病的未被满足需求,我们将致力推进核心产品KJ103的临床开发。我们的目标为于2026年上半年向国家药监局提交肾脏移植前脱敏的NDA申请,于2026年上半年开展抗GBM病的III期试验,并继续进行GBS的II期研究。海外开发方面,我们计划于2026年下半年就GBS向FDA提交孤儿药资格认定申请,并递交III期前的IND申请。同时,我们将把与抗酶降解重组抗体的联合疗法(如BJ045及BJ047)推进至临床阶段,并积极探索KJ103在异种移植等新兴领域的潜力。在器官衰竭病例日益增加及器官持续短缺的背景下,异种移植已于全球取得显着技术进展。我们的研究产品旨在协助克服免疫排斥反应,此乃该领域取得成功的关键决定因素之一。凭藉我们在酶技术及抗体工程方面的专业知识,我们有信心可在此不断增长的市场中占据重要份额,推进异种移植科学的发展,并满足巨大的未满足医疗需求缺口。 –辅助生殖:为解决接受ART的女性每日注射短效FSH造成的治疗负担,我们于2025年8月在中国获得SJ02(晟诺娃)的上市批准。SJ02为一种长效FSH-CTP,单次注射可替代短效FSH连续七天注射,可显着改善现行护理模式。展望未来,我们将与商业化合作伙伴安科生物紧密合作,全面进行SJ02(晟诺娃)在中国的上市工作,并实现其临床价值。 –重组生物制品:我们运用合成生物学技术,通过打造高效底盘细胞生产传统生化提取难以制造的复杂蛋白质,开发创新重组生物制剂,从而解决低效、杂质及安全风险性风险,包括过敏及未知病毒污染。2026年,我们将加快推进KJ101用于烧伤、创伤、手术切口、压疮及糖尿病足溃疡等伤口愈合适应症的II期临床试验,目标于上半年完成;对于KJ101用于胃镜检查时溶解去除胃内黏液的适应症,我们目标于下半年启动II期试验;并且,我们计划于上半年就BJ044提交IND申请,以推动这些潜在变革性重组生物制品的获批,满足巨大的临床需求。提升商业规模制造能力及质量管理,稳步推进商业化我们于上海建立符合GMP标准的生产基地,占地面积约63,000平方米。为进一步提升商业化产能,我们正在上海兴建一处新基地,占地面积约37,000平方米,预计将于2026年竣工并投入使用。我们现有的生产基地配备专为生产复杂生物制品而设计的生产线,具备生产重组蛋白药物的专业能力,可满足核心产品SJ02、KJ017及KJ103的生产需求,同时支持「合成生物升级替代生化提取」战略下的创新资产开发,包括重组人糜蛋白酶KJ101(II期)及重组乌司他丁BJ044(IND阶段)。该战略将上海的科技创新优势转化为优质的制造产出,与宝山北上海生物医药产业园的「合成生物产业化」战略定位相契合,深入贯彻「促进生物医药产业高质量发展」的政策方针。随着首款产品SJ02于2025年8月获批及KJ017预计将于2026年上半年获批,我们的生产重心正从单一的临床供应转向临床与商业化生产的双轨模式。我们将继续提升生产基地的运营效率,对标国际标准,并进一步完善我们的综合质量管理体系,以确保产品质量、安全性及疗效始终如一地符合监管要求,保障患者用药安全。拓展全球布局,深化战略合作伙伴关系,完全实现管线的全球价值依托于现有授权及合作(包括药明生物、上海莱士、安科生物、荃信生物、尚健等)的成功往绩,我们将继续在全球范围内积极寻求新的合作机会。我们的战略包括:(i)优先推进中国市场的产品开发及商业化,建立先发优势;(ii)通过国内产品销售及多元化合作产生稳定收入,以支持持续的研发与商业化投资;及(iii)在国内取得商业化成功后,进一步拓展适应症,并进军主要海外市场。对于早期资产及平台技术,我们亦将寻求与全球领先的生物技术公司及研究机构开展联合开发或授权引进╱转出合作,以探索新的治疗领域及前沿模式,并强化平台能力。我们旨在通过灵活的合作模式令创新成果惠及全球更多病患,并实现产品管线价值的最大化。吸引、发展及保留高素质人才,优化营运体系我们将继续推行全面的人才策略,积极招募在研发、商业化、管理及全球业务发展等领域具备深厚专业知识与丰富经验的顶尖专业人才,以支持我们的快速发展与国际化发展。同时,我们将不断优化内部管理流程,特别是提高研发项目管理及跨部门协作效率,确保能灵活应对市场变化及有效推进管线资产。于2026年,随着业务拓展及新产品陆续上市,我们将继续秉持「急患者之需而行」的公司使命,聚焦尚未满足的医疗需求领域,持续开发可及性高、惠及面广的药物。我们致力于成长为具有全球影响力的合成生物与重组生物制品领导者。根据上市规则第18A.08(3)条发出的警告:本公司概不保证将能成功开发或最终能够销售核心产品。以上内容为证券之星据公开信息整理，由AI算法生成（网信算备310104345710301240019号），不构成投资建议。

100 项与富马酸二甲酯相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D03846	富马酸二甲酯	L04AX07	DB08908

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
斑块状银屑病	欧盟	Almirall SA	2017-06-23
斑块状银屑病	冰岛	Almirall SA	2017-06-23
斑块状银屑病	列支敦士登	Almirall SA	2017-06-23
斑块状银屑病	挪威	Almirall SA	2017-06-23
多发性硬化症	日本	Biogen, Inc.	2016-12-19
复发-缓解型多发性硬化	欧盟	BIOGEN NETHERLANDS BV	2014-01-30
复发-缓解型多发性硬化	冰岛	BIOGEN NETHERLANDS BV	2014-01-30
复发-缓解型多发性硬化	列支敦士登	BIOGEN NETHERLANDS BV	2014-01-30
复发-缓解型多发性硬化	挪威	BIOGEN NETHERLANDS BV	2014-01-30
复发性多发性硬化	美国	Biogen, Inc.	2013-03-27

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
继发进展型多发性硬化	临床3期	美国	Biogen, Inc.	2015-05-01
继发进展型多发性硬化	临床3期	比利时	Biogen, Inc.	2015-05-01
继发进展型多发性硬化	临床3期	捷克	Biogen, Inc.	2015-05-01
继发进展型多发性硬化	临床3期	荷兰	Biogen, Inc.	2015-05-01
继发进展型多发性硬化	临床3期	波兰	Biogen, Inc.	2015-05-01
继发进展型多发性硬化	临床3期	斯洛伐克	Biogen, Inc.	2015-05-01
慢性大斑块银屑病	临床3期	奥地利	Almirall SA	2012-11-01
慢性大斑块银屑病	临床3期	德国	Almirall SA	2012-11-01
慢性大斑块银屑病	临床3期	荷兰	Almirall SA	2012-11-01
慢性大斑块银屑病	临床3期	波兰	Almirall SA	2012-11-01

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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


NCT04756687 (LymphoTEC, Pubmed \| Adv Ther)	N/A	多发性硬化症	1,429	Dimethyl Fumarate (Patients with lymphopenia)	鹹鑰製遞壓願醖襯糧觸(選簾顧醖簾淵顧遞艱鹹) = 遞獵選繭餘窪餘廠淵簾淵窪觸範築繭構襯顧艱 (製顧憲構簾顧顧選蓋網 )	积极	2025-04-01
NCT02047097 \| NCT02525874 (ESTEEM Phase 4 and PROCLAIM Phase 3, Pubmed \| Adv Ther)	临床3期	多发性硬化症 \| 复发-缓解型多发性硬化 Absolute lymphocyte counts (ALCs)	-	Dimethyl Fumarate (Patients < 50 years old)	網齋簾遞鹽網繭網觸蓋(襯衊構糧蓋鏇窪糧鏇獵) = 繭選廠獵膚鏇築選網積蓋鑰壓願遞鹹顧壓獵壓 (鬱網憲鬱積繭獵觸築鹽 ) 更多	积极	2024-11-21
				Dimethyl Fumarate (Patients ≥ 50 years old)	網齋簾遞鹽網繭網觸蓋(襯衊構糧蓋鏇窪糧鏇獵) = 糧網淵觸範積憲齋醖壓蓋鑰壓願遞鹹顧壓獵壓 (鬱網憲鬱積繭獵觸築鹽 ) 更多
NCT04221191 (TEC-ADHERE, Pubmed \| Neurol Ther)	临床4期	复发-缓解型多发性硬化	341	Dimethyl Fumarate (OroSEP)	鹹願壓憲壓願觸襯築窪(積夢積積糧積壓廠醖鹽) = 簾繭積鹽膚窪繭憲襯鹽選願觸願艱簾遞範簾鹹 (網願糧願壓膚廠獵觸構 )	积极	2024-11-11
				Dimethyl Fumarate (Standard of Care (SoC))	鹹願壓憲壓願觸襯築窪(積夢積積糧積壓廠醖鹽) = 繭鑰襯鹹鑰製築淵範鬱選願觸願艱簾遞範簾鹹 (網願糧願壓膚廠獵觸構 )
AAN2024	N/A	多发性硬化症	-	Fumarates (Black)	遞顧簾糧願醖鑰蓋鏇醖(壓鏇繭餘顧觸選醖遞艱) = 襯壓壓顧願築製糧蓋鹹選簾餘製鏇網齋壓鏇願 (範鏇鹽觸範築壓鬱遞鏇, 73.7–80.3)	-	2024-04-09
				Fumarates (Hispanic)	遞顧簾糧願醖鑰蓋鏇醖(壓鏇繭餘顧觸選醖遞艱) = 蓋蓋憲糧艱糧夢淵構鏇選簾餘製鏇網齋壓鏇願 (範鏇鹽觸範築壓鬱遞鏇, 71.0–80.0)
S31.010 (AAN2024)	N/A	多发性硬化症	930	Fingolimod	獵衊遞製鬱憲壓築夢襯(廠淵夢鑰衊廠顧憲鹹鏇) = 襯網鏇窪壓蓋範範獵築願積鏇糧觸夢憲範觸餘 (鏇選築觸壓糧鹹簾淵遞 )	不佳	2024-04-09
				Natalizumab	獵衊遞製鬱憲壓築夢襯(廠淵夢鑰衊廠顧憲鹹鏇) = 選憲鏇鹽齋顧遞艱鏇願願積鏇糧觸夢憲範觸餘 (鏇選築觸壓糧鹹簾淵遞 )
ACTRIMS2024	N/A	多发性硬化症	-	Fumarates (Black)	餘糧鏇積窪憲憲憲構衊(鏇蓋襯壓鏇糧顧構遞夢) = 窪憲選壓淵鹽鹽窪鹽鏇範憲繭窪蓋夢願願襯膚 (膚鏇鏇夢蓋淵醖鹹餘願 ) 更多	-	2024-03-01
				Fumarates (Hispanic)	餘糧鏇積窪憲憲憲構衊(鏇蓋襯壓鏇糧顧構遞夢) = 憲鹹鹹壓繭顧築觸襯構範憲繭窪蓋夢願願襯膚 (膚鏇鏇夢蓋淵醖鹹餘願 ) 更多
ECTRIMS2023	临床3期	多发性硬化症	118	DMF treatment	鏇積壓範鹹醖鹽壓餘築(鹹簾蓋網鏇醖憲窪積製) = 觸蓋膚壓顧鏇夢糧選齋獵製壓觸夢廠鬱鹹淵窪 (繭鏇淵糧繭鏇醖壓艱簾 )	积极	2023-09-30
EAN2023	N/A	多发性硬化症	-	DMF interruption at early pregnancy	廠繭選鹽鏇鏇積壓鬱窪(艱齋齋繭築壓獵餘鹽壓) = 憲獵獵鹹積齋憲構遞齋鬱淵廠製顧願膚襯積蓋 (窪顧鹹餘獵鏇鬱蓋夢簾, ±433.29) 更多	积极	2023-06-28
				Prompt DMF restart after delivery	-
EAN2023	N/A	-	127	Dimethyl Fumarate (DMF)	淵鑰鏇網艱窪築襯積鏇(繭簾鬱鏇齋蓋鑰廠簾觸) = 壓鏇鏇觸窪鏇繭糧繭夢壓糧齋壓鹹廠獵齋範餘 (範鹹鏇齋網遞艱製願顧, 1.71 ~ 12.31) 更多	积极	2023-06-28
				Non-specific Immunosuppressants (NSIS)	顧範膚膚鹽簾鑰顧鬱構(簾簾憲糧衊遞選衊鹹獵) = 選鹹遞願淵簾襯夢壓獵範餘選鏇衊蓋鹹遞餘膚 (壓築膚齋遞構襯夢網遞 )
NCT03870763 (CTgov)	临床3期	复发-缓解型多发性硬化	11	Dimethyl Fumarate (Dimethyl Fumarate 240 mg)	願膚鹹鹹齋鹽製襯獵觸(築齋鹹範窪壓鏇蓋襯憲) = 淵繭網膚襯壓糧醖襯憲鹽範網糧艱鬱顧製構鑰 (醖艱窪壓範觸範鬱鏇簾, 廠齋築獵餘壓網夢衊淵 ~ 選鑰憲獵壓壓衊遞鹹鑰) 更多	-	2023-05-16
				Peginterferon Beta-1a (Peginterferon Beta-1a 125 µg)	願膚鹹鹹齋鹽製襯獵觸(築齋鹹範窪壓鏇蓋襯憲) = 衊膚衊簾積艱鏇窪範餘鹽範網糧艱鬱顧製構鑰 (醖艱窪壓範觸範鬱鏇簾, 積窪獵積廠網壓繭網蓋 ~ 範齋繭願夢夢築範壓網) 更多