DiaPrecise, A Phase II Open Label Study to Evaluate the Safety and Feasibility of Intralymphatic Administration of Diamyd® in Individuals at Risk for Type 1 Diabetes Carrying the HLA DR3-DQ2 Haplotype

A 2-arm randomized Phase II Open Label Study to evaluate the safety and feasibility of intralymphatic administration of Diamyd® (Diamyd) also known as retogatein in individuals at risk of Type 1 diabetes carrying the HLA DR3-DQ2 haplotype.

开始日期2024-07-09

申办/合作机构

Diamyd Medical AB

CTIS2024-513350-30-00

/ Recruiting临床2期

DiaPrecise, A Phase II Open Label Study to evaluate the safety and feasibility of intralymphatic administration of Diamyd® in individuals at risk for Type 1 diabetes carrying the HLA DR3-DQ2 haplotype

开始日期2024-07-09

申办/合作机构

Diamyd Medical AB

CTIS2023-509021-53-00

/ Not yet recruiting临床2期IIT

A follow-up project on the GADinLADA trial to test for the continued impact of HLA-DR3DQ2 on diabetes status and immune parameters 3 years after treatment with intranodal GAD-alum.

开始日期2024-05-01

申办/合作机构

Norwegian University of Science & Technology

100 项与 Retogatein 相关的临床结果

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100 项与 Retogatein 相关的转化医学

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100 项与 Retogatein 相关的专利（医药）

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项与 Retogatein 相关的文献（医药）

2025-08-01·JOURNAL OF IMMUNOLOGICAL METHODS

Development and performance evaluation of a novel, fully automated, high-throughput GAD65 autoantibody assay on the Phadia platform

Article

作者: Ettner, Virginie ; Filchtinski, Daniel ; Kayser, José ; Holz, Sanja ; Fiedler, Raimund ; Kristensen, Bjarne ; Schulte-Pelkum, Johannes ; Wilhelm, Isabel

OBJECTIVES:

In clinics, diabetes is determined by blood glucose levels or by glycosylated hemoglobin (HbA1c). However, to distinguish between Type 1 and Type 2 diabetes (T1D and T2D) for adequate treatment, patients are tested for the presence of Type 1 diabetes-relevant autoantibodies in the second tier. Anti-GAD65 autoantibody is one of the main markers for T1D in both pediatric and adult T1D patients. If the anti-GAD65 autoantibody is detectable, the patients have type 1 diabetes and need to be treated with insulin. In the clinical routine diagnostics of type 1 diabetes, GAD65-serology is performed using a special assay architecture known as bridging ELISA. Adapting these assays onto automated ELISA systems has shown to be a constant challenge, so they can be hardly applied for high throughput testing in a routine clinical laboratory.

METHODS:

We developed the first fully automated, high-throughput GAD65 autoantibody fluorescence enzyme immunoassay based on the bridging immunoassay format on Phadia instrument platform, termed EliA GAD65 and compared its performance with the bridging ELISA from RSR™ (GADAb ELISA) and the fully automated MAGLUMI® GAD65 chemiluminescence assay (CLIA) from Snibe Diagnostics.

RESULTS:

When tested for clinical performance, ROC Analysis of EliA GAD65 showed an Area under the curve (AUC) of 0.906 (95 % CI 0.864 to 0.949) compared to the AUC of 0.923 (95 % CI 0.884to 0.963) of GADAb ELISA with no significant difference between the two assays (p = 0.384). When adjusted to a specificity of 96 %, EliA GAD65 showed a sensitivity of 76 % (CI 95 % 66.4 % to 84.0 %) compared to a sensitivity of 74 % (95 % CI 64.6 % to 81.6 %) of GADAb ELISA. When the fully automated MAGLUMI® GAD65 CLIA was compared to GADAb ELISA, it showed a total agreement of 92.9 % (95 % CI 86.6 % to 96.4 %) with less positive samples detected in contrast to the total agreement of 100 % (95 % CI 96.7 % to 100 %) of EliA GAD65 to GADAb ELISA.

CONCLUSIONS:

Fully automated EliA GAD65 assay displays similar clinical characteristics as the well-established bridging GADAb ELISA and a higher agreement with the GADAb ELISA than the automated MAGLUMI® GAD65 CLIA.

2023-11-01·Diabetes, obesity & metabolism

A 1‐year pilot study of intralymphatic injections of GAD‐alum in individuals with latent autoimmune diabetes in adults (LADA) with signs of high immunity: No safety concerns and resemblance to juvenile type 1 diabetes

Article

作者: Ludvigsson, Johnny ; Hals, Ingrid K. ; Grill, Valdemar ; Björklund, Anneli ; Balasuriya, Chandima ; Casas, Rosaura

Abstract:

Aims:

To test, for the first time in latent autoimmune diabetes in adults (LADA), the effects of autoantigen‐specific immunotherapy by intralymphatic administration of aluminium‐formulated recombinant human glutamic acid decarboxylase 65 (GAD‐alum); specifically, to test if this treatment is safe, to test whether it induces a strong immunological response akin to a similar protocol in type 1 diabetes and to look for associations with preserved beta‐cell function.

Materials and Methods:

Three GAD‐alum injections, 4 μg each, were administered 1 month apart into an inguinal lymph node in 14 people with newly diagnosed LADA (age 30‐62 years) presenting with high levels of antibodies against glutamic acid decarboxylase (GADA). Adverse effects, immunological variables and beta‐cell function were monitored, with detailed measurements at 5 and 12 months from baseline.

Results:

Clinical adverse effects were minor and transient and measured laboratory variables were unaffected. All participants completed the study. Treatment raised levels of GADA, elicited strong effects on reactivity of peripheral blood mononuclear cells to GAD and raised cytokine/chemokine levels. Beta‐cell function appeared stable preferentially in the seven participants carrying human leukocyte antigen (HLA) haplotypes DR3DQ2, as assessed by C‐peptide glucagon tests ( P < 0.05 vs. seven non‐carriers).

Conclusion:

Intralymphatic treatment with GAD‐alum in LADA is without clinical or other safety concerns over a 12‐month period. As in a similar protocol used in type 1 diabetes, treatment exerts a strong immunological impact and is compatible with protection of beta‐cell function preferentially in HLA‐DR3DQ2 LADA patients. These findings pave the way for a randomized controlled trial in this important subgroup of LADA patients.

2022-10-01·BMJ open

Phase III, randomised, double-blind, placebo-controlled, multicentre trial to evaluate the efficacy and safety of rhGAD65 to preserve endogenous beta cell function in adolescents and adults with recently diagnosed type 1 diabetes, carrying the genetic HLA DR3-DQ2 haplotype: the DIAGNODE-3 study protocol

Article

作者: Casas, Rosaura ; Nowak, Christoph ; Lind, Marcus ; Eriksson, Linnea ; Widman, Martina ; Teixeira, Pedro F ; Lindqvist, Anton ; Hannelius, Ulf ; Ludvigsson, Johnny

Introduction:

Type 1 diabetes (T1D) is an autoimmune disease leading to the destruction of the insulin-producing beta cells resulting in insulin deficiency and hyperglycaemic. Today, no approved therapy exists to halt this detrimental immunologic process. In a recent phase 2b study, intralymphatic administration of recombinant human glutamic acid decarboxylase 65 kDa (rhGAD65) adsorbed to Alhydrogel adjuvant to individuals recently diagnosed with T1D and carrying the HLA DR3-DQ2 haplotype showed promising results in preserving endogenous insulin secretion, confirming the results of a large meta-analysis of three randomised placebo-controlled trials of subcutaneous rhGAD65. The aim of the current precision medicine phase 3 study is to determine whether intralymphatic administration of rhGAD65 preserves insulin secretion and improves glycaemic control in presumed responder individuals with recently diagnosed T1D carrying HLA DR3-DQ2.

Methods and analysis:

Individuals ≥12 and <29 years recently diagnosed with T1D (<6 months) will be screened for the HLA DR3-DQ2 haplotype, endogenous insulin production estimated by fasting C-peptide and presence of GAD65 antibodies. 330 patients are planned to be randomised to 3 monthly intralymphatic injections of rhGAD65 or placebo (both accompanied by oral vitamin D supplementation), followed by 22 months of follow-up. The study is powered to detect a treatment effect in the two coprimary endpoints; change from baseline in AUC (0-120min) C-peptide levels during a mixed meal tolerance test, and change from baseline in glycaemic control estimated by haemoglobin A1c at 24 months. Secondary endpoints include effects on glucose patterns collected by masked continuous glucose monitoring, proportion of patients in partial remission and number of episodes of severe hypoglycaemia and/or diabetic ketoacidosis.

Ethics and dissemination:

The trial is approved by Ethics Committees in Poland (124/2021), the Netherlands (R21.089), Sweden (2021-05063), Czech Republic (EK-1144/21), Germany (2021361) and Spain (21/2021). Results will be published in international peer-reviewed scientific journals and presented at national and international conferences.

Trial registration number:

EudraCT identifier: 2021-002731-32, NCT identifier: NCT05018585 .

项与 Retogatein 相关的新闻（医药）

2026-03-29

·diamyd.com

Diamyd Medical updates on the interim efficacy analysis of Phase 3 DIAGNODE-3 trial

Diamyd Medical on Friday March 27th announced that the pre-specified interim efficacy analysis on 174 out of 321 participants of the ongoing Phase 3 DIAGNODE-3 trial yielded results that were unexpected and not aligned with prior retrospective or prospective data. This lack of alignment underscores the need for thorough evaluation of the interim analysis results. Based on the interim dataset, no treatment effect on C-peptide was observed, neither in the overall population nor in pre-specified subgroups, including the previously identified potential super responder group. “This outcome is highly unexpected and not consistent with the totality of prior data,” said Ulf Hannelius, CEO of Diamyd Medical. “The absence of any observable effect on C-peptide, including in pre-specified subgroups, makes it critical to conduct a thorough evaluation. Our priority is to understand these surprising findings that we received late on Friday and determine the most appropriate path forward.” The Company will conduct a comprehensive assessment of the analysis results that were received late on Friday March 27. The Company’s core operations, including manufacturing activities, will continue as planned during this period. To ensure patient safety and full compliance with ethical and regulatory standards Diamyd Medical will engage with regulatory authorities to determine appropriate next steps. “The interim outcome is disappointing and not aligned with earlier results,” said Dr. Joshua Vieth, Senior Director of Research at Breakthrough T1D. “A thorough evaluation of the interim results will be essential to understand these findings. We support Diamyd Medical in this work that will inform next steps in the development of retogatein.” Diamyd Medical will report further findings as analyses progress and will continue to communicate transparently on conclusions and next steps. The Company will hold live conference calls tomorrow, Monday, March 30, 2026 at 8:30 am CET (Direkt Studios, link to access call at: https://www.youtube.com/live/XPy2VdMUArI) and 3:00 pm CET (DNB Carnegie, link to access call at: https://qcnl.tv/p/r0_qafjpYZQbdjGvj3SYbg). About Diamyd Medical Diamyd Medical develops precision medicine therapies to prevent and treat type 1 diabetes. Retogatein (rhGAD65) formulated with alum is an investigational antigen-specific immunotherapy, designed to induce antigen-specific immune tolerance to GAD65 and preserve endogenous insulin production in individuals with type 1 diabetes who carry the HLA DR3-DQ2 gene. Retogatein has been granted Orphan Drug Designation in the U.S. as well as Fast Track Designation by the U.S. FDA for the treatment of Stage 3 (clinically diagnosed symptomatic) type 1 diabetes. Fast Track Designation has also been granted for the treatment of Stage 1 and 2 (pre-symptomatic) type 1 diabetes. DIAGNODE-3, a confirmatory Phase 3 trial in patients with recent-onset (Stage 3) type 1 diabetes was discontinued following a pre-planned interim futility analysis which indicated that the study was unlikely to meet its primary endpoint. Significant results in preserving endogenous insulin production have previously been shown in a large genetically predefined patient group – both in a large-scale meta-analysis as well as in the Company’s prospective European Phase 2b trial. The DIAGNODE-3 trial has only included patients from this specific patient group that carries the common genotype known as HLA DR3-DQ2, which constitutes approximately 40 % of patients with type 1 diabetes in Europe and the US. A biomanufacturing facility is under development in Umeå, Sweden, for the manufacture of retogatein (recombinant GAD65 protein), the active ingredient in the antigen-specific immunotherapy. Diamyd Medical is a major shareholder in the stem cell company NextCell Pharma AB and in the artificial intelligence company MainlyAI AB. Diamyd Medical’s B share is traded on Nasdaq First North Growth Market under the ticker DMYD B. FNCA Sweden AB is the Company’s Certified Adviser. For further information, please contact: Ulf Hannelius, President and CEO Phone: +46 736 35 42 41 E-mail: ulf.hannelius@diamyd.com Diamyd Medical AB (publ) Box 7349, SE-103 90 Stockholm, Sweden. Phone: +46 8 661 00 26, Fax: +46 8 661 63 68 E-mail: info@diamyd.com Reg. no.: 556242-3797 Website: https://www.diamyd.com The information was provided by the contact person above, for publication on 22.45 CET, March 29, 2026. Attachments: PDF version

临床结果临床3期临床2期孤儿药快速通道

2026-03-27

·生物通

免疫细胞图谱揭示抗GAD65抗体相关神经系统综合征中干细胞样记忆T细胞的扩增

在我们身体这座精密运转的“城市”中，免疫系统扮演着恪尽职守的“防卫军”，负责识别并清除入侵的病原体。然而，有时这支军队会“敌我不分”，错误地将自身的组织当成敌人发起攻击，由此引发自身免疫病。抗谷氨酸脱羧酶65（anti-GAD65）抗体相关的自身免疫性神经系统综合征（AINS）就是这样一种情况。患者体内产生的抗体错误地攻击了合成大脑关键抑制性神经递质GABA的限速酶GAD65，导致一系列严重的神经系统症状，如边缘性脑炎伴颞叶癫痫、僵人综合征或小脑性共济失调等。尽管检测到高水平的自身抗体，但现有免疫疗法对许多患者效果有限，疾病常导致终身神经功能残疾，其确切的免疫发病机制长期以来一直是个“黑箱”。抗体的致病性也存有争议，因为GAD65位于细胞内部，抗体难以直接接触。越来越多的证据，包括脑组织病理中发现的大量细胞毒性T细胞浸润，提示T细胞免疫应答可能在疾病过程中扮演着更核心的角色。为了揭开这一复杂疾病的神秘面纱，厘清究竟是抗体、T细胞还是二者协同“作案”，一个国际研究团队在《Proceedings of the National Academy of Sciences》上发表了他们的最新发现。研究者们综合运用了多项前沿技术来绘制抗GAD65 AINS的详细免疫图谱。他们建立了包含患者和对照组（非炎症性特发性颅内高压及健康人）的队列，获取了外周血单个核细胞和脑脊液样本。核心技术方法包括：单细胞RNA测序结合免疫受体谱分析，用于在单细胞分辨率下无偏倚地解析细胞类型、基因表达和T细胞受体（TCR）/B细胞受体（BCR）克隆性；多维流式细胞术，用于验证和定量特定免疫细胞亚群（如干细胞样记忆T细胞）；以及多重免疫荧光组织学分析，用于在脑组织原位确认特定免疫细胞的存在。此外，研究还从脑脊液BCR序列中重组表达了单克隆抗体，以检测其抗原反应性。研究结果 1. 人口统计学和基本临床数据研究纳入了8名抗GAD65 AINS患者作为测序队列，19名患者作为流式细胞术队列，并与非炎症对照组及健康对照进行比较。患者主要表现为边缘性脑炎/颞叶癫痫、僵人综合征和小脑性共济失调等临床表型。采样时患者基本未接受免疫治疗，可视为治疗初治状态。 2. 抗GAD65 AINS患者外周血和脑脊液中TSCM的扩增通过单细胞RNA测序分析，研究人员发现，与对照组相比，抗GAD65 AINS患者脑脊液中活化的CD4+干细胞样记忆T细胞比例显著增加。在外周血中，CD4+TSCM的频率也更高，但受样本量限制未达统计学显著性。进一步的多维流式细胞术验证证实，抗GAD65 AINS患者外周血中的TSCM，尤其是CD4+TSCM比例显著高于健康对照，并且这些CD4+T细胞表现出更高的活化标志物HLA-DR和组织归巢标志物CD69的表达。这种TSCM的增多在疾病长短程患者中持续存在。更重要的是，组织学分析在四分之三的患者脑实质中确认了CD8+TSCM的存在，并在一位患者的脑膜和脑实质中发现了CD4+TSCM。 3. 抗GAD65 AINS与IIH个体在炎症免疫反应调控相关基因表达上的差异差异基因表达分析揭示了患者与对照组在多基因表达上的不同。在外周血单个核细胞中，涉及细胞迁移、BCR信号、T细胞活化等的基因（如EID2、TCFL5、MAP2K2、TPT1、PCBP2）表达发生变化，且先前全基因组关联研究鉴定的HLA-C基因在患者中表达下调。在脑脊液中，与免疫细胞分化、活化、调节性T细胞生成等相关的基因（如GNAS、KLF2、CD69、TGFB1）表达出现差异。在特定的脑脊液细胞簇中，如活化的CD4+TSCM中LINC01578表达降低，提示其可能参与疾病相关的转录调控。 4. 大量脑脊液BCR具有GAD65反应性且发生体细胞高频突变，但源自非克隆扩增的B细胞对脑脊液B细胞受体的分析显示，患者脑脊液中几乎没有克隆性扩增的B细胞。然而，当研究人员随机选取脑脊液BCR序列重组表达成单克隆抗体后，惊人地发现40个抗体中有10个（25%）能够特异性结合GAD65。这些GAD65反应性抗体均为IgG1亚型，并且其体细胞高频突变数量显著高于非反应性抗体，表明它们很可能经历了抗原驱动的亲和力成熟过程。不过，这些抗体来源于未发生克隆扩增的B细胞。 5. 抗GAD65 AINS中区室特异性的TCR克隆扩增 T细胞受体谱分析揭示了疾病特异的克隆扩增模式。在抗GAD65 AINS患者脑脊液中，存在显著扩增的T细胞克隆，这些克隆的转录组显示细胞毒性、TCR信号、增殖和炎症反应相关基因上调。相反，在对照组中，克隆扩增更多见于外周血。这种“脑脊液扩增、外周血多样性降低”的倒置模式提示，病原性T细胞可能从外周迁移并聚集于中枢神经系统。网络分析进一步显示，患者脑脊液中的CD4+TSCM存在明显的克隆扩增和互连，支持了抗原驱动选择的存在。 6. 抗GAD65 AINS个体脑脊液中CD4+TSCM的扩增对脑脊液CD4+TSCM的深入分析显示，与对照组相比，患者该细胞群体中存在显著的克隆扩增。克隆网络分析显示患者存在更大、更互连的扩增克隆簇，定量分析也证实患者脑脊液TSCM中扩增克隆组的频率更高，突出了疾病特异性、聚焦的免疫反应。研究结论与意义本研究在单细胞分辨率下系统描绘了抗GAD65 AINS的免疫景观，得出了一系列重要结论，极大深化了对该疾病病理机制的理解。首先，研究确立了干细胞样记忆T细胞，特别是CD4+TSCM，在疾病中的核心地位。这些细胞在患者外周血和脑脊液中持续增多，并在脑脊液中发生克隆性扩增，展现出活化和细胞毒性的基因特征。同时，研究在脑实质中鉴定出CD8+TSCM，在脑膜/脑实质中鉴定出CD4+TSCM。这支持了一个“双区室”病理模型：脑脊液和脑膜中的CD4+TSCM作为自我更新的储备库，持续支持脑实质中CD8+TSCM的效应反应，二者协同驱动慢性的自身免疫过程。这解释了为何该病对主要靶向体液免疫的传统治疗反应不佳，并为开发靶向T细胞的疗法（如抗CD3治疗）提供了强有力的理论依据。TSCM本身也有潜力成为评估疾病活动度和治疗效果的生物标志物。其次，研究揭示了独特的T细胞受体库区室化特征。患者脑脊液中存在克隆扩增且转录活跃的T细胞，而外周血TCR多样性降低，这印证了病原性T细胞向中枢神经系统迁移和聚集的假说，是中枢神经系统自身免疫的一个潜在标志。再者，研究澄清了B细胞的作用。尽管脑脊液中B细胞缺乏明显的克隆扩增，但通过重组抗体技术，发现其中存在相当比例（25%）的、经过亲和力成熟的GAD65特异性抗体。这表明抗原特异性的B细胞应答确实存在，并且可能参与了疾病的启动或辅助T细胞应答，但可能并非慢性期持续损伤的主要直接“执行者”。这有助于解释为何靶向B细胞的疗法（如抗CD19 CAR-T）对部分患者也可能有效。此外，差异基因表达分析发现的多个差异表达基因（如HLA-C、KLF2、CD69等），为理解疾病涉及的免疫调控网络提供了新线索，其中一些可能成为新的治疗靶点。综上所述，这项研究通过高分辨率的系统生物学方法，将抗GAD65 AINS的病理机制从“抗体致病”的争议中，推向了一个“以T细胞（特别是TSCM）驱动为核心，B细胞辅助”的更全面模型。它不仅为这种难治性疾病提供了新的机制见解，更重要的是，指明了未来药物研发的潜在方向：即针对这些长期存活、具有自我更新能力的干细胞样记忆T细胞进行精准干预，有望为患者带来更有效、更持久的治疗选择。研究也展示了整合单细胞多组学、免疫谱分析和功能性验证在解析复杂自身免疫病中的强大威力。

2026-03-25

·diamyd.com

Quarterly Report 2 25/26

September 2025 – February 2026, Diamyd Medical AB (publ), Fiscal year 2025/2026 Cover photo Precision Medicine for type 1 diabetes Fully enrolled registrational Phase 3 program Diamyd Medical develops a proprietary platform of precision medicines for type 1 diabetes, a progressive autoimmune disease in which the immune system destroys the body’s own insulin production. The Company is advancing the fully enrolled registrational Phase 3 trial DIAGNODE-3 in the United States and Europe with an upcoming interim readout end of March 2026. Diamyd Medical’s B-share is traded on Nasdaq First North Growth Market under the ticker DMYD B. Further information is available on https://www.diamyd.com September 1, 2025 – February 28, 2026 Net sales: MSEK 0.3 (0.1), second quarter MSEK 0.1 (0.0) Net result: MSEK -111.7 (-81.0), second quarter MSEK -62.9 (-44.9) Result per share before and after dilution: SEK -0.6 (-0.8), second quarter: SEK -0.3 (-0.4) Cash flow from operating activities: MSEK -83.2 (-90.2), second quarter MSEK -39.5 (-48.4) Cash and short-term investments at February 28, 2026: MSEK 190.7 (103.8) Events during the second quarter Diamyd Medical deepens long-term manufacturing collaboration for retogatein Diamyd Medical announced the completion of screening in pivotal Phase 3 DIAGNODE-3 trial Diamyd Medical’s lead immunotherapy for type 1 diabetes received global non-proprietary name retogatein Diamyd Medical accelerated primary efficacy readout by 9 months in type 1 diabetes Phase 3 trial following FDA alignment and guidance Other events after the second quarter Diamyd Medical gives notice of extraordinary general meeting on April 8th, 2026 Diamyd Medical enters into equity financing agreements with new U.S. sector specialist investors for up to USD 125 million and announces full enrollment in Phase 3 trial Diamyd Medical receives Notice of Grant in Japan for insulin-based antigen therapy patent in type 1 diabetes Diamyd Medical announced that ADA will feature retogatein Phase 3-trial in dedicated symposium Diamyd Medical finalized the database for interim analysis readout in DIAGNODE-3 trial Diamyd Medical announced the strengthening of its IP-position in the US Comments by CEO Ulf Hannelius Dear Shareholders, Diamyd Medical has now reached a defining stage in the development of retogatein as we prepare for the interim efficacy readout from our pivotal Phase 3 trial DIAGNODE-3, anticipated on March 27, 2026. Earlier this week, we achieved a major operational milestone with the completion of randomization in the study. A total of 321 patients has now been randomized across 57 clinical sites in Europe and the United States, bringing this groundbreaking precision medicine trial to full enrollment. Importantly, and directly aligned with this milestone, we have secured significant new funding to support DIAGNODE-3 through its next phase. We recently entered into directed equity financing agreements with new U.S. sector specialist investors, raising approximately USD 25 million before transaction costs. In addition, the newly issued warrants may provide an additional USD 100 million upon full exercise. Together with the existing TO5 warrants, which can contribute approximately USD 35 million, this establishes a total potential financing capacity of around USD 160 million. Both the new warrants and the TO5 warrants can be exercised following the upcoming interim analysis, directly linking this potential additional financing to a key value-inflection point. This structure provides financial flexibility to scale investment in line with clinical outcomes, supporting completion of the Phase 3 program, preparations for potential commercialization as well as life cycle management and pipeline opportunities. The financing was executed at a pivotal moment for the program—following full enrollment and ahead of the interim analysis—and the participation of specialized U.S. healthcare investors further validates both the quality of our data and the potential of our precision medicine approach in type 1 diabetes. The upcoming interim analysis evaluates preservation of C-peptide from baseline to month 15, reflecting the body’s own insulin production, in a genetically defined patient population representing approximately 40 percent of individuals with the disease in Europe and the United States. Should the interim analysis demonstrate statistically significant efficacy, the results may support further regulatory interactions with the FDA regarding the potential Biologics License Application (BLA) pathway in the United States. The trial continues with follow-up of all participants toward the full study readout expected in the third quarter of 2027, intended to support a potential BLA for full approval. Scientific interest in our Phase 3 program continues to grow, and we are particularly pleased that DIAGNODE-3 has been selected for a dedicated symposium at the American Diabetes Association’s 86th Scientific Sessions in June 2026. The symposium will highlight the scientific rationale for precision medicine in type 1 diabetes and present the interim Phase 3 outcomes together with leading international experts in the field. Being selected for a dedicated scientific session at one of the world’s largest diabetes conferences underscores both the scientific relevance of our program and the increasing recognition of precision medicine approaches in autoimmune diabetes. In parallel with the clinical progress, our manufacturing capabilities in Umeå continue to advance. During the quarter, the Swedish Medical Products Agency conducted an inspection of our biomanufacturing facility as part of the ongoing process toward Good Manufacturing Practice (GMP) certification for the production of material for clinical studies. The inspection outcome was positive, and the observations raised were limited to minor findings that are currently being addressed in dialogue with the Agency ahead of their final assessment. Importantly, we also received constructive feedback from the Agency regarding requirements for future commercial manufacturing, providing valuable guidance as we continue developing our long-term production capabilities for retogatein. In parallel, we continue to work closely with our manufacturing partners to advance process validation, analytical methods and other CMC activities required for late-stage development and future commercialization. I am also very pleased to report that the long-term incentive program launched three years ago reached its first vesting during the quarter, with participants receiving shares after successfully meeting the program’s performance criteria, defined as a 50 percent increase in the Company’s market capitalization since its inception. This outcome reinforces the strong alignment between employees and shareholders. The team has executed in an exemplary fashion to advance both the Phase 3 program and our manufacturing capabilities in circumstances that periodically have been challenging. As we approach the interim analysis of DIAGNODE-3, the entire organization remains focused on disciplined execution across clinical development, regulatory engagement and manufacturing readiness. Our focus now is on delivering the upcoming milestone whilst continuing to advance the program toward the full Phase 3 readout and the next stages of regulatory engagement. I would like to extend my sincere gratitude to our employees, clinical investigators, partners and shareholders for their continued commitment and support. Stockholm, March 25, 2026 Ulf Hannelius President and CEO *** The above is an excerpt from the report. To read the complete report, please visit https://www.diamyd.com, or see attached PDF *** INVITATION TO PRESENTATION OF THE QUARTERLY REPORT 2 2025/2026 Diamyd Medical’s CEO Ulf Hannelius and CFO Niklas Axelsson will be interviewed in connection to the Company’s second quarterly report for the financial year 2025-26, published on March 25. The presentation will be held in English When: Wednesday March 25 at 09:00 am CEST Live webcast: https://www.youtube.com/live/qj2_tVpxIfY The YouTube chat is open for questions. The broadcast is produced by Biostock. The presentation will also be made available on www.diamyd.com About Diamyd Medical Diamyd Medical develops precision medicine therapies to prevent and treat type 1 diabetes. Retogatein (rhGAD65) formulated with alum is an investigational antigen-specific immunotherapy, designed to induce antigen-specific immune tolerance to GAD65 and preserve endogenous insulin production in individuals with type 1 diabetes who carry the HLA DR3-DQ2 gene. Retogatein has been granted Orphan Drug Designation in the U.S. as well as Fast Track Designation by the U.S. FDA for the treatment of Stage 3 (clinically diagnosed symptomatic) type 1 diabetes. Fast Track Designation has also been granted for the treatment of Stage 1 and 2 (pre-symptomatic) type 1 diabetes. DIAGNODE-3, a confirmatory Phase 3 trial with potential for an accelerated approval pathway in the US, is being conducted at 57 clinics in eight European countries and in the US in patients with recent-onset (Stage 3) type 1 diabetes. Significant results in preserving endogenous insulin production have previously been shown in a large genetically predefined patient group – both in a large-scale meta-analysis as well as in the Company’s prospective European Phase 2b trial. The DIAGNODE-3 trial has only included patients from this specific patient group that carries the common genotype known as HLA DR3-DQ2, which constitutes approximately 40 % of patients with type 1 diabetes in Europe and the US. A biomanufacturing facility is under development in Umeå, Sweden, for the manufacture of retogatein (recombinant GAD65 protein), the active ingredient in the antigen-specific immunotherapy. Diamyd Medical is a shareholder in the stem cell company NextCell Pharma AB and in the artificial intelligence company MainlyAI AB. Diamyd Medical’s B share is traded on Nasdaq First North Growth Market under the ticker DMYD B. FNCA Sweden AB is the Company’s Certified Adviser. Further information is available on https://www.diamyd.com For more information, please contact: Ulf Hannelius, President and CEO, ulf.hannelius@diamyd.com Niklas Axelsson, CFO, niklas.axelsson@diamyd.com Diamyd Medical AB (publ), Box 7349, SE-103 90 Stockholm, Sweden Phone: +46 8 661 00 26 E-mail: info@diamyd.com Reg. no: 556242-3797 The information was submitted for publication, through the agency of the contact persons set out above, at 08.15 CET on March 25, 2026. Attachments: Quarterly Report 2 2025/2026

临床3期快速通道孤儿药

100 项与 Retogatein 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	-	-	DB04963

研发状态

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适应症	最高研发状态	国家/地区	公司	日期
1型糖尿病	临床3期	芬兰	Diamyd Medical AB	2008-07-01
1型糖尿病	临床3期	法国	Diamyd Medical AB	2008-07-01
1型糖尿病	临床3期	德国	Diamyd Medical AB	2008-07-01
1型糖尿病	临床3期	意大利	Diamyd Medical AB	2008-07-01
1型糖尿病	临床3期	荷兰	Diamyd Medical AB	2008-07-01
1型糖尿病	临床3期	斯洛文尼亚	Diamyd Medical AB	2008-07-01
1型糖尿病	临床3期	西班牙	Diamyd Medical AB	2008-07-01
1型糖尿病	临床3期	瑞典	Diamyd Medical AB	2008-07-01
1型糖尿病	临床3期	英国	Diamyd Medical AB	2008-07-01
成人隐匿性自身免疫性糖尿病	临床2期	瑞典	Diamyd Medical AB	2004-12-01

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临床结果

适应症

分期

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT04262479 (GADinLADA, CTgov)	临床2期	成人隐匿性自身免疫性糖尿病	14	recombinant human glutamic acid dehydrogenase (rhGAD65), formulated in aluminium hydrogel+Vitamin D	齋築構鹽鏇顧選構願餘 = 鏇積獵繭糧衊鏇襯艱齋願遞醖窪鑰築壓觸膚壓 (糧築觸構獵憲糧醖艱願, 膚蓋壓淵憲蓋繭網艱鹽 ~ 齋壓餘繭鏇鬱範網願齋) 更多	-	2025-06-08
NCT03345004 (DIAGNODE-2, CTgov)	临床2期	1型糖尿病	109	Vitamin D+Diamyd (Active Arm)	鏇廠齋鹹築夢製淵齋鹽(廠醖鑰壓鏇遞鏇製夢鏇) = 繭膚夢壓鹽鑰網襯觸膚選壓憲顧鏇願廠製積憲 (製鑰鹹餘積築構簾範衊, 1.715) 更多	-	2023-01-09
NCT03345004 (DIAGNODE-2, CTgov)	临床2期	1型糖尿病	109	Placebo for Diamyd (Placebo Arm)	鏇廠齋鹹築夢製淵齋鹽(廠醖鑰壓鏇遞鏇製夢鏇) = 艱觸蓋築遞餘艱艱糧顧選壓憲顧鏇願廠製積憲 (製鑰鹹餘積築構簾範衊, 2.163) 更多	-	2023-01-09
NCT02352974 (DIAGNODE \| DIAGNODE-1, CTgov)	临床1期	1型糖尿病	12	Vitamin D+GAD-Alum	衊獵夢鹹鬱淵膚鑰觸選 = 築襯窪遞廠積糧鬱遞鏇壓鹽鬱觸積築艱襯製積 (窪鏇襯鬱齋憲衊醖鏇簾, 鏇鏇遞鹹顧顧廠窪觸蓋 ~ 齋餘鬱顧壓鏇衊鹹糧衊) 更多	-	2020-04-27
NCT02352974 (DIAGNODE-1, Biospace) 人工标引	临床1/2期	1型糖尿病	12	Diamyd	鏇窪壓壓遞襯糧簾夢憲(壓積壓醖壓範鹹衊繭淵) = 鹹醖選網願淵製壓齋餘網艱廠網鬱構繭觸築糧 (窪鬱齋鹽夢襯鏇鹽願觸 ) 更多	积极	2019-12-20
NCT02464033 (CTgov)	临床2期	1型糖尿病	20	Vitamin D+Etanercept	願簾憲餘齋積醖衊憲繭 = 鹽夢蓋廠積憲廠餘夢顧鹹顧獵顧鬱鹽齋鑰製壓 (壓憲繭蓋醖觸醖糧積膚, 齋鹽構夢鏇繭鹹獵窪鹹 ~ 夢繭壓餘壓廠壓繭夢窪) 更多	-	2019-09-25
NCT01122446 (DIAPREV-IT, CTgov)	临床2期	1型糖尿病 \| 糖尿病前期	50	Placebo comparator (Placebo Comparator)	製艱遞範鹽顧衊艱製淵 = 築築構構鏇壓衊蓋齋鹹鑰憲餘膚遞構範積醖淵 (壓範窪艱觸積壓廠網醖, 襯顧廠窪遞膚獵願窪獵 ~ 醖襯鑰製衊繭範窪觸觸) 更多	-	2019-05-06
NCT01122446 (DIAPREV-IT, CTgov)	临床2期	1型糖尿病 \| 糖尿病前期	50	Diamyd (Alum-GAD (Diamyd))	製艱遞範鹽顧衊艱製淵 = 壓衊遞艱繭顧鹹餘築齋鑰憲餘膚遞構範積醖淵 (壓範窪艱觸積壓廠網醖, 淵窪蓋糧鹽淵糧構壓構 ~ 廠鹽蓋醖鹹遞餘鏇齋衊) 更多	-	2019-05-06
NCT00529399 (TN08, CTgov)	临床2期	1型糖尿病	145	GAD-Alum (GAD-alum)	夢鬱觸襯鹹積願範齋窪(製簾蓋襯製觸憲憲願網) = 選窪蓋遞齋糧鹽憲艱繭淵壓觸範鬱壓簾夢觸獵 (醖構餘選顧觸鹹蓋獵齋, 醖遞糧顧壓範繭糧淵鬱 ~ 窪鹽壓選淵選壓鹹淵鏇) 更多	-	2016-12-07
NCT00529399 (TN08, CTgov)	临床2期	1型糖尿病	145	Alum (GAD-alum Plus Alum)	夢鬱觸襯鹹積願範齋窪(製簾蓋襯製觸憲憲願網) = 鏇衊夢膚選願衊淵製艱淵壓觸範鬱壓簾夢觸獵 (醖構餘選顧觸鹹蓋獵齋, 獵範獵餘範醖鏇顧觸構 ~ 窪築構夢襯製餘簾廠壓) 更多	-	2016-12-07
NCT00837759 (CTgov)	临床2期	1型糖尿病	7	Insulin+Sitagliptin+Lansoprazole+GAD65	膚餘願積選範淵網壓壓(憲觸觸獵襯醖艱鹽鹹淵) = 襯窪選願衊鬱獵蓋遞襯顧鑰鏇顧鬱夢顧願選鑰 (憲膚淵構糧醖夢夢獵壓, 0.53) 更多	-	2012-10-24