A Double-blind, Oral, Multiple-dose, Parallel, Randomized Study to Evaluate Efficacy and Safety of Endoxifen in Bipolar I Disorder Patients With Acute Mania Episodes With or Without Mixed Features

Bipolar disorder (BPD) is a chronic debilitating illness characterized by drastic swings in mood, energy and functional ability that affects the adult population. Endoxifen is an active metabolite of the marketed drug Tamoxifen and the present study aims to evaluate the efficacy and safety of 8 mg endoxifen in the Bipolar I disorder patient population compared to a placebo arm. Endoxifen will be compared to a placebo to demonstrate that the test product is active and to establish that the study is sufficiently sensitive to detect differences between the investigational products. Thus, Endoxifen will be compared to placebo to demonstrate that the test product is safe and active.

开始日期2024-03-19

申办/合作机构

Jina Pharmaceuticals, Inc.

[+1]

NCT06075953

/ Recruiting临床2期

DCIS: RECAST Trial -Ductal Carcinoma In Situ: Re-Evaluating Conditions for Active Surveillance Suitability as Treatment: a Breast Cancer Prevention Pilot Study

The goal of this trial is to see if active surveillance monitoring and hormonal therapy in patients diagnosed with ductal cell carcinoma in situ (DCIS), an early stage of breast cancer, can be an effective management of the disease.
Participants will be asked to receive control hormonal therapy or an investigational hormonal therapy treatment. Participants will be asked to return for evaluation with MRI at three months and six months. Depending on the evaluation participants will have the option to continue on the treatment. If the evaluation suggests surgery is recommended, the participant will discontinue the study treatment and will undergo surgery. In addition to the treatment and MRI evaluation, participants will be asked to provide blood sample to understand their immune status, provide saliva sample for genetic testing, provide the study with a portion of the tissue or slides generated from tissue removed during surgery performed as part of their standard of care.

开始日期2024-02-17

申办/合作机构

Quantumleap Healthcare Collaborative

100 项与内昔芬相关的临床结果

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100 项与内昔芬相关的转化医学

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100 项与内昔芬相关的专利（医药）

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515

项与内昔芬相关的文献（医药）

2026-02-01·STEROIDS

The effects of tamoxifen and its metabolites on circulating estrogen metabolites among pre- and postmenopausal women

Article

作者: Turner, Paul C ; He, Xin ; Dallal, Cher M ; Ramin, Cody ; Xu, Xia ; Ghosh, Rajrupa ; Pfeiffer, Ruth M ; Fan, Shaoqi ; Gierach, Gretchen L ; Troisi, Rebecca

PURPOSE:

Circulating estrogen metabolites of the 2-, 4- or 16-hydroxyestrone (OH) pathways may be differentially associated with breast cancer risk due to varying estrogenic and genotoxic activity. However, little is known about the influence of tamoxifen, an effective endocrine therapy, or its metabolites on estrogen metabolism.

METHODS:

Among women referred to tamoxifen therapy, 15 circulating estrogens and estrogen metabolites (EMs) were measured at baseline (pre-tamoxifen) and 12 months post-tamoxifen initiation using liquid chromatography-tandem mass spectrometry. Changes in EMs were assessed among women postmenopausal at baseline (n = 23) using paired t-tests. Using linear regression, cross-sectional associations between circulating tamoxifen, its three metabolites, and EMs were assessed 12-months post-tamoxifen among pre- (n = 33) and postmenopausal women (n = 27; includes four women who transitioned to postmenopausal during follow-up).

RESULTS:

Twelve months post-tamoxifen initiation, mean total EM concentrations decreased by 13.8% among postmenopausal women, primarily driven by decreases in 2-OH (15.3%) and 16-OH (17.2%) metabolites (p < 0.05). Among women premenopausal at 12-month follow-up, circulating tamoxifen was positively associated with estrone (β = 1.24), estradiol (β =1.39), 2-OH metabolites (2-OHE1: β = 0.72, 2-ME1: β = 1.15), and all 16-OH metabolites (p < 0.05). The tamoxifen metabolite, endoxifen, was positively associated with estrone (β = 10.1) and estradiol (β = 12.4), and select 2-OH and 16-OH metabolites (p < 0.05). Positive associations were also observed between 4-hydroxy-tamoxifen and estrone, 2-OHE1, 2-ME1, 4-ME1, and E3 (p<0.05). Among postmenopausal women, only N-desmethyltamoxifen was significantly associated with 3ME1 (β = 0.18, p = 0.02).

CONCLUSION:

Tamoxifen and its metabolites were associated with changes in circulating EMs. Further research is needed to understand tamoxifen-induced EM changes in breast cancer prevention and management.

2026-01-01·JOURNAL OF PHOTOCHEMISTRY AND PHOTOBIOLOGY B-BIOLOGY

Optical and random laser properties of the novel multifunctional endoxifen derivative (FLTX3) and its potential for the diagnosis of breast cancer resistance

Article

作者: de Armas-Rillo, Sergio ; Diaz, Mario ; Boto, Alicia ; Hernández, Dácil ; Valdes-Baizabal, Catalina ; Canerina-Amaro, Ana ; Lobo, Fernando ; Marín, Raquel ; Rodriguez, Mitzi ; Delgado, Pablo ; Pereda de Pablo, Daniel ; Lahoz, Fernando ; Soler-Carracedo, Kevin

Endoxifen is the most powerful metabolite of tamoxifen (TX), the main endocrine therapy administered worldwide for the treatment of estrogen-receptor (ER) positive metastatic breast cancer. Tamoxifen itself is a prodrug with weak affinity for ER, but it is converted into endoxifen, with up to 100-fold higher affinity for ER than TX. In this study, we introduce the first fluorescent endoxifen derivative (FLTX3), formed by covalent attachment of the small fluorophore NBD to the basic side chain of endoxifen. We have characterized the optical properties of FLTX3, demonstrating its ability as a laser dye. FLTX3 is an efficient target-directed fluorescent probe for the cellular labelling of ER in MCF7 breast cancer cell line as well as in uterine tissues. FLTX3 is also endowed with an intrinsic photodynamic effect when irradiated at the optimal excitation wavelength of FLTX3. Further, we show that FLTX3 has an optical gain behaviour that leads to random laser (RL) when the light emitted by the drug is scattered in the cell cultures. Indeed, analyses of coherent spectra by power function Fourier transform revealed a RL dominant cavity in the range of average cell sizes. As one of the main causes for tamoxifen treatment failure is resistance, we explored the potential discriminative value of FLTX3-induced RL between tamoxifen-resistant and tamoxifen-sensitive MCF7 cells. Using multivariate approaches, we unravelled significant differences in the RL signal between tamoxifen-sensitive and tamoxifen-resistant cells. These findings indicate that FLTX3-generated RL might provide a target-directed diagnostic tool for tamoxifen resistance in metastatic ER+ breast cancer.

2025-12-01·Toxicology reports

Accidental tamoxifen ingestion in a two-year-old child without major immediate symptoms: Case report

Article

作者: Mian, Paola ; Meer, Hester van ; Huitema, Alwin D R ; de Vries, Niels ; Hof, Willemien F J ; de Groot, Alise D E ; Touw, Daan J

Tamoxifen is a selective oestrogen receptor modulator indicated for the treatment of breast cancer in adults. The recommended dose is 20 mg orally once daily. We report an accidental tamoxifen ingestion in a 2-year-old female who accessed the tamoxifen from a pill bottle stored in a bag while unsupervised. Activated charcoal and sodium sulphate were administered approximately 2.5 h after ingestion. Plasma concentrations of both tamoxifen and active metabolite endoxifen were determined. The maximum plasma concentration of tamoxifen was 53.8 ng/mL, occurring two hours after ingestion. In contrast, the plasma concentration of endoxifen measured at 22 h after ingestion was 0.771 ng/L, which is considered subtherapeutic in adults. However, given the prolonged half-life of endoxifen, the plasma levels may still be rising at this point. The ingested dose was estimated to be around 31-37 mg, based on the plasma levels of tamoxifen and endoxifen. The actual ingested amount may have been higher due to early oral administration of activated charcoal. No direct serious events occurred during a hospital admission of 31 h. Given the anti-oestrogen properties of tamoxifen and the critical role of oestrogen in pubertal development long-term follow-up is recommended to monitor potential delayed effects.

项与内昔芬相关的新闻（医药）

2026-01-19

·递因递药

【聚焦DMD】FDA授予(Z)-Endoxifen治疗DMD孤儿药资格，加速药物研发进程

2026年1月19日，美国临床阶段生物制药公司Atossa Therapeutics宣布，其正在开发的DMD潜在疗法(Z)-Endoxifen，已正式获得美国FDA授予的“孤儿药认定”（Orphan Drug Designation）。这一重要里程碑，预示着DMD创新治疗的研发正在加速，为无数在困境中挣扎的DMD患者家庭带来希望。一、国际DMD研发新动态：(Z)-Endoxifen获FDA重要认定根据Atossa Therapeutics于2026年1月19日发布的公告，美国FDA孤儿产品开发办公室（OOPD）已正式批准授予(Z)-Endoxifen用于治疗杜氏肌营养不良症（DMD）的“孤儿药认定”。在此之前，(Z)-Endoxifen已获得了“罕见儿科疾病认定”（Rare Pediatric Disease Designation），这进一步巩固了其在罕见病领域，特别是DMD治疗方向的研发地位。 Atossa Therapeutics总裁兼首席执行官Steven C. Quay博士表示：“继此前获得罕见儿科疾病认定之后，(Z)-Endoxifen在杜氏肌营养不良症方面获得孤儿药认定，是Atossa推进该严重致残性疾病药物开发的重要里程碑。” 这一认定不仅是对(Z)-Endoxifen治疗DMD潜力的一种官方认可，更将为Atossa Therapeutics在未来的药物研发和上市过程中带来一系列激励措施，从而加速这款潜在疗法走向DMD患者的步伐。二、什么是“孤儿药认定”？为何重要？对于许多患者家庭而言，“孤儿药认定”可能是一个相对陌生的概念。简单来说，美国FDA的“孤儿药认定”是专门授予那些用于治疗罕见疾病或病症的药物的。其主要目的是鼓励制药公司投入资源研发这类药物，因为罕见病患者数量少，药物研发成本高，市场回报可能不足以覆盖投入，导致药企研发意愿不强。获得“孤儿药认定”通常意味着：监管支持：FDA将为药物开发提供更直接的指导和支持。研发激励：可能包括税收抵免、研究经费补助等。市场独占期：如果该药物最终获批上市，通常可以享有7年的市场独占期，在此期间其他公司不能生产或销售相同适应症的相同药物，这为药企提供了研发回报的保障。然而，需要明确的是，“孤儿药认定”：并非上市批准：它只是研发过程中的一个重要里程碑，并不意味着药物已经安全有效并可以上市销售。不缩短审批时间：它并不会自动加速药物的开发或监管审查时间。因此，(Z)-Endoxifen获得孤儿药认定，是研发路上的一大步，但距离最终惠及患者，仍需通过严格的临床试验验证其有效性和安全性，并最终获得上市批准。三、认识潜在疗法(Z)-Endoxifen (Z)-Endoxifen是一种强效的选择性雌激素受体调节剂/降解剂（SERM/SERD）。在DMD的病理进展中，多种复杂的机制交织在一起，导致肌肉损伤和功能丧失。(Z)-Endoxifen的独特之处在于其展现出多种作用机制：雌激素受体靶向效应：作为SERM/SERD，它能够影响与雌激素受体相关的细胞信号通路。 PKC抑制：同时，它还具有蛋白激酶C（PKC）抑制活性。这些多重机制的结合，使其有望从不同的角度干预DMD的病理过程，例如减轻炎症、保护肌肉细胞、或改善肌肉功能。Atossa Therapeutics正评估其在肿瘤学和罕见病领域的潜在应用。目前，(Z)-Endoxifen的口服制剂已显示出良好的安全性和药理学特征。这一作用机制的多元性，为DMD的治疗提供了新的思路，也意味着(Z)-Endoxifen有潜力成为一款能够适用于更广泛DMD患者群体的药物。关于(Z)-Endoxifen更详细介绍，请看本公众号的历史报道：【聚焦DMD】DMD治疗新曙光？小分子药物(Z)-Endoxifen多通路出击，女性携带者或也将受益！四、杜氏肌营养不良症杜氏肌营养不良症（DMD）是一种罕见、进行性、X连锁的神经肌肉疾病，由肌营养不良蛋白基因突变引起。DMD是儿童期最常见、最严重的肌营养不良症，其主要特征是：进行性肌肉无力：症状通常在幼儿期出现，表现为走路不稳、易跌倒，并逐渐丧失行走能力。多系统受累：随着病情进展，心肌和呼吸肌也会受到影响，最终常因呼吸衰竭或心力衰竭而导致英年早逝。尽管近年来DMD的治疗取得了显著进展，包括基因疗法和外显子跳跃疗法等，但DMD仍然是一种致死性疾病，对于安全、有效且可及的治疗方案，全球仍存在巨大的未满足医疗需求。因此，每一次新药物的研发进展，都承载着患者家庭沉甸甸的希望。五、最后最后，虽然这又是一个令人“沮丧”的国外药物获得国外认定的新闻，但毫无疑问这是一个积极信号，表明全球科研力量正在不懈探索DMD的多元化治疗路径。孤儿药认定所提供的监管激励和市场保障，将有助于Atossa Therapeutics加速(Z)-Endoxifen的临床试验进程，使其更快地完成必要的研发步骤。需要理解的是，国际上罕见病药物研发的不断突破，也将进一步推动中国在罕见病领域的政策制定和完善，包括加速审批、医保准入、临床试验支持等，从而使更多创新药物能更快惠及中国患者。我们期待(Z)-Endoxifen能够顺利通过后续的临床试验，最终证明其在DMD治疗中的安全性和有效性。也希望，未来，随着药物研发的不断推进和国际合作的深化，我们有理由相信，(Z)-Endoxifen以及更多创新疗法将有机会被引入中国！免责声明：本文旨在传递国际医疗进展信息，(Z)-Endoxifen目前仍处于临床试验阶段，尚未在中国或任何国家获批上市用于治疗杜氏肌营养不良症。任何药物的使用请务必在专业医生指导下进行，并遵循国家相关法律法规。请患者家庭理性看待研发进展，保持积极心态，并与您的主治医生保持密切沟通。参考文献： Atossa Therapeutics, Inc. Press Release: Atossa Therapeutics Receives FDA Orphan Drug Designation for (Z)-Endoxifen for the Treatment of Duchenne Muscular Dystrophy. January 19, 2026. 关于DMD疾病描述的相关医学资料。

孤儿药上市批准

2026-01-17

·基因狐

突破，不限基因型，DMD创新疗法再获FDA孤儿药资格认定！

点击蓝字关注“基因狐” 点亮星标获取更多精彩内容~ ▉ 导语杜氏肌营养不良症 (Duchenne muscular dystrophy, DMD) 是一种由抗肌萎缩蛋白 (dystrophin) 编码基因突变引起的X染色体连锁的隐性遗传病，临床表现为进行性肌肉萎缩，患者多于20岁左右死于心力衰竭或呼吸衰竭。目前，与DMD基因相关的变异已超过7000个，绝大多数为外显子区域的缺失、重复和点突变。据统计，DMD男婴发病率约1/5000-1/3500，已纳入我国《第一批罕见病目录》。尽管目前多款DMD疗法已经取得了突破性进展，但其发病机制复杂且治疗方法存在疗效有限、成本较高等问题，亟需进一步的研究和创新。近日，Atossa Therapeutics公司宣布，其用于治疗治疗DMD的核心在研管线(Z)-Endoxifen已获得美国FDA授予的孤儿药资格认定 (ODD)。这是继其获得儿科罕见疾病资格认定 (RPDD) 后在DMD这一充满挑战的领域取得的又一重要监管里程碑，有望为全球DMD患者带来一种有望超越现有基因靶向疗法的治疗希望。 ▲ 来源 iStock ▲ 好物推荐 ▉ 监管双重加持，加速DMD新药研发进程当地时间2026年1月16日，Atossa Therapeutics公司 (Nasdaq: ATOS) 宣布，其用于治疗治疗DMD的核心在研管线(Z)-Endoxifen已获得美国FDA授予的孤儿药资格认定 (Orphan Drug Designation, ODD)，这将为Atossa提供包括临床试验方案设计、FDA会议支持在内的多项监管便利，若最终获批上市，更有望获得长达7年的市场独占权。此前，(Z)-Endoxifen已获得儿科罕见疾病资格认定 (Rare Pediatric Disease Designation, RPDD)，并有望在未来获得一张价值上亿美元的优先审评券 (PRV)，为其后续开发铺平了道路。 ▲ 新闻稿截图 DMD是一种致命的X连锁遗传病，由于编码抗肌萎缩蛋白 (dystrophin) 的DMD基因突变导致进行性肌肉退化，患儿通常在3至5岁时就会出现近端肌肉无力、步态异常和小腿肌肉假性肥大。DMD基因全长约2220kb，含79个外显子，其cDNA长14 kb，是人类最大的编码基因。在DMD患者中，外显子缺失(Exon Deletions) 是最常见的类型，约占所有病例的60-70%，其次为外显子重复 (Exon Duplications)，约占所有病例的10%–15%。DMD基因的缺失和重复突变集中于45-55和3-9号外显子，分别有47%和7%的DMD患者携带这两处突变。 ▲ DMD基因外显子结构及功能结构域过去的几十年，DMD的治疗经历了从支持性治疗 (如物理治疗、呼吸支持) 到药物干预的演变，治疗策略主要集中于恢复抗肌萎缩蛋白 (dystrophin) 的表达、改善患者表型以及通过药理学手段减缓病程。皮质类固醇 (如泼尼松、地夫可特) 是长期以来的标准疗法，它们能减缓肌肉力量的衰退，但副作用显著，无法从根本上改变疾病进程。以Sarepta Therapeutics公司的Exondys 51® (eteplirsen) 为代表的反义寡核苷酸 (ASO) 药物，通过“外显子跳跃”技术，跳过有缺陷的外显子，使得细胞能够翻译产生一个缩短但仍有部分功能的抗肌萎缩蛋白。2023年获批的Sarepta/罗氏的基因疗法Elevidys，则通过AAV载体将一个微型抗肌萎缩蛋白 (micro-dystrophin) 基因递送到肌肉细胞中，试图从源头上“修复”问题。然而，无论是外显子跳跃还是基因疗法，都面临着共同的挑战：覆盖面有限: 外显子跳跃药物仅适用于特定类型的基因突变，例如eteplirsen仅适用于13%的DMD患者。递送与免疫原性: 基因疗法的AAV载体面临靶向效率、潜在的严重免疫反应 (如肝毒性、血小板减少) 以及长期安全性的考验，且价格极其高昂。无法逆转损伤: 这些疗法旨在延缓疾病进程，但对于已经发生的肌肉纤维化和炎症损伤，作用有限。 ▲ DMD治疗策略 (Front. Med, 2022) 尽管已有多种药物获得加速批准，但目前尚无疗法能实现肌肉中蛋白质的永久性、高水平恢复。因此，在“对因治疗”的道路上，是否还需要一种能够广泛适用、直击疾病核心病理生理过程 (如炎症、纤维化、细胞坏死) 的“普适性”疗法，一直是科学家们探索的方向。 ▉ (Z)-Endoxifen: 基于下游机制的“广谱”策略 (Z)-Endoxifen作为乳腺癌药物他莫昔芬 (Tamoxifen) 在人体内的活性代谢产物，被是一种强效的选择性雌激素受体调节剂/降解剂 (SERM/SERD)，其主要作用机制是通过调节雌激素受体活性和抑制蛋白激酶C (PKC)发挥作用。 ▲ Endoxifen代谢机制临床前研究显示，雌激素受体 (特别是ERα) 信号通路在免疫调节中扮演关键角色，(Z)-Endoxifen可能通过调节肌肉微环境中的免疫细胞功能，抑制关键炎症因子的产生，从而为肌肉的自我修复创造一个更有利的环境。这在理论上可以实现比皮质类固醇更具靶向性的抗炎效果，并可能避免其带来的全身性副作用。 PKC是一个庞大的激酶家族，参与了细胞增殖、分化、凋亡等多种信号通路。在DMD中，PKC的异常激活与炎症、纤维化和细胞死亡密切相关。(Z)-Endoxifen对PKC的抑制作用 (尤其是对PKC-β1)，为其在DMD治疗中提供了一个独立于ER靶点之外的、同样重要的作用机制，形成了“多靶点、协同增效”的治疗潜力。 ▲ Endoxifen作用机制值得一提是，(Z)-Endoxifen的作用机制不依赖于患者具体的抗肌萎缩蛋白基因突变类型。这使其有望成为一种覆盖全体DMD患者的“基础疗法”，可以与现有的基因靶向药物联用，实现“标本兼治”的协同效应。Atossa公司下一步的核心工作将是与FDA沟通并设计首次人体临床试验，主要涉及：安全性与耐受性: (Z)-Endoxifen虽源于他莫昔芬，但其药理特性不尽相同，尤其是长期用药的安全性，将是I/II期临床试验的首要评估目标。需要关注其对生长发育、骨骼健康、内分泌系统的潜在影响。 PK/PD研究: 确定在患儿体内的最佳给药剂量和频率，并探索能够反映药物生物学活性的生物标志物。例如，是否可以通过检测血液中的某些炎症因子 (如TNF-α, IL-6) 或纤维化标志物 (如TGF-β) 的水平变化，来早期判断药物是否起效。临床疗效终点: 早期的疗效探索可能会聚焦于功能性终点，如经典的“6分钟步行测试 (6MWT)”、北极星移动评价量表 (NSAA) 、起立-行走-上楼-下楼时间等。此外，影像学评估 (如肌肉MRI) 检测肌肉脂肪浸润和纤维化程度的变化，也将是评价疗效的客观指标。联合用药潜力: 未来的临床设计中，非常值得探索(Z)-Endoxifen作为“基础疗法”与外显子跳跃药物或基因疗法的联合应用。理论上，(Z)-Endoxifen改善肌肉微环境，可能为基因靶向疗法发挥更佳效果创造条件，实现“1+1>2”的效应。尽管前景广阔，但该项目仍处于非常早期的阶段。未来的临床试验将面临诸多挑战，例如：ERα和ERβ在肌肉中的功能并非完全一致，有时甚至相互拮抗，阐明(Z)-Endoxifen对这两种亚型的亲和力、调节/降解活性的差异，对于深入理解其作用机制、预测其在人体中的效果至关重要。此外，DMD主要影响男性，而(Z)-Endoxifen作用于雌激素信号通路，因此还必须在临床试验中评估(Z)-Endoxifen在男性患儿体内的药代动力学、药效学以及安全性，特别是对青春期发育的潜在影响。 (Z)-Endoxifen在DMD领域的大胆探索，代表了药物研发从“线性思维” (一个基因->一个蛋白->一个靶点) 向“网络思维” (一个基因缺陷->网络失衡->多节点干预) 的转变。这种系统生物学的视角，尤其适用于那些病理机制复杂的遗传性疾病。与此同时，(Z)-Endoxifen这种“不限基因型”的治疗潜力，如果能在临床上得到证实，将极大地改变DMD的治疗格局，让更多无法从现有精准疗法中获益的家庭看到希望。 ▉ 关于孤儿药资格认定孤儿药资格认定 (Orphan-drug designation, ODD)，是美国FDA授予符合条件的用于预防、诊断或治疗罕见病的药物或生物制品的一种资格认定，旨在鼓励研发机构为罕见病开发创新治疗方案。FDA授予的孤儿药资格认定适用于在美国每年患病人数低于20万人的罕见病的药物和生物制剂。获得FDA孤儿药资格认定的药物将享有包括临床试验费用的税收抵免、免除新药申请费、获批后七年的市场独占权等一系列政策支持，以加速其开发进程，早日惠及患者。信息基于公开资料，仅供参考，不构成医疗或投资建议。原创文章，未经授权允许严禁转载！资料来源 [1]. https://www.atossatherapeutics.com [2]. Remmel HL et al. A Hypothesized Therapeutic Role of (Z)-Endoxifen in Duchenne Muscular Dystrophy (DMD). Degener Neurol Neuromuscul Dis. 2025 Mar 15;15:1-15. [3]. 其他公开资料版权声明：本公众号署名原创的文章仅为个人学习笔记，且只用于交流学习目的，无意剽窃和抄袭，若涉及版权问题或标记有误，烦请留言联系，将第一时间更正或删除。免责声明：本公众号对转载、分享的内容、陈述、观点判断保持中立，不对所包含内容的准确性、可靠性或完善性提供任何明示或暗示的保证，仅供读者参考。推荐阅读 2020-2025：AAV基因治疗安全警示录！重磅，全球首个DMD AAV基因疗法Ⅲ期临床2年随访数据公布！ 2026突破之年：值得关注的六项杜氏肌营养不良症(DMD)创新疗法！突破，又一款DMD外显子跳跃疗法冲刺2027年上市！ FDA批准全球首款DMD基因疗法标签更新，严格适用范围和强化临床监测，Sarepta迎来监管“压力测试”！又一款罕见病AAV基因疗法获FDA批准上市，定价259万美元！ Nature | 刘如谦教授团队发布“通用型”基因编辑策略，罕见病有望进入“广谱治疗”新时代！重大突破！FDA开启个性化基因编辑“平台化”审评时代，罕见病治疗有望迎来新纪元！重磅突破！首款体内(in vivo)基因编辑疗法临床前数据惊艳，即将进入人体试验，我们离治愈遗传性罕见病还有多远？ 100%响应率，又一款罕见病AAV基因疗法获美FDA突破性疗法认定，上市进展加速！千亿AAV赛道大洗牌：巨头退场、天价困局，基因疗法的“下半场”怎么玩？突破，首款罕见病体内基因编辑疗法完成III期临床患者入组，冲刺2027年上市！《自然-生物技术》| 个体定制化CRISPR基因编辑治疗迈入新纪元！ “点赞”“分享”“推荐”，让更多人看到撰文/排版/编辑：基因狐转载、投稿、合作请联系：25378137@qq.com

孤儿药优先审批

2026-01-16

·PRNewswire

Atossa Therapeutics Receives FDA Orphan Drug Designation for (Z)-Endoxifen for the Treatment of Duchenne Muscular Dystrophy

Designation further supports (Z)-Endoxifen program into rare pediatric neuromuscular disease along with previously received Rare Pediatric Disease Designation SEATTLE, Jan. 16, 2026 /PRNewswire/ -- Atossa Therapeutics, Inc. (Nasdaq: ATOS) ("Atossa" or the "Company"), a clinical-stage biopharmaceutical company developing novel therapies in oncology and other areas of high unmet clinical need, today announced that the U.S. Food and Drug Administration ("FDA") Office of Orphan Products Development ("OOPD") has granted Orphan Drug Designation to (Z)-endoxifen for the treatment of Duchenne muscular dystrophy ("DMD"). "In addition to the previously received Rare Pediatric Disease designation, Orphan Drug Designation for (Z)-endoxifen in Duchenne muscular dystrophy is an important milestone for Atossa as we move forward developing (Z)-endoxifen for this serious and debilitating disease," noted Steven C. Quay, M.D., Ph.D., Atossa Therapeutics President and Chief Executive Officer. Atossa plans to continue engaging with FDA as it advances development efforts and will provide updates as appropriate. About Orphan Drug Designation Orphan Drug Designation is granted by FDA to therapies intended to treat rare diseases or conditions. The designation is designed to encourage drug development by offering certain potential incentives, such as regulatory support and, if the product ultimately receives marketing approval for the designated indication, eligibility for a period of market exclusivity. FDA also notes that if an "otherwise same drug" is approved first for the same indication, a sponsor may need to demonstrate that the new drug is clinically superior to the previously approved drug in order to be eligible for orphan-drug exclusivity. Orphan Drug Designation neither shortens the development time or regulatory review time of a drug nor gives the drug any advantage in the regulatory review or approval process. About Duchenne Muscular Dystrophy Duchenne Muscular Dystrophy is a rare, progressive, X-linked neuromuscular disorder caused by mutations in the dystrophin gene. Symptoms typically emerge in early childhood and include progressive muscle weakness, loss of ambulation, respiratory compromise, and cardiomyopathy. DMD is uniformly fatal, often in early adulthood, and despite recent therapeutic advances, there remains a substantial unmet medical need for safe, effective, and accessible treatments. About (Z)-Endoxifen (Z)-Endoxifen is a potent Selective Estrogen Receptor Modulator/Degrader (SERM/SERD) with demonstrated activity across multiple mechanisms of interest. Atossa is evaluating its potential applications in oncology and rare diseases. The Company's proprietary oral formulation has shown a favorable safety profile and pharmacology distinct from tamoxifen, including ER-targeted effects and PKC inhibition. Atossa's (Z)-Endoxifen is not approved for any indication. Atossa's (Z)-Endoxifen program is supported by a growing global intellectual property portfolio, including multiple recently issued U.S. patents and numerous pending applications worldwide. About Atossa Therapeutics Atossa Therapeutics, Inc. (Nasdaq: ATOS) is a clinical-stage biopharmaceutical company developing innovative medicines in oncology and other areas of significant unmet need. The Company's lead product candidate, (Z)-Endoxifen, is currently in development across several clinical settings. More information is available at . Forward-Looking Statements This press release contains certain "forward-looking statements" within the meaning of applicable securities laws, including but not limited to, our expectations regarding the Company's development and regulatory strategy and related milestones, the potential indications that the Company may pursue for (Z)-Endoxifen, the potential for (Z)-Endoxifen to receive regulatory approval and the timing thereof, and the potential market and growth opportunities for the Company. Words such as "expect," "potential," "continue," "may," "will," "should," "could," "would," "seek," "intend," "plan," "estimate," "anticipate," "believe," "design," "predict," "future," or other similar expressions or statements regarding intent, belief or current expectations, are forward-looking statements. Forward-looking statements in this press release are subject to risks and uncertainties that may cause actual results, outcomes, or the timing of actual results or outcomes to differ materially from those projected or anticipated, including, without limitation, risks and uncertainties associated with: our ability to successfully execute our strategy to shorten our clinical development timelines and pursue a metastatic breast cancer indication, DMD indication or other indications for our lead program, (Z)-Endoxifen; expected timing, completion and results of our preclinical studies, clinical trials and research and development programs; the unpredictable relationship between preclinical study results and clinical study results; the timing or likelihood of regulatory filings and approvals; the outcome or timing of necessary regulatory approvals; our ability to receive orphan-drug exclusivity for (Z)-Endoxifen for DMD; our ability to regain and maintain compliance with Nasdaq listing requirements; our ability to establish and maintain intellectual property rights covering our products; the impact of general macroeconomic conditions on our business; our ability to raise capital; and other risks and uncertainties detailed from time to time in Atossa's filings with the SEC, including, without limitation, its Annual Reports on Form 10-K and Quarterly Reports on Form 10-Q. Forward-looking statements are presented as of the date of this press release. Except as required by law, we do not intend to update any forward-looking statements. SOURCE Atossa Therapeutics Inc 21% more press release views with Request a Demo

孤儿药

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适应症	最高研发状态	国家/地区	公司	日期
躁狂	临床3期	美国	Jina Pharmaceuticals, Inc.	2024-03-19
躁郁症1型	临床3期	美国	Jina Pharmaceuticals, Inc.	2020-08-03
ER阳性乳腺癌	临床2期	美国	Inclin, Inc.	2023-02-14
ER阳性乳腺癌	临床2期	美国	Atossa Therapeutics, Inc.	2023-02-14
GRPR阳性/ER阳性/HER2阴性乳腺癌	临床2期	美国	Atossa Therapeutics, Inc.	2023-02-14
GRPR阳性/ER阳性/HER2阴性乳腺癌	临床2期	美国	Inclin, Inc.	2023-02-14
HER2 阴性乳腺癌	临床2期	美国	Inclin, Inc.	2023-02-14
HER2 阴性乳腺癌	临床2期	美国	Atossa Therapeutics, Inc.	2023-02-14
浸润性乳腺癌	临床2期	美国	Atossa Therapeutics, Inc.	2023-02-14
浸润性乳腺癌	临床2期	美国	Inclin, Inc.	2023-02-14

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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


NCT05607004 (EVANGELINE, SABCS2025) 人工标引	临床2期	ER阳性/HER2阴性乳腺癌新辅助 Estrogen receptor positive \| HER2 Negative	22	(Z)-endoxifen (40mg vs 80mg) ± Goserelin (Part 1 (PK Run-in))	憲鏇觸遞廠遞齋願網淵(淵糧鹹餘範淵簾廠願構) = 襯憲簾廠願襯繭構壓糧夢構鏇齋製築築衊衊製 (範艱積遞糧鬱壓鬱鑰獵 )	积极	2025-12-09
NCT05068388 (KARISMA-Endoxifen, Company_Website) 人工标引	临床2期	乳腺癌	-	(Z)-endoxifen 1 mg	蓋鏇齋觸襯膚鹹壓獵廠(積憲艱遞遞簾遞夢顧觸) = 壓夢獵齋襯顧艱壓齋網鹹憲獵廠構願構艱襯壓 (衊壓餘鏇獵積淵簾醖築 ) 更多	积极	2024-11-04
				(Z)-endoxifen 2 mg	蓋鏇齋觸襯膚鹹壓獵廠(積憲艱遞遞簾遞夢顧觸) = 蓋夢鑰淵餘襯獵築繭醖鹹憲獵廠構願構艱襯壓 (衊壓餘鏇獵積淵簾醖築 ) 更多
NCT05607004 (EVANGELINE, AACR2024) 人工标引	临床2期	绝经前乳腺癌新辅助 HER2 Negative \| Proliferation marker protein Ki-67 \| ER Positive	7	(Z)-endoxifen 40 mg/day	糧廠窪艱廠鑰糧壓顧鹽(鹹壓餘夢醖構製鬱顧淵) = 鏇蓋淵獵膚襯鹽網鑰淵膚膚築願襯鑰淵鏇願範 (製窪衊蓋簾壓構鹹壓積, 180.3 ~ 376.6) 更多	积极	2024-04-05
NCT03317405 (CTgov)	临床1期	乳腺导管癌 \| 乳腺原位癌	32	Endoxifen Hydrochloride (Endoxifen Hydrochloride Gel 10 mg Daily)	簾繭顧壓鏇艱糧壓淵鹹 = 衊艱鬱憲餘願夢鏇網簾襯選築觸鹽夢鹽糧製廠 (觸範糧襯網製構鹽遞簾, 艱壓願觸觸選鬱窪鹽壓 ~ 簾壓獵網簾艱築醖膚鏇) 更多	-	2023-06-13
				Endoxifen Hydrochloride (Endoxifen Hydrochloride Gel 20 mg Daily)	簾繭顧壓鏇艱糧壓淵鹹 = 齋鬱衊顧憲築蓋鹹壓衊襯選築觸鹽夢鹽糧製廠 (觸範糧襯網製構鹽遞簾, 齋鏇餘蓋構顧艱餘構鑰 ~ 選夢窪憲鏇網繭淵鑰觸) 更多
NCT02311933 (PD7-06, CTgov)	临床2期	复发性乳腺癌 \| 局部晚期乳腺癌 \| 转移性乳腺癌 ... 更多	81	Pharmacological Study+Endoxifen Hydrochloride (Arm I (Z-endoxifen Hydrochloride))	憲衊積壓遞淵夢繭壓淵(築夢製餘鏇壓夢範淵鹹) = 簾鹹繭範簾鑰蓋餘鬱鹽範簾襯鬱獵範淵淵淵壓 (繭遞齋憲壓範鏇艱襯鬱, 廠淵餘鹽獵鹹蓋醖窪繭 ~ 簾鏇鹽顧積簾網鹹衊淵) 更多	-	2021-09-28
				Pharmacological Study+Tamoxifen Citrate (Arm II (Tamoxifen Citrate))	憲衊積壓遞淵夢繭壓淵(築夢製餘鏇壓夢範淵鹹) = 窪蓋鹽醖構鑰鹹壓淵選範簾襯鬱獵範淵淵淵壓 (繭遞齋憲壓範鏇艱襯鬱, 淵餘繭襯蓋網鬱衊積糧 ~ 窪餘廠顧壓壓願範願齋) 更多
NCT04616430 (CREME, CTgov)	临床2期	乳腺疾病	90	Placebo (Control)	鹹壓範壓齋蓋繭築鑰繭(積積簾糧窪衊窪願築餘) = 齋鬱艱淵製觸選壓蓋糧廠餘鹹憲膚鬱醖範鏇築 (襯製鑰鬱範餘襯積獵淵, 5.58) 更多	-	2021-06-18
				Topical endoxifen (Topical Endoxifen 10mg/Breast/Day)	鹹壓範壓齋蓋繭築鑰繭(積積簾糧窪衊窪願築餘) = 憲鹽構夢築淵製觸觸製廠餘鹹憲膚鬱醖範鏇築 (襯製鑰鬱範餘襯積獵淵, 6.50) 更多
NCT02311933 (AACR2020) 人工标引	临床2期	HER2 阴性乳腺癌 \| ER阳性乳腺癌 HER2 negative \| ER Positive	77	Z-endoxifen HCL	齋鹹築範窪顧夢繭鑰網(壓構襯鏇糧廠繭築鹹願) = 廠壓範艱膚糧顧範憲積壓夢醖築艱襯廠淵顧範 (糧淵製鬱範膚蓋願製齋, 76 ~ 138)	非优	2020-02-15
				tamoxifen	齋鹹築範窪顧夢繭鑰網(壓構襯鏇糧廠繭築鹹願) = 觸齋選遞蓋衊製簾襯製壓夢醖築艱襯廠淵顧範 (糧淵製鬱範膚蓋願製齋, 24 ~ 129)
NCT01273168 (ASCO 12018 \| ASCO 22018) 人工标引	临床1期	HR阳性实体瘤 HR Positive	40	Endoxifen	鹽衊繭築網鑰獵壓糧遞(鹹壓構鹽獵廠襯簾觸遞) = hypertension (13%), hyponatremia (8%), hypophosphatemia (8%), neutropenia (8%), dehydration (5%), and elevated ALT (5%) 鏇鬱範網鏇鹽糧遞鏇廠 (蓋鬱獵夢範齋鹹獵鹽鹹 ) 更多	积极	2018-06-04
NCT01327781 (MC093C, ASCO2017)	临床1期	激素难治性乳腺癌	41	Z-endoxifen	構夢壓壓齋衊獵鏇餘夢(獵繭糧繭衊製顧鏇選獵) = 淵鑰鹹餘製衊範夢網網壓夢選簾鏇範鏇鏇衊鏇 (鹽觸餘衊獵鹹簾鑰蓋願, 13.4 ~ 43.1) 更多	-	2017-05-30
NCT01327781 (phase I study of Z-Endoxifen hydrochloride, SABCS2016)	临床1期	转移性乳腺癌 ER+	41	Z-Endoxifen hydrochloride (Z-Endx)	獵範夢鑰蓋蓋衊糧蓋鏇(齋顧窪壓齋鏇顧餘鹹艱) = 觸鹽淵艱艱簾鏇簾繭壓遞膚餘憲鏇積鹽蓋衊簾 (糧醖餘膚憲蓋鑰夢顧壓 )	积极	2016-02-15