Atossa Highlights Emerging Opportunity for (Z)-Endoxifen in Duchenne Muscular Dystrophy, Including Symptomatic Female Carriers, Following Peer-Reviewed Publication and Scientific Presentation
Atossa (Nasdaq: ATOS) highlighted a newly published peer‑reviewed hypothesis article and an invited scientific presentation supporting the investigational therapy (Z)-endoxifen for Duchenne muscular dystrophy (DMD) and symptomatic female carriers (D‑CAPs).
The paper maps multi‑pathway mechanisms — ER modulation, PKC‑β1 inhibition, AKT/mTOR and NF‑κB effects — and notes potential advantages over tamoxifen due to more consistent exposure. Atossa plans preclinical validation and fit‑for‑purpose clinical studies focused on safety, PK/PD, functional and cardiac endpoints; a follow‑up manuscript on utrophin modulation is under review.
Atossa (Nasdaq: ATOS) ha evidenziato un articolo di ipotesi peer‑reviewed recentemente pubblicato e una presentazione scientifica invitata che supportano la terapia sperimentale (Z)-endoxifen per la distrofia muscolare di Duchenne (DMD) e per i portatori femminili sintomatici (D‑CAPs).
Il documento mappa meccanismi multi‑via — modulazione dell'ER, inibizione PKC‑β1, effetti AKT/mTOR e NF‑κB — e segnala potenziali vantaggi rispetto al tamoxifen dovuti a un'esposizione più costante. Atossa pianifica convalida preclinica e studi clinici adeguati per sicurezza, PK/PD, endpoint funzionali e cardiaci; un manoscritto di follow‑up sulla modulazione dell’utrofina è in revisione.
Atossa (Nasdaq: ATOS) destacó un artículo de hipótesis recién publicado y una presentación científica invitada que respaldan la terapia experimental (Z)-endoxifen para la distrofia muscular de Duchenne (DMD) y para portadoras sintomáticas femeninas (D‑CAPs).
El artículo traza mecanismos multiruta — modulación del receptor de estrógeno (ER), inhibición de PKC‑β1, efectos en AKT/mTOR y NF‑κB — y señala posibles ventajas frente al tamoxifeno por una exposición más constante. Atossa planea validación preclínica y estudios clínicos adecuados para seguridad, PK/PD, endpoints funcionales y cardíacos; un manuscrito de seguimiento sobre la modulación de la utrofina está en revisión.
Atossa (Nasdaq: ATOS)는 최근 발표된 동료 심사(peer‑reviewed) 가설 논문과 초청 과학 발표를 강조하며 신약 치료제 (Z)-엔독시펜을 Duchenne 근이영양증(DMD) 및 증상성 여성 보인자(D‑CAPs)에 대해 지지합니다.
그 논문은 다중 경로 메커니즘을 제시합니다 — ER 조절, PKC‑β1 억제, AKT/mTOR 및 NF‑κB 효과 — 그리고 더 일관된 노출로 Tamoxifen에 비해 잠재적 이점이 있다고 언급합니다. Atossa는 안전성, PK/PD, 기능적 및 심장 지표를 중심으로 한 전임상 검증 및 목적에 맞는 임상 연구를 계획하고 있으며, utrophin 조절에 관한 후속 원고도 심사 중입니다.
Atossa (Nasdaq: ATOS) a mis en évidence un article d’hypothèse par peer‑review récemment publié et une présentation scientifique invitée qui soutiennent la thérapie expérimentale (Z)-endoxifen pour la dystrophie musculaire de Duchenne (DMD) et les porteuses féminines symptomatiques (D‑CAPs).
L’article cartographie des mécanismes multi‑voies — modulation des ER, inhibition de PKC‑β1, effets AKT/mTOR et NF‑κB — et note des avantages potentiels par rapport au tamoxifène en raison d’une exposition plus constante. Atossa prévoit une validation préclinique et des études cliniques adaptées axées sur la sécurité, PK/PD, objectifs fonctionnels et cardiaques ; un manuscrit de suivi sur la modulation de l’utrophine est en cours de revue.
Atossa (Nasdaq: ATOS) hob eine neu veröffentlichte, von Peer-Review begutachtete Hypothesen‑Artikel und eine eingeladene wissenschaftliche Präsentation hervor, die die experimentelle Therapie (Z)-Endoxifen für Duchenne‑Muskeldystrophie (DMD) und symptomatische Trägerinnen (D‑CAPs) unterstützen.
Der Artikel skizziert Multi‑Pfad‑Mechanismen — ER‑Modulation, PKC‑β1‑Inhibition, AKT/mTOR und NF‑κB‑Effekte — und weist auf potenzielle Vorteile gegenüber Tamoxifen aufgrund einer konsistenteren Exposition hin. Atossa plant präklinische Validierung und passgenaue klinische Studien zu Sicherheit, PK/PD, funktionalen und kardialen Endpunkten; ein Folgemanuskript zur Utrophin‑Modulation befindet sich in der Prüfung.
Atossa (المدرجة في ناسداك: ATOS) سلطت الضوء على مقال فرضي محكّس من قبل زميل-مراجعة وعرض علمي مدعو يدعمان العلاج التجريبي (Z)-Endoxifen لضمور Duchenne عضلي (DMD) ومحولات إناث حاملة أعراض (D‑CAPs).
يخطيط الورقة يوضح آليات متعددة المسارات — تعديل ER، تثبيط PKC‑β1، آثار AKT/mTOR و NF‑κB — ويشير إلى مزايا محتملة مقارنة بالتاموكسين بسبب التعرض الأكثر اتساقًا. تخطط Atossa للتحقق قبل الإكلينيكي ودراسات سريرية مناسبة للأمان، PK/PD، ونقاط نهاية وظيفية وقلبيّة؛ مخطوط متابعة حول تنظيم الأوتروفين قيد المراجعة.
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Insights
Peer‑reviewed hypothesis and invited presentation build a plausible, multi‑mechanism case for (Z)-endoxifen in DMD but no clinical efficacy data yet.
The publication frames how (Z)-endoxifen could act on inflammation, fibrosis, calcium handling, mitochondrial function, lipid biology, ER signaling, PKC (notably PKC-β1), AKT/mTOR and NF-κB pathways relevant to dystrophic muscle. The paper highlights potential pharmacokinetic advantages over tamoxifen by avoiding CYP2D6 variability and notes tissue concentrations that may exceed plasma, creating a biological rationale for sustained muscle activity.
Key dependencies and risks include absence of clinical efficacy data, the hypothesis nature of the article, and ongoing preclinical/clinical validation needs. Monitor the
Published article outlines rationale for multi-pathway efficacy for (Z)-endoxifen in Duchenne Muscular Dystrophy (DMD); November scientific presentation to spotlight potential in Duchenne carrier–associated pathologies
Newly published hypothesis article outlines why (Z)-endoxifen may matter in DMD
The article about (Z)-Endoxifen in Duchenne Muscular Dystrophy (DMD), surveys the DMD treatment landscape and details how (Z)-endoxifen's pharmacology could address multiple downstream drivers of disease, including inflammation, fibrosis, calcium dysregulation, mitochondrial dysfunction, and lipid abnormalities. A video abstract of the paper can be found here.
The paper emphasizes (Z)-endoxifen's direct estrogen-receptor (ER) modulation, allosteric inhibition of PKC (notably PKC-β1), and effects along AKT/mTOR and NF-κB axes, mechanisms that together may help slow disease progression when used as an adjunct to standard care. Notably, the authors underscore (Z)-endoxifen's potential to deliver more consistent therapeutic exposures than tamoxifen by bypassing CYP2D6 metabolic variability, an important limitation of the pro-drug approach. As illustrated in the mechanistic diagram, page 7 of the publication, the paper maps (Z)-endoxifen's ER-dependent and ER-independent signaling effects relevant to dystrophic muscle.
- Clinical context: Prior tamoxifen studies in DMD showed safety and encouraging trends but were underpowered due to premature termination during the pandemic, supporting continued exploration of more potent, exposure-reliable metabolites like (Z)-endoxifen.
- Tissue exposure: (Z)-endoxifen tissue concentrations may substantially exceed plasma levels in certain settings, supporting a rationale for sustained pharmacodynamic activity at the muscle level.
- Cardio-skeletal relevance: The paper reviews pathways tied to cardiomyopathy, now a leading cause of death in DMD, and discusses how ER/PKC-linked modulation could complement existing standards of care, including glucocorticoids and recently approved agents.
- Pragmatic access: As a small-molecule candidate, (Z)-endoxifen could, if proven safe and effective, offer a potentially more scalable and accessible option alongside high-cost genetic approaches, while remaining mechanistically complementary.
As a follow-up to our initial publication, Atossa recently submitted a second manuscript investigating the potential mechanism of action of (Z)-endoxifen in Duchenne muscular dystrophy (DMD). This study focuses specifically on the role of (Z)-endoxifen in modulating utrophin expression and signaling pathways. The manuscript, entitled "(Z)-Endoxifen as a Modulator of Utrophin Pathways in Duchenne Muscular Dystrophy," is currently under review with the Journal of Degenerative Neurological and Neuromuscular Disease. Utrophin is a structural and functional analog of dystrophin that can compensate for the loss of dystrophin in DMD, stabilizing the sarcolemma and mitigating muscle fiber damage. Pharmacological upregulation of utrophin represents a promising therapeutic strategy that is independent of the underlying dystrophin mutation. Our work explores how (Z)-endoxifen influences utrophin-related pathways, potentially offering a novel, mutation-agnostic therapeutic approach for DMD.
Scientific presentation to focus on female carriers
H. Lawrence Remmel, Director of Atossa Therapeutics will present "Endoxifen: A Potential Novel Therapy for Duchenne Carrier-Associated Pathologies" at the 2nd International Conference on Women's Health, Reproduction & Obstetrics in
Management commentary
"Duchenne remains one of the highest-need pediatric diseases. The science we and our collaborators summarized points to a coherent, multi-pathway rationale for (Z)-endoxifen that is distinct from, and potentially synergistic with, genetic strategies," said Steven Quay, M.D., Ph.D., Atossa Therapeutics Founder and CEO. "We believe this framework supports responsible next steps aimed at rigorously testing endoxifen as a potential adjunct in DMD and as an investigational option for symptomatic carriers."
"Having led the first IND for a Duchenne therapy, I've seen how crucial it is to pair solid biology with a pragmatic development plan. (Z)-Endoxifen's small-molecule profile, exposure-guided strategy, and phase-appropriate GxP controls position it for a rigorous, stepwise program focused on safety, PK/PD, and functional endpoints. Our goal is to engage regulators early and, if data support, pursue rare-disease pathways that can responsibly accelerate development for patients and families who have waited too long," said Janet Rea, Senior Vice President, R&D of Atossa Therapeutics.
Mr. Remmel added, "Our Rome presentation will focus on Duchenne carrier–associated pathologies, where unmet need and biological plausibility intersect. The published hypothesis provides the mechanistic scaffolding, now the task is to translate this into data-driven clinical exploration."
Why investors should care
- Large unmet need, multiple shots on goal: DMD demands more than one solution; (Z)-endoxifen's combinable, small-molecule profile offers a potentially capital-efficient path to add value alongside genetic and anti-inflammatory standards.
- Mechanistic differentiation: Direct ER modulation plus PKC-β1 inhibition (with downstream AKT/mTOR and NF-κB effects) targets convergent disease nodes implicated across skeletal and cardiac muscle pathology. (See mechanistic diagram, page 7 of the publication.)
- Broader population reach: Beyond boys with DMD, symptomatic female carriers represent a clinically meaningful extension opportunity aligned with our publication and the upcoming presentation.
Atossa intends to leverage the published framework to guide prioritized preclinical validation and the design of fit-for-purpose clinical studies. The Company expects they will be designed to assess safety, pharmacokinetics, pharmacodynamics, and functional endpoints relevant to upper-limb, diaphragmatic, and cardiac performance, while exploring biomarker-enriched and combination strategies consistent with standard of care. Investigational plans are subject to refinement and regulatory feedback.
About Duchenne Muscular Dystrophy (DMD)
DMD is an X-linked, progressive neuromuscular disease driven by dystrophin loss, leading to muscle degeneration, loss of ambulation, respiratory compromise, and cardiomyopathy, with substantial morbidity, mortality, and economic burden. Cardiomyopathy is now a leading cause of death in DMD.
About (Z)-Endoxifen
(Z)-Endoxifen is Atossa's investigational ER-modulating small molecule. In oncology and CNS studies to date, (Z)-endoxifen has shown a favorable safety profile and pharmacology distinct from tamoxifen, including ER-targeted effects and PKC inhibition. (Z)-endoxifen is not approved for any indication.
About Atossa Therapeutics
Atossa Therapeutics, Inc. (Nasdaq: ATOS) is a clinical-stage biopharmaceutical company developing innovative therapies for significant unmet needs in breast cancer. Atossa's strategy emphasizes disciplined capital allocation, focusing resources on programs and data packages that can enable future regulatory submissions and potential commercialization. For more information, visit www.atossatherapeutics.com and refer to Atossa's filings with the
Forward-Looking Statements
This press release contains forward-looking statements, including statements regarding the potential safety, efficacy, development plans, regulatory strategy, clinical trial design, timelines, and market opportunity for (Z)-endoxifen in DMD and in Duchenne carrier–associated pathologies. Forward-looking statements are based on current expectations and involve risks and uncertainties that could cause actual results to differ materially. These risks include, but are not limited to, preclinical and clinical development risks, regulatory uncertainties, manufacturing and supply constraints, competition, intellectual property risks, and funding availability. Atossa undertakes no obligation to update forward-looking statements except as required by law.
Medical Disclaimer
(Z)-Endoxifen is investigational and has not been approved by the
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SOURCE Atossa Therapeutics Inc
FAQ
What did Atossa announce about (Z)-endoxifen for Duchenne muscular dystrophy (ATOS) on November 17, 2025?
How does (Z)-endoxifen differ from tamoxifen according to Atossa (ATOS)?
What mechanisms does the November 17, 2025 Atossa (ATOS) publication cite for (Z)-endoxifen in DMD?
Will Atossa (ATOS) pursue clinical studies for (Z)-endoxifen in DMD and female carriers?
When and where did Atossa present on (Z)-endoxifen for Duchenne carrier pathologies (ATOS)?
What follow‑up research did Atossa (ATOS) report about (Z)-endoxifen and utrophin?
