Atossa's New Research Findings on DMD
Written by Emily J. Thompson, Senior Investment Analyst
Updated: May 20 2026
0mins
Source: PRnewswire
- Publication Achievement: Atossa Therapeutics' manuscript titled '(Z)-Endoxifen as a Potential Modulator of Utrophin Pathways in Duchenne Muscular Dystrophy' has been accepted for publication in Degenerative Neurological and Neuromuscular Disease, highlighting utrophin's compensatory role in the absence of dystrophin, potentially offering new avenues for DMD treatment.
- Mechanistic Insights: The study indicates that (Z)-endoxifen may enhance utrophin expression and function by influencing multiple signaling pathways, such as PKC beta-1 and estrogen receptor signaling, thereby supporting muscle cell stability and regeneration, which holds significant clinical application potential.
- Future Research Plans: The manuscript outlines next steps including preclinical evaluations in dystrophin-deficient models and the development of biomarkers focused on utrophin expression and localization to further validate its therapeutic effects.
- Market Potential: Atossa's (Z)-endoxifen has received FDA orphan drug and Rare Pediatric Disease designations, and upon approval, it may benefit from a Priority Review Voucher, with substantial market potential evidenced by past sales of such vouchers ranging from $100 million to $205 million over the last 18-24 months.
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Analyst Views on ATOS
Wall Street analysts forecast ATOS stock price to rise
4 Analyst Rating
4 Buy
0 Hold
0 Sell
Strong Buy
Current: 5.190
Low
7.00
Averages
18.33
High
40.00
Current: 5.190
Low
7.00
Averages
18.33
High
40.00
About ATOS
Atossa Therapeutics, Inc. is a clinical-stage biopharmaceutical company. It is engaged in developing medicines in areas of unmet medical need in oncology with a focus on women’s breast cancer and other breast conditions. Its lead drug candidate under development is oral (Z)-endoxifen, which it is developing for both the prevention and treatment of breast cancer. (Z)-endoxifen is the potent Selective Estrogen Receptor Modulator for estrogen receptor inhibition and causes estrogen receptor degradation. In addition to its potent anti-estrogen effects, (Z)-endoxifen has been shown to target PKCB1, a known oncogenic protein, at clinically attainable blood concentrations. It is developing a form of (Z)-endoxifen which is administered orally for the potential treatment of breast cancer and reduction of breast density. It has completed four Phase I clinical studies and two Phase II clinical studies with its proprietary (Z)-endoxifen (including oral and topical formulations).
About the author
Emily J. Thompson
Emily J. Thompson, a Chartered Financial Analyst (CFA) with 12 years in investment research, graduated with honors from the Wharton School. Specializing in industrial and technology stocks, she provides in-depth analysis for Intellectia’s earnings and market brief reports.
Atossa Presents New Data Highlighting (Z)-Endoxifen's Potential in Breast Cancer
- Clinical Data Presentation: Atossa Therapeutics announced that two abstracts accepted for the 2026 American Society of Clinical Oncology Annual Meeting highlight the significant estrogen receptor inhibition of (Z)-endoxifen in ER+ breast cancer, potentially providing new treatment options.
- Preclinical Research Findings: New data demonstrate that (Z)-endoxifen achieves robust estrogen receptor inhibition across clinically relevant ESR1 mutations, which commonly lead to endocrine therapy resistance, indicating the drug's potential for patients with limited treatment options.
- EVANGELINE Trial Progress: The ongoing EVANGELINE study evaluates the effects of (Z)-endoxifen combined with goserelin as neoadjuvant therapy for ER+/HER2- premenopausal women, aiming to improve clinical response and safety outcomes.
- Broad Market Potential: Atossa's (Z)-endoxifen program is supported by a growing global intellectual property portfolio, showcasing its extensive application potential in oncology and rare diseases, which may lead to significant market growth opportunities for the company.
See More
Atossa Presents Robust ER Inhibition Data for (Z)-Endoxifen in Breast Cancer
- Clinical Data Highlights: Atossa Therapeutics' preclinical data reveals that (Z)-endoxifen exhibits robust estrogen receptor inhibition in clinically relevant concentrations, reducing ER activity to 16-26% of control in MCF-7 cells, indicating its potential in treating endocrine-resistant breast cancer.
- EVANGELINE Study Progress: The ongoing EVANGELINE study evaluates the combination of daily 40 mg (Z)-endoxifen and goserelin as neoadjuvant therapy, aiming to determine if patients with baseline Ki-67 >10% can achieve levels of 10% or less after four weeks, showcasing the treatment's promise in premenopausal women.
- Clinical Trial Design: Utilizing a Simon two-stage design, the study initially enrolls 20 patients, with plans to recruit an additional 25 if results are promising, focusing on assessing the efficacy and safety of the treatment, thereby advancing the clinical application of (Z)-endoxifen.
- Market Potential: Given the prevalence of ESR1 mutations in ER+ breast cancer patients, (Z)-endoxifen, as a selective estrogen receptor modulator/degrader, may offer new treatment options for these patients, addressing significant unmet needs and presenting substantial market opportunities.
See More
Atossa's New Research Findings on DMD Treatment
- Publication Announcement: Atossa Therapeutics' manuscript titled '(Z)-Endoxifen as a Potential Modulator of Utrophin Pathways in Duchenne Muscular Dystrophy' has been accepted by Degenerative Neurological and Neuromuscular Disease, highlighting the significance of utrophin in muscle stability and potentially offering new insights for DMD treatment.
- Mechanistic Exploration: The study indicates that (Z)-endoxifen may enhance utrophin expression and function, influencing multiple signaling pathways relevant to DMD, including PKC beta-1 signaling and calcium homeostasis, thereby providing new therapeutic options for DMD patients.
- Preclinical Evaluation Plans: Atossa plans to conduct preclinical evaluations in dystrophin-deficient models and develop biomarkers to monitor utrophin expression and localization, aiming to lay the groundwork for future clinical trials.
- Market Potential Analysis: Atossa's (Z)-endoxifen has received FDA orphan drug and Rare Pediatric Disease designations, which could lead to a Priority Review Voucher upon approval, indicating significant market potential, with past sales of such vouchers ranging from $100 million to $205 million over the last 18-24 months.
See More
Atossa's New Research Findings on DMD
- Publication Achievement: Atossa Therapeutics' manuscript titled '(Z)-Endoxifen as a Potential Modulator of Utrophin Pathways in Duchenne Muscular Dystrophy' has been accepted for publication in Degenerative Neurological and Neuromuscular Disease, highlighting utrophin's compensatory role in the absence of dystrophin, potentially offering new avenues for DMD treatment.
- Mechanistic Insights: The study indicates that (Z)-endoxifen may enhance utrophin expression and function by influencing multiple signaling pathways, such as PKC beta-1 and estrogen receptor signaling, thereby supporting muscle cell stability and regeneration, which holds significant clinical application potential.
- Future Research Plans: The manuscript outlines next steps including preclinical evaluations in dystrophin-deficient models and the development of biomarkers focused on utrophin expression and localization to further validate its therapeutic effects.
- Market Potential: Atossa's (Z)-endoxifen has received FDA orphan drug and Rare Pediatric Disease designations, and upon approval, it may benefit from a Priority Review Voucher, with substantial market potential evidenced by past sales of such vouchers ranging from $100 million to $205 million over the last 18-24 months.
See More
Atossa Hosts Key Opinion Leader Event on (Z)-Endoxifen
- Expert Participation: Atossa Therapeutics announced a virtual key opinion leader event on May 19, 2026, featuring internationally recognized surgical oncologist Dr. Laura Esserman, aimed at discussing clinical and translational data supporting the development of (Z)-endoxifen for innovative breast cancer treatment.
- Therapeutic Potential: The company believes that (Z)-endoxifen may serve as more than a single-indication therapy, potentially becoming an endocrine therapy platform across multiple ER-positive breast cancer settings, a perspective that will be further validated through Dr. Esserman's insights.
- Clinical Research Outlook: The event will address various topics of interest to clinicians, investors, and potential strategic partners, emphasizing the significance of (Z)-endoxifen in personalized breast cancer treatment, which could attract more investment and collaboration opportunities.
- Knowledge Dissemination: The event will be conducted via Zoom, allowing participants to submit questions in advance, and a replay of the webinar will be available on the company's investor section of the website post-event, ensuring broad dissemination and access to information.
See More
Atossa Therapeutics Q1 Financial Results
- Loss per Share: Atossa Therapeutics reported a GAAP EPS of -$1.11 for Q1 2026, indicating ongoing challenges in profitability that could undermine investor confidence.
- Increased Operating Expenses: The total operating expenses for the three months ended March 31, 2026, reached $9.9 million, up $2.5 million from $7.4 million in the same period of 2025, reflecting rising costs that may impact future financial flexibility.
- Reasons for Expense Growth: The increase in operating expenses is primarily attributed to higher spending on research and marketing, suggesting that Atossa is preparing for future product launches and market expansion, but may exert pressure on cash flow in the short term.
- Financial Health Risks: The ongoing losses and rising operating expenses could lead to greater pressure on Atossa regarding financing and investor trust, potentially affecting its long-term strategic development and market competitiveness.
See More
Atossa Presents New Data Highlighting (Z)-Endoxifen's Potential in Breast Cancer
- Clinical Data Presentation: Atossa Therapeutics announced that two abstracts accepted for the 2026 American Society of Clinical Oncology Annual Meeting highlight the significant estrogen receptor inhibition of (Z)-endoxifen in ER+ breast cancer, potentially providing new treatment options.
- Preclinical Research Findings: New data demonstrate that (Z)-endoxifen achieves robust estrogen receptor inhibition across clinically relevant ESR1 mutations, which commonly lead to endocrine therapy resistance, indicating the drug's potential for patients with limited treatment options.
- EVANGELINE Trial Progress: The ongoing EVANGELINE study evaluates the effects of (Z)-endoxifen combined with goserelin as neoadjuvant therapy for ER+/HER2- premenopausal women, aiming to improve clinical response and safety outcomes.
- Broad Market Potential: Atossa's (Z)-endoxifen program is supported by a growing global intellectual property portfolio, showcasing its extensive application potential in oncology and rare diseases, which may lead to significant market growth opportunities for the company.
See More
Atossa Presents Robust ER Inhibition Data for (Z)-Endoxifen in Breast Cancer
- Clinical Data Highlights: Atossa Therapeutics' preclinical data reveals that (Z)-endoxifen exhibits robust estrogen receptor inhibition in clinically relevant concentrations, reducing ER activity to 16-26% of control in MCF-7 cells, indicating its potential in treating endocrine-resistant breast cancer.
- EVANGELINE Study Progress: The ongoing EVANGELINE study evaluates the combination of daily 40 mg (Z)-endoxifen and goserelin as neoadjuvant therapy, aiming to determine if patients with baseline Ki-67 >10% can achieve levels of 10% or less after four weeks, showcasing the treatment's promise in premenopausal women.
- Clinical Trial Design: Utilizing a Simon two-stage design, the study initially enrolls 20 patients, with plans to recruit an additional 25 if results are promising, focusing on assessing the efficacy and safety of the treatment, thereby advancing the clinical application of (Z)-endoxifen.
- Market Potential: Given the prevalence of ESR1 mutations in ER+ breast cancer patients, (Z)-endoxifen, as a selective estrogen receptor modulator/degrader, may offer new treatment options for these patients, addressing significant unmet needs and presenting substantial market opportunities.
See More
Atossa's New Research Findings on DMD Treatment
- Publication Announcement: Atossa Therapeutics' manuscript titled '(Z)-Endoxifen as a Potential Modulator of Utrophin Pathways in Duchenne Muscular Dystrophy' has been accepted by Degenerative Neurological and Neuromuscular Disease, highlighting the significance of utrophin in muscle stability and potentially offering new insights for DMD treatment.
- Mechanistic Exploration: The study indicates that (Z)-endoxifen may enhance utrophin expression and function, influencing multiple signaling pathways relevant to DMD, including PKC beta-1 signaling and calcium homeostasis, thereby providing new therapeutic options for DMD patients.
- Preclinical Evaluation Plans: Atossa plans to conduct preclinical evaluations in dystrophin-deficient models and develop biomarkers to monitor utrophin expression and localization, aiming to lay the groundwork for future clinical trials.
- Market Potential Analysis: Atossa's (Z)-endoxifen has received FDA orphan drug and Rare Pediatric Disease designations, which could lead to a Priority Review Voucher upon approval, indicating significant market potential, with past sales of such vouchers ranging from $100 million to $205 million over the last 18-24 months.
See More
Atossa's New Research Findings on DMD
- Publication Achievement: Atossa Therapeutics' manuscript titled '(Z)-Endoxifen as a Potential Modulator of Utrophin Pathways in Duchenne Muscular Dystrophy' has been accepted for publication in Degenerative Neurological and Neuromuscular Disease, highlighting utrophin's compensatory role in the absence of dystrophin, potentially offering new avenues for DMD treatment.
- Mechanistic Insights: The study indicates that (Z)-endoxifen may enhance utrophin expression and function by influencing multiple signaling pathways, such as PKC beta-1 and estrogen receptor signaling, thereby supporting muscle cell stability and regeneration, which holds significant clinical application potential.
- Future Research Plans: The manuscript outlines next steps including preclinical evaluations in dystrophin-deficient models and the development of biomarkers focused on utrophin expression and localization to further validate its therapeutic effects.
- Market Potential: Atossa's (Z)-endoxifen has received FDA orphan drug and Rare Pediatric Disease designations, and upon approval, it may benefit from a Priority Review Voucher, with substantial market potential evidenced by past sales of such vouchers ranging from $100 million to $205 million over the last 18-24 months.
See More
Atossa Hosts Key Opinion Leader Event on (Z)-Endoxifen
- Expert Participation: Atossa Therapeutics announced a virtual key opinion leader event on May 19, 2026, featuring internationally recognized surgical oncologist Dr. Laura Esserman, aimed at discussing clinical and translational data supporting the development of (Z)-endoxifen for innovative breast cancer treatment.
- Therapeutic Potential: The company believes that (Z)-endoxifen may serve as more than a single-indication therapy, potentially becoming an endocrine therapy platform across multiple ER-positive breast cancer settings, a perspective that will be further validated through Dr. Esserman's insights.
- Clinical Research Outlook: The event will address various topics of interest to clinicians, investors, and potential strategic partners, emphasizing the significance of (Z)-endoxifen in personalized breast cancer treatment, which could attract more investment and collaboration opportunities.
- Knowledge Dissemination: The event will be conducted via Zoom, allowing participants to submit questions in advance, and a replay of the webinar will be available on the company's investor section of the website post-event, ensuring broad dissemination and access to information.
See More
Atossa Therapeutics Q1 Financial Results
- Loss per Share: Atossa Therapeutics reported a GAAP EPS of -$1.11 for Q1 2026, indicating ongoing challenges in profitability that could undermine investor confidence.
- Increased Operating Expenses: The total operating expenses for the three months ended March 31, 2026, reached $9.9 million, up $2.5 million from $7.4 million in the same period of 2025, reflecting rising costs that may impact future financial flexibility.
- Reasons for Expense Growth: The increase in operating expenses is primarily attributed to higher spending on research and marketing, suggesting that Atossa is preparing for future product launches and market expansion, but may exert pressure on cash flow in the short term.
- Financial Health Risks: The ongoing losses and rising operating expenses could lead to greater pressure on Atossa regarding financing and investor trust, potentially affecting its long-term strategic development and market competitiveness.
See More
