Atossa's Rare Pediatric Disease Designation for (Z)-endoxifen in neuromuscular diseases qualifies for a future PRV upon FDA approval About Rare Pediatric Disease Designation The FDA's Rare Pediatric Disease designation is reserved for serious or life-threatening diseases that primarily affect individuals from birth to 18 years old and that meet the definition of a rare disease or condition within the meaning of Section 526 of the Federal Food, Drug & Cosmetic Act ("FD&C Act"). Drugs granted RPD designation ...

The Healthcare Technology Report is pleased to announce The Top Healthcare Technology CEOs of 2025.NEW YORK, Jan. 23, 2026 /PRNewswire-PRWeb/ -- The Healthcare Technology Report is pleased to announce The Top Healthcare Technology CEOs of 2025. In 2025, the pressure points in healthcare stayed the same—access, time, staffing, cost—but the expectations for technology changed. Leaders were measured by whether products could integrate into day-to-day work, produce reliable outcomes, and expand beyond early dep ...

Core Insights - Atossa Therapeutics, Inc. has announced that its Founder and CEO, Dr. Steven Quay, has been recognized as one of "The Top 50 Healthcare Technology CEOs of 2025" for his leadership in advancing innovative biotechnology solutions [1][2][3] Company Overview - Atossa Therapeutics is a clinical-stage biopharmaceutical company focused on developing novel therapies in oncology and other areas of significant unmet clinical need [4] - The company's lead product candidate, (Z)-endoxifen, is a precision-engineered endocrine therapy currently undergoing multiple Phase 2 clinical trials [2][4] - Atossa's strategy emphasizes disciplined capital allocation, focusing on programs that can enable future regulatory submissions and potential commercialization [4] Product Development - (Z)-endoxifen has been optimized as a potent selective estrogen receptor modulator/degrader (SERM/D) with additional PKC1 inhibition, designed for consistent systemic exposure independent of CYP2D6 metabolism [2] - The company is also exploring the application of (Z)-endoxifen for Duchenne Muscular Dystrophy (DMD) [2]

Several biotech and healthcare stocks posted notable gains in after-hours trading on Friday, driven by regulatory developments, financing updates, and investor sentiment.Atossa Therapeutics, Inc. (ATOS) rose 12.98% to $0.69, up $0.080 after announcing that the U.S. Food and Drug Administration's Office of Orphan Products Development granted Orphan Drug Designation to (Z)-endoxifen for the treatment of Duchenne muscular dystrophy. The designation marks a significant milestone for the company's pipeline.Immu ...

Core Insights - Atossa Therapeutics has received Orphan Drug Designation from the FDA for (Z)-endoxifen to treat Duchenne muscular dystrophy (DMD), which is a significant milestone for the company in its development efforts for this serious disease [1][2] Group 1: Company Overview - Atossa Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing innovative therapies in oncology and other areas with high unmet medical needs [7] - The company's lead product candidate, (Z)-endoxifen, is being evaluated for its potential applications in oncology and rare diseases [5][7] - Atossa has a growing global intellectual property portfolio supporting the (Z)-endoxifen program, including multiple recently issued U.S. patents and numerous pending applications worldwide [6] Group 2: Product Information - (Z)-endoxifen is a potent Selective Estrogen Receptor Modulator/Degrader (SERM/SERD) with demonstrated activity across multiple mechanisms of interest, showing a favorable safety profile distinct from tamoxifen [5] - The drug is not yet approved for any indication, and the company plans to continue engaging with the FDA as it advances its development efforts [2][5] Group 3: Disease Context - Duchenne Muscular Dystrophy (DMD) is a rare, progressive, X-linked neuromuscular disorder caused by mutations in the dystrophin gene, leading to severe symptoms and a substantial unmet medical need for effective treatments [4]

Financial Position - As of December 31, 2025, Atossa Therapeutics, Inc. estimated cash and cash equivalents of approximately $40 million[6] - The financial results for the year ended December 31, 2025, are still subject to finalization and should not be solely relied upon[7] Development Strategy - The company is focusing on the development and regulatory strategy for (Z)-Endoxifen, with potential market opportunities being explored[10] Risks and Compliance - Forward-looking statements indicate potential risks and uncertainties related to clinical development timelines and regulatory approvals[11] - The company aims to regain and maintain compliance with Nasdaq listing requirements as part of its strategic objectives[11]

Core Insights - Atossa Therapeutics has received a "Study May Proceed" letter from the FDA for its clinical study of (Z)-endoxifen in metastatic breast cancer, marking a significant regulatory milestone for the company [1][2] Group 1: Product Development - (Z)-endoxifen is a potent Selective Estrogen Receptor Modulator/Degrader (SERM/D) with demonstrated activity across multiple mechanisms, currently under evaluation for oncology and rare diseases [3] - The proprietary oral formulation of (Z)-endoxifen has shown a favorable safety profile and distinct pharmacology compared to tamoxifen, including ER-targeted effects and PKC inhibition [3] - The (Z)-endoxifen program is supported by a growing global intellectual property portfolio, including multiple recently issued U.S. patents and numerous pending applications worldwide [4] Group 2: Company Overview - Atossa Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing innovative medicines in oncology and other areas of significant unmet medical need [5] - The company's lead product candidate, (Z)-endoxifen, is currently in development across several clinical settings [5]

Core Insights - Atossa Therapeutics has been awarded the Research and Development Award in the Precision Endocrine Therapy category at the 2025 Clinical Trials Arena Excellence Awards for its work on (Z)-endoxifen [1][3] Group 1: Product Development - (Z)-endoxifen is a selective estrogen receptor modulator/degrader (SERM/D) designed to provide consistent systemic exposure independent of CYP2D6 metabolism, with applications in metastatic, neoadjuvant, adjuvant breast cancer, and Duchenne Muscular Dystrophy (DMD) [2][4] - The therapy has shown a favorable safety profile and pharmacology distinct from tamoxifen, including ER-targeted effects and PKC inhibition [4] Group 2: Company Strategy - Atossa emphasizes disciplined capital allocation, focusing on programs that can enable future regulatory submissions and potential commercialization [6] - The company is supported by a growing global intellectual property portfolio, including multiple recently issued U.S. patents and numerous pending applications worldwide [5] Group 3: Leadership and Innovation - The award reflects the company's commitment to precision endocrine therapy and its integrated approach to developing (Z)-endoxifen for patients with high unmet medical needs [3] - The Clinical Trials Arena Excellence Awards recognize companies demonstrating scientific rigor, innovation, and leadership in clinical research and drug development [3]

Core Insights - Atossa Therapeutics presented four clinical trial updates on (Z)-endoxifen at the San Antonio Breast Cancer Symposium, highlighting its potential in breast cancer treatment and risk reduction [1][2] Clinical Trial Updates - The company emphasized the therapeutic value of (Z)-endoxifen across the breast care continuum, with ongoing enrollment in the Phase 2 EVANGELINE study for neoadjuvant ER+/HER2- breast cancer [2] - Initial results from the RECAST trial suggest that short-term endocrine therapy combined with MRI response assessment may help identify low-risk DCIS patients who can avoid surgery [3][7] - Low-dose (Z)-endoxifen demonstrated excellent tolerability and biological activity, with significant reductions in Ki-67 (–72% median), MRI tumor volume, and ctDNA clearance in 70% of initially ctDNA-positive patients [5][8] - The EVANGELINE trial is the first to evaluate (Z)-endoxifen with ovarian function suppression as a neoadjuvant therapy for premenopausal ER+/HER2- breast cancer, showing strong early biological activity with 86% of patients achieving a Week 4 Ki-67 of 10% [10][15] Mechanistic Insights - (Z)-endoxifen maintains potent ER antagonist activity against key ESR1 mutations, stabilizing inactive receptor conformations and demonstrating strong suppression of ER signaling [6][14] - Transcriptomic analyses indicate that (Z)-endoxifen reverses multiple mutant ESR1-associated oncogenic pathways while restoring beneficial programs, highlighting its therapeutic potential for ER+/ESR1 mutant breast cancer [14]

Core Viewpoint - Atossa Therapeutics is advancing its regulatory strategy for (Z)-endoxifen, focusing on expedited pathways for metastatic, neoadjuvant, and risk-reduction settings in breast cancer treatment [1][2]. Regulatory Strategy - The company completed a Type C meeting with the FDA on November 17, 2025, which provided feedback on potential expedited regulatory pathways and development options for (Z)-endoxifen [1][2]. - The meeting clarified routes to accelerate clinical development and regulatory review, allowing Atossa to pursue a faster development strategy across multiple breast cancer indications [2][3]. Clinical Development - Atossa has submitted an Investigational New Drug (IND) application for its metastatic breast cancer program and anticipates additional IND submissions in 2026 to explore combination strategies [4]. - The clinical program has involved nearly 800 participants across multiple trials, with a focus on creating value-creating milestones [5]. Product Profile - (Z)-endoxifen is a potent Selective Estrogen Receptor Modulator/Degrader (SERM/D) that can inhibit and potentially degrade estrogen receptors, showing activity in tumors resistant to other therapies [5]. - The company is developing a proprietary oral formulation of (Z)-endoxifen that bypasses stomach acid, enhancing bioavailability and therapeutic integrity [6]. Intellectual Property - Atossa's (Z)-endoxifen program is supported by a growing global intellectual property portfolio, including four recently issued U.S. patents and numerous pending applications worldwide [7]. Clinical Trials and Indications - The company is preparing a dose-ranging study for metastatic breast cancer and continues enrollment in the Phase 2 EVANGELINE trial for neoadjuvant ER+/HER2- breast cancer [9]. - Development efforts also include a low-dose strategy targeting mammographic breast density and overall breast cancer risk [9].