Insilico Medicine and Atossa Therapeutics Publish AI-Driven Study in Nature Scientific Reports Identifying (Z)-Endoxifen as a Potential Therapeutic Candidate for Glioblastoma
MWN-AI** Summary
Insilico Medicine and Atossa Therapeutics have collaborated on a groundbreaking AI-driven study published in *Nature Scientific Reports* that identifies (Z)-endoxifen as a potential therapeutic candidate for glioblastoma multiforme (GBM). Glioblastoma, a highly aggressive brain tumor, has a dismal five-year survival rate of approximately 4%. The study leverages Insilico's advanced PandaOmics platform, which allows for extensive analysis of cancer mechanisms and drug repurposing opportunities.
Endoxifen, an active metabolite of tamoxifen, is well-known for its efficacy in hormone-resistant breast cancer but had not been properly explored in the context of GBM until now. The researchers evaluated over 900 cancer indications using transcriptomic signatures from endoxifen-treated datasets. Results revealed GBM as a top candidate for further research.
The study utilized multi-omics approaches, including transcriptomic integration and single-cell sequencing, to identify more than 1,400 shared genes between GBM tumors and endoxifen-treated cells. The findings demonstrated that endoxifen could reverse tumor growth and treatment resistance by counteracting pathways associated with proliferation, inflammation, and metabolic dysregulation. In laboratory validations, (Z)-endoxifen showed significant suppression of GBM cell proliferation and apoptotic effects, outperforming standard treatments like temozolomide.
Dr. Steven Quay, CEO of Atossa, emphasized the potential to expand treatment indications for endoxifen beyond its established use in breast cancer to include GBM, which represents an urgent medical need. Insilico's rapid drug discovery capabilities further enhance the feasibility of developing (Z)-endoxifen for GBM. Together, the collaboration signifies a promising step in utilizing artificial intelligence to identify new therapeutic pathways for challenging malignancies.
MWN-AI** Analysis
The recent collaboration between Insilico Medicine and Atossa Therapeutics, which led to the publication of their AI-driven study identifying (Z)-endoxifen as a potential therapeutic candidate for glioblastoma, presents a noteworthy development in the oncology sector. Insilico's advanced AI capabilities have enabled the rapid analysis of over 900 cancer indications, successfully identifying glioblastoma as a target, a market characterized by high unmet medical needs and limited therapeutic options.
Investors should consider the implications of this study for both Insilico and Atossa. For Insilico Medicine, this breakthrough reinforces its reputation as a leader in AI-driven drug discovery and positioning within the oncology landscape. By showcasing its proprietary PandaOmics platform, Insilico is not only enhancing its drug development efficiency but also demonstrating its potential for repurposing existing drugs. The swift identification of (Z)-endoxifen as a viable candidate for glioblastoma highlights the strategic advantages of leveraging artificial intelligence in drug discovery.
Atossa Therapeutics, meanwhile, is broadening its therapeutic focus and venturing into the lucrative brain cancer market. With glioblastoma being one of the most aggressive tumor types, this could represent a significant growth opportunity and bolster investor confidence in Atossa's pipeline. Successful results in clinical trials could lead to substantial valuation uplifts, making this a prime candidate for portfolio consideration.
Given the encouraging preliminary data and laboratory results indicating the efficacy of (Z)-endoxifen against GBM, market participants may view shares in both companies favorably. While the road ahead includes the usual clinical trials and regulatory hurdles, the combination of innovative drug repurposing strategies and AI technology positions both companies for potential breakthroughs in the highly competitive oncology market. Thus, active investors should monitor developments closely, as positive outcomes may lead to upward momentum for both stocks.
**MWN-AI Summary and Analysis is based on asking OpenAI to summarize and analyze this news release.
PR Newswire
CAMBRIDGE, Mass., Dec. 2, 2025 /PRNewswire/ -- Insilico Medicine ("Insilico"), a global leader in AI-powered drug discovery, and Atossa Therapeutics ("Atossa") (Nasdaq: ATOS), a clinical-stage biopharmaceutical company developing novel treatments for breast cancer and other serious conditions, announce the publication of a joint study evaluating the potential of (Z)-endoxifen for glioblastoma multiforme (GBM). The peer-reviewed article, now published in Nature's Scientific Reports, represents one of the most comprehensive AI-enabled analyses to date exploring whether endoxifen, an active metabolite of tamoxifen with known activity in endocrine-resistant breast cancer, may offer new therapeutic opportunities for one of the deadliest malignant brain tumors in adults. The study aimed to identify new oncology indications with high therapeutic potential for endoxifen, as monotherapy or in combination, by applying Insilico's AI-powered PandaOmics platform across a wide range of cancer types based on its mechanisms of action. Through this systematic evaluation, GBM emerged as a top candidate for further investigation.
The publication highlights progress in understanding glioblastoma, an aggressive primary brain tumor with a five-year survival rate of roughly 4%. By applying multi-omics and AI-enabled methods—including transcriptomic integration, computational modeling, single-cell sequencing, survival analysis, and experimental validation—the study offers mechanistic insights that support further exploration of endoxifen's potential role in this setting.
(Z)-Endoxifen, already clinically active in hormone-resistant breast cancer and known to act through both estrogen-dependent and estrogen-independent mechanisms, has long been considered a promising molecule for broader oncology applications, but has not been systematically explored in GBM. Using PandaOmics, researchers evaluated more than 900 cancer indications with weighted transcriptomic signatures from endoxifen-treated datasets. PandaOmics combined differential expression, pathway enrichment, protein–protein interaction networks, mechanistic NLP, and disease unmet-need modeling, ultimately ranking GBM among one of the highest-opportunity indications.
AI-driven analyses identified more than 1,400 genes shared between GBM tumors and endoxifen-treated cells, revealing strong reversal of biological programs that drive tumor growth and treatment resistance. Endoxifen was predicted to counteract pathways associated with uncontrolled proliferation, inflammation, metabolic dysregulation, and aggressive tumor behavior. Single-cell sequencing further pinpointed key malignant-cell genes linked to poor survival and aggressive subtypes, all of which were downregulated by endoxifen.
"This collaboration with Insilico Medicine provides a whole new indication in which we might explore the utility of endoxifen, and potentially significant new opportunities to address an extremely underserved set of cancer patients," said Steven Quay, M.D., Ph.D., CEO of Atossa Therapeutics. "Most of our joint projects have focused on advancing women's health, but this study allowed us to extend our work into glioblastoma, a disease with an urgent and unmet need, and not unlike other highly ranked indications, including but not limited to Duchenne muscular dystrophy (DMD) and multiple gynecologic-related cancers for which (Z)-Endoxifen may have excellent therapeutic outcomes. The results of our collaboration with Insilico highlight how AI-enabled discovery can uncover entirely new opportunities for a well-characterized molecule like endoxifen."
Laboratory validation closely matched the computational predictions. In vitro, (Z)-endoxifen significantly suppressed GBM cell proliferation and induced apoptosis, demonstrating greater cytotoxic activity than high-dose temozolomide and showing enhanced effects in combination. In vivo studies confirmed that endoxifen was well tolerated across all doses. Together, the multi-omic analyses and experimental findings highlight endoxifen as a promising therapeutic candidate for GBM and showcase how AI-powered discovery can reveal new opportunities for drug repurposing and cancer treatment innovation.
"Atossa is deeply committed to scientific leadership in the prevention and treatment of high risk breast cancers, and Insilico is honored to collaborate with the company to advance genuine innovation and expand indications across different areas of oncology," said Alex Zhavoronkov, Ph.D., Founder and CEO of Insilico Medicine. "This publication represents one of the early fruits of our joint research, and much of our collaborative work remains unpublished. We are hopeful that these efforts will ultimately translate into meaningful therapeutic programs."
Harnessing state-of-the-art AI and automation technologies, Insilico has significantly improved the efficiency of preclinical drug development. While traditional early-stage drug discovery typically requires 3 to 6 years, from 2021 to 2024 Insilico nominated 20 preclinical candidates, achieving an average turnaround - from project initiation to preclinical candidate (PCC) nomination - of just 12 to 18 months per program, with only 60 to 200 molecules synthesized and tested in each program.
About Insilico Medicine
Insilico Medicine, a leading and global AI-driven biotech company, utilizes its proprietary Pharma.AI platform and cutting-stage automated laboratory to accelerate drug discovery and advance innovations in life sciences research. By integrating AI and automation technologies and deep in-house drug discovery capabilities, Insilico is delivering innovative drug solutions for unmet needs including fibrosis, oncology, immunology, pain, and obesity and metabolic disorders. Additionally, Insilico extends the reach of Pharma.AI across diverse industries, such as advanced materials, agriculture, nutritional products and veterinary medicine.
For more information, please visit www.insilico.com.
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 U.S. Securities and Exchange Commission (SEC).
SOURCE Atossa Therapeutics Inc
FAQ**
How does the collaboration between Insilico Medicine and Atossa Therapeutics Inc. ATOS utilize AI to identify (Z)-endoxifen as a therapeutic candidate for glioblastoma compared to traditional drug discovery methods?
What specific mechanisms of action for (Z)-endoxifen in glioblastoma were revealed through the multi-omics and AI-enabled analyses conducted by Insilico Medicine and Atossa Therapeutics Inc. ATOS?
Given that (Z)-endoxifen is already clinically active in hormone-resistant breast cancer, what are the anticipated challenges Atossa Therapeutics Inc. ATOS might face in transitioning this compound into glioblastoma treatment?
How do laboratory validation results of (Z)-endoxifen's effectiveness against GBM compare with existing treatments, and what statistical outcomes can be shared by Atossa Therapeutics Inc. ATOS to support its potential as a new therapy?
**MWN-AI FAQ is based on asking OpenAI questions about Atossa Therapeutics Inc. (NASDAQ: ATOS).
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