On December 2, 2025 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, reported 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.

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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.

(Press release, Insilico Medicine, DEC 2, 2025, View Source [SID1234661063])

On December 2, 2025 Electra Therapeutics, a clinical stage biotechnology company pioneering therapies against novel targets for diseases in immunology and cancer, reported it will present two posters on the ELA026 clinical program in secondary hemophagocytic lymphohistiocytosis (sHLH) at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, taking place December 6-9 in Orlando, Florida.

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A trial-in-progress poster, presented by Dr. Swaminathan Iyer (MD Anderson Cancer Center), will highlight the SURPASS Phase 2/3 pivotal clinical study, which is evaluating the safety, efficacy, pharmacokinetics, and pharmacodynamics of ELA026 in newly diagnosed, treatment‑naïve adult and pediatric patients with sHLH. ELA026 is the first investigational therapy designed to treat sHLH regardless of underlying trigger, leveraging a novel mechanism of action that targets signal regulatory proteins (SIRP) on immune cells. By selectively depleting pathological myeloid cells and T lymphocytes that drive the cytokine storm and disease severity, ELA026 has the potential to reset the hyperinflammatory cascade at its source. The SURPASS study is actively enrolling at academic medical centers across the U.S. and Europe. Additional information is available at clinicaltrials.gov (NCT05416307).

A second poster, presented by Dr. Catherine Broome (MedStar Georgetown University) and Dr. Daniel Hausrath (Vanderbilt University Medical Center), in collaboration with EpidStrategies, will share results from an Electra-sponsored natural history study characterizing the disease burden and unmet need in patients with sHLH. This study identified a cohort of 232 adult patients over a 16‑year period through electronic health record queries to assess prognostic factors, clinical features, treatment patterns, healthcare utilization, and survival outcomes. The analysis underscored the life-threatening nature of sHLH, its high mortality, and the limited effectiveness of currently available therapies, reinforcing the significant unmet need for improved treatment options.

"We are delighted to connect with the scientific community at ASH (Free ASH Whitepaper) and share the clinical development progress of ELA026 in sHLH," said Kim-Hien Dao, DO, PhD, Chief Medical Officer of Electra Therapeutics. "Every day, patients with sHLH around the world face a devastating prognosis and very limited treatment options. We are committed to generating pivotal data to demonstrate ELA026’s potential to meaningfully improve outcomes for these patients."

Poster Details:

The SURPASS Trial: A Phase 2/3 Registrational Trial of ELA026 in Subjects with Treatment-Naïve Secondary Hemophagocytic Lymphohistiocytosis

Session Number and Name: 201. Granulocytes, Monocytes, and Macrophages: Poster I
Session Date and Time: Saturday, December 6, 5:30 – 7:30 p.m. EST
Location:West Halls B3-B4
Publication Number:#1223

Survival Outcomes of Patients with Secondary Hemophagocytic Lymphohistiocytosis (sHLH) at Two Academic Medical Centers in the United States

Session Number and Name: 905. Outcomes Research: Non-Malignant Conditions Excluding Hemoglobinopathies: Poster III
Session Date and Time: Monday, December 8, 6:00 – 8:00 p.m. EST
Location:West Halls B3-B4
Publication Number:#6243

ELA026 is the first investigational therapy to receive U.S. Food and Drug Administration Breakthrough Designation (BTD) and European Medicines Agency Priority Medicines (PRIME) designation for sHLH, reflecting its potential to address this life-threatening condition with significant unmet medical need.

About Secondary Hemophagocytic Lymphohistiocytosis (sHLH)
Secondary hemophagocytic lymphohistiocytosis (sHLH) is a rare, life-threatening hyperinflammatory disease with a lack of treatment options. It can be triggered by cancer, infection, autoimmune disease, or immunotherapy. sHLH is associated with a severe inflammatory response that requires immediate intervention. Without effective treatment, patients may experience multiorgan failure and death. sHLH is associated with high mortality early in the disease course, with malignancy-associated HLH (mHLH) patients having a mortality rate of approximately 50% at two months with available therapies.

(Press release, Electra Therapeutics, DEC 2, 2025, View Source [SID1234661045])

On December 2, 2025 Physician-scientists from Rutgers Cancer Institute and RWJBarnabas Health reported it will share new discoveries from their basic science and clinical research programs to advance the understanding of blood cancers, such as lymphoma, leukemia and multiple myeloma, as well as classical hematology disorders at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, taking place December 6–9, 2025, in Orlando, Florida (and online). Rutgers Cancer Institute, New Jersey’s only National Cancer Institute-designated Comprehensive Cancer Center together with RWJBarnabas Health, will be featured prominently at this year’s meeting with a total of 93 accepted peer-reviewed scientific abstracts, including 32 oral presentations, 60 poster presentations and 1 satellite symposium.

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"ASH is a vital platform to present critical breakthroughs that are transforming the science and clinical care in blood diseases, and our prominent involvement reflects our commitment to deliver meaningful progress for patients and the exceptional scientific leadership of our research teams," said Andrew Evens, DO, MBA, MSc, Deputy Director for Clinical Services and Chief Physician Officer, Rutgers Cancer Institute and Jack & Sheryl Morris Cancer Center and System Director of Medical Oncology, RWJBarnabas Health. "Our faculty’s remarkable work highlights the innovation and ground-breaking discoveries that define Rutgers Cancer Institute and RWJBarnabas Health. With the Jack & Sheryl Morris Cancer Center, New Jersey’s only freestanding cancer hospital, now open, we are entering a new era of care and research that brings world-class inpatient and outpatient treatment together with leading science to accelerate discoveries and improve outcomes for patients and families across our state and beyond."

Key scientific contributions from Rutgers Cancer Institute and RWJBarnabas Health at ASH (Free ASH Whitepaper) 2025:

Abs25-2080: Data from a comparative study evaluated PET-adaptive BEACOPP- versus ABVD-based therapies for advanced-stage classic Hodgkin lymphoma (cHL). Researchers compared outcomes from escBEACOPP and ABVD across 2,381 adults using data from four clinical trials and adjusting for baseline risk with the A-HIPI prognostic score. escBEACOPP was associated with significantly better progression-free survival, and multistate modeling showed a lower likelihood of treatment failure both before and after achieving one-year remission. These findings suggest that escBEACOPP offers stronger disease control in PET-adaptive strategies, while the A-HIPI score remains a powerful predictor of risk throughout the treatment course.

Abs25-2547: A study assessed the performance of the advanced-stage Hodgkin lymphoma international prognostic index (A-HIPI) in the SWOG S1826 randomized trial of nivolumab-AVD versus brentuximab-AVD. Using locked model parameters and Cox regression analyses, the A-HIPI demonstrated strong prognostic capability, with clear separation of 3-year progression-free survival across predicted risk quartiles and superior discrimination compared with the historic IPS-7. The A-HIPI performed similarly across treatment arms and remained superior in multivariable modeling, confirming it as a more precise, treatment-agnostic tool for risk stratification in newly diagnosed advanced-stage classical Hodgkin lymphoma.

Abs25-12859: Researchers conducted a large multicenter real-world analysis evaluating the use and safety of peripheral intravenous catheters (PIV) versus central venous catheters (CVC) for frontline anthracycline-based chemotherapy in lymphoma. Among 1,414 patients treated with CHOP- or ABVD-based regimens across eight U.S. centers, extravasation was rare in both groups, with only one documented case in the CVC cohort and two suspected cases in the PIV cohort. No significant differences were observed in induration, phlebitis/cellulitis, venous thromboembolism, or erythema. While PIV use was associated with higher rates of local pain/tenderness and infiltration, events were uncommon and self-limited. Overall, PIV access was found to be safe with a low rate of serious complications over more than 9,000 treatments, supporting its selective use despite the common default to CVC placement.

Abs25-8890: A first report from Part 1 of the Phase 3 OLYMPIA-3 study examined odronextamab plus chemotherapy in previously untreated diffuse large B-cell lymphoma (DLBCL). The Part 1A analysis assessed two dose levels in 22 patients, with primary endpoints focused on dose-limiting toxicities and treatment-emergent adverse events. No dose-limiting toxicities occurred, and the safety profile was manageable, with neutropenia, cytokine release syndrome, anemia, and infections as the most common adverse events. All patients experienced Grade ≥3 TEAEs, though CRS events were limited to Grade 1–2 and no tumor lysis syndrome was observed. Preliminary efficacy was encouraging, with ORR of 77.8% and CR rate of 66.7% at DL1 and both ORR and CR of 100% at DL2, with median duration of response not reached. These early results suggest rituximab may not be required to achieve depth of response in this frontline setting.

Abs25-7103: A retrospective real-world evidence study investigated racial and ethnic inequities in access to guideline-preferred first-line therapies for chronic lymphocytic leukemia (CLL). Using data from 4,452 patients in the Flatiron Health Research Database linked with neighborhood-level social determinants of health, researchers found that Black and Hispanic patients were more likely to live in high-deprivation areas and were less likely than White patients to receive NCCN-preferred novel therapies. Structural factors—including residential segregation, socioeconomic status, limited internet access, vehicle ownership, and health insurance—were associated with lower use of preferred first-line treatments. Findings underscore the role of neighborhood-level barriers in limiting equitable access to novel therapies.

The full list of presentations at this year’s American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition can be found here.

(Press release, Rutgers Cancer Institute of New Jersey, DEC 2, 2025, View Source [SID1234661064])

On December 2, 2025 Enterome, a clinical-stage company pioneering OncoMimics peptides, a new class of off-the-shelf, multi-targeted in vivo immune therapies that induce a fast and potent expansion of memory T cells to fight cancer, reported new survival analyses from Cohort 3 of the Phase 1/2 ROSALIE clinical trial. The data were reported at the 2025 Society for Neuro-Oncology (SNO) Annual Meeting. The ROSALIE trial is evaluating Enterome’s OncoMimics immune therapy EO2401 in combination with immune checkpoint inhibitor nivolumab, with or without anti-VEGF therapy bevacizumab, in patients with glioblastoma at first progression/recurrence. Top-line data from the trial had previously been released in November 2023.

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The analyses demonstrated a statistically significant survival benefit for patients who underwent a second surgery after their first glioblastoma recurrence when treated with EO2401 in combination with nivolumab and bevacizumab, compared with patients who did not undergo surgery (p = 0.027). Importantly, no survival benefit was observed for the supportive therapies, nivolumab or bevacizumab on a stand-alone basis after surgery, compared with no surgery. The investigators concluded in their poster that: "The data indicate that a randomized study evaluating EO2401 is warranted."

The poster was presented at SNO by the lead investigator of the trial, David Reardon M.D., and Professor of Medicine at Harvard Medical School and Clinical Director of the Center for Neuro-Oncology at Dana-Farber Cancer Institute, under the title: "EO2401 peptide immunotherapy + nivolumab +/- bevacizumab in first recurrent glioblastoma: treatment strategy optimization in the phase 1/2 study EOGBM1-18 / ROSALIE (NCT04116658)". The poster will be made available on the Enterome website.

ROSALIE is a multicenter, open-label, first-in-human study of EO2401 in 100 patients with glioblastoma. EO2401 / nivolumab +/- bevacizumab was well tolerated with a safety profile consistent with the safety profile of nivolumab, and when applicable bevacizumab, except the addition of local administration site reactions. EO2401 plus nivolumab generated fast, strong, and durable specific CD8 T cell immune responses against the EO2401-mimic peptides and target epitopes on tumor associated antigens. Furthermore, the investigators wrote in their poster that the addition of bevacizumab (to EO2401/nivolumab), which exerts strong antiedema properties, and putatively counteracts immunosuppression by VEGF, increased treatment duration and efficacy.

"This finding from the ROSALIE trial in this incredibly challenging patient population is another strong indication of the potential benefit that OncoMimics peptides can offer patients across a broad range of cancers," said Pierre Belichard, Chief Executive Officer of Enterome. "This, and the earlier clinical results make clear that larger, randomized studies of EO2401 are warranted."

EO2401 is an innovative, off-the-shelf OncoMimics multi-targeted in vivo immune therapy composed of three synthetically produced, short non-self HLA-A2 peptides with sequences derived from gut-bacteria (EO2316, EO2317, and EO2318). It is designed to rapidly expand – through peptide molecular mimicry – pre-existing CD8 T cells that cross-react with key glioblastoma tumor associated antigens (TAAs; IL13Ra2, BIRC5/survivin, and FOXM1). EO2401 also includes a universal CD4 helper epitope, UCP2 derived from hTERT, to support and enhance the immune response.

OncoMimics peptides consist of bacteria-derived peptide antigens that closely mimic tumor-associated antigens (TAAs). These antigens induce a fast and potent in vivo expansion of cytotoxic memory CD8+ T cells that were primed by gut bacteria, and are cross-reactive with TAAs. Because the peptides are "non-self", OncoMimics peptides avoid the self-tolerance that limits many cancer immunotherapies, enabling rapid, potent, and durable responses. The synthetically produced peptides are designed in silico, mining Enterome’s proprietary database of 23 million commensal bacteria genes. Each product combines multiple high-affinity peptides to broaden target coverage and mitigate tumor heterogeneity.

OncoMimics peptides are easy to manufacture, store, distribute and administer as an "off-the-shelf" subcutaneous injection. OncoMimics peptides have achieved rapid and potent responses in clinical testing in over 230 patients to date, with a benign safety profile.

(Press release, Enterome, DEC 2, 2025, View Source [SID1234661046])

On December 2, 2025 Solu Therapeutics, a biotechnology company pioneering novel therapies to eliminate disease-driving cells in cancer, immunology, and other therapeutic areas, reported that it will present details from its first-in-human Phase 1 clinical trial of STX-0712, the company’s novel CCR2-CyTAC (Chemokine Receptor Type 2 Cytotoxicity Targeting Chimera) for the treatment of chronic myelomonocytic leukemia (CMML) and acute myeloid leukemia (AML) in a poster session at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting being held December 6–9, 2025, in Orlando, Florida.

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The poster will highlight the design of the ongoing Phase 1, open-label, multicenter study, which is evaluating STX-0712 as monotherapy in patients with refractory or resistant CMML and relapsed or refractory monocytic or monocytic-predominant AML. Primary objectives include safety, determination of dose limiting toxicities, and determination of recommended Phase 2 dose. Secondary objectives include pharmacokinetic and pharmacodynamic characterization of STX-0712, tolerability, and preliminary evidence of antitumor activity. Key elements of the study design include dose escalation using a Bayesian Optimal Interval approach, planned dose expansion, and exploratory analyses assessing CCR2+ cell depletion, biomarkers, immune cell profiling and patient-reported outcomes.

STX-0712 is a CyTAC targeting the G-Protein Coupled Receptor CCR2, a selective marker expressed at high levels on malignant monocytes in these indications, which are key drivers in certain hematologic cancers. By targeting CCR2, STX-0712 is designed to selectively eliminate these malignant cells.

Presentation Details:
Abstract Title: A First-in-Human Study of STX-0712, a CCR2+ Cytotoxicity Targeting Chimera (CyTAC), in Patients with Chronic Myelomonocytic Leukemia (CMML) and Acute Myeloid Leukemia (AML)
Presenter: Guillermo Montalban Bravo, M.D., Department of Leukemia, Division of Cancer Medicine, MD Anderson Cancer Center
Date: Sunday, December 7, 2025
Time: 6:00pm-8:00pm EST
Location: Room OCCC- West Halls B3-B4
Session: 637. Myelodysplastic Syndromes: Clinical and Epidemiological: Poster II
Abstract Number: 14421

(Press release, Solu Therapeutics, DEC 2, 2025, View Source [SID1234661065])