On October 27, 2025 OncoNano Medicine, Inc. ("OncoNano") reported encouraging first-in-human results from Part 1 of its Phase 1 ON-5001 trial evaluating ONM-501, a dual-acting STING agonist, as monotherapy and in combination with Libtayo (cemiplimab), Regeneron’s PD-1 inhibitor, in patients with advanced solid tumors and lymphomas. The findings were shared during a poster presentation at the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) in Boston, Massachusetts.

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ONM-501 is designed to activate the STING pathway and stimulate both innate and adaptive immune responses within the tumor microenvironment. By linking a cyclic dinucleotide (cGAMP) to a proprietary polymer, ONM-501 prolongs STING activation and overcomes key limitations of earlier STING agonists. In preclinical models, this approach produced durable anti-tumor responses without triggering systemic inflammation, an effect now supported by emerging clinical results.

In the dose-escalation and dose-finding study, ONM-501 was well tolerated and demonstrated early signs of clinical benefit. Among 39 patients treated (15 with ONM-501 monotherapy and 24 in combination with cemiplimab), no dose-limiting toxicities were observed, and the most common adverse events were mild fatigue and injection-site reactions. Systemic interferon-γ levels remained within normal limits across all cohorts.

In the monotherapy arm, one patient achieved an objective response and three patients had prolonged stable disease, including a case of recurrent, immunotherapy-refractory uveal melanoma with disease control lasting over one year (390 days). In the combination arm, five patients experienced objective responses, including two complete in patients with cutaneous squamous cell carcinoma (cSCC).

"These data provide the first clinical evidence that sustained STING activation through our proprietary polymer conjugate can drive meaningful immune responses in solid tumors," said Kartik Krishnan, MD, PhD, Chief Executive Officer of OncoNano Medicine. "The safety profile and emerging efficacy signals, particularly in patients with advanced cutaneous malignancies, highlight the potential of ONM-501 to expand the reach of STING-based immunotherapy."

Part 2 of the ON-5001 study (NCT06022029) is now open and enrolling patients with advanced basal cell carcinoma, cutaneous squamous cell carcinoma and melanoma.

About ONM-501
ONM-501 is a dual-activating agonist of the stimulator of interferon genes ("STING") pathway composed of cGAMP (the endogenous activator of STING) linked to a proprietary pH-activated polymer (the OMNI polymer). ONM-501 is presently being studied in a Phase 1 clinical trial (ON-5001). In preclinical models, the dual activation of STING by ONM-501 has been shown to lead to a direct anti-tumor effect, as well as leading to an anti-tumor immune response over an extended period of time. Development of ONM-501 was funded in part by the Cancer Prevention and Research Institute of Texas.

(Press release, OncoNano Medicine, OCT 27, 2025, View Source [SID1234657048])

On October 27, 2025 Tolerance Bio, a biopharmaceutical company pioneering innovative approaches to extending healthspan by preserving, restoring, and manipulating the function of the thymus, the master regulator of immune tolerance, reported the publication of a scientific article titled "Patient-specific autologous thymic organoids support functional T-cell education leading to antitumor activity in humanized mice with melanoma xenografts" in Cancer Research Communications, a journal of the American Association for Cancer Research (AACR) (Free AACR Whitepaper). The research was led by Antonio Jimeno, M.D., Ph.D., from the Division of Medical Oncology, Department of Medicine, University of Colorado Anschutz School of Medicine, and Holger A. Russ, Ph.D., from the Diabetes Institute and the Department of Pharmacology and Therapeutics, College of Medicine at the University of Florida, Gainesville, and scientific co-founder of Tolerance Bio.

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The contributing teams utilized a novel approach, implanting patient-derived thymic cells generated via direct differentiation of induced pluripotent stem cells (iPSCs) into thymectomized and humanized immune-deficient mice, along with matched tumor tissues from a patient with metastatic melanoma. The key finding was that tumors implanted in mice with human thymic tissue grew significantly slower compared to tumors implanted in control and in humanized mice without thymic tissue. Viable melanoma content was also significantly decreased in mice bearing thymic organoids, associated with an increase in intra-tumoral activated T-cells. Whole gene sequencing identified multiple candidate neoantigens that were eliminated in cancer cells in thymus-bearing mice, suggesting a more effective identification and T-cell-driven tumor clearance.

"An experiment is only as good as the models used to conduct it. Over the last 15 years, we have generated increasingly complex laboratory and mouse models that can bear both human immune and cancer cells, which we call humanized mice, to realize the promise of immune therapy. A fundamental limitation of humanized mice, generated with donor blood stem cells and implanted with mismatched patient tumors, is that the fidelity of the interaction between the immune and tumor cells is limited by lack of the organ that facilitates immune cell education, the thymus," said Dr. Jimeno.

"Here we report the successful generation of a novel model that incorporates functional thymic organoids derived from iPSC generated using patient peripheral blood with tumors from the same patient, providing an important step towards improved personalized models," remarked Dr. Russ.

"These results represent meaningful advance in oncology, both from a therapeutic and a diagnostic point of view, as iPSC thymic cells have the potential to serve as a cell therapy for cancer, alone or along immune agents, the patient derived xenograft model can improve new drug development by more accurately identifying promising drugs and vaccines, and it will allow for personalized medicine by testing which immune therapy is more likely to work for a specific patient," added Dr. Jimeno. "We have a lot of work ahead, but I am looking forward to extending our interdisciplinary work into the future to realize this potential."

"Tolerance Bio is proud to collaborate with Dr. Jimeno and Dr. Russ, and their superb teams at U. Colorado and U. Florida. Their work lays the foundation for the use of iPSC thymic cells in immune diseases, including cancer," said Dr. Francisco Leon, M.D., Ph. D., CEO of Tolerance Bio. "The changes in the tumor growth kinetics provide critical proof of principle for the ability of iPSC thymic cells to reconstitute a T cell immune system in vivo with beneficial functional consequences. As we advance towards the clinic, we have developed a robust manufacturing process for our allogenic, off-the-shelf iPSC thymic cell product, and we plan to initiate pre-investigational new drug (IND) studies in the near future."

(Press release, Tolerance Bio, OCT 27, 2025, View Source [SID1234657047])

On October 27, 2025 IDEAYA Biosciences, Inc. (NASDAQ: IDYA), a precision medicine oncology company committed to the discovery and development of targeted therapeutics, reported its participation in the upcoming investor relations events.

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Citi’s 2025 SMID Cap Biopharma Call Series
Thursday, November 6th, 2025 at 12:00 PM ET

Fireside chat with Yujiro S. Hata, President and Chief Executive Officer, hosted by Yigal D. Nochomovitz, Ph.D., Director, SMid Cap Biotech Analyst

Jefferies Global Healthcare Conference in London
Tuesday, November 18th, 2025 at 1:00 PM GMT | 8:00 AM ET

Fireside chat with Yujiro S. Hata, Chief Executive Officer, hosted by Maury Raycroft, Ph.D. Equity Research Analyst, Biotechnology
A live audio webcast of the conference events, as permitted by the conference host, will be available at the "Investors/Events" section of the IDEAYA website at View Source and/or through the conference host. A replay of available webcasts will be accessible for 30 days following the live event.

(Press release, Ideaya Biosciences, OCT 27, 2025, View Source [SID1234657046])

On October 27, 2025 OncoC4 reported that China’s National Medical Product Administration (NMPA) has granted Breakthrough Therapy Designation (BTD) for gotistobart (BNT316/ONC392) for the treatment of patients with squamous non-small cell lung cancer (sqNSCLC) who have progressed on prior standard immuno-oncology therapies (IO). Gotistobart is a next-generation anti-CTLA-4 antibody candidate which is being jointly developed by BioNTech and OncoC4.

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Squamous non-small cell lung cancer (sqNSCLC) is an aggressive subtype of lung cancer accounting for approximately 25-30% of all NSCLC cases, the most common type of lung cancer1. The 5-year relative survival rate for patients with sqNSCLC is 15%2. While novel first line treatments combining immune-checkpoint inhibition and chemotherapy have improved outcomes of patients with NSCLC, the medical need remains high for novel treatments options, especially for patients with lung cancer that have progressed on first-line therapy and have limited treatment options.

"For the past 5 years, limited advances in treatment outcomes have been reported for EGFR wild-type NSCLC patients who progressed on the first-line IO and chemotherapies. We believe gotistobart has the potential to broaden the reach of CTLA-4-targeting immunotherapy and to improve outcomes for patients living with sqNSCLC." said Pan Zheng, MD, PhD, Co-founder and Chief Medical Officer of OncoC4.

"Gotistobart is a next-gen acid pH-sensitive anti-CTLA4 mAb that conferred an improved therapeutic index compared with approved anti-CTLA-4 antibodies by preserving CTLA-4 on regulatory T cells as demonstrated in preclinical models. The emerging data support clinical proof of concept for this new approach", added Yang Liu, PhD, Co-Founder, Chief Executive Officer, and Chief Scientific Officer of OncoC4.

Gotistobart is currently being evaluated in a registrational two-stage randomized Phase 3 trial, PRESERVE-003 (NCT05671510). The clinical trial assesses the efficacy and safety of gotistobart as monotherapy compared to the standard-of-care chemotherapy (docetaxel) in patients with metastatic sqNSCLC that progressed under previous PD-(L)1-inhibitor treatment. The initiation of the Phase 3 trial was based on positive safety and efficacy data from an ongoing Phase 1/2 trial (NCT04140526) with gotistobart alone and in combination with pembrolizumab in patients with advanced solid tumors. The program received Fast Track Designation from the U.S. Food and Drug Administration (FDA) in 2022 and represents an innovative approach which aims to leverage the full potential of CTLA-4-targeting therapies.

Breakthrough Therapy Designation is an NMPA program designed to expedite the development and regulatory review of investigational therapies that are designed to address serious or life-threatening conditions. To receive the designation, the candidate needs to demonstrate preliminary clinical evidence that indicates that it may offer substantial improvement over existing therapies on one or more clinically significant endpoints. The NMPA’s BTD designation is based on the Stage 1 safety and efficacy data of the PRESERVE-003 trial. The detailed data will be reported in an upcoming medical conference in December 2025. With the Breakthrough Therapy designation, the development of gotistobart may benefit from more frequent engagement with the NMPA, which will support the collection of appropriate data needed to accelerate development and may also allow for priority review if the relevant criteria are met.

About PRESERVE-003

The two-stage, open-label, randomized Phase 3 trial, PRESERVE-003 (NCT05671510), will assess the efficacy and safety of gotistobart (BNT316/ONC-392) as monotherapy compared to the standard-of-care chemotherapy (docetaxel) in patients with metastatic NSCLC that progressed under previous PD-(L)1-inhibitor treatment. Stage 1, the non-pivotal dose-confirmation stage, assessed the efficacy and safety of two gotistobart dosing regimens in comparison to docetaxel in patients with squamous and non-squamous non-small cell lung cancer (NSCLC). Stage 2 is the pivotal stage of the trial and will assess the safety and efficacy of gotistobart in squamous cell NSCLC. The primary endpoint measure is overall survival. Secondary endpoints include overall response rate, progression-free survival and adverse event profile. Approximately 600 patients are planned to be enrolled at clinical sites in 12 countries and regions globally, including China, Germany, Italy, South Korea, Türkiye, the United Kingdom, and the United States.

About gotistobart (BNT316/ONC392)

Gotistobart is licensed to BioNTech for commercialization and jointly developed clinically by OncoC4 and BioNTech for oncology indications. There are several ongoing clinical trials in different tumor types, investigating gotistobart either as monotherapy or in combination with other therapeutic agents.

(Press release, OncoC4, OCT 27, 2025, View Source [SID1234657045])

On October 27, 2025 Kazia Therapeutics Limited ("Kazia" or the "Company") reported its intention to request and hold a follow-up Type C meeting with the U.S. Food & Drug Administration (FDA) to discuss overall survival (OS) findings in newly diagnosed glioblastoma (GBM) patients treated with paxalisib and to seek agency feedback on a potential regulatory pathway aligned with the FDA Oncology Center of Excellence’s Project FrontRunner initiative.

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"GBM remains one of the most lethal cancers with limited therapeutic options. In line with the FDA Oncology Center of Excellence’s Project FrontRunner initiative, we intend to engage the Agency to discuss whether the overall survival data generated in newly diagnosed GBM patients treated with paxalisib may be adequate to support a conditional approval pathway," said Dr. John Friend, M.D., Chief Executive Officer of Kazia Therapeutics. "Consistent with this framework, Kazia will propose initiation of the post-approval, randomized Phase 3 confirmatory study prior to submission of the NDA, ensuring that our regulatory strategy fully reflects the FDA’s renewed emphasis on overall survival as the most meaningful endpoint for patients and clinicians."

In its recently issued draft guidance, the FDA stated that overall survival is the "gold standard" endpoint in oncology and "should be prioritized as the primary endpoint when feasible," particularly in diseases with a short natural history where survival can be reliably assessed. Kazia believes GBM is precisely such a setting and intends to present survival analyses, supporting clinical safety, and planned confirmatory trial design for FDA discussion.

Project FrontRunner is an FDA Oncology Center of Excellence initiative encouraging sponsors to consider when it may be appropriate to seek approval of cancer drugs for advanced or metastatic disease in an earlier clinical setting, rather than the traditional approach of developing therapies only for patients who have exhausted available treatment options.

As announced in July 2024, in the prespecified secondary analysis in newly diagnosed (up-front) unmethylated GBM patients, median OS was 15.54 months in the paxalisib arm (n = 54) versus 11.89 months for concurrent standard of care (SOC) (n = 46). Kazia intends to reference Project FrontRunner principles in its Type C briefing package, including an OS-driven confirmatory study plan in newly diagnosed GBM.

"We are moving decisively to bring paxalisib forward in GBM using the endpoints that matter most to patients and physicians," added Dr. Friend. "Our objective is to work collaboratively with the FDA under the guiding principles of Project FrontRunner to pursue a conditional approval in the front-line treatment setting of glioblastoma. In parallel, Kazia will initiate the post-approval, randomized Phase 3 study prior to filing the NDA, ensuring that our development plan fully aligns with the Agency’s modernized, patient-focused framework."

Kazia also notes that leading oncology companies have begun publicly referencing Project FrontRunner in successful FDA actions, underscoring the initiative’s growing relevance for sponsors developing first-line or earlier-setting therapies.

(Press release, Kazia Therapeutics, OCT 27, 2025, View Source [SID1234657044])