On November 10, 2025 Ontada, a leader in real-world oncology data and insights, reported it will present new research findings in an oral presentation at the ISPOR Europe 2025 conference, being held this week in Glasgow, Scotland. ISPOR is the leading professional society for health economics and outcomes research globally. The study, titled, "A Framework for Accelerating Clinical Development with Real-World External Control Arms (ECA) in Phase 2 Trials," addresses a critical gap in early-stage clinical research by offering a scalable approach to contextualizing single-arm trial data.

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This research highlights the potential of ECAs to enhance Phase 2 trials, which often lack traditional control groups and face challenges related to small sample sizes and slow patient accrual, particularly in oncology and rare diseases.

Key Data Findings

Real-world data (RWD) curation: The framework outlines a systematic approach to identifying and curating structured and unstructured RWD sources to build robust ECAs.
Trial emulation: It applies rigorous eligibility criteria from the trial to real-world cohorts, ensuring comparability.
Adaptive matching strategies: To address limited trial sample sizes, the framework uses simulation techniques to create synthetic datasets that support more robust patient matching.
Pilot application: The framework was successfully applied to a Phase 2 single arm trial of Tucatinib and Doxil in HER2+ metastatic breast cancer, demonstrating feasibility and value.
"Phase 2 trials are essential for identifying promising therapies, but their inherent design poses challenges to achieving rapid and robust inferences," said Jessica K. Paulus, ScD, vice president, Real World Research, Ontada. "This framework leverages high-quality real-world data and extends state-of-the-science methods in ECA development to overcome barriers in Phase 2 trial conduct, offering a practical path to accelerate development while maintaining scientific rigor. It’s a meaningful step toward more efficient clinical research."

Study Methodology

The framework was developed through a literature review of best practices in Phase 3 ECA design and adapted for Phase 2 single-arm trials. It incorporates simulation-based matching to address the challenge of limited trial patient data and was piloted using real-world data for a newly launched Phase 2 trial in HER2+ metastatic breast cancer.

Research at ISPOR Europe 2025

Ontada is showcasing its research capabilities at ISPOR with five accepted abstracts on topics such as evolving trends in patient demographics and diagnosis in gastric cancer, improving data quality in oncology electronic health record-derived databases, assessing secular trends in ovarian cancer diagnoses, and real-world associations between smoking status and genetic driver mutations in metastatic lung cancer. For a complete list of Ontada presentations, visit Ontada’s ISPOR Europe 2025 site. Additionally, visit Ontada at booth #616 at the Scottish Event Campus in Glasgow, November 10–12, to explore our latest research and real-world oncology solutions.

"At Ontada, we are committed to advancing oncology research through innovative applications of real-world data," said Christine Davis, president, Ontada. "Our presence at ISPOR Europe reflects the depth and diversity of our real-world research capabilities. From advancing trial design with external control arms to uncovering trends in cancer diagnosis and treatment, our work is helping shape the future of oncology through data-driven insights."

Ontada is a part of McKesson, which has an unmatched portfolio of oncology and multispecialty solutions and partners that provide research, insights, technologies and services that help to address barriers and improve cancer and multispecialty care.

(Press release, Ontada, NOV 10, 2025, View Source [SID1234659748])

On November 10, 2025 Leucid Bio ("Leucid" or the "Company"), a privately-held biotechnology company developing innovative Chimeric Antigen Receptor T-cell (CAR-T) therapies using its proprietary lateral CAR platform, reported an update on the Phase I/IIa AERIAL trial evaluating the safety and clinical activity of LEU011 in patients with relapsed/refractory solid tumours.

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Preliminary data from the ongoing AERIAL trial have established proof-of-concept for LEU011 by demonstrating key biological activity through evidence of encouraging pharmacokinetic (PK) and pharmacodynamic (PD) profiles. In particular, analysis of post-treatment biopsies (as evaluated by ddPCR and RNAScope) has shown tumour infiltration by LEU011 cells.

To date, treatment with LEU011 has been generally well tolerated. In addition, disease control (based on RECIST criteria) has been observed in multiple patients treated with the lowest dose of LEU011. Dose-escalation in the AERIAL trial continues as planned, with additional data from the trial anticipated during the first half of 2026.

Filippo Petti, Chief Executive Officer, Leucid Bio , said: "Although preliminary, we are highly encouraged by these initial data from the AERIAL trial which establish proof-of-concept for LEU011 in the treatment of solid tumours. Furthermore, they highlight LEU011’s dual mechanism of action targeting NKG2D stress ligands for tumour recognition and using CXCR2 signalling to enhance T-cell infiltration into the tumour microenvironment."

Dr. John Maher, Chief Scientific Officer, Leucid Bio, added: "Traditionally, CAR-Ts have struggled to deliver robust clinical responses in the treatment of solid tumours. We believe these preliminary data highlight the functional activity of LEU011 in the treatment of solid tumours given its unique mechanism of action. We anticipate that the early signals observed to date for LEU011 will continue to improve as we advance through the dose-escalation segment of the AERIAL trial."

AERIAL is a multi-centre, dose-escalation trial designed to evaluate the safety and clinical activity of LEU011 in patients with relapsed/refractory solid tumours, following a single intravenous dose of LEU011 after preconditioning chemotherapy. Additional information on the AERIAL trial can be found on ClinicalTrials.gov under the identifier NCT06193902.

Details of the ongoing AERIAL trial were presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting 2025.

Poster Title: A First-in-human Phase I/IIa dose escalation trial evaluating the safety and preliminary efficacy of LEU011, a novel CAR-T, in subjects with relapsed/refractory solid tumors (AERIAL)
Author / Presenter: R. Kristeleit
Session: Clinical Trials In Progress
Date: Saturday, November 8, 2025
Abstract Number: 578

About LEU011
LEU011 is a lateral CAR-T cell therapy targeting NKG2D stress ligands, which are overexpressed on more than 80% of human tumour cells and the cells within the surrounding tumour microenvironment. In addition to its novel architecture, LEU011 also co-expresses the chemokine receptor CXCR2 which is engineered to enhance cell trafficking and tumour infiltration, providing an extra mechanism to overcome significant limitations of CAR-T therapies currently in development for the treatment of relapsed/refractory solid tumours.

(Press release, Leucid Bio, NOV 10, 2025, View Source [SID1234659747])

On November 10, 2025 Akeso, Inc. (HKEX: 9926. HK) reported that the first patient has been dosed in a Phase I clinical trial evaluating the personalized mRNA vaccine AK154. This trial is investigating AK154 both as a monotherapy and as a combination with the company’s first-in-class bispecific antibodies, cadonilimab (PD-1/CTLA-4) and ivonescimab (PD-1/VEGF), for the adjuvant treatment of pancreatic cancer following surgical resection.

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AK154 is Akeso’s first mRNA-based therapeutic candidate to enter clinical development. This achievement marks a significant breakthrough for the company in the field of mRNA technology, following its established leadership in multi-specific antibodies, and entry into the clinic for its antibody-drug conjugates (ADCs).

AK154 is a personalized neoantigen vaccine developed by Akeso using its mRNA platform. It designs sequence-specific mRNA vaccines by sequencing tumor tissue and identifying immunogenic mutations with high affinity. This approach aims to overcome the "cold tumor" phenotype seen in pancreatic cancer.

AK154 shows a synergistic therapeutic effect with Akeso’s bispecific antibodies in enhancing anti-tumor immunity. Preclinical data demonstrated strong immunogenicity, potent anti-tumor activity, and a favorable safety profile. The combination of AK154 with cadonilimab or ivonescimab offers potentially promising new therapeutic options for pancreatic cancer patients.

(Press release, Akeso Biopharma, NOV 10, 2025, View Source [SID1234659746])

On November 10, 2025 Vascarta Inc., a healthspan focused, clinical stage biopharmaceutical company advancing safe, patient friendly therapies for pain, inflammation, and, in collaboration with the City University of New York (CUNY), reported the publication of a preclinical study demonstrating that STO-1, a first-in-class drug candidate, can selectively eliminate glioblastoma (GBM) cells in mice while avoiding harmful autoimmune reactions.

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STO-1 is a proprietary hybrid molecule in which curcumin is linked to paclitaxel with a linker that gets broken when STO-1 enters a cell. In the study, mice treated with STO-1 experienced a 67% long-term survival rate, with several animals achieving complete tumor clearance. The therapy works in part by reprogramming deactivated tumor-associated immune cells to attack the tumor, while leaving healthy immune function intact. This minimizes the prospect of undesirable side effects often seen with immunotherapies.

Dr. Probal Banerjee, the senior author and Professor, Biochemistry, Biology, Neuroscience, Chemistry at the College of Staten Island, CUNY, said:
"Unlike other immune therapies, which can trigger dangerous autoimmune reactions, STO-1 targets only the tumor-associated cells. This study demonstrates a promising new approach to treating glioblastoma safely."

Dr. Richard Prince, Chairman, Chief Executive and President, Vascarta, stated:
"These results highlight the potential of STO-1 to offer a safe and effective option for patients with glioblastoma without the side effects."

(Press release, City University of New York, NOV 10, 2025, View Source [SID1234659745])

On November 10, 2025 Verismo Therapeutics, a clinical-stage CAR T company developing a novel KIR-CAR platform technology, reported the presentation of new preclinical data from its lead clinical pipeline, SynKIR-110 to target mesothelin on solid tumors, at the Society of Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 2025 Annual Meeting. The oral presentation showcased their latest preclinical data demonstrating SynKIR-110’s improved safety profile and enhanced anti-tumor activity compared to conventional CAR T therapies.

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The oral presentation, "A Novel NK-cell Based Split-Signaling Killer Immunoglobulin Receptor (KIR)-Based CAR T Targeting Mesothelin, SynKIR-110, Shows Increased Safety Profile and Increased Efficacy in vitro and in vivo" was delivered by Dr. Nora Yucel, Principal Scientist at Verismo Therapeutics.

Key Findings:

Enhanced Tumor-Specific Serial Killing: In vitro, SynKIR-110 showed sustained killing of MSLN-expressing tumor cells while minimizing off-target activation and cytokine release.
Reduced Exhaustion and Improved Functional Persistence: Unlike conventional 41BB-CD3ζ CAR T cells, SynKIR-110 exhibited reduced activation and exhaustion markers in vitro, suggesting less tonic signaling and functional exhaustion.
Improved Anti-Tumor Activity: In NSG mouse models of mesothelioma, SynKIR-110 induced deep and prolonged regression of implanted tumors and metastatic spread.
Less Off-tumor Activity: SynKIR-110 cells were selectively enriched in tumors and reduced in, normal tissues – contrasting with the off-tumor accumulation in lung and normal tissues observed with conventional 41BB-CD3ζ CAR T designs.
"Conventional CAR T therapies have faced significant safety and efficacy challenges in treating solid tumors," said Dr. Laura Johnson, CSO/COO of Verismo Therapeutics. "Our SynKIR platform presents a truly novel signaling platform approach designed to allow CAR T cells to rest and recover when not engaged with tumors, which in turn may reduce T cell exhaustion and improve safety and efficacy of tumor-specific killing."

SynKIR-110 is currently being evaluated in a Phase 1 clinical trial (NCT05568680) in patients with advanced ovarian cancer, cholangiocarcinoma, and mesothelioma, and has received both Orphan Drug and Fast Track Designations from the U.S. Food and Drug Administration (FDA) for the treatment of mesothelioma.

(Press release, Verismo Therapeutics, NOV 10, 2025, View Source [SID1234659744])