TG4050 / Transgene, NEC Corp - LARVOL DELTA

A viral-based individualized neoantigen vaccine as adjuvant treatment in resected head and neck squamous cell carcinoma: a randomized Phase I trial. (PubMed, Nat Commun) - P1/2 | "Vaccine neoantigen-specific CD8+ T cells had an effector phenotype, displayed high expression of cytotoxic and tissue-resident markers, were polyclonal and comprised both de novo responses and amplification of pre-existing tumor-infiltrating T cell clones. Together, these translational data are consistent with the hypothesis in which single-agent delivery of TG4050 induces long-lasting tumor neoantigen-specific cytotoxic T cell responses that can prevent tumor recurrence."

Clinical • Journal • P1 data • Head and Neck Cancer • Oncology • Solid Tumor • Squamous Cell Carcinoma • Squamous Cell Carcinoma of Head and Neck • CD8

Transgene Completes Patient Randomization in Phase 2 Part of Clinical Trial Evaluating TG4050 in the Adjuvant Treatment of Head and Neck Cancer (The Manila Times) - "The primary endpoint of the trial is 2-year disease-free survival (DFS) and is expected to read out as soon as all patients from the Phase 2 part achieve 2-year follow-up from randomization unless an event (relapse, death) occurs earlier, with results anticipated by the end of Q1 2028....Data from patients in the Phase 1 part of the trial have already shown that multiple subcutaneous injections of TG4050 were well-tolerated with no unexpected safety signals. 3-year DFS follow-up of Phase 1 patients is expected in Q2/Q3 2026."

P1 data • P2 data • Trial status • Squamous Cell Carcinoma of Head and Neck

Personalized vaccine TG4050 induces polyepitopic immune responses against private neoantigens in resected HPV negative head and neck cancers (AACR 2024) - "TG4050 design and manufacturing was feasible in Head and Neck cancers. Vaccination was safe and induced polyclonal immune responses against vaccine targets alone or during concurrent treatment with immunotherapy. The association of vaccine treatment with lower rates of relapse warrants further clinical confirmation in this indication."

IO biomarker • Late-breaking abstract • Tumor-specific neoantigens • Head and Neck Cancer • Oncology • Oral Cancer • Solid Tumor • Squamous Cell Carcinoma • Squamous Cell Carcinoma of Head and Neck • CD8 • IFNG

Transgene and NEC Bio Sign License Agreement to Prepare the Next Steps of the Development of TG4050 in Head and Neck Cancer (GlobeNewswire) - "Under the terms of the license agreement, Transgene secures access to NEC’s AI-based neoantigen prediction platform for further development of TG4050 in the adjuvant treatment of resected HPV-negative head and neck cancer while conferring rights to enable Transgene’s further clinical development and to support commercialization and potential partnering of the program."

Licensing / partnership • Head and Neck Cancer

Personalized and HPV cancer vaccines in head and neck squamous cell carcinoma: from concept to clinical implementation. (PubMed, Transl Oncol) - "Compounds such as TG4050, mRNA-4157, and PGV001 have shown early, hypothesis-generating signals of activity (from small early-phase studies), particularly in the adjuvant setting. HPV-targeted vaccines, including PDS0101, BNT113, and CUE-101, are being explored in HPV-positive HNSCC, mainly in combination with ICIs...The greatest clinical potential of therapeutic vaccination may lie in earlier disease stages, where the tumor burden is lower and immune modulation more feasible. Further clinical and translational research is needed to define the optimal integration of vaccine-based therapies into multimodal treatment paradigms for HNSCC."

IO biomarker • Journal • Review • Head and Neck Cancer • Immune Modulation • Immunology • Oncology • Solid Tumor • Squamous Cell Carcinoma • Squamous Cell Carcinoma of Head and Neck

A Trial Evaluating TG4050 in Ovarian Carcinoma. (clinicaltrials.gov) - P1 | N=13 | Not yet recruiting | Sponsor: Transgene

New P1 trial • Fallopian Tube Cancer • Gynecologic Cancers • Oncology • Ovarian Cancer • Peritoneal Cancer • Solid Tumor

A Trial Evaluating TG4050 in Ovarian Carcinoma. (clinicaltrials.gov) - P1 | N=13 | Recruiting | Sponsor: Transgene | Not yet recruiting ➔ Recruiting

Enrollment open • Fallopian Tube Cancer • Gynecologic Cancers • Oncology • Ovarian Cancer • Peritoneal Cancer • Solid Tumor

A Trial Evaluating TG4050 in Ovarian Carcinoma. (clinicaltrials.gov) - P1 | N=64 | Completed | Sponsor: Transgene | Active, not recruiting ➔ Completed | N=23 ➔ 64 | Trial completion date: Dec 2025 ➔ Oct 2024

Enrollment change • Trial completion • Trial completion date • Fallopian Tube Cancer • Oncology • Ovarian Cancer • Peritoneal Cancer • Solid Tumor

A Trial Evaluating TG4050 in Ovarian Carcinoma. (clinicaltrials.gov) - P1 | N=23 | Active, not recruiting | Sponsor: Transgene | Recruiting ➔ Active, not recruiting | Trial completion date: Sep 2024 ➔ Dec 2025 | Trial primary completion date: Sep 2023 ➔ Dec 2024

Enrollment closed • Trial completion date • Trial primary completion date • Fallopian Tube Cancer • Oncology • Ovarian Cancer • Peritoneal Cancer • Solid Tumor

A Trial Evaluating TG4050 in Ovarian Carcinoma. (clinicaltrials.gov) - P1 | N=23 | Recruiting | Sponsor: Transgene | Trial completion date: Jun 2023 ➔ Sep 2024 | Trial primary completion date: Jun 2022 ➔ Sep 2023

Trial completion date • Trial primary completion date • Fallopian Tube Cancer • Oncology • Ovarian Cancer • Peritoneal Cancer • Solid Tumor

A Trial Evaluating TG4050 in Ovarian Carcinoma. (clinicaltrials.gov) - P1 | N=23 | Recruiting | Sponsor: Transgene | N=13 ➔ 23 | Trial completion date: Dec 2022 ➔ Jun 2023 | Trial primary completion date: Dec 2021 ➔ Jun 2022

Enrollment change • Trial completion date • Trial primary completion date • Fallopian Tube Cancer • Oncology • Ovarian Cancer • Peritoneal Cancer • Solid Tumor

TG4050.02: A Clinical Trial Evaluating TG4050 in Head and Neck Cancer (clinicaltrials.gov) - P1/2 | N=80 | Active, not recruiting | Sponsor: Transgene | Trial completion date: Dec 2028 ➔ Dec 2031

Trial completion date • Head and Neck Cancer • Oncology • Solid Tumor • Squamous Cell Carcinoma • Squamous Cell Carcinoma of Head and Neck

Profiling of the neoantigen-specific T cell response after adjuvant TG4050 individualized therapeutic vaccination in a randomized phase I trial for locally advanced resected HPV-negative HNSCC (SITC 2025) - P1/2 | "All participants provided written, informed consent before taking part. France: Comité de Protection des Personnes Ile de France 5 ID: 35725 (EUCT 2023-508561-33) Initial approval on 01 Oct 2019 UK: South Central - Oxford A Research Ethics Committee ID: 19/SC/0500 (EudraCT 2018-003267-58) Initial approval on 26 Mar 2020 US: Mayo Clinic Institutional Review Boards ID: 20-007421 Initial approval on 19 Feb 2021"

Clinical • Metastases • P1 data • Tumor-specific neoantigens • Head and Neck Cancer • Oncology • Squamous Cell Carcinoma of Head and Neck • CD8 • CX3CR1 • GZMB • IFNG • PRF1

Transgene to Present New Immunological Data from Phase I Trial of Individualized Therapeutic Cancer Vaccine, TG4050, at SITC 2025 (GlobeNewswire)

P1 data • Squamous Cell Carcinoma of Head and Neck

Transgene Completes Initial Patient Screening in Phase II Part of TG4050 Trial in Operable Head and Neck Cancer (GlobeNewswire) - "Transgene...announced the completion of initial patient screening in the randomized multicenter Phase II part of its Phase I/II clinical trial with TG4050, an individualized neoantigen therapeutic vaccine, as a single agent in the adjuvant treatment of HPV-negative squamous head and neck cancers (NCT04183166)....Transgene expects to complete randomization of all patients in the Phase II part by the end of 2025, following a second screening of patients conducted after surgery and adjuvant (chemo)radiotherapy....First immunogenicity data from the Phase II part of the trial are expected to be available in H2 2026, and preliminary efficacy data are expected in H2 2027....'Consequently, Transgene is starting initial preparations for a new Phase I trial in a second, undisclosed indication in an early treatment setting, with the aim to initiate the trial in Q4 2025'."

New P1 trial • P2 data • Trial status • Squamous Cell Carcinoma of Head and Neck

TG4050.02: A Clinical Trial Evaluating TG4050 in Head and Neck Cancer (clinicaltrials.gov) - P1/2 | N=80 | Active, not recruiting | Sponsor: Transgene | Recruiting ➔ Active, not recruiting

Enrollment closed • Head and Neck Cancer • Oncology • Solid Tumor • Squamous Cell Carcinoma • Squamous Cell Carcinoma of Head and Neck

Randomized phase I trial of adjuvant personalized cancer vaccine TG4050 in resected locally advanced (LA) head and neck squamous cell carcinoma (HNSCC) patients (pts). (ASCO 2025) - P1/2 | "TG4050 is safe and induces immune responses in pts with resected LA HNSCC. No relapse occurred in the vaccine arm as opposed to 19% in the control arm. With the evolution of the landscape, adjuvant anti-PD1 therapy may become standard in resected LA HNSCC."

Clinical • IO biomarker • Metastases • P1 data • Head and Neck Cancer • Oncology • Solid Tumor • Squamous Cell Carcinoma • Squamous Cell Carcinoma of Head and Neck

Transgene to Host Webcast on Positive TG4050 Head and Neck Cancer Data Presented at ASCO 2025 (GlobeNewswire) - "Transgene...announced it will host a live webcast to discuss the latest positive clinical data from its individualized neoantigen cancer vaccine, TG4050, presented at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting....They will discuss the new findings, the unmet medical need and the current treatment landscape and perspectives for early-stage HPV-negative head and neck cancer."

Clinical data • Head and Neck Cancer

Transgene and NEC Present Durable Disease-Free Survival and Sustained T Cell Responses at 24 Months with Individualized Cancer Vaccine TG4050 (The Manila Times) - P1/2 | N=80 | NCT04183166 | Sponsor: Transgene | "All patients in the treatment arm remain disease-free after a minimum of 2-year follow-up in the randomized Phase I trial in resected HPV-negative locally advanced head and neck cancer - Demonstrating clinical proof of principle for TG4050, Single agent TG4050 induced long-lasting neoantigen-specific CD8+ T cell responses...Treatment was well-tolerated with no unexpected safety signals...The Phase II part of the trial, aimed at confirming the encouraging results in a larger patient population and evaluating both immunological and clinical outcomes, is currently underway. All patients are expected to be randomized by Q4 2025."

P1 data • Trial status • Squamous Cell Carcinoma of Head and Neck

Transgene to Present 24-month Disease-Free Survival Data from All Patients in Phase I Part of Trial of Individualized Cancer Vaccine, TG4050, at ASCO 2025 (GlobeNewswire) - "Transgene...today announces that it will present two-year disease-free survival (DFS) data from all patients in the randomized Phase I part of the trial of its individualized neoantigen therapeutic cancer vaccine TG4050. The data will be shared during a rapid oral presentation at the American Society of Clinical Oncology (ASCO) Annual Meeting, taking place in Chicago from May 30 to June 3, 2025....In addition, Transgene will present a poster at ASCO on TG4001, presenting randomized Phase II data in combination with avelumab in a cervical cancer subgroup."

P1 data • P2 data • Cervical Cancer • Head and Neck Cancer

Transgene to Provide Updated Phase I Data in a Rapid Oral Presentation on Individualized Therapeutic Cancer Vaccine TG4050 at ASCO 2025 (GlobeNewswire) - "Transgene...will be delivering a rapid oral presentation of randomized Phase I trial data of individualized neoantigen therapeutic cancer vaccine TG4050 in head and neck cancer at the 2025 American Society of Clinical Oncology (ASCO)....The company will also present a poster on randomized Phase II data of TG4001 in combination with avelumab in the cervical cancer subgroup."

P1 data • P2 data • Cervical Cancer • Squamous Cell Carcinoma of Head and Neck

Randomized phase I trial of adjuvant individualized TG4050 vaccine in patients with locally advanced resected HPV-negative head and neck squamous cell carcinoma (HNSCC) (SITC 2024) - P1/2 | "All participants provided written, informed consent. •France: Name: Comité de Protection des Personnes Ile de France 5 ID: 35725 (EUCT 2023-508561-33) •UK: Name: South Central - Oxford A Research Ethics Committee ID: 19/SC/0500 (EudraCT 2018-003267-58) •US: Name: Mayo Clinic Institutional Review Boards ID: 20-007421."

Clinical • IO biomarker • Metastases • P1 data • Head and Neck Cancer • Oncology • Solid Tumor • Squamous Cell Carcinoma • Squamous Cell Carcinoma of Head and Neck • CD8

Transgene and NEC Present New Data Confirming Clinical Proof of Principle for Neoantigen Cancer Vaccine, TG4050, in Head & Neck Cancer at SITC 2024 (GlobeNewswire) - P1/2 | N=80 | NCT04183166 | Sponsor: Transgene | "Transgene...announced 24.1-month median follow-up data from the ongoing randomized Phase I trial of TG4050...all 16 patients who received TG4050 as adjuvant immunotherapy after completion of adjuvant standard of care remain disease-free and have not relapsed. Out of the 16 patients in the control observation arm, 3 patients have relapsed. There remains a high medical need for these head and neck cancer patients, as approximately 30% of them are expected to experience a relapse within 24 months after standard surgery and adjuvant chemoradiotherapy...All treatment-related adverse events continue to be mild to moderate...Building on these promising data, the randomized Phase I trial has been expanded to a randomized Phase I/II trial in the adjuvant setting of head and neck cancer (NCT04183166), which is currently enrolling patients in the Phase II part."

Enrollment status • P1 data • Head and Neck Cancer • Oncology • Solid Tumor • Squamous Cell Carcinoma • Squamous Cell Carcinoma of Head and Neck

Transgene to Present a Poster on Updated Data for TG4050 at SITC 2024 (GlobeNewswire) - "Transgene...will present a poster highlighting median 24-month follow up from the ongoing randomized Phase I trial of its individualized therapeutic cancer vaccine, TG4050 at the 39th Society for ImmunoTherapy of Cancer (SITC) annual meeting. This presentation will emphasize efficacy and disease-free survival outcomes in patients with head and neck cancers. SITC annual meeting will take place in Houston, Texas, USA, from November 6 to 10, 2024....Transgene and NEC are continuing the joint development of TG4050 in this indication with a Phase II extension of the trial, which is currently enrolling patients."

Enrollment status • P1 data • Head and Neck Cancer • Oncology • Solid Tumor • Squamous Cell Carcinoma • Squamous Cell Carcinoma of Head and Neck

Transgene to Report Major Clinical Data before the end of 2024 – Confirmed Financial Visibility until Q4 2025 (GlobeNewswire) - "Neoantigen therapeutic cancer vaccine: TG4050...The Phase II part started enrolling patients in Q2 2024 within the framework of an extended collaboration between Transgene and NEC. Patient enrollment is progressing at a good pace...Additional data on the 24-month median follow-up of Phase I patients will be reported in Q4 2024...In H1 2024, Transgene has completed the enrollment of 90 patients in the ongoing randomized Phase II trial evaluating TG4001 in HPV-positive anogenital cancers (NCT03260023) in combination with an immune checkpoint inhibitor. Transgene confirms that topline readouts are expected in Q4 2024....The Phase I Delivir trial (NCT05788926) is evaluating TG6050 in patients with advanced non-small cell lung cancer who have failed standard therapeutic options. Initial data from the trial is expected in Q4 2024."

Clinical data • Enrollment status • Anal Carcinoma • Genito-urinary Cancer • Head and Neck Cancer • Lung Cancer • Non Small Cell Lung Cancer • Oncology • Solid Tumor • Squamous Cell Carcinoma of Head and Neck