On June 1, 2025 Transgene (Euronext Paris: TNG), a biotech company that designs and develops virus-based immunotherapies for the treatment of cancer, and NEC Corporation (NEC; TSE: 6701), a leader in IT, network and AI technologies, reported to have presented new positive data on TG4050 in a rapid oral presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (Press release, Transgene, JUN 1, 2025, View Source [SID1234653559]).

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These positive data confirm that individualized neoantigen therapeutic vaccine TG4050 is safe and feasible in the adjuvant setting of resectable HPV-negative locally advanced head and neck squamous cell carcinoma (HNSCC). TG4050 induces, as monotherapy, long-lasting immune responses to vaccine neoantigens sustained for up to 2 years, and these results met all trial endpoints (NCT04183166) including safety, feasibility, immune activation and disease-free survival (defined as survival without recurrence or death for any cause).

TG4050 is based on Transgene’s myvac platform and powered by NEC’s cutting-edge AI capabilities designed to optimize antigen selection.

Positive data from Phase I, confirming proof of principle
of Transgene’s viral vector based individualized cancer vaccine TG4050
in HPV-negative locally advanced head and neck cancer

100% disease free survival at a minimum of 2-year follow-up of treated patients (median follow-up: 30 months) in the Phase I part of the trial: all patients in the TG4050 treatment arm remain disease free while 3 patients in the observational arm have relapsed.
Persistence of neoantigen-specific CD8+ T cell responses over 2 years after the start of TG4050 has been observed.
Dr. Alessandro Riva, CEO of Transgene, commented: "The sustained clinical and immunogenicity outcomes observed over two years of TG4050 monotherapy, along with the positive safety profile, mark an important milestone for Transgene. These results reinforce both the clinical promise of TG4050 and our commitment to accelerate the development of this individualized immunotherapy in adjuvant setting for patients with HPV-negative, locally advanced head and neck cancer."

Motoo Nishihara, Corporate EVP, and CTO, at NEC, commented: "This positive readout, combined with the durability of the efficacy data at two years, underscore the clinical potential of individualized cancer vaccine programs. It is a strong validation of our innovative AI platform and our dedication to advancing solutions that deliver meaningful, long-term value to patients and healthcare systems alike."

Ongoing Phase II part of Phase I/II clinical trial of individualized neoantigen therapeutic cancer vaccine TG4050

TG4050 is being evaluated in a randomized multicenter Phase I/II trial as a single agent in the adjuvant treatment of HPV-negative head and neck cancers (NCT04183166). Based on the promising data obtained in the Phase I part of the trial, Transgene and NEC extended the joint development of TG4050 in this indication with a Phase II extension of the trial.

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. Altogether, the Phase I/II study will comprise approximately 80 patients.

Dr Christian Ottensmeier, MD, PhD, FRCP (University of Liverpool, La Jolla Institute for Immunology), will discuss the data presented at ASCO (Free ASCO Whitepaper) 2025, the unmet medical need and current treatment landscape for patients suffering from head and neck cancers in a live virtual event taking place on June 6, 2025 (9:00 p.m. ET; 3:00 p.m. CET).

On June 1, 2025 Bicara Therapeutics Inc. (Nasdaq: BCAX), a clinical-stage biopharmaceutical company committed to bringing transformative bifunctional therapies to patients with solid tumors, reported updated data from the company’s Phase 1/1b clinical trial of ficerafusp alfa in combination with pembrolizumab in patients with first line (1L) recurrent/metastatic (R/M) head and neck squamous cell carcinoma (HNSCC) at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (Press release, Bicara Therapeutics, JUN 1, 2025, View Source [SID1234653544]). Ficerafusp alfa is a first-in-class bifunctional antibody designed to enhance tumor penetration by breaking barriers in the tumor microenvironment that have challenged the treatment of multiple solid tumor cancers. Specifically, ficerafusp alfa combines two clinically validated targets: an epidermal growth factor receptor (EGFR) directed monoclonal antibody with a domain that binds to human transforming growth factor beta (TGF-β).

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"Ficerafusp alfa was specifically designed to impact the tumor stroma and drive tumor penetration with the goal of leading to deeper and more durable responses. We’ve now begun to see this translate clinically, with responses lasting nearly two years and contributing to prolonged overall survival in HPV-negative patients, a population that typically faces poor outcomes due to resistance to available therapies," said David Raben, MD, Chief Medical Officer of Bicara Therapeutics. "The strength of the totality of this updated dataset, including 80% of responders achieving a deep response of at least 80% tumor shrinkage, median duration of response of 21.7 months, median progression free survival of 9.9 months, overall survival of 21.3 months, and a 2-year overall survival rate of 46%, provides compelling support for the continued advancement of FORTIFI-HN01, our pivotal Phase 2/3 trial in the first-line recurrent/metastatic setting."

Key highlights from the presentation include:

In the Phase 1/1b trial, ficerafusp alfa in combination with pembrolizumab resulted in deep and durable anti-tumor activity with improved overall survival (OS) compared to historical benchmarks in patients with 1L R/M human papillomavirus (HPV)-negative HNSCC with a PD-L1 combined positive score (CPS) of ≥1 and with at least 24 months of follow-up.
In the efficacy evaluable human papillomavirus (HPV)-negative population (n=28):
Median duration of response (DOR) of 21.7 months amongst responders (n=15).
Median OS of 21.3 months; 2-year OS rate of 46%.
54% (15/28) confirmed objective response rate (ORR); 64% (18/28) ORR, including an additional three unconfirmed responses.
21% (6/28) complete response rate.
80% (12/15) of responders achieved a deep response (≥80% tumor shrinkage).
Disease control rate of 89% (25/28 patients).
Median progression-free survival of 9.9 months.
Manageable safety profile consistent with previously reported adverse events.
"These latest Phase 1/1b data are impressive, particularly the duration of response, which represents a significant advance over historical controls in patients with HPV-negative recurrent/metastatic head and neck squamous cell carcinoma, including anti-PD-1 combinations with chemotherapy or EGFR inhibitors," said Christine H. Chung, MD, Chair of the Department of Head and Neck-Endocrine Oncology and Deputy Physician-in-Chief at the Moffitt Cancer Center. "This reflects the enhanced ability of ficerafusp alfa to remodel the tumor microenvironment allowing the tumor penetration of immune cells required for deep and durable responses in these patients. In addition, the prolonged overall survival, highlighted by a median OS of 21.3 months, reinforces the potential of ficerafusp alfa to address a critical unmet need by delivering durable anti-tumor responses and meaningful improvements in patients’ quality of life."

Conference Call and Webcast Information
Bicara Therapeutics will host a conference call and webcast today, June 1, 2025 at 3:00 p.m. CT / 4:00 p.m. ET. Individuals may register for the conference call by clicking the link here. Once registered, participants will receive dial-in details and a unique PIN which will allow them to access the call. An audio webcast will be accessible through the Investor Relations section of Bicara’s website under Events and Presentations. Following the live webcast, an archived replay will also be available.

About Head and Neck Squamous Cell Carcinoma
Head and neck squamous cell carcinomas (HNSCCs) develop from the mucosal epithelium in the oral cavity, pharynx and larynx and are the most common malignancies that arise in the head and neck. HNSCC is one of the most common cancers in the United States and globally with a rising incidence anticipated to reach one million new global cases annually by 2030. Ten percent of HNSCC patients are diagnosed with metastatic disease and up to 30% develop a recurrence or metastases over time after receiving initial treatment for advanced HNSCC.

Most cases of HNSCC are thought to result from accumulated mutations caused by carcinogenic exposures such as tobacco smoke or HPV infection. Approximately 80% of patients with R/M HNSCC are HPV-negative. These HPV-negative tumors often exhibit a recurrence pattern that is primarily local and are associated with severe morbidities, including fatal tumor bleeding, intense pain, difficulty swallowing, significant weight loss, and cachexia. This highlights a critical unmet need for therapies that have the potential to deliver durable anti-tumor responses, ultimately leading to meaningful improvements in patients’ quality of life.

About Ficerafusp Alfa
Ficerafusp alfa is a first-in-class bifunctional antibody designed to drive tumor penetration by breaking barriers in the tumor microenvironment that have challenged the treatment of multiple solid tumor cancers. Specifically, ficerafusp alfa combines two clinically validated targets: an epidermal growth factor receptor (EGFR) directed monoclonal antibody with a domain that binds to human transforming growth factor beta (TGF-β). Through this targeted mechanism, ficerafusp alfa reverses the fibrotic and immune-excluded tumor microenvironment driven by TGF-β signaling to enable tumor penetration that drives deep and durable responses.

Ficerafusp alfa is currently being evaluated in FORTIFI-HN01, a pivotal Phase 2/3 clinical trial in patients with first line (1L) recurrent/metastatic (R/M) head and neck squamous cell carcinoma (HNSCC).

On June 2, 2025 Imugene Limited (ASX: IMU), a clinical-stage immuno-oncology company, reported the first site activation within Australia, as part of the investigator sponsored Phase II Neo-POLEM clinical trial (Press release, Imugene, JUN 2, 2025, https://mcusercontent.com/e38c43331936a9627acb6427c/files/0af44840-0a27-5641-dade-e0642353307d/1st_Australian_site_activated_PD1_Vaxx_Neo_POLEM_Ph_II_trial.pdf [SID1234653561]).

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Neo-POLEM is a Phase II study investigating the potential of PD1-Vaxx, a neoadjuvant PD-1 vaccine, to improve treatment outcomes for patients with mismatch repair-deficient / microsatellite instability high (dMMR/MSI-high) colorectal cancer. Approximately 15% of patients with colorectal cancer have the dMMR/MSI-high subtype.

The trial is an IST being conducted by Cancer Research UK Southampton Clinical Trials Unit in collaboration with Royal Surrey Hospital NHS Foundation Trust and the Australasian Gastro-Intestinal Trial Group (AGITG).

The primary objective of the study is to determine major pathological response rates, a measure of tumour size reduction post-treatment with PD1-Vaxx before surgery, with secondary objectives to assess the safety of PD1-Vaxx, evaluate biomarkers, and evaluate the objective response rates and overall survival.

This trial builds upon compelling early evidence that immunotherapy can deliver significant benefits in this patient population. The trial will recruit patients in both Australia and the United Kingdom.

Imugene’s CEO and Managing Director Leslie Chong said: "We’re very pleased to see this Neo-POLEM IST open and enrolling in Australia. PD1-Vaxx has the potential to offer a durable immune response and improve treatment outcomes, and we look forward to further progress in both Australia and the UK."

Colorectal cancer (CRC), also known as bowel cancer, is the third most common cancer, with a worldwide annual incidence of more than 1.2 million cases and a mortality rate of approximately 50%. About 80% of patients with colon cancer have localised and resectable disease at diagnosis. Approximately 15% of CRC is dMMR/MSI-high.

On June 1, 2025 J INTS BIO, a company specializing in the development of therapeutics for cancer and rare diseases, reported interim results from the global Phase 1/2 clinical trial of JIN-A02, its fourth-generation EGFR-TKI drug candidate, at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2025 (ASCO 2025), the world’s largest oncology conference, held in Chicago, USA (Press release, J INTS BIO, JUN 1, 2025, View Source [SID1234653563]).

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JIN-A02 is an oral, fourth-generation EGFR-TKI designed to overcome resistance mutations (such as C797S) that develop after treatment failure with third-generation EGFR-TKIs, which are currently the first-line therapy for EGFR-mutant Non-small cell lung cancer (NSCLC). It is currently undergoing clinical trials in South Korea, the United States, Thailand, and other countries. Key efficacy and safety results from Part A (dose escalation) of the multi-center clinical trial (NCT05394831) were presented.

‘Confirmed’ clinical response observed in patients with disease progression after 3rd Generation EGFR TKI and chemotherapy

One of the key findings of the ASCO (Free ASCO Whitepaper) presentation was that tumor responses were sustained in specific dose cohorts, with confirmed partial responses (PRs) verified by independent evaluation. Confirmed PRs were observed in 50mg,100mg and 300mg QD dose groups, providing clinical evidence of anti-tumor activity.

In the 50 mg dose group, patients achieved a 77.3% reduction in tumor size, maintaining a PR over six consecutive treatment cycles (from cycle 3 to cycle 13). In the 300 mg dose group, a confirmed PR was observed with a 39.7% tumor size reduction, including a significant reduction in brain metastatic lesions. In the 100 mg dose group, a PR was also reported with a 35.3% reduction in tumor size, and brain metastatic lesions remained stable, further supporting the potential of JIN-A02 in treating brain metastases.

Safety confirmed up to 300 mg, with therapeutic signals in brain metastases

No dose-limiting toxicities (DLTs) or serious adverse events were observed with JIN-A02 at doses up to 300mg, which is six times the dose level when PR was first observed, demonstrating a favorable safety profile even at higher dose levels. The majority of adverse events reported at 300mg were mild (Grade 1-2) and included for the first time, Grade 1 skin rash, diarrhea, and skin desquamation in 3 out of 5 subjects – events commonly associated with EGFR inhibitors and generally considered clinically manageable. Importantly, there were no reports of systemic toxicities such as cardiovascular events or hepatotoxicity, supporting the drug’s excellent safety profile. This safety and tolerability have translated into extended treatment durations in real-world settings, with a patient still on JIN-A02 after one year and seven months.

In addition, the trial demonstrated notable responses in brain metastases first noted at 100mg, suggesting that JIN-A02 achieves therapeutically relevant concentrations in brain tissue.

JIN-A02 gains national spotlight as government grants accelerate development

In addition to the ASCO (Free ASCO Whitepaper) announcement, J INTS BIO continues to achieve noteworthy results in Korea. Recently, it was selected for the ‘2025 Baby Unicorn Fostering Project’ by the Ministry of SMEs and Startups, officially recognizing both our scientific technology and commercialization potential. The ‘Baby Unicorn Fostering Project’ is a government project that focuses on developing promising startups with innovative technologies and growth potential in the global market as ‘potential unicorns’. Companies selected for this project will receive full government support in recognition of their technological prowess and growth potential.

With these accolades, JIN-A02 will receive dual funding for its Phase 2 trial and global expansion over the next two years, which is expected to be a decisive point to significantly accelerate the pace of clinical and commercialization in parallel with private investment. It is also significant as a case of securing confidence in the 3 arena of technology, marketability, and global scalability, as the recognition came from different government departments.

Based on these achievements, we plan to initiate the Phase 2 clinical trial of JIN-A02 in before the end of this year, in discussions with the US FDA.

"The ASCO (Free ASCO Whitepaper) presentation of JIN-A02’s ability to induce anti-cancer responses and its ability to respond to central nervous system metastatic lesions is of great significance," said Dr. Anna Jo, CEO. "The government’s continued support is a recognition of our technical capabilities and potential for growth. We will take advantage of this opportunity to accelerate global clinical expansion, technology transfer, and indication expansion to realize our goal of early commercialization"

On June 1, 2025 Kura Oncology, Inc. (Nasdaq: KURA, "Kura") and Kyowa Kirin Co., Ltd. (TSE: 4151, "Kyowa Kirin") reported the U.S. Food and Drug Administration (FDA) has accepted Kura’s New Drug Application (NDA) seeking full approval for ziftomenib as a treatment for adult patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) with a nucleophosmin 1 (NPM1) mutation (Press release, Kura Oncology, JUN 1, 2025, View Source [SID1234653547]). The application has been granted Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) target action date of November 30, 2025.

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"The FDA’s acceptance of our New Drug Application marks a significant milestone for Kura and Kyowa Kirin and, more importantly, for patients living with this genetic subset of AML, who face an aggressive form of the disease with few treatment options," said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. "This achievement reflects the strength of the clinical data for ziftomenib as well as the incredible commitment of our teams. Along with our partners at Kyowa Kirin, we look forward to continuing to work closely with the FDA throughout the review process and to prepare for the anticipated launch of this treatment, which holds potential to meaningfully impact the lives of patients and their families."

The NDA is based on results from the Phase 2 KOMET-001 registrational trial in R/R NPM1-mutant (NPM1-m) AML (NCT #04067336). The KOMET-001 trial achieved its primary endpoint of complete remission (CR) plus CR with partial hematological recovery (CRh) and the primary endpoint was statistically significant. Ziftomenib was well‑tolerated with limited myelosuppression and 3% ziftomenib-related discontinuations. The safety and tolerability of ziftomenib were consistent with previous reports, and the benefit-risk profile for ziftomenib is highly encouraging.

"Adult R/R NPM1-m AML patients face a significantly poor prognosis, highlighting the urgent need for innovative treatment options that can improve their outcomes," said Takeyoshi Yamashita, Ph.D., Executive Vice President and Chief Medical Officer of Kyowa Kirin. "The acceptance of this NDA is a crucial step in our ongoing efforts to explore and evaluate various therapeutic strategies for AML through our comprehensive clinical trials. Our dedicated teams at Kyowa Kirin and Kura are fully committed to working tirelessly to ensure that, once approved, ziftomenib is made available to AML patients as quickly as possible. We recognize the importance of this endeavor and are excited about the possibility of making a meaningful impact on the lives of those affected by this challenging disease."

The KOMET-001 registration-directed trial is designed to assess evidence of clinical activity, safety and tolerability of ziftomenib, the only investigational therapy to receive Breakthrough Therapy Designation (BTD) from the FDA for treatment of R/R NPM1-mutant AML. In addition to BTD, ziftomenib has received Fast Track and Orphan Drug Designations. The full data analyses from the KOMET-001 trial of ziftomenib in R/R NPM1-m AML patients have been selected for oral presentation on Monday, June 2nd at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, and an encore presentation is planned at the 2025 European Hematology Association (EHA) (Free EHA Whitepaper) Congress.

About NPM1-Mutant AML

AML is the most common acute leukemia in adults and begins when the bone marrow makes abnormal myeloblasts (white blood cells), red blood cells or platelets. Despite the many available treatments for AML, prognosis for patients remains poor and a high unmet need remains. The menin pathway is considered a driver for multiple genetic alterations of the disease, of which NPM1 mutations are among the most common, representing approximately 30% of AML cases. While patients with NPM1-m AML have high response rates to frontline therapy, relapse rates are high and survival outcomes are poor, with only 30% overall survival at 12 months in the R/R setting. Additionally, NPM1 mutations frequently occur with co-mutations in other disease-associated genes, including FLT3, DNMT3A, and IDH1/2, with prognosis heavily influenced by the presence of such co-occurring mutations. Adult patients with NPM1-m AML and select co-mutations and/or R/R disease have a poor prognosis, with median overall survival of only approximately 7.8 months in 2nd line, 5.3 months in 3rd line, and 3.5 months following the 4th line1. There are currently no FDA-approved therapies targeting NPM1-m AML.

About Ziftomenib

Ziftomenib is a potent and selective, oral, investigational menin inhibitor currently in development for the treatment of genetically defined AML patients with high unmet need. In April 2024, ziftomenib received BTD from the FDA for the treatment of adult patients with R/R AML with an NPM1 mutation based on data from Kura’s KOMET-001 clinical trial. Additional information about clinical trials for ziftomenib can be found at www.kuraoncology.com/clinical-trials/#ziftomenib.