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Transgene’s Individualized Neoantigen Therapeutic Cancer Vaccine TG4050 Delivers Positive Randomized Phase I Data Randomization to be Completed in Phase II Part in Q4 2025

On September 16, 2025 Transgene (Euronext Paris: TNG), a biotech company that designs and develops virus-based immunotherapies for the treatment of cancer, reported its financial results for the six-month period ended June 30, 2025, and provides an update on the myvac platform, its lead asset TG4050, and upcoming plans (Press release, Transgene, SEP 16, 2025, View Source [SID1234656003]).

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Dr. Alessandro Riva, MD, Chairman and CEO of Transgene, commented: "We are extremely proud that all patients treated with TG4050 in our Phase I trial remained disease-free after a median follow-up of 30 months. These results, selected for oral presentation at ASCO (Free ASCO Whitepaper) 2025, represent a pivotal milestone for Transgene and underscore the potential of our viral vector-based individualized therapeutic cancer vaccine platform. The durable clinical benefit and robust immune responses we have observed, together with the strong enthusiasm from clinicians, reinforce our vision to deliver transformative therapies to people living with operable head and neck cancer. TG4050 continues to progress through Phase II and we look forward to sharing first data from his trial in 2026. We will also present further immunological data from our Phase I study later this year."

TG4050: Individualized Neoantigen Therapeutic Cancer Vaccine (INTV)

ASCO 2025: TG4050, the first candidate from Transgene’s myvac platform, achieves all Phase I endpoints, with 100% disease-free survival (DFS) after more than 2-years

Transgene presented positive data from the randomized Phase I part of the ongoing multicenter Phase I/II trial (NCT04183166) in an oral presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) (ASCO 2025) Annual Meeting (see press release). TG4050 was administered as a single agent in the adjuvant treatment of HPV-negative operable head and neck squamous cell carcinoma (HNSCC).

– All patients who received TG4050 remained disease-free for at least two years (median follow-up: 30 months), providing strong clinical proof of principle. TG4050 also induced durable and specific T cell responses persisting 24 months after the start of treatment.

-> In addition, the results successfully met all trial endpoints (including safety and feasibility). Additional immunological data from Phase I patients will be presented at an upcoming congress in Q4 2025. These data will provide further insight into the phenotyping of patients’ immune responses against selected epitopes. In addition, Transgene expects to communicate on the 3-year follow-up of Phase I patients in H1 2026.

Based on the positive Phase I data presented at ASCO (Free ASCO Whitepaper) and further immunological data (that will be communicated in Q4 2025), Transgene is currently evaluating the most efficient regulatory pathway to accelerate the development of TG4050 and bring it to patients with operable HNSCC as quickly as possible.

Ongoing Phase II part: critical milestones in 2026 and 2027

Initial patient screening has been completed in the randomized Phase II part of the Phase I/II clinical trial with TG4050 (see press release).

Randomization of all patients in the Phase II part is expected to be completed by the end of 2025.

First immunogenicity data and preliminary efficacy data from the Phase II part of the trial are expected in H2 2026 and Q4 2027, respectively.

Expanding the myvac potential in operable solid tumors

Transgene’s INTV platform myvac could be applied across a range of solid tumors where in many cases a significant unmet medical need remains.

In parallel with the development plan in HNSCC, Transgene is setting up a new Phase I trial in a second indication in an early treatment setting, with the aim to initiate it as soon as all conditions are met.

Other viral vector-based assets

BT-001 (oncolytic virus – intratumoral administration): Updated data on Phase I/II trial to be presented at ESMO (Free ESMO Whitepaper) 2025

Transgene and BioInvent will present a poster on updated data from the ongoing Phase I trial (NCT04725331) evaluating BT-001, an armed oncolytic virus expressing an anti-CTLA4 monoclonal antibody, in combination with MSD’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab)[1], at the European Society for Medical Oncology Annual Meeting (ESMO 2025 — see press release). The abstract will be available on the ESMO (Free ESMO Whitepaper) website on October 13, 2025, at 0:05 am CEST.

In previously reported data (ESMO 2024), BT-001 was well tolerated with limited adverse events and no dose-limiting toxicities. BT-001 in monotherapy was shown to shrink injected lesions in patients with advanced solid tumors.

In combination with pembrolizumab, BT-001 showed promising efficacy data with partial responses in patients with relapsed and refractory advanced melanoma and leiomyosarcoma.

Transgene and BioInvent are pursuing clinical development opportunities with clinicians for BT-001 administered intratumorally.

TG4001 (HPV16 therapeutic vaccine)

Transgene presented a poster (available here) on randomized Phase II data of TG4001 in combination with avelumab in a cervical cancer subgroup at the 2025 ASCO (Free ASCO Whitepaper) conference.

Transgene is currently evaluating potential partnership opportunities to determine the best path forward for the program.

TG6050 (oncolytic virus — intravenous administration)

The Phase I dose-escalation Delivir trial (NCT05788926), evaluating TG6050 administered by intravenous infusion, has enrolled 22 patients with advanced non-small cell lung cancer who have failed standard therapeutic options.

TG6050 has demonstrated good tolerability as monotherapy, with no new safety signals identified. However, the analysis of preliminary efficacy and translational data did not demonstrate a clear efficacy signal in the context of intravenous administration in this indication. Patient recruitment of the Phase I trial is completed, and the Company is evaluating the best way forward for this candidate.

Pasithea Therapeutics Announces Activation of Clinical Trial Sites in South Korea for Phase 1/1b Trial of PAS-004 in Adult NF1 Patients

On September 16, 2025 Pasithea Therapeutics Corp. (NASDAQ: KTTA) ("Pasithea" or the "Company"), a clinical-stage biotechnology company developing PAS-004, a next-generation macrocyclic MEK inhibitor reported activation of two South Korean clinical trial sites participating in its Phase 1/1b open label study to assess the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of PAS-004, in adult participants with neurofibromatosis type 1 (NF1) with symptomatic and inoperable, incompletely resected, or recurrent plexiform neurofibromas (Press release, Pasithea Therapeutics, SEP 16, 2025, View Source [SID1234656001]).

The South Korea clinical trial sites, ASAN Medical Centre and Severance Hospital Yonsei University Health System, are now actively recruiting NF1 trial participants.

Professor Lee Beom-Hee of the Department of Pediatrics at Asan Medical Center said, "I am very pleased to partner with the Pasithea team to initiate testing of PAS-004 in adult patients with plexiform neurofibromas associated with NF1 at Asan Medical Center. Our institution has the largest NF1 caseload in South Korea and a long history of research leadership in this field. Our team was among the first to report the therapeutic benefits of MEK inhibition on neurocognitive decline, café-au-lait spots, and growth retardation caused by neurofibromatosis. We are eager to evaluate PAS-004, a next-generation MEK inhibitor that to date has demonstrated a distinct pharmacokinetic profile and a more convenient dosing regimen, which we believe may provide important benefits for our NF1 patients."

Dr. Tiago Reis Marques, chief executive officer of Pasithea commented, "With access to world-class facilities and an estimated 10,000 NF1 patients in South Korea, we believe our clinical sites in the country will play a pivotal role in the success of this trial. We are excited to include South Korean patients in our NF1 study and look forward to advancing meaningful treatment options for this community."

Asan Medical Center is a reference hospital and the teaching hospital of the University of Ulsan College of Medicine, located in Seoul, South Korea. With 2,432 beds for patients and a total floor area of approximately 280,000 square meters, it is the largest hospital in South Korea.

Severance Hospital is a teaching hospital located in Sinchon-dong, Seodaemun District, South Korea. It is one of the oldest and biggest university hospitals in South Korea. It has 2,437 beds and treats approximately 2,500,000 outpatients and 840,000 inpatients annually.

About the Phase 1/1b Clinical Trial in Adult NF1 Patients

The primary objective of the Phase 1/1b study (NCT06961565) is to evaluate the safety and tolerability of PAS-004 when administered for one 28-day treatment cycle in adult NF1 participants with at least one and up to two additional target plexiform neurofibromas (PNs) that are symptomatic and inoperable, incompletely resected, or recurrent. Secondary objectives are (i) to identify the recommended Part B dose ("RPBD") or Maximum Tolerated Dose (MTD) of PAS-004, (ii) to characterize the PK and PD profile of PAS-004, (iii) to evaluate the preliminary efficacy of PAS-004 on target PN volume, (iv) to evaluate the preliminary efficacy of PAS-004 on the size, appearance, and associated symptoms of cutaneous neurofibromas (CNs), and (v) to evaluate the impact of PAS-004 on quality of life ("QOL") and any physical symptoms attributed to the target PN. Experimental objectives are (i) to evaluate the impact of PAS-004 on QOL and any physical symptoms attributed to CNs, (ii) to evaluate the impact of PAS-004 on pain and function attributed to PNs, and (iii) to investigate PAS-004 effects on CN tumor cellular and molecular biology.

The trial will be conducted in two parts. In Part A (dose escalation phase), following a screening period of up to 28 days, up to 24 eligible participants will be enrolled sequentially to receive one of four planned dose levels of PAS-004 tablets (4mg, 8mg, 12mg, 18mg) in a modified 3+3 design. Part A will identify the recommended RPBD. During Part B (expansion phase), approximately 24 eligible participants will be enrolled in parallel to receive one of two planned dose levels of PAS-004 tablets. Participants will be dosed at the RPBD level and at a dose level below the RPBD for up to six continuous 28-day treatment cycles. Part B will identify the recommended phase 2 dose (RP2D).

The study is planned to be conducted at five clinical trial sites in Australia, South Korea and the U.S.

Oncolytics Biotech® Provides Update on GOBLET Study Progress and U.S. Site Expansion

On September 15, 2025 Oncolytics Biotech Inc. (Nasdaq: ONCY) ("Oncolytics" or the "Company"), a clinical-stage immunotherapy company developing pelareorep, reported updates on enrollment progress, U.S. site expansion, and expected data readouts from the ongoing GOBLET trial evaluating pelareorep in gastrointestinal cancers (Press release, Oncolytics Biotech, SEP 16, 2025, View Source [SID1234656000]). The study is supported in part by a grant from the Pancreatic Cancer Action Network (PanCAN).

"The GOBLET trial is designed to rigorously evaluate pelareorep’s potential across gastrointestinal cancers," said Dr. Dirk Arnold, Director of Asklepios Tumorzentrum Hamburg and Primary Investigator of the GOBLET study. "The strong early efficacy signals give us confidence that pelareorep may become a foundational immunotherapy for these difficult-to-treat tumors, and expanding enrollment into the U.S. will broaden the scope and impact of these data."

Cohort 4 (Second-Line or later Squamous Cell Carcinoma of the Anal Canal, "SCAC")
Pelareorep is being studied in combination with atezolizumab in the rare but deadly relapsed, unresectable SCAC indication. In January, the Company released efficacy data showing a 33% overall response rate (ORR) in 12 patients. This nearly triples the ORR achieved by retifanlimab in second-line or later SCAC patients. Enrollment is currently at 20 evaluable patients and is expected to be completed by the end of 2025.

The Company expects to provide an efficacy update regarding ORR in Cohort 4 in the fourth quarter of 2025.

Cohort 5 (First-Line Metastatic Pancreatic Ductal Adenocarcinoma, "mPDAC")
In a randomized two-arm cohort, pelareorep is being evaluated in combination with modified FOLFIRINOX with or without atezolizumab to gain greater clarity regarding the contribution of the checkpoint inhibitor to the efficacy achieved in GOBLET Cohort 1. In that cohort, pelareorep combined with gemcitabine/nab-paclitaxel and atezolizumab achieved a 62% ORR in 13 evaluable patients. Enrollment into Cohort 5 is approximately 40% complete and is expected to be fully enrolled by the end of 2026.

The Company anticipates providing a Cohort 5 interim efficacy update, including overall survival, in the first quarter of 2026.

GOBLET Expansion to U.S. Sites
The Company recently submitted a protocol amendment to allow the GOBLET study to open U.S. clinical sites within the next few months. Upon approval, Northwestern University and other academic institutions are expected to serve as U.S. sites for GOBLET.

"We are pleased to bring this important study to U.S. patients with pancreatic cancer, a population urgently in need of novel immunotherapy strategies," said Dr. Devalingam Mahalingham, who is expected to lead the Cohort 5 study at Northwestern University. "The combination of pelareorep and a checkpoint inhibitor with chemotherapy has shown early signs of durability, and I am excited to be part of advancing this program toward registration."

"We expect to build on this clinical momentum to lay the foundation for our regulatory strategy," said Jared Kelly, Chief Executive Officer of Oncolytics. "It’s imperative that we leverage our clinical data to obtain regulatory clarity and position pelareorep as a platform immunotherapy in these gastrointestinal tumors where patients desperately need treatment options."

NUCLIDIUM to Present Clinical Data from Phase I/II Study for Copper-based PET-Diagnostic in Neuroendocrine Tumors at EANM 2025

On September 16, 2025 NUCLIDIUM AG, a clinical-stage radiopharmaceutical company developing a proprietary copper-based theranostic platform, reported that the company’s abstract featuring first clinical data from its ongoing Phase I/II trial has been selected for an oral presentation at the European Association of Nuclear Medicine (EANM) congress, taking place from October 4 – 8, 2025 in Barcelona, Spain (Press release, NUCLIDIUM, SEP 16, 2025, View Source [SID1234655999]). The Phase I/II trial is evaluating the company’s PET tracer, 61Cu-TraceNetTM [61Cu]Cu-NODAGA-LM3 for the detection of SSTR-positive gastroenteropancreatic & bronchopulmonary neuroendocrine tumors (GEP & BP-NETS). Dr. Guillaume Nicolas, Deputy Head of Nuclear Medicine at the Department of Theragnostics, University Hospital Basel, Switzerland, and principal investigator of the study, will present the data.

Oral Presentation Details
Session Title: Clinical Oncology Track – Top Rated Oral Presentation – Oncology & Theranostics Committee: NET Imaging
Abstract Title: "A phase I/II study of [61Cu]Cu -NODAGA-LM3 for the detection of neuroendocrine tumours: Preliminary results of the COPPER PET in NET Trial"
Date and Time: Monday, October 6, 2025, at 9:45 AM CEST
Location: Barcelona International Convention Centre Room 117

Kura Oncology Highlights Preclinical Data Demonstrating Potential of Farnesyl Transferase Inhibitors to Overcome Drug Resistance in Combination with Key Targeted Therapies Across Multiple Tumor Types

On September 16, 2025 Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer, reported the first of two analyst/investor events focused on its farnesyl transferase inhibitor (FTI) program (Press release, Kura Oncology, SEP 16, 2025, View Source [SID1234655998]). The session features compelling preclinical data illustrating the potential of FTIs to address a common resistance pathway, thereby enhancing the anti-tumor activity of PI3Kα inhibitors, KRAS inhibitors and antiangiogenic tyrosine kinase inhibitors (TKIs) across a range of diverse tumor types. These findings, drawn from Kura’s pioneering research on FTI mechanisms, offer important context to interpret the preliminary clinical data to be presented next month at the ESMO (Free ESMO Whitepaper) Congress 2025.

"Innovation in cancer therapy demands not just new drugs, but smarter combinations to confront resistance head-on," said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. "Today’s preclinical presentations underscore the transformative potential of KO-2806 – also known as darlifarnib – as a versatile combination therapy to major classes of precision medicines, paving the way for our upcoming presentations of its first, preliminary clinical data at ESMO (Free ESMO Whitepaper) 2025 next month."

Topics discussed during today’s event include:

Overcoming Resistance: Innate and adaptive resistance mechanisms can significantly limit the long-term efficacy of monotherapy with PI3Kα inhibitors, KRAS inhibitors and antiangiogenic tyrosine kinase inhibitors (TKIs), underscoring the need for combination therapies.

Pioneering FTI Innovation: Kura has pioneered the discovery and development of farnesyl transferase inhibition and the targeting of mTOR – a clinically validated target – to overcome drug resistance and amplify the impact of targeted oncology therapeutics when paired with an FTI.

KO-2806, A Next-Generation FTI: Also known as darlifarnib, KO-2806 is Kura’s optimized, next-generation FTI, which was designed to provide superior potency, pharmacokinetics and physicochemical properties compared to first-generation candidates. Preclinical studies support the use of KO-2806 in combination with other agents to target pathways of resistance across a range of large indications.

Robust Preclinical Activity: In a broad panel of genetically-defined, in vivo tumor models, FTIs potently suppress mTOR signaling, driving enhanced anti-tumor activity when combined with antiangiogenic TKIs, PI3Kα inhibitors and KRAS inhibitors.

Class-Wide Applicability: Preclinical results with multiple agents from each targeted therapy class indicate broad mechanistic overlap, suggesting KO-2806’s potential extends across these classes.

Re-Sensitization in Relapsed Models: In preclinical non-small cell lung cancer (NSCLC) and colorectal cancer (CRC) models, KO-2806 re-sensitizes tumors to both mutant-selective or pan-KRAS inhibitors, restoring responsiveness in these models of relapsed settings.

Upcoming Clinical Milestones: In advance of its three presentations of FTI clinical data at the ESMO (Free ESMO Whitepaper) 2025 Congress, Kura reviewed the rationale, design and objectives of its ongoing FTI Phase 1 trials.

Expansive Patient Opportunity: KO-2806 combinations with standard-of-care agents could reach a substantial patient population. Combining with cabozantinib or other TKIs positions KO-2806 to address critical gaps in the treatment of renal cell carcinoma (RCC) and neuroendocrine tumors (NET). Extending to KRAS- and PI3Kα-mutant cancers in NSCLC, CRC, breast cancer and beyond, KO-2806 has the potential to impact more than 200,000 incident patients in the U.S. annually.
Today’s event will be held at 1:30 p.m. PT / 4:30 p.m. ET.

Kura plans to host a second event on October 18, 2025, at 10:30 a.m. PT / 1:30 p.m. ET to review clinical data from Kura’s three scheduled presentations at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2025.

A live webcast and archived replay of each event will be available on the Events page in the Investors section of Kura’s website.