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).

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"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."

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]).

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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.

On September 16, 2025 Oryzon Genomics, S.A. (ISIN Code: ES0167733015, ORY), a clinical-stage biopharmaceutical company and a European leader in epigenetics, reported that it continues to expand its patent portfolio for iadademstat and vafidemstat, Oryzon’s clinical-stage LSD1 inhibitors for oncology and central nervous system (CNS) disorders, following new Decisions to Grant from the Australian and European patent offices (Press release, Oryzon, SEP 16, 2025, View Source;utm_medium=email&utm_campaign=NdP.27+2025-09-16+IP+additional+decision+grant+ENG778 [SID1234655986]).

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The Australian Patent Office has issued a Decision to Grant for Oryzon’s patent application AU2020249493, titled "Combinations of iadademstat for cancer therapy". The allowed claims cover combinations of iadademstat with PD1 or PD-L1 inhibitors for the treatment of cancer, including small cell lung cancer (SCLC). A Decision to Grant is an official communication from a national patent office indicating that a patent application has met all requirements for issuance as a patent. Once formally granted, this Australian patent will remain in force until at least 2040, not including any potential patent term extensions. A corresponding patent has also been granted in Russia, and applications are pending in Europe, the United States, Japan, China, and other countries.

Iadademstat is currently being evaluated in combination with PD-L1 inhibitors (atezolizumab or durvalumab) in first line SCLC patients with extensive disease in a Phase I/II trial conducted and sponsored by the U.S. National Cancer Institute (NCI) under a Cooperative Research and Development Agreement (CRADA) with Oryzon. More than 30 sites accross the U.S. participate in the trial, including leading institutions such as Memorial Sloan Kettering Cancer Center, Johns Hopkins, City of Hope, Yale University and the University of Chicago, among others.

Oryzon has also received a Decision to Grant from the European Patent Office for its patent application EP24205125.8, titled "Vafidemstat for treating behavior alterations". The allowed claims cover the use of vafidemstat for the treatment of aggressiveness and social withdrawal associated with CNS diseases. Among the allowed claims, there are claims specifically aimed at the treatment of aggressiveness associated with Borderline Personality Disorder (BPD), Autism Spectrum Disorder (ASD), Alzheimer’s disease and other conditions, as well as claims directed to treating social withdrawal associated with diseases such as schizophrenia or ASD. Once formally granted, this European patent will remain in force until at least 2038, not including any potential patent term extensions. Additional patents in this family have already been granted or allowed in Europe, Australia, Canada, Hong Kong, Israel, South Korea, Malaysia, the Philippines, and Russia, with applications pending in other countries.

Vafidemstat is in advanced clinical development for the treatment of aggression in psychiatric disorders, with an upcoming Phase III trial in aggression in BPD (protocol submitted), and a Phase II trial in aggression in ASD patients under preparation. In addition, a Phase II trial is ongoing in schizophrenia, with a focus on negative symptoms. One of the most prominent negative symptoms of schizophrenia is social withdrawal.

"These new patent grants strengthen Oryzon’s global IP position by protecting key therapeutic indications under clinical development for iadademstat and vafidemstat, thereby extending the commercial life for both compounds", said Neus Virgili, Oryzon’s Chief IP Officer.

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.

On September 16, 2025 Lantern Pharma Inc. (NASDAQ: LTRN), a leading artificial intelligence (AI)-driven oncology company leveraging its proprietary RADR platform to accelerate targeted cancer therapies, reported the successful completion of its Phase 1a clinical trial (NCT05933265) for LP-184 (Press release, Lantern Pharma, SEP 16, 2025, View Source [SID1234656004]). The trial met all primary endpoints, demonstrating a favorable safety and pharmacokinetic (PK) profile, and early signs of antitumor activity. Enrollment is complete, with several patients continuing treatment due to ongoing clinical benefit.

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The open-label, multicenter, non-randomized study evaluated LP-184 in 63 patients with advanced relapsed or refractory solid tumors, including GBM. Primary objectives focused on safety, tolerability, PK, and determining a recommended Phase 2 dose (RP2D) when administered on Days 1 and 8 of a 21-day cycle.

Summary of Preliminary Phase 1a Safety and Pharmacokinetic Observations

LP-184 exhibited a robust safety profile, with no dose-limiting toxicities in the majority of cohorts and low incidence of discontinuations, interruptions, or delays due to drug-related adverse events. Adverse events were predominantly Grade 1 or 2, including manageable nausea and vomiting—consistent with alkylating agents—that resolved without significant intervention. The low rate of Grade 3+ events (minimal across the study) underscores LP-184’s tolerability, making it well-suited for potential monotherapy or combinations with agents like PARP inhibitors and immunotherapies, where preclinical synergies have been observed.

PK data confirmed that therapeutic concentrations were achieved at dose levels 8 (0.25 mg/kg) and above, aligning with preclinical models and supporting dose optimization for future trials. These observations will help further de-risk LP-184, enabling efficient advancement in biomarker-enriched populations identified via our RADR AI platform.

Summary of Preliminary Phase 1a Antitumor Observations

Promising antitumor activity emerged, particularly at dose levels 8 (0.25 mg/kg) and above, where therapeutic exposures were attained. Disease control was achieved in 48% (10/21) of evaluable patients after two cycles, including in heavily pre-treated cases. The median number of prior lines of therapy was 3; some patients had up to 8 prior lines of therapy.

Notable highlights from the overall study include:

Clinical benefit observed in 4 of 16 recurrent GBM patients previously exposed to temozolomide, lomustine, and/or radiation.
Marked reductions in target cancer lesions among patients with CHK2, ATM, BRCA1 and STK11/KEAP1 mutations, spanning colon cancer, thymic carcinoma, gastrointestinal stromal tumor (GIST), and NSCLC.
A NSCLC patient with DNA damage response (DDR) mutations, refractory to immunotherapy, achieved nearly two years of clinical benefit and remained on treatment.
Two patients at dose level 10 (0.39 mg/kg) maintain disease control beyond six months and continue on therapy.
These signals in DDR-deficient tumors further support LP-184’s synthetic lethal mechanism and highlight its potential in precision oncology.

"On behalf of our dedicated team, we extend our sincere gratitude to the patients, families, investigators, and clinical staff whose commitment drove the success of our Phase 1a LP-184 trial, establishing a robust safety profile with encouraging signals of activity at therapeutic doses," said Panna Sharma, Chief Executive Officer of Lantern Pharma. "Leveraging our RADR AI platform, we’re now positioning LP-184 for targeted Phase 1b and Phase 2 studies. Our goals are to position LP-184 to address critical unmet needs in TNBC, NSCLC, and other DDR-deficient cancers, which can unlock significant value for patients and investors alike."

Recommended Phase 2 Dose and LP-184 Future Development Plan

The Safety Review Committee supported an RP2D of 0.39 mg/kg in this regimen, with provisions for intra-patient escalation, based on the trial’s evaluation of safety, tolerability, and PK data. Building on these results, Lantern is developing plans to advance multiple Phase 1b/2 trials, prioritizing the following disease indications:

TNBC in combination with Olaparib (a PARP inhibitor), with potential for ctDNA as an early response biomarker to support accelerated approval pathways.
NSCLC with STK11/KEAP1 co-mutations, with or without immunotherapy.
Bladder cancer in a trial to be conducted as an investigator-led study in Denmark with patients harboring DNA damage repair genetic alterations.
These initiatives target high-value indications with substantial market potential and high clinical need.

Data Cutoff Date and Future Publications and Presentations

The data cutoff for the observations described in this release is August 26, 2025. Comprehensive results from the LP-184 Phase 1a trial are being prepared for peer-reviewed publications and presentations at upcoming oncology conferences.