On September 18, 2025 PDS Biotechnology Corporation (Nasdaq: PDSB) ("PDS Biotech" or the "Company"), a late-stage immunotherapy company focused on transforming how the immune system targets and kills cancers, reported details of a sub-analysis of the cohort of patients with low PD-L1 expression (CPS 1-19) from the final data for its recently completed VERSATILE-002 Phase 2 clinical trial (Press release, PDS Biotechnology, SEP 18, 2025, View Source [SID1234656073]). The VERSATILE-002 trial evaluated PDS0101 (Versamune HPV) + Keytruda in patients with HPV16-positive first-line recurrent and/or metastatic head and neck squamous cell cancer ("1L R/M HNSCC").

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"This is great news for these patients who may now have the possibility of a well-tolerated treatment without chemotherapy" stated Prof. Kevin Harrington, M.D., Head of the Division of Radiotherapy, The Institute of Cancer Research, London.

"Patients with low levels of PD-L1 expression have typically shown poor response to immune checkpoint inhibitor therapy, leaving HNSCC patients in this sub-type with few viable treatment options and a poor prognosis," said Kirk Shepard, M.D., Chief Medical Officer of PDS Biotech. "This sub-analysis of survival data from the CPS 1-19 patient cohort appears to show that the multifunctional T cell immune response of PDS0101 treatment may overcome the documented limitations of immune checkpoint inhibitor therapy and significantly improve survival in one of the most difficult-to-treat patient populations. This is a very encouraging finding, pointing to the potential of PDS0101 to improve treatment outcomes in a patient group that has historically derived limited benefit from current standards of care."

Approximately 60% of the patients enrolled (n=53) in the VERSATILE-002 trial had low PD-L1 expression, a difficult to treat subset of the overall HNSCC patient population. The sub-analysis of this patient cohort shows:
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Median overall survival (mOS) for patients within the CPS 1-19 cohort (n=32) was 29.5 months.
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Published mOS in this CPS cohort was 10.8 months with Keytruda monotherapy and 12.3 months with Keytruda plus chemotherapy*

The Company announced mOS results for the full study population of 39.3 months from the VERSATILE-002 trial on August 25, 2025, and the full data set for the trial is expected to be published later this year.

*No head-to-head studies have been performed comparing Keytruda (pembrolizumab) monotherapy and Keytruda plus chemotherapy with PDS0101 *

About the VERSATILE-002 Trial
VERSATILE-002 (NCT04260126) is an open-label, multi-center Phase 2 clinical trial evaluating the safety and efficacy of PDS0101, an HPV16-targeted immunotherapy, in combination with pembrolizumab for unresectable, recurrent or metastatic HPV16-positive HNSCC. The trial is designed to assess the combination therapy’s impact on patients who are either naive to or refractory to immune checkpoint inhibitors.

On September 17, 2025 Imugene Limited (ASX: IMU), a clinical-stage immuno-oncology company, reported further encouraging efficacy data from its Phase 1b clinical trial evaluating azer-cel (azercabtagene zapreleucel) in patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL), an aggressive form of blood cancer (Press release, Imugene, SEP 17, 2025, https://mcusercontent.com/e38c43331936a9627acb6427c/files/eb0aad51-27f2-1fa7-4e15-300441221a2f/Overall_Response_Rate_increases_to_81_in_azer_cel_trial.pdf [SID1234656038]).

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In August 2025, Imugene announced that a total of eleven out of fourteen patients had achieved an ORR of 79%, defined as either Complete Response, (the disappearance of signs of cancer in response to treatment) or Partial Response, (defined as cancer reduction by at least 50%). Since then, two new patients have become evaluable for responses with both achieving a Partial Response and another patient transitioning from PR to CR at Day 90 scan evaluation increasing the best ORR to 81% with thirteen out of sixteen patients showing response to treatment. The Complete Response (CR) rate continues to evolve as enrollment progresses and patients transition from partial to complete response, with an average time to best response seen in 1–3 months. The durability of response is also deepening in patients treated with azer-cel in combination with interleukin-2 (IL-2).

Azer-cel is being developed as a potential allogeneic, off-the-shelf, CAR T-cell therapy, addressing key limitations of approved autologous CAR T drugs, including geographical access to treatment centres, manufacturing complexity and time to receive treatment (on-demand).

Imugene is actively enrolling patients to the Phase 1b azer-cel trial at ten US sites with up to six sites in Australia planned, after the first Australian patient was dosed in January 2025 at Royal Prince Alfred Hospital in Sydney, resulting in a Complete Response.

About the Phase 1b azer-cel trial

The azer-cel allogeneic CAR T trial is an ongoing, open-label, multi-centre Phase 1b clinical trial in the U.S. and Australia, for CAR T relapsed patients with DLBCL. The study has recently expanded to include and treat CAR T naïve patients diagnosed with a broad range of Non-Hodgkins lymphomas including primary central nervous system lymphoma (PCNSL), chronic lymphocytic leukemia (CLL)/ small lymphocytic lymphoma (SLL), marginal zone lymphoma (MZL), Waldenstrom macroglobulinemia (WM) and follicular lymphoma (FL). Treatment with azer-cel, lymphodepletion (LD) and IL-2 is showing promising results with evidence of meaningful clinical activity, and durability of response. Additionally, the safety profile is manageable and generally well tolerated.

About diffuse large B cell lymphoma (DLBCL)

DLBCL is an aggressive and fast-growing type of non-Hodgkin’s lymphoma (NHL), a type of blood cancer. DLBCL is the most common type of NHL, with approximately 160,000¹ global cases per year and approximately 30,000 new cases per year in the U.S. Relapsed/refractory DLBCL has a high unmet medical need; ~60% of patients treated with approved autologous CD19 CAR T relapse.

¹Science Direct Volume 60, Issue 5, November 2023

About primary central nervous system lymphoma (PCNSL)

PCNSL is a rare and aggressive form of non-Hodgkin lymphoma (NHL), a type of blood cancer that originates in the brain, spinal cord, leptomeninges, or eyes, usually without evidence of systemic disease. In the U.S., there are approximately 1,500 to 1,800 new cases per year with limited approved treatment options and is a high unmet need. Currently, there are no CAR T-cell products approved for the treatment of PCNSL providing a unique opportunity for azer-cel to treat CART naïve patients.

About other types of B Cell Lymphoma

Other subtypes of non-Hodgkin lymphoma (NHL) include chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL), the most common slow growing leukemia that can become resistant to therapy; marginal zone lymphoma (MZL), a slow-growing B-cell lymphoma that arises in lymphoid tissues associated with mucosal sites like the stomach and lung; Waldenström macroglobulinemia (WM), a rare slow-growing lymphoma characterized by excess IgM production, which can cause multiple complications ; and follicular lymphoma (FL), a common slow-growing NHL that can become more aggressive. While several targeted therapies and monoclonal antibodies are available for these types of B Cell Lymphoma, relapsed or refractory disease remains an ongoing challenge, highlighting the ongoing need for continued innovation and new and better treatments.

About Interleukin 2 (IL-2)

IL-2 is a cytokine (a protein that affects what happens between cells in the immune system) that helps T-cells (which are part of the immune system that help fight cancer) grow and survive. IL-2 has been shown to help T cells live longer and to enhance the cancer killing functions of CAR T cells, making them more effective at targeting and killing cancer cells.

On June 3, 2025 Agenus and Zydus Pharmaceuticals (USA) Inc. entered agreements pursuant to which, (i) under an asset purchase agreement Zydus will acquire assets comprising the Company’s manufacturing operations, (ii) under a stock purchase agreement Zydus will acquire a minority position in the Company and (iii) under a license agreement Zydus will receive certain commercial rights in India and Sri Lanka relating to intellectual property associated with BOT/BOL (Filing, 8-K, Agenus, SEP 17, 2025, View Source [SID1234656166]). The Company and Zydus jointly submitted a filing to the Committee on Foreign Investment in the United States (CFIUS) to commence the CFIUS review process.

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On September 17, 2025, CFIUS requested that the Company and Zydus voluntarily submit a full notice application with CFIUS related to the transactions.

Based on the anticipated CFIUS review time related to the full notice filing the anticipated closing of these transactions has now shifted to the fourth quarter of 2025.

More detailed descriptions of the asset purchase agreement and stock purchase agreement are contained in our Current Report on Form 8-K filed with the SEC on June 4, 2025.

On September 17, 2025 CARsgen Therapeutics Holdings Limited (Stock Code: 2171.HK), a company focused on developing innovative CAR T-cell therapies, reported that the updated long-term follow-up results of Phase I clinical trial of zevorcabtagene autoleucel (zevor-cel, R&D code: CT053, an autologous CAR T-cell product targeting BCMA) have been presented as a poster at the 22nd International Myeloma Society ("IMS") Annual Meeting (Press release, Carsgen Therapeutics, SEP 17, 2025, View Source [SID1234656039]). The poster was titled "Long term Follow-up of Zevor-cel in Patients with Relapsed/Refractory Multiple Myeloma" (Abstract number: PA-029).

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In this study, a total of 14 patients with relapsed or refractory multiple myeloma (R/R MM) received a single infusion of zevor-cel. As of February 22, 2025, the median follow-up duration was 53.3 months (range:14.8, 63.5).

Regarding safety: There were no reports of ≥Grade 3 cytokine release syndrome (CRS), immune effector cell-associated neurotoxicity syndrome (ICANS), delayed neurotoxicities, second primary malignancy or other delayed AEs on the study.

Regarding efficacy: The overall response rate was 100% (95% CI: 76.8, 100.0) with 11 (78.6%) patients achieving complete response (CR) or stringent complete response (sCR). All patients who achieved CR or better were minimal residual disease (MRD) negative at 10−5 threshold. One patient remained in sCR at 59.3 months in the study. The median progression-free survival (mPFS) and the median duration of response (mDoR) were 44.1 months and 43.2 months in CR/sCR patients, respectively. The median overall survival (OS) was not reached. The proportion of patients surviving at 24, 36, 48 and 60 months after infusion were 100%, 92.3%, 84.6% and 76.9%, respectively.

At approximately 5 years of follow-up, zevor-cel demonstrates manageable safety profile while eliciting deep and durable responses in R/R MM patients.

About Zevor-cel

Zevor-cel is a fully human, autologous BCMA CAR T-cell product for the treatment of Multiple Myeloma (MM). Zevor-cel was approved by the NMPA on February 23, 2024 for the treatment of adult patients with R/R MM who have progressed after at least 3 prior lines of therapy (including a proteasome inhibitor and an immunomodulatory agent). Zevor-cel received Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug designations from the U.S. FDA in 2019.

On September 17, 2025 Ono Pharmaceutical Co., Ltd. (Headquarters: Osaka, Japan; President and COO: Toichi Takino; "Ono"), reported that the European Commission (EC) has approved ROMVIMZA (vimseltinib) in the European Union (EU) for the treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT) associated with clinically relevant physical function deterioration and in whom surgical options have been exhausted or would induce unacceptable morbidity or disability (Press release, Deciphera Pharmaceuticals, SEP 17, 2025, View Source [SID1234656040]).

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"The European Commission’s approval of vimseltinib for TGCT is a significant milestone for Deciphera, ONO, and TGCT patients across the European Union who are in need of a non-invasive treatment option. We are excited to leverage our global commercial infrastructure to bring vimseltinib to these patients," said Ryota Udagawa, President and Chief Executive Officer of Deciphera. "We look forward to working with health authorities to ensure all eligible patients who can benefit from vimseltinib have access as quickly as possible."

"This is welcome news for the TGCT community as vimseltinib is now the first approved therapy for TGCT in Europe," said Jean-Yves Blay, M.D., Ph.D., Leon Berard Center. "TGCT can significantly impact the daily lives of patients by causing pain, stiffness and mobility limitations. Vimseltinib is a differentiated treatment that has demonstrated the ability to address these unmet patient needs while remaining well-tolerated."

The EC approval is supported by compelling efficacy and safety results from the pivotal Phase 3 MOTION study of vimseltinib in patients with TGCT not amenable to surgery with no prior anti-CSF1/CSF1R therapy (prior therapy with imatinib or nilotinib allowed), compared to placebo, as well as the Phase 1/2 study of vimseltinib1. The primary endpoint was supported by statistically significant and clinically meaningful improvements in active range of motion, patient-reported physical functioning, and patient-reported pain observed in the vimseltinib arm compared to the placebo arm at week 251. The secondary endpoint was supported by statistically significant and clinically meaningful improvements versus placebo in all six key secondary endpoints assessed at Week 25 including objective response rate (ORR) by tumor volume score (TVS), active range of motion (ROM), physical function, stiffness, quality of life, and pain1. In a descriptive analysis at Week 97, 23% (n=19/83) of the patients randomized to receive vimseltinib had best overall response of complete response (CR) according to RECIST v1.1, as assessed by blind independent radiological review (IRR), with a median time to CR of 11.5 months1. The safety profile of vimseltinib is manageable and consistent with results previously disclosed in the Phase 1/2 clinical trial1. For a full list of side effects and information on dosage and administration and other precautions, please refer to the Summary of Product Characteristics for further information.

About Tenosynovial Giant Cell Tumor (TGCT)

TGCT is caused by a translocation in colony-stimulating factor 1 (CSF1) gene resulting in overexpression of CSF1 and recruitment of colony-stimulating factor 1 receptor (CSF1R)-positive inflammatory cells into the lesion.2 TGCT is also known as giant cell tumor of the tendon sheath (GCT-TS) or pigmented villonodular synovitis (PVNS). TGCT is a rare, locally aggressive neoplasm that can grow and cause damage to surrounding tissues and structures inducing pain, swelling, and limitation of movement of the joint. Surgery is the main treatment option; however, these tumors tend to recur, particularly in diffuse-type TGCT. If untreated or if the tumor continually recurs, damage and degeneration may occur in the affected joint and surrounding tissues, which may cause significant disability. For a subset of patients, surgical resection will potentially cause worsening functional limitation or severe morbidity, systemic treatment options are limited and a new therapeutic option for TGCT is needed.