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").

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"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:
•
Median overall survival (mOS) for patients within the CPS 1-19 cohort (n=32) was 29.5 months.
•
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 18, 2025 Replimune Group, Inc. (NASDAQ: REPL), a clinical stage biotechnology company pioneering the development of novel oncolytic immunotherapies, reported that the company completed a Type A meeting with the U.S. Food and Drug Administration (FDA) on September 16th to discuss the complete response letter (CRL) for the Company’s Biologics License Application (BLA) for RP1 in combination with nivolumab for the treatment of advanced melanoma (Press release, Replimune, SEP 18, 2025, View Source [SID1234656074]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The company is evaluating the feedback from the FDA provided during the meeting to determine next steps. At this time, a path forward under the accelerated approval pathway has not been determined.

"The feedback from the melanoma community, including patients and physicians, clearly highlights the unmet need in advanced melanoma and the compelling risk-benefit profile of RP1 observed in the IGNYTE trial," said Sushil Patel, Ph.D., CEO of Replimune. "We remain committed to working with the FDA to determine an expeditious path forward for RP1."

About RP1
RP1 (vusolimogene oderparepvec) is Replimune’s lead product candidate and is based on a proprietary strain of herpes simplex virus engineered and genetically armed with a fusogenic protein (GALV-GP R-) and GM-CSF intended to maximize tumor killing potency, the immunogenicity of tumor cell death, and the activation of a systemic anti-tumor immune response.

On September 18, 2025 Marengo Therapeutics, Inc., a clinical-stage biotechnology company pioneering novel approaches for precision immunotherapy in oncology, inflammation and immunology (I&I), reported the completion of the safety run-in and the determination of recommended Phase 2 dose (RP2D) for its ongoing Phase 1b/2 STARt-002 trial (Press release, Marengo Therapeutics, SEP 18, 2025, View Source [SID1234656077]). The study, evaluating Marengo’s dual T-cell agonist invikafusp alfa in combination with TRODELVY (sacituzumab govitecan-hziy), Gilead’s approved TROP2-directed antibody-drug conjugate (ADC), is now advancing into two expansion cohorts in HR+/HER2− and TNBC metastatic breast cancer, respectively.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Breast cancer remains an area of tremendous unmet need, particularly for patients with advanced HR+/HER2− and triple-negative disease," said Erika Hamilton, M.D. FASCO, Director of Breast Cancer Research at Sarah Cannon Research Institute (SCRI). "The ability to combine a novel T cell agonist like invikafusp with a proven ADC such as sacituzumab govitecan represents an exciting step toward potentially broadening the benefit of immunotherapy-based combination regimens for patients with historically limited treatment options. The enthusiasm we’re seeing among investigators and patients to participate in STARt-002 reflects the promise of this approach."

"As highlighted at recent oral presentations at major scientific conferences, invikafusp has already demonstrated promising single-agent activity in PD-1 resistant tumors including breast cancer," said Kevin Chin, M.D., Chief Medical Officer of Marengo Therapeutics. "Advancing STARt-002 into Phase 2 expansion cohorts reinforces invikafusp’s potential as an immunotherapy backbone, particularly in combination with ADCs for immunologically cold tumors such as breast cancer. We look forward to building on this momentum as we generate additional clinical data in both monotherapy and combination settings."

The STARt-002 study (NCT06827613) is currently enrolling patients across leading North American cancer centers including Massachusetts General Hospital, SCRI, Princess Margaret Cancer Centre, University of Southern California, University of California Los Angeles, University of Texas at San Antonio and Ohio State University, with participation from additional sites in the near term.

On September 18, 2025 Biovance Capital Partners, a biotech venture capital firm based in Lisbon, Portugal, alongside co-investors OrbiMed, a leading healthcare investment firm, with over $17 billion in assets under management and Torrey Pines Investment, a specialty life-science investment company, reported the Series A funding of Mondego Bio (Press release, Mondego Bio, SEP 18, 2025, View Source [SID1234656078]). Headquartered in Portugal’s main biotech park, Biocant, Mondego Bio is developing best-in-class protein tyrosine phosphatase non-receptor type 2 (PTPN2) inhibitors, providing a potential immuno-oncological therapy with an improved safety and tolerability profile.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

As a critical negative regulator of the JAK-STAT pathway, PTPN2 functions to directly regulate signaling through cytokine receptors, including IFNγ receptor. Thus, enhancing IFNγ sensing and signaling through the inhibition of PTPN2 is a potential therapeutic strategy to improve the efficacy of cancer immunotherapy regimens.

"This marks the first investment by Biovance Capital Fund I, which looks to provide crucial capital to develop transformational therapies for unmet medical needs, and attract world-class scientists and investors to Portugal," said Ricardo Perdigão Henriques, PhD, Managing Partner of Biovance Capital, the investment firm leading the round. "We are excited to join forces with top-tier US healthcare investors to help accelerate safer, more effective treatments—and, ultimately, better outcomes for patients battling cancer," added João Incio, MD PhD, General Partner of Biovance Capital. "We see this investment as an great excellent opportunity to advance a groundbreaking innovation with the potential to reshape current immunotherapy in oncology," said André Albergaria, PhD, Principal of Biovance Capital.

"With very compelling science, we are excited to back Mondego Bio’s work on PTPN2 inhibitors in immuno-oncology, marking our first investment in Southern Europe and a successful collaboration with OrbiMed and Biovance Capital," said Nikolay Savchuk, PhD, Managing Partner of Torrey Pines Investment.

"It is great to have Biovance Capital as a local partner in Portugal supporting the development of this company," added Iain Dukes, PhD, Venture Partner at OrbiMed.

The lead programs are based on research initially developed by Eilean Therapeutics, an AI/ML supported oncology platform co-founded by OrbiMed and Torrey Pines.

On September 18, 2025 K36 Therapeutics, Inc. ("K36"), a privately held clinical-stage biotechnology company developing novel, targeted therapies for cancers with unmet medical need, reported the presentation of two posters at the 22nd Annual International Myeloma Society (IMS) Meeting in Toronto, taking place September 17-20 (Press release, K36 Therapeutics, SEP 18, 2025, View Source [SID1234656079]). The presentations will feature preclinical and translational data from K36’s lead program, KTX-1001, a first-in-class targeted therapy currently being evaluated in an ongoing Phase 1 clinical trial for patients with relapsed or refractory multiple myeloma (MM).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

KTX-1001 is a selective inhibitor of NSD2, also known as MMSET, a histone methyltransferase frequently dysregulated in patients with the t(4;14) chromosomal translocation, a high-risk subtype of multiple myeloma. K36 recently began dosing patients in multiple cohorts of its Phase 1b dose-expansion study assessing KTX-1001 in combination with standard of care and mezigdomide for t(4;14) MM.

The new data presented at IMS highlight the mechanism of action and potential clinical utility of KTX-1001. The data include bone marrow analyses from patients enrolled in the Phase 1 trial that support its clinical relevance. Additional findings from large multi-omics datasets identify a subgroup of newly diagnosed patients with high NSD2 expression in the absence of the t(4;14) translocation, highlighting the opportunity to expand investigation of NSD2 inhibition in patients that lack the translocation. Together, these findings provide important validation of KTX-1001’s differentiated mechanism and support its continued development as a first-in-class targeted therapy for multiple myeloma.

"These new findings strengthen the scientific rationale supporting KTX-1001 as a treatment for multiple myeloma and demonstrate how its differentiated mechanism of action can be translated into the clinic," said Erin Flynt, PhD, Executive Director of Translational Medicine, K36 Therapeutics. "Importantly, the identification of a distinct subgroup of patients with high NSD2 expression beyond the t(4;14) population highlights the potential to broaden the clinical development of KTX-1001 and expand its impact for patients with multiple myeloma."

Poster Details:

Anti-adhesion Properties of KTX-1001, a Selective NSD2/MMSET Inhibitor, Enhance Carfilzomib Sensitivity in Multiple Myeloma

Poster Number: PA-293
Location: Exhibit Hall, Hall E
Presentation Time: Friday, September 19, 12:15 – 1:15 p.m. ET
High Expression of NSD2 in Non-t(4;14) Newly-diagnosed Multiple Myeloma Patients May Mimic t(4;14) Biology

Poster Number: PA-259
Location: Exhibit Hall, Hall E
Presentation time: Thursday, September 18, 12:00 – 1 p.m. ET
Full abstracts can be found at the IMS Annual Meeting website.

About KTX-1001
KTX-1001 is a novel, first-in-class, potent, and selective methyltransferase inhibitor of the catalytic activity of MMSET/NSD2. It is an orally administered small molecule developed initially for the treatment of relapsed and refractory multiple myeloma, with a focus on patients with the t(4;14) translocation. This inhibitor offers a promising avenue for addressing this challenging high risk patient population.

About Multiple Myeloma
Multiple myeloma (MM) is the second most common hematologic malignancy, driven by the uncontrolled proliferation of plasma cells in the bone marrow. According to the American Cancer Society, approximately 36,000 new cases are diagnosed each year. While recent therapeutic advances have extended survival, MM remains incurable, and most patients eventually relapse. High-risk MM, defined by genetic abnormalities such as t(4;14) and other adverse prognostic markers, is associated with aggressive disease biology, shorter survival, and limited benefit from standard-of-care regimens. Addressing this high-risk population represents one of the greatest unmet needs in MM research and treatment.