On September 18, 2025 Geneos Therapeutics, a clinical-stage biotherapeutics company developing personalized immunotherapies for cancer (PICs), reported that two patients with aggressive cancers – one with glioblastoma multiforme (GBM), and another with advanced hepatocellular carcinoma (HCC) treated in the second line – remain on personalized immunotherapy for cancer (PIC) monotherapy, recurrence free and healthy, after more than six years and five years of ongoing treatment, respectively (Press release, Geneos Therapeutics, SEP 18, 2025, View Source [SID1234656084]). Both achieved complete responses and neither has experienced any PIC-related adverse event greater than Grade 1 nor any PIC-related serious adverse event. Additional clinical trial patients with GBM and advanced HCC receiving PIC monotherapy continue to progress toward five years of recurrence-free survival.

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"Geneos’ PICs as monotherapy have now enabled patients with two distinct, difficult-to-treat, rapidly progressing cancers to live beyond five years, recurrence free and healthy – living rich, fulfilling lives," said Niranjan Sardesai, Ph.D., President and Chief Executive Officer of Geneos. "These cases, together with our broader clinical trial results, highlight the durability and tolerability we believe to be achievable with our DNA-based PIC therapy. These results are encouraging and we look forward to continuing to advance our clinical program so that we may bring this potential new treatment option to people living with aggressive cancers rapidly."

The U. S. Food and Drug Administration (FDA) recently released draft guidance identifying overall survival (OS) as a key endpoint in oncology trials. Geneos believes the long-term survival being seen in PIC-treated patients aligns with these regulatory expectations and strengthens the case for broader clinical development.

"Durable overall survival of five years or more in GBM and advanced HCC are uncommon, while recurrence-free survival is almost unheard of," said Ildiko Csiki, M.D., Ph.D., Geneos Chief Medical Officer. "If confirmed in larger datasets, we expect these results to align with FDA’s guidance on overall survival as a primary endpoint for registrational studies."

The patient with GBM presented at age 21 with an IDH-positive, methylated tumor. She received standard of care treatment of surgery, radiation, and temozolomide, as well as a single dose each of two experimental treatments. One year after diagnosis, the patient began PIC monotherapy. Median recurrence-free survival in this setting is 26 months and OS is 40 months. This patient has now reached 75 months of recurrence-free survival and is 87 months from surgical resection. She recently completed a master’s degree and works supporting other cancer patients.

The HCC patient presented at age 61 with a beta catenin mutated form of HCC, having progressed despite liver resection and treatment with an oncolytic virus and sorafenib. Upon enrollment in Geneos’ GT-30 Phase 1b/2a trial, the patient was treated for two years with the combination of PIC and pembrolizumab, after which, per protocol, she was converted to PIC monotherapy. Median OS for such PD-1 treated HCC patients averages 14 months with three-to-four months of progression-free survival. This patient has now reached 60 months of recurrence-free survival and recently welcomed her first grandchild.

PICs are DNA-based tumor-infiltrating lymphocyte (TIL)-inducing agents containing up to 43 of a patient’s specific cancer neoantigens. They have been shown to have a 100% success rate in patients at inducing CD8+ activated cytotoxic T effector memory cells which traffic to tumors, the first such immunotherapeutic ever to achieve this metric. Unlike experimental mRNA-based personalized immunotherapeutics generally administered for no more than nine months, the tolerability of PICs supports uninterrupted treatment over years to maintain TIL response and minimize the odds of recurrence or progression.

Geneos is actively preparing to advance PIC monotherapy development in the upcoming GT-31 Phase 2b randomized, controlled clinical trial as adjuvant immunotherapy of patients with HCC.

On September 18, 2025 Immuto Scientific reported the closing of an oversubscribed $8 million Seed 2 financing round and a drug discovery collaboration with Daiichi Sankyo, a global pharmaceutical company. Immuto Scientific is applying its AI-enabled structural surfaceomics platform to a new approach in drug discovery (Press release, Immuto Scientific, SEP 18, 2025, https://www.businesswire.com/news/home/20250912125211/en/Immuto-Scientific-Announces-%248M-Seed-2-Financing-and-Daiichi-Sankyo-Collaboration-to-Uncover-a-New-Class-of-Therapeutic-Targets-with-Structural-Surfaceomics [SID1234656083]). Backed by the financing, the company will advance its internal oncology pipeline toward IND-enabling studies and extend the platform into additional areas such as immunology and inflammation.

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"The greatest challenge in drug discovery today is not the lack of modalities; it’s the shortage of truly novel, disease‑specific targets," said Faraz A. Choudhury, Ph.D., co‑founder and CEO of Immuto Scientific. "The vast majority of drug targets overlap with healthy tissues, which drives toxicity and narrow therapeutic windows. Our data‑driven platform reveals a hidden dimension of the cell surfaceome that conventional omics approaches cannot see, opening new therapeutic opportunities across cancer and other diseases."

For its first drug discovery collaboration, Immuto will work with Daiichi Sankyo Research Institute Boston to discover new targets in solid tumors using its proprietary structural surfaceomics platform and to develop antibodies against these targets. Under the terms of the agreement, Daiichi Sankyo holds an option to license resulting assets. Financial terms were not disclosed.

"Our collaboration with Daiichi Sankyo underscores how exploring the structural conformations of cell‑surface proteins represents an untapped frontier in drug discovery," said Dan Benjamin, Chief Technology Officer of Immuto Scientific. "By targeting cancer‑specific surface structures, we have the opportunity to develop first‑in‑class therapies with truly differentiated precision and selectivity."

Building on its success collaborating with pharmaceutical and biotechnology partners in high‑resolution structural proteomics, Immuto applies its target discovery platform and structural epitope‑mapping engine to identify disease‑specific surface protein conformations that are invisible to other omics approaches, unlocking highly selective, novel therapeutic targets. The platform integrates high‑resolution structural proteomics, live‑cell protein structural assessment, and advanced AI‑enabled analytics to interrogate conformational differences across thousands of proteins simultaneously—even in heterogeneous, patient‑derived samples.

By identifying epitopes specific to disease states, the platform enables discovery of first‑in‑class targets ideally suited to highly disease‑specific modalities, providing opportunities for improved therapeutic indices and translational success. Immuto has demonstrated target identification in multiple tumor types and is advancing its lead program through in vivo studies.

"Immuto Scientific’s approach is highly differentiated, creating and leveraging data that no other group in the world has," said Spencer Maughan, Founder and Managing Partner, DYDX. "The company is driving a step‑change in predictive protein structure based on its data and AI engine. This opens a new world of targets for much‑needed therapies with exceptional specificity. We are excited to be partnering with Immuto and believe the company will be a key part of the new era of data‑driven drug discovery."

The financing was led by DYDX, a venture fund investing in the Data SuperCycle, with participation from WARF Ventures, Gravity Fund, Great Oaks Venture Capital, among others.

On September 18, 2025 IASO Biotherapeutics ("IASO Bio"), a biopharmaceutical company focused on the discovery, development, manufacturing, and commercialization of innovative cell therapies, reported that the 36-month long-term follow-up data from the FUMANBA-1 study of its independently developed fully human anti-BCMA CAR-T cell therapy Fucaso (Equecabtagene Autoleucel, Eque-cel), for the treatment of relapsed/refractory multiple myeloma (R/R MM), were presented at the 2025 International Myeloma Society (IMS) Annual Meeting (Press release, IASO Biotherapeutics, SEP 18, 2025, View Source;iaso-bio-highlights-three-year-follow-up-data-of-car-t-cell-therapy-fucaso-for-multiple-myeloma-treatment-302561198.html [SID1234656080]). The results further demonstrate that Fucaso delivers deep and durable efficacy in patients with relapsed/refractory multiple myeloma (R/R MM), including those with high-risk features, along with a manageable long-term safety profile that significantly enhances overall survival quality.

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The results of this study were presented by Professor Lugui Qiu from Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences in an oral presentation at the IMS Annual Meeting (Abstract Number: 2142568). This presentation is based on findings from FUMANBA-1, a phase 1b/2 study evaluating the safety and efficacy of Fucaso, which was conducted at 14 sites in China. R/R MM patients who had received at least three prior lines of therapy and with progressive disease after the last line of therapy were enrolled. And patients with extramedullary disease (EMD) or prior exposure to anti-BCMA CAR-T therapy were included.

As of December 31, 2024, a total of 109 patients with R/R MM had received Fucaso, 12.8% had EMD and 11% had received prior BCMA CAR-T therapy. Following lymphodepletion with cyclophosphamide and fludarabine for three consecutive days, a single infusion of CAR-T cells (1×10⁶ cells/kg) was administered.

Deep and durable efficacy

Among 107 evaluable patients, the overall response rate (ORR) was 96.3%, including a complete or stringent complete response (CR/sCR) in 83.2%. In CAR-T–naïve patients, ORR and CR/sCR rates were 98.9% and 88.4%, respectively. Among 109 patients who received Eque-cel, the median progression-free survival (PFS) was 30.5 months, extending to 35.9 months in CAR-T–naïve patients. Median overall survival (OS) was not reached. Minimal residual disease (MRD) negativity was achieved in 95.3% (102/107) of evaluable patients, including all patients who achieved CR or sCR, the median duration of MRD negativity was 36.5 months.

Manageable long-term safety profile

CRS occurred in 93.6% of patients, with only one case≥grade 3;
ICANS was reported in two patients (grade 1–2);
No late-onset neurotoxicity or secondary malignancies were observed.
Conclusion: At a median follow-up of 36.0 months, Fucaso therapy demonstrated deep, durable responses and sustained MRD negativity in heavily pretreated R/R MM patients, including those with high-risk features. The long-term safety profile remained manageable, with no new safety signals identified.

The principal investigators of this study, Professor Lugui Qiu, from Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences, and Professor Chunrui Li, from Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology, remarked: "We are delighted to present the three-year follow-up data from the FUMANBA-1 study of Eque-cel injection at this year’s IMS Annual Meeting, and the results are highly encouraging. Due to its moderate antigen affinity, Eque-cel enables rapid binding and dissociation between CAR-T cells and tumor cells, contributing to a rapid onset of action and potent tumor clearance, thereby leading to deep responses in patients with relapsed or refractory multiple myeloma (R/R MM). Furthermore, as a human-derived CAR-T product, it exhibits low immunogenicity. While maintaining a low exhaustion phenotype, it achieves prolonged persistence, resulting in sustained antitumor activity and extended patient survival. It is particularly noteworthy that Eque-cel achieved a median progression-free survival (mPFS) of 35.9 months in CAR-T–naïve patients. These results indicate that the therapy can provide a longer treatment-free interval, significantly improve patients’ quality of life[1,3]."

Ms. Jinhua Zhang, Founder, Chairwoman, and CEO of IASO Biotherapeutics, remarked: "We are greatly encouraged to see that the three-year follow-up data from the FUMANBA-1 study of Fucaso has once again confirmed its outstanding long-term efficacy and reliable safety profile. Notably, the complete response/stringent complete response rate among patients receiving BCMA CAR-T therapy for the first time has increased to 88.4%. Delivering sustained clinical benefits to patients with relapsed or refractory multiple myeloma remains our greatest motivation. The achievement of this important milestone is made possible through the collective efforts and unwavering dedication of both the investigator team and the IASO team. Building on the remarkable efficacy and safety profile of Fucaso, we are vigorously advancing the FUMANBA-3 clinical study for its second- and third-line treatments, while accelerating global registration and market access efforts to actively expand its presence in international markets. We look forward to making this high-quality CAR-T cell therapy accessible to a broader patient population worldwide."

About Equecabtagene Autoleucel (Fucaso)

Equecabtagene Autoleucel (Fucaso) is an innovative fully human anti-BCMA CAR-T cell therapy which uses lentivirus as a gene vector to transfect autologous T cells. The CAR contains a fully human scFv, CD8a hinge and transmembrane domain, and 4-1BB co-stimulatory molecule and CD3ζactivation domains. Based on rigorous molecular structure screening and comprehensive in vitro and in vivo functional evaluations, Fucaso demonstrates rapid and potent efficacy, accompanied by exceptional long-term persistence in vivo, enabling patients to achieve deep and durable remission, providing continuous protection and care for patients with multiple myeloma.

About Multiple Myeloma (MM)

Multiple myeloma (MM) is the second most common hematological malignancy globally. According to Globocan data, the global incidence of multiple myeloma in 2022 was 1.8 per 100,000 people, with a 5-year prevalence of 6.8 per 100,000. Despite progress in current anti-myeloma treatments, MM remains largely incurable with multiple relapses and tendency to develop refractoriness to several drug classes, presenting a major therapeutic challenge. Thus, there is an unmet need for new treatment options beyond these current anti-myeloma therapies for the treatment of relapsed or refractory MM, capable of achieving deep and durable responses.

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).

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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.

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.

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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.