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.

On September 17, 2025 ProteinQure, a Toronto-based biotech company pioneering computational peptide drug discovery, reported the successful dosing of the first patient in its Phase I clinical trial evaluating PQ203 – a novel, rationally designed peptide therapeutic for advanced metastatic solid tumors (Press release, ProteinQure, SEP 17, 2025, View Source [SID1234656043]).

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"Dosing our first patient with PQ203 represents a defining moment not just for ProteinQure, but for the vision from the founding of the company — that computational tools can unlock new medicines," said Lucas Siow, CEO and Co-Founder of ProteinQure. "Having created PQ203 in Toronto and starting the trial with the exceptional clinical team at Princess Margaret underscores the ability to advance world-class science in Canada."

The trial is being started at Princess Margaret Cancer Centre in Toronto, one of the world’s top five cancer research centers. There, the trial is led by investigator Dr. Philippe Bedard,

"It’s exciting to be part of a groundbreaking program that was discovered using the latest in computational science," said Dr. Bedard. "Peptides open up a new and promising path for targeted cancer treatments, and we’re looking forward to seeing how PQ203 might help patients who currently have limited treatment options."

About the Trial

The Phase 1 trial is a first-in-human trial with three parts: dose escalation, dose expansion (in multiple tumor types), and dose optimization. The study will evaluate the safety, tolerability, pharmacokinetics, preliminary activity, and pharmacodynamics of PQ203. It will take place across Canada and the US at clinical sites including Princess Margaret Cancer Centre, McGill, Yale, MD Anderson, and Next Oncology.

For more information about the PQ203 trial, visit www.clinicaltrials.gov (NCT# pending) or contact [email protected].

About PQ203

PQ203 is the company’s first internally owned AI designed peptide therapeutic entering the clinic 3 years after the program was started. PQ203 is composed of a peptide targeting the Sortilin receptor conjugated to the cytotoxic agent MMAE. The Sortilin receptor is expressed in a high percentage of diseased tissue from Triple Negative Breast Cancer (TNBC) patients. ProteinQure has generated data that PQ203 exhibits potent efficacy in a patient-derived xenograft (PDX) model resistant to Sacituzumab Govitecan (Trodelvy), an antibody drug conjugate that is the standard of care for metastatic TNBC.

On September 17, 2025 Purdue Pharma L.P. ("Purdue") reported that it has entered into an agreement to have its pipeline medication tinostamustine included in the Phase 2/3 GBM AGILE (Glioblastoma Adaptive Global Innovative Learning Environment) adaptive clinical trial for glioblastoma patients led by the Global Coalition for Adaptive Research (GCAR) (Press release, Purdue Pharma, SEP 17, 2025, View Source [SID1234656046]).

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Tinostamustine is a first-in-class, new chemical entity that combines two potentially synergistic mechanisms of action, bifunctional alkylating activity and pan histone deacetylase inhibition (or HDAC inhibition). Tinostamustine is under investigation in patients with glioblastoma, an aggressive brain cancer that is very challenging to treat and for which there is no cure.1 Most patients do not survive more than 15 months with current treatment approaches.1 Tinostamustine has the potential to be a first-line treatment. Nearly 14,000 people in the U.S. are diagnosed with glioblastoma each year. Tinostamustine will be investigated in patients with newly diagnosed glioblastoma as an adjuvant therapy following standard treatment with surgery, chemotherapy and radiation, as well as in patients with recurrent disease.

GBM AGILE is a pioneering, international adaptive platform trial designed to accelerate the identification of effective treatments for glioblastoma. It is led by GCAR, a non-profit corporation, and is supported by a global network of oncologists, neuro-oncologists, clinicians, researchers, biostatisticians, and patient advocates. Unlike traditional trials, GBM AGILE offers a seamless phase 2/3 design that supports a well-defined path for FDA registration. It allows for the simultaneous assessment of multiple investigational therapies against a common control arm, significantly reducing development time by adapting to emerging data and prioritizing promising new treatments.

"We are pleased to partner with GCAR in GBM AGILE to further study tinostamustine, which has shown promise in early trials," said Dr. Julie Ducharme, Vice President and Chief Scientific Officer, Purdue. "GBM AGILE accelerates the clinical trial timeline to speed medicines to patients, which aligns with our mission to address serious, unmet medical needs."

"We are pleased to be evaluating tinostamustine in GBM AGILE," said Dr. Meredith Buxton, Chief Executive Officer/President, GCAR. "Glioblastoma is the deadliest brain cancer, and our mission is to find promising new treatments to improve overall survival as compared to standard treatments. We are now hard at work collaborating on the Investigational New Drug Application (IND) and Clinical Trial Application (CTA) submissions necessary to include tinostamustine in our innovative and patient-centric adaptive trial."

The study is anticipated to begin following IND submission and FDA approval of the protocol with activation in Canada, Europe, and Australia to follow.

On September 17, 2025 TATUM bioscience, a preclinical biotechnology company developing a new immunotherapy to fight cancer, reported the publication of new research showing strong antitumor activity from its proprietary bioengineered multimodal nanofilaments (Press release, TATUM bioscience, SEP 17, 2025, View Source [SID1234656047]). The study, published in the new issue of the Journal for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) (JITC), details how this groundbreaking therapy exposes ("uncloaks") cancer cells and orchestrates a comprehensive immune response that eradicates tumors.

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"Our innovative nanofilament-directed immunotherapy overcomes cancer’s ability to hide from the immune system and unleashes a coordinated immune response against tumor cells," said Jean-François Millau, Ph.D., co-founder and Chief Executive Officer of TATUM Bioscience.

"The immune system can be compared to an orchestra: for a powerful antitumor response, each instrument — or cellular component — must play its part at the right moment. That’s what our drug candidate, TAT003, achieves." Dr. Millau is a co-author on the article, "Nanofilament immunotherapy induces potent antitumor vaccine responses."

How TAT003 Works

TAT003 is administered directly into the tumor ("intratumorally") and combines three biological functions to drive the immune system’s antitumor response:

Checkpoint Blockade: Anti–PD-L1 single-chain antibody fragments at one end of the nanofilament bind to tumor cells, decorating the tumor and blocking the PD-L1 immune checkpoint. This allows the immune system to recognize and target the cancer.
Innate Activation: Encapsulated within the nanofilament is a TLR9 agonist, which activates macrophages to engulf tumor cells. Activated macrophages present tumor antigens to the broader immune system.
T Cell Stimulation: IL-2 molecules on the nanofilaments stimulate T cells, triggering them to seek out and destroy other cells bearing tumor antigens — mimicking a vaccine-like response.
"Strikingly, the antitumor response triggered by TAT003 nanofilament immunotherapy was both systemic and durable in animal models," said Kevin Neil, Ph.D., co-founder and Chief Scientific Officer of TATUM. "Replicating these results in patients is our goal as we move toward clinical development."

Unique Advantages Over Personalized Vaccines

TATUM’s engineered nanofilaments offer distinct advantages over other therapeutic strategies. According to Dr. Neil, "TAT003 elicits a vaccine-like effect without the complexity and cost of individualized neoantigen vaccines, which require tumor sequencing and custom manufacturing for each patient. Our approach is designed to be more practical and scalable."

Manufacturing complex, multi-specific biologics remains a major challenge in biopharma. TAT003 addresses this with a proprietary synthetic biology platform: "We have engineered bacteria to function as miniature drug factories, producing fully assembled therapeutic nanofilaments in a single step," explained Dr. Millau. "We simply culture the bacteria, and they secrete the complete, functional drug."

Dr. Gerald Batist, director of the Segal Cancer Centre at Jewish General Hospital in Montréal, commented, "These results are highly promising and suggest that nanofilament-directed immunotherapy could overcome some of the limitations seen with today’s personalized neoantigen approaches. Engaging both innate and adaptive immunity, as shown here, is likely key to improving outcomes for patients who do not benefit from current immunotherapies. This represents a major step forward in the evolving landscape of immuno-oncology." Dr. Batist is a medical advisor to TATUM.

Looking Ahead

These findings represent a major milestone for TATUM Bioscience and the advancement of nanofilament-directed immunotherapy, paving the way for a powerful and innovative approach to treating solid tumors. TATUM is currently raising funds to accelerate development and bring TAT003 into clinical trials. The company welcomes interest from investors, partners, and clinicians seeking to be part of this next era in cancer immunotherapy.