PALETTE- Adaptive Platform Trial for Personnalisation of Sepsis Treatment in Children and Adults: a Multi-national, Treatable Traits-guided, Adaptive, Bayesian Basket Trial"

PALETTE is a perpetual adaptive platform to efficiently study sepsis interventions within 'treatable traits' in all-ages patients enabling prompt evaluation of pandemic treatments. Treatable traits, therapeutic targets identified by phenotypes or endotypes (defined by biological mechanism or by treatment response) through validated biomarkers (measurable characteristic reflecting normal or pathogenic processes, or treatment responses), may include multi-omics, cellular, immune, metabolic, endocrine features, or intelligent algorithms. PALETTE Bayesian adaptive design enables parallel investigations of multiple interventions for sepsis, and quick inclusion of pandemic pathogens. PALETTE's new conceptual model will respond to the challenges of standard approaches, i.e. series of sepsis trials, each investigating one or two interventions, expensive, time consuming, and inappropriate in pandemic context.

Start Date01 Apr 2026

Sponsor / Collaborator

Assistance Publique des Hôpitaux de Paris SA

NCT03787290

/ SuspendedPhase 2IIT

Examining Immune-Based Mechanisms of Action for Mild-Intensity Whole Body Hyperthermia (WBH) in the Treatment of Major Depressive Disorder

Overall, the objective of this pilot study is to utilize the IL-6 receptor antagonist tocilizumab to prospectively evaluate the role of IL-6 in the antidepressant and immunological effects of whole body hyperthermia (WBH). The study seeks to replicate findings thus far that WBH has an antidepressant effect by administering the intervention at a site not involved in studies to date. Moreover, the current proposal may help the investigators better understand the role of IL-6 in the pathogenesis and treatment of depression which might point to novel immune-based interventions for Major Depressive Disorder (MDD). Finally, the current proposal holds promise for better understanding of a novel treatment for MDD, which is among the leading causes of health-related disability in the world.

Start Date01 Dec 2025

Sponsor / Collaborator

The General Hospital Corp.

[+1]

NCT06016517

/ Not yet recruitingNot ApplicableIIT

Application of the Personalized N-of-1 Trial Design in Patients With Rheumatoid Arthritis

The goal of this N-of-1 study is to learn about treatment for individual patients who have rheumatoid arthritis (RA,) for which many treatments are available. The treatments are different in how they work, the way they are given, side- effects, and cost. While treatment guidelines are available, finding the best treatment order of treatments is often based on physician choice. The main question this study aims to answer are:
* What are the effects of different treatments on RA symptoms and condition for each individual patient
* What is the effectiveness of different treatments across all patients enrolled in the N-of-1 study
Participants will be enrolled and randomized to a sequence of three U.S. Food and Drug Administration (FDA) approved RA medications: 1. etanercept, 2. adalimumab, 3. upadacitinib 4. tocilizumab. Participants will be asked to complete questionnaires about their condition and quality of life fortnightly, monthly and/or quarterly (either in clinic or remotely) and report their level of pain on alternate days (remotely).

Start Date15 Nov 2025

Sponsor / Collaborator

Tufts Medical Center, Inc.

100 Clinical Results associated with Tocilizumab

100 Translational Medicine associated with Tocilizumab

100 Patents (Medical) associated with Tocilizumab

7,021

Literatures (Medical) associated with Tocilizumab

01 Jan 2026·INTERNATIONAL JOURNAL OF FOOD MICROBIOLOGY

Physicochemical properties, microbial diversity, metabolites and their potential relationships of traditional Chinese Zhacai fermented for different periods

Article

Author: Wang, Xinhui ; Ren, Ting ; Wang, Xinyi ; Gu, Yuanqing ; Nie, Xin ; Liu, Dayu ; Zhao, Zhiping ; Luo, Yuanli ; Zhao, Nan ; Liu, Yuling

In this study, we investigated the physicochemical properties, dominant microorganisms, non-volatile metabolites and their relationships of traditional Chinese Zhacai (TCZ) at the "second-time pickling and pressing" stage fermentation. pH and amino acid nitrogen contents exhibited an increasing and then decreasing trend. Metagenomics analysis revealed the dominant bacterial species Lactiplantibacillus (Lpb.) plantarum and Latilactobacillus (Lat.) sakei, and fungal species Debaryomyces (D.) hansenii. LEfSe analysis identified a total of 47 differentially abundant bacteria, including Lpb. plantarum and Lat. Sakei. Metabolomics analysis indicated organic acids and their derivatives, organic heterocyclic compounds, lipids and lipid-like molecules as the major differential metabolites. Spearman's correlation results showed significant correlations among differential bacteria, non-volatile metabolites and physicochemical properties. Unexpectedly, differential fungi were not observed in TCZ at this fermentation period. Pathway enrichment identified phenylpropanoid biosynthesis and tryptophan metabolism as dominant metabolic pathways, both influencing the TCZ flavor development. This study contributes to further understanding of quality formation of TCZ, providing a foundational resource for future optimization and industrial application.

01 Dec 2025·Journal of Translational Autoimmunity

Acylcarnitine enrichment as a characteristic of rheumatoid arthritis fibroblast-like synoviocyte metabolic fingerprint

Article

Author: Hørslev-Petersen, Kim ; Østergaard, Mikkel ; van Vollenhoven, Ronald ; Sureshkumar, Reshmi ; Guma, Monica ; Nurmohamed, Michael ; Hultgård Ekwall, Anna-Karin ; Gröndal, Gerdur ; Nordström, Dan ; Vasileiadis, Georgios K ; Fatima, Tahzeeb ; Zhang, Yuan ; Haavardsholm, Espen A ; Lend, Kristina ; Rudin, Anna ; Maglio, Cristina ; Gudbjornsson, Bjorn ; Borén, Jan ; Lampa, Jon ; Uhlig, Till ; Hetland, Merete L ; Sokka-Isler, Tuulikki ; Laudette, Marion

Objective:

In rheumatoid arthritis (RA), fibroblast-like synoviocytes (FLS) alter their metabolism to support their activation. We aimed to analyse the full spectrum of metabolic alterations associated with RA by performing untargeted metabolomics in RA FLS vs. non-inflamed (NI) FLS.

Methods:

Untargeted annotated metabolomics was performed using mass spectrometry on ten primary RA and seven NI FLS culture extracts and 220 serum samples from participants with early RA from the randomised controlled NORD-STAR trial. Carnitine-related proteins were measured with Western blot. FLS bioenergetic profile was assessed with a Seahorse flux analyser.

Results:

Metabolomics analysis based on 138 annotated metabolites revealed a distinct metabolic fingerprint between RA and NI FLS. Of the 12 metabolites enriched in RA FLS, 11 were acylcarnitines. Pro-inflammatory stimulation of NI FLS also led to acylcarnitine accumulation. RA FLS exhibited lower levels of CD36, a fatty acid transporter, but similar levels of L-carnitine transporter, and carnitine palmitoyltransferase 1 A and 2 compared to NI FLS. Seahorse analyses showed no difference in fatty acid oxidation between RA and NI FLS; however, RA FLS displayed mitochondrial dysfunction and energetic impairment. Serum acylcarnitine content decreased after 24 weeks of treatment with methotrexate combined with abatacept or tocilizumab in patients with early RA achieving remission.

Conclusion:

Acylcarnitine accumulation is a characteristic of RA FLS metabolic fingerprint and could be linked to mitochondrial dysfunction. In patients with early RA, acylcarnitine content in serum decreases after successful anti-rheumatic treatment. These results indicate a dysregulation in acylcarnitine metabolism in RA at the joint level and systemically.

01 Dec 2025·JOURNAL OF MEDICAL ECONOMICS

Comparison of utilization and total medicare fee-for-service expenditures for subcutaneous versus intravenous versions of a select group of therapies.

Article

Author: Sarocco, Phillip ; Kardel, Peter ; Maynard, Jason ; Sheetz, Caitlin

INTRODUCTION:

Comparisons of subcutaneous (SC) and intravenous (IV) delivery of biotherapeutics in Europe have revealed differences in cost, convenience, and preference. In the US, claims-based studies comparing SC and IV delivery have been limited. This study compared resource utilization and expenditures between cohorts of patients receiving the same drug by the SC or IV route of administration (ROA) across several indications.

METHODS:

Medicare Fee-For-Service claims were compared between cohorts of patients who received SC- or IV-delivered abatacept, belimumab, daratumumab, infliximab, tocilizumab, trastuzumab/pertuzumab, or vedolizumab. For each drug, utilization rates and healthcare expenditures per service site were compared between ROAs. Observed differences in baseline characteristics were accounted for using Inverse Probability Treatment Weighting models that balanced pre-index differences between ROAs per drug. Each ROA comparison per drug and service site was weighted and conducted independently.

RESULTS:

Across seven drugs, a total sample size of 158,632 (72,820 IV; 85,812 SC) was analyzed. For most comparisons, high-spend sites were utilized at a higher rate for IV administration. In particular, all comparisons revealed more frequent hospital outpatient department utilization for the IV ROA. For five of the seven drugs, SC treatment was associated with lower mean total Medicare expenditures, with savings of up to $56,000 per patient annually. Although three SC treatments had higher medication index spend, the total spend for these drugs across sites was significantly lower for SC delivery.

LIMITATIONS:

Limitations of this study include differences in billing between the SC and IV ROAs, potential treatment selection bias, and the assumption of equivalent efficacy.

CONCLUSIONS:

To our knowledge, this study was the first and largest US Medicare claims analysis, comparing patients receiving SC or IV versions of the same therapeutic across multiple drugs and indications. Findings demonstrated that SC delivery may facilitate reduced resource utilization and expenditures across drugs and disease indications.

260

News (Medical) associated with Tocilizumab

20 Oct 2025

·www.globenewswire.com

BioAtla Presents Promising Interim Data from its Phase 1 Trial with BA3182 in Patients with Treatment Refractory Metastatic Adenocarcinoma at ESMO 2025

Adverse events (AEs) generally transient and readily manageable; only 2 cases of cytokine release syndrome (CRS)Prolonged tumor control with increasing doses of BA3182; confirmed partial response (cPR) at 0.6 mg in a patient with intrahepatic cholangiocarcinoma without progression for >6 monthsMaximally tolerated dose not yet defined; dose escalation continues; currently testing 1.8 mg dose level SAN DIEGO, Oct. 20, 2025 (GLOBE NEWSWIRE) -- BioAtla, Inc. (Nasdaq: BCAB), a global clinical-stage biotechnology company focused on the development of Conditionally Active Biologic (CAB) antibody therapeutics for the treatment of solid tumors, today announced preliminary clinical data from a Phase 1 study of BA3182 during a poster presentation titled “Preliminary Results from a First-in-Human Phase 1 Study of BA3182, a Dual-Conditionally Binding Biologic (CAB) EpCAM x CD3 Bispecific T-cell Engager in Patients with Treatment Refractory Metastatic Adenocarcinoma” at the European Society for Medical Oncology (ESMO) Congress 2025, held in Berlin, Germany. The poster was featured in the Investigational Immunotherapy session on Sunday, October 19. EpCAM is broadly expressed among adenocarcinomas of the colon, stomach, pancreas, biliary tract, lung, breast, prostate, and thyroid, making it a compelling bispecific T-cell engager (TCE) target when reliably restricting antibody binding in the tumor microenvironment (TME). EpCAM is also broadly expressed on healthy epithelial tissues, and this broad expression is associated with on-target, off-tumor toxicities when targeted by traditional antibodies. BA3182 is a dual-conditionally binding bispecific TCE targeting EpCAM and CD3, designed to selectively bind within the acidic TME, and eliminate on-target, off-tumor toxicity. “We continue to observe encouraging preliminary prolonged tumor control and a manageable safety profile with our dual-CAB EpCAM x CD3 bispecific TCE that combines, and thereby multiplies, the binding selectivity of the CAB targets for directing the body’s T-cells to kill cancer cells, while protecting healthy tissues,” said Jay M. Short, Ph.D., Chairman, Chief Executive Officer and co-founder of BioAtla, Inc. “BA3182 has tremendous promise across a broad range of adenocarcinomas with the potential to serve over one million patients globally.” Key Highlights from the Poster PresentationBA3182 demonstrates a manageable safety profile with preliminary evidence of antitumor activity 35 patients were dosed subcutaneously with BA3182 once weekly; all but one received 1 or 2 priming doses 4 to 7 days prior to treatment (as of September 10, 2025)Patients were heavily pre-treated receiving a median of 3 prior lines of therapy (range 2 to 9)Patients treated at the 0.6 mg dose level received a greater number of doses, mean of 11+ doses, relative to all preceding lower doses, with 7 patients presently continuing on therapy. Safety BA3182 priming and first treatment doses were associated with transient and reversible elevations in hepatic analytes, consistent with cholestasis, that resolved with ongoing, higher treatment exposuresMinimal, transient, low-grade CRS observed to date (G1, N=1; G2, N=1)Priming dose of 0.1 mg safely enabled scheduled delivery of higher continuing treatment dosesObserved DLTs (N=3) were transient and reversible, enabling continued treatment 0.1 mg prime followed by 0.3 mg treatment dose observed G3 non-febrile neutropenia possibly related to tocilizumab; dose escalated to 0.6 mg with no neutropenia recurrence 0.3 mg prime followed by 0.6 mg treatment dose observed transient G4 ALT0.6 mg prime followed by 1.2 mg treatment dose observed reversible G3 diarrhea associated with concomitant CMV enteritis; no diarrhea recurrence upon BA3182 rechallenge with 0.6 mg and continuing treatment No treatment-related deaths occurred; only one patient discontinued treatment due to an AE Safety profile supports continued dose escalation; maximally tolerated dose not yet defined Preliminary assessment of efficacy Response-evaluable patients included those with at least one scan; preliminary assessment of anti-tumor activity demonstrated prolonged tumor control with increasing doses of BA3182For those treated with BA3182 at doses of 0.6 mg and higher (N=24): 14 CRC patients: among 7 patients who had one scan, 5 achieved stable disease (SD) at 1st scan6 pancreatic cancer patients: 2 had SD at 1st scan, and 2 continued treatment beyond potential pseudo-progression2 cholangiocarcinoma patients: 1 achieved a cPR and 1 is pending first scan1 Adenoid Cystic Carcinoma (ACC) patient experienced SD; 1 patient with appendiceal cancer withdrew consent before 1st scanAmong patients treated at higher doses, patients achieved SD at a higher rate and remained on active treatment for prolonged intervals, generally longer than those who received lower doses A copy of the presentation materials can be accessed on the “Publication” section of the Company’s website at www.bioatla.com. About CAB-EpCAM x CAB-CD3 Bispecific T-cell Engager Antibody BioAtla is developing BA3182 as a potential anticancer therapy for patients with advanced adenocarcinoma. BA3182 is a (CAB) EpCAM x (CAB) CD3 bispecific T-cell engager antibody that contains two binding sites for EpCAM and two binding sites for CD3ε. The binding sites for EpCAM and CD3ε have been designed to bind their respective targets specifically and reversibly under the acidic conditions found in the TME and to have reduced binding in the normal alkaline environment outside of the TME. The CAB selective binding to both the CAB EpCAM and CAB CD3ε arms are required to activate the T-cell engagement against the tumor, thus enabling the combined selectivity of each CAB binding arm in the bispecific antibody. BioAtla continues to advance the ongoing Phase 1 study to evaluate the safety, pharmacokinetics, and efficacy of BA3182 in advanced adenocarcinoma patients. About CAB technologyCAB technology is a proprietary technology engineered to activate only in acidic, inflammatory conditions found in diseased microenvironments, but not in normal tissues. BioAtla utilizes its CAB technology to develop novel, reversibly active monoclonal and bispecific antibodies and other protein therapeutic product candidates. CAB product candidates are designed to have more selective targeting, greater efficacy with lower toxicity, and more cost-efficient and predictable manufacturing than traditional antibodies. About BioAtla®, Inc. BioAtla is a global clinical-stage biotechnology company with operations in San Diego, California, and in Beijing, China through its contractual relationship with BioDuro-Sundia, a provider of preclinical development services. BioAtla has extensive and worldwide patent coverage for its CAB platform technology and products with greater than 780 active patent matters, more than 500 of which are issued patents. Broad patent coverage in all major markets includes methods of making, screening and manufacturing CAB product candidates in a wide range of formats and composition of matter coverage for specific products. To learn more about BioAtla, Inc., visit www.bioatla.com. Forward-looking StatementsStatements in this press release contain "forward-looking statements" that are subject to substantial risks and uncertainties. Forward-looking statements contained in this press release may be identified by the use of words such as "anticipate," "expect," "believe," "will," "may," "should," "estimate," "project," "outlook," "forecast," "has potential to" or other similar words. Examples of forward-looking statements include, among others, statements we make regarding BioAtla’s business plans and prospects; results, progress and timing of our research and development programs and clinical trials; expectations with respect to enrollment and dosing in our clinical trials; and the potential size of the addressable patient population for BA3182. Forward-looking statements are based on BioAtla's current expectations and are subject to inherent uncertainties, risks and assumptions, many of which are beyond our control, difficult to predict and could cause actual results to differ materially from what we expect. Further, certain forward-looking statements are based on assumptions as to future events that may not prove to be accurate. Factors that could cause actual results to differ include, among others: factors that raise substantial doubt about our ability to continue as a going concern and that we will need additional funding to continue development of our CAB technology platform and our CAB product candidates; the risk that preliminary or interim clinical results may not be indicative of results from later cohorts or larger populations; potential delays in clinical and preclinical trials; the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for clinical trials, regulatory submission dates, or regulatory approval dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; whether regulatory authorities will be satisfied with the design of and results from the clinical studies or take favorable regulatory actions based on results from the clinical studies; our dependence on the success of our CAB technology platform; our ability to enroll patients in our ongoing and future clinical trials; the successful selection and prioritization of assets to focus development on selected product candidates and indications; our ability to form collaborations and partnerships with third parties and the success of such collaborations and partnerships; our reliance on third parties for the manufacture and supply of our product candidates for clinical trials; our reliance on third parties to conduct our clinical trials and some aspects of our research and preclinical testing; the accuracy of our estimates regarding addressable patient populations and market opportunity; potential adverse impacts due to geopolitical or macroeconomic events outside of our control, including health epidemics or pandemics; and those other risks and uncertainties described in the section titled "Risk Factors" in our Annual Report on Form 10-K filed with the Securities and Exchange Commission (the “SEC”) on March 27, 2025, our Quarterly Report on Form 10-Q filed with the SEC on May 6, 2025, and August 7, 2025 and our other reports as filed with the SEC. Forward-looking statements contained in this press release are made as of this date, and BioAtla undertakes no duty to update such information except as required under applicable laws. Internal Contact: Richard Waldron Chief Financial Officer BioAtla, Inc. rwaldron@bioatla.com 858.356.8945 External Contact: Joyce Allaire LifeSci Advisors, LLC jallaire@lifesciadvisors.com

Phase 1Clinical ResultImmunotherapy

20 Oct 2025

·www.prnewswire.com

CARsgen Presents Preliminary Results on Satri-cel for Adjuvant Therapy of Pancreatic Cancer at ESMO Congress 2025

SHANGHAI, Oct. 20, 2025 /PRNewswire/ -- CARsgen Therapeutics Holdings Limited (Stock Code: 2171.HK), a company focused on developing innovative CAR T-cell therapies, announces that the research results of the Phase Ib registrational clinical trial of satricabtagene autoleucel ("satri-cel", CT041) (an autologous CAR T-cell product candidate against protein Claudin18.2) for pancreatic cancer (PC) adjuvant therapy in China (CT041-ST-05, NCT05911217) has been presented in poster session at European Society for Medical Oncology (ESMO) Congress 2025. The poster was titled "Adjuvant Therapy with Claudin18.2-specific CAR T Cells (Satri-cel) in High-Risk Pancreatic Cancer (CT041-ST-05)" (Poster number: 2220P). The trial represents the world's first proof-of-concept (POC) study exploring CAR T-cell therapy for the adjuvant treatment of solid tumors. Professor Xianjun Yu from Fudan University Shanghai Cancer Center serves as the principal investigator. Pancreatic ductal adenocarcinoma (PDAC) is characterized by a dismal prognosis even among patients who undergo surgical resection. Local recurrence and distant metastasis are common, often leading to treatment failure. Elevated carbohydrate antigen 19-9 (CA19-9) levels post resection indicate aggressive tumor biology and higher risk of recurrence. The median interval is approximately 3 months between CA19-9 elevation and radiological recurrence. [1][2] Current standard adjuvant therapies have limited effectiveness for high-risk patients, highlighting the urgent need for novel strategies. This trial enrolled patients with Claudin18.2 positive PDAC who have undergone curative-intent resection, with abnormal CA19-9 after 3 months adjuvant chemotherapy and no evidence of recurrence. From Sep 15, 2023 to April 11, 2025 (data cut-off date), six patients received satri-cel infusion and completed at least 4 weeks of follow-up. With a median follow-up of 6.05 months from infusion, only one patient experienced disease recurrence, while others are still under disease free. The median disease-free survival (DFS) and median overall survival (OS) were not reached (NR). The 9-month DFS rate from surgery was 83.3%. Notably, one patient who has completed 52-week follow-up post infusion is still under follow-up without disease recurrence. Moreover, significant decline in CA19-9 levels post infusion was observed in five (83.3%) patients, with reductions ranging from 51.3% to 96.1%. All patients developed Grade 1 or 2 cytokine release syndrome (CRS) after the first satri-cel infusion. For the second infusion administered in one patient, grade 3 CRS accompanied by hypotension was observed, which was resolved within three days following tocilizumab treatment. All patients experienced gastrointestinal disorders, such as nausea and vomiting, which were all Grade 1 or 2. Only one case of Grade 3 gastritis occurred. No immune effector cell-associated neurotoxicity syndrome (ICANS) was reported. Dr. Zonghai Li, Founder, Chairman of the Board, Chief Executive Officer, and Chief Scientific Officer of CARsgen Therapeutics, said, "We are pleased to see that satri-cel has shown promising preliminary efficacy with a manageable safety profile in the highly challenging setting of pancreatic cancer adjuvant therapy. For patients at high risk of recurrence after surgical resection of pancreatic cancer, there are currently very few effective treatment options. In this trial, the sustained disease-free survival and marked declines in CA19-9 levels suggest that satri-cel, an innovative cellular immunotherapy, may clear minimal residual disease and potentially alter the disease course for these patients. Furthermore, we are actively advancing clinical trials exploring satri-cel for gastric cancer adjuvant therapy and as a sequential treatment following first-line gastric cancer therapy, with the goal of providing better curative opportunities for a broader patient population." About Satri-cel Satri-cel is an autologous CAR T-cell product candidate against the protein Claudin18.2 that has the potential to be the first-in-class globally. Satri-cel targets the treatment of Claudin18.2-positive solid tumors with a primary focus on gastric/gastroesophageal junction adenocarcinoma (G/GEJA) and pancreatic cancer (PC). Initiated trials include investigator-initiated trials (CT041-CG4006, NCT03874897), a confirmatory Phase II clinical trial for advanced G/GEJA in China (CT041-ST-01, NCT04581473), a Phase Ib registrational trial for PC adjuvant therapy in China (CT041-ST-05, NCT05911217), an investigator-initiated trial for satri-cel be used as consolidation treatment following adjuvant therapy in patients with resected G/GEJA (CT041-CG4010, NCT06857786), an investigator-initiated trial for satri-cel as a sequential therapy following first-line treatment in patients with advanced G/GEJA (CT041-CG4011, NCT07179484), and a Phase 1b/2 clinical trial for advanced gastric or pancreatic adenocarcinoma in North America (CT041-ST-02, NCT04404595). The Center for Drug Evaluation (CDE) of National Medical Products Administration (NMPA) of China has accepted the New Drug Application (NDA) for satri-cel for the treatment of Claudin18.2-positive advanced G/GEJA in patients who have failed at least two prior lines of therapy on June 25, 2025. It has been granted Priority Review in May 2025 and Breakthrough Therapy Designation in March 2025 by the CDE. Satri-cel was granted Regenerative Medicine Advanced Therapy designation by U.S. FDA for the treatment of advanced G/GEJA with Claudin18.2-positive tumors in January 2022. Satri-cel received Orphan Drug designation from the U.S. FDA for the treatment of G/GEJA in September 2020. About CARsgen Therapeutics Holdings Limited CARsgen is a biopharmaceutical company focusing on developing innovative CAR T-cell therapies to address the unmet clinical needs including but not limited to hematologic malignancies, solid tumors and autoimmune diseases. CARsgen has established end-to-end capabilities for CAR T-cell research and development covering target discovery, preclinical research, product clinical development, and commercial-scale production. CARsgen has developed novel in-house technologies and a product pipeline with global rights to address challenges faced by existing CAR T-cell therapies. Efforts include improving safety profile, enhancing the efficacy in treating solid tumors, and reducing treatment costs, etc. CARsgen's mission is to be a global biopharmaceutical leader that provides innovative and differentiated cell therapies for patients worldwide and makes cancer and other diseases curable. Forward-looking Statements All statements in this press release that are not historical fact or that do not relate to present facts or current conditions are forward-looking statements. Such forward-looking statements express the Group's current views, projections, beliefs and expectations with respect to future events as of the date of this press release. Such forward-looking statements are based on a number of assumptions and factors beyond the Group's control. As a result, they are subject to significant risks and uncertainties, and actual events or results may differ materially from these forward-looking statements and the forward-looking events discussed in this press release might not occur. Such risks and uncertainties include, but are not limited to, those detailed under the heading "Principal Risks and Uncertainties" in our most recent annual report and interim report and other announcements and reports made available on our corporate website, . No representation or warranty is given as to the achievement or reasonableness of, and no reliance should be placed on, any projections, targets, estimates or forecasts contained in this press release. SOURCE CARsgen Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Cell TherapyClinical ResultPhase 1Orphan DrugPhase 2

19 Oct 2025

·www.prnewswire.com

Case Report | Preliminary Clinical Data of CARsgen's Allogeneic BCMA CAR-T Product CT0596 for the Treatment of Primary Plasma Cell Leukemia

SHANGHAI, Oct. 19, 2025 /PRNewswire/ -- Primary plasma cell leukemia (pPCL) is a rare and highly aggressive plasma cell malignancy, often associated with complex genetic abnormalities. There is currently no standard treatment regimens, and conventional therapies for multiple myeloma are typically used. While targeted agents and autologous hematopoietic stem cell transplantation can slightly extend the overall survival of pPCL patients to 1.5–3 years, relapsed/refractory pPCL after multiple lines of therapy presents a significant clinical challenge due to the extremely limited treatment options and rapid disease progression, making it difficult to re-induce remission with existing therapies. CT0596 is an allogeneic BCMA-targeted CAR-T therapy developed using CARsgen's proprietary THANK-u Plus™ platform. It is currently being evaluated in investigator-initiated trials for plasma cell malignancy. As of the data cutoff date (Oct 17, 2025), two patients with relapsed/refractory pPCL had been enrolled. Details are as follows: pPCL-01: A 62-year-old male with IgG-λ type experienced poor disease control and rapid progression despite previous autologous hematopoietic stem cell transplantation and treatment with triple classes of drugs (proteasome inhibitor, immunomodulatory agent, and CD38 monoclonal antibody). By the screening period, his serum free light chain level had reached 10,374.86 mg/L. The patient underwent two CAR-T cell infusions approximately two months apart: he first received low-dose lymphodepletion and a relatively lower dose of CAR-T cells per protocol, then followed one cycle of bridge therapy. About one month after the failure of bridge therapy, he received a second CAR-T cell infusion. Following this infusion, he developed Grade 2 CRS, Grade 4 cytopenia, and a lung infection, but recovered after supportive treatment including tocilizumab, corticosteroids, autologous stem cell infusion, and anti-infective therapy. CAR-T cells expanded robustly, with a peak copy number (Cmax) of 161,971 copies/μg gDNA, and copy numbers remained at 10³ by Week 8. Efficacy assessments at both Week 4 and Week 8 post-infusion showed stringent complete response (sCR), and bone marrow minimal residual disease (MRD) was negative (< 10⁻⁶) at Week 4. pPCL-02: A 70-year-old male with κ light chain had previously been treated with triple classes of drugs (proteasome inhibitor, immunomodulatory agent, and CD38 monoclonal antibody). The patient developed Grade 1 CRS, Grade 4 neutropenia, and thrombocytopenia following lymphodepletion and CAR-T cell infusion. The CRS and cytopenias were resolved after treatment with tocilizumab and other supportive measures. CAR-T cells expanded robustly, with a Cmax of 151,654 copies/μg gDNA. The patient achieved sCR at the Week 4, Week 8, and Week 12 assessments post-infusion, with bone marrow MRD negative (< 10⁻⁶) at both Week 4 and Week 12. Based on the current findings, CT0596 has exhibited robust and rapid efficacy in heavily pretreated patients with rapidly progressive relapsed/refractory pPCL. Both pPCL patients achieved sCR following CT0596 infusion, with peak CAR-T cell copy numbers exceeding 10⁵ copies/μg gDNA. Aside from expected CAR-T-associated toxicities such as CRS and hematologic adverse events, no significant organ toxicities were observed, indicating a manageable safety profile. These results lend strong support to the continued investigation of CT0596 across a broader spectrum of plasma cell neoplasms. About CT0596 CT0596 is an allogeneic BCMA-targeted CAR-T therapy developed using CARsgen's proprietary THANK-u Plus™ platform. It is currently being evaluated in investigator-initiated trials for relapsed/refractory multiple myeloma (R/R MM) or plasma cell leukemia (PCL). CT0596 demonstrated preliminary favorable tolerability and encouraging efficacy signals. Further investigation is planned in additional plasma cell malignancies and autoimmune diseases mediated by autoreactive plasma cells. The company anticipates submitting an Investigational New Drug (IND) application in the second half of 2025. About CARsgen Therapeutics Holdings Limited CARsgen is a biopharmaceutical company focusing on developing innovative CAR T-cell therapies to address the unmet clinical needs including but not limited to hematologic malignancies, solid tumors and autoimmune diseases. CARsgen has established end-to-end capabilities for CAR T-cell research and development covering target discovery, preclinical research, product clinical development, and commercial-scale production. CARsgen has developed novel in-house technologies and a product pipeline with global rights to address challenges faced by existing CAR T-cell therapies. Efforts include improving safety profile, enhancing the efficacy in treating solid tumors, and reducing treatment costs, etc. CARsgen's mission is to be a global biopharmaceutical leader that provides innovative and differentiated cell therapies for patients worldwide and makes cancer and other diseases curable. Forward-looking Statements All statements in this press release that are not historical fact or that do not relate to present facts or current conditions are forward-looking statements. Such forward-looking statements express the Group's current views, projections, beliefs and expectations with respect to future events as of the date of this press release. Such forward-looking statements are based on a number of assumptions and factors beyond the Group's control. As a result, they are subject to significant risks and uncertainties, and actual events or results may differ materially from these forward-looking statements and the forward-looking events discussed in this press release might not occur. Such risks and uncertainties include, but are not limited to, those detailed under the heading "Principal Risks and Uncertainties" in our most recent annual report and interim report and other announcements and reports made available on our corporate website, . No representation or warranty is given as to the achievement or reasonableness of, and no reliance should be placed on, any projections, targets, estimates or forecasts contained in this press release. SOURCE CARsgen Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Cell TherapyClinical ResultImmunotherapy

100 Deals associated with Tocilizumab

External Link

KEGG	Wiki	ATC	Drug Bank
D02596	Tocilizumab	L04AC07	DB06273

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Interstitial lung disease due to systemic disease	United States	Genentech, Inc.	04 Mar 2021
Still's Disease, Adult-Onset	Japan	Chugai Pharmaceutical Co., Ltd.	22 May 2019
Takayasu Arteritis	Japan	Chugai Pharmaceutical Co., Ltd.	25 Aug 2017
COVID-19	European Union	Roche Registration GmbH	16 Jan 2009
COVID-19	Iceland	Roche Registration GmbH	16 Jan 2009
COVID-19	Liechtenstein	Roche Registration GmbH	16 Jan 2009
COVID-19	Norway	Roche Registration GmbH	16 Jan 2009
Cytokine Release Syndrome	European Union	Roche Registration GmbH	16 Jan 2009
Cytokine Release Syndrome	Iceland	Roche Registration GmbH	16 Jan 2009
Cytokine Release Syndrome	Liechtenstein	Roche Registration GmbH	16 Jan 2009
Cytokine Release Syndrome	Norway	Roche Registration GmbH	16 Jan 2009
Giant Cell Arteritis	European Union	Roche Registration GmbH	16 Jan 2009
Giant Cell Arteritis	Iceland	Roche Registration GmbH	16 Jan 2009
Giant Cell Arteritis	Liechtenstein	Roche Registration GmbH	16 Jan 2009
Giant Cell Arteritis	Norway	Roche Registration GmbH	16 Jan 2009
Juvenile Idiopathic Arthritis	European Union	Roche Registration GmbH	16 Jan 2009
Juvenile Idiopathic Arthritis	Iceland	Roche Registration GmbH	16 Jan 2009
Juvenile Idiopathic Arthritis	Liechtenstein	Roche Registration GmbH	16 Jan 2009
Juvenile Idiopathic Arthritis	Norway	Roche Registration GmbH	16 Jan 2009
Oligoarticular Arthritis	European Union	Roche Registration GmbH	16 Jan 2009

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Hypoxic respiratory failure	Phase 3	United States	Genentech, Inc.	20 Apr 2020
Osteoarthritis	Phase 3	France	Hoffmann-La Roche, Inc.	20 Nov 2015
Pain	Phase 3	France	Hoffmann-La Roche, Inc.	20 Nov 2015
Inflammation	Phase 3	Denmark	Hoffmann-La Roche, Inc.	28 May 2014
Inflammation	Phase 3	Finland	Hoffmann-La Roche, Inc.	28 May 2014
Inflammation	Phase 3	Norway	Hoffmann-La Roche, Inc.	28 May 2014
Inflammation	Phase 3	Sweden	Hoffmann-La Roche, Inc.	28 May 2014
Scleroderma, Systemic	Phase 3	United States	Hoffmann-La Roche, Inc.	01 Mar 2012
Scleroderma, Systemic	Phase 3	Canada	Hoffmann-La Roche, Inc.	01 Mar 2012
Scleroderma, Systemic	Phase 3	France	Hoffmann-La Roche, Inc.	01 Mar 2012

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02767557 (Pubmed \| J Clin Oncol)	Phase 2	Pancreatic Cancer First line growth differentiation factor 15 (GDF15)	147	Gemcitabine/nab-paclitaxel with Tocilizumab	tbussnwckk(jzibxfdegd) = bgzjmqjrbi nrlevbstos (cawjbewjbg, 56.3 - 78.1) View more	Positive	20 Jun 2025
				Gemcitabine/nab-paclitaxel without Tocilizumab	tbussnwckk(jzibxfdegd) = wrhclebywi nrlevbstos (cawjbewjbg, 49.6 - 72.1) View more
EULAR2025	Not Applicable	Giant Cell Arteritis	30	Tocilizumab-biosimilar	auriclzrmk(fmjlxceigp) = No patient developed adverse events. udtyrjogyl (wyrlijesnb )	Positive	11 Jun 2025
EULAR2025	Not Applicable	Takayasu Arteritis	121	Molecular targeted drugs	gsdeonjdzb(jlmmwpdneu) = arupkljwbt wrsfwtpvnx (aushqwgzfh ) View more	Positive	11 Jun 2025
EULAR2025	Not Applicable	Rheumatoid Arthritis	165	Tumour necrosis factor inhibitors (TNFi)	ofuxfqzlnb(kdzujgcfsw) = blcdgasxag adsmqmhkdu (qubklytrwo ) View more	Positive	11 Jun 2025
				Janus kinase inhibitors (JAKi) or IL-6 inhibitors (tocilizumab, TCZ)	ofuxfqzlnb(kdzujgcfsw) = nhnaiezrit adsmqmhkdu (qubklytrwo ) View more
EULAR2025	Not Applicable	Giant Cell Arteritis	60	Tocilizumab	yzvpteomrv(gvjwtbxcip) = The half of the patient experienced an infectious adverse event, including two serious respiratory infections and one complicated diverticulitis. The other common side effects, such as cytopenias and lipid and hepatic abnormalities, were also seen and did not affect treatment continuation zxqaigeomo (ezwjthlrhz ) View more	Positive	11 Jun 2025
EULAR2025 \| ACR2025	Not Applicable	Takayasu Arteritis	330	biologic immunosuppressive (bISs)	ubrjklnfgk(xquqrdbwcj) = nujpqvngho zgetwgncap (grfspyvdpd ) View more	Positive	11 Jun 2025
				conventional immunosuppressive (cISs)	ubrjklnfgk(xquqrdbwcj) = xusbjnjfje zgetwgncap (grfspyvdpd ) View more
JPRN-UMIN000024574 (EULAR2025)	Phase 3	Granulomatosis With Polyangiitis \| Microscopic Polyangiitis	39	Tocilizumab	rtbquideog(dhjpkmiyhc) = srzndectwe vrsnsoqyfk (ijdwhzmgsb ) View more	Non-inferior	11 Jun 2025
				Cyclophosphamide	rtbquideog(dhjpkmiyhc) = jgxrpzhksf vrsnsoqyfk (ijdwhzmgsb ) View more
EULAR2025	Not Applicable	Takayasu Arteritis	5	Tocilizumab 8 mg/kg/monthly alongside GCs and other csDMARDs	bjqexbqxnf(xfywrwibrg) = One patient experienced moderate hepatotoxicity, which resolved after dose adjustment kcdwsnoaoo (wkrberldxr ) View more	Positive	11 Jun 2025
NCT04479358 (COVIDOSE-2, CTgov)	Phase 2	COVID-19	85	Standard of Care (Sub-study A, Tocilizumab-Free Standard of Care)	zkaznzibsz(gfytylzrrf) = bspbtnzndq zxcbmvrvqv (hueajdzgdg, cjmrohhpku - hfvpggrnfa) View more	-	05 Jun 2025
				Tocilizumab (Sub-study A, Tocilizumab 40mg)	zkaznzibsz(gfytylzrrf) = tatwqxrafm zxcbmvrvqv (hueajdzgdg, amsbvsonlq - iikthhxhsl) View more
ASCO2025	Not Applicable	Relapse multiple myeloma BCMA	20	Teclistamab	tlsgtymudk(xydhhijwcp) = CRS occurred in 2 subjects (10%), all grade 1 ifywflbfqo (sxfxizpbri ) View more	Positive	30 May 2025

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