This is an open label, Phase 1b, multiple ascending dose, and dose-expansion study of IDP-023 administered in combination with interleukin-2 (IL-2) and ocrelizumab to evaluate the safety, tolerability, and biologic activity on autoreactive immune cells in patients with refractory progressive multiple sclerosis.

Start Date30 Jun 2026

Sponsor / Collaborator

Indapta Therapeutics, Inc.

NCT06815003

/ Not yet recruitingPhase 2IIT

Phase-2 Study of Vedolizumab Plus Post-Transplant Cyclophosphamide and Short Course Tacrolimus for Graft-versus-Host Disease Prevention After Reduced Intensity Conditioning Peripheral Blood Stem Cell Allogeneic Hematopoietic Cell Transplantation

This phase II trial studies how well vedolizumab plus post-transplant cyclophosphamide (PTCy) and short course tacrolimus work for the prevention of graft versus host disease (GVHD) in patients undergoing allogeneic hematopoietic cell transplantation (HCT) after reduced intensity conditioning. Allogeneic HCT is a procedure in which a person receives blood-forming stem cells (cells from which all blood cells develop) from a donor. Giving reduced conditioning chemotherapy before an allogeneic HCT helps kill cancer cells in the body and helps make room in the patient's bone marrow for new stem cells to grow using less than standard doses of chemotherapy. Sometimes, the transplanted cells from a donor can attack the body's normal cells (called graft-versus-host disease). Vedolizumab is a monoclonal antibody, which is a type of protein that can bind to certain targets in the body, such as molecules that cause the body to make an immune response (antigens). It may reduce inflammation. Cyclophosphamide is in a class of medications called alkylating agents. It works by damaging the cell's deoxyribonucleic acid and may kill cancer cells. It may also lower the body's immune response. Tacrolimus suppresses the immune system by preventing the activation of certain types of immune cells. Giving vedolizumab plus PTCy and short course tacrolimus may be effective at preventing GVHD after allogeneic HCT.

Start Date08 Jan 2026

Sponsor / Collaborator

City of Hope National Medical Center

[+1]

NCT05521087

/ WithdrawnPhase 1

A Phase I/Ib Study of JNJ-75276617 in Combination With Conventional Chemotherapy for Pediatric and Young Adult Participants With Relapsed/Refractory Acute Leukemias Harboring KMT2A, NPM1, or Nucleoporin Gene Alterations

The purpose of this study is to determine the recommended Phase 2 dose(s) (RP2Ds) of JNJ-75276617 in combination with a conventional chemotherapy backbone in pediatric and young adult participants with relapsed/refractory acute leukemia harboring histone-lysine N-methyltransferase 2A1 ([KMT2A1], nucleophosmin 1 gene (NPM1), or nucleoporin alterations in Part 1 (Dose Escalation) and to further evaluate safety at the RP2D(s) of JNJ-75276617 in combination with chemotherapy in pediatric and young adult participants with relapsed/refractory acute leukemia harboring KMT2A1, NPM1, or nucleoporin alterations and safety at the RP2D(s) of JNJ-75276617 as monotherapy in a select low burden of disease cohort in Part 2 (Dose Expansion).

Start Date26 Dec 2025

Sponsor / Collaborator

Janssen Research & Development LLC

100 Clinical Results associated with Fludarabine Phosphate

100 Translational Medicine associated with Fludarabine Phosphate

100 Patents (Medical) associated with Fludarabine Phosphate

8,278

Literatures (Medical) associated with Fludarabine Phosphate

31 Dec 2025·JOURNAL OF MEDICAL ECONOMICS

US consumer and healthcare professional preferences for combination COVID-19 and influenza vaccines

Article

Author: Ghaswalla, Parinaz ; Kent, Cannon ; Poulos, Christine ; Rudin, Deborah ; Mehta, Darshan ; Buck, Philip O.

AIMS:

To quantify preferences for an adult combination vaccine for influenza and COVID-19 (flu + COVID) compared with standalone influenza and COVID-19 vaccines.

MATERIALS AND METHODS:

This survey study used a series of direct-elicitation questions to assess preferences for a single-shot combination flu + COVID, standalone influenza, and standalone COVID-19 vaccines among US consumers (N = 601) and healthcare professionals (HCPs) (N = 299). Response frequencies described the proportion of each sample that would prefer a flu + COVID vaccine to standalone influenza and COVID-19 vaccines. A multivariate logit regression model explored how certain characteristics influenced the odds of selecting the flu + COVID vaccine over a standalone influenza vaccine.

RESULTS:

Most consumers (398/601; 66.2%) and HCPs (250/298; 83.9%) preferred a flu + COVID vaccine to a standalone influenza vaccine. When not forced to choose between flu + COVID and standalone influenza vaccines, most consumers again selected the flu + COVID vaccine (62.3%); 14.7% would prefer separate standalone influenza and COVID-19 vaccines, 8.3% a standalone influenza vaccine only, 7.3% a COVID-19 vaccine only, and 7.4% neither vaccine. Consumers aged ≥50 years with a body mass index ≥40, those aged ≥65 years who previously received a COVID-19 vaccine, and those who had previously experienced severe impacts from influenza were more likely to choose a flu + COVID vaccine over a standalone influenza vaccine than were consumers without these characteristics. HCPs whose practice stocks high-dose influenza vaccines were more likely to choose the flu + COVID vaccine for patients aged ≥65 with no risk factors and patients aged 18-64 with ≥1 risk factor over the standalone influenza vaccine.

LIMITATIONS:

Results are subject to potential hypothetical, responder, selection, and information biases.

CONCLUSIONS:

Most US consumers and HCPs would likely prefer a single-shot combination flu + COVID vaccine compared with standalone influenza and COVID-19 vaccines. Given the low COVID-19 vaccination coverage rates in the US, the availability of a combination flu + COVID vaccine could help increase COVID-19 vaccine coverage.

31 Dec 2025·ANNALS OF MEDICINE

Efficacy and safety of low-dose TBI combined MAC regimen for HSCT in high-risk AML patients with active disease

Article

Author: Chen, Can ; Qian, Shenxian ; Tan, Junfeng ; Shi, Pengfei ; Huang, Xilian ; Gao, Daquan ; Chen, Kuang ; Xu, Ying ; Liu, Lirong ; Xie, Yaping ; Fan, Yang

BACKGROUND:

The management of high-risk acute myeloid leukaemia (AML) remains challenging, highlighting the need for innovative conditioning strategies beyond current regimens.

METHODS:

In the present single-arm study, a FACT regimen comprised of low-dose total body irradiation (TBI) with fludarabine, cytarabine and cyclophosphamide was employed to treat cytogenetically high-risk AML patients exhibiting pre-transplant active disease. This clinical trial is registered in the Chinese Clinical Trial Registry with the registration number ChiCTR2000035111.

RESULTS:

In this study, 21 high-risk AML patients with pre-transplant disease statuses including primary induction failure, relapse and measurable residual disease positivity, were enrolled to undergo FACT conditioning. The FACT group demonstrated a 1-year non-relapse mortality (NRM) rate of 9.5%, indicating a similar level of safety and tolerability among the conditioning regimens. The estimated cumulative incidence of grade 2-4 acute graft-versus-host disease (GVHD) at one year was 30.7%. Additionally, the cumulative incidence of chronic GVHD was 36.0% at one year and increased to 43.0% at two years.

CONCLUSIONS:

The FACT regimen is an effective myeloablative conditioning (MAC) strategy for high-risk AML patients, potentially reducing relapse risk without increasing NRM, warranting further research.

01 Dec 2025·JOURNAL OF CLINICAL IMMUNOLOGY

Early Haploidentical Hematopoietic Stem Cell Transplantation Provides Rapid Leukocyte and Immune Reconstitution in AK2 Patient Identified by TREC Newborn Screening

Article

Author: Meisel, Roland ; Schuster, Friedhelm R ; Hoenig, Manfred ; Boyle, Janel O ; Cicek, Alphan ; Ghosh, Sujal

Abstract:

Reticular dysgenesis (RD) is a rare inborn error of immune cell formation defined by severe combined immunodeficiency, agranulocytosis and sensorineural deafness. We report a case of successful haploidentical maternal hematopoietic stem cell transplantation (HSCT) in a boy with RD detected by TREC newborn screening. The patient was admitted to our hospital at 2 weeks of age and was kept in laminar-air flow / hepa-filtered isolation until HSCT was performed at 8 weeks of age with a busulfan, fludarabine conditioning regime. Except few episodes of acute skin graft-versus-host disease (aGVHD) the peritransplant period was uneventful. The patient was discharged 7 weeks post-HSCT. At 18 months of age cochlear implants were placed. The patient was thriving well, showed full donor chimerism and a T cell count > 1000 TCRab + CD3 + cells/µl after one year. Our case highlights that severely immune-compromised patients with RD benefit from early diagnosis by newborn screening, immediate isolation to prevent infections, and early haploidentical HSCT to overcome neonatal neutropenia and establish protective immunity.

357

News (Medical) associated with Fludarabine Phosphate

13 May 2025

·ir.fatetherapeutics.com

Fate Therapeutics Reports First Quarter 2025 Financial Results and Business Updates

Additional Phase 1 Data for FT819 Off-the-Shelf CAR T-cell Product Candidate in Moderate-to-Severe Systemic Lupus Erythematosus to be Featured in Oral Presentation at EULAR 2025 Congress in June Regenerative Medicine Advanced Therapy Designation Granted by the FDA for FT819 in Moderate-to-Severe Systemic Lupus Erythematosus Expanding FT819 Phase 1 Study to Include Treatment of Multiple Additional B Cell-mediated Autoimmune Diseases and Ex-US Territories $273 Million in Cash, Cash Equivalents, and Investments with Projected Operating Runway through 1H27 SAN DIEGO, May 13, 2025 (GLOBE NEWSWIRE) -- Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to bringing a first-in-class pipeline of induced pluripotent stem cell (iPSC)-derived off-the-shelf cellular immunotherapies to patients, today reported business highlights and financial results for the first quarter ended March 31, 2025. “We continue to be pleased with the initial clinical profile of our FT819 off-the-shelf CAR T-cell program in patients with moderate-to-severe SLE, and we look forward to sharing new clinical data from our ongoing FT819 Phase 1 study at the EULAR Conference in June,” said Bob Valamehr, Ph.D. MBA, President and Chief Executive Officer of Fate Therapeutics. “Our primary focus remains on driving patient enrollment and achieving therapeutic differentiation in SLE, including administration of FT819 with fludarabine-free conditioning and as an add-on to maintenance therapy without conditioning. We plan to work closely with the FDA under our FT819 RMAT designation to align on a registrational pathway for FT819 in SLE, and we have now initiated regulatory submissions in Europe to expand the geographic reach of the program for lupus patients. The expansion of patient population and reach is well supported by the ability to produce FT819 in large quantities from a master cell bank, ensuring consistent on-demand product supply for a large number of patients.” FT819 iPSC-derived 1XX CAR T-cell Program Phase 1 Study Ongoing using Flu-free Conditioning Regimen for SLE. The Company’s ongoing multi-center, Phase 1 clinical trial of FT819 for moderate-to-severe systemic lupus erythematosus (SLE) (NCT06308978) is designed to evaluate the safety, pharmacokinetics, and efficacy of a single dose of FT819 following a fludarabine (flu)-free conditioning regimen, consisting of either bendamustine alone or cyclophosphamide alone. The Company is currently enrolling patients at two dose levels – a single dose of 360 million cells and a single dose of 900 million cells – with the intent of identifying a recommended dose for later-stage development. In addition, the Company is assessing the safety, pharmacokinetics, and anti-B cell activity of a single dose of FT819 at 360 million cells as an add-on to maintenance therapy without conditioning chemotherapy. The Company plans to present new clinical data from its FT819 Phase 1 study during an oral session at the European Alliance of Associations for Rheumatology (EULAR) 2025 Congress in Barcelona, Spain on June 11. RMAT Designation Received from the FDA for SLE. In April 2025, the Company was granted Regenerative Medicine Advanced Therapy (RMAT) designation by the U.S. Food and Drug Administration (FDA) for FT819 to treat moderate-to-severe SLE. The RMAT designation was established under the 21st Century Cures Act to expedite the development and review of regenerative medicine therapies for serious or life-threatening diseases or conditions. The Company’s RMAT application included initial clinical safety and activity data from patients treated with FT819 in its ongoing multi-center, Phase 1 clinical trial. The Company plans to pursue differentiated treatment approaches, including treatment of patients in community centers without hospitalization, and novel registrational strategies with the FDA under its RMAT designation. Expanded Phase 1 Study to Include Multiple Additional B Cell-mediated Autoimmune Diseases. In December 2024, the Company reached agreement with the FDA to allow for clinical investigation of multiple B cell-mediated autoimmune diseases under its current Phase 1 clinical trial of FT819. The Company has submitted an amended clinical protocol to the FDA that enables the conduct of independent dose-expansion cohorts for anti-neutrophilic cytoplasmic antibody-associated vasculitis (AAV), idiopathic inflammatory myositis (IIM), and systemic sclerosis (SSc). The Company plans to initiate dose-expansion cohorts in each of AAV, IIM, and SSc in 2025. FT825 / ONO-8250 iPSC-derived CAR T-cell Program Phase 1 Study Ongoing for Advanced Solid Tumors. Under its collaboration with Ono Pharmaceutical Co., Ltd. (Ono), the Company is conducting a multi-center, Phase 1 study to assess the safety, pharmacokinetics, and activity of FT825 / ONO-8250, a multiplexed-engineered CAR T-cell product candidate targeting human epidermal growth factor receptor 2 (HER2), in patients with advanced solid tumors (NCT06241456). Dose escalation is currently ongoing, with each patient administered conditioning chemotherapy and a single dose of FT825 / ONO-8250 either as monotherapy or in combination with epidermal growth factor receptor (EGFR)-targeted monoclonal antibody therapy. FT825 / ONO-8250 has demonstrated a favorable safety profile with no dose-limiting toxicities (DLTs) to date. Next-generation iPSC-derived CAR T-cell Programs FT836 MICA/B-targeted CAR T-cell Program. FT836 is the Company’s multiplexed-engineered CAR T-cell product candidate uniquely targeting major histocompatibility complex (MHC) proteins A (MICA) and B (MICB). The expression of MICA/B cell-surface proteins is induced by cellular stress or malignant transformation and is detectable across many types of cancer cells with limited expression on healthy tissue. At the American Society of Gene & Cell Therapy (ASGCT) 28th Annual Meeting being held in New Orleans on May 13-17, the Company plans to present preclinical data showing that FT836 exerted potent and durable anti-tumor activity in vivo across a broad array of solid tumors. FT836 is the Company’s first product candidate to incorporate its novel Sword & Shield technology that couples its novel Alloimmune Defense Receptor (ADR) technology with the complete knock-out of CD58 (CD58KO), which is uniquely designed to both target and evade host alloreactive immune cells and to reduce or eliminate the need for administration of conditioning chemotherapy to patients receiving cell therapies. In January 2025, the Company secured a $4 million award from the California Institute of Regenerative Medicine (CIRM) to support IND-enabling activities for FT836. FT839 Dual CAR T-cell Program. FT839 is the Company’s dual CAR T-cell product candidate that incorporates its novel Sword & Shield technology and is designed to express two unique CARs: a first CAR targeting CD19+ B cells, and a second CAR targeting additional disease-causing cells. At the ASGCT Annual Meeting in May, the Company plans to present preclinical data demonstrating iPSC-derived CAR T cells targeting CD19 and the cell-surface glycoprotein CD38 specifically eliminated a variety of malignant cell types, including CD19+ lymphoma and CD38+ multiple myeloma cell lines. In addition, using unmatched peripheral blood mononuclear cells sourced from a patient with SLE, dual CAR T cells showed robust eradication of aberrant CD19+ B cells, CD38+ plasma cells, and CD38+ activated T cells. FT522 iPSC-derived CAR NK Cell Program New Phase 1 Translational Data to be Presented at ASGCT. FT522 is the Company’s off-the-shelf CAR NK cell product candidate and its first to incorporate Alloimmune Defense Receptor (ADR) technology. The FDA has allowed the Company’s Investigational New Drug (IND) application to assess the safety, pharmacokinetics, and activity of FT522 across a basket of B cell-mediated autoimmune diseases. The Company is currently evaluating opportunities and timelines for the clinical development of FT522 in autoimmunity without administration of conditioning chemotherapy. At the ASGCT Annual Meeting in May, the Company plans to present new clinical and translational data from its multi-center, Phase 1 clinical trial of FT522 in patients with relapsed / refractory B-cell lymphoma (BCL) (NCT05950334), where initial translational data demonstrated the potential of FT522 to persist and function in the presence of an unmatched, fully-intact immune system. First Quarter 2025 Financial Results Cash & Investment Position: Cash, cash equivalents, and investments as of March 31, 2025 were $272.7 million. Total Revenue: Revenue was $1.6 million for the first quarter of 2025, which was derived from the conduct of preclinical development activities for a second collaboration candidate targeting an undisclosed solid tumor antigen under the Company’s collaboration with Ono Pharmaceutical. Total Operating Expenses: Total operating expenses were $42.9 million for the first quarter of 2025, including research and development expenses of $29.1 million and general and administrative expenses of $13.8 million. Such amount included $7.4 million of non-cash stock-based compensation expense. Shares Outstanding: As of March 31, 2025, common shares outstanding were 114.6 million, pre-funded warrants outstanding were 3.9 million, and preferred shares outstanding were 2.8 million. Each preferred share is convertible into five common shares. About Fate Therapeutics’ iPSC Product Platform Human induced pluripotent stem cells (iPSCs) possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. The Company’s proprietary iPSC product platform combines multiplexed-engineering of human iPSCs with single-cell selection to create clonal master iPSC lines. Analogous to master cell lines used to mass produce biopharmaceutical drug products such as monoclonal antibodies, the Company utilizes its clonal master iPSC lines as a starting cell source to manufacture engineered cell products which are well-defined and uniform in composition, can be stored in inventory for off-the-shelf availability, can be administered in combination with other therapies, and can potentially reach a broad patient population. As a result, the Company’s platform is uniquely designed to overcome numerous limitations associated with patient- and donor-sourced cell therapies. Fate Therapeutics’ iPSC product platform is supported by an intellectual property portfolio of over 500 issued patents and 500 pending patent applications. About Fate Therapeutics, Inc. Fate Therapeutics is a clinical-stage biopharmaceutical company dedicated to bringing a pipeline of induced pluripotent stem cell (iPSC)-derived cellular immunotherapies to patients. Using its proprietary iPSC product platform, the Company has established a leadership position in creating multiplexed-engineered master iPSC lines and in the manufacture and clinical development of off-the-shelf, iPSC-derived cell products. The Company’s pipeline includes iPSC-derived T-cell and natural killer (NK) cell product candidates, which are selectively designed, incorporate novel synthetic controls of cell function, and are intended to deliver multiple therapeutic mechanisms to patients. Fate Therapeutics is headquartered in San Diego, CA. For more information, please visit www.fatetherapeutics.com. Forward-Looking Statements This release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 including statements regarding the Company’s results of operations, financial condition, anticipated operating expenses and cash runway, and sufficiency of its cash and cash equivalents to fund its operations, as well as statements regarding the advancement of and plans related to the Company's product candidates, clinical studies and preclinical research and development programs, the Company’s progress, plans and timelines for the clinical investigation of its product candidates, including the Company’s plans to complete IND-enabling studies and to submit IND applications and other regulatory submissions, including regulatory submissions in Europe, for its product candidates, the initiation and continuation of enrollment in the Company’s clinical trials, the initiation of additional clinical trials, including in new indications, and additional dose cohorts in ongoing clinical trials of the Company’s product candidates, the availability of data from the Company’s clinical trials and the Company’s plans to provide updates on its clinical trials, the therapeutic and market potential of the Company’s research and development programs and product candidates, the Company’s clinical and product development strategy, the Company’s plans to work with the FDA on a registrational pathway for FT819 in SLE, the Company’s ability to benefit from RMAT designation for FT819, and the Company’s expectations regarding progress and timelines, and the objectives, plans and goals of its collaboration with Ono. These and any other forward-looking statements in this release are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that the Company’s research and development programs and product candidates, including those product candidates in clinical investigation, may not demonstrate the requisite safety, efficacy, or other attributes to warrant further development or to achieve regulatory approval, the risk that results observed in prior studies of the Company’s product candidates, including preclinical studies and clinical trials, will not be observed in ongoing or future studies involving these product candidates, the risk of a delay or difficulties in the manufacturing of the Company’s product candidates or in the initiation and conduct of, or enrollment and continued participation of patients in, any clinical trials, the risk that the Company may cease or delay preclinical or clinical development of any of its product candidates for a variety of reasons (including requirements that may be imposed by regulatory authorities on the initiation or conduct of clinical trials, changes in the therapeutic, regulatory, or competitive landscape for which the Company’s product candidates are being developed, the amount and type of data to be generated or otherwise to support regulatory approval, difficulties or delays in patient enrollment and continuation in the Company’s ongoing and planned clinical trials, difficulties in manufacturing or supplying the Company’s product candidates for clinical testing, failure to demonstrate that a product candidate has the requisite safety, efficacy, or other attributes to warrant further development, and any adverse events or other negative results that may be observed during preclinical or clinical development), the risk that its product candidates may not produce therapeutic benefits or may cause other unanticipated adverse effects, the risk that the Company may not comply with its obligations under and otherwise maintain its collaboration agreement with Ono, the risk that research funding and milestone payments received by the Company under its collaboration may be less than expected, and the risk that the Company may incur operating expenses in amounts greater than anticipated. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company’s actual results to differ from those contained in the forward-looking statements, see the risks and uncertainties detailed in the Company’s periodic filings with the Securities and Exchange Commission, including but not limited to the Company’s most recently filed periodic report, and from time to time in the Company’s press releases and other investor communications. Fate Therapeutics is providing the information in this release as of this date and does not undertake any obligation to update any forward-looking statements contained in this release as a result of new information, future events or otherwise. Contact: Christina Tartaglia Precision AQ 212.362.1200 christina.tartaglia@precisionaq.com Source: Fate Therapeutics, Inc.

Phase 1Cell TherapyImmunotherapyFinancial StatementFast Track

12 May 2025

·www.prnewswire.com

CARsgen Announces Preliminary Clinical Data for Allogeneic BCMA CAR-T CT0596, Demonstrating Favorable Safety and Efficacy

SHANGHAI, May 11, 2025 /PRNewswire/ -- CARsgen Therapeutics Holdings Limited (Stock Code: 2171.HK), a company focused on developing innovative CAR T-cell therapies, announces preliminary clinical data for CT0596, an allogeneic BCMA-targeted CAR-T developed using the THANK-u Plus™ platform. CT0596 is currently being evaluated in an early exploratory clinical study for relapsed/refractory multiple myeloma (R/R MM) or relapsed/refractory plasma cell leukemia (R/R PCL) to assess its safety, and preliminary efficacy. As of May 6, 2025, 8 patients with R/R MM who had received at least three prior lines of therapy were enrolled and infused with CT0596 following lymphodepletion with the FC regimen (fludarabine 22.5-30 mg/m² and cyclophosphamide 350-500 mg/m²). Key findings from up to four months of follow-up include: No dose-limiting toxicities (DLTs), ≥Grade 3 cytokine release syndrome (CRS), immune effector cell-associated neurotoxicity syndrome (ICANS), or graft-versus-host disease (GVHD) were reported. The product demonstrated a favorable safety profile, with no patients discontinuing treatment due to adverse events. 1) Among 5 patients who completed the first efficacy assessment at Week 4, 3 patients (60%) achieved stringent complete response/complete response (sCR/CR), and 4 patients (80%) achieved minimal residual disease (MRD)-negativity in the bone marrow. 2) Early efficacy data from 2 patients at Day 14 showed reductions in measurable lesions by ≥92% and ≥65%, respectively. 3) 1 patient had not yet reached the protocol-specified efficacy assessment timepoint. All sCR/CR patients remained in ongoing responses, including the first patient who completed the four-month follow-up. Based on the preliminary safety and efficacy data, CT0596 demonstrated favorable tolerability and encouraging efficacy signals in R/R MM patients across all predefined dose levels, with CAR-T expansion observed. These findings warrant further exploration not only in R/R MM, but also in other plasma cell malignancies and autoimmune diseases mediated by autoreactive plasma cells. CARsgen plans to present detailed clinical data at upcoming scientific conferences. The company anticipates submitting an Investigational New Drug (IND) application for this product candidate in the second half of 2025. About THANK-u Plus™ CARsgen has developed the THANK-u Plus™ platform as an enhanced version of its proprietary THANK-uCAR® allogeneic CAR-T technology to address the potential impact of NKG2A expression levels on therapeutic efficacy. THANK-u Plus™ demonstrates sustained expansion regardless of varying NKG2A expression levels on NK cells and exhibits significantly improved expansion compared to THANK-uCAR®. Preclinical studies show that THANK-u Plus™ delivers superior antitumor efficacy in the presence of NK cells compared to THANK-uCAR®. Allogeneic BCMA or dual-targeting CD19/CD20 CAR-T cells developed using this platform exhibit robust antitumor activity in the presence of NK cells, indicating that THANK-u Plus™ has broad potential for developing diverse allogeneic CAR-T therapies. CARsgen is developing allogeneic CAR-T products using THANK-u Plus™ platform to increase CAR T cell expansion, persistence and efficacy. 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. Contact CARsgen For more information, please visit 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

Clinical ResultCell TherapyImmunotherapyPhase 1

08 May 2025

·www.globenewswire.com

Artiva Biotherapeutics Reports First Quarter 2025 Financial Results, Recent Business Highlights

IND clearance and initiation of global basket trial exploring AlloNK® + rituximab in refractory rheumatoid arthritis, Sjögren’s disease, idiopathic inflammatory myopathies and systemic sclerosis First trial to explore an allogeneic cell therapy in refractory rheumatoid arthritis and Sjögren’s disease in the U.S. Initial safety, translational data, and lead indication selection to be presented by year-end 2025; initial clinical response data in the lead indication to be presented in 1H2026 Longer-term clinical data from Phase 1/2 trial exploring AlloNK + rituximab in B-NHL in heavily pretreated CAR-T naïve patients to be presented at ASGCT, demonstrating complete response rates and median duration of responses in line with approved auto-CAR-T therapies, support deep B-cell depletion Cash runway extension into Q2 2027, with cash, cash equivalents and investments of $166.0 million as of March 31, 2025 SAN DIEGO, May 08, 2025 (GLOBE NEWSWIRE) -- Artiva Biotherapeutics, Inc. (Nasdaq: ARTV) (Artiva), a clinical-stage biotechnology company whose mission is to develop effective, safe, and accessible cell therapies for patients with devastating autoimmune diseases and cancers, today announced financial results for the first quarter ended March 31, 2025, and highlighted recent progress. “We are encouraged by our early experience with AlloNK® in autoimmune disease. We have initiated regulatory submissions globally for our company-sponsored basket trial in refractory rheumatoid arthritis, Sjögren’s disease, myositis and scleroderma. Notably, we are first to announce a company-sponsored allogeneic cell therapy trial in rheumatoid arthritis and Sjögren’s disease in the U.S.; two sizeable indications that could benefit from AlloNK’s potential ease of use and well-tolerated safety profile in the community setting,” said Fred Aslan, M.D., Chief Executive Officer of Artiva. “Since the IPO last July, we have transformed our development team and now have a deep bench of autoimmune expertise to expand and refine the clinical trial infrastructure and development strategy for AlloNK in autoimmune disease. By the end of 2025, we plan on sharing initial safety and translational data supporting AlloNK’s mechanism of action and tolerability profile across multiple autoimmune diseases, as well as announcing our lead indication. In the first half of 2026, we expect to share clinical response data in our lead indication with longer follow-up to substantiate AlloNK’s emerging target product profile, and to have initial insights on registrational approaches.” Dr. Aslan continued, “Our longer-term aggressive B-NHL data is maturing into a leading data set, in line with approved auto-CAR-T therapies, providing proof of concept of AlloNK’s ability to deplete B-cells deeply and durably. We remain well capitalized to generate meaningful data sets and to differentiate ourselves over time from other therapeutic approaches in areas of significant unmet need in the community setting.” Recent Business Highlights AlloNK® (also known as AB-101) Updates Autoimmune clinical program updates: Company-sponsored clinical trials: Phase 2a company-sponsored basket clinical trial: Following Investigational New Drug application clearance by the U.S. Food and Drug Administration, initiating global Phase 2a company-sponsored basket clinical trial for AlloNK + rituximab for refractory rheumatoid arthritis (RA), Sjögren’s disease, idiopathic inflammatory myopathies (myositis, or IIM) and systemic sclerosis (scleroderma, or SSc), with site initiation underwayFirst company-sponsored trial exploring an allogeneic cell therapy in refractory RA and Sjögren’s disease in the U.S.Protocol allows for continuous enrollment with no stagger within dose levels and no hospitalization/inpatient requirement for patients dosed with AlloNKInitiation of regulatory submissions across multiple geographic regions Phase 1/1b company-sponsored trial in systemic lupus erythematosus (SLE) with or without lupus nephritis (LN): Treatment of patients in combination cohort with AlloNK + monoclonal antibodies (mAb) initiated in late 2024Prioritization of patient enrollment in AlloNK + obinutuzumab cohort Currently exploring 1 billion cells per AlloNK dose x 3 weekly doses in company-sponsored trials with plan to escalate to 4 billion cells per AlloNK dose x 3 weekly doses across both trials Investigator-initiated trial (IIT): Ongoing IIT Basket Trial: IIT assessing the safety, tolerability, and clinical activity of AlloNK + rituximab in patients with RA, pemphigus vulgaris, granulomatosis with polyangiitis/microscopic polyangiitis, and SLE. The trial is being conducted by Integral Rheumatology & Immunology Specialists (IRIS), in an outpatient setting at a community rheumatology clinic. Initial tolerability supports prioritization of community sites for clinical development of AlloNK AlloNK + rituximab in B-cell non-Hodgkin lymphoma (B-NHL) updates: Longer-term clinical data from Phase 1/2 trial exploring AlloNK + rituximab in patients with relapsed/refractory B-NHL to be presented at The American Society of Gene & Cell Therapy (ASGCT) 28th annual meeting on May 13, 2025. Abstracts are currently available on the ASGCT websiteUpdated clinical data in heavily pre-treated, CAR-T naïve patients with B-NHL shows prolonged duration of response, with complete response rates and median duration of response in line with approved auto-CAR-T therapies in aggressive B-NHL patients with multiple prior lines of treatment Other program updates: Preclinical data on the ablation of SLE donor B-cells with AlloNK in combination with either an anti-CD19 or anti-CD20 monoclonal antibody presented at the 2025 Pan-American League of Rheumatology Congress (PANLAR) and to be presented at the 16th International Congress on Systemic Lupus Erythematosus (LUPUS 2025), being held May 21-24, 2025, in Toronto, CanadaPoster presentation featuring the scalability and consistency of the manufacturing process for AlloNK to be presented at the ASGCT 28th annual meeting on May 15, 2025 Data highlights consistent and high expression of CD16, uniform expansion across greater than 40 manufacturing lots, and high viability and cytotoxicity after 4 years of shelf life for AlloNK Corporate Updates Appointment of Subhashis Banerjee, M.D., as Chief Medical Officer announced on April 8, 2025, culminating Artiva’s efforts to build a seasoned development team with strong expertise in autoimmune disease and cell therapy Upcoming Milestones By Year-End 2025: Initial safety and translational data for AlloNK + mAb across multiple autoimmune indications from ongoing clinical trials and disclosure of lead autoimmune indication for further development Mechanistic and translational data for AlloNK in autoimmune indicationsInsights into tolerability of AlloNK + mAb, and the patient journey in community rheumatology sites, including the potential ease of use of conditioning regimens with cyclophosphamide and fludarabineDisclosure of lead indication for AlloNK development in autoimmune diseases 1H 2026: Initial clinical response data in the lead autoimmune indication from ongoing clinical trials with longer follow-up to inform registrational strategy First Quarter 2025 Financial Results Cash, Cash Equivalents and Investments. As of March 31, 2025, Artiva had cash, cash equivalents, and investments of $166.0 million which is expected to fund operations into Q2 2027.Research and Development Expenses. Research and development expenses were $17.1 million for the three months ended March 31, 2025, compared to $11.2 million for the three months ended March 31, 2024.General and Administrative Expenses. General and administrative expenses were $5.1 million for the three months ended March 31, 2025, compared to $3.6 million for the three months ended March 31, 2024.Other Income, net. Other income, net, was $1.9 million for the three months ended March 31, 2025, compared to other income, net, of $0.5 million for the three months ended March 31, 2024.Net Loss. Net loss totaled $20.3 million for the three months ending March 31, 2025, as compared to net loss of $14.0 million for the three months ending March 31, 2024, with non-cash stock-based compensation expense of $2.1 million and $1.4 million for the three months ended March 31, 2025 and 2024, respectively. About Artiva Biotherapeutics Artiva is a clinical-stage biotechnology company whose mission is to develop effective, safe and accessible cell therapies for patients with devastating autoimmune diseases and cancers. Artiva’s lead program, AlloNK® (also known as AB-101), is an allogeneic, off-the-shelf, non-genetically modified, cryopreserved NK cell therapy candidate designed to enhance the antibody-dependent cellular cytotoxicity effect of monoclonal antibodies to drive B-cell depletion. AlloNK is currently being evaluated in three ongoing clinical trials for the treatment of B-cell driven autoimmune diseases. This includes two company-sponsored trials, one in systemic lupus erythematosus for patients with or without lupus nephritis, and a basket trial across autoimmune diseases including rheumatoid arthritis and Sjögren’s disease, as well as an investigator-initiated basket trial in B-cell driven autoimmune diseases. Artiva’s pipeline also includes CAR-NK candidates targeting both solid and hematologic cancers. Artiva was founded in 2019 as a spin out of GC Cell, formerly GC Lab Cell Corporation, a leading healthcare company in the Republic of Korea, pursuant to a strategic partnership granting Artiva exclusive worldwide rights (excluding Asia, Australia and New Zealand) to GC Cell’s NK cell manufacturing technology and programs. Artiva is headquartered in San Diego, California. For more information, please visit www.artivabio.com. Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Statements in this press release that are not statements of historical fact are forward-looking statements. Such forward-looking statements include, without limitation, statements regarding: expectations of Artiva Biotherapeutics, Inc. (the “Company”) regarding the potential benefits, accessibility, ease of use, effectiveness and safety of AlloNK; the Company’s ability to advance AlloNK in autoimmune disease; the Company’s ability to demonstrate progress and clinical validation of its approach; the belief that of Phase 1/2 trial data in B-NHL provides support for B-cell depletion as a MoA and its comparison to approved auto-CAR-T therapies; the Company’s expectations regarding design, timing and availability of data from the Company’s clinical trials or the basket IIT; the timing related to the selection of a lead autoimmune indication; the timing, likelihood or success of the Company's business strategy, as well as plans and objectives of management for future operations; and the Company’s future results of operations and financial position, including cash runway. These forward-looking statements are based on the beliefs of the management of the Company as well as assumptions made by and information currently available to the Company. Such statements reflect the current views of the Company with respect to future events and are subject to known and unknown risks and uncertainties. In light of these risks and uncertainties, the events or circumstances referred to in the forward-looking statements may not occur. These and other factors that may cause the Company’s actual results to differ from current expectations are discussed in the Company’s filings with the Securities and Exchange Commission (the “SEC”), including the section titled “Risk Factors” in the Company’s Quarterly Report on Form 10-Q for the quarter ended March 31, 2025. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date this press release is given. Except as required by law, the Company undertakes no obligation to publicly update any forward-looking statements, whether as a result of new information, future events or otherwise. Artiva Biotherapeutics, Inc.Condensed Balance Sheets(Unaudited)(in thousands) March 31, 2025 December 31, 2024Assets Cash, cash equivalents and investments$165,963 $185,428 Property and equipment, net 7,057 6,370 Operating and financing lease right-of-use assets 13,728 14,055 Other assets 4,515 3,728 Total assets$191,263 $209,581 Liabilities and stockholders' equity Accounts payable and accrued expenses$8,411 $8,513 Operating and financing lease liabilities 13,996 14,354 Other liabilities 73 73 Total liabilities 22,480 22,940 Stockholders' equity 168,783 186,641 Total liabilities and stockholders' equity$191,263 $209,581 Artiva Biotherapeutics, Inc.Condensed Statements of Operation and Comprehensive Loss(Unaudited)(in thousands, except share and per share data) Three Months Ended March 31, 2025 2024License and development support revenue$- $251 Operating expenses: Research and development 17,052 11,156 General and administrative 5,119 3,587 Total operating expenses 22,171 14,743 Loss from operations (22,171) (14,492)Other income, net Interest income 1,864 650 Change in fair value of SAFEs — (268)Other income (expense) (4) 147 Total other income, net 1,860 529 Net loss$(20,311) $(13,963)Net loss per share, basic and diluted$(0.83) $(17.24)Weighted-average common shares outstanding, basic and diluted 24,341,978 809,758 Comprehensive loss: Net loss$(20,311) $(13,963)Other comprehensive income (loss) 129 (101)Comprehensive loss$(20,182) $(14,064) ContactsInvestors: Neha Krishnamohan, Artiva Biotherapeutics, ir@artivabio.comMedia: Jessica Yingling, Ph.D., Little Dog Communications Inc., jessica@litldog.com, +1.858.344.8091 Source: Artiva Biotherapeutics, Inc.

Cell TherapyFinancial StatementLicense out/inPhase 2ASH

100 Deals associated with Fludarabine Phosphate

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KEGG	Wiki	ATC	Drug Bank
D01907	Fludarabine Phosphate	L01BB05	DB01073

R&D Status

Approved

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Indication	Country/Location	Organization	Date
Lymphoma	Japan	Sanofi KK	26 Mar 2019
Multiple Myeloma	Japan	Sanofi KK	30 Jun 2015
Myelodysplastic Syndromes	Japan	Sanofi KK	30 Jun 2015
Philadelphia chromosome positive chronic myelogenous leukemia	Japan	Sanofi KK	30 Jun 2015
Mantle-Cell Lymphoma	Japan	Sanofi KK	06 Nov 2009
Non-Hodgkin Lymphoma	Japan	Sanofi KK	06 Nov 2009
Hematopoietic stem cell transplantation	Japan	Sanofi KK	20 May 2008
B-Cell Lymphoma	Japan	Sanofi KK	26 Jan 2007
Thrombocytopenia	Japan	Sanofi KK	29 Sep 1999
Chronic Lymphocytic Leukemia	United States	Genzyme Corp.	18 Apr 1991

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Pulmonary Disease, Chronic Obstructive	Phase 3	Australia	Novartis Pharmaceuticals Corp.	01 Apr 2012
Pulmonary Disease, Chronic Obstructive	Phase 3	New Zealand	Novartis Pharmaceuticals Corp.	01 Apr 2012
Disorder related to transplantation	Phase 3	United States	Sanofi	02 Nov 2011
Infectious Diseases	Phase 3	United States	Sanofi	02 Nov 2011
Acute Graft Versus Host Disease	Phase 3	United States	Fred Hutchinson Cancer Research Center	01 Nov 2010
Acute Graft Versus Host Disease	Phase 3	Denmark	Fred Hutchinson Cancer Research Center	01 Nov 2010
Acute Graft Versus Host Disease	Phase 3	Germany	Fred Hutchinson Cancer Research Center	01 Nov 2010
Recurrent Multiple Myeloma	Phase 3	United States	Fred Hutchinson Cancer Research Center	01 Nov 2010
Recurrent Multiple Myeloma	Phase 3	Denmark	Fred Hutchinson Cancer Research Center	01 Nov 2010
Recurrent Multiple Myeloma	Phase 3	Germany	Fred Hutchinson Cancer Research Center	01 Nov 2010

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03615105 (CTgov)	Phase 2	Hodgkin's Lymphoma \| Ph-Like Acute Lymphoblastic Leukemia \| Acute Myeloid Leukemia ... View more	9	Hyperfractionated total body irradiation+Cyclophosphamide+Rituximab+Thiotepa (Radiation, Thiotepa & Cyclophosphamide)	wnqvydiojy = rqvbdhyswk jbxcejdfmk (vmlsimfnlz, xzwuaufaqk - aqxxlxahbs) View more	-	06 Apr 2025
				HPC(A) stem cell allograft+Fludarabine+Busulfan+Melphalan+Rituximab (Busulfan, Fludarabine & Melphalan)	wnqvydiojy = wcmfrpyhwv jbxcejdfmk (vmlsimfnlz, etriyqrbib - obujsiwgfq) View more
NCT05108805 (Prospective Clinical Trial of Outpatient Administration of Axicabtagene Ciloleucel, CTgov)	Phase 4	Diffuse Large B-Cell Lymphoma	25	Telemedicine Visit+Axicabtagene Ciloleucel+Fludarabine+Cyclophosphamide	fsknvsjjjo = pzaznvqmkc uqxousbcgz (emmwszeiwg, sbajzvdjwa - vwgcohuofj) View more	-	27 Mar 2025
NCT02566304 (CTgov)	Phase 2	Anemia, Refractory, With Excess of Blasts \| Refractory cytopenia with multilineage dysplasia and ringed sideroblasts \| Relapsing acute myeloid leukemia ... View more	35	Peripheral Blood Stem Cell Transplantation+Fludarabine+Cyclophosphamide+Tacrolimus+Mycophenolate mofetil	kvynwiqxit = rnyweqzwhp kwbfqwygvx (sqrfrkbhwk, nceirzeocf - bsfwfzdpoh) View more	-	20 Mar 2025
NCT04191187 (Phase II Trial of Reduced-Intensity Fludarabine, Melphalan 70 Mg/m 2 , and Total Body Irradiation Conditioning \| 4900, CTgov)	Phase 2	Marginal zone lymphoma recurrent \| Acute Myeloid Leukemia \| Recurrent Follicular Lymphoma ... View more	34	Total Body Irradiation+Fludarabine+Melphalan	vqdwuxvpos = ifecevbful ozzjxenzlz (veaybklewt, lrgcwpquaw - ugjqdlikqn) View more	-	07 Mar 2025
NCT04432506 (CTgov)	Phase 2	Primary mediastinal large B-cell lymphoma refractory \| Refractory High-Grade B-Cell Lymphoma \| Primary mediastinal large B-cell lymphoma recurrent ... View more	22	Anakinra (Cohort 1)	pghhplcxde = ujualhqbkd dqdutzadoi (fsutwqrzfp, kwrldpmamj - jiegtordon) View more	-	28 Jan 2025
				Anakinra (Cohort 2)	pghhplcxde = atpjspsrkr dqdutzadoi (fsutwqrzfp, mfchqueqgc - iyjmivvtaw) View more
NCT04339101 (1043, ASH2024)	Phase 2	Myelofibrosis \| acute leukemia \| Myelodysplastic Syndromes HGF \| IL17 \| TNFaR ... View more	59	Itacitinib + Tacrolimus/Sirolimus	qzvlbmineu(eskuqefdkl) = vqchhjazuq ypwvevfyeq (ycberjsafy, 41 - 66) View more	Positive	09 Dec 2024
				Tacrolimus/Sirolimus	qzvlbmineu(eskuqefdkl) = cflejjjskn ypwvevfyeq (ycberjsafy )
ASH2024	Not Applicable	Hematopoietic stem cell transplantation	-	Fludarabine 30mg/m2 + Thiotepa 5mg/kg	rhtssmblmw(cdhkmkjtjv) = rsosswtukg okqxjurufx (lghuskcboy ) View more	-	09 Dec 2024
				Fludarabine 30mg/m2 + Busulfan 3.2mg/kg	wniylubuuk(egglybetya) = hwchwntwcc jdnmbknuia (pfunmyfwdf ) View more
NCT05322733 (cwCLL-001, ASH2024) Manual	Phase 2	Chronic Lymphocytic Leukemia First line	25	Orelabrutinib+Fludarabine+Cyclophosphamide+Obinutuzumab	ydpajpbini(xdsrmocxmx) = fiugervbwk ayfutsndeo (hnsefujcyb ) View more	Positive	08 Dec 2024
NCT04708054 (ASH2024)	Phase 2	Acute Myeloid Leukemia \| Myelodysplastic Syndromes	-	Busulfan, Fludarabine, Cladribine, Thiotepa and Venetoclax (Cladillac) Conditioning Regimen	kwzlycvamc(exdofuxsib) = xrbjxvqono xznqeflrhg (vmldnxzexj, 23 - 50) View more	-	08 Dec 2024
ASH2024	Not Applicable	Peripheral Stem Cell Transplantation	-	3-day fludarabine	rfbkyeqqtp(fgoaahclfb) = jfkdrmysej yojmlrvybu (eyhcaralne ) View more	-	08 Dec 2024
				5-day fludarabine	rfbkyeqqtp(fgoaahclfb) = faqvhfsxwh yojmlrvybu (eyhcaralne ) View more

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