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.

NCT06996119

/ Not yet recruitingPhase 1IIT

Pilot Study of Emapalumab With Post-Transplant Cyclophosphamide as Graft-Versus-Host Disease Prophylaxis for Reduced-Intensity Allogeneic Hematopoietic Cell Transplantation

This phase I trial tests the safety, side effects and effectiveness of emapalumab with post-transplant cyclophosphamide, tacrolimus, and mycophenolate mofetil in preventing graft-versus-host disease (GVHD) in patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) after reduced-intensity donor (allogeneic) hematopoietic cell transplant (HCT). Giving chemotherapy, such as fludarabine, melphalan, or busulfan, before a donor [peripheral blood stem cell] transplant helps kill cancer cells in the body and helps make room in the patient's bone marrow for new blood-forming cells (stem cells) to grow. When healthy stem cells for a donor are infused into a patient (allogeneic HCT), they may help the patient's bone marrow make more healthy cells and platelets. Allogeneic HCT is an established treatment, however, GVHD continues to be a major problem of allogeneic HCT that can complicate therapy. GVHD is a disease caused when cells from a donated stem cell graft attack the normal tissue of the transplant patient. Emapalumab binds to an immune system protein called interferon gamma. This may help lower the body's immune response and 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 is a drug used to help reduce the risk of rejection by the body of organ and bone marrow transplants. Mycophenolate mofetil is a drug used to prevent GVHD after organ transplants. It is also being studied in the prevention of GVHD after stem cell transplants for cancer, and in the treatment of some autoimmune disorders. Mycophenolate mofetil is a type of immunosuppressive agent. Giving emapalumab with post-transplant cyclophosphamide, tacrolimus and mycophenolate mofetil may be safe, tolerable and/or effective in preventing GVHD in patients with AML or MDS after a reduced-intensity allogeneic HCT.

Start Date25 May 2026

Sponsor / Collaborator

City of Hope National Medical Center

[+1]

NCT07164469

/ Not yet recruitingPhase 2IIT

Phase 2 Trial of CD70.CAR NK Cells for Patients With Primary Refractory or Early Relapsed Diffuse Large B-Cell Lymphoma and Hodgkin Lymphoma

This clinical research study is to learn if CD70.CAR NK cell therapy can help to control early relapsed or primary refractory DLBCL and cHL.

Start Date28 Feb 2026

Sponsor / Collaborator

The University of Texas MD Anderson Cancer Center

[+1]

100 Clinical Results associated with Fludarabine Phosphate

100 Translational Medicine associated with Fludarabine Phosphate

100 Patents (Medical) associated with Fludarabine Phosphate

7,908

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·OncoImmunology

Lymphodepletion, tumor-infiltrating lymphocytes, and high versus low dose IL-2 followed by pembrolizumab in patients with metastatic melanoma

Article

Author: Patel, Sapna P. ; Amaria, Rodabe N. ; McQuade, Jennifer L. ; Bernatchez, Chantale ; Forget, Marie-Andrée ; Gershenwald, Jeffrey E. ; Haymaker, Cara ; Ross, Merrick I. ; Hasanov, Merve ; Hwu, Patrick ; Glitza, Isabella C. ; Diab, Adi ; Tawbi, Hussein A. ; Wargo, Jennifer A. ; Lee, Jeffrey E. ; Bassett, Roland ; Davies, Michael A. ; Wong, Michael K. ; Kiany, Simin ; Lucci, Anthony

This study evaluated the efficacy and safety of unengineered tumor-infiltrating lymphocytes (TILs) combined with pembrolizumab and either high (HD, Arm-1) or low (LD, Arm-2) doses of IL-2 in patients with metastatic melanoma (MM). Patients were lymphodepleted with cyclophosphamide and fludarabine, followed by TIL infusion and IL-2 (Arm-1: 720,000 IU/kg IV q 8 hrs up to 15 doses; Arm-2: 2 million IU SC for 14 days). Patients received pembrolizumab 200 mg IV starting 21 days post-TIL infusion, and every 3 weeks for up to 2 years. The primary endpoint was overall response rate (ORR) per RECIST 1.1. Blood samples were collected for longitudinal flow cytometry and cytokine analysis. In Arm-1 (n = 7), one patient had a partial response (PR) for 10 months, two had stable disease (SD), three had progressive disease (PD), and one was not evaluable (NE). In Arm-2 (n = 7), one patient had an ongoing PR for over 76 months, one had SD, and five had PD. The toxicity profiles were comparable; however, patients in Arm-2 had lower grade 3 febrile neutropenia (57% vs. 71%) and shorter hospitalization (median 16 days vs. 18 days). No correlation was observed between TIL phenotype and clinical response, although PR patients received high numbers of TIL with a high CD8⁺/CD4⁺ T cell ratio. IL-2 dose did not affect the frequency, phenotype, or proliferation of circulating T cell subsets, and anti-PD-1 did not boost T-cell proliferation. No significant differences were observed between IL-2 doses, suggesting low-dose IL-2 as an alternative to high-dose IL-2 after TIL administration.

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

380

News (Medical) associated with Fludarabine Phosphate

30 Oct 2025

·www.bioheng.com

Imviva Biotech Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for CTD402 in Relapsed/Refractory T-ALL/LBL

The announcement follows the recent IND clearance for CTD402 to be used in Imviva Bio’s Phase 1b/2 trial CTD402 is a CD7 Allogeneic CAR T-cell therapy designed for use in patients with T-cell Acute Lymphoblastic Leukemia (T-ALL) and T-cell Lymphoblastic Lymphoma (T-LBL) October 28, 2025 – Imviva Biotech, a clinical-stage biotechnology company developing next-generation allogeneic CAR-T cell therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to CTD402 for the treatment of relapsed or refractory T-cell acute lymphoblastic leukemia (T-ALL). T-cell acute lymphoblastic leukemia represents a particularly challenging subset of ALL with a paucity of therapeutic option and poor outcomes in the relapsed/refractory setting. CTD402 is an off-the-shelf allogeneic CAR-T cell therapy designed to target CD7 while addressing key challenges in T-cell malignancies. RMAT designation is granted to regenerative medicine therapies that demonstrate the potential to address unmet medical needs for serious or life-threatening conditions. The designation is intended to facilitate efficient development and expedite review of therapies through early interactions with the FDA. "We are pleased to receive RMAT designation for CTD402. This designation underscores the significant unmet need in relapsed/refractory T-ALL and the potential of our allogeneic CAR-T platform to address this challenging malignancy," Jan Davidson-Moncada, MD, PhD, CMO of Imviva Biotech. "RMAT designation enables more efficient collaboration with the FDA as we advance CTD402 through clinical development, bringing Imviva closer to delivering this potentially transformative therapy to patients who have limited treatment options." Robust Clinical Data Supporting RMAT Designation The RMAT designation is supported by compelling clinical data from ongoing Phase I/II trials involving approximately 100 patients. At the recommended Phase 2 dose (RP2D) of 400 million cells, CTD402 demonstrated: 64.1% (76% CR/ 24% CRi) complete remission rate (CRR) in 41 heavily pretreated R/R T-ALL/LBL patients 91.7% MRD-negative rate among responders, indicating deep remissions Durable responses with median duration of response of 9.7 months (95% CI: 5.0 - not reached) Efficacy in high-risk populations: 60.9% CRR in patients with high-risk molecular profiles and 59% CRR in patients with extramedullary disease Favorable safety profile: predominantly mild-to-moderate cytokine release syndrome (78% of patients experienced CRS, with 66% Grade 1-2), with no cases of immune effector cell-associated neurotoxicity syndrome (ICANS) or graft-versus-host disease The patient population treated represented a particularly challenging cohort: 37% were primary induction failures, 24% had relapsed post-allogeneic stem cell transplant, and 56% had high-risk molecular profiles. These results far exceed current standard of care outcomes in this difficult-to-treat population. "T-ALL/LBL in the relapsed or refractory setting has historically been associated with very poor outcomes and limited treatment options," said Jan, “CTD402's high rates of MRD-negative complete remissions in heavily pretreated patients, including those with high-risk disease features, represents a meaningful therapeutic advance in this disease. Moreover, CTD402 armed with the company’s proprietary ANSWERTM technology is administered following standard lymphodepleting chemotherapy (fludarabine 30 mg/m²/day and cyclophosphamide 500 mg/m²/day for 3 days), in contrast to many allogeneic CAR-T approaches that require intensive or augmented conditioning regimens. This standard dosing regimen contributes to CTD402's favorable safety profile while maintaining robust therapeutic efficacy, offering a safer treatment option for heavily pretreated patients who may be unable to tolerate more intensive lymphodepletion protocols.” About CTD402 CTD402 is an investigational ‘off-the-shelf’ allogeneic anti-CD7 CAR-T cell therapy designed for T-cell hematologic malignancies. The product incorporates T cell receptor (TCR) and HLA class II knockout, along with Imviva's proprietary ANSWER™ inhibitory ligands to enhance resistance to host immune rejection. The robustness of CTD402’s manufacturing process, showing product consistency across multiple donors and production lots, promises to deliver an 'off-the-shelf' allogeneic platform with the critical advantage of immediate availability, eliminating manufacturing delays that can be life-threatening for patients with rapidly progressive disease. A global Phase 1b/2 clinical trial is currently open and enrolling patients (NCT07070219). About RMAT Designation The RMAT designation program was established under the 21st Century Cures Act to facilitate efficient development and expedite review of regenerative medicine therapies for serious or life-threatening conditions. Benefits of RMAT designation include: Early interactions with FDA to discuss development plans and clinical trial design Potential eligibility for priority review and accelerated approval if relevant criteria are met Opportunity for rolling review of marketing applications About T-ALL/LBL T-cell acute lymphoblastic leukemia and lymphoblastic lymphoma are aggressive hematologic malignancies with particularly poor outcomes in the relapsed/refractory setting. Current treatment options are limited, and patients who fail initial therapy or relapse after stem cell transplantation face dismal prognoses with median survival measured in months. The development of effective targeted therapies for R/R T-ALL/LBL represents a critical unmet medical need. About Imviva Biotech Imviva Biotech is a clinical-stage biotechnology company dedicated to developing innovative allogeneic CAR-T cell therapies for patients with cancer and autoimmune diseases. The company's proprietary platform incorporates advanced cell engineering technologies to create off-the-shelf cellular immunotherapies. Imviva’s pipeline includes programs in both oncology and autoimmune indications. Forward-Looking Statements This press release contains forward-looking statements regarding the development and potential of CTD402. These statements involve risks and uncertainties, and actual results may differ materially from those expressed or implied. Contact: Investor relations: InvestorRelations@Imvivabio.com Media contact: Media@Imvivabio.com

Cell TherapyImmunotherapyClinical ResultINDFast Track

30 Oct 2025

·www.bioheng.com

Imviva Biotech Presents Latest Clinical Data on Allogeneic CAR-T Therapy CTA313 for Patients with Autoimmune Diseases at ACR Convergence 2025

Strong CAR-T persistence: CTA313 achieved >67% Day-28 persistence, exceeding typical allo-CAR-T benchmarks and supports the durability of ImvivaBio’s immune-evasion design. Deep and durable clinical responses: Among 14 evaluable patients with systemic lupus erythematosus (SLE) or lupus nephritis (LN), SRI-4 100%, LLDAS 79%, and DORIS 43% remission were observed at a median 4-month follow-up. All cytokine release syndrome (CRS) cases were all Grade 1 (40%), with no ICANS or GvHD reported. Expanding access through an allogeneic platform: CTA313 is a ready-at-point-of-care therapy that removes the barriers of autologous CAR-T manufacturing, offering a scalable solution that enables global clinical trials and expands access to patients who otherwise cannot receive autologous treatment. October 29, 2025 – Imviva Biotech, a clinical-stage biotechnology company developing next-generation allogeneic CAR-T cell therapies, today announced the presentation of data from an investigator-initiated trial (IIT) evaluating CTA313 in patients with active systemic lupus erythematosus (SLE) and lupus nephritis (LN). "The clinical data presented at ACR reinforce the potential of CTA313 as an 'off-the-shelf' therapy to deliver durable, drug-free clinical responses with a single infusion across multiple autoimmune diseases," said Jan Davidson-Moncada, MD, PhD, CMO of Imviva Biotech. "The positive clinical outcomes in SLE and LN patients, including early signs of immunological reset observed in the study, enhance Imviva Biotech's leadership credentials in allogeneic cell therapies. This progress demonstrates our potential to bring groundbreaking treatment solutions to autoimmune disease patients worldwide." CTA313 Data This single-arm, open-label, dose-escalation study evaluated the safety and efficacy of CTA313 in patients with SLE/LN. Three dose levels were explored (DL1: 3×106 CAR+ T cells/kg; DL2: 6×106 CAR+ T cells/kg; DL3: 10×106 CAR+ T cells/kg). All patients received lymphodepletion similar to that used for autologous CAR T therapy, consisting of fludarabine (30 mg/m2/day × 3 days) and cyclophosphamide (300 mg/m2/day × 3 days) prior to CTA313 infusion. As of September 20, 2025, 15 patients had been enrolled, including 40% with active or refractory systemic lupus erythematosus (SLE) and 60% with lupus nephritis (LN). The highest SLEDAI-2K score was 23, and 40% of patients had previously been treated with at least one biologic agent. Key highlights are outlined below: Safety: CTA313 was well-tolerated. Cytokine Release Syndrome (CRS) was observed in only 40% of patients, all of which were Grade 1. Only two patients required tocilizumab treatment. No cases of Immune Effector Cell-associated Neurotoxicity Syndrome (ICANS) or Graft-versus-Host Disease (GvHD) were observed at any dose level. Infections were manageable and fully resolved with antimicrobial therapy. Efficacy & Cellular Kinetics: With a median follow-up of 4 months, the 14 evaluable patients showed rapid and durable disease control. 100% of patients (14/14) achieved an SRI-4 response. 79% of patients (11/14) achieved Lupus Low Disease Activity State (LLDAS). 43% of patients (6/14) achieved Definitions of Remission in SLE (DORIS). Robust in vivo expansion of CAR T cells was observed, with persistence at 28 days in 67% of patients and lasting up to 90 days after infusion. Data also showed significant and sustained seroconversion, with marked decreases or normalization of anti-dsDNA antibodies and complement C3 levels. Reconstituted B cells displayed a naïve B-cell phenotype, accompanied by a marked reduction in memory B cells and plasmablasts , providing early evidence of a potential "immune reset". "CTA313 demonstrates rapid and sustained disease control in patients with difficult-to-treat systemic lupus erythematosus and lupus nephritis,” said Jan, "The mild CRS profile is especially promising - potentially enabling future outpatient treatment. These findings strongly support expanded development in SLE and broader autoimmune indications." Ongoing clinical studies are further evaluating CTA313 in patients with other B cell-driven autoimmune diseases, global study is planned for early 2026. Presentation Details Title: Preliminary Safety, Efficacy, and Cellular Kinetics of CTA313, a CD19/BCMA Dual-Targeted Universal CAR-T Therapy, for Active Systemic Lupus Erythematosus Session: Systemic Lupus Erythematosus – Treatment Poster II Date and Time: Monday, October 27, 10:30 AM - 12:30 PM CT About CTA313 CTA313 is an allogeneic, CD19/BCMA dual-targeting allogeneic CAR T-cell product derived from healthy donors for the treatment of refractory, active autoimmune diseases. CTA313 is developed based on Imviva Biotech’s proprietary allogeneic CAR-T platform—ANSWER®—which incorporates genetic modifications to prevent graft-versus-host disease (GvHD) and host-versus-graft rejection (HvG). These products can be manufactured in advance and stored for multiple patients, providing an "off-the-shelf" solution for patients in need of CAR-T cell therapy. Leveraging the ANSWER® platform, Imviva Biotech is actively developing a broad pipeline of allogeneic CAR-T programs. These programs have demonstrated robust and durable persistence of allogeneic CAR-T cells, highlighting strong therapeutic potential in both autoimmune diseases and oncology. About Imviva Biotech Imviva Biotech is a clinical-stage biotechnology company dedicated to developing innovative allogeneic CAR-T cell therapies for patients with cancer and autoimmune diseases. The company's proprietary platform incorporates advanced cell engineering technologies to create off-the-shelf cellular immunotherapies. Imviva’s pipeline includes programs in both oncology and autoimmune indications. Forward-Looking Statements This press release contains forward-looking statements regarding the development and potential of CTA313. These statements involve risks and uncertainties, and actual results may differ materially from those expressed or implied. Contact: Investor relations: InvestorRelations@Imvivabio.com Media contact: Media@Imvivabio.com

ImmunotherapyClinical ResultCell TherapyClinical StudyAACR

28 Oct 2025

·www.bioheng.com

ACR 2025 | Bioheng Therapeutics Presents Latest Clinical Data on Allogeneic CAR-T Therapy CTA311 and CTA313 for Patients with Autoimmune Diseases

From October 24 to 29, 2025, the American College of Rheumatology (ACR) Convergence 2025 was grandly held in Chicago, USA. At this event, Bioheng Therapeutics presented the latest clinical results of CTA311, its allogeneic anti-CD19 CAR-T product for systemic lupus erythematosus (SLE) and ANCA-associated vasculitis (AAV), and CTA313, its allogeneic anti-CD19/BCMA CAR-T product for SLE and lupus nephritis (LN), in the form of academic posters. Abstract Title: Preliminary Safety, Efficacy, and Cellular Kinetics of CTA311, a CD19 Targeted Universal CAR-T Therapy, for Active Autoimmune Diseases Abstract Code: 2129320 Poster Code: 2452 Session: Systemic Lupus Erythematosus – Treatment Poster III Presentation Time: Tuesday, October 28, 10:30 AM - 12:30 PM CT The poster at ACR Convergence 2025 detailed the results of an investigator-initiated trial. This single-arm, open-label, dose-escalation study evaluated the safety and efficacy of CTA311 in patients with SLE and AAV. Four dose levels were explored (DL1: 0.2×106 CAR+ T cells/kg; DL2: 1.0×106 CAR+ T cells/kg; DL3: 3.0×106 CAR+ T cells/kg; DL4: 6.0×106 CAR+ T cells/kg). All patients received lymphodepletion with fludarabine (30 mg/m2/day × 3 days) and cyclophosphamide (300 mg/m2/day × 3 days) prior to CTA311 infusion. As of August 30, 2025, a total of 11 patients were enrolled, including 10 with SLE and 1 with AAV.Ten patients (91%) were female, with a median age of 31 years (range: 21-55), and 64% patients previously received biologics including belimumab and telitacicept. Safety: CTA311 was well tolerated with no dose-limiting toxicities (DLT), neurotoxicity or graft-versus-host disease (GvHD) events reported. Cytokine release syndrome (CRS) events occurred in 82% (n=9), all low grade (G) (7 G1 and 2 G2). Three patients experienced treatment related infections (1 pneumonia (G3) and CMV reactivation (G1), 1 fungal infection (G2), and 1 infection NOS (G1)), which all fully resolved with antimicrobial treatment. Efficacy and Cellular Kinetics: In SLE/LN, preliminary analysis indicates elimination of autoantibodies, improvement in C3 levels, and decline in proteinuria leading to improvement in disease activity scores SLEDAI-2K and PGA. Responses were dose dependent with 100% LLDAS and 50% DORIS at DL4. In AAV, the patient achieved a BVAS score of 0 at M3 and M4 from baseline 8. A minor symptom (arthritis) reappeared at M6 but resolved at M9 without any adjustment in medication. PK analysis revealed peak expansion of CAR-T cells approximately 7-21 days post-infusion, with a dose-dependent pattern, suggesting the potential for deeper remission at higher dose levels. Pharmacodynamics: Complete B cell depletion was observed by Day 4 post-infusion with recovery starting Day 42. In patients that achieved anti-dsDNA antibodies eradication, the anti-dsDNA antibodies remained undetected up to 12 months, and ongoing, despite B-cell recovery. CTA311 exhibited an acceptable safety profile in patients with SLE, LN and AAV, and displayed robust expansion coincided with profound B-cell depletion and concomitant elimination of pathogenic autoantibodies, leading to marked clinical improvement in these patients. Future clinical studies are warranted to further evaluate and characterize the efficacy and safety of CTA311 in patients with SLE or other B cell-driven autoimmune diseases. Abstract Title: Preliminary Safety, Efficacy, and Cellular Kinetics of CTA313, a CD19/BCMA Dual-Targeted Universal CAR-T Therapy, for Active Systemic Lupus Erythematosus Abstract Code: 2129706 Poster Code: 1532 Session: Systemic Lupus Erythematosus – Treatment Poster II Presentation Time: Monday, October 27, 10:30 AM - 12:30 PM CT The poster at ACR Convergence 2025 detailed the results of an investigator-initiated trial. This single-arm, open-label, dose-escalation study evaluated the safety and efficacy of CTA313 in patients with SLE/LN. Three dose levels were explored (DL1: 3×106 CAR+ T cells/kg; DL2: 6×106 CAR+ T cells/kg; DL3: 10×106 CAR+ T cells/kg). All patients received lymphodepletion with fludarabine (30 mg/m2/day × 3 days) and cyclophosphamide (300 mg/m2/day × 3 days) prior to CTA313 infusion. As of September 20, 2025, a total of 15 patients were enrolled, including 6 (40%) with SLE and 9 (60%) with LN, with a median age of 37 years (range: 21-50). In terms of safety, no immune effector cell-associated neurotoxicity syndrome (ICANS) or graft-versus-host disease (GvHD) was observed at any dose level. Only Grade 1 Cytokine release syndrome (CRS) was observed in 6 patients (40%), largely self-limiting. All Grade 3 infections fully resolved with antimicrobial treatment. Regarding efficacy, with a median follow-up of 4 months, 100% of patients achieved the Systemic Lupus Erythematosus Responder Index-4 (SRI-4), 79% achieved Lupus Low Disease Activity State (LLDAS), and 43% achieved Definitions of Remission in SLE (DORIS). Robust in vivo expansion of CAR-T cells was observed, with 28-day persistence in 67% of patients. A single infusion of CAR-T cells induced complete B-cell depletion, sustained undectable anti-dsDNA antibodies, and notable reductions in other autoantibodies, including anti-Sm, anti-histone, and anti-nucleosome antibodies. Preliminary results indicate that CTA313 can rapidly and durably control disease activity in patients with SLE and LN, with an acceptable safety profile. Ongoing clinical studies are further evaluating CTA313 in patients with other B cell-driven autoimmune diseases. About CTA311 and CTA313 Both CTA311 and CTA313 are allogeneic CAR-T products derived from healthy donors. CTA311 targets CD19, whereas CTA313 targets both CD19 and BCMA. Both products are developed based on Bioheng Therapeutics’ proprietary allogeneic CAR-T platform—ANSWER®—which incorporates genetic modifications to prevent graft-versus-host disease (GvHD) and host-versus-graft rejection (HvG). These products can be manufactured in advance and stored for multiple patients, providing an "off-the-shelf" solution for patients in need of CAR-T cell therapy. Leveraging the ANSWER® platform, Bioheng Therapeutics is actively developing a broad pipeline of allogeneic CAR-T programs. These programs have demonstrated robust and durable persistence of allogeneic CAR-T cells, highlighting strong therapeutic potential in both autoimmune diseases and oncology.

Clinical ResultImmunotherapyCell TherapyAACRClinical Study

100 Deals associated with Fludarabine Phosphate

External Link

KEGG	Wiki	ATC	Drug Bank
D01907	Fludarabine Phosphate	L01BB05	DB01073

R&D Status

Approved

10 top approved records.

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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.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
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
Small Lymphocytic Lymphoma	Phase 3	United States	Fred Hutchinson Cancer Research Center	01 Nov 2010
Small Lymphocytic Lymphoma	Phase 3	Denmark	Fred Hutchinson Cancer Research Center	01 Nov 2010

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03263936 (CTgov)	Phase 1	Refractory acute myeloid leukemia	37	Decitabine (Dose Level 1)	qlmbbaylya = zkatcqlmqg qfszgoieyk (pqbkegxefk, avakwbqqiy - arnbbxjupl) View more	-	16 Oct 2025
				Decitabine (Dose Level 2)	qlmbbaylya = rhaqprztaf qfszgoieyk (pqbkegxefk, qjfifskufb - zyoyyebyqv) View more
NCT05579769 (CTgov)	Phase 2	Myeloid Tumor \| Graft vs Host Disease	3	Cyclophosphamide (Cy) (Total Body Irradiation (TBI)/Cyclophosphamide (Cy))	hrienmiejq = zkgibjfyhe gztlddewqp (sibxknyzdb, txhbavmmlt - xhbxrnpwzd) View more	-	02 Oct 2025
				Fludarabine (TBF)+Busulfan+Thiotepa (Thiotepa, Busulfan, and Fludarabine (TBF))	hrienmiejq = ynnzrdnfyq gztlddewqp (sibxknyzdb, cyswcahpdr - hvgdtmgqdo) View more
NCT00920972 \| NCT03128996 (Pubmed \| Transplant Cell Ther)	Phase 2	Refractory Severe Aplastic Anemia	22	Alemtuzumab+fludarabine+melphalan±thiotepa (MSD/unrelated/haploidentical)	efveduomzg(ivnebrptga) = Complications before day +180 were primarily transient viral reactivations. Thyroid and ovarian hypofunction were the commonest effects noted at long-term follow-up. zmypguyeiw (bchotdhqra )	Positive	01 Sep 2025
NCT04904185 (CTgov)	Phase 1	Metastatic melanoma	8	Multiple Antigen Specific Endogenously derived T cells+Fludarabine Phosphate+Cyclophosphamide (Part A)	msphtilqwy = ayfipszjsg lkidagzblx (jvbfutdvpy, ffscrylxut - foftyjqvla) View more	-	25 Jul 2025
				Multiple Antigen Specific Endogenously derived T cells+Fludarabine Phosphate+Cyclophosphamide+Pembrolizumab (Part B)	msphtilqwy = zexafwsiyt lkidagzblx (jvbfutdvpy, hrtahnspca - ueriowsfmk) View more
NCT02722668 (CTgov)	Phase 2	Relapse multiple myeloma \| Chronic Lymphocytic Leukemia \| Acute Lymphoblastic Leukemia ... View more	15	TBI+Fludarabine+Cyclophosphamide+Sirolimus+MMF+Umbilical cord blood cell infusion (No Anti-thymocyte Globulin (ATG))	gmgpxvzhwz = jqrtcilnak nwnyuvtjrg (ardswqagpn, bfjwsuupan - somcoripcd) View more	-	04 Jul 2025
				TBI+Fludarabine+Cyclophosphamide+Sirolimus+MMF+Umbilical cord blood cell infusion (Anti-thymocyte Globulin (ATG))	gmgpxvzhwz = dwvukaxalx nwnyuvtjrg (ardswqagpn, whehtcxqny - nqvhtziuif) View more
NCT04530487 (CTgov)	Phase 2	Refractory Malignant Solid Neoplasm \| Relapsed Solid Neoplasm \| Neuroblastoma recurrent ... View more	1	ATG+cyclosporine+etoposide+mephalan+tacrolimus+MMF+thiotepa	bpcjdyfqoe(autcvkbgvx) = isezyqlccy uhzuvrfmfx (pffjgmwvvx, jeegjpjxaq - cjbbjikqch) View more	-	29 Jun 2025
PF1188 (EHA2025)	Not Applicable	Transfusion-dependent Thalassemia	51	F-BMT conditioning regimen	yeymfdlxap(qpsztsclxf) = umpahatnwv qkevbvimdc (ilccrivdlf ) View more	Positive	14 May 2025
PB3435 (EHA2025)	Not Applicable	Myeloid Tumor	21	Fludarabine and Treosulfan	mwcllxacoy(fyapzyeokg) = qxmwpytldo adfuacciam (dehhmzufon ) View more	Positive	14 May 2025
EHA2025	Not Applicable	Acute myeloid leukemia with mutated NPM1 NPM1 \| FLT3-ITD \| DNMT3A ... View more	44	Fludarabine (FLT3-ITD mutation)	dskpflkkwe(nrisfsgzuc) = btjnrdkptg drsamzlwwt (zjnwwoawkz ) View more	-	14 May 2025
				Fludarabine (DNMT3A mutations)	dskpflkkwe(nrisfsgzuc) = gijmbrfula drsamzlwwt (zjnwwoawkz ) View more
PS1482 (EHA2025)	Not Applicable	Refractory acute myeloid leukemia DNMT3A \| TP53 \| KIT ... View more	-	FLAG Mitox and Venetoclax	unznuelayd(stvoagtyoq) = Grade 3 and 4 adverse events occuring in 10% of pts included febrile neutropenia, pneumonia, sepsis, and gastrointestinal disorders oojgimakgb (uqcezusfjj ) View more	Positive	14 May 2025

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