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Last update 20 Mar 2025

Methotrexate

Last update 20 Mar 2025

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

SummaryRASUVO®, an antineoplastic medication that impedes dihydrofolate reductase (DHFR), has gained notoriety since its approval by the US in 1953. Its multifarious applications include treatment for acute lymphoblastic leukemia, breast cancer, severe psoriasis, rheumatoid arthritis, and juvenile rheumatoid arthritis. Indeed, Methotrexate's versatility in managing such afflictions has made it a vital resource in the medical field, elevating patient outcomes and reducing the burden of debilitating illnesses. Pfizer Inc., the pharmaceutical company responsible for its development, has achieved a remarkable feat in crafting such a compound that serves such a wide array of purposes. Through the ages, Methotrexate has proven its efficacy and usefulness, paving the way for a brighter future in the realm of modern medicine.

Drug Type

Small molecule drug

Synonyms

METHOTREXATE PARENTERAL, MTX, Methotrexate (JP17/USP/INN)

+ [39]

Target

DHFR

Action

inhibitors

Mechanism

DHFR inhibitors(Dihydrofolate reductase inhibitors)

Therapeutic Areas

NeoplasmsImmune System DiseasesInfectious Diseases+ [10]

Active Indication

Adult Acute Lymphocytic LeukemiaNon-Hodgkin's lymphoma refractoryRecurrent Non-Hodgkin Lymphoma+ [96]

Inactive Indication

Acquired Immunodeficiency SyndromeAcute Erythroblastic LeukemiaAcute Graft Versus Host Disease+ [116]

Originator Organization

Pfizer Inc.

Active Organization

National Cancer Institute

Shanghai Sine Pharmaceutical Co. Ltd.

National Heart, Lung & Blood Institute

+ [16]

Inactive Organization

Millennium Pharmaceuticals, Inc.

National Eye InstituteWyeth AB

+ [9]

Drug Highest PhaseApproved

First Approval Date

Japan (01 Feb 1968),

LeukemiaTrophoblastic Neoplasms

RegulationOrphan Drug (European Union), Priority Review (United States)

Structure/Sequence

Molecular FormulaC20H22N8O5

InChIKeyFBOZXECLQNJBKD-ZDUSSCGKSA-N

CAS Registry59-05-2

1,688

Clinical Trials associated with Methotrexate

NCT06015750

/ Not yet recruitingPhase 4

An Interventional, Prospective Open-Label Study of Immunosuppressive Therapies to Mitigate Immune-Mediated Loss of Therapeutic Response to Asfotase Alfa (STRENSIQ®) for Hypophosphatasia (RESTORE)

The primary objective of this study is to evaluate the effect of immunosuppressive therapy (IST) in participants treated with asfotase alfa who demonstrate immune-mediated loss of effectiveness (LoE).

Start Date02 Jul 2025

Sponsor / Collaborator

Alexion Pharmaceuticals, Inc.

NCT06315309

/ Not yet recruitingPhase 2IIT

Phase II Clinical Trial of 2 Step ATG Combined with Tacrolimus and Mini Methotrexate for Prevention of Acute GVHD Post Myeloablative Allogeneic Stem Cell Transplant

The purpose of this study is to test whether the combination of the drugs called tacrolimus (Tac), methotrexate (MTX) and new dosing strategy of another drug called (rabbit Anti-thymocyte Globulin [ATG]) will help prevent the development and/or improve severity of acute and/or chronic GVHD.

Start Date29 Jun 2025

Sponsor / Collaborator

The University of Alabama at Birmingham

NCT06124157

/ Not yet recruitingPhase 3IIT

A Randomized International Phase 3 Trial of Imatinib and Chemotherapy With or Without Blinatumomab in Patients With Newly-Diagnosed Philadelphia Chromosome-Positive or Philadelphia Chromosome-Like ABL-Class B-Cell Acute Lymphoblastic Leukemia

This phase III trial compares the effect of the combination of blinatumomab with dasatinib and standard chemotherapy versus dasatinib and standard chemotherapy for treating patients with Philadelphia chromosome positive (PH+) or Philadelphia chromosome-like (Ph-Like) ABL-class B-Cell acute lymphoblastic leukemia (B-ALL). Blinatumomab is a bispecific antibody that binds to two different proteins-one on the surface of cancer cells and one on the surface of cells in the immune system. An antibody is a protein made by the immune system to help fight infections and other harmful processes/cells/molecules. Blinatumomab may bind to the cancer cell and a T cell (which plays a key role in the immune system's fighting response) at the same time. Blinatumomab may strengthen the immune system's ability to fight cancer cells by activating the body's own immune cells to destroy the tumor. Dasatinib is in a class of medications called tyrosine kinase inhibitors. It works by blocking the action of an abnormal protein that signals cancer cells to multiply, which may help keep cancer cells from growing. Giving blinatumomab and dasatinib in combination with standard chemotherapy may work better in treating patients with PH+ or Ph-Like ABL-class B-ALL compared to dasatinib and chemotherapy alone.

Start Date29 May 2025

Sponsor / Collaborator

National Cancer Institute

100 Clinical Results associated with Methotrexate

100 Translational Medicine associated with Methotrexate

100 Patents (Medical) associated with Methotrexate

70,896

Literatures (Medical) associated with Methotrexate

31 Dec 2025·Annals of Medicine

Effect of curcumin on methotrexate-induced ovarian damage and follicle reserve in rats: the role of PARP-1 and P53

Article

Author: Karaoluk, Nesibe ; Keçeci, Mete

BACKGROUNDMethotrexate (MTX) is an agent used in the treatment of many neoplastic and non-neoplastic diseases and is known to cause oxidative damage in normal tissues. Curcumin (Cur) is a natural polyphenol compound with powerful antioxidant and antiapoptotic effects. In this study we investigate the effects of Cur on MTX-induced ovarian damage.MATERIALS AND METHODSThirty-two young adult female Wistar albino rats were divided into four groups: (1) Control (n = 8): only vehicle group, (2) Cur (n = 8): Cur-only group (200 mg/kg/day), (3) MTX (n = 8): MTX-only group (0.35 mg/kg/day), (4) MTX+Cur (n = 8): The group was given MTX (0.35 mg/kg/day) and Cur (200 mg/kg/day) for 28 days. Then, SOD, CAT, MDA, AMH levels were measured using ELISA kits. Follicle count was performed on H&E stained slides. In addition, the expressions of P53 and PARP-1 were analysed by immunohistochemistry.RESULTSMDA levels were seen to be higher in the MTX group than in the MTX+Cur group (p < 0.05). Cur treatment lowered MDA levels and increased SOD and CAT levels (p < 0.05 for all). In the MTX+Cur group, atretic follicle count decreased (p < 0,05), however, primordial follicle count increased (p < 0,01). Secondary follicle count and AMH levels were higher in MTX-treated groups (p < 0,05 and p < 0,01, respectively). Expressions of p53 and Poly [ADP-ribose] polymerase 1 (PARP-1) increased significantly in the MTX group compared to the other groups (p < 0,05).CONCLUSIONCur pretreatment prior to MTX administration may be an effective option in preserving the ovarian follicle pool by regulating P53 and PARP-1 expressions with its antioxidant effect.

31 Dec 2025·Journal of Obstetrics and Gynaecology

Predictors of treatment failure of tubal pregnancy with single-dose methotrexate regimen – a systematic review and meta-analysis

Review

Author: Zhu, Qian ; Tang, Lili ; Ling, Ling ; Nie, Sipei

BACKGROUNDEctopic pregnancies represent a potentially life-threatening medical emergency, with 95% being tubal. This meta-analysis aimed to identify early predictors for single-dose methotrexate (MTX) treatment failure in tubal pregnancies.METHODSA literature search was conducted across several databases from their inception to December 2023, with references in the selected studies manually reviewed. 14 studies involving 2,804 patients were included in this meta-analysis.RESULTSThe results revealed that higher serum beta-human chorionic gonadotropin (β-hCG) levels on Day 1 (SMD = 1.25, 95% CI 0.73-1.77), foetal cardiac activity presence (OR = 12.64, 95% CI 3.15-50.75), adnexal mass presence (OR = 4.66, 95% CI 2.02-10.74), yolk sac presence (OR = 5.35, 95% CI 2.33-12.27), thicker endometrium (MD = 1.74, 95% CI 0.30-3.19), more number of previous ectopic pregnancies (MD = 0.21, 95% CI 0.13-0.30), history of pelvic inflammatory disease (PID) (OR = 3.97, 95% CI 2.02-7.79), higher progesterone levels on Day 1 (SMD = 0.22, 95% CI 0.07-0.36), a higher 48-hour pre-treatment increment in serum β-hCG percentage (MD = 11.46, 95% CI 2.95-19.98), and a higher percentage of serum β-hCG change from Day 4 to Day 0/1 (SMD = 2.58, 95% CI 1.02-4.14) were early predictive factors for treatment failure of tubal pregnancy with the MTX single-dose regimen.CONCLUSIONSThis review clarifies early predictive factors for treatment failure with the MTX single-dose regimen in tubal pregnancies. High-risk tubal pregnancies likely to fail MTX monotherapy could be identified earlier, allowing for personalised intervention measures to be implemented at an early stage to prevent harm and improve treatment outcomes.

01 Dec 2025·Journal of Clinical Immunology

Successful Allogeneic Hematopoietic Cell Transplantation for Patients with IL10RA Deficiency in Japan

Article

Author: Kato, Motohiro ; Uhlig, Holm H ; Saida, Satoshi ; Sasahara, Yoji ; Keino, Dai ; Eguchi, Katsuhide ; Ishige, Takashi ; Hagiwara, Shin-Ichiro ; Wada, Taizo ; Suzuki, Tasuku ; Kudo, Takahiro ; Morio, Tomohiro ; Matsuda, Yusuke ; Ishimura, Masataka ; Arai, Katsuhiro ; Ito, Yoshiya ; Goto, Kimitoshi ; Kanegane, Hirokazu ; Takeuchi, Ichiro ; Tomomasa, Dan

BACKGROUNDIL10RA (IL10 receptor subunit alpha) deficiency is an autosomal recessive disease that causes inflammatory bowel disease during early infancy. Its clinical course is often fatal and the only curative treatment is allogeneic hematopoietic cell transplantation (HCT). In Japan, only case reports are available, and there are no comprehensive reports of treatment outcomes.METHODSWe retrospectively analyzed patients with IL10RA deficiency in Japan.RESULTSTwo newly identified and five previously reported patients were included in this study. Five patients underwent HCT; one untransplanted patient survived to age 14, and one died of influenza encephalopathy before transplantation. All five HCT recipients underwent HCT at the age before 2 years. They all were conditioned with fludarabine/busulfan- or fludarabine /melphalan-based regimens. The donor source was human leukocyte antigen haploidentical donor bone marrow (BM) for two patients and unrelated umbilical cord blood (CB) for two patients. One patient experienced graft failure with unrelated CB and required a second transplant with unrelated BM. All patients who underwent HCT survived and demonstrated an improved performance status.CONCLUSIONIn cases of IL10RA deficiency, the need for transplantation should be promptly assessed, and early transplantation should be considered. (190/250).

474

News (Medical) associated with Methotrexate

18 Mar 2025

·pipelinereview.com

Orca Bio Announces Positive Results from the Pivotal Phase 3 Study of Investigational Orca-T® Compared to Allogeneic Stem Cell Transplant for the Treatment of Hematologic Malignancies

Precision-T study met the primary endpoint of a statistically significant improvement in survival free of moderate-to-severe chronic graft versus host disease (cGvHD), showing 78% with Orca-T versus 38% with conventional allogeneic stem cell transplant (alloHSCT) at one year (HR 0.26, p<0.00001) Overall survival with Orca-T was 94% compared to 83% with alloHSCT at one year, and the cumulative incidence of moderate-to-severe cGvHD was 13% versus 44%, respectively Orca Bio is preparing to submit these findings in a BLA to the U.S. FDA in 2025 Results will be presented at the 51st Annual Meeting of the EBMT MENLO PARK, CA, USA I March 17, 2025 I Orca Bio, a late-stage biotechnology company committed to transforming the lives of patients through high-precision cell therapy, today announced positive results from the pivotal Phase 3 Precision-T study of Orca-T, its lead investigational allogeneic T-cell immunotherapy, in patients with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), high-risk myelodysplastic syndrome (MDS) and mixed-phenotype acute leukemia (MPAL). Orca-T is manufactured using highly purified regulatory T-cells, hematopoietic stem cells and conventional T-cells derived from peripheral blood from either related or unrelated matched donors. In the randomized Precision-T study, Orca-T met the primary endpoint of a statistically significant improvement in survival free of moderate-to-severe chronic graft versus host disease (cGvHD) with Orca-T. At one year, the rate for patients who received Orca-T was 78% compared to 38% for patients who received a conventional allogeneic hematopoietic stem cell transplant (alloHSCT). Patients in the Orca-T group achieved an estimated overall survival (OS) of 94% compared to 83% in the alloHSCT arm at one year. “Today, treating patients with serious blood cancers using allogeneic stem cell transplants requires a difficult risk-benefit trade-off as clinicians aim to cure the disease while avoiding potentially deadly treatment-related toxicities, like GvHD,” said presenting author Everett Meyer, M.D., Ph.D., hematologist and associate professor of medicine in Blood and Marrow Transplantation and Cellular Therapy at Stanford Health Care. “The Precision-T study showed double the rate of survival free from GvHD with Orca-T versus a conventional transplant, a relapse-free survival rate of 76% and no new safety concerns. These findings are highly encouraging and provide compelling new evidence as we work to solve for the critical factors contributing to the needs of this patient population.” Precision-T Study Results In the study, all patients (n=187) with a median age of 43.5 years (range 19-65 years) were randomized 1:1 to Orca-T plus single-agent tacrolimus (TAC) or alloHSCT plus TAC methotrexate (TAC/MTX). Patients across both groups received myeloablative conditioning (MAC) and used a related or unrelated matched donor. Patients had a median follow-up time of 11.4 months (range 0.2-24.3 months) across both arms. Key results from the Precision-T study at one year are summarized below: Exploratory endpoints at one year include the rate of relapse-free survival which was 76% and 74% in the Orca-T and alloHSCT arms, respectively (HR 0.80, p=0.49). The cumulative incidence of non-relapse mortality was 3% in the Orca-T arm and 13% in the alloHSCT arm. Additionally, the cumulative incidence of Grade 3 or 4 acute GvHD was 6% and 17% in the Orca-T and alloHSCT arms, respectively. No new safety issues were identified with Orca-T. Grade ≥ 4 infections per CTCAE scoring were noted in 6% and 10% of patients in the Orca-T and alloHSCT arms, respectively. Orca-T was manufactured in Orca Bio’s centralized GMP facility and delivered to patients at 19 treatment centers across the U.S., with all infusions occurring within a vein-to-vein time of 72 hours or less. “Approximately 46,000 people are diagnosed with AML, ALL and MDS in the U.S. each year, but only a fraction of them receive an allogeneic stem cell transplant within the current paradigm,” said Rawan Faramand, M.D., Blood and Marrow Transplant and Cellular Immunotherapy, Moffitt Cancer Center. “Additional treatment options are needed, and the introduction of a cell therapy like Orca-T that leverages a precision-based approach could pave the way for a new standard of care for patients with various hematologic malignancies.” “These exciting results underscore Orca Bio’s vision of transforming the treatment landscape for patients living with serious blood cancers, potentially standardizing curative treatment for diseases like AML, ALL and MDS,” said Ivan Dimov, Ph.D., co-founder and chief executive officer at Orca Bio. “We are working closely with the FDA and expect to submit a Biologics License Application this year. These results support the validity of our high-precision platform as we continue to advance our robust pipeline of allogeneic cell therapies for the treatment of hematologic malignancies, autoimmune diseases and beyond.” Orca Bio is grateful to the patients and families, donors and trial site investigators who participated in the Precision-T study. The complete results will be presented on April 2, 2025, at the 51st Annual Meeting of the EBMT in Florence, Italy. About Precision-T Precision-T (NCT05316701) is a randomized, open-label multi-center study that evaluated the safety, efficacy and tolerability of Orca Bio’s lead investigational allogeneic T-cell immunotherapy, Orca-T, compared to conventional allogeneic hematopoietic stem cell transplant (alloHSCT). Orca Bio received guidance from the U.S. Food and Drug Administration on the design of Precision-T, which evaluated Orca-T in patients with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), high-risk myelodysplastic syndrome (MDS) and mixed-phenotype acute leukemia (MPAL). There are 19 leading treatment centers participating in the trial, which enrolled 187 patients across the U.S. About Orca-T Orca-T is an investigational allogeneic T-cell immunotherapy being evaluated for the treatment of multiple hematologic malignancies. Orca-T is composed of highly purified regulatory T-cells, CD34+ stem cells and conventional T-cells derived from peripheral blood from either related or unrelated matched donors. Orca-T has received Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug Designation for the prevention of graft versus host disease or death in patients eligible for hematopoietic stem cell transplant from the U.S. Food and Drug Administration. About Orca Bio Orca Bio is a late-stage biotechnology company developing high-precision cell therapies for the treatment of cancer and autoimmune diseases. Our manufacturing platform uses single-cell precision to create proprietary, uniquely-defined products designed to replace a patient’s diseased blood and immune system with a healthy one. At Orca Bio, we are on a mission to redefine what’s possible for patients by transforming the field of curative allogeneic cell therapy. For more information, visit www.orcabio.com . SOURCE: Orca Bio

Clinical ResultImmunotherapyCell TherapyPhase 3Orphan Drug

17 Mar 2025

·www.globenewswire.com

Lirum Therapeutics Announces Debut of LX-101 Results in Thyroid Eye Disease, Selected for Presentation at the 2025 NANOS Annual Meeting

New York, NY, March 17, 2025 (GLOBE NEWSWIRE) -- Lirum Therapeutics, Inc. (“Lirum”), an innovative clinical-stage biopharmaceutical company focused on the treatment of debilitating diseases, announced today that its first data on LX-101 in Thyroid Eye Disease (TED) have been selected for presentation at the 51st Annual Meeting of the North American Neuro-Ophthalmology Society (NANOS), March 15-20 in Tucson, Arizona. These new results demonstrate the ability of LX-101 to effectively target the underlying processes that drive TED pathogenesis. Principal findings demonstrate that LX-101 can: 1) inhibit TED-derived orbital fibroblasts; 2) decrease production of key inflammatory cytokines; 3) reduce production of pro-fibrotic components of the extracellular matrix; and 4) modulate activity of TED-derived T-cells. TED is a serious autoimmune disorder characterized by progressive inflammation, driven by activated orbital fibroblasts and infiltrating T-cells, that can lead to orbital tissue expansion, eye bulging (proptosis), double vision (diplopia), pain, vision impairment, and possible sight-threatening complications. The results will be presented by Lirum’s academic collaborator, Collynn F. Woeller, Ph.D., from the Flaum Eye Institute at the University of Rochester. LX-101 is a clinical stage, novel, payload-bearing, targeted therapy directed to IGF-1R. LX-101 enables precise delivery of methotrexate, a drug widely used in autoimmune disease and with a history of use in TED, directly to key effector cells contributing to disease (i.e. IGF-1R+ activated T-cells and orbital fibroblasts). As such, LX-101, directed to a clinically and commercially validated target, may offer a novel and differentiated approach to treating TED. Given these promising results and a strong scientific rationale, combined with LX-101’s novel mechanism of action and prior positive clinical experience in oncology, Lirum is planning new clinical trials with LX-101 in TED and cancer. Presentation Details Abstract#:337Title:LX-101, a Novel Payload-bearing Insulin-like Growth Factor-1 Receptor Targeted Therapy, Inhibits Thyroid Eye Disease InflammationDate/Time:Monday, March 17th at 5 PM MSTSession Title:Analytical Studies, Orbital and Eyelid Disorders Following the session, the presentation will be available on the Lirum website (www.lirumtx.com) under the Investors and Media tab. About Lirum Therapeutics, Inc.Lirum is an innovative clinical-stage biopharmaceutical company focused on the treatment of debilitating diseases through the acquisition, development and commercialization of novel drug candidates with compelling mechanisms of action, regulatory pathways and commercial opportunities. Lirum’s lead candidate, LX-101, is a novel clinical-stage targeted therapy directed to the insulin-like growth factor 1 receptor (IGF-1R) with a differentiated mechanism of action. Lirum is developing LX-101 in oncology and autoimmune indications, including thyroid eye disease (TED). For more information on Lirum, please visit www.lirumtx.com. Forward Looking StatementsThis press release contains certain “forward-looking statements” within the meaning of the “safe harbor” provisions of the US Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by words such as: “target,” “believe,” “expect,” “will,” “may,” “anticipate,” “estimate,” “would,” “positioned,” “future,” and other similar expressions that predict or indicate future events or trends or that are not statements of historical matters. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based only on Lirum’s current beliefs, expectations and assumptions. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that are difficult to predict and many of which are outside of our control. Actual results and outcomes may differ materially from those indicated in the forward-looking statements. Therefore, you should not rely on any of these forward-looking statements. Important factors that could cause actual results and outcomes to differ materially from those indicated in the forward-looking statements include, among others, the following: (1) the ability of Lirum to successfully develop its product candidates, including obtaining positive results from planned clinical trials, 2) expectations for the clinical development, manufacturing, regulatory approval and commercialization of our product candidates or other products we may acquire or in-license(3) expectations for incurring capital expenditures and generating revenue, 4) estimates of the sufficiency of our existing cash and cash equivalents and investments to finance operations, 5) changes in applicable laws or regulations; (6) the possibility that Lirum may be adversely affected by other economic, business, and/or competitive factors; (7) the impact of health epidemics, including the COVID-19 pandemic, on Lirum’s business and the actions Lirum may take in response thereto; and (8) other risks and uncertainties indicated from time to time. There may be additional risks that Lirum considers immaterial or which are unknown. Any forward-looking statement made by us in this press release is based only on information currently available to Lirum and speaks only as of the date on which it is made. Lirum undertakes no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise, except as required by law. Also, the information found on our website is not incorporated by reference into this press release and is included for reference purposes only. Company Contact: Matt HobermanInvestor RelationsIR@lirumtx.com Phone: (646) 389-6015 Lirum Therapeutics, Inc.1270 Ave of the Americas, 7th FloorNew York, NY 10020

17 Mar 2025

·orcabio.com

Orca Bio Announces Positive Results from the Pivotal Phase 3 Study of Investigational Orca-T® Compared to Allogeneic Stem Cell Transplant for the Treatment of Hematologic Malignancies

MENLO PARK, CA, March 17, 2025 – Orca Bio, a late-stage biotechnology company committed to transforming the lives of patients through high-precision cell therapy, today announced positive results from the pivotal Phase 3 Precision-T study of Orca-T, its lead investigational allogeneic T-cell immunotherapy, in patients with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), high-risk myelodysplastic syndrome (MDS) and mixed-phenotype acute leukemia (MPAL). Orca-T is manufactured using highly purified regulatory T-cells, hematopoietic stem cells and conventional T-cells derived from peripheral blood from either related or unrelated matched donors. In the randomized Precision-T study, Orca-T met the primary endpoint of a statistically significant improvement in survival free of moderate-to-severe chronic graft versus host disease (cGvHD) with Orca-T. At one year, the rate for patients who received Orca-T was 78% compared to 38% for patients who received a conventional allogeneic hematopoietic stem cell transplant (alloHSCT). Patients in the Orca-T group achieved an estimated overall survival (OS) of 94% compared to 83% in the alloHSCT arm at one year. "Today, treating patients with serious blood cancers using allogeneic stem cell transplants requires a difficult risk-benefit trade-off as clinicians aim to cure the disease while avoiding potentially deadly treatment-related toxicities, like GvHD," said presenting author Everett Meyer, M.D., Ph.D., hematologist and associate professor of medicine in Blood and Marrow Transplantation and Cellular Therapy at Stanford Health Care. "The Precision-T study showed double the rate of survival free from GvHD with Orca-T versus a conventional transplant, a relapse-free survival rate of 76% and no new safety concerns. These findings are highly encouraging and provide compelling new evidence as we work to solve for the critical factors contributing to the needs of this patient population." Precision-T Study Results In the study, all patients (n=187) with a median age of 43.5 years (range 19-65 years) were randomized 1:1 to Orca-T plus single-agent tacrolimus (TAC) or alloHSCT plus TAC methotrexate (TAC/MTX). Patients across both groups received myeloablative conditioning (MAC) and used a related or unrelated matched donor. Patients had a median follow-up time of 11.4 months (range 0.2-24.3 months) across both arms. Key results from the Precision-T study at one year are summarized below: The primary endpoint of survival free of cGvHD was 78% (95% CI: 65%, 87%) in the Orca-T arm (n=93) and 38% (95% CI: 26%, 51%) in the alloHSCT arm (n=94) (HR 0.26; p<0.00001). An interim analysis of the secondary endpoint of OS was 94% (95% CI: 86%, 97%) in the Orca-T arm and 83% (95% CI: 73%, 90%) in the alloHSCT arm (HR 0.49; p=0.11823). An additional secondary endpoint of cumulative incidence of moderate-to-severe cGvHD was 13% (95% CI: 5%, 23%) and 44% (95% CI: 31%, 56%) in the Orca-T and alloHSCT arms, respectively (HR 0.19; p<0.00002). Exploratory endpoints at one year include the rate of relapse-free survival which was 76% and 74% in the Orca-T and alloHSCT arms, respectively (HR 0.80, p=0.49). The cumulative incidence of non-relapse mortality was 3% in the Orca-T arm and 13% in the alloHSCT arm. Additionally, the cumulative incidence of Grade 3 or 4 acute GvHD was 6% and 17% in the Orca-T and alloHSCT arms, respectively. No new safety issues were identified with Orca-T. Grade ≥ 4 infections per CTCAE scoring were noted in 6% and 10% of patients in the Orca-T and alloHSCT arms, respectively. Orca-T was manufactured in Orca Bio’s centralized GMP facility and delivered to patients at 19 treatment centers across the U.S., with all infusions occurring within a vein-to-vein time of 72 hours or less. “Approximately 46,000 people are diagnosed with AML, ALL and MDS in the U.S. each year, but only a fraction of them receive an allogeneic stem cell transplant within the current paradigm,” said Rawan Faramand, M.D., Blood and Marrow Transplant and Cellular Immunotherapy, Moffitt Cancer Center. “Additional treatment options are needed, and the introduction of a cell therapy like Orca-T that leverages a precision-based approach could pave the way for a new standard of care for patients with various hematologic malignancies.” “These exciting results underscore Orca Bio’s vision of transforming the treatment landscape for patients living with serious blood cancers, potentially standardizing curative treatment for diseases like AML, ALL and MDS,” said Ivan Dimov, Ph.D., co-founder and chief executive officer at Orca Bio. “We are working closely with the FDA and expect to submit a Biologics License Application this year. These results support the validity of our high-precision platform as we continue to advance our robust pipeline of allogeneic cell therapies for the treatment of hematologic malignancies, autoimmune diseases and beyond.” Orca Bio is grateful to the patients and families, donors and trial site investigators who participated in the Precision-T study. The complete results will be presented on April 2, 2025, at the 51st Annual Meeting of The EBMT in Florence, Italy.

Clinical ResultCell TherapyImmunotherapyPhase 3

100 Deals associated with Methotrexate

External Link

KEGG	Wiki	ATC	Drug Bank
D00142	Methotrexate	L04AX03 L01BA01	DB00563

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Lung Cancer	China	Jiangsu Hengrui Pharmaceuticals Co., Ltd.	01 Jan 1995
Lung Cancer	China	Shanghai Sine Pharmaceutical Co. Ltd.	01 Jan 1995
Uterine Cervical Cancer	China	Jiangsu Hengrui Pharmaceuticals Co., Ltd.	01 Jan 1995
Stomach Cancer	Japan	Pfizer Japan, Inc.	04 Oct 1991
acute leukemia	Australia	Pfizer Australia Pty Ltd.	09 Jul 1991
Acute Lymphoblastic Leukemia	Australia	Pfizer Australia Pty Ltd.	09 Jul 1991
Breast Cancer	Australia	Pfizer Australia Pty Ltd.	09 Jul 1991
Bronchogenic Carcinoma	Australia	Pfizer Australia Pty Ltd.	09 Jul 1991
Choriocarcinoma	Australia	Pfizer Australia Pty Ltd.	09 Jul 1991
Hydatidiform Mole	Australia	Pfizer Australia Pty Ltd.	09 Jul 1991
Mycosis Fungoides	Australia	Pfizer Australia Pty Ltd.	09 Jul 1991
Osteosarcoma	Australia	Pfizer Australia Pty Ltd.	09 Jul 1991
Psoriasis	Australia	Pfizer Australia Pty Ltd.	09 Jul 1991
Sarcoma	Japan	Pfizer Japan, Inc.	15 Feb 1984
Lymphoma	Japan	Pfizer Japan, Inc.	01 Feb 1984
Lymphoid Leukemia	Japan	Pfizer Japan, Inc.	10 Sep 1982
Philadelphia chromosome positive chronic myelogenous leukemia	Japan	Pfizer Japan, Inc.	10 Sep 1982
Leukemia	Japan	Pfizer Japan, Inc.	01 Feb 1968
Trophoblastic Neoplasms	Japan	Pfizer Japan, Inc.	01 Feb 1968
Rheumatoid Arthritis	Canada	Nordic Group BV	-

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Non-Hodgkin Lymphoma	Discovery	United States	Accord Healthcare, Inc.	24 Aug 2020
Osteosarcoma	Discovery	United States	Accord Healthcare, Inc.	24 Aug 2020
Psoriasis	Discovery	United States	Nordic Group BV	27 Nov 2019
Rheumatoid Arthritis	Discovery	United States	Nordic Group BV	27 Nov 2019

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT00390793 (CTgov)	Phase 2	Acute lymphoblastic leukemia recurrent \| Philadelphia positive acute lymphocytic leukaemia \| Recurrent Adult Acute Lymphoblastic Leukemia \| Blast Phase Chronic Granulocytic Leukemia	107	dasatinib+cyclophosphamide+Prednisone+Doxorubicin+Cytarabine+Dexamethasone+Methotrexate+Vincristine	xrfruzumiy(sblmlathdw) = qyipoupgwp nykewebipd (gkpwflhnwr, daoshlmrni - akbrrhqjhu) View more	-	17 Feb 2025
NCT00801216 (SCNSL1, CTgov)	Phase 2	Central Nervous System Diseases \| B-Cell Lymphoma	40	etoposide+cyclophosphamide+carmustine+Rituximab+methotrexate+Thiotepa+cytarabine	(ubcpibevbq) = owlqjcljbd lauyhvsbaf (gueiwhmnvl, shrrghkdez - bzfylofrja) View more	-	10 Dec 2024
ASH2024 Manual	Not Applicable	Primary Central Nervous System Lymphoma First line	22	Orelabrutinib + Rituximab + Methotrexate	jreiobaxqg(vnscxbbhck) = tnbthpewsb kxgxijtzfq (ahprikwcrz ) View more	Positive	09 Dec 2024
ASH2024 Manual	Not Applicable	Primary Central Nervous System Lymphoma First line	17	Orelabrutinib + Rituximab + Thiotepa	jtlhcarhha(dokyxapzxi) = bhonesdadn fyizglaslp (lppzaduasi ) View more	Positive	09 Dec 2024
ASH2024 Manual	Not Applicable	Primary Central Nervous System Lymphoma First line	17	Orelabrutinib + Rituximab + Thiotepa + High-Dose Methotrexate	jtlhcarhha(dokyxapzxi) = fvmhwzldtj fyizglaslp (lppzaduasi ) View more	Positive	09 Dec 2024
ASH2024	Not Applicable	Immune Reconstitution Inflammatory Syndrome \| Graft vs Host Disease	-	Calcineurin inhibitor plus methotrexate (CNI+MTX)	(qnlbecefnu) = rttrhnejrb bhqmbgiuxh (cgxbpalcbm )	-	09 Dec 2024
ASH2024	Not Applicable	Acute Lymphoblastic Leukemia	-	Methotrexate	zkrcvvkbqz(owiwrtjvba) = n=4 loaowklydb (kkclpfuqbi ) View more	-	09 Dec 2024
ASH2024	Not Applicable	-	-	Post-transplant cyclophosphamide (PTCy) + calcineurin inhibitor (CnI) and mycophenolate mofetil (MMF)	nbmaqcwpdx(cpavyvppvq) = ntwwwpdwyn xkqvkujrfp (fgzgmiumgz, 42 - 75) View more	-	09 Dec 2024
ASH2024	Not Applicable	-	-	Calcineurin inhibitor (CnI) and methotrexate (MTX)	nbmaqcwpdx(cpavyvppvq) = nulnpvaplb xkqvkujrfp (fgzgmiumgz, 10 - 27) View more	-	09 Dec 2024
ASH2024	Not Applicable	Histiocytosis, Langerhans-Cell	-	Methotrexate and Cytarabine (MA) therapy	(sgmhqjvlyy) = sjqitcxyei kosabpkrhm (saiepvrlxk ) View more	-	08 Dec 2024
ASH2024 Manual	Not Applicable	Primary Central Nervous System Lymphoma First line	26	Rituximab, Methotrexate, Thiotepa, and Orelabrutinib	(snqqmeaakx) = grade 3 hematologic toxicity were observed in 30.77% patients, three patients with grade 4 hematologic toxicity. pppazddnsy (ctndnvuwyf )	Positive	07 Dec 2024
ASH2024	Not Applicable	Acute Lymphoblastic Leukemia \| Down Syndrome	-	Methotrexate (DS-ALL)	rdbrrywfur(eecuzzszxj) = individuals with DS-ALL have slower PK clearance of HDMTX and are at a greater risk of HDMTX-induced AEs despite doses lower than non-DS-ALL bgtlmposjr (dbvwkovyml )	-	07 Dec 2024
ASH2024	Not Applicable	Acute Lymphoblastic Leukemia \| Down Syndrome	-	Methotrexate (non-DS-ALL)		-	07 Dec 2024

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