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Imetelstat for use in treating myelodysplastic syndrome

Resumen de: ZA202000070B

This disclosure provides methods of treating a myelodysplastic syndrome (MDS) in a subject that is naive to treatment with an agent selected from a hypomethylating agent (HMA) and lenalidomide, or both. The method includes administering to the subject an effective amount of a telomerase inhibitor, such as e.g. imetelstat or imetelstat sodium. In some cases, the subject treated is classified as low or intermediate-1 IPSS risk MDS and/or have MDS relapsed/refractory to Erythropoiesis-Stimulating Agent (ESA).

ANTI-CD79B ANTIBODY DRUG CONJUGATES

Resumen de: EP4670742A1

The present disclosure relates to anti-CD79b antibodies and anti-CD79b antibody drug conjugates (anti-CD79b ADCs) comprising thereof, where the anti-CD79b ADCs further comprise a topoisomerase 1 inhibitor (Topli) drug as a payload. The present disclosure further relates to methods of using such anti-CD79b ADCs for treating B-cell non-Hodgkin lymphoma (B-NHL), diffuse large B cell lymphoma (DLBCL), Burkitt's lymphoma, mantle cell lymphoma, and follicular lymphoma.

PRODRUG NANOPARTICLE FOR TUMOR-TARGETED ULTRASOUND-ACTIVIATED CANCER THERAPY

Resumen de: EP4670735A1

A prodrug nanoparticle for targeted accumulation and activation (treatment) in tumor tissue, that consists of a Pt(IV) complex with the following formula:wherein R₁ und R₂ are the same or different branched or unbranched alkyls having at least 14 carbon atoms, a sonosensitizer and a biocompatible drug delivery system can be used to treat tumors and in particular metastases in a way that is well tolerated by the body. The prodrug nanoparticles according to the invention are particularly suitable for the treatment of mammary carcinomas, pancreatic carcinomas, prostate carcinomas, bronchial carcinomas, endometrial carcinomas, germ cell tumors, non-Hodgkin's lymphomas and malignant mesotheliomas and, in particular, colorectal carcinomas, and their respective metastases.

PHARMACEUTICAL COMBINATION COMPRISING ANTI-CD20 ANTIBODY-DRUG CONJUGATE AND USE THEREOF FOR TREATING NON-HODGKIN LYMPHOMA

Resumen de: WO2025261276A1

An anti-CD20 antibody-drug conjugate, use thereof in combination with a therapeutic agent for treating relapsed or refractory non-Hodgkin lymphoma, and a composition thereof. The present invention relates to the field of biopharmaceuticals. The combined use of the anti-CD20 antibody-drug conjugate and at least one additional therapeutic agent has better efficacy than existing clinical second-line standard therapies.

BIOMARKERS OF CBL-B INHIBITION

Resumen de: WO2025265023A1

Disclosed herein are methods for predicting responsiveness to an immune checkpoint inhibitor, such as a Casitas B-lineage lymphoma proto-oncogene b inhibitor (CBL-Bi), using a biomarker signature. Additionally disclosed herein are methods for determining a biomarker signature indicative of responsiveness to an immune checkpoint inhibitor, such as a Casitas B-lineage lymphoma proto-oncogene b inhibitor (CBL-Bi). Additionally disclosed herein are methods for determining effects of a Casitas B-lineage lymphoma proto-oncogene b inhibitor (CBL-Bi) administered to a subject.

MATERIALS AND METHODS FOR TREATING MYELOID NEOPLASMS

Resumen de: WO2025265055A1

This document provides methods and materials involved in treating myeloid neoplasms (e.g., myeloid cancers such as acute myeloid leukemia (AML)). For example, methods and materials for making and/or using T cells expressing (e.g., engineered to express) (a) one or more chimeric antigen receptors (CARs) having the ability to bind to a myeloid-specific polypeptide (e.g., a CD33 polypeptide) and (b) one or more inhibitory CARs (iCARs) having the ability to bind to a class I major histocompatibility complex (MHC) polypeptide (e.g., an HLA-A polypeptide such as an HLA-A2 polypeptide). In some cases, T cells provided herein can be administered to a mammal (e.g., a human) having a myeloid neoplasm (e.g., a myeloid cancer such as AML) and having received a haploidentical bone marrow transplant to target (e.g., target and destroy) the mammal's myeloid cells while sparing the donor-derived myeloid cells.

HUMAN ANTI-CD45 ANTIBODIES AND USES THEREOF

Resumen de: WO2025264864A1

Human anti-CD45 antibodies are described. The anti-CD45 antibodies can be engineered into numerous formats, such as antibody-radioisotope conjugates, antibody-immunotoxin conjugates, antibody-drug conjugates (ADCs), antibody-detectable label conjugates, antibody-nanoparticle conjugates, antibody-bead conjugates, multi-domain binding molecules, single chain variable fragments (scFv), and recombinant receptors and can be used as research, diagnostic, or therapeutic tools against CD45-related disorders. Examples of CD45 related disorders include cancers (solid tumors and hematologic malignancies such as acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL)), infections, autoimmune disorders, and metabolic disorders, among other disorders described herein.

COMBINATION THERAPY USING MALT1 AND BCL-2 INHIBITORS AND METHODS OF REDUCING REGULATORY T CELLS

Resumen de: WO2025262113A1

The present disclosure is directed to a combination therapy comprising an inhibitor of mucosa-associated lymphoid tissue lymphoma translocation protein and an inhibitor of an anti-apoptotic Bcl-2 family protein, and methods of use thereof.

METHODS OF TREATMENT USING TOPICAL ROFLUMILAST COMPOSITIONS

Resumen de: WO2025265054A2

Methods of treating a patient suffering from one or more of epidermal disorders of persistent inflammation—cell kinetics and differentiation disorders, epidermal disorders of persistent inflammation—altered reactivity disorders, disorders of the lips, oral, or vaginal mucosa, epidermal disorders of cohesion—vesicular and bullous disorders, cutaneous lymphomas, skin manifestations of rheumatologic diseases, hypomelanoses and hypermelanoses, or skin manifestations related to oncology treatments. The methods include topical administration of a pharmaceutical composition comprising a therapeutically effective amount of roflumilast.

COMBINATION THERAPY USING A MALT1 INHIBITOR AND A BTK INHIBITOR

Resumen de: WO2025262118A1

The present disclosure is directed to a combination therapy comprising an inhibitor of mucosa-associated lymphoid tissue lymphoma translocation protein and an inhibitor of BTK, and methods of use thereof.

METHODS FOR TREATING LYMPHOMA

Resumen de: US2025388691A1

Provided herein, in certain aspects, are methods for the treatment of lymphoma, comprising administration of a CD19 antibody, a CD3×CD20 multispecific antibody, and 3-(4-amino-1-oxo 1,3-dihydro-2H-isoindol-2-yl) piperidine-2, 6-dione (Compound A).

ANTIBODIES HAVING SPECIFICITY FOR CD38 AND USES THEREOF

Resumen de: US2025388692A1

CD38 is also expressed in a variety of malignant hematological diseases, including multiple myeloma. In the present invention, the inventors have generated a new antibody against CD38 that could be suitable for producing bispecific antibodies as well as CAR-T cells. In particular, the inventors report the development of Bi38-3, a new bispecific T cell engager that targeted CD38 on MM cells and recruited cytotoxic T cells through the CD3ε. Bi38-3 lacked the Fc region of natural mAb, which contributes to resistance processes, but triggered T cells to proliferate, release cytokine and lyse CD38 positive MM cells in vitro. Similarly, Bi38-3 induced autologous T cells to eliminate tumor plasma cells isolated from MM patients both at diagnosis and at relapse. The cytotoxicity triggered by Bi38-3 was restricted to cells expressing high levels of CD38 and preserved the integrity of T, B and NK lymphocytes in vitro. Importantly, Bi38-3 rapidly reduced tumor cells in an MM1.S xenograft mouse model of human MM. Taken together, the results show that the antibody of the present invention is an effective reagent to specifically eliminate CD38 positive malignant cells without significantly affecting CD38 lowly expressing cells and represents a promising novel immunotherapeutic tool for the treatment of malignant hematological diseases, and especially multiple myeloma.

CAR T-CELLS AGAINST CD79B FOR THE TREATMENT OF NON-HODGKIN LYMPHOMA

Resumen de: EP4667494A2

The present invention provides therapeutics for Non-Hodgkin Lymphoma. In particular, the present invention provides chimeric antigen receptor (CAR) T-cells that can target CD79b.

Benzimidazolone derived inhibitors of bcl6

Resumen de: NZ800047A

The present invention relates to compounds of Formula II and IV that function as inhibitors of BCL6 (B-cell lymphoma 6) activity: wherein X1, X2, R1, and R2 are each as defined herein. The present invention also relates to processes for the preparation of these compounds, to pharmaceutical compositions comprising them, and to their use in the treatment of proliferative disorders, such as cancer, as well as other diseases or conditions in which BCL6 activity is implicated.

SMALL MOLECULE INHIBITORS OF DYRK/CLK AND USES THEREOF

Resumen de: US2025382301A1

This invention is in the field of medicinal chemistry. In particular, the invention relates to a new class of small-molecule compounds having a 6,6-heterocyclic structure (e.g., compounds having a naphthyridine, pyrido-pyridazine, pyrido-pyrazine, quinoline, pyrazino-pyridazine, pyrimido-pyrimidine, quinazoline, quinoxaline or cinnoline ring system) which function as inhibitors of DYRK1A, DYRK1B, DYRK2, DYRK3, CLK1, CLK2, CLK3, CLK4, CDK7, CDK8/19, PI3K, PDGFrA/B, mTOR, WNT, homeodomain-interacting kinases (HIPKs), and/or CMGC kinases leading to inhibition of WNT signaling, and their use as therapeutics for the treatment of Alzheimer's disease, down syndrome, Parkinson's disease, Huntington's disease, diabetes, autoimmune diseases, inflammatory disorders (e.g., airway inflammation, osteoarthritis (e.g., knee related osteoarthritis)), cancer (e.g., glioblastoma, prostate cancer, metastatic breast cancer, metastatic lung cancer, multiple myeloma, secondary metastatic tumors of the brain, colorectal cancer and metastatic colorectal cancer (e.g., metastatic colorectal cancer in the liver)), and other diseases.

HUMANIZED ANTIBODY MOLECULES TO CD138 AND USES THEREOF

Resumen de: US2025382379A1

Humanized antibody molecules that specifically bind to CD138 are disclosed. The humanized antibody molecules can be used to treat, prevent, and/or diagnose disorders, such as multiple myeloma.

TREATMENT METHODS FOR SECOND LINE THERAPY OF CD19-TARGETED CAR T CELLS

Resumen de: US2025381272A1

Provided are adoptive cell therapy involving the administration of doses of cells for treating subjects with certain B cell malignancies, and related methods, compositions, uses and articles of manufacture. The cells generally express recombinant receptors such as chimeric antigen receptors (CARs). In some embodiments, the disease or condition is a large B cell lymphoma (LBCL) relapsed or refractory to first-line chemoimmunotherapy.

COMPOUND FOR DEGRADING USP7 PROTEIN BY MEANS OF TARGETED UBIQUITINATION, PHARMACEUTICAL COMPOSITION THEREOF AND USE THEREOF

Resumen de: WO2025256654A1

A compound for degrading USP7 protein by means of targeted ubiquitination, a pharmaceutical composition thereof and the use thereof. The structural formula thereof is shown as formula (I), wherein A is a specific protein ligand in an E3 ubiquitin ligase complex, and L is a divalent linker group between a small molecule ligand of the USP7 protein and the specific protein ligand in the E3 ubiquitin ligase complex. The protein degradation chimera has both USP7 protein inhibitory activity and USP7 protein-degrading activity, can effectively inhibit malignant proliferation of acute lymphoblastic leukemia cells, and thus can be used for diseases associated with abnormal USP7 protein expression.

MOLECULES THAT ARE VITAMIN D RECEPTOR AGONISTS AND HISTONE DEACETYLASE INHIBITORS

Resumen de: WO2025255667A1

There is provided a compound of formula I or a pharmaceutically acceptable salt or solvate thereof, (Formula I) wherein W is a 5 or 6 membered heteroaryl group comprising at least one nitrogen atom; X1 is CO or O; X2, is NH or CH2; Y is O or CH2, and n is an integer of from 2 to 5. The compound is particularly useful for the treatment of proliferative diseases such as psoriasis or a cancer selected from the group consisting of leukemia, non-small cell lung cancer, colon cancer, central nervous system cancer, melanoma, ovarian cancer, renal cancer, prostate cancer, and breast cancer.

SMALL MOLECULE INHIBITORS OF DYRK/CLK AND USES THEREOF

Resumen de: US2025382277A1

This invention is in the field of medicinal chemistry. In particular, the invention relates to a new class of small-molecule compounds having a 6,6-heterocyclic structure (e.g., compounds having a naphthyridine, pyrido-pyridazine, pyrido-pyrazine, quinoline, pyrazino-pyridazine, pyrimido-pyrimidine, quinazoline, quinoxaline or cinnoline ring system) which function as inhibitors of DYRK1A, DYRK1B, DYRK2, DYRK3, CLK1, CLK2, CLK3, CLK4, CDK7, CDK8/19, PI3K, PDGFrA/B, mTOR, WNT, homeodomain-interacting kinases (HIPKs), and/or CMGC kinases leading to inhibition of WNT signaling, and their use as therapeutics for the treatment of Alzheimer's disease, down syndrome, Parkinson's disease, Huntington's disease, diabetes, autoimmune diseases, inflammatory disorders (e.g., airway inflammation, osteoarthritis (e.g., knee related osteoarthritis)), cancer (e.g., glioblastoma, prostate cancer, metastatic breast cancer, metastatic lung cancer, multiple myeloma, secondary metastatic tumors of the brain, colorectal cancer and metastatic colorectal cancer (e.g., metastatic colorectal cancer in the liver)), and other diseases.

METHODS FOR THE TREATMENT OF CHEMOTHERAPY-INDUCED CARDIOTOXICITY IN A SUBJECT IN NEED THEREOF

Resumen de: WO2025257167A1

The evidences provided by the present patent application demonstrate the therapeutic effect of succinate dehydrogenase inhibition to counteract the cardiotoxicity of chemotherapies used in cancer, such as acute myeloid leukemia and breast cancer. This combination also remarkably exhibits a chemosensitizing capacity of cancer cells. The use of Malonate in patients affected by these pathologies would therefore protect the heart from the toxic side effects of chemotherapy and eliminate the most resistant cells for an ever better risk-benefit ratio for these patients. Accordingly, the present invention relates to a method for the treatment of chemotherapy-induced cardiotoxicity in a subject in need thereof comprising administering to said subject a therapeutically effective amount of a succinate dehydrogenase inhibitor.

COMBINATION THERAPY FOR TREATING CANCER SUCH AS LYMPHOMA

Resumen de: WO2025257171A1

The present disclosure relates to a combination therapy for treating cancer comprising belinostat or a pharmaceutically acceptable salt, tautomer, stereoisomer, mixture of stereoisomers, prodrug, or deuterated analog thereof and at least one nicotinamide phosphoribosyltransferase (NAMPT) inhibitor. Belinostat and the NAMPT inhibitor can either be administered separately or in the form of a single composition. Thus, the disclosure relates to a kit comprising Belinostat and at least one NAMPT inhibitor, wherein Belinostat and the NAMPT inhibitor are in separate formulations and, alternatively, to a composition comprising belinostat and at least one NAMPT inhibitor, such as a pharmaceutical composition. It also relates to such kit or such composition for use in the treatment of cancer, preferably for the treatment of lymphoma(s).

COMBINATION THERAPY FOR TREATING CANCER SUCH AS LYMPHOMA

Resumen de: WO2025257173A1

The present disclosure relates to a combination therapy for treating cancer comprising belinostat or a pharmaceutically acceptable salt, tautomer, stereoisomer, mixture of stereoisomers, prodrug, or deuterated analog thereof and at least one Bcl-2 family protein inhibitor. Belinostat and the Bcl-2 family protein inhibitor can either be administered separately, or in the form of a single composition. Thus, the disclosure relates to a kit comprising Belinostat and at least one Bcl-2 family protein inhibitor, wherein Belinostat and the Bcl-2 family protein inhibitor are in separate formulations and, alternatively, to a composition comprising belinostat and at least one Bcl-2 family protein inhibitor, such as a pharmaceutical composition. It also relates to such kit or such composition for use in the treatment of cancer, preferably for the treatment of lymphoma(s).

COMBINATION THERAPY FOR TREATING CANCER SUCH AS LYMPHOMA

Resumen de: WO2025257176A1

The present disclosure relates to a combination therapy for treating cancer comprising belinostat or a pharmaceutically acceptable salt, tautomer, stereoisomer, mixture of stereoisomers, prodrug, or deuterated analog thereof and at least one glucose transporters (GLUT) inhibitor. Belinostat and the GLUT inhibitor can either be administered separately or in the form of a single composition. Thus, the disclosure relates to a kit comprising Belinostat and at least one GLUT inhibitor, wherein Belinostat and the GLUT inhibitor are in separate formulations and, alternatively, to a composition comprising belinostat and at least one GLUT inhibitor, such as a pharmaceutical composition. It also relates to such kit or such composition for use in the treatment of cancer, preferably for the treatment of lymphoma(s).

NON-INVASIVE MYELODYSPLASTIC SYNDROME DIAGNOSTICS

Nº publicación: WO2025257825A1 18/12/2025

Solicitante:

YEDA RES AND DEVELOPMENT CO LTD [IL]
YEDA RESEARCH AND DEVELOPMENT CO. LTD

Resumen de: WO2025257825A1

Non-invasive methods of diagnosing myelodysplastic syndrome (MDS) in a subject comprising applying a trained machine learning model to a set of parameters from the subject, wherein the machine learning model outputs an MDS score and wherein an MDS score beyond a predetermined threshold indicates the subject suffers from MDS is provided.