American Society of Hematology

Oncotartis’ OT-82 enhances venetoclax efficacy in AML

Tue, 30 Dec 2025 08:00:00 -0500

Venetoclax has shown good results for adult acute myeloid leukemia (AML) in combination with azacitidine, but there is increasing evidence of inherent and acquired resistance. High expression of nicotinamide phosphoribosyltransferase (NAMPT) has been associated with cancer aggressiveness and poor prognosis due to increased nicotinamide metabolism.

Development and characterization of anti-CD19 in vivo CAR T therapy

Mon, 29 Dec 2025 08:00:00 -0500

Shenzhen Grit Biotechnology Co. Ltd. and Shanghai Vitalgen Biopharma Co. Ltd. recently presented their work to develop and evaluate a novel anti-CD19 in vivo CAR T candidate, named GT-801.

Preclinical results of anti-ADAMTS13 antibody in acquired von Willebrand syndrome

Mon, 29 Dec 2025 08:00:00 -0500

The acquired von Willebrand syndrome (AVWS) is a rare bleeding disorder with laboratory findings similar to those of inherited von Willebrand disease. Researchers from the Nara Medical University and collaborating institutions presented a potential therapeutic approach for AVWS.

AND-017 increases RBC and hemoglobin in sickle cell disease

Wed, 24 Dec 2025 08:00:00 -0500

Sickle cell disease (SCD) is an inherited hemoglobinopathy caused by a mutation in the gene encoding β-globin that results in hemoglobin S polymerization, red blood cell (RBC) sickling and hemolytic anemia, among others.

IMV-101 shows promise for B-cell malignancies

Wed, 24 Dec 2025 08:00:00 -0500

IMV-101 is a new CAR T-cell therapy targeting CD19 developed by Suzhou Immunofoco Biotechnology Co. Ltd. for the potential treatment of B-cell malignancies and autoimmune diseases. The company has presented results of the evaluation of its in vitro and in vivo properties.

Orum’s ORM-1153 outperforms venetoclax in AML

Wed, 24 Dec 2025 08:00:00 -0500

Acute myeloid leukemia (AML) is a hematological cancer with limited treatment options and characterized by frequent relapse and poor prognosis. The only approved antibody-drug conjugate for AML is gemtuzumab ozogamicin, which targets CD33.

Eilean’s ZE74-0282 shows promise in JAK2 V617 mutant diseases

Tue, 23 Dec 2025 08:00:00 -0500

Eilean Therapeutics LLC has presented data for ZE74-0282, a novel small molecule that targets the JAK2 JH2 domain harboring the V617F mutation with no impact on wild-type JAK2.

LBL-076: a first-in-class trispecific TCE for refractory MM

Tue, 23 Dec 2025 08:00:00 -0500

Despite therapeutic advances, multiple myeloma (MM) remains incurable, with most patients relapsing and developing resistance, especially those refractory to proteasome inhibitors, immunomodulatory drugs and anti-CD38 antibodies. Limited options and poor prognosis highlight the need for new agents with distinct mechanisms and better safety.

Crossbow Therapeutics highlights activity of TERT-targeting CBX-663

Tue, 23 Dec 2025 08:00:00 -0500

Crossbow Therapeutics Inc. has provided details on the preclinical characterization for the telomerase reverse transcriptase (TERT)-targeting T-cell engager CBX-663.

DXO-1801 shows promise for inflammation-driven anemia

Mon, 22 Dec 2025 08:00:00 -0500

Dexoligo Therapeutics has presented data for their liver-targeted siRNA DXO-1801 for the potential treatment of inflammation-driven anemia, a serious complication of chronic diseases such as chronic kidney disease, myelofibrosis or advanced solid tumors characterized by elevated pro-inflammatory cytokines where the availability for iron for erythropoiesis is limited due to increased hepcidin levels.

AJ1-11095 outperforms ruxolitinib in myeloproliferative neoplasm models

Mon, 22 Dec 2025 08:00:00 -0500

JAK2 inhibitors (JAK2i) are the standard treatment for myelofibrosis (MF), offering symptom relief and reducing spleen size. However, all FDA-approved JAK2i are type I inhibitors, which fail to eliminate the mutant MPN clone, leading many patients to treatment discontinuation.

EMU-116 performs better than plerixafor in sickle cell disease

Fri, 19 Dec 2025 08:00:00 -0500

Using C-X-C chemokine receptor type 4 (CXCR4) antagonists as cell mobilization agents has resulted in some FDA approved agents, such as Plerixafor, for hematopoietic stem cell transplantation and neutropenia. Oral cell mobilizers could result in using them in conditions such as sickle-cell disease (SCD) and chronic neutropenia. Emory University has developed and presented data for their CXCR4 antagonist EMU-116.

AND-017 ameliorates anemia in MDS mice

Thu, 18 Dec 2025 08:00:00 -0500

Most patients with myelodysplastic syndrome (MDS) exhibit variable degrees of anemia due to impaired erythropoiesis. Ameliorating anemia and reducing the dependence on transfusion may enhance the quality of life of these patients and improve their survival rates.

MDX-2003 exerts potent activity in preclinical B-cell malignancies

Wed, 17 Dec 2025 08:00:00 -0500

Modex Therapeutics Inc. has developed MDX-2003, a first-in-class tetraspecific T-cell engager targeting CD19 and CD20 on B cells, while co-engaging CD3 and CD28 on T cells.

AERA-109 successfully fights B-cell-mediated autoimmune disorders

Wed, 17 Dec 2025 08:00:00 -0500

Researchers from Aera Therapeutics Inc. reported the preclinical profile of AERA-109, a novel, targeted in vivo CAR T therapy designed to treat B-cell-mediated autoimmune diseases.

Preclinical characterization of Sumitomo’s DSP-5336

Tue, 16 Dec 2025 08:00:00 -0500

Sumitomo Pharma Co. Ltd. is developing the Menin (MEN1)-myeloid/lymphoid or mixed-lineage leukemia (MLL) interaction inhibitor DSP-5336 (enzomenib) for the treatment of MLL-rearranged acute myeloid leukemia.

Johnson & Johnson’s JNJ-5322 shows improved efficacy in MM

Mon, 15 Dec 2025 08:00:00 -0500

Antibodies targeting CD269 and GPRC5D have shown unprecedented clinical efficacy in the treatment of multiple myeloma (MM), but many patients still develop progressive disease. It was hypothesized that dual-targeting T-cell immunotherapies might improve the efficacy by addressing the difficulty of heterogenous target expression and preventing resistance development due to antigen escape.

Apollo’s APL-4098 shows potent antileukemic effects

Fri, 12 Dec 2025 08:00:00 -0500

Apollo Therapeutics Ltd. has developed APL-4098, a small-molecule general control nonderepressible 2 (GCN2) inhibitor for the potential treatment of AML.

Astrazeneca reports data on AZD-4512 for B-cell malignancies

Fri, 12 Dec 2025 08:00:00 -0500

Astrazeneca plc has provided data for their CD22-targeting antibody-drug conjugate (ADC) AZD-4512 under development for the treatment of B-cell malignancies, which still have significant rates of disease resistance and relapse, as well as treatment-related toxicities.

Amphista’s AMX-883 shows synergy with venetoclax in AML

Thu, 11 Dec 2025 08:00:00 -0500

Amphista Therapeutics Ltd. has developed and presented data for AMX-883, a novel orally bioavailable bromodomain-containing protein 9 (BRD9) degradation inducer for acute myeloid leukemia (AML) treatment.

ASH 2025: Ianalumab data offer break from chronic ITP therapy

Wed, 10 Dec 2025 12:00:00 -0500

Novartis AG’s monoclonal antibody, ianalumab (VAY-736), when added to standard-of-care eltrombopag, extended disease control of primary immune thrombocytopenia (ITP) by 45%, according to data presented Dec. 9 during a late-breaker abstract session at the 67th American Society of Hematology’s annual meeting in Orlando, Fla.

GB-3226: a first-in-class dual ENL-YEATS/FLT3 inhibitor for AML

Wed, 10 Dec 2025 08:00:00 -0500

ENL-YEATS is an epigenetic reader that sustains transcriptional programs essential for AML, whereas FLT3 mutations, present in approximately 30% of patients, drive malignant proliferation. Dual inhibition of ENL-YEATS and FLT3 may therefore more effectively disrupt complementary drivers of leukemogenesis than FLT3 targeting alone.

Exicure phase II mobilizer burixafor rallies in MM war

Tue, 09 Dec 2025 12:00:00 -0500

Exicure Inc.’s buyout early this year of GPCR Therapeutics Inc. is paying off in a big way with data from the finished phase II trial testing burixafor (GPC-100). The agent is used with propranolol and granulocyte colony-stimulating factor to mobilize hematopoietic progenitor cells in patients with multiple myeloma (MM) undergoing autologous hematopoietic cell transplant.

ASH 2025: Tec-dara shines on ‘functional cure’ Majestec-3 MM data

Tue, 09 Dec 2025 10:00:00 -0500

Impressive results of a potential second-line combination treatment for multiple myeloma from the Majestec-3 trial of teclistamab plus daratumumab raised eyebrows at the American Society of Hematology’s 67th annual meeting, with the combination showing an 83.4% rate of progression-free survival at three years vs. 29.7% for standard of care.

ASH 2025: New blood cancer therapies trump chemo, older drugs

Mon, 08 Dec 2025 12:00:00 -0500

Targeted therapies and immunotherapies continue to show better results than chemotherapy in investigator-initiated and company-sponsored cancer trials, and newer options demonstrate improvements over older ones, supporting potential shifts in how patients are treated.

ASH 2025: Speeding AML research with MRD surrogate endpoint

Mon, 08 Dec 2025 12:00:00 -0500

While several targeted therapies are approved for acute myeloid leukemia, a 2023 U.K. study found that median survival following diagnosis was only about seven months, highlighting the need not only for new therapies, but for a faster regulatory strategy. At the American Society of Hematology’s 67th annual meeting in Orlando Dec. 6, researcher Jesse Tettero presented data supporting the use of a measurable residual disease (MRD) surrogate endpoint in acute myeloid leukemia (AML) research.

ASH 2025: Casgevy for kids? Expanding, improving SCD gene therapies

Mon, 08 Dec 2025 07:00:00 -0500

Only a couple of years since the first sickle cell disease (SCD) gene therapies gained U.S. FDA approval, researchers are working to expand access for younger children, and to improve manufacturing and commercialization to reach patients faster.

Ono terminates development of Chordia’s cancer drug candidate

Tue, 29 Apr 2025 11:00:00 -0400

Ono Pharmaceutical Co. Ltd. terminated development of CTX-177 (ONO-7018), its ex-oncology candidate in-licensed from Chordia Therapeutics Inc. in December 2020 for up to ¥52.9 billion (US$370.37 million).

Radiance wins ROR1 ADC rights from CSPC in potential $1B+ deal

Tue, 25 Feb 2025 12:00:00 -0500

Radiance Biopharma Inc. bought its way into the ROR1 antibody-drug conjugate (ADC) space through a potential $1 billion-plus licensing deal, including a $15 million up-front payment, with CSPC Megalith Biopharmaceutical Co. Ltd. for rights to RB-164 (SYS-6005) in the U.S. and select countries.

Radiance wins ROR1 ADC rights from CSPC in potential $1B+ deal

Thu, 20 Feb 2025 12:00:00 -0500