Research Updates

The FDA has accepted an application for INQOVI® (decitabine + cedazuridine) with venetoclax to treat newly diagnosed AML patients ineligible for intensive chemotherapy. If approved, it would be the first all-oral frontline option. A decision is expected by February 25, 2026.

The European Hematology Association (EHA) 2025 Annual Congress, held June 12–15 in Milan, Italy, featured over 100 presentations focused on myelodysplastic syndromes (MDS). This year’s research spotlighted promising advances across diagnosis, risk stratification, anemia treatment in lower-risk MDS, strategies for high-risk disease, and transplantation. Notably, new insights into AI-powered diagnostics, the evolving use of imetelstat and luspatercept, and emerging transplant protocols were shared. Explore our full summary for key data and takeaways from featured abstracts that may shape the future of MDS care.

Bone marrow biopsies play a critical role in diagnosing, staging, and monitoring treatment response in AML. In this expert-led educational video, Thomas LeBlanc, MD, MA, MHS, hematologist-oncologist, associate professor of medicine and population health sciences at Duke Cancer Institute, and Keri Halsema, MSN, NP, nurse practitioner and senior instructor in the Cell Therapy Center at University of Colorado, walk through essential best practices for performing and interpreting bone marrow biopsies in AML care. Topics include key diagnostic tests, biopsy techniques, outpatient procedural considerations, and strategies to improve provider confidence through training and simulation. The video also highlights the importance of patient education, interdisciplinary coordination, and timely lab processing to support optimal outcomes.

Research is the pathway towards a cure for MDS. From May 30 to June 3, 2025, doctors from around the world met to talk about new treatments at the annual meeting of the American Society of Clinical Oncology (ASCO).

Dr. Goldberg has prepared an easy-to-understand summary of the key MDS studies presented.

The MDS Foundation is proud to expand its Journey of Empowerment (JOE) initiative to the acute myeloid leukemia (AML) community - an interactive platform to support patients and caregivers with trusted information, engaging education, and tailored resources. 

Johnson & Johnson (NYSE:JNJ) today announced new Phase 1b data showing encouraging antileukemic activity and a promising safety profile for bleximenib (JNJ-75276617) in combination with venetoclax and azacitidine (VEN + AZA) for the treatment of acute myeloid leukemia (AML) harboring KMT2A gene rearrangements (KMT2Ar) or NPM1 gene mutations (NPM1m). The study evaluated patients with newly diagnosed, intensive chemo-ineligible AML and relapsed or refractory AML.¹ The results were featured in an oral presentation at the 2025 European Hematology Association (EHA) Congress ( S137).

Faron Pharmaceuticals Ltd. (AIM: FARN, First North: FARON), a clinical-stage biopharmaceutical company focused on tackling cancers via novel immunotherapies, today announced the publication of the results of its Phase I BEXMAB study in the prestigious Lancet Haematology which reinforced the safety and efficacy of the treatment in patients with high-risk myelodysplastic syndrome (HR-MDS) and relapsed/refractory (r/r) acute myeloid leukemia (AML).

Auron Therapeutics announced that the first patient has been dosed with AUTX-703 in a first-in-human, Phase 1 clinical trial for patients with advanced hematologic malignancies (NCT06846606).