The ENHANCE-3 study: venetoclax and azacitidine plus magrolimab or placebo for untreated AML unfit for intensive therapy

N Naval Daver (1The University of Texas MD Anderson Cancer Center, Houston, TX) P Paresh Vyas G Gerwin Huls (3Department of Hematology, Universitair Medisch Centrum Groningen, Groningen, The Netherlands) H Hartmut Döhner (1Department of Internal Medicine III, University Hospital of Ulm, Ulm, Germany) S Sébastien Maury (12Service d’Hématologie Clinique, Hôpital Henri-Mondor, Assistance Publique-Hôpitaux de Paris, Paris, France) J Jan Novak C Cristina Papayannidis (2IRCCS Azienda Ospedaliero-Universitaria di Bologna, Istituto di Ematologia “Seràgnoli”, Bologna, Italy) C Carmen Martinez Chamorro (Hospital Universitario Quironsalud Madrid and Universidad Europea de Madrid, Pozuelo de Alarcon, Spain) P Pau Montesinos (Hospital Universitari i Politecnic La Fe, Valencia, Spain) R Rabin Niroula (2Rhode Island Hospital, Division of Hematology-Oncology, Department of Medicine, Providence, United States) P Pierre Fenaux J Jordi Esteve (13Hematology Department, Institute of Cancer & Blood Diseases (ICAMS), Hospital Clinic, Barcelona, Spain) S Shang-Ju Wu A Adrien De Voeght (1University Of Liège, Montefiore Institute, Department of Electrical Engineering and Computer Science, Artificial Intelligence and Deep Learning, Liège, Belgium) J Jiri Mayer (6Department of Internal Medicine, Hematology and Oncology, University Hospital Brno and Masaryk University Brno, Brno, Czech Republic) P Peter J. M. Valk (2Erasmus MC Cancer Institute, University Medical Center Rotterdam, Rotterdam, The Netherlands) L Lisa Johnson (17Gilead Sciences, Inc, Foster City, CA) M Mei Dong (State Key Laboratory of Coal Conversion) K Ke Liu S Sowmya Kuwahara (17Gilead Sciences, Inc, Foster City, CA) K Kenneth Caldwell (17Gilead Sciences, Inc, Foster City, CA) G Guru Subramanian Guru Murthy (Medical College of Wisconsin, Milwaukee, Wisconsin, United States)

Abstract

Abstract Patients with acute myeloid leukemia (AML) ineligible for intensive chemotherapy (IC) have limited treatment options. The phase 3 ENHANCE-3 study aimed to determine whether magrolimab (magrolimab arm) was superior to placebo (control arm) when either was combined with venetoclax and azacitidine. Adults with previously untreated AML who were ineligible for IC were randomized to receive magrolimab (1 mg/kg on days 1 and 4, 15 mg/kg on day 8, 30 mg/kg on days 11 and 15, then weekly for 5 weeks, and then every 2 weeks) or placebo, venetoclax (100 mg on day 1, 200 mg on day 2, and 400 mg daily thereafter), and azacitidine (75 mg/m2 days 1-7) in 28-day cycles. The primary end point was overall survival (OS); key secondary end points included complete remission (CR) rate and safety. After randomization of 378 patients, the trial was stopped at a prespecified interim analysis owing to futility. At final analysis, with median follow-up of 7.6 months (magrolimab arm) vs 7.4 months (control arm), median OS was 10.7 vs 14.1 months (hazard ratio, 1.178; 95% confidence interval, 0.848-1.637). The CR rate within 6 cycles was 41.3% vs 46.0%. Addition of magrolimab to venetoclax and azacitidine resulted in more fatal adverse events (19.0% vs 11.4%), primarily driven by grade 5 infections (11.1% vs 6.5%) and respiratory events (2.6% vs 0%). There were similar incidences of any-grade infections, febrile neutropenia, and neutropenia between arms. These results highlight the difficulty in improving outcomes for patients with AML who were ineligible for IC. This trial was registered at www.clinicaltrials.gov as #NCT05079230.

Article Details

Journal Blood
Volume / Issue Vol. 146, Issue 5
Published July 31, 2025
Pages 601-611
ISSN 0006-4971
Publisher Elsevier BV

Journal Info

Blood

Elsevier BV

ISSN: 0006-4971 Health Sciences

Authors (22)

N

Naval Daver

1The University of Texas MD Anderson Cancer Center, Houston, TX

P

Paresh Vyas

G

Gerwin Huls

3Department of Hematology, Universitair Medisch Centrum Groningen, Groningen, The Netherlands

H

Hartmut Döhner

1Department of Internal Medicine III, University Hospital of Ulm, Ulm, Germany

S

Sébastien Maury

12Service d’Hématologie Clinique, Hôpital Henri-Mondor, Assistance Publique-Hôpitaux de Paris, Paris, France

J

Jan Novak

C

Cristina Papayannidis

2IRCCS Azienda Ospedaliero-Universitaria di Bologna, Istituto di Ematologia “Seràgnoli”, Bologna, Italy

C

Carmen Martinez Chamorro

Hospital Universitario Quironsalud Madrid and Universidad Europea de Madrid, Pozuelo de Alarcon, Spain

P

Pau Montesinos

Hospital Universitari i Politecnic La Fe, Valencia, Spain

R

Rabin Niroula

2Rhode Island Hospital, Division of Hematology-Oncology, Department of Medicine, Providence, United States

P

Pierre Fenaux

J

Jordi Esteve

13Hematology Department, Institute of Cancer & Blood Diseases (ICAMS), Hospital Clinic, Barcelona, Spain

S

Shang-Ju Wu

A

Adrien De Voeght

1University Of Liège, Montefiore Institute, Department of Electrical Engineering and Computer Science, Artificial Intelligence and Deep Learning, Liège, Belgium

J

Jiri Mayer

6Department of Internal Medicine, Hematology and Oncology, University Hospital Brno and Masaryk University Brno, Brno, Czech Republic

P

Peter J. M. Valk

2Erasmus MC Cancer Institute, University Medical Center Rotterdam, Rotterdam, The Netherlands

L

Lisa Johnson

17Gilead Sciences, Inc, Foster City, CA

M

Mei Dong

State Key Laboratory of Coal Conversion

K

Ke Liu

S

Sowmya Kuwahara

17Gilead Sciences, Inc, Foster City, CA

K

Kenneth Caldwell

17Gilead Sciences, Inc, Foster City, CA

G

Guru Subramanian Guru Murthy

Medical College of Wisconsin, Milwaukee, Wisconsin, United States