All content on this site is intended for healthcare professionals only. By acknowledging this message and accessing the information on this website you are confirming that you are a Healthcare Professional. If you are a patient or carer, please visit Know AML

The AML Hub uses cookies on this website. They help us give you the best online experience. By continuing to use our website without changing your cookie settings, you agree to our use of cookies in accordance with our updated Cookie Policy

SNDX-5613 shows encouraging overall response rate in relapsed/refractory acute leukemias

Apr 22, 2021
Share:

SNDX-5613, a potent, highly selective, oral inhibitor of the menin-mixed lineage leukemia (MLL) binding interaction, is currently under investigation in the phase I/II AUGMENT-101 trial (NCT04065399) for the treatment of patients with relapsed/refractory acute leukemias. Very recently, it was announced that SNDX-5613 yielded an overall response rate of 48% in patients harboring MLL rearrangement and NPM1c mutation.1

Preclinical and preliminary clinical data from the AUGMENT-101 trial demonstrated single-agent activity and rapid response after a single 28-day cycle. For more information on the study design and initial results, click here.

By a cutoff date of March 12, 2021, a total of 43 heavily pretreated patients, with a median of three prior therapies, received SNDX-5613 in the phase I trial. Among evaluable patients for efficacy (n = 31), the overall response rate in patients with MLL-rearranged (MLL-r) and those with NPM1c mutation was 54% and 29%, respectively. Among responders, 67% achieved minimal residual disease negativity. There were no treatment discontinuations due to treatment-related adverse events, suggesting good tolerability.

The trial will continue into phase II and the efficacy of SNDX-5613 will be evaluated in three different expansion cohorts, including MLL-r acute lymphoblastic leukemia, MLL-r acute myeloid leukemia (AML), and NPM1c+ AML.

SNDX-5613 holds orphan drug designation by the U.S. Food and Drug Administration (FDA) for adult and pediatric AML.2

  1. BioSpace. Syndax posts positive phase I data in genetically-defined acute leukemias. https://www.biospace.com/article/syndax-posts-positive-phase-i-data-in-genetically-defined-acute-leukemias/. Published Apr 20, 2021. Accessed Apr 21, 2021.
  2. Futubull. Syndax Pharmaceuticals: FDA grants orphan drug designation to SNDX-5613 for adult and pediatric acute myeloid leukemia. https://news.futunn.com/en/post/5958332?report_type=stock&report_id=12314844&seo_redirect=1&level=1&data_ticket=1619085509316429. Published Apr 28, 2020. Accessed Apr 21, 2021.

Share: