Sumitomo Pharma America has received the fast track designation from the US Food and Drug Administration (FDA) for DSP-5336 to treat certain patients with acute myeloid leukaemia (AML).
The FDA granted the designation to treat patients with relapsed or refractory AML characterised by a KMT2A rearrangement. This condition is also known as mixed lineage leukaemia rearrangement (MLLr) or nucleophosmin mutation (NPM1m).
DSP-5336 is an experimental small molecule inhibitor targeting the interaction between the menin and mixed-lineage leukemia (MLL) proteins.
In June 2022, the FDA awarded the orphan drug designation to DSP-5336 for the treatment of AML.
The asset is currently being assessed in the Phase 1/2 trial. The updated data from the dose-escalation and dose-optimisation phase of the study was presented at the 2024 European Hematology Association (EHA) Hybrid Congress.
As per the last gathered data, objective response was observed in 57% of patients, which included responses in patients with both Nucleophosmin 1 (NPM1) mutation and KMT2A (MLL) rearrangement.
Additionally, 24% of these patients achieved either complete remission (CR) or CR with partial haematologic recovery (CRh).
As of now, DSP-5336 continues to be well-tolerated, with no observed dose-limiting toxicity (DLT) and no significant cardiac signals.
There have been no treatment-related discontinuations or deaths reported.
In preclinical studies, DSP-5336 had demonstrated selective inhibition of growth in human acute leukaemia cell lines.
The experimental agent has been shown to decrease the expression of leukaemia-associated genes such as HOXA9 and MEIS1.
These findings highlight DSP-5336’s potential to target specific molecular pathways involved in leukaemia pathogenesis and differentiation.
Sumitomo Pharma America president and CEO Tsutomu Nakagawa said: “For patients and families facing a diagnosis of relapsed or refractory acute myeloid leukaemia, significant unmet medical needs remain – and we share in their urgency to identify and advance new treatment pathways.
“We are encouraged by FDA’s decision and look forward to working closely with the agency as we continue our clinical development of DSP-5336.”
Sumitomo Pharma America is a US subsidiary of Japan-based Sumitomo Pharma.
In May 2024, the FDA accepted Sumitomo Pharma America’s supplemental new drug application (sNDA) for vibegron (Gemtesa).
Vibegron is a beta-3 adrenergic receptor (β3) agonist for the treatment of men with overactive bladder (OAB) symptoms.
