Sumitomo Pharma Receives FDA Fast Track Designation for DSP-5336 in Relapsed or Refractory Acute Myeloid Leukemia Treatment
Monday, July 15, 2024
Sumitomo Pharma America, Inc. (SMPA) has announced that the U.S. Food and Drug Administration (FDA) granted Fast Track designation to DSP-5336 for the treatment of patients with relapsed or refractory acute myeloid leukemia (AML) characterized by a KMT2A rearrangement, also known as mixed lineage leukemia rearrangement (MLLr), or nucleophosmin mutation (NPM1m). DSP-5336 is an investigational small molecule inhibitor targeting the interaction between menin and mixed-lineage leukemia (MLL) proteins, crucial in pathways such as gene expression, protein interactions, cell growth, and hematopoiesis.
The FDA's Fast Track Designation is reserved for therapies addressing serious conditions with unmet medical needs.
SMPA, expressed, "There remains a significant need for new treatment options for patients and families facing relapsed or refractory AML. We are encouraged by the FDA's decision and look forward to advancing DSP-5336 through clinical development."
Updated data from the Phase 1/2 study, presented at the European Hematology Association (EHA) 2024 Hybrid Congress, revealed an objective response in 57% of patients (12/21), including those with NPM1 mutation and KMT2A rearrangement. Complete remission or complete remission with partial hematologic recovery was achieved in 24% (5/21 patients).
DSP-5336 continues to demonstrate good tolerability, with no observed dose-limiting toxicity, significant cardiac signals, or treatment-related discontinuations or deaths. Reports of differentiation syndrome (DS), managed without intensive care unit (ICU) stays or treatment discontinuation, were noted in 5% of patients.
Dr. Jatin Shah, Chief Medical Officer – Oncology at SMPA, highlighted, "The management of AML remains challenging, particularly with the limited availability of targeted therapies. DSP-5336 has shown promising clinical activity, especially in AML cases featuring KMT2A rearrangements or NPM1 mutations. We are excited about these findings and the Fast Track Designation, aiming to provide a safe and effective treatment option."
AML, characterized by rapid blood cell proliferation in the bone marrow, necessitates immediate intervention. Approximately 30% of AML patients have NPM1 mutations, while 5-10% present with KMT2A rearrangements.
DSP-5336 acts as a menin inhibitor, selectively targeting leukemia-associated genes like HOXA9 and MEIS1, and increasing differentiation gene CD11b expression in preclinical studies. Clinical evaluation continues in Phase 1/2 trials (NCT04988555), supported by FDA-granted Orphan Drug Designation and Fast Track Designation.
Source: prnewswire.com
