Sumitomo Pharma's Promising AML Therapy Earns Fast Track Status

Sumitomo Pharma America, announced on July 15, 2024, that DSP-5336 has received Fast Track designation from the FDA for treating relapsed or refractory acute myeloid leukemia (AML) with KMT2A rearrangement (MLLr) or nucleophosmin mutation (NPM1m). DSP-5336 is an experimental small molecule inhibitor targeting interactions between menin and MLL proteins, influencing various biological processes such as cell growth, genomic stability, and hematopoiesis.

Tsutomu Nakagawa, Ph.D., President and CEO of SMPA, expressed optimism following FDA's Fast Track designation for DSP-5336 in treating relapsed or refractory acute myeloid leukemia (AML). He emphasized the urgent need for new treatment options and highlighted ongoing clinical advancements. Updated findings from the Phase 1/2 study, presented at the EHA 2024 Congress, showed a 57% objective response rate among patients, with significant responses observed in those with Nucleophosmin 1 (NPM1) mutation and KMT2A (MLL) rearrangement. Complete remission or remission with partial hematologic recovery was achieved in 24% of patients.

DSP-5336 has demonstrated excellent tolerability so far, with no dose-limiting toxicity, cardiac issues, treatment discontinuations, or deaths reported. It also shows minimal pharmacokinetic accumulation and lacks significant interactions with azoles. Differentiation syndrome occurrences were manageable, with no need for intensive care or treatment discontinuations. Dr. Jatin Shah, SMPA's Chief Medical Officer in Oncology, highlighted the challenging landscape of AML treatment, particularly for patients with KMT2A (MLL) rearrangements or NPM1 mutations. He underscored DSP-5336's promising clinical results and potential impact, expressing enthusiasm for advancing the program with FDA Fast Track designation to meet urgent medical needs in AML therapy.