创新药周报：首款menin抑制剂获批上市治疗KMT2A基因易位的急性白血病

Investment Rating - The report indicates a positive investment rating for the innovative drug sector, particularly focusing on the approval of the first menin inhibitor for treating KMT2A rearranged acute leukemia [20]. Core Insights - The approval of Revuforj® (revumenib) marks a significant advancement in the treatment of KMT2A rearranged acute leukemia, providing a new therapeutic option for patients [20][21]. - Revuforj® demonstrated a complete response (CR) rate of 21.2% in clinical trials, with a median duration of response (mDOR) of 6.4 months [21][23]. - The market potential for Revuforj® is estimated at approximately $750 million in the U.S. for KMT2A rearranged acute leukemia patients, with a broader potential market exceeding $4 billion when considering additional indications [28]. Summary by Sections Innovative Drug Focus - The report highlights the recent approval of the first menin inhibitor, Revuforj®, for treating KMT2A rearranged acute leukemia [20]. - The mechanism of action for menin inhibitors involves disrupting the interaction between menin and KMT2A fusion complexes, leading to leukemia cell differentiation and apoptosis [10][21]. Domestic Innovative Drug Review - The report notes that 28 new drug IND applications were submitted in China, with one NDA approval [58]. - The report provides insights into various companies and their drug development statuses, indicating a vibrant domestic innovative drug landscape [58]. Global New Drug Updates - The report discusses the competitive landscape for menin inhibitors, including several candidates in various stages of clinical development [17][38]. - Ziftomenib, another menin inhibitor, has shown promising results in treating NPM1 mutated acute myeloid leukemia, with a CR rate of 35% in clinical trials [38][42].