Adaptimmune Announces U.S. FDA Breakthrough Therapy Designation Granted to Letetresgene Autoleucel (lete-cel) for Treatment of Myxoid/Round Cell Liposarcoma (MRCLS)

Core Insights - Adaptimmune Therapeutics has received U.S. FDA breakthrough therapy designation for letetresgene autoleucel (lete-cel) aimed at treating unresectable or metastatic myxoid/round cell liposarcoma (MRCLS) in patients who have undergone prior anthracycline-based chemotherapy and express the NY-ESO-1 antigen [1][3][4] Group 1: FDA Designation and Clinical Data - The breakthrough therapy designation for lete-cel was based on results from the Phase II IGNYTE-ESO trial, where 42% of patients with synovial sarcoma or MRCLS showed RECISTv1.1 responses, including 6 complete responses and 21 partial responses [3] - The response rates were 41% for synovial sarcoma and 43% for MRCLS, with a median duration of response of 12.2 months for MRCLS and 18.3 months for synovial sarcoma [3] - Safety findings were consistent with previous data, with common adverse events including cytopenias, cytokine release syndrome (CRS), and rash, all of which were manageable [3] Group 2: Future Plans and Presentations - The company plans to present updates on its sarcoma franchise and cell therapy pipeline at the Annual J.P. Morgan Healthcare Conference on January 14, 2025 [2][6] - A rolling Biologics License Application for lete-cel is expected to be initiated later this year, targeting both sarcoma indications [4][5] - The Allo-T program will also be featured at the Biotech Showcase™ and the Wuxi Global Forum 2025 [6] Group 3: Company Overview - Adaptimmune is focused on redefining cancer treatment through its engineered T cell receptor (TCR) platform, developing personalized medicines for difficult-to-treat solid tumors [7]