Adaptimmune announced the U.S. FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to ADP-A2M4 (MAGE-A4), a SPEAR T-cell therapy, for the treatment of synovial sarcoma. RMAT designation will enable expedited development and review of ADP-A2M4 with the goal of commercialization in 2022.
Established under the 21st Century Cures Act, RMAT designation is a dedicated program designed to expedite the drug development and review processes for promising pipeline products. A product is eligible for RMAT designation if it is a regenerative medicine therapy, such as a T-cell therapy, and is intended to treat, modify, reverse or cure a serious or life-threatening disease or condition and preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for such disease or condition.
Results from a phase 1 trial were presented in November at the Connective Tissue Oncology Society (CTOS) annual meeting in Tokyo, Japan. Data are from patients with synovial sarcoma treated in the expansion phase of the Phase 1 trial. There was an overall response rate of 50%, and a disease control rate of 93% with 13 out of 14 patients showing clinical benefit with best overall responses of partial responses (confirmed or unconfirmed; n=7) or stable disease (n=6).
About ADP-A2M4 (MAGE-A4)
ADP-A2M4 (MAGE-A4) is a SPEAR T-cell therapy is directed to a member of the MAGE family of cancer testis antigen expressed in a number of solid tumor cell types. The MAGE- A4 antigen is among the most commonly expressed cancer testis antigens.
About Synovial SarcomaSynovial sarcoma is a rare and aggressive soft tissue sarcoma. In the early stages of the condition, it may cause no noticeable signs or symptoms. However, as the tumor grows larger, affected people may notice a lump or swelling. In some cases, the tumor can limit range of motion or cause numbness and/or pain if it presses on nearby nerves. Although synovial sarcoma does not have a clearly defined cause, genetic factors are believed to influence the development of this disease. Cells in these tumors are usually characterized by the presence of a translocation involving chromosomes X and 18. This translocation is specific to synovial sarcoma and is often used to diagnose the condition. Treatment generally includes a combination of surgery, radiation therapy and/or chemotherapy.