FDA Approves Adaptimmune's Tecelra as First Engineered Cell Therapy for Solid Tumor

Patients with a particular type of synovial sarcoma now have a new therapeutic option for the first time in over ten years, as the FDA has granted approval to Adaptimmune's Tecelra, marking it as the first engineered T-cell therapy for a solid tumor. This approval signifies several important milestones, including being the first engineered cell therapy for solid tumors, the first TCR-T therapy to reach the market, and the first new treatment for this condition in more than a decade. Additionally, this marks the first approval for Adaptimmune, a company established 16 years ago that has engaged in, and then dissolved, partnerships with major pharmaceutical companies like GSK, Astellas, and Roche, driven by the potential of Adaptimmune's unique T-cell receptor (TCR) platform. This platform enables the engineering of T-cells designed to target and destroy various solid tumor cancers.

Tecelra represents a significant breakthrough not just for Adaptimmune but also for the broader cell therapy landscape. John Lunger, Adaptimmune’s chief patient supply officer, highlighted the novelty of using a lentiviral vector to insert an engineered T-cell receptor into the cell. In contrast, Iovance’s Amtagvi, the first T-cell treatment for solid tumors approved by the FDA in February for melanoma, utilizes immune cells called tumor-infiltrating lymphocytes (TILs) collected from a patient’s tumor, whereas CAR-T cell therapies are derived from a patient’s circulating blood.

Lunger emphasized that 90% of cancer cases involve solid tumors, indicating the significant impact of cell-based therapies targeting these types of cancers. The cost for Tecelra, a one-time therapy, is set at $727,000, with approximately 400 patients in the US qualifying annually. Tecelra is approved for adults with advanced MAGE-A4+ synovial sarcoma of specific human leukocyte antigen (HLA) types who have previously undergone chemotherapy. Patients are screened for their genetic HLA types and MAGE-4A expression through companion diagnostic tests.

Synovial sarcomas are rare solid tumor cancers that form in bones and soft tissues like fat, muscle, nerves, and blood vessels, often affecting young adults. The US sees around 13,400 new cases of soft tissue sarcomas yearly, with synovial sarcomas accounting for 5 to 10% of these. The five-year survival rate is about 20%, with most patients undergoing multiple treatment lines.

The FDA's approval of Tecelra is based on the results of the SPEARHEAD-1 study involving 44 patients, where afami-cel achieved a 43% overall response rate, and 4.5% of patients experienced complete tumor disappearance. For those who responded to Tecelra, the median treatment-free interval in the trial was 17 months. According to Lunger, approximately 70% of responding patients were still alive after two years, although this data is still evolving. While there were no treatment-related deaths in the study, Tecelra can cause serious side effects like cytokine release syndrome and neurotoxicity.

Lunger compared the patient experience with Tecelra’s one-time therapy to the standard treatments involving chemotherapy, radiation, and surgery, describing it as extraordinary. A confirmatory trial will be required as part of the accelerated approval process.

Adaptimmune, headquartered in Oxford, England, aims to have six to ten designated treatment centers operational by the end of the year, expanding to 30 across the US by the end of 2025. The company faced challenges earlier this year when Genentech exited a $3 billion partnership to develop allogenic T-cell therapies. However, Adaptimmune rebounded through a $665 million deal with Galapagos for the rights to uza-cel, a MAGE-4A TCR T-cell therapy in phase 2 trials for ovarian cancer.

The deal with Galapagos, which includes $100 million upfront, will support the launch of Tecelra. Adaptimmune manufactures Tecelra in Philadelphia and outsources viral vector production to Miltenyi Biotec’s subsidiary Lentigen in Maryland, while it handles manufacturing for uza-cel in its UK facilities. The approval of Tecelra is a crucial development for patients with synovial sarcoma and signifies a major milestone for cell therapies targeting solid tumors.

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