Adaptimmune Therapeutics PLC (NASDAQ: ADAP) announced that the U.S. Food and Drug Administration (FDA) granted a Breakthrough Therapy designation for the company’s affinity enhanced T-cell therapy targeting NY-ESO in synovial sarcoma for HLA-A*201, HLA-A*205 or HLA-A*206 allele-positive patients with inoperable or metastatic synovial sarcoma who have received prior chemotherapy and whose tumor expresses the NY-ESO-1 tumor antigen.

Ultimately, the Breakthrough designation was based on the results of a Phase 1/2 trial in patients with unresectable, metastatic or recurrent synovial sarcoma who have received prior chemotherapy. Patients were treated with lymphodepleting chemotherapy followed by immunotherapy with T-cells engineered to recognize an HLA-A2 restricted NY-ESO-1 peptide.

The Annual Meeting of the Society of Immunotherapy for Cancer in November 2015 was the most recent update from this study. In the primary efficacy analysis, 60% of the 10 patients receiving the target dose of cells responded, and there was a 50% overall response rate in the 12 patients receiving any dose of cells. Some 90% of those patients who received the target dose and 75% of all patients were alive and on long-term follow-up.

The most common adverse events included nausea, anemia, pyrexia, lymphopenia and neutropenia. Cytokine release syndrome (CRS) was seen in four of 12 subjects, with grade 3 CRS observed in two subjects; no grade 4 CRS events were observed.

Dr. Rafael Amado, Adaptimmune’s chief medical officer, commented:

We are committed to investigating the potential of our NY-ESO-1-T cell therapy across a variety of cancers. We are pleased that the FDA has granted Breakthrough Therapy designation for our T-cell therapy in synovial sarcoma, recognizing both the unmet need for patients suffering from this disease as well as the promise of these early data. We look forward to working closely with the FDA to expedite the clinical development of this therapeutic candidate.

Shares of Adaptimmune were trading down nearly 7% at $6.65 on Tuesday, with consensus analyst price target of $19.75 and a 52-week trading range of $6.19 to $21.57.

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