MatchMe™, our proprietary cell selection platform, is being developed to select the most appropriate treatments for patients.
Pipeline
Tab-cel®
Our most advanced T-cell immunotherapy, tab-cel®, is in Phase 3 development for the treatment of patients with EBV+ PTLD who have failed rituximab or rituximab plus chemotherapy. Based on our market research, we estimate there were several hundred EBV+ PTLD patients who failed rituximab or rituximab plus chemotherapy in the U.S. in 2019. Tab-cel® is also under development for other EBV+ diseases with significant unmet medical need through a Phase 2 multi-cohort study that was initiated in the third quarter of 2020.
EBV+ PTLD
We continue to advance development of tab-cel® in Phase 3 for patients with EBV+ PTLD following hematopoietic cell transplants, or HCT, or solid organ transplants, or SOT. We have obtained Breakthrough Therapy Designation, or BTD, in the U.S. and PRIority MEdicines, or PRIME, designation in the European Union, or EU.
We presented a comprehensive data package to the FDA in October 2020 and reached alignment on several key topics related to the tab-cel® regulatory package, including that (i) a rolling submission is acceptable for the biologics license application, or BLA, (ii) we can complete the BLA submission with currently enrolled patients with at least six months follow-up for duration of response, and (iii) the FDA will consider the data from the Phase 2 trials conducted at Memorial Sloan Kettering Cancer Center, or MSK, our Phase 2 multicenter expanded access protocol, or EAP, and the Single Patient Use, or SPU, program as supportive data to the pivotal study in the BLA clinical module.
We remain on track to initiate a BLA submission for patients with EBV+ PTLD by the end of 2020. We plan to continue engaging with the FDA as part of our rolling BLA and BTD status and expect to finalize the BLA submission in the third quarter of 2021.
Data from the interim analysis we conducted for the Phase 3 study in the third quarter of 2020 showed a 50% objective response rate, or ORR, to tab-cel® with independent oncologic and radiographic assessment, or IORA, in patients with EBV+PTLD following HCT or SOT, that had reached at least 6 months follow-up after achieving a response. This ORR is consistent with previously published investigator assessed data. The tab-cel® safety profile is also consistent with previously published data, with no new safety signals. Data from this pivotal study will be presented at an appropriate forum in 2021.
We remain in active discussions with the Pediatric Committee, or PDCO, of the European Medicines Agency, or EMA, regarding a Pediatric Investigation Plan, or PIP, and received a positive opinion from PDCO on the PIP in November 2020. We expect EMA ratification of the PIP in December 2020, and we plan to submit a tab-cel® EU marketing authorization application for patients with EBV+ PTLD in the second half of 2021.
While clinical study operations and the opening of additional Phase 3 study sites in the United States, Canada and Europe have been impacted by the spread of COVID-19, most sites are currently open for patient enrollment.
Other EBV+Diseases
We continue to pursue development of tab-cel® in earlier lines of therapy with the goal of expanding the potential label in PTLD and closely related diseases. We initiated a Phase 2 multi-cohort study in third quarter of