ATA129

ATA129 is a cytotoxic T lymphocyte product candidate precision targeted against antigens expressed by the Epstein-Barr Virus (EBV). ATA129 is our most advanced development program and has received:

  • Orphan and breakthrough designation from FDA
  • Orphan and advanced therapy medicinal product (ATMP) designation from EMA
  • Access to priority medicines regulatory support (PRIME) for the lead indication from EMA

ATA129 is ready to enter Phase 3 trials to support approval in two separate indications for EBV-PTLD, a hematologic malignancy:

  • Rituximab refractory EBV-PTLD following allogeneic hematopoietic stem cell transplant – The MATCH Trial
  • Rituximab refractory EBV-PTLD following solid organ transplant – The ALLELE Trial

Pursuant to parallel scientific advice from the EMA’s Scientific Advice Working Group and several national Health Technology Assessment, or HTA, agencies in the EU, in 2018, we plan to submit an application for Conditional Marketing Authorization, or CMA, for ATA129 in the treatment of patients with rituximab refractory EBV-PTLD following HCT.  The CMA will be based on clinical data from Phase 1 and 2 trials conducted at MSK and supported by available data from our Phase 3 trials in rituximab refractory EBV-PTLD after HCT and SOT, which will be ongoing at the time of filing.

To complement the Phase 3 trials, we are conducting an expanded access trial for ATA129 that will enroll patients with a broad range of EBV associated tumors (such as Burkitt’s lymphoma, Hodgkin’s lymphoma, DLBCL) for which there is no comparable or satisfactory alternative therapy.

We have also begun to generate data utilizing ATA129 to treat nasopharyngeal carcinoma (NPC). Our collaborating investigator, MSK, presented clinical results at the June 2016 American Society of Clinical Oncology, or ASCO, meeting on the use of ATA129 in patients with NPC. We intend to continue to evaluate this product candidate in the treatment of NPC, and expect to initiate a Phase1/2 clinical trial evaluating ATA129 in combination with a checkpoint inhibitor for the treatment of NPC following the initiation of our ATA129 Phase 3 trials.

EBV-PTLD

The Epstein-Barr virus (EBV) is a ubiquitous virus that causes infectious mononucleosis in people with normal immune function; however, in immunocompromised patients such as those undergoing hematopoietic cell transplants (HCT) or solid organ transplants (SOT), it can cause lymphoma and other cancers. In the US and European Union, there are an estimated 3100 cases annually of EBV-PTLD following HCT and SOT.

EBV-CTLs are a third-party, donor-derived, “off-the-shelf” T-cell product candidate designed to target and destroy EBV-infected lymphoma cells.

In February 2015, the US Food and Drug Administration granted breakthrough therapy designation for EBV-CTLs in the treatment of patients with rituximab-refractory, EBV-associated lymphoproliferative disease (EBV-PTLD) following allogeneic hematopoietic cell transplant (alloHCT). AlloHCT is a transplant of bone marrow stem cells from one person to another as a means to treat a variety of serious diseases, primarily blood cancers.

In June 2016, ATA129 received Advanced Therapy Medicinal Product Classification (ATMP) from EMA and in October 2016, was granted access to the Priority Medicines (PRIME) regulatory pathway for our lead indication, providing enhanced regulatory support from EMA.

Nasopharyngeal Carcinoma (NPC)

EBV virus is associated with several tumors including EBV-PTLD, DLBCL, Burkitt’s lymphoma, NPC, and Gastric Cancer.

With respect to NPC, nearly all cases of the most common form of NPC, non-keratinizing, are EBV positive (have evidence of EBV infection). Our collaborating investigators at MSK, presented clinical results at the June 2016 American Society of Clinical Oncology, or ASCO, meeting on the use of ATA129 in patients with NPC.  In the study, 11 of 14 patients on the study were alive at a median follow up of 18.1 months, including one complete response and two partial responses. These results are encouraging when compared to historical median survival rates that range from five to eleven months for patients with metastatic disease after progression following standard chemotherapy.

We plan to continue to evaluate this product candidate in the treatment of NPC, and expect to initiate a Phase1/2 clinical trial evaluating ATA129 in combination with a checkpoint inhibitor for the treatment of NPC following the initiation of our ATA129 Phase 3 trials.