T-Cell Therapy Racks Fast Track, Orphan Designations for EBV-Nasopharyngeal Carcinoma


The FDA cleared the IND for BRG01 in February 2023.

The FDA has granted both fast track and orphan drug designations to Biosyngen’s T-cell therapy BRG01 for the treatment of Epstein-Barr virus (EBV)-positive relapsed/metastatic nasopharyngeal carcinoma.1,2

BRG01 is an engineered, allogeneic, EBV-targeting T-cell therapy that has previously demonstrated preliminary safety and efficacy in exploratory clinical trials. The therapy received investigational new drug (IND) approval to initiate clinical trials in the United States (US) in February 2023 and in China in December 2022. The FDA granted orphan drug designation to BRG01 on June 6, 2023, and fast track designation on July 6, 2023.

Cancers commonly associated with EBV include gastric cancers, lymphoma, and lymphoproliferative diseases in addition to nasopharyngeal cancer. According to the World Health Organization, an estimated 133,000 new cases of nasopharyngeal cancer were reported worldwide in 2020, around half of which were diagnosed in China. Within China, over 60% of these cases are within provinces including Guangdong and Guangxi. Standard of care treatment for refractory nasopharyngeal cancer includes immune checkpoint inhibitors but overall response rates are generally below 30%, represting a high unmet need for this population.

WATCH NOW: Amer Beitinjaneh, MD, MSc, MPH, FACP, on Treating EBV+ PTLD With Tab-cel

Biosyngen plans to apply for IND clearance for other immunotherapies targeting lung and liver cancer indications in both the US and China. The company also plans to seek fast track designations for candidates to treat hepatocellular cancer, gastric cancer, and digestive track cancers in Singapore, China, and the US.

Biosyngen isn’t the only company targeting EBV-associated conditions. Atara Biotherapeutics’ tabelecleucel (tab-cel) is a similar allogeneic EBV-specific T-cell immunotherapy being investigated for the treatment of EBV-positive post-transplant lymphoproliferative disease (EBV+ PTLD). Data from the phase 3 ALLELE clinical trial (NCT03394365) were presented at the 2023 Tandem Meetings |Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR, in February 2023. Among patients who received at least 1 dose of tab-cel, the overall response rate was 51.2%, the complete response rate was 27.9%, and the median overall survival was estimated to be 18.4 months.3

“What is remarkable is those responses being durable, with a median duration of response of about 23 months, and they were quick responses...” Amer Beitinjaneh, MD, University of Miami/Jackson Memorial Hospital, said during his presentation of the data.3 “The median time to response was 1 month, so basically 1 cycle of the treatment.”

1. Biosyngen’s first-in-class cell therapy BRG01 receives FDA Fast Track designation. News release. Biosyngen. July 10, 2023. https://www.biosyngen.com/index.php?m=home&c=View&a=index&aid=107
2. Biosyngen's Cell Therapy BRG01 Granted Orphan Drug Designation by the U.S. FDA for Treatment of Nasopharyngeal Cancer. News release. Biosyngen. June 6, 2023. https://www.asiaone.com/business/biosyngens-cell-therapy-brg01-granted-orphan-drug-designation-us-fda-treatment
3. Beitinjaneh A, Baiocchi R, Chaganti S, et al. New and updated results from a multicenter, open-label, global phase 3 (P3) study of tabelecleucel (tab-cel) for Epstein–Barr virus-positive post-transplant lymphoproliferative disease (EBV+ PTLD) following allogeneic hematopoietic cell (HCT) or solid organ transplant (SOT) after failure of rituximab (R) or rituximab and chemotherapy (R+CT) (ALLELE). Presented at: 2023 Tandem Meetings | Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR.February 15-19, 2023; Orlando, FL. Abstract 25
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