FDA Grants RMAT Designation to TScan’s Two Lead TCR-T Candidates for Blood Cancers

Rare Daily Staff

The U.S. Food and Drug Administration granted Regenerative Medicine Advanced Therapy designation to TScan Therapeutics for its two lead TCR-T therapy candidates for the treatment of rare blood cancers.

“This is an important milestone that recognizes the transformative potential of our engineered TCR-T therapy candidates, TSC-100 and TSC-101, in multiple difficult-to-treat cancers.” said Chrystal Louis, chief medical officer at TScan Therapeutics.

The candidates are part of TScan’s heme program designed to treat patients with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), and myelodysplastic syndrome (MDS) undergoing allogeneic hematopoietic cell transplantation with reduced intensity conditioning, based on encouraging initial data from the ALLOHA trial.

TSC-100 and TSC-101 target patients with specific minor histocompatibility antigens have are paired with donors who are negative for these antigens.

TSC-100 and TSC-101 are designed to eliminate all recipient hematopoietic cells, including malignant, pre-malignant or normal cells, that persist post-transplant, while leaving donor-derived cells unaffected. Approximately 40 percent of patients with AML, ALL, and MDS who undergo allogeneic haploidentical HCT with reduced intensity conditioning relapse within two years of transplant, at which point there are limited treatment options and poor prognosis. The goal of this program is to increase the cure-rate for patients receiving HCT.

TScan recently provided an update on its phase 1 heme malignancies program. The update included additional follow-up on all eight treatment-arm patients as well as data on two additional control-arm patients. With a median follow-up of >10 months, all eight patients treated with TSC-100 or TSC-101 remain relapse-free with no detectable disease. No dose-limiting toxicities were observed. In contrast, two of eight control-arm patients relapsed approximately six months post-transplant and one of these patients died approximately three months later. A third control-arm patient required clinical intervention because of concerns of impending relapse, and a fourth control-arm patient died post-transplant.

Established under the 21st Century Cures Act, RMAT designation is a dedicated program designed to expedite the drug development and review processes for promising pipeline products, including gene and cell therapies. A regenerative medicine therapy is eligible for RMAT designation if it is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug or therapy has the potential to address unmet medical needs for such a disease or condition. It provides the benefits of intensive FDA guidance on efficient drug development, including the ability for early interactions with FDA to discuss surrogate or intermediate endpoints, potential ways to support accelerated approval and satisfy post-approval requirements, potential priority review of an Investigational New Drug application, and other opportunities to expedite development and review.