Data from the TRANSCEND trial of JCAR017 in relapsed and refractory aggressive B cell non-Hodgkin lymphoma (NHL) was presented on June 17th at the 2017 International Conference on Malignant Lymphoma in Lugano, Switzerland.

Trial summary notes include:

• 66% (21/32) overall response and 50% (16/32) complete response at three months in core group moving to pivotal trial; of those patients in response at three months, 90% (9/10) continue in response at six months
• 66% (29/44) of patients experienced no cytokine release syndrome or neurotoxicity in core group moving into pivotal trial
• 18% (8/44) of core group patients experienced severe neurotoxicity and 2% (1/44) experienced severe cytokine release syndrome
• Defined cell composition and 4-1BB co-stimulatory domain deliver predictable cell expansion and prolonged CAR T cell persistence
• Product was available for 98% (86/88) of patients

JCAR017 is an investigative chimeric antigen receptor (CAR) T cell product candidate that targets CD19, a protein expressed on the surface of almost all B cell malignancies, and uses a defined composition of CD4 to CD8 T cells and a 4-1BB costimulatory domain, which differentiates it from other current CD19-directed CAR T product candidates.

“CAR T cell therapy represents an important step forward in providing options for these highly chemotherapy refractory patients and addresses a significant unmet medical need,” said Dr. Jeremy Abramson, M.D., of Massachusetts General Hospital Cancer Center. “I am particularly excited about the emerging efficacy and safety profile with JCAR017, which could ultimately prove to be optimal therapy for patients with relapsed and refractory DLBCL.”

JCAR017 has been granted Breakthrough Therapy designation by the FDA for treatment of r/r aggressive large B cell NHL and PRIME designation by the European Medicines Agency for treatment of r/r diffuse large B cell lymphoma (DLBCL), a type of NHL.

TRANSCEND NHL 001 is a dose-finding study of JCAR017, which is administered following fludarabine/cyclophosphamide lymphodepletion. Patients received one of two dose levels (50 or 100 million cells).

They were then evaluated for pharmacokinetics, disease response, and safety outcomes, including common CAR T side effects such as cytokine release syndrome (CRS) and neurotoxicity (NT).

Notably, the TRANSCEND NHL 001 protocol includes patients with forms of B cell NHL that would exclude them from trials of other CAR T product candidates, including those with ECOG 2 performance status, central nervous system (CNS) involvement of their lymphoma, and those relapsed after allogeneic bone marrow transplant.

Data for the DLBCL cohort were presented in two groups: core and full.

The core analysis group (N=44) includes patients that represent the population that are planned to move forward into a pivotal trial in the second half of 2017.

The core group includes patients with DLBCL (de novo and transformed from follicular lymphoma) that are ECOG Performance Status 0-1. The full analysis group includes all r/r patients in the DLBCL cohort (N=55), including 11 patients with poor performance status or niche subtypes of aggressive NHL.

Both the core and full groups received conforming product, with at least one month follow-up, and with a data cutoff date of May 4, 2017, for this presentation.

Source: BusinessWire