U.S. FDA approves investigational new drug (IND) application for NEXI-002

On the 19^th December 2019 it was announced that the United States (U.S.) Food & Drug Administration (FDA) has approved an investigational new drug (IND) application for NEXI-002 for the treatment of patients with multiple myeloma (MM) who have failed three or more prior lines of therapy.¹

NEXI-002

NEXI-002 is a novel, immune-based, endogenous T-cell therapy that contains populations of primed antigen-specific CD8⁺ T cells. These T cells are directed at multiple, specific MM antigen targets: WT1, CD138, NY-ESO-1 and CS1.^1,2 This autologous therapy contains T-cell subtypes with mainly memory-based phenotypes (stem cell- and central-memory), which are associated with anti-tumor activity and durable responses.¹

The pharmaceutical company responsible for the development of this therapy are able to expand multi-antigen specific T cells, without the requirement for genetic manipulation, using a two-step nanotechnology platform^1,2:

• Synthetic nanoparticles act as an artificial antigen-presenting cell to prime and activate T cells against tumor antigen targets
• A T-cell enrichment and expansion process controls ex vivo proliferation and differentiation

Clinical trials

The IND clearance means a multicenter, open-label, single-arm, phase I/II study can begin recruiting patients with MM, who have received three or more prior lines of therapy, to evaluate the safety, tolerability, and initial efficacy of NEXI-002.¹ The study will enroll patients in sites across the U.S., such as the Dana Farber Cancer Institute, MD Anderson, Memorial Sloan Kettering Cancer Center, and The Karmanos Cancer Institute. The trial will initially evaluate safety in three patients (safety cohort). Following this, the expansion phase will enroll up to an additional 20 patients, who will be followed for a minimum of one year.¹

Other applications

Similar products are also in development for the treatment of acute myeloid leukemia (AML) and solid tumors. These include an autologous product, NEXI-003 for transplant ineligible patients with AML, and an allogeneic product, NEXI-001, for patients with AML who relapse post allogeneic stem cell transplant. Both AML products target the WT1, PRAME, and Cyclin A1 antigens.² The U.S. FDA previously granted IND status to NEXI-001 in October 2019, meaning a phase I/II trial in the U.S. could begin enrolling patients.³