Immix Biopharma gains FDA Regenerative Medicine Advanced Therapy designation for NXC-201, an optimised CAR-T treatment for relapsed or refractory AL amyloidosis
Tuesday, February 11, 2025
Immix Biopharma, a clinical-stage biopharmaceutical company focused on cell therapies for AL amyloidosis and immune-mediated diseases, has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA) for NXC-201. This sterically optimised CAR-T therapy is being developed for the treatment of relapsed or refractory AL amyloidosis. The RMAT designation is granted to regenerative medicines that target serious conditions and show potential to address unmet medical needs based on preliminary clinical evidence.
The RMAT designation is designed to accelerate the development and review of promising therapies, including cell-based treatments. It provides opportunities for early and frequent interactions with the FDA to discuss regulatory pathways, potential surrogate endpoints, and post-approval requirements, potentially expediting market approval.
NXC-201 is currently the only CAR-T therapy in development for AL amyloidosis and was highlighted in a review article on systemic light chain amyloidosis published in the New England Journal of Medicine in June 2024. It is a BCMA-targeted chimeric antigen receptor T (CAR-T) cell therapy, designed to provide effective treatment while minimising neurotoxicity. Initial results from the Phase 1b/2 NEXICART-1 study conducted outside the U.S. have demonstrated high complete response rates with no reported neurotoxicity in patients with relapsed or refractory AL amyloidosis.
The therapy is being evaluated in a comprehensive clinical programme in the U.S., with potential applications in other immune-mediated diseases. The ongoing NEXICART-2 trial (NCT06097832) builds on existing clinical data and is an open-label, single-arm, multi-site Phase 1b/2 dose expansion study. The trial is set to enrol 40 patients with preserved heart function who have not previously received BCMA-targeted therapy. A safety-run phase involving six patients will assess two dose levels, both of which have shown complete responses in prior studies. The primary goals of the study are to assess safety and efficacy, focusing on complete and overall response rates.
NXC-201 has also been granted Orphan Drug Designation (ODD) by both the FDA in the U.S. and the EMA in the EU.
Source: immixbio.com
