Immix Biopharma's NXC-201 CAR-T Gets FDA RMAT Status for Relapsed/Refractory AL Amyloidosis

Immix Biopharma, Inc., a clinical-stage biopharmaceutical company, has announced that its CAR-T cell therapy, NXC-201, has received the Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA) for the treatment of relapsed/refractory AL amyloidosis. This designation is significant, as it could streamline the path to market approval, allowing for frequent interactions with the FDA and potential routes to accelerated approval and priority review.

NXC-201 is a sterically-optimized BCMA-targeted chimeric antigen receptor T (CAR-T) cell therapy. It is currently the only CAR-T therapy under development for AL amyloidosis, a condition characterized by the production of misfolded amyloid proteins by abnormal plasma cells in the bone marrow. These proteins accumulate in organs such as the heart, kidney, and liver, leading to serious health issues and high mortality rates.

The RMAT designation is designed to expedite the development and review of promising therapies for serious or life-threatening conditions, provided there is preliminary clinical evidence showing the product's potential to address unmet medical needs. For Immix Biopharma, this designation underscores the promise of NXC-201 as a potential new treatment option for patients with relapsed/refractory AL amyloidosis, a condition for which no FDA-approved drugs currently exist.

Gabriel Morris, Chief Financial Officer of Immix Biopharma, expressed satisfaction with the accelerated pace of enrollment in their ongoing NEXICART-2 clinical trial, following the successful completion of its safety run-in phase. The company plans to provide further updates on the trial's progress in the first half of 2025.

The NEXICART-2 clinical trial is an open-label, single-arm, multi-site U.S. Phase 1b/2 study designed to evaluate the safety and efficacy of NXC-201. Expected to enroll 40 patients, the trial will focus on those with preserved heart function who have not previously been treated with BCMA-targeted therapies. The primary endpoints of NEXICART-2 are the complete response rate and overall response rate.

NXC-201 has already demonstrated high complete response rates and an absence of neurotoxicity in prior trials, specifically the ex-U.S. NEXICART-1 study. This promising data supports its potential expansion into treating select immune-mediated diseases beyond AL amyloidosis.

According to recent studies, the prevalence of relapsed/refractory AL amyloidosis in the U.S. is increasing by 12% annually, with an estimated 33,277 patients affected in 2024. The amyloidosis market, valued at $3.6 billion in 2017, is projected to grow to $6 billion by 2025.

Immix Biopharma is committed to advancing the development of NXC-201 and addressing the unmet needs of patients with AL amyloidosis and other immune-mediated diseases. By achieving the RMAT designation, the company hopes to expedite the availability of this innovative therapy to patients who desperately need new treatment options.