Nexcella (NASDAQ: IMMX) has announced updated clinical data for its novel chimeric antigen receptor T (CAR-T) cell therapy, NXC-201, for the treatment of patients with relapsed or refractory multiple myeloma and light chain (AL) amyloidosis.

The data, which was presented at the 26th Annual Meeting of The American Society of Gene and Cell Therapy, showcased impressive results, suggesting that NXC-201 could be a valuable option for AL amyloidosis patients who have not responded to current standards of care.

The clinical data demonstrated highly encouraging outcomes. Eight DARZALEX relapsed or refractory AL amyloidosis patients were treated with NXC-201, and the highlights of the data include:

• An overall response rate of 100%
• A complete response rate of 63% (with minimal residual disease at a rate of 10-5)
• An organ response rate of 75%
• Zero grade 4 cytokine release syndrome (CRS) events reported
• The best responder showed a duration of response of 16.5 months as of the data cutoff of May 11, 2023, with response ongoing

These results hold great promise for AL amyloidosis patients, particularly those who have experienced relapse or refractory cases.

Currently, recent trials with DARZALEX-based treatments show an overall response rate of 55%. Nexcella's NXC-201 provides a potentially efficacious alternative for patients who have progressed on existing standards of care. The rapid organ response observed in the study indicates the therapy's ability to reduce free light chain toxicity quickly.

In addition to its efficacy, NXC-201 demonstrated a favorable safety profile, with no grade 4 cytokine release syndrome events reported. This indicates that the BCMA CAR-T therapy is well tolerated, further supporting its potential as a treatment option for advanced, relapsed/refractory AL amyloidosis.

Following the successful Phase 1b portion of the ongoing clinical trial, Nexcella now plans to submit an IND application to the FDA for a Phase 1b/2 study of NXC-201 in relapsed/refractory multiple myeloma and AL amyloidosis, aiming to expand the trial to the United States.

The company says it will aim to submit data to the FDA once 100 patients are treated with NXC-201 for multiple myeloma and 30-40 patients for AL amyloidosis.

AL amyloidosis is a rare systemic disorder with significant mortality rates, affecting thousands of patients worldwide.

The current market size for amyloidosis treatments is substantial, with estimates projecting it to reach $6 billion by 2025. Nexcella's CAR-T therapy represents a significant advancement in the field, offering hope to patients who face limited treatment options.

Nexcella's updated clinical data for NXC-201 presents a compelling case for its potential as an effective treatment for relapsed or refractory AL amyloidosis.

The impressive overall response rates, favorable safety profile, and rapid organ response demonstrated in the study make NXC-201 a promising candidate in the fight against this debilitating disease.

As Nexcella continues its efforts to expand the clinical trial and gain regulatory approval, the future looks promising for AL amyloidosis patients in need of new treatment options.