Nexcella, Inc., a subsidiary of Immix Biopharma, Inc., has announced the completion of a Pre-Investigational New Drug (Pre-IND) meeting with the U.S. Food and Drug Administration (FDA) concerning its NXC-201 cell therapy. The meeting focused on the planned manufacturing and clinical trials of NXC-201 in the United States for the treatment of AL amyloidosis and multiple myeloma.
The Pre-IND meeting provided Nexcella with an opportunity to discuss its proposed Investigational New Drug (IND) filing and to receive guidance from the FDA on the design and execution of initial clinical studies for NXC-201. The FDA reviewed Nexcella's pre-IND package, which included clinical data, a manufacturing plan, and a synopsis of the Phase 1b/2 study protocol for NXC-201.
NXC-201: A Novel CAR-T Therapy
NXC-201 (formerly HBI0101) is an investigational chimeric antigen receptor T (CAR-T) cell therapy targeting B-cell maturation antigen (BCMA). It is currently under evaluation in a comprehensive clinical development program for patients with relapsed/refractory multiple myeloma and relapsed/refractory AL amyloidosis.
The ongoing Phase 1b/2a study, known as NEXICART-1 (NCT04720313), is an open-label trial assessing the safety and efficacy of NXC-201 in adults with relapsed/refractory multiple myeloma and relapsed/refractory AL amyloidosis. The Phase 1b portion of the study successfully determined the recommended Phase 2 dose (RP2D) of 800 million CAR+ T cells.
Clinical Trial Expansion Plans
Nexcella intends to submit an IND application to the FDA to expand the ongoing clinical trial to include sites in the United States. The primary endpoint for the Phase 2 portion of the trial in relapsed/refractory multiple myeloma is overall response rate (ORR) and duration of response, assessed according to the International Myeloma Working Group (IMWG) Uniform Response Criteria. For relapsed/refractory AL amyloidosis, the primary endpoint is ORR, based on consensus recommendations.
Nexcella plans to submit data to the FDA for relapsed/refractory multiple myeloma after 100 patients have been treated with NXC-201 and for relapsed/refractory AL amyloidosis after 30-40 patients have been treated.
Promising Early Results
According to Nexcella, NXC-201 has demonstrated a 92% response rate in relapsed/refractory multiple myeloma and a 100% response rate in relapsed/refractory AL amyloidosis as of February 9, 2023, across 58 patients. The company believes NXC-201 has the potential to be the world’s first outpatient CAR-T therapy.
"Completion of our planned Pre-IND meeting with FDA is an important milestone towards opening U.S. clinical trial sites for NXC-201," said Ilya Rachman, MD, PhD, Chief Executive Officer of Immix Biopharma. Gabriel Morris, Chief Financial Officer of Immix Biopharma, added, "We appreciate the FDA’s guidance and believe that with regulatory clarity in hand, our next step is to proceed with submitting a US IND application to the FDA for NXC-201."
