Nexcella Announces Patients Already Treated With CAR-T NXC-201; Estimates 100-Patient Total Enrollment for FDA Approval BLA Submission

Nexcella, Inc. recently announced 50 relapsed/refractory multiple myeloma patients have already been dosed with next-generation CAR-T NXC-201 in its ongoing Phase 1b/2a clinical trial. Nexcella anticipates 100-patient open-label total enrollment to seek US FDA BLA approval.

Robust enrollment of up to 5 patients per month continues in the NXC-201 ongoing Phase 1b/2a clinical trial.  Nexcella is on track to present this 50-patient cohort multiple myeloma data later this year at a premier scientific forum.

“Few companies are as far advanced with the quantity and quality of patient data that we have at Nexcella in multiple myeloma and AL amyloidosis,” said Gabriel Morris, President of Nexcella. “50-patient data represents significant progress on our path to BLA submission to seek FDA approval of NXC-201.”

Ilya Rachman, MD, Executive Chairman of Nexcella, added “There are waiting lists with thousands of patients across America who are currently unable to receive these life-saving multiple myeloma CAR-T therapies like NXC-201. Our team is working tirelessly to bring this groundbreaking treatment to patients in the US and worldwide.”

NXC-201 (formerly HBI0101) is a BCMA-targeted investigational chimeric antigen receptor T (CAR-T) cell therapy that is being studied in a comprehensive clinical development program for the treatment of patients with relapsed or refractory multiple myeloma and AL amyloidosis. The design consists of a structurally differentiated CAR-T, with our proprietary BCMA-targeting CAR, which has demonstrated reduced toxicity in NEXICART-1, supporting investigating NXC-201 as an outpatient therapy.

As of the October 23, 2022 data cutoff, updated clinical data from the ongoing Phase 1b portion of the NEXICART-1 (NCT04720313) study of the novel, autologous, BCMA-targeted chimeric antigen receptor T (CAR-T) cell therapy NXC-201 for the treatment of patients with relapsed or refractory multiple myeloma and light chain amyloidosis (AL) showed:

• Multiple Myeloma – 90% overall response rate (59% complete responses) for NXC-201 at the therapeutic dose in an ongoing 42-Patient Phase 1 expansion trial (Haematologica https://doi.org/10.3324/haematol.2022.281628, 5th European CAR-T cell meeting https://www.nexcella.com/publications/) in relapsed/refractory multiple myeloma. All patients treated with NXC-201 were triple-class refractory (to at least 1 immunomodulatory drug, 1 proteasome inhibitor and 1 anti-CD38 antibody)
• AL Amyloidosis – 100% organ response rate, 100% complete responses (MRD negativity 10^-5), published for NXC-201 in 5 relapsed/refractory patients (Clinical Cancer Research  https://doi.org/10.1158/1078-0432.CCR-22-0637, 5th European CAR-T cell meeting https://www.nexcella.com/publications/)
• The therapeutic dose of NXC-201 (800 million CAR+T cells) has already been established as the recommended Phase 2 dose (RP2D)

Additional information on NXC-201 clinical data as of October 23, 2022 is available here.

NEXICART-1 (NCT04720313) is an ongoing Phase 1b/2, open-label study evaluating the safety and efficacy of NXC-201 (formerly HBI0101), in adults with relapsed or refractory multiple myeloma, all of which as of October 23, 2022 were triple-class refractory (to at least 1 immunomodulatory drug, 1 proteasome inhibitor and 1 anti-CD38 antibody).

The primary objective of the Phase 1b portion of the study, is to characterize the safety and confirm the Maximally Tolerated Dose (MTD) and Phase 2 dose of NXC-201. The Phase 2 portion of the study will evaluate the efficacy and safety of NXC-201 with endpoints of overall survival, progression-free survival and response rates according to International Myeloma Working Group (IMWG) Uniform Response Criteria.

Multiple myeloma (MM) is an incurable blood cancer of plasma cells that starts in the bone marrow and is characterized by an excessive proliferation of these cells. Despite initial remission, unfortunately, most patients are likely to relapse. There are 34,470 patients in the US diagnosed with MM each year. Prognosis for patients who do not respond to or relapse after treatment with standard therapies, including protease inhibitors and immunomodulatory agents remains poor.

Nexcella, Inc., a subsidiary of Immix Biopharma, Inc (NASDAQ:IMMX), is a clinical-stage biopharmaceutical company engaged in the discovery and development of novel cell therapies for oncology and other indications. Our N-GENIUS platform allows us to discover, develop, and manufacture cutting-edge cell therapies for patients in need. For more information, visit www.nexcella.com.