Nexcella Announces FDA Approval of IND Application for CAR-T NXC-201, Enabling U.S. Patient Dosing
News provided byNexcella, Inc.
Nov 21, 2023, 9:56 AM ET
- NEXICART-2 to expand studies of NXC-201 in relapsed/refractory AL Amyloidosis to U.S. sites based on IND clearance
- 72 patients previously dosed with NXC-201 ex-U.S.
- First CAR-T program for light-chain (AL) Amyloidosis
- Favorable tolerability enables expansion into autoimmune indications
LOS ANGELES, Nov. 21, 2023 (GLOBE NEWSWIRE) -- Nexcella, Inc. (“Nexcella”, “Company”, “We” or “Us”), a clinical-stage biopharmaceutical company engaged in the discovery and development of novel cell therapies for oncology and other indications, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for BCMA CAR-T NXC-201 (formerly HBI0101). With this clearance, NEXICART-2 (NCT06097832) is to expand studies of NXC-201 in relapsed/refractory AL Amyloidosis to the United States. Favorable tolerability enables potential expansion into autoimmune indications.
“Building on encouraging NXC-201 clinical data to-date, we are thrilled that multiple leading U.S. sites are currently planning to enroll patients in the coming months,” said Ilya Rachman, MD PhD, Executive Chairman of Nexcella. “No approved treatment options currently exist for relapsed/refractory AL Amyloidosis patients."
72 patients have been dosed (9 in AL Amyloidosis, 63 in multiple myeloma) with NXC-201 ex-U.S. Relapsed/refractory AL Amyloidosis dose escalation levels included: 150 x 106 (n=1), 450 x 106 (n=2), and 800 x 106 (n=6) CAR+T cells, demonstrating a 100% overall response rate (9/9) (median 6 lines of prior therapy) (as of the September 20, 2023 AL Amyloidosis data cutoff). Relapsed/refractory multiple myeloma dose escalation levels included: 150 x 106 (n=6), 450 x 106 (n=7), and 800 x 106 (n=50) CAR+T cells, demonstrating a 95% overall response rate with median 11.9 months of follow-up (as of the July 17, 2023 multiple myeloma data cutoff). NXC-201 publications, presentations and posters include the 65th American Society of Hematology (ASH) Annual Meeting & Exposition, 27th Annual American Society for Gene and Cell Therapy (ASGCT), 20th International Myeloma Society (IMS) Annual Meeting, Clinical Cancer Research (CCR), 49th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT) and Haematologica.
“We credit our world-class cell-therapy expert team in achieving this IND clearance in-line with our previously communicated timelines,” said Gabriel Morris, President of Nexcella. “NXC-201’s favorable tolerability profile, including overcoming neurotoxicity, potentially enables expansion beyond AL Amyloidosis into autoimmune indications.”
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/refractory AL amyloidosis, relapsed/refractory multiple myeloma, and potentially expanding into autoimmune indications: systemic lupus erythematosus (SLE), myasthenia gravis (MG), and multiple sclerosis (MS).
NXC-201 has been awarded Orphan Drug Designation (ODD) by the FDA in both AL Amyloidosis and multiple myeloma.
NEXICART-1 (NCT04720313) is an ongoing Phase 1b/2a, open-label study evaluating the safety and efficacy of NXC-201 (formerly HBI0101), in adults with relapsed/refractory multiple myeloma and relapsed/refractory AL amyloidosis.
The primary objective of the Phase 1b portion of the study is to characterize the safety and confirm the recommended Phase 2 dose (RP2D) and Phase 2 dose of NXC-201. The Phase 1b portion has been successful in determining the recommended Phase 2 dose (RP2D) of 800 million CAR+T cells. The expected primary endpoint for the Phase 2 portion in relapsed/refractory multiple myeloma is overall response rate and duration of response. Nexcella plans to submit data to the FDA in relapsed/refractory multiple myeloma once 100 patients are treated with NXC-201. The expected primary endpoint for NXC-201 in relapsed/refractory AL Amyloidosis is overall response rate. Nexcella plans to submit data to the FDA in relapsed/refractory AL amyloidosis once 30-40 patients are treated with NXC-201.
About AL Amyloidosis
AL amyloidosis is a rare systemic disorder caused by an abnormality of plasma cells in the bone marrow. Misfolded amyloid proteins produced by these cells cause a buildup of misfolded immunoglobulin proteins in and around tissues, nerves and organs, gradually affecting their function. This can cause progressive and widespread organ damage and high mortality rates.
AL amyloidosis affects roughly 30,000 – 45,000 patients in the U.S. and Europe, and it is estimated that there are approximately 3,000 – 4,000 new cases annually in the U.S. The estimated annual global incidence of AL Amyloidosis is ~15,000 patients. The Amyloidosis market was $3.6 billion in 2017, expected to reach $6 billion in 2025, according to Grand View Research.
About Nexcella, Inc.
Nexcella, Inc., is a Los Angeles, California based clinical-stage biopharmaceutical company engaged in the discovery and development of novel cell therapies for oncology and other indications. Our lead candidate, next generation BCMA-targeted CAR-T NXC-201 for relapsed/refractory AL amyloidosis and relapsed/refractory multiple myeloma, has produced 100% and 95% response rates in each indication, respectively, as of July 17, 2023 across 72 patients. NXC-201 has been awarded Orphan Drug Designation (ODD) by the FDA in both AL Amyloidosis and multiple myeloma. We believe NXC-201 has the potential to be the world’s first “Single-Day CRS” CAR-T, enabling faster return home for patients. Expansion into autoimmune indications is planned: systemic lupus erythematosus (SLE), myasthenia gravis (MG), and multiple sclerosis (MS). Our N-GENIUS platform allows us to discover, develop, and manufacture cutting-edge cell therapies for patients in need. To learn more about Nexcella, Inc. visit us at www.nexcella.com
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