- 100% (10/10) overall response rate (ORR) and 70% (7/10) complete response (CR) rate observed in standard of care (Dara-CyBorD) relapsed/refractory AL Amyloidosis patients with median 6 lines of prior therapy in updated Phase 1/2 data as of December 10, 2023
- Best responder duration of response was 23.7 months with response ongoing as of December 10, 2023
- U.S. observed prevalence of relapsed/refractory AL Amyloidosis is growing 12% per year according to Staron, et al Blood Cancer Journal, estimated to reach 29,712 patients in 2023
LOS ANGELES, Dec. 11, 2023 (GLOBE NEWSWIRE) -- Nexcella, Inc. (“Nexcella”, “Company”, “We” or “Us”), today announced new clinical data from its Phase 1b/2a NEXICART-1 (NCT04720313) study of novel, autologous, BCMA-targeted chimeric antigen receptor T (CAR-T) cell therapy, NXC-201, in patients with relapsed/refractory AL Amyloidosis (R/R ALA) at an oral presentation by study investigator Moshe E. Gatt, MD at the 65th American Society of Hematology (ASH) Meeting being held in San Diego, CA. The updated results include follow-up and clinical data from one new patient. All patients were relapsed/refractory to standard-of-care Dara-CyBorD (daratumumab combined with cyclophosphamide, bortezomib, and dexamethasone) and had experienced a median of 6 prior lines of therapy that failed to stop worsening of disease prior to receiving NXC-201.
“There is a rising prevalence of relapsed/refractory AL Amyloidosis patients who have no approved options for treatment,” said Polina Stepensky, M.D., Director of the Hadassah Medical Organization’s Department of Bone Marrow Transplantation and Immunotherapy for Adults and Children, and principal study investigator. “We continue to be encouraged by NXC-201’s 100% overall response rate, including in this 10th relapsed/refractory AL amyloidosis patient.”
“We believe NXC-201 is the first and only CAR-T in clinical development for AL amyloidosis. With our recent IND clearance, we are thrilled to be now activating sites to bring this first-of-a-kind study to U.S. relapsed/refractory AL Amyloidosis patients,” said Ilya Rachman, M.D., Ph.D., Executive Chairman of Nexcella. Gabriel Morris, President of Nexcella, added, “We believe NXC-201’s favorable tolerability profile, including ‘Single Day CRS’ and the ability to overcome neurotoxicity, enables a new potential option for relapsed/refractory AL Amyloidosis patients and potential expansion into select autoimmune indications.”
Nexcella Announces 100% Overall Response Rate (n=10); 23.7 months Best Response Duration (ongoing) for CAR-T NXC-201 in Relapsed/Refractory AL Amyloidosis Patients at ASH 2023
Nexcella, Inc.
At the NXC-201 ASH 2023 oral presentation, data were presented from 10 relapsed/refractory AL amyloidosis patients (including one new patient) in the ongoing Phase 1b/2a NEXICART-1 study, with median 6 lines of therapy prior to NXC-201. Patients were infused with CAR+T cells at doses of 150 x 106 (n=1), 450 x 106 (n=2), and 800 x 106 (n=7).
Patient characteristics:
- 90% (9/10) had high-risk cytogenetics
- 80% (8/10) had cardiac involvement
- 50% (5/10) had New York Heart Association (NYHA) stage 3 or 4 heart failure (3 stage 4, 2 stage 3)
- 40% (4/10) had Mayo stage 3 (1 stage 3b, 3 stage 3a) AL amyloidosis disease
- 40% (4/10) had t(11;14) translocation
- Relapsed/refractory to a median 6 lines of prior therapy (range: 3-10)
Safety and efficacy data:
- Overall response rate of 100% (10/10)
- Complete response + very good partial response rate of 90% (9/10)
- Complete response rate of 70% (7/10) (6 out of 7 were MRD 10-5)
- Organ response rate of 60% (6/10)
- Best responder had a duration of response of 23.7 months as of December 10, 2023, with response ongoing
- Transformation to complete response at month 2 observed in a patient with 7 lines of prior therapy and cardiac involvement
- There were no immune effector cell-associated neurotoxicity syndrome (ICANS) events
- “Single Day CRS”: Median CRS duration was 1 day (range: 1-4):
- No grade 4 cytokine release syndrome (CRS) events
- 2 experienced no CRS; 2 experienced grade 1 CRS; 4 Experienced grade 2 CRS; 2 experienced grade 3 CRS
For the 8 patients with cardiac involvement:
- Overall response rate of 100% (8/8)
- Complete response rate of 63% (5/8) (4 out of 5 were MRD 10-5)
- Organ response rate of 63% (5/8)
For the 4 patients with t(11;14) disease:
- Overall response rate of 100% (4/4)
- Complete response rate of 75% (3/4) (MRD 10-5)
- Organ response rate of 50% (2/4)
The NXC-201 65th American Society of Hematology Meeting oral presentation video can be accessed on the ASH website: https://annualmeeting.hematology.org/session/227510
The NXC-201 65th American Society of Hematology Meeting oral presentation can be accessed on the Nexcella corporate website at this link: https://nexcella.com/publications/
ASH Presentation Details:
| Title | “Feasibility of a Novel Academic Anti-BCMA Chimeric Antigen Receptor T-Cell (CART) (HBI0101) for the Treatment of Relapsed and Refractory AL Amyloidosis” |
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| Presentation Replay |
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About NXC-201
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, with potential expansion into autoimmune indications.
NXC-201 has been awarded Orphan Drug Designation (ODD) by the FDA in both AL Amyloidosis and multiple myeloma.
About NEXICART-1
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) of NXC-201. The Phase 1b portion has been successfully completed, with a recommended Phase 2 dose (RP2D) of 800 million CAR+T cells.
- 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.
- 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.
About AL Amyloidosis
U.S. observed prevalence of relapsed/refractory AL Amyloidosis is growing 12% per year according to Staron, et al Blood Cancer Journal, estimated to reach 29,712 patients in 2023.
The Amyloidosis market was $3.6 billion in 2017, expected to reach $6 billion in 2025, according to Grand View Research. AL amyloidosis is a 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.
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 December 10, 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. 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
Forward Looking Statements
This press release contains “forward-looking statements.” Forward-looking statements reflect our current view about future events. When used in this press release, the words “anticipate,” “believe,” “estimate,” “expect,” “future,” “intend,” “plan,” or the negative of these terms and similar expressions, as they relate to us or our management, identify forward-looking statements. Such statements, include, but are not limited to, statements contained in this press release relating to our business strategy, our future operating results, continuing development of our product candidates, including development timelines, timing of FDA submissions and expected endpoints, long-term visions and strategies, evaluations and judgements and beliefs regarding potential efficacy and safety of our product candidates, future clinical development of our product candidates, including any implication that results or observations in initial data, data observed to date, or earlier clinical trials will be representative of results or observations in later data or clinical trials, the expected timing of such results and the potential market size and benefits for our product candidates. Forward-looking statements are based on our current expectations and assumptions regarding our business, the economy and other future conditions. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that are difficult to predict. Our actual results may differ materially from those contemplated by the forward-looking statements. They are neither statements of historical fact nor guarantees of assurance of future performance. We caution you, therefore, against relying on any of these forward-looking statements. Important factors that could cause actual results to differ materially from those in the forward-looking statements include, without limitation, our ability to raise capital to fund continuing operations; our ability to protect our intellectual property rights; the impact of any infringement actions or other litigation brought against us; competition from other providers and products; our ability to develop and commercialize products and services; changes in government regulation; our ability to complete capital raising transactions; that our product candidates may not realize the anticipated responses discussed in this release or that their development may suffer delays that materially and adversely affects future commercial viability; that the market for our product candidates may not grow at the rates anticipated or at all; and other factors relating to our industry, our operations and results of operations. Actual results may differ significantly from those anticipated, believed, estimated, expected, intended or planned, including: the uncertainties related to market conditions and other factors described more fully in the section entitled ‘Risk Factors’ in Immix Biopharma’s Annual Report on Form 10-K for the year ended December 31, 2022, and other periodic reports subsequently filed with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and we specifically disclaim any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise. Factors or events that could cause our actual results to differ may emerge from time to time, and it is not possible for us to predict all of them. We cannot guarantee future results, levels of activity, performance or achievements.
Contacts
Mike Moyer
LifeSci Advisors
mmoyer@lifesciadvisors.com
Company Contact
irteam@nexcella.com
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