Immix Biopharma Announces FDA Approval of IND Application for CAR-T NXC-201, Enabling U.S. Patient Dosing

Rhea-AI Summary

Immix Biopharma, Inc. (IMMX) receives FDA clearance for IND application of BCMA CAR-T NXC-201, expanding studies to U.S. sites for relapsed/refractory AL Amyloidosis. Favorable tolerability allows potential expansion into autoimmune indications. 72 patients previously dosed with NXC-201 ex-U.S. have shown promising overall response rates in clinical trials.

Positive

• FDA clearance for IND application of BCMA CAR-T NXC-201 is a significant milestone for Immix Biopharma, allowing expansion of studies to the U.S.
• Promising overall response rates in clinical trials for NXC-201 in both AL Amyloidosis and multiple myeloma indicate the potential effectiveness of the treatment.
• Favorable tolerability of NXC-201, including overcoming neurotoxicity, opens up possibilities for expansion into autoimmune indications.
• No approved treatment options currently exist for relapsed/refractory AL Amyloidosis patients, indicating a significant unmet medical need.

Negative

• The small sample size of patients dosed with NXC-201 ex-U.S. may limit the generalizability of the overall response rates observed in the clinical trials.

• 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) --  Immix Biopharma, Inc. (Nasdaq:IMMX) (“ImmixBio”, “Company”, “We” or “Us”), a clinical-stage biopharmaceutical company advancing personalized therapies for oncology and immunology, 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, Chief Executive Officer of Immix Biopharma. “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 65^th American Society of Hematology (ASH) Annual Meeting & Exposition, 27^th Annual American Society for Gene and Cell Therapy (ASGCT), 20^th International Myeloma Society (IMS) Annual Meeting, Clinical Cancer Research (CCR), 49^th 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, Chief Financial Officer of Immix Biopharma. “NXC-201’s favorable tolerability profile, including overcoming neurotoxicity, potentially enables expansion beyond AL Amyloidosis into autoimmune indications.”

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, 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.

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) 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. ImmixBio 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. ImmixBio 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 Immix Biopharma, Inc.
Immix Biopharma, Inc. (ImmixBio) (Nasdaq: IMMX) is a clinical-stage biopharmaceutical company pioneering personalized therapies for oncology and immunology with more than 100 patients treated to-date. Our lead cell therapy asset is CAR-T NXC-201 for relapsed/refractory AL Amyloidosis and relapsed/refractory multiple myeloma, for which we have observed overall response rates of 100% and 95%, respectively, in the ongoing Phase 1b/2a NEXICART-1 (NCT04720313) clinical trial (July 17, 2023). NXC-201 has the potential to be the world’s first “Single-Day CRS” CAR-T, enabling a faster return home for patients. Expansion into autoimmune indications is planned: 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. Our lead tissue specific therapeutic (TSTx) asset IMX-110, currently in Phase 1b/2a clinical trials as a monotherapy and IMMINENT-01 combination clinical trial with BeiGene’s anti-PD-1 antibody tislelizumab in relapsed/refractory solid tumors, holds Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) by the FDA. Learn more at www.immixbio.com.

About Nexcella, Inc.
Nexcella, Inc., a subsidiary of Immix Biopharma, Inc. (Nasdaq:IMMX), 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

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@immixbio.com

FAQ

What is the significance of the FDA clearance for the IND application of BCMA CAR-T NXC-201 for Immix Biopharma (IMMX)?

The FDA clearance allows Immix Biopharma to expand studies of NXC-201 to U.S. sites for relapsed/refractory AL Amyloidosis, indicating progress in the development of potential treatments for this condition.

What are the key findings from the clinical trials of NXC-201 in patients with AL Amyloidosis and multiple myeloma?

The clinical trials have shown promising overall response rates for NXC-201 in both AL Amyloidosis and multiple myeloma, demonstrating the potential effectiveness of the treatment.

What potential does the favorable tolerability of NXC-201 hold for Immix Biopharma?

The favorable tolerability of NXC-201, including overcoming neurotoxicity, opens up possibilities for expansion into autoimmune indications, indicating potential for broader applications of the treatment.

What are the limitations of the current treatment options for relapsed/refractory AL Amyloidosis patients?

There are currently no approved treatment options for relapsed/refractory AL Amyloidosis patients, indicating a significant unmet medical need in this patient population.

How many patients have been dosed with NXC-201 ex-U.S. and what are the overall response rates observed in the clinical trials?

72 patients have been dosed with NXC-201 ex-U.S., with promising overall response rates observed in both AL Amyloidosis and multiple myeloma, indicating potential effectiveness of the treatment.