Immix Biopharma Announces Primary Endpoint Met in positive NXC-201 Interim Results Presented at ASCO, Enabling Path to Best-in-Class Therapy for relapsed/refractory AL Amyloidosis

Rhea-AI Summary

Immix Biopharma (IMMX) announced positive interim results from its NEXICART-2 Phase 1/2 clinical trial of NXC-201 for relapsed/refractory AL Amyloidosis at ASCO 2025. The therapy met its primary endpoint with a remarkable 70% complete response rate (7/10 patients), significantly outperforming current treatments which typically achieve less than 10% CR. The remaining 3 patients showed bone marrow minimum residual disease negativity, suggesting potential future CRs. The trial demonstrated strong safety profile with no neurotoxicity, no relapses, and only low-grade cytokine release syndrome observed. Patients in the trial had received a median of 4 prior lines of therapy. Following treatment, all patients normalized pathological disease markers and showed cardiac and renal organ improvements. The company plans to submit a Biologics License Application (BLA) for FDA approval.

Positive

• Exceptional 70% complete response rate (7/10 patients) vs typical <10% CR with current treatments
• Strong safety profile with no neurotoxicity, no relapses, and only low-grade cytokine release syndrome
• All patients normalized pathological disease markers with cardiac and renal organ improvements
• Remaining 3 patients show bone marrow MRD negativity, suggesting potential future complete responses
• Meeting primary endpoint enables path to BLA submission for FDA approval

Negative

• Small patient sample size of only 10 patients in the trial
• Early interim results may not reflect final efficacy outcomes

Insights

Biotechnology Research Scientist

NXC-201 shows exceptional 70% complete response rate in AL Amyloidosis trial, significantly outperforming current treatments' <10% response rate.

Immix Biopharma's announcement represents a significant clinical milestone for NXC-201, their cell therapy candidate for relapsed/refractory AL Amyloidosis. The interim results from their Phase 1/2 NEXICART-2 trial demonstrated a 70% complete response (CR) rate (7/10 patients), substantially outperforming current treatment options which typically achieve less than 10% CR rates in this difficult-to-treat patient population.

What makes these results particularly compelling is the trial's patient composition – subjects had received a median of 4 prior treatment lines (range: 2-12), representing a heavily pre-treated population with limited therapeutic options. The remaining 3 patients achieved bone marrow minimum residual disease (MRD) negativity at the 10^-6 threshold, suggesting potential future conversion to complete response status.

The absence of relapses to date is noteworthy, as is the favorable safety profile with no neurotoxicity observed – only low-grade cytokine release syndrome has been reported. This safety profile is particularly important for AL Amyloidosis patients who often have multi-organ involvement and may be more vulnerable to treatment-related adverse events.

The company's intent to pursue a Biologics License Application suggests confidence in their data package. If approved, NXC-201 would address a significant unmet need in AL Amyloidosis, a rare and often fatal disease characterized by misfolded protein deposits damaging organs. The downstream clinical improvements in cardiac and renal function following complete responses further validate the potential disease-modifying effects of this therapy.

Immix Biopharma today presented results at ASCO from its U.S. multi-center NEXICART-2 Phase 1/2 clinical trial of NXC-201, meeting its primary endpoint.

– NXC-201 met primary endpoint with a complete response (CR) rate of 70% (7/10 patients) –

– No relapses recorded to-date; no safety signals identified –

– Immix plans Biologics License Application (BLA) for FDA approval –

– Results were presented by Heather Landau, MD, of Memorial Sloan Kettering Cancer Center –

– KOL event to discuss significance Tuesday, June 3, 2025 3:00pm ET after ASCO presentation: https://lifescievents.com/event/immix-asco –

LOS ANGELES, CA, June 03, 2025 (GLOBE NEWSWIRE) -- Immix Biopharma, Inc. (“ImmixBio”, “Company”, “We” or “Us” or ”IMMX”), a clinical-stage biopharmaceutical company developing cell therapies for AL Amyloidosis and other serious diseases, today announced meeting its primary endpoint of complete response (CR) rate for cell therapy NXC-201 at an oral presentation of interim results at the 2025 American Society of Clinical Oncology Annual Meeting (ASCO 2025) in Chicago, Illinois. The oral presentation summarized meeting its interim results endpoint in from the U.S. multi-site NEXICART-2 clinical trial evaluating NXC-201 in relapsed/refractory AL Amyloidosis with a data cutoff of April 11, 2025.

A Key Opinion Leader (KOL) event to discuss the significance will be held Tuesday, June 3, 2025 3:00pm ET with live Q&A (register here to participate).

“Meeting primary endpoint at this interim data readout is a testament to the groundbreaking efficacy of NXC-201, as we work toward the first approval in the challenging indication of relapsed/refractory AL Amyloidosis,” said Ilya Rachman, MD, PhD, Chief Executive Officer of Immix Biopharma. Gabriel Morris, Chief Financial Officer of Immix Biopharma, added, “This inflection point further accelerates our progress toward BLA submission for FDA approval.”

Immix’s Phase 1/2 NEXICART-2 clinical trial is a U.S. multi-site, single-arm study to evaluate the efficacy and safety of NXC-201 in patients who are refractory to, or relapse on treatment for relapsed/refractory AL Amyloidosis.

ASCO Presentation Results
Prior to NXC-201 treatment, the median lines of therapy was 4 (range: 2-12). All patients had baseline relapsed/refractory AL Amyloidosis organ involvement.  After NXC-201 treatment, all patients normalized pathological disease markers. Complete responses (CRs) were observed in 70% (7 out of 10) of patients treated with NXC-201. The remaining 3 patients are bone marrow minimum residual disease (MRD) negative (10^-6), predicting future CR (Immix believes remaining three MRD negative (10^-6) patients could be confirmed as CRs in the coming weeks and months). Downstream clinical improvement, including cardiac and renal organ responses, were recorded after CRs. There have been no relapses recorded to-date and no safety signals identified. No neurotoxicity has been observed. Only low-grade cytokine release syndrome has been observed. The ASCO presentation contains clinical data as of April 11, 2025.

Current treatments typically result in a lower than 10% complete response (CR) rate in relapsed/refractory AL Amyloidosis according to Zanwar, et al 2024, indicating a high unmet medical need.

KOL Event to Discuss NXC-201 ASCO Clinical Data Presentation
A Key Opinion Leader (KOL) event with lead investigator Heather Landau, MD, of Memorial Sloan Kettering Cancer Center, Shahzad Raza, MD of Cleveland Clinic and Jeffrey Zonder, MD of Karmanos Cancer Center will be held Tuesday, June 3, 2025 at 3:00 pm ET to discuss these results. Register here: https://lifescievents.com/event/immix-asco. 

About NEXICART-2
NEXICART-2 (NCT06097832) is an ongoing single-arm multi-site U.S. Phase 1/2 clinical trial of sterically-optimized CAR-T NXC-201 in relapsed/refractory AL Amyloidosis. NEXICART-2 is expected to enroll 40 patients with preserved heart function (excluding patients with pre-existing heart failure) who have not been exposed to prior BCMA-targeted therapy. The primary endpoint of the Phase 1 portion is safety. The primary endpoint of the Phase 2 portion is efficacy.

About NXC-201
NXC-201 is a sterically-optimized BCMA-targeted chimeric antigen receptor T (CAR-T) cell therapy with a “digital filter” that filters out non-specific activation. Initial data from ex-U.S. study NEXICART-1 has demonstrated high complete response rates in relapsed/refractory AL Amyloidosis. U.S. Phase 1/2 study NEXICART-2 is ongoing. NXC-201 has been awarded Regenerative Medicine Advanced Therapy (RMAT) by the FDA, and Orphan Drug Designation (ODD) by the US FDA and in the EU by the EMA.

About AL Amyloidosis
AL amyloidosis is caused by abnormal plasma cells in the bone marrow, which produce misfolded amyloid proteins that circulate in the blood, then build-up in the heart, kidney, liver, and other organs. This build-up causes progressive and widespread organ damage, including heart and renal failure, leading to high mortality rates.

The U.S. observed prevalence of relapsed/refractory AL Amyloidosis is estimated to be growing at 12% per year according to Staron, et al Blood Cancer Journal, to approximately 33,277 patients in 2024.

The Amyloidosis market was $3.6 billion in 2017, and is 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 developing cell therapies for AL Amyloidosis and other serious diseases. Our lead candidate is sterically-optimized BCMA-targeted chimeric antigen receptor T (CAR-T) cell therapy NXC-201. NXC-201 is being evaluated in the U.S. multi-center study NEXICART-2 (NCT06097832). NXC-201 has demonstrated no neurotoxicity in AL Amyloidosis, supporting expansion into other serious diseases. NXC-201 has been awarded Regenerative Medicine Advanced Therapy (RMAT) by the US FDA and Orphan Drug Designation (ODD) by FDA and in the EU by the EMA. Learn more at www.immixbio.com and www.BeProactiveInAL.com.

Forward Looking Statements
This press release contains forward-looking statements regarding Immix Biopharma, Inc., its results of operations, prospects, future business plans and operations and the matters discussed above, including, but not limited to, the potential benefits of our product candidate CAR-T NXC-201 and the timing and results related clinical trials. These statements involve risks and uncertainties, and actual results may differ materially from any future results expressed or implied by the forward-looking statements. Forward-looking statements also include, but are not limited to, our plans, objectives, expectations and intentions and other statements that contain words such as “expects”, “contemplates”, “anticipates”, “plans”, “intends”, “believes”, “estimates”, “potential”, and variations of such words or similar expressions that convey the uncertainty of future events or outcomes, or that do not relate to historical matters. Those forward-looking statements involve known and unknown risks, uncertainties and other factors that could cause actual results to differ materially. Among those factors are: (i) the risk that the further data from the ongoing Phase 1/2 clinical trials for CAR-T NXC-201 will not be favorably consistent with the data readouts to date, (ii) the risk that the Company may not be able to continue the NEXICART-2 multi-site U.S. Phase 1/2 clinical trial; (iii) the risk that the Company may not be able to advance to registration-enabling studies for CAR-T NXC-201 or other product candidates, (iv) that success in early phases of pre-clinical and clinicals trials do not ensure later clinical trials will be successful; (v) that no drug product developed by the Company has received FDA pre-market approval or otherwise been incorporated into a commercial drug product, (vi) the risk that the Company may not be able to obtain additional working capital with which to continue the clinical trials for CAR-T NXC-201, or advance to the initiation of registration-enabling studies, for such product candidates as and when needed and (vii) those other risks disclosed in the section “Risk Factors” included in the Company’s Annual Report on Form 10-K filed with the SEC on March 25, 2025 and other periodic reports subsequently filed with the Securities and Exchange Commission. These reports are available at www.sec.gov. Immix Biopharma cautions that the foregoing list of important factors is not complete. Immix Biopharma cautions readers not to place undue reliance on any forward-looking statements. Immix Biopharma does not undertake, and specifically disclaims, any obligation to update or revise such statements to reflect new circumstances or unanticipated events as they occur, except as required by law. If we update one or more forward-looking statements, no inference should be drawn that we will make additional updates with respect to those or other forward-looking statements.

Contacts
Mike Moyer
LifeSci Advisors
mmoyer@lifesciadvisors.com

Company Contact
irteam@immixbio.com

FAQ

What were the clinical trial results for IMMX's NXC-201 in AL Amyloidosis?

NXC-201 achieved a 70% complete response rate (7/10 patients) in the NEXICART-2 Phase 1/2 trial, with no relapses and strong safety profile. The remaining 3 patients showed bone marrow MRD negativity.

How does NXC-201's efficacy compare to current AL Amyloidosis treatments?

NXC-201's 70% complete response rate significantly outperforms current treatments, which typically achieve less than 10% complete response rates in relapsed/refractory AL Amyloidosis.

What safety concerns were identified in the IMMX NXC-201 trial?

No major safety concerns were identified. The trial showed no neurotoxicity, no relapses, and only low-grade cytokine release syndrome was observed.

What are the next steps for IMMX's NXC-201 development?

Following the positive interim results, Immix Biopharma plans to submit a Biologics License Application (BLA) for FDA approval of NXC-201.

How many prior treatments did patients in the IMMX NXC-201 trial receive?

Patients in the trial had received a median of 4 prior lines of therapy, with a range of 2-12 treatments before receiving NXC-201.