Immix Biopharma Accelerates Enrollment in U.S. AL Amyloidosis Trial of NXC-201 CAR-T
Immix Biopharma (NASDAQ: IMMX) has successfully completed the six-patient Phase 1b safety run-in segment of its NEXICART-2 study for NXC-201, a CAR-T therapy targeting AL Amyloidosis. The trial, which evaluated three patients at 150 million CAR-T cells and three at 450 million cells, is now advancing to accelerated enrollment across U.S. study sites in January 2025.
The NEXICART-2 study consists of two segments: the completed six-patient safety run-in and a 34-patient dose expansion segment. NXC-201 is currently the only CAR-T therapy in development for AL Amyloidosis, a life-threatening disorder affecting approximately 33,000 patients in the U.S. The company previously announced positive data from the first four patients in December 2024 and expects to provide the next program update in Q1 2025.
Immix Biopharma (NASDAQ: IMMX) ha completato con successo il segmento di sicurezza di sei pazienti della Fase 1b del suo studio NEXICART-2 per NXC-201, una terapia CAR-T mirata all'amiloidosi AL. Lo studio, che ha valutato tre pazienti con 150 milioni di cellule CAR-T e tre con 450 milioni di cellule, ora passerà a un arruolamento accelerato in vari siti di studio negli Stati Uniti a gennaio 2025.
Lo studio NEXICART-2 è composto da due segmenti: il segmento di sicurezza completato di sei pazienti e un segmento di espansione della dose di 34 pazienti. NXC-201 è attualmente l'unica terapia CAR-T in fase di sviluppo per l'amiloidosi AL, una malattia potenzialmente letale che colpisce circa 33.000 pazienti negli Stati Uniti. L'azienda ha precedentemente annunciato dati positivi dai primi quattro pazienti nel dicembre 2024 e prevede di fornire il prossimo aggiornamento sul programma nel primo trimestre del 2025.
Immix Biopharma (NASDAQ: IMMX) ha completado con éxito el segmento de seguridad en fase 1b con seis pacientes de su estudio NEXICART-2 para NXC-201, una terapia CAR-T dirigida a la amiloidosis AL. El ensayo, que evaluó a tres pacientes con 150 millones de células CAR-T y a tres con 450 millones de células, ahora avanzará a un reclutamiento acelerado en los sitios de estudio de EE. UU. en enero de 2025.
El estudio NEXICART-2 consiste en dos segmentos: el segmento de seguridad de seis pacientes que ya se completó y un segmento de expansión de dosis con 34 pacientes. NXC-201 es actualmente la única terapia CAR-T en desarrollo para la amiloidosis AL, una enfermedad potencialmente mortal que afecta a aproximadamente 33.000 pacientes en EE. UU.. La empresa había anunciado anteriormente datos positivos de los primeros cuatro pacientes en diciembre de 2024 y espera proporcionar la próxima actualización del programa en el primer trimestre de 2025.
Immix Biopharma (NASDAQ: IMMX)는 AL 아밀로이드증을 표적으로 하는 CAR-T 치료제 NXC-201의 NEXICART-2 연구에서 6명의 환자를 포함한 1b상 안전성 시험을 성공적으로 완료했습니다. 이 시험은 150밀리온 CAR-T 세포를 가진 환자 3명과 450밀리온 세포를 가진 환자 3명을 평가하였으며, 2025년 1월부터 미국 연구 사이트에서 가속 등록을 진행할 예정입니다.
NEXICART-2 연구는 완료된 6명 환자의 안전성 시험과 34명 환자의 용량 확장 부분으로 구성됩니다. NXC-201은 AL 아밀로이드증에 대한 유일한 CAR-T 치료제 개발 중이며, 이 질환은 미국에서 약 33,000명의 환자에게 영향을 미치는 생명을 위협하는 질병입니다. 이 회사는 지난 2024년 12월에 첫 4명의 환자에 대한 긍정적인 데이터를 발표했으며, 2025년 1분기에 다음 프로그램 업데이트를 제공할 예정입니다.
Immix Biopharma (NASDAQ: IMMX) a réussi à achever le segment d'essai de sécurité de la phase 1b avec six patients dans son étude NEXICART-2 pour le NXC-201, une thérapie CAR-T ciblant l'amyloïdose AL. L'essai, qui a évalué trois patients avec 150 millions de cellules CAR-T et trois avec 450 millions de cellules, passe maintenant à une enrôlement accéléré sur les sites d'étude aux États-Unis en janvier 2025.
L'étude NEXICART-2 se compose de deux segments : le segment de sécurité achevé de six patients et un segment d'expansion de dose de 34 patients. NXC-201 est actuellement la seule thérapie CAR-T en développement pour l'amyloïdose AL, une maladie potentiellement mortelle touchant environ 33 000 patients aux États-Unis. La société a précédemment annoncé des données positives des quatre premiers patients en décembre 2024 et s'attend à fournir la prochaine mise à jour du programme au premier trimestre de 2025.
Immix Biopharma (NASDAQ: IMMX) hat erfolgreich das Sicherheitssegment der Phase 1b mit sechs Patienten der NEXICART-2-Studie für NXC-201, eine CAR-T-Therapie zur Behandlung von AL-Amyloidose, abgeschlossen. Die Studie, die drei Patienten mit 150 Millionen CAR-T-Zellen und drei mit 450 Millionen Zellen bewertet hat, wird nun im Januar 2025 zu einer beschleunigten Rekrutierung an verschiedenen US-Studienstätten übergehen.
Die NEXICART-2-Studie besteht aus zwei Segmenten: dem abgeschlossenen Sicherheitssegment mit sechs Patienten und einem Dosiserweiterungssegment mit 34 Patienten. NXC-201 ist derzeit die einzige CAR-T-Therapie, die für die AL-Amyloidose entwickelt wird, eine lebensbedrohliche Erkrankung, die ungefähr 33.000 Patienten in den USA betrifft. Das Unternehmen hatte zuvor positive Daten von den ersten vier Patienten im Dezember 2024 bekannt gegeben und plant, im ersten Quartal 2025 das nächste Programm-Update bereitzustellen.
- Successfully completed Phase 1b safety run-in with 6 patients
- Advancing to accelerated enrollment phase across U.S. sites
- Only CAR-T therapy in development for AL Amyloidosis
- Previous doses produced complete responses in ex-US NEXICART-1 study
- Large addressable market of 33,000 patients in the U.S.
- No FDA-approved drugs currently available for relapsed/refractory AL Amyloidosis
- Extended timeline for next update (Q1 2025)
Insights
This clinical milestone marks a important advancement in IMMX's NXC-201 development program. The successful completion of the safety run-in phase with 6 patients demonstrates two critical aspects: the therapy's safety profile at both 150M and 450M CAR-T cell doses and the company's ability to execute its clinical development strategy. The progression to expanded enrollment across multiple sites significantly de-risks the program.
The AL Amyloidosis market represents a substantial opportunity with 33,000 U.S. patients and no FDA-approved treatments for relapsed/refractory cases. NXC-201's position as the only CAR-T therapy in development for this indication provides a significant first-mover advantage. Publication recognition in the prestigious New England Journal of Medicine adds credibility to the program and could facilitate patient recruitment.
The planned Q1 2025 update will be pivotal - investors should watch for efficacy data, particularly complete response rates, which could validate the therapy's potential market position. The accelerated enrollment could expedite the path to potential FDA submission if positive results continue.
The successful safety evaluation at both dose levels is particularly noteworthy for CAR-T therapy in AL Amyloidosis. The ability to safely administer 450M CAR-T cells - the higher dose that previously showed complete responses - suggests a potentially optimal therapeutic window. The completion of post-dosing safety evaluations in all six patients without apparent issues indicates a manageable safety profile, important for CAR-T therapies which can have significant side effects.
The multi-site expansion phase targeting 34 additional patients is well-designed to generate robust efficacy data. The previous complete responses observed in NEXICART-1 at these dose levels provide encouraging precedent. For a rare disease like AL Amyloidosis, this sample size is substantial and could provide compelling evidence for regulatory submission if positive outcomes are maintained.
- Successfully dosed 6 patients in safety run-in segment, now accelerating enrollment
- Positive data from first four patients announced December 2024
- NXC-201 is the only one-time CAR-T therapy in development for AL Amyloidosis
- Next program update Q1 2025
- AL Amyloidosis is a life-threatening disorder of plasma cells in the bone marrow affecting ~33,000 patients in the U.S.
LOS ANGELES, Jan. 07, 2025 (GLOBE NEWSWIRE) -- Immix Biopharma, Inc. (Nasdaq: IMMX) (“ImmixBio”, “Company”, “We” or “Us”, “IMMX”), a clinical-stage biopharmaceutical company developing cell therapies for AL Amyloidosis and select immune-mediated diseases, today announced successful completion of the six-patient Phase 1b safety run-in segment in the U.S. NEXICART-2 study of NXC-201, an investigational CAR-T therapy, in patients relapsed/refractory (R/R) AL Amyloidosis. Achievement of this milestone is expected to accelerate enrollment across U.S. study sites beginning in January 2025.
NEXICART-2 is an open-label study designed to evaluate NXC-201 in patients with R/R AL Amyloidosis. The study has two segments: a six-patient “safety run-in” segment and a 34-patient dose expansion segment. The Phase 1b “safety run-in” segment dosed three patients at 150 million CAR-T cells and three patients at 450 million CAR-T cells (these doses also produced complete responses in the prior ex-US NEXICART-1 study). All six patients successfully completed a post-dosing safety evaluation, and enrollment can now proceed across U.S. study sites at 450 million CAR-T cells.
“We are pleased to report the successful completion of the NEXICART-2 safety run-in segment. Achievement of this milestone positions us to accelerate enrollment in the NEXICART-2 study and brings us one step closer to providing a new treatment option for patients with relapsed/refractory AL Amyloidosis, where no drugs are currently FDA approved,” said Ilya Rachman, MD, PhD, Chief Executive Officer of Immix Biopharma. Gabriel Morris, Chief Financial Officer of Immix Biopharma, added, “We credit the resolute efforts of our investigators, sites, and team as we continue with robust enrollment in our multi-site U.S. NEXICART-2 study. We are on track for the next program update in Q1 2025.”
NXC-201 is the only CAR-T therapy currently in development in AL Amyloidosis, mentioned in a review article entitled “Systemic Light Chain Amyloidosis” published in June, 2024 New England Journal of Medicine .
About NXC-201
NXC-201 is a sterically-optimized BCMA-targeted chimeric antigen receptor T (CAR-T) cell therapy. Initial data from Phase 1b/2 ex-U.S. study NEXICART-1 has demonstrated high complete response rates and no neurotoxicity of any kind in AL Amyloidosis.
NXC-201 is being studied in a comprehensive U.S. clinical development program for the treatment of patients with relapsed/refractory AL amyloidosis, with the potential to expand into select immune-mediated diseases. The NXC-201 NEXICART-2 (NCT06097832) U.S. clinical trial builds on a robust clinical dataset. NXC-201 has been awarded Orphan Drug Designation (ODD) in AL Amyloidosis by the US FDA and in the EU by the EMA.
About NEXICART-2
NEXICART-2 (NCT06097832) is an open-label, single-arm, multi-site U.S. Phase 1b/2 clinical trial of 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 study is designed with a standard 6 patient safety-run in to evaluate two doses (three patients each at 150 million CAR+T cells and 450 million CAR+T cells) (both dose levels were evaluated in the NEXICART-1 study and have produced complete responses in relapsed/refractory AL Amyloidosis patients). The study aims to evaluate the safety and efficacy of NXC-201. Primary endpoints are complete response rate and overall response rate, according to consensus recommendations (Palladini et al. 2012).
About AL Amyloidosis
AL amyloidosis is caused by abnormal plasma cells in the bone marrow, which produce misfolded amyloid proteins that build-up in the heart, kidney, liver, and other organs. This build-up causes progressive and widespread damage to multiple organs, including heart failure, and leads to high mortality rates.
The U.S. observed prevalence of relapsed/refractory AL Amyloidosis is estimated to be growing at
The Amyloidosis market was
About Immix Biopharma, Inc.
Immix Biopharma, Inc. (ImmixBio) (Nasdaq: IMMX) is a clinical-stage biopharmaceutical company developing cell therapies for AL Amyloidosis and select immune-mediated 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. Phase 1b/2 trial NEXICART-2 (NCT06097832) as well as the ex-U.S. study NEXICART-1 (NCT04720313). NXC-201 has demonstrated no neurotoxicity of any kind in AL Amyloidosis and short duration of cytokine release syndrome (CRS), supporting expansion into select immune-mediated diseases. NXC-201 has been awarded Orphan Drug Designation (ODD) in AL Amyloidosis by the US 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 CAR-T product candidate, 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 further data from the ongoing Phase 1b/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 1b/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 29, 2024 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 risk 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 are the results of IMMX's NEXICART-2 safety run-in trial for NXC-201?
How many patients will be enrolled in IMMX's NEXICART-2 expansion phase?
When will IMMX release the next update for the NXC-201 AL Amyloidosis trial?
What is the market size for IMMX's AL Amyloidosis treatment in the US?
What makes IMMX's NXC-201 unique in the AL Amyloidosis treatment landscape?
