Immix Biopharma Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for NXC-201, sterically-optimized CAR-T for relapsed/refractory AL Amyloidosis
Immix Biopharma (NASDAQ: IMMX) has received FDA Regenerative Medicine Advanced Therapy (RMAT) designation for NXC-201, their sterically-optimized CAR-T therapy for relapsed/refractory AL Amyloidosis. This designation follows positive proof-of-concept U.S. clinical data from the NEXICART-2 trial.
The RMAT designation, granted to less than half of applications in the past eight years, potentially accelerates the path to market approval through frequent FDA interactions and routes to Accelerated Approval and Priority Review. NXC-201 is currently the only CAR-T therapy in development for AL Amyloidosis, addressing an area with no FDA-approved drugs.
The company reports accelerated enrollment in the NEXICART-2 study following successful completion of the safety run-in segment, with the next update planned for H1 2025.
Immix Biopharma (NASDAQ: IMMX) ha ricevuto la designazione FDA di Terapia Avanzata in Medicina Rigenerativa (RMAT) per NXC-201, la loro terapia CAR-T ottimizzata stericamente per l'amiloidosi AL in recidiva/rifrangente. Questa designazione segue dati clinici positivi di prova-concetto negli Stati Uniti provenienti dallo studio NEXICART-2.
La designazione RMAT, concessa a meno della metà delle domande negli ultimi otto anni, potrebbe accelerare il percorso verso l'approvazione sul mercato attraverso interazioni frequenti con la FDA e vie per l'Approvazione Accelerata e Revisione Prioritaria. NXC-201 è attualmente l'unica terapia CAR-T in sviluppo per l'amiloidosi AL, affrontando un'area priva di farmaci approvati dalla FDA.
L'azienda riporta un arruolamento accelerato nello studio NEXICART-2 dopo il completamento con successo del segmento di sicurezza, con il prossimo aggiornamento pianificato per il primo semestre del 2025.
Immix Biopharma (NASDAQ: IMMX) ha recibido la designación de Terapia Avanzada en Medicina Regenerativa (RMAT) por parte de la FDA para NXC-201, su terapia CAR-T optimizada estéricamente para la amiloidosis AL en recaída/refractaria. Esta designación sigue a datos clínicos positivos de prueba de concepto en EE. UU. provenientes del ensayo NEXICART-2.
La designación RMAT, otorgada a menos de la mitad de las solicitudes en los últimos ocho años, potencialmente acelera el camino hacia la aprobación en el mercado a través de interacciones frecuentes con la FDA y vías para la Aprobación Acelerada y Revisión Prioritaria. NXC-201 es actualmente la única terapia CAR-T en desarrollo para la amiloidosis AL, abordando un área sin medicamentos aprobados por la FDA.
La empresa informa un reclutamiento acelerado en el estudio NEXICART-2 tras la exitosa finalización del segmento de seguridad, con la próxima actualización programada para el primer semestre de 2025.
Immix Biopharma (NASDAQ: IMMX)는 재발/불응 AL 아밀로이도시스에 대한 생체외 최적화 CAR-T 치료제인 NXC-201에 대해 FDA의 재생 의학 고급 치료(RMAT) 지정 승인을 받았습니다. 이 지정은 NEXICART-2 시험에서의 긍정적인 개념 증명 미국 임상 데이터를 기반으로 합니다.
RMAT 지정은 지난 8년 간의 신청 중 50% 미만에게 부여되며, FDA와의 빈번한 상호작용 및 가속 승인 및 우선 검토 경로를 통해 시장 승인으로의 경로를 가속화할 수 있습니다. NXC-201은 AL 아밀로이도시스를 치료하기 위해 개발 중인 유일한 CAR-T 치료제이며, FDA에서 승인된 약물이 없는 분야를 다룹니다.
회사는 안전성을 고려한 초기 단계 완료 후 NEXICART-2 연구의 모집이 가속화되고 있으며, 다음 업데이트는 2025년 상반기로 계획하고 있습니다.
Immix Biopharma (NASDAQ: IMMX) a reçu la désignation de Thérapie Avancée en Médecine Régénérative (RMAT) de la part de la FDA pour NXC-201, leur thérapie CAR-T optimisée stériquement pour l’amyloïdose AL en rechute/réfractaire. Cette désignation fait suite à des données cliniques américaines positives de preuve de concept issues de l'essai NEXICART-2.
La désignation RMAT, accordée à moins de la moitié des demandes au cours des huit dernières années, pourrait accélérer le chemin vers l'approbation sur le marché grâce à des interactions fréquentes avec la FDA et des voies pour une Approbation Accélérée et un Examen Prioritaire. NXC-201 est actuellement la seule thérapie CAR-T en développement pour l'amyloïdose AL, répondant à un domaine sans médicaments approuvés par la FDA.
L'entreprise rapporte que l'inscription dans l'étude NEXICART-2 a été accélérée suite à l'achèvement réussi du segment de sécurité, avec la prochaine mise à jour prévue pour le premier semestre 2025.
Immix Biopharma (NASDAQ: IMMX) hat die FDA-Zulassung für Regenerative Medizin mit dem Status Advanced Therapy (RMAT) für NXC-201, ihre sterisch optimierte CAR-T-Therapie für rezidivierende/refraktäre AL-Amyloidose, erhalten. Diese Zulassung folgt positiven Beweisen aus der klinischen Studie NEXICART-2 in den USA.
Die RMAT-Zulassung, die in den letzten acht Jahren weniger als der Hälfte der Anträge erhalten hat, könnte den Weg zur Marktzulassung durch häufige Interaktionen mit der FDA sowie durch Wege zur Beschleunigten Zulassung und Prioritätsprüfung beschleunigen. NXC-201 ist derzeit die einzige CAR-T-Therapie in der Entwicklung für AL-Amyloidose und spricht ein Gebiet an, für das es keine von der FDA genehmigten Medikamente gibt.
Das Unternehmen berichtet von einer beschleunigten Rekrutierung in der NEXICART-2-Studie nach erfolgreichem Abschluss des Sicherheitssegments, wobei das nächste Update für das erste Halbjahr 2025 geplant ist.
- Received FDA RMAT designation, a significant regulatory milestone granted to less than 50% of applicants
- NXC-201 is the only CAR-T therapy in development for AL Amyloidosis
- Accelerated enrollment in NEXICART-2 study following successful safety run-in
- Potential expedited path to market through FDA Accelerated Approval and Priority Review
- No FDA-approved products yet in portfolio
- Next clinical update not expected until H1 2025
Insights
The FDA's RMAT designation for NXC-201 marks a pivotal development for Immix Biopharma's novel CAR-T therapy. AL Amyloidosis, a rare and life-threatening condition where abnormal protein deposits damage organs, currently lacks FDA-approved treatments for relapsed/refractory cases, representing a significant unmet medical need.
Three critical aspects make this announcement particularly significant:
- With less than
50% of RMAT applications approved historically, this designation validates NXC-201's preliminary clinical evidence and potential therapeutic impact. - As the only CAR-T therapy in development for AL Amyloidosis, NXC-201 could establish a first-mover advantage in this untapped market. Its mention in the prestigious New England Journal of Medicine further validates its scientific merit.
- The accelerating enrollment in NEXICART-2 following successful safety run-in suggests growing physician confidence and patient demand, potentially expediting the development timeline.
The RMAT designation provides significant strategic advantages, including frequent FDA interactions and potential accelerated approval pathways. This could substantially reduce time-to-market and development costs, critical factors for a small-cap biotech company like Immix. The upcoming H1 2025 update on NEXICART-2 will be important in validating the therapy's potential and could serve as a significant catalyst for the company's valuation.
- FDA RMAT designation follows positive proof-of-concept U.S. clinical data from the NXC-201 NEXICART-2 clinical trial in relapsed/refractory AL Amyloidosis
- RMAT designation potentially streamlines the path to market approval by allowing frequent interactions with FDA and routes to FDA Accelerated Approval and Priority Review
- Enrollment in NEXICART-2 study accelerating; next update planned for H1 2025
LOS ANGELES,CA, Feb. 10, 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 that the U.S. Food and Drug Administration (FDA) has granted RMAT designation to sterically-optimized CAR-T NXC-201 for the treatment of relapsed/refractory AL amyloidosis. As of June 2024 public information, FDA approved less than half of RMAT applications submitted to the agency during the last eight years. FDA RMAT designation requires that a drug is an advanced regenerative medicine, targets a serious condition, with the potential to treat, modify, reverse, or cure, and preliminary clinical evidence has indicated that the drug has the potential to address these unmet medical needs.
“Receipt of FDA RMAT designation underscores the strength of our NXC-201 data and the potential for NXC-201 to provide a new treatment option for patients with relapsed/refractory AL amyloidosis, where no drugs are FDA approved today,” said Ilya Rachman, MD, PhD, Chief Executive Officer of Immix Biopharma. Gabriel Morris, Chief Financial Officer of Immix Biopharma, added, “We are also pleased to report that the pace of enrollment in NEXICART-2 has accelerated, following successful completion of the safety run-in segment. We look forward to sharing further information on our progress, including an update on NEXICART-2, in the first half of 2025.”
The RMAT designation is intended to accelerate the development and review of promising investigational products, including cell therapies. To qualify, a product must be designed to treat, modify, reverse, or cure a serious or life-threatening disease, with preliminary clinical evidence suggesting it can address unmet medical needs. The RMAT designation offers several key advantages, including early and frequent interactions with the FDA to discuss potential surrogate or intermediate endpoints, as well as strategies to meet post-approval requirements, potentially streamlining the path to market approval.
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 relapsed/refractory 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 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 NEXICART-2
NEXICART-2 (NCT06097832) is an open-label, single-arm, multi-site U.S. Phase 1b/2 dose expansion 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, supporting expansion into select immune-mediated 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 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 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 does the RMAT designation mean for IMMX's NXC-201 development?
When will IMMX release the next update for the NEXICART-2 trial?
What is the current competition for IMMX's NXC-201 in AL Amyloidosis treatment?
What percentage of RMAT applications have been approved by the FDA?
How is the NEXICART-2 trial progressing for IMMX?
