California Institute for Regenerative Medicine Awards Funding for CAR-T NXC-201 U.S. AL Amyloidosis Clinical Trial (NEXICART-2)
Immix Biopharma (Nasdaq: IMMX) has been awarded an $8 million CLIN2 grant from the California Institute for Regenerative Medicine (CIRM) to support the clinical development of NXC-201, a CAR-T cell therapy for relapsed/refractory AL Amyloidosis. The funding will be used for the NEXICART-2 U.S. clinical trial, which aims to evaluate the safety and efficacy of NXC-201 in patients with adequate cardiac function who have not received prior BCMA-targeted therapy.
The decision follows promising results from the ex-U.S. NEXICART-1 study, which showed a 92% overall response rate in relapsed/refractory AL Amyloidosis patients. NXC-201 is currently the only CAR-T therapy in development for AL Amyloidosis, a rare disease with treatment options. The therapy's potential has been recognized in a recent New England Journal of Medicine review article.
Immix Biopharma (Nasdaq: IMMX) ha ricevuto un finanziamento CLIN2 di 8 milioni di dollari dall'Istituto della California per la Medicina Rigenerativa (CIRM) per supportare lo sviluppo clinico di NXC-201, una terapia CAR-T per l'amiloidosi AL recidivante/refrattaria. Il finanziamento sarà utilizzato per il trial clinico NEXICART-2 negli Stati Uniti, volto a valutare la sicurezza e l'efficacia di NXC-201 in pazienti con funzione cardiaca adeguata che non hanno ricevuto terapie precedenti mirate al BCMA.
Questa decisione segue risultati promettenti dallo studio NEXICART-1 al di fuori degli Stati Uniti, che ha mostrato un tasso di risposta complessivo del 92% nei pazienti con amiloidosi AL recidivante/refrattaria. NXC-201 è attualmente l'unica terapia CAR-T in sviluppo per l'amiloidosi AL, una malattia rara con poche opzioni di trattamento. Il potenziale della terapia è stato riconosciuto in un recente articolo di revisione pubblicato nel New England Journal of Medicine.
Immix Biopharma (Nasdaq: IMMX) ha recibido una subvención CLIN2 de 8 millones de dólares del Instituto de Medicina Regenerativa de California (CIRM) para apoyar el desarrollo clínico de NXC-201, una terapia de células CAR-T para la amiloidosis AL recidivante/refractaria. La financiación se utilizará para el ensayo clínico NEXICART-2 en EE. UU., que tiene como objetivo evaluar la seguridad y la eficacia de NXC-201 en pacientes con función cardíaca adecuada que no han recibido terapias anteriores dirigidas al BCMA.
La decisión sigue a resultados prometedores del estudio NEXICART-1 fuera de EE. UU., que mostró una tasa de respuesta general del 92% en pacientes con amiloidosis AL recidivante/refractaria. NXC-201 es actualmente la única terapia CAR-T en desarrollo para la amiloidosis AL, una enfermedad rara con pocas opciones de tratamiento. El potencial de la terapia ha sido reconocido en un reciente artículo de revisión del New England Journal of Medicine.
Immix Biopharma (Nasdaq: IMMX)는 800만 달러의 CLIN2 보조금을 캘리포니아 재생의학 연구소(CIRM)로부터 수여받아 NXC-201의 임상 개발을 지원하게 됩니다. NXC-201은 재발성/불응성 AL 아밀로이드증에 대한 CAR-T 세포 치료법입니다. 이 자금은 NEXICART-2 미국 임상 시험에 사용되며, 이는 BCMA 타겟 치료를 받지 않은 적절한 심장 기능을 가진 환자에서 NXC-201의 안전성과 유효성을 평가하는 것을 목표로 합니다.
이 결정은 AL 아밀로이드증 환자에서 92%의 전체 반응률을 보인 미국 외 NEXICART-1 연구의 유망한 결과에 따른 것입니다. NXC-201은 현재 AL 아밀로이드증에 대해 개발 중인 유일한 CAR-T 치료법으로, 치료 옵션이 제한된 희귀 질환입니다. 최근 뉴잉글랜드 의학 저널 리뷰 기사에서도 치료의 잠재력이 인정되었습니다.
Immix Biopharma (Nasdaq: IMMX) a reçu un financement CLIN2 de 8 millions de dollars de l'Institut californien de médecine régénérative (CIRM) pour soutenir le développement clinique de NXC-201, une thérapie CAR-T pour l'amylose AL récalcitrante/récidivante. Ce financement sera utilisé pour l'
Cette décision fait suite à des résultats prometteurs de l'étude NEXICART-1 en dehors des États-Unis, qui a montré un taux de réponse global de 92% chez des patients atteints d'amylose AL récalcitrante/récidivante. NXC-201 est actuellement la seule thérapie CAR-T en développement pour l'amylose AL, une maladie rare avec peu d'options de traitement. Le potentiel de cette thérapie a été reconnu dans un article de revue récent du New England Journal of Medicine.
Immix Biopharma (Nasdaq: IMMX) hat einen CLIN2-Zuschuss in Höhe von 8 Millionen Dollar vom California Institute for Regenerative Medicine (CIRM) erhalten, um die klinische Entwicklung von NXC-201 zu unterstützen, einer CAR-T-Zelltherapie für rezidivierende/refraktäre AL-Amyloidose. Die Finanzierung wird für die NEXICART-2-Studie in den USA eingesetzt, die darauf abzielt, die Sicherheit und Wirksamkeit von NXC-201 bei Patienten mit ausreichender Herzfunktion zu evaluieren, die zuvor keine BCMA-targetierte Therapie erhalten haben.
Die Entscheidung folgt auf vielversprechende Ergebnisse der NEXICART-1-Studie außerhalb der USA, die bei Patienten mit rezidivierender/refraktärer AL-Amyloidose eine Gesamtansprechrate von 92% zeigte. NXC-201 ist derzeit die einzige CAR-T-Therapie, die für AL-Amyloidose entwickelt wird, einer seltenen Erkrankung mit wenigen Behandlungsoptionen. Das Potenzial dieser Therapie wurde in einem kürzlich veröffentlichten Übersichtsartikel im New England Journal of Medicine anerkannt.
- $8 million CLIN2 grant awarded by CIRM for NXC-201 development
- 92% overall response rate in NEXICART-1 study for relapsed/refractory AL Amyloidosis
- Best responder showed 28.0 month duration of response in NEXICART-1 study
- NXC-201 is the only CAR-T therapy in development for AL Amyloidosis
- Recognition in New England Journal of Medicine review article
- None.
Insights
The
From a financial perspective, this grant provides several benefits:
- Cash Conservation: The grant allows Immix Biopharma to advance its clinical trials without depleting its own cash reserves, potentially extending its runway.
- Validation: CIRM's support lends credibility to Immix Biopharma's research, which could attract additional investors or partners.
- Reduced Dilution Risk: By securing non-dilutive funding, the company avoids the need for immediate equity financing, protecting shareholder value.
However, investors should note that while this grant is positive, it doesn't guarantee commercial success. The company still faces significant clinical and regulatory hurdles before potential commercialization. Additionally, the grant's impact on the company's overall financial position should be considered in the context of its total cash burn rate and development timeline.
The CIRM grant for NXC-201's development in AL Amyloidosis is a noteworthy advancement in the field of rare disease treatment. Several aspects of this news are particularly intriguing from a medical research perspective:
- Unmet Medical Need: AL Amyloidosis currently lacks FDA-approved treatments, making NXC-201 a potential game-changer if successful.
- Promising Early Results: The
92% overall response rate in the NEXICART-1 study is impressive, especially considering the 28-month duration of response in the best responder. - Novel Approach: As the only CAR-T therapy in development for AL Amyloidosis, NXC-201 represents a innovative treatment modality for this condition.
The mention of NXC-201 in the New England Journal of Medicine further underscores its potential significance. However, it's important to remember that early-stage results don't always translate to late-stage success or FDA approval. The upcoming NEXICART-2 study will be critical in confirming the therapy's safety and efficacy in a U.S. patient population. Investors should closely monitor the progress of this trial, as its results could significantly impact Immix Biopharma's future prospects in the competitive CAR-T therapy landscape.
LOS ANGELES, CA, July 25, 2024 (GLOBE NEWSWIRE) -- Immix Biopharma, Inc. (“ImmixBio”, “Company”, “We” or “Us”, “IMMX”) (Nasdaq: IMMX), a clinical-stage biopharmaceutical company trailblazing cell therapies in AL Amyloidosis and autoimmune disease, today announced that the California Institute for Regenerative Medicine (CIRM) has awarded Immix Biopharma cell therapy division Nexcella an
“AL amyloidosis is an unmet medical need, and current approved therapies for this rare disease are mostly not well tolerated and have not led to sustained remissions,” said Dr. Abla Creasey, PhD, Vice President of Therapeutics Development at CIRM. “This one-time therapy would be an innovative treatment for patients with AL Amyloidosis, and the preliminary data are encouraging.”
“We are grateful for the recognition from CIRM of the importance of NXC-201 in relapsed/refractory AL Amyloidosis, for which there are no FDA approved drugs today,” said Ilya Rachman, M.D., Ph.D., Chief Executive Officer of Immix Biopharma. Gabriel Morris, Chief Financial Officer of Immix Biopharma, added, “We are excited to join California’s thriving CIRM biotechnology innovation ecosystem as we proceed on-plan with NEXICART-2.”
The NEXICART-2 U.S. study is intended to evaluate the safety and efficacy of NXC-201 in relapsed/refractory AL Amyloidosis patients with adequate cardiac function who have not been exposed to prior BCMA-targeted therapy. The study builds on positive data from the initial ex-U.S. study, NEXICART-1, presented at the 27th Annual Meeting of The American Society of Gene and Cell Therapy (ASGCT 2024) which showed a
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 the California Institute for Regenerative Medicine (CIRM)
At CIRM, we never forget that we were created by the people of California to accelerate stem cell treatments to patients with unmet medical needs, and act with a sense of urgency to succeed in that mission. To meet this challenge, our team of highly trained and experienced professionals actively partners with both academia and industry in a hands-on, entrepreneurial environment to fast track the development of today’s most promising stem cell technologies.
With
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 adequate cardiac function 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), with the potential for further escalation to 800 million CAR+T cells (all 3 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 in this patient population. Primary endpoints are complete response rate and overall response rate, according to consensus recommendations (Palladini et al. 2012).
About NEXICART-1
NEXICART-1 (NCT04720313) is an open-label, ex-U.S. Phase 1b/2a clinical trial of NXC-201 (formerly HBI0101) in patients with relapsed/refractory multiple myeloma and relapsed/refractory AL amyloidosis (including AL Amyloidosis patients with impaired cardiac function and including AL Amyloidosis patients exposed to prior BCMA-targeted therapy). The primary objective of the study is to characterize the safety and efficacy, as well as confirm the recommended Phase 2 dose (RP2D) of NXC-201 (which has already been confirmed). NEXICART-1 clinical results, most recently from ASGCT 2024, are available at https://immixbio.com/pipeline/#publications.
About NXC-201
NXC-201 is a sterically-optimized BCMA-targeted chimeric antigen receptor T (CAR-T) cell therapy. Initial data from Phase 1b/2a ex-U.S. study NEXICART-1 has demonstrated short duration of cytokine release syndrome (CRS) and no grade 3 or delayed neurotoxicity in high-volume disease, as well as short duration CRS and no neurotoxicity of any kind in AL Amyloidosis.
NXC-201 is being studied in a comprehensive clinical development program for the treatment of patients with relapsed/refractory AL amyloidosis, with the potential to expand into autoimmune indications. The NXC-201 NEXICART-2 U.S. clinical trial builds on a robust clinical dataset. NXC-201 has been awarded Orphan Drug Designation (ODD) in the US by the FDA and in the EU by the EMA in AL Amyloidosis.
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 trailblazing cell therapies in AL Amyloidosis and autoimmune 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/2a trial NEXICART-2 (NCT06097832) as well as the ex-U.S. study NEXICART-1 (NCT04720313). NXC-201 has demonstrated short duration of cytokine release syndrome (CRS) and no grade 3 or delayed neurotoxicity in high-volume disease, as well as short duration CRS and no neurotoxicity of any kind in AL Amyloidosis, supporting expansion into autoimmune indications. NXC-201 has been awarded Orphan Drug Designation (ODD) in the US by the FDA and in the EU by the EMA in AL Amyloidosis. Learn more at www.immixbio.com and www.BeProactiveInAL.com.
About Nexcella, Inc.
As of May 2024, Nexcella, Inc. is a wholly-owned subsidiary of, and the cell therapy division of, Immix Biopharma, Inc. (ImmixBio) (Nasdaq: IMMX).
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 receipt of, timing of receipt, finalization of the terms of, and allocation of funds in connection with, the grant discussed above and potential benefits of our product candidate CAR-T NXC-201. 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/2a clinical trials for CAR-T NXC-201 will not be favorably consistent with the initial data initial data readouts, (ii) the risk that the Company may not be able to advance to registration-enabling studies for CAR-T NXC-201 or other product candidates, (iii) that success in early phases of pre-clinical and clinicals trials do not ensure later clinical trials will be successful; (iv) that no drug product developed by the Company has received FDA pre-market approval or otherwise been incorporated into a commercial drug product, (v) 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 is the purpose of the $8 million CIRM grant awarded to Immix Biopharma (IMMX)?
What were the results of the NEXICART-1 study for NXC-201 in AL Amyloidosis patients?
How is NXC-201 unique in the treatment of AL Amyloidosis?
What is the focus of the NEXICART-2 U.S. clinical trial for Immix Biopharma's (IMMX) NXC-201?
