Immix Biopharma Advances CAR-T NXC-201 to Expansion Cohort Dose Level in U.S. AL Amyloidosis Trial NEXICART-2
Immix Biopharma (IMMX) has advanced its CAR-T therapy NXC-201 to the expansion cohort dose level in the U.S. NEXICART-2 trial for AL Amyloidosis. The study is now dosing at 450 million NXC-201 CAR+T cells, following successful completion of the first cohort at 150 million cells. Both doses have previously produced complete responses in clinical studies.
NXC-201 is the only one-time CAR-T treatment being studied for relapsed/refractory AL Amyloidosis in U.S. clinical trials. The NEXICART-2 study, led by Memorial Sloan Kettering Cancer Center, aims to evaluate the safety and efficacy of NXC-201 in patients with adequate cardiac function who haven't received prior BCMA-targeted therapy.
Previous data from an ex-US study showed a 92% overall response rate and a 28.0 month duration of response. AL Amyloidosis, affecting approximately 33,000 patients in the U.S., currently has no FDA-approved drugs for relapsed/refractory cases.
Immix Biopharma (IMMX) ha portato avanti la sua terapia CAR-T NXC-201 al livello di dose della coorte di espansione nel trial NEXICART-2 negli Stati Uniti per l'amiloidosi AL. Lo studio sta ora somministrando 450 milioni di cellule CAR+T NXC-201, dopo il completamento con successo della prima coorte di 150 milioni di cellule. Entrambe le dosi hanno precedentemente prodotto risposte complete negli studi clinici.
NXC-201 è l'unico trattamento CAR-T a una sola somministrazione attualmente in fase di studio per l'amiloidosi AL recidivante/refrattaria negli studi clinici statunitensi. Lo studio NEXICART-2, guidato dal Memorial Sloan Kettering Cancer Center, ha l'obiettivo di valutare la sicurezza e l'efficacia di NXC-201 in pazienti con adeguata funzione cardiaca che non hanno ricevuto precedenti terapie mirate al BCMA.
Dati precedenti da uno studio al di fuori degli Stati Uniti hanno mostrato un tasso di risposta complessivo del 92% e una durata della risposta di 28,0 mesi. L'amiloidosi AL, che colpisce circa 33.000 pazienti negli Stati Uniti, attualmente non ha farmaci approvati dalla FDA per i casi recidivanti/refrattari.
Immix Biopharma (IMMX) ha avanzado su terapia CAR-T NXC-201 al nivel de dosis de la cohorte de expansión en el ensayo NEXICART-2 en EE. UU. para Amiloidosis AL. El estudio ahora está administrando 450 millones de células CAR+T NXC-201, tras la finalización exitosa de la primera cohorte con 150 millones de células. Ambas dosis han producido previamente respuestas completas en estudios clínicos.
NXC-201 es el único tratamiento CAR-T único que se estudia para la amiloidosis AL recaída/refractaria en ensayos clínicos en EE. UU. El estudio NEXICART-2, dirigido por el Memorial Sloan Kettering Cancer Center, tiene como objetivo evaluar la seguridad y eficacia de NXC-201 en pacientes con función cardíaca adecuada que no han recibido terapia previa dirigida a BCMA.
Los datos previos de un estudio fuera de EE. UU. mostraron un tasa de respuesta global del 92% y una duración de respuesta de 28,0 meses. La amiloidosis AL, que afecta aproximadamente a 33,000 pacientes en EE. UU., actualmente no tiene medicamentos aprobados por la FDA para casos recaídos/refractarios.
Immix Biopharma (IMMX)는 알레르기성 아밀로이드증(AL 아밀로이드증)을 위한 미국 NEXICART-2 시험에서 CAR-T 치료제 NXC-201의 확장 코호트 용량 수준으로 전환했습니다. 이 연구는 1차 코호트에서 1억 5천만 세포가 성공적으로 완료된 후, 4억 5천만 NXC-201 CAR+T 세포를 투여하고 있습니다. 두 용량 모두 임상 연구에서 완전 반응을 나타낸 바 있습니다.
NXC-201은 미국 임상 시험에서 재발/내성 AL 아밀로이드증에 대해 연구되고 있는 유일한 단회 CAR-T 치료제입니다. Memorial Sloan Kettering Cancer Center가 주도하는 NEXICART-2 연구는 BCMA 표적 치료를 이전에 받지 않은 심장 기능이 충분한 환자에서 NXC-201의 안전성과 효능을 평가하는 것을 목표로 하고 있습니다.
이전 외국 연구에서 얻은 데이터에 따르면 92%의 전체 반응률과 반응 지속 시간 28.0개월을 보였습니다. AL 아밀로이드증은 미국에서 약 33,000명의 환자에게 영향을 미치며, 현재 재발/내성 사례에 대해 FDA 승인 약물이 없습니다.
Immix Biopharma (IMMX) a fait progresser sa thérapie CAR-T NXC-201 au niveau de dose de la cohorte d'expansion dans l'essai NEXICART-2 aux États-Unis pour l'amyloïdose AL. L'étude administre maintenant 450 millions de cellules CAR+T NXC-201, après avoir réussi à compléter la première cohorte de 150 millions de cellules. Les deux doses ont précédemment produit des réponses complètes dans des études cliniques.
NXC-201 est le seul traitement CAR-T à dose unique étudié pour l'amyloïdose AL en rechute/résistante dans des essais cliniques aux États-Unis. L'étude NEXICART-2, dirigée par le Memorial Sloan Kettering Cancer Center, vise à évaluer la sécurité et l'efficacité de NXC-201 chez les patients ayant une fonction cardiaque adéquate qui n'ont pas reçu de thérapie ciblée BCMA antérieure.
Des données antérieures provenant d'une étude hors des États-Unis ont montré un taux de réponse global de 92% et une durée de réponse de 28,0 mois. L'amyloïdose AL, qui touche environ 33 000 patients aux États-Unis, n'a actuellement aucun médicament approuvé par la FDA pour les cas de rechute/résistance.
Immix Biopharma (IMMX) hat seine CAR-T-Therapie NXC-201 im NEXICART-2-Studie in den USA auf die Dosierungsebene der Erweiterungsgruppe vorangebracht für AL-Amyloidose. Die Studie verabreicht jetzt 450 Millionen NXC-201 CAR+T-Zellen, nachdem die erste Gruppe erfolgreich mit 150 Millionen Zellen abgeschlossen wurde. Beide Dosen haben zuvor vollständige Antworten in klinischen Studien gezeigt.
NXC-201 ist die einmalige CAR-T-Behandlung, die derzeit in klinischen Studien in den USA für rezidivierende/refraktäre AL-Amyloidose untersucht wird. Die NEXICART-2-Studie, geleitet vom Memorial Sloan Kettering Cancer Center, hat zum Ziel, die Sicherheit und Wirksamkeit von NXC-201 bei Patienten mit ausreichender Herzfunktion, die keine vorherige BCMA-zielgerichtete Therapie erhalten haben, zu bewerten.
Frühere Daten aus einer Studie außerhalb der USA zeigten eine gesamtansprechrate von 92% und eine Ansprechdauer von 28,0 Monaten. AL-Amyloidose, die etwa 33.000 Patienten in den USA betrifft, hat derzeit keine von der FDA genehmigten Medikamente für rezidivierende/refraktäre Fälle.
- Advancement to expansion cohort dose level in NEXICART-2 trial
- NXC-201 is the only one-time CAR-T treatment for AL Amyloidosis in U.S. clinical trials
- 92% overall response rate in previous ex-US study
- 28.0 month duration of response in best responder from previous study
- Both dose levels have produced complete responses in prior studies
- None.
Insights
The advancement of NXC-201 to the expansion cohort dose level in the NEXICART-2 trial is a significant milestone for Immix Biopharma. This CAR-T therapy, targeting relapsed/refractory AL Amyloidosis, shows promise with a 92% overall response rate and a 28.0-month duration of response in previous studies. The expansion to a 450 million CAR+T cell dose after successfully completing the 150 million cell cohort indicates positive safety and efficacy signals.
The unmet medical need in AL Amyloidosis is substantial, with approximately 33,000 patients in the U.S. and no FDA-approved drugs for relapsed/refractory cases. NXC-201's potential as a one-time treatment option could be transformative for patient care. The robust enrollment and investigator enthusiasm suggest strong clinical interest, which could accelerate the development timeline.
Investors should note that while these developments are promising, CAR-T therapies face challenges in manufacturing, logistics and potential side effects. The upcoming interim and final read-outs will be critical in assessing NXC-201's true potential and its path to potential FDA approval.
Immix Biopharma's progress with NXC-201 could significantly impact its market position and financial outlook. As the only CAR-T therapy in development for AL Amyloidosis mentioned in a recent New England Journal of Medicine review, IMMX has a potential first-mover advantage in a niche market with high unmet need.
With a current market cap of
Investors should monitor cash burn rates and potential capital raises as clinical trials progress. Positive interim results could lead to increased investor interest and potential partnerships, which might alleviate financial pressures. However, the path to commercialization for cell therapies is long and capital-intensive, so risk assessment is crucial.
- Now dosing at dose expansion level of 450 million NXC-201 CAR+T cells. First cohort at 150 million CAR+T cells already successfully completed
- Each of these two doses have produced complete responses in prior clinical studies
- Lead site Memorial Sloan Kettering Cancer Center (MSKCC); 4 disclosed clinical sites so far
- NXC-201 is the only one-time CAR-T treatment option being studied for relapsed/refractory AL Amyloidosis in U.S. clinical trials
- AL Amyloidosis, a disorder of plasma cells that leads to progressive damage to the heart, kidney and liver, affects approximately 33,000 patients in the U.S.
LOS ANGELES, CA, Oct. 02, 2024 (GLOBE NEWSWIRE) -- Immix Biopharma, Inc. (“ImmixBio”, “Company”, “We” or “Us”, “IMMX”) (Nasdaq: IMMX), a clinical-stage biopharmaceutical company developing cell therapies for AL Amyloidosis and select immune-mediated diseases, today announced advances in the NEXICART-2 clinical study of its CAR-T NXC-201 for relapsed/refractory AL Amyloidosis, a plasma cell disorder that leads to progressive damage to the heart, kidney and liver, which currently affects approximately 33,000 patients in the U.S. Immix Biopharma’s sterically-optimized CAR-T NXC-201 is the only one-time CAR-T treatment option being studied for relapsed/refractory AL Amyloidosis in U.S. clinical trials.
“We are pleased to report that the NEXICART-2 study is making excellent progress. We are now one step closer to providing a new treatment option for patients with relapsed/refractory AL Amyloidosis, where no FDA drugs are approved today,” said Ilya Rachman, M.D., Ph.D., Chief Executive Officer of Immix Biopharma. “Robust enrollment reflects the enthusiasm of clinical investigators for CAR-T NXC-201.”
“Positive data from our ex-US study of NXC-201, the largest CAR-T clinical study in relapsed/refractory AL Amyloidosis to-date, showed a
The NEXICART-2 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. Advancement to the second dosing cohort of three patients at 450 million CAR-T cells will dose one patient with NXC-201 every 28-days, subsequently enabling multiple patients to be dosed per month in NEXICART-2.
Each of the NEXICART-2 dose levels have produced complete responses in relapsed/refractory AL Amyloidosis patients in the ex-U.S. NEXICART-1 study presented at ASGCT 2024.
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 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 dose expansion at 450 million CAR+T cells following. During dose expansion, multiple patients are allowed to be dosed per month. The NXC-201 dosing levels being evaluated in NEXICART-2 study have been 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 NXC-201
NXC-201 is a sterically-optimized BCMA-targeted chimeric antigen receptor T (CAR-T) cell therapy. Initial NXC-201 clinical data in relapsed/refractory AL Amyloidosis from Phase 1b/2 ex-U.S. study NEXICART-1 has demonstrated a high overall and complete response rate, as well as no neurotoxicity of any kind and short duration of cytokine release syndrome, presented at the American Society of Hematology (ASH) and the American Society of Gene and Cell Therapy (ASGCT) annual meetings.
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 select immune mediated diseases. The NXC-201 NEXICART-2 U.S. clinical trial builds on an existing 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 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 a high overall and complete response rate, as well as no neurotoxicity of any kind and short duration of cytokine release syndrome, presented at the American Society of Hematology (ASH) and the American Society of Gene and Cell Therapy (ASGCT) annual meetings. NXC-201 has been awarded Orphan Drug Designation (ODD) in the U.S. by the FDA and in the EU by the EMA in AL Amyloidosis. Learn more at www.immixbio.com and www.BeProactiveInAL.com.
Forward Looking Statements
This press release contains forward-looking statements regarding Immix Biopharma, Inc., including the potential benefits of our product candidate CAR-T NXC-201. Forward-looking statements 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. 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.
Contacts
Mike Moyer
LifeSci Advisors
mmoyer@lifesciadvisors.com
Company Contact
irteam@immixbio.com
FAQ
What is the current dosing level for NXC-201 in the NEXICART-2 trial for AL Amyloidosis?
How many patients are affected by AL Amyloidosis in the United States?
What was the overall response rate for NXC-201 in the ex-US study for AL Amyloidosis?
Which institution is the lead site for the NEXICART-2 trial of IMMX's NXC-201?
