Immix Biopharma Announces 75% Complete Response Rate (n=16); 31.5 months Best Response Duration (ongoing) for CAR-T NXC-201 in Relapsed/Refractory AL Amyloidosis Patients at ASH 2024
Immix Biopharma (IMMX) has presented updated Phase 1/2 clinical data for NXC-201 in treating relapsed/refractory AL Amyloidosis at ASH 2024. The study showed a 75% complete response rate (12/16 patients) with a median of 4 prior therapy lines. The best responder maintained a complete response for 31.5 months ongoing.
Key results from 16 patients include: 94% overall response rate, 62% organ response rate, and favorable safety profile with no ICANS events. Patient characteristics showed 81% had cardiac involvement, 38% had NYHA stage 3/4 heart failure, and 31% had Mayo stage 3 AL amyloidosis. The treatment demonstrated manageable cytokine release syndrome with a median duration of 2 days.
Immix Biopharma (IMMX) ha presentato dati aggiornati della fase 1/2 dello studio clinico per NXC-201 nel trattamento dell'amiloidosi AL recidivante/ripetitiva durante l'ASH 2024. Lo studio ha mostrato un tasso di risposta completa del 75% (12/16 pazienti) con una mediana di 4 linee di trattamento precedenti. Il miglior rispondente ha mantenuto una risposta completa per 31,5 mesi in corso.
I risultati chiave di 16 pazienti includono: tasso di risposta complessivo del 94%, 62% di tasso di risposta organica e un profilo di sicurezza favorevole senza eventi di ICANS. Le caratteristiche dei pazienti mostrano che l'81% aveva coinvolgimento cardiaco, il 38% aveva insufficienza cardiaca di stadio NYHA 3/4 e il 31% aveva amiloidosi AL di stadio Mayo 3. Il trattamento ha dimostrato una sindrome da rilascio di citochine gestibile con una durata mediana di 2 giorni.
Immix Biopharma (IMMX) ha presentado datos clínicos actualizados de la fase 1/2 para NXC-201 en el tratamiento de la amiloidosis AL en recaída/refractaria en el ASH 2024. El estudio mostró una tasa de respuesta completa del 75% (12/16 pacientes) con una mediana de 4 líneas de tratamiento previas. El mejor respondedor mantuvo una respuesta completa durante 31.5 meses.
Los resultados clave de 16 pacientes incluyen: tasa de respuesta global del 94%, 62% de tasa de respuesta orgánica y un perfil de seguridad favorable sin eventos de ICANS. Las características de los pacientes mostraron que el 81% tenía afectación cardíaca, el 38% tenía insuficiencia cardíaca en estadío NYHA 3/4 y el 31% tenía amiloidosis AL en estadío Mayo 3. El tratamiento demostró un síndrome de liberación de citoquinas manejable con una duración mediana de 2 días.
Immix Biopharma (IMMX)는 2024 ASH에서 재발성/치료 저항성 AL 아밀로이드증 치료를 위한 NXC-201의 1/2상 임상 데이터 업데이트를 발표했습니다. 연구 결과 75%의 완전 반응률 (16명 중 12명)과 4개의 이전 치료 라인의 중앙값이 나타났습니다. 최고의 반응자는 현재까지 31.5개월 동안 완전 반응을 유지했습니다.
16명 환자로부터의 주요 결과는: 전체 반응률 94%, 62%의 장기 반응률, ICANS 사건이 없는 우수한 안전성 프로필입니다. 환자 특성에서는 81%가 심장 관련 문제가 있었고, 38%가 NYHA 3/4 단계의 심부전, 31%가 Mayo 3단계 AL 아밀로이드증이 있었습니다. 치료는 중앙값 2일 동안 관리 가능한 사이토카인 방출 증후군을 나타냈습니다.
Immix Biopharma (IMMX) a présenté des données cliniques mises à jour de phase 1/2 pour NXC-201 dans le traitement de l'amyloïdose AL récidivante/réfractaire lors de l'ASH 2024. L'étude a montré un taux de réponse complet de 75% (12/16 patients) avec une médiane de 4 lignes de thérapie précédentes. Le meilleur répondant a maintenu une réponse complète pendant 31,5 mois.
Les résultats clés des 16 patients comprennent : taux de réponse global de 94%, taux de réponse organique de 62 % et un profil de sécurité favorable sans événements ICANS. Les caractéristiques des patients ont montré que 81 % avaient une implication cardiaque, 38 % avaient une insuffisance cardiaque au stade NYHA 3/4, et 31 % avaient une amyloïdose AL au stade Mayo 3. Le traitement a démontré un syndrome de libération de cytokines gérable avec une durée médiane de 2 jours.
Immix Biopharma (IMMX) hat aktualisierte klinische Daten der Phase 1/2 für NXC-201 zur Behandlung von rezidivierenden/refraktären AL-Amyloidose auf dem ASH 2024 vorgestellt. Die Studie zeigte eine Vollansprechrate von 75% (12 von 16 Patienten) mit einer Medianzahl von 4 vorherigen Therapielinien. Der beste Ansprecher hielt eine vollständige Antwort über 31,5 Monate aufrecht.
Schlüsselresultate von 16 Patienten umfassen: eine Gesamtansprechrate von 94%, eine Organansprechrate von 62% und ein günstiges Sicherheitsprofil ohne ICANS-Ereignisse. Die Patientenmerkmale zeigten, dass 81% eine Herzbeteiligung hatten, 38% in NYHA-Stadium 3/4 Herzinsuffizienz hatten und 31% eine AL-Amyloidose im Stadium Mayo 3 hatten. Die Behandlung zeigte ein handhabbares Zytokinfreisetzungssyndrom mit einer medianen Dauer von 2 Tagen.
- High complete response rate of 75% (12/16 patients)
- Strong overall response rate of 94% (15/16 patients)
- Significant duration of response up to 31.5 months (ongoing)
- Favorable safety profile with no ICANS events
- 62% organ response rate in evaluable patients
- None.
Insights
The updated Phase 1/2 trial results for NXC-201 in relapsed/refractory AL Amyloidosis demonstrate remarkable efficacy with a 75% complete response rate and durable responses extending up to 31.5 months. The high response rates in heavily pretreated patients (median 4 prior lines) with significant cardiac involvement (81%) are particularly impressive.
The safety profile appears favorable with manageable cytokine release syndrome and notably, no cases of neurotoxicity (ICANS). This is important for AL amyloidosis patients who often have compromised organ function. The 94% overall response rate and 62% organ response rate suggest potential disease-modifying effects beyond just hematologic responses.
These results position NXC-201 as a promising therapy in an indication with no FDA-approved treatments for relapsed/refractory disease. The durability of responses and high MRD negativity rate (56%) suggest potential for long-term disease control.
This clinical data release represents a significant milestone for IMMX, potentially accelerating the path to market for NXC-201 in an indication with no approved therapies. The robust efficacy data in a difficult-to-treat patient population could translate to substantial market opportunity.
With a market cap of just
Key value drivers include the high complete response rate, manageable safety profile and potential first-mover advantage in the relapsed/refractory setting. These factors could make IMMX an attractive partnership or acquisition target for larger pharmaceutical companies.
75% (12/16) complete response (CR) rate observed in standard of care (Dara-CyBorD) relapsed/refractory AL Amyloidosis patients with median 4 lines of prior therapy in updated Phase 1/2 data as of December 9, 2024- Best responder duration of response was 31.5 months with complete response ongoing as of December 9, 2024
- Conference call to discuss results Tuesday, December 10, 2024 4:30 p.m. ET at https://zoom.us/j/94736340854?pwd=LTBtu2LuvwSb6S6ISuH5yKTDLsI2vt.1
LOS ANGELES, CA, Dec. 10, 2024 (GLOBE NEWSWIRE) -- Immix Biopharma, Inc. (“ImmixBio”, “Company”, “We” or “Us” or ”IMMX”), a clinical-stage biopharmaceutical company developing cell therapies for AL Amyloidosis and select immune-mediated diseases, today announced that new NXC-201 NEXICART-1 clinical data in relapsed/refractory AL Amyloidosis has been presented at 66th American Society of Hematology (ASH) Annual Meeting being held in San Diego, California. The updated results include follow-up and clinical data from 3 new NEXICART-1 patients.
“We are encouraged by the updated NXC-201 results being presented by our academic collaborators at ASH 2024. We believe the high percentage of complete responders, combined with the consistent, attractive tolerability profile is critically important in relapsed/refractory AL Amyloidosis. This expanded NXC-201 dataset continues to bolster our leadership in relapsed/refractory AL Amyloidosis, where no drugs are FDA approved 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 looking forward to bringing this promising therapy to U.S. relapsed/refractory AL Amyloidosis patients.”
Immix Biopharma Announces
Immix Biopharma, Inc. (Nasdaq:IMMX)
At the NXC-201 ASH 2024 oral presentation, data were presented from 16 relapsed/refractory AL amyloidosis patients (including 3 new patients) in the ongoing Phase 1b/2 NEXICART-1 study, with median 4 lines of therapy prior to NXC-201. Patients were infused with CAR+T cells at doses of 150 x 106 (n=1), 450 x 106 (n=2), and 800 x 106 (n=13).
Patient characteristics:
81% (13/16) had cardiac involvement38% (6/16) had New York Heart Association (NYHA) stage 3 or 4 heart failure (3 stage 4, 3 stage 3)
31% (5/16) had Mayo stage 3 (1 stage 3b, 4 stage 3a) AL amyloidosis disease44% (7/16) had t(11;14) translocation- Relapsed/refractory to a median 4 lines of prior therapy (range: 3-10)
Safety and efficacy data:
- Overall response rate of
94% (15/16) - Complete response rate of
75% (12/16) (9 out of 16 were MRD- 10-5) - Organ response rate of
62% (8/13 evaluable) - Best responder had a duration of response of 31.5 months as of December 9, 2024, with complete response ongoing
- There were no immune effector cell-associated neurotoxicity syndrome (ICANS) events
- Median CRS duration was 2 days (range: 1-5):
- No grade 4 cytokine release syndrome (CRS) events
- 2 experienced no CRS; 3 experienced grade 1 CRS; 8 Experienced grade 2 CRS; 3 experienced grade 3 CRS
The NXC-201 66th American Society of Hematology Meeting oral presentation video can be accessed on the ASH website: https://annualmeeting.hematology.org/session/250954.
The NXC-201 66th American Society of Hematology Meeting oral presentation can be accessed on the ImmixBio corporate website at this link: https://www.immixbio.com/.
ASH Presentation Details (CAR-T NXC-201 in relapsed/refractory AL Amyloidosis).
| Event | 66th ASH Annual Meeting and Exposition, San Diego, CA |
| Title | “Efficacy and Safety of Anti-BCMA Chimeric Antigen Receptor T-Cell (CART) for the Treatment of Relapsed and Refractory AL Amyloidosis” |
| Presentation Date/Time (Pacific Time) |
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About NEXICART-1
NEXICART-1 (NCT04720313) is an open-label, ex-U.S. Phase 1b/2 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 of NXC-201. NEXICART-1 clinical results are available at https://immixbio.com/the-science-pipeline-and-publications/ .
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) (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 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 clinical development program for the treatment of patients with relapsed/refractory AL amyloidosis in the U.S., 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 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 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 commence 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
Attachment
FAQ
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