Poseida Therapeutics Reports Positive Interim Phase 1 Results for Allogeneic CAR-T Therapy P-BCMA-ALLO1 with High Overall Response Rates in Heavily Pretreated Relapsed/Refractory Multiple Myeloma Patients
Poseida Therapeutics reported positive interim Phase 1 results for its allogeneic CAR-T therapy P-BCMA-ALLO1 in relapsed/refractory multiple myeloma patients. The study showed a 91% overall response rate (ORR) in the optimized lymphodepletion arm, including a 100% ORR in BCMA-naïve patients. Key findings include:
- High efficacy in heavily pretreated patients
- Differentiated safety profile with no dose-limiting toxicities
- Low rates of CRS and ICANS (all Grade 2 or less)
- No graft vs. host disease or Parkinsonism observed
P-BCMA-ALLO1 received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA. The therapy is being developed in collaboration with Roche for blood cancers using Poseida's TSCM-rich CAR-T platform.
Poseida Therapeutics ha riportato risultati intermedi positivi della Fase 1 per la sua terapia CAR-T allogenica P-BCMA-ALLO1 in pazienti con mieloma multiplo recidivante/refrattario. Lo studio ha mostrato una percentuale di risposta complessiva (ORR) del 91% nel braccio con linfodeplezione ottimizzata, inclusa una ORR del 100% nei pazienti naïve al BCMA. Le principali scoperte includono:
- Alta efficacia in pazienti gravemente pretrattati
- Profilo di sicurezza differenziato senza tossicità limitanti la dose
- Bassi tassi di CRS e ICANS (tutti di Grado 2 o inferiore)
- Nessuna malattia del trapianto contro l'ospite o Parkinsonismo osservato
P-BCMA-ALLO1 ha ricevuto la designazione Regenerative Medicine Advanced Therapy (RMAT) dalla FDA. La terapia è in fase di sviluppo in collaborazione con Roche per i tumori del sangue utilizzando la piattaforma CAR-T ricca di TSCM di Poseida.
Poseida Therapeutics informó sobre resultados interinos positivos de la Fase 1 para su terapia CAR-T alogénica P-BCMA-ALLO1 en pacientes con mieloma múltiple en recaída/refractario. El estudio mostró una tasa de respuesta global (ORR) del 91% en el brazo de linfodepleción optimizado, incluyendo una ORR del 100% en pacientes naïve al BCMA. Los hallazgos clave incluyen:
- Alta eficacia en pacientes con tratamiento previo intensivo
- Perfil de seguridad diferenciado sin toxicidades limitantes de dosis
- Bajas tasas de CRS e ICANS (todas Grado 2 o menos)
- No se observó enfermedad injerto contra huésped ni parkinsonismo
P-BCMA-ALLO1 recibió la designación de Terapia Avanzada de Medicina Regenerativa (RMAT) de la FDA. La terapia se está desarrollando en colaboración con Roche para cánceres de sangre utilizando la plataforma CAR-T rica en TSCM de Poseida.
Poseida Therapeutics는 재발/불응성 다발성 골수종 환자에 대한 자가면역 CAR-T 치료제 P-BCMA-ALLO1의 1상 임상시험에서 긍정적인 중간 결과를 보고했습니다. 연구 결과 전체 응답률(ORR) 91%를 보였으며, 최적화된 림프 제거 그룹에서 BCMA 미경험 환자에서 100% ORR이 포함되었습니다. 주요 발견 사항은 다음과 같습니다:
- 고용량 치료를 받은 환자에 대한 높은 효능
- 용량 제한 독성이 없는 차별화된 안전성 프로필
- CRS 및 ICANS의 낮은 발생률(모두 2 등급 이하)
- 응식 대 숙주 질병이나 파킨슨병이 관찰되지 않음
P-BCMA-ALLO1은 FDA로부터 재생 의학 첨단 치료(RMAT) 지정을 받았습니다. 이 치료법은 Poseida의 TSCM-풍부 CAR-T 플랫폼을 활용하여 혈액 암 치료를 위해 Roche와 협력하여 개발되고 있습니다.
Poseida Therapeutics a rapporté des résultats intermédiaires positifs de la Phase 1 pour sa thérapie CAR-T allogène P-BCMA-ALLO1 chez des patients atteints de myélome multiple récidivant/réfractaire. L'étude a montré un taux de réponse global (ORR) de 91% dans le groupe de lymphodéplétion optimisé, y compris un ORR de 100% chez les patients naïfs de BCMA. Les principales conclusions comprennent :
- Haute efficacité chez les patients fortement prétraités
- Profil de sécurité différencié sans toxicités limitantes de dose
- Faibles taux de CRS et d'ICANS (tous de Grade 2 ou moins)
- Pas de maladie du greffon contre l'hôte ni de parkinsonisme observé
P-BCMA-ALLO1 a reçu la désignation Thérapie Avancée de Médecine Régénérative (RMAT) de la FDA. La thérapie est développée en collaboration avec Roche pour les cancers du sang en utilisant la plateforme CAR-T riche en TSCM de Poseida.
Poseida Therapeutics berichtete über positive Zwischen Ergebnisse der Phase 1 für ihre allogene CAR-T Therapie P-BCMA-ALLO1 bei Patienten mit rückfallendem/refraktärem multiplem Myelom. Die Studie zeigte eine gesamtreaktionsrate (ORR) von 91% in der optimierten Lymphodepletion-Gruppe, einschließlich einer ORR von 100% bei BCMA-naiven Patienten. Wichtige Ergebnis sind:
- Hohe Wirksamkeit bei stark vorbehandelten Patienten
- Differenziertes Sicherheitsprofil ohne dosislimitierende Toxizität
- Niedrige Raten von CRS und ICANS (alle Grad 2 oder weniger)
- Keine Transplantat-gegen-Wirt-Krankheit oder Parkinsonismus beobachtet
P-BCMA-ALLO1 erhielt die Bezeichnung für fortschrittliche regenerativmedizinische Therapie (RMAT) von der FDA. Die Therapie wird in Zusammenarbeit mit Roche zur Behandlung von Blutkrebs unter Verwendung von Poseidas TSCM-reicher CAR-T-Plattform entwickelt.
- 91% overall response rate (ORR) in the optimized lymphodepletion arm
- 100% ORR in BCMA-naïve patients
- 86% ORR in patients with prior BCMA- and/or GPRC5D-targeting treatment
- 22% of patients achieved complete response (CR) or stringent complete response (sCR)
- 48% of patients achieved very good partial response or higher (VGPR+)
- Median duration of response of 232 days for study Arms A and B
- Granted Regenerative Medicine Advanced Therapy (RMAT) designation by FDA
- No dose-limiting toxicities observed
- Low rates of CRS and ICANS (all Grade 2 or less)
- No graft vs. host disease or Parkinsonism observed
- None.
Insights
The interim Phase 1 results for P-BCMA-ALLO1 in relapsed/refractory multiple myeloma are highly encouraging. The 91% overall response rate in the optimized lymphodepletion arm, including 100% ORR in BCMA-naïve patients, is remarkable for this heavily pretreated population. Key highlights:
- High efficacy even in patients with prior BCMA-targeting therapy exposure (86% ORR)
- Compelling safety profile with no dose-limiting toxicities and low rates of CRS/ICANS
- Rapid treatment initiation without manufacturing delays
- Potential for outpatient administration
The RMAT designation from the FDA underscores the therapy's potential. If these results hold in larger trials, P-BCMA-ALLO1 could become a significant advancement in multiple myeloma treatment, offering an off-the-shelf option with efficacy comparable to autologous CAR-T therapies but with improved accessibility and potentially better safety.
This data release is highly positive for Poseida Therapeutics and its development partner Roche. Key financial implications include:
- Potential for accelerated regulatory pathway and earlier market entry due to RMAT designation
- Large market opportunity in multiple myeloma, with potential for both BCMA-naïve and previously treated patients
- Competitive advantage in the allogeneic CAR-T space, potentially capturing market share from autologous therapies
- Reduced manufacturing costs and improved scalability compared to autologous approaches
The collaboration with Roche adds significant value, providing development expertise and future commercialization capabilities. Investors should monitor upcoming Phase 1b data and any discussions around pivotal trial designs. If successful, P-BCMA-ALLO1 could become a major revenue driver for Poseida, potentially transforming the company's financial outlook.
Data showed a
Differentiated P-BCMA-ALLO1 safety results with no dose-limiting toxicities, low rates of CRS and ICANS all Grade 2 or less and no graft vs. host disease or Parkinsonism
P-BCMA-ALLO1 was recently granted Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA and is being evaluated in a Phase 1/1b clinical trial in patients with relapsed/refractory multiple myeloma who have previously received three or more prior lines of therapy
Company to host webcast and conference call tomorrow at 1 p.m. ET / 10 a.m. PT with multiple myeloma experts to review the Phase 1 IMS oral presentation data and provide business updates
P-BCMA-ALLO1 is an investigational non-viral, stem cell memory T cell (TSCM)-rich allogeneic CAR-T cell therapy in Phase 1/1b clinical development for the treatment of patients with RRMM. The Company is developing this investigational off-the-shelf allogeneic CAR-T cell therapy with Roche as part of a broader collaboration focused on addressing blood cancers with Poseida's TSCM-rich CAR-T platform.
"The compelling and differentiated results from the optimized lymphodepletion arms of the ongoing Phase 1 trial of P-BCMA-ALLO1 showed deep responses and a high response rate in patients with heavily pre-treated relapsed or refractory multiple myeloma, regardless of prior exposure to B-cell maturation antigen (BCMA)-targeting therapy. The high overall response rate of
"P-BCMA-ALLO1 is one of the most advanced allogeneic CAR-T in clinical development for multiple myeloma, manufactured using non-viral technology to produce a TSCM-rich therapy that has shown a compelling emerging product profile," said Kristin Yarema, Ph.D., president and chief executive officer of Poseida Therapeutics. "We are encouraged to see such a high overall response rate in an arm of the Phase 1 trial with optimized lymphodepletion, along with standout safety results across all arms, given that the study population was heavily pretreated, high-risk, and in general had many features that historically have led to a poor prognosis. We are excited to build on these data as we advance P-BCMA-ALLO1 in the Phase 1b part of the trial, which is currently enrolling patients."
New Interim Clinical Data from Phase 1 P-BCMA-ALLO1 Trial
The ongoing open-label, multicenter Phase 1/1b dose-escalation and expansion trial in patients with RRMM is assessing the safety and maximum tolerated dose of P-BCMA-ALLO1 (primary objective) and its anti-myeloma activity (secondary objective). As of September 6, 2024, 72 unique patients were enrolled as an intent-to-treat (ITT) population and were treated across four study arms (S, A, B and C) that included different P-BCMA-ALLO1 doses and lymphodepletion regimen combinations. Study participants were required to have received three or more prior lines of therapy, including a prior proteasome inhibitor, immunomodulatory drug and anti-CD38 monoclonal antibody. The trial enrolled a heavily pretreated patient population with
In the ITT population,
The ORR across all four study arms was
Data from Arm C of the Phase 1 P-BCMA-ALLO1 Trial
Results from 23 study participants in Arm C were highlighted in the oral session at IMS. Patients received cyclophosphamide 750 mg/m2/day and fludarabine 30 mg/m2/day and approximately 2x106 cells/kg P-BCMA-ALLO1. Some patients were treated in an outpatient setting. Arm C patient details include:
- Nearly half (
48% ) were age 65 or older - All were heavily pretreated, with a median of six prior lines of anti-myeloma therapy and a maximum of 14
62% of patients had received prior BCMA-targeting therapy29% had failed both a BCMA CAR-T and a bispecific TCE, and29% had failed both a BCMA-targeting therapy and the GPRC5D-targeting TCE, talquetamab- Approximately two-thirds of patients (
62% ) had high-risk disease by cytogenetics and38% had extramedullary disease
Efficacy results, which are still evolving, for the 23 patients in Arm C showed:
- An ORR of
91% , with a100% ORR in BCMA-naïve patients, an86% ORR in those who had received at least one prior BCMA-targeting treatment (all had received prior CAR-T and/or TCE), and an86% ORR in those who had received at least one prior BCMA-targeting treatment and/or talquetamab 22% achieved a CR or an sCR48% achieved VGPR+- Median DoR could not be estimated at the time of data cut-off because the current median follow-up is less than 3.5 months (for pooled arms A and B, the median DoR was more than seven months (estimated range of five-10 months), with a median time to response of only 16 days)
P-BCMA-ALLO1 was well-tolerated with key safety results from Arm C, including:
- No dose-limiting toxicities, Grade 3 or higher cytokine release syndrome (CRS) or immune effector cell neurotoxicity syndrome (ICANS). The incidence of Grade 1 or 2 CRS was
39% and the incidence of Grade 1 or 2 ICANS was13% - The incidence of infections was
48% , including30% that were Grade 1 or 2 and17% that were Grade 3 - Rapid cytopenia recovery in the vast majority of cases
- No graft-vs-host disease (GvHD), hemophagocytic lymphohistiocytosis (HLH), Parkinsonism or cranial neuropathies observed
- The safety results of P-BCMA-ALLO1 in arm C have been consistent with those observed in the other three arms of the Phase 1 trial, with a total safety database, including 72 unique patients
The ongoing P-BCMA-ALLO1 Phase 1/1b trial is enrolling patients using the Arm C lymphodepletion regimen described above, across two dosing cohorts, with dose optimization ongoing in Arm C.
Company-Hosted IMS Live Webcast and Conference Call Information
Poseida will host a live webcast and conference call tomorrow, Saturday, September 28, 2024, at 1 p.m. ET / 10 a.m. PT. The webcast will feature an expert panel of clinicians who will discuss the new clinical data and multiple myeloma treatment landscape. The panel will be moderated by Dr. Rizvi and include Dr. Dholaria and Thomas G. Martin, M.D., Clinical Professor of Medicine, Adult Leukemia and Bone Marrow Transplantation Program and Associate Director of the Myeloma Program at the University of
The conference call can be accessed by dialing 800-225-9448 or 203-518-9708 (International) with the conference ID PSTX0928. The live webcast can be accessed using the link here, or by visiting the Events and Presentations section of the Poseida website at investors.poseida.com. After the live webcast, the event will remain archived on the Poseida website for approximately 90 days.
About P-BCMA-ALLO1
P-BCMA-ALLO1 is an allogeneic CAR-T product candidate licensed to Roche targeting B-cell maturation antigen (BCMA) for the treatment of relapsed/refractory multiple myeloma. This allogeneic program includes a VH-based binder that targets BCMA, and interim clinical data presented at IMS in September 2024 support the Company's belief that T stem cell (TSCM)-rich allogeneic CAR-Ts have the potential to offer effective, safe and reliable treatment addressing unmet needs in multiple myeloma. The
P-BCMA-ALLO1 is currently being evaluated in a Phase 1/1b trial in patients with multiple myeloma. Additional information about the Phase 1/1b trial is available at www.clinicaltrials.gov using identifier: NCT04960579.
About Poseida Therapeutics, Inc.
Poseida Therapeutics is a clinical-stage biopharmaceutical company advancing differentiated allogeneic cell therapies and genetic medicines with the capacity to cure certain cancers and rare diseases. The Company's pipeline includes investigational allogeneic CAR-T cell therapies for both solid tumors and hematologic cancers as well as investigational in vivo genetic medicines that address patient populations with high unmet medical need. The Company's approach is based on its proprietary genetic editing platforms, including its non-viral piggyBac® DNA Delivery System, Cas-CLOVER™ Site-Specific Gene Editing System, Booster Molecule and nanoparticle gene delivery technologies, as well as in-house GMP cell therapy manufacturing. The Company has formed strategic collaborations with Roche and Astellas to unlock the promise of cell therapies for cancer patients. Learn more at www.poseida.com and connect with Poseida on X and LinkedIn.
Forward-Looking Statements
Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, expected plans with respect to clinical trials; the potential capabilities and benefits of the Company's technology platforms and product candidates; the quotes from Drs. Dholaria and Yarema; and the Company's plans and strategy with respect to developing its technologies and product candidates. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These forward-looking statements are based upon the Company's current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, the fact that interim data from the Company's clinical trials may change as more patient data become available and remain subject to audit and verification procedures that could result in material differences from the final data; the Company's reliance on third parties for various aspects of its business; risks and uncertainties associated with development and regulatory approval of novel product candidates in the biopharmaceutical industry; the Company's ability to retain key scientific or management personnel; the Company's ongoing and planned clinical trials; whether any of the Company's product candidates will be shown to be effective, safe and reliable; and the other risks and uncertainties described in the Risk Factors section of Poseida's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on August 5, 2024, and in other filings Poseida makes with the SEC from time to time. All forward-looking statements contained in this press release speak only as of the date on which they were made. The Company undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.
1 Based on an efficacy cutoff date of September 6, 2024 and a safety cutoff date of July 31, 2024
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FAQ
What were the key results of Poseida Therapeutics' P-BCMA-ALLO1 Phase 1 trial for multiple myeloma (PSTX)?
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How does P-BCMA-ALLO1 differ from other CAR-T therapies for multiple myeloma (PSTX)?