Poseida Therapeutics Receives Regenerative Medicine Advanced Therapy (RMAT) Designation from FDA for P-BCMA-ALLO1 to Treat Relapsed/Refractory Multiple Myeloma
Poseida Therapeutics (Nasdaq: PSTX) has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for P-BCMA-ALLO1, their investigational allogeneic CAR-T cell therapy for relapsed/refractory multiple myeloma. This designation, based on positive Phase 1 clinical data, offers benefits like Fast Track and Breakthrough Therapy designations, enabling increased FDA interaction. P-BCMA-ALLO1 has shown promising efficacy, safety, and rapid 'off-the-shelf' access. New clinical data will be presented at the 21st International Myeloma Society Annual Meeting. The therapy has demonstrated responses in refractory patients, including those previously treated with BCMA-targeted therapies. Poseida plans additional clinical updates in the second half of 2024, in coordination with their strategic partner Roche.
Poseida Therapeutics (Nasdaq: PSTX) ha ricevuto la designazione di Terapia Avanzata in Medicina Rigenerativa (RMAT) dalla FDA per P-BCMA-ALLO1, la loro terapia con cellule CAR-T allogeniche in fase di sperimentazione per il mieloma multiplo relapsato/refrattario. Questa designazione, basata su dati clinici positivi della Fase 1, offre vantaggi come le designazioni Fast Track e Breakthrough Therapy, consentendo un maggior contatto con la FDA. P-BCMA-ALLO1 ha mostrato un'efficacia promettente, sicurezza e un rapido accesso 'pronto all'uso'. Nuovi dati clinici saranno presentati al 21° Congresso Annuale della Società Internazionale del Mieloma. La terapia ha dimostrato risposte nei pazienti refrattari, inclusi quelli trattati in precedenza con terapie mirate a BCMA. Poseida prevede ulteriori aggiornamenti clinici nella seconda metà del 2024, in coordinamento con il partner strategico Roche.
Poseida Therapeutics (Nasdaq: PSTX) ha recibido la designación de Terapia Avanzada en Medicina Regenerativa (RMAT) de la FDA para P-BCMA-ALLO1, su terapia experimental de células CAR-T alogénicas para el mieloma múltiple en recaída/refractario. Esta designación, basada en datos clínicos positivos de la Fase 1, ofrece beneficios como las designaciones de Fast Track y Breakthrough Therapy, lo que permite una mayor interacción con la FDA. P-BCMA-ALLO1 ha mostrado eficacia prometedora, seguridad y un rápido acceso 'listo para usar'. Nuevos datos clínicos serán presentados en el 21° Congreso Anual de la Sociedad Internacional de Mieloma. La terapia ha demostrado respuestas en pacientes refractarios, incluidos aquellos previamente tratados con terapias dirigidas a BCMA. Poseida planea actualizaciones clínicas adicionales en la segunda mitad de 2024, en coordinación con su socio estratégico Roche.
포세이다 테라퓨틱스 (Nasdaq: PSTX)는 P-BCMA-ALLO1에 대해 FDA로부터 재생 의학 진보 치료(RMAT) 지정을 받았습니다. 이는 재발/불응성 다발성 골수종에 대한 조사 중인 동종 CAR-T 세포 치료법입니다. 이 지칭은 긍정적인 1상 임상 데이터에 기반하여, 빠른 추적 및 혁신 치료 지정을 포함한 이점들을 제공하여 FDA와의 상호작용을 증가시킵니다. P-BCMA-ALLO1은 약속된 효능, 안전성 및 빠른 '즉시 사용 가능' 접근을 보여주었습니다. 새로운 임상 데이터는 제21회 국제 다발성 골수종학회 연례 회의에서 발표될 예정입니다. 이 치료법은 BCMA 표적 치료로 이전에 치료받은 환자를 포함하여 불응성 환자에게 반응을 나타냈습니다. 포세이다는 2024년 하반기에 전략적 파트너인 로슈와 협력하여 추가 임상 업데이트를 계획하고 있습니다.
Poseida Therapeutics (Nasdaq: PSTX) a reçu la désignation de Thérapie Avancée en Médecine Régénérative (RMAT) de la FDA pour P-BCMA-ALLO1, sa thérapie expérimentale par cellules CAR-T allogéniques pour le myélome multiple en rechute/réfractaire. Cette désignation, basée sur des données cliniques positives de la phase 1, offre des avantages tels que les désignations Fast Track et Breakthrough Therapy, permettant une interaction accrue avec la FDA. P-BCMA-ALLO1 a montré une efficacité prometteuse, une sécurité et un accès rapide 'prêt à l'emploi'. De nouvelles données cliniques seront présentées lors de la 21ème réunion annuelle de la Société Internationale du Myélome. La thérapie a démontré des réponses chez les patients refractaires, y compris ceux précédemment traités par des thérapies ciblées BCMA. Poseida prévoit des mises à jour cliniques supplémentaires au second semestre 2024, en coordination avec leur partenaire stratégique Roche.
Poseida Therapeutics (Nasdaq: PSTX) hat von der FDA die Bezeichnung für Fortschrittliche Therapie in der regenerativen Medizin (RMAT) für P-BCMA-ALLO1 erhalten, deren experimentelle allogene CAR-T-Zelltherapie für rezidiviertes/refraktäres multiples Myelom ist. Diese Bezeichnung, die auf positiven klinischen Daten der Phase 1 basiert, bietet Vorteile wie die Bezeichnungen Fast Track und Breakthrough Therapy und ermöglicht eine erhöhte Interaktion mit der FDA. P-BCMA-ALLO1 hat vielversprechende Wirksamkeit, Sicherheit und schnellen 'off-the-shelf'-Zugang gezeigt. Neue klinische Daten werden auf dem 21. Jahrestreffen der International Myeloma Society vorgestellt. Die Therapie hat bei refraktären Patienten, darunter solche, die zuvor mit BCMA-zielgerichteten Therapien behandelt wurden, Reaktionen gezeigt. Poseida plant, in der zweiten Hälfte des Jahres 2024 zusätzliche klinische Updates in Koordination mit ihrem strategischen Partner Roche zu geben.
- Received RMAT designation from FDA for P-BCMA-ALLO1
- Positive early clinical data from ongoing Phase 1 study
- Demonstrated clinical responses in refractory patients, including those with prior BCMA-targeted therapies
- Strategic collaboration with Roche for multiple allogeneic CAR-T therapies
- Potential for accelerated approval and increased FDA interaction
- None.
Insights
The FDA's RMAT designation for P-BCMA-ALLO1 is a significant milestone for Poseida Therapeutics. This designation, based on promising Phase 1 data, acknowledges the therapy's potential to address unmet needs in relapsed/refractory multiple myeloma patients. Key points to consider:
- P-BCMA-ALLO1 has shown efficacy in heavily pretreated patients, including those who've received prior BCMA-targeted therapies.
- As an allogeneic CAR-T therapy, it offers potential for "off-the-shelf" availability, addressing manufacturing and timing challenges of autologous CAR-T treatments.
- The RMAT status provides accelerated development pathways and increased FDA interaction, potentially expediting the therapy's path to market.
While promising, investors should note that Phase 1 data is preliminary and further studies are needed to confirm efficacy and safety profiles.
This RMAT designation is a positive development for Poseida Therapeutics, potentially accelerating P-BCMA-ALLO1's path to market. Financial implications include:
- Reduced development costs and timelines due to streamlined regulatory processes.
- Increased investor confidence, potentially boosting stock value.
- Enhanced competitive position in the lucrative multiple myeloma market.
The collaboration with Roche adds financial stability and development resources. However, investors should consider that commercialization is still distant and success isn't guaranteed. The upcoming data presentation at the International Myeloma Society Annual Meeting could be a near-term catalyst for stock movement, depending on the strength of the results presented.
FDA evaluated RMAT application based on positive clinical data from ongoing Phase 1 study of P-BCMA-ALLO1; new clinical data from the study will be presented at the 21st International Myeloma Society Annual Meeting this month
RMAT designation recognizes potential of P-BCMA-ALLO1 to address significant unmet needs of multiple myeloma patients and enables increased dialogue with FDA throughout the development process
RMAT designation includes all the benefits of the Fast Track and Breakthrough Therapy designation programs, including early interactions with the FDA. Poseida's RMAT application was evaluated based on encouraging early data from its ongoing Phase 1 study of P-BCMA-ALLO1, which demonstrated P-BCMA-ALLO1's potential to offer promising efficacy, safety profile and rapid 'off-the-shelf' patient access.
"The RMAT designation for P-BCMA-ALLO1, our lead program, is based on impressive early clinical data from our ongoing Phase 1 study and further validates its potential to address the unmet needs of patients with relapsed/refractory multiple myeloma," said Kristin Yarema, Ph.D., president and chief executive officer of Poseida Therapeutics. "Importantly, our data has shown clinical responses in very sick, refractory patients, including those that have received prior BCMA-targeted therapies. With both RMAT and Orphan Drug designations for P-BCMA-ALLO1, we look forward to working closely with the FDA as we continue to advance this next-generation, off-the shelf allogeneic CAR-T therapy, including the recently initiated Phase 1b portion of the trial."
The Company will report new clinical data from the P-BCMA-ALLO1 Phase 1 study in an oral session at the 21st International Myeloma Society Annual Meeting, which is being held in
The RMAT designation is a program under the 21st Century Cures Act that is intended to expedite the development and review of regenerative medicine therapies for serious or life-threatening diseases or conditions. A regenerative medicine therapy is eligible for RMAT designation if it is intended to treat, modify, reverse or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the regenerative medicine therapy has the potential to address unmet medical needs for such disease or condition.
RMAT designation includes all Breakthrough Therapy designation features, including early interactions to discuss any potential surrogate or intermediate endpoints. RMATs may be eligible for accelerated approval based on previously agreed-upon surrogate or intermediate endpoints that are reasonably likely to predict long-term clinical benefit, or reliance upon data obtained from a meaningful number of sites, including through expansion to additional sites, as appropriate.
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 clinical data presented at ASH in December 2023 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 FDA has granted P-BCMA-ALLO1 Regenerative Medicine Advanced Therapy (RMAT) designation for adult patients with relapsed/refractory multiple myeloma after three or more prior lines of therapies including a proteasome inhibitor (PI), an immunomodulatory agent (IMiD), and anti-CD38 antibody in addition to Orphan Drug designation for multiple myeloma. Additional information about the Phase 1/1b study 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, including timing of regulatory submissions and approvals and clinical data updates; anticipated timelines and milestones with respect to the Company's development programs and manufacturing activities and capabilities; the potential capabilities and benefits of the Company's technology platforms and product candidates, including the efficacy and safety profile of such product candidates; the quote from Dr. Yarema; the potential benefits from receiving Regenerative Medicine Advanced Therapy designation for P-BCMA-ALLO1; 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 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; and the other risks described in the Company's filings with the Securities and Exchange Commission. 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.
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FAQ
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