Allogene Therapeutics Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for ALLO-316, an AlloCAR T™ Investigational Product for Adult Patients with Advanced or Metastatic Renal Cell Carcinoma (RCC)
Allogene Therapeutics announced that the FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to ALLO-316 for treating adult patients with CD70 positive advanced or metastatic renal cell carcinoma (RCC). The designation was based on clinical data from the TRAVERSE trial, showing potential in treating patients who failed multiple standard RCC therapies. ALLO-316 utilizes the Dagger® technology as a next-generation allogeneic platform for single-infusion 'off-the-shelf' CAR T therapy. The company will present updated Phase 1 data at the upcoming SITC annual meeting. ALLO-316 previously received Fast Track Designation in March 2022.
Allogene Therapeutics ha annunciato che la FDA ha conferito la designazione Regenerative Medicine Advanced Therapy (RMAT) per ALLO-316 nel trattamento di pazienti adulti con carcinoma a cellule renali (RCC) avanzato o metastatico positivo al CD70. Questa designazione si basa su dati clinici provenienti dallo studio TRAVERSE, che mostrano potenzialità nel trattare pazienti che hanno fallito molteplici terapie standard per il RCC. ALLO-316 utilizza la tecnologia Dagger® come piattaforma allogenica di nuova generazione per la terapia CAR T 'pronta all'uso' con infusione singola. L'azienda presenterà i dati aggiornati della Fase 1 al prossimo incontro annuale della SITC. ALLO-316 ha precedentemente ricevuto la designazione Fast Track a marzo 2022.
Allogene Therapeutics anunció que la FDA otorgó la designación de Regenerative Medicine Advanced Therapy (RMAT) a ALLO-316 para el tratamiento de pacientes adultos con carcinoma de células renales (RCC) avanzado o metastásico positivo para CD70. La designación se basó en datos clínicos del ensayo TRAVERSE, que muestran el potencial para tratar a pacientes que no respondieron a múltiples terapias estándar para RCC. ALLO-316 utiliza la tecnología Dagger® como una plataforma alogénica de próxima generación para la terapia CAR T 'lista para usar' con infusión única. La compañía presentará datos actualizados de la Fase 1 en la próxima reunión anual de la SITC. ALLO-316 recibió previamente la designación de Fast Track en marzo de 2022.
Allogene Therapeutics는 FDA가 ALLO-316에 대해 CD70 양성의 진행성 또는 전이성 신세포암(RCC) 성인 환자를 치료하는 데 재생의학고급치료(RMAT) 지정을 부여했다고 발표했습니다. 이 지정은 여러 표준 RCC 치료에 실패한 환자를 치료하는 데 잠재력을 보여주는 TRAVERSE 시험의 임상 데이터를 기반으로 했습니다. ALLO-316은 단일 주입형 '즉시 사용' CAR T 치료를 위한 차세대 동종 이식 플랫폼으로 Dagger® 기술을 활용합니다. 이 회사는 다가오는 SITC 연례 회의에서 업데이트된 1상 데이터를 발표할 예정입니다. ALLO-316은 2022년 3월에 Fast Track 지정을 받았습니다.
Allogene Therapeutics a annoncé que la FDA a accordé la désignation de Regenerative Medicine Advanced Therapy (RMAT) à ALLO-316 pour le traitement des patients adultes atteints d'un carcinome rénal à cellules fraîches (RCC) avancé ou métastatique positif au CD70. Cette désignation est basée sur des données cliniques de l'essai TRAVERSE, montrant un potentiel pour traiter des patients ayant échoué à plusieurs thérapies standards pour le RCC. ALLO-316 utilise la technologie Dagger® comme plateforme allogénique de nouvelle génération pour la thérapie CAR T 'prête à l'emploi' par infusion unique. L'entreprise présentera des données actualisées de phase 1 lors de la prochaine réunion annuelle de la SITC. ALLO-316 a précédemment reçu la désignation Fast Track en mars 2022.
Allogene Therapeutics gab bekannt, dass die FDA die Regenerative Medicine Advanced Therapy (RMAT) Auszeichnung für ALLO-316 zur Behandlung von Erwachsenen mit CD70-positivem fortgeschrittenem oder metastasierendem Nierenzellkarzinom (RCC) erteilt hat. Die Auszeichnung basierte auf klinischen Daten aus der TRAVERSE-Studie, die das Potenzial zur Behandlung von Patienten zeigen, die mehrere Standardtherapien für RCC nicht erfolgreich durchlaufen haben. ALLO-316 nutzt die Dagger®-Technologie als Plattform für die next-gen-allogenen CAR-T-Therapie mit einer Einfusa. Das Unternehmen wird aktualisierte Phase-1-Daten auf dem kommenden SITC-Jahrestreffen präsentieren. ALLO-316 erhielt zuvor im März 2022 die Fast-Track-Auszeichnung.
- Received FDA RMAT designation for ALLO-316, potentially accelerating development and review process
- Promising clinical data from TRAVERSE trial showing efficacy in treatment-resistant RCC patients
- Development of 'off-the-shelf' CAR T therapy could enable faster and more accessible treatment options
- Product still in Phase 1 trials, indicating early stage of development
- Efficacy to specific subset of RCC patients (CD70 positive)
Insights
The RMAT designation for ALLO-316 represents a significant regulatory milestone with tangible implications for accelerated development. This advanced therapy status, combined with the existing Fast Track Designation, creates a powerful regulatory framework that could expedite the path to market. The designation specifically targets CD70-positive advanced RCC patients who have failed both checkpoint inhibitors and VEGF therapies - a substantial unmet medical need.
The Dagger® technology platform shows promise in solid tumors, historically resistant to CAR-T approaches. Early clinical evidence from the TRAVERSE trial suggests meaningful therapeutic potential, though detailed efficacy data remains pending. The "off-the-shelf" nature of ALLO-316 could address major limitations of current autologous CAR-T therapies, including manufacturing time and reliability.
This regulatory milestone significantly enhances ALLO-316's market potential in the $4.5 billion RCC treatment market. The RMAT designation provides important competitive advantages, including enhanced FDA interaction and potential for accelerated approval pathways. For Allogene, this represents a strategic opportunity in solid tumors, expanding beyond traditional CAR-T applications in blood cancers.
The allogeneic approach could capture market share by offering faster treatment initiation and potentially lower production costs compared to autologous alternatives. However, investors should note that commercialization remains distant, with Phase 1 data still emerging and larger trials needed to confirm efficacy and safety profiles.
- RMAT Designation Follows Positive Proof-of-Concept Data from the ALLO-316 TRAVERSE Trial in Adult Patients with Advanced or Metastatic CD70 Positive Renal Cell Carcinoma (RCC) Who Received Prior Immune Checkpoint Inhibitor and VEGF-Targeting Therapy
- ALLO-316 Advances Scientific Understanding and Applicability of the Dagger® Technology as the Next-Generation Allogeneic Platform to Maximize the Potential of a Single Infusion of an “Off-the-Shelf” CAR T Product
- Company to Present Updated Phase 1 Data from the TRAVERSE Trial at the Society for Immunotherapy of Cancer (SITC) Annual Meeting
- ALLO-316 was Previously Granted Fast Track Designation (FTD) in March 2022 by the U.S. Food and Drug Administration
SOUTH SAN FRANCISCO, Calif., Oct. 29, 2024 (GLOBE NEWSWIRE) -- Allogene Therapeutics, Inc. (Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T™) products for cancer and autoimmune disease, today announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to ALLO-316 for the treatment of adult patients with CD70 positive advanced or metastatic renal cell carcinoma (RCC). The RMAT designation was based on clinical data from the TRAVERSE trial indicating the potential of ALLO-316 to address the unmet need for patients with difficult-to-treat RCC who have failed multiple standard RCC therapies, including an immune checkpoint inhibitor and a VEGF-targeting therapy. The ongoing development of ALLO-316 continues to advance the scientific understanding and applicability of the Dagger® technology as the next-generation allogeneic platform to maximize the potential of a single infusion of “off-the-shelf” CAR T product in solid tumors.
“The RMAT designation for ALLO-316 highlights the transformative potential of our AlloCAR T™ platform to offer new hope for heavily pretreated patients with renal cell carcinoma who have exhausted standard treatment options,” said Zachary Roberts, M.D., Ph.D., Executive Vice President of Research & Development and Chief Medical Officer. “This important milestone moves us closer to fulfilling the promise of “off-the-shelf” CAR T therapy—delivering faster, more reliable, and widely accessible treatments. We remain optimistic about the future of ALLO-316 and its potential to be an important advancement for patients.”
The ongoing Phase 1 TRAVERSE trial is designed to evaluate the safety, tolerability, and activity of ALLO-316 in patients with advanced or metastatic RCC. Initial results from the TRAVERSE trial were presented in an oral presentation at the American Association for Cancer Research (AACR) Annual Meeting in Orlando, Florida, in 2023. The Company will present updated Phase 1 data from the TRAVERSE trial at the Society for Immunotherapy of Cancer (SITC) annual meeting.
The RMAT designation is intended to accelerate the development and review of promising investigational products, including cell therapies. To qualify, a product must be designed to treat, modify, reverse, or cure a serious or life-threatening disease, with preliminary clinical evidence suggesting it can address unmet medical needs. The RMAT designation offers several key advantages, including early and frequent interactions with the FDA to discuss potential surrogate or intermediate endpoints, as well as strategies to meet post-approval requirements, potentially streamlining the path to market approval.
About ALLO-316 (TRAVERSE)
ALLO-316, an AlloCAR T™ investigational product, targets CD70 which is highly expressed in renal cell carcinoma (RCC). CD70 is also selectively expressed in several cancers, creating the potential for ALLO-316 to be developed across a variety of both hematologic malignancies and solid tumors. ALLO-316 utilizes the Dagger® technology to optimize CAR T cell expansion and persistence to maximize the potential efficacy in solid tumors with a one-time infusion. The ongoing Phase 1 TRAVERSE trial is designed to evaluate the safety, tolerability, and activity of ALLO-316 in patients with advanced or metastatic clear cell RCC. In March 2022, the U.S. Food and Drug Administration (FDA) granted Fast Track Designation (FTD) to ALLO-316, and in October 2024 the FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to ALLO-316 based on its potential to address the unmet need for adult patients with CD70 positive advanced or metastatic RCC who have failed standard RCC therapies.
About Allogene Therapeutics
Allogene Therapeutics, with headquarters in South San Francisco, is a clinical-stage biotechnology company pioneering the development of allogeneic chimeric antigen receptor T cell (AlloCAR T™) products for cancer and autoimmune disease. Led by a management team with significant experience in cell therapy, Allogene is developing a pipeline of “off-the-shelf” CAR T cell product candidates with the goal of delivering readily available cell therapy on-demand, more reliably, and at greater scale to more patients. For more information, please visit www.allogene.com, and follow @AllogeneTx on X and LinkedIn.
Cautionary Note on Forward-Looking Statements for Allogene
This press release contains forward-looking statements for purposes of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. The press release may, in some cases, use terms such as “potential,” “continue,” “intend,” “will,” “advance,” “move,” “offer,” “goal,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Forward-looking statements include statements regarding intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things: the extent to which ALLO-316 TRAVERSE Trial data may establish proof-of-concept or advance scientific understanding; whether the Dagger® Technology will become the next-generation allogeneic platform or maximize the potential of a one-time Infusion of a CAR T product; the Dagger® technology’s ability to optimize CAR T cell expansion and persistence and maximize the potential efficacy in solid tumors with a one-time infusion; the potential of ALLO-316 to address the unmet need for any patient population; the transformative potential of our AlloCAR T™ platform; ALLO-316’s potential to be an important advancement for patients; potential advantages of the RMAT designation, including acceleration of the development and review of our investigational products, early and frequent interactions with the FDA, or streamlining the path to market approval; the potential for ALLO-316 to be developed across a variety of both hematologic malignancies and solid tumors; or our ability to deliver cell therapy on-demand, faster, more reliably, and at greater scale to more patients. Various factors may cause material differences between Allogene’s expectations and actual results, including, risks and uncertainties related to: RMAT designation may not lead to a faster development or regulatory review or approval process and it does not increase the likelihood that our product candidates will receive marketing approval and the designation can be revoked if the criteria for eligibility ceases to be met; our product candidates are based on novel technologies, which makes it difficult to predict the time and cost of product candidate development and obtaining regulatory approval; the limited nature of our Phase 1 data from our clinical trials and the extent to which such data may or may not be validated in any future clinical trial; our product candidates may cause undesirable side effects or have other properties that could halt their clinical development, prevent their regulatory approval or limit their commercial potential; the extent to which the Food and Drug Administration disagrees with our clinical or regulatory plans or the import of our clinical results, which could cause future delays to our clinical trials, including initiation of clinical trials, or require additional clinical trials; we may encounter difficulties enrolling patients in our clinical trials; we may not be able to demonstrate the safety and efficacy of our product candidates in our clinical trials, which could prevent or delay regulatory approval and commercialization; and our ability to obtain additional financing to develop our products and implement our operating plans. These and other risks are discussed in greater detail in Allogene’s filings with the SEC, including without limitation under the “Risk Factors” heading in its Form 10-Q filed for the quarter ended June 30, 2024, filed with the SEC on August 7, 2024. Any forward-looking statements that are made in this press release speak only as of the date of this press release. Allogene assumes no obligation to update the forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release.
AlloCAR T™ and Dagger® are trademarks of Allogene Therapeutics, Inc.
Allogene’s investigational AlloCAR T™ oncology products utilize Cellectis technologies. The anti-CD70 AlloCAR T program is licensed exclusively from Cellectis by Allogene and Allogene holds global development and commercial rights to this AlloCAR T™ program.
Allogene Media/Investor Contact:
Christine Cassiano
EVP, Chief Corporate Affairs & Brand Strategy Officer
Christine.Cassiano@allogene.com
FAQ
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