Cartesian Therapeutics Announces FDA Special Protocol Assessment Agreement for Phase 3 AURORA Trial of Descartes-08 in Myasthenia Gravis
Cartesian Therapeutics (NASDAQ: RNAC) has secured FDA Special Protocol Assessment (SPA) agreement for its Phase 3 AURORA trial of Descartes-08 in myasthenia gravis (MG). The trial is scheduled to begin in first half of 2025. The SPA agreement confirms that the trial design could support a future Biologics License Application.
The Phase 3 AURORA trial will be randomized, double-blind, and placebo-controlled, involving approximately 100 participants with AChR Ab+ MG. The study will evaluate Descartes-08 versus placebo with six weekly infusions without preconditioning chemotherapy. The primary endpoint will measure improvement in MG-ADL score.
Recent Phase 2b trial results showed promising outcomes, with treated participants (n=12) achieving an average MG-ADL reduction of 5.5 at Month 4. The therapy demonstrated good tolerability, supporting outpatient administration without requiring lymphodepleting chemotherapy.
Cartesian Therapeutics (NASDAQ: RNAC) ha ottenuto l'accordo di Valutazione del Protocollo Speciale (SPA) della FDA per il suo studio di Fase 3 AURORA su Descartes-08 nella miastenia grave (MG). Lo studio è previsto per iniziare nella prima metà del 2025. L'accordo SPA conferma che il disegno dello studio potrebbe supportare una futura Domanda di Licenza per Prodotti Biologici.
Il trial di Fase 3 AURORA sarà randomizzato, in doppio cieco e controllato con placebo, coinvolgendo circa 100 partecipanti con MG AChR Ab+. Lo studio valuterà Descartes-08 rispetto al placebo con sei infusioni settimanali senza chemioterapia di precondizionamento. L'obiettivo primario misurerà il miglioramento nel punteggio MG-ADL.
I recenti risultati del trial di Fase 2b hanno mostrato esiti promettenti, con i partecipanti trattati (n=12) che hanno raggiunto una riduzione media di 5,5 nel punteggio MG-ADL al quarto mese. Il trattamento ha dimostrato una buona tollerabilità, supportando l'amministrazione ambulatoriale senza richiedere chemioterapia linfodepletiva.
Cartesian Therapeutics (NASDAQ: RNAC) ha conseguido un acuerdo de Evaluación de Protocolo Especial (SPA) por parte de la FDA para su ensayo de Fase 3 AURORA de Descartes-08 en miastenia grave (MG). Se espera que el ensayo comience en la primera mitad de 2025. El acuerdo SPA confirma que el diseño del ensayo podría respaldar una futura Solicitud de Licencia de Biológicos.
El ensayo de Fase 3 AURORA será aleatorio, doble ciego y controlado por placebo, involucrando aproximadamente a 100 participantes con MG AChR Ab+. El estudio evaluará Descartes-08 frente al placebo con seis infusiones semanales sin quimioterapia de preacondicionamiento. El objetivo primario medirá la mejora en el puntaje MG-ADL.
Los recientes resultados del ensayo de Fase 2b mostraron resultados prometedores, con los participantes tratados (n=12) logrando una reducción promedio de 5.5 en el puntaje MG-ADL al mes 4. El tratamiento demostró una buena tolerabilidad, apoyando su administración ambulatoria sin requerir quimioterapia linfodepletiva.
Cartesian Therapeutics (NASDAQ: RNAC)는 Descartes-08의 중증근무력증(MG) 치료를 위한 3상 AURORA 임상시험에 대한 FDA의 특별 프로토콜 평가(SPA) 합의를 확보했습니다. 임상시험은 2025년 상반기에 시작될 예정입니다. SPA 합의는 임상시험 설계가 향후 생물학적 제품 허가 신청을 지원할 수 있음을 확인합니다.
3상 AURORA 임상시험은 무작위 이중 맹검 및 위약 대조 방식으로 진행되며, 약 100명의 AChR Ab+ MG 참가자가 포함됩니다. 연구에서는 기존 화학요법 없이 6주마다 Descartes-08과 위약을 비교합니다. 주요 목표는 MG-ADL 점수의 개선을 측정하는 것입니다.
최근 2b상 임상시험 결과는 치료를 받은 참가자들(n=12)이 4개월 차에 평균 5.5점의 MG-ADL 점수 감소를 달성하며 유망한 결과를 보여주었습니다. 이 치료는 좋은 내약성을 나타내어 림프 감량 화학요법 없이도 외래에서 투여할 수 있음을 입증했습니다.
Cartesian Therapeutics (NASDAQ: RNAC) a obtenu l'accord de l'évaluation du protocole spécial (SPA) de la FDA pour son essai de Phase 3 AURORA sur Descartes-08 dans la myasthénie grave (MG). L'essai devrait commencer dans la première moitié de 2025. L'accord SPA confirme que la conception de l'essai pourrait soutenir une future demande de licence pour les produits biologiques.
L'essai de Phase 3 AURORA sera randomisé, en double aveugle et contrôlé par placebo, impliquant environ 100 participants atteints de MG AChR Ab+. L'étude évaluera Descartes-08 par rapport au placebo avec six perfusions hebdomadaires sans chimiothérapie de préconditionnement. Le critère principal mesurera l'amélioration du score MG-ADL.
Les résultats récents des essais de Phase 2b ont montré des résultats prometteurs, les participants traités (n=12) ayant atteint une réduction moyenne de 5,5 du score MG-ADL au bout de 4 mois. Le traitement a démontré une bonne tolérance, permettant une administration en ambulatoire sans nécessiter de chimiothérapie lymphodéplétante.
Cartesian Therapeutics (NASDAQ: RNAC) hat eine FDA-Sonderprotokollbewertung (SPA) für seine Phase-3-Studie AURORA zu Descartes-08 bei Myasthenia gravis (MG) gesichert. Die Studie soll in der ersten Hälfte des Jahres 2025 beginnen. Die SPA-Vereinbarung bestätigt, dass das Studiendesign einen zukünftigen Antrag auf Zulassung biomedizinischer Produkte unterstützen könnte.
Die Phase-3-Studie AURORA wird randomisiert, doppelblind und placebo-kontrolliert sein und umfasst etwa 100 Teilnehmer mit AChR Ab+ MG. Die Studie wird Descartes-08 im Vergleich zu Placebo mit sechs wöchentlichen Infusionen ohne vorbehandelte Chemotherapie evaluieren. Der primäre Endpunkt wird die Verbesserung des MG-ADL-Scores messen.
Die aktuellen Ergebnisse der Phase-2b-Studie zeigten vielversprechende Ergebnisse, da behandelte Teilnehmer (n=12) im 4. Monat einen durchschnittlichen Rückgang des MG-ADL-Scores um 5,5 erreichten. Die Behandlung zeigte eine gute Verträglichkeit und unterstützte die ambulante Verabreichung ohne erforderliche lymphodepleting Chemotherapie.
- FDA SPA agreement provides regulatory clarity for Phase 3 trial design
- Phase 2b results showed significant MG-ADL score reduction of 5.5 points
- Treatment can be administered in outpatient setting without chemotherapy
- Phase 3 trial design finalized with clear pathway to potential approval
- Phase 3 trial not yet started, with timeline extending to 1H25
- Small sample size (n=12) in Phase 2b trial primary efficacy dataset
Insights
The FDA's Special Protocol Assessment agreement for Cartesian's Phase 3 AURORA trial is a crucial de-risking event that substantially increases the probability of eventual approval for Descartes-08. The SPA effectively provides a regulatory roadmap, reducing uncertainty about the trial design's adequacy for future marketing approval.
The Phase 2b results are particularly compelling from both clinical and commercial perspectives. The observed 5.5-point reduction in MG-ADL scores represents a clinically meaningful improvement that exceeds the Phase 3 trial's targeted 3-point improvement threshold. This suggests a robust therapeutic effect that could position Descartes-08 competitively in the
Two key differentiating factors enhance Descartes-08's commercial potential:
- Outpatient administration capability without requiring preconditioning chemotherapy, significantly reducing treatment burden and healthcare costs
- The mRNA cell therapy approach offers potential for deeper, more durable responses compared to conventional treatments
The Phase 3 trial design with 100 participants is well-powered to detect the targeted treatment effect, while the 1:1 randomization and double-blind design ensure robust data quality. The focus on AChR Ab+ patients, who represent approximately
The planned trial initiation in 1H25 positions Cartesian for potential market entry by 2027-2028, assuming standard regulatory review timelines. This timing could allow them to capture significant market share in the growing MG treatment landscape, particularly given the convenient administration profile that could drive strong adoption among both physicians and patients.
Trial on track to commence in 1H25
FREDERICK, Md., Jan. 27, 2025 (GLOBE NEWSWIRE) -- Cartesian Therapeutics, Inc. (NASDAQ: RNAC) (the “Company”), a clinical-stage biotechnology company pioneering mRNA cell therapy for autoimmune diseases, today announced that it has received written agreement from the U.S. Food and Drug Administration (FDA) under the Special Protocol Assessment (SPA) process on the overall design of the Company’s planned Phase 3 AURORA trial for Descartes-08, its lead mRNA cell therapy candidate, in myasthenia gravis (MG). The SPA agreement indicates that the FDA has determined that the proposed trial design is acceptable to support a future Biologics License Application for Descartes-08 in MG, subject to the ultimate outcome of the trial.
“This SPA agreement marks an important milestone in the development of Descartes-08 for MG, providing critical regulatory clarity and a clear path toward potential approval,” said Carsten Brunn, Ph.D., President and Chief Executive Officer of Cartesian. “Supported by compelling Phase 2b results where we observed deep and durable improvements in patients with MG, we firmly believe that Descartes-08 has the potential to serve as a meaningful new therapy that can be delivered in the convenient outpatient setting without the need for preconditioning chemotherapy. We look forward to commencing the Phase 3 AURORA trial in the first half of this year.”
The randomized, double-blind, placebo-controlled Phase 3 AURORA trial is designed to assess Descartes-08 versus placebo (1:1 randomization) administered as six once weekly infusions without preconditioning chemotherapy in approximately 100 participants with acetylcholine receptor autoantibody positive (AChR Ab+) MG. The primary endpoint will assess the proportion of Descartes-08 participants with an improvement in MG-ADL score of three points or more at Month 4 compared to placebo.
In December 2024, the Company announced positive updated results from the Phase 2b trial of Descartes-08 in participants with MG. Deepening responses were observed over time, with Descartes-08-treated participants included in the primary efficacy dataset (n=12) experiencing an average MG Activities of Daily Living (MG-ADL) reduction of 5.5 (±1.1) at Month 4. Consistent with previously reported data, Descartes-08 was observed to be well-tolerated, supporting outpatient administration without the need for lymphodepleting chemotherapy.
About Myasthenia Gravis
Myasthenia gravis (MG) is a chronic autoimmune disorder that causes disabling muscle weakness and fatigue. For most people with MG, the disease is characterized by the presence of antibodies against the acetylcholine receptor, a protein found on the surface of nerve cells that plays a key role in muscle contraction. There is currently no cure for MG, and treatment typically requires chronic immunosuppressive medicines, with their attendant risks and side effects.
About Descartes-08
Descartes-08, Cartesian’s lead mRNA cell therapy candidate, is an autologous mRNA-engineered chimeric antigen receptor T-cell therapy (mRNA CAR-T) product targeting B-cell maturation antigen (BCMA) in clinical development for generalized myasthenia gravis (MG) and systemic lupus erythematosus. In contrast to conventional DNA-based CAR T-cell therapies, mRNA CAR-T administration is designed to not require preconditioning chemotherapy, can be administered in the outpatient setting, and does not carry the risk of genomic integration associated with cancerous transformation. Descartes-08 has been granted Orphan Drug Designation and Regenerative Medicine Advanced Therapy Designation by the U.S. Food and Drug Administration for the treatment of MG, and Rare Pediatric Disease Designation for the treatment of juvenile dermatomyositis.
About Cartesian Therapeutics
Cartesian Therapeutics is a clinical-stage company pioneering mRNA cell therapy for the treatment of autoimmune diseases. The Company’s lead asset, Descartes-08, is an mRNA CAR-T in Phase 2b clinical development for patients with generalized myasthenia gravis and Phase 2 development for systemic lupus erythematosus, with a Phase 2 basket trial planned in additional autoimmune indications. The Company’s clinical-stage pipeline also includes Descartes-15, a next-generation, autologous anti-BCMA mRNA CAR-T. For more information, please visit www.cartesiantherapeutics.com or follow the Company on LinkedIn or X, formerly known as Twitter.
Forward Looking Statements
Any statements in this press release about the future expectations, plans and prospects of the Company, including without limitation, statements about the Company’s planned Phase 3 AURORA trial, statements regarding observations and data from the myasthenia gravis Phase 2a/2b trial, the ability of the Company’s product candidates to be administered in an outpatient setting or without the need for preconditioning lymphodepleting chemotherapy, the potential of Descartes-08, Descartes-15, or any of the Company’s other product candidates to treat myasthenia gravis, systemic lupus erythematosus, juvenile dermatomyositis, or any other disease, the anticipated timing or the outcome of ongoing and planned clinical trials, studies and data readouts, the anticipated timing or the outcome of the FDA’s review of the Company’s regulatory filings, the Company’s ability to conduct its clinical trials and preclinical studies, the timing or making of any regulatory filings, the novelty of treatment paradigms that the Company is able to develop, the potential of any therapies developed by the Company to fulfill unmet medical needs, and enrollment in the Company’s clinical trials and other statements containing the words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “hypothesize,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would,” and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including, but not limited to, the following: the uncertainties inherent in the initiation, completion and cost of clinical trials including proof of concept trials, including uncertain outcomes, the availability and timing of data from ongoing and future clinical trials and the results of such trials, whether preliminary results from a particular clinical trial will be predictive of the final results of that trial and whether results of early clinical trials will be indicative of the results of later clinical trials, the ability to predict results of studies performed on human beings based on results of studies performed on non-human subjects, the unproven approach of the Company’s technology, potential delays in enrollment of patients, undesirable side effects of the Company’s product candidates, its reliance on third parties to conduct its clinical trials, the Company’s inability to maintain its existing or future collaborations, licenses or contractual relationships, its inability to protect its proprietary technology and intellectual property, potential delays in regulatory approvals, the availability of funding sufficient for its foreseeable and unforeseeable operating expenses and capital expenditure requirements, the Company’s recurring losses from operations and negative cash flows, substantial fluctuation in the price of the Company’s common stock, risks related to geopolitical conflicts and pandemics and other important factors discussed in the “Risk Factors” section of the Company’s most recent Annual Report on Form 10-K and subsequently filed Quarterly Reports on Form 10-Q, and in other filings that the Company makes with the Securities and Exchange Commission. In addition, any forward-looking statements included in this press release represent the Company’s views only as of the date of its publication and should not be relied upon as representing its views as of any subsequent date. The Company specifically disclaims any intention to update any forward-looking statements included in this press release, except as required by law.
Investor Contact
Megan LeDuc
Associate Director, Investor Relations
megan.leduc@cartesiantx.com
Media Contact
David Rosen
Argot Partners
david.rosen@argotpartners.com
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