Cartesian Therapeutics Announces Presentation of Results from Phase 2b Trial of Descartes-08 in Patients with Myasthenia Gravis at the 2024 MGFA Scientific Session of the AANEM Annual Meeting
Cartesian Therapeutics (NASDAQ: RNAC) presented data from its Phase 2b trial of Descartes-08 in patients with generalized myasthenia gravis (MG) at the 2024 MGFA Scientific Session of the AANEM Annual Meeting. The trial achieved its primary endpoint with statistical significance, with 71% of Descartes-08 treated patients showing ≥5-point improvements in MG Composite score at Month 3 compared to 25% in the placebo group (p=0.018).
Descartes-08, an autologous mRNA-engineered CAR-T therapy targeting BCMA, demonstrated a favorable safety profile supporting outpatient administration without preconditioning chemotherapy. The treatment was associated with reduced anti-AChR antibody titer levels and did not lead to decreased vaccine titers or increased infection rates.
Cartesian plans to hold an End-of-Phase 2 meeting with the FDA by year-end 2024 to discuss initiating a Phase 3 clinical trial of Descartes-08 in MG.
Cartesian Therapeutics (NASDAQ: RNAC) ha presentato dati dal trial Fase 2b di Descartes-08 in pazienti con miastenia gravis generalizzata (MG) durante la Sessione Scientifica MGFA dell'Annual Meeting AANEM 2024. Il trial ha raggiunto il endpoint primario con significatività statistica, con il 71% dei pazienti trattati con Descartes-08 che mostrano miglioramenti di almeno 5 punti nel punteggio composito MG al Mese 3 rispetto al 25% del gruppo placebo (p=0.018).
Descartes-08, una terapia CAR-T ingegnerizzata con mRNA autologo che mira a BCMA, ha dimostrato un profilo di sicurezza favorevole che supporta la somministrazione ambulatoriale senza chemioterapia di precondizionamento. Il trattamento è stato associato a livelli ridotti di anticorpi anti-AChR e non ha portato a diminuzioni dei titoli vaccinali o a un aumento delle infezioni.
Cartesian prevede di tenere un incontro di fine Fase 2 con la FDA entro la fine del 2024 per discutere l'avvio di un trial clinico di Fase 3 di Descartes-08 in MG.
Cartesian Therapeutics (NASDAQ: RNAC) presentó datos del ensayo de Fase 2b de Descartes-08 en pacientes con miastenia gravis generalizada (MG) en la Sesión Científica MGFA de la Reunión Anual AANEM 2024. El ensayo logró su punto final primario con significancia estadística, con el 71% de los pacientes tratados con Descartes-08 mostrando mejoras de ≥5 puntos en el puntaje compuesto de MG en el Mes 3 en comparación con el 25% en el grupo placebo (p=0.018).
Descartes-08, una terapia CAR-T ingenierizada con mRNA autólogo que apunta a BCMA, demostró un perfil de seguridad favorable que soporta la administración ambulatoria sin quimioterapia de preacondicionamiento. El tratamiento fue asociado a niveles reducidos de anticuerpos anti-AChR y no condujo a disminuciones en los títulos de vacuna ni a un aumento en las tasas de infección.
Cartesian planea realizar una reunión de Fin de Fase 2 con la FDA para finales de 2024 para discutir el inicio de un ensayo clínico de Fase 3 de Descartes-08 en MG.
카르테시안 치료제(Cartesian Therapeutics) (NASDAQ: RNAC)는 2024 AANEM 연례 회의의 MGFA 과학 세션에서 일반화형 중증 근무력증(MG) 환자를 대상으로 한 Descartes-08의 2b 단계 시험 데이터를 발표했습니다. 이 시험은 통계적으로 유의한 주요 목표를 달성했습니다, Descartes-08 치료를 받은 환자의 71%가 3개월 동안 MG 복합점수에서 5점 이상의 개선을 보였으며, 이는 플라세보 그룹의 25%와 비교됩니다 (p=0.018).
Descartes-08은 BCMA를 표적으로 하는 자가유래 mRNA 조작 CAR-T 치료법으로, 사전 화학요법 없이 외래 환자에게 시행할 수 있는 안전성을 나타냈습니다. 이 치료법은 anti-AChR 항체 역가 수준을 감소시키며 백신 역가 감소나 감염률 증가로 이어지지 않았습니다.
카르테시안은 2024년 말까지 FDA와의 2단계 종결 회의를 개최할 계획이며, MG에 대한 Descartes-08의 3단계 임상 시험 시작에 대해 논의할 예정입니다.
Cartesian Therapeutics (NASDAQ: RNAC) a présenté des données de son essai de Phase 2b pour Descartes-08 chez des patients atteints de myasthénie grave généralisée (MG) lors de la session scientifique MGFA de la réunion annuelle AANEM 2024. L'essai a atteint son point final principal avec une signification statistique, avec 71% des patients traités par Descartes-08 montrant des améliorations de ≥5 points dans le score composite de MG au mois 3, contre 25% dans le groupe placebo (p=0.018).
Descartes-08, une thérapie CAR-T à ARN messager autologue ciblant le BCMA, a démontré un profil de sécurité favorable permettant une administration en ambulatoire sans chimiothérapie de préconditionnement. Le traitement était associé à des niveaux réduits d'anticorps anti-AChR et n'a pas conduit à une diminution des titres de vaccins ni à une augmentation des taux d'infection.
Cartesian prévoit de tenir une réunion de fin de phase 2 avec la FDA d'ici la fin de l'année 2024 pour discuter de l'initiation d'un essai clinique de phase 3 de Descartes-08 dans la MG.
Cartesian Therapeutics (NASDAQ: RNAC) präsentierte Daten aus der Phase 2b-Studie zu Descartes-08 bei Patienten mit generalisierter Myasthenia gravis (MG) während der MGFA-Wissenschaftssitzung der AANEM-Jahrestagung 2024. Die Studie erreichte ihr primäres Endziel mit statistischer Signifikanz, wobei 71% der mit Descartes-08 behandelten Patienten im Monat 3 ≥5 Punkte Verbesserungen im MG-Kompositwert im Vergleich zu 25% in der Placebogruppe zeigten (p=0.018).
Descartes-08, eine autologe mRNA-entwickelte CAR-T-Therapie, die auf BCMA abzielt, zeigte ein günstiges Sicherheitsprofil, das eine ambulante Verabreichung ohne vorgelagerte Chemotherapie unterstützt. Die Behandlung war mit verringerten Anti-AChR-Antikörpertitern assoziiert und führte nicht zu verminderten Impftitern oder erhöhten Infektionsraten.
Cartesian plant, bis Ende 2024 ein Ende-der-Phase-2-Treffen mit der FDA abzuhalten, um den Beginn eines Phase-3-Klinikversuchs von Descartes-08 in MG zu besprechen.
- Phase 2b trial of Descartes-08 achieved primary endpoint with statistical significance (p=0.018)
- 71% of Descartes-08 treated patients showed ≥5-point improvements in MG Composite score at Month 3
- Descartes-08 demonstrated a favorable safety profile supporting outpatient administration
- Treatment associated with reduced anti-AChR antibody titer levels
- No observed decrease in vaccine titers or increased infection rates
- FDA granted Regenerative Medicine Advanced Therapy (RMAT) Designation and Orphan Drug Designation for Descartes-08
- None.
Insights
The Phase 2b trial results for Descartes-08 in myasthenia gravis (MG) patients are highly encouraging. The treatment demonstrated statistically significant efficacy, with
Importantly, Descartes-08 showed a favorable safety profile, allowing for outpatient administration without chemotherapy preconditioning. This could be a significant advantage over existing therapies. The observed reduction in anti-AChR antibody titers and maintenance of vaccine titers suggest potential for long-term efficacy without compromising immune function.
With plans for an End-of-Phase 2 FDA meeting by year-end, Cartesian is well-positioned to advance to Phase 3. If successful, Descartes-08 could become a game-changing treatment for MG patients, potentially offering improved efficacy and convenience over current options.
This clinical progress represents a significant milestone for Cartesian Therapeutics. The positive Phase 2b results for Descartes-08 in myasthenia gravis could substantially increase the company's market valuation, especially considering its current market cap of
The MG treatment market is projected to grow, with some estimates suggesting it could reach
Investors should note that the company is advancing towards a critical Phase 3 trial, which will require substantial funding. This may lead to dilutive financing rounds in the near future. However, positive trial results and FDA interactions could attract partnership opportunities or acquisition interest from larger pharmaceutical companies, potentially driving up RNAC's stock price.
Company continues to expect to hold an End-of-Phase 2 meeting with the FDA by year-end
GAITHERSBURG, Md., Oct. 15, 2024 (GLOBE NEWSWIRE) -- Cartesian Therapeutics, Inc. (NASDAQ: RNAC) (the “Company”), a clinical-stage biotechnology company pioneering mRNA cell therapy for autoimmune diseases, today announced the presentation of data from its Phase 2b trial of Descartes-08 in patients with generalized myasthenia gravis (MG) during the 2024 Myasthenia Gravis Foundation of America (MGFA) Scientific Session of the American Association of Neuromuscular and Electrodiagnostic Medicine (AANEM) Annual Meeting being held today in Savannah, Georgia. Positive topline results from the trial were previously announced in July 2024. A copy of today’s presentation will be available in the Publications and Presentations section of the Company’s website.
Descartes-08, Cartesian’s lead mRNA cell therapy candidate, is an autologous mRNA-engineered chimeric antigen receptor T-cell therapy (mRNA CAR-T) product candidate targeting B-cell maturation antigen (BCMA). Descartes-08 is designed to be administered without preconditioning chemotherapy and does not use integrating vectors. Descartes-08 was previously granted Regenerative Medicine Advanced Therapy (RMAT) Designation and Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for the treatment of MG.
“Descartes-08 has strong potential for deep and durable improvements in patients with MG treated in the convenient outpatient setting without preconditioning chemotherapy and with no observed-to-date increased risk of infection,” said Carsten Brunn, Ph.D., President and Chief Executive Officer of Cartesian. “We look forward to continuing to collaborate with the FDA to determine next steps for the Descartes-08 program for MG and plan to hold an End-of-Phase 2 meeting by the end of this year. We are steadfast in our commitment to advancing our pipeline of innovative mRNA cell therapy candidates to extend the reach of this powerful modality to patients with autoimmune conditions.”
Trial Overview and Topline Results
In the Phase 2b double-blind, placebo-controlled, crossover trial, a total of 36 heavily pre-treated, highly symptomatic patients with MG were randomized 1:1 to receive either Descartes-08 or placebo administered as six weekly outpatient infusions without preconditioning chemotherapy. At the conclusion of the trial’s Month 3 blinded follow-up assessment, patients receiving placebo were eligible to cross over to Descartes-08 treatment.
- The trial achieved its primary endpoint with statistical significance in the pre-specified modified intent-to-treat efficacy population, with
71% (10/14) of patients treated with Descartes-08 observed to have 5-point or greater improvements in MG Composite (MGC) score at Month 3 compared to25% (3/12) of patients treated with placebo (p=0.018). - Responders that reached their four-month and six-month assessments were observed to have deep, durable, and clinically meaningful improvements in their MGC severity scores.
- Descartes-08 was observed to have a favorable safety profile supporting outpatient administration without the need for lymphodepleting chemotherapy.
Additional Data Featured in Presentation
In addition to the previously announced topline results, data presented today included the following:
- Patients treated with Descartes-08 were observed to have a reduction in anti-acetylcholine receptor (AChR) antibody titer levels at three months, which is in line with findings from the previously completed Phase 2a trial, compared to an increase observed in patients receiving placebo. Reductions in AChR antibody titer levels are believed to be an early prognostic indicator for clinical improvement in patients with MG.
- Treatment with Descartes-08 was not observed to lead to a decrease in vaccine titers for common viruses and was not associated with increased rates of infection or hypogammaglobulinemia.
The Company expects to hold an End-of-Phase 2 meeting with the FDA by year-end 2024 to review data from the Phase 2b trial and discuss plans for initiating a Phase 3 clinical trial of Descartes-08 in MG.
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 Cartesian Therapeutics
Cartesian Therapeutics is a clinical-stage company pioneering mRNA cell therapies 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 systematic 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 regarding the ability of Descartes-08 and Descartes-15 to be administered in an outpatient setting or without the need for preconditioning lymphodepleting chemotherapy or integrating vectors, the potential of the Company’s technology to enable precision control and optimization of engineered cells for diverse cell therapies leveraging multiple modalities, the potential of Descartes-08, Descartes-15, or any of the Company’s other product candidates to treat myasthenia gravis, systemic lupus erythematosus, multiple myeloma, 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, reductions in AChR antibody titer levels being believed to be an early prognostic indicator for clinical improvement in patients with myasthenia gravis, the Company’s ability to conduct its clinical trials and preclinical studies, the timing or making of any regulatory filings, the anticipated timing or outcome of selection of developmental product candidates, the ability of the Company to consummate any expected agreements and licenses and to realize the anticipated benefits thereof, 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 or preclinical studies 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
Melissa Forst
Argot Partners
cartesian@argotpartners.com
Media Contact
David Rosen
Argot Partners
david.rosen@argotpartners.com
FAQ
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