Cartesian Therapeutics’ Descartes-08 Observed to Provide Deep and Sustained Benefits Through Month 12 After a Single Course of Therapy in Phase 2b Myasthenia Gravis Trial
Cartesian Therapeutics (NASDAQ: RNAC) announced promising 12-month data from its Phase 2b trial of Descartes-08, a CAR-T cell therapy for myasthenia gravis (MG). After a single six-week treatment course, patients showed sustained benefits with an average 4.8-point reduction in MG-ADL at Month 12.
Key highlights:
- Patients without prior biologic therapy exposure showed the strongest response, with a 7.1-point reduction in MG-ADL and 57% maintaining minimum symptom expression at Month 12
- 83% of evaluable participants maintained clinically meaningful response through Month 12
- Safety profile remained favorable with no cytokine release syndrome or neurotoxicity cases
- Treatment was administered in outpatient setting without chemotherapy
The company received FDA's RMAT and Orphan Drug Designations for Descartes-08, and plans to initiate the Phase 3 AURORA trial in Q2 2025.
Cartesian Therapeutics (NASDAQ: RNAC) ha annunciato dati promettenti a 12 mesi dal suo studio di Fase 2b su Descartes-08, una terapia CAR-T per la miastenia grave (MG). Dopo un singolo ciclo di trattamento di sei settimane, i pazienti hanno mostrato benefici sostenuti con una riduzione media di 4,8 punti nell'MG-ADL al mese 12.
Principali punti salienti:
- I pazienti senza esposizione precedente a terapie biologiche hanno mostrato la risposta più forte, con una riduzione di 7,1 punti nell'MG-ADL e il 57% ha mantenuto un'espressione minima dei sintomi al mese 12
- Il 83% dei partecipanti valutabili ha mantenuto una risposta clinicamente significativa fino al mese 12
- Il profilo di sicurezza è rimasto favorevole, senza casi di sindrome da rilascio di citochine o neurotossicità
- Il trattamento è stato somministrato in regime ambulatoriale senza chemioterapia
La società ha ricevuto le designazioni RMAT e Orphan Drug dalla FDA per Descartes-08 e prevede di avviare lo studio di Fase 3 AURORA nel secondo trimestre del 2025.
Cartesian Therapeutics (NASDAQ: RNAC) anunció datos prometedores de 12 meses de su ensayo de Fase 2b sobre Descartes-08, una terapia CAR-T para la miastenia gravis (MG). Después de un único ciclo de tratamiento de seis semanas, los pacientes mostraron beneficios sostenidos con una reducción promedio de 4.8 puntos en el MG-ADL al mes 12.
Puntos destacados:
- Los pacientes sin exposición previa a terapia biológica mostraron la respuesta más fuerte, con una reducción de 7.1 puntos en el MG-ADL y el 57% mantuvo una expresión mínima de síntomas al mes 12
- El 83% de los participantes evaluables mantuvieron una respuesta clínicamente significativa hasta el mes 12
- El perfil de seguridad se mantuvo favorable sin casos de síndrome de liberación de citoquinas o neurotoxicidad
- El tratamiento se administró en un entorno ambulatorio sin quimioterapia
La empresa recibió las designaciones RMAT y de Medicamento Huérfano de la FDA para Descartes-08, y planea iniciar el ensayo de Fase 3 AURORA en el segundo trimestre de 2025.
Cartesian Therapeutics (NASDAQ: RNAC)는 중증 근무력증(MG)을 위한 CAR-T 세포 치료제인 Descartes-08의 2b상 시험에서 12개월의 유망한 데이터를 발표했습니다. 6주간의 단일 치료 과정을 거친 환자들은 12개월 차에 MG-ADL에서 평균 4.8점 감소를 보이며 지속적인 혜택을 나타냈습니다.
주요 하이라이트:
- 이전 생물학적 치료를 받지 않은 환자들이 가장 강한 반응을 보였으며, MG-ADL에서 7.1점 감소와 함께 57%가 12개월 차에 최소한의 증상 표현을 유지했습니다.
- 평가 가능한 참가자의 83%가 12개월 차까지 임상적으로 의미 있는 반응을 유지했습니다.
- 안전성 프로필은 사이토카인 방출 증후군이나 신경독성 사례 없이 긍정적으로 유지되었습니다.
- 치료는 항암치료 없이 외래 환자 환경에서 시행되었습니다.
회사는 Descartes-08에 대해 FDA의 RMAT 및 희귀의약품 지정을 받았으며, 2025년 2분기에 3상 AURORA 시험을 시작할 계획입니다.
Cartesian Therapeutics (NASDAQ: RNAC) a annoncé des données prometteuses à 12 mois de son essai de Phase 2b sur Descartes-08, une thérapie CAR-T pour la myasthénie grave (MG). Après un seul traitement de six semaines, les patients ont montré des bénéfices durables avec une réduction moyenne de 4,8 points dans le MG-ADL au mois 12.
Points clés :
- Les patients sans exposition préalable à une thérapie biologique ont montré la réponse la plus forte, avec une réduction de 7,1 points dans le MG-ADL et 57 % ont maintenu une expression minimale des symptômes au mois 12
- 83 % des participants évaluables ont maintenu une réponse cliniquement significative jusqu'au mois 12
- Le profil de sécurité est resté favorable, sans cas de syndrome de libération de cytokines ou de neurotoxicité
- Le traitement a été administré en ambulatoire sans chimiothérapie
L'entreprise a reçu les désignations RMAT et médicament orphelin de la FDA pour Descartes-08, et prévoit de lancer l'essai de Phase 3 AURORA au deuxième trimestre de 2025.
Cartesian Therapeutics (NASDAQ: RNAC) hat vielversprechende 12-Monats-Daten aus seiner Phase-2b-Studie zu Descartes-08, einer CAR-T-Zelltherapie für Myasthenia gravis (MG), bekannt gegeben. Nach einer einzigen sechsmonatigen Behandlungsreihe zeigten die Patienten nachhaltige Vorteile mit einer durchschnittlichen Reduktion von 4,8 Punkten im MG-ADL nach 12 Monaten.
Wichtige Highlights:
- Patienten ohne vorherige Exposition gegenüber biologischen Therapien zeigten die stärkste Reaktion, mit einer Reduktion von 7,1 Punkten im MG-ADL und 57% der Patienten hielten nach 12 Monaten eine minimale Symptomausprägung aufrecht.
- 83% der bewertbaren Teilnehmer hielten bis zum 12. Monat eine klinisch bedeutsame Reaktion aufrecht.
- Das Sicherheitsprofil blieb günstig, ohne Fälle von Zytokinfreisetzungssyndrom oder Neurotoxizität.
- Die Behandlung wurde in einer ambulanten Umgebung ohne Chemotherapie durchgeführt.
Das Unternehmen erhielt von der FDA die RMAT- und Orphan-Drug-Zulassungen für Descartes-08 und plant, die Phase-3-Studie AURORA im 2. Quartal 2025 zu starten.
- Strong efficacy with 4.8-point reduction in MG-ADL at Month 12
- 83% of patients maintained clinically meaningful response through Month 12
- Superior results in treatment-naive patients (7.1-point MG-ADL reduction)
- Favorable safety profile with no serious adverse events
- FDA RMAT and Orphan Drug Designations received
- Special Protocol Assessment agreement obtained for Phase 3 trial
- Three participants were lost to follow-up after Month 3 assessments
- 60% of patients experienced infusion-related reactions
Insights
Cartesian's 12-month data for Descartes-08 in myasthenia gravis delivers impressive durability with a single treatment course, addressing a critical unmet need in this chronic autoimmune disease. The 4.8-point reduction in MG-ADL scores at month 12 demonstrates sustained clinical benefit well beyond the initial treatment period.
Most striking is the 7.1-point MG-ADL reduction in biologic-naïve patients, with 57% achieving minimum symptom expression through month 12. This suggests Descartes-08 may be particularly effective as an earlier-line therapy rather than after biologic failure.
The clean safety profile represents a significant advantage over traditional CAR-T approaches - no cytokine release syndrome or neurotoxicity was observed, enabling outpatient administration without lymphodepleting chemotherapy. This dramatically improves the risk-benefit profile for a chronic condition like MG.
From a regulatory perspective, the FDA's Special Protocol Assessment agreement for the Phase 3 AURORA trial substantially de-risks the approval pathway. Combined with RMAT and Orphan Drug designations, Cartesian has secured significant regulatory advantages.
This data positions Descartes-08 as potentially transformative in MG treatment by offering what appears to be durable efficacy from a single treatment course rather than continuous therapy. The strong 12-month data should accelerate enrollment in the upcoming Phase 3 trial.
The 12-month data for Descartes-08 represents a potential paradigm shift in myasthenia gravis management. Current treatments including acetylcholinesterase inhibitors, immunosuppressants, complement inhibitors and FcRn antagonists require chronic administration, while this data suggests sustained benefit for 12 months following a single 6-week treatment course.
The 4.8-point reduction in MG-ADL and 6.0-point reduction in QMG scores at month 12 exceed the clinically meaningful thresholds for MG symptom improvement. Most impressive is that 83% of participants maintained clinically meaningful responses throughout the follow-up period.
The subgroup analysis showing enhanced efficacy in biologic-naïve patients (7.1-point MG-ADL reduction) provides strong rationale for potential positioning earlier in the treatment algorithm. The achievement of minimum symptom expression in 57% of these patients represents a disease control level rarely seen with current therapies.
From a safety perspective, the absence of CRS, ICANS, hypogammaglobulinemia, or increased infection rates addresses key concerns that have CAR-T adoption in non-oncology settings. These results suggest Descartes-08 achieves its therapeutic effect through a more targeted immunomodulation rather than broad immunosuppression.
If replicated in the Phase 3 AURORA trial, this approach could fundamentally change how we manage myasthenia gravis by providing long-term disease control with minimal treatment burden.
After a single course of therapy, Descartes-08-treated participants were observed to sustain deep responses through long-term follow-up, with an average 4.8-point reduction in MG-ADL at Month 12
Deepest and most compelling sustained responses observed in Descartes-08-treated participants who did not have prior exposure to biologic therapies, with an average 7.1-point reduction in MG-ADL and
Safety profile consistent with previously reported data and continues to support outpatient administration
Phase 3 AURORA trial on track to dose first patient in 2Q25
FREDERICK, Md., April 08, 2025 (GLOBE NEWSWIRE) -- Cartesian Therapeutics, Inc. (NASDAQ: RNAC) (the “Company”), a clinical-stage biotechnology company pioneering cell therapy for autoimmune diseases, today announced 12-month efficacy and safety data from the Phase 2b trial of Descartes-08 in participants with generalized myasthenia gravis (MG). Participants dosed with a single six-week course of treatment of Descartes-08 were observed to continue to experience a sustained benefit in symptoms of MG at the 12-month assessment. The data will be discussed by management at the 24th Annual Needham Virtual Healthcare Conference today, April 8, 2025, and presented tomorrow, April 9, 2025, by Tuan Vu, M.D., Professor of Neurology at the University of South Florida Morsani College of Medicine, at the 2025 American Academy of Neurology Annual Meeting being held in San Diego.
Descartes-08, Cartesian’s lead cell therapy candidate, is an autologous engineered chimeric antigen receptor T-cell therapy (CAR-T) product candidate targeting B-cell maturation antigen (BCMA). Descartes-08 is designed to be administered without preconditioning chemotherapy in an outpatient setting and does not use integrating vectors.
12-Month Phase 2b Trial Results
In the Phase 2b double-blind, placebo-controlled, crossover trial, a total of 36 heavily pretreated, highly symptomatic participants with MG were randomized 1:1 to receive either Descartes-08 or placebo administered as six weekly outpatient infusions without preconditioning chemotherapy. As previously announced, the trial met its primary endpoint and demonstrated a safety profile supporting outpatient administration of Descartes-08. In December 2024, the Company reported updated efficacy and safety data from the trial in which deepening responses were observed over time and some participants were observed to have durable responses through to Month 12.
The primary efficacy dataset for the follow-up portion of the trial consisted of a modified intent-to-treat (mITT) population of all subjects enrolled at academic medical centers who received at least one dose of Descartes-08 and completed at least one post-Month 3 MG Activities of Daily Living (MG-ADL) score follow-up assessment.
As of a March 31, 2025 cutoff date, 12 out of 15 participants who received Descartes-08 in the primary efficacy dataset completed their Month 12 follow-up assessments. Three participants, two of whom were MG Composite (MGC) responders at Month 3, were lost to follow-up after their Month 3 assessments.
12-Month Efficacy Results
- Deep and sustained responses observed through Month 12 (n=12).
- Participants treated with Descartes-08 were observed to have deep responses following initial treatment and sustained symptom improvement, with an average MG-ADL reduction of 5.5 (±1.1) at Month 4 and 4.8 (±1.4) at Month 12.
- Participants treated with Descartes-08 were observed to have an average Quantitative Myasthenia Gravis Score (QMG) reduction of 4.8 (±1.7) points at Month 4, which deepened through Month 12 (6.0±2.1).
33% (4/12) of participants achieved minimum symptom expression (MSE), defined as an MG-ADL score of 0 or 1, at Month 6, all of whom maintained MSE through Month 12.83% (10/12) of evaluable participants maintained a clinically meaningful response through Month 12. Clinically meaningful response is defined as a reduction in MG-ADL score of at least 2 points.
- Deepest and most compelling sustained responses observed in participants without prior biologic therapies (n=7).
- The subset of participants who did not have exposure to prior biologic therapies, including complement or neonatal fragment crystallizable receptor (FcRn) inhibitors, were observed to exhibit a deepening of responses throughout the year, with an average MG-ADL reduction of 6.6 (±1.5) at Month 4 and 7.1 (±1.9) at Month 12.
- The participants treated with Descartes-08 without exposure to prior biologic therapies were observed to have an average QMG reduction of 5.9 (±2.4) points at Month 4, which deepened through Month 12 (9.4±2.6).
57% (4/7) of these participants were observed to achieve MSE at Month 6 which was maintained through Month 12.100% (7/7) of these participants were observed to maintain at least a clinically meaningful response through Month 12.
Safety
- Well-tolerated safety profile supports outpatient administration without the need for lymphodepleting chemotherapy.
- Consistent with previously reported data, Descartes-08 was observed to be well-tolerated across the safety dataset through Month 12 (n=12), and adverse events were transient and mostly mild, with no new adverse events reported in the 12-month follow-up data. Notably, there were no cases of cytokine release syndrome (CRS), and no cases of immune effector cell-associated neurotoxicity syndrome (ICANS). In addition, 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.
- There were no Descartes-08-related adverse events reported in Month 4 through Month 12 follow-up. As previously reported, common side effects through the Month 3 primary endpoint observed in participants who received any dose of Descartes-08 were infusion-related reactions manifesting as fever (
60% of participants receiving Descartes-08), chills (60% of participants receiving Descartes-08), headache (55% of participants receiving Descartes-08) and nausea (45% of participants receiving Descartes-08), all of which typically resolved within 24 hours of infusion.
"This impressive data highlights the potential of Descartes-08 to serve as an important therapeutic option to deliver deep and sustained reductions in MG-ADL for patients with myasthenia gravis,” said Tuan Vu, M.D., Professor of Neurology at the University of South Florida Morsani College of Medicine, Division Director for Neuromuscular Medicine and EMG and investigator in the Phase 2b trial. “The data in participants who had not received prior biologic therapy is particularly striking as this population is most comparable to the patient populations in trials of standard-of-care biologics. Participants in this subgroup who were randomized to Descartes-08 were observed to experience a profound average 7.1-point reduction in MG-ADL and
“The remarkable results presented today underscore the potential of utilizing our cell therapy to deliver deep and durable responses in MG patients one year after receiving a single course of therapy in a convenient outpatient setting with no preconditioning chemotherapy,” said Carsten Brunn, Ph.D., President and Chief Executive Officer of Cartesian. “The impressive strength and duration of response shown in the data reinforce our confidence in the potential of Descartes-08 to transform the current treatment landscape in MG, offering patients a safe, flexible, and durable treatment option. We look forward to dosing the first patient in our Phase 3 AURORA trial in the second quarter of this year.”
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. Cartesian received written agreement from the FDA under the Special Protocol Assessment (SPA) process indicating the overall design of the planned Phase 3 AURORA trial of Descartes-08 is acceptable to support a future Biologics License Application in MG, subject to the ultimate outcome of the trial. Cartesian remains on track to commence the Phase 3 AURORA trial of Descartes-08 in MG in the second quarter of 2025.
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 cell therapy candidate, is an autologous chimeric antigen receptor T-cell therapy (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, Cartesian’s 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 cell therapy for the treatment of autoimmune diseases. The Company’s lead asset, Descartes-08, is a CAR-T entering Phase 3 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. A Phase 3 trial of Descartes-08 in patients with generalized myasthenia gravis has received written agreement from the FDA under the Special Protocol Assessment process. The Company’s clinical-stage pipeline also includes Descartes-15, a next-generation, autologous anti-BCMA CAR-T currently being evaluated in a Phase 1 trial in patients with multiple myeloma. 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 observations and data from the Company’s clinical trials of Descartes-08 in myasthenia gravis, the anticipated timing or the outcome of ongoing and planned clinical trials, studies and data readouts, 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 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, whether results of early clinical trials will be indicative of the results of later clinical trials, and whether results observed in certain patient subgroups will be indicative of the results in such subgroups in later clinical trials or are reflective of a product candidate’s overall characteristics, 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 of Investor Relations
megan.leduc@cartesiantx.com
Media Contact
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Argot Partners
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FAQ
What were the key efficacy results of RNAC's Descartes-08 Phase 2b trial in myasthenia gravis?
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