Cabaletta Bio Announces Updated Clinical Data Demonstrating Deepening Clinical Responses across Multiple Indications with Rese-cel at February Scientific Meetings
Cabaletta Bio (NASDAQ: CABA) has reported updated clinical data for resecabtagene autoleucel (rese-cel) across multiple autoimmune indications. The data covers 10 patients treated through the RESET clinical development program, showing significant clinical responses:
Key highlights include three SLE patients achieving DORIS remission, the first lupus nephritis patient reaching complete renal response, and the first dermatomyositis patient maintaining major TIS improvement. Notably, all these patients discontinued immunosuppressants and steroids. The safety profile remains favorable, with 90% of patients experiencing either no or Grade 1 CRS, and 90% showing no ICANS.
The company's clinical trial network has expanded to 50 sites across the U.S. and Europe, with 26 patients enrolled in the RESET program as of February 13, 2025. Deep B cell depletion was observed in all patients post-treatment, with B cell repopulation typically starting around 2 months post-infusion.
Cabaletta Bio (NASDAQ: CABA) ha riportato dati clinici aggiornati per resecabtagene autoleucel (rese-cel) in diverse indicazioni autoimmuni. I dati riguardano 10 pazienti trattati attraverso il programma di sviluppo clinico RESET, mostrando risposte cliniche significative:
I punti salienti includono tre pazienti con SLE che hanno raggiunto la remissione DORIS, il primo paziente con nefrite lupica che ha ottenuto una risposta renale completa e il primo paziente con dermatomiosite che ha mantenuto un notevole miglioramento del TIS. È importante notare che tutti questi pazienti hanno interrotto l'uso di immunosoppressori e steroidi. Il profilo di sicurezza rimane favorevole, con il 90% dei pazienti che ha sperimentato nessun effetto o un CRS di Grado 1, e il 90% che non ha mostrato ICANS.
La rete di studi clinici dell'azienda si è ampliata a 50 siti negli Stati Uniti e in Europa, con 26 pazienti arruolati nel programma RESET al 13 febbraio 2025. È stata osservata una profonda deplezione delle cellule B in tutti i pazienti dopo il trattamento, con la ripopolazione delle cellule B che inizia solitamente circa 2 mesi dopo l'infusione.
Cabaletta Bio (NASDAQ: CABA) ha reportado datos clínicos actualizados para resecabtagene autoleucel (rese-cel) en múltiples indicaciones autoinmunes. Los datos cubren a 10 pacientes tratados a través del programa de desarrollo clínico RESET, mostrando respuestas clínicas significativas:
Los puntos clave incluyen a tres pacientes con LES que lograron remisión DORIS, el primer paciente con nefritis lúpica que alcanzó una respuesta renal completa y el primer paciente con dermatomiositis que mantuvo una mejora importante en el TIS. Notablemente, todos estos pacientes suspendieron los inmunosupresores y esteroides. El perfil de seguridad sigue siendo favorable, con el 90% de los pacientes experimentando ningún efecto o un CRS de Grado 1, y el 90% mostrando ninguna ICANS.
La red de ensayos clínicos de la compañía se ha expandido a 50 sitios en EE. UU. y Europa, con 26 pacientes inscritos en el programa RESET a partir del 13 de febrero de 2025. Se observó una profunda depleción de células B en todos los pacientes después del tratamiento, con la repoblación de células B que generalmente comienza alrededor de 2 meses después de la infusión.
카발레타 바이오 (NASDAQ: CABA)는 여러 자가면역 질환에 대한 레세카브타겐 오토류셀(레세-셀)의 업데이트된 임상 데이터를 보고했습니다. 이 데이터는 RESET 임상 개발 프로그램을 통해 치료받은 10명의 환자를 포함하며, 중요한 임상 반응을 보여줍니다:
주요 하이라이트로는 SLE 환자 3명이 DORIS 관해를 달성했으며, 첫 번째 루푸스 신염 환자가 완전 신장 반응에 도달했고, 첫 번째 피부근육염 환자가 주요 TIS 개선을 유지한 사례가 있습니다. 특히 이 모든 환자는 면역억제제와 스테로이드를 중단했습니다. 안전성 프로필은 우호적이며, 90%의 환자가 CRS가 없거나 1등급 CRS를 경험했으며, 90%가 ICANS를 보이지 않았습니다.
회사의 임상 시험 네트워크는 미국과 유럽 전역에 50개 사이트로 확장되었으며, 2025년 2월 13일 기준으로 RESET 프로그램에 26명의 환자가 등록되었습니다. 치료 후 모든 환자에서 깊은 B 세포 고갈이 관찰되었고, B 세포 재생은 일반적으로 주입 후 약 2개월 경과 후 시작됩니다.
Cabaletta Bio (NASDAQ: CABA) a rapporté des données cliniques actualisées pour le resecabtagene autoleucel (rese-cel) dans plusieurs indications auto-immunes. Les données concernent 10 patients traités dans le cadre du programme de développement clinique RESET, montrant des réponses cliniques significatives :
Les points clés incluent trois patients atteints de LES ayant atteint la rémission DORIS, le premier patient atteint de néphrite lupique ayant obtenu une réponse rénale complète, et le premier patient atteint de dermatomyosite ayant maintenu une amélioration majeure du TIS. Il est à noter que tous ces patients ont arrêté les immunosuppresseurs et les stéroïdes. Le profil de sécurité reste favorable, avec 90 % des patients ne présentant aucun effet ou un CRS de grade 1, et 90 % montrant aucune ICANS.
Le réseau d'essais cliniques de l'entreprise s'est élargi à 50 sites aux États-Unis et en Europe, avec 26 patients inscrits au programme RESET au 13 février 2025. Une déplétion profonde des cellules B a été observée chez tous les patients après le traitement, avec une repopulation des cellules B commençant généralement environ 2 mois après l'infusion.
Cabaletta Bio (NASDAQ: CABA) hat aktualisierte klinische Daten zu resecabtagene autoleucel (rese-cel) für mehrere autoimmune Indikationen veröffentlicht. Die Daten beziehen sich auf 10 Patienten, die im Rahmen des RESET-Klinikentwicklungsprogramms behandelt wurden und zeigen signifikante klinische Antworten:
Zu den wichtigsten Highlights gehören drei SLE-Patienten, die eine DORIS-Remission erreicht haben, der erste Patient mit Lupusnephritis, der eine vollständige Nierenreaktion erzielt hat, und der erste Patient mit Dermatomyositis, der eine erhebliche Verbesserung des TIS aufrechterhalten hat. Bemerkenswert ist, dass alle diese Patienten die Einnahme von Immunsuppressiva und Steroiden eingestellt haben. Das Sicherheitsprofil bleibt günstig, da 90 % der Patienten entweder keine oder Grad 1 CRS erlebten und 90 % keine ICANS zeigten.
Das klinische Studiennetzwerk des Unternehmens hat sich auf 50 Standorte in den USA und Europa erweitert, wobei bis zum 13. Februar 2025 26 Patienten im RESET-Programm eingeschrieben sind. Nach der Behandlung wurde bei allen Patienten eine tiefe B-Zell-Depletion beobachtet, wobei die B-Zell-Repopulation in der Regel etwa 2 Monate nach der Infusion beginnt.
- Multiple patients achieved disease remission across different indications
- Patients successfully discontinued all immunosuppressants and steroids
- Favorable safety profile with 90% of patients showing minimal or no adverse effects
- Rapid enrollment pace averaging one patient per week since November
- Expanded clinical trial network to 50 sites across U.S. and Europe
- Immune-mediated necrotizing myopathy (IMNM) patients showed slower improvement compared to other conditions
- B cell repopulation occurs relatively quickly at around 2 months post-infusion
Insights
The latest clinical data from Cabaletta Bio's rese-cel program represents a potentially transformative development in autoimmune disease treatment. The achievement of disease remission while discontinuing all immunosuppressants and steroids across multiple indications - including SLE, lupus nephritis, and dermatomyositis - suggests a paradigm-shifting therapeutic approach.
The data's significance lies in several key aspects: First, the ability to achieve complete disease control without chronic immunosuppression addresses a critical unmet need in autoimmune disease management. Current standard-of-care treatments typically require lifelong immunosuppression, carrying significant risks and quality-of-life impact. Second, the consistent efficacy across multiple indications suggests a platform potential that could dramatically expand the therapeutic and commercial opportunity.
The safety profile is particularly noteworthy, with 90% of patients experiencing either no or only mild cytokine release syndrome, and 90% showing no neurotoxicity (ICANS). This favorable safety profile could position rese-cel as a first-line cellular therapy option for autoimmune diseases, rather than being relegated to last-line treatment.
The robust enrollment rate of approximately one patient per week since November, coupled with 50 active clinical sites across the US and Europe, indicates strong execution capability and suggests significant investigator interest. This momentum could accelerate the path to potential regulatory submissions.
The confirmed B cell depletion in both peripheral blood and tissue (demonstrated by lymph node biopsy) provides mechanistic validation and suggests potential durability of response. The emergence of transitional naïve B cells upon repopulation may indicate restoration of immune tolerance, a key goal in autoimmune disease treatment.
– Clinical efficacy continued to deepen over time with three SLE patients in DORIS remission, the first LN patient achieving complete renal response, and the first dermatomyositis patient maintaining a major TIS improvement; each of these patients discontinued all immunosuppressants and are off steroids as of the latest follow-up –
– Safety profile continues to suggest favorable risk-benefit in the first 10 patients dosed;
– Deep B cell depletion observed in all patients after rese-cel infusion with a transitional naïve B cell phenotype upon repopulation; tissue-resident B cell elimination confirmed by a lymph node biopsy in a scleroderma patient –
– 50 clinical sites in the U.S. and Europe actively recruiting with 26 patients enrolled across the RESET™ clinical development program as of February 13, 2025 –
PHILADELPHIA, Feb. 18, 2025 (GLOBE NEWSWIRE) -- Cabaletta Bio, Inc. (Nasdaq: CABA), a clinical-stage biotechnology company focused on developing and launching the first curative targeted cell therapies designed specifically for patients with autoimmune diseases, today announced new and updated clinical data from the first 10 patients dosed with resecabtagene autoleucel (rese-cel, formerly referred to as CABA-201) across the RESET clinical development program. These data were presented by Aimee Payne, M.D., Ph.D., Co-founder of and Scientific Advisory Board Co-chair at Cabaletta Bio in the ‘Science Breakthroughs’ session at the 2025 annual meeting of the American Association for the Advancement of Science, which was held in Boston, MA from February 13-15, 2025, and are being presented by Samik Basu, M.D., Chief Scientific Officer at Cabaletta Bio at the 5th International Conference on Lymphocyte Engineering, which is being held in Munich, Germany from February 20-22, 2025.
“The expanding clinical experience with rese-cel underscores its potential to provide compelling clinical responses without the need for immunosuppressants or steroids in patients with active, refractory autoimmune disease. With patients across the ongoing myositis, lupus and systemic sclerosis trials achieving DORIS remission in SLE, complete renal response in LN, and major TIS improvement in dermatomyositis, all while off all immunosuppressants and steroids, we believe rese-cel has the potential to transform the lives of patients with autoimmune disease,” said David J. Chang, M.D., Chief Medical Officer of Cabaletta. “We intend to include these data when we meet with the FDA to align on registrational trial designs in the first half of 2025. We believe our expanding footprint of clinical sites in the US and Europe has facilitated our ability to accelerate the pace of enrollment and dosing across the RESET program. With an average of one patient enrolling per week since November, we anticipate that we will generate sufficient data to further clarify rese-cel’s profile across multiple indications this year to rapidly deliver its therapeutic potential for autoimmune patients.”
Cabaletta is currently evaluating rese-cel in the RESET (REstoring SElf-Tolerance) clinical development program, which includes six company-sponsored Phase 1/2 clinical trials with disease-specific cohorts, spanning the therapeutic areas of rheumatology, neurology and dermatology. All cohorts are evaluating a weight-based single infusion of rese-cel following a preconditioning regimen of fludarabine and cyclophosphamide, except for the RESET-PV™ trial, which is evaluating weight-based dosing of rese-cel without preconditioning.
New and Updated Clinical Data Summary
As of the data cut-off date of January 8, 2025, 10 patients had been dosed with rese-cel across the RESET-Myositis™, RESET-SLE™ and RESET-SSc™ trials with sufficient follow-up to be evaluable, providing the following key insights:
- In the RESET-Myositis trial, the first adult dermatomyositis patient maintained a major total improvement score (TIS) improvement at 3 months post-infusion, off all immunosuppressants and steroids, showing potential for achieving drug-free remission in patients with refractory myositis. In addition, initial clinical responses in the first 2 immune-mediated necrotizing myopathy (IMNM) patients continued to show more gradual improvement, consistent with published academic data, suggesting response kinetics may differ among myositis subtypes.
- In the RESET-SLE trial, 3 out of 4 patients in the non-renal systemic lupus erythematosus (SLE) cohort achieved DORIS (definition of remission in SLE) remission as of the most recent follow-up visit. The first patient dosed with rese-cel in the lupus nephritis (LN) cohort achieved a complete renal response (CRR). All 6 SLE and LN patients dosed, including these patients, demonstrated clinical responses off all immunosuppressants and steroids as of the data cut-off date.
- In the RESET-SSc trial, the first patient dosed with rese-cel in the severe skin cohort continued to demonstrate clinically meaningful skin improvements across an increasing number of body areas at 3 months post-infusion, in addition to improvement in lung function, after discontinuing all disease-specific therapies.
- Rese-cel consistently demonstrated deep depletion of B cells in the periphery within the first month of infusion. Tissue resident depletion consistent with the deep B cell depletion in circulation was confirmed by a lymph node biopsy in a systemic sclerosis patient. B cell repopulation has typically started around 2 months post-infusion and exhibited a transitional naïve phenotype, reflecting the production of new B cells after deep systemic depletion.
- Across the first 10 patients dosed with rese-cel with at least one month of follow-up,
90% experienced either no cytokine release syndrome (CRS) or grade 1 CRS (fever) and90% experienced no immune effector cell-associated neurotoxicity syndrome.
Additional information can be accessed on the website of each scientific meeting. Presentation materials will be made available on the Posters & Publications section of the Company’s website following each event.
About rese-cel (formerly referred to as CABA-201)
Rese-cel is a 4-1BB-containing fully human CD19-CAR T cell investigational therapy for patients with autoimmune diseases where B cells contribute to the initiation and/or maintenance of disease. Following a one-time infusion of a weight-based dose, rese-cel is designed to transiently and deeply deplete all CD19-positive cells in both the peripheral circulation and within tissues. This approach has the potential to reset the immune system and result in profound clinical responses without chronic therapy requirements in patients. Cabaletta is currently evaluating rese-cel in the RESET™ (REstoring SElf-Tolerance) clinical development program which includes multiple disease-specific, company-sponsored clinical trials across expanding portfolios of autoimmune diseases in a broad range of therapeutic areas, including rheumatology, neurology and dermatology.
About Cabaletta Bio
Cabaletta Bio (Nasdaq: CABA) is a clinical-stage biotechnology company focused on developing and launching the first curative targeted cell therapies designed specifically for patients with autoimmune diseases. The CABA™ platform encompasses two complementary strategies which aim to advance the discovery and development of engineered T cell therapies with the potential to become deep and durable, perhaps curative, treatments for a broad range of autoimmune diseases. The lead CARTA (Chimeric Antigen Receptor T cells for Autoimmunity) strategy is prioritizing the development of rese-cel, a 4-1BB-containing fully human CD19-CAR T cell investigational therapy. Rese-cel is currently being evaluated with a single weight-based dosing regimen across the RESET™ (REstoring SElf-Tolerance) clinical development program spanning multiple therapeutic areas, including rheumatology, neurology and dermatology. Cabaletta Bio’s headquarters and labs are located in Philadelphia, PA. For more information, please visit www.cabalettabio.com and connect with us on LinkedIn.
Forward-Looking Statements
This press release contains “forward-looking statements” of Cabaletta Bio within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including without limitation, express or implied statements regarding: Cabaletta’s business plans and objectives as a whole; Cabaletta’s ability to realize its vision of launching the first curative targeted cell therapy designed specifically for patients with autoimmune diseases; Cabaletta’s ability to successfully complete research and further development and commercialization of its drug candidates in current or future indications, including the timing and results of Cabaletta’s clinical trials and its ability to conduct and complete clinical trials; expectation that clinical results will support rese-cel’s safety and activity profile; statements regarding the timing of interactions with regulatory authorities, including such authorities’ review of safety information from Cabaletta’s ongoing clinical trials and potential registrational pathway for rese-cel; Cabaletta’s expectations around the potential success and therapeutic benefits of rese-cel, including its belief that rese-cel has the potential to reset the immune system and result in profound clinical responses without chronic therapy requirements in patients; the Company’s advancement of separate Phase 1/2 clinical trials of rese-cel in patients with SLE, myositis, SSc and gMG and advancement of the RESET-PV and RESET-MS trials, including updates related to status, safety data, efficiency of clinical trial design and timing of data read-outs or otherwise; the clinical significance of the clinical data read-out at upcoming scientific meetings; Cabaletta’s belief that its expanding clinical experience with rese-cel underscores its potential to provide compelling clinical responses without the need for immunosuppressants or steroids in patients with active, refractory autoimmune disease, as well as its belief that rese-cel has the potential to transform the disease outcome and the lives of patients with autoimmune disease; and Cabaletta’s belief that its growing number of sites will allow it to continue accelerating the pace of enrollment and dosing across the RESET program, further enabling it to evaluate the emerging clinical profile of rese-cel and its therapeutic potential for autoimmune patients.
Any forward-looking statements in this press release are based on management’s current expectations and beliefs of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to regulatory filings and potential clearance; the risk that signs of biologic activity or persistence may not inform long-term results; Cabaletta’s ability to demonstrate sufficient evidence of safety, efficacy and tolerability in its preclinical studies and clinical trials of rese-cel; the risk that the results observed with the similarly-designed construct employed in academic publications, including due to the dosing regimen, are not indicative of the results we seek to achieve with rese-cel; risks that modifications to trial design or approach may not have the intended benefits and that the trial design may need to be further modified; risks related to clinical trial site activation, delays in enrollment generally or enrollment rates that are lower than expected; delays related to assessment of clinical trial results; risks related to unexpected safety or efficacy data observed during clinical studies; risks related to volatile market and economic conditions and public health crises; Cabaletta’s ability to retain and recognize the intended incentives conferred by Orphan Drug Designation and Fast Track Designation or other designations for its product candidates, as applicable; risks related to Cabaletta’s ability to protect and maintain its intellectual property position; risks related to fostering and maintaining successful relationships with Cabaletta’s collaboration and manufacturing partners; uncertainties related to the initiation and conduct of studies and other development requirements for its product candidates; the risk that any one or more of Cabaletta’s product candidates will not be successfully developed and/or commercialized; and the risk that the initial or interim results of preclinical studies or clinical studies will not be predictive of future results in connection with future studies. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Cabaletta’s actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in Cabaletta’s most recent annual report on Form 10-K as well as discussions of potential risks, uncertainties, and other important factors in Cabaletta’s other subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Cabaletta undertakes no duty to update this information unless required by law.
Contacts:
Anup Marda
Chief Financial Officer
investors@cabalettabio.com
FAQ
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