“ No CRS, ICANS, infections or severe hostile occasions noticed in both of the primary two sufferers by way of information cut-off of Could 28, 2024 “
“ CABA-201 exhibited anticipated profile of CAR T cell growth and contraction with full B cell depletion noticed in each sufferers by day 15 post-infusion “
“ Enhancements in each sufferers’ particular illness measures, in step with educational expertise of the same 4-1BB CD19-CAR T, recommend rising scientific profit with CABA-201 whereas discontinuing all disease-specific therapies apart from a deliberate steroid taper in a single affected person “
“ Immature, naïve B cell repopulation in first IMNM affected person noticed at week 8 in step with a possible immune system reset “
“ 18 websites open and recruiting throughout 4 Part 1/2 RESET™ trials with 5 sufferers enrolled as of June 12, 2024; preliminary scientific and translational information help continued improvement of CABA-201 on the present dose “
“ Firm to host reside investor convention name and webcast right now at 8:00 a.m. ET “
PHILADELPHIA, June 14, 2024 (GLOBE NEWSWIRE) — Cabaletta Bio, Inc. (Nasdaq: NASDAQ:), a clinical-stage biotechnology firm centered on creating and launching the primary healing focused cell therapies designed particularly for sufferers with autoimmune illnesses, right now reported constructive preliminary scientific information from every of the primary two sufferers dosed with CABA-201 within the Part 1/2 RESET-Myositis and RESET-SLE trials. These information might be introduced right now at 8:15 a.m. CEST (2:15 a.m. ET) at a EULAR European Congress of Rheumatology 2024 Trade Symposia session titled Immune Reset: The Potential of CAR T Cell Remedy to Rework the Remedy of Sufferers with Autoimmune Illness in Vienna, Austria. Slides from the presentation might be discovered on the corporate’s web site right here.
We’re inspired by the preliminary security, scientific and translational information from the RESET-Myositis and RESET-SLE trials which we imagine present essential early validation relating to the potential of the chosen scientific dose of CABA-201 to allow an immune system reset for sufferers with autoimmune illnesses. By demonstrating a doubtlessly well-tolerated security profile together with preliminary scientific and translational information in step with the educational expertise of the same 4-1BB CD19-CAR T assemble, we imagine CABA-201 could also be uniquely positioned to satisfy unmet affected person wants throughout a broad vary of autoimmune illnesses, stated David J. Chang, M.D., Chief Medical Officer of Cabaletta. With the RESET-SSc™ and RESET-MG™ trials just lately opening for enrollment, a further cohort evaluating sufferers with juvenile myositis included into the RESET-Myositis trial and the momentum offered by the promising early scientific information, we’re trying ahead to accelerating scientific trial enrollment within the RESET scientific program. We proceed to count on to report preliminary scientific information from the Part 1/2 RESET-SSc and RESET-MG trials in addition to further information from the RESET-Myositis and RESET-SLE trials within the second half of this 12 months.
Cabaletta designed CABA-201, a 4-1BB-containing totally human CD19-CAR T cell investigational remedy, to deeply and transiently deplete CD19-positive B cells following a one-time infusion which will allow a reset of the immune system with the potential for sturdy remission with out power remedy in sufferers with autoimmune illnesses. Cabaletta is advancing 4 Part 1/2 RESET trials evaluating CABA-201 inside a complete of ten cohorts with six sufferers in every cohort. All cohorts are evaluating the identical single, weight-based dose of 1 x 106 cells/kg, following a preconditioning routine of fludarabine and cyclophosphamide in step with the dosing routine used within the educational expertise, with no dose escalation requirement.
As of Could 28, 2024, the information closing date, one affected person handled within the immune-mediated necrotizing myopathy (IMNM) cohort within the RESET-Myositis trial had accomplished three months of follow-up and one affected person enrolled within the systemic lupus erythematosus (SLE) non-renal cohort within the RESET-SLE trial had accomplished one month of follow-up. The affected person with IMNM is a 33-year-old male with a two-year historical past of illness, constructive for anti-SRP antibody and who had prior disease-specific remedy that included IVIg, rituximab, methotrexate and glucocorticoids. The affected person with SLE is a 26-year-old male with a six-year historical past of illness, constructive for anti-dsDNA antibody and who had prior illness particular remedy that included cyclophosphamide, voclosporin, belimumab, tacrolimus, mycophenolate mofetil, hydroxychloroquine and glucocorticoids. Each sufferers have been administered a one-time infusion of CABA-201 at 1 x 106 cells/kg, following a preconditioning routine of fludarabine and cyclophosphamide. The first endpoint of every trial is security and tolerability inside 28 days of infusion. Secondary endpoints embody translational assessments and scientific outcomes.
Preliminary Medical Knowledge Abstract
Security and Tolerability
- CABA-201 was administered throughout a four-day hospital keep, as presently required by the protocol, and was usually well-tolerated with no severe hostile occasions reported for both affected person by way of the follow-up interval.
- No proof of cytokine launch syndrome (CRS) or immune effector cell-associated neurotoxicity syndrome (ICANS) of any grade was noticed for both affected person by way of the follow-up interval. Tocilizumab was not administered for both affected person.
- No infections have been noticed for both affected person by way of the follow-up interval.
- All power upkeep remedy or concomitant medicines have been discontinued for each sufferers by way of the follow-up interval, apart from a deliberate prednisone taper for the SLE affected person.
Medical and Translational Profile
- Full B cell depletion was noticed inside 15 days post-infusion with CABA-201 in each sufferers. Each sufferers had early, transient leukopenia, as anticipated with the preconditioning routine.
- CAR T cell growth related to CABA-201 reached its peak magnitude at day 15 post-infusion in each sufferers and the magnitude of growth was in step with the educational expertise with the same 4-1BB CD19-CAR T assemble.
- At week 12 of follow-up for the IMNM affected person, the information present a decline in creatinine kinase from 617 at infusion to 308 and a complete enchancment rating (TIS) of 30, which is in step with the clinically significant enchancment seen within the educational expertise of the same 4-1BB CD19-CAR T assemble that additionally just lately reported information from an IMNM affected person.
- At week 4 of follow-up for the SLE affected person, the information demonstrated an enchancment within the SLEDAI-2K (systemic lupus erythematosus illness exercise index) rating from 26 at baseline to 10.
- B cell repopulation was noticed within the IMNM affected person at week 8 with immature, naïve B cell phenotypes as demonstrated by movement cytometry, suggesting potential immune system reset with confirmatory analyses ongoing.
Investor Convention Name and Webcast Data
Cabaletta will host a convention name and webcast right now, June 14, 2024, at 8:00 a.m. ET to evaluation the preliminary scientific information introduced on the satellite tv for pc symposium on the EULAR 2024 Congress and supply an replace on the RESET scientific improvement program. A webcast of the reside name might be accessed on the Information and Occasions part of the Firm’s web site at www.cabalettabio.com. An archived replay might be obtainable on the Firm’s web site.
In regards to the RESET-Myositis™ Trial
The RESET-Myositis™ trial is a Part 1/2 open-label research of CABA-201 in topics with lively idiopathic inflammatory myopathy (IIM, or myositis), together with the subtypes of dermatomyositis (DM), anti-synthetase syndrome (ASyS), immune-mediated necrotizing myopathy (IMNM) and juvenile myositis (JM), every evaluated in particular person cohorts. Topics will obtain a one-time infusion of CABA-201 at a dose of 1 x 106 cells/kg, following a preconditioning routine of fludarabine and cyclophosphamide. Key inclusion standards for the DM, ASyS and IMNM cohorts embody sufferers between ages 18 to 75 (inclusive), proof of lively illness and illness exercise regardless of prior or present remedy with customary of care remedies. Key exclusion standards for the DM, ASyS and IMNM cohorts embody cancer-associated myositis, vital lung or cardiac impairment, remedy with a B cell depleting agent throughout the prior roughly six months or remedy with a biologic agent throughout the prior roughly three months.
In regards to the RESET-SLE™ Trial
The RESET-SLE™ trial is a Part 1/2 open-label research of CABA-201 in topics with systemic lupus erythematosus (SLE) and lupus nephritis (LN), every evaluated in particular person cohorts. Topics will obtain a one-time infusion of CABA-201 at a dose of 1 x 106 cells/kg, following a preconditioning routine of fludarabine and cyclophosphamide. Key inclusion standards embody sufferers between ages 18 to 65 (inclusive), proof of lively illness and illness exercise regardless of prior or present remedy with customary of care remedies. Key exclusion standards embody remedy with a B cell depleting agent throughout the prior roughly six months or remedy with a biologic agent throughout the prior roughly three months.
About CABA-201
CABA-201 is designed to deeply and transiently deplete CD19-positive cells following a one-time infusion, which can allow an immune system reset with the potential for sturdy remission with out power remedy in sufferers with autoimmune illnesses. Cabaletta is evaluating CABA-201 in a number of autoimmune circumstances inside 5 disease-specific firm sponsored INDs together with myositis (idiopathic inflammatory myopathy, or IIM), systemic lupus erythematosus (SLE), systemic sclerosis (SSc), generalized myasthenia gravis (gMG) and pemphigus vulgaris (PV; a sub-study to judge CABA-201 with out preconditioning).
About Cabaletta Bio
Cabaletta Bio (Nasdaq: CABA) is a clinical-stage biotechnology firm centered on the invention and improvement of engineered T cell therapies which have the potential to offer a deep and sturdy, maybe healing, remedy for sufferers with autoimmune illnesses. The CABA™ platform encompasses two methods: the CARTA (chimeric antigen receptor T cells for autoimmunity) technique, with CABA-201, a 4-1BB-containing totally human CD19-CAR T, because the lead product candidate being evaluated within the RESET™ (REstoring SElf-Tolerance) scientific trials in systemic lupus erythematosus, myositis, systemic sclerosis and generalized myasthenia gravis and within the RESET-PV™ sub-study throughout the DesCAARTes™ scientific trial in pemphigus vulgaris, together with the CAART (chimeric autoantibody receptor T cells) technique, with a number of clinical-stage candidates, together with DSG3-CAART for mucosal pemphigus vulgaris and MuSK-CAART for MuSK-associated myasthenia gravis. The increasing CABA™ platform is designed to develop doubtlessly healing therapies that supply deep and sturdy responses for sufferers with a broad vary of autoimmune illnesses. Cabaletta Bio’s headquarters and labs are situated in Philadelphia, PA.
Ahead-Wanting Statements
This press launch incorporates forward-looking statements of Cabaletta Bio throughout the which means of the Non-public Securities Litigation Reform Act of 1995, as amended, together with with out limitation, categorical or implied statements relating to: Cabaletta’s means to develop its autoimmune pipeline; Cabaletta’s future plans and techniques for its CAAR T and CARTA applied sciences and the corporate’s enterprise plans and goals as a complete; Cabaletta’s expectations across the potential security and therapeutic advantages of CABA-201, together with its perception that CABA-201 might allow an immune system reset with the potential for sturdy remission with out power remedy in sufferers with autoimmune illnesses; the Firm’s development of separate Part 1/2 scientific trials of CABA-201 in sufferers with SLE, myositis, SSc and gMG and development of a RESET-PV sub-study throughout the ongoing DesCAARTes trial in PV, together with the Firm’s expectations for the effectivity of the trial designs and updates associated to standing, security information, or in any other case and the anticipated timing of the associated information read-outs; Cabaletta’s means to speed up its pipeline, develop significant therapies for sufferers and leverage its analysis and translational insights; the scientific significance of the preliminary scientific information read-out on the EULAR 2024 Congress in June 2024 for sufferers with myositis and SLE handled with CABA-201; Cabaletta’s further deliberate preliminary scientific information read-outs for sufferers with SSc and gMG handled with CABA-201 or in any other case; Cabaletta’s development of the method to activate scientific trial websites and pursue affected person enrollment; and Cabaletta’s deliberate evaluation of its DesCAARTes™ and MusCAARTes™ trials.
Any forward-looking statements on this press launch are primarily based on administration’s present expectations and beliefs of future occasions and are topic to quite a few dangers and uncertainties that would trigger precise outcomes to vary materially and adversely from these set forth in or implied by such forward-looking statements. These dangers and uncertainties embody, however aren’t restricted to: dangers associated to regulatory filings and potential clearance; the danger that indicators of biologic exercise or persistence might not inform long-term outcomes; Cabaletta’s means to reveal adequate proof of security, efficacy and tolerability in its preclinical research and scientific trials of CABA-201; the danger that the outcomes noticed with the similarly-designed assemble employed in educational publications, together with because of the dosing routine, aren’t indicative of the outcomes we search to realize with CABA-201; dangers associated to scientific trial web site activation, delays in enrollment usually or enrollment charges which are decrease than anticipated; delays associated to evaluation of scientific trial outcomes; dangers associated to sudden security or efficacy information noticed throughout scientific research; dangers associated to unstable market and financial circumstances and public well being crises; Cabaletta’s means to retain and acknowledge the meant incentives conferred by Orphan Drug Designation and Quick Observe Designation or different designations for its product candidates, as relevant; dangers associated to Cabaletta’s means to guard and keep its mental property place; dangers associated to fostering and sustaining profitable relationships with Cabaletta’s collaboration and manufacturing companions, together with in gentle of latest laws; uncertainties associated to the initiation and conduct of research and different improvement necessities for its product candidates; the danger that anybody or extra of Cabaletta’s product candidates is not going to be efficiently developed and/or commercialized; and the danger that the preliminary or interim outcomes of preclinical research or scientific research is not going to be predictive of future ends in reference to future research. For a dialogue of those and different dangers and uncertainties, and different essential components, any of which might trigger Cabaletta’s precise outcomes to vary from these contained within the forward-looking statements, see the part entitled Threat Elements in Cabaletta’s most up-to-date annual report on Type 10-Okay in addition to discussions of potential dangers, uncertainties, and different essential components in Cabaletta’s different filings with the Securities and Alternate Fee. All info on this press launch is as of the date of the discharge, and Cabaletta undertakes no obligation to replace this info except required by legislation.
Contacts:
Anup Marda
Chief Monetary Officer
traders@cabalettabio.com
William Gramig
Precision AQ
william.gramig@precisionaq.com
