PHILADELPHIA, PA — Cabaletta Bio, Inc. (Nasdaq: CABA) recently reported financial results for the third quarter ended September 30, 2023, and provided a business update.
“Inspired by the recent flow of academic clinical publications and industry sponsored case reports of multiple other CD19-CAR T candidates suggesting that a single dose of CD19-CAR T can provide deep and durable responses in patients across an increasing number of autoimmune diseases, our team has continued to expand the breadth of our program in the U.S. with what we believe are the first U.S. IND clearances for a CD19-CAR T product candidate in myositis, systemic sclerosis and generalized myasthenia gravis. With the opening of our initial U.S. clinical site in lupus and now four Phase 1/2 studies incorporating a total of nine cohorts that could enroll in parallel, we believe we are in a position to realize our vision of developing and launching the first curative targeted cellular therapies for patients with autoimmune diseases,” said Steven Nichtberger, M.D., Chief Executive Officer and Co-founder of Cabaletta. “We look forward to reporting initial clinical data on patients treated with CABA-201 in the first half of next year.”
Recent Operational Highlights and Upcoming Anticipated Milestones
Chimeric Antigen Receptor T cells for Autoimmunity (CARTA) Strategy
CABA-201: Autologous, engineered T cells with a chimeric antigen receptor containing a fully human CD19 binder and a 4-1BB co-stimulatory domain as a potential treatment for a broad range of autoimmune diseases where B cells contribute to the initiation and/or maintenance of disease.
- Initial clinical data from Phase 1/2 trials in lupus and/or myositis expected by the first half of 2024: Cabaletta anticipates reporting initial clinical efficacy and tolerability data for patients treated with CABA-201 from the Phase 1/2 trials in lupus and/or myositis in the first half of 2024. The Phase 1/2 trial of CABA-201 in systemic lupus erythematosus (SLE) will consist of two separate parallel cohorts, including six SLE patients with active lupus nephritis (LN) and six patients with active SLE without renal involvement. The Phase 1/2 trial of CABA-201 in myositis will consist of three separate parallel cohorts, including six patients with dermatomyositis (DM), six patients with anti-synthetase syndrome (ASyS) and six patients with immune-mediated necrotizing myopathy (IMNM). The CABA-201 starting dose of 1 x 106 cells/kg is equivalent to the CD19-CAR T dose used in the academic studies in SLE and myositis.
- Clinical development program expanded to include SSc and gMG: In October 2023, Cabaletta announced the Company’s third Investigational New Drug (IND) application for CABA-201 was cleared by the U.S. Food and Drug Administration (FDA) for a Phase 1/2 study in patients with systemic sclerosis (SSc). In November 2023, Cabaletta announced the Company’s fourth IND application for CABA-201 was cleared by the FDA for a Phase 1/2 study in patients with generalized myasthenia gravis (gMG). The Company believes that these IND clearances represent the first in each of these diseases for a CD19-CAR T product candidate in the U.S. Consistent with the previously announced CABA-201 IND application clearances for lupus and myositis, the separate Phase 1/2 studies in patients with SSc and gMG will feature a starting dose of 1 x 106 cells/kg and parallel cohort design.
- WuXi ATU selected as a GMP manufacturing partner and Oxford Biomedica as a lentiviral vector supplier for CABA-201 clinical trials: In August 2023, Cabaletta announced the Company entered into additional work orders under the master services agreement with WuXi Advanced Therapies (WuXi ATU), a global Contract Testing, Development and Manufacturing Organization (CTDMO), to include Good Manufacturing Practice (GMP) manufacturing for CABA-201. Through the work orders, WuXi ATU will serve as a cell processing manufacturing partner, in addition to the University of Pennsylvania, for the planned global clinical development of CABA-201 in multiple indications, including potential late-stage clinical trials and commercial readiness activities for CABA-201. In August 2023, Cabaletta also entered into an amendment to its licensing and supply agreement and vector supply agreement with Oxford Biomedica (UK) Limited (Oxford), a leading gene and cell therapy group and established commercial supplier of lentiviral vector. The vector supply agreement granted Cabaletta a non-exclusive license to Oxford Biomedica’s LentiVector® platform for its application in CABA-201. Cabaletta continues to explore multiple paths to scale cell processing and vector manufacturing production in a rapid and reliable manner for CABA-201.
- Translational data published by Cabaletta scientists in collaboration with Dr. Georg Schett to be presented at ACR Convergence 2023: In September 2023, Cabaletta scientists published “Cytokine and reactivity profiles in SLE patients following anti-CD19 CART therapy” in Molecular Therapy: Methods and Clinical Development, highlighting studies performed on serum samples from the first six SLE patients treated with CD19-CAR T by Dr. Georg Schett. The publication reports that in the three months following CD19-CAR T infusion, cytokine markers of systemic inflammation resolved, SLE-associated antibodies were reduced, and pre-existing humoral immunity was maintained. Data that characterize the serologic factors associated with CD19-CAR T treatment in autoimmune patients will also be presented in a poster presentation at the upcoming American College of Rheumatology (ACR) Convergence 2023.
Chimeric AutoAntibody Receptor T (CAART) cells Strategy
- DSG3-CAART: Cabaletta is evaluating desmoglein 3 chimeric autoantibody receptor T (DSG3-CAART) cells as a potential treatment for patients with mucosal pemphigus vulgaris (mPV). Enrollment in the combination cohort of the DesCAARTes™ trial is ongoing, where patients are pre-treated with intravenous immunoglobulin (IVIg), cyclophosphamide and fludarabine prior to DSG3-CAART infusion, with the aim of improving persistence and activation of DSG3-CAART.
- MuSK-CAART: Cabaletta is evaluating muscle-specific kinase (MuSK) chimeric autoantibody receptor T (MuSK-CAART) cells as a potential treatment for patients with MuSK-associated myasthenia gravis (MG). Enrollment in the Phase 1, open-label MusCAARTes™ study of MuSK-CAART in patients with MuSK autoantibody-positive MG is ongoing.
Upcoming Events
Cabaletta plans to participate in the following upcoming scientific conference:
- ACR Convergence 2023, which is being held at the San Diego Convention Center in San Diego, CA from November 10-15, 2023. Cabaletta will present new preclinical data for CABA-201 in a poster presentation and Cabaletta Bio Scientific Advisory Board members Carl June, M.D., and Georg Schett, M.D. will be featured at an Innovation Theater fireside chat presentation titled “Pioneering CAR T Cell Therapy in Autoimmune Diseases” on Tuesday, November 14, 2023, at 12:30 p.m. PT.
Cabaletta plans to participate in the following upcoming investor conferences:
- Stifel 2023 Healthcare Conference, which is being held from November 14-15, 2023 in New York, NY.
- 6th Annual Evercore ISI HealthCONx Conference, which is being held from November 28-30, 2023 in Miami, FL.
Third Quarter 2023 Financial Results
- Research and development expenses were $13.8 million for the three months ended September 30, 2023, compared to $8.2 million for the same period in 2022.
- General and administrative expenses were $4.9 million for the three months ended September 30, 2023, compared to $3.6 million for the same period in 2022.
- As of September 30, 2023, Cabaletta had cash, cash equivalents and short-term investments of $164.4 million, compared to $106.5 million as of December 31, 2022.
The Company expects that its cash, cash equivalents and short-term investments as of September 30, 2023, will enable it to fund its operating plan into the fourth quarter of 2025.
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