Summary of Cabaletta Bio FY Conference Call - September 10, 2025 Company Overview - **Company**: Cabaletta Bio (NasdaqGS:CABA) - **Focus**: Development of autologous CAR T therapies for autoimmune diseases, particularly myositis Key Industry Insights - **Competitive Landscape**: The CAR T therapy space for autoimmune diseases is highly competitive, with many companies targeting similar indications, particularly lupus, which has 35 competitors [4][19] - **Market Opportunity**: Myositis is identified as a less crowded market with fewer competitors, providing a strategic advantage for Cabaletta Bio [4][5] Core Product and Data Presentation - **Lead Product**: ResiCel (CABA-201), an autologous CD19 CAR T product for autoimmune patients, with a focus on myositis as the lead indication [3][4] - **Upcoming Data**: Data from phase one and two trials will be presented at multiple medical meetings in October, including ACR and AANEM [8][9] - **Regulatory Agreement**: Cabaletta has an agreement with the FDA to run a trial with 14 patients to access a market of approximately 70,000 myositis patients, with 16,000 to 20,000 likely eligible at launch [6][7] Clinical Development Strategy - **Trial Design**: The trial will include a primary endpoint based on the TIS score, similar to the approval process for IVIG in myositis [5][7] - **Focus on Myasthenia Gravis**: Initial clinical data for myasthenia gravis will also be presented, highlighting an unmet need in this area [8][9] - **No Preconditioning Approach**: A trial will assess the efficacy of ResiCel without preconditioning, which could simplify the treatment process and expand market access [10][11][20] Market Dynamics and Pricing Strategy - **Pricing Considerations**: The company is evaluating how to price ResiCel in relation to existing therapies like IVIG and Vivgart, which are expensive and not curative [30][31] - **Value Proposition**: The potential for ResiCel to provide significant patient benefits (85% off all medications) positions it favorably against existing therapies [15][31] Enrollment and Patient Demand - **Enrollment Progress**: The company is on track to meet its enrollment milestones, with a focus on ensuring the right patients are included in trials [38][44] - **Patient Interest**: There is strong demand from both patients and physicians for the treatment, with many wanting to enroll additional patients after seeing positive outcomes [44][46] Competitive Threats and Differentiation - **Manufacturing Speed**: Competitors are exploring faster manufacturing processes, but efficacy and safety remain the top priorities for physicians [49][55] - **Focus on Efficacy and Safety**: Cabaletta emphasizes that the most critical factors for physicians are the depth and durability of response, rather than turnaround time [55][57] Conclusion - **Strategic Positioning**: Cabaletta Bio is well-positioned in the CAR T therapy market for autoimmune diseases, particularly with its focus on myositis and the upcoming data presentations that could validate its approach and product efficacy [34][35]