Cabaletta Bio (CABA) 2025 Conference Transcript

Summary of Caballetta Bio's Conference Call Company Overview - Company: Caballetta Bio - Event: Jefferies Global Healthcare Conference - CEO: Steven Key Industry Insights - Regulatory Alignment: Caballetta Bio has aligned with the FDA on the registrational trial design for Reza cel, focusing on myositis as a target indication [3][4] - Clinical Endpoint: The primary endpoint for the trial will be the TIS score, a validated clinical endpoint previously used for IVIG approval in dermatomyositis [3][4] - Cohort Design: The trial will include two cohorts, one for ACEs and dermatomyositis subtypes (85% of myositis patients) and another for the necrotizing form (IMNM subtype, 15% of patients) [6][8] Efficacy and Safety Data - Patient Enrollment: The trial will enroll approximately 15 patients per cohort, focusing on those refractory to other therapies [4][10] - Safety Database: A safety database of over 100 patients will be utilized for the filing, with 35 patients having myositis-specific data [10][11] - Weight-Adjusted Dosing: Caballetta Bio is the only company using weight-adjusted dosing, which is believed to enhance safety and efficacy [18][19] Market Dynamics - Competitive Landscape: There are currently 35 cell therapies IND cleared for lupus, indicating a crowded market where payers require specific data for reimbursement [15] - Patient Expectations: Patients desire to eliminate symptoms and stop immunosuppressants, while physicians prioritize organ protection [22][24] Future Milestones - Upcoming Presentations: Caballetta Bio will present data at EULAR, including safety and efficacy results from 18 patients dosed for at least one month [31][36] - Regulatory Alignment: Expected regulatory alignments with the FDA for lupus and scleroderma in the second half of the year [45][46] - Enrollment Projections: Enrollment is exceeding aggressive forecasts, with ongoing updates expected at medical meetings [44][46] Additional Considerations - Unmet Need: There is a profound unmet need in the myositis and scleroderma markets, with significant patient enrollment observed [29][28] - Cost Reduction Potential: Collaborations with Solaris may lead to reduced manufacturing costs and increased scalability [48] Conclusion Caballetta Bio is positioned to make significant strides in the treatment of autoimmune diseases with its innovative approach to CAR T therapies, focusing on safety, efficacy, and regulatory alignment. The upcoming data presentations and regulatory milestones will be critical in shaping the company's future trajectory in the market.