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.