Cabaletta Bio (CABA) Jefferies Global Healthcare Conference 2026 summary

Key data highlights and clinical progress

• Over 50 patients treated with rese-cel across multiple autoimmune diseases, showing strong efficacy and safety data presented at EULAR.

• In myositis, over 80% of phase I/II patients met the primary endpoint, with 100% durability up to 1.5 years and no need for additional myositis drugs.

• Juvenile Dermatomyositis cohort initiated, with the first pediatric patient showing a durable response similar to adults.

• Scleroderma patients demonstrated improvement in lung function (FVC) and skin, with a majority achieving the ACR-CRISS endpoint and a favorable safety profile.

• Early lupus and pemphigus data with preconditioning-free regimens show promising immune reset at low doses, with plans to escalate dosing.

Durability, safety, and payer perspectives

• All responders in myositis maintained their response for up to 1.5 years, exceeding the 52-week durability threshold required by payers and physicians.

• Rese-cel shows an unprecedented safety profile for CAR T, with 94% of patients experiencing no CRS and no ICANS in myositis; scleroderma cases had only isolated, manageable events.

• Transient B cell depletion (6–12 weeks) allows patients to avoid chronic immunosuppression and return to a healthy state.

• Outpatient administration is feasible due to low toxicity, reducing costs and expanding access for providers and patients.

• Payers are focused on durability and off-therapy status, with market research confirming alignment with clinical outcomes.

Commercial strategy and manufacturing

• Commercial preparations are underway, focusing on payer engagement and demonstrating value through durable, one-time therapy.

• Outpatient go-to-market model is enabled by the safety profile, allowing broader provider participation and cost savings.

• Partnership with Cellares enables automated, industrialized manufacturing, reducing cost of goods and capital requirements.

• Cellares-produced rese-cel is comparable to other manufacturing sites, supporting scalability and margin improvement.

• Anticipated BLA filing for dermatomyositis in the second half of 2027, with scleroderma registrational trial starting soon.