Cabaletta Bio (CABA) investor relations material

Strategic development and clinical progress

• CABA-201 was prioritized to replicate promising academic results in autoimmune diseases, with multiple indications now fully enrolled or completed in phase 1/2 RESET trials as of late 2025.

• Recent data from myositis, scleroderma, lupus, and pemphigus vulgaris trials show strong efficacy, including durable responses and improvements in lung function and skin, with pivotal myositis trial enrollment imminent.

• CAR-T in autoimmunity was a major focus at ACR, marking a tipping point in rheumatology, with broad recognition of its transformative potential.

• Pivotal trials for myositis, scleroderma, and lupus are aligned with FDA, with key data and regulatory milestones expected by end of 2025 and into 2026.

• Outpatient administration is a core strategy, enabled by a favorable safety profile and supported by FDA-reviewed protocols.

Safety, outpatient potential, and manufacturing

• CRS rates are low (about 33%, mostly grade 1), with minimal ICANS, attributed to weight-based dosing and 4-1BB co-stimulatory domain.

• Outpatient dosing is feasible due to the safety profile, with plans to publish supporting data before launch.

• Manufacturing out-of-spec rates are extremely low (about 1 in 60+ patients), attributed to healthier autoimmune patient populations.

• Launch will leverage CDMOs like Lonza, with plans for broad site activation and no anticipated supply constraints.

• Partnership with Cellares enables fully automated, closed-system manufacturing, supporting scalable, cost-efficient production.

Commercialization, competition, and value proposition

• Outpatient therapy offers a fundamentally better financial profile, with most patients covered by commercial insurance rather than Medicare.

• CABA-201 is positioned as a cost-effective alternative to high-cost annual therapies, with potential to keep 85% of patients drug-free after one infusion.

• The company plans a focused commercial launch at 77 sites, leveraging existing outpatient infrastructure and referral networks.

• Competition from bispecifics and in vivo CAR-T is acknowledged, but CAR-T efficacy and service predictability are seen as key differentiators.

• The company is open to partnerships but is confident in its ability to launch and scale independently, with profitability expected from early commercialization.