Cabaletta Bio (CABA) investor relations material

Clinical data and program updates

• Rese-cel demonstrated durable, immunomodulator-free responses in over 50 patients, with 80% of dermatomyositis and most anti-synthetase syndrome patients achieving remission at week 16; durability in dermatomyositis extends to 1.5 years so far.

• Safety profile is highly favorable, with minimal ICANS and CRS, enabling outpatient dosing and rapid adoption in clinical practice.

• Dose-ranging and preconditioning-free regimens are being explored in lupus and pemphigus, with early data showing promising immune reset and safety, especially for women of childbearing age.

• Systemic sclerosis program showed increasing response magnitude over time, with a 7.5% improvement at week 36 and plans for a 25-patient registrational study in Q4.

• Outpatient infrastructure and rapid uptake are expected due to the favorable safety profile and existing CAR T experience in oncology.

Competitive landscape and differentiation

• Rese-cel stands out among autologous CAR Ts for its safety and tolerability, with 97% of patients experiencing no ICANS and most CRS limited to mild fever.

• Durability and safety are key differentiators versus in vivo CAR T and bispecifics, with rese-cel patients regaining normal B-cell populations by 116 days.

• The approach allows for patients to discontinue all immunomodulators, contrasting with chronic therapies that require ongoing medication.

• Manufacturing advances are expected to lower cost of goods, supporting broader access and commercial viability.

Regulatory strategy and commercial outlook

• The pivotal myositis study is single-arm, weight-based, and includes both adult and juvenile dermatomyositis, aiming for BLA submission in 2H 2027 with robust 16- and 52-week data.

• Inclusion of juvenile dermatomyositis may enable a priority review voucher, potentially providing $180–$200 million in non-dilutive capital.

• Frequent FDA interactions have shaped study design, with outpatient dosing and safety data supporting label expansion.

• Recent $150 million financing brings cash reserves to $250 million, supporting operations and pivotal data delivery into 2027.

• Economic impact models are being developed to demonstrate cost savings to payers and providers, leveraging the drug-free, durable response profile.