Cabaletta Bio (CABA) investor relations material

Key clinical insights and program updates

• Rese-cel, an autologous CAR T-cell therapy, is designed specifically for autoimmune diseases, using a nine-day manufacturing process and weight-based dosing, showing strong efficacy and safety in nearly 100 patients dosed so far.

• Over 80-85% of patients achieve major or complete responses, often coming off all medications after a single dose, with durable effects observed up to two years and evidence of immune system "reset."

• No cases of IEC-HS have been observed in over 90 patients, with 94% experiencing no or only grade 1 CRS and 97% with no ICANS, differentiating rese-cel from other CD19 CAR T therapies that have reported serious safety events.

• The company plans to initiate a pivotal program in scleroderma within 12 months, alongside ongoing pivotal studies in myositis, with data readouts expected in mid-next year and a BLA filing by year-end.

• Early data in preconditioning-free settings show promise, with ongoing dose exploration to optimize efficacy and safety, particularly for populations where chemotherapy is less desirable.

Differentiation and competitive landscape

• Rese-cel’s nine-day manufacturing process is believed to contribute to its favorable safety profile, contrasting with competitors using shorter processes that may increase pro-inflammatory cytokine release and adverse events.

• Recent safety concerns have led major competitors to pause their autoimmune programs, highlighting the importance of manufacturing choices and patient-specific immune responses.

• The therapy’s ability to be administered in outpatient settings and its potential for use without preconditioning could further distinguish it in the market.

• Market research indicates strong interest from insurers in a one-time, curative therapy that could disrupt the current additive cost model of chronic autoimmune treatments.

• The company is cautious in dose escalation without preconditioning, recognizing the unique risks in autoimmune versus oncology settings.

Manufacturing and commercialization strategy

• Multiple manufacturing partners, including Lonza and ElevateBio, are in place to ensure supply and scalability for launch, with Cellares’ automated platform expected to add flexibility and redundancy post-launch.

• Cellares-manufactured product has shown comparability to traditional processes, and regulatory acceptance has been achieved for this manufacturing approach.

• Commercial prospects are strong, with the potential for a priority review voucher in juvenile myositis and urgent unmet needs in scleroderma and pediatric populations.

• The therapy’s cost model and clinical profile may shift payer and provider preferences, especially as data matures and durability is confirmed beyond two years.

• Ongoing innovation in manufacturing and clinical development aims to maintain differentiation and address evolving market and patient needs.