Cabaletta Bio (CABA) 2024 Cantor Fitzgerald Global Healthcare Conference summary

Program overview and clinical progress

• Lead asset CABA-201, a CD19-CAR T therapy, is in phase I/II trials for four autoimmune indications using a consistent starting dose modeled after Professor Schett's work.

• 28 clinical sites are open across the U.S. for lupus, myositis, myasthenia gravis, and scleroderma, with rapid site expansion and enrollment pace.

• Enrollment accelerated from five patients in eight months to four more in the next seven weeks, with a current pace of about one patient every two weeks.

• Safety events included a grade 4 ICANS case, managed successfully with no sequelae, leading to the addition of seizure prophylaxis and a two-week wait post-cardiac events.

• FDA and DSMB supported continued enrollment and protocol with minor safety adjustments; no dose changes were required.

Strategic site expansion and industry positioning

• Early and aggressive site recruitment has positioned the program as a leader in U.S. clinical site count for autoimmune CAR T trials.

• Over 100 U.S. sites are being engaged, with global expansion plans underway to support large-scale enrollment.

• Clinical site access is seen as a critical differentiator, with competitors struggling to open sites at similar scale.

• Trials are designed for flexibility, allowing continued enrollment beyond initial cohorts while engaging with FDA for potential registrational designs.

• Other companies are approaching to leverage established site networks for faster, cost-effective development.

Data, safety, and regulatory outlook

• All trials use the same dose, with FDA permission to escalate if needed; safety and efficacy data are being collected across nine disease subtypes.

• The program aims to demonstrate drug-free, disease-free outcomes with fewer patients than traditional standards, focusing on clear statistical efficacy.

• A new sub-study is underway to test CABA-201 without preconditioning, potentially changing the value proposition for allogeneic competitors.

• Discussions with FDA for registrational cohorts are ongoing, with lupus and IMNM myositis as likely first registration indications.

• Major data updates are anticipated at upcoming conferences, with ACR in November as a likely venue.