Lexeo Therapeutics (LXEO) investor relations material

Recent progress and clinical updates

• Advanced gene therapy program for Friedreich's ataxia shows improvement in cardiac hypertrophy and neurologic scales, with effect sizes comparable to approved treatments.

• Pivotal study for Friedreich's ataxia expected to start this quarter, with top-line readout anticipated next year and BLA submission planned.

• PKP2 arrhythmogenic cardiomyopathy program completed phase I/II enrollment, showing up to 65% reduction in non-sustained ventricular tachycardia at nine months.

• Data readout for PKP2 program expected in the second half of the year, with regulatory updates targeted for 2026.

• Preclinical TNNI3 hypertrophic cardiomyopathy program progressing, with novel delivery approaches in collaboration with Johnson & Johnson.

Regulatory and study design insights

• Ongoing alignment with FDA on pivotal study protocol, with recent feedback extending review timelines but not impacting overall development milestones.

• Natural history study underway to facilitate rapid transition to treatment study upon FDA alignment.

• Study design for Friedreich's ataxia centers on LVMI as the primary endpoint, with a 10% effect size agreed upon and nearly 30% achieved in earlier studies.

• Secondary endpoints include biomarkers such as troponin and mFARS, with durability demonstrated in long-term data.

• Confirmatory trial design remains under discussion, with focus on endpoints that correlate with clinical outcomes and potential adaptation for European regulatory requirements.

Manufacturing and safety profile

• Manufacturing process transitioned from HEK to Sf9 cells, with FDA comparability requirements met and commercial-scale batches ready.

• High yields achieved, supporting commercial supply and cost efficiency.

• Favorable safety profile observed in both Friedreich's ataxia and PKP2 programs, with minimal liver enzyme elevations and no serious adverse events.