Lexeo Sets Q2 2026 Start for SUNRISE-FA 2 with 15% LVMI Endpoint
LXEO•Lexeo finalized its SUNRISE-FA 2 pivotal trial protocol for LX2006 gene therapy in Friedreich ataxia cardiomyopathy, with a 6-month primary endpoint of 15% LVMI reduction in 13 treated versus 13 untreated subjects and crossover eligibility. Initiation remains on track for Q2 2026 with first patient expected by end of June.
1. Trial Design and Endpoints
SUNRISE-FA 2 is an open-label pivotal study enrolling 13 participants aged 16 and older receiving a single intravenous dose of LX2006 at 1.2×10^12 vector genomes/kg, alongside 13 untreated controls. The primary endpoint is LVMI measured by cardiac MRI at 6 months, powered to detect a ≥15% effect size, with secondary measures including mFARS, KCCQ, hs-troponin-I and lateral wall thickness. Participants are randomly allocated to eliminate selection bias and untreated controls may crossover after 6 months.
2. Regulatory Timeline
The protocol and statistical analysis plan for SUNRISE-FA 2 are finalized, targeting trial initiation in Q2 2026 and first patient enrollment by end of June. Lexeo aims to submit a Biologics License Application for accelerated approval in 2028, with the FDA agreeing to remove the frataxin protein co-primary endpoint and allowing pediatric cohorts following adult safety data.
3. Manufacturing and Enrollment
Clinical-scale drug product has been manufactured using an optimized Sf9-baculovirus process and is immediately available for dosing. Inclusion criteria require abnormal baseline LVMI defined as ≥2 standard deviations above normal mean, mirroring criteria in the parallel CLARITY-FA natural history study to streamline enrollment.
4. Natural History Support Study
CLARITY-FA enrollment is ongoing to provide external natural history data supporting both accelerated and full approval. Participants in CLARITY-FA share identical inclusion criteria with SUNRISE-FA 2 and may transition into the pivotal trial, enhancing consistency in disease course comparisons.
