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Lexeo Therapeutics Announces Regulatory Update and Registrational Trial Design for LX2006 Gene Therapy in Friedreich Ataxia
SUNRISE-FA 2 study parameters include LVMI primary endpoint, 6-month topline efficacy analysis, inclusion criteria focused on abnormal baseline LVMI and open-label trial design BLA supportive manufacturing strategy includes flexible process validation, including reduced PPQ manufacturing batches SUNRISE-FA 2 initiation on track for Q2 2026, with first patient expected to be enrolled by end of June Company to host webcast today at 8:00 AM ET NEW YORK, June 15, 2026 (GLOBE NEWSWIRE) -- Lexeo Thera
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SUNRISE-FA 2 study parameters include LVMI primary endpoint, 6-month topline efficacy analysis, inclusion criteria focused on abnormal baseline LVMI and open-label trial design BLA supportive manufacturing strategy includes flexible process validation, including reduced PPQ manufacturing batches SUNRISE-FA 2 initiation on track for Q2 2026, with first patient expected to be enrolled by end of June Company to host webcast today at 8:00 AM ET NEW YORK, June 15, 2026 (GLOBE NEWSWIRE) -- Lexeo Therapeutics, Inc. (Nasdaq: LXEO), a clinical stage genetic medicine company dedicated to pioneering novel treatments for cardiovascular diseases, today announced that the Company has finalized the SUNRISE-FA 2 pivotal trial protocol and statistical analysis plan (SAP) intended to provide clinical evidence to support the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for gene therapy candidate LX2006 under the accelerated approval pathway in 2028. “We have reached a major milestone with the finalization of the SUNRISE‑FA 2 pivotal study design, establishing a clear and rigorous path to evaluate LX2006 in Friedreich ataxia (FA) cardiomyopathy,” said Narinder Bhalla, M.D., Chief Medical Officer of Lexeo Therapeutics. “Patients living with FA, particularly those with cardiac involvement, have a significant unmet need for new treatment options and remain at the center of our efforts. This progress brings us one step closer to delivering a potential new therapy, and we remain focused on execution as we work to initiate the pivotal study and enroll the first patient by the end of the month.” "FARA congratulates the Lexeo Therapeutics team on this important milestone and is deeply grateful for their commitment to advancing the first gene therapy program for Friedreich ataxia," said Jennifer Farmer, Chief Executive Officer of the Friedreich's Ataxia Research Alliance (FARA). "We also thank the participants and investigators in the SUNRISE-FA Phase I/II study, whose courage paved the way for this pivotal trial. We commend Lexeo for designing SUNRISE-FA 2 with scientific rigor while recognizing that a sham or placebo design is neither necessary nor appropriate in the context of gene therapy and adding a pediatrics arm to this study, putting patients first as we work urgently toward the first approved tre...
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