Lexeo Therapeutics Advances Cardiac Gene Therapy for Friedreich Ataxia

Lexeo Therapeutics has made significant strides in developing a gene therapy for cardiac dysfunction in Friedreich ataxia (FA), with updated clinical data supporting plans for a registrational trial. The biotech company has outlined its strategy for a pivotal study set to commence in early 2026, bolstered by encouraging results from its LX2006 gene therapy candidate.

Updated Clinical Data Shows Promise

Recent data from a pooled analysis of two studies involving LX2006 have demonstrated improvements in key cardiac measures. Five out of six patients with abnormal left ventricular mass index (LVMI) at baseline experienced a 10% or greater improvement within 12 months of treatment. This marks an increase from the previously reported three out of four patients showing improvement.

The gene therapy, designed to increase expression of the heart muscle protein frataxin, also showed dose-dependent increases in frataxin levels. Notably, patients in the third dose cohort exhibited a substantial 115% jump in frataxin expression.

FDA Alignment and Trial Design

Lexeo Therapeutics has secured agreement from the FDA on using frataxin expression and LVMI as co-primary endpoints in the planned registrational trial. CEO Nolan Townsend emphasized the continuity in their interactions with the FDA, stating, "The [FDA] team that we've been working with has not changed and they continue to work in the same way."

The company expects to finalize the statistical plan with the FDA this year, setting the stage for the pivotal trial's initiation in early 2026. Townsend projects study enrollment to be completed within 2026, with data anticipated in 2027.

Financial Outlook and Future Plans

With a strong financial position, Lexeo has announced that its current cash reserves are expected to provide a runway into 2027. This timeline aligns with the company's clinical development plans, allowing for the execution of the registrational trial and subsequent data analysis.

As Lexeo Therapeutics advances its cardiac gene therapy program, the pharmaceutical industry watches closely. The potential for LX2006 to address cardiac dysfunction in Friedreich ataxia patients represents a significant step forward in treating this rare genetic disorder, offering hope for improved cardiac outcomes in affected individuals.