Lexeo Soars on FDA Fast-Track Talks for Gene Therapy
Regulator signals openness to an accelerated approval pathway for LX2006, a promising treatment for Friedreich's Ataxia cardiomyopathy.
Lexeo Therapeutics (NASDAQ: LXEO) saw its stock gain investor attention following the announcement of significant progress in its discussions with the U.S. Food and Drug Administration (FDA) regarding an accelerated approval pathway for its lead gene therapy candidate, LX2006.
The therapy is being developed to treat cardiomyopathy associated with Friedreich's Ataxia (FA), a rare, debilitating genetic disease that currently has no approved treatments for its cardiac manifestations. The company revealed that the , a move that could significantly reduce the size and duration of the required pivotal study, potentially bringing the treatment to patients sooner.
According to the company, Lexeo has reached an alignment with the FDA on the key components of the registrational development plan. This includes co-primary endpoints focused on left ventricular mass index (LVMI) and the expression of the frataxin protein, which is deficient in FA patients. This regulatory clarity is a critical de-risking milestone for the development program.
The positive regulatory feedback is supported by from the ongoing Phase I/II SUNRISE-FA trial. Data from the study showed that LX2006 was generally well-tolerated and led to sustained improvements in cardiac and neurologic measures. Notably, patients with abnormal LVMI at baseline experienced a mean reduction of 18% to 23%, comfortably exceeding the 10% improvement threshold agreed upon with the FDA.
The potential for a faster route to market is further bolstered by the multiple special designations LX2006 has already received. The therapy has been granted , all of which are intended to expedite the development and review of drugs that address serious unmet medical needs.
Lexeo Therapeutics plans to initiate the pivotal study for LX2006 in the first half of 2026. This latest development provides a much clearer regulatory path forward and marks a significant step in the company's mission to develop a transformative treatment for Friedreich's Ataxia cardiomyopathy.