Lexeo Therapeutics’ investigational gene therapy has shown promise as a treatment for Friedreich’s ataxia (FA) cardiomyopathy, according to new phase 1/2 data shared by the company.
Affecting approximately one in every 40,000 people, FA is a neurodegenerative movement disorder and the most common form of inherited ataxia.
The typical age of disease onset is between ten and 15 years, with initial symptoms including unsteady posture, frequent falling, fatigue and progressive difficulty walking due to an impaired ability to coordinate voluntary movements.
Administered intravenously, Lexeo’s LX2006 is designed to treat FA cardiomyopathy, the most common cause of death in this patient population.
Clinically meaningful improvements in cardiac biomarkers were observed in patients receiving the therapy across the Lexeo SUNRISE-FA phase 1/2 trial and the Weill Cornell Medicine investigator-initiated phase 1A study, with increasing improvements seen over time.
A mean reduction in left ventricular mass index (LVMI) of 11.4% was observed at 12 months among patients with elevated LVMI at baseline, increasing to 18.3% at 18 months.
Left ventricular lateral wall thickness, an early indicator of left ventricular hypertrophy, was reduced by 13.6% and troponin I, a biomarker of myocardial injury, dropped by 53.3% on average in all patients at 12 months.
An increase in levels of the protein which is deficient in FA, frataxin, was also observed in all patients and LX2006 was well tolerated.
Dr Sandi See Tai, chief development officer at Lexeo, said: “Together with the increases in frataxin protein expression observed in SUNRISE-FA cardiac biopsies to date, these results further highlight the potential of LX2006 to positively impact outcomes for people with FA cardiomyopathy.”
LX2006 has already received rare paediatric disease, fast track and orphan drug designations from the US Food and Drug Administration to treat FA cardiomyopathy.
Dr Eric Adler, chief medical officer and head of research at Lexeo, said: “We are very encouraged by this data and the potential of LX2006 to treat FA cardiomyopathy, a devastating and fatal condition with no currently approved therapies.
“Based on the favourable safety profile and clinical benefits observed to date, we are excited to explore expedited clinical development of LX2006, including potential for accelerated approval of this possibly life-saving treatment.”