Lexeo’s gene therapy shows promising phase 1/2 results for Friedreich's ataxia cardiomyopathy

The results​ are from eight patients with 6-12 months of follow up data and show reductions in left ventricular mass, thickening of the heart wall and myocardial injury as a result of the treatment.

The therapy, LX2006, is being tested by the company, but also by Weill Cornell medical school in New York and the trial is ongoing. As of July 15, 13 patients have been treated with the therapy across two trials. Next steps include testing a higher dose of the therapy.

“We are very encouraged by these data and the potential of LX2006 to treat FA cardiomyopathy, a devastating and fatal condition with no currently approved therapies,” said Eric Adler, Chief Medical Officer and Head of Research, at Lexeo in a press statement.

“Based on the favorable 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.”

As well as a positive impact on cardiac symptoms, no significant side effects were seen in patients given the therapy and no participants discontinued the trial.

Focus on rare disease​

Lexeo is focused on developing gene therapies for rare genetic cardiac and neurological conditions, including for people with APOE4-linked Alzheimer’s disease. 

Friedreich's ataxia is inherited in an autosomal recessive manner and is caused by mutations in a gene that encodes the protein frataxin (FXN​) on chromosome 9. People with this disease produce less frataxin than normal and this causes degeneration of nerve tissue that leads to difficulty walking, loss of coordination and impaired speech.

Symptoms of this condition can start as early as 5 years of age and are usually present by the age of 20. Although it is a rare condition, it is one of the most common inherited ataxias and impacts between 1 in 20,000 and 1 in 50,000 people of Caucasian descent, in whom it is most common.

Many people with Friedreich’s ataxia also develop cardiomyopathy, where the heart struggles to pump blood to the body, mostly caused by abnormal thickening of the heart wall in people with this condition. Life span varies in people with Friedreich’s ataxia but cause of death is related to cardiomyopathy in up to 80% of people with the disease.

LX2006 is an adeno-associated viral vector therapy designed to restore the function of FXN​ and increase levels of the protein in people with Friedreich’s ataxia to treat symptoms of cardiomyopathy.

The first therapy, omaveloxolone, to treat the condition was approved by the US FDA last year and the EMA​ earlier this year. However, it is not specifically focused on cardiac symptoms of the condition, which Lexeo hopes to target, and is focused on treating neurological symptoms of the condition.