Lexeo Therapeutics is a pharmaceutical company developing a gene therapy called LX2006 for people with Friedreich’s ataxia (FA) cardiomyopathy (a condition involving stretching, thickening, or stiffening of the heart muscle, making it harder to pump blood around the body).
Frataxin is a protein that is deficient in people with FA. LX2006 is designed to transfer the FXN gene (which codes for the frataxin protein) to heart cells and increase frataxin protein levels in the energy centres of cells called mitochondria. The increase in frataxin levels in the mitochondria restores energy production in heart cells.
Lexeo announced the completion of their first cohort in their SUNRISE-FA phase 1/2 clinical trial of LX2006 in those with FA cardiomyopathy in June 2023, where the therapy was well tolerated with no unexpected events observed.
On 11th March 2024, Lexeo announced preliminary data from a subset of their second dose cohort of the trial, showing an increase in frataxin levels at 3 months following treatment with LX2006 compared to pre-treatment baseline levels. SUNRISE-FA is the first clinical trial to evaluate frataxin levels in the heart via cardiac biopsy, where heart tissue is taken from a person for examination before and after a treatment. 3 participants in the trial showed an increase in frataxin 3 months after treatment with LX2006, as shown in cardiac biopsy data. Lexeo expects further data from the trial in mid-2024.
Read their press release here.
Read more about the SUNRISE-FA trial here.
Read our previous article on the trial here.
Update on 16th April 2024:
On 16th April, Lexeo announced that they have been granted a fast-track designation by the FDA for LX2006 for people with FA cardiomyopathy.
Fast-track designations are designed to support the development and expedite the review of new drugs to treat serious conditions. In this case, the fast-track designation was granted based on preclinical data on LX2006 for FA cardiomyopathy.
Read the press release from Lexeo Therapeutics here.
