FRIEDREICH ATAXIA CARDIOMYOPATHY (FA-CM)

FA: The Heart Of The Matter

If you are living with Friedreich ataxia (FA), you may be familiar with the
idea of a heavy heart. For many people with FA, the heart itself can
become affected over time, working harder than it should — a disease
known as Friedreich ataxia cardiomyopathy (FA-CM).

At the same time, there is another kind of heaviness: the emotional weight
of living with a disease that raises questions about what comes next.

Still, the heart holds hope. It seeks answers, and it looks toward a future
shaped by possibility. Lexeo’s clinical trials are designed with all of this in
mind and aim to get to the heart of the matter.

FA is a multisystem genetic disease — involving the nervous system and
heart — in which heart issues are the leading cause of a shortened lifespan.
Though considered rare, FA is the most common form of hereditary ataxia
in the U.S.

FA is caused by changes in the FXN gene, which provides instructions for
the body to make a protein called frataxin. Without enough frataxin, cells
can become energy-starved and vulnerable to damage. This is particularly
important in the heart, brain, and muscles.

Most people can recognize the classic signs and symptoms associated with
FA like movement difficulty and muscle weakness.¹ What often goes
unnoticed is damage to the heart.

Heart issues associated with FA are particularly dangerous because FA-CM
can begin silently but lead to severe consequences including abnormal
heart rhythms, heart failure, and shortened lifespan.^2,3

There are currently no approved treatments that address the underlying
cause of FA-CM. This makes clinical trial research for treating the heart-related
issues in FA crucial.

At Lexeo, we have made this unmet need a top priority and are currently
researching an investigational gene therapy called LX2006 in clinical trials.