LX2006 for Friedreich ataxia

Lexeo is currently conducting a Phase 1/ 2 clinical trial called SUNRISE-FA to evaluate the safety and efficacy of LX2006, an investigational gene therapy in patients with cardiomyopathy associated with Friedreich ataxia

About SUNRISE-FA

• The main goal of this trial is to learn about the safety and tolerability of 3 different doses of the investigational gene therapy, LX2006.
• All trial participants will receive a one-time intravenous injection of LX2006.

Key Eligibility Criteria

• Adults 18-50 years of age.
• Genetically confirmed diagnosis of Friedreich ataxia, with onset of disease on or before 25 years of age.
• Evidence of cardiomyopathy based on specific criteria.
• Full eligibility criteria will be evaluated by the clinical trial doctor to determine if an individual is the right candidate for this trial.
• Participants taking Skyclarys® (omaveloxolone) must be on a stable dose for a minimum of 12-weeks prior to screening. If a participant wishes to start omaveloxolone after enrolling, they may begin treatment one year after LX2006 administration.

Study Duration

This trial will last approximately 5.5 years in total, and will include:

• A screening period that may last up to 5 months.
• A 52-week study assessment period which will consist of 15 study center visits.
• A long-term follow up period consisting of approximately 5 study center visits and 3 remote visits over the next 4 years.

Participation Support

• The investigational gene therapy and all trial-related assessments will be provided at no cost to study participants.
• Any reasonable trial-related expenses such as fees for parking and meals will be covered by the study Sponsor, Lexeo.
• Travel and accommodation will be arranged for participants in the trial and for one caregiver.

Friedreich Ataxia Overview

Friedreich ataxia (FA) is a multisystem disorder primarily known for its debilitating impact on coordination and movement. Though neurological impairment is a hallmark of FA, endocrine and cardiac involvement often occur. Cardiomyopathy, a disease of the heart muscle, is a common complication in Friedreich ataxia or FA. Cardiomyopathy causes the heart muscle to thicken, which can lead to stiffening and scarring of the heart muscle, also called fibrosis. Cardiomyopathy makes it harder for the heart to pump blood to the rest of the body and can cause symptoms such as shortness of breath, fatigue, or a rapid heart rate. If this heart disease progresses, it can become heart failure. Cardiac disease, notably congestive heart failure or life‑threatening arrhythmias, associated with cardiomyopathy is the most reported cause of death in patients with FA.

In our bodies, there is a gene called FXN that gives instructions to make frataxin. Frataxin is an essential protein for our nervous system and heart to produce energy to work properly. In FA, there is a mutation in the FXN gene that limits the production of frataxin causing. many complications, including cardiomyopathy.

LX2006 Approach

LX2006 is an investigational gene therapy using an AAV vector, that aims to deliver a functional FXN gene, to cardiac cells to increase frataxin protein levels, thereby improving mitochondrial function and increasing energy production.

To learn more about gene therapy, visit the Gene Therapy Overview page.