Aurora - Phase 1/2 clinical trial for RHO mediated retinitis pigmentosa

What is Aurora?

Aurora is the name of a clinical trial which aims to study whether the investigational RNA therapy QR-1123 is effective and safe for people with retinitis pigmentosa (RP) due to the P23H mutation, also known as c.68C>A, in the rhodopsin (RHO) gene. Mutations in the RHO gene cause an autosomal dominant form of RP (adRP) and the P23H mutation is the most common mutation causing adRP in the United States. This Phase 1/2 trial was launched in December 2019 and is the first clinical study to investigate QR-1123. If you are interested in joining this trial, please read about the eligibility criteria.

What is QR-1123?

QR-1123 is an investigational RNA therapy that aims to stop vision loss or restore vision in people that have RP due to the P23H mutation in the RHO gene.

How is the Aurora trial set up?

Clinical trials are used by researchers to find out whether new medicines are effective and safe. Key aspects of the Aurora trial are the following:

• Aurora aims to find out whether QR-1123 is safe and well-tolerated, and whether it is effective at stabilizing or improving vision;
• The study will enroll adults (age 18 and up);
• Study participants will be randomly assigned to one of the following study groups:
• Single dose group: will receive one intravitreal injection (injection into the eye) with QR-1123.
• Multiple dose group: will receive up to four intravitreal injections with QR-1123 or up to four sham procedures (intravitreal injection is mimicked but no injection and no study medicine are given).

Participants will receive the study medicine or the sham procedure in the chosen (study) eye, which is the eye with the worse vision.

What is RHO mediated retinitis pigmentosa?

Retinitis pigmentosa (RP) is an inherited retinal disease that leads to vision loss and blindness. Symptoms usually start with night blindness during adolescence or early adulthood and further progress with loss of side (peripheral) vision leading to tunnel vision. Loss of central vision appears which frequently progresses to blindness in adulthood.

RP is a genetic disease that causes a mistake, or mutation, in the patient’s RNA. Because of the mutation, an essential protein in the eye cannot function, and this leads to the deterioration of the light detecting cells in the retina. RP can be caused by mutations in many different genes. In the United States, people often have RP because of the P23H mutation in the RHO gene causing a version of RP called autosomal dominant RP. Because of the mutation the RHO RNA produces a mutated version of the rhodopsin protein that is toxic to the retina. 

In the United States, approximately 2,500 people have vision loss due to the P23H mutation. To date, there are no other therapies approved or in clinical development that treat the vision loss associated with this mutation.