News update Initial results from QR-1123 Aurora study in adRP

We are pleased to announce an update on our third clinical program QR-1123 and our Phase 1/2, Aurora clinical trial, for autosomal dominant retinitis pigmentosa (adRP) due to the P23H mutation.

The Aurora logo with a green stamp 'Results' displayed on a desk scattered with a plant, an eye chart, USB stick, and a study design schematic.

Autosomal dominant retinitis pigmentosa (adRP)

AdRP 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. Specifically, the P23H mutation, also known as c.68C>A, in the rhodopsin (RHO) gene is the most prevalent mutation associated with adRP in the US, accounting for over 2,500 cases.

QR-1123 for RHO-mediated retinitis pigmentosa

ProQR develops investigational RNA therapy QR-1123 that aims to prevent vision loss or restore vision in people with adRP due to the P23H mutation. QR-1123 works by binding to the mutated version of RHO RNA so that it is removed, and the normal version of the rhodopsin protein can perform its function in the retina again.

Aurora Phase 1/2 clinical trial

Aurora is the name of a clinical trial which aims to study whether QR-1123 is effective and safe for people with adRP due to the P23H mutation. The eleven study participants received a single intravitreal injection (injection into the eye) with QR-1123 at one of five different doses.

This Phase 1/2 trial was launched in December 2019 and is the first clinical study to investigate QR-1123.

Initial results

Today we can announce that our key objectives were met. A single dose of QR-1123 was found to have a manageable safety profile and appeared to be well tolerated by the study participants. Across all doses evaluated no serious adverse events were observed.

With regards to efficacy signals in Aurora we are pleased to announce that we have seen signs of target engagement across the majority of trial participants, and across different endpoints studied.

For further details on the results see the QR-1123 section of the 2021 Analyst Event presentation.

Next steps

With the announcement today, we hope to move to a new stage of clinical development with QR-1123 and plan to proceed to a Phase 2 repeated dose study in 2022.

Interested to learn more?

Want to find out more about QR-1123? Visit our page for QR-1123 for autosomal dominant retinitis pigmentosa.

If you would like to learn more about how genetics play a role in our clinical research? Check out our dedicated webpage what are inherited retinal diseases?

Do you have questions about what kind of sight loss you have? Please reach out to us on patientinfo@proqr.com.

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