Ann: Successful Major Milestone - Human Retina in a Dish Models, page-41

In the July presentation on PYC’s flagship program, one advantage listed for the path to market (Slide 15) was that ‘Proof of Concept’ for ASOs in retina had already been established by ProQR in its early results of sepofarsen in the inherited retinal disease, Leber congenital amaurosis 10 (LCA).

An 11 patient Phase 1/2 trial of sepofarsen in LCA was commenced in October 2017 and positive interim results were released in September last year.

Today, ProQR has released (early) top-line results for its Phase 1/2 trial, ahead of the American Academy of Ophthalmology's annual meeting being held in San Francisco.

It was reported that patients in the trial had demonstrated “a clinically meaningful improvement in vision, and in some cases the patient’s vision improved to a level that could be deemed life changing.” The company stated that the top-line data strengthened its confidence in the design of its Phase 2/3 trial for sepofarsen and boosted its hopes that this might be the sole registration trial required. That trial has already been commenced, based on last year’s positive interim results, and top-line data is expected in the first half of 2021.

The positive results announced today were achieved with the target registration dose (80 µg with a 160 µg loading dose) and a six-month dosing frequency.  The response observed at 12 months in the target registration dose was equal to or greater than the response at the 3-month interim analysis.

https://ir.proqr.com/news-releases/...nces-positive-top-line-results-phase-12-study