The following transcript has been edited for clarity.
Hi, this is Aleksandra V. Rachitskaya, MD, FASRS, from Cleveland Clinic Cole Eye Institute, and I just finished my presentation here at the Retina Society meeting in Los Angeles, looking at the results of the phase 2 DAWN trial. Now, the DAWN trial was a trial that used laruparetigene zovaparvovec (laru-zova; Beacon Therapeutics), which is a subretinal gene therapy for patients with X-linked retinitis pigmentosa (RP). As we know, X-linked RP affects mostly male patients and leads from loss of peripheral vision to central vision and even legal blindness, and there’s currently no treatment.
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Now, laru-zova has been studied pretty extensively along different studies. This study was the phase 2 DAWN study in which we treated the fellow eyes of the patients who were previously treated in SKYLINE and HORIZON. So what did we see?
First, of course, in the phase 2 trial, we looked at safety, and overall laru-zova was very well tolerated. There were no suspected/unexpected serious adverse reactions (SUSARs), no retinal detachments, no endophthalmitis cases. We saw some changes mostly related to the surgical procedure and to use of the corticosteroids. The inflammation was mild and usually controlled as needed with steroids.
When we look at the visual outcomes, the 2 visual outcomes that were studied were a change in low-luminance visual acuity (LLVA), and we saw that patients in the study eye had more gain of 2 and 3 lines of LLVA compared to the fellow eyes. And we also saw improvements on microperimetry.
So overall, as we look at this phase 2 DAWN study, which was the treatment of the fellow eye of patients who were recruited in SKYLINE and HORIZON, it was well tolerated with an acceptable safety profile. And we saw this improvement in LLVA.
What is exciting is, just recently there’s been a press release looking at the results of the pivotal phase 2/3 VISTA trial in which laru-zova was administered in high and low doses, and it met its primary endpoint looking at LLVA.
So to see and hear more results of the VISTA phase 3 trial, those are going to be presented at the American Academy of Ophthalmology Subspecialty Day in the next couple of weeks. RP







