Dr Byron Lam Reviews Current Treatments and Therapies in the Pipeline for Inherited Retinal Disease

The most exciting treatment for inherited retinal disease was approved in 2017, but there are many novel treatments in phase 2 or phase 3 trials at this point, said Byron L. Lam, MD, professor of ophthalmology, University of Miami Miller School of Medicine's Bascom Palmer Eye Institute.

Transcript

What does the current treatment landscape look like for inherited retinal disease?

I think the most exciting treatment, that was approved in 2017 and first patients treated in 2018, is, no question, the approved gene therapy for RPE65-associated inherited renal disease [Luxturna, voretigene neparvovec-rzyl]. A number of patients have been treated, and the results and the efficacy of the treatment is very compatible with the original phase 3 study. There have been some new findings in terms of the real-world experience. One of then is this development of atrophy of the retina that can occur, sort of in the perifoveal region as well as the peripheral retinal region. However, in those patients, despite the development of the retinal atrophy, they still have very good efficacy. What's the reason for the atrophy? We're still learning, but in general, the rules hold: The earlier you'll treat these patients, the more responsive they will be. So, it's really a major breakthrough.

What are some key therapies in the pipeline?

We have many different novel treatments that are under clinical trials for inherited retinal disease. I think if you're looking at human clinical trials that are either in phase 2 or phase 3, or about to go into phase 3, I think the most exciting inherited retinal disease [therapies] that are going to be in pivotal trials will certainly be the X-linked retinitis pigmentosa, RPGR, because Janssen is working on this with MeiraGTx, as well as AGTC [Applied Genetic Technologies Corporation]. We certainly look forward to the pivotal phase 3 clinical trials, and then we want to know whether these treatments indeed work. I think that's the exciting part.

I think there are a few exciting results that have occurred in other inherited retinal diseases in earlier phases. Certainly, some of them are too early to tell. We'll have to wait until the phase 2 and phase 3 [trial results].