How Revakinagene Taroretcel Can Benefit Patients With MacTel: Dr Charles Wykoff

Revakinagene taroretcel-lwey (Encelto; Neurotech Pharmaceuticals) recently became the first and only FDA-approved treatment for macular telangiectasia type 2 (MacTel), a progressive retinal condition that can cause irreversible vision loss.

The newly approved therapy fills a significant unmet need for patients with MacTel and employs a novel method of administration with potential in a broader range of retinal conditions, clinical investigator Charles C. Wykoff, MD, PhD, Retinal Consultants of Texas, explained. In part 1 of this interview, Wykoff discussed revakinagene taroretcel’s mechanism of action and the data supporting its FDA approval.

This transcript has been lightly edited; captions were auto-generated.

Transcript

What are the clinical implications of revakinagene taroretcel’s approval for patients with MacTel?

It’s an exciting time for patients with MacTel. We've been fortunate, as a retina field, to have great therapeutics for many diseases in retina, but many diseases have significant unmet needs, as well, in retina. MacTel has been one of those lingering unmet needs where patients have had no potential therapies to slow their disease progression, and so to have now a potential therapy is exciting. But of course, the main question is, “Well, great. Now we have the surgical procedure. Who do I put this in? Who do I treat with this?” And I think using the inclusion criteria in the phase 3 trial is a good place to start. There are some patients with MacTel who have very minimal disease and have a lifetime of essentially normal vision, and those patients probably do not need a surgical procedure for treatment. But many of our patients with MacTel do have progressive distortion of their visual field and loss of visual function, as we've seen in the clinical trial. And it's those patients with clearly visible areas of ellipsoid zone loss on OCT [optical coherence tomography] with visual symptoms, that I think it's worth having a discussion with patients about the risks and potential benefits of treatment and considering treatment. I've been pleasantly surprised by the community of MacTel patients. I think they are uniquely unified online. There are all sorts of groups that support MacTel, and it's important that these data be disseminated and that patients become aware of this potential treatment option.

Can you discuss the method of administration and how it sets revakinagene taroretcel apart from other therapies?

This is a fascinating mechanism for drug delivery, and I think the most interesting thing broadly about this is just the platform technology itself, because you can imagine having a different cell line that was engineered to produce a different therapeutic protein to treat other diseases. This is a technology that is exciting and potentially could be here for a long time in retina as new indications potentially come online in the future. There's nothing in the near future for that, as far as I'm aware, but certainly something down the road. This is a surgically implanted device, so you need to go to an operating room and a sterile setting, and then the surgical procedure has been incredibly well thought out at a very detailed level. And every step, we sort of know a lot of the potential risks to be aware of, and the potential adverse events that can happen if things aren't done in a certain way. It's really important when surgeons are beginning to consider doing this that they really consider those surgical steps carefully before proceeding.