Future Research Directions for Revakinagene Taroretcel in MacTel

With the recent FDA approval of revakinagene taroretcel-lwey (Encelto; Neurotech Pharmaceuticals), patients with macular telangiectasia type 2 (MacTel) now have an approved treatment option for the progressive retinal condition characterized by irreversible vision loss.

In an interview with The American Journal of Managed Care^®, clinical investigator Charles C. Wykoff, MD, PhD, of Retinal Consultants of Texas, said that there is still more to learn about the cell-based gene therapy and novel encapsulated cell therapy (ECT) technology used to deliver the treatment. He also discussed the importance of patient groups in the MacTel space.

In part 1 of this interview, Wykoff discussed revakinagene taroretcel’s mechanism of action and the data supporting its FDA approval. In part 2, he discussed the unmet needs the therapy addresses and the ECT delivery system.

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

Transcript

What questions remain that may be answered through additional research?

With new drugs that come into the market and new surgical approaches, there's always a lot to learn. That's what's exciting about doing what we do to develop new drugs and new therapies for patients. There's always more we can understand. I think one of the lingering questions here is that both of these clinical trials, trial A and trial B for Encelto, or revakinagene, were both positive, and that's why the drug got FDA approved. But if you look, the magnitude of benefit appeared to be different, whereas 1 trial had a larger magnitude of benefit than the other. And related to that, one had a significant benefit related to microperimetry protection for patients and reducing visual function loss over time compared with the other trial, which was not significant. The question is, "Well, why?" Are there certain patients that can be better responders than others or certain patients that may be more appropriate for this treatment than others? I think we have a lot to learn about exactly who those patients are.

Some of the early post hoc analyses from this data set suggest that patients with earlier stages of disease—so those, in particular, with ellipsoid zone area loss of less than 0.5 mm², which was about the average in the trial—patients with less than 0.5 mm² may have a particularly higher chance of being a high responder, meaning that they have a 50% reduction in loss of ellipsoid zone area over time. Because if you're a patient getting a surgical procedure, you want to be more likely to have a high response if you can. And what it suggests is that earlier stages of the disease may be a sweet spot for getting maximal benefit from what we've seen in these post hoc analyses. But of course, those are hypothesis-generating. We need to validate that with prospective work and really look forward to looking at this data set in more detail and then hopefully have more prospective trials to better understand this disease process and how this treatment can benefit patients over time.

Do you have anything else to add about the MacTel treatment space?

I appreciate the opportunity to be able to talk about MacTel and this new, exciting treatment. Related to what we were talking about before, I think the online community for MacTel patients is remarkably robust for a relatively infrequent disease. And I think if patients have MacTel, they should be encouraged to get engaged. I think that one of the reasons why this therapy is now FDA approved is that there's a very robust, non-profit movement behind MacTel. There's the MacTel Foundation, there's the Lowy Foundation, and there are a lot of organizations that support MacTel patients. So if you're a patient with MacTel, I would encourage you to reach out to other patient groups related to MacTel and get involved.