VIDEO: ‘Mind-shattering’ potential of gene therapy for neurotrophic keratitis

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One of them is varenicline nasal spray, and we know that as over the counter, as a dry eye treatment, as Tyrvaya (Viatris). Now remember how it works, right? It stimulates your parasympathetic nerve ending inside of your nostril, and then it increases lacrimal tear production. But remember, it’s stimulating your nerves. If it’s stimulating your nerves, can it be used for other things? And that’s what they're looking into, in a much higher dosage though.

They have an ongoing, phase 2, multicenter trial that’s investigating intranasal varenicline with 0.12 mg, which I believe is four times the concentration we currently have on the market. They’re looking at the utilization of that higher concentration for symptom relief in patients with stage 1 NK. Remember, it’s symptom relief. It’s unknown whether or not that’s actually going to have some sort of nerve-regenerating function in the long term.

Two, remember we talked about different growth factors, different peptides that can promote wound healing. There are two in this category that I’m rather excited to see what would happen. One is thymosin beta-4, which is a naturally occurring polypeptide that ranges in effect clinically have been observed, such as stem cell recruitment migration and anti-inflammatory properties and so on. So safe to say, can help with neurotrophic patients. And I believe that they are now entering a phase 3 trial or about to. It’s called SEER-2. I think they’re trying to get a slightly larger cohort than in their phase 2.

The next one is being produced by Brim Biotechnology and the compound that they’re studying is coded BRM-424. It’s a regenerative peptide that supposedly also stimulate limbal stem cell regeneration, very similar to the thymosin beta-4 that I just talked about. And again, it may then have regenerative properties to treat NK patients. And the reason why I think two or both of them are very exciting is because a lot of growth factors, clinically, if you look at their studies, trying to figure out what’s the best vehicle and best concentration. But most studies that I know, looking at growth factors, they seem to have more struggle in generating a positive outcome for patients with limbal stem cell deficiency because there’s nothing to recruit from stem cell? The fact that both of these compounds they may be able to do something with stem cell, may possibly be, in my mind, may have a little bit better outcome in the future, or at least wider ranging patient targets that we could use this for.

And the very last one, remember we talked about varenicline, which is coded OCO-1, the same maker is actually looking at, I believe it’s going to be an injectable, gene therapy. They’re looking at delivering the gene that is responsible for nerve growth factor production in the lacrimal gland. They’re looking at delivering that with the adeno-associated virus vectors, which again, obviously probably later than the first three that I talked about in terms of timeline is my belief. But again, you can imagine that if we could actually utilize gene therapy in NK space, it would be mind-shattering to me. It’s blowing my mind just kind of reading about it. I will watch out for that as well.