MeiraGTx Holdings plc (MGTX) Earnings Call Transcript
December 3, 2025
Earnings Call Speaker Segments
Let's go ahead and get started here. So next up, really happy to be joined by Zandy Forbes, who is the President and CEO of MeiraGTx. Only 20 minutes, so we're going to dive right into it. So Zandy, over to you for a quick introduction of the company where things stand today? And then we'll go into some questions.
Okay. So Meira is a genetic medicines company. We founded the company to optimize the ability to use DNA as a therapeutic modality, and in doing that, we started with AAV, and we have capsids that we've developed, 250,000 promoters that we've developed with AI, and most importantly, the thing that we set the company up to do, is to control genes, trans-genes with pills. So we are now in a position, and we have done this in-vivo with many targets, every large pharma's antibody, so like a bio-similar as well as PTH, EPO, growth hormone, GLP-1, GIP, leptin, any peptide, you can -- we can take the natural sequence of any biologic, put that into the muscle like a vaccine, you take a pill everyday. And every time you take a pill, you dose very specifically with that particular therapeutic. It's a very powerful technology. We didn't know if we'll be able to do it, but we've done it. So we were a genetic medicines are a genetic medicines technology company who've developed some of the most innovative technology in gene therapy. And we recently -- you can look at our various partnerships around all of those technologies. which I think if you look at who those partners are, Sanofi, Lilly and J&J, that's validation of the technology. But in addition to that, we had a deep clinical program -- pipeline and all of the programs that we started early in the company used very small doses, locally delivered to avoid immune response, but also where we saw that there was really good proof-of-concept. And because of that, we now have 4 late-stage programs. They're not targeting only inherited diseases. We're a company that wanted to use DNA as a modality broadly. So our first two programs are in the eye in inherited diseases, awaiting filing. One is for an ultra-rare disease that is the most severe form of blindness in infants. 11 children were treated all under the age of 4, all 11 children went from completely blind to being able to see and function normally. So that we're filing globally with our partner, Lilly. Second is a much, much bigger indication. It's called RPGR. We have Phase III data, which we believe is approvable for sure in Europe and with discussion in the U.S. as well. That is currently with our partner, Johnson & Johnson. The two other programs are not for inherited disease. One is for Parkinson's, where we have two double-blind studies that have been statistically significant and positive. No one else has ever done that in Parkinson's disease with an intervention like gene or cell therapy, and we have a very interesting program in xerostomia, which is in a pivotal Phase II. Both of those have an RMAT and both of them, therefore, have Fast Track and breakthrough. And we have our own internal manufacturing end-to-end as well as process development in a very valuable process that we've developed over the last 10 years to manufacture our products, which speeds everything up. It's allowed us to be in this position of having all these late-stage programs.
Perfect. All right. Let's go program by program. Xerostomia. You've been so active on the BD front. I guess one of the questions is, is xerostomia going to be the first program that you take to the market totally yourselves?
It can be, yes, but we actually also have interest from others in that. That is a really interesting market because that's one of the reasons we took on xerostomia. It's not necessarily well known, but it's a very, very severe and the most important consequence of radiation that cures head and neck cancer. 30% of people who are cured of their head and neck cancer after 2 or 3 years, so this isn't concurrent with radiation. It's afterwards, so you're otherwise healthy, have severe or Grade 2, 3 xerostomia, very severe. To explain how severe it is, not only do you lose your teeth and can't sleep and can't eat, but for example, you can't exercise, because it turns out that if you have a dry mouth, you can't breathe any faster. So you literally sometimes can't run up the stairs. And so we have a treatment for xerostomia. We had really impressive Phase I data, Phase I/II data. And we started a Phase II. The FDA wrote to us after we filed our IND amendment and looked at our manufacturing and said, because this is manufactured by you, if your manufacturing does come within the parameters of your release specs that you've written, we consider this a pivotal, which is sort of extraordinary because manufacturing tends to be the issue for a lot of late-stage gene therapy programs. It is not with us. We have done everything in-house, and we have cross-referenced every filing with the FDA. So many, many reviewers have seen our manufacturing. We have commercial licenses in the U.K., in Ireland, we manufacture plasmid. We do the whole lot. As a result, our Phase II turned into pivotal. We then filed for an RMAT. We then got an RMAT meeting. We got alignment with the FDA on the endpoints, the stats, the clinical outline of the program. So we are currently approaching -- finishing enrollment of the higher dose cohorts of that Phase II. Sometime around the end of this year with data most likely in the first quarter of 2027 with a 12-month endpoint with filing shortly thereafter. We're targeting approval in '27. This is a sitting duck market. I know all markets require you to sell. But in this case, every patient is in the healthcare system. Every patient has been cured of their head and neck cancer. Every patient is going to their physician at least once a year, to have their tumor checked to see if it's come back, and this is their #1 complaint. One of the first people we spoke to about this was Dave Pfister, who's obviously oncologist, Head of head and neck at Sloan Kettering, and this was like his #1 problem outside even cured people with head and neck cancer, he could do nothing about this. So it actually is very severe. We have treated quite a few patients, as you can appreciate, and we have incredible feedback from physicians and patients about how this changes their life. So that's our first late-stage program. Second is in Parkinson's, where we completely avoid the quagmire of dealing with dopamine replacement. Rather, we do something that allows you to physiologically change the circuitry of the brain and avoid the need for dopamine in later-stage Parkinson's, a very small dose of the enzyme that makes GABA, the inhibitory neurotransmitter is put into the subthalamic nucleus. This is via an intervention that doesn't require -- it doesn't require general anesthetic. It's almost the same as deep brain stimulation, but you don't leave anything in the brain. And again, we've had 2 sham-controlled studies, which have shown statistically significant benefit, which is unprecedented, going into Phase III in the next several months.
Perfect. Just on the xerostomia side, can you just remind...
So can we market it ourselves? Yes, we can because all of those patients are sitting there. And it's a rather small oncology sales force, and there's a huge patient advocacy movement.
Perfect. For the xerostomia study, can you just remind us how you powered it?
We -- well, this is interesting because there's no precedent for placebo. So what we actually did is we looked at what's significant with respect to -- what's clinically significant in the xerostomia score, which is the agreed primary and a 7-ish point to 8-point change is significant. I think in our bilateral cohort, we saw a 21-point change against baseline and an average 17. So what our assumption was is that we allowed for a large placebo and we were looking for, I think it's 90 -- I can't remember the exact powering, a 7-point change between one of our arms and the placebo. There are two arms, which are pooled.
How much variability is there in this endpoint? Like is that something you monitor on a blinded basis and maybe it will be kind of more of a next year thing?
We absolutely -- we don't look at the data at all. It's -- it's now a Phase III. So it's super, super regulated.
Yes. What about pricing in this market?
So we did an L.E.K survey some years ago, and they went out to payers globally, and we had 95% coverage in the U.S. for 100,000 onetime treatment, which actually, if you look at the number of patients, currently, the patients eligible for this, not everyone treated with head and neck cancer is about 170,000 and 15,000 new patients a year, reach that 3-year time point when they're eligible for treatment. So that's just in the U.S., a really large market, complete unmet need because these patients have de facto failed any other possible treatment.
All right. Going over to the Parkinson's and the Hologen AI side for JD. When are you presenting the full data at a conference? I think you mentioned you're going into a Phase III in the next few months.
Yes, probably sometime next year.
Okay. That makes sense. What's your economic stake for Hologen AI also, it's maybe worthwhile to kind of introduce that.
We formed a joint venture. We own 30% of that joint venture. The joint venture is 100% paid-for all R&D, all clinical studies up to $230 million, which we felt was way -- well, was certainly sufficient to do the Phase III study.
Yes. That makes sense.
[ Almirall ] received $200 million to use for our...
Perfect. Let's go over to riboswitch actually. I think that's going to be an area of focus next year. Maybe you could just introduce the platform in a little more detail on why you chose Leptin as the first target?
Well, as I initially described, this allows you for the first time to deliver to the body a very accurate dose at a very accurate time of anything that can be encoded by DNA. So like Moderna used RNA, which you inject, makes the protein and then disappears. We use DNA. And the power of that is the sequence that we choose can be the biosimilar if it's an antibody, right, Dupixent or Erbitux or whatever it may be, or the native form of any peptide or hormone. And we've shown over a year in animals for Leptin that you can stop the pill, you can start the pill. And this last -- the dynamics are completely the same, day 2, day 370. So this is really a way it can be used in any context. So you don't -- we deliver through -- into the muscle with AAV. We can deliver through our lentivirus. We can knock it in. So we've also controlled the CAR in CAR-T, and we increase efficacy. This is most important when you're dealing with agonists like GLP-1. GLP-1 switches on its receptor. It's very well known that if it's long-acting, that receptor down-regulates, you need more GLP-1 and you control insulin less well. We deliver GLP-1 with a pill in the morning to the mice, and it works way, way, way better than long-acting GLP-1. So in the case of agonist biologics, this is the first time you can physiologically dose them, which changes what the amount of protein you need, but also the efficacy. Coming to Leptin, where there is an injectable Leptin, Metreleptin. However, it's a synthetic Leptin that's manufactured outside the body and it's immunogenic. So 10% of those people make neutralizing antibodies to Leptin, which is one thing, and that has catastrophic effects because when you have no Leptin, every calorie you eat turns to fat, your whole metabolism goes kaput, it's literally lethal, right? So it can't be used in many, many contexts. So we have made the natural Leptin sequence, inject it into the muscle of mice, and we can completely rescue in a dose response fashion the reduction or the absence of Leptin in mice. And this has been over a year, and that will be the first that we take into the clinic. We have this optimized sequence for injection. We have GMP material for the small molecules. We actually got two, which work really well. So full tox, everything has been done. The material is waiting for the clinic, and that will be our first in the clinic. And we'll probably be working with Sadaf Farooqi, who's the expert on genetic leptin deficiency.
Perfect. What's the status of the regulatory discussions and kind of time line for when its starting?
We're in interact. So we're working out how we do this as a filing with SEB A right, at the moment.
That makes sense. As you look towards the Phase I in humans, like how do you actually prove out the whole platform? Like are you doing continuous PK monitoring? How is that going to work?
In humans?
Yes.
We would. We would look at leptin levels as well as everything else, but yes.
And essentially, like as you think about what's a good outcome in humans, you're kind of replicating what you showed in the animal models. Is there any difference as we're thinking about translating?
Does it work? Normal differences, right? I mean we all look at mouse experiments, but this injectable leptin works. We have very, very precise dosing. Really super precise. One small molecule makes RNA and it's really precise, way better than injecting something. So we have very, very strong confidence that it works.
Yes. All right. Perfect. Maybe on the manufacturing side, you kind of alluded to this briefly, but are there any public comps out there for how to think about the value of this business on a stand-alone basis? Obviously, it's a little complicated since there's a lot of strategic value to what you guys are working on.
Yes. So with manufacturing for each of our partners, we're the manufacturer because in gene therapy, if you manufacture -- when we manufacture because of our FDA and global regulatory interactions, our manufacturing process, which has been built on 20, 30 different viral vectors, actually gives us a GMP process fit for commercial, right, which is very, very valuable. Now there have been similar companies sold, who don't have commercial experience. We actually have commercial license and have not done as many runs or batches or assays as we have because we do QC. The whole of QC is validated and in-house, we've got commercial license. And I think it's sold for around GBP 600 million. But from a strategic perspective, we really wouldn't be in this late-stage situation, particularly if we hadn't bought QC in-house. And that's quite difficult. We use our preclinical guys. They do our potency assays. We translate them to cell-based assays. And it's very hard to explain how valuable it is. But from a timing perspective, it allows us to have very clear quick conversations with regulatory agencies about what commercial looks like and not what a clinical batch looks like. And that's very -- we learned that with J&J, our first partner, who we do commercial manufacturing for.
Perfect. Well, right on time. So we'll wrap it up there. Thanks so much for joining us Zandy.
Thank you.
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