Viridian Therapeutics, Inc.\DE (VRDN) September 2026 Conference Transcript

Good afternoon, everyone. We'll get started here with the next fireside discussion. My name is Derek Archilla, one of the senior biotech analysts here at Wells. Very excited to have with us Viridian Therapeutics from the company. We have Steve Mahoney, president and CEO, as well as Shan Wu, chief business officer. Thanks for coming. Lots to talk about.

Thanks for having us.

So maybe, you know, it would be a good place to start, just state of the business. You guys are amidst the launch.

You've got a couple other things going on in terms of the pipeline. but maybe just kind of yeah give us that state of the business and then we can kind of dig in some some of the more you know nuanced questions here yeah sure uh so we're two months into the lumvoa iv launch uh which is great so uh all systems go there um well we can i'm sure we'll get into more details as to what we're looking for there we are preparing our boa submission for our sub-q program for, this is all for thyroid eye disease, and so we expect to submit that BOA for our sub-Q program in the first quarter of 27, so right around the corner now. We have a TSHR program, a TSHR antagonist that we are expecting to submit to IND and and get into the clinic. IND will go in this year, Q4. We'll get into the clinic early next year. That's an exciting program with applicability in both thyroid eye disease and with Graves' disease, and there's some overlap in those diseases in terms of the population, so that's a good complementary part of the portfolio. And then we also have FCRN data coming as well, so we have a half-life extended FCRN program that we're working through the first in human trials and we're gonna collect that data and we said we would come out and present it or disclose it and give our indication or clinical development plans going forward for the half-life extended program we also have another program which is an FC fragments very similar to the VivGuard approach where we've already characterized that data that studies complete so we know that data hit the IgG suppression thresholds that you'd want to see and that it was albumin sparing. So we decided we wanted to see how the Half-Life Extended program played out in the first Inhuman, and, again, we'll have that this year, and then we'll be able to decide what to do going forward.

So that's the portfolio in a nutshell. Awesome, high level. All right, well, let's dig into the Lumvoa launch, obviously top of mind for most folks. But maybe, you know, what are we seeing kind of in those first two months? What's kind of the feedback from physicians, and how operationally have you guys kind of executed thus far?

Yeah, operationally we are doing very, very well. Teams are doing a great job and that goes with sales reps, it goes with medical affairs, patient access liaisons, which is basically a patient support services team, supply chain. All systems are clicking, and that's really important to see, and all the support teams that go with that. So operationally, very exciting to see. We came out in, so our Purdue date was June 30th. We actually got approval on June 26th, which has some advantages in terms of J-code. I'm sure we'll get into that detail. But we had our Q2 earnings in mid-August. So we were about six weeks into launch at that point. We indicated there were three main categories that we're tracking to understand how well launch is going. First is field engagement. Are we getting access to the physicians that make up the core prescribing base? We know from profiling that there are about 2,000 core prescribers that make up 80 to 90% of all Tepeza scripts that are written today. And so our strategy is to make sure that we're engaging with them and trying to make sure that they're aware of our data and our new label. We did say in that Q2 earnings release that we had engaged with 95% of that 2,000 core prescriber group within six weeks after launch. So really good field execution. Great to see. That's a metric that's really important to us. And this is actually real engagement. This is not just sending emails. This is actually talking. So really important there. Second element is market access. We have to get out and make sure that we're talking to the payers and trying to get policy adoption for Lumevola. We have been doing payer research for a number of years. We did our pre-approval information exchange with payers starting in January, knowing that we had a June 30th PDUFA date. So those conversations were productive. Essentially, the guidance that we got from payers at that time was for parity pricing, we could expect parity coverage. This is a reminder, Tepeza roughly has 85% of covered lives today. I mean, it took them five, six years to get there, but that's a great footprint for us to step into, particularly with the guidance that we got from payers. Now we're just simply working through those conversations in a post-approval setting, and we did guide to parity pricing on our approval call. So again, we're going to be able to step into that footprint of coverage. So that's a second element that we're tracking for launch. And then finally, and obviously critically important, is demand, right? What kind of physician demand are we seeing? How is that translating into patient demand? And we're obviously paying very close to that. We see what we said in the Q2 Arnie's release was that based on the patient enrollment forms that we were seeing, that we saw broad and actually deep demand, too. So we were seeing physicians not only on the breadth side, but also seeing multiple scripts coming out of them. So all of the three categories that we track, and there's tons of subparts that go underneath those, but they all look good.

What are the couple of things that kind of go from engagement to utilization among these docs? I mean, obviously, you've got an entrenched competitor. So what is the sales force and kind of the commercial messaging to these high prescribers?

Yeah, so there's three key elements. This is the basis of our breakthrough therapy designation. We applied based on three elements. and as you know, we got breakthrough therapy and priority review for Lumevola. The application was based on rapid onset of treatment effect. We were seeing a majority of patients respond on their proptosis. So just as a reminder to folks in the audience that this disease primarily affects women in their 40s and 50s. Proptosis is a bulging of the eyes. It can be disfiguring. And then there's also an element of diplopia, which is double vision. So can't read, can't drive, can't, you know, it's very difficult to live your daily life with double vision. And there's also elements of pain and friction and redness that come in the eye too. So when we're talking to physicians and we're talking, you know, and they're having their conversations with patients, we're seeing rapid proptosis response after just one infusion. That's an element that we want people to see, the majority of patients achieving it after just one infusion. The second element is we ran chronic patient population studies, very robust. We ran the full spectrum of chronic patients. We did it as part of our registration studies, which Tipeza did not do. They ran theirs as a phase four. So we got our chronic data in our label, and what we saw in our chronic data consistent with active in terms of the outcomes, in Diplopia, we saw really good response rates, and we saw really good resolution rates, which is really important. So instead of an improvement, you're actually seeing complete resolution, and that hadn't really been seen before in chronic. So that's another talking point with physicians pointing out that level of data. And then lastly, the basis of our application for breakthrough was the fact that we are, we're five infusions, Tepeza has eight infusions, we put in 10 mg per kg versus 20 mg per kg, we have shorter infusion time, the 12-week treatment period versus 21 weeks. And when we did market research, the patients would say that they were declining to go on to PESA simply from the standpoint that the treatment burden was just too much, was one of the main elements as to why they would decline. So we think we've made that easier, and then obviously, and I'm sure we'll get to this, obviously we think that the sub-Q will make things even easier from there, and then we have an answer for every patient that walks in the door.

Got it. No, super helpful. It might be good to illustrate kind of like the current process from a patient enrollment form to first infusion, just kind of not only like is there friction to write the start form or the enrollment form, and then, you know, there's usually the doc writing and then the infusion center doing the infusion. So maybe just kind of remind us how all the dynamics play here. Do you want to walk through that?

Yeah, sure, definitely. So given the profile advantages and the very robust clinical profile that Steve just talked about in those three points of potential differentiation, we feel really good about this new start market where patients really get to choose every time that they're coming in to have a conversation about a treatment option for TED. They get to choose between Lomboa and potentially Tepeza, and so we feel good about the clinical profile. What actually happens as a patient, if they and their physician chooses LIMBOA, is an enrollment form gets submitted electronically. And that's sort of how physicians write scripts today. They're not writing it on a pad of paper anymore. And so the enrollment form activates our Viridian CARES program, which is the patient services program that we have built to very specifically walk through and hold the hands of the patients, the physicians' offices, and then ultimately the infusion centers through that process of getting a patient on treatment and through treatment. So one of the first things that happens is a benefit verification that a patient has the insurance coverage to be able to support lymphoma and connecting that patient then with a infusion center that is convenient for them, getting them scheduled. and then the infusion center will ultimately do the robust prior authorization through the insurance company. Starting out, before we're at a critical mass of coverage at payers, many of these Lomboa scripts will go through a medical exception process at the payer side. But again, that's why Viridian Cares is so important to have built, and we had that available on day one right after we launch so that patients are supported, physicians' offices are supported, and the infusion centers are supported through getting this prior authorization process. Tepeza today, just to use as an example, it's also a high-priced biologic IGF-1R. It takes, on average, 60 to 90 days based on our data for a patient to go through that prior authorization process. For us, if we're going through a medical exception process, it can take a little bit longer, So we would expect to be on the higher end of that range, closer to the 90 days on average. Obviously, every patient and every insurance plan is going to be different. There will be a range. But once the patient is through that prior authorization, then that's when the patient sits down at the infusion center to start to get their infusions. The way that the vials are ordered and shipped is the infusion center would order from a specialty distributor in time, just in time for that patient's infusion that is scheduled. So there's minimal stocking at the infusion center. And in terms of revenue recognition, the revenues come in for us when we ship to that specialty distributor, which is then filling vials as the infusion centers are ordering the vials. So this process makes it such that in the early days after launch, revenues will naturally lag behind leading indicators of field execution, of payer coverage, as well as demand for LEMVOA. And so those are the areas that we will be looking at that we'll talk about on the Q3 earnings call in November. And revenues, once we get into 2027, when we have a funnel of patients who have stacked up, the revenues will naturally then catch up and be much more representative of the number of patients on therapy.

So, I mean, you just kind of answered a lot of the questions I had. But looking at, you know, start forms is going to be the most kind of key, you know, indicator of the demand, right? So when we get to 3Q, it'll be more about kind of, you know, those enrollment forms. I guess, you know, it sounds like there's probably, well, maybe you tell me, but how much friction is there to even, like, write a starter form? Because it seems like, at least from our checks, that's been fairly easy for what you just said, the Verdean cares. A lot of this has, you know, really helped from the patient side and the physician side, but maybe just kind of communicate to us, you know, what friction exists in that component, and we can kind of talk about the downstream stuff on the J code and whatnot, but that's more on the revenue side, not the starter form side.

Yeah. So I think start forms, enrollment forms are a reasonable indication of demand. We've not said exactly what the demand metric will be once we get to Q3 earnings. We'll report what we think is the most meaningful for us as well as for investors. We're obviously tracking a number of different metrics as it comes to demand. In terms of friction, it's really the typical things that are more buy and build logistics that I kind of walk through and so directionally speaking those are the kinds of things you're getting through onto coverage for payers so that we no longer have to go through that medical exceptions process getting the prior authorization time from an enrollment form to that first infusion down as much as we can in terms of an enrollment form that is really driven by the profile of the drug if the profile of the drug which we really think feel very confident about and it kind of speaks for itself that's what will drive the enrollment form and the enrollment form we've specifically made to be very simple very familiar to the physician a couple of tweaks to actually streamline it from the existing enrollment forms that they might be used to but really trying to replicate that experience for them as much as possible to reduce the friction that comes from submitting a enrollment form for lymphoma got it and then maybe on the flip side so with the infusion centers you guys have been working with these guys um to essentially sure you know future reimbursement things so maybe you can kind of talk about you know things that you did there to just ensure that there is utilization there's less friction point there as well yeah we we've been talking uh with those infusion centers for quite a while and there's national ones that where you can

get you know they have you know several hundred infusion centers under their umbrella so we can we've had access to them we've explained what was coming they have experience with the PESA the the key issue that you touched on is they don't want to be underwater but they don't want to order drug and front money and then not and be at risk for reimbursement so we're very it's very simple to cover for that. We just adjust payment terms in that short period until we get that permanent J-code. They're very familiar. Infusion centers are very familiar with temporary J-codes versus permanent. So in this particular case, we actually don't think this is going to be problematic for us.

Gotcha. And again, that's not something that is novel to you. This is something that is used across the industry in terms of extended payment terms and ensuring that drugs available.

100%. And our team is very, very experienced in buying bills, so they know exactly where the pain points are. Gotcha.

And we were able to submit our JCO application as well before the start of the third quarter since we got approval at the end of June. So we were able to get that in before July 1. So we would expect a permanent JCO on January 1.

Excellent. Perfect. So I guess when you think about, you know, the market here, so Tepeza has been on the market for a number of years, U.S. market kind of like maybe stagnating a little bit. But I guess do you think there'll be maybe a resurgence of growth overall? Like are there other patients to pull in at least, you know, active, and you'd love to get your thoughts on chronic with IV, but I don't know, like, how do you think the overall TED market grows potentially with Lymvoa in the market? And then the follow-on to that is just, you know, with the physicians, you know, now with two choices, you know, kind of to make it, there's some differentiation here. Is there a possibility of just, is it like a zero-sum game where they just go all the way, all in on one, or is there reasons to use one or the other for different types of patients?

Yeah, I think we'll have to see how that plays out. And we're still two months in, so let's see how that plays out as the zero-sum. I'm not sure I would expect zero-sum, but I think that we'll certainly be watching for that to understand. Now, to your first question as to whether there'll be a natural expansion, there could be an element of a natural expansion with two products on the market, another voice at the table, there's a lot of patient education going on out there you know between us and Amgen to try to you know increase disease awareness, increase diagnosis rates but just a reminder our commercial strategy particularly in this early days is to convert like we want to go with if someone's writing a Tepeza script we want to be in front of them we want to we want to make sure that they're aware of the limboa label they already know the mechanism it's IGF-1R inhibition so there's some level of comfort already by virtue of them writing to PESO scripts so that's our first and foremost and so of the you know of the 6,000 patients roughly that that are treated annually today the patient mix is about 80% active and then the other 20% is kind of split between chronic patients and retreated patients. More chronic than retreated, but it kind of varies. And I think we would expect a similar patient mix coming out because of our conversion strategy with those Tepezo writers. But again, we're still early days, and we'll see how it plays out.

I mean, does that leave, I don't know, chronic TED with LOA as kind of an underappreciated opportunity just because you have the most robust data set with chronic right i think one of the things that i would say with tepeza and maybe why they have so few patients with chronic is data is not it was like a phase four trial it wasn't really as robust does that i don't know like is that a message that you guys are trying to send certainly i mean we are so we have chronic data in our label we ran um as you said a robust study where uh clinical activity score is on a scale of 07, we ran the full scale of patients because we think that's representative of the actual chronic patients that are out there.

There's a lot of heterogeneity in the disease, and so you'll see some chronic patients that have proptosis. They may not have diplopia. They may have both. They may have an active-like flare in that chronic population just by virtue of the they've been living with the disease, but no one knows what the underlying cause is. It could be lack of sleep, it could be stress at work, it could be a number of things. But do we think that the Lomvoa profile can start to help penetrate that population? Certainly, because we do have the data, it is in our label, the physicians are familiar with it, and we do have really strong results in the chronic data set with respect to proptosis reductions. And really importantly, as I said, in the breakthrough therapy designation, that diplopia response and resolution was a key component of our application for breakthrough therapy because that type of response hadn't been seen. Yeah, understood.

So I guess looking forward, and then we have the launch still really early, but based on what you know, are you more convicted about becoming the market leader as the IGF-1R, like the lead IGF-1R, or I guess what would take it to get to that level? What would you need to see?

Yeah, I mean, I think we really love the data set that we have for lymphoma in both the active population and the chronic population, again, across the key metrics, which is proptosis and diplopia. And so, yeah, I mean, we do think that we have a great drug for these patients. We think we, by virtue of five infusions and 10 mg per kg and shorter infusion time, we think we make things easier for patients, and that's a big part of what we're trying to do. That actually carries forward into the sub-Q story as well, because that's even easier for patients. Now we're mailing an auto-injector pen to your home, and you're able to self-administer in under 10 seconds. So we think that is just the next step forward in terms of making things easier for this patient population, which, given the numbers, given the prevalence, given the incidence rate of 40,000 patients that fit into that active phase of the disease, this is an underpenetrated market. I mean, it's a single-digit penetration of the market. We need to bring people in off the sidelines, and we think the lymphoma profile, and then when we get there the sub-q profile we think that the combination of those is going to is going to be able to do that.

Gotcha and you're still early in the launch but what's kind of been the Amgen counter detail like what are you seeing anything or like what are we learning so far about how they're responding to the new entrant?

Yeah I um I would say we are there was a new commercial and if you watch the the nightly news it comes on uh so that there's a new commercial just in that commercial references that Tepeza's been around for a while and that physicians and patients should take comfort from the fact that it's been available which you know understandable as a counter detail but I think that's certainly not something that you know overly concerns us so again we we feel really strong about the profile that we have and our job is to get out there educate physicians educate patients make sure they're aware of what we have to offer, and I think that's going to carry the day for us.

There are a couple of things here, too, that Tepeza, or Amgen, I should say, would typically be able to do that they're not able to do. So because the prescribing physicians are not the ones who are doing the infusion, there is no buy-and-bill economics for anyone, including Amgen, to provide rebates to the physician to try to incentivize them to prescribe one drug versus the other. The other thing that came from the Horizon Amgen acquisition process is Amgen had a consent decree that, to our understanding, we believe that Amgen is not able to bundle Amgen products with legacy Horizon products, including Tepeza. So those things we think work in our favor and can benefit us and are levers that Amgen may have wanted to be able to deploy but wouldn't actually have the ability to in this special circumstance.

Yeah, that's helpful. So how do you think about the kind of evolution of the TED market? So we'll talk about sub-queue, but what about like OBI and some of these other low-volume sub-queues that might come out from competitors, but also as you guys already have, phase three data, positive. So you'll be first, but, you know, how should we kind of think about this as not only just replacing maybe IV or switching or whatever, but also kind of the chronic opportunity? So going back to our kind of thinking of lymphoma, maybe expanding that, but sub-Q is probably more the driver there.

Yeah, I agree. Sub-Q is probably the driver. I think there's going to be a, what we look forward to the most, I can say, at Viridian is, as you know, coming out of our sub-Q trial, we had very good proptosis and diplopia results for the Q4 weekly dose regimen. And then we saw really good proptosis results for the Q8 weekly regimen. And so there's a spectrum of patients. As I said earlier, there's a heterogeneity to the disease. So in the moderate to severe patient spectrum, if you've got proptosis but diplopia is not your main concern, Q8 weekly is three administrations and you're done. So you dose on day one, week eight, week 16, and you're done. And you're doing that at home in under 10 seconds. That's a really easy option for people at that end of the spectrum. As you move towards the middle or towards the severe end, Q4 weekly from a sub-Q standpoint, and you've got proptosis and diplopia, now Q4 weekly's got those results that you'd be able to see. And then obviously as you move further into the severe side, IV's available for you. So we love the fact that Viridian is going to have an answer for anybody who walks in the door. And so that suite of products is really important for us as we plan to be the leader in this disease. And so when you talk about the on-body device, not really clear where that fits into that spectrum, just given the fact that it's a device that's about four and a half inches long, it's two inches thick, it's got batteries, gears, a cartridge you have to load, you have to adhere it to your stomach, it's more of an infusion pump, it takes up to 30 minutes to infuse. We haven't really seen any data on it yet. We saw a proptosis number, but we didn't see any details behind any of that. Not clear what's gonna happen there, but the fact that it's dosed, you have to do it every two weeks for 24 weeks, it's arguable that lumbola five infusions is not more convenient than that. And the final thing is it's not clear that that particular on-body device is gonna be at home administration. It's just not clear. We haven't that has not been stated. So, again, we love the fact that we have this suite of products that we expect to have, I should say, available to these patients. And it's not really clear to us where the OBI fits in.

And correct me if I'm wrong. I thought that trial was only an active.

Yeah, it was. So it's not even really addressing the chronic data. Yeah, good point. That's a good point. Yeah.

So, yeah, I think that's interesting. So, and also, I guess, when you think about the chronic opportunity, so I think about a quarter of the sales from Depeza are in chronic, so maybe like 500 million, let's say. Is there an opportunity, like, where do you see the growth? Is it, like, a double from there? Like, is it going to be a billion dollars just in chronic? Like, what do you actually think the expansion opportunity is?

Yeah, we definitely feel that the penetration into the, the chronic population is bigger than the active population. You know, in terms of numbers, we think there's roughly 200,000 people in the U.S. that have moderate to severe thyroid eye disease. As I mentioned, 40,000 of those probably fit into that active phase of the disease. And then so that leaves you roughly 160,000 that are in the chronic population. They may or may not have been treated before. They may have tried to learn how to live with the disease. Their symptoms may wax and wane. and so there's a huge opportunity for us to get in there and that's why we ran those for both IV and for sub-Q. We ran the biggest studies possible or the biggest studies to date, I should say and we think there's opportunity there and we think those patients deserve a chance at both a lighter burden on the IV but then obviously administration at home. So that's definitely penetration. We think there's more penetration to be made in the active population. You know, if we're talking about treating 6,000 patients total, that's still a small percentage of the active population. And that's going to come from having multiple products on the market. It's going to come from disease awareness and the profiles making it easy as possible for people.

So you were talking about, you know, the BLA being submitted first quarter of 27 for LA Grover. What else are kind of the gating factors? Because I know you were waiting for safety, but where's kind of the BLA package currently and what else needs to be done before that?

Well, we put the safety data out with the top-line efficacy data, too. So that all looked in line. That looked like it was supposed to, which is great. And the gating item is the fact we need to finish the study. So that was top-line at 24 weeks. It's a 52-week study. So you have to get last patient, last visit in. But it's the same setup as we had with the IV. We had put the top line out, but then we needed to finish the studies. So we simply need to do that, which is why we think Q1-27 is good guidance for that.

Is it because you need long-term efficacy, or is it long-term safety that's really the requirement?

Yeah, I mean, we'll look at durability data as well. So it'll be – I mean, they're no longer on drug, right? They come off drug at week 21. but we have to finish the safety follow-up and then we'll see their ability data as well.

I mean, what do you think about, you know, do you think you get more of a traditional review there or a priority review? What's your expectation?

Yeah, I think we're going to, we always try to move things faster. So we're going to, we'll give that, we haven't applied for that yet, but we will. And we'll see how that goes. I mean, that's a pretty standard course that we would try. and we'll just have to see how that plays out. But I think, you know, we're ready for any scenario there.

So I want to spend a few minutes on, you know, beyond IGF-1R. So you do, like as you mentioned, have this data coming out for the extended FCRN. What do you want to see here to be competitive? I mean, Argenix is there. They've got a variety of different programs, a couple other extended half-life FCRNs out there. What do you think you need to show to be competitive in kind of this evolving market? and it's always interesting that you were saying so you'll communicate where you want to go like when we get this data. So what are kind of like, you know, what's the phenotype of an indication that you'd want to pursue given that there's a lot of indications already being pursued with FCRNs?

Yeah, so I think from a profile perspective, we need to see competitive levels of IgG suppression. I think there's a range that we've seen. I think there's a deeper is better camp and then there's a threshold camp based on the Argenix data so IgG suppression is part of it we want to see albumin sparing obviously you know we you know I think the field learned from the protocol lab experience in that respect and then it's a matter of indication selection as to where we go from there as Sean pointed out there's there's a lot of translatability from primates to humans and so we would expect to see that when when the data comes in and then we'll decide which indication makes the most sense for these programs and so we

just need to see the data set before we make that decision I mean do you think you need to be greater than one month those things versus like we're kind of our Jackson's with their first program we don't know their second program we haven't seen data yet for that but I guess you know what do you think you need every eight weeks or twelve like what what kind of like what what starts to actually be like oh wow like this is differentiated I mean the IGG reduction As you said, maybe the jury, we were just here talking to Roy Vanna in Argenics this morning, and it's like, again, as you just said, threshold versus greater is better. So we don't know. Maybe we do know, maybe we don't know, but there's two camps. But at least if you show competitive IgG reduction, what sort of profile do you want to have on a dosing side to basically be like, oh, yeah, we can go into MG and win, or we could go into other indications where this would be a big benefit?

Yeah, we've talked about, from the get-go on this program, we've talked about trying to achieve monthly dosing. We think that would shift the paradigm. Patients now are essentially, everyone's doing weekly except for Amavi, which is an IV, right? But we think if we can get monthly, that would certainly help with the patient burden. And again, if we're hitting those IgG levels, if the safety profile looks good from the albumin sparing side of it. Gotcha.

And then just a moment on your TSHR antibodies. So this is an area where we've seen a lot more competitive intensity in early programs coming. So I guess where do you think your program can fit in? And then also kind of the indications, whether it be Graves or Ted, where would you want to focus first?

Yeah, we're really excited about the TSHR program. It's good to see validation as well of others being interested and excited about the same mechanism the Molecule that we have generated is a antagonist of the receptor we've half-life extended that molecule and plan to It has been formulated for subcutaneous delivery and we believe that it will fit into an auto injector So from a profile standpoint, we think this could be the potential best-in-class TSHR inhibitor in terms of infrequent auto injector sub-q dosing being the target we do plan to take this into both TED and Graves from a TED standpoint we believe TSHR can complement IGF-1R exactly how the two will complement each other will depend on the clinical profile for TSHR ultimately the main question is whether TSHR antagonism can rival IGF-1R in terms of efficacy but it's a nice place to be for us to have our own TSHR and then of course there's a natural expansion into Graves disease which also has an overlapping commercial infrastructure with TED so strategically it's an exciting molecule for us to be investing in and bring we look forward to 3Q and the earnings and learning more about the launch but thank you so much for joining us today thank you Derek

Thank you.