Good afternoon, everyone. We'll get started here with the next fireside discussion. My name's Derek Archila, 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.
Sure.
Lot to talk about.
Thanks. Thanks for having us.
Excellent. So maybe it would be a good place to start, just state of the business. You guys are amidst the launch, and you've got a couple other things going on in terms of the pipeline, but maybe just give us that state of the business, and then we can dig into some of the more nuanced questions here.
Yeah. Sure. We are two months into the Lumvoa IV launch, which is great. All systems go there. I am sure we will get into more details as to what we are looking for there. We are preparing our BLA submission for our sub-Q program, elegrobart for thyroid eye disease. We expect to submit that BLA for our sub-Q program, in the first quarter of 2027, so right around the corner now. We have a TSHR program, a TSHR antagonist that we are expecting to submit the IND and get into the clinic. IND will go in this year, Q4. We will get into the clinic early next year. That is an exciting program with applicability in both thyroid eye disease and with Graves' disease, and there is some overlap in those diseases in terms of population, so that is a good complementary part of the portfolio.
We also have FcRn data coming as well, so we have a half-life extended FcRn program that we are working through the first-in-human trials. We are going to collect that data, and we said we would come out and present it or disclose it and give our indication, our clinical development plans going forward, for the half-life extended program. We also have another program which is an Fc fragment, very similar to the VYVGART approach, where we have already characterized that data. That study is complete, so we know that data hit the IgG suppression thresholds that you would 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-in-human, and again, we will have that this year, and then we will be able to decide what to do going forward.
Excellent. That is great.
That is the portfolio in a nutshell.
Awesome. High level. All right. Let's dig into the Lumvoa launch. Obviously top of mind for most folks, but maybe what are we seeing in those first two months? What's the feedback from physicians, and how operationally have you guys executed thus far?
Yeah. Operationally we are doing very well. The teams are doing a great job, and that goes with the 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. Operationally, very exciting to see. Our PDUFA date was June 30. We actually got approval on June 26, which has some advantages in terms of J-code, and 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%-90% of all TEPEZZA scripts that are written today. 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. 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 Lumvoa.
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 30 PDUFA date. Those conversations were productive. Essentially the guidance that we got from payers at that time was for parity pricing. We could expect parity coverage. Just as a reminder, TEPEZZA roughly has 85% of covered lives today. 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. We did guide to parity pricing on our approval call.
Again, we're going to be able to step into that footprint of coverage. That's a second element that we're tracking for launch. Then finally, and obviously critically important, is demand. What kind of physician demand are we seeing? How's that translating into patient demand? We're obviously paying very close to that. What we said in the Q2 earnings release was that based on the patient enrollment forms that we were seeing, that we saw broad and actually deep demand, too. We were seeing physicians not only on the breadth side, but also seeing multiple scripts coming out of them. 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 go from engagement to utilization among these docs? Obviously, you got an entrenched competitor.
Sure.
What is the sales force and the commercial messaging to these high prescribers?
Yeah. There's three key elements. This is the basis of our Breakthrough Therapy designation. We applied based on three elements. As you know, we got Breakthrough Therapy and Priority Review for Lumvoa. The application was based on rapid onset of treatment effect. We were seeing a majority of patients respond on their proptosis. 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. Then there's also an element of diplopia, which is double vision. Can't read, can't drive. It's very difficult to live your daily life with double vision. There's also elements of pain and friction and redness that come in the eye, too.
When we are talking to physicians and they are having their conversations with patients, we are seeing rapid proptosis response after just one infusion. That is an element that we want people to see, a 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 TEPEZZA did not do. They ran theirs as a phase IV. 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. Instead of an improvement, you are actually seeing complete resolution. That had not really been seen before in chronic.
That is another talking point with physicians pointing out that level of data. Lastly, the basis of our application for Breakthrough Therapy designation was the fact that we are five infusions. TEPEZZA has eight infusions. We put in 10 mgs per kg versus 20 mgs per kg. We have a shorter infusion time, the 12-week treatment period versus 21 weeks. When we did market research, the patients would say that they were declining to go on TEPEZZA simply from the standpoint that the treatment burden was just too much, was one of the main elements as to why they would decline. We think we have made that easier, and obviously, I am sure we will get to this, obviously, we think that the subcu will make things even easier from there. We have an answer for every patient that walks in the door.
Got it. No, super helpful. It might be good to illustrate the current process from a patient enrollment form to first infusion. Not only is there friction to write the start form or the enrollment form, and then there is usually the doc writing and then the infusion center doing the infusion. Maybe just remind us how all the dynamics play here.
You want to walk through that?
Yeah, sure. Definitely. Given the profile advantages and the very robust clinical profile that Steve just talked about and 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 are coming in to have a conversation about a treatment option for TED. They get to choose between Lumvoya and potentially TEPEZZA. We feel good about the clinical profile. What actually happens as a patient, if they and their physician chooses Lumvoya, is an enrollment form gets submitted electronically. That is sort of how physicians write scripts today. They are not writing it on a pad of paper anymore.
The enrollment form activates our ViridianCares 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. One of the first things that happens is a benefit verification that a patient has the insurance coverage to be able to support Lumvoya, and connecting that patient then with an infusion center that is convenient for them, getting them scheduled. Then the infusion center will ultimately do the robust prior authorization through the insurance company. Starting out, before we are at a critical mass of coverage at payers, many of these Lumvoya scripts will go through a medical exception process at the payer side.
Again, that is why ViridianCares is so important to have built, and we had that available on day 1 right after we launched so that patients are supported, physicians' offices are supported, and the infusion centers are supported through getting this prior authorization process. TEPEZZA today, just to use as an example, it is also a high-priced biologic, IGF-1R. It takes on average 60-90 days based on our data for a patient to go through that prior authorization process. For us, if we are going through a medical exception process, it can take a little bit longer. 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.
Once a patient is through that prior authorization, then that is 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 just in time for that patient's infusion that is scheduled. There is minimal stocking at the infusion center. 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. This process makes us 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 LINVOYA. Those are the areas that we will be looking at, that we will talk about on the Q3 earnings call in November.
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.
You just kind of answered a lot of the questions that I had, but looking at start forms is going to be the most kind of key indicator of the demand, right? When we get to 3Q, it will be more about kind of those enrollment forms. I guess, it sounds like there is probably, well, maybe you tell me, but how much friction is there to even write a start form? Because it seems like, at least from our checks, that has been fairly easy for what you just said, the ViridianCares. A lot of this has really helped from the patient side and the physician side, but maybe just kind of communicate to us what friction exists in that component, and we can kind of talk about the downstream stuff on the J-code and whatnot. But that is more on the revenue side, not the start form side.
Yeah. I think start forms, enrollment forms are a reasonable indication of demand. We have not said exactly what the demand metric will be once we get to Q3 earnings. We will report what we think is the most meaningful for us as well as for investors. We are obviously tracking a number of different metrics as it comes to demand. In terms of friction, it is really the typical things that are more buy and bill logistics that I kind of walk through. Directionally speaking, those are the kinds of things, getting 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.
Yep.
If the profile of the drug, which we really feel very confident about, and it kind of speaks for itself, that is what will drive the enrollment form. The enrollment form we have 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 an enrollment form for LENVIMA.
Got it. Then maybe on the flip side, with the infusion centers, you guys have been working with these guys to essentially ensure future reimbursement things. So maybe you can kind of talk about things that you did there to just ensure that there is utilization, there's less friction point there as well.
Yeah. We've been talking with those infusion centers for quite a while, and there's national ones where you can get. They have several hundred infusion centers under their umbrella, so we've had access to them. We've explained what was coming. They have experience with TEPEZZA. The key issue that you touched on is they don't want to be underwater.
Right.
They don't want to order drug and front money and be at risk for reimbursement. So it's very simple to cover for that. We just adjust payment terms in that short period until we get that permanent J-code. 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.
Got you. 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 drug's available, things like that.
100%. Our team is very, very experienced in buy and bill, so they know exactly where the pain points are.
Got you.
We were able to submit our J-code 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. We would expect a permanent J-code on January 1.
Excellent. Perfect. I guess when you think about the market here, TEPEZZA has been on the market for a number of years. U.S. market kind of like maybe stagnating a little bit. I guess, do you think there will be maybe a resurgence of growth overall? Are there other patients to pull in, at least active and would love to get your thoughts on chronic with IV. I do not know, how do you think the overall TED market grows potentially with Lumvoa in the market? The follow on to that is just with the physicians now with two choices to make, and there is some differentiation here.
Is there a possibility of just them, 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 will have to see how that plays out, and we are still two months in, so let us see how that plays out as this zero sum. I am not sure I would expect zero sum, but I think that we will certainly be watching for that to understand. To your first question as to whether there will 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 is a lot of patient education going on out there between us and Amgen to try to increase disease awareness, increase diagnosis rates. But just a reminder, our commercial strategy, particularly in this early days, is to convert. If someone is writing a TEPEZZA script, we want to be in front of them. We want to make sure that they are aware of the Lumvoa label.
They already know the mechanism. It is IGF-1R inhibition. There is some level of comfort already by virtue of them writing TEPEZZA scripts. That is our first and foremost. Of the 6,000 patients roughly 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. I think we would expect a similar patient mix coming out because of our conversion strategy.
with those TEPEZZA script writers. But again, we're still early days.
Yeah
and we'll see how it plays out.
Does that leave, I don't know, chronic TED with Lumvoa as kind of an underappreciated opportunity just because you have the most robust data set with chronic, right?
Right.
I think one of the things that I would say with TEPEZZA and maybe why they have so few patients with chronic is the data is not It was like a phase IV trial. It wasn't really as robust. Does that I don't know. Is that a message that you guys are trying to send?
Certainly. We have chronic data in our label. We ran, as you said, a robust study where Clinical Activity Score is on a scale of zero to seven. 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 you'll see some chronic patients that have proptosis. They may not have diplopia, or they may have both. They may have an active-like flare in that chronic population just by virtue of the fact 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 we think that the Lumvoa 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 dataset 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. I guess, looking forward, and again, the launch is 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 think we really love the dataset that we have for Lumvoa in both the active population and the chronic population, again, across the key metrics, which is proptosis and diplopia. And so yeah, we do think that we have a great drug for these patients. We think we, by virtue of five infusions and 10 mgs 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. 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 under-penetrated market. This is single-digit penetration of the market. We need to bring people in off the sidelines, and we think the Lumvoa profile, and then when we get there, the sub-Q profile, we think that the combination of those is going to be able to do that.
Got you. It's still early in the launch, but what's been the Amgen counter detail? Are you seeing anything, or what are we learning so far about how they're responding to the new entrant?
Yeah. I would say there was a new commercial.
Yeah
It comes on.
Okay.
There's a new commercial, and that commercial references that TEPEZZA's been around for a while.
And that physicians and patients should take comfort from the fact that it's been available, which, understandable as a counter detail, but I think that's certainly not something that overly concerns us. Again, 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's-
There are a couple of things here, too, that TEPEZZA, or Amgen, I should say, would typically be able to do that they're not able to do. Because the prescribing physicians are not the ones who are doing the infusion, there's 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 TEPEZZA. 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. No, that's helpful. I guess, how do you think about the kind of evolution of the TED market? We'll talk about sub-Q, but what about OBI and some of these other low-volume sub-Qs that might come out from competitors, but also as you guys already have phase III data positive, so you'll be first. But how should we think about this as not only just replacing maybe IV or switching or whatever, but also the chronic opportunity? Going back to our thinking of Lumvoa and maybe expanding that, but sub-Q's probably more the driver there.
Yeah, agree sub-Q's 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. 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'll be able to see. Then obviously as you move further into the severe side, IV is available for you. So we love the fact that Viridian is going to have an answer for anybody who walks in the door. That suite of products is really important for us as we plan to be the leader in this disease. 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 4.5 in long, it's 2 in 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 going to happen there. But the fact that you have to do it every two weeks for 24 weeks, it's arguable that LENVIMA five infusions is not more convenient than that. The final thing is it's not clear that particular on-body device is going to be at-home administration. It's just not clear. 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. It's just not really clear to us where the OBI fits in.
Got it. Correct me if I'm wrong, I thought that trial was only in active.
Yeah. It was only in active.
It's not even really addressing-
No cross data there either.
the chronic data.
Yeah, good point. That's a good point.
Yeah. I think that's interesting. And also, I guess when you think about the chronic opportunity, I think about a quarter of the sales from TEPEZZA in chronic, something like $500 million, let's say. Is there an opportunity? Where do you see the growth? Is it like a double from there? Is it going to be a billion dollars just in chronic? What do you actually think the expansion opportunity is?
Yeah. We definitely feel that the chronic population is bigger than the active population.
Yeah.
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. 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. 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. 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. That's definitely penetration.
We think there's more penetration to be made in the active population. If we're talking about treating 6,000 patients total, that's still a small percentage of the active population. 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.
Got it. You were talking about the BLA being submitted first quarter of 2027 for elegrobart. What else are the gating factors? I know you were waiting for safety, but where's 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. That all looked in line. That looked like it was supposed to, which is great. The gating item is the fact we need to finish the study. That was top line at 24 weeks. It's a 52-week study, so you have to get last patient, last visit in. 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. We simply need to do that, which is why we think Q1 2027 is good guidance for that.
Okay. Is it because of that you need long-term efficacy, or is it long-term safety that is really the requirement?
Yeah. We will look at durability data as well.
Okay.
They are no longer on drug, right?
Yeah.
They come off drug at week 21. But we have to finish the safety follow-up, and then we will see durability data as well.
Got you. Understood. What do you think about, do you think you get more of a traditional review there or a Priority Review? What's your expectation?
Yeah, we always try to move things faster. We haven't applied for that yet, but we will. We'll see how that goes. That's pretty standard course that we would try. We'll just have to see how that plays out. But I think we're ready for any scenario there.
Got you. I want to spend a few minutes on beyond IGF-1R. 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? argenx 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 this evolving market? It's always interesting that you were saying, you'll communicate where you want to go, like when we get this data.
Right.
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. 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 the threshold camp.
Yeah
based on the argenx data. IgG suppression is part of it. We want to see albumin sparing, obviously.
Yeah.
I think the field learned from the batoclimab experience in that respect. Then it's a matter of indication selection as to where we go from there. As Shan pointed out, there's a lot of translatability from primates to humans. We would expect to see that when the data comes in, and then we'll decide which indication makes the most sense for these programs. We just need to see the data set before we make that decision.
Do you think you need to be greater than one-month dosing versus like where kind of argenx is with their first program? We don't know their second program. We haven't seen data yet for that. I guess, do you think you need every eight weeks or every 12? What starts to actually be like, "Oh, wow, this is differentiated"? I mean, the IgG reduction, as you said, maybe the jury, we were just here talking to Roy van Annen, argenx this morning, and it's like, again, as you just said, threshold versus greater is better. 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. 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 IMAAVY, which is an IV, right?
But we think if we can get monthly, that would certainly help with the patient burden. Again, if we're hitting those IgG levels, if the safety profile looks good from the albumin sparing side of it.
Got you. Then just a moment on your TSHR antibodies. This is an area where we've seen a lot more competitive intensity in early programs coming. I guess, where do you think your program can fit in? Then also, kind of the indications, whether it be Graves' or TED, where would you want to focus first?
Yeah. We are really excited about the TSHR program. It is good to see validation as well of others being interested and excited about the same mechanism. The molecule that we have generated is an antagonist of the receptor. We have 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 subcu dosing being the target. We do plan to take this into both TED and Graves'. From a TED standpoint, we believe TSHR can complement IGF1R. 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 IGF1R in terms of efficacy.
It is a nice place to be for us to have our own TSHR. Then, of course, there is a natural expansion into Graves' disease, which also has overlapping commercial infrastructure with TED. Strategically, it is an exciting molecule for us to be investing in and bring.
Got it. Excellent. We look forward to 3Q and the earnings and learning more about the launch. Thank you so much for joining us today.
Yeah. Great to see you.
Got you.
Thanks for having us.
Thank you, Derek.
Appreciate it. Good to see you.
Thank you.