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Goldman Sachs 45th Annual Global Healthcare Conference

Jun 11, 2024

Moderator

All right, good morning. So for our next session, I am excited to be hosting Steve and Shan from Viridian. Steve Mahoney, CEO, and Shan Wu, CBO. And welcome, and great to have you guys here. So first of all, congrats on the positive Type C meeting that you guys announced this morning, and finalizing phase 3 program design for VRDN-003, which is now called REVEAL, the program. So we saw the PR this morning, and there's a lot of exciting details there that we want to get into. Before diving into our questions, maybe I'll turn the stage to you guys to give an opening remark, and then discuss the journey so far, and then we'll jump into the questions.

Steve Mahoney
CEO, Viridian Therapeutics

Sure. Thanks, Rich. Yeah, very excited to release this morning the news about our Phase 3s both in active and chronic TED, with our subcutaneous program, long-awaited, and so very happy to be moving forward. Yeah. That is an exciting part of our overall thyroid eye disease approach. As you know, we have Phase 3 readouts this year. We have top-line readout for our active study in September, and we have a top-line readout for our chronic study at the end of the year. So really exciting year for us. We're, as we indicated in our press release this morning, we'll initiate the Phase 3s for active and chronic, simultaneously in August.

And we expect to have top-line data in early 2026, first half of 2026, and then followed by a BLA filing at the end of 2026. So we're moving rapidly, we're executing, we've got an exciting thyroid eye disease portfolio. We also have a FcRn portfolio, anti-FcRn portfolio, which we have announced back in October. And we have an IND for our first program there, which is 006, which is a Fc fragment approach, which, as you know, from argenx and VYVGART, that is a great place to be. We are the only other that we know of in clinical development with a Fc fragment. So exciting for us there.

And then we also announced that we have a program that we refer to as 008 , which is a half-life extension approach for anti-FcRns-

Moderator

Mm-hmm.

Steve Mahoney
CEO, Viridian Therapeutics

which would be, in our view, a game changer.

Moderator

Mm-hmm.

Steve Mahoney
CEO, Viridian Therapeutics

So really exciting portfolio as it's advancing. We'll have NHP data for the half-life extension in the second half of this year. So moving ahead.

Moderator

Sounds good. So before diving into the phase 3 program for the subcutaneous agent, why don't we start with a couple general questions? So both of you guys joined Viridian in late 2023. How has that journey been for you so far? What was the state of the company when you guys just joined? What was working, what was not working, and what changes did you guys have to implement in the company, and where is the company now?

Steve Mahoney
CEO, Viridian Therapeutics

Yeah, I think operationally, the company was in good shape. I think we were able to come in and reiterate the timelines that the company had already set. We unveiled the FCRN portfolio, which the company had actually been working on for a few years prior to that, and just that was the original disclosure when we came on board. The team at Viridian is very good. We're adding and building that out with a lot of really great people who are working hard. And as you can tell from our track record of execution, we've delivered our subQ data on time in December, even though we had just started essentially in November.

That was a great win because we were able to bring the subQ, the 003 subQ program forward, which was exciting for us. We enrolled the THRIVE study, the active form for the IV study. We enrolled that on time, and in fact, exceeded enrollment, so which speaks to execution.

Moderator

Mm-hmm.

Steve Mahoney
CEO, Viridian Therapeutics

Then we just announced this morning, as you mentioned, the subQ program trial design, which was a successful execution with our interactions with FDA. So everything was. From that standpoint, we came in and we have a team that executes, so that's all really positive.

Moderator

Fantastic. So the stock has been traded down since the beginning of 2024. What has been the biggest pushbacks that you guys have been hearing from investors, and what are investors missing?

Steve Mahoney
CEO, Viridian Therapeutics

Well, I think, there is pushback that the investors are watching Amgen sales of TEPEZZA. You know, we think there's a number of factors there. We're very, very confident that they'll course correct that. I think they're in the process of doing that. They did just close the acquisition in October, so work to be done there. I'm sure they have acknowledged on their calls that they're in the single digit penetration of the market. We agree. As I mentioned, we enrolled our active study, half of which was in the U.S. We exceeded enrollment. That speaks to market demand that remains out there. There's opportunities to grow the revenue side of it for all the IGF-1Rs with respect to TEPEZZA and us.

When you look at, they filed in Europe, they filed in Japan, we'll do the same. And subQ is a game changer.

Moderator

Right.

Steve Mahoney
CEO, Viridian Therapeutics

SubQ comes in, it'll increase penetration in the market, simply from the standpoint of more convenience for patients and better access. We can mail an auto injector to your house as opposed to, you know, having to go to an infusion center.

Moderator

Right.

Steve Mahoney
CEO, Viridian Therapeutics

You know, people often forget that not everybody lives near an infusion center. So this will really broaden out the market. So we think there's plenty of room to grow in both. We think TEPEZZA sales will increase. We also think that when we get there, we'll be able to penetrate even further.

Moderator

I see. Got it. So just looking at the U.S. market, right? So their sales was, like, $1.8 billion in 2023, which was a little down when you look at just the last two quarters compared to the year before. How do you think that the U.S. market, forget about the ex-U.S. and expansion there-

Steve Mahoney
CEO, Viridian Therapeutics

Mm-hmm.

Moderator

Just the U.S. market, how do you think that's gonna grow, and where do you think it's gonna be at, at steady state?

Steve Mahoney
CEO, Viridian Therapeutics

Yeah, well, I mean, I think I would remind everybody that it's still a $1.8 -2 billion market. The U.S. is a pretty healthy market. Even if you assume they're never gonna grow it, that's still a good, healthy market. And this is a new start market. This is where, you know, it, you... It's not a situation where a patient gets put on therapy, and they stay on therapy forever. And so we're not in a position where we have to convince people to switch. In a new start market, you have, it's really symptoms driven, whether you're an active form of disease or whether you're in chronic form of disease, you present at the physician's office with symptoms.

Moderate to severe TED patients or thyroid eye disease patients have severe proptosis, means bulging of the eyes, redness, pain in the eye, and diplopia, which is double vision, which impairs their ability to read, write, drive, work. Quality of life can be really difficult for moderate to severe TED patients. So we have the ability with our IGF-1 program to address those, and there's plenty of patients out there, given the single-digit penetration of the market by TEPEZZA. And as evidenced by our enrollment in our trial, we think there's plenty of demand there to be had. As a reminder, TEPEZZA did not run a chronic study in their registration study.

Moderator

Mm-hmm. Mm.

Steve Mahoney
CEO, Viridian Therapeutics

So they didn't generate the data for payers, and they went back and did it later.

Moderator

Went back, yeah.

Steve Mahoney
CEO, Viridian Therapeutics

On a post-approval basis. We are doing that in our original study.

Moderator

Right, right.

Steve Mahoney
CEO, Viridian Therapeutics

So we'll generate that data upfront, and so we expect to be able to piggyback off of their efforts there too. So I think it's actually the market set up very nicely for us to come in and expand it.

Moderator

Mm-hmm. Fantastic. So why don't we move into the Phase 3 design for 003 that you guys announced this morning? Perhaps you guys, you have two studies going on there, and they look similar to 001 in some ways. And why don't you guys go over some of the highlights from the design that you guys for both trials that you guys announced?

Steve Mahoney
CEO, Viridian Therapeutics

Yeah, sure. Maybe Shan can take that.

Shan Wu
Chief Business Officer, Viridian Therapeutics

Sure, I can start. Yeah, we're really excited to announce the details of the two phase 3 programs, which will form the crux of the pivotal programs for our subcutaneous 003 program. It's two studies, REVEAL-1 and REVEAL-2 , in active and chronic TED. Very similar to what we have done and are doing for 001 . What we have decided to do is take into each of those trials two active dose regimens every four weeks and every eight weeks.

A reminder that VRDN-003 is a half-life extended molecule against IGF-1R, shares the same binding domain as VRDN-001, which when we disclosed the data back in December, we showed that with that PK profile, that VRDN-003 can, based on PK modeling, can achieve or even exceed levels of VRDN-001, that were shown to be clinically, really clinically robust and active in a phase 2 study. So we feel great about the potential efficacy of both of these arms, and of course, Q4 weekly with an extended half-life antibody that is a at-home auto-injector, self-administered, would be a transformative profile for patients, and then Q8 weekly would be an even better profile.

Moderator

Mm-hmm.

Shan Wu
Chief Business Officer, Viridian Therapeutics

And so we're really excited about taking those two, dosing arms forward. Top-line data expected in the first half of 2026, with a BLA filing by the end of 2026, which we believe puts us ahead of, the other sub Q. So, also very fast, development here that we're excited about bringing best-in-class and first to, market sub Q.

Moderator

Mm-hmm. Fantastic. So 003 has only been tested in healthy volunteers, so not in patients so far. So what gave you guys the confidence to go into a pivotal study straight and then using both the Q4W and the Q8W?

Steve Mahoney
CEO, Viridian Therapeutics

Yeah, well, I think the healthy volunteer study just helped us identify our doses. We didn't need to go into a phase 2 to figure out what our dosing regimen should look like. As Shan just mentioned, 003 and 001 have the same binding domain.

Moderator

Mm-hmm.

Steve Mahoney
CEO, Viridian Therapeutics

CDR. So, the only difference being the half-life extension. So we feel really confident about, obviously, the mechanism of action, but also the data from the phase 1 healthy volunteers, and the PK/PD modeling showed us what our doses should be.

Moderator

Right.

Steve Mahoney
CEO, Viridian Therapeutics

There's no need to go through a phase, the traditional phase 2. We can jump right into phase 3.

Moderator

I see. Got it. So when I look into the exposure levels, right? Looking at the Q4W, Q8W, it looks equivalent to the 3 mg per kg and 10 mg per kg from the IV.

Steve Mahoney
CEO, Viridian Therapeutics

Mm-hmm.

Moderator

So why, why choose a 24-week primary endpoint? And I think for IV, it's 15 weeks. Is it possible to do Q4W, Q8W, say, at 16 weeks instead of 24 weeks?

Shan Wu
Chief Business Officer, Viridian Therapeutics

... Yeah, it's a great question. You know, firstly, the subQ is just a different molecule. So we're not trying to compare to the IVs anymore. The IVs with our VRDN-001 IV doses is designed to be maximally differentiated versus TEPEZZA IV. But that's a different game. To keep going with that analogy, the VRDN-003 subQ with the half-life extension and potential best-in-class profile is a game changer for these patients. And so with that backdrop, and just the next example, the every eight-week dosing regimen would be three total administrations, one upfront, one at eight weeks, and one at 16 weeks, and the Q4 weekly would be six administrations. That's a really fantastic dosing regimen for patients.

Why wouldn't we preserve the efficacy of IGF-1R and go out to 24 weeks when it's already maximally convenient and a great profile for a patient?

Moderator

Hmm, okay.

Steve Mahoney
CEO, Viridian Therapeutics

Essentially, we've established the convenience, and now we can just match those exposures to bring the efficacy.

Moderator

Right. Right. Okay, got it. So you guys also announced part of the plan that you guys will have an auto-injector. So, so far, you guys are the only company that actually gave such specific details and commitment to that auto-injector. So where are you now with that auto-injector development, with the pairing of the 003? And when should we expect that pairing to go into the study, into clinic?

Steve Mahoney
CEO, Viridian Therapeutics

Yeah, we haven't really gotten into those details-

Moderator

Yeah.

Steve Mahoney
CEO, Viridian Therapeutics

-in terms of disclosing that. Yeah, as you know, devices have their own timeline-

Moderator

Yeah

Steve Mahoney
CEO, Viridian Therapeutics

That runs in parallel with drug development. So we and as you mentioned, we have an auto-injector that's commercially available. It's so we're, we have the device that's available, and now we just need to do the testing to get our drug into it. And that runs in parallel until we can merge the two, and we just, we haven't. And we've got plans to do that.

Moderator

Right. I see. Got it.

Steve Mahoney
CEO, Viridian Therapeutics

Our launch will be in the auto-injector.

Moderator

Got it.

Steve Mahoney
CEO, Viridian Therapeutics

That's our plan.

Moderator

Right. So the study will be in the prefilled syringe, and then at some point,

Steve Mahoney
CEO, Viridian Therapeutics

Yeah, we'll, we'll start vial syringe, but then we have a, we have plans to introduce it in a normal, typical fashion-

Moderator

Right

Steve Mahoney
CEO, Viridian Therapeutics

... how you introduce a device into these types of studies.

Moderator

Right. Which are usually at-

Steve Mahoney
CEO, Viridian Therapeutics

Nothing unusual.

Moderator

Small, small studies.

Steve Mahoney
CEO, Viridian Therapeutics

Bridging studies, whatever it is.

Moderator

Yeah.

Steve Mahoney
CEO, Viridian Therapeutics

We haven't disclosed exactly what it is, but, again, pretty typical-

Moderator

I see

Steve Mahoney
CEO, Viridian Therapeutics

in terms of how to introduce a device.

Moderator

I see. Got it. How do you define a successful VRDN-003 in respect to VRDN-001 in terms of safety and efficacy? Do you think it has to show similar efficacy and safety to VRDN-001 or TEPEZZA? If there's a drop in efficacy compared to VRDN-001, is there still value in VRDN-003?

Steve Mahoney
CEO, Viridian Therapeutics

Yeah, that's a hypothetical, right?

Moderator

Yeah.

Steve Mahoney
CEO, Viridian Therapeutics

I don't know what you mean by a drop in efficacy because it could be offset by something else, right?

Moderator

Yeah.

Steve Mahoney
CEO, Viridian Therapeutics

So, so hard to answer hypotheticals, but I think you nailed it in the sense that, a similar efficacy and safety profile would be a great place to start. Simply because for moderate to severe patients, they need that IGF-I treatment effect size. We have, as Shan mentioned, we have the convenience lockdown with the subQ. And now if we can just, if we can match that, that efficacy treatment effect size, that would be fantastic. So that, that's what we're aiming for.

Shan Wu
Chief Business Officer, Viridian Therapeutics

I'll add to that. With the subQ and dosing at lower exposure levels than the IV, but exposure levels that have already been shown to be to have clinical activity, we uniquely are in a position to be able to test this hypothesis that lower exposure levels can preserve the IGF-I efficacy, because we know that's really important for moderate to severe patients, but potentially make the profile, the safety profile, even better than the IV versions.

Moderator

I see. Got it. So basically, efficacy expectation is to be comparable to the 001, the IV, and then safety, perhaps a better, slightly better safety profile.

Steve Mahoney
CEO, Viridian Therapeutics

Well, we'll see. I mean, I think that to the extent... I think Shan's point is right. I think to the extent lower exposures-

Moderator

Yep

Steve Mahoney
CEO, Viridian Therapeutics

translates to a better safety profile.

Moderator

Yeah

Steve Mahoney
CEO, Viridian Therapeutics

Fantastic. We're in a great place to see that unfold.

Moderator

Right.

Steve Mahoney
CEO, Viridian Therapeutics

Obviously, the difference between subQ and IV would be Cmax, right?

Moderator

Right.

Steve Mahoney
CEO, Viridian Therapeutics

The Cmax in an IV is a lot higher than subQ. It's smoothed out quite a bit. We're not hanging our hat on Cmax as a driver here, but to the extent it plays a role, then we're in a great place to see that, too.

Moderator

Okay, sounds good. Okay, so the clinical study, is that gonna be an IGF-1 naive patients, similar to 001's pivotal study?

Steve Mahoney
CEO, Viridian Therapeutics

Yeah, it's very similar-

Moderator

Okay

Steve Mahoney
CEO, Viridian Therapeutics

... in the approach, yes.

Moderator

Is there... Do you guys plan to do an IGF-1R experience patient population at some point?

Steve Mahoney
CEO, Viridian Therapeutics

Oh, for retreatment.

Moderator

For retreatment, yeah.

Steve Mahoney
CEO, Viridian Therapeutics

Yeah. Retreatment is a great opportunity, commercially, certainly. I think we're an opportunity.

Moderator

Okay, fantastic. So what 001. There's data coming out, there's pivotal studies coming out. Only a couple of months away, so it's very exciting-

Steve Mahoney
CEO, Viridian Therapeutics

Yeah, very exciting.

Moderator

Time for 001. And there's a lot of hot topics to talk about, so why don't we switch gears? Okay, can you talk about the phase 2 data that you guys generated and how that compares to TEPEZZA's phase three in your view?

Steve Mahoney
CEO, Viridian Therapeutics

Yeah, maybe I'll start, and Shan can jump in. But I think, I think that's what got people really excited about Viridian, is when that phase 2 data came out. And we showed across the relevant endpoints, which is proptosis, CAS score, and diplopia. We saw after two infusions at the week six time point, we showed that we were as good, if not, in some cases, significantly better, than TEPEZZA at that time point. So that was really encouraging. A good proof of concept study, and that allowed us to, you know, really gave us the confidence to move forward into these phase 3 studies.

Shan Wu
Chief Business Officer, Viridian Therapeutics

That's exactly right.

Moderator

Right. So in the two patient populations, the active and chronic, we saw some pretty high placebo effects there. And so besides running a larger pivotal study program, is there anything else that you guys can do to mitigate the potential of these high placebo effects?

Shan Wu
Chief Business Officer, Viridian Therapeutics

Yeah, I think the high placebo effects in the phase 2 is largely attributed to the small size, and the way that proptosis is measured is by what's known as a Hertel exophthalmometer, which is an eye ruler, and the trial...

Moderator

Yeah

Shan Wu
Chief Business Officer, Viridian Therapeutics

- conductors are measuring millimeters of changes in proptosis, and that those small sizes, especially in a placebo arm-

Moderator

Mm-hmm

Shan Wu
Chief Business Officer, Viridian Therapeutics

The noise is quite big. So we have certainly powered our phase 3s appropriately for that. So I think that's a great place to be, and it's a metric that is cross trials, of course, but will be comparable-

Moderator

Right.

Shan Wu
Chief Business Officer, Viridian Therapeutics

- back to TEPEZZA.

Moderator

I see. And have you guys reviewed the powering assumptions for both of these studies, for VRDN-001 and VRDN-003?

Steve Mahoney
CEO, Viridian Therapeutics

No, we have not.

Moderator

I see.

Steve Mahoney
CEO, Viridian Therapeutics

Yeah. We have not disclosed the powering assumptions, but, you know, what we've done is, to Sean's point, we've powered it sufficiently-

Moderator

Right

Steve Mahoney
CEO, Viridian Therapeutics

... to overcome those small n's that were in the phase 2.

Moderator

I see.

Steve Mahoney
CEO, Viridian Therapeutics

Which is just a little bit noisy, but they're certainly directional, so we again, we feel good.

Moderator

I see. Okay. So looking at the active versus the chronic population, you see a lower response in terms of the chronic group, consistently across yours and TEPEZZA's trials. Does this suggest a lower unmet need for IGF-1R biologics in these patients?

Steve Mahoney
CEO, Viridian Therapeutics

No. Go ahead.

Shan Wu
Chief Business Officer, Viridian Therapeutics

Yeah, no, we don't think so. So there may be something that's a bit more different about the chronic patients from their response, but in terms of the symptoms that they have, they are still moderate to severe with proptosis, redness, inflammation, swelling, which is measured by the clinical activity score-

Moderator

Okay

Shan Wu
Chief Business Officer, Viridian Therapeutics

... and also diplopia. So these are still patients that have an urgency to treat, if not more, because they are the ones who have, experienced that initial, when they were first diagnosed, flare-up of symptoms, gotten over it, knew how debilitating that was. And what we hear is the patients who are chronic, because they've been diagnosed a long time ago, when they see those symptoms flare up again, they actually want to treat that even more because they remember how, terrible and debilitating the symptoms were. So no, we think these patients, there's still a very high unmet need in the moderate to severe type patients.

Moderator

Okay, got it. So you guys are differentiating VRDN-001 from TEPEZZA based on the shorter treatment course of five doses compared to TEPEZZA's eight doses. So your treatment course, the duration 16 weeks, compared to TEPEZZA's 24 weeks. How do you guys decide on that five doses versus, say, like four or six?

Steve Mahoney
CEO, Viridian Therapeutics

Well, I think, it's worth to look at the. When we were looking at it, TEPEZZA had its placebo-adjusted Phase 3 results, and proptosis showed pretty clearly that they were maxing out the benefit at around the fifth dose. The incremental benefit of doses six, seven, and eight with TEPEZZA is certainly not clear. And as a reminder here, TEPEZZA did not do any dose ranging when it introduced the drug. And so, to Sean's earlier point, we're really the first ones that are gonna be able to explore the therapeutic index here. And we think with our full antagonist in five doses, we will match their eight doses in terms on both efficacy and safety, and/or be similar to.

I think that's just a function of looking at TEPEZZA and the gaps in that development. It's a great drug, no criticism of the development, other than the fact that it presents a real good opportunity for us.

Moderator

Right

Steve Mahoney
CEO, Viridian Therapeutics

... given the fact that they didn't do any dose ranging. And I think it's pretty clear that if you have unnecessary doses, you know, why would you give them? And so I think we can come in, and again, in the new start market, this is, like, the critical piece, is that because it's symptom driven, physicians will... Patients will come in to see their physicians, they'll have a choice to make when our IV is on the market.

Forget subQ for a second, but if you, if you have IV versus IV, and you've got our five doses versus their eight doses, 10 mg per kg versus 20 mg per kg, 30-minute infusion time versus 60-90 minutes, and all of that plays into we deliver about a third of the drug, two-thirds less drug for the same outcomes. And if, if we have a similar profile, I think that's a no-brainer in terms of when you come in and you have choices to make in a new start market. So that's why we feel really good about where we're situated.

Moderator

I see. Okay. So, why don't you expect 001 to differentiate in, say, like, safety or maybe efficacy? Like, for example, do you see any chance of faster onset of action or something like that, or?

Steve Mahoney
CEO, Viridian Therapeutics

Yeah, sure. I think there, there's upsides on either side.

Moderator

Yeah.

Steve Mahoney
CEO, Viridian Therapeutics

But I think our base expectations in a great place to be. I mean, TEPEZZA has great outcomes for patients, both on the efficacy and the safety. I mean, I think, and so if we can be a similar profile, like I said, in that new start market dynamic, that's a great place for us to land. So yeah.

Moderator

I see. So how compelling is this competitive advantage of the five-dose treatment course? Why can't TEPEZZA just, like, mimic that five-dose in a competitive setting?

Steve Mahoney
CEO, Viridian Therapeutics

Yeah, well-

Moderator

In response to when you guys commercialize 001 .

Steve Mahoney
CEO, Viridian Therapeutics

Sure. I think that's, I think that sounds good in theory. I think that's difficult practically from the standpoint that that's their label, and that is also how they're priced. So it gets. That's gonna be difficult for them to be able to do. The vast, vast majority of physicians refer to the label. And so I think, you know, that I think competitively, we are in a good position to make the argument that we're still, you know, fewer doses, less drugs, for a similar outcome.

Moderator

I see.

Shan Wu
Chief Business Officer, Viridian Therapeutics

It's also a different molecule, so they're a partial antagonist-

Moderator

Yeah.

Shan Wu
Chief Business Officer, Viridian Therapeutics

- and we're a full antagonist. And so, when if and when we get to the point we hope to be, where our label is clearly for five infusions, the outside of the KOL community, the more general prescribers of TEPEZZA will want to go by label, and that's what we understand-

Moderator

Mm-hmm.

Shan Wu
Chief Business Officer, Viridian Therapeutics

is happening in the marketplace is right now, that the majority of these patients are completing the full 8 doses of TEPEZZA.

Steve Mahoney
CEO, Viridian Therapeutics

Right.

Moderator

I see.

Steve Mahoney
CEO, Viridian Therapeutics

Because they're different molecules, there's no clinical evidence to suggest that their five doses would... You know, in terms of, there's just not enough evidence out there to suggest that there would—their five doses would match our five doses.

Moderator

Right. Okay, got it. So among the chronic patients, it's estimated that small subset of these patients experience these inflammatory flares. How are you thinking about that overall opportunity for chronic patients?

Steve Mahoney
CEO, Viridian Therapeutics

Well, the chronic patients, again, as TEPEZZA's generating the data, has generated the data, and we, in our chronic study, we showed that, you know, these are patients that still have these symptoms of proptosis and pain, and inflammation, and redness, as well as diplopia. So there's certainly a market there to address those symptoms, and that's really what it boils down to, is: Can you address the symptoms for people with moderate to severe symptoms?

Moderator

I see. Okay, so, you guys plan to file the BLA not until late 2025? What are all the activities that you need to do to complete before then?

Steve Mahoney
CEO, Viridian Therapeutics

Yeah, I mean, it really boils down to, and we've said this before, it really boils down to just the follow-up period for THRIVE-2.

Moderator

Yeah.

Steve Mahoney
CEO, Viridian Therapeutics

So the chronic study, we have year-end top-line data this year, and then we just need to complete the follow-up period. We do have a safety study that's ongoing as well-

Moderator

Right.

Steve Mahoney
CEO, Viridian Therapeutics

That fits well within the THRIVE-2 timelines. It's really driven by THRIVE-2 at this point.

Moderator

I see.

Steve Mahoney
CEO, Viridian Therapeutics

And then we'll file as soon as we can after that.

Moderator

Right. Is there any chance to file ahead of that?

Steve Mahoney
CEO, Viridian Therapeutics

Yeah, we're looking at options.

Moderator

We-

Steve Mahoney
CEO, Viridian Therapeutics

Yeah, we're looking at different options as to how we can do that, and we'll engage. But, you know, we have to just work through the regulators on that in terms of-

Moderator

Yeah

Steve Mahoney
CEO, Viridian Therapeutics

... you know, whether it can be done on a rolling basis or not, but we're looking at everything.

Moderator

Right. I see. Got it. So you guys are using both the MRI and the, the EXO for, for your, to measure the proptosis response at week 15. What's the purpose of using these two measurements, and what happens if these two measurements sort of... If they're different from each other, does it present any risk to the, to the FDA, regarding how the reliability of these results?

Steve Mahoney
CEO, Viridian Therapeutics

Yeah, I mean, I think, you know, Hertel has been Hertel, the EXO-

Moderator

Yeah

Steve Mahoney
CEO, Viridian Therapeutics

... has been traditionally used in this space.

Moderator

Mm-hmm.

Steve Mahoney
CEO, Viridian Therapeutics

I think it's well known that there's some variability in there.

Moderator

Yeah.

Steve Mahoney
CEO, Viridian Therapeutics

And which is why, you know, you saw our phase 2 when we had the Hertel or the EXO measurement. But the small numbers makes it a little bit noisy, so that's why we power the studies. MRI certainly is a more objective-

Moderator

Mm-hmm

Steve Mahoney
CEO, Viridian Therapeutics

... because the EXO is basically an eye ruler-

Moderator

Yeah

Steve Mahoney
CEO, Viridian Therapeutics

- that people, you, the physician holds up against your head.

Moderator

Right. Right.

Steve Mahoney
CEO, Viridian Therapeutics

So there's a lot of variability that can come with that type of approach.

Moderator

Okay.

Steve Mahoney
CEO, Viridian Therapeutics

and MRI is just a more objective way. So we want to collect both sets of data and just see, and see what that looks like. MRI certainly could end up being, you know, something that just gives people a better sense of what the-

Moderator

Right

Steve Mahoney
CEO, Viridian Therapeutics

... true proptosis response rate looks like.

Moderator

I see. But if those two results are not consistent, would that present a risk, regulatory risk?

Shan Wu
Chief Business Officer, Viridian Therapeutics

We've seen so far that they're consistent with each other. You know, the MRI does have better precision, but would generally say that the two methods should be showing directionally the same results. So we wouldn't expect that to be different.

Moderator

I see. Okay. So we have a couple more minutes left. If the audience have any questions, please raise your hand and we'll come to you with a microphone. So, with that said, why don't we keep going? Okay, so what is the long-term aspiration for Viridian? What would the company look like 10 years from now?

Steve Mahoney
CEO, Viridian Therapeutics

I think it's the long-term prospects are fantastic. I mean, we're building a great team. We're executing. We have multiple product candidates that we're moving through clinical, late stage and early stage. So we've got a whole portfolio in autoimmune, including thyroid eye disease, but then with the FcRn, the whole host of autoimmune. So we have an opportunity to, on a de-risked opportunity because we're, you know, going after clinically validated targets that we'll have multiple approved products, commercial products, and we'll just keep looking to grow the company in that way. So I'm we're really excited about what we're getting started with.

Moderator

I see. And how do you see that longer-term sort of commercial strategy or how VRDN-001 and VRDN-003 is gonna play out in the market? Like, how do you see these two products, sort of like from a market share perspective, how they will roll out, and what's your strategy there?

Steve Mahoney
CEO, Viridian Therapeutics

Yeah, I mean, we certainly recognize that the subQ, with the potential best-in-class offering, whether it's, you know, Q4 weekly or Q8 weekly, the convenience for patients, again, an auto-injector delivered to your home, you can self-administer, you don't have to go to an infusion center. We certainly understand that that is the big opportunity for thyroid eye disease, could change the landscape. But we still think there's a spot for IV. I mean, there are... You know, other products have shown when they do IV to subQ conversion, that IV sticks around and that there are certainly patients and physicians that still like to do IV.

Moderator

Right.

Steve Mahoney
CEO, Viridian Therapeutics

So we expect that there'll be a split in that market share, heavily skewed towards the subq, certainly.

Moderator

Yeah.

Steve Mahoney
CEO, Viridian Therapeutics

You know, we've talked about north of 70%-

Moderator

Okay.

Steve Mahoney
CEO, Viridian Therapeutics

On subq. So that's a great—I mean, in our view, that's a great one-two punch to have for TED patients that's differentiated, and again, with subq potentially best-in-class.

Moderator

Right. So before we conclude, I don't think we can. We have to touch on the FcRn portfolio a little bit as well.

Steve Mahoney
CEO, Viridian Therapeutics

Sure.

Moderator

So VRDN-006 and VRDN-008. So maybe just an overview of where these are, what are the upcoming catalysts for this? When, when should we expect to see data?

Steve Mahoney
CEO, Viridian Therapeutics

Yeah, great. So VRDN-006 is an Fc fragment, very very much like VYVGART. Again, we really like the Fc fragment approach. We feel like we're the-- we, as far as we know, we're the only other fragment in development, in clinical development. We like that differentiation versus what we've seen so far from the full-length antibodies. And so we have an IND for that program at the end of this year, and we're on track with that. We've reiterated that guidance several times. And then we have our VRDN-008 program, which is a half-life extended approach, and that could be a game changer. That could be a game changer in FcRns, given how quickly they clear and how frequently you need to dose.

So to the extent we can have IgG suppression, and we showed this in our humanized mice, where we had very good IgG suppression, and we're able to sustain that for a period of time, that would allow us to change the dosing regimen. Again, that would be a game changer for FcRn. So we've seen the humanized mice data, and now we want to see the primate data.

Moderator

Right.

Steve Mahoney
CEO, Viridian Therapeutics

The primate data is coming in the second half of this year. What's exciting there as a catalyst is that, primate data for FcRns has proven to be very translatable into the clinic. That's a catalyst in and of itself.

Moderator

Right.

Steve Mahoney
CEO, Viridian Therapeutics

So we're excited. Yeah, the FcRn portfolio is moving right along.

Moderator

Got it. Last question from me. I don't see any hands raised. So, so when you look into the, you know... when you would start preparing for commercial, is that people usually start to prepare when the phase three data, pivotal data, becomes available. And how is that commercial preparation going? Because you have data coming up very soon.

Steve Mahoney
CEO, Viridian Therapeutics

Yeah, we're ahead of that.

Moderator

Are you guys already sort of ... like, where are you guys in terms of the commercial preparation and-

Steve Mahoney
CEO, Viridian Therapeutics

Yeah, we're ahead of that timeline. And we think that's really important. You can't be prepared enough. We have a great commercial team, a highly experienced group, that we'll continue to build out, obviously, as we ramp up. But yeah, we're certainly gonna ramp up and be ready for that, and yeah, pretty excited about our ability to compete.

Moderator

Right. And are you guys looking for a commercial partner in U.S. and ex-U.S.? Have you thought about that?

Steve Mahoney
CEO, Viridian Therapeutics

Yeah, look, what we do is we keep our options open, and we talk to people. But with this, these are certainly for thyroid eye disease in particular, I mean, just given the nature of how far along it is, that's certainly programs we could commercialize ourselves. So a partner is not particularly necessary.

Moderator

Yeah.

Steve Mahoney
CEO, Viridian Therapeutics

But again, we keep our options open.

Moderator

I see. Well, Steve and Shan, pleasure to be hosting you. Very exciting time for Viridian, that we look forward to seeing the data in September.

Steve Mahoney
CEO, Viridian Therapeutics

Thank you.

Moderator

Final remarks or final conclusion?

Steve Mahoney
CEO, Viridian Therapeutics

No, we're, you know, as we've said, we're really excited about all the things in our portfolio. From thyroid eye disease, we had a great announcement on our press release this morning for 003. Really exciting about the prospects there. We do think that's gonna be potentially best-in-class and could really help a lot of patients with thyroid eye disease. And then we have our autoimmune portfolio with FcRNs. So we're executing across the board, and we've got a long, long way to go, and we're looking forward to it.

Moderator

All right. Sounds good. All right. Thanks, everyone.

Shan Wu
Chief Business Officer, Viridian Therapeutics

Thanks for having me.

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