Conference Transcript - VRDN 2026-06-09

Rich, Analyst — Other

All right. Good afternoon. Let's kick off our next session. It is my pleasure to host Viridian Therapeutics. And with me, we have Steve Mahoney and John Wu, CEO and CBO of the company. So a lot to talk about. Very, very exciting year. But before we go do it, I'm going to kick it off

Speaker 2

to... Yeah, thanks, Rich. Thanks for having us. We appreciate it. We are on the verge of commercial approval. We have a PDUFA date of June 30th, so a very short time period

Rich, Analyst — Other

until we expect to be on the market.

Speaker 2

All of our regulatory interactions have been consistent and positive and give us a lot of confidence going into that PDUFA date, we are right on track with everything. So that's exciting. We can talk about launch dynamics and that type of thing in more detail as we go. We obviously also had our top-line readout from our subcutaneous studies, Reveal 1 and Reveal 2, both highly positive studies, both of them proptosis response and diplopia. We are advancing our Q4 and our Q8 weekly dosing regimens with a BLA submission targeted for Q1 of 27. So also exciting because we think that has a lot of potential in the TED market going forward. We also have a TSHR program that we have guided to an IND in Q4 of this year. So that's exciting, too, because that gives us applicability both in thyroid eye disease but also in Graves' population, which is exciting and complementary to the portfolio. And then finally, we have our FCRN portfolio is continuing to move forward. We have finished our first in human for our 006 program, which looked just like it should in terms of IgG suppression and albumin disparate, and that looked great. And now we're waiting on the results from the first in human. We're working through our first in human study on the 008 program, which is a half-life extended approach for FCRN. So a very exciting portfolio turning into a commercial company, but a lot of great stuff.

Rich, Analyst — Other

Got it. So any time, right? June 30th is the Padufa date. I mean, I think we've seen it where some of these Padufas are moving ahead of time, too. So with that, can you give us an update on the launch prep? Are you guys just ready to go? You just push the button and the sales forces all train up. Where are you with that process?

Speaker 2

Yeah, we are all ready to go. So we have the sales team has been on board for, or they came in in certain ways. But they're trained on a – we know the label is somewhat predictable, so we've been able to train them. They're obviously not physicians about anything yet, but they do have the ability to – so that stuff is all good. We've had our medical affairs team out in the field for quite some time. Support services, which is also built and ready to go. Supply chain is ready to go. so everything is ready to go. So we are operationally launched. We are all ready to go. We can, so we're ready to go.

Rich, Analyst — Other

More questions on the CRN. I think you mentioned that. We'll see updates.

Speaker 1

I can take that one. So we have two programs with a CRN 006, which is a FC fragment, and we really like the FC fragment. We think there may be something special about a fragment that the full-length antibodies so far have not been fully able to replicate the safety and efficacy of the currently available fragment, and 006 would be the other one that is in development. We've communicated that phase one studies from in healthy volunteers, the data looked as expected, so IGF-1 or IGG suppressions in line with the XDRN class, sparing of LDL and albumin, well-tolerated, so a very clean profile across the board. So that's exciting for that program. And then 008, which has the half-life extension that we've talked about, it's difficult to engineer half-life extension into an FCRM molecule, but we've been able to do it, and that is an ongoing healthy volunteer studies right now, phase one, and we expect data second half of this year. And with that, we will plan to look at and disclose and share the Healthy Volunteers data with regards to IgG suppression. We want to see that in line, again, with prior studies and what we have seen in non-human primates, which historically have been very translatable to humans. We'll want to see tolerability, in particular with regards to LDL and albumin, to see that being spared, which again we saw in primates, so looking to confirm that. And then importantly, looking to confirm the half-life extension, which again, head-to-head versus FGAR in the primate studies, we saw longer half-life, three times the half-life, as well as more sustained IgG suppression. So that would be very exciting data from a healthy volunteer standpoint to confirm that in humans, which will allow us to really extend the dosing interval between doses of a FCRN, which we think could be a game changer for patients. Both of these programs we've designed to be subcutaneous and patient self-administered, which is still an unmet need for patients in FCRN. And so we believe that we will be able to move the field forward with either one of these profiles.

Rich, Analyst — Other

Right, I see. Okay. And then you will be disclosing the indication and then sort of the development plan for those

Speaker 1

indications. Yeah, that's right. So for each of these programs, we do plan to update everyone on how we're thinking about next steps in development indication strategy.

Rich, Analyst — Other

Correct. Fantastic. Okay. And you guys recently raised $300 million due equity and convertible senior note. And I remember you guys mentioned that you guys already have enough capital to kind of sufficiently take the company to cash flow positive. So was that just to pay down, I think, the Hercules capital credit facility? Is there anything beyond that? Is there any consideration to pay off the DRI financing deal as well?

Speaker 2

Anything there? No. Well, so what we did is, yeah, we were able to pay down the Hercules facility. We also talked about, and Ellie, we're already covered. We thought it was a good idea that we're guiding towards profitability. But again, we've got a lot of really...

Rich, Analyst — Other

Are you sure, like, doctors, or are they more like some are for endo, some are for ophthalmologists, and some are for surgeons? Like, how are you guys thinking that?

Speaker 2

It's more geographic. So we have all of the TAPASA prescriptions, so that is our target sized for them.

Rich, Analyst — Other

And then, so given all that, consider, like...

Speaker 2

Yeah, so a couple points. First, in terms of advertising, I don't think you'll see a lot of us do it. But I think more importantly, we applaud. We get to take it available to them. We think there's a lot of physician excitement about that from a resource standpoint.

Speaker 1

And remember that this is a new start market. So we're not looking to switch patients off of any treatment on and doing well on because everything is fixed dose. The currently approved treatment is fixed dose. Veli IV will be fixed dose. It's a shorter course in awareness and channeling those patients to physicians to be a potential new start patient for VELI as well. We feel really good about the clinical profile and soon to be, we anticipate, commercial profile for VELI, given the data that we generate in both Thrive and Thrive 2, in particular the points of differentiation that we've talked about before that form the basis for our applications for breakthrough therapy designation as well as priority review. First of all, rapid onset of treatment effect on the active side. Chances are a patient who starts therapy will have already had a proptosis response after just one infusion after three weeks. And then secondly, diplopia improvement and resolution for these patients, in particular on the chronic side, which Veli is the first drug candidate, I should say, to have shown that level of diplopia benefit and statistically significant in chronic patients. And we offer all of that with a shorter duration of treatment in just 12 weeks that a patient will complete the entire full course of treatment. And then each infusion is only 30 to 45 minutes long.

Speaker 2

And remember that this is a market that's primarily made up of women in their 40s and 50s. It's an autoimmune disease. These are people with very busy lives, with families and jobs and just the activities of daily life in that age group. And so the ability to bring, as Sean just referenced, a shorter treatment period of just five infusions, shorter infusion times, just on a logistics basis of, you know, the clinical profile is what it is with the differentiation that we think that Sean just referenced, but then simply just the use of the treatment. Right.

Rich, Analyst — Other

So the PDUFA is coming. We're going to see your label coming out very soon. How do you think it's going to be, how do you think it's going to differentiate against the PESLA's label? I mean, I think a lot of, Sean, I think you highlighted a lot of things there that some of these advantages that we saw from the Thrive trial, do you think, like how would those be reflected in the label relative to how, and also obviously I think the, but what are some of the other key?

Speaker 1

Yeah, those points that I mentioned in terms of rapid onset of treatment effect, diplopia in response and complete resolution, all in a shorter course of treatment, those are the key data points from Thrive and Thrive 2 are two pivotal studies. So we would anticipate having that data in our label in both active and chronic TED, in which case we would be the first drug approved for thyroid eye disease with both active and chronic data in our label. So that's a major differentiation point for the label. Remember that when Tepeza was first approved, the indication was broad. It was for not just restricted to active TED, which was the basis for their registrational trials, but inclusive of all TED patients. And so we do think the FDA views this population as a continuum of disease and does not necessarily differentiate between active and chronic TED. And so we expect to also have a broad indication. But the labeling, again, this is where Horizon has done very good work in getting a clean label on both the efficacy as well as safety data that's in there, and we get to benefit from that, but replacing the data with the data that we generated from Thrive and Thrive 2, which we think are very compelling.

Rich, Analyst — Other

I see. Okay. And then, so I know part of the launch prep, you know, you guys go, and I mean, every company do a lot of these qualitative, quantitative market research with payers, subscribers, and patients. What are the findings there in terms of, like, how many of them actually recognize that differentiation that people actually recognize it and and and and would be see that as an advantage for them to I think what's more important when it

Speaker 2

comes to the payers is in the feedback that we've gotten in communication with the payers for quite some time including the pre-approval information exchanges that you can do now well I think what's really critical and important is that now Now that Tepeza has 85% coverage, it has taken a bit of time, but it's in our interactions with IL-6, our studies, IL-11, obviously that didn't go forward either, and then FCRN as well. So when the payers, that'll take a bit of time.

Rich, Analyst — Other

One thing that they're going to use. No, we haven't seen that. Again, going back to the market, how many wide trials?

Speaker 2

So now our job is to make...

Speaker 1

As a prescriber, it's in our clinical trial. So many of these physicians already have experience with VELI and actually, in fact, probably have had experience with ELI since there was good overlapping trial sites. And, of course, as we mentioned, our medical affairs team has been out there talking with these 2,000 core prescribers so that they are well-educated on, first, aware of VELI as a potential option and also well-educated on the data that we've generated from Thrive and Thrive 2. And it's helpful that it's the same mechanism. It's IGF-1R. We are a full antagonist. And so for physicians, understanding that it is the same mechanism is also very helpful.

Rich, Analyst — Other

I see. Okay. Let's talk about the chronic patients a little bit here. TAPESA has been struggling with this with these patients, single digit penetration what's the potential of VALA in that chronic test setting is it just because three less infusion suddenly that opens up like how should we think about that chronic setting is it more or should we have to wait for the subcutaneous or for ALI to come out in order to be better penetrate that

Speaker 2

that population? I would say that our strategy out of the box is, and currently they prescribe probably on an 80-20 basis. We do think that the profile has the potential, the valley profile, because it can possibly make the logistics, that might bring some. But in the short term, our strategy is to, the ability to come in, have an option, grab some of that market share in those prescribing habits. That's the, we'll see how the, we do think it's going to be attractive to chronic patients, depending on where they are. And there's, it's such a continuum.

Rich, Analyst — Other

I mean, given that these, yeah, so we ran the two, these are patients that by definition to

Speaker 2

get into the study, these, the symptomology, because there's a lot of variability, we did enroll clinical activity scores or they had high clinical activity. And that's a, So there's a lot of variability, and the population particularly gravitated toward primarily, in our view, if you think about it, they've been living with the disease, particularly, again, as I mentioned. And so our whole approach here, both try to make things easier, but we think that's primarily.

Speaker 1

We enrolled both of those chronic studies very quickly, so Thrive 2 on the IV side as well as Reveal 2 on the sub-Q side. and the majority of the REVEAL-2 study came from the U.S., 56%, and that's with a commercially available therapy that's here in the U.S. So that is good signal for the motivation that these patients have to seek treatment and their willingness to come on to treatment to have the symptoms of their thyroid eye disease be addressed. We think that they're just being underserved right now by what is currently available.

Rich, Analyst — Other

I see. Let's move on to Ellie. We don't have a lot of time left, so there's a lot to talk about there, too. So you guys presented the Phase 3 results for the Review 1 in active and Review 2 in the chronic study. Review 1 fell short of your own expectation that within TAPESA's proptosis response. But Review 2 showed IV-like results that fell between TAPESA and VALIE's proptosis response. How do you sort of reconcile the difference between these two studies?

Speaker 2

Yeah, look, I think as a reminder, reveal one was highly stat-sig on the primary endpoint, proptosis reduction for Q4, responses in diplopia in the Q4 arm, and then the Q8 weekly arm is an option for patients where we saw really good proptosis responses. That was in the active study. So then we saw that reinforced, to your point, on Reveal 2. And our intention, where dipopia may not be their main complaint, but proptosis is. And just if you think about getting, this is IGF-1R in an auto-injector pen. So everyone knows that IGF-1R is the mechanism that actually is the most effective in this disease population. And so the ability to put IGF-1R in an auto-injector pen that's very simple, it's at home, patient-administered at home, that is going to be very meaningful for this patient population. because if you think about where we fit, we've got IV, we've got an IV that we just talked about for all the reasons that that's a very attractive profile, Q4 weekly for proptosis in diplopia patients, and Q8 weekly for proptosis. So we have an answer, we believe we have an answer for any patient that walks in with moderate to severe TAD, we can address it across, and so those studies reinforce both of those profiles.

Rich, Analyst — Other

Got it. So even though at least inhibition for the IGF-1 are, excuse me, and you guys show similar PKPD profiles, why do you think that WP1 and 2 resources were lower?

Speaker 2

Yeah, so just, right, so we had the PKPD both looked like they were supposed to in terms of on the PK side what we saw there plus the IGF-1 levels that we would see from the PD side. So the drug did what we expected it to do. We may have seen some clinical variability in Reveal 1, but then Reveal 2, as you pointed out, brought that as expected, brought that IV-like efficacy. So I think that kind of reinforces the profile. You may have seen some variability in Reveal 1, but at the end of the day, we've tested these profiles with physicians, and the response has been overwhelmingly, okay, that looks like IGF-1R in an auto-injector pen. That's a great profile for us. I think that's the bottom line. It's very simple. Everyone knows that IGF-1R is the right mechanism, and now we're, you know, we expect to be able to spend an auto-injector.

Rich, Analyst — Other

Okay, got it. So when you think about the patients that are suitable for LD versus VALID, how do you think about that setting? You have these products come out. In the active setting and the chronic setting, how do you think, you know, how do patients

Speaker 2

Yeah, I mean, a little bit to repeat myself, I guess, but on the IV side, we think there's an IV appropriate patient population, no doubt. As you go up the index in severity and that urgency to treat, they're very well, we expect forever, we expect there to be patients that will be appropriate for IV, whether that's because their physician wants them in a controlled setting like an infusion center or the patient wants to be in a controlled setting. Like a site-threatening patient, you would want to put them on IV, and that's what we have Valley 4, that kind of severe. And we think it's obviously done very well with the moderate end of that spectrum as well, so we think that's a great option. When sub-Q is available, we do expect that the patients that are non-IV, that Q4 weekly has that impact on proptosis and diplopia, and then the Q8 weekly is really geared towards the proptosis. Again, we cover that spectrum of patients, and we have an answer for anybody who walks in.

Rich, Analyst — Other

I see. Okay. So I always look at chronic and the active patients as two very different and sort of distinct populations in some way. And therefore, because of that, you need different products that cater to what they're needs. Do you share this view that these are sort of two kind of distinct patients? And then also, if that's the case, then what product attributes do you believe that are more suitable for active and chronic patients?

Speaker 2

Yeah, the way we look at it is there's certainly the active population, again, along that spectrum that I described from moderate to severe. But there's also that same – you'll have patients that have lived with the disease, but they're not necessarily complaining about the pain and the inflammation that goes with it. They may have settled into their proptosis. Diplopia is a little harder to settle into, but there is some element of that. So we call them kind of like a chronic stable population, which is really where Tepeza was studied in that population. But then there's a chronic flaring population is what we call it. So these are people, as I said, we had people to get into our studies. Again, the largest studies ever run in chronic. To get into our studies, you had to have above normal proptosis, and a large percentage had diplopia as well. So there are patients in that chronic population that, again, to Sean's point, they enrolled that study quickly. They're the biggest studies ever run. And so it speaks to the market demand that's still out there for these patients. But, yes, there is a distinction. There's a lot of chronic patients. It's very underpenetrated, which is the opportunity in front of us with both VELA and then, obviously, LE sub-Q. Right. Got it. And then I do want to ask you this question.

Rich, Analyst — Other

So besides the OPI from Amgen, they have another IGF-11R sub-Q asset in development called AMG-732. So this is, I believe this is another legacy product from Horizon's acquisition. I think it's in phase 1-2 development, and it's a new modular entity. So it's designed for sub-Q, not like PESA OPI, which is really just more like PESA we formulated for subcutaneous. So given that this asset and then tapestral OBI is not being developed for Quantic Head, and my guess is that this one could be, have you heard much about this asset and your thoughts about it so far?

Speaker 2

Yeah, let's start with the OBI for a second. The OBI is four inches long, two inches thick. It's got gears, batteries. You have to insert the cartridge in fusion. So not really clear when they compare that to the auto-injector pen that we have.

Rich, Analyst — Other

So we don't think that's real.

Speaker 2

32, yeah, we're aware of it. We don't know exactly what it is. We think it's IGF-1R. They just got started with Phase 2. Fantastic. So we're out of time. Thank you so much.

Rich, Analyst — Other

It's been a pleasure hosting you guys, as always. I'll turn it to you guys for any final remarks.

Speaker 2

No, look, we're about to be commercial. We've got a great profile, very highly supported by the Phase 3 data. We've got a great commercial team. I'm very familiar with buy and build dynamics, and we are...so we're ready to go operationally and just in regulatory interactions have been great, and then we're looking forward to advancing ELI with BLA and Q127, and then the SDRN portfolio is coming too, along with TSHR. I look forward to it.