Viridian Therapeutics Q1 2024 Earnings Call Transcript

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Provided by Quartr
May 8, 2024

There are 10 speakers on the call.

Operator

Welcome to the Viridian Therapeutics First Quarter 2024 Conference Call. At this time, all participants are in a listen only mode. Later, we will conduct a question and answer session and instructions will be given at that time. As a reminder, this conference call is being recorded. I would now like to hand the call over to Ms.

Operator

Louisa Stone, Manager of Investor Relations at Viridian. Please go ahead.

Speaker 1

Thank you, and welcome, everyone, to our Q1 2024 earnings conference call. The press release reporting our financial results and corporate updates is available on the Investors page of our corporate website at www.veridiantherapeutics.com. Joining me on the call this morning are Steve Mahoney, our President and CEO Tom Sciulla, our Chief Medical Officer and Sean Wu, our Chief Business Officer. Before we begin, I would like to remind everyone that this conference call and webcast will contain forward looking statements regarding our financial outlook in addition to regulatory, product development and commercialization plans and research activities. These statements are subject to risks and uncertainties that could cause actual results to materially differ from those forecasted.

Speaker 1

A description of these risks can be found in our most recent Form 10Q and 10 ks on file with the SEC. I would now like to turn the call over to Steve Mahoney, our President and CEO.

Speaker 2

Thanks, Louisa, and welcome, everyone, to our Q1 earnings call. I'll start by giving a brief overview of Viridian, and then we'll get into more detail about our programs and recent progress. For those of you who are new to the Viridian story, our strategy is to identify market opportunities where there's a clear unmet need and where there is potential for us to develop differentiated products. We then aim to engineer the best possible therapeutics and then move rapidly to advance our programs to patients. Turning to Slide 4.

Speaker 2

Slide 4 shows our pipeline, which includes both a thyroid eye disease or TED portfolio, as well as an FcRn targeting autoimmune portfolio. We have several exciting updates to provide across our pipeline today, which we'll get into next. We're really excited to highlight for you today the significant progress that we've made across the business so far this year. Beginning with our 1 IV program, we are pleased to announce that THRIVE, our Phase 3 trial evaluating 1 in patients with active TED completed enrollment in March. In fact, not only did Thrive reach the target enrollment of 90 patients in mid March, but we exceeded enrollment for a total of 113 patients due to strong patient demand at our clinical trial sites.

Speaker 2

We expect to share top line results for Thrive in September 2024. Thrive II, our Phase 3 trial initiating 1 IV in patients with chronic TED continues enrolling and is on track for top line data at the end of this year. Lastly, for 1, we are announcing that we anticipate filing a BLA for the 1 program in the second half of twenty twenty five. For our subcutaneous 3 program, which we believe has substantially to be best in class, we recently completed a positive Type C meeting with the FDA, and we are moving forward with our preparations for our pivotal program in line with our previous guidance. We will provide additional updates for the 3 program before we start that pivotal program, which remains on track for mid year.

Speaker 2

We are also progressing our FcRn portfolio as planned. We are aiming to file an IND for 6 by the end of this year, and we plan to share 8 non human primate data in the second half of twenty twenty four. Lastly, we ended the quarter with 613 $1,000,000 in cash, cash equivalents and short term investments and we maintain our cash runway into the second half of twenty twenty six also as previously guide. Now I'd like to turn to our TED portfolio and talk more about the programs and our progress in a bit more detail. As a reminder, TED is an autoimmune condition characterized by inflammation and damage to the tissues around and behind the eyes.

Speaker 2

This leads to symptoms including proptosis or bulging of the eyes, redness, swelling, double vision and retraction to the eyelids. In severe cases TED can be sight threatening. With those symptoms as a backdrop, there is already a large market opportunity in TED that comes with global growth potential and an expansion that can come with better options for patients. There is an estimated 190,000 people in the U. S.

Speaker 2

Alone who are living with moderate to severe TED. These patients are only served by 1 marketed IGF-1R IV therapy currently, which generated approximately $1,800,000,000 in sales in just the U. S. Alone in 2023. This approved therapy requires 8 infusion every 3 weeks, which can be a significant burden for patients.

Speaker 2

We see opportunity for us to provide differentiated options for TED patients with both our IV and subcu programs. Because TED is an autoimmune disease characterized by flaring symptoms, patients with moderate to severe symptoms struggle with quality of life issues that make it hard for them to drive, work and even sleep. Because it is a flare based disease, it is considered a new start market, which means that it doesn't matter when a patient was diagnosed, because you need to treat the flare or the onset of those symptoms. This new start market also means that all patients experiencing symptoms will have the opportunity to choose from available treatment options with no chronic treatment to displace. Once a flare is treated, patients do not remain on an anti IGF-1R therapy.

Speaker 2

So when a subsequent flare arises, physicians will have the opportunity to choose from available treatment options, including potential new options to manage these flares in their patients. This is a great position for our IV and subcu programs in TED because we believe that we are developing potential best in class therapies in a drug class that is shown to be highly effective in inhibiting IGF-1R and in treating TED. Turning to the specifics of our product candidates. Viridian is developing 2 anti IGF-1R antibodies for TED, 1, which is delivered intravenously and 3, which is delivered subcutaneously with the potential for self administration. As you can see here, 3 and 1 have the same binding domain and we expect them to bind IGF-1R similarly.

Speaker 2

They differ because 3 is engineered to have an extended half length, which we have shown to be 40 to 50 days in healthy volunteers, which is 4 to 5 times that of 1, its parent molecule. With 1, we hope to have a fast to market differentiated IV therapy for patients with fewer doses and a shorter infusion time than the current standard of care. With DOH-three, we hope to have we hope to develop a convenient, less frequent, low volume therapy that patients can self administer at home. Let's review the 1 program, our progress and what makes us excited about the Phase 3 readout that we expect in September. On Slide 11, this is a reminder that we've already shown robust clinical activity with O-one after just 2 infusions in a Phase 2 clinical trial in active TED.

Speaker 2

This robust activity is across all key areas of the disease, proptosis or the bulging of the eyes, clinical activity score and diplopia or double vision. We have added data from the type of clinical trials after 2 doses on this slide to show the data side by side. While cross trial comparisons are difficult, we are encouraged by the clinical responses observed after just 2 doses of 1. On Slide 12, you could see that 1 was well tolerated in active TED patients with no serious adverse events, no infusion reactions and no discontinuations. Similarly, to active TED, in patients with chronic TED, just after just 2 infusions, 1 meaningfully reduced disease burden across each disease point as well.

Speaker 2

On Slide 14, 1 was also well tolerated in chronic TED patients. Based on this Phase 2 data, we believe that clinical regimen of 1 with fewer infusions, shorter infusion time and lower cumulative drug exposure has a potential to be a better choice for moderate to severe patients with TED. Now turning to our Phase III trials for OVA1. I would like to take a moment to thank all the patients and clinical trial site teams who have participated in our THRIVE trial. We're not done yet and our achievements so far would not be possible without them.

Speaker 2

As we announced today, we completed enrollment for THRIVE in March with 113 patients, which exceeded our enrollment target of 90 patients due to strong demand and interest at our clinical sites. About half of THRIVE's patients were from the U. S. And the other half came from Europe. We expect to provide top line results for this study in September of this year.

Speaker 2

THRIVE II, our 2nd pivotal study in TED is ongoing and are track for top line readout at the end of this year. In addition to Thrive and Thrive II, we recently initiated STR1VE, which is a planned safety study. STR1VE is a study of 1 in TED patients to complete the sufficient safety database for BLA submission alongside the patient numbers from Thrive and Thrive-two. In conclusion with 1, we are developing a potentially differentiated IV therapy for patients with fewer doses and shorter infusion time than the current standard of care, while inhibiting the same IGF-1R target, which has been shown clinically and commercially to be effective in treating TED. We are very excited about bringing FLOR-two zero one as a potential option for patients, which could mean significantly less drug for patients, fewer visits in the infusion center, lower volumes and less infusion share time.

Speaker 2

Our next program, subcutaneous 3 will take this differentiation even further with the possibility of lower frequency sub lower frequency subcutaneous administrations and potential for at home self administration using auto injectors. We know from market examples that a later entrant subcu therapy can convert meaningful portions of an existing IV market. We've included 2 of those examples here. In each of the cases on this slide, subcu offerings grew the overall market size of the class. In addition to quickly commanding a significant share of the IV markets.

Speaker 2

And keep in mind that these subcu examples have the same or more frequent dosing than their IV counterparts. This would not be the case with 3, which is designed to have potentially a best in class dosing profile. Also, it is important to point out in both examples that neither of these are a new start market. Again, the TED market is a new start market where we need to treat flaring disease or onset of symptoms as opposed to trying to convert patients from a long term chronic therapy. With 3, we hope to provide patients with an anti IGF-1R therapy that is a better option with respect to less overall drug exposure and more convenience.

Speaker 2

On Slide 19, we show the complete data set from our Phase 1 healthy volunteer study of OO3 to assess PK and PD. The update here is the inclusion of the last cohort, Cohort 5, where participants received 2 doses of 3 28 days apart. The data confirms the differentiated PK differentiated PK and PD for 3 seen in the first four cohorts with an extended half life of 40 to 50 days and sustained increased levels of the PD biomarker IGF-one. On Slide 20, you can see that the subcutaneous 3 was well tolerated in the Phase I study, including in the latest Cohort 5, with no serious adverse events or discontinuations related to treatment and observed adverse events were generally Grade 1 and mild. As we shared previously, our pharmacokinetic modeling for 3 that showed that or predicts that 3 potential dosing regimens are available to us.

Speaker 2

3 every 8 weeks, every 4 weeks and every 2 weeks could achieve or exceed the exposure levels of 1 that we saw in the active and chronic studies that were correlated to robust clinical activity for our Phase 2 clinical trials in TED. This gives us a lot of optionality as we move forward move towards our pivotal studies for 3 and importantly gives us the potential to develop for patients a best in class low volume subcu delivery option. We are pleased to announce today that we have completed our Type C meeting with the FDA and we are on track to initiate pivotal clinical trials for the 3 program. We will share more details on the pivotal trial design before we start those studies. Now turning to our FcRn portfolio.

Speaker 2

On Slide 24, in addition to TED and consistent with our development strategy, we are developing exciting portfolio of potential best in class FcRn inhibitors to address the unmet needs of patients living with auto antibody mediated autoimmune diseases. FcRn inhibitors represent a large market opportunity. The first FcRn inhibitor, FgART or VIVGART is approved for myasthenia gravis and is in registration for CIPD. It's already annualizing over to over $1,000,000,000 in annual sales. Myasthenia gravis alone is a large market with projected sales of over $4,000,000,000 annually by 2028.

Speaker 2

In addition to myasthenia gravis, as you can see from the slide, there are additional sizable autoimmune indications that would meaningfully add to the FcRn opportunity. Our FcRn franchise includes 2 assets, 6 and 8. With 6, we are excited to have the only other FcRn targeting Fc fragment in development other than efgartigimod. Argenx has shown that its Fc fragment achieves substantial efficacy while sparing an effect on albumin or LDL and shows better tolerability than the full length antibodies. We are on track to submit an IND for 6 by the end of this year and look forward to sharing more about the program in the future.

Speaker 2

Next on the right is 8. Our protein engineering efforts identified a molecule derived from Fc fragments that both extended the half life and generated meaningfully deeper IgG reductions in animal models. We believe 8 is a potential durably suppress IgG. We are on track to provide 8 non human primate data in the second half of this year as guided, and we are excited to bring forward this portfolio of next generation FcRns to potentially offer patients a more convenient dosing profile compared to current weekly IV or subcu infusions. In addition, by aiming to improve the duration and depth of IgG suppression with 8, we hope to offer a best in class option for patients.

Speaker 2

Our team is executing. We have made excellent progress across the company in the Q1 of the year, and we look forward to continuing that momentum through the rest of the year to deliver on our multiple upcoming catalysts. As I mentioned previously, we plan to report Thrive top line in September and Thrive II top line is on track for the end of the year. The FcRn programs are also proceeding as expected. It has been my pleasure today to provide these exciting updates across our portfolio and in particular for 1 and 3, reflecting the work that we've completed during this quarter.

Speaker 2

This progress and our recent achievements reflect our team's ability to execute and we are well positioned to continue to work and deliver on our exciting upcoming catalysts. Last but not least, we remain well capitalized and in the quarter was $613,000,000 and the runway is into the second half of twenty twenty six. So with that, I'll ask the operator to open the call for questions. Operator?

Operator

Thank you. We will now begin the question and answer session. And the first question comes from the line of Laura Chico with Wedbush. Please go ahead.

Speaker 3

Good morning. Thanks very much for taking my question and congrats on the progress here. I guess I have two questions for you. First, with respect to the THRIVE data that's coming in September, I'm wondering if you can kind of help us frame what success looks like on the efficacy side. But then also with respect to reduced drug exposure, how would you think this might impact the rate of hearing impairment events that you might see in Thrive?

Speaker 3

And then secondarily, just related to STR1VE, I'm wondering if you could talk a little bit more about the inclusion of the active control arm? Thanks.

Speaker 2

Yes, sure. Thanks, Laura, for the question. In terms of what is good look like for Thrive efficacy, as we've stated previously, we think a profile that looks like Tepesa is similar to Tepesa would be a really good place for us to land. So with respect to hearing, yes, certainly, I think what we're looking for in the same vein on efficacy for safety, getting a similar profile on the safety because the safety profile for TAPPEZZA is good, it's benign. And you referenced hearing in particular.

Speaker 2

To the extent the lower exposures improve upon that, that would be great. To the extent it's C MAX driven. We obviously have a lower Cmax just by virtue of the volume that we deliver versus Tepesa. So that could possibly be helpful. We'll have to see.

Speaker 2

But I think in terms of what good looks like, we'd love to see a similar profile. With respect to your question on STR1VE, yes, I mean, look, the STR1VE is simply to complete the safety database, which is just normal blocking and tackling for a BLA submission. So nothing there unusual. On the active control arm, it's just you got to run a well controlled study. And so we have the option of an active control arm of 3 mgs per kg.

Speaker 2

It randomizes 3:one, so the numbers will be heavily weighted towards the 10 mgs per kg. Again, it's all for the safety side of it. So I hope that answers the question. Yes,

Speaker 3

it does. Thanks, Steve.

Operator

Your next question comes from the line of Alex Thompson with Stifel. Please go ahead.

Speaker 4

Great. Thanks for taking our questions. I guess I wanted to drill in a little bit more on safety in particular. I guess when you look back at the TEPEZZA safety profile from the Phase 3s versus the more recent chronic TED study. Like do you feel like the chronic TED study might represent a better if you ran a TEDSA study today with more focused hearing measuring if that's more of a bar for safety?

Speaker 4

Or how are you thinking about like what TEPEZZA safety actually looks like today versus when those Phase 3s were started? And then for the top line for 1 in Phase 3,

Speaker 5

do you expect to be able to

Speaker 4

share any data beyond 15 weeks as part of that either for safety or efficacy? Thanks.

Speaker 2

Well, I can take the second one first, Alex. No, top line is top line. So it'll be a readout at the 15 week endpoint. With respect to your question on what would it sounds like you're asking what is TAPAZA post the clinical trials and what that real world experience is. Yes, that is all part of it.

Speaker 2

Like we're trying to understand that as well. I mean, I know Amgen is trying to understand that. The physician community, the patient community, they're all trying to understand that. And maybe, Tom, do you want to just explain how we're approaching our reporting of AEs in the same way that that depends a bit?

Speaker 6

Sure. So Alex, as you know, the field is evolving. As you alluded to, there's updated guidance from the FDA which led to a label change for tepro as you know. And in current clinical practice, physicians are assessing patients hearing at baseline during and after treatment. So we are recording adverse events using Medra terms.

Speaker 6

And as you know, this is just a standard way of recording patient reported changes in their health, including hearing. This is standard for any clinical trial and including TEPEZ in their pivotal trials. We're also assessing audiometry as is done in clinical practice at baseline in pre specified points. So we're essentially doing what's done currently in the evolving clinical practice.

Speaker 4

Great. Thanks.

Operator

Your next question comes from the line of Michael Yee with Jefferies. Please go ahead.

Speaker 2

Hello?

Speaker 5

Okay, great. I guess we can hear you. So two questions. First was on the ongoing THRIVE study. Can you talk a little bit about how you can control for the hearing impairment and hearing loss events?

Speaker 5

I know that if you actually go look back at some of the TEPEZZA post marketing studies that there is some commentary and analysis around how patients have some of this hearing loss already and there is factors already going on with some of these TED patients in the background. And so just trying to think about how you can screen and protect for that and think about that as you go through your Phase 3. And then on the subcu plans for Phase 3, I think you said that you met with the FDA and you're planning to start Phase 3. Can you just talk a little bit about how that meeting went? And I know there were some uncertainties about going directly into Phase 3.

Speaker 5

So just talk a little bit about your confidence there or anything else that you need to do order to start the Phase 3 for subcu? Thank

Speaker 2

you. Yes, great. Thanks. Thanks, Mike. Let me take the second one first, and then I'll ask Tom Chula to weigh in on the baseline hearing question that you had first.

Speaker 2

So with respect to the 3 program, we did have a positive meeting. We have not received the minutes yet. So but

Speaker 7

we had

Speaker 2

a positive meeting and we are reiterating our guidance that we are going to start a pivotal program mid year this year. So we will provide a lot more detail once we get on the other side of minutes, but before we start the study. So more to come, but to answer your question, positive meeting, we feel good about our reiterating our guidance on starting that pivotal program. So we're pretty excited there. With respect to the Thrive and the baseline hearing, I'll ask Tom Chula to jump in there, please.

Speaker 6

Thanks, Michael. So as we said in the previous answer, adverse events in the study is reported via the measure of terms with the standard methodology for reporting patient changes in their health including hearing and this was done in the TESSA trials. As I mentioned, we're also using audiometry for monitoring and that's consistent with the current clinical practice and FDA guidance. We do have an exclusion criteria for hearing loss at baseline. You can see that exclusion on clinicaltrials.gov.

Speaker 5

Okay. Thank you.

Operator

Your next question comes from the line of Gavin Clark Gardner with Evercore. Please go ahead.

Speaker 8

Good morning and thanks for taking my questions. Just had 2. First, on the Type C meeting for 3, does the FDA want to see any dose ranging work in TED patients as part

Speaker 7

of that pivotal? Or do

Speaker 8

you believe you can start dosing immediately in like a blinded pivotal portion of the trial?

Speaker 2

Yes. So thanks, Gavin. Like I said, give us a little bit more time. We'd like to see the minutes. But just take comfort from the fact that we are we feel positive coming out of that meeting and that we are starting our pivotal program, which is what we've been what we were guiding to previously, but we've now had that meeting.

Speaker 2

So we feel good about where we're going. But give us a little bit more time and we'll be able to break down those details for you. But that's kind of where we are and we feel good.

Speaker 8

Sounds good. We'll await more details. And are you able to provide any details on how the Thrive baseline characteristics compared to TEPESIS Phase 3?

Speaker 2

Yes, that's another one, Gavin. I mean, it's a great question. I totally appreciate it. It's just that we're just not there yet. We don't have all that information for Baseline Thrive.

Speaker 2

We just completed enrollment. And so it's going to take us a bit to just get all that together. So more to come on that one as well.

Speaker 8

Makes sense. Thank you.

Operator

Your next question comes from the line of Raynik Aguda with LifeSci Capital. Please go ahead.

Speaker 7

Hey guys, congrats on all the progress and thanks for taking my questions as well. You touched on the significant ex U. S. Market opportunity in TED. I guess, are you planning to file for approval of 1 in the U.

Speaker 7

S. And Europe in parallel once all the data is in hand? And how large would have been opportunity could that ultimately represent?

Speaker 2

Yes. I think the epidemiology in Europe is very similar. So we know that to be the case, similar to the U. S. That is.

Speaker 2

With respect to our ex U. S. Plans, again, that's something we'll probably talk a bit more about later. We are as you can imagine, we are absolutely thinking about all that and the best approaches in these different geographies even beyond Europe. So that's all in the works.

Speaker 2

It will have more to say at a later time.

Speaker 7

Got it. Sounds good. Thanks.

Operator

Your next question comes from the line of Derek Archila with Wells Fargo. Please go ahead.

Speaker 7

Hey, this is Adam on for Derek. Thanks for taking our questions today. I guess just a couple of questions on the timeline really. So given Thrive is still reading out like mid-twenty 24 ish and Thrive II reading out end of year, what factors are driving a second half 20 25 BLA submission in TED? And is this related to the STR1VE study then?

Speaker 7

And in that sense, is an interim cut, would that be sufficient from STR1VE for a BLA submission? Or does the whole STR1VE trial need to be completed to support the BLA submission?

Speaker 2

Great. Thanks, Adam. Appreciate that question. Yes, so what's driving the timeline is remember, so we talked about SRAIGHT 2, that's a top line readout at the end of the year. So by definition, it's not the completed study, right?

Speaker 2

So we have to let that we have a follow-up period. There's a total of the 52 week follow-up period, but only 37 weeks post the last dose. So there's a follow-up period that is have to be taken into account. And it's primarily Thrive 2 that's driving the timeline. It really doesn't we're not expecting STRIVE to have an impact there.

Speaker 2

That STRIVE should fit squarely within that timeline. And so we feel that that's probably the driver and we don't need all of STRiVE. What you see there is that on the ct.gov, you see 212 patients. That's the maximum number that we'll need. We can do a data cut as soon as we reach the requisite number for the safety database.

Speaker 2

And there's a little there's a there are moving parts that go with that in terms of you can have dropout rates in your Thrive OR Thrive 2. So we kind of over we over engineer or over set up the STR1VE study, but we can do a data cut when we hit that threshold. And again, all of this is really typical. You have to have safety database that accompanies your BLA submission. So it all kind of is normal blocking and tackling from our perspective.

Speaker 2

But just kind of take into account that that Thrive 2 is just a top line readout. So there's more to do after a top line readout, which drives the second half twenty twenty five filing.

Speaker 7

Got you. That makes sense. And I guess, can you provide any numbers and on what that threshold is? And then just kind of following up on the STR1VE study too, is this the safety database you expect to leverage then with any potential regulatory path for VRDN003? Thanks.

Speaker 2

Yes. So 3, as we've talked about previously, is a different molecular entity. So it will have its own path. So the answer to that question is no. On the first question, the threshold is the threshold, again, like I said, $212,000,000 is the max number.

Speaker 2

We're not we'll see where we end up. The total number is 300, but that includes Thrive and Thrive 2 active patients on 10 mgkg. So, we're not anticipating needing the entire STRIDE study. And again, most importantly, STRIDE is not expected to drive the timeline for BLA submission. It's more the THRIVE 2.

Speaker 7

Got it. Thank you.

Operator

Your next question comes from the line of Gregory Renzo with RBC. Please go

Speaker 5

ahead. Hey, Steve. Congrats on the progress. Thanks for taking my question. Maybe Steve for you and perhaps Tom, just wanted to get your latest views on the competition for patients and clinical trials certainly as your trials are heating up and appreciate all of the progress you have made.

Speaker 5

There are others out there as well, tepro with the subcutaneous. What's the latest on driving demand and what levers are you pulling to really accelerate the enrollment as well as the trial execution? Thanks.

Speaker 2

Yes. So I'll turn this over to Tom in a second. But yes, I think if you could see that we enrolled not only did we enroll Thrive on time, we exceeded enrollment. We had really strong patient demand to drive that all within the month of March. I think that should be a clear sign to the world that there are lots of patients out there with TED that want to access IGF-one therapy.

Speaker 2

So that's a really good sign for us. Don't forget also that we had roughly half of the patients were enrolled in the U. S. I know that was a question mark for people. I think we've definitively answered that the U.

Speaker 2

S. Is very we've got the opportunity within the U. S. And then the other half in Europe where the as I mentioned, the epidemiology is the same. So that might be just a general answer.

Speaker 2

Tom, Chula, do you want to talk about how competition for trials is shaking up? Sure. Yes.

Speaker 6

Thanks for your question, Gregory. I'm out in the field a lot talking to investigators and KOLs both in the U. S. And ex U. S.

Speaker 6

And I can tell you there's a lot of excitement about our entire portfolio. As you know, our Phase 2 trial showed really promising results and that's driven a lot of interest. Hence, the over enrollment that Steve referenced. Also I can tell you that with respect to STR1VE, we have an active control. There is no placebo and we think that's going to drive enrollment there.

Speaker 6

So I think overall just lots of positivity around our TED portfolio in our trials. I really can't comment on competitors, but what I can say is just lots of excitement and enthusiasm around our portfolio.

Speaker 5

Great. Thanks guys.

Operator

Your next question comes from the line of Julian Harrison with BTIG. Please go ahead.

Speaker 5

Hi, good morning. Thank you for taking my question. I understand FcRn is kind of in the background this year, but I'm wondering if there's maybe an IGF-one hundred FcRn sequence in TED that could be worthwhile to study in the future? Or are you mainly interested in FcRn opportunities beyond TED at this point?

Speaker 2

Yes, it's the latter, Julian. Yes, we're we would we just don't take for TED patients, we firmly believe that IGF-1R is the key to that disease or the heart of that disease. That's where the cell signaling is taking place. You've got to hit that receptor in order to disrupt that. And so FcRn, the IL-six is the other modalities are not or other mechanisms.

Speaker 2

We don't feel are on target for moderate to severe TED patients. So for us, the IGF-1R is the key to TED. So FCRM, we'll take that in different places as we alluded to in the deck.

Speaker 8

Thank you.

Operator

Your next question comes from the line of Trevor Allred with Oppenheimer. Please go ahead.

Speaker 9

Hey guys, good morning. Thanks for taking the question. So with the presence of sales trending down slightly, can you give us some perspective on why you think the new start market there appears to be somewhat stagnant and how you see this as a potential opportunity for you?

Speaker 2

Yes. I mean, I think it's hard for to comment on Amgen sales. I think they are they did report on their call last week, they did report year over year growth, which is the first time they've done that since the announcement of the merger. So we see that as a really good sign. Amgen was also really confident on their call that they believe the market continues to be under penetrated, which we agree with.

Speaker 2

And they believe that it's going to continue to grow. Growth areas, don't forget growth areas also include the other geographies, the introduction of subcu. And so they've now they've filed in Japan and Europe, which is good as they're continuing to kind of blaze that trail for us. And don't forget, even in the backdrop of all this, they did close to $1,800,000,000 or close to $2,000,000,000 in sales in 2023 in the backdrop of all of this as a first entrant. So again, we feel that there's plenty of room to run-in TED, not only in the U.

Speaker 2

S. But elsewhere as well. And then we think sub Q, particularly our sub Q, which we think is potentially going to be best in class, where we can have patients that can access it just good delivery at home and they can self administer at home. We think that's a game changer for TED patients. And I think the physician community agrees with us on that.

Speaker 2

So, yes, we're not particularly worried about IGF-1R being the right place for TED patients and I think Amgen is going to prove that as well.

Speaker 9

Okay, great. That's super helpful. And I guess could you give us some perspective on when we might expect to see initial FcRn data with the IND coming in the year, maybe second half mid year 2025?

Speaker 2

Yes. In our deck, you can see that we've got some healthy volunteer data that's pegged to the second half 'twenty five for that 6 program. It's a little ways out, but so we'll look to see if we can pull that timeline in. But we feel we're on track for that IND and then we'll get the healthy volunteer study going and so we'll get some data there. I think don't forget the 8 non human primate data that has in other FcRns that proven to be pretty translatable.

Speaker 2

So we're pretty excited to see that. We saw great humanized mice data for 8, but obviously that's mice data. We want to see the NHPs. We'll get that in the second half of this year. So we think that's actually a relatively important thing for us to get.

Speaker 2

So we're looking forward to FcRn moving forward. We've got a lot to do there.

Speaker 9

Okay, great. Thanks, Steve.

Operator

Great. Thank you. Sorry, at this time, we have reached the conclusion of the question and answer session. I would now like to turn the call back to Viridian's President and CEO, Steve Mahoney, for closing remarks.

Speaker 2

Great. Thank you, operator, and thanks to everyone for joining the call this morning. We've made a lot of progress, and we are executing. We're delivering on what we've said and that's important. We know that's important to you as well.

Speaker 2

So thank you for your participation today, and we look forward to talking to you in the future.

Operator

This concludes today's conference call. You may disconnect your lines. Thank you for participating.

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Key Takeaways

  • Viridian announced completion of the THRIVE Phase 3 trial in active TED with 113 patients (exceeding the 90-patient target) and expects top-line results in September 2024.
  • THRIVE II in chronic TED remains on track for top-line data at year-end 2024, supporting a planned BLA submission for VRDN-001 in the second half of 2025.
  • After a positive FDA Type C meeting, the subcutaneous candidate VRDN-003 is gearing up for pivotal trials mid-year, aiming to deliver a low-frequency, self-administered therapy.
  • The FcRn portfolio is advancing with a VRDN-006 IND submission targeted by year-end 2024 and non-human primate data for VRDN-008 expected in the second half of 2024.
  • Viridian ended Q1 with $613 million in cash and equivalents, extending its operational runway into the second half of 2026.
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Earnings Conference Call
Viridian Therapeutics Q1 2024
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