Viridian Therapeutics Q1 2024 Earnings Report

Viridian Therapeutics EPS Results

• Actual EPS: -$0.79
• Consensus EPS: -$1.07
• Beat/Miss: Beat by +$0.28
• One Year Ago EPS: -$1.61

Viridian Therapeutics Revenue Results

• Actual Revenue: $0.07 million
• Expected Revenue: $0.09 million
• Beat/Miss: Missed by -$20.00 thousand
• YoY Revenue Growth: -26.50%

Viridian Therapeutics Announcement Details

• Quarter: Q1 2024
• Date: 5/8/2024
• Time: Before Market Opens
• Conference Call Date: Wednesday, May 8, 2024
• Conference Call Time: 8:00AM ET

Viridian Therapeutics (NASDAQ:VRDN) (NASDAQ: VRDN) is a clinical-stage biopharmaceutical company focused on the discovery and development of targeted antibody therapeutics for patients suffering from rare and serious diseases. The company’s lead program, VRDN-001, is a fully human monoclonal antibody that antagonizes the insulin-like growth factor-1 receptor (IGF-1R), with an initial focus on thyroid eye disease (TED). By selectively inhibiting IGF-1R signaling, VRDN-001 aims to reduce inflammation and tissue remodeling associated with TED and related disorders.

In addition to VRDN-001, Viridian is advancing a second antibody program, VRDN-002, which targets complement-mediated pathways implicated in autoimmune and inflammatory diseases. Both candidates are being evaluated in multiple clinical trials across North America and Europe, underscoring the company’s commitment to building a robust pipeline of precision biologics. Viridian’s research platform leverages advanced antibody engineering to enhance potency, selectivity and pharmacokinetic properties, supporting the development of differentiated therapies with the potential for best-in-class profiles.

Founded in 2020 and headquartered in Boston, Massachusetts, Viridian was established by a team of experienced biotechnology entrepreneurs and scientists. The company completed its initial public offering in 2021 to secure funding for clinical development and manufacturing scale-up. Under the leadership of Chief Executive Officer Mara Aspinall and Chief Medical Officer Michael Rosenzweig, Viridian has formed strategic partnerships with contract development and manufacturing organizations to support its late-stage programs. Viridian’s global footprint extends through clinical sites and collaborative networks aimed at delivering novel treatments to patients worldwide.

Viridian Therapeutics Q1 2024 Earnings Call Transcript

Key Takeaways

• THRIVE Phase 3 enrollment exceeded target with 113 patients due to strong site demand, and topline results are expected in September 2024.
• THRIVE II in chronic TED remains on track for a year-end topline readout, supporting a planned BLA submission for the IV program in the second half of 2025.
• Subcutaneous VRT-003 completed a positive Type C meeting with the FDA and is proceeding toward a mid-year pivotal trial start, leveraging its 40- to 50-day half-life for less frequent dosing.
• FcRn portfolio progress includes an IND filing for VRDN-006 by year-end and anticipated non-human primate data for VRDN-008 in the second half of 2024.
• Strong cash position with $613 million in cash, equivalents and short-term investments, extending runway into the second half of 2026.

Presentation

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

[Louisa Stone, Manager of Investor Relations at Viridian Therapeutics]

Thank you, and welcome everyone to our first quarter 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.viridiantherapeutics.com. Joining me on the call this morning are Steve Mahoney, our President and CEO; Tom Ciulla, our Chief Medical Officer; and Shan 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. A description of these risks can be found in our most recent Form 10-Q and 10-K on file with the SEC.

[Louisa Stone, Manager of Investor Relations at Viridian Therapeutics]

I would now like to turn the call over to Steve Mahoney, our President and CEO.

[Steve Mahoney, President and CEO at Viridian Therapeutics]

Thanks, Louisa, and welcome everyone to our first quarter 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's 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 four, slide four 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.

[Steve Mahoney, President and CEO at Viridian Therapeutics]

Beginning with our 001 IV program, we are pleased to announce that THRIVE, our phase 3 trial evaluating 001 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. We expect to share top-line results for THRIVE in September 2024. THRIVE-2, our phase 3 trial initiating 001 IV in patients with chronic TED, continues enrolling and is on track for top-line data at the end of this year. Lastly, for 001, we are announcing that we anticipate filing a BLA for the 001 program in the second half of 2025.

[Steve Mahoney, President and CEO at Viridian Therapeutics]

For our subcutaneous 003 program, which we believe has the potential 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 003 program before we start that pivotal program, which remains on track for mid-year. We are also progressing our FcRn portfolio as planned. We are aiming to file an IND for 006 by the end of this year, and we plan to share 008 non-human primate data in the second half of 2024. Lastly, we ended the quarter with $613 million in cash, cash equivalents, and short-term investments, and we maintain our cash runway into the second half of 2026, also as previously guided.

[Steve Mahoney, President and CEO at Viridian Therapeutics]

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. 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. alone who are living with moderate to severe TED. These patients are only served by one marketed IGF-1R IV therapy currently, which generated approximately $1.8 billion in sales in just the U.S. alone in 2023.

[Steve Mahoney, President and CEO at Viridian Therapeutics]

This approved therapy requires 8 infusions every 3 weeks, which can be a significant burden for patients. 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.

[Steve Mahoney, President and CEO at Viridian Therapeutics]

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 two anti-IGF-1R antibodies for TED: VRDN-001, which is delivered intravenously, and VRDN-003, which is delivered subcutaneously with the potential for self-administration. As you can see here, VRDN-003 and VRDN-001 have the same binding domain, and we expect them to bind IGF-1R similarly.

[Steve Mahoney, President and CEO at Viridian Therapeutics]

They differ because 003 is engineered to have an extended half-life, which we have shown to be 40-50 days in healthy volunteers, which is 4-5 times that of 001, its parent molecule. With 001, 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 003, we hope to develop a convenient, less frequent, low-volume therapy that patients can self-administer at home. Let's review the 001 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 have already shown robust clinical activity with 001 after just 2 infusions in a phase 2 clinical trial in active TED.

[Steve Mahoney, President and CEO at Viridian Therapeutics]

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 TEPEZZA 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 001. On slide 12, you can see that 001 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 2 infusions, 001 meaningfully reduced disease burden across each disease point as well. On slide 14, 001 was also well-tolerated in chronic TED patients.

[Steve Mahoney, President and CEO at Viridian Therapeutics]

Based on this phase 2 data, we believe that the clinical regimen of 001 with fewer infusions, shorter infusion time, and lower cumulative drug exposure has the potential to be a better choice for moderate to severe patients with TED. Now turning to our phase 3 trials for 001. 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 are not done yet, and our achievements so far would not be possible without them. 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.

[Steve Mahoney, President and CEO at Viridian Therapeutics]

We expect to provide top-line results for this study in September of this year. THRIVE-2, our second pivotal study in TED, is ongoing and on track for top-line readout at the end of this year. In addition to THRIVE and THRIVE-2, we recently initiated STRIVE, which is a planned safety study. STRIVE is a study of 001 in TED patients to complete the sufficient safety database for BLA submission alongside the patient numbers from THRIVE and THRIVE-2. In conclusion with 001, 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.

[Steve Mahoney, President and CEO at Viridian Therapeutics]

We are very excited about bringing forward 001 as a potential option for patients, which could mean significantly less drug for patients, fewer visits to the infusion center, lower volumes, and less infusion chair time. Our next program, subcutaneous 003, will take this differentiation even further with the possibility of lower-frequency subcutaneous administrations and potential for at-home self-administration using autoinjectors. We know from market examples that a later entrant subcu therapy can convert meaningful portions of an existing IV market, and we've included two 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. Keep in mind that these subcu examples have the same or more frequent dosing than their IV counterparts.

[Steve Mahoney, President and CEO at Viridian Therapeutics]

This would not be the case with 003, 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 in new start markets. 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 003, 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. On slide 19, we show the complete dataset from our phase 1 healthy volunteer study of 003 to assess PK and PD. The update here is the inclusion of the last cohort, cohort 5, where participants received 2 doses of 003 28 days apart.

[Steve Mahoney, President and CEO at Viridian Therapeutics]

The data confirms the differentiated PK and PD for 003 seen in the first four cohorts with an extended half-life of 40-50 days and sustained increased levels of the PD biomarker IGF-1. On slide 20, you can see that the subcutaneous 003 was well-tolerated in the phase 1 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 003 showed or predicts that three potential dosing regimens are available to us: 003 every eight weeks, every four weeks, and every two weeks could achieve or exceed the exposure levels of 001 that we saw in the active and chronic studies that were correlated to robust clinical activity for our phase 2 clinical trials in TED.

[Steve Mahoney, President and CEO at Viridian Therapeutics]

This gives us a lot of optionality as we move towards our pivotal studies for 003 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 003 program. We will share more details on the pivotal trial design before we start those studies. Now turning to our FcRn portfolio. On slide 24, in addition to TED and consistent with our development strategy, we are developing an exciting portfolio of potential best-in-class FcRn inhibitors to address the unmet needs of patients living with autoantibody-mediated autoimmune diseases. FcRn inhibitors represent a large market opportunity.

[Steve Mahoney, President and CEO at Viridian Therapeutics]

The first FcRn inhibitor, efgartigimod or VYVGART, is approved for myasthenia gravis and is in registration for CIDP, and it's already annualizing to over $1 billion in annual sales. Myasthenia gravis alone is a large market with projected sales of over $4 billion annually by 2028. 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 two assets: 006 and 008. With 006, 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.

[Steve Mahoney, President and CEO at Viridian Therapeutics]

We are on track to submit an IND for 006 by the end of this year and look forward to sharing more about the program in the future. Next on the right is 008. 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 008 is a potential best-in-class extended half-life molecule targeting FcRn with the potential to more durably suppress IgG. We are on track to provide 008 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.

[Steve Mahoney, President and CEO at Viridian Therapeutics]

In addition, by aiming to improve the duration and depth of IgG suppression with 008, we hope to offer a best-in-class option for patients. Our team is executing. We have made excellent progress across the company in the first quarter 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-2 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 001 and 003, reflecting the work that we've completed during this quarter.

[Steve Mahoney, President and CEO at Viridian Therapeutics]

This progress and our recent achievements reflect our team's ability to execute, and we are well-positioned to continue the work and deliver on our exciting upcoming catalysts. Last but not least, we remain well-capitalized ending the quarter with $613 million, and the runway is into the second half of 2026. 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. If you have dialed in and would like to ask a question, please press star 1 on your telephone keypad to raise your hand and join the queue. If you would like to withdraw your question, simply press star 1 again. If you are called upon to ask your question and are listening via loudspeaker in your device, please pick up your handset and ensure that your phone is not on mute when asking your question. Again, press star 1 to join the queue. Your first question comes from the line of Laura Chico with Wedbush. Please go ahead.

[Laura Chico, Analyst at Wedbush]

Good morning. Thanks very much for taking that 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? And then secondarily, just related to STRIVE, I'm wondering if you could talk a little bit more about the inclusion of the active control arm. Thanks.

[Steve Mahoney, President and CEO at Viridian Therapeutics]

Yeah, sure. Thanks, Laura, for the question. In terms of what does good look like for THRIVE efficacy, as we've stated previously, we think a profile that looks like TEPEZZA is similar to TEPEZZA would be a really good place for us to land. So with respect to hearing, yeah, 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 TEPEZZA is good. It's benign. And you referenced hearing in particular. To the extent the lower exposures improve upon that, that would be great. To the extent it's CMAX-driven, we obviously have a lower CMAX just by virtue of the volume that we deliver versus TEPEZZA. So that could possibly be helpful. We'll have to see, but I think in terms of what good looks like, we'd love to see a similar profile.

[Steve Mahoney, President and CEO at Viridian Therapeutics]

With respect to your question on STRIVE, yeah, I mean, look, STRIVE 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 mg/kg. It randomizes 3 to 1, so the numbers will be heavily weighted towards the 10 mg/kg. Again, it's all for the safety side of it. So I hope that answers the question.

[Laura Chico, Analyst at Wedbush]

That does. Thanks, Steve.

[Operator]

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

[Alex Thompson, Analyst at Stifel]

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, do you feel like the chronic TED study might represent a better if you ran a TEPEZZA study today with more focused hearing measuring, if that's more of a par for safety? Or how are you thinking about what TEPEZZA safety actually looks like today versus when those phase 3s were started? And then for the top line for 001 in phase 3, do you expect to be able to share any data beyond 15 weeks as part of that, either for safety or efficacy? Thanks.

[Steve Mahoney, President and CEO at Viridian Therapeutics]

Well, you 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 it sounds like you're asking what is TEPEZZA post the clinical trials and what that real-world experience is. Yeah, that is all part of it. We're trying to understand that as well. I mean, I know Amgen's trying to understand that. The physician community, the patient community, they're all trying to understand that. And maybe, Tom Ciulla, do you want to just explain how we're approaching our reporting of AEs in the same way that TEPEZZA did?

[Thomas Ciulla, CMO at Viridian Therapeutics]

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 TEPEZZA, as you know. And in current clinical practice, physicians are assessing patients' hearing at baseline during and after treatment. So we're recording adverse events using MedDRA terms. 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, including TEPEZZA and their pivotal trials. We're also assessing audiometry, as is done in clinical practice, at baseline and prespecified points. So we're essentially doing what's done currently in the evolving clinical practice.

[Alex Thompson, Analyst at Stifel]

Great. Thanks.

[Operator]

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

[Michael Yee, Managing Director at Jefferies]

Hello? 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? I know that if you actually go look back at some of the TEPEZZA post-marketing studies, that there's some commentary and analysis around how patients have some of this hearing loss already, and there's factors already going on with some of these TED patients in the background. And so just trying to think about how you can screen or 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?

[Michael Yee, Managing Director at Jefferies]

I know there were some uncertainties about going directly into phase 3, so just talk a little bit about your confidence there or anything else that you need to do in order to start the phase 3 for subcu? Thank you.

[Steve Mahoney, President and CEO at Viridian Therapeutics]

Yeah, great. Thanks. Thanks, Mike. Let me take the second one first, and then I'll ask Tom Ciulla to weigh in on the baseline hearing question that you had first. So with respect to the 003 program, excuse me. We did have a positive meeting. We have not received the minutes yet, but we had 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 it 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 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 Ciulla to jump in there, please.

[Thomas Ciulla, CMO at Viridian Therapeutics]

Thanks, Michael. So as we said in the previous answer, adverse events in the studies were reported via the MedDRA terms, which is a standard methodology for reporting patient changes in their health, including hearing. This was done in the TEPEZZA 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.

[Alex Thompson, Analyst at Stifel]

Okay. Thank you.

[Operator]

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

[Gavin Clark-Gartner, Analyst at Evercore]

Good morning, and thanks for taking my questions. Just had two. First, on the Type C Meeting for 003, does the FDA want to see any dose-ranging work in TED patients as part of that pivotal, or do you believe you can start dosing immediately in a blinded pivotal portion of the trial?

[Steve Mahoney, President and CEO at Viridian Therapeutics]

Yeah. 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 feel positive coming out of that meeting and that we are starting our pivotal program, which is what we were guiding to previously, but we've now had that meeting, 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.

[Gavin Clark-Gartner, Analyst at Evercore]

Sounds good. We'll await more details. Are you able to provide any details on how the THRIVE baseline characteristics compare to TEPEZZA's Phase 3?

[Steve Mahoney, President and CEO at Viridian Therapeutics]

Yeah, that's another one, Gavin. I mean, it's a great question, and I totally appreciate it. We're just not there yet. We don't have all that information for baseline THRIVE. 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.

[Gavin Clark-Gartner, Analyst at Evercore]

Makes sense. Thank you.

[Operator]

Your next question comes from the line of Rami Katkhuda with LifeSci Capital. Please go ahead.

[Rami Katkhuda, Analyst at LifeSci Capital]

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 001 in the U.S. and Europe in parallel once all the data is in hand, and how large of an opportunity could that ultimately represent?

[Steve Mahoney, President and CEO at Viridian Therapeutics]

Yeah, I think the epidemiology in Europe is very similar, so we know that to be the case, similar to the U.S., that is. With respect to our ex-US plans, again, that's something we'll probably talk a bit more about later. 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. It will have more to say at a later time.

[Rami Katkhuda, Analyst at LifeSci Capital]

Got it. Sounds good. Thanks.

[Operator]

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

[Analyst]

Hey, this is Adam Ohm 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 mid-2024-ish and THRIVE-2 reading out end of year, what factors are driving a second-half 2025 BLA submission in TED? And is this related to the STRIVE study then? And in that sense, is an interim cut, would that be sufficient from STRIVE for a BLA submission, or does the whole STRIVE trial need to be completed to support the BLA submission?

[Steve Mahoney, President and CEO at Viridian Therapeutics]

Great. Thanks, Adam. Appreciate that question. Yeah, so what's driving the timeline is remember, so we talked about THRIVE-2. That's a top-line readout at the end of the year. So by definition, it's not the completed study, right? So we have to let that we have a follow-up period. There's a total of a 52-week follow-up period, but only 37 weeks post the last dose. So there's a follow-up period that has to be taken into account. It's primarily THRIVE-2 that's driving the timeline. We're not expecting THRIVE to have an impact there. That THRIVE should fit squarely within that timeline. So we feel that that's probably the driver. We don't need all of THRIVE. What you see there is on the ct.gov, you see 212 patients. That's the maximum number that we'll need.

[Steve Mahoney, President and CEO at Viridian Therapeutics]

We can do a data cut as soon as we reach the requisite number for the safety database. And 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-engineer or overset up the STRIVE 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 a safety database that accompanies your BLA submission. So it all kind of is normal blocking and tackling from our perspective, but just kind of take into account 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 2025 filing.

[Analyst]

Gotcha. That makes sense. I guess, can you provide any numbers then on what that threshold is? Then just kind of following up on the STRIVE study too, is this a safety database you expect to leverage then with any potential regulatory path for VRDN-003? Thanks.

[Steve Mahoney, President and CEO at Viridian Therapeutics]

Yeah. So 003, 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 is the max number. We'll see where we end up. The total number is 300, but that includes THRIVE and THRIVE-2 active patients on 10 mg/kg. So we're not anticipating needing the entire THRIVE study. And again, most importantly, THRIVE is not expected to drive the timeline for BLA submission. It's more the THRIVE-2.

[Analyst]

Got it. Thank you.

[Operator]

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

[Gregory Renza, Analyst at RBC]

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 the progress you have made. There are others out there as well, TEPEZZA 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.

[Steve Mahoney, President and CEO at Viridian Therapeutics]

Yeah. So I'll turn this over to Tom in a second. But yeah, I think if you could see that we enrolled, not only did we enroll STRIVE on time, we exceeded enrollment. We had really strong patient demand to drive that all within the month it was 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-1R therapy. So that's a really good sign for us. Don't forget also that we had roughly half of the patients who were enrolled in the US. I know that was a question mark for people. I think we've definitively answered that the US is very we've got the opportunity within the US.

[Steve Mahoney, President and CEO at Viridian Therapeutics]

And then the other half in Europe, where, as I mentioned, the epidemiology is the same. So that might be just a general answer. Tom Ciulla, do you want to talk about how competition for trials is shaking up?

[Thomas Ciulla, CMO at Viridian Therapeutics]

Sure. Yeah. 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. 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 STRIVE, we have an active control. There is no placebo, and we think that's going to drive enrollment there. I think overall, just lots of positivity around our TED portfolio in our trials. I really can't I really can't comment on competitors, but what I can say is just lots of excitement and enthusiasm around our portfolio.

[Gregory Renza, Analyst at RBC]

Great. Thanks, guys.

[Operator]

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

[Julian Harrison, Managing Director at BTIG]

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-1R to 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?

[Steve Mahoney, President and CEO at Viridian Therapeutics]

Yeah, it's the latter, Julian. Yeah, we just don't think 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's taking place. You've got to hit that receptor in order to disrupt that. And so FcRns, the IL-6s, the other modalities are not or other mechanisms, 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 FcRn, we'll take that in different places, as we alluded to, in the deck.

[Julian Harrison, Managing Director at BTIG]

Thank you.

[Operator]

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

[Trevor Allred, Analyst at Oppenheimer]

Hey, guys. Good morning. Thanks for taking the question. So with TEPEZZA 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?

[Steve Mahoney, President and CEO at Viridian Therapeutics]

Yeah. I mean, I think it's hard for us to comment on Amgen sales. I think 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 believed that the market continues to be under-penetrated, which we agree with, and they believe that it's going to continue to grow. Don't forget that growth areas also include the other geographies, the introduction of subQ. And so they've filed in Japan and Europe, which is good, as they're continuing to kind of blaze that trail for us.

[Steve Mahoney, President and CEO at Viridian Therapeutics]

Don't forget, even in the backdrop of all of this, they did close to $1.8 billion or close to $2 billion 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.S. but elsewhere as well. Then we think subQ, particularly our subQ, which we think is potentially going to be best in class, where we can have patients that can access it just by 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. So yeah, we're not particularly worried about IGF-1R being the right place for TED patients, and I think Amgen's going to prove that as well.

[Trevor Allred, Analyst at Oppenheimer]

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?

[Steve Mahoney, President and CEO at Viridian Therapeutics]

Yeah. In our deck, you can see that we've got some healthy volunteer data that's pegged to the second half of 2025 for that 006 program. It's a little ways out, 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 008 non-human primate data. In other FcRns, that has proven to be pretty translatable. So we're pretty excited to see that. We saw great humanized mice data for 008, 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.

[Steve Mahoney, President and CEO at Viridian Therapeutics]

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

[Trevor Allred, Analyst at Oppenheimer]

Okay. Great. Thanks, Steve.

[Steve Mahoney, President and CEO at Viridian Therapeutics]

Great. Thank you.

[Operator]

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.

[Steve Mahoney, President and CEO at Viridian Therapeutics]

Great. Thank you, operator. 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. 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.

Participants

Executives

• Louisa Stone, Manager of Investor Relations
• Steve Mahoney, President and CEO
• Thomas Ciulla, CMO

Analysts

• Alex Thompson, Analyst at Stifel
• Gavin Clark-Gartner, Analyst at Evercore
• Gregory Renza, Analyst at RBC
• Julian Harrison, Managing Director at BTIG
• Laura Chico, Analyst at Wedbush
• Michael Yee, Managing Director at Jefferies
• Rami Katkhuda, Analyst at LifeSci Capital
• Trevor Allred, Analyst at Oppenheimer
• Analyst