Viridian Therapeutics at Jefferies Conference: Strategic TED and FcRn Developments

On Wednesday, 19 November 2025, Viridian Therapeutics (NASDAQ:VRDN) presented at the Jefferies London Healthcare Conference 2025. The company outlined its strategic advancements in the Thyroid Eye Disease (TED) and FcRn portfolios, highlighting potential commercial launches and financial strategies. While optimistic about their pipeline and market opportunities, Viridian also addressed challenges and competitive benchmarks.

Key Takeaways

• Viridian anticipates a mid-2026 commercial launch for its IV program, veligrotug (Veli), for TED, with a subcutaneous version expected a year later.
• The TED market in the US is valued at $2 billion annually, with Viridian targeting the moderate to severe patient population.
• The company's FcRn portfolio includes VRDN-006 and VRDN-008, with ongoing studies and expected data readouts in 2026.
• A recent royalty deal and equity raise position Viridian to achieve profitability based on current cash and projected revenues.
• Consistent FDA interactions have been pivotal, with a priority review requested for Veligrotug's BLA submission.

Financial Outlook

• Viridian completed a royalty deal and equity raise in October, strengthening its financial position.
• The company expects to reach a break-even stage based on cash on hand and anticipated revenues.
• Profitability is projected as the company advances its TED and FcRn portfolios.

Operational Updates

• The Veligrotug IV program for TED has completed Phase 3 trials, with a BLA submitted in October and a potential launch in mid-2026.
• The subcutaneous program (VRDN-003) aims to offer a self-administration option, with pivotal study enrollment completed and data expected in Q1 and Q2 2026.
• Viridian plans a modest commercial launch infrastructure, targeting 2,000 core prescribers with a team of 50 reps.

Future Outlook

• Viridian's FcRn portfolio includes VRDN-006, which shows promising IgG suppression, and VRDN-008, with an IND filing expected by the end of 2025.
• Healthy volunteer data for VRDN-008 is anticipated in the second half of 2026, with the potential for once-a-month dosing.
• The company is exploring large markets like Myasthenia Gravis and CIDP for its FcRn products.

Regulatory Interactions

• Viridian has maintained constructive conversations with the FDA's ophthalmology division, which remains stable despite organizational changes.
• The company requested a priority review for Veligrotug's BLA, supported by its breakthrough therapy designation.
• Viridian expects to know the status of BLA acceptance and review timeline by the end of the year.

Q&A Highlights

• Clara Dunn from Jefferies inquired about the TED and FcRn portfolios, with Viridian expressing confidence in their market strategies and clinical progress.
• The company emphasized its ability to enroll trials on time, despite the presence of competing products.

Viridian's comprehensive presentation at the Jefferies Conference underscores its strategic focus on innovative treatments for TED and FcRn-related conditions. For a detailed analysis, readers are encouraged to refer to the full conference call transcript.

Full transcript - Jefferies London Healthcare Conference 2025:

Clara Dunn, Biotech Analyst, Jefferies: Good morning. Thanks, everyone, for joining Jefferies Healthcare Conference in London. My name is Clara Dunn, and I'm a biotech analyst in Jefferies. Sitting next to me, I'm joined by the team of Viridian Therapeutics, Stephen Shan. Welcome.

Stephen Shan, Viridian Therapeutics: Thanks, Clara.

Jan, Viridian Therapeutics: Thanks, Clara.

Clara Dunn, Biotech Analyst, Jefferies: Before we kick off, maybe I will hand it to you to just give the audience here an overview of Viridian. We have kept you busy for the past years. A lot going on. It's a very exciting time.

Stephen Shan, Viridian Therapeutics: Yes, we have a lot going on, that's for sure. Thank you, and thank you for having us here today. Our approach at Viridian is to take de-risked science into large, established markets. By way of example, we have an IGF-1R program that's a validated target for thyroid eye disease. We also have an FCRN portfolio, which is a validated target for a number of diseases so far, with more to come. I'll start with the thyroid eye disease portfolio, or TED. Again, it's IGF-1 receptor inhibition that has been shown by the only one currently marketed approved product where that inhibition results in a reduction of key symptoms associated with the disease, including proptosis, which is a bulging of the eyes, diplopia, or double vision. There is an endpoint called clinical activity score, which is essentially a measure of inflammation and pain associated.

This is a very debilitating disease. It affects women primarily in their 40s and 50s. It's largely a commercial insurance market that we're working our way into. It's currently annualizing at a $2 billion in the United States alone as a market size. Obviously, we think we can grow that with our two programs in that part of the portfolio. We have an IV program, which is with a drug referred to as veligrotug, or Veli. And then we have a subcutaneous program too. Just a status update on where we are with those two programs. Veli is the IV program. We finished our phase threes. We got breakthrough therapy designation this past spring. We just recently, at the end of October, filed our BLA, despite the government shutdown, I'll add.

We have submitted the BLA because we got breakthrough therapy designation, in spite of the fact that the currently marketed approved product also inhibits the same target. We believe there is an increased odds for our ability to get priority review from the FDA, which, if we do achieve that, means we could be commercially launching the product in mid-2026. That is very exciting. The commercial story is right around the corner. We are preparing for commercial launch on that basis, on that timeline basis, because we do think priority review is a real possibility. Behind that, we have our subcutaneous program. The subcutaneous program is essentially the same antibody, the only difference being that it has been half-life extended. It has been formulated to fit into a commercially validated autoinjector pen. It is the same pen that is used by Dupixent and several other marketed approved products, so a validated device.

We announced back in September that we had finished enrollment in the pivotal studies for that program. That is an exciting option for patients, as you can imagine, because the currently available products and our first product are going to be infusions, whereas the subQ program will enable us to mail an autoinjector to a person's home, and they can self-administer on their own schedule. That would be, in our view, a game changer and a massive potential to expand the market quite significantly. We have a pretty great setup from the standpoint of the timing. We think the programs are approximately a year apart. With commercial launch of Veligrotug this coming mid-2026, that would allow us to build a relatively small infrastructure. Horizon launched the currently marketed approved product, started with only 50 reps.

It's a concentrated prescriber base of 2,000 core prescribers, which is certainly reachable with a relatively small infrastructure. When the subQ program comes along, assuming everything goes well with the data, we can simply plug that subQ program into that already built infrastructure. We think that'll be an accelerator for that launch. We are in great shape on the TED portfolio side. We are hitting all our timelines. Our clinical enrollment has been robust. We've enrolled well over 500 patients in TED trials this year. We are, in fact, the only company who has been able to enroll trials on time or early. In each case, we have over-enrolled those studies with very good representation of enrollment in the United States, despite the fact that there is a marketed product commercially available for folks.

In fact, our subQ trial, Reveal 1, in the active population, 67% of the patients who enrolled were in the United States, and for Reveal 2, 56% in a bigger trial. We think that's really good read-through for commercial demand. That's the TED portfolio. We also have an FCRN portfolio, which I'm sure people are familiar with. Vyvgart has been doing an amazing job of setting that market. There are many, many indications that are possibly addressable with an anti-FCRN approach. Our portfolio, we have two programs, VRDN-006, which is a fragment approach, very much like Vyvgart. We like the fragment. We think there's something special about the efficacy and safety profile generated by the fragment. We are completing a first-in-human study there.

We have recently disclosed that our 006 program looks very much like it should with respect to IgG suppression thresholds and the ability to spare albumin, so you do not see the LDL spikes that prior FCRN programs had shown. Obviously, from a safety profile, that's not what you want to see. We are very happy that our 006 program is very much in line with the class. We also have a 008 program, which is a program that is designed to be half-life extended. If you're familiar with FCRNs, because of the clearance mechanism, they are dosed frequently, once a week. We think our half-life extended approach could possibly extend that. We showed a model where we could get out to once-a-month dosing in the NHPs.

We're working through, we have an IND on file for, or we are filing our IND, excuse me, at the end of this year, and we'll generate our healthy volunteer data in that program this coming year in 2026. That will, excuse me, that's a pretty exciting prospect. We really look forward to seeing that. That could be a potential game changer for the FCRN franchise and be a really great development for patients. Just finally, we're very well capitalized. We just recently, we did a royalty deal and an equity raise back in October. With our cash on hand, plus our anticipated revenues, we have guided that we expect to be profitable on this basis. We're at a break-even stage based on the cash. With that, that is my summary.

Clara Dunn, Biotech Analyst, Jefferies: Great, Stephen. Thanks for the summary. Maybe I want to take a step back and just talk about your TED portfolio. You mentioned this is really a large market in the U.S., $2 billion a year with one approved product. How do you see Veli really fitting this space? You also mentioned you expect to really expand the market. Maybe just elaborate, how do you expect to kind of achieve that as well?

Stephen Shan, Viridian Therapeutics: Yeah, I think first and foremost, what's really, really critical to this market is this is a new start market. We do not need to switch anyone off of the currently approved product. It is not chronic dosing. Therefore, it's a one cycle, and patients hopefully are free of their symptoms for significant periods of time. We, because it's a new start market, we represent a treatment option for every patient that comes in. This is an interesting market because we believe there's a 20,000 patient, just in the U.S. alone, 20,000 patient incident rate annually. Interestingly enough, the recent onset or newly diagnosed patients with those flaring symptoms stay in that state for roughly two years. That's roughly 40,000 patients that are in that state for two years. That replenishes, obviously, on an annual basis.

From a prevalence side, you've got 500,000 people in the United States that have thyroid eye disease, 200,000 of which we believe have moderate to severe. That's our target population is moderate to severe thyroid eye disease. The incident rate, as I mentioned, 20,000. The currently approved product is, as acknowledged by Amgen, still in single-digit penetration of that population, but still annualizing $2 billion. In a new start dynamic, we can step in, represent a treatment option for these patients. What we, you know, I can't do a cross-trial comparison, but from a treatment regimen standpoint, we're a 12-week treatment period. We're a full antagonist, so we believe we have a more potent antibody. We're a 12-week treatment period versus a 21-week treatment period. We are 10 mg per kg versus they're 20 mg per kg.

We have a faster infusion time in the chair. And cumulatively, we put in 70% less drug. You can look at our data from our phase threes, which were the largest studies ever run in thyroid eye disease. The data were fantastic. We were seeing great proptosis response rates. We saw, for the first time ever, diplopia response and resolution in the chronic population, like meaningful response and resolution in that population. The safety profile was as expected, which is generally mild and completely manageable by physicians. It is a great step forward for patients. They are really looking forward to that. We think that because of the way our treatment regimen is structured and because of some of the data that we have been able to generate, we think we can expand the market on that IV basis alone.

Obviously, subQ is a different profile from the standpoint of being able to allow people to administer at home.

Clara Dunn, Biotech Analyst, Jefferies: You also submitted BLA last month. I think we are all very interested to know the possibility of priority review, as you mentioned. How have the FDA interactions been for the past maybe six months? Obviously, there have been some dynamic changes over there as well.

Stephen Shan, Viridian Therapeutics: Yeah, I mean, that's a great question. What's really important for us is that it's not just the last six months with the FDA. This is the team at FDA, the division of ophthalmology is completely intact. They were not impacted by the changes that have been occurring down there. They've been incredibly productive and constructive in our conversations with them. They've been around for years. The leadership is there. The reviewers are there. We’ve had consistent conversations with them over several years now. We feel good about the alignment, and we feel good about the fact that they're still intact and able to review the filing with the context that they've had based on the conversations that we've had over the past several years. We feel very fortunate. Again, they were very helpful in terms of helping us submit the BLA during the shutdown as well.

We think that's all very positive.

Clara Dunn, Biotech Analyst, Jefferies: We also think that we have, obviously, when we submitted the BLA, we requested for priority review. We were granted breakthrough therapy designation back in May of this year, which the breakthrough therapy designation supports eligibility for priority review. We think the odds are in our favor to be able to get priority review. Obviously, can't guarantee it, but we are on that 60-day clock right now after having submitted the BLA and should find out before the end of the calendar year the status of the acceptance of that BLA and the review timeline, which again, we think we have a really good shot at getting priority review, which would mean a mid-2026 potential launch for Veligrotug. Great. Let's also talk about your launch preparation. I mean, you talked a little in your opening remarks.

What kind of lesson, or the most important lessons you've learned from Tepezza's launch in terms of pricing, reimbursement, and patient awareness, physicians' engagement, et cetera, et cetera?

Stephen Shan, Viridian Therapeutics: Yeah, I mean, our perspective on that is we are very fortunate to be coming on the heels of them with respect to patient education on IGF-1R, physician education, payer education. In a lot of respects, they've cleared the way for us to come in and talk about our data and our treatment regimen. We get the benefit of all that type of work that they've been able to do. This was, as I mentioned in the beginning, this was a product that had a spectacular launch. I think it's one of the top 20 launches of all time. They did, Horizon was a small company at the time, 50 reps to start.

They moved up to 80, but they had a pretty broad reach in terms of the prescriber base that they needed to get to and did $800 million in sales in the first year of launch in spite of being doing so in the middle of the pandemic. That is certainly not revenue guidance. I'm just pointing out that there's a lot of excitement around it, and we're able to come in, offer that additional treatment option, and take advantage of a lot of the things they've done. You referenced pricing. We and the fact that pricing goes hand in hand with insurance coverage. They've made a lot of progress, particularly recently in the past year with respect to getting commercial insurance coverage. I think the last time that they spoke about it, they had 85% of commercial plans in the U.S. covering. That was not the case a year ago.

A lot of the obstacles within those plans with respect to step-through steroids and having people work their way through to get to IGF-1R, those obstacles have been cleared out. That is, again, a benefit to us as we step into the space.

Clara Dunn, Biotech Analyst, Jefferies: Great. Maybe let's turn to subQ 003. Your top-line readout has recently been accelerated to Q1 2026 for Review 1 and narrowed to Q2 for Review 2 for both active and chronic. Maybe just help us set the expectation for the upcoming pivotal readouts. What are the strongest evidence that we should feel confident about those two readouts?

Jan, Viridian Therapeutics: Thanks, Clara. On the 003 readouts, we're really excited about the two top-line readouts, phase three, that are coming up in the first half of next year, as you mentioned, Q1 for the active study and Q2 for the chronic study. We've designed both of these studies very similarly to Thrive and Thrive 2 on the Veligrotug IV side. In particular, we're enrolling patients on the chronic side, a very broad CAS inclusion at the initial inclusion criteria so that we're getting the most representative and robust representation of chronic patients across all those CAS scores of zero to seven. Going back to the confidence that we have for the readouts that are coming up, we go back to how we designed the 003 molecule to begin with. We took the Veli IV molecule, which is a full antagonist against IGF-1R, and we engineered into a half-life extension.

What we kept the same from the Veli molecule are the CDRs, the variable domain of the antibody. We would expect, we believe, at the same exposures for the 003 molecule to interact with the IGF-1R target in the same way. When we did this back in the phase I healthy volunteer study, this is a couple of years ago now, what we had at the time were Veli IV data from a phase II study that looked at both 3 mg per kg IV as well as 10 mg per kg IV. Both of these were shown to be very clinically active. What we then did with the 003 molecule is we modeled the PK based on different regimens of dosing, Q4 weekly and Q8 weekly.

What was nice and was a pleasant surprise was the Q4 weekly was able to achieve the C mins of 10 mg per kg IV from that phase two study, which was the data that we had at the time. An even more pleasant surprise was that Q8 weekly could achieve and actually exceed the C mins of 3 mg per kg IV. I mention the C mins in particular because we believe C mins drive the efficacy of antagonistic antibodies. What this gives us is a nice reference point of being able to achieve, we believe, the exposures already of Veli IV levels based on that PK modeling with both Q4 weekly and Q8 weekly subQ dosing. We think both of these exposure levels are de-risked based on the clinical activity that we had already seen with Veli IV.

This sets us up in a really nice way into the Reveal study readouts. As you mentioned, asking about expectations for that, we think in general, the expectations for efficacy are set quite high for IGF-1Rs. Tepezza actually is still the standard of care and the only approved therapy that's available. When we look at what the efficacy is with Tepezza, we reference back to the original pivotal studies that they ran. We think that's the bar for success for 003 as well, is to be able to match that efficacy. Cross-trial comparisons aside, I'm not trying to do cross-trial comparisons, but if you look at the Tepezza registrational trials, they did see a range of proptosis response on a placebo-adjusted basis from about 50%-70% placebo-adjusted proptosis response rate.

We think if 003 efficacy on the primary endpoint of proptosis response is able to be anywhere in that ballpark, that will have met the bar for success for the high efficacy that physicians and patients have come to expect for an IGF-1R.

Clara Dunn, Biotech Analyst, Jefferies: You mentioned the autoinjector. Maybe just tell us what kind of data or potentially a trial you will need for autoinjector to get approval as well.

Jan, Viridian Therapeutics: Yeah, so we plan to launch 003 in an autoinjector, a small volume, very quick pen that is commercially validated. It's been approved in multiple other commercially available products. We think it's de-risked in that regard as well. It's a very typical device development path where we plan to do, or actually we have already opened a bridging study into the autoinjector. All of that will supplement the pivotal trial data and be submitted together in the BLA to the FDA so that the FDA can evaluate the totality of data set. We expect that total data set to be sufficient to support a launch with the autoinjector for 003.

Clara Dunn, Biotech Analyst, Jefferies: For the last two minutes, I also want to talk about the FCRN portfolio. You reported 006 proof of concept data in healthy volunteers. For the next step, how are we thinking about the indication selections for this asset?

Jan, Viridian Therapeutics: Yeah, for both of these programs, 006 and 008, we think the opportunity is really a large one given the number of indications that have the scientific rationale for being able to be addressed with FCRN reduction. The way that we think about it is actually a number of options are available to us, both in the large existing approved markets, for example, Myasthenia Gravis, CIDP, but also, as I mentioned, dozens and potentially more additional indications that FCRN is implicated in. At this point, we have all of that available to us. It is actually very exciting to have two separate programs. There are opportunities here to be able to go into potentially different indications.

Or actually with 008, the next generation, what is half-life extended FCRN, we see this being potentially highly differentiated from a dosing regimen standpoint and could really be a game changer for the FCRN market.

Clara Dunn, Biotech Analyst, Jefferies: When should we expect data from 006 and 008?

Jan, Viridian Therapeutics: Yeah, so 006, we have already characterized the phase one data, which Steve talked about earlier, and it is as we expected, which is a great place to be. 008, we plan to submit the IND end of 2025, end of this year, and we'll have healthy volunteer data second half of next year.

Clara Dunn, Biotech Analyst, Jefferies: Great. Any other milestones you would like to highlight here?

Stephen Shan, Viridian Therapeutics: I think we talked about the potential for priority review at the end of this year coming up real soon. We have our subQ readouts, top-line readouts in Q1 and Q2 for the active and chronic populations. Then we have commercial launch. Then we have FCRN data. We have a lot coming up. 2026 is going to be a big year for us, and we're looking forward to that.

Clara Dunn, Biotech Analyst, Jefferies: We are looking forward to that as well. Thank you, Steve. Thank you, Jan. Thank you.

Stephen Shan, Viridian Therapeutics: Thank you, Clara.

Clara Dunn, Biotech Analyst, Jefferies: Thank you, audience, for joining us here. Enjoy the rest of the conference. Thank you.

Jan, Viridian Therapeutics: Thank you.

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