All right. Good afternoon or evening, I guess, everyone. Thank you for those of you in the room, and also those of you on the webcast. If you're in the room, we are, I think, the last thing between you and the cocktail hours, so especially appreciate you being here. My name is Faisal Khurshid. I'm one of the Senior Biotech Analysts at Jefferies, reporting live here from the Global Healthcare Conference in New York. Really pleased to have this today, the management team of Viridian Therapeutics, Steve Mahoney, CEO, Shan Wu, CBO. Just to kick it off, Steve, can you start by introducing the company?
Sure. Great. Thank you very much for having us. Viridian Therapeutics, we are developing drugs for thyroid eye disease and other autoimmune conditions. Just very briefly, we have a PDUFA date coming up for our first commercial product. The PDUFA date is 30 June 2026, we're right around the corner. We have an IV program for veligrotug. The PDUFA date applies to that, we can talk about the positive outcomes of those phase III studies as we march towards commercial. We also have a subcutaneous, recently a top-line readout for both active and chronic thyroid eye disease, both positive studies. We're marching towards BLA submission in Q1 of 2027 for that program. We have announced recently a TSHR program that will have applicability in both thyroid eye disease and Graves' disease. Finally, we have an FcRn portfolio where we're in first-in-human studies.
We'll have more data in the latter half of this year. A lot going on, and we are turning the corner towards commercial.
Excellent. With the PDUFA date approaching just in a few weeks now, can you talk to us about launch preparedness and what investors should expect in terms of, is this a day one kind of a launch, or what does the scale-up of the launch look like?
Yeah. Operationally, the teams are all ready to go. We have the field team hired, largely trained until we see the final label. We have a medical affairs team that's been in the field for quite some time. Patient support services, that team is also built. All the systems that are necessary to support supply chain commercial product is already in the U.S. We are in good position to launch as soon as we get approval.
Got it. Then with respect to payer discussions related to veli, can you update us on how that is going and what gives you confidence and what should give investors confidence on that?
Yeah, we've been doing payer research for quite some time, several years now. Recently, at the beginning of this year, we turned the corner to being able to do the pre-approval information exchanges with payers where we actually get to show them the target profile for the product and get their reaction to that. That has been very productive. The guidance that we've been given as an outcome of those conversations is that if we can price at a parity level with the existing current therapy that's available, then we could expect parity coverage, which is a great place to be because they're at 85% of covered lives currently after several years on the market. We get to step into those shoes once we work through the process. It's a great infrastructure to step in with respect to payer feedback.
Those information exchanges and the research have gone very well and suggestive of our price.
Got it. Do the parity access policies, do those allow for redosing of an IGF-1R?
They currently do not allow for that, but there are retreatments that do take place that are currently in the market. There are paths to be able to do that. We see that as an additional commercial opportunity. We did talk recently about our plans to generate data in that setting as well, which will only help.
Got it. In terms of what would be needed to enable that from an access perspective, can you describe what that would entail? Is that dependent on additional data, or would you be able to negotiate that just with the package that you have?
Retreatment already occurs with TEPEZZA, even though it's not in a particular policy. Well, most policies are. There are few exceptions to that. They are successful in getting retreatment at certain physicians' offices and certain hospitals, so we'd still have the same ability to do that.
Got it, okay. I think even regardless of retreatment, there is meaningful headroom for growth within the TED market. Could you just remind us the active TED market, how large that is and what proportion of that TEPEZZA is currently penetrating on an annual basis?
Yeah. It's single-digit penetration with TEPEZZA currently in the market. Our view is that that is more of a TEPEZZA profile problem than it is the TED market itself. Because it's so under-penetrated, there are a number of patients that are sitting on the sidelines. We think that veligrotug, our IV program, can unlock a lot of that in terms of being an attractive option for patients, which is a pretty exciting prospect for physicians and patients at this point. We would expect, we have our chronic data, which we expect to be in our label. That chronic data was quite robust, and we saw really good responses on proptosis, which is a bulging of the eyes and diplopia. We expect to see some element of that expansion in the market there and drive better penetration.
Obviously when we introduce our sub Q, we would expect even better penetration expansion in the market.
In terms of the current existing market as well, it's important to remember that thyroid eye disease is a new start market because it's a fixed course of treatment. Every patient that comes onto therapy is new to therapy. Given the low penetration of the current therapy, most patients have not previously been on therapy. Whether it's active patients, active TED or chronic TED, we do expect the profile of veli, which we've talked about before, having received breakthrough therapy designation from the FDA as well as priority review from the FDA. This is a very compelling product profile that we think will be very competitive in this new start market, where every patient gets to choose the optimal drug therapy for them.
We've talked about some of these differentiation points before with the veli profile, namely rapid onset of treatment effect, which is not the current experience of physicians. After just one infusion, the majority of active patients already had a proptosis response. Diplopia resolution and response on both the active and the chronic side. Veli is the first drug to have demonstrated statistically significant improvements on diplopia and complete resolution of diplopia in that chronic setting. Of course, we get to provide all of this with a shorter duration of treatment, not just shorter infusions, each one about half the time as the current therapy, but overall full course of therapy in only 12 weeks versus the existing 21 weeks, almost six months. That makes a lot of difference to patients. Again, in this new start market, we think we have a very compelling profile.
Got it. Then in terms of the single-digit percent penetration of the existing IGF-1R therapy, can you remind us or walk us through what are the numbers that underlie that?
Yeah. There's 20,000 patients per year who are diagnosed with thyroid eye disease. The acute or active form of the disease lasts two years, so that's basically a 40,000 incident patient population every year. We think that TEPEZZA is being used for 6,000 or 7,000 patients per year. You could see the math just based on the incidence population alone. When you graduate from the active form of the disease and you get past that two-year period, that's usually considered the chronic phase of the disease. Since it's autoimmune disease, there is a lot of patient variability. Even within the chronic population, you have chronic stable patients they very well might have proptosis and diplopia, but they're not complaining of the inflammation and the redness and the pain that goes.
You might have patients in that chronic population who are more like active patients in terms of their symptoms with the proptosis and the diplopia. There's a lot of variability, and I think there's a lot of opportunity for our program of veligrotug to come in and help offer treatment options for these patients.
Great. How should we think about the timeline from approval date to scale-up of revenue growth, given some of the factors at play here, like insurance approvals, getting the J-codes in place, the ophthalmologist getting the infusion center on board, these different steps that need to happen before you get dollars in the door?
Yeah. Again, we're ready to go to launch, so we can start accepting enrollment forms relatively soon after we get approval. What'll happen is patients will come in. It's a prior auth. It's a high-priced biologic, so TEPEZZA has the same prior authorization process that they need to go through. That can take 60 to 90 days currently. We would expect that we'll work through that process. It is just-in-time inventory as well, so infusion centers, after they get the prior authorization sorted out, they'll order for that first infusion. They won't order for the full course. You'll see just in time, you'll see a revenue lag that comes with that. In terms of J-code, we'll have a temporary J-code to start, and then it takes six to nine months to get a permanent J-code.
We don't think that's going to necessarily play a big factor in this because the infusion centers are used to dealing with temporary J-codes. It's really just a matter of making sure that they are not underwater, that we can set up the payment terms to prevent that. That part is certainly manageable. It's just a matter of, because it's a new start market, as Shan said, and we're not actually having to switch anybody off, it'll be new patients come in, and then they'll start working through that prior authorization period. The first two quarters, we would expect to work through these processes, and then the revenue will start to catch up.
Got it. Is it fair then from the investor perspective that at least for the first couple of quarters of the launch, the investor metric that should matter more would be enrollment forms as opposed to revenue numbers?
Yeah. I think if you take that prior authorization period into account and you're looking at a 60, 90 days from the start of the process to try to get that prior authorization, you would expect that that first quarter is not going to be a revenue story as much as it will be patient enrollment forms and those types of metrics, prescriber engagement, how many prescribers, what's their level of activity. We'll be focused on that, and I think that's very typical. It's entirely typical of a buy and bill market with a high-priced biologic where you have to work through prior authorization. Instead of a revenue focus for those first two quarters, it'll be more of those types of metrics.
When we turn the corner into 2007, that's when we stack up the patients in a way that we start to see all that revenue start to come up.
Got it. Have you guys thought about, with respect to some of those metrics, like enrollments and prescribers, what you would consider a good outcome in the first couple quarters of the launch?
Yeah, we haven't talked externally about what our expectations are. We haven't guided to those. We do think that there's a lot of enthusiasm for this treatment option to be available. This is the first time that there's been an option available since the introduction of TEPEZZA, and Shan talked about the different attributes of the drug with respect to rapid onset of treatment effect, the diplopia outcomes that we saw in the chronic population for the first time, and then obviously the fact that the treatment burden is five infusions versus eight. It's a lot less drug, faster infusion times. We think that package, that profile is going to be attractive to both physicians and the patients.
Great. I want to switch over to talking about the sub Q, but before we switch over to that, maybe we can talk about the pseudo sub Q, the TEPEZZA OBI. My words, not yours. Can I at least get your reaction to the OBI results and also how you think that compares to both of your products and your understanding of where that is from a regulatory process?
Yeah, it's hard to refer to that as a sub Q.
That's why I know.
Yeah. It's more of an infusion pump that's attached to your abdomen. It is pretty sizable. It has to deliver what we think is a 10 mL dose, which takes up to 30 minutes to deliver. It's a big device. It's four inches long, it's two inches thick. It's got a battery, it's got gears. It's pretty cumbersome for patients. I think they had a lot of difficulty enrolling that trial, multiple delays. We think that that, as a commercial product or a commercial profile, is going to be very difficult. In fact, the last on-body device that they have launched or commercialized was the Repatha on-body device, which they have since discontinued in favor of an auto-injector pen, which is exactly what the profile that we've developed.
Our auto-injector pen, just for reference, is the same auto-injector pen that's used by DUPIXENT and a number of other marketed drugs. From a patient experience perspective, it's a lot easier. It's not clear to us We haven't really seen all the data yet. Let's remember that. We saw one proptosis number, a high-level proptosis number, but we haven't seen much since that. We look forward to seeing what that actual profile looks like, if we get to that point. Commercially, we don't think that's entirely competitive with us.
It's also every two weeks for 12 doses, so it takes 24 weeks for that full course of regimen. No matter how you compare it to, whether it's our veli IV, which is only 12 weeks every three weeks, or the ELE subcutaneous auto-injector, as few as three doses, the OBI is more frequent and more doses than either one of those options.
Yeah, makes sense. Let's jump into talking about sub Q ELE. Within the past couple of months, you had the REVEAL-1 and the REVEAL-2 results. Could you just give us a quick recap about how you guys feel about those results now that we're on the other side of that?
I can start there. ELE, as we mentioned, is the drug that we anticipate being in an auto-injector. In both REVEAL-1, which was the active study, and REVEAL-2, the chronic study, we looked at every four weeks or every eight weeks of ELE given to patients. Both studies were exceedingly positive with meeting the primary endpoint and multiple secondary endpoints as well with very low P values. Consistently across both studies, we saw very persuasive proptosis responses in both Q4 as well as Q8. In addition, Q4 also led to meaningful benefits in diplopia for both the active patients and the chronic patients. What we're looking at, and we hear this in the reactions in our market research with physicians, is essentially IV-like efficacy in an auto-injector that is as few as three doses.
We see extreme potential for this profile in bringing a much simpler, much more convenient therapy to patients, and we hear that feedback from physicians as well in our market research. On the active side, we know a number of these patients today are being offered TEPEZZA and turn it down. We think that's a wonderful opportunity for first veli IV, which is a shorter course of treatment, but especially when ELE sub Q auto-injector comes to market. In that chronic patient segment, today, very few of these chronic patients are choosing therapy. Most of this patient population is completely untapped, and what we hear from physicians is something that is efficacious, something that is safe, and something that is simple for these patients will be really motivational for them to come off of the sidelines.
We think the unlock for that is this ELE auto-injector profile that we hope to be able to bring.
We talk about the under-penetration. It's still a $2 billion market with a single product. That's exciting prospect for us to walk into with the IGF-1R mechanism. Based on the REVEAL readouts on the sub Q, our expectation is that when we get all of these commercialized, we will have IV available wherever people fall on the patient spectrum in terms of moderate to severe. If they're towards the more severe end of that continuum, we think they might be IV appropriate. We're really happy to have the veligrotug profile for IV. We expect to advance both Q4 and Q8 weekly in the sub Q.
We could have the IV Q4 weekly for those who have proptosis and diplopia, and then Q8 weekly, which we think will be primarily the go-to dose, Q8 weekly available for patients who have the proptosis who may not have the diplopia. It's a suite of product options for any patient who walks into a physician's office and asks, "What are my options?" Viridian is going to have the answer.
Great. Shan, you mentioned this, in your research, you guys have learned about these patients who are offered TEPEZZA and then turn it down even with an active TED. I hadn't heard that before. Could you just expand on that a little bit of what is driving that and why you believe being able to offer more convenient alternatives can help overcome that?
Yeah. One of the main reasons patients are turning down or not even being offered treatment options to begin with is the burden of the IV treatment course. You think about the demographics of thyroid eye disease patients, primarily women in their 40s and 50s, have full lives, full jobs. It's a lot to ask these patients, even if they have active disease. Even more so when they have chronic disease that they've learned to live with this disease for many years. It's a lot to ask them to come in every three weeks for 60-90-minute infusions for six months of their lives. That's for a patient who even has access close enough to an infusion center. If the patients have diplopia, they may not be able to drive themselves to the infusion center.
We, again, going back to veli IV, I know we're talking about the sub Q here, but the veli IV is a incremental step to make that process easier for patients. With a auto-injector with ELE, we think that this is going to be the game changer for patients, active or chronic, because it's something that they can just go home and take, again, in as few as three doses with the Q8 weekly arm. It's self-administered, a commercially validated auto-injector that's been approved for about a dozen other indications and super simple one step, a few seconds in an auto-injector.
Great. As we look at REVEAL-1 and REVEAL-2, like I said, REVEAL-1 underperformed the IV comp a little bit. REVEAL-2 was right there in line with it. Can you talk to us about how you guys interpret those results?
Yeah. Maybe I'll start, Shan. Just a reminder, positive study, highly stats sig in the primary endpoint, the clinically meaningful responses in the secondary endpoints. Positive study, it captures the vast majority of IGF-1R efficacy based on that data in an auto-injector pen. That's where we're headed. When you look at that in combination with the REVEAL-2 study, where that probably exceeded expectations, you could see the profile as it emerges ultimately ends up with IGF-1R efficacy in an auto-injector pen. That's what we're referring to in terms of being the game changer for this market.
Got it. Okay. Shan, do you have something to add?
I'm good.
Okay. With respect to chronic TED, I think investors struggle a little bit with believing that chronic TED is a real market opportunity because the currently approved product has essentially not penetrated that TAM at all. What do you say to that to help build investor confidence that chronic TED, those patients not only exist but can be activated to seek treatment?
We talked about how there are about 40,000 prevalent active patients, and after two years, as Steve said, those active patients enter that chronic phase of the disease. You have this disease for life. The prevalence in that chronic population is much larger than the active population. There are a number of things about our drug profiles, in particular the ELE profile, where we plan to launch with an auto-injector that we think will attract patients to treatment. First of all, we talked about the patients who are currently today turning down the TEPEZZA because of large part due to the burden of the IV treatment. That's low-hanging fruit. Secondly, the prescriber base for the chronic patients are for a lot of these patients, more endocrinologists, general ophthalmologists who are managing, for example, the underlying Graves' disease.
They may not necessarily be seeing a TED specialist. These are physicians, the endos and the general ophthalmologists, who are not used to prescribing an infusion drug. By providing them a subcutaneous option to prescribe, we think they would be much more receptive to prescribing a subcutaneous auto-injector for patients. We look at these patients. I mentioned that these patients have been living with the disease for many years in many of these cases. They still have proptosis and may have diplopia, but they've learned to compensate for these symptoms in their lives. It's, again, a pretty hard sell for these patients to consider an IV treatment course.
Where the ELE sub Q auto-injector really makes the difference is the simplicity of it, the convenience of it that will attract these patients off of the sidelines. That's the unlock that is the large market expansion potential that we see in particular in the chronic patient population.
Great. Does having two products here, an IV and a sub Q, for the sub Q specifically, how are you thinking about pricing and access? We started the conversation talking about pricing and access and the kind of once for lifetime reimbursement dynamics that exist on the IVs. Would you try to commercialize the sub Q in a different way to get around that?
No. It's a bit early to talk. We just put the top-line data out for REVEAL. Yeah, I think more to come on pricing for this. It seems that we know that parity coverage comes with parity pricing on the IV side, and I think we could expect the same for the sub Q. We also think that the sub Q has got its own market opportunity, too. I think just we've got to sort through that, but it's a great place to start.
Got it.
We would think about both on the IV and sub Q side that course of therapy as the comparator, as the payers think about potential pricing, because it's the full value that a course of therapy provides to patients. When we talk about parity pricing, we're talking about parity on a per course basis.
Right. Of course. In terms of your cash position, I think you've guided to having what you need to bridge profitability. Could you explain a little bit what assumptions underlie that?
It's relatively simple. Our current cash position, where we guided, we had the $775 million at the end of last quarter. We did a recent financing, added a bit north of $350 million to the balance sheet. Our current cash position, we did a royalty deal in October of last year, so we would expect royalty milestone payments that'll come in through that. We have our Japanese partnership with Kissei Pharmaceutical, that has milestones associated with that as well. Finally, obviously, we're going to be revenue generating in a very short period of time here.
Got it. I believe you recently de-levered part of the balance sheet as well. Can you explain the thought process there?
Yeah. We had a debt facility with Hercules, and it was at a higher rate, so we thought from a cost of capital perspective, we could end that and lower our cost of capital.
Great. I want to shift gears in our last couple of minutes here to the FcRn franchise. Can you help set expectations for the updates that we should see in the second half of the year?
Definitely. We have a really exciting second half of the year coming up. First of all, we have two programs in our FcRn portfolio, 006, which is in the lead, has completed phase I healthy volunteer studies. The data looked great, as we were expecting FcRn-like IgG reductions, as well as sparing of albumin and LDL. That's on track. Our second program is 008. This is the potential to be best-in-class half-life extended FcRn, and we have healthy volunteer data coming second half of this year. This is a program that we're very excited about. It's very difficult to engineer half-life extension into FcRn as a mechanism. We believe we have the only other one that's in development and also in line in terms of the competitive product and timelines. For the healthy volunteer study, we will be looking at IgG reductions.
That's the PD biomarker in this space. Also looking at PK and the sustained PK and sustained PD would be the anticipated outcome here with that half-life extension and generally sparing of albumin and LDL. Historically speaking, primate data has been very translatable for FcRns to clinical data. We feel very confident about the data that is coming up, but look forward to confirming that with healthy volunteer data.
You know investors love to have a bar or a number that they're looking for. On IgG reductions, what should investors consider to be a level that supports moving forward?
Yeah, I think in terms of IgG suppression, the bar that has been set is still being set by VYVGART, which is that kind of 60%-70% IgG reductions with repeated dosing. Others in this space have talked about deeper suppression, and I think the debate, the jury is out on whether deeper is truly better. Being able to hit that threshold of 60%-70% would be a great place to be.
Got it. Excellent. Well, I think that's all the time we have. Really appreciate you joining us today.
Yeah, thank you for having us. Appreciate it.
Thanks so much.