Viridian Therapeutics (VRDN), a biotechnology company specializing in the treatment of thyroid eye disease (TED) and autoimmune diseases, held its first quarter 2024 earnings call, announcing significant advancements in its clinical programs. The company completed enrollment for its Phase 3 trial of the VRDN-001 program for active TED and is preparing to share top-line results in September 2024. They also plan to file a Biologics License Application (BLA) in the second half of 2025. Progress was noted in its subcutaneous VRDN-003 program, following a positive Type C meeting with the FDA, which is set to enter pivotal trials mid-year. Viridian also intends to file an Investigational New Drug (IND) application for its FcRn inhibitor VRDN-006 by year-end and to present non-human primate data for VRDN-008 in the second half of 2024. President and CEO Steve Mahoney highlighted the underpenetrated TED market and the potential of their subcutaneous delivery method. The company concluded the quarter with a strong cash position, maintaining a runway into the second half of 2026.
Key Takeaways
- Phase 3 trial for VRDN-001 in active TED completed enrollment; top-line results expected in September 2024.
- Anticipated BLA filing for VRDN-001 program in the second half of 2025.
- Positive Type C meeting with the FDA for the subcutaneous VRDN-003 program, with pivotal trials starting mid-year.
- Plans to file an IND for VRDN-006 by year-end; non-human primate data for VRDN-008 due in the second half of 2024.
- The company ended the quarter with $613 million in cash, ensuring a runway into the second half of 2026.
Company Outlook
- Viridian Therapeutics is on track with its clinical program timelines and regulatory milestones.
- The company is preparing for pivotal trials for VRDN-003 after a successful Phase 1 study and a constructive Type C meeting with the FDA.
- The TED market remains underpenetrated, offering significant growth potential for Viridian's therapies.
Bearish Highlights
- The company did not mention any negative outcomes or setbacks during the earnings call.
Bullish Highlights
- VRDN-001 program is progressing smoothly, with potential market expansion for TED treatments.
- VRDN-003 shows promise with positive Phase 1 results and an extended half-life.
- The financial position of the company remains strong with sufficient capital to support ongoing operations.
Misses
- There were no reported misses or disappointments in the clinical development or financial results.
Q&A Highlights
- CEO Steve Mahoney emphasized the importance of targeting the IGF-1R receptor for TED treatment.
- Mahoney noted Amgen (NASDAQ:AMGN)'s success and year-over-year sales growth in the TED market, suggesting a positive outlook for Viridian's programs.
- The potential of subcutaneous administration of TED treatments was highlighted as a game-changing approach for patient care.
Viridian Therapeutics continues to execute its strategic plan for developing innovative treatments for thyroid eye disease and autoimmune diseases. With a clear path outlined for its clinical programs and a robust financial standing, the company is poised to make significant strides in the biotechnology industry. The TED market, which is seen as underpenetrated, presents a valuable opportunity for Viridian to grow and serve unmet medical needs. With pivotal trials and regulatory milestones on the horizon, Viridian Therapeutics maintains a strong position for continued progress and potential market expansion.
InvestingPro Insights
Viridian Therapeutics (VRDN) has shown a commitment to advancing its clinical programs, as evidenced by the recent completion of enrollment for its Phase 3 trial of VRDN-001. While the company's strategic initiatives appear promising, a closer look at the financial metrics provided by InvestingPro offers a nuanced view of its market position and potential challenges.
InvestingPro Data reveals a market capitalization of $891.6 million, reflecting investor valuation of the company. However, the P/E Ratio stands at -3.16, with an adjusted P/E Ratio for the last twelve months as of Q1 2024 at -4.12, indicating that the company is not currently profitable. These figures are underscored by a significant revenue decline of -82.59% over the last twelve months as of Q1 2024, hinting at potential headwinds in sales performance.
The InvestingPro Tips provide further insights, noting that although Viridian holds more cash than debt, which is a positive indicator of financial health, the company is rapidly burning through cash. Analysts are also anticipating a sales decline in the current year, and they do not expect the company to be profitable within this period. Additionally, Viridian Therapeutics is trading at a high revenue valuation multiple, suggesting that its stock price may be premium-priced relative to its sales.
For readers interested in a deeper analysis of Viridian Therapeutics, there are additional InvestingPro Tips available, which could offer more comprehensive insights into the company's financial health and market prospects. Readers can find these tips by visiting InvestingPro's dedicated page for Viridian at https://www.investing.com/pro/VRDN. To access these valuable insights, use the coupon code PRONEWS24 to get an additional 10% off a yearly or biyearly Pro and Pro+ subscription.
In summary, while Viridian Therapeutics' clinical developments are progressing, potential investors should weigh these advancements against the company's financial metrics and market valuation. The InvestingPro Tips and Data provide a critical lens through which to assess the company's current standing and future outlook.
Full transcript - Signal Genetics (VRDN) Q1 2024:
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. [Operator Instructions] 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: 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. I’d now like to turn the call over to Steve Mahoney, our President and CEO.
Steve Mahoney: Thank you, 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 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, 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 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. 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. 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. This approved therapy requires eight infusion every three 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 flared-baseddisease, 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. 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. 001, which is delivered intravenously, and 003, which is delivered subcutaneously with the potential for self administration. As you can see here, 003 and 001 have the same binding domain and we expect them to bind IGF-1R similarly. They differ because 003 is engineered to have an extended half-life, which we have shown to be 40 to 50 days in healthy volunteers, which is 4 to 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 have – 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’ve already shown robust clinical activity with 001 after just two infusions in a Phase 2 clinical trial in active TED. 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 TEPEZZA clinical trials after two 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 two 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 just two infusions, 001 meaningfully reduced disease burden across each disease point as well. On Slide 14, VRDN-001 was also well tolerated in chronic TED patients. Based on this Phase 2 data, we believe that the clinical regimen of VRDN-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 trial for VRDN-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’re 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 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. 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 VRDN-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 VRDN-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. We are very excited about bringing forward VRDN-001 as a potential option for patients, which could mean significantly less drug for patients, fewer visits than the infusion center, lower volumes and less infusion share time. Our next program, subcutaneous VRDN-003 will take this differentiation even further with the possibility of lower frequency subcutaneous administrations and potential for at home self administration using auto injectors. We know from market examples that a later entrance subcu therapy can convert meaningful portions of an existing IV market. 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. 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 VRDN-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 VRDN-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 data set from our Phase 1 healthy volunteer study of VRDN-003 to assess PK and PD. The update here is the inclusion of the last cohort, cohort 5, where participants receive two doses of 003 28 days apart. The data confirms the differentiated PK and PD for VRDN-003 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-1. On Slide 20, you can see that the subcutaneous VRDN-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 VRDN-003 that showed that or predicts that three potential dosing regimens are available to us. VRDN-003 every eight weeks, every four weeks and every two weeks could achieve or exceed the exposure levels of VRDN-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. This gives us a lot of optionality as we move towards our pivotal studies for VRDN-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 VRDN-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. The first FcRn inhibitor, efgart or Vyvgart is approved for myasthenia gravis and is in registration for CIPD, 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, VRDN-006 and VRDN-008. With VRDN-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. We are on track to submit an IND for VRDN-006 by the end of this year and look forward to sharing more about the program in the future. Next on the right is VRDN-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 VRDN-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 VRDN-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. In addition, by aiming to improve the duration and depth of IgG suppression with VRDN-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 VRDN-001 and VRDN-003, reflecting the work that we’ve completed during this quarter. 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, ending the quarter was $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. [Operator Instructions] And the first question comes from the line of Laura Chico with Wedbush. Please go ahead.
Laura Chico: 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: 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 TEPEZZA is similar to TEPEZZA 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 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. With respect to your question on STRIVE, yes, I mean, look, the 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 three to one, 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: That does. Thanks, Steve.
Operator: Your next question comes from the line of Alex Thompson with Stifel. Please go ahead.
Alex Thompson: 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 TEPEZZA study today with more focused hearing, measuring, if that’s more of a par for safety 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 VRDN-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: 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 end point. With respect to your question on what would – it sounds like you’re asking what is TEPEZZA post the clinical trials and what that real world experience is. Yes, that is all part of it. 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 Ciulla, do you want to just explain how we’re approaching our reporting of AEs in the same way that depends a bit.
Tom Ciulla: 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’re recording adverse events using [indiscernible] 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 in their pivotal trials. We’re also assessing audiometry as is done in clinical practice at baseline and pre-specified points. So we’re essentially doing what’s done currently in the evolving clinical practice.
Alex Thompson: Great. Thanks.
Operator: Your next question comes from the line of Michael Yee with Jefferies. Please go ahead.
Michael Yee: 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 somebody’s 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, 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: Yes, 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 VRDN-003 program, excuse me, we did have a positive meeting. We have not received the minutes yet. So – but we had a positive meeting and we are reiterating our guidance that we are going to start a pivotal program mid-year 20 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 Ciulla to jump in there, please.
Tom Ciulla: Thanks, Michael. So as we said in the previous answer, adverse events in the studies reported via the [indiscernible] standard methodology for reporting patient changes in their health, including hearing. And 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.
Michael Yee: Okay, thank you.
Operator: Your next question comes from the line of Gavin Clark-Gartner with Evercore. Please go ahead.
Gavin Clark-Gartner: Good morning and thanks for taking my questions. Just had two. First, on the Type C meeting for VRDN-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 like a blinded pivotal portion of the trial?
Steve Mahoney: 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. 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: Sounds good. We’ll await more details. And are you able to provide any details on how the THRIVE baseline characteristics compared to TEPEZZA’s Phase 3?
Steve Mahoney: 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. 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: Makes sense. Thank you.
Operator: Your next question comes from the line of Rami Katkhuda with LifeSci Capital. Please go ahead.
Rami Katkhuda: 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 VRDN-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: 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. 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. It will have more to say at a later time.
Rami Katkhuda: Got it. Sounds good. Thanks.
Operator: Your next question comes from the line of Derek Archila with Wells Fargo. Please go ahead.
Unidentified Analyst: Hey, this is Adam on for Derek. Thanks for taking our questions today. I guess just a couple questions on the timeline really. So given THRIVE is still reading out like mid-2024-ish and THRIVE-2 reading out end of year. What factors are driving a second half 2025 BLA submission in TED? 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: Great. Thanks, Adam. Appreciate that question. Yes. 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 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, 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. And there is a – there are moving parts that go with that in terms of you can have dropout rates in your THRIVE and THRIVE-2. So we kind of over engineer or over set 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 safety database that accompanies your BLA submission. So it all kind of is normal blocking and tackling from our perspective, but just got to 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 finally.
Unidentified Analyst: Got you. That makes sense. And I guess, can you provide any numbers on what that threshold is and then just kind of following-up on the STRIVE study too? Is this the safety database you expect to leverage then with any potential regulatory path for VRDN-003? Thanks.
Steve Mahoney: Yes. 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’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 mg/kg. So we’re not anticipating needing the entire STRIVE study. And again, most importantly, STRIVE is not expected to drive the timeline for BLA submission. It’s more the THRIVE-2.
Unidentified Analyst: Got it. Thank you.
Operator: Your next question comes from the line of Gregory Renza with RBC. Please go ahead.
Gregory Renza: 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. 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.
Steve Mahoney: 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’s a – 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 were enrolled in the U.S. I know that was a question mark for people. I think we’ve definitively answered that the U.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. Tom Ciulla, do you want to talk about how competition for trials shaking out?
Tom Ciulla: Sure. Yes. 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., and I can tell you there’s a lot of excitement about our 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. 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.
Gregory Renza: Great. Thanks, guys.
Operator: Your next question comes from the line of Julian Harrison with BTIG. Please go ahead.
Julian Harrison: 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 and 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: Yes, it’s the latter, Julian. Yes, we’re – we would – we do – so 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 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 will take that in different places, as we alluded to in the deck.
Julian Harrison: Thank you.
Operator: Your next question comes from the line of Trevor Allred with Oppenheimer. Please go ahead.
Trevor Allred: 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: Yes. I mean, I think it’s hard for us 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 that the market continues to be underpenetrated, which we agree with, 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 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. And then we think subcu, particularly our subcu, 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 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.
Trevor Allred: 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 it – with the IND coming in the year, maybe second half to mid-year 2025?
Steve Mahoney: Yes. 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 that has 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. So we’re looking forward to FcRn moving forward. We got a lot to do there.
Trevor Allred: Okay. Great. Thanks, Steve.
Steve Mahoney: Great. Thank you.
Operator: At this time – 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.
Steve Mahoney: 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. 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.
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.