Conference Transcript - CLDX 2026-04-14

Joe Pangenis, Analyst — H.C. Wainwright

Okay. Hi, everybody. Welcome to the next session of H.C. Wainwright's Annual Inflammation and Skin Disease Conference. My name is Joe Pangenis, Managing Director and Senior Research Analyst here at the firm. Very happy to have with us Celldex. Presenting for the company is Dr. Diane Young, Senior Vice President and Chief Medical Officer. Terry Lawler, Senior Vice President, Chief Commercial Officer, recent nice addition to the company. And of course, Sarah Kavanaugh, Senior Vice President of Corporate Affairs at Celdex. So guys, thank you very much for being here. And, you know, look, having covered Celdex for well over 15 years plus, it's so completely gratifying to me to really see the progress that you've made and really being on the cusp of potential approvability of barzololumab, which will be the bulk of our conversation today in about a year or so, depending on how the studies go with urticaria being in phase threes. And with that said, I, of course, want to make sure we dive right in based on time today and hit the salient points on the time that we have. So again, ladies, thank you very much for being here. Thank you for having us. So with Barzo being your lead asset, obviously, and it's been in a nice development program, I think there needs to be a quick reminder, plus a little bit of a compare and contrast. I know sometimes it's difficult to talk about the competitive landscape, but this is at the forefront of investor minds, obviously, as to the antibodies mechanism of action, and also, you know, relative, I said, to the competitive of assets also targeting urticaria and beyond? Yeah, so I'll start. So barzavolimab is an

Diane Young, Other

anti-kit monoclonal antibody, and this is a novel mechanism of action that is really targeting the root cause of the disease, the diseases we're dealing with, which is mast cells. So kit is a receptor that is expressed by mast cells, and it's really critical for their function and survival. So barzavolumab, with a unique mechanism, blocks the KIT receptor and leads to mast cell depletion. And this is a novel mechanism. The other drugs that are out there for urticaria are targeting things that are triggering the urticaria, you know, various IgE, or they're targeting mediators such as IL-4, IL-13 with Dupixin. But barzavolumab is going for the mast cells itself. So it's, you know, a very broad and novel mechanism.

Joe Pangenis, Analyst — H.C. Wainwright

No, that's really helpful. And of course, there was additional news with Dupixent today. So maybe we'll touch on that a little bit later. Look, I guess part of the bulk of this conversation and leading up to the current phase threes, you know, data are always key. I was hoping we can get a bit of a review of the phase two data set, or I should say even broad phase two data set in CSU as well as COLD, U, and SD that were so incremental in driving your quickly progressing pivotal programs.

Diane Young, Other

Yeah, so we conducted phase two studies in CSU and Sindhu. In CSU, we studied antihistamine refractory patients, including those who had had prior biologics and other advanced therapies. And really, in that study, we saw that barzavalumab has rapid, profound, and durable effects on urticaria symptoms. We reported very high complete response rates. So we had up to 51% complete response rate at week 12, meaning no itch, no hives. When we continued the therapy out to week 52, that went up to 70%. And even off therapy for seven months, we still had a 41% complete response rate. And that's really unprecedented. And in addition, the other endpoints in the study, notably angioedema and quality of life, also showed, you know, really dramatic improvements. And then we did a study in inducible urticaria, which is urticaria, which is triggered by various conditions. And there we studied cold urticaria and symptomatic dermographism. And the endpoint for those studies is provocation tests and the ability to provoke the urticaria. And we saw that we had statistically significant benefits in terms of patients having negative provocation tests on barzovalumab. And that was in both cold urticaria and symptomatic dermographism with both doses of barzoval in the study versus placebo. And as well, we showed benefits in terms of urticaria control test and quality of life. So, you know, really nice clinical responses as well as the provocation tests.

Joe Pangenis, Analyst — H.C. Wainwright

Now, that's fantastic. And, you know, maybe just a little more color than I know, you know, will help Terry's conversation later as her, you know, job gets more broad as well, is the durability component. I'm hoping you can just maybe dive into that a little bit more, especially when we consider the landscape.

Diane Young, Other

Yeah, so the durability, so two parts of that. In the CSU study, we treated patients for 52 weeks, and then they went off therapy for six months. And in that study, we observed that patients really, you know, maintained their response. And when they were, you know, when they were recurring, they were coming back at a milder level of disease. And this really raised, this is totally novel. You know, other drugs in this space don't do this. And it raises the question about whether we're seeing some sort of disease modification here. And the companion to the durability that we saw in the CSU study was in the Sindhu study, we had a different design where after 20 weeks of treatment, they went off therapy. And then if they recurred, we allowed them to go back on to Barzavolimab. And part of the results of that study that we recently presented were that with retreatment, you get the exact same response as you had at the beginning. so that really you know sets us up with some some flexibility you know in the future for as to how

Joe Pangenis, Analyst — H.C. Wainwright

physicians will treat treat patients no got it and i guess you know again with the potential for disease modification here you know not to repeat or re-emphasize but i and i don't want to overstate unless it's really true there is you know targeting kit as the core target here you know as the key area of disease. So I'm not, you know, having you to repeat that, but I mean, it just seems like pretty obvious, not real scientific term there, but it's a nice correlation.

Diane Young, Other

Yeah, I agree. It's really interesting and it's definitely differentiates us from the other agents so far. Got it.

Sarah Cavanaugh, Head of Investor Relations

It's interesting to add to that when you look at the biology, tryptase is a marker of mast cell burden. And for these patients on study, at the end of study, if their disease is back, it's typically also milder in form. And when you look at their tryptase, their tryptase levels are now much more normalized, whereas when they were at baseline, they were much higher. So, you know,

Joe Pangenis, Analyst — H.C. Wainwright

we're seeing that change in biology too. Now that's perfect. I appreciate those comments. And so, look, as I alluded to earlier, all eyes right now are on the fact that you guys are a pivotal stage company that brings on a lot more eyes on the investor front. And, of course, you know, building interest in the physician community that might not necessarily know about Barzo or, you know, broadening the experience there. And again, you know, bringing in Terry's future responsibilities and that what she's also building now. So with that said, your phase three CSU data are due approximately in the fourth quarter. Of course, in biotech, things can waver a little bit. One of the things that we've been impressed with is that this study has enrolled quite And I was wondering, so first, can you comment on that? But even more importantly, can you discuss the endpoints of the study and how we, the street, and even physicians should benchmark, you know, the success of this study and what really represents a positive outcome, you know, beyond the, of course, required STAT-SIG?

Diane Young, Other

Yes, so we were very pleased that our enrollment went so well and that the, you know, really completed, you know, six months of where we had guided and really faster than the competitors trials. And these were very large studies. And these are the largest studies that have been conducted in this antihistamine refractory population, including patients refractory to advanced therapies. And, you know, we think that this really speaks to the unmet need out there. You know, there are clearly a lot of patients in need of new therapies. There's also great enthusiasm for the drug. You know, we were very gratified by the participation of both allergists and dermatologists in the study. So we were really pleased with how well it went. I would also say that our operations team did a fantastic job, and we had a CRO who has had experience in this area. So all these things really work together. In terms of the endpoints, so our endpoint is the mean change from baseline in urticaria activity score 7, which is the daily diary that patients do. And this is a traditional endpoint for CSU, as you mentioned. We have, you know, we've sized the study. It's 90% powered to see a 10-point change in the UAS 7 between placebo and active. That being said, and I would say it's 90% powered to do that in the omelizumab refractory group. So it's overpowered in a sense for the larger group of the study. But I would say that when we are looking at the results, we would really like to see similar results to what we saw in Phase 2, which we believe are differentiating. So we're really looking for high complete response rates because we believe that complete response is the most important thing for patients. No itch, no hives. And we're also interested in looking at the group of patients who had prior omelizumab and were refractory to omelizumab because that's really an area of unmet need.

Joe Pangenis, Analyst — H.C. Wainwright

No, that's great. And then, look, I think I'll ask a devil's advocate question, but before I do that, you know, what, if you could just give us a quick reminder of the size and design of the study, because obviously you mentioned about OMA refractory patients as well.

Diane Young, Other

Yeah, so it's a, yeah, so it's that we had 1900 patients on the study. It looked at two different dose regimens of barzvolumab and placebo, has a six-month placebo-controlled period with a 12-week endpoint. and then the patients go on to active treatment for, you know, another 36 weeks. We have tried to keep all elements of the study very similar to what we did to phase two in terms of inclusion-exclusion criteria, endpoints, things like that. The only real difference is that we added a loading dose to our two-dose regimens, and that was based on data that we saw in phase two.

Joe Pangenis, Analyst — H.C. Wainwright

Okay, got it, got it. And then sort of the devil's advocate question, and you see a lot of these, and of course, it's not related, so it's loose analogy here in cardiovascular diseases where, okay, there's a bogey that the street's expecting. So you're talking about being powered for a 10-point difference in UAS 7, and it's like, well, what happens if you come in with a 9, 8, or a 7? And, you know, it's still clinically meaningful and, you know, you could still hit very important numbers. But, again, it's like you always have versus, you know, cardiovascular mace where the street might have wanted a 15 percent impact on survival. And they got a 13 percent, which is still doctors say that's awesome, you know, but the street got disappointed. So I guess, you know, there's this balance that we're trying to hit here, even though the phase two data support, you know, what you're looking for. But again, just playing strictly devil's advocate.

Diane Young, Other

You know, I think, you know, I think, again, we have tried, I think we've tried as best we can to keep everything, you know, as similar as we can to to phase two. You know, you know, we tried to monitor the study very closely going along. But, you know, beyond that, we have to we have to see what happens.

Joe Pangenis, Analyst — H.C. Wainwright

No, of course. I get it. It's like, you know, just it's always to me just the constant balance of, you know, physicians, what they're looking for and, you know, what investors are looking for. And, you know, sometimes they're they're different in the interpretations. But again, just very encouraged about what you've shown so far. So thanks for that. Look, so, Terry, like I said, you've got a lot of things going on in the background since you've joined, obviously, and your position is going to be really growing in prominence, hopefully with, you know, as we look towards potential approval. So phase three data are not too far off. I think a key point to this discussion today and beyond, obviously, is the commercial profile of Barzo. You know, this really also needs to include a view towards the competitive landscape, which we already started to touch on, including Novartis. And like I said, there was additional news today with Depixent and, you know, the role in following drugs like Zolaire. So a little long-winded sort of intro on my part, but I guess from the baseline, what kind of market sizes are we looking at for the two top indications here with regard to CSU and cold?

Terry Lawler, Other

We see significant headroom for growth in these early indications. If we take CSU in the U.S. just as a starting point, approximately 1.8 million people in the U.S. suffering from this disease, but only 32% receiving a proper diagnosis. And that diagnosis can take anywhere from seven months to three years when it happens. So the new entrance into the category, as we've seen with other I&I diseases, typically create a tailwind for both diagnosis, speed of diagnosis, and movement on to advanced therapies. With those tailwinds, we see the worldwide market for chronic urticaria, advanced therapies, so that's CSU and Sindhu, reaching peak sales of $12 billion with potential upside.

Joe Pangenis, Analyst — H.C. Wainwright

Got it. Got it. And I think there's another aspect of numbers here and potential disconnects about how investors view the size of these markets when they hear these indications that they may not have heard about or just learning about. So I think those comments are extremely helpful. And of course, this talks to physicians potential use, but also the understanding of investors, you know, where do you think, you know, Barzo at least initially may fit in the treatment landscape, you know, with regard to targeted populations or stages of disease or what have you, because obviously the phase threes right now, you know, excuse me, include

Terry Lawler, Other

OMA-resistant patients? There are two large and growing patient populations where we believe barzobolimab is really uniquely positioned to address the unmet need. The first of those is in first-line advanced therapy, so patients who are refractory to background antihistamines, who are presenting with severe disease and angioedema. So if we look at the angioedema scores from our phase two data, and not only the overall scores, which were 65% angioedema-free at 12 weeks and that deepening to almost 8 out of 10 patients at one year. When we look at the placebo-adjusted delta on angioedema-free, it's more than double what's been seen with any historical therapies before us. And when we quantify that first-line opportunity, in our phase two studies, we had 36% of the population with both severe disease and presenting with angioedema. So even if we assume the broader population is, say, half as severe as those who enrolled in our studies, that's still 15% of the first-line market. And keep in mind, for angioedema, these patients have historically been treated with chronic corticosteroids, oral corticosteroids, and the entire community, both the physicians and the CSU patients, understand how toxic that is. So they really need an alternative. The second opportunity for entry point is as the preferred therapy of choice for all second-line advanced therapies. So for patients who have tried any of the current three approved advanced therapies and are still symptomatic, Barzivolumab offers a really important option being, we believe, potentially the first product with, in our label, demonstration of efficacy in a biorefractory population, as well as efficacy regardless of IgE levels. So when we speak with physicians and patients who are at this critical juncture of having tried an advanced therapy still suffering with their symptoms, Barzivolumab can take the guesswork out and give people a lot of confidence in the opportunity. When we quantify that second entry point, if we look at, we assume, say, a 50% flow-through from first-line advance to a next therapy in a given year, which is consistent with what we've seen both with Zolaire in this category, but also what we've seen in other INI categories, that would make the second-line-plus advance therapy market eclipse first-line from a market opportunity within the first few years. So those two really nice entry points for Barzal.

Joe Pangenis, Analyst — H.C. Wainwright

Okay, got it. And you know what? That links nicely, I think. And of course, this is open to all of you as well. You know, you talked about the landscape when, you know, where it would fit here. And to me, there's also this, I don't want to keep saying disconnects between physicians and investors. You know, docs always talk about having multiple tools in the bag. You know, it's not like you're going to supplant Dupixent or Novartis or what have you. You know, they want multiple tools. They're going to look at it at different stages, use it in different patient populations as you build market penetration for the indications you talked about. so as your education moves forward of course not just on the phase three data which will be absolutely critical you know i also see the linking again back to the underlying mechanism of action you know with regard to the disease modification so maybe you could talk to the multiple tools and the bags right now and sort of get the initial doctor feedback you know from the current phase two data and as you know you're looking forward and doing a lot of your initial

Terry Lawler, Other

research here? Let me start addressing the unmet need and where Barzol fits even in this competitive landscape. And then maybe Diane can talk about the uniqueness of the mechanism behind that. So even with these entries, as we say, we believe that's a tailwind for Barzolimab in terms of bringing people into treatment. Now, there are three really important areas of unmet need that barzavolimab can address in a unique way. The first, and Diane touched on these, but the straight-up differentiation in three critical areas that we're hearing from physicians and patients are absolutely pivotal. First is those patients who are in complete response, not UAS7 less than six, but zero. So absence of itch and hives. And barzavolimab in our phase II trial was the first trial to show a majority of patients in complete response at 12 weeks. And the second area we think about is the deepening of response, right? So that deepened to 71% at 52 weeks, and then that off-drug durability, so differentiation, deepening of response, and durability. And we see that across complete response. We see that in return to normal quality of life, which is the number one factor that patients point to in terms of what they're looking for in a treatment. And for those patients with angioedema, again, complete absence of angioedema, 65% at 12 weeks, 8 out of 10 at 52 weeks, and then even off treatment at 76 weeks, still a majority angioedema-free. So that differentiation, deepening, and durability is a really unique profile. And as Diane alluded to, where we have the option to show off-drug durability, typically INI drugs, when they talk about durability, are talking about on-drug durability. We're seeing the same level of complete response off-drug that other companies are seeing on-drug. So that gives us an immense amount of optionality in terms of utilization, paradigms, in terms of differentiation with the payers and in terms of really filling big needs that while these other new therapies are really good additions to the armamentarium, there are some things they won't be able to address that Barzavolimab will.

Joe Pangenis, Analyst — H.C. Wainwright

Just making sure, because you alluded to Diane a little bit, making sure you didn't have any additional comments there.

Diane Young, Other

No, just to reiterate, I think our mechanism in depleting mast cells is really targeting the root cause of the disease. So, you know, omelizumab targets IgE, which is one trigger. Dupilumab targets, you know, IL-4, IL-13, which is a mediator and affects a subset of patients. And BTK is, you know, a pathway that's downstream of, you know, IgE receptor. So, but we think, so we think we have, you know, a broader effect. And I will just, I'll just reiterate, I think physicians are very excited about having different options for CSU. You know, we're seeing that now with new drugs becoming available after they're only having omalizumab for a long period of time. And, you know, they definitely talk about how it's all about discussions with their patients. And, you know, they're going to take into account how severe are the patients, what are their symptoms, you know, how convenient are the dosing regimens. And so they're really happy to have, you know, new things. And they're pretty enthusiastic about Barsovolumab.

Joe Pangenis, Analyst — H.C. Wainwright

No, perfect. I appreciate that. So I'm going to ask the broader question here that sometimes, you know, as a company gets closer to filing or has filed or is awaiting FDA decision, you know, has negatively impacted some companies in the past. We won't mention any names. but the broader question here is how are your manufacturing preparedness here with regard to Barzo and being able to hit the ground running? Yeah, so I can say we're, you know, the team is

Diane Young, Other

working very hard on that. You know, we're obviously working with contract manufacturing and we're, you know, we're really monitoring the whole process. We're, you know, preparing to enter the market with a pre-filled syringe and then with, we're developing plans for an auto

Joe Pangenis, Analyst — H.C. Wainwright

injector to follow that. Got it. And sort of like, it's easy then to just plug and play with your CROs to be able to get more, you know, slots available and pre-planned for those, you know, fashion. Yep. Okay. So again, I'll just stick to the broader profile of the company right now. And if we have time, I'll ask another question here. How are we doing? Yep. So over the years that I've been covering you, you've always had and been able to keep a big war chest going with regard to cash on hand. I'm hoping you can sort of discuss that. You guys obviously just did another sizable raise over $300 million, which again, shows that you continue to attract important investment into the company. And I'll also maybe so discuss that, but then I would also link that to providing leverage in any potential or upcoming or ongoing BD discussions. So that's the second part of the question with regard to, you know, what are your general views

Sarah Cavanaugh, Head of Investor Relations

towards BD at this point? Sure. So as you alluded to, we ended 2025 with $518 million in the bank, and our cash guidance at that point was through 2027. We just recently completed a raise and brought in an additional $345 million, and I'm sure we'll update our guidance when we report out Q1 relatively soon. So from that perspective, it was fantastic to have that money raised. It was a great mix of some current investors and some new investors who've been very excited by the story. I think, you know, what really drove that excitement was seeing that enrollment get completed early. And it certainly spoke to the enthusiasm around the program. And then also, as you mentioned at the start of the call, it brought those top line results into the fourth

Diane Young, Other

quarter of this year so we're really all looking forward to that from a business development

Sarah Cavanaugh, Head of Investor Relations

perspective it certainly always makes sense to have cash on hand you know how important that is we have been very clear that we want to take this drug forward on our on our own in the united states um it's part of why terry is here and we are working through all those pieces um we'll certainly be smart you know we'll look at the opportunities that are out there and evaluate them you know as they make sense but we are really looking forward to launching this drug

Joe Pangenis, Analyst — H.C. Wainwright

Oh, perfect. You know what? So I am going to ask my last question because, again, it's also different personalities in the investment community. So I can't say 100% focuses on Barzo right now, but it's very, very high. But, you know, you always have the mantra in people's heads that's saying, okay, so what have you done for me lately? Got Barzo going on? What do you have going on in the background? So maybe you could take a minute or, you know, a minute and a half just to maybe discuss your pipeline assets, at least from a high-level standpoint.

Sarah Cavanaugh, Head of Investor Relations

Sure. Let me do two things. I'll have Diane quickly talk through 622, and then maybe what we can do is I can end by capping through the milestones for both Barzol and 622 over the course of the year.

Diane Young, Other

Yeah, so we're very excited about CDX622. This is a bispecific antibody that targets TSLP as well as stem cell factor. And stem cell factor is the ligand for the kit receptor. So that part of it is a different way of targeting mast cells. So we have entered this into phase one, which is fully enrolled. We presented some of the single ascending dose data, and it looks very, very promising. We will have the multiple ascending dose data later this year, and we have initiated a proof of mechanism study in asthma because, you know, we believe with the two targets that, you know, pulmonary diseases are, you know, a good direction to go with this, but there's actually a lot of potential. So we're really excited, and I can tell you that our chief scientific officer, Tibor, has a number of other things behind that that are not clinical stage yet.

Joe Pangenis, Analyst — H.C. Wainwright

Absolutely. Tibor has always delivered for the long term coming up with assets, you know.

Sarah Cavanaugh, Head of Investor Relations

He has. So then if we look at, from a milestone perspective, for Barzivalamab, we really focused on CSU and on inducible uticaria. And as we said, you'll see top line data from the phase three and CSU in Q4. um we also will continue to enroll to the phase three inducible utacaria study we recently completed enrollment in two phase two studies one in paragonodularis and one in atopic dermatitis the pn data is expected in the summer of 2026 and then the atopic dermatitis data is expected in late 2026 so you're going to get pn you're going to get both phase three studies in csu reading out at the same time in Q4 to be followed by the atopic dermatitis data. And then Diane mentioned the 622 study. So you'll have the phase one multiple ascending dose healthy volunteer data, and then you'll also have the sub-Q multiple ascending dose data. So that'll all come around Q3 of 2026. So lots of opportunities to look at data and add to the story.

Joe Pangenis, Analyst — H.C. Wainwright

no absolutely so look um we're great on time here terry diane sarah thank you so much for doing this great commentary um wish you the best of luck coming into the phase three data and uh really a lot going on with all the catalysts that sarah just mentioned as well so and for those on the call here please enjoy all the other sessions that you know of course for our fourth annual inflammatory skin disease conference and again ladies thank you very very much

Sarah Cavanaugh, Head of Investor Relations

Thank you, Joe. We really appreciate the time. Thank you.