Everyone, welcome to Guggenheim Emerging Outlook Biotech Summit 2026. My name is Yatin Suneja, one of the biotech analysts here at the firm. My pleasure to introduce my next presenting company, Celldex Therapeutics. From the company, we have three executives here, who I will be moderating the discussion with. I have Anthony Marucci, who's the President and CEO. We have Diane Young, she's the Chief Medical Officer, and then we also have Tibor Keler, who's the Chief Scientific Officer. Thank you so much to you all for coming to our conference. Anthony, why don't you make some opening comments? Obviously, there is a lot going on from a drug development standpoint, multiple indication that you are evaluating with barzolvolimab.
Orient our investor in terms of the key milestone that are upcoming, and then I've prepared the Q&A, which, we'll go in a little bit more detail.
Sure, Yatin, thank you, and thank you for inviting us today. So, 2025 was a really busy year for us, on the execution front and as well on the development front. We ended 2025 with starting our second phase 3 study with cold urticaria and symptomatic dermographism. We started our phase 3 studies there in December. We also finished up our phase 2 PN studies and our phase 2 atopic dermatitis study, in December and in January of this year. So those are studies that we concluded and are moving forward. But beyond that is the CSU study, which I think Yatin is alluding to. In the CSU program, we have two phase 3 studies that are ongoing.
We've guided, since we started these three phase 3 studies in 2024, that we would finish accrual in the summer of 2026. So Yatin just asked me if we can give some additional guidance, and we will do so at the end of this month when we put out our earnings release. We'll update you on guidance there, so... But we do plan on completing the phase 3 study enrollment for CSU. They are two large phase 3 studies being conducted in over 500 centers and 43 countries around the world. We expect to have at least 1,830 patients in the two studies combined. And we're very hopeful, looking forward to duplicating the phase 2 data that we presented in this program back then.
As I said, the ColdU and the SD studies are ongoing. That is a 240-patient phase III studies, 120-patient in each-
Mm-hmm
... of the ColdU and the symptomatic dermographism arms. Timing around that is around 18 months. And as you know, that's how we guide. We want to be pretty conservative about it, and then hopefully meet the timelines like we have. The data from the AD and the PN studies, we're guiding towards the second half of 2026, and based on the data we see there, we'll start up the phase III studies in those programs. And then behind that, we're looking at additional indications-
Mm-hmm
... for development, either later on this year or 2027. That would include food allergy, allergic rhinitis, and perhaps, chronic pruritic itch of unknown origin. Those are three things that we're looking at, as well as some others. So, as you can imagine, this year is gonna be very busy from an execution standpoint. We certainly have the team to do that, and we look forward to finishing studies up.
Great. Thank you, Anthony, for those comments. Indeed, execution year for sure. So let's start with the CSU program. I think you've detailed or presented a lot of data, both on CSU and CIndU, including the off-drug durability data. So the question is, don't-- not to repeat the data, but just what you are hearing from physician, how are they viewing the drug now that you have evaluated and presented all this data, especially the long-term durability data?
I'll let Diane answer that question.
Yeah. So, you know, the physicians that we speak with are very excited about our data. You know, they, they believe that the data is really unprecedented. And, I think, you know, first of all, we have, you know, our data on symptoms, so UAS7. And in that case, you know, we have rapid responses. We have, you know, the highest complete response rate that's been reported, you know, up to 70%. And we have this persistence, of effect, 41% are still responding, seven months after the last dose. And then, you know, accompanying that, we've been presenting all these endpoints.
You know, we really show, you know, fantastic improvements in quality of life and in angioedema, and, you know, you know, putting it all together, I think it's making our, you know, our investigators very excited and, you know, very enthusiastic about our trials.
Got it.
Yeah.
And I think, you know, from a global perspective, I mean, you may have seen that Quad AI-
Yeah
... put out a PR.... and, and that was their PR around our 76-week data. So I think that there's a lot of excitement around that. There's gonna be a lot of discussion around that. You know, there's a lot of discussion around how to treat patients now that these data sets have come out, and it'll probably change the paradigm for a long time.
Yeah. Like, even at this AAAAI in the next few weeks, I think you have three posters, right? Are we gonna get a little bit more, even longer prolonged off treatment effect data? Can you maybe preview what we should be expecting? Like, what are some of the key presentations that you have on the data?
Yeah. So we have three presentations. One, as Anthony mentioned-
Yeah
... was actually just press released by Quad AI. That's additional data regarding the long-term response.
Mm-hmm.
So it's the subset of patients who were well controlled at 52 weeks, and it follows them out with detailed reports on, on their, their symptoms, you know, over the 7 months after therapy. And it really shows that, you know, the majority of those maintain a complete response, and those that, you know, do have a recurrence are recurring with milder disease.
Mm-hmm.
So it really supports, you know, the really effective long-term responses we're seeing. The other two posters that we have are going to be related to the phase 2 CIndU study.
Mm-hmm.
One is the 20-week placebo-controlled period data on quality of life and urticaria control test, just, you know, supporting the positive efficacy in CIndU. And then the other one is that is a late breaker, is retreatment data.
Yeah.
In the CIndU study, after the 20-week placebo-controlled period, they had the option, if their symptoms recurred, to be retreated with barzolvolimab, and we have shown that we are able to successfully retreat the patients.
Got it. Helpful. So that's on the efficacy side. What about safety? You know, what is the perception, and how is the safety in the real world, whether it's related to neutropenia, hair color changes, or skin pigmentations?
So I think the safety profile, you know, with all the data we've shown so far, is very consistent, and our investigators are feeling, you know, very, very comfortable with it. You know, the, you know, most of the common side effects are, you know, mediated by KIT. They only occur in a subset of patients, they're mild, and, you know, we've shown that they're reversible.
Got it. Then let's touch on these, the phase 2 CSU program. Are there key features of this study that are different than the phase 2? And then in terms of the primary endpoint, it's at 12 weeks, but I do think that you have to follow everybody for 24 weeks. So just help us understand, can you announce the data before 24 weeks?
Yeah. So in terms of the design, you know, it's a randomized placebo-controlled phase 3. We have tried to keep everything as close as possible to the phase 2 as we possibly could in terms of, you know, inclusion, exclusion criteria. The only major difference is that we have added a loading dose-
Mm-hmm
... to each of the doses. In terms of the, you know, the. So the primary endpoint is the change from baseline of UAS7 at 12 weeks, which is the typical endpoint. But we, as you mentioned, so we have a 24-week placebo-controlled period, and we are going to do the first analysis after all the patients have completed the 24-
Nice
... week placebo control, although the endpoint is at 12 weeks. So we'll have a more... You know, we'll have the full set of the 6-month safety data and the, and the efficacy endpoint as well.
Got it. Got it.
Sorry, is this the phase 3 CSU I got?
Yeah.
Yes.
Thank you.
I think if you look at ClinicalTrials.gov, the primary completion says October 2026. Is that sort of a reasonable timeframe for data, or it's just more study enrollment completion?
It's more study enrollment completion.
I see. Then in terms of the various scenarios for efficacy and safety from the readout, how would you describe various scenarios? Like, what would you like to see, in a successful outcome?
We definitely want to see, you know, results like we saw in phase 2. You know, we want to see, you know, high complete response rates, and, you know, we'd be, we'd be very happy with results as we saw in phase 2.
Yeah. We want to make sure we hit our endpoints in the antihistamine refractory. We want to make sure we hit our endpoints in the biological refractory. You know, similar efficacy, if you can get it, is fantastic.
Yeah.
I mean, it's the best efficacy that's out there, so, it's a high bar, but I'll take it if it happens, that's for sure.
Got it.
And certainly, you know, the fact that it's gonna be in over 1,800 patients will build up that safety database that is gonna required by the FDA. So as we go through phase 3s and other indications like cold and SD, the study sizes will be much smaller. They're not gonna be required to be 1,800 patients. So, we're doing all the hard work with CSU-
Yeah
... as far as the safety, the databases, the longevity, it'll all be done with this study. So, you know, we're confident that the data is gonna look good. We'll see what happens.
Got it. Then on the commercial side, could you touch on the commercial dynamic there? How should we think about positioning, size? I mean, now we have Novartis's Rapsido, which is actually priced a little bit higher than what I think people were expecting in the $45,000-$72,000 range. So how are you thinking about pricing there?
Yeah. So I think with, you know, remibrutinib now being approved, you have Novartis, you have Sanofi, you have Roche-
Yeah
... to an extent, with Xolair now, but food allergies out there. I think that's only gonna grow the market, and I think that we can all agree that the three big players in there are gonna grow that market. We've always felt, and I think I've told you a million times, we think the market is 750,000 biologically eligible patients. Novartis seems to think that the market is a lot bigger than that, so time will tell. But either way, if the market is bigger, the pie gets larger as well. I think our entry point, you know, even though, you know, we're making it perfectly clear, we can treat the antihistamine refractory, we can treat the biologically refractory patients.
We think our entry point is gonna be, you know, behind Xolair with the allergist and, you know, Remi or Dupixent. Go through one of those, and we'll think we'll be behind them as well. And we also think that as they grow that pie, that second line biologic market is gonna continue to get larger and larger over the next 5-10 years.
Got it. I mean, is Remi a good proxy for price now?
I think Remy is a good proxy for Dupixent. I mean, certainly, I you know, we think that the data we've generated you know requires a premium price, but we'll see. You know, we have to certainly-
Got it
... go through that process. The data needs to bear out.
I mean, how would you any view on the launch so far for Remi and the cadence?
We haven't heard a lot.
Okay.
Certainly, we saw that Dupixent believes their uptake-
Yeah
... has been good. We'll see what happens. Usually, I don't think they report out before they have a couple of quarters on the ripple.
Yeah.
But we imagine that it would be good.
Okay. On AD side, so that readout is coming later this year. Again, what are the goals? What exactly... How would you envision success there? What exactly you would like to show in the atopic dermatitis study?
Yeah. So, atopic dermatitis, we've done that study. Based on, you know, the information that we had from, you know, prurigo nodularis in terms of the effect on, on itch. You know, so we'd certainly like to see, you know, itch, itch reduction. And, you know, we're gonna see what effect we have on clearing of the lesions and so forth. You know, we hope to have, you know, very, very good efficacy, particularly in terms of itch.
Yeah, we think there will be a read-through from PN AD, especially on the itch component. Again, we'll see where else mast cells play a key role in AD, but from the itch component, I think our PN data's will be a good read-through for us.
Is the endpoint 12 weeks, 6 or 24 weeks?
Sixteen.
Sixteen weeks.
16 weeks, yeah.
For AD. For the PN study, which is, that's a 12-week endpoint.
That's 12 weeks, yes.
But you do have a but it goes to 24 weeks.
Twenty-four weeks.
Same thing.
So you will-
Yeah, the same thing as what we described for CSU, that we will do the analysis after all the patients have completed at least the 6, the 24 weeks.
24 weeks. But for AD, 16 weeks is what we're gonna get.
Yeah.
Yeah, AD-
Both data are coming second now. Okay. Then I wanna go into the... So you touched on food allergy, right? Other indications that you are thinking. What work you have to do before you go into food allergy? Obviously, a significant area that is just opening up with Xolair.
Yeah, so I think there's gonna be, you know, CSU, CIndU, there's a lot of comorbidities.
Yeah.
One of the comorbidities is food allergy.
Yeah.
We've been looking through our phase II data. Certainly, as the phase III data comes out, we'll see that there's a lot of comorbidities there, and we'll certainly get a sense if Barzol's having an effect. We do believe that food allergy is a mast cell-
Yeah
... driven disease. So certainly that makes sense for us. So again, I want that direct evidence coming from studies and see what the comorbidities, and we'll take it from there.
Okay, okay. Moving on to the bispecific, the anti-TSLP and SCF. Could you just explain to us what is this molecule? What have you generated so far in healthy volunteer? And I think there is a MAD portion that is ongoing. What should be the expectation from the MAD?
Yeah, I'd be happy to do that. So, with our bispecific approach, the overall concept is to build off the data that we're generating and our learnings with barzolvolimab about targeting mast cells, particularly in settings where mast cells are important in the disease pathology, but not necessarily the only driver. So the idea is to combine mast cell inhibition with another, important and validated pathway. For the bispecific approach, we've decided to target stem cell factor, the ligand for KIT. We think there is advantages of that approach, and so we've generated a bispecific that neutralizes stem cell factor, and in this case, for CDX-622, also neutralizes the alarmin TSLP. So we're really excited.
We started the healthy volunteer study earlier last year, reported some of the single ascending dose in the fall, and everything's going extremely well. We have over a 24-day half-life at the highest dose, which is great for a bispecific. From a safety point of view, we've seen absolutely no, you know, safety signals. And very importantly, we're seeing, you know, significant and sustained reductions in tryptase, indicating that we're knocking down mast cells. So that's step one. As you mentioned, we're in the multiple ascending dose, where we're giving 4 doses every other week. And that data is going to be presented sometime around this summer. And that's all with the IV formulation.
We've also developed a subcutaneous formulation for CDX-622 that's been added to the healthy volunteer study, and we'll be reporting on that SubQ dosing as well from the healthy volunteer study. So, lots of data, but we're very excited about it.
Got it. And you also have proof of mechanism study in asthma, right?
Right.
Is that initiated, I think that-
Yes, so we've initiated this study in mild to moderate asthma patients with the idea to generate phase 1 data in a patient population, but really importantly, to validate both ends of the molecule. So, we'd like to demonstrate that the TSLP side of CDX-622 generates the type of impact in patients that you would expect from a TSLP blocking molecule, such as knocking down Th2, IL-5, eosinophils. Those biomarkers are very clearly established-
Mm-hmm
... mostly by the data presented by Tezspire. But we are also collecting sputum samples from these patients, so we can analyze directly what's happening in the lung, understanding our impact on lung mast cells, as well as other, you know, inflammatory biomarkers.
Got it. What about the dosing? So if the MAD is ongoing, like, how do we know you are at the optimal dose in this proof of mechanism study?
Yeah, well, the data will tell us that, of course. We have a pretty wide dose range, up to 9 milligrams-
Nice
... per kilogram in the IV. So I think we'll learn a tremendous amount, both from IV and subQ dosing, that will help us inform the, you know, future studies.
Even in these 12 patient asthma study, it's multiple doses?
No, this is a single IV dose at our highest dose-
Ah, mm-hmm
... milligrams per kilogram.
I see. Yes, yes, yes. Okay, that's good. Good, it's good.
We think we get plenty of exposure for the endpoints that we're looking for.
Got it. So once we establish the proof of concept and we validate sort of the biology, how should we or how are you thinking about list of indications that you would pursue with this dual mechanism?
So we're evaluating a lot of different possibilities. Clearly, what we're focused on is where there's validation for both pathways. And that centers initially around pulmonary indications. Asthma is clearly one of those, COPD might be a very interesting indication, other fibrotic conditions as well. And we're also considering food allergy in this setting as well as we're trying to understand better the role of TSLP in that setting. So those are decisions we'll be making as this data comes out this year.
Got it. How is the immunogenicity or the ADA profile of the asset? What have you seen so far?
Yeah. So far, so good. So we reported in the single ascending dose a very good, you know, PK without any immunogenicity measurable. So we certainly hope that's something that we can maintain, obviously, an important hurdle for bispecifics.
Got it. Got it. Got it. Very good. Then finally, on the cash position and the runway, run rate, and the expenses, and the burn rate.
Yeah. So we had $583 million at the end of the third quarter, and, you know, we've guided that. That's enough money to get us through 2027. So obviously, we flipped a card on CSU. That's plenty of money to get us through there. We flipped a card on CIndU as well.
Yeah.
We're there. But certainly, you know, we will need more capital for a launch-
Got it
... and other development.
Got it. So very good. Thank you so much. That's all I have.
Thank you. Appreciate it.