Aclaris Therapeutics at Jefferies Conference: Strategic Pipeline Advances

On Wednesday, 04 June 2025, Aclaris Therapeutics (NASDAQ:ACRS) presented at the Jefferies Global Healthcare Conference 2025, highlighting its robust pipeline and strategic financial positioning. The company shared promising updates on its clinical-stage biopharmaceutical developments, despite the competitive landscape. Aclaris emphasized its strong financial health, with a cash runway extending to mid-2028, while actively seeking partnerships to bolster its respiratory franchise.

Key Takeaways

• Aclaris is advancing its clinical programs, focusing on O45 and the ITK/JAK3 inhibitor, 2138.
• The company boasts a solid financial position with $191 million in cash, extending its runway to mid-2028.
• Strategic partnerships are being explored to enhance the US respiratory franchise.
• Upcoming data readouts and clinical trials are expected to drive future growth.

Pipeline and Clinical Development

Aclaris’s pipeline features both large and small molecule targets, with significant focus on:

• O45 (T-slip mAb):

- Phase 2 study in atopic dermatitis (AD) aims to replicate previous successful results, with data expected in the second half of 2026.

- Positive data from a China partner in asthma and CRS supports clinical potency.

- US partnerships are being explored for the respiratory franchise.

• 2138 (ITK/JAK3 inhibitor):

- Imminent data readout from an open-label study to assess efficacy and safety in AD.

- Potential development in alopecia areata is on the horizon.

- A next-generation ITK selective will enter the clinic in 2026.

• Bispecific Antibody (T-slip and IL-4R):

- Phase 1 study to start this quarter, with data on safety and tolerability expected by year-end.

- Phase 1b trial planned for 2026 in respiratory or AD.

Financial Position and Strategy

Aclaris has strategically managed its finances to support its ambitious clinical goals:

• Cash Runway: Extended to mid-2028, thanks to prudent capital management.
• Balance Sheet: $191 million in cash provides a strong foundation.
• Capital Allocation: Funds are allocated to support key clinical catalysts through 2027.
• Partnerships and IP Monetization: Additional funding avenues are being pursued to supplement cash reserves.

O45 (T-slip mAb) Discussion

The discussion around O45 highlighted its competitive edge:

• Potency: Extensive diligence confirmed its high potency compared to competitors.
• Phase 2 Powering: The study is designed to achieve robust EASI-75 and IGA 0/1 responses.
• Confidence in AD Program: Based on strong Phase 2a data and positive respiratory data from China.
• Mechanism: Features long tissue residue and low dissociation rate.

Conclusion

Readers interested in more detailed insights can refer to the full transcript for comprehensive information.

Full transcript - Jefferies Global Healthcare Conference 2025:

Roger Song, Senior Analyst, Jefferies: Alright. Alright. Welcome everyone to Jefferies twenty twenty five global health care conference. My name is Roger Song, one of the senior analysts cover semi cap biotech in The US. It’s my pleasure to introduce our next printing company, Clarus, Niu, and Hugh.

Welcome, gentlemen. Thank you. Awesome. Alright. So we have this fireside chat.

Even we’re standing here. And then we why not we start with the high level elevator pitch for the update no recent updates for Aclaris because you have some change in terms of the strategy and then the pipeline, and then we can dive into those, you know, details.

Unidentified speaker: Sure. So Aclaris is a clinical stage biopharmaceutical company focused on both large and small molecule targets. We have a drug discovery engine that underpins everything that we do, including a proprietary chemical library with world class expertise in the development of small molecule inhibitors. And as Roger alluded to, we recently onboarded a large molecule portfolio and with that brought, five to six full time employees from Biocean who we license the assets from who also have decades of experience at large companies like like J and J. Currently, we have three clinical stage assets.

First one is two thousand one hundred thirty eight. It is a oral small molecule targeting ITK and JAK three, very unique pharmacology. Nothing like that in the market. And then we have a t slip mAb which we in licensed from Biocean that’s in phase two. And then we have a bispecific that we recently announced had an IND allowed and we will be starting phase one SADMAD work this quarter.

And then finally, we have a next gen ITK inhibitor that is due to get into the clinic in 2026. So we have a lot going on, a lot of catalysts in ’26 for sure, and we also announced in our in our latest earnings release that we were able to effectively extend our cash runway by about a quarter and a half through, mid year twenty twenty eight. So well capitalized with a hundred 91,000,000 on the balance sheet and, three clinical stage assets, again, spanning large and small molecules.

Roger Song, Senior Analyst, Jefferies: Excellent. Great updates. Maybe we can focus on the o 45 for now for the your t slip monoclonal antibody because that’s, you know, in the clinic and in phase three phase two. I believe you recently started the phase two, and then that just announced. Quick timing.

And so how much the the study design you can tell? I believe you have some high level, but any like, a dose dose regimen and anything additional detail you can tell us?

Unidentified speaker: Yeah. Sure. So o forty five is the t slip mAb, and, we had a phase two a, where we were in AD with twenty four weeks of therapy, and we’re recapitulating that same study design in the phase two with placebo control. And so it’s highly powered study, with, high moderate to severe patients in AD, with a three hundred milligram dose, which is the same dose we used in the two a where we saw, you know, ninety plus percent easy 75 response and eighty eight percent IJ zero one response. So we’re looking to recapitulate that with a placebo control.

Roger Song, Senior Analyst, Jefferies: Got it. Yeah. You mentioned the phase two a in The US open label study without the placebo control, but the effect side, the drug effect is extremely high. So when you design the phase two, you say it’s well well powered. What is your statistical assumption in terms of the drug versus the placebo?

Unidentified speaker: Yeah. So we we’ve seen, you know, the the higher placebo rates in recent months, and so we’re really focused on ensuring that we we maintain a a patient population that’s gonna be highly relevant. And we’re using a central reader to really make sure that that’s gonna work around pictures coming into the trial. And then, again, we’re looking to show, again, that robust activity and efficacy along with, obviously the same safety profile that we had earlier. And so it’s really about robustness of response, but really maintaining a really solid, normal placebo rate.

Roger Song, Senior Analyst, Jefferies: Okay. Normal placebo rate, but likely similar phase two, drug effect. That’s the power.

Unidentified speaker: Yeah. And I I I think, you know, if we got that same drug effect, we’d be ecstatic, and that would be the best AD drug of all time for sure. You know, I I I think when we modeled it out, we assumed a little bit of a discount factor for that just because of the single arm bias. But even with that, when you’re talking about IgA response rates of 88%, you know, easy 75 north of 90%, Even if you dis discount that by a third, it’s still industry leading. So the design of this study, just to be clear, we we don’t wanna squint at the end and and just, oh, it it’s slightly better than maybe standard of care.

We think there’s an opportunity. This is why we’re dosing every two weeks even though we think, the the PKPD supports q, two months. We’re we’re trying to max the effect out, and we wanna see, obviously, early onset of action, but that sustained action through, twenty six weeks, and and further in in the in the drug free period. So this is you know, we feel very strongly about this, and I know there’s a lot of debate about t slip and AD just due to the Amgen, TESI study that that didn’t work out. However, I think the key difference here is is the potency, and and we’ve made a lot of this narrative.

And and I do really think it matters, and I think it’s being missed that if you assume a certain concentration gets into the skin, then potency has to matter. If you’re if you have a an off rate that is exceedingly long, then you’re going to block effectively block that alarmant from initiating the whole cascade. And and why wouldn’t that be more meaningful? And we have a couple of slides that we’re gonna add to our deck in in the coming days to weeks where we’ve done some of that work showing the distinction between TESI and our molecule as it relates to potency in the skin specifically.

Roger Song, Senior Analyst, Jefferies: Yeah. That that would be very helpful, and then understand when you acquire the molecule, you’re a decent diligence in terms of the, you know, PKPD and then the mechanism. Right? So you see the very low disassociation, a very long tissue residue, and then you have the phase two data. Right?

That’s that really leads to why you think this drug can work in AD while Amgen drug doesn’t work. Right? So that’s the Yeah.

Unidentified speaker: No. That that’s correct. I I think that’s a very important point to mention. We we’ve done an extensive amount of work pre deal to fully characterize the molecule versus not the entirety, but the relevant competitive landscape. And it’s pretty clear just looking at the data.

We repeated these experiments numerous times. You add on to that the the respiratory data, which we we talked about qualitatively in our last earnings release, and then you layer on to that the phase two a, data that Hugh and the team generated and albeit single arm, it still showed such a effect size that that, you know, it’s hard to ignore. So I think the abundance of all of that data all points in the same direction, and and we’re really, we’re really excited about that program.

Roger Song, Senior Analyst, Jefferies: Yeah. Excellent. Just a one quick, you know, final question about the powering. So this is phase two. Right?

So you have multiple efficacy endpoint. I believe you’re looking through all the EZ and then the fifty, seventy five, 90, and the IGA. So in terms of powering, what what are the endpoints that are powered? And then, so how should we think about when you read all the data and then we see the statistics?

Unidentified speaker: Yeah. I mean, well, it’s the standard way. So we we looked across, not only our own data, but across what’s going on in AD and looking at a delta, that would make sense from, you know, a p value. Obviously, a a easy 75 response is a primary endpoint, but IgA zero one is a real key, secondary, which is important for this because, again, we wanna show robustness of response, for these patients.

Roger Song, Senior Analyst, Jefferies: Got it. You power for those two endpoints. And I believe once you power for that and then the reduction probably already powered, but that’s a little that typically is more powered than the the responder analysis. Correct. Okay.

Got it. Alright. So we should though and then you start the phase two, and then when we potentially can see the data?

Unidentified speaker: Second half of of twenty six.

Roger Song, Senior Analyst, Jefferies: Alright. Yeah. That’s exciting timing. Okay. So that’s for AD, which is, I I believe, your key main focus for the O45 program.

But also we know recently you have because this is acquired from China, you have a China partner. They released some top line data, very qualitative, and then in the asthma and the CRS. So you have some common, very interesting, you say, you know, basically validate the clinical potency is higher than TESI. And then just tell us a little bit more what to the extent you can disclose to us and how much data you have seen and then what what’s the how you’re gonna how you’re gonna categorize the data?

Unidentified speaker: Yeah. So I think I’ll stick with what we mentioned in the earnings release, but the the suffice to say we’re very happy with what we’ve seen. It is a partial dataset. So, unfortunately, until we get the complete data, you know, we’re not going to put put data out, and we just don’t have that that ability at the moment. But the the key things from our standpoint were, one, is to validate the potency narrative because up to that point, what we had had was the in vitro work, which is meaningful but still not clinical work, And we had the single arm two a AD data which was conducted in The US.

So it was helpful just for us to validate that hypothesis with larger data sets and another indication. So that was kind of the the biggest point. The second point was to get a sense of the efficacy and we certainly got that. And then third, and this wasn’t a surprise, just get a sense of the safety profile which is of course good. So the good news is is along all those parameters, we’re we’re very happy.

And, you know, I I don’t usually like just putting out qualitative statements, but it it is the box that we’re in at the moment. And, you know, I think that in conjunction with all the other data we just mentioned, makes us really excited about this program. And and so now we kind of turn our attention to partnering activities. We’ve already had a lot of of inbound interest on the respiratory front post that that earnings release, and it’s because how advanced that asset is that’s going into phase two large phase three studies in both indications in China. And so I think it’s it’s one of arguably the most advanced t slit assets out there.

And and, so, yeah, we’re excited about that. It gives us a way to to potentially bring in some nondilutive capital, get get a partner engaged in that, validate what we’ve already said about the asset, and, and I think it’s exciting exciting times over the next few months.

Roger Song, Senior Analyst, Jefferies: Got it. Understand that the, the focus for the respiratory franchise for o forty five in The US is, through the partnering. Right? So we can talk about that in a moment. But, the reason investor, including myself, is interested in knowing this China data is how what’s the risk through to your AD.

Right? So how would you how would you say the risk through based on what your data you see to give you more or less confidence that AD gonna work?

Unidentified speaker: So I think, you know, as you know, in those studies, you have all sorts of endpoints. So what you’d like to see is some kind of translation of of the potency narrative into clinical effect. And and so when we say we’ve seen some of that, that’s kinda what that means. Right? So and I would say that is meaningful.

However, we actually have direct read through from the AD study that was single arm. Again, it was it was, you have to discount that. Right? Because there’s some bias in a single arm study for sure. But, again, the effect size was so robust, you know, we just haven’t seen numbers like that that, in an indication where another company previously failed, to me, that in conjunction with all of the assay work, I’m talking about, you know, a dozen different assays, makes us feel really confident about that read through, and and, you know, hopefully, we get the opportunity to surprise people late next year.

Roger Song, Senior Analyst, Jefferies: Okay. Very good. Yeah. I know people are waiting for that surprise positive surprise for sure. And then so okay.

Just one last one. I just want to press one more time and then you can tell me you cannot say anything more than that. You say they enhance the potency. Just want to clarify that’s more on the biomarker side or on the clinical efficacy endpoint side?

Unidentified speaker: That would be on both.

Roger Song, Senior Analyst, Jefferies: Excellent. Thank you, and thank you for the clarification. Okay. I think that may be oh, so maybe the partner discussion. So you already know the data, I think, couple weeks, and then I believe you start a conversation with the potential strategics before that.

So what’s the conversation so far?

Unidentified speaker: Yeah. We we’ve had interest both global ex China and regional, and, you know, that that was that predated, you know, our earnings release disclosure. So that’s since accelerated. Look. You can’t predict BD.

Who knows what happens with that? But it it’s good to have inbound interest. I don’t think it’s surprising when you think about that this particular target is is only, you know, there’s only two large pharmaceutical companies working on this this target or has it in their portfolio. So to me, that’s not a surprise. And and I think the safety profile with a mAb like this is is quite attractive.

So we’ll continue to have those dialogue and and hopefully hopefully have something, you know, towards the back part of the year.

Roger Song, Senior Analyst, Jefferies: Awesome. Alright. Great. So that’s for we spent almost half of the time for 45, which makes sense because that’s a lead program. But you do have other pipeline as well.

So one is the ITKJAK3 inhibitor small molecule two thousand one hundred thirty eight. So you will have data very soon. So but that’s also our organ label, no placebo controlled. So how should we expect the data coming and then if you can give us some context to interpret the data when you have the

Unidentified speaker: Sure. So we tried to accomplish a lot with with this study. It was, numerous kind of outputs and and, you know, one would like to see efficacy on par with with JAK inhibition in AD. I think that makes sense. We have JAK three within that molecule and the way to think about it is it’s about a fifty fifty contribution from the ITK side and the JAK three side.

And so that’s on the efficacy front. On the safety front, this was the first time we took it through twelve weeks in patients with disease and so certainly this will give us a first real look at at the safety profile and we would expect to see a pretty well tolerated molecule there. And then on the PD side, what we’re trying to do is you can run assays that tease out the ITK effect versus a JAK three effect and we have a very heavy PD component. We’re doing biopsies, tape strips, everything in between, ex vivo stimulated work. And what we hope to do there is make it very clear the contribution of ITK as to the the efficacy.

And and that does two things. One is it paints a picture for that particular molecule as we transition into indications like alopecia areata where we know JAK three and ITK both work. And then it also paints a picture for our ITK selective that comes into view in 2026 for AD and other t h two related diseases like various respiratory diseases. So we’re we’re really excited about the the ITK selective where we’ve engineered out the the JAK component. That has very real potential to be a massive drug because you basically have an oral medication that that’s knocking out all the key cytokines that a lot of the biologics do and can do so in a safe in a safe manner.

So, that’s the objective of that study. So it’s really to to to really lay the groundwork for for both the asset itself, 2,138, but also the next gen to come.

Roger Song, Senior Analyst, Jefferies: Yeah. Got it. To be clear, 02/1938, the next step will potentially be the alopecia areata. Right? So and then for AD, you will start to select the the selective IgG for the next step.

Unidentified speaker: That’s correct. And and we think, you know, twenty one thirty eight is a better molecule for alopecia. There’s a little bit less sensitivity to having JAK in a compound because the entire, alopecia areata commercial landscape is composed of JAK inhibitors, and we happen to have one that, you know, we think is going to be more effective. It’s, you know, five x more potent on JAK three than ritlacitinib, forty x more potent on ITK. And so we think we have a a great approach to fast follow ritlacitinib, which is doing quite well in the market.

So that just seems to be a better place for us.

Roger Song, Senior Analyst, Jefferies: Yep. Makes sense. So can you give us a sense what are the key assay can differentiate the contribution between the ITK and the JAK-three? You know, I know they have some overlap, but also it may have some distinct kind of biomarker effect you can see. Because, clinically, you cannot see that.

Unidentified speaker: Yeah. One one of the key things to look at I mean, first, you can generate gene signatures, and then that’s some of the work that we’re we’re outsourcing. But also looking at c d three, c d eight, c d 28 stimulated work on an ex vivo basis where you can look at the differential effect of ITK.

Roger Song, Senior Analyst, Jefferies: Got it. Okay. Good. I think that’s the ITK side, and then you have a two path forward. Right?

So that’s how we’re gonna see the see the data pretty soon. And then you have very exciting bispecifics. So I know the I nine the future is probably the bispecific, you know, different ways to combine, so multi complex. So you choose to use t slip and r four, so that’s your and the receptor, so that’s your approach. So maybe just take a step back, say, okay.

Why you choose that as your lead, bispecific, and then, we move on to the clinical development?

Unidentified speaker: Yeah. So so with the T slip portion, we already know we had that real long residence time, and the molecule is actually chosen based upon its lack of or or a great amount of lack of dissociation off of T slip. So you’re inhibiting this alarming, and and we wanted to do that completely. So the four hundred hour residence time and neutralization of TSLP was key. So that’s the first component.

And then on the IL four r side, we wanted to have something that was a similar affinity as dupi because that’s already shown good effectiveness, because we also wanted to inhibit IL four and IL 13, and IL four antagonist would do that. So in effect, we have a a trispecific, you will, where we’re inhibiting both the action of TSLP IL four and IL 13 with this bispecific. And we also have a YTE mutation in order to to enhance the, the half life on that. So, so it was built with the idea to completely, neutralize the TSLP over activation in disease and then be able to have, additional potency, on top of the I l four r act side of the equation.

Roger Song, Senior Analyst, Jefferies: Got it. So how should we think about the synergy or additive effect between the IL four and the t slip? I believe they have some overlap, but, also, they try to kind of broaden the coverage for the the past four I think, the lead lead indication and a couple a couple different indications.

Unidentified speaker: Yeah. That’s a good question. The way we looked at it was by using that same, peripheral blood mononuclear cell assay where you activate with TSLP and IL four, you can then see c c l 17 produced. It’s a chemokine. What we did with the bispecific is we showed that if you use TESI and DUPI combined to inhibit that c c l 17 release from the activated PBMCs, the o fifty two bispecific is about three to five times more potent than DUPI and TESI combined in inhibiting the chemokine, secretion.

So we know this molecule, not only has that long residence time on TSLP, but it also has enhanced potency.

Roger Song, Senior Analyst, Jefferies: Got it. Okay. Great. So you are this is not I think you already cleared the IND, so it’s not that far away from the clinical. So where are you in the development stage, then what will we what we’ll be able to see in the near future?

Unidentified speaker: Right. Well, we’re initiating the phase one in healthy volunteers, SADMAD, this this month. And and the we would expect that we would have output from that by the end of the year in terms of PK safety and tolerability. And then we would move, in ’26 into a patient population that we haven’t yet defined. But, you know, you can imagine it could be in respiratory and or atopic derm, in order to show, you know, activity and safety in a patient population in ’26.

Roger Song, Senior Analyst, Jefferies: And then for the the end of the year phase one PKPD data, what will give you the confidence for the different indication considering you already have the T slip side and then maybe you wanna see a little bit better on the I l four side. And then very interesting because that data will come before you have your own t slip US controlled AD study. So how should we link those two data and then some research?

Unidentified speaker: Yeah. That’s that’s a good question because what you have is PK safety and tolerability. So we need to make sure that hitting both pathways, TSLP at the top of the t h two cascade and the immune component with T cells at the bottom with IL four and thirteen, we need to make sure that that still has the same safety profile that you would expect, for TESSI and and DUPI. We also are really interested in the PK because, as you know, DUPI is half life of eight or ten days, and so it’s really limited the dosing interval for patients. And so our expectation is that this this molecule should have an enhanced longer half life.

And so, there, we’ll have some really good understanding of what we would take into a phase one b in terms of, extended dosing beyond two week dosing.

Roger Song, Senior Analyst, Jefferies: So longer half life, very safe when you combine. And on the PD side, what, you will give us some contacts between o fifty two and then o forty five and then maybe do p and then the tezi? That’s something you will give us some head to head?

Unidentified speaker: Yeah. I mean, you you could certainly do, you know, PD types of of, where you could do ex vivo activation and and see how the drug is working, over time. So, you know, that that would be more on the exploratory side. But yes.

Roger Song, Senior Analyst, Jefferies: Got it. And, and then you have the the o forty five, which is a monoclonal antibody for t slip, and then I think the indication pretty clear for your focus is AD. And then the o fifty two, you just mentioned, can be respiratory and or the AD. So how we think about how should we think about the strategic decision between those, you know, large, you know, group of indications?

Unidentified speaker: You know, it’s it’s gonna depend. But, you know, if if o forty five can show the same sort of activity, that we saw in the two a, then then that obviously has a path forward in both AD and respiratory on its own. But, also, if we do have this enhanced potency with the bispecific, there’s gonna be populations that are gonna require an extra boost, if you will, of of activity. And so we could, think about other types of populations where that would even be more effective. And, certainly, I’m not saying COPD is is one of them, but, you know, we know that both TESI and DUPI, even with DUPI with an approval, you know, you’re looking at 35% response rates for approval.

So there is a huge unmet medical need there for having a much more potent molecule. And TESI itself, you know, with their phase two data, same thing, thirty five percent response rate. So there are populations that are still in dire need of a more effective drug.

Roger Song, Senior Analyst, Jefferies: Yeah. Makes sense. Okay. And then in terms of the corporate strategy and then the the the capital allocation, How should we think about your cash? And then what are the I know you’re in the low burn rate and then which long very long runway.

And then how should we think about the cash will support your pipeline considering you have a multiple, I know, direction you can you work up?

Unidentified speaker: Yeah. I think, obviously, based on success, you know, we would be looking at the traditional ways, to to finance that and and also supplement that with with the various partnerships. And we also have, you know, an IP estate that we started monetizing last year, and we’ll continue to do that throughout this year. So we’re we’re well capitalized to deliver all the catalysts we mentioned through ’27 with with runway to spare. And, you know, as we continue to flip over the data cards, we’ll we’ll make the right moves to to continue to capitalize the company.

Roger Song, Senior Analyst, Jefferies: Yeah. Maybe just to be more specific because you have the phase two a d and then you have the, potential phase two for ITK for alopecia areata and then you have this o 52 phase one, phase one b. So are those all included in the current cash flow?

Unidentified speaker: That’s correct. We we deliver, I’ll just repeat it, we deliver two thousand one hundred thirty eight in alopecia areata in addition to the top line that we’ll put out imminently on AD. We deliver the o 45 in atopic dermatitis on the t slit mAb. We deliver the bispecific and sad mAb plus we have in our budget two one b’s in rest one in respiratory, one in derm. Deliver both of those in in ’26.

And then we deliver the ITK through next gen through a POC. So all of that comes within the current cash runway.

Roger Song, Senior Analyst, Jefferies: Excellent. Thank you. And then you do have this small molecule platform. Now you have a large molecule t slip platform, not just tslip but you know it’s a biologic platform. So what are the highlights you want to give us for the earlier pipeline in terms of small molecule and the large molecule?

Unidentified speaker: Yeah, so you know, ITK obviously we’re really excited about that next gen and that includes a portfolio of compounds. And then we’ve also started to look at, again, based on the data we saw out of China, utilizing giving having the confidence we do in the t slit mAb, looking at various bispecific constructs utilizing that that, in our mind, best in class t slip map component. We haven’t disclosed what those are, but, work has already started. And good news is it doesn’t cost a lot of money to do that in the early days.

Roger Song, Senior Analyst, Jefferies: Yeah. No. I agree. Okay. Alright.

I think that may be, the most of the question I have. And anything else you wanna highlight to people and that we haven’t really discussed?

Unidentified speaker: No. I just say again, we, you know, have a hundred 91,000,000 on the balance sheet, three clinical stage assets with meaningful readouts through ’26. Really exciting preclinical program with the with the ITK next gen and pretty pretty prudent use of capital with the ability to to bring in additional nondilutive capital over the short term that gets us, you know, well into 2028. So I think it’s a good setup given where we’re trading.

Roger Song, Senior Analyst, Jefferies: Agree. Alright. Thank you. Thank you everyone for listening. Thank you.

Thank you.

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