Okay, I think we're ready to get started here. Thanks, everyone, for joining us for our second annual Healthcare Innovation Conference here in Boston. I'm Valmont Devon, one of the Bio pharma analysts here at Guggenheim. Arseniy Sagasvili from the team also joining us up here on stage. Very pleased now to have next presenting in this room is Absci. Joining us here, we have Sean McClain, the Founder, CEO, and the Director of the company, Zach Jonasson, who's a CFO and CBO of the company. Thanks so much for both of you joining us again this year. Obviously, exciting times for the company. A lot of good progress over the last year. Remember last year, we were sort of talking about your R&D Day that was coming up in December, and there's been a lot of progress since then, and the story's evolved a lot too.
Maybe just for people who are a little less familiar with the story, you can talk a little bit about the background of the company and some of your core technology, and then we'll dive more deeply into your lead programs, especially the 201 program, which I think has generated a lot of interest.
Yeah, yeah, absolutely. Thanks for having us here. I founded the company 14 years ago. We originally were not a generative AI drug creation company. We had a background on technology to scale protein-protein interactions, how antibodies interact with targets of interest, looking at their overall functionality. That was right around the time that deep learning was taking off at Google.
We had this idea of being able to take this functionality data with these models to really be able to go from this paradigm in R&D, where you're searching for a needle in the haystack, to actually being able to create the needle, in our case, an antibody, and being able to start to test hypotheses that scientists have always wanted to test but haven't been able to, and being able to actually engineer in the properties that we want, activate a particular pathway, or create different switches, being able to bind in the tumor microenvironment but now not bind in healthy tissues. The strategy that we've implemented over the past couple of years is really applying this generative design technology to these hard-to-drug targets that have known biology. Think of GPCRs and ion channels.
We really see a lot of really exciting upside with some of these targets, because again, you have the biology. They have just been difficult to drug. We see this as really being a way to leverage the technology, stay really competitive to create first-in-class and best-in-class assets. Additionally, on our own pipeline, we have been able to develop some exciting assets. We have a TL1A asset, which we are going to be disclosing some of the data here very soon. Additionally, we have ABS-201, which is targeting the prolactin receptor. There are two really exciting indications on this. The first is Androgenetic alopecia. Think of common baldness that affects 80 million Americans alone.
What we have been able to demonstrate with this is that by blocking the prolactin receptor, you are actually able to shunt the follicle from the catagen state, where you have apoptosis and regression of the hair, to an active state. What we are seeing is really strong durability with this, as well as superior efficacy to minoxidil in preclinical studies. We have actually brought in the timelines in terms of getting this drug into the clinic, as well as a phase two proof of concept. We have brought our guidance into dosing our first participant December of this year. Roughly six to nine months later, we plan to have a proof of concept readout second half of the following year. That will be looking at the hair regrowth. We are just really excited about this particular indication and overall market. I think it is very analogous to the GLP-1s.
You're seeing patients pay out of pocket for this. You're seeing this because they're able to essentially get their identity back, their confidence back. People don't like the shame of being overweight. I think the same sentiment is very true with hair loss. You talk to these patients, and they'll do anything to ultimately get their hair back. We see this as a huge market opportunity. I'm just really excited about the next couple of years. The last point I'll make on 201 is the other major indication, as well as endometriosis, which we'll be talking more about here in the coming months.
Okay, great. Thanks for that perfect overview. Let's dive deeper into 201, because I think that is clearly where a lot of the attention is. You touched on some of this upfront, but just in terms of the market opportunity, obviously a huge potential market. How are you sort of seeing the competitive landscape there? Where are your sort of areas that you can potentially differentiate? Then we'll talk about your ongoing work you're doing and the data that we might see next year.
Yeah, absolutely. You have not seen much innovation at all in hair regrowth. You have had finasteride and minoxidil. With women in particular, they are unable to take finasteride, and minoxidil gives them hair in unwanted places. The other issue is that you do not get full efficacy. Some patients respond really well to minoxidil. Others do not. The total area hair count that you are getting is roughly 20%-30% with minoxidil and finasteride. You are having to take this daily. You are actually having to take it for life. Once you commit to oral minoxidil, you actually have to stay on it for life, because you do not actually revert to your baseline. You revert to what you would have lost during the time you have been on minoxidil. These drugs out there right now are not condition modifying. They are just preventing hair loss from occurring.
Again, once you're off of these drugs, you immediately lose your hair. I think the major difference, and we'll actually talk more about this data at the KOL Summit coming up, is the mechanism is actually condition modifying, where you're actually able to restimulate the follicle stem cell growth. With that, you're able to see the follicle go from the catagen state into the anagen state. From a biology standpoint, once you're in that active growth state and the follicle is reactivated, you stay in that state for anywhere from two to six years, based on your biology. We believe that two to three injections will give durability for potentially up to two to three years.
When we talk to KOLs, when we talk to patients, that's something that they're really excited to see, is that durability, in addition to better efficacy than what you see with minoxidil and finasteride.
Okay. You mentioned you're hoping to get the patients started next month, get the trial going in December. Data, I think you said, looks like another next year. What should we expect in terms of the design of that, and what are you hoping to see out of this trial?
Yeah. Yeah, so it's a very well-powered trial. I'll start by saying that. It's segmented into two portions. The first portion is an SAD, where we'll evaluate safety and tolerability, primarily in healthy volunteers. Although we do have an optional cohort, we may look at efficacy just for a single dose in the SAD portion. Following the SAD, we have three to four doses we'll be testing in the MAD portion. That's the portion that's powered for us to look at efficacy for hair regrowth. The primary endpoints, again, beyond safety and tolerability, will be looking at the target area hair count, which is the validated measure for FDA. That's where you're using a TrichoScan, essentially a way to magnify and count the number of hairs in a square centimeter in different points on each patient's scalp. That's a quantitative measure, again, that's accepted by FDA.
We're also going to look at some really interesting secondary endpoints, in particular hair density, the thickness of the hair, the pigmentation of the hair, which we think are also very important to patients and consumers.
Okay. And then maybe thinking a little bit more on the commercial side, you touched on this a little bit upfront, but maybe two, three injections could give you a few years' worth of benefit. I guess the questions we sort of have, and I'm sure we'll learn more about this at your summit, just the sort of market strategy here, is this something that patients would be administering to themselves? Is this something that's more sort of channeled through a physician's office, which may actually help with, we've seen it with Botox and kind of bringing patients into an aesthetics practice? How are you sort of thinking about this? Is this more of a, again, doctor-driven, or is it more sort of patient DTC sort of driven? Or maybe some combination of both, yeah.
We see it as a combination of both. I think right away, it's important to get the physicians excited and bring in the patients to actually administer. Long term, we see this as a direct-to-consumer play, I think very similar to how you're seeing the GLP-1s ultimately play out. I think you could see an auto injector, and this is something where you partner with a Hims & Hers to ultimately get the script, and then you can go direct-to-consumer that way. We do believe that partnering early on with the physicians is a critical step for ensuring that this is ultimately well-known and well-accepted within the physician community.
I will just add that the setup for the go-to-market is remarkable, right? Because you have got dermatology offices and clinicians. You have got medispa and plastic surgeons. If you put all those together, there are more of those in this country than there are Starbucks. They are all looking for products like this. I think the go-to-market for the initial part is very well laid out for us. Longer term, as Sean pointed out, we could go direct-to-consumer.
Okay. Maybe one more from me that I'll turn to. Anyway, just as you think of this big potential market, and obviously, we're a few years away from this, but is this something that you would be sort of commercializing on your own, or is it where you mentioned there are Hims & Hers, or how would you sort of think about this strategy? Is this something that you, again, hold on to all the rights to, or is it more of a partnership or working with others, just given the size of the market?
I would say this is one of these indications where the clinical development is straightforward. There are objective endpoints. The patient recruitment and the trials are straightforward. We're told there'll be waitlists to get into the trials. When you get to commercialization, like I said, you've got this perfect setup for go-to-market. Our belief is we can take this all the way into commercialization ourselves. We think that's the big opportunity here.
Yeah. When you talk to folks that have been at Allergan that have commercialized Botox, we've been able to talk to a former CEO, the head of commercialization. This is definitely not pushing a boulder up the hill. This is definitely a boulder going down the hill. I think the sales force is going to be a lot more streamlined. It's actually going to be smaller. It's going to be a completely different marketing play than traditional pharma. I think that it's ultimately going to require, I think, less of a kind of traditional sales force that you see with other drugs.
Okay. I'm sure.
Yeah. And as we think about the safety of this approach, what is the natural function of the prolactin receptor, and what are potential issues, if any, in blocking it in men and women over time?
Yeah. The safety profile looks remarkably clean. It's supported by human genetics, our GLP talks work. And then most recently, Hope Medicine, which has a, we consider a very much inferior antibody, just read out a phase two trial in endometriosis, where they didn't see any what we would call really severe AEs in that trial at all. The mechanism looks very clear. And just to speak a little bit about the genetics, there are patient case reports in the literature looking at patients that have loss of function mutations. These patients are perfectly healthy, normal hormone levels other than prolactin, normal serum electrolytes, childbearing, no sexual dysfunction.
The only phenotype, though, that's associated with it is the inability to breastfeed. That's what would be expected. We don't think that's in any way going to inhibit what we're doing here for hair regrowth, because, again, it's a pulse treatment. You do this treatment for two to three injections over six months, and then you're off therapy. If you become pregnant during that time, you just go off therapy. We don't think that actually diminishes the market. We see no safety signal based on all the work that we've done, the genetics, or what Hope Medicine are in their phase two trial for the mechanism.
Great. Let me pass it back to Valmont to talk about your KOL-MA program.
Yeah. I guess one other question on this one before we go there. You mentioned endometriosis. When do you expect to just hear more about the opportunity there?
Yeah, I would say in the near future, we plan to discuss more about endometriosis and how that ultimately is going to be baked into our overall strategy.
Okay. And then this KOL Summit in December, I know you don't want to front-run it too much, but what should we expect there? Is there more of a discussion about the market and sort of the commercial side? Is it more about the data? What's the plan?
Yeah, absolutely. You'll hear from KOLs talking about the market opportunity. You'll hear from patients and ultimately what hair means to them and what a treatment would, what a treatment could look like where they would ultimately get excited. Additionally, we'll be going over the Ex vivo data that really supports the overall mechanism of the prolactin receptor, and then the overall kind of market opportunity and some of our thoughts around commercialization.
Okay.
Maybe I'll just tease one thing that he mentioned. The Ex vivo data is some new data we generated on human biopsies from scalp, looking at the effect of our antibody in that system, in a human system, Ex vivo system, which is some really nice data there that we look forward to share.
Okay. Sounds great. Maybe we can just move on then just to one-on-ones at the top of your lead program. We have some data here coming up soon. Maybe again, if you can just sort of talk through the product in this competitive landscape here and obviously a lot of companies in the space and what we should be seeing here in the next month, I guess.
Yeah. Yeah, I mean, we can't say too much because we're just getting the data in now, and we're going to release some results shortly. I would say we're looking for safety and tolerability, number one. Then we'll be looking at the PD and the PK and the half-life. One other element we'll be tracking very carefully is we believe our antibody has a differentiation in terms of tissue distribution. We'll be looking to evaluate that. That'll probably come a little bit later into December when we have that data, but we expect to see that as well.
Okay. Maybe you could just talk then on the competitive side, maybe not on your data, but just I think that's one question we certainly guess there's a lot of companies in the space now. Kind of how do you see the space evolving? Where do you see the openings maybe to show differentiation with your program?
Yeah. I would say we would acknowledge the IBD space is becoming very competitive. It's not just from TL1A monotherapy. It's other targets. It's combination therapy. It's bispecifics. One of the things we've been actively looking at at Absci are other indications, and particularly some indications where you could be first in class. Indications that other competitors aren't pursuing currently. I would say as well, we're actively and have been having discussions with potential partners, but we're looking to find a partner, particularly if it's going to be advanced in IBD. We think that's the kind of indication a large pharma is much better equipped to execute on in comparison to AGA, for example, where we think we can execute those trials given our size and balance sheet quite easily.
Okay.
Yeah, there is a particular indication that no one has yet explored. There is some new recent data that has come out. This would open up the buyer universe. This is outside of INI. I think that this is also where we see differentiation. We actually think that the higher tissue distribution is actually going to help in that particular indication. In that area, we would be first in class there. Again, I think you have both an IBD play, but now with this outside of INI, you have kind of a bigger buyer universe that opens up. We are definitely exploring other areas as well.
Interesting. Yeah. It gives us some stuff to dig into to try and figure that out.
Yeah.
Okay. All right. Great. Let me turn back to our senior list. We've kind of dug into the programs. Just a couple of big picture questions in the last few minutes here.
Yeah. Maybe just a quick recap of your programs that are further back in development, which I think is the 301 is your immuno-oncology program that's still in preclinical development. You have the 501, which is a next-generation HER2 antibody. I think you also mentioned a bispecific that you're thinking of developing with the TL1A. Anything to highlight there as recent developments or anything we should look forward to hearing more about at your upcoming R&D Day, just any updates on those earlier programs?
Yeah, absolutely. 301 is a novel IO target. We've been able to show target validation with that. We are actually validating it with the drug that we've designed towards this particular target. Right now, those in vivo efficacy studies are wrapping up. We are hoping in the near future to be able to take that data and partner this particular asset. Given the portfolio that we're building up around INI as well as in AGA, we do believe partnering that and not taking actually both the oncology assets into the clinic is the best strategy for us. We have been engaging with parties. I think if we do see positive in vivo data there, we would then look to partner that asset. We are wrapping those up in the near future.
You previously guided to announcing one more large pharma partnership in 2025.
That is still on the roadmap, right?
That is still on the roadmap. We very much are still sticking with the guidance we had given. Definitely expect that in the near term.
Okay. Let me turn it back to Valmont to talk about your financials.
Yeah. Maybe you just talked, obviously, a lot going on now with the company, a lot of different programs. Can you just give us an update on sort of where you stand from a cash position and your runway?
Yeah. We'll report out here in earnings Q3 numbers. As of Q2, we had about $117.5 million on the balance sheet, cash equivalents, short-term investments. We subsequently, in July, raised another $64 million through two transactions. One was a direct offering. The other was through the ATM. With that additional capital, we have runway into the first half of 2028. A full year beyond the readout, the proof of concept, interim readout we're going to have on the AGA program next year. I think we're in a great position. It also enables us to advance other programs forward as well on that same run rate. The last thing I'd mention is that's a fairly conservative forecast.
We have additional upside, including from the partnership that Sean was alluding to that we're working on today, as well as if we were to transact on any of the assets in the portfolio.
That would all be accretive to that runway.
Okay. So maybe just kind of bring things together here because we've talked about a lot of different programs. Just to sort of the catalyst path here. So we have obviously the 101 data coming up, I guess, next. Maybe you can just sort of walk us through the next 12-18 months. What are the key things we should be looking out for these various programs?
Yeah. So we'll have the large pharma partnership announcement in the near term. We'll be entering the clinic with 201 December of this year. Then second half of next year, we'll have the 12-week interim readout on the alopecia trial looking at the hair regrowth. From there, we will have the opportunity for a phase two readout as well, which we'll be discussing more about here in the coming weeks.
Okay. Great. Maybe just as a little tease from our side, we are hosting you guys again in a couple of weeks for a deep dive under the 201 program. So talking with all of you in the KOL too in the space. Looking forward to kind of learning more there. We've been focused on the conference this week, but it's November 24th, and you'll see that in your emails shortly. Thanks so much for joining us. Obviously, great progress. Looking forward to following all this progress over the next 12 months going forward.
Yeah, absolutely. Thanks so much.
Okay. Thank you. Thanks.
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