Everyone, I think we're ready to get started here. Thanks again for joining us for the Guggenheim Healthcare Innovation Conference this year on day two, moving to the afternoon sessions. I'm Vamil Divan, one of the Biopharma Analysts here at Guggenheim. Joined on the Guggenheim side up on stage with me, Arseniy Shabashvili, one of the members of the team. From the company side, very pleased to have Absci join us.
We have Alex Khan, Corporate Vice President for Finance and Investor Relations, joining us here. Thanks, Alex, for coming to the conference again. Obviously, a very busy time at Absci. Lots changed over the last 12 months since we saw you here last. Maybe for those less familiar with the company or maybe following it as closely, just a little bit of background on the Absci story and some of the key developments, and then we'll go from there on the questions.
Absolutely. Thank you, Vamil and Guggenheim, for hosting us again. Always a pleasure to be here. For those who've been following us for a while, you've probably seen our evolution over time from an AI drug creation platform company that was working with a number of partners on the pharma side to where we are today, which is a pipeline-owning company that is building out a pipeline of our own antibody programs. Right now, our lead program, ABS-201, is in a phase I/IIa study for pattern hair loss and also for endometriosis, are the two indications we're looking at. That's an anti-prolactin receptor antibody, which I know we'll get into some details.
That's what we see as our flagship program, and then we have some potential additional indications we're looking at for prolactin biology that we think could be very interesting to look at, whether it be within the ABS-201 molecule as an indication expansion, like we went from pattern hair loss to endometriosis, or some additional prolactin antibodies behind that. There's also some other molecules we have earlier stage in our pipeline that are outside of prolactin that we think could be interesting as well. Continuing to build out that pipeline that we have.
Okay, great. Let's dive into 201, because I think that's obviously where most of the focus is right now. You mentioned prolactin receptor. What was it about that receptor that drove the company to choose that as a target for your lead program here?
Yeah, no, there's a very interesting history on it. Our now retired Head of R&D, Andreas Busch, he had previously been at Bayer, and he was working with the team there that had been looking at the prolactin mechanism for endometriosis at that time. They had, he and the team, a serendipitous discovery that the prolactin mechanism was not only implicated in endometriosis, but also in androgenetic alopecia or pattern hair loss, and they determined that while doing these animal studies. At the time, they had looked at those results in mice and also in stump-tailed macaques, which is a species of non-human primate that naturally goes bald after adolescence, much like humans. Andreas had been looking at moving that forward, and the team there decided to deprioritize that indication and look at some other things.
When Andreas had come over to Absci, first as a Board Member and then as our Head of R&D In-house, this was a program and an indication that he was very excited about. This was biology that he was saying was very overlooked, very underappreciated, and that it was something that Absci should be pursuing. We started on that program a couple years ago now. We're in the phase I/IIa right now. We put out that SAD data in June from the IV portion of that, and we have the MAD portion ongoing right now. But it's a very unique mechanism. It looks to be regenerative in nature, very different from other programs in development, and very different from any other hair regrowth agents that we see out there right now.
Okay. Maybe you can talk more specifically around that as you think about the biology and the connection here, both for hair loss and endometriosis.
Yeah.
Just elaborate further on what gets you excited.
Yeah, I mean, when the team there and now at Absci have been looking into the biology of, well, what exactly is the connection between prolactin receptor, and the hair cycle, it does look like the buildup of prolactin in the follicle is actually responsible for pushing the follicle into the apoptosis or regression phase of the hair cycle, which is the catagen phase. By blocking that buildup of prolactin in the follicle, you should be able to push the follicle back into the active phase, which is that anagen phase, which, depending on your biology for a particular human, can last a few years, right? Two years, four years, six years. Similarly, with endometriosis, excuse me, we see in the literature is that the buildup of prolactin in the endometrium could be responsible for two things.
One is the actual creation and formation of the lesions there, but also the pain perception that people feel when those lesions are growing. The study that we'll be planning to do next year as our phase II for endometriosis will be measuring pain growth, so looking at non-menstrual pain, dysmenorrhea. We do believe that the mechanism has the potential to be disease-modifying in that it could help with the lesion growth as well.
Okay. Let's talk pattern hair loss first, and then we'll come back to the endometriosis side. You mentioned how this looks different than anything else that's available or in development for pattern hair loss. Maybe you can elaborate on this. Kind of from the sort of profile you're looking at to develop with 201.
Yeah, I mean, I think what we look at as being a potential winning product for ABS-201 or for just a hair loss agent in general is something that would be safe, efficacious, convenient, and durable. When we think about the ABS-201 target product profile, what we are looking to develop is something that would be convenient in either getting two or three doses over a six-month period that can then give you multi-year durability after that, once you are off treatment, with obviously a good safety and then some good efficacy there. We can contrast that to something that is a chronic daily administration, a daily pill, a daily topical, or certain administrations like platelet-rich plasma treatments, like nutraceuticals, like hair transplants.
The ABS-201 target product profile is designed to be something that a consumer can take a few infrequent injections and see multi-year durability after that, versus something like a minoxidil, for example, where a lot of consumers in practice will be using this on a daily basis. If you come off treatment, you do not see that durability that you would have if you were continuing to take it. Again, contrast that to something like a prolactin receptor antibody, where the preclinical data we saw with the stump-tailed macaque suggests that even if you are having this infrequent dosing and then come off the dosing, you will still see that durability as you saw in the stump-tailed macaques.
What they saw was in, I think, 28 weeks, getting dosed every two weeks, but after those 28 weeks were up, you look out six months, two years, and four years later, those macaques still had all the hair that they had grown while they were on treatment. We can contrast that with something like a minoxidil, where once you come off that treatment, once you are no longer taking it, you are more likely or you are likely to lose that hair that you would have been growing in that time period.
Okay, great. You have obviously moved this program now into phase I, the HEADLINE trial ongoing now. Maybe you could just walk us through that trial, the setup of the trial, and where things stand right now in terms of enrollment and next steps.
Yeah, we had the single ascending dose data out in June, so everything looked generally safe and tolerable. The half-life was supportive of the extended dosing interval we're looking for. Now we're in the multiple ascending dose portion. So in that MAD portion, we have three cohorts, 49 individuals each, looking at doses of 300 mg, 600 mg, and 1,200 mg. It's a double-blind placebo-controlled study, so a 3:1 randomization with placebo in there. We're looking to have our first interim analysis data cut coming out next month. That'll be a 13-week analysis, just interim by design. In that analysis, we're looking to share target area hair count, which is a metric that looks at these non-vellus sort of thick hairs.
Also looking at total hairs, which is a metric that looks at both the non-vellus and also the vellus thin hairs, which we think could be an interesting leading indicator. Then also target area hair width, which is looking at the width of the hairs. Those should be part of the data that we're sharing in the interim analysis next month. When it comes to the full proof of concept data, which is the 26 weeks data early next year, we'd be looking at the same metrics and then some additional metrics too, patient-reported outcomes, global images, things like that.
Then as an exploratory additional upside, we'd be looking at that 26-week readout. More likely, the time point that this would show up, if at all, would be the target area hair darkening and pigmentation. We do think the mechanism has potential to restore someone's natural hair color, but again, that's just an upside scenario, not baked into any of our estimates or assumptions right now.
Okay, great. One question we get, I just want to make sure it's clear because we're getting close now to this-
the data release. We will see data from all three of the dosing arms and 13-week data for all 49 patients. I know there are some women in the trial too, but I think it is only going to be the males. Maybe you can clarify exactly what we should expect to see when you release that interim data.
Yeah, you are right that the 13-week interim analysis upcoming is going to be just male data only. As for the specific number of people per cohort, we have not fully shared that at this point, but as you can imagine, just the way that the trial is designed for enrollment, we will have more people who are coming from those earlier cohorts than from the later cohorts. It would not be all 49 people from each cohort at this time point. When we have the 26-week readout early next year, we would expect to have that fuller, more robust data set to share.
Okay. I think a lot of questions we get around what to expect. I know you have made comments about how it may take some time for this mechanism to really show its impact. This earlier 13-week time point, what would you be happy with? What do you think investors should be hoping to see?
Yeah, I mean, really at this early time point, the 13-week interim analysis, we just want to see a directionally positive signal of hair growth at that point. And we think back to the stump-tailed macaques, who in that case it was 12 weeks and 28 weeks, we think could be something similar in humans if we kind of follow that analogy. At the 13-week interim analysis, we would want to see a directionally positive signal of hair growth. At the 26 weeks, want to see that strong result growing from the 13 week out to the 26. We also think beyond that, there is a potential to see the continued durability or even further growth from the 26-week time point onward. 13 weeks, look to see a directionally positive signal of hair growth.
26 weeks, look to see a strong hair growth result, and then beyond that, potential durability and continued growth.
Okay. Maybe one last one for me, then I will turn to Arseniy. Just again, just because we have so many questions, I will make sure it is clear. In the 26 weeks is when we should expect maybe the first time you will share more around the pigmentation and some of the other endpoints you are looking at.
This one, the 13-week, be more focused just on the count. Is that
Yeah, that makes sense. I mean, the 13-week, again, just interim by design, so looking to see that target area hair count, which is that standard metric that we all look at, so that would be the non-vellus hairs. Also look at the total hairs and then the target area hair width at that interim analysis. The later time point, the full POC, is when we would expect to show that more comprehensive data set. The reason for the 13-week, we think an interim, interesting additional point to look at is that total hairs, which again is the metric that includes the vellus hairs, the thin ones, in addition to the non-vellus thick hairs. We think this mechanism, compared to some others, has the potential to generate hairs in what were these dormant follicles.
It may be the case that when those hairs do come in, they come in as vellus, thinner hairs, with the potential to, at a later point, convert to additional non-vellus, thick hairs. So we will be showing how many non-vellus, thick hairs grew at that time, plus how many vellus, thin hairs grew at that time, which could kind of give a hint as to how many additional of those vellus hairs, the thin ones, could convert to the thicker, non-vellus ones at a later time point. So, a little bit of a more comprehensive, robust data set as we can.
Okay. Sorry, one last one from me, actually. How are you thinking about the placebo group? You having at 13 weeks and then 26?
Yeah, so we'll have, again, as a placebo-controlled study, and we as the company and the trial will remain blinded through the end of the study, but we do plan to report out the results by cohort and by placebo. So, we should be able to have a placebo-adjusted number that people can kind of compare to other agents out there.
Okay. All right, great. Let me turn it over.
Yeah, so turning to endometriosis. The original molecule from Bayer, which I think is now called HMI-115 was also studied in endometriosis. They have some publications out. What did that data tell you, and how did that inform your study design?
Yeah, no, we thought that was a good data set they put out. It did show some good proof of concept. They saw the reduction in dysmenorrhea at the high dose. That is more encouraging for us, more validation in the proof of the mechanism. We also think there were some potential areas of improvement on their trial design, such as the geographic and ethnic concentration and diversity, the run-in period. We are planning in our study to have a run-in period of two cycles, for example, a little bit more broad on the geographic and ethnic diversity.
A lot of the ways that we're looking to design our study come from the expertise of our CMO, Ransi Somaratne, who had been at Vertex previously and worked with their genetics program, but also some of the learnings from Hope Medicine's trial, which again, they saw good data, which we're encouraged to see, but probably some areas of improvement on their study design. We're looking to have, and this is all subject to finalization, but 150 people, 10 + countries, and probably going to look at two or three doses in that study.
What is the cadence of readouts from that study, and what would follow after?
Yeah, we haven't specified that yet, but we'll be looking to kick off the phase II middle of next year.
Okay. How are you thinking about the unmet need in endometriosis and the potential size of the addressable market?
Yeah, I mean, the endometriosis treatment market is very lacking. There's a lot of trade-offs in existing treatments, so things that can cause essentially early menopause or bone mineral density loss. There's a lot of side effects that aren't great, honestly. We think this could be something that, as a commercial product, could potentially fit in a second-line area, right? There's NSAIDs, there's GnRHs. We think ABS-201 has a potential if we hit that TPP, which we're going to be measuring in the study as a pain-based outcome, but we do believe this could be potentially disease-modifying too. We think this could be, estimate peak sales above $4 billion, based on our estimates.
Great. How would it be dosed in endometriosis, or that yet to be determined?
Yeah, TBD on the dosing levels and frequency of administration, but we see it being likely different than the PHL dosing.
Understood. Turning to your pipeline, what would come after ABS-201?
Yeah, I mean, so a few interesting things we're looking at that are also within the prolactin franchise, so things outside of hair loss and endometriosis. We have ABS-202, for example, which is for an undisclosed I&I indication, but there's also some other indications that we see as having potential for prolactin. Outside of prolactin, there's a few other targets we're looking at that we're keeping a little quieter for now, just for competitive reasons, but some things that are getting us excited. I think when we think about our business strategy and what programs we want to prosecute ourselves, or what programs we want to prosecute in general, I think a lot of what we're looking at now kind of fits into, well, how does this fit into a potential direct-to-consumer franchise or potential direct-to-consumer channel?
We think for ABS-201 for pattern hair loss, that's kind of the blueprint here. That is a large unmet need with a lacking standard of care that we think is just primed to be direct-to-consumer. There is, for pattern hair loss, an in-place provider channel where right now 80% of people in the U.S. who are looking for hair treatment will go to one of a dermatologist, a med spa, plastic surgeon. So there is that in-place provider channel, but there's also a large and growing telemedicine and DTC channel, and we think that we could potentially leverage those trends for an ABS-201 potential commercial launch, go through those channels, go through DTC. We have the flexibility there.
So my last question before I turn it back to Vamil is on your platform and how that sort of evolved over time, because I know initially you were very focused on antibody discovery, antibody development, which you still are. But what role does AI sort of play in the company beyond that? For example, target selection is obviously very important.
Yeah, I mean, we recently talked about our Atlas platform, which is part of our target discovery engine. So to your point, we've always had AI integrated into our platform as a molecule generation, like de novo design, but also for lead optimization, and then now we have that target discovery platform as well, so we can kind of look at it end-to-end. It's not that every program will use all components of that, but with the way that the de novo model's been evolving over time is we're at the point now where we're not just trying to develop a molecule or generate a molecule that would be a hit, so to speak. We want to generate something that would be candidate-ready.
I mean, that would be coming out of the model with much less screening than necessary compared to a few years ago, and be able to go essentially direct into IND-enabling studies. We leverage our platform to be able to look at designing molecules with the attributes that we would want from the start, so that includes developability, half-life extension, lower immunogenicity, and then also looking at, depending on the target, epitope specificity, so we can map out which epitope we want to hit specifically. Those are the types of things that we use our platform for.
When you think about target selection, are you looking for targets that maybe no one pursued before, or targets where there is maybe people pursued before, but there was a signal that was overlooked? How are you thinking about it?
Yeah, I mean, it depends really, and I think prolactin is a very unique example of something where a lot of the information that we are uncovering and discovering has been in the literature for quite some time, and just has been very overlooked. We think we want to use our platform and our target discovery to find what we are thinking of as the next prolactin. What else is out there where there may be some good biology that just needs to be further elucidated and built upon? But we can always look at potential first in class, but also potential best in class. We are not married to one strategy or the other.
Let me turn this back to Vamil Divan.
Yeah, so maybe a couple, just going back to the pattern hair loss, couple other questions we get, if you can touch on quickly. One, you mentioned this initial data will focus on the male population.
When do you think you'll have sufficient data to release something on the female side?
Given the mechanism, do you see limitations in terms of the female population who may or may not be able to use 201?
Yeah, so we'll have separate additional female cohorts, and we'll share some more information about that pretty soon. So those will have their own datasets coming after these male-focused datasets. But we do think one of the very interesting and compelling things about the prolactin receptor mechanism compared to something like minoxidil is that we don't see it creating unwanted hair growth. So we don't envision it having hypertrichosis issues as you would with other agents. So we think for a female population, which would have to potentially deal with that for minoxidil and would not be patients for finasteride, this could be a solution for them if it works out the way we envision it as being a very targeted treatment therapy that will just grow hair on the scalp and nowhere else.
Okay. Then, you touched on this a little bit, but I think there's obviously a lot of dynamics on the commercial side that people are curious about with this sort of product, relative to maybe what's currently available or what's in development. I know you can't say much here, but I have to ask this. Kind of your latest thoughts around pricing and kind of where, if this profile holds up somewhere near what you're looking at or we've laid out as a potential target product profile.
Yeah.
How do you think about entering this market relative to where the competitors are?
Yeah, I mean, the bottom line is that the pricing would be commensurate with the efficacy and overall TPP we hit. But we do think that if we hit the TPP we're looking for, this would be positioned as a premium product in the market. If you look at what's out there now, something like a nutraceutical, those things, I think people will pay, say, $100 a month for that. Think over three years, $3,600. Even something like a platelet-rich plasma PRP treatment, I think that'll be about $5,000 over three years. So there's a market out there for consumers that are looking for hair growth treatments that'll be paying sums like that. Then even for a hair transplant in the U.S., a good one, I think $10,000-$14,000. So it's a very large market out there.
Our consumer research for the ABS-201 TPP, if we achieve it, does suggest a total addressable market of about $25 billion in the U.S., including the fact that those that we've surveyed in that study were told it would be a premium-priced product. We didn't disclose what exactly that product would be when we published the survey results, but when we spoke to those individuals, they were presented with a premium price, and it still tested extremely well. We do see this being positioned, if we hit our target product profile, as a premium product.
Okay. Maybe a last question, actually has two parts. You did a raise as a company back in June. Maybe one, if you can just sort of share your cash position now. I know you're going to report earnings here shortly, but just kind of your current cash position now and your runway. Then also as part of that raise, Lilly invested as part of that in that raise. Maybe you can just talk about the Lilly relationship with the company.
Yeah, speaking to the June numbers, end of Q2, about $201 million in the balance sheet. That gets us runway into second half of 2028, so through all the key upcoming milestones. The interim analysis for pattern hair loss next month, the full POC for pattern hair loss early next year, and then kicking off the phase II for endometriosis middle of next year. To your point, when we did the June raise and Eli Lilly was an anchor in that, they were just a strategic investor, and we have a member of their team joining our endometriosis advisory board, which the board is advisory by nature. But the investment comes with no program rights or any sort of affiliation or claim to the ABS-201 program.
That's wholly unencumbered, wholly owned by Absci, and we are committed to continuing to take that forward, bring it to potential approval and commercialization ourselves.
Okay, perfect. I think why don't we wrap it up there. Congrats on all the progress. Look forward to seeing your earnings on Thursday, I think you've been out so soon. Then, obviously a big update next month and early next year.
Yeah, no, exciting times. Thanks again for having us.
Okay, great. Thank you.