Good afternoon, and thanks for joining us to have a conversation with Zach Jonasson, Chief Financial Officer and Chief Business Officer of Absci, and Alex Khan, Corporate Vice President and Head of IR at Absci. Absci describes itself as advancing the future of drug discovery with generative design to create better biologics for patients faster, pairing its Integrated Drug Creation platform with synthetic biology data engine in a continuous lab-in-the-loop cycle. The company now has three clinical- stage programs anchored by ABS-201, an AI-designed anti-prolactin receptor antibody, which is in a phase I/II-A headline trial for pattern hair loss with an indication expansion into endometriosis. In June, Absci reported positive interim phase I safety data, safety and PK data, showing an estimated half-life of at least 65 days and raised $100 million, including a $40 million strategic investment from Eli Lilly.
Our headline is a 13-week interim proof of concept readout in the second half of this year. A phase II endometriosis start in the fourth quarter and the full 26-week data, which we are expecting in early 2027. To discuss Absci's platform, pipeline, and strategy, let's get started with Zach and Alex. Zach, Alex, thank you very much for being here, and I appreciate you accepting our invitation.
Thanks, RK.
Absci suddenly has changed its strategy, starting from a platform partnership story and now to a clinical- stage company with three clinical- stage programs and a wholly owned prolactin receptor franchise. Zach, for people who are new to Absci, can you frame the long-term strategy for us and how you decide what stays on your balance sheet versus what gets partnered?
Yeah, absolutely. Again, thanks for having us. I've been involved with Absci since 2016. I used to manage a venture firm, so I've been a board member and now I'm in operations. I like to say I voted with my wallet and then with my feet. I can say it's a very exciting journey we're on. Our vision is to create a portfolio of DTC therapeutics that can be direct to consumer, leveraging telemedicine, and that can really be more oriented towards partnering with the consumer to improve their own health. Our first molecule, which we think will reach approval, is ABS-201 for pattern hair loss, which we think is a phenomenal market opportunity. There hasn't been innovation in that space for 30 years, and there's 80 million patients in the U.S. alone that have pattern hair loss, and they're very broadly dissatisfied with standard of care.
ABS-201 offers the kind of program that we really want to proselytize and invest in. It's a brand new category of therapy for a market that's desperate for solutions. Rather than an oral or topical you'd have to take daily with a lot of side effects and limited efficacy, this could be a product where you take two to three injections, kind of like a GLP-1, and you're set for multiple years of hair growth. So v ery convenient and durable, and that resonates very strongly with consumers. Our second program, as RK mentioned, is endometriosis. Another area where there's a fantastic, or unfortunately, a fantastic unmet medical need, where there's a really high patient burden. About 10% of women globally suffer with this disease. We think the timing for this program couldn't be better. You now have clinical guidelines for diagnosing endometriosis.
We think this program not only addresses a significant unmet need, but also could be brought to market as a DTC as well. Behind that, RK, we're using our platform, and I think you will probably have some questions around the platform, but we're using our platform today to generate additional programs that we think could fit into that broader strategy.
Talking about the platform, one of the pitch to the story is you can industrialize discovery, especially trying to bring a program into IND enabling studies with under two and a half years and under $15 million, which is fantastic compared to what other biotechs and large- cap pharma spend, both in terms of time and money. What is the advantage that your platform gives that it is difficult to replicate, at least in your point of view?
Yeah. I will start with the metrics. Those are metrics that we have succeeded on with our prior programs, ABS-201 as an example. We think we can do better, though. Today we are investing heavily in an entire agentic platform that interfaces with our AI models that design the antibodies. This agentic workflow involves disease biology, it involves selecting targets and selecting indications, and it plugs into or incorporates that AI design model or Origin model so that we can rapidly construct antibodies to different epitopes and commence testing those for proof of concept. Our goal is to cut those timelines in half and cut those costs down even further, and we have a number of means of doing that. Part of that is leveraging the agentic platform we have built, which we have called Atlas.
Part of that is leveraging some of the things we are going to be doing in China for faster, cheaper clinical trial development.
Let us start with the ABS-201 program. The June interim data showed no serious adverse events across four cohorts and an estimated half-life of 65 days. Walk us through what that PK profile actually does in terms of the target product profile itself, and you are also modeling two or three subcutaneous injections over six months, generating close to 70% bioavailability. What is the significance of what you have seen so far, and how do you think, at least for the next catalyst, which is the 13-week study data, how does that feed into that?
Yeah. Let me comment on the PK, and I'll let Alex comment a little bit about what we're looking for at 13 weeks. We're very happy to see the PK profile, 65 days and counting. We think that that's going to enable at a minimum every two-month dosing, which is very convenient for patients. There's a potential upside to be something more like every three months. We'll see as we collect more data on PK as the trial moves forward. Think about the convenience factor for patients, and we've tested this in consumer studies. What they have today in standard of care is a daily application of minoxidil or finasteride, and if they stop, they lose their hair. Think about a product where you have maybe two injections over six months, and you don't have to worry about it for two years.
That's a game changer in this market. This PK profile is going to enable this convenience. Whether it's one or two doses or three doses over six months, we'll find out. Either one of those tests extremely well with consumers. The other thing that this gives us, which we're really happy about, is flexibility.
As you recall, we're taking ABS-201 into a second indication, endometriosis, and we want to have a different dose and dose frequency across those two indications. We have a lot of flexibility now, and we couldn't be happier with the PK profile. Alex, do you want to comment on the 13 week?
Yeah. In terms of what we're hoping to show in terms of the efficacy, everything we've seen from the preclinical model, particularly that stump-tailed macaque data we've talked about, and just all the literature on the underlying biology gives us strong confidence that we'll see some good efficacy at the full 26-week readout, but even at the interim. The interim, the 13-week readout that we're hoping to have in the near term, we want to have obviously the most robust, comprehensive, and informative data set as possible. As we're collecting all that data, we'll be sharing that probably in the December timeframe for the 13-week readout. That'll be people who have had two doses from the MAD portion already.
They will have had people in the 300 mg, 600 mg, and 1,200 mg cohorts, each having had a baseline dose and another dose eight weeks later. In the first half of next year, we will have the 26-week readout, which will be that full proof of concept.
Okay. You have framed the mechanism as actually regenerative in the sense, protecting follicle stem cells, restoring CD34 progenitors, raising IGF-1 and FGF7. So, two questions. What in the 13 or the 26-week data actually could demonstrate that regeneration rather than just a symptomatic effect? When do investors get a real read on their two to three year durability that you are talking about? It will end up in the pricing eventually.
Well, to your first question, I do not think there is anything that is going to be symptomatic for results at 26-week. Recall, we have looked at this translational model in the stump-tailed macaque, which is a gold standard. Minoxidil and finasteride have been run in that model, and you see the same phenotype that you see in patients. What you see with the prolactin-blocking antibodies, you see this durable effect that lasts multiple years. We took that as an impetus to go run a study in human biopsies, and that is where we elucidated the mechanism that really revolves around repopulating the stem cell niche. So that is the progenitor cells as well as the.
K15+ stem cells. So that is the mechanism in human tissue. If we see growth at 26 week, I think we can be confident that is the mechanism that is underlying it. To your second question, though, we are going to be following the patients in these trials a full year after their final dose. By follow them, I mean we are going to be taking hair measurements. So we are going to collect data on the durability.
We actually expect they will probably see additional hair growth the second six months. That is what you saw in the stump-tailed macaque model. In longer term, we are looking at doing an open- label trial extension. We have not announced what that will look like, but that is something we are looking at right now. The strategy is to achieve endpoints that the FDA accepts, so looking at the 26 week for approval.
But ultimately to establish the durability through a medical affairs strategy where we commission publications, and we share data from these follow-up studies. I think that sort of helps us with the pricing.
Okay. So recently you changed your expectations on the label from androgenetic alopecia to pattern hair loss. Actually, which added quite a bit of a TAM on it, going from 30 million- 80 million U.S. patients. How do you, one, what made you think about that change? Two, how are investors reacting to that framing? Is there any expectations in terms of including females as well into that cohort?
Yeah. A few things. To take a step back, it's purely a terminology matter, right? Our assumptions around the market size, the population size, the community size is wholly unchanged. Still 80 million Americans, 50 million men, 30 million women within the population. But a few reasons we decided to make that change is we've been getting a lot more attention from the broader community, including the medical community, investor community, and just general population. I think there had been, in the broader market, some confusion about, well, what does androgenetic alopecia mean, right? Everyone knows they can look at themselves in the mirror and think, "I have pattern hair loss. I'm losing my hair." Some people might not understand that that could be one and the same with androgenetic alopecia for themselves.
It almost sounds a little too clinical for a general consumer to want to admit and diagnose it themselves. So that was one reason. There's also the biological rationale. We do think that prolactin sits upstream of androgen. We do think there's a little more to the story than just the androgen component. Then just from a consumer-facing standpoint, we think it's just a more consumer-friendly, well-understood terminology there. But it's been well understood that the population we're talking about is the same as it has been all along.
Okay. So regarding the second indication, which is endometriosis, you have guided to start the study in the fourth quarter of this year, and the expectation is to have some initial proof of concept data in the second half of 2027. Just trying to understand the start of the study. Is that because you want to see the 13-week data from the pattern hair loss study before you get started, or these are totally two different events and independent of each other?
Yeah. So our goal is to start that study at the end of this year. What we want to see is the safety and the PK data. The efficacy data for pattern hair loss isn't as germane to endometriosis, but we'd like to see the PK data so we can select our dose and dose frequency for that study.
Okay.
As you can imagine, the safety portion of that study will be part of what is submitted to FDA.
Okay. In terms of the Eli Lilly & Company investment, there's been a little bit of a conversation going on around that. So what is the Eli Lilly & Company investment about? Do they have an option to do more than just the investment, or what else is included?
We've been in discussions and dialogue with Lilly for over a year, and they have a strong interest in prolactin biology. I think we, as a company, are at the forefront of prolactin biology. They're interested in pattern hair loss and endometriosis and some other indications. We tried to find a good solution for how we could work together. Our goal was to not encumber the program with any rights. I think we were able to strike a really great partnership with Eli Lilly, whereby they invested $40 million, anchored $100 million financing, and took a seat on our advisory board for endometriosis. I think for us, this is a big win because we welcome their advice on endometriosis. They get front-row seats. They bought tickets to the game, is the analogy we use. But we did not encumber the program in any way.
No preferential information rights, no rights of first refusal or negotiation. That program is wholly owned and unencumbered.
Okay. When we started talking about ABS-201, you were saying that you wanted to look at the DTC market as the first market to enter into. Is there a certain reason and what is the real strategy behind it, so that you don't have a huge commercial team or what is it that made that decision?
We still are going to work with practitioners. I think the derms have a big role to play. They're very excited about this program. I think they'll help us establish it as the premium new category of therapy. But if you think about the long-term vision for this program, it really belongs in a DTC channel, we think. We're talking about 80 million potential consumers, right? With the rise of telemedicine and DTC, if you think about this product and the way it works, it's a really perfect fit. If you think about how we can structure the pricing through DTC, I think it gives us even more flexibility on pricing and to keep a higher margin. Long term, I think this is a huge opportunity to take this DTC.
But again, I would say we're still planning to work with practitioners, particularly dermatologists, when we launch.
Okay. Regarding the AI platforms that you have, you unveiled Atlas on the second quarter call. It's a target discovery engine, pulling literature, human genetics data, and also single-cell data that feeds into the Origin [models]. So it reportedly helped in understanding the prolactin receptor genetics and also the indications that you're currently on. Can you highlight for us what Atlas can do? Also, what sort of targets is it more like a finding a new target sort of a missionary, or is it something that can actually improve whatever is currently out there in the market?
I'll pull Alex into this, too, but I would say that it's a platform that leverages agentic workflows. It leverages omics data, genetics data. It also helps us design experiments to answer questions for identifying new targets and indications. A lot of it is novel targets, novel indication, or novel targets for an indication. But we have seen its capability in finding new indications for a known target, and that's something that we've used for prolactin. So we initially used it to validate what we were doing in endometriosis and in pattern hair loss, and you'll see some updates in our corporate deck that show you some of the MD work we've done that comes out of Atlas. But we've also used it to identify some new indications.
As you may recall, we announced two quarters ago that we have another anti-prolactin antibody program, ABS-202, that is going to be focused on an indication we haven't announced, but that indication came out of the Atlas workflow. I don't know if you want to comment.
Yeah. Even internally, we see it as a tool that our scientists and our scientific team are able to use to really scale themselves, to be able to do more with fewer resources and find those greater efficiencies. It's a great addition to our end-to-end platform to do that target discovery, experiment design, and then feed into our de novo design model, which as you know, kind of feeds our loop.
You initially started off with the Origin-1, right? Origin-1 actually generated quite a few partnerships for you folks over the time period. Now that you have Atlas, is Atlas also going to be used in generating partnerships, or is Atlas more for proprietary work, but Origin is for partnerships?
Just to make sure we run the flow diagram correctly, Origin is incorporated in Atlas.
Okay.
Origin helps you define a target and an indication. When Atlas helps you define a target indication, then Origin can immediately go to work at helping you design antibodies to specific epitopes that then you can go test in the lab. It's all part of an integrated agentic workflow. To Alex's point, we're seeing a compression of time, and we think better quality as well.
Yeah.
So.
As for who's actually benefiting in this and utilizing it, our business model has really evolved in a way such that programs that we initiate now, we intend to take forward ourselves.
Sorry, no, go ahead.
We do preserve optionality to find partnerships along the way, but as you've seen over the years, the business model of finding outside partners alongside our programs has really kind of shifted to now our internal programs are the key focus and strategic priority of the platform or of the company, and that's where we see the Origin and Atlas parts of the platform really being utilized by the Absci team.
Talking about partnerships, looking at your pipeline, outside of ABS-201, you have quite a few programs, but you also stated that some of these programs are up for partnerships. When you are having these conversations with potential partners, are they waiting for ABS-201 data to come out to move to the next stage of conversations, or ABS-201 data does not really matter in those conversations?
I wouldn't say it doesn't matter because it's further validation. If you look at all the companies trying to, or talking about at least, there's a lot of AI sprinkled on top, but trying to use AI to design molecules, most of them don't have any clinical validation. So it's important in that sense, right? It validates what the model's producing, even though it's looking back two years to when the current molecules were designed. It nonetheless validates the approach we're taking. So I think it's important in that sense. But in another sense, it's really always all about the asset, right?
Yeah.
For any partnership, and our focus, to Alex's point, is not renting out the platform, tying up capacity. That way, the probability of partnerships. If we design a great molecule in a partnership, it still gets killed in portfolio review half the time. You just take on all these unknown risks and timeline. The better strategy, and we've put numbers around this internally, is to build assets, and then some of those we'll take forward, some of them we may partner. And the partnership around assets is always based on the merits of the asset.
Okay. In the interest of time, just to close out on the financials, what's your current cash position and the runway that you can get out of it?
Yeah. We ended the quarter of June with a little over $200 million on the balance sheet, and that's runway into second half of 2028. So that with our current plan, should get us past all the upcoming readouts that we have. So that would be the interim 13-week analysis for the pattern hair loss coming in December, the full 26-week POC coming in the first half of next year. And then endometriosis phase II POC shortly after that, potentially end of next year as well. So really excited times with that first data cut coming in December, and just really excited to share that with everyone.
Okay. Thank you very much. Thanks for being here and appreciate it.
Thanks, RK.