2025 Healthcare Conference. It's my pleasure to now introduce Sean McClain, CEO, and Zach Jonasson, CFO of Absci Corporation. Just a reminder, there will be a 20-minute presentation following with a 5-minute Q&A. Thank you.
Thank you. I'm Sean McClain, the founder and CEO of Absci. We are a generative design company focused on using AI to really tackle some of the hardest problems in drug discovery, being able to go after some of these undruggable targets that have known biology that traditional approaches have been struggling to ultimately drug. Now, if you look at AI drug discovery over the past few years, there's been a lot of companies that have been emerging, and they talk about new model improvements and ultimately what the efficiencies of these models are. At the end of the day, we really need to see the translation in the clinic. In the next 24 months, we're going to see how that translation looks with two of Absci's assets, ABS-201 in AGA, as well as endometriosis. We'll have two phase two readouts in the next 24 months.
If you look at our team, we're not just AI scientists, but we're multilingual. We have an AI team, a disease biology team, as well as a bunch of drug hunters that ultimately work together to leverage this technology to ultimately go after diseases that have been difficult to address in the past. Additionally, we have a data flywheel where we're constantly learning. We're able to take data from our 77,000 sq ft automated wet lab and be able to use that data to ultimately train our models and rapidly learn. In a six-week time period, we can go from data in the wet lab to training our models and continuing to learn what AI architectures are ultimately best for generative design. This has led to us being able to have really big breakthroughs in de novo design of antibodies.
In particular, we've been able to address some really hard and challenging targets, such as ion channels. In a partnership with Almirall, we've been able to actually block an ion channel that has been difficult to drug for the past 30 years. Additionally, we've been able to drug the HIV capsid region in partnership with Caltech. As I said at the beginning, we are seeing exciting translation of this technology. With ABS-201, we have very exciting catalysts in the next 24 months: two phase two readouts, one in androgenic alopecia, this is common baldness, and then additionally in endometriosis. If we look at Absci's strategy, we're looking to apply our de novo design technology to tackle hard, challenging targets that have yet to be drugged, that have known biology. These are GPCRs, ion channels.
How the model works is you take a target of interest, you identify the epitope you want the antibody to bind to, and you're able then to generate the CDRs that can bind to that particular epitope of interest. Now our scientists are actually able to start to test hypotheses that they have been unable to attain previously. This is where we can specifically engineer in agonism versus antagonism, turning on or off a particular pathway, or let's say creating switches where you can bind in the tumor microenvironment but not bind in healthy tissue. These are all ways that we are leveraging our AI platform to really create differentiated assets. We, as a team, figure out which of these we want to partner and then which we want to ultimately take forward ourselves.
In particular, we see a really big opportunity with ABS-201, and this is going to stay wholly owned by Absci. The rest of the assets we do look to partner. At the end of the day, it's the team that has gotten us to where we're at. We have incredible AI scientists and wet lab and disease biologists that work together to ultimately get these cutting-edge therapies to patients. We call ourselves Unlimiters, really being able to achieve the impossible. Roughly 140 Unlimiters strong. We have a 77,000 sq ft campus in Vancouver, Washington, where we're able to leverage that wet lab capability not only for generating data for training our models, but also being able to do all of the disease biology validation for the particular targets we're going after.
Currently, as of Q3, we have $150 million on the balance sheet, and that gets us runway into the first part of 2028. I like to think of this industry as a team sport. We all need each other to ultimately get these breakthrough therapies to patients. We partner with large pharma in disease areas that we are not focused in on to ultimately co-develop assets together. We've had partnerships with AstraZeneca, Merck, and then additionally, we have data and compute partnerships with NVIDIA and AMD. AMD is actually one of our top shareholders. They recently made a $20 million investment in Absci, and we're using that partnership to really drive innovation within our AI models. Today, I'll be going through one particular asset, ABS-201. This is an anti-prolactin receptor antibody. The two indications that we are pursuing with this are androgenic alopecia as well as endometriosis.
As I stated previously, within the next 24 months, we'll have phase two readouts on both these indications. Now, diving into ABS-201 in androgenic alopecia. Hair is really important to individuals. If you talk to patients that are losing their hair, it is very personal. It's about their identity. It's about confidence. When patients are losing their hair, they lose their confidence. They end up becoming, a lot of them are depressed. By regaining their hair, they're not just getting vanity back, but they're really getting identity. This is a condition and a disease that has been underfunded and underlooked for quite some time. We see this as a really exciting opportunity. The patient population is extremely large. 80 million Americans alone suffer from androgenic alopecia. The standard of care is extremely poor.
Patients are ultimately not getting the efficacy they want as well as the durability. If you look at minoxidil, minoxidil only works on roughly 50% of patients. The topical is not very efficacious. Oral, you have to take that for life or you will lose your hair. There is no regenerative aspect with minoxidil. This is where we see a huge opportunity for ABS-201 because we see actual durable hair regrowth with ABS-201. It is truly regenerative. We will be diving into what that looks like on the following slides. Diving into the actual mechanism of action for the prolactin receptor, if we step back and just look at the hair follicle growth cycle, it has three stages. First is the anagen phase. This is the active growth phase.
You stay in this phase based on your genetics for anywhere from two to six years. You go into the catagen phase where you get apoptosis and regression. Ultimately, the telogen phase is where you have shedding. What happens over time is individuals, as they age, end up building more prolactin locally on the scalp. This drives the follicle into the catagen state. It continues to stay there due to the high levels of prolactin. By blocking the receptor, you're able to ultimately shunt the follicle back into the anagen state.
We will be sharing exciting ex vivo data at our KOL Summit on December 11, where we actually are able to show by blocking the prolactin receptor, you are actually able to restimulate the follicle stem cells and actually see the hair shaft growth as well as melanin production as well. We believe that, and you will see on the next slide here, that this should lead to very efficacious hair growth, but also durable hair regrowth. This target was actually discovered by our Chief Innovation Officer, Andreas Busch, when he was at Bayer. They were actually looking at the prolactin receptor for endometriosis. When they dosed with the drug, they ended up finding out that the mice that were on drug regrew their hair faster than the control arm, which led them to actually investigate prolactin in AGA and hair growth.
They did a study in the stumptail macaques that really got him excited and got us excited as well. What you are seeing here is the tops of these stumptail macaques. The stumptail macaques naturally go bald. The treatment was over 28 weeks. After 28 weeks, treatment stopped, and the monkeys were monitored for up to four years. You can see when the treatment started, they were completely bald, and they had gray hair. After 28 weeks of treatment, they have most of their hair regrown. Not only is it regrown, it is repigmented as well. They have gone from their gray hair to their naturally colored hair. What really blew us away and got us really excited was the durability.
Post-treatment for up to four years, they continue to regrow their hair, which then ties back really nicely to the mechanism of action. Once you're in that anagen state, you stay there for anywhere from two to six years. We believe that this will be actually a condition or disease-modifying drug where you'll essentially have post-treatment, and you could get durability for two to three years by blocking the prolactin receptor. Additionally, we took ABS-201 and wanted to show the superiority versus standard of care, which is minoxidil. This is a mouse shaving study. These mice are naturally in the telogen phase. We then dosed these mice with both ABS-201 as well as minoxidil.
You can see that with ABS-201, very rapidly, you're able to see that the hair follicle is shunted into the anagen phase, and you get really pretty instantaneous hair regrowth compared to minoxidil. Again, nicely validating the mechanism of action here. All right, great. The efficacy is there, durability. What about safety? Are there any issues? If we're going direct to consumer, we really need to make sure that this is a safe mechanism and a safe drug. There is really great human genetic data out there where a few families were actually studied where they had loss of function mutations of the prolactin receptor. These individuals were completely healthy. The only issue that the women had was the inability to lactate. We do see this pathway, given this human genetic data, as a very safe mechanism.
Tying out the AGA story here, we'll end with the upcoming catalyst. December of this year, we will start our phase one two-way study. This study will start with a SAD component, which will have healthy participants. Then we'll quickly go into the MAD study where we will be looking at healthy volunteers that have androgenic alopecia. The second half of next year, we'll have the first efficacy interim readout looking at the hair regrowth. We're really excited to start this trial in December. We've actually accelerated our timelines from previous guidance, and we'll have, again, that efficacy readout in the second half of next year. All right, diving into endometriosis. The other major indication we can go after with the prolactin receptor is endometriosis.
Endometriosis and women's health in general has been underlooked, underserved, underfunded, and there has not really been much innovation. Yet, this is a disease that is prevalent in so many women. One out of ten women have endometriosis. This ends up leading to pelvic and menstruation pain for these women. It is a completely debilitating disease. The standard of care is extremely poor. It is off-the-shelf pain medication. There are some hormonal treatments such as GnRH. These hormonal treatments really have pretty poor side effects. There are not a lot of women that ultimately take these hormonal treatments. The opportunity for innovation in this space is huge. We are really excited about the prolactin receptor as not only being able to treat the pain, but actually be disease-modifying. Let us just take a look at how prolactin is involved in endometriosis.
What ends up happening, very similar to AGA, instead of building prolactin locally in the scalp, women develop prolactin within the lesion. This prolactin is actually what drives the overall growth of the lesion as well as sensitizes the neurons, which ends up leading to the pain, the pelvic pain that they experience as well as the pain during menstruation. By being able to block the prolactin receptor, you can actually shut down the pain as well as reduce the overall lesion development. There are two pieces of fundamental preclinical research that got us really excited about this mechanism and were able to show both the reduction in overall pain as well as decreasing the lesion formation. The first major work is here on the left.
A group was able to show by blocking the prolactin receptor in mice, they were able to dramatically decrease the overall pain that was experienced within these mice. Additionally, there's another study that was able to show that by blocking, again, the prolactin receptor, you're able to decrease the lesion size and actually bring the disease state back to a normal level. We then took ABS-201 and wanted to compare it to standard of care, the GnRHs, within a mouse pain model. We were able to show that we could indeed reduce overall pain by blocking the prolactin receptor, as well as we were able to show a decrease in the inflammation cytokines as seen here on the right. As I mentioned, this is a large addressable market. One in ten women have endometriosis.
There are currently no non-hormonal treatments out there for women to, again, reduce overall pain and lesion formation. We believe, based on the preclinical data and some clinical data on HMI-115, that we actually believe that this could be a disease-modifying treatment. If this truly is disease-modifying, we do believe that that actually will increase the diagnostic rate of these women that have endometriosis. Based on all of that, we do see potential peak sales of greater than $5 billion. That is due to the fact that there is very low or poor standard of care, and it's a very large unmet medical need. To summarize, we again have a very exciting 24 months coming up. We have ABS-101 that we just dropped up in our phase one. We're looking to out-license that particular asset in IBD.
Additionally, we do anticipate signing one or more new partnerships with a large pharma either this year or next. Additionally, we have two phase two readouts coming in the next 24 months in AGA and Indo. With that, I'll open it up to questions. Your AI platform, do you guys also have, is this your own platform? Second question would be, do you have molecular dynamics that you can see, that you can test in actual life? Yeah, absolutely. This is our own model that we have developed in-house. Actually, the molecular dynamics is a great question. We are obviously using structure-based data along with sequence and function data. One of the things that we are seeing as an exciting area of development is actually using MD simulation to generate synthetic data.
Actually, combining both the synthetic data along with the wet lab data that's generated is definitely a key part of our overall strategy. Certainly, human genetic repairs, whether for lower prolactin or lower prolactin receptor, have less basic cytokines? That is a great question. I don't believe that has been studied, but that's definitely something I can follow up with the team on. Yeah, that is a great question. That molecule, first off, the safety that was done or the phase I safety study, they only went up to 280 milligrams, which we believe, based on the data that they saw both in AGA as well as endometriosis, that they're severely underdosing and they're not reaching greater than 90% receptor occupancy. In order to do that, they'd have to repeat their phase I study.
Additionally, they have a poor formulation and a poor half-life. In order to be commercially viable in AGA, they'd have to have 24 doses to ultimately achieve the efficacy in the monkey versus we would have two to three doses over that six-month period, which we see being commercially viable. We do believe being able to actually get to that 90% receptor occupancy is ultimately going to be key for overall development. We do believe that we have a superior drug that has been developed compared to the HMI-115 molecule. I'm a woman. Just how do you prove that this isn't a party? It's really incredible talent. Again, it has been studied with the human genetic data of the women that have had prolactin receptor knockout. They have been fertile and they have had children.
They just have not been able to lactate. I think that there is strong human genetic data that supports that this should not affect fertility within women. Hello? Hello. Oh, hi. Yeah. Thank you for your presentation. I'm thinking about the alopecia program. You mentioned that the prolactin is localized mainly in the scalp. Yes. In your non-human primate data, are you seeing mostly just hair growth in the scalp, or are you also seeing hair growth in other regions of the macaque? The hair growth was specific to the overall scalp. There was not hair growth seen elsewhere. Again, that is just driven by the fact that, over time, you see the prolactinemia on the scalp, but that does not necessarily occur elsewhere on the body. The other thing I will mention is that you are not going to grow hair in unwanted places.
It's essentially just restimulating follicles that have already been created. It's not creating new ones. Great. Thank you.