Absci Corporation ($ABSI)

A.J. Welcome to the Jefferies New York Global Healthcare Conference. It's my pleasure to introduce Sean and Zack from Absci Corporation. Happy to see you today.

Great. Thank you. I'm Sean McClain, Founder and CEO at Absci. Absci is an AI drug creation company. We've built an AI native platform at Absci, where we're able to accelerate the time it takes to get new drugs to the clinic and overall reducing the time lines, and we'll talk more about that on later slides. And really creating an exciting differentiated pipeline that's focused on prolactin receptor biology, 2 programs, 1 focused on hair regrowth, angiogenic alopecia and the other focused on endometriosis. We see drug discovery as being -- or AI drug discovery as being able to really industrialize drug discovery being able to have a wet lab in the loop to rapidly design molecules. And now we're actually shifting with Agentic AI, being able to start to use these models to reason through data, being able to help us select the right targets and then fully integrate into our design platforms to design the drug -- and what we've been able to show to date is we've been able to dramatically reduce overall cost and time lines. So traditionally, it takes $50 million to $100 million just to get 1 drug into the clinic. We've been able to show with ABS 101 and 201 that we were able to get these drugs into the clinic with an investment of roughly $10 million to $15 million. Additionally, it takes 5 to 6 years, again, just to give 1 drug into the clinic. We've been able to show that we can get new drugs into the clinic and roughly 24 months and these time lines are continuing to come down and get cheaper. And we see this as being able to fail faster and be able to get the right drugs into the clinic that are ultimately going to impact patients. As I've mentioned, we have 2 drugs that have come out of our pipeline, ABS 201 for androgenic alopecia, which is in a Phase II a study right now as well as endometriosis, which will be starting a Phase II at the end of this year. We're wanting to use AI to create differentiated assets, being able to go after modalities and targets that have been traditionally hard to drug such as ion channels, PCR where we have known biology, but it's been difficult to drug. We've been able to show success with this platform, not only on our own pipeline but also with partners in particular. Overall, we were able to show that we could actually drug an ion channel that had been known for 30 years, but no one has been able to successfully drug it. We recently released an exciting state-of-the-art model, Origin one. This is a design -- an antibody design model where we're able to, for the first time, able to show that you could go after what's called 0 prior epitopes. So targets that have no known binders or structural known binders to that epitope -- and the significance of this is being able to go after hard-to-drug targets, where traditional approaches have to pay out. And if you start to look at it in terms of integrating into an Agentic AI workflow that discovers novel antigens and targets, it can then feed directly into this Origin 1 platform to design the molecule and then design the killer experiment to validate that and rapidly be discovering new novel biology, really faster than we've seen to date. And we see this as a very, very scalable approach. We see the AI platform, not just as a technology, but really putting the value in the assets and making sure that we are investing in the right targets and ultimately taking those into the clinic because that's where the value is at. It's not necessarily on the platform. It's what the platform can ultimately achieve, and that's with these particular assets. And we're really excited to be focusing on prolactin biology. And we have, again, as I've mentioned, 2 assets, that's going after 2 indications, androgenic alopecia, so I think hair regrowth as well as endometriosis. So ABS-201, this is a really exciting asset for us. This is targeting the prolactin receptor for androgenic alopecia. This really hits on a new trend that you're starting to see this total vitality market. We saw that the GLP-1s were able to provide weight loss and a lot of other benefits -- and what we're seeing is that patients and the consumer are not just wanting weight loss. They're wanting their hair to be regrown. They want their wrinkles remove. -- this total vitality market. And we see just an absolutely massive market here, 80 million men and women in the United States suffer from androgenic alopecia. Additionally, there is a massive psychological burden that's associated with losing your hair. It's not just men but women also, especially perimenopausal women that are losing their hair. They have 0 solutions out there. They can't take finestride and Minoxyil gives them hair and unwanted areas. And so there is a wide open space here, a massive market, and we see this being on par with the size of the GLP-1 market. So how is prolactin and prolactin receptor involved in hair regrowth. What we've found is over time, you build up prolactin or you have on the scalp. And that is driving the follicle into a miniaturization state, essentially taking it from this active growth state, this antigen state and driving it into the state. And we'll talk more about the mechanism in detail. But essentially, what goes on is you start to lose the stem cells within the hair follicle -- and over time, that leads to the miniaturization and ultimately, the hair loss that you see. And by blocking the prolactin receptor, you're able to have the follicle go back into the antigen state and convert those vous hairs into robust terminal hair growth. And this is a novel mechanism. For the past 30 years, everyone has been going after the same targets. This is a brand-new target to hit the androgenic alopecia space. Now when you compare this to standard of care, oral minoxidil in mice, we were able to show that was able to provide superior hair regrowth compared to 5% oral minoxidil. You can see the mice, these are shaved mice and you have the control arm, 5% and ABS-201. And you can see the dramatic hair regrowth that these mice experience when compared to oral monoxidil. And what got us really excited in the first place about this particular mechanism were these stem til. So our Chief Innovation Officer, Andreas Busch, prior to his time at Absa was at Bayer, and they were studying the prolactin receptor for endometriosis. And what they ended up finding was when you shave the mice, the mice that were on drug regrew their hair faster. And so they commissioned the stem tail study. And what you see here are baled stem tail -- these are stem cell attacks that naturally go bald. So there's -- they are not -- there is no chemical induction or any modification that go on, which really make us the perfect androgenic alopecia model. And they were on drug for 28 weeks on antiprolactin receptor antibody. And after 6 months of treatment, it's pretty remarkable what ended up happening. They go from their ball gray hair to a full head of hair, jet block -- and what's even more remarkable is post treatment, they continue to regrow their hair. There was a regenerative effect that was going on for up to 4 years. And we believe that the regenerative effect is the ability to rebuild the SEMCO niche. We know that miniaturization happens slowly over time. So you can almost think of it as we're rebuilding the wall. We're rebuilding the machinery and that gives that durable effect that regenerative effect that we have yet to see in this space. So we saw efficacy in mice. We saw it in the same town Macs. The next model we wanted to run was an ex-vivo sample from punch biopsies from humans. And we worked with Professor Ralph House, a leading hair expert in this space and performed a 72-hour ex-vivo model where we compared hair growth and looks at the mechanism when comparing ABS 201 while adding prolactin -- and what we saw was really consistent with the Stump town. So -- what you see here is a microscopic hair imaging of the various different cycles. So as I mentioned, antigen is the active growth state. Catagen is where you start to see the miniaturization occur. And what you see in a very short amount of time is that ADS-201 alone was able to drive the follicle into the antigen state. And when you add prolactin, you can see that it drives the follicle into the catagen state. And when you have prolactin as well as ABS 201, you're able to rescue the follicle. So again, seeing that same mechanism play out in humans or at least human biopsies. I'd mentioned that our belief is that ABS-201 is able to rescue the stem cell niche. And once you rescue that stem cell niche, you're able to drive the rest of the machinery that is necessary to drive the follicle into a sustained antigen state -- and that's exactly what we see is that ABS-201 is able to promote K15 stem cell growth while Prolactin drives the apoptosis of K15 stem cells and so we're able to preserve and prevent their exhaustion that's seen -- and what's really quite interesting as well is that these stem cells are anchored by collagen 17A, and it looks like in studies that we followed up on that prolactin on its own is able to drive the increase of Collagen 17A, which is ensuring that these stem cells stayed anchored to the niche and the bulge, again, allowing them to continue to proliferate and give that sustained durable hair regrowth. Some other pieces that we were excited about from this study was not only the stem cells that we are able to preserve, but additionally, the other growth factors that are important hair regrowth. That's FGF7, IGF-1, IGF-1 were all upregulated, again, important for driving the follicle into the antigen state. And additionally, we did see a decrease of TGF beta, which is a known Collagen driver. So early this year, we started a Phase I/IIa study. We started off with a single ascending dose in non-AGA patients. These were 4 doses, 150 milligrams all the way up to 1,800 milligrams. So far, everything is looking good with that. We do see favorable safety and PK profile. In June or later this month, we will be disclosing the initial safety and PK data that's coming out of the single ascending dose. And we additionally have started the MAD portion, which is in healthy volunteers that have AGA and this is a subcu dosing, and we have 3 different doses here, 300 milligrams, 600 milligrams and 1,200 milligram, and this is going to be dosed every 8 weeks. And we'll have a 13-week interim readout and a 26-week interim read -- or the final radar at 26 weeks. The 13-week readout will be the second half of this year. This will -- the primary end point on that is safety and tolerability. The secondary endpoint is the efficacy. So looking at total area hair count thickness and darkening. And for this we just want to see hair regrowth showing that the mechanism is working. It's a directional readout. It will be a responder analysis. So it will stay blinded looking at the growth of the responders, again, second half of this year, that data will be available. And then the study will complete early next year, and this will be the '26 readout unblinded looking at safety, tolerability, total area hair count, darkness and hair width. As I mentioned, we see this as a large massive market, the standard of care hasn't changed in the last 20 to 30 years. It's still minoxidil and fenesteride. And additionally, you do have hair transplants. And what we've seen from physicians and patients when we've interviewed them is that they are not happy with the current standard of care in terms of advocacy hair regrowth and just being able to take a daily to. And we believe that if we are able to achieve efficacy that is around oral monoxide, call it 30 to 50 hairs per square centimeter and we're able to have a durable hair regrowth. So I think 2 to 3 doses over a 6-month period and you have hair regrowth for the next 1 to 2 years. We believe that is a home run product from the consumer quant studies that we've done. And if we're able to achieve greater than that, we do see the TAM growing much larger. So we did do a consumer quant study looking at those that would be interested in the TPP that I had just laid out. And as I mentioned, there is a large patient population, 80 million men and women suffer from androgenic alopecia. And if you kind of work your way down the funnel here, we do see an overall TAM within the U.S. of $25 billion and globally being $40 billion. And this market is just getting started. Again, standard of care hasn't changed. This is a brand-new novel mechanism and a massive opportunity that we're looking to tap into. The second indication that we're going into with ABS-201 is another underserved patient population, and that is endometriosis in women 1 in 10 women suffer from endometriosis. This is a debilitating disease. It's extremely painful for women during administration. It's an inflammatory-based disease. And current standard of care is not great. And for too long, there hasn't been a lot of investment here. The diagnostic tests aren't great, which we believe is actually underestimating the overall patient population and really excited to be pursuing this particular indication, just due to standard of care being so low and just a massive unmet medical need here.

So how is Prolactin involved in endometriosis?

What we're seeing is very similar to what we see in the scalp that you get high levels of prolactin in the endometrial tissue -- and what that does is it drives inflammation within the endometrial tissue and drives lesion formation as well as drives pain sensitization. And what we've seen from both publicly available data as well as some data we've generated preclinically is that it does look like indeed, at least in mouse studies that Prolactin is driving the lesion formation not that women get on the endometrial tissue and additionally, Prolactin is driving the pain sensitization that women experience during dysmenorrhea. So as I mentioned, this is a very large patient population, 1 in 10 women have endometriosis is a very debilitating disease -- and we see the market opportunity for this being over $4.5 billion and excited to be pursuing this in an area that has been very much overlooked and underappreciated, and we do believe that we could be bringing forward a treatment that is truly disease-modifying. And so over the next 24 months, we have exciting readouts that are occurring first off, June of this year, this month, we'll be having the safety PK readout on the Phase I/IIa study for the portion, looking at initial safety, and PK data. Later this year, in the second half, we'll have the 13-week interim readout on AGA. And then we will be starting the endometriosis study at the end of this year, Phase II study. And in the beginning of next year, we will be having the full 26-week readout on. So it's a very exciting 24 months for us with multiple readouts and yes, 2 Phase readouts...

We'll open it up for questions. Thank you.

Thank you.