Good morning, everyone. Thank you for joining the 25th Annual Needham Healthcare Conference. My name is Joey Stringer, and I'm one of the biotech analysts at Needham & Company. And it's my pleasure to introduce our next company, Annexon Biosciences. And joining us today from Annexon is President and CEO Doug Love. For those of you joining on the webcast, if you would like to ask a question, you can do so at any time. You can submit a question using the chat box feature at the bottom of your screen. So that will get started. Doug, thank you so much for joining us today.
Thanks, Joey. Appreciate you having us.
2026 certainly looks to be shaping up to be a pivotal year for an Exxon. But before we get into the details of the pipeline, what are the company's top priorities? And can you briefly touch on some of the key milestones and catalysts for this year?
Yeah, we're super excited. Thanks once again, Joey, you and Lidam. We always appreciate this conference, an opportunity to talk to folks. Super excited about 2026. So as you know, we've been on a 12-year journey to get to this year. This we call inside a win year for multiple reasons. So as you know, from a mechanistic perspective, what we're doing is very different in bringing forward targeted immunotherapies in that we're stopping all of the classical complement pathway before it gets started. I imagine we'll talk more about that. But over the course of 12 years, we've prosecuted this approach in a range of different diseases across the body, the brain, and eye. All of this is homegrown, wholly owned technology with a really robust research foundation in the work that we're doing. That's led us to a series of late-stage registrational programs, which we're really encouraged by. So from a milestone perspective over 2026, I'll just call out a few. First, starting with our most advanced program, Guillain-Barre syndrome, or GBS, on the backs of a very successful phase three pivotal study there, we filed for regulatory approval with Europe, and we anticipate approval in the first part of 2027. We also anticipate filing for regulatory approval later this year with the FDA, so a key milestone event for us there, getting that BLA on file. Second to that would be ANX1502, and I'm really going in order of the milestones as I anticipate them coming over the course of the year. ANX1502, a small molecule, the first small molecule targeting the classical pathway. We think this is a really important drug candidate, not only for us, but for the marketplace as a whole. The target activated form of C1S has been clinically validated and is regularly treated with IVs or sub-Q approaches, which are now being studied in the clinic. The opportunity to bring patients a small molecule to take in the comforter of their home to treat devastating neuromuscular and other diseases is really encouraging for us. We're in a proof of concept study, which we anticipate reading out also this year before what I'll talk about next, which is our kind of last and most important, I would say, milestone this year, which is our phase three readout in geographic atrophy. This is yet a third drug candidate, again, targeting the classical pathway, voniprumit, and there on the backs of what we consider a very informative proof of concept study where it's the only program to demonstrate significant vision preservation, as well as significant preservation of photoreceptor neurons in the eye responsible for visual acuity in the center of the eye, which is the locus of where visual acuity is housed. So, on the backs of that data, we've run a large phase three program, which we anticipate reading out in Q4 of this year. So, a milestone rich year for us after, you know, 10, 12 years on this journey.
Doug, in lead asset fund improvement, the C1Q mechanism, can you walk us through that and what differentiates it from some of the other complement targeting approaches, such as targeting C3 and C5?
Yeah, really good question. So as we like to say around here, all complement is not the same. C1Q is the most unique complement component in the classical pathway in that it is a recognizing molecule that localizes on diseased tissue, anchors, and activates the entire inflammatory cascade. So that's really, really important. That is what we're targeting. In the case of geographic atrophy, which is very different from the downstream approaches C5 and C3, C1Q localizes on photoreceptor synapses in cells responsible for visual acuity, drives harmful inflammation to damage and ultimately kill these photoreceptors that are necessary for vision. So our approach is simply to block C1Q right where it's localized on disease tissue. It's really akin to stopping this inflammatory cascade, which amplifies and gets far more aggressive as it continues to trigger before it starts. So you never get on the cascade by blocking C1Q. And so that's led to very differentiated outcomes in our GA program. But not only that, we've also looked at C1Q, as you know, in GBS, where C5 has been studied in phase two and phase three studies, very different outcomes. We've looked at C1Q in ALS, very different outcomes where C3 and C5. So this is a very consistent finding in what we're seeing in geographic atrophy, stopping harmful inflammation before it starts, right where the locus of the disease is, is essential to provide function, in this case, vision preservation, which we have not
seen with the downstream approaches. And continuing along the theme of your geographic atrophy program, the most advanced in the latest stage, really a lot has changed in the GA space since when you first started your clinical program. So can you give your high-level thoughts on the evolution of the field over the last several years and its current state, and what are the true unmet needs here and value creation opportunities in the GA field as you see it?
Yeah, really good question, because it indeed has been an evolution. If you go back, You know, eons ago, giraffe atrophy was always known as a neurodegenerative disease. That storyline got lost over the last 10 years or so, which I would consider the advent of various technologies to assess various structures in the eye, one being fundus autofluorescence, which measures or assesses RPE cells that are underneath the photoreceptor neurons in the eye. Because you could now image what was going on in the eye, and RPE cells are thought to provide and do indeed provide trophic support for your photoreceptor neurons, everyone really kind of globbed on to that is the approach to geographic atrophy. Bear in mind, no preclinical data that I'm aware of showing that by protecting RPE cells, it translates to functional benefit. Be that as it may, you know, companies move forward with that. And we were delighted to see that the regulators were really quite supportive of that approach and frankly, really helped advance the field by allowing companies to start study a biomarker surrogate for vision in a chronic neurodegenerative disease. That's very rarely done, pick the disease, whether it's in the brain or in the eye. That was done here in GA. It brought in a lot of investors and a lot of companies to study the disease, which we think is a very good thing. Unfortunately, targeting RPE cells has not bore out to show preservation of function or protection against vision loss. And so now that we're four or five years into data sets looking at protection against RPE growth, we now know that to be the case. And we also know a second thing, which is you're not going to get worldwide approval by pursuing RPE fun structure unless it shows functional benefit. So Europe has not approved those drugs. What we are seeing now more recently is a return to the story of geographic atrophy is indeed a neurodegenerative disease. Photoreceptors are the neurons in the eye that are responsible for visual acuity. So whatever you are doing in this disease, you probably need to have an effect on the photoreceptors and protecting them before they die. When neurons die, they do not regenerate. That is classic neurodegeneration. So you want to preserve photoreceptors in the eye. And that's exactly what we're doing with our approach. is what led to the discovery of an exon is the underlying technology discovered in Stanford by the late Dr. Ben Barish, Chair of Neurobiology there, on the role of C1Q in protecting against the loss of photoreceptor synapses and neurons to preserve vision.
Can you describe the key characteristics of voniprumen and compare this to the approved GA drugs, Sifovir and Iservay? How do you think voniprumen is differentiated?
Yeah, it's a really unique drug candidate. So first, it's a FAB fragment of our full-lift monoclonal antibody, Tanrubivar, which is what we're using for GBS, which has been really well tolerated in GBS, Huntington's Disease, ALS, and others. Vonoprumid is a FAB fragment. So it's a small 50KD FAB antibody. It's non-pegulated with low viscosity and high potency. That's really important in terms of administering the drug and hopefully doing away with some off-target safety events that we've seen in some of the other programs. The other thing I would say is, you know, it's a 5-mig administered dose, 25 microliters. That's about a quarter of the size of the approved drugs. They're about, you know, 100 microliters. So it allows for a really rapid administration of the drug as well, which we think is a competitive advantage. So we like vonaprumic quite, quite well. Last thing I'll say is we were able to demonstrate by pulling aqueous humor from patient eyes that we have full C1Q target engagement before we even started our Phase II study. So it's a well-designed drug candidate, and it does exactly what we wanted to do in the eye.
The Vaughn Improvement Phase II Archer trial in geographic atrophy, that readout was in 2024. Now, we did miss on the 12-month primary endpoint of lesion growth, but showed improvements across a variety of visual acuity measures. So can you just review the key data from this trial and put those results into context for the ongoing Phase III trial?
Yeah, absolutely. So first, as it relates to the primary endpoint, RPE preservation against lesion growth, we showed a trend on that at 12 months, which was increasing over the course of the study. In fact, it more than tripled in the second half of the study, and we think that's, from a mechanistic perspective, makes a ton of sense. We are targeting the loss of photoreceptors, which you lose before you lose RPE sales. So, in effect, our approach is top-down. So, imagine protecting your photoreceptors and then your RPEs. So, it will take longer for us to protect RPEs, which gets stronger as you protect your photoreceptors, and we see that in our data. Importantly, turning to the vision aspect of things, we did two things. First, on the structural element, photoreceptors themselves, the neurons I referred to previously, we showed a really significant preservation of those photoreceptors across the entire eye, the pan macula, and importantly, an even more pronounced protection against photoreceptor loss in the center of the eye, in the center of the retina that is responsible for visual acuity. So we're really encouraged by that. we have not seen that data set from any other company protecting photoreceptors in the one and a half to two millimeters in the central retina. Really, really encouraging because that is the how, which then results in the what. The what is, what that led to is significant vision preservation on every measurement of visual acuity in this study. Best corrected visual acuity, 15 letter loss was the pre-specified secondary endpoint on that study. And there we showed a significant preservation of that endpoint at month 12. We reduced the risk of losing 15 letters at month 12 by 73 percent, so a really significant protection there. We also showed the protection of vision in low light visual acuity, which is really best visual BCVA in low light conditions, as well as other measures. So really encouraged. Perhaps the most important aspect of the data that we showed from a vision protection perspective is this study was designed as a 12-month study with a six-month off-treatment follow-up period. So we were able to assess what happened with patients once we removed the drug. And what we see there is that we protected patients' vision over the course of the 12 months, in effect, resetting their baseline for vision loss. Once we stopped drug, these patients didn't rebound and immediately lose vision on the same level in which the sham arm did, but they did begin to lose vision once we stopped drug. So we really like that kind of confirmation that that's a biological effect from your drug's intervention and protecting of photoreceptor cells. And Doug, I'm curious, what's the current
level of understanding of von improvement's differentiated mechanism here amongst GA treating physicians? And how well is the vision preservation aspect of the drug resonating with
docs? Yeah, really good question. Growing is the short answer on that, Joey. I will say that when we first came out with the data, our approach to targeting photoreceptors and preserving vision was very rarely talked about. The storyline had been all around RPE growth. And for good reason, you had two companies coming through at exactly the same time, both through phase three and ultimately get approval on an RPE story. So a large share of voice. However, as we have continued to educate, we and others on the, this is a neurodegenerative disease, you have to preserve photoreceptors and walk physicians through our data. We have seen a real swing on that and physicians really globbing on to our program and what we're doing. The best evidence of that is our phase three study that's underway. That study, we were able to enroll 659 patients. We over enrolled it by 30 patients and had to stop this study or would still be enrolling candidly. And we did that roughly two months ahead of schedule. So physicians have really poured into this program as they've begun to understand what we're doing from a neuroprotective perspective, which we're really encouraged by. And we know that patients are remaining on drug. So I'm sure we'll talk about the phase three, but we're encouraged that physicians are now really leaning into this
approach. Yeah. And the phase three ARCHER trial, you mentioned it. Just could you level set us on the trial design here, when we can expect the data, and then what data do you plan to disclose
in the top line readout? Yeah, it's very similar to the phase two study, which was called ARCHER. This is ARCHER. This is now ARCHER II. It is a study that has two arms. So we've got an every month treatment arm versus sham, two-to-one randomization on that. The primary endpoint is at 15 months. And this is the first and only program we're aware of in geographic atrophy where the primary endpoint is indeed preservation of vision. So protection against best corrected visual acuity, 15 letter loss at month 15. We'll be looking at a host of key secondary endpoints as well, but that is the primary endpoint for which we have alignment across the globe from a regulatory perspective. So we're really encouraged by that. Again, the study is very similar to what we did with the Archer study. I would say there are two things that we did that we think enhanced the overall PTS of the phase three, however, and that's just with regard to making sure we really honed in on the patients who are most likely to lose vision in this study and so that we can show protection. What we saw in the phase two study is about 20 percent of the patients were so far advanced in their geographic atrophy when they came into the study that there was no more vision to protect. So we set a cutoff for patients at their baseline vision, visual acuity, so that we can assure ourselves that we'll get patients who will progress, but there's also room for show protection with our treatment, which we really are encouraged by. The second thing we did was we made sure that we enrolled roughly half of the patients are foveal patients. Foveal patients are patients who have more aggressive disease. They're advancing more quickly. And again, really with the aim of making sure that we're picking up patients that are showing this decline in vision, but they're not so far gone. So those two enrichment criteria we like, we think, as I said, adds to the overall PTS of the study. But other than that, we're just looking to replicate
what we did in the phase two study. Yeah. And just a further ask on those two points, Doug, the phase two versus the phase three. Can you remind us what the requirements were in phase two in terms of the percent foveal patients and the BCVA baseline or the cutoff there? Just in terms of, I know that you're enriching for those patients in phase three, but how does
it compare to what you did in phase two? Yeah. So in the phase two, we really focused on lesion size versus BCVA cutoffs in the phase two, because again, the primary endpoint was RPE. So it was really based on the learnings from our phase two study, coupled with a really large natural history data set, the LAP and Luzumab data set from Roche, 2000 patient data set that we were able to get access to. We were able to really understand that you do not want patients whose BCVA scores are too low in baseline. We cut it off at 45 letters. So we did not do that in the phase two. And that is really the key difference in the phase three, ensuring ourselves that every patient that is in the study is one that we can show benefit in. And I just think that's really, really important. Can you disclose anything on the powering assumptions of the trial, of the phase three trial? Yeah, we've not given specific numbers, but, you know, what we've done in that study is we've powered it really well for a phase three study. So it's a really well-powered phase three study. Again, based on number of events of BCVA 15-letter loss over a 15-month time period, we have a real good understanding of what to anticipate with regard to that, again, based on our data from the Phase II study and the Lampaluzumab data set. So with those two data sets in mind, we looked at the sham arm, which lost about 16.9% over a 12-month period of time, very similar to what you saw in the Lampaluzumab study. That's what we saw in our Phase II study. We took that number and we were conservative for powering in our phase three. So we took a haircut on that number, you know, anticipating a bit of a regression to the We also protected vision with our every month treatment arm, about four and a half percent of patients. And so there we also were conservative. So we're conservative on both bookends from a powering perspective. And we really stand on two feet really solid about really well powered for phase three
Got it. Got it. Makes sense. And obviously, BCBA, primary endpoint, what about secondary endpoints here? What are the most important secondaries for investors to focus on?
Yeah, we like them all. Again, we know so much more about this disease and visual acuity than what was known prior to the Phase 2 study that we ran there. So we'll be looking at both structure and functional secondary endpoints. On the structural side, we'll be looking at EZ and just making sure it's consistent with what we saw in our Phase 2. and importantly, what we see in the phase three from a clinical perspective. And that really, again, is the how. And it really, from a commercial perspective, is really nice to go out and talk to physicians about impact on structure as well as functional benefit. We'll also be looking at low light visual acuity. It's an endpoint that there's less known about. Just no one's studied it as a primary endpoint, but it's been in studies. And we just think it's upside. You know, we rarely do have perfect light. So we'll be looking at both of those in particular, among other things.
For the EZ loss secondary, what's considered a minimally important clinical difference? And what's the bar for success on this endpoint? And can you maybe expand upon why this could be important from a label or a commercial uptake perspective?
Yeah, well, maybe in reverse order. I think it could be really important because, again, it's the how. And you think about retina specialists, they really do focus on structure as well as function. So it gives you two things in your argument, if you will, for why this drug versus something else. And they all are really well adept on that is your photoreceptors, your neurons in the eye responsible for visual acuity. So showing protection on that, I think, is really quite important in terms of uptake from a commercial perspective. The first part of your question on what is considered clinically meaningful, again, it needs to be directional and consistent with what we see with the functional data. This is, as I am aware, of the first kind of phase three readout with EZ as a likely, we haven't made the final determination, but likely to be a pre-specified secondary endpoint. And so there will be learnings with regard to that. But so the way we've had the discussions internally with regulators, directionally supporting the functional outcomes will be useful for us.
And what are the regulatory requirements needed for approval from both FDA and EMA?
Yeah, there it's quite simple. It's winning on best corrected visual acuity, 15-letter loss. And this is the gold standard endpoint for visual acuity studies. If you go back, you know, a few decades, a couple of decades to wet AMD and then various other ophthalmic diseases thereafter, best corrected visual acuity, 15 letter loss is the end point. And the reason for that is it's a really high bar. It represents 50 percent of your vision loss over a 12 month period of time. You, in effect, lose your independence. By law, you're required to turn in your driver's license. And, you know, there's been 34 or so phase two, phase three studies that have not hit on this endpoint. We are the only ones to do that. And so it is a recognized endpoint that if you show statistical significance on best corrective visual acuity, 15 letter loss, you have an approvable drug.
Can you talk about the FDA requirements for two adequately well-controlled trials that that phrase is used needed for approval? So I guess, does the Phase II ARCHER trial satisfy one of the requirements in FDA's eyes, or what are the puts and takes of doing two separate analyses here on the two sub-studies in Phase III on the primary endpoint versus just doing a single analysis and then perhaps using the Phase II ARCHER trial to meet the other trial requirement for FDA?
Really good question. So we think at a minimum, Archer, the phase two study is supportive. For purposes of the FDA, historically, to get a label in ophthalmology and certainly in the retina space, you needed two well-controlled phase three studies. We initially were going to run two well-controlled phase three studies, one against SIFOVRI, one against SHAM. When we went in to meet with the regulators, SIFOVRI had had some safety events that were deemed potentially unmasking in a phase three study, so we were unable to compare against that. So the FDA, which we really appreciate, made the recommendation to us that we run a single protocol study and do two sub-analyses of the patient population. And so what we did was we went from a study that was going to be 300 and something patients and increased it up to 659 to ensure that each sub study is individually powered at a phase three level. And so we, in effect, have two studies under one protocol. What we love about it is, is that it really simplified the execution of the study and it squeezed out some of the risk from the study. By doing this under one protocol, we can assure ourselves that we won't have misalignment in patient populations between the two sub-studies, among other things, which we're really, really are encouraged by. Excuse me. So really straightforward analysis. We'll get the data, and we will then do the two sub-study analysis. In Europe, they're not seeking that. It's just a single analysis of the one protocol, 659 patients. So it's exceedingly well-powered. I will just note, we are going back to the FDA to have a discussion on what, you know, Dr. McCary, the commissioner, has more recently said that a single phase three study is all that's necessary for approval. Don't know if that applies to ophthalmology. We'll learn that answer. We consider that an upside scenario. So, yeah, that's the overarching answer for that.
And the phase three trial that's ongoing, you're tracking progress in a blinded fashion. So what are you seeing on event rates, both in terms of efficacy and safety? Are these tracking with your internal expectations?
Yeah, it's really important. So the key to winning on this study is that you have enough BCVA 15-letter events over the course of the 15 months. If you don't have enough events, you can't show a delta with the protection of your drug. And so we are tracking that in a blinded fashion. And we set that number, again, based on what we saw in our phase two and what we see in the lap and luzumab data set and the event rates there. So 2,000 patients can't really do better than that from a source of information to set your event rates. And what we're seeing is that from a blinded fashion, we very much are on top of our targeted event rates. We do not know who's on drug versus not on drugs, so it's blinded with regard to that, but we're very encouraged by that. Secondly, the DSMB continues to meet regularly. And so far, everything has gone really, really well with regard to that. I think one of the things from an execution perspective that also gives us a lot of encouragement is that we're having a really high compliance rate with regard to this study and very low withdrawal rate. I mean, this is an elderly patient population. The average age is in and around 80 years old coming into the study. So it's something you want to be mindful of, both compliance and withdrawal. And both are also exceeding our targets with regard to that as well. So we're really encouraged with the way this study is being executed.
Great. Well, so I guess if the phase three data are similar to what the phase two showed and the drug is ultimately approved, where do you see Fonaprumit fitting into the GA treatment paradigm and what could a potential label look like? And I guess what types of GA patients would this drug see the biggest uptake in?
Yeah, well, we think this drug makes a lot of sense for all patients first. I will say that. And one of the things that we saw in our phase two data set that I didn't refer to is that patients who had, in effect, healthier eyes or earlier in their disease process, zero out of 56 of those patients lost vision on our drug versus roughly 17 percent in the treatment arm. What that tells you, like virtually all neurodegenerative diseases, the earlier you treat, the better your outcomes. You really want to stop the damage and death of neurons early in the process to give yourself a better outcome. So we think certainly treating earlier in the disease is important, but also later in the disease, as we showed in our data in the phase two, foveal patients and others that were a little further along in their disease, we had a pronounced effect in that population as well. So we think this drug becomes the market winner, if you will, if I can say it that way, in fact, it is the only drug on the market that offers an advantage with regard to vision protection, protection of the structure in your eye that's responsible for vision. And, you know, again, the fact that it is not peculated, low viscosity, one of the things we saw from a safety perspective in our phase two study was virtually no difference in conversion to wet AMD in our program versus the sham arms. That's really, really important. By targeting C1Q in the classical pathway, we are allowing the other two complement pathways, the alternative and lectin pathways, to continue to function, which perform an immune function and really help support the overall health of the eye. That's different than the downstream approaches, C3 and C5, where they're seeing conversions to wet AMD somewhere in that 12 to 15 percent range. So it's a really different differentiated product, both on efficacy as well as on safety. And the last thing I'll just say is the administration, as I said before, you know, 25 microliters, 50 KD. It's just a really fast, quick administration into the eye, which makes it more tolerable for the patient.
Yeah. Now, you're also working on a pre-filled syringe for Vonna Prumant. What's the status of this and how important is it to have a PFS for commercial success?
Yeah, it's very important. Yeah, so we think, you know, obviously we want to partner with the retina specialists in the world. We're treating patients, lots of patients. These are big, big, big patient populations, both this and AMD. Like all the other drugs, we will launch with the current presentation, which is not pre-filled syringe, but we'll be then transitioning to a pre-filled syringe solution, which is underway. We're running, we will launch with what the presentation is in the phase three, though, which is standard. But that work is underway and we look forward to bringing that
to the market as well. Great. Well, we'll transition from Geographic Atrophy to Guillain-Barre, your GBS program. You mentioned you submitted the MAA to the European regulators earlier this year, but BLA submission to FDA is still in the works. So what's the latest update on how the talks with the agency around requirements are going needed for BLA filing? What are some of the key sticking
points? Yeah, really good question. First, for those who may be less familiar with GBS, really important disease. It's number one cause of acute neuromuscular paralysis. You're healthy one day, you have this aberrant attack of your immune system, and three or four days later, you can't get out of bed. One in four patients are on a ventilator, you're in the ICU. So really devastating disease. What's important about our data is a couple of few things. One, these are placebo-controlled studies. So although it's a rare disease, we did not follow the typical rare disease development approach where we were comparing against natural history data sets or something or another, or biomarkers. These are placebo-controlled studies, which we really love, and we were highly statistically significant on the primary endpoint and many other endpoints with regard to this. So this is a drug or disease for which there are no approved therapies. We need a therapy for this disease. It's robbing patients of their lives. As you alluded to, We filed for this program for approval with Europe, really with the understanding that it was the same understanding in the U.S., that we need to demonstrate two things to get approval. One, show substantial evidence of effectiveness from your Phase III study. Check that box. Two, demonstrate that the patients in our Phase III study are consistent or comparable with the patients that we see in the U.S. and Europe. We ran the study in Southeast Asia because you could not run placebo-controlled studies here for various reasons, mostly ethical reasons. And so we had the same agreement with both Europe honored that we filed. In the U.S., FDA has sought some additional data, really in the U.S. and Europe, to confirm that the data that we saw in our Phase III is consistent with what we would expect to see here in actual patients in the U.S. That's, in effect, a bridging-type study from our perspective. And so we are in that study now. It is called the FORGE study, where we're collecting PKPD data as well as efficacy data in patients treated in the U.S. and Europe, really encouraged by what we're seeing with that. And so we anticipate submitting the package that we submitted to Europe to the FDA, but with this additional data from this FORGE study in the U.S. and Europe at some point this year.
Got it. You mentioned the forward study. What's the risk here that you'd have to run another GBS trial, even if it is small, in Western patients to satisfy the FDA or at least prior to BLA submission? Do you feel confident that the data you submit from the open label forward study would be sufficient in FDA's eyes?
Yeah, well, we do. And really because of the consistency of the data. And the reason I say that is so PKPD, this is an antibody you would expect. It would look exactly the same across all territories in the world. That's what we've seen. This is also a drug where we've already treated U.S. and European patients in diseases like Huntington's disease and ALS. So we have a lot of PKPD data. And now we're collecting specific PKPD data in GBS patients here in the U.S. So I feel really good about that. The second thing is, you know, roughly 90% of our patients show benefit by week one in our study, in the phase three study. What that says is most everybody is going to show benefit in the study. And to the extent we see that here in the U.S. and in Europe, we're really encouraged by what we're seeing. The consistency of the data should answer the question. The question is not, do we need to establish substantial evidence of effectiveness here in the West in a program that has never been run in the U.S. and Europe? There's never been a placebo-controlled study, or in 40 years, there hasn't been a placebo-controlled study in the U.S. and Europe to demonstrate effectiveness. It's really a difficult study to do because of the rare disease nature. So we're just looking to confirm with this data that it is consistent with what we saw in our Phase III program and then filed straight away. And so we feel confident about it, but you can never count your chickens until you actually look at it, and FDA fully reviews the package.
That's right. Well said. Lastly, Doug, ANX1502, a lot of investor interest in the program, kind of flying under the radar. It's an oral complement drug. So what's the current status of this program, and when could we anticipate to see the next data update?
Yeah, we like the program quite a bit. Now, look, it's a first-in-kind program. It's earlier than the other two. We've talked about GBS and GA. It's at the translational stage. But we're in a proof-of-concept study. We are dosing as we speak now and getting this thing towards the end. So I'm encouraged by that, and I anticipate we will be able to have some – we will certainly have some data this year to update the marketplace on it. We think it has the potential to really be a market winner, whether it's in its current presentation or whether we do some additional formulation. We have seen some things over the course of your study with regard to a food effect. So when you take the drug with food, the drug is washing out in the gut. We can go into that. It's pretty technical. But when you take it without food, our target drug levels are exceeding what we hope to see in the study. And so the patients that we are now dosing are fully fasted on both doses, about two hours in and around the dose. And so we'll get that data, pull it all together, figure out our next steps and get it out into the marketplace.
What would be an ideal kind of target product profile for 1502? And what type of data would you need to see from the next update that would be enough to, say, give you kind of a go-no-go decision for advancing it forward?
Yeah, we definitely, well, so we certainly need to, from a target product profile perspective, need to, well, so the profile is that it's BID dosing, twice a day dosing. So I want to make sure we can do that comfortably and consistently. Secondly, it's incredibly important that we show we can normalize complement markers, which are elevated in these disease populations, meaningfully elevated. So being able to normalize those over a four-week period of time is really important. We know in the disease we're studying it in and a bunch of other diseases, by doing that, that translates to efficacy outcomes. We're also looking at biomarkers of efficacy, however. or hemolysis, and importantly, so bilirubin is the key measurement, it's super objective in the disease we're looking at, codaglutinin disease, and so we're looking to see normalization in that as well. So the complement measurements, the bilirubin normalization, and then of course safety tolerability will be important in making the no-go decision, yeah.
And what autoimmune indications do you think an oral therapy like 1502 could be well suited for
as a potential therapeutic? Yeah, I think, well, look, I think most all antibody-mediated diseases where the classical pathway is the key effector of the autoantibody attack, and there's a wide list for that. We will most likely start in those that have already been clinically validated. So you can think about things like myasthenia gravis as well as CIDP. In effect, cousins, if you will, of GBS, but in the chronic form. And so those are really nice entry points for us, and then
and then onward from there. Lastly, Doug, what's your current cash position? What's your expected runway here? And what clinical catalyst does this cash runway get you through?
Yeah. So we reported year end 2025, I think about $238 million or so in the bank. Importantly, that gets us well into the second half of 2027 through really important catalysts. So it includes You know, the GBS approval in Europe as well, hopefully the GBS approval in the U.S., ANX 1502, the proof of concept data as well, and then certainly the GA phase three data readout with some cushion beyond that, quite a ways beyond that to be able to figure out, you know, what we would do next with regard to that asset. So, yeah, we're encouraged where we are now, as we said at the start of this, Joey. It's a win year for us after 12 years, so we're super focused and excited by it, and we're going for it.
Great, great. Well, Doug, thank you so much for participating. It was an excellent discussion.
Thank you, Joey. Really good questions. And thank all of you for listening in.
Yes, and thanks, everyone, for joining us on the webcast. Have a good day and a good rest of the conference.