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. It's my pleasure to introduce our next company, Annexon Biosciences. 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. With that, we'll get started. Doug, thank you so much for joining us today.
Thanks, Joey. We appreciate you having us.
2026 certainly looks to be shaping up to be a pivotal year for Annexon. 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 Needham. We always appreciate this conference and opportunity to talk to folks. Super excited about 2026. 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. 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. 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.
From a milestone perspective over 2026, I'll just call out a few. First, starting with our most advanced program, Guillain-Barré Syndrome, or GBS, on the backs of a very successful phase III 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 comfort 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 III readout in geographic atrophy. This is our yet a third drug candidate, again, targeting the classical pathway, vonaprument.
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. On the backs of that data, we've run a large phase III program, which we anticipate reading out in Q4 of this year, a milestone-rich year for us after 10, 12 years on this journey.
Doug, in lead asset vonaprument, 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. 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. 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. Our approach is simply to block C1q right where it's localized on diseased 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. You never get on the cascade by blocking C1q.
That's led to very differentiated outcomes in our GA program. Not only that, we've also looked at C1q, as you know, in GBS, where C5 has been studied in phase II and phase III studies, very different outcomes. We've looked at C1q in ALS, very different outcomes where C3 and C5. 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 essential to provide function, in this case, vision preservation, which we have not seen with the downstream approaches.
Continuing along the theme of your geographic atrophy program, the most advanced and the latest stage, really a lot has changed in the GA space since when you first started your clinical program. 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 eons ago, geographic atrophy was always known as a neurodegenerative disease.
Yeah.
That storyline got lost over the last 10 years or so, with what 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 onto that is the approach to treat 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, companies move forward with that. 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.
Yeah.
Now that we're four or five years into datasets looking at protection against RPE growth, we now know that to be the case. We also know a second thing, which is, you're not going to get worldwide approval by pursuing RPE structure unless it shows functional benefit. 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.
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. You want to preserve photoreceptors in the eye, and that's exactly what we're doing with our approach. It's what led to the discovery of Annexon is the underlying technology discovered in Stanford by the late Dr. Ben Barres, 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 vonaprument and compare this to the approved GA drugs, SYFOVRE and Izervay? How do you think vonaprument is differentiated?
Yeah, it's a really unique drug candidate . First, it's a Fab fragment of our full-length monoclonal antibody, tanruprubart, which is what we're using for GBS, which has been really well-tolerated in GBS, Huntington's disease, ALS, and others. Vonaprument is a Fab fragment. It's a small 50 kDa Fab antibody. It's non-PEGylated 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, it's a 5 mg administered dose, 25 μl. That's about a quarter of the size of the approved drugs. They're about 100 μl. It allows for a really rapid administration of the drug as well, which we think is a competitive advantage. We like vonaprument 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. It's a well-designed drug candidate, and it does exactly what we want it to do in the eye.
The vonaprument, the 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. Can you just review the key data from this trial and put those results into context for the ongoing phase III trial?
Yeah, absolutely. 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 from a mechanistic perspective, makes a ton of sense. We are targeting the loss of photoreceptors, which you lose before you lose RPE cells. In effect, our approach is top-down. Imagine protecting your photoreceptors and then your RPE. It will take longer for us to protect RPEs, which get 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, the center of the retina that is responsible for visual acuity. We're really encouraged by that. We have not seen that dataset from any other company protecting photoreceptors in the 1.5 mm-2 mm in the central retina. 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. 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%, 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. We are 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. We were able to assess what happened with patients once we removed the drug. 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. We really like that kind of confirmation that that's a biological effect from your drug's intervention in protecting the photoreceptor cells.
Doug, I'm curious, what's the current level of understanding of vonaprument's differentiated mechanism here amongst GA treating physicians? 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 III and ultimately get approval on an RPE story, a large share of voice. However, as we have continued to educate others on 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 glomming on to our program and what we're doing.
The best evidence of that is our phase III 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 we're still re-enrolling candidly. We did that roughly two months ahead of schedule. 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. We know that patients are remaining on drug. I'm sure we'll talk about the phase III, but we're encouraged that physicians are now really leaning into this approach.
Yeah, the phase III 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 topline readout?
Yeah, it's very similar to the phase II study, which was called ARCHER. This is now ARCHER II. It is a study that has two arms. We've got an every month treatment arm versus sham, two-to-one randomization on that. The primary endpoint is at 15 months. This is the first and only program we're aware of in geographic atrophy where the primary endpoint is indeed preservation of vision. 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. 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 III, 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 II study is about 20% 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. We set a cutoff for patients at their baseline visual acuity so that we can assure ourselves that we'll get patients who will progress, but there's also room for trial 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. A gain, really with the aim of making sure that we're picking up patients that are showing this decline in vision, but are 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 at replicating what we did in the phase II study.
Yeah. Just a further ask on those two points, Doug, the phase II versus the phase III, can you remind us what the requirements were in phase II 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 III, but how does it compare to what you did in phase II?
Yeah. In the phase II, we really focused on lesion size versus BCVA cutoffs in the phase II because, again, the primary endpoint was RPE. It was really based on the learnings from our phase II study, coupled with a really large natural history dataset, the lampalizumab dataset from Roche, 2,000 patient dataset 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 at baseline. We cut it off at 45 letters. We did not do that in the phase II, and that is really the key difference in the phase III, ensuring ourselves that every patient that is in the study is one that we can show benefit in. We just think that's really important.
Got it. Can you disclose anything on the powering assumptions of the trial?
Yeah.
Of the phase III trial?
Yeah. We've not given specific numbers, but what we've done in that study is we've powered it really well for a phase III study. It's a really well-powered phase III 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 lampalizumab data set. 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 lampalizumab study. That's what we saw in our phase II study. We took that number, and we were conservative for powering in our phase III, so we took a haircut on that number, anticipating a bit of a regression to the mean.
Yeah.
We also protected vision with our every-month treatment arm, about 4.5% of patients. There we also were conservative. We're conservative on both bookends from a powering perspective and we really stand on two feet, really solid about really well-powered for a phase III study.
Got it. Makes sense. Obviously BCVA 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 II study that we ran there. 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 II and, importantly, what we see in the phase III from a clinical perspective. 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. We rarely do.
Got it.
We have perfect light. 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? 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. You think about retina specialists, they really do focus on structure as well as function. It gives you two things in your argument, if you will, for why this drug versus something else. They all are really well adept on that is your photoreceptors, your neurons in the eye responsible for visual acuity. 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.
Yeah.
This is, as I am aware, of the first kind of phase III readout with EZ as a likely, we haven't made the final determination, but likely be a pre-specified secondary endpoint. There will be learnings with regard to that. The way we've had the discussions internally with regulators, directionally supporting the functional outcomes will be useful for us.
What are the regulatory requirements needed for approval from both FDA and EMA?
Yeah. There it's quite simple. It's, meaning, our best corrected visual acuity, 15-letter loss. This is the gold standard endpoint for visual acuity studies. If you go back a couple decades to wet AMD and then various other ophthalmic diseases thereafter, best corrected visual acuity, 15-letter loss is the endpoint. The reason for that is it's a really high bar. It represents 50% 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. There's been 34 or so phase II, phase III studies that have not hit on this endpoint. We are the only ones to do that. It is a recognized endpoint that if you show statistical significance on best corrected visual acuity, 15-letter loss, you have an approvable drug.
Can you talk about the FDA requirements for two adequately well-controlled trials, that phrase is used, needed for approval? 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. We think at a minimum, ARCHER, the phase II 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 III studies. We initially were going to run two well-controlled phase III studies, one against SYFOVRE, one against sham. When we went in to meet with the regulators, SYFOVRE had had some safety events that were deemed potentially unmasking in a phase III study, so we were unable to compare against that. 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.
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 III level. We, in effect, have two studies under one protocol. What we love about it 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 really are encouraged by. Excuse me.
Really straightforward analysis, w e'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. It's exceedingly well-powered. I will just note, we are going back to the FDA to have a discussion on what Dr. Makary, the Commissioner, has more recently said that a single phase III 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.
Got it.
Yeah, that's the overarching answer for that.
The phase III trial that's ongoing, you're tracking progress in a blinded fashion. 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. The key to winning on this study is that you have enough BCVA 15-letter events over the course of the 15 months.
Yeah.
If you don't have enough events, you can't show a delta with the protection of your drug. We are tracking that in a blinded fashion, and we set that number again based on what we saw in our phase II and what we see in the lampalizumab data set and the event rates there. 2,000 patients can't really do better than that from a source of information to set your event rates. 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 drug.
Sure.
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 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. 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. We're really encouraged with the way this study's being executed.
Great. Well, I guess if the phase III data are similar to what the phase II showed and the drug is ultimately approved, where do you see vonaprument fitting into the GA treatment paradigm, and what could a potential label look like? 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. One of the things that we saw in our phase II 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% 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. We think certainly treating earlier in the disease is important.
Also later in the disease, as we showed in our data in the phase II, foveal patients and others that were a little further along in their disease, we had a pronounced effect in that population as well. We think this drug becomes the market winner, if you will, if I can say it that way, in that it is the only drug on the market that offers advantage with regard to vision protection of the structure in your eye that is responsible for vision. Again, the fact that it is not PEGylated, low viscosity, one of the things we saw from a safety perspective in our phase II study was virtually no difference in conversion to wet AMD in our program versus the sham arms. That is really important.
By targeting C1q and 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%-15% range. It's a really differentiated product, both on efficacy as well as on safety. The last thing I'll just say is the administration, as I said before, 25 μl, 50 kDa. 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 vonaprument. What's the status of this, and how important is it to have a PFS for commercial success?
Yeah, it's very important. Yeah. We think, obviously we want to partner with the retina specialists in the world who are treating lots of patients. These are big patient populations, both this and wet 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 will launch with what the presentation is in the phase III, though, which is standard. 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-Barré, 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. What's the latest update on how the talks with the agency around requirements are going needed for BLA filing, and what are some of the key sticking points?
Yeah, really good question. Well, first, for those who may be less familiar with GBS, really important disease. It's the 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. Really devastating disease. What's important about our data is a couple of few things. One, these are placebo-controlled studies. 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.
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. We had the same agreement with both. Europe honored that we filed.
In the U.S., the 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 FORWARD study, where we're collecting PK/PD data as well as efficacy data in patients treated in the U.S. and Europe. Really encouraged by what we're seeing with that. We anticipate submitting the package that we submitted to Europe to the FDA, but with this additional data from this FORWARD 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, it would be sufficient in FDA's eyes?
Yeah. Well, we do, and really because of the consistency of the data. The reason I say that is PK/PD, 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. We have a lot of PK/PD data, and now we're collecting specific PK/PD data in GBS patients here in the U.S. We feel really good about that. The second thing is roughly 90% of our patients show benefit by week one in the phase III study. What that says is most everybody is going to show benefit in the study. 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., in 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. Just it's really a difficult study to do because of the rare disease nature. We're just looking to confirm with this data that it is consistent with what we saw in our phase III program, and then file straight away. We feel confident about it, but you can never count your chickens until you actually submit 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. 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. We're in a proof- of- concept study. We are dosing as we speak now, and getting this thing towards the end. I'm encouraged by that, and I anticipate 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 the study with regard to a food effect. When you take the drug with food, the drug is washing out in the gut. We can go into that. It's pretty technical. When you take it without food, our target drug levels are exceeding what we hope to see in the study. The patients that we are now dosing are fully fasted on both doses, about two hours in and around the dose. 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 ANX1502? What type of data would you need to see from the next update that would be enough to, say, give you a kind of a go/no-go decision for advancing it forward?
Yeah. Well, from a target product profile perspective, the profile is that it's BID dosing, twice a day dosing. We 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. 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 translates to efficacy outcomes.
We're also looking at biomarkers of efficacy, however, hemolysis importantly. Bilirubin is the key measurement. It's super objective in the disease we're looking at, cold agglutinin disease, and we're looking to see normalization in that as well. The complement measurements, the bilirubin normalization, and then of course safety tolerability will be important in making the go/ no-go decision. Yeah.
What autoimmune indications do you think an oral therapy like ANX1502 could be well suited for as a potential therapeutic?
Yeah. 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. 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. Those are really nice entry points for us, 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. 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. It includes the GBS approval in Europe, as well, hopefully, the GBS approval in the U.S., ANX1502, the proof- of- concept data as well, and then certainly the GA phase III data readout, with some cushion beyond that, or quite a ways beyond that, to be able to figure out what we would do next with regard to that asset. Yeah, we're encouraged with where we are now. As we said at the start of this, Joey, it's a win year for us after 12 years. We're super focused and excited by it, and we're going for it.
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.