All right, let's go ahead and get started. Welcome, everyone, to the J.P. Morgan Healthcare Conference. My name is Anupam Rama. I'm one of the senior biotech analysts here at J.P. Morgan, joined by my squad, Priyanka Grover, Malcolm Kuno, and Rada Pinhasi. Our next presenting company is Annexon. I'm presenting on behalf of the company. We have CEO Doug Love. Doug?
Excellent. Excuse me. Thank you, Anupam and J.P. Morgan. We're delighted to be here today to give an update on Annexon Biosciences and our path towards a breakthrough 2025. I'll be making forward-looking statements for which you can review our materials on file. Founded 10 years ago, Annexon started with the premise that we are going to completely transform the complement landscape and drive immense value by translating our pioneering complement science into effective therapies for millions of patients suffering from complement-mediated diseases. Grounded in our scientific platform, it has now been clinically validated in an array of indications and has broad applicability for multiple therapeutic areas. Leading the charge is our most advanced program, Guillain-Barré Syndrome, which is poised to replace the standard of care with a recent phase III positive program, which is differentiated from the products that have been used for 40 years in this space.
This is a blockbuster space, which we're quite excited about, and I'll talk more too. Following Guillain-Barré Syndrome is our phase III program in geographic atrophy, which also has produced differentiated data from the marketplace, as it is the only significant vision-sparing product in development, having produced statistically significant data there, and is poised to disrupt a multi-billion-dollar blockbuster market, and last but not least is our first-in-kind oral classical complement inhibitor, which is designed to disrupt the space by providing robust efficacy coupled with convenience in the form of a pill in diseases that are currently treated by frequent IV infusions and subcutaneous injections. In total, we have multiple ways to win with this platform, each one of the programs having the potential to be best in class, and so we're excited for the outlook for 2025 and beyond.
Turning to our science, it is where we are founded and grounded. We are taking a pioneering scientific approach to stop complement-mediated-driven neuroinflammation, where it starts on disease tissue in autoimmune, neurodegenerative, and ophthalmic diseases. Based on discoveries of our scientific co-founder, the late Dr. Ben Barres, former chair of neurobiology, we are targeting C1q, the initiator of the classical pathway, to shut down both upstream and downstream complement so as to halt neuroinflammation in diseases of the body, the brain, and the eye. With that founding discovery, we have spent 20 years studying this approach and the role of neuroinflammation in a host of neurodegenerative diseases and have produced more than 30 novel assays around this pathway to best understand the role where complement is playing a key driving role in the disease process versus a supportive role. That, of course, increases our overall PTS.
We're taking those learnings into what we call a strategic drug development approach. It's a bit old school in that in each of our programs, we have started with a signal-finding study to further elucidate the role of complement in a diverse array of diseases with strong execution across those. The net result is we've been able to produce really what is game-changing data across our flagship programs, which we will talk more about. Now, on the basis of this data, we've been able to develop the leading complement-focused pipeline with multiple ways to win. Led by our flagship programs, each has a diverse drug candidate that's leading the charge there. And as I've said before, we've produced very unique data. And as a result, we have the potential to treat millions of patients suffering from complement-mediated diseases.
Our flagship programs have also informed and de-risked our next wave of programs, which gives us the opportunity to continue to generate value for many years to come. Turning to 2025, really centered upon three key catalysts to drive really breakthrough value. The first is in ANX005 in Guillain-Barré Syndrome. There, we're poised to submit our first BLA submission in the first half of this year while we continue to advance our discussions with EMA towards MAA over the course of the year. Secondly, with ANX007, our phase III pivotal program, a large program that is global in nature, we anticipate completing that enrollment of that study in the second half of the year and reading out the data in the second half of 2026.
And then finally, with regard to our oral ANX1502 program, first-in-kind oral program in the classical complement space, we will produce clinical proof-of-concept data in the first quarter of this year in cold agglutinin disease. And from there, we will also update on future indications in which we look to take this program forward. We're really excited by a host of indications to move forward into that have been clinically validated, and so more to come on that. Finally, as it relates to the overall health of the company, we have cash runway into the second half of 2026 to achieve all of the milestones you see here on this slide. And so we're really well positioned to really break through this year and transform the company and taking it to the next stage of its life. Now to double-click into Guillain-Barré Syndrome.
This is a first-in-kind targeted therapy for GBS. There have been no advancements in this space in over 40 years. This represents our third study here. And we're excited by the differentiated data that's been produced out of this program and really has set it up to replace the standard of care. A few headlines from our recent phase III program where GBS ANX005 and GBS showed neurological success in this late-stage study, a really promising agent improving just disability in this disease across all measures related to GBS. And I'll show you some of that data. First, however, just kind of to paint the picture, this is a devastating disease, really an acute neuromuscular disease that causes paralysis overnight and really robs patients of their ability to live their best lives. And notwithstanding standard of care, IVIg, folks are really still quite suffering from this disease.
In fact, almost 50% of patients treated with IVIg are admitted to ICU immediately upon coming into the emergency room. One in four require mechanical ventilation. One in five can't walk after a one-year period of time. You can just only imagine that one day you were healthy, you got an infection, and the next day you can no longer move and function independently. Really quite devastating. And it's not a cheap disease to treat either. It costs the healthcare system more than $2 billion a year to treat this disease, really associated with patients in ICU, on ventilation, skilled nursing facility, and the like. And unfortunately, there are no FDA-approved therapies for this disease.
As Hugh Willison noted, this is a neurological emergency, and we really do need therapies who are available to provide immediate and aggressive stopping of the disease process and a fast recovery to get patients back to their normal lives. And we're encouraged by that's what we've seen in our phase III program. Here's just a snapshot of some of the data. Importantly, what we saw in our phase III program, which is, again, a large 240-patient placebo-controlled study, we saw very rapid benefit in this study where we improved muscle strength by week one in the patients treated with ANX005. It's a really important time period. That's a time when you're making decisions on whether to move patients into ICU or provide additional alternative therapies, which really are not quite as effective. The impact on muscle strength at week one translated into very durable results.
You can see by week 26, excuse me, or six months, two and a half times more patients treated with ANX005 were able to recover to full strength at that time point, so a really whopping effect early was translated to full recovery six months thereafter. Excuse me. We also did a real-world evidence study, which is really quite important for us to evidence the impact of ANX005 against standard of care or IVIg. There, very similar to what we saw in our placebo-controlled study, we saw a more complete recovery and fast recovery, so very much like what I showed on the prior slide, ANX005 versus IVIg showed a 10-point improvement in muscle strength by week one, and you can see that IVIg, those patients are still declining at that time point.
Then looking out to six months, you can see with treatment with ANX005 on the right, we saw almost two times as many patients return to full recovery versus the standard of care at six months. A marked improvement on what the therapies are currently available to treat this devastating disease. We also went further to look at the impact of ANX005 on key measurements of disease burden. There, too, you see a robust effect with ANX005 versus standard of care, particularly IVIg. We had half as many patients treated with ANX005 actually go on to ventilation versus IVIg. For those patients who did move on to ventilation or ICU, we got them off the ventilator and got them out of ICU almost two weeks sooner than IVIg or plasma exchange.
You can imagine every day that you are in the ICU, every day that you're on a ventilation is a game-changing event for the patients, the hospitals, their families, et cetera. We're really pleased by the outcomes here. All of that positions ANX005 to really own what is a blockbuster market. GBS occurs in 150,000 patients worldwide. Importantly, 22,000 patients annually in Europe and the U.S. As I said before, the current standard of care is suboptimal to treat this population. There's been no placebo-controlled studies ever conducted with IVIg, so we don't really know its true treatment effect. Notwithstanding that, 90% of patients are treated with IVIg off-label due to the severity of the disease. You really have to do something for this patient population that was otherwise normal and now can no longer function.
Although contraindicated on the treatment guidelines, unfortunately, half of the patients treated with IVIg are getting a second course of treatment. It's now been clinically established that IVIg and a second course of treatment results in worse clinical outcomes and, more importantly, higher safety events. Physicians are still putting patients in a second course of treatment because they just have no other options. And it really speaks to the need and the debilitating aspect of this disease. Last thing I'll note is that IVIg carries a black box warning, and so that's not trivial. At a time that you're fighting a disease like GBS or taking therapies, that could really have an impact on your overall health as well. By contrast, as I showed in the prior slides, ANX005 has really differentiated outcomes. We got patients better and faster and more completely on function. It was generally well tolerated.
We avoided ICU and ventilation and reduced ventilation time. All of that leads to what we think is a robust commercial opportunity. One of the things to note about GBS is everyone gets treated. As I said before, 90% of patients take some form of therapy because you just have to, given the debilitating natures. But it's a very concentrated circumstance. This is an incidence-based disease. And what we've determined with extensive research is that 440 hospitals account for 80% of the GBS population on an annualized basis in the U.S. And 3,000 physicians are responsible for writing scripts and treating these patients. What that means is it's a very tractable commercial opportunity where we can have a very focused commercial footprint to target these physicians and these patients and help them get better. We're well underway in building out our commercial operations.
We have a very strong Medicare group that's out in the field now calling on these hospitals and educating the physicians, managed care folks, and the like. So we're really encouraged by the outlook to, day one, really get after this disease and drive immense value for patients and our shareholders. On the go forward, we've checked the two most important boxes here to fore. One, obviously winning on a large placebo phase III study and also demonstrating that in real-world evidence that ANX005 is superior to IVIg and plasma exchange in that analysis. In terms of next steps, we're actively engaged in discussions with the U.S. and E.U. regulatory bodies. We plan to submit our BLA submission in the first half of this year, and we are, of course, ramping our education and commercial opportunities in this disease.
This offers the opportunity to be Annexon's first commercially available drug. We're really encouraged and excited to be bringing this forward as it is a game changer in this space. Second, ANX007, geographic atrophy, the first program in geographic atrophy to provide significant visual preservation in this really devastating patient or disease. Excuse me. Some headlines from our phase II proof-of-concept study where our neuroprotective approach by blocking C1q showed significant preservation against vision loss with the opportunity to preserve sight in an elderly population. The average age of the patients in our study coming into the study were 80 years old and above. So it's the population that you really want to get after.
You also should note headlines here, where this is the only program, first and only program, that has received PRIME designation from Europe. In effect, breakthrough, really evidencing the import of our functional data to preserve vision in this population. Very much like GBS, the unmet need here is quite high. GA is a severe disease that limits an elderly patient population's independence. Looking at vision loss here, three lines on the standard endpoint BCVA 15 letter means you've lost 50% of your vision. You have to turn in your driver's license. You can no longer operate independently. Many of these folks are moved into skilled nursing facilities where they have to have family members come move in with them. So it's a really devastating disease. It's the leading cause of blindness. It impacts 8 million patients worldwide. There are no FDA-approved treatments impacting vision in this population.
Unfortunately and indeed, there are no treatments at all approved in Europe or in Asia. In the words of Dr. Eleonora Lad, Vice Chair of Clinical Research at Duke Eye Center, she says, "Unfortunately, currently treatments have not translated to protection of clinically meaningful vision for patients," and our mission is to do exactly that, and so we've embarked on a really robust program, starting with a proof-of-concept study that we conducted last year or we read out on last year. This is a 270-patient study that was run here in the U.S. And what we like about it is the comprehensive nature of the data. Shown in the top panel on the left, we've shown that we were able to preserve significantly vision in normal life conditions.
You can see there that 6% of the patients in the every-month group shown in red lost vision versus 21% of the patients in the sham arm. Moving to the right in a setting of low-light conditions, we also significantly protected vision there. 7.6% of the patients in the every-month group shown in red lost vision versus 20% in the sham group, and importantly, in those patients who have earlier onset geographic Atrophy, none of our patients in the every-month group lost vision. That's 0 out of 56 versus 17% in the sham arm. That's a really significant finding. It means treating earlier with ANX007 is going to result in bigger and better outcomes and probably expands the pool, and that's what you would expect to see in all neurodegenerative diseases. If you have an effective therapy, it should work better earlier.
And that's what we see in our data. And then last but not least, it's really important, excuse me, to call out our functional benefits. Targeting photoreceptors in the eye, these are neurons in the eye which are responsible for visual acuity and the only structure in the eye that directly correlates with visual preservation. There you can see in the central fovea of the eye where you have vision loss, we preserved almost 60% loss of photoreceptors versus sham in that group. So what we've done here is shown that we have protected vision and the structures that are associated with vision in our data set, which is a really comprehensive package that has been well received by KOLs, patients, and importantly, the regulators who we've talked to around the globe. In terms of next steps, we've checked the box on a really robust proof-of-concept study.
We've got an EMA PRIME designation and a robust alignment with the FDA. And our next steps are to complete the phase III study, which is really designed to replicate what we did in our proof-of-concept study. So we have not deviated much from that and really just going deep on that. This is a large study, 630 patients globally, enrolled predominantly in the U.S. and Europe. And we're really excited by the outlook on this with it reading out in the second half of 2026. Finally, turning to the first and only oral classical complement inhibitor in this phase, we're really excited with this potential to completely disrupt the biologics-treated marketplace for antibody-mediated autoimmune conditions. As you all well know, these conditions, many of them neuromuscular, like myasthenia gravis, CIDP, MMN, are treated by frequent IV infusions, whether that's weekly, every other week, and occasionally once a month.
That's how this market is being managed. These are severe diseases of anemia. Patients have inability to get out of bed at times and to manage their daily living, so our aim is to give them an oral tablet where they can get better from home, regain their freedom, and have flexibility in the way they go about treating their disease, and this is a large opportunity. What I like about it is the indications that are chiefly in our mind are those indications that have already been clinically validated from a clinical perspective, so the PTS is relatively high, and we're bringing forward a drug that really meets the patient's needs in these disease spaces. Our drug, ANX1502, is targeting the activated form of C1s that will be familiar to many of you who have studied the antibody-mediated autoimmune space.
In our healthy volunteer studies, it's been generally well tolerated in a tablet formulation, twice-a-day dosing, which is really quite convenient. It's shown robust inhibition of the classical pathway comparable to autoantibodies or antibodies that have been used in this space previously, like ANX005 or sutimlimab, which has been approved for use here. You see a bit of that data here on the right where our drug levels achieve greater than 100 nanomolar, which is the target trough level that you would want to see in diseases for which we are pursuing. In terms of next steps, we've completed our healthy volunteer work there. We've had initial in vivo pharmacodynamic signals in those studies as well, which is really quite important. We're now looking to really confirm the dosing profile for our drug in a patient population.
We're using cold agglutinin disease to do so, a very objective autoantibody disease, and there, the endpoints are objective impact on reduction of hemolysis as measured by bilirubin, as well as showing the ability to shut down the entire classical complement pathway. We anticipate data in the first quarter of this year, and with that data, we will announce the next set of indications we will go into. We think this is a game changer, candidly, and has an opportunity to really disrupt the marketplace, so we're excited by that. In closing, I would just quickly say we've got multiple ways to win in 2025 and beyond. It's been a 10-year journey to disrupt the complement space. We're excited with what we've done in terms of validating this clinical platform.
We've got three flagship programs, each designed to be best in class in their respective space, multiple winning data sets across those diseases, and they each have a differentiated profile and are positioned to completely transform the space in which they're playing. Really, really important. I love our team. They've been able to execute exceedingly well. I mean, think about the diversity of programs that we have. GBS and acute neuromuscular disease that's been run in Southeast Asia. GA of chronic ophthalmic disease that's been run in Europe and in the US. And so we love that we've been on time on budget, and we will continue to fight to do so. Love the team's fight from a cultural perspective. Warrior spirit is one of our key mantras, as well as all for one. You guys have all been in drug development for a while.
You know, it's all about how do you solve it. We've been able to do that. And then our catalysts in 2025 and 2026 really are game changing. So we think we're well positioned for breakthrough. There's more to do, and we're excited to do that. So thank you for your time, and happy to answer any questions you have.
Thanks, Doug. I just want to remind folks that there's three ways to ask a question. The first is you can raise your hand, and I'll call on you. The second is you can put a question in the portal, and I'll ask it on your behalf, or you can email me. But I will kick off the questions here, maybe on ANX005 and GBS.
So now that you have the real-world evidence in hand, what are the final sort of gating steps of the NDA submission in the first half?
Yeah, great question. So we're next set to meet with the FDA for our pre-BLA meeting. Importantly, to get a label in this disease, given that it was run outside of the U.S. And just maybe a little backdrop on that. We ran the program outside of the U.S. because it was deemed unethical to withhold standard of care in the U.S., IVIg, given the debilitating nature of the disease. So from the FDA, we need two things to get a label. One, win on your phase III program. We check that box. And two, demonstrate that the patients in our study are comparable to patients in the Western world.
In other words, is the data that we saw in our phase 3 study generalizable to the US? It's why we conducted the large real-world evidence data that we released in December. And we're really pleased that we were able to match the patients one-for-one in our study with patients in a large registry of Western world patients. So we feel like we've checked that box as well. But we will go down and have that discussion with the regulator shortly and then look to follow BLA shortly thereafter.
And are there plans to publish or present some of the real-world data to the, say, broader physician community?
Yeah, absolutely. So really pleased that IGOS, the registry that has done the analysis in the real-world evidence fashion, will have submitted, and that will be published at the upcoming AAN conference.
And we'll be at every conference thereafter with both the real-world evidence data as well as the data from our phase III study. We think it's really important to continue to publish our data.
Questions from the audience? Yeah.
For the, you were mentioning continuing education for physicians on GBS. You were mentioning continuing education for GBS within the physician community. Would you be focusing predominantly in the 440 hospitals that you had mentioned within the p resentation?
It's a good question. So it is a prioritized approach to education. But the first thing I will say is we want to treat all GBS patients. I mean, this is such a debilitating disease. We can't emphasize that enough. Normal one day, the next day you can't get out of bed. The 440 hospitals I alluded to do represent 80% of the patients in the US.
And so we certainly have started to go in very deeply there with our Medicare group and others to ensure that they're educated both in person as well as digital media, et cetera. We will continue to do that. But we are pushing out further than that. It's just important to try and treat as many patients as possible with this, what we think is a very unique approach to this disease. So thank you.
What are the key pre-commercial activities that you're going to be doing kind of in 2025 to prep the market?
Yeah, really good question there. So it's multifaceted, as you alluded to. So we, of course, are engaged in heavy medical education. I mean, the thing to note about GBS is there's been literally no advancements in 40 years. And so patients are typically treated in the emergency room.
The physicians have had no reps or MSLs calling on them, so we're making sure that people really understand the disease, how to diagnose the disease, the course of the disease, and how important it is to treat quickly. I mean, what you hear in this space is time is nerves. The more quickly you can treat, the better. We started off with a really robust medical education campaign, both in person, digitally, and print. From there, we are very active on the managed care front, meeting with hospitals and payers to ensure that we can get this therapy on formulary. One of the things that we did in this study, which is really intentional, is we looked at measurements of disease burden. What are the impacts to patients and physicians that they care about from this disease as well as from payers?
And so when you look at things like days on ventilation, days in the hospital, days in the ICU, these are the things they really care about. These are the discussions we're having with the payers and the hospitals now, which will allow us to get reimbursement for this drug, we think, rapidly and at rates that are really beneficial to us and the patient population. So really active on the managed care front. And then really a robust commercial plan, excuse me, to launch this drug initially in the 440. Excuse me, I got a little frog today. In the 440 hospitals. Distribution is the last thing I'll just talk about on that. It's really important. Obviously, this is a neurological emergency. You have to have drug there. You don't necessarily know which hospitals it will be at immediately.
We do know in the ED, they're going to see repeat cases of GBS. And so there we'll be providing drug buy and bill. It'll be on site there. We also can provide our drug to these folks on consignment so they can have it there and they'll pay for it when they use it. And last but not least, it's just kind of a standard offering from some of the specialty distributors. We can do it drop shipment in a matter of hours. What's important about ANX005, it's really very stable. The shelf life is four years with just standard refrigeration. So the drug can pretty much always be on site or we can get it to you in a matter of hours. And again, our aim is to just treat patients.
We're starting with that 440, but we want you wherever you are in the U.S. and Europe, Malcolm.
So it's pretty clear GBS data in terms of days in the hospital. Do you have any data cuts or even anecdotal evidence about when it comes to readmission?
Readmission?
Yeah.
Yeah, very low readmission rate. Once we get you through, so it's a disease of muscular damage and ultimately full-body functional damage. You gain muscle first, and then you start being able to move functionally. So you're able to use a remote or whatever with your hands. Then ultimately, you're able to get out of bed. Once you do that, assuming you're getting the right kind of physical therapy, et cetera, you're just on your road to recovery. Hopefully, you're getting to full recovery. We've been really pleased with what we've seen.
I will say this is the first study to look out to six months. Typically, what you see if you go back and look at IVIg plasma exchange studies, you're seeing four-week data points. Occasionally, you'll see an eight-week data point. We carry this all the way out to six months so that we can demonstrate the durability of our treatment effect. And that's what we like about some of this data here on the right, where you're seeing patients get to full recovery on the GBS disability scale. That means they're able to walk, run without assistance. So they're able to be back to their full selves. And you can imagine if you're an employee and you want to get back to work, that's what you're looking to get to.
Yeah, thank you.
Additional questions from the audience? Go ahead.
I have the mic.
As a follow-up to that, in your six-month duration, have you picked up any side effects because you're completely blocking C1q?
Yeah, we've been really, really pleased with the safety profile for this drug. So this is a single infusion. So you're stopping this autoantibody attack, which typically lasts anywhere from a week to two weeks right at the start. So you're shutting it down completely. Your drug does its thing. And then you allow complement to come back. So once you've had that initial infusion, if you really haven't seen a safety event related to the drug at that point, it's unlikely you're going to see it thereafter. And so the safety profile has been really quite nice. We've seen a bit of tolerability upon the initial infusion in the form of a skin rash that's transient and goes away.
But outside of that, we've been really encouraged by the safety profile of this drug.
Thank you. Another question?
Yes.
Yeah, go ahead. Still have the mic.
For 1502, does the molecule get into the CNS?
It does not. This initial formulation is targeting just C1q in the periphery. It's a good question.
So on 1502, so how are you going to define a win scenario when you flip the card in terms of what key biomarkers you're going to be looking at and what should we be expecting?
Yeah, I mean, so we went into cold agglutinin disease because it's a very objective disease, very objective measurements on your drug's pharmacodynamic and efficacy effects. And so the key measurements in a hemolysis disease or in a disease like cold agglutinin disease is hemolysis.
So we're looking to show that we can bring down bilirubin in a matter of a week or two, like in a very rapid period of time. And so that's a real important measure for us from a go, no-go decision. Secondly, as I said before, we really do want to evidence the ability to shut down the entire classical complement cascade. If you don't, you'll begin to have complement come back and have bad effects for patients there. And so both of those are the two key measurements for that. And assuming success there, we'll make a go decision, but we'll have to wait to see the data.
And in your presentation, Doug, you talked about moving forward in kind of clinically validated indications, but not all clinically de-risked sort of indications are the same competitively.
This is very true.
How do we think about where you would go from a competition standpoint?
The first thing I will say is irrespective of the indication, coming in with the oral is going to be best in class. I mean, assuming we've got comparable efficacy and a safety profile. Just again, thinking about where complement has been effective in the clinic thus far, it has predominantly been in these antibody-mediated neuromuscular diseases where folks have a hard time just managing their day-to-day living. We talk to patients. They talk about if they go to a grocery store, they really have to schedule that because the next two or three days thereafter, they really don't have the ability to function at normal levels, and so giving them an ability to take pills at home at their own convenience and the freedom of their home really is a game changer.
Beyond that, in terms of the players and the differing indications, we have assessed that very closely. We will reveal the indications. We will target first and second thereafter. Part of this is just kind of the number of drugs that are in clinical development in some of these indications, although we think a pill will be really rapid to recruit, so we're assessing all of that, but we think all of them are in play, candidly, and not only the clinically validated indications, there are a host of other antibody-mediated diseases, which are really difficult to pursue from a clinical perspective with IV infusions that you have to do weekly or every other week. Patients just don't sign up for those studies. Giving them a pill gives us an opportunity to move into indications that have not been fully tapped here too far, and we're really excited about that as well.
Questions from the audience?
Maybe switching gears a little bit to ANX007. How is enrollment going in the phase III Archer II study relative to kind of your expectations?
Yeah, I mean, the short answer is, without overselling it, it's exceeding our expectations. We're thrilled by this. I mean, the thing to note about this program, again, is that it's an elderly patient population that is losing their vision and their independence associated with it. And if you look at what's occurred over the last seven to 10 years, these folks have participated in studies that are really designed to show some impact on structure, but that has not translated to vision.
The fact that we have now demonstrated in a robust 270-patient proof of concept study, the ability to protect your vision both in normal conditions and low-light conditions, and if you're earlier in your disease, it's even better. It's a very comfortable conversation for the retina specialist to have with patients about participating in our study and the potential to have preservation of their vision at a vulnerable time in their lives. And so enrollment has been really, really robust in the U.S. We started first with those physicians who participated in our proof of concept study. So they had experience with the drug. We then migrated out to the broader kind of community, if you will, in the U.S., and now punching over into Europe where they have no other options, right? So there are no complement therapies over there that have been approved.
This is the only kind of game in town for them, and it's just been really quite gratifying the way they dug into the data and understand the science and how they decided to participate into our program. So we're really encouraged by this.
And then on 007, as a clarification point, just given the competitive landscape, are you still considering the Archer study, or are you just going to focus on Archer?
It's a good question. So the Archer II study, as I said, is a 630-patient global study. That is a sham-controlled study. We have assessed looking at running a head-to-head study against Syfovre over the Archer study. We're still doing that assessment. We've had good discussions with the FDA on that.
The reason why you would do a head-to-head against Syfovre in the ARCHER study is, candidly, because at the time we were thinking about it, that was the leading drug in the space. That's come back a bit, and so you've got multiple competitors that are not the king of the mountain, and so it may not be, from a commercial perspective, necessary to go after Syfovre in a head-to-head study. We know they don't have vision preservation, so you would just be confirming what you already know, and so that may be something that's better handled from a marketing perspective. As I said, we have been having discussions with the regulators with regard to doing that or not doing that. I anticipate we will update the broader market on our development plan with regard to that study in Q1 more fully.
Any final questions from the audience?
In ANX005, are you sure that you have the right dose? I recall that you had a higher dose that wasn't optimal.
Yeah, it's a good question. We took two doses into the phase III study really because of what we saw in the phase II study. So the thing to know about GBS is it is an acute monophasic disease where you have an autoantibody attack over about a two-week period of time. You want to stop that attack as quickly as possible by shutting down complement. After that attack, you actually want complement to come back because it's part of the repair process. That's complement's normal role. It's part of the healing process.
And what we saw in our phase II proof of concept study was that patients who were inhibited complement for about a week period of time did better for patients who were inhibited where we inhibited complement for more than a week period of time, two to three weeks period of time. That was a really small study. That was 18 patients. So out of an abundance of caution, we took both doses into our phase III, both a one-week inhibitory dose as well as a three-week inhibitory dose just to be empowered each separately to see if we can win on both. And what we saw in that study is very much like what we saw in the proof of concept study. The one week of inhibition did much better.
I mean, the three-week inhibition did better than placebo on every time point, but the one week did better statistically on every measure. So we're really encouraged by that. And so we feel like we've really defined the window for which you would target Guillain-Barré syndrome. This has also been demonstrated with IVIg, as I said before. IVIg, one course of therapy, that's probably their best data. We know from large studies that have been conducted with IVIg, with two courses of treatment, those patients do much worse and have worse safety outcomes. So we now see it in two different therapies. You want to hit GBS during the active disease phase and then allow complement to come back and be part of the cleanup. Thank you for that question. It's a good question.
Any final questions?