Annexon, Inc. (ANNX) Earnings Call Transcript & Summary

Greetings, and welcome to the Annexon Biosciences GBS program update. [Operator Instructions] As a reminder, this conference is being recorded. I would now like to turn the conference over to your host, Ms. Jennifer Lew, Chief Financial Officer for Annexon Biosciences. Thank you. You may begin.

Thank you. Good morning, and welcome to Annexon's GBS program update conference call and webcast. Earlier this morning, Annexon issued a press release reporting positive top line data from real-world evidence supporting our registrational program for ANX005 in Guillain-Barré Syndrome or GBS. A copy of this press release is available on the company's website and through our SEC filings. Joining me on the call today are Doug Love, President and Chief Executive Officer; Dr. Jamie Dananberg, Chief Medical Officer; and Dr. Ted Yednock, Chief Innovation Officer. Before we open the call up for questions, we will be joined by Dr. Hugh Willison, Professor Emeritus of Neurology at the University of Glasgow in Scotland, a member of the International GBS Outcome Steering Committee and Dr. Ken Gorson, Professor of Neurology at Tufts University School of Medicine and Chair of the Data Safety Monitoring Board for Annexon's Phase III trials. Please note that on today's call, we will be making forward-looking statements, including statements relating to the existing clinical data and the therapeutic and commercial potential of our investigational product candidate, ANX005. These forward-looking statements are based on current expectations and assumptions, which are subject to risks and uncertainties. These statements reflect our views as of today should not be relied upon as representative of our views of any subsequent date, and we undertake no obligation to revise or publicly release the results of any revision to these forward-looking statements in light of new information or future events. These statements are subject to a variety of risks and uncertainties that could cause actual results to differ materially from expectations. For a further discussion on the material risks and other important factors that could affect our financial results, please refer to our filings with the SEC, including our most recent annual report on Form 10-K and quarterly report on Form 10-Q. With that, I will now turn the call over to Doug. Please go ahead.

Thank you, Jen, and thank you all for joining us on the call this morning. We're very excited to be reporting today positive top line results from a real-world evidence study supporting the treatment of ANX005 and GBS. Building upon our prior clinical results, this study strengthens the body of evidence for ANX005 and GBS that supports our overall registrational program with a planned U.S. BLA submission in the first half of 2025. After decades of nonspecific immunotherapy, ANX005 has the potential to be the first targeted therapy for GBS, and we are committed to helping people with this said and devastating neurological disease get better sooner. At Annexon, we are pioneering a novel and intentional approach to tacking an array of diseases mediated by the immune system classical complement pathway. Our company was founded two years ago on the bold thesis that stopping classical complement inflammation where it starts on disease tissue could result in a unique functional benefit. Since then, ANX005 and other of our drug candidates have generated compelling functional data in serious diseases like GBS in geographic atrophy and have reinforced the power and the potential of our platform. As a result, we remain sharply focused on developing first-in-class therapies with game-changing potential for patients living with severe neuroinflammatory diseases of the body, the brain and the eye. Importantly, ANX005 is the most advanced targeted immunotherapy and development for GBS. It is designed to rapidly block C1q in the classical component activity with a single dose to halt progressive nerve damage during the critical acute phase of the disease. GBS is a severe and underrecognized disease with high unmet need. Currently, there are no FDA-approved therapies and no targeted treatments approved worldwide, often striking without warning, GBS is a rapid acute and indiscriminate neurological emergency. It has a narrow window for therapeutic invention and results in the hospitalization of over 22,000 people annually in the United States and Europe. Globally, it is the most common cause of acute neuromuscular paralysis and arrives too many of their independents and ability to live their best lives. GBS typically follows an infection in otherwise healthy individuals that leads to the generation of autoantibodies to activate complement to directly cause nerve damage and acute paralysis. Please note that GBS can happen to anyone at any time. As such, the burden of this disease urgently calls for new, more targeted options that are designed specifically to meet the needs of GBS patients and their providers. Our goal at Annexon is to position ANX005 for the treatment of GBS worldwide to address this unmet need. Today represents another step in the journey for the GBS community and for Annexon. We're excited by this highly supportive real-world data generated in collaboration with the international GBS outcome study for IGOS. Not only does it strengthen our overall clinical package, but it shows for the first time in comparative analysis, ANX005 benefit over current standard of care, IVIg or plasma exchange. We'll discuss this in more detail shortly. Turning to Slide 4. First, let me briefly review our Phase III pivotal trial. Recall that in our successful placebo-controlled 240-patient Phase III trial, a single dose of ANX005 30 mg per kg led to rapid, robust and consistent benefit across multiple endpoints important to patients and the health care community. In the trial, ANX005 30 mg per kg met the primary endpoint, demonstrating a statistically significant 2.4-fold higher likelihood of being in a better state of health on the GBS disability scale at week 8 versus placebo. Multiple sensitivity analysis of the primary endpoint showed consistent improvements with an even more robust effect observed in a subgroup with Western population baseline therapeutics. Both early and durable treating effects were also evident in the Phase III trial with improvements over placebo across multiple measures, including muscle strength, less time on ventilation and less overall disability. And importantly, approximately 2.5 more ANX005-treated patients returned to normal at month 6 compared to placebo. On the safety front, ANX005 is generally well tolerated with mild to moderate and season-related events the overall safety profile that was comparable to placebo. There were no new safety signals, nor increased rate of infection while not requiring vaccination of prophylactic antibiotics and no difference in all-cause mortality. The GBS Phase III outcomes represented an important scientific breakthrough, reinforcing our founding thesis that C1q and classical pathway inhibition is a powerful mechanism of action to stop the start on neural inflammation. Since our Phase III trial is conducted outside the U.S., we've collaborated with IGOS under a formal protocol with prespecified endpoints to identify a cohort of patients from the IGOS global registry that match patients treated with ANX005 30 mg per kg from our Phase III trial. IGOS is a global prospective observational multi-center cohort study that has enrolled 2,000 patients who were followed for 1 to 3 years. I will now turn the call over to Jamie, who will discuss the top line real-world evidence findings of ANX005 for the treatment of GBS. Jamie, over to you, sir.

Thanks, Doug. Good morning, everybody. We're excited about the progress across our GBS program and the therapeutic potential of ANX005. I'll start with a brief overview of our real-world study methodology and its findings, and then I'll dive deeper into the data. As Doug mentioned, our randomized double-blind multicenter Phase III study was conducted in Bangladesh and the Philippines due to the high prevalence of GBS of all types, the country's internationally recognized scientific and clinical leadership in GBS and the limited availability of IVIg or plasma exchange, thereby enabling the conduct of a placebo-controlled trial. This is the first placebo-controlled trial in decades with all patients receiving best supportive care comparable to that delivered in the West, except for the use of IVIg or plasma exchange. It is important to understand that GBS is an acute neurologic emergency that can lead to long-term sequelae requiring an immediately effective and targeted intervention. Currently, there are no treatments that specifically and rapidly target complement-mediated nerve damage. The current standard of care for GBS is plasma exchange and more often, IVIg, but the latter is not approved by the FDA for use in this disease. While GBS is an acute emergency that requires rapid intervention, IVIg involves a 5-day infusion regimen and has an ill-defined mechanism of action. Even with IVIg or plasma exchange treatment, GBS often results in severe weakness in paralysis that can require long stays in the ICU and in many patient's mechanical ventilation. Ultimately, the early nerve damage caused in GBS can result in slowly reversible or permanent loss of function, leading to incomplete recovery that prevents patients from returning to a normal life. There were two main objectives of the real-world study conducted in collaboration with the IGOS team. The first was to demonstrate patients treated with ANX005 30-milligrams per kilogram from the Phase III study were represented within the IGOS global registry population using propensity score matching methodology. And second, to compare outcomes against current global standards of care, IVIg or plasma exchange. Comparing these propensity match cohorts, ANX005 showed faster and more complete recovery than IVIg or plasma exchange on multiple measures, including early gains in muscle strength that we won and the greater improvement in GBS disability scale or GBS-DS, I'll refer to it from here. In addition, fewer ANX005-treated patients required mechanical ventilation and patients were observed to spend fewer days on ventilation for those who required it and less time in the intensive care unit. A key takeaway from this RWE analysis is that the outcomes were consistent with the observations of -- benefits of ANX005 from the Phase III trial. On Slide 6, let's begin with how we establish the matching of patients from our Phase III trial with the IGOS population using a widely accepted matching methodology, a 1:1 propensity score matched IGOS cohort into ANX005 30 milligram per kilogram population was established based on prespecified key prognostic factors of baseline muscle strength and GBS disability score. Baseline MRC sum score, the measure of muscle strength and GBS-DS are the most prognostic measures for disability outcomes based on long-standing literature and on the analysis of IGOS patients themselves. The propensity score matching process was conducted blinded to outcomes. Nearly half of non-Bangladeshi patients in the IGOS database would have qualified for the ANX005 Phase III study. Using the prespecified factors, a one-to-one match was established between patients in the IGOS registry and patients treated with ANX005 30 milligrams per kilogram from the Phase III study, meaning 79 IGOS patients matched with 79 treated with ANX005. I want to acknowledge you the value of the IGOS database and the importance of our collaboration with the Erasmus Medical Center team that oversees the registry to use patient-level data for matching with patients from our Phase III study. We greatly appreciate the work of IGOS to create the most robust database of GBS patients worldwide. Most patients in the ANX005 Phase III population had moderate to severe disease and the matching and balance of disease characteristics between the cohorts demonstrates that the Phase III population is represented within the global GBS population spectrum captured within the IGOS registry. Recognizing the common role of complement biology and GBS pathogenesis. It's reasonable to expect these results could translate well to a broad population of GBS patients. Now, let's turn to muscle strength as a measure of MRC sum score, as measured by MRC sum score, muscle weakness is a hallmark feature of GBS and MRC sum score at week 1 is a sensitive measure of the acute disease process of neuroinflammation, nerve damage and destruction. Importantly, early improvement in muscle strength is a sensitive measure of halting disease progression is, therefore, highly prognostic for functional improvement on disability and need for mechanical ventilation that requires intensive care. The MRC sum score uses a quantitative scale to measure muscle strength across 12 muscle groups in both arms and legs with a maximum of 60 points. At week one, ANX-treated patients demonstrated a greater than 10-point improvement in muscle strength compared to IVIg or plasma-exchange-treated patients who continued to decline. This statistically significant early impact of ANX005 is a remarkable and unprecedented finding and is consistent with both our Phase III results and the rapid mechanism of action of ANX005. Now, let's discuss the GBS disability scale, the GBS-DS. ANX005 treated patients also showed improvement on the GBS-DS in a consistent direction at every time point compared to IVIg or plasma exchange. ANX005 30 milligram per kilogram treated patients demonstrated an approximately twofold higher likelihood of being in a better state of health at weeks 1, 4, 8 and 26 on the GBS-DS, achieving statistical significance at week-8, which was consistent with the primary endpoint for the ANX005 Phase III trial. Now, let me highlight some of the measures impacting the burden of care from GBS. These data are compelling and point to the broad benefits of an early and complete targeted immunotherapy in an acute care hospital setting. A few notable points stood out to us in this analysis. First, approximately half as many ANX005-treated patients required mechanical ventilation. Furthermore, patients treated with ANX005 were observed to spend less time on mechanical ventilation or in the ICU, a meeting of 12 fewer days compared to those treated with IVIg or plasma exchange. What these data suggest is that ANX005 is helping patients get better sooner and regain their independence. We are also intrigued by the finding that ANX005 patients may require a less intense resource utilization, which is a key component of our value proposition for ANX005. To summarize the data from the real-world study, these findings are consistent with the outcomes in our Phase III trial and provide the first insights on how ANX005 may improve upon the current standard of care. ANX005 continues to demonstrate a differentiated ability to halt disease progression and restore patients to a healthy state sooner with his study providing context in comparison to IVIg or plasma exchange. As a targeted therapy, ANX005 may enable patients to resume normal activities of daily living. Moreover, the single dose administration of ANX005 and it's generally well-tolerated safety profile hold promise in addressing the high unmet need and significant acute and long-term burden on patients and the health care system. With that, I will now turn the call back over to Doug, our CEO, for closing remarks and to discuss the next steps for our GBS program. Doug, over back to you.

Thank you, Jamie. As you heard today, this is a pivotal time for ANX005 program in GBS and a transformational advancement for GBS patients. These findings are a win for the GBS community and underscore the potential of ANX005 to go beyond the use of nonspecific approaches to become the first targeted therapy approved for GBS. We are confident that the results from our Phase III trial demonstrate the potential for ANX005 with its novel and unique mechanism of action to change the standard of treatment in GBS. Today, we've now shared real-world data strictly the body of evidence ANX005 and showing the potential for improved outcomes over IVIg or plasma exchange. As a result, we are very encouraged by the consistent and robust effects demonstrated by ANX005 across our GBS registrational program. And we look forward to discussing these data in our overall regulatory package of regulators in preparation for our planned BLA submission in the first half of 2025. In closing, I want to specifically thank the patients, families, caregivers, physicians and medical teams who participated in our Phase III trial. We're grateful for your support and contributions. I also want to thank our collaborators and advisers as well as our employees who operate daily with a purpose and a commitment to thrive by helping others live their best lives. Finally, we'd like to thank the GBS CIDP Foundation International, IGOS and countless others, including our speakers here today for their continued support and partnership. Before we open the call for Q&A, I would like to introduce our expert panel joining us today for this segment of the call. Dr. Hugh Willison, Professor Emeritus in Neurology at the University of Glasgow in Scotland, and member of the IGOS Steering Committee; and Dr. Ken Gorson, professor of Neurology at the Tufts University School of Medicine and Chair of the Data Safety Monitoring Board for Annexon's Phase III trial. Will each of you, please make a brief comment starting with you, Dr. Willison?

Yes. Good morning, everybody. I'm Hugh Willison Emeritus' Professor of Neurology at the University of Glasgow in Scotland. I have a long-standing clinical and research interest in Guillain-Barré Syndrome and been involved in the clinical care of hundreds of patients with GBS over 4 decades. As Doug has pointed out, I'm a founding member of the IGOS executive and have represented the U.K.'s efforts in IGOS and my research focus has always been on exploring the complement-mediated pathways for GBS and developing targeted immunotherapies. In this context, the data developed in the Annexon GBS program, including the IGOS data that you've seen today seems the whole great promise for our patients with GBS, and I look forward to answering your questions.

Thank you. Dr. Gorissman?

Yes. I'm Ken Gorson from Boston. I'm a clinical neurologist with expertise in the evaluation and management of patients with neuromuscular disorders and in particular, GBS and related inflammatory neuropathies. As you've already heard, I'm a Professional of Neurology at Tufts University School of Medicine. I was the U.S. country coordinator for IGOS as well as serving on the steering committee from the study's inception. And as you've heard also is Chair of the Data Safety Monitoring Board. Like Dr. Willison, over my 30-year career, I've had an opportunity to personally care for several hundred patients with GBS and completely concur the data are so promising for ANX005.

Thank you, Dr, Gorson. We thank you both for joining us today. With that, operator, we'd like to begin our Q&A session.

[Operator Instructions] Our first question comes from the line of Anupam Rama with JPMorgan.

Thanks so much for taking the question and congrats on the update. One quick one for the company and then one quick one for the KOLs on the line. For the company, now with this sort of real-world evidence in hand, is there a publication strategy or a scientific counter strategy to kind of get these comparison data out to U.S. physicians? And then for the KOLs, with what you know about ANX005 to date, how do you incorporate -- how do you think about incorporating this product into the treatment paradigm? And since you guys are also involved in the community, are there guideline strategies that could help the broader physician community like when they think about what product to reach for?

Thanks, Anupam. Appreciate you joining. On the first question with regard to the publication strategy. Yes, the short answer is yes. We have a very extensive comprehensive registration or publication and congress strategy, both around the Phase III data set as well as around the real-world evidence matching and outcomes data set. There's the overall set of data that we, of course, like to publish. But there are multiple sub analyses that really add credibility and strength to the overall data package that you'll see from us starting shortly and over the next 12 months. Then as it relates to how this ANX005 may fit within the treatment paradigm and treatment guidelines that may be available to help treating physicians. Maybe I'll turn it over to you, Dr. Willison and Dr. Gorson, to provide your perspective.

Yes. Thanks. It's Hugh here in Scotland. Yes, thanks for that question. Yes, there are guidelines. In fact, if you go into partners, you can see the guidelines for the treatment of management of GBS, which we very recently published in a couple of journals, a huge international effort. And we also have this package called the Brighton criteria, which were involved in grading diagnostic certainty for the disease. So the community is very well versed in diagnostic and treatment guidelines. At the moment, of course, the guidelines are centered around the use of intravenous immunoglobulins and plasma exchange because as we sit here today, there are no other registered or available products for treating the disease. However, as this information becomes more widespread and 005 progresses through the licensing and regulatory authorities. I would expect a set of specific guidelines to be built up around the use of it of 005 in clinical practice. And Ken, do you want to discuss anything else?

Yes. So I would just comment in terms of a paradigm shift. I mean I really see an enormous potential to potentially revolutionize the care patients with GBS, the data are so strong from the GBS 02 trial regarding efficacy, it would seem to be, at least from what we could see comparison to real-world data that it's quite superior. And so I anticipate over time that this will displace the use of IVIg or plasma exchange as the primary achievement for GBS and 005 will become the desired treatment of choice moving forward.

Our next question comes from the line of Andrew Tsai with Jefferies.

Good morning. Congrats on this analysis, and thanks for taking my question. So I guess the first one you did kind of hint that superiority versus IVIG. But how confident are you guys that this will be used as a first-line drug in the U.S. as well as the EU and elsewhere in the world? And then secondly, do you think there could be a chance these analyses can be included in your eventual label?

Yes. Maybe I'll start and bring our experts in on that. With regard to the used frontline in the U.S. Of course, we need to get through the regulatory process, which we are actively working on. And as the prior question alluded to, we need to make sure folks are aware of the data of how to effectively use the therapy. That being said, our thought is just given the data that's been produced here in contrast to candidly much less data that's available for the standard of care. We think over time that ANX005 has not changed for a strong position in the marketplace, but maybe I'll turn it over to our experts to weigh in on that.

Yes, so Ken Gorson. So just to support Doug's comments, I'm actually personally quite confident of the data of its use throughout the United States and in the EU initially. And then I think over time, as the world becomes educated with the data, it will spread elsewhere to other countries. I think the issue is going to be around educating the neurological community like any new drug coming out. It's going to take some efforts to spread the word, so to speak, to bring the drug online. But I think I'm quite confident from the data thus far that we'll be able to execute that.

Yes. Thanks, Ken. It's Hugh here. I think it's very important to recognize that GBS is really a medical emergency. It's like treating somebody with meningitis with antibiotics or somebody with a stroke or an infant with a clock-busting drug. You don't have time to try one treatment and then decide on another because the disease has such a short, aggressive window at the early phase. And so it's very important to get it immediately with a completely disease ablating therapy. And of course, this is what 005 provides because it has immediate aggressive and complete mechanism of action. Now immunoglobulin has served us well over the years, but this has a much slower onset. We don't know how it works. It takes many days to deliver it. And when you add these different things up, of course, plasma exchange is even more prolonged and the immunoglobulin in that it takes a couple of weeks deliver a full course of plasma exchange by which time the disease is largely over. So I think having the opportunity to have an instant aggressive disease ablation therapy is game-changing. And I think it will set the therapeutic scene for the foreseeable future.

Thank you, gentlemen. Andrew, did you have an additional question?

yes, Thanks. Do you think there's a chance this real-world analysis can be included in the eventual label?

Yes. We certainly conducted this analysis with some intentionality. So it is done pursuant to a protocol, which prespecified the assessment, including the key prognostic factors for doing the assessment and it was done behind it, meaning we did not have access to outcomes if it was done of course, through IGOS and the Erasmius experts there. So that will be a conversation to be had with the regulators before we make a determination on that before we answer that question, but we certainly set it up with that hope in mind. And our publication strategy will pick it up in any event.

Our next question comes from the line of Derek Archila with Wells Fargo.

Congrats on the data, well done. Just two questions from us. The first one, I was just wondering if you could provide some more color on the trends on MRC after week 1 versus IVIg. And then second question, on the regulatory front, I was just wondering if you have already scheduled a pre-BLA meeting? Also, I guess, post this data, would you look to pursue breakthrough designation?

Andrew, good questions -- or Derek, good questions. I'll maybe start with the regulatory and then Jamie can kick it over to you all to talk about MRC after week 1. On the regulatory front, we are seeking to have formal meetings with the regulators early in the year to stay on track for BLA submission in the first half of the year. And so more to come on that. And in those discussions, we will be discussing breakthrough as you probably know, to achieve breakthrough status, you need to demonstrate a likelihood of being better than the standard of care, the real-world evidence analysis. We had that in mind when we did so, but of course, this would be a regulatory determination. And so we will have the discussion with the regulators early in the new year and hopefully I have an update on that at some point in time. Jamie, Ted, do you guys want to comment on MRC and time points after week 1 and what that tells us?

Yes. I'm happy to start and then turn it over to Ted. So we use MRC sum score as a muscle strength as an early indicator of improving neurologic and neuromuscular function. And as such, it is a very sensitive measure of that restoration of function. Once people start to -- and in the data that we have from this study, they begin to converge, not surprisingly since the disease is self-emitting. However, overall functionality at the -- beyond early time points is best described by the overall GBS disability score, which is why we present those data to show what all that means. In other words, people can regain strength but there's a lot more to neuromuscular function than just simple strength measures that the MRC sum score is meant to assess, which is why -- which includes sensory function, coordinative function, et cetera, which is not part of the MRC sum score but it's clearly part of the GBS disability score, which is why we shipped over to show that on the long term. Ted, anything more to add to that?

He may be muted. He may have gone town. Listen, I'll just -- maybe if you have anything else to add with regard to just MRC and its interrelation.

Sorry, I was on mute. I'll let you speak.

Hugh, I'm not sure if you heard the question. The question really is around.

Sorry, are you asking me? Hugh? Sorry.

Yes. If you had any additional thoughts.

Yes. Well, there's only a limit -- I mean the spectacular improvement in MRC strength in week 1 is really impressive. There's only a certain amount it can improve by before it gets close to normal. So you can't expect it to accelerate indefinitely. Otherwise, you would have people who were super normal by the end of it. So I think it's very early improvement is in itself an extremely important finding and can't be expected to carry on week on week at that level while you would go beyond the top of the scale. So if I've got you right in your question, I hope that answers it.

Our next question comes from the line of Phil Nadeau with TD Cowen.

Congratulations on the data. Two for the company and then one for the physicians. For the company, first, was there a statistical hierarchy for the comparisons between IVIg and 005 in the trial or in this analysis. And then second, can you remind us specifically what the FDA requested from this analysis, what in the FDA's mind is likely to be a positive result? And then the question for the physicians. We've seen a lot of data now from both the Phase III trial as well as this real-world extension protocol. What data do you think will be most persuasive to your colleagues to the treating physicians?

Thanks, Phil for your questions. Maybe I'll start with the regulatory requirements and then turn it over to Jamie to talk about hierarchy. On the regulatory requirements, what they were seeking really is around generalizability on outcomes from our Phase III study or ANX005, which was conducted outside of the U.S. with a patient profile in the U.S., i.e., are the patients who were treated and shown a treatment effect with our drug outside the U.S. similar or comparable to what we see here in the U.S. And so the matching exercise that Jamie nicely described really goes towards answering that question, and hopefully, it's sufficient, but that will be a regulatory discussion. The analysis on outcomes versus standard of care is more than what we had talked about. I think certainly, it's informative for the regulators to see what ANX005 does versus the standard of care and including for a potential breakthrough designation, but it was not an absolute requirement of our real-world evidence analysis. We're thrilled to provide that information to the marketplace. As you know, there just has not been a lot of innovation over the last several decades. And the more information we can provide treating physicians and patients on GBS, the disease and how to effectively treat it, we think, is really important. It's a strong commitment by the company. And so with that, maybe, Jamie, you can talk about the hierarchy, if any, that was used in the global analysis.

Yes. Thanks, Doug. So there was a hierarchy, as Doug had mentioned, you did conduct this with an intention to share this with regulatory authorities that was conducted under a protocol with mass blinding and in S&P as well. And the hierarchy as much as we shared, it was the GBS-DS at the mid-time points of 4 and 8 weeks, followed by the MRC sum score earlier and then the rest of the assessments. So yes, Ted, anything else on you?

Maybe over to Hugh and Ken, the question on just kind of the use of the drug in the market, et cetera.

Thanks for that question. It's Hugh Willison here in Glasgow. To my mind, the really impressive finding is the early improvement, I mean most patients who we treat, not most, but a significant proportion of patients who we currently treat with intravenous immunoglobulin on plasma exchange, continue to deteriorate right after initiation of treatment. What is absolutely remarkable about 005 is that it does seem to stop the disease in its tracks and leads to rapid improvement in the things that we measure disability, scale, MRC sum score, time on ventilation, whether or not you need to go to become ventilated at all time on intensive care for the patients with GBS. These are absolutely critical improvements over current best treatment. And so to me, it's this early first few weeks of the illness in which we really see this extraordinary benefit of 005 compared with existing practice. And I think that's where it will really make its mark.

Ken, can you...

Ken Gorson here. Just to give some perspective about caring for these patients. The vast majority of patients who are non-ambulatory or ventilated will remain that down, wheelchair-bound or on a ventilator going into several weeks into the illness and often they'll progress through treatment with IVIg and plasma exchange. That's not at all uncommon. So just from a 30,000-foot perspective, seeing a 10-point change in the MRC in week 1 is just extraordinary. I mean that's just not seen in the care of these severely affected patients. And then the other side of this is just, as you had said, recognizing getting off the ventilator and half the time out of the ICU, you just prevent all of these complications of critically ill paralyzed patients on ventilators and the ventilator-associated pneumonias, the sepsis, the pulmonary emboli, all of these issues that add to the morbidity in caring for GBS patients in an ICU setting.

Our next question comes from the line of Pete Stavropoulos with Cantor Fitzgerald.

Thank you for taking our questions and congratulations on the data. Great to see the [indiscernible]. First question for the KOLs, it could be hard to break physician behavior in terms of the way they treat certain conditions. And it's my understanding that neurologists are familiar and comfortable with IVIg, would you imagine 005 will be used as a monotherapy when it's initially available? If so, first line or just in patients who cannot be administered IVIg for some reason?

It's Hugh here. Maybe I'll answer that first. I think what this -- what this targeted immunotherapy will do as a -- with a known mechanism of action and an instant virtually instant full therapeutic effect. I think what it will do is shift people into recognizing that here we have a drug, which can be used in an emergency to treat evolving paralysis in a patient with -- diagnosed with GBS. And at the moment, we just don't have that opportunity. So I think that is where it is fundamentally different from the current approach. And it should and I expect will from that point of view alone become very widely used. It's not a second-line treatment. There's no point -- peak -- the majority of patients with Guillain-Barré Syndrome get to their peak disability by 10 days and certainly all by two weeks after clinical onset. So you haven't got time to wait, this isn't a drug which you'll come along with after a couple of weeks after they've had immunoglobulins here and, well, maybe we're just -- we'll try this. 005 is a drug which needs to be given urgently as soon as diagnosis has been established in a patient. And I think that is where it's fundamentally different from current therapy with immunoglobulin. I'll close there.

Thank you for that color. For Doug and the team, you showed data from multiple time points. From a payer perspective, what are the key data that you think could result in premium pricing. And second question I have is what are the differences in terms of safety that 005 could differentiate on? And do you expect a black box warning?

Yes, good question, Pete. Too early to get too far into the value-based pricing proposition and what we may do with regard to that. But I will say that we're encouraged by multiple aspects of the data. Firstly, I mean, our charges to help patients get better sooner. As you've heard today, that's what we see in our data, both in the Phase III study as well as in the real-world evident by week 1, you see a very consistent powerful effect in patients getting better. And then it's durable. And turning back to our Phase III study where you look all the way out to 6 months, where 2.5 more patients are likely to be in a better state of health versus placebo. So it's rapid and it's durable. And of course, as you also heard today, getting patients off a ventilator and out of the ICU and able to walk sooner is just paramount to them living their best lives. And so we think all of that is relevant for discussions with payers on how to appropriately position this drug out in the marketplace. So overall, we're really encouraged by this, but more work to do to bring it to folks on the bedside.

Okay. Any differences in terms of safety of 005 could differentiate?

Yes. Sorry about that, Pete. Yes, I mean, listen, maybe I'll turn this over to Jamie and Ted, before I go too long and I get excited by that topic. Jamie, Ted, you guys want to weigh in?

Yes. Thanks for the question, Pete. The -- as we know, IVIg does have a black box warning. This is clearly in a different class than IVIg. So I do not expect that kind of statements to carry over so far. All I can say is that the data in general -- the product is generally well tolerated. And we've not seen items that raise significant concerns, but obviously, the final determination is to interact with the regulators and that negotiation.

Our next question comes from the line of Joseph Stringer with Needham & Company.

Question for the company on the data. What percentage of the patients in the IGOS group were treated with either IVIg or PV? And did you see any differences in either of those subgroups compared to the ANX005 Phase III data?

Yes, Jamie, Ted, do you guys want to weigh in on that?

Ted, do you know that? I have that actually.

Yes, I think it was for IVIg was about 3/4 of the patients, and I'm not aware of any differences that is 3/4 for IVIg, 1/4 of plasma exchange, and there were no differences in those data sets, those populations.

Ladies and gentlemen, that concludes our question-and-answer session. I'll turn the floor back to Mr. Love for any final comments.

All right. Thank you so much. Listen, thank you all for joining us early this morning at late in the year. We really appreciate your attention and your good questions. We're excited, as you probably hear from the call. Taken together with our Phase III results, we think this real-world study really strengthens the body of evidence supporting ANX005 used as a treatment for GBS. We are now sharply focused on partnering with the regulators worldwide as well as, of course, the physician community and our patients. And so more to come into the year, and we wish you all a very nice holiday season, and thanks once again. Take care.

This concludes today's conference call. You may disconnect your lines at this time. Thank you for your participation.