Great. Good afternoon, everybody. Thanks for sticking around for our last fireside today. We are really pleased to have Vera with us. I am going to let Marshall make some introductory comments, and then we will jump into Q&A. Over to you, Marshall. Thanks.
Matthew, thanks for having us and me at your conference. Great to have an interview with you today. I am Marshall Fordyce. I am the Founder and CEO of Vera Therapeutics. Great day for us today and for patients. Today, we shared the final efficacy analysis from our pivotal phase III trial, showing outstanding efficacy by GFR and by kidney composite endpoint. It was an important update for the field and for Vera, as well as an indication of how launch is going. We shared that so far, about 10 weeks into our first commercial launch, we have over 350 patient start forms or roughly prescriptions as an early indication of demand. Two important updates for the company. Great activity happening now and in the coming time. Here we are in September. The data that we top-lined today are being submitted or have been submitted both for presentation and publication.
It would be great if we could get that done at the next month's major kidney conference, which is called ASN, where we would hope to have an opportunity to present these data in a more fulsome way to the academic world. In conjunction with that, we of course, are pursuing an sBLA in Q4 to target full approval with these top-line data and more for mid-2027. A fantastic moment for us as a company. Happy to dive into detail about both the clinical results that we have shared today, as well as the commercial progress early on in our first launch in the disease IgA nephropathy with TRUTAKNA.
Wonderful. Yeah, no, I mean, really exciting updates for today. Maybe first, can we put the clinical data into context? What does what you share mean to patients? How should people think about progression of the kinds of patients that you have put into the study and how you have changed that progression?
Sure. I mean, we ran a global randomized double-blind placebo-controlled trial in the typical patient who is at high risk of disease progression. To put it into context, think about who are these patients and what do they experience on current standard of care. They are 40 years old. Their GFR is on average 60, which means that they have already lost roughly 40% of their kidney function by 40 years old. If you follow the placebo arm, they are losing up to 5 mLs- 10 mLs per year in our trial. These are patients who are 40 on their way to dialysis by the age of 50. That puts you into the context of what disease situation are we trying to alter. What we have shared is data from the final efficacy analysis. We have ended the randomized phase.
What we have shared is that the GFR difference, meaning the kidney function difference between TRUTAKNA and placebo, is 5 mLs per minute per year. That is significant. That means that over a two-year period, 104 weeks, which is the extent of the data we looked at, if you are on placebo, you are losing 10% of your kidney function. That is a bad situation. We essentially have normalized the GFR for patients on TRUTAKNA. The GFR slope for those patients over that 104-week period was -0.6, so just under 1. People without disease lose about 1 mL per minute per year. This is on average what a person without disease experiences. It is also what international guidelines are trying to target for patients to reverse these bad outcomes for patients.
We shared the primary endpoint, the confirmatory endpoint that we agreed with FDA, which is at one year at 52 weeks, and we also looked at the totality of the data where lots of patients went out to up to two years, and that is the two-year GFR slope comparison. So at 5.0 as a treatment delta is the largest number we have seen in the field. Moreover, we shared clinical outcome data. What happens to those patients on placebo? Well, in our trial, right of those patients met hard, tragic clinical outcomes. They required dialysis, transplant, or they died. Eight of them on placebo. On TRUTAKNA, none met that outcome. If you add another criteria, which is a significant reduction in GFR of greater than 30%, so that plus the hard clinical outcomes, we call that the composite kidney endpoint, we showed a hazard ratio of 0.24.
The inverse of that, we also call the risk reduction, was 76%. That is a massive risk reduction. That really is the best we have seen in any IgAN trial, and you can look even broader at other kidney trials. That is a very big effect size for a new medicine. On these data, we plan to continue to lead the field and set a new standard of care for patients. Today, TRUTAKNA is now available to patients in the U.S. through accelerated approval. I think these data will be incredibly important for patients, for providers, and for payers as we advance our effort to change standard of care.
And obviously, you showed some stuff on the commercial side, and I want to get to that, but maybe just a couple of other questions on the profile. Obviously, there is another therapy with a similar mechanism filed. Otsuka has their drug on the market. Can you talk a little bit about the breadth of the data of the competitors and how you think you compare?
Sure. First, I think it is really important when you look at a disease area that has a standard of care with a lot of hope that we can raise the bar. It is great that there are multiple players in that space. That increases the share of voice for changing standard of care. We actually think that is very helpful for patients and for Vera's potential to really penetrate that market. That mechanism is an APRIL-only mechanism as opposed to dual BAFF/APRIL. From an efficacy perspective, we see our delta between active and placebo at slope for two years is 5.0. Theirs is 4.5. We have also seen no data yet on the composite kidney endpoint that I am describing. We look forward to seeing that. We are really the first among the B-cell modulators to share that.
Safety looks strong for TRUTAKNA, and we also have the first autoinjector on the market. This is a very simple device that is simple to use on a weekly basis. I have it here in my hand. No button push needed. It simply is an injection once per week, and it takes just a few seconds. That is what is being asked of patients today as we are out in the field, and this is something that I think we really see a lot of acceptance from both patients and physicians.
Then I think the last thing, maybe just comment, you have a dual mechanism, APRIL and BAFF. There are other B-cell modulators in this class that have chosen one of the mechanisms. Any differences you think you are seeing from that in the clinical data so far?
Well, I think the clinical data need to mature further, and we just happen to be the first BAFF/APRIL on the market today. I think I would be looking at long-term kidney outcomes that we've shown today and also GFR delta as being the most important to patients, providers, and payers. I think it remains to be seen how that plays out across the field. But look, this is a really important new step for patients, for the field in setting a new standard of care.
Great. So why don't we talk about commercial? You obviously gave some updates, but maybe before we get there, can we just talk a little bit about market size, how you think about the market size, and what sort of standard of care for patients is now and what that tells you about patients that are being treated versus how you might expand that market?
Sure. This is a very serious unmet need, as I characterized by the patients who we enrolled in this trial. We would estimate this to be, in the U.S. alone, roughly 160,000 patients are biopsy-confirmed IgAN patients. We think that that's likely an underestimate. By the time they come on to treatment or into a trial, they tend to be pretty advanced with a GFR of 60 and already 40 years old. So we hope to see that shift. That's a large number of patients. The market size is likely very large, in the $10 billion-$20 billion range when you look at the value that's being delivered for that number of patients. Not all of them will be considered at risk for rapid progression, but the label that TRUTAKNA has is broad. It's for any patient at risk of disease progression.
Today, most nephrologists define that by those on maximally tolerated standard of care, an ACE or an ARB, most often an SGLT2 inhibitor. For us in phase III, it was about 60%. Then with that background, they're still producing over a gram of protein in their urine. That is characterizing the patients who have the placebo GFR trajectory and the hard clinical outcomes that we've reported in a relatively short amount of time and in a low number of patients. So really, that is the market. Already, we're seeing from Otsuka, one of the strongest, maybe the strongest renal drug launch that we've ever seen in history. They've guided to a $380 million sales number in 2026 in their first full year of launch. That's a good indicator that this is likely a very large market.
What we shared this morning is that we have over 350 patient start forms in the first 10 weeks. That tracks very nicely and demonstrates early signs of a growing and large market. We think this will continue to grow as others come to market, and this is a great thing for patients and for the field of nephrology. We see already feedback from the field telling us about our data. That means that we've really been out there. We have good relationships with the physicians in this space, and there's a lot of excitement for a new medicine that can actually stop kidney function decline and change hard outcomes for patients. That'll be really important data for our launch.
Can you characterize, I mean, you characterized a little bit the 350 start forms, but how should people think about that relative to Otsuka's initial trajectory and anything you can share in terms of payers or how people should think about what you need to do on the payer side?
Sure. We have fantastic commercial leadership within Vera. We hit the ground running. Patient start forms were generated immediately. Drug was in channel within three weeks. We have made good progress. It would be a mistake to say it was linear for the 10 weeks. Of course, it's taken time to get going, and we hope to accelerate that. You can make a model, but we know that we're generating that market every single day in the field. Vera is entirely focused on that execution. So that feels really good to see that growth. On the payer side, we see an objective for us would be, to have similar access to other drugs in the market. We think that is the right way to ensure that physicians keep prescribing decision is in their hands and more so than in the payer's hands.
Our objective is really parity, and that's an important feature of what we'd like to do in this market.
Great. Maybe just talk about in the context of what you need to do to resource this launch. Can you give people a sense of how big the commercial infrastructure build is and how that impacts your path to profitability?
Sure. We have resourced the launch. It was really important for us to hit the ground running. We have the right-sized sales force. We have 82 reps. We think that is appropriate for the U.S. area. We're targeting about 6,000 practicing nephrologists. We have additional resources to take up the tail of the remaining thousands in the U.S. for prescribing IgAN clinicians. For those 6,000, 82 reps really has the right contact. We already have early telemetry from our launch that feels right-sized in terms of our ability to touch decile eight, nine, 10 physicians, even multiple times within the first 10 weeks of launch. Feels great for where we are. There's other resourcing to be done on the marketing side, and we'll see that evolve this year.
Last year at ASN, we had the number one share of voice among the IgAN developers, and that was from third-party data. We feel really good about how we've been out in the community, leading the conversation about B-cell modulation with TRUTAKNA and dual BAFF/APRIL. We've presented really field-leading data. We're recognized as the leaders by the physicians out there. We continue to do that. We did it today. Coming up at ASN next month, we'll continue to target that.
Any changes you think you need to make? Obviously, there's a third competitor potentially coming on market later this year. Presumably, you've already resourced for that, but any other changes or how do you think that changes the market dynamic?
Yeah. It does not change our resourcing plan. I think, again, it is a positive that we will have another entrant that is talking about dual BAFF/APRIL inhibition. We think that is a positive for Vera. We welcome the added voice for B-cell modulation. It remains to be seen what that profile looks like. We do think that GFR data and renal composite endpoint data is going to be important out there. I think we will have that for a long time before we see that from competitors. I think that will be an important differentiator in the near term at least, and sets a high bar for others to try to reach.
Great. As we sort of think about additional updates from you, obviously, at your next quarterly, you will report sort of the initial launch number. I mean, other metrics that investors should be focused on that you will be talking about more?
Sure. Revenue. We will be talking about revenue in November. Q3 update roughly November is our time for our earnings call. That will be key. We will continue to provide quarterly updates on patient start form. We give the market an indication of demand. We will not be very granular beyond that, frankly. I think it is important to expect a quarterly cadence for these updates.
That will be an exciting moment for us to continue to start to put points on the board and show this launch trajectory. Happiness for us is seeing growth for TRUTAKNA and seeing growth for the other B-cell modulators. We firmly believe this is a $10 billion-$20 billion market. It is all about giving access to patients who really need it and otherwise face really grave outcomes. That is really where we are focused. Near term, we are going to see a lot of expansion.
With the profile that TRUTAKNA now has, the efficacy that we highlighted today, with the safety that is also consistent with what we have shown before, which is very tolerable, and finally, the form factor of the autoinjector, we are looking at global expansion. We will be filing in Europe in the near term. We will be filing in Japan. We are also starting to show data outside of IgA nephropathy. We are hoping by the end of this year, we will have the opportunity to present data in IgAN patients who would not have qualified for our phase III trial. That includes patients who are adolescents, who have lost kidney function due to IgAN and have a transplant, are now worried about maintaining their transplant, patients with concomitant vasculitis, patients with lower proteinuria than would qualify them for ORIGIN 3. These are important questions for physicians.
They're thrilled we're studying it, and we're going to be able to provide some clinical data. Already today, our label for Accelerated Approval is broad and would capture much of that population, but providing that clinical data, I think, is useful for the field and continues to demonstrate our leadership in the IgAN space. We'll also start to show data in adjacent autoantibody-driven kidney disease, starting with membranous nephropathy, and we hope we'll have that opportunity by the end of the year.
Maybe just touching on that, can you talk a little bit more about PIONEER, that study, the kinds of patients you've enrolled there? You talked about membranous nephropathy, but I think you also enrolled FSGS and MCD patients, if I remember right. What's that study? How should people think about those market opportunities and the path for those indications?
Sure. Look, I think the big idea that Vera got invested in six years ago when we began developing atacicept is that you can treat autoimmune disease in a way that doesn't immunosuppress a patient the way that high-dose steroids or B-cell depletion does. That big idea is now increasingly supported by the available clinical data in our phase II and phase III trial. That's not just mechanism, it's not just BAFF/APRIL, it's also what molecule are you using? We're using the native TACI receptor and a fusion protein. What dose did you select? We're selecting the 150 milligram weekly dose. With that as the totality of our approach to dual BAFF/APRIL inhibition, that gives us a profile that has a safety and efficacy profile that we now can take in other areas.
In the PIONEER study, as I mentioned, there are sort of two buckets. There's the IgAN population that we didn't study in phase III. An important one, of course, is concomitant vasculitis, which is a question we often get from physicians, but also autoantibody-driven kidney disease and, as I mentioned, membranous nephropathy. There is a portion of FSGS or minimal change disease that is autoantibody driven. We actually don't know the portion. There's some studies that put that into the 15%-20% of FSGS patients, but it could be higher. We will find out. That's just the beginning of what the expansion opportunity looks like for TRUTAKNA. If you leave the nephrology space and go into the rheumatology, neurology, and dermatology spaces, suddenly you have a much broader population that could benefit from a medicine with this type of profile.
Among indications that there's strong validation for, whether it's a study in China or elsewhere, could include Sjögren's disease, myasthenia gravis, and a variety of other diseases that we think this kind of profile could be highly competitive in. We're thrilled about the ability to expand near term our commercial execution in the U.S., and this large double-digit billion-dollar market is the near-term priority, but we are expanding quickly into other areas. The pathway in adjacent indications we haven't shared yet, but we will in the near future.
Okay. From a data perspective, it sounds like people should expect to see at a minimum the other populations you haven't studied in IgAN and maybe a little bit of some of these additional populations towards the end of the year. Is that the right?
Yes.
Okay. You talked about other geographies. Maybe just talk a little bit about your plans from a commercial perspective for them.
Sure. It begins with clinical regulatory progress with the data set that we've now disclosed and finalized in the U.S. We're taking that to Europe, Japan, and elsewhere, and those are really the first steps. We've done the basics we need to do to start to expand into Europe. These are stage appropriate steps for global expansion. But for now, it begins with the clinical regulatory progress. We've run our global studies in the U.S., Europe, Japan, China, really in multiple geographies.
Great. Maybe just I guess a couple other things. Maybe just remind people, patent profile here, how long you have. Obviously, you're very beginning of your launch, but just how people should think about your ability if you can expand into a bunch of other diseases.
Sure. TRUTAKNA is a biologic, so we do begin with a 12-year regulatory exclusivity in the U.S. But we have awarded patents through 2042 when it comes to formulation process methods of use, so we don't anticipate a biosimilar entrant on that basis until the mid to late 2040s.
Any other. Obviously, and we've talked about this a little bit, right? There are different frequencies of dosing, right? Have you thought about other frequencies that you might explore beyond the weekly that you currently are, your current presentation?
Yeah. Well, in the last 10 weeks of our launch, we've had over 12,000 interactions with healthcare providers around TRUTAKNA, and we never hear that dosing frequency is an issue. Given what I've demonstrated here in eight seconds, this is not a burden for patients, so it doesn't come up currently. On the other hand, we do think that providing additional options for patients in the future could be beneficial. So we're doing two things. We're running a monthly dosing study that we began last year. It's a study in IgAN patients at three different doses, and we'll be evaluating that data later this year. So that has the opportunity to give us a line extension for monthly dosing. We in-licensed a molecule from Stanford University in January of 2025, what we call VT-109, which is a different construct for BAFF APRIL inhibition.
It's still preclinical but has the characteristics that could look at a longer dosing interval. We'd like to continue to gather data on those, but I would tell you that currently, the autoinjector weekly small volume, eight seconds a week is a pretty acceptable profile in today's market and delivers really practice-changing efficacy and very tolerable safety background. We think we're in a very good position with respect to dosing interval today.
Great. Maybe two final things on my mind. As you think about the data package that you have now and the label that supports, anything that would change as you go for full approval from Accelerated Approval and anything important in that from a payer perspective, from an ability to market perspective, from a demand perspective that you think is important to highlight?
Yeah. Look, all of those things are important. The more mature data set that we're top-lining today that we hope to put out into the nephrology field later this year in a presentation publication, getting that reviewed by FDA, seeing if that is appropriate for the label, that would be our expectation in a final approval and in a label. Incredibly important and informative to prescribing physicians and the patients who may take that medicine. That is important. It's important for continued effort to change standard of care. Yeah, critical updates. Although important that we have it out there in the public today and important that we will have it in a peer-reviewed setting in the near term. Yeah, incredibly important. I would also say that guideline committees are going to probably be meeting and changing guidelines are important.
That's all going to be driven by what is the benefit and what is the risk. We think today's data update is really important for that.
And then I guess final question just around cash runway, how you think about how you are financed as a company. Maybe just sort of comment on where you stand there and your ability to finance the launch.
Sure. Well, it is great we are generating revenue today. We have $500 million in cash. We have a facility with Oxford for an additional $425 million, so access to close to a billion to continue in this current mode. The launch curve is looking good, and we are going to continue to draw dots on that curve. We got a lot of options at this point. So we feel good from a resourcing perspective. As you asked, wouldn't change anything in terms of what we are doing in terms of our launch. We hit the ground running. Don't expect to really change that a whole lot given what we see today.
Awesome. Great. Marshall, thanks for being here. Appreciate it.
Thanks for the opportunity, Matthew. All right.