Welcome, everyone, to the 44th Annual JP Morgan Healthcare Conference. My name's Anupam Rama. I am one of the Senior Biotech Analysts here at J.P. Morgan. I'm joined by my squad: Rati Pinhey, Priyanka Grover, and Joyce Zhou. Our next presenting company is Vera Therapeutics, and presenting on behalf of the company, we have CEO Marshall Fordyce. Marshall.
Great, thank you so much, Anupam. Good afternoon, everyone. Welcome. Thanks for the opportunity to present to you today. I'm Dr. Marshall Fordyce. I'm the founder and CEO of Vera Therapeutics. Our mission is to lead a paradigm shift to a more targeted way of modulating the immune system and free patients from the burdens of their disease. Today, I'm very pleased to present, at the outset of 2026, our outlook as we approach the commercial launch of our lead product candidate, atacicept. Before I get started, I want to remind you that my remarks contain forward-looking statements under the Safe Harbor Act, and as such, we'll present this disclaimer regarding at-risk statements. Vera was founded here in San Francisco in 2016, and we licensed atacicept in 2020.
Atacicept is the first-in-class dual BAFF/APRIL inhibitor, a mechanism that holds promise to control autoimmune disease activity while avoiding the challenges of immune suppression. Based on positive phase III results last year, Vera submitted a BLA to the U.S. FDA and was just last week awarded priority review with a PDUFA date of July 7th, 2026. Atacicept is on track for commercialization in IgAN this July, and it's also under investigation in a phase II trial in adjacent autoimmune kidney diseases: primary membranous nephropathy, autoantibody-driven FSGS, and MCD.
Vera has two other molecules in our pipeline: a phase II asset called MAU868, a monoclonal antibody against BK virus, which is a leading cause of kidney transplant failure, and a preclinical asset, VT-109, that we licensed from Stanford last year, a novel fusion protein with the potential for immune modulation with much less frequent dosing intervals on the order of two to four per year. Vera maintains ownership of each of these molecules in all indications and all geographies. Vera's management team brings deep leadership experience and a track record of success in clinical development and blockbuster commercialization. We are the leading innovators in nephrology through rigorous clinical science, patient-centric development, and engagement with the physician community. We have a strong cash position with pro forma cash of $779 million, with 71.3 million shares outstanding.
In addition, we have access to $425 million in non-dilutive capital through our Oxford Finance financing facility. I want to give you the big idea first. Atacicept's mechanism of action of dual BAFF/APRIL inhibition has broad therapeutic potential for many autoimmune diseases, which are substantially driven by abnormal or overactive B-cell function. The two known circulating cytokines, BAFF and APRIL, are both important for the survival and maturation of the B-cell lineage. Elevations of both BAFF and APRIL are found in patients with IgA nephropathy, lupus, and certain other autoimmune diseases that I'll highlight later in my presentation, and both play a key role in disease pathogenesis driving autoantibody production and damage to the body. Atacicept is a biologic fusion protein that makes use of the true unaltered TACI receptor and has picomolar binding to both BAFF and APRIL in binding assays, as expected from TACI's natural role in B-cell biology.
IgAN, or IgA nephropathy, is our lead indication. It is the most common primary glomerular disease worldwide, with an incidence of 2.5 per 100,000 people. This is a disease of young people, with approximately half of all diagnosed patients reaching kidney failure or death within 10 years, meaning before their 50th birthday. Current treatment guidelines for nephrologists recommend reducing the rate of kidney function decline as measured by estimated glomerular filtration rate, or eGFR, which is calculated from a blood test that you would get when you see your doctor, a serum creatinine. A new therapy that could stabilize kidney function decline and potentially avoid dialysis or transplant at 50 years old or beyond would be completely novel and transformative for these patients. IgAN patients currently face a very challenging life ahead.
If their kidney function decline leads to end-stage kidney disease or ESKD, the need for dialysis or kidney transplant dramatically alters their lives and those of their families, with mortality over five years similar to stage IV colorectal cancer. Through rigorous clinical science, we have aimed to demonstrate that the inhibition of immune complex formation in IgAN through dual BAFF/APRIL inhibition offers the potential for these patients to avoid kidney failure over their lifetime. IgAN is a disease of the immune system in which BAFF and APRIL levels are elevated, driving circulating immune complexes, causing a cascade of downstream damage to the kidney through inflammation, fibrosis, and loss of organ function. Atacicept, as I mentioned, takes the unaltered TACI receptor that binds both BAFF and APRIL, and in patients with IgAN, this mechanism holds the promise of targeting the disease at its source.
And so, as we conceive of what this target and this way to drug the target could imply for clinical outcomes, we imagine that at the right dose, we should be able to demonstrate four measures of clinical effect: reduction of immune complexes in the blood as measured by Gd-IgA1 in the upper left; resolution of inflammation in the kidney as measured by hematuria or blood in the urine in the upper right; reduction of protein in the urine, a sign of glomerular dysfunction. The filters in the kidney are letting protein through abnormally. And finally, in the lower right, the most important stabilization of eGFR, stopping kidney function decline. In Vera's phase II-B study, for which we have reported two-year follow-up, we have shown just that. Halting kidney function decline is completely novel in IgAN, and the implications for young patients facing dialysis are profound.
Subsequently, in phase III, we designed and are conducting a multinational randomized double-blind placebo-controlled trial comparing atacicept 150 mg to placebo with a primary endpoint of proteinuria at nine months and a secondary endpoint of eGFR at two years, in alignment with the U.S. FDA. In this consort diagram, at the 36-week interim analysis presented last year, retention on atacicept was high at 93% versus 87% on placebo. The baseline characteristics include enrolled subjects that were similar both in phase II-B as well as phase III, and these are patients who are at high risk of disease progression and were on current optimized background chronic kidney disease treatment. Baseline characteristics reflect a relevant and diverse population. Mean age was 40 years old, mean eGFR of 65, mean proteinuria of 1.7.
This pivotal phase III trial met its primary endpoint at 36 weeks, achieving a statistically and clinically significant reduction in proteinuria with a 42% placebo-adjusted delta. Importantly, in phase III, the treatment effect on proteinuria reduction was robust across all prespecified subgroups according to demographics, baseline kidney disease by both proteinuria and eGFR, as well as background treatment. Secondary endpoints were also consistent with what we showed in phase II. We've reported secondary endpoints Gd-IgA1, the measure of immune complexes, and hematuria, the measure of active kidney inflammation. These results are similar to those in phase II. Per FDA's recommendation, we have not reported eGFR results, but of course, these are part of our BLA submission, but the registrational trial is still ongoing, and I'll highlight that we plan to share these eGFR results when available, estimated in early 2027.
Importantly, the clinical safety profile of atacicept was similar to placebo. Atacicept was well tolerated in IgAN patients with no reported deaths, a low rate of serious adverse events of 0.5%, and a low rate of adverse events leading to discontinuation. There was no evidence of opportunistic infections. No significant imbalance of infectious adverse events, either serious or mild, was seen in the active arm. Some increased rate of injection site reactions was observed on atacicept, but these were mostly mild or moderate in severity. And recall that our experience in phase II-B has shown over 90% retention of patients self-administering weekly atacicept for over two years. These results were presented at the 2025 Kidney Meeting, ASN, in Houston last November at the opening plenary session and simultaneously published in the New England Journal of Medicine.
With our cumulative clinical data in both phase II-B and phase III, Vera is preparing for commercialization this year with a winning profile, with the longest-term efficacy data from a randomized controlled trial in the B-cell modulator class, with a rapid and sustained response seen consistently across our two global trials and across all subgroups. With a differentiated safety profile and desirable patient-centric features of delivery, Vera is well positioned to address a significant unmet need for IgAN patients in the United States this year. Atacicept has product characteristics similar to other blockbuster biologic drugs. We have presented atacicept for review by FDA as an at-home, self-administered 1 mL autoinjector using a 27-gauge needle, a rapid injection time with once-weekly frequency. Recent third-party surveys indicate that nephrologists view atacicept as one of the most desirable IgAN agents in the development pipeline.
There is strong interest in B-cell modulators for IgAN, with most participants willing to prescribe them for high-risk patients as well as patients earlier in the treatment paradigm. It's a highly dynamic category-creating moment for new therapies in IgAN. How many patients are there? In the United States alone, we estimate that the U.S. population of diagnosed IgAN patients is roughly 160,000 patients, with roughly half that number in the highest-risk category matching our pivotal trial study population. Vera is also studying atacicept's potential benefit in moderate and low-risk patients in the ongoing PIONEER trial, with results expected this year. There are six cohorts in the PIONEER trial, which includes not only moderate and low-risk patients, but also patients who are adolescents, patients who have ongoing or recurrent IgAN after kidney transplant, and the like, which serves a very important data gap for these patients with a high unmet need.
New practice guidelines are recognizing the potential opportunity to stop disease progression and specifically call to initiate treatment at lower proteinuria thresholds, currently at 0.5 g per day, which falls into that moderate-risk category. The treatment targets described in guidelines speak directly to the treatment effects we have shown with atacicept, including prevention of immune complex formation, reduction of inflammation, reducing proteinuria, and most importantly, stopping eGFR loss to a rate of less than 1 mL per year. Over the past five years or so, five new drugs have been approved for IgAN, each with premium pricing annualized on this slide. The IgAN market has many hallmarks of an attractive commercial opportunity, and atacicept is well positioned to meet this substantial unmet need. There is a large and growing market with a favorable payer mix and a significant opportunity to deliver a differentiated product.
The key measure of efficacy recognized by FDA, nephrologists, and of most concern to patients is their overall kidney function eGFR, where atacicept has long-term data supporting a return to normal eGFR slope. Moreover, with the clinical profile that has emerged in our developing program, atacicept represents a paradigm shift in how we might treat a much broader array of immune diseases, which represents an even bigger opportunity beyond kidney disease alone, and I'll touch on that before I close. Therapeutic potential in other autoimmune diseases depends on a mechanistic match in which BAFF and APRIL are elevated and apparent key drivers of disease, and we see an opportunity to expand into a double-digit billion-dollar market as we build our pipeline.
We identify here at least 12 distinct indications for which there is significant unmet medical need, a strong mechanistic fit, significant clinical validation, attractive regulatory pathway, and opportunity to differentiate from the emerging standard of care. Among these indications, we estimate about 1.2 million addressable patients between nephrology and non-nephrology diseases in the U.S. alone. In closing, Vera is poised for a transformative year in 2026 as we gather momentum towards our U.S. commercial launch. Priority review is currently underway with a PDUFA date of July 7th. Our phase III trial is ongoing with a two-year confirmatory GFR result expected in 2027, with full approval projected to 2028. In addition, our Extend and Pioneer clinical trials studying atacicept in additional IgAN cohorts and adjacent autoimmune kidney diseases are enrolling very well, and we'll be sharing clinical data results at a nephrology conference later this year.
I want to thank you for your time and interest and would be happy to answer questions. Thank you very much.
Thank you, Marshall. As always, I'll ask the first couple of questions, and then if there are questions in the audience, just raise your hand and I'll call on you. Marshall, I want to start out with a question that I've been getting a lot here in the last week or so, which is just if you could comment on the IP estate for atacicept?
Sure. Atacicept is a biologic product. We licensed this from Merck KGaA in 2020, and it was a phase III-ready program at that time. Formulation and process, since we've brought it in-house over the last five years, has had substantial improvements. We have full control of the supply chain, and that's generated not only new IP but also trade secrets and know-how.
So it's a very well-protected molecule. Most recently, there's been some public updates on the way that we've built our picket fence around this asset, including methods of use that get us to 2041. Our target is to get to 2047 and beyond, and this is a very standard playbook for a molecule like this. Yeah.
And then as you have a PDUFA now, right? So what is going to be kind of your key medical education market prep work here in the first half as we look to PDUFA action date?
Yeah, I like the surprise in the voice. We're not surprised. We expected this. That was a good surprise.
Yeah, a good surprise.
We've been preparing for this for a long time, and it's been great to have our Chief Operating Officer, DJ Johnson, who joined us from Global Blood Therapeutics and previously from Gilead Sciences.
He oversees the overall commercial organization, CMC, and certain G&A activities. We have Matt Skelton, our EVP of Commercial and leading the U.S. launch. They're really building on work that has been going on at Vera for four years. We believe that when you have the right target molecule data, you need to communicate about that data, and we've been very active out there. So disease state awareness, this is a category-creating launch. We've taken that seriously now for years. We invested heavily in our ability to communicate to the nephrology community with a focus on the United States and we're very pleased at the end of 2025 to see the slide I showed you, which is a very good awareness of atacicept, its data, and ensuring that there's broad understanding of this disease, its mechanism, and the data that we have coming forward.
There's a lot of work that has been done and that will continue to be done as we prepare for launch. But the team is fully in place. This is a commercial company. We have all of our sales leadership in place, and sales force is being hired in the very near future.
Questions from the audience? What's going to be the size and scope of your sales force as you look to a launch here? And when could we get an update on what that structure looks like?
Yeah, happy to share it right now. We're hiring 82 sales reps. We envision a very strong coverage and the right sizing of that sales force. There are about 11,000 nephrologists in the United States. We'll target a bit more than half of those. This is a size and structure that makes a lot of sense to our experienced leadership.
Matt, overseeing the U.S. commercial launch, has chosen to have good span of control. We have a Western U.S. sales leader and an Eastern U.S. sales leader who I've been in the field with who are fantastic. They've hired their regional managers, and the next step is hiring and sales training. So some of it's about size and structure. A lot of it's about the quality of people.
And competitively, there's going to be a bunch of updates in the space, right? You're going to have Vertex's initial proteinuria data, Otsuka eGFR data. How do you think about that in the context of what you've shown with atacicept already?
Yeah, I think we've set the context. We've set the bar that one needs to meet. And that really, when we think about new products, we used to say efficacy is king. The most important thing is that GFR stability.
If you're a 35-year-old with a diagnosis like this, you want to take a drug that's going to work over the long term. We've shown two-year data that gets a 35-year-old to 37. That's not enough. We're interested in decades of durability. So we like that we're dual BAFF-APRIL inhibition. We like that we are the native TACI receptor. We like that we have the longest-term data out there in the field. And it's our view that it would be very challenging given the unknown pathophysiology. Once you get to fibrosis and you show up with a GFR of 60, you can't reverse that. You need an antifibrotic for that. So the best another program can do in our view is to match it, and that sets a really high bar. I will also say that it's good to not be alone.
We're not the only ones raising awareness about this unmet need, about the importance of modulating B cells in a safe manner with a good therapeutic window, and so it helps us that we're not alone in that, and we welcome the chorus of interest in helping these patients.
Questions from the audience? Marshall, if I could just push a little bit on what you just said, right, so I agree with you. This is an eGFR game. I agree with you. These patients are young. They're going to be on these products chronically, right, so if you had to think about it, what is the minimum eGFR delta between two products, right? Because this is a chronic disease, and even 0.5 mL will cumulatively add, so what is that minimal delta that you think is clinically meaningful as you think about these patients chronically?
I love that question.
So first, those of us who are lucky enough not to have a chronic kidney disease lose 1% or 1 mL per minute per year. So that's normal. In phase II, we showed a - 0.6 slope, which we would round to 1. So that's why we were excited. You have to think about what does the disease do. If you're at high risk, you're losing 5 mL - 10 mL per minute per year. You're losing up to 10% of their kidney function every year, which puts you on dialysis by 50. So I think that's important to keep in mind. Look, we're standing on the shoulders of giants. We're not the first drug to market. Others who have brought drugs to market have been able to improve that slope from losing 10% to losing a bit less. And that's fantastic.
You are actually saving months to years of kidney function and keeping someone off dialysis. So what's clinically meaningful in this disease has already been shown, which is great. We'd like to really transform what that target is and normalize it. And that's what's so exciting about the dataset that we pulled forward. So you can make these inferences and extend out, but you need to do the data. And I think that's where Vera has been very strong and rigorous in its clinical science. We know that others have moved quickly from initial proof of concept and open-label trials to registrational trials without doing careful dose finding. We think that incurs meaningful risk with respect to safety and getting the right therapeutic window.
I think it's hard to know what the effect size is until you've done a proper, fully powered phase III trial that's not only controlled but randomized and double-blinded and run for the full duration.
Questions from the audience? Maybe, Marshall, you could give us an update on your monthly formulation of atacicept, which I know you're working on. When could we learn more about what that profile looks like?
Sure. It could be later this year. We're running a dose range finding study, and I would put this in the category of creating new options in the Vera pipeline for the future. I do think that dosing frequency eventually in a market can become an important option for patients. So we've known that and invested in potential monthly.
That ongoing study should yield data for us around mid-year, and then we'll be looking at that data and determining the path forward in concert with regulatory authorities. So that's the plan. That's roughly the timing. And then VT-109, again, as I mentioned, has the opportunity to do even less frequent dosing. That's important in the leadership position that we have to continue to create options for patients.
What would you say to those people who say, "Vera has to have a monthly atacicept formulation out there ahead of or around if Vertex comes to market"?
Yeah, I guess I would say that the data doesn't support that view. The data that we have in hand, two-year data in open-label atacicept weekly with a prefilled syringe, 90% retention is a home run number. You got to pay attention to that if you think the patients aren't going to take it.
I do think that prescribing and maintaining on a drug does depend also on the dataset that you have forward. So that's not just the frequency, but also the overall dataset's important as well. So we do have this thesis. We're now seeing it come out in our own long-term data and retention, and we also see it in third-party surveys where this is not going to be a meaningful hurdle for us as we seek to bring this to patients.
Questions from the audience? Yeah, go ahead.
The KDIGO guidelines last time they were updated, there were no approved therapies. Can you talk about any changes you'd expect to happen now that we have approved therapies coming to market? Thank you.
Sure. Good question. What influences how these patients are managed among nephrologists?
There is an international guideline, so distinct from other areas, subspecialties of medicine. There's the KDIGO guidelines. It's not U.S.-specific, which is a little unusual relative to other fields. They were updated recently, as I reviewed on the slide, and they really indicate the dataset that I'm describing to you. So these are a bit anticipatory, but as you'd expect, they don't include any non-approved drugs. We would expect an update in these guidelines sometime after our approval. So the second half of this year is our general estimate of when that will happen. But there isn't a clear date coming from KDIGO. I'd also point out that not all nephrologists go to that as the singular source. Sources like UpToDate and other guidance documents that physicians use are also important and sometimes are updated faster. Yeah.
I have one more question, but additional questions from the audience? Final chance.
The final one for me, as we think about the ORIGIN Extend, and PIONEER updates in 2026, for Extend, any chance we may get that ahead of a PDUFA as part of a more medical education on durability and things like that? And then kind of the size and scope of what we should think about PIONEER?
Absolutely. I would just speak for our Chief Medical Officer in saying, at this stage, making sure that our fresh clinical data is presented at conferences is important. In the U.S., it's ERA, which is in the fourth quarter. In Europe, there tends to be a late May, early June meeting called ERA, which precedes our PDUFA date. So that is a possible target for some new data from our additional trials.
Yeah, I think you meant ASN in the US, right?
I did mean ASN. Sorry. I might have switched them.
ASN in the U.S., fourth quarter, ERA in Glasgow in June. Yeah.
All right. Thank you, Marshall.
Good. Thanks so much for the opportunity.