All right. Welcome everyone to Jefferies 2026 Global Healthcare Conference. My name is Roger Song, covering mid-cap biotech. It is my pleasure to have the fireside chat with our company, Akebia Therapeutics. We have the CFO, Erik, and the Chief Commercial Officer, Nick here. Welcome, gentlemen.
Great. Thank you.
Awesome. Erik, why not you kick off by giving us some overview of Akebia, where Akebia is, the state of R&D, what should we be looking in the coming months, year?
Yeah, that sounds great. Yeah, thanks Roger for having us. Thanks, Jefferies as well. Really excited to talk to you about Akebia Therapeutics today. We are a commercial stage company focused on kidney disease. We have two commercial products. Our key focus is a product called Vafseo, which is approved for the treatment of anemia caused by CKD for patients on dialysis. This is a significant market opportunity. We have a very differentiated product. We launched the product last year. Nick will be talking a whole lot more about that. Our other product is Auryxia. It's a phosphate binder. It's been on the market for some time. Lost IP exclusivity last year. Going forward, it will be a smaller component of the business. In the meantime, it continues to generate cash flow for us. We also have a really exciting advancing pipeline.
We announced back in December the establishment of our rare renal disease pipeline, which has two core assets. The first is called praliciguat, which is an sGC stimulator. We believe it has applicability in a number of kidney diseases. We're initially evaluating it in FSGS, and that's in a phase II trial that's underway. The other asset is what we refer to as AKB-097. This is a tissue-targeted complement inhibitor. We believe it has some really unique advantages in that it targets complement locally versus systemically, and we can talk more about that. We are evaluating AKB-097 initially in a rare renal basket trial, which we plan to start in the second half of this year. Finally, we have a program called AKB-9090, and this utilizes our internally developed HIF-PHI technology, and we're evaluating that in cardiac surgery-associated AKI or acute kidney injury.
That's in a phase I trial. Yeah, a whole lot of interesting things here for us to dig into.
Yeah, I like company in the commercial stage, but also you have a significant upside potential from the pipeline. I think that applies very well to Akebia. Why not we zoom in a little bit on the commercial franchise for Vafseo. We see the Vafseo last quarter back to the growth trajectory, and then we probably just focus on the last year, this year, because that's within the TDAPA period. In second quarter and then the rest of the year, that's still the second year of the TDAPA. How should we think about the growth trajectory and then also you have the TIW seems to be being adopted. How should we think about the growth trajectory compared to the last year and the first quarter?
Yeah, it's a great question. We're not going to get too specific around the dynamics for the rest of the year. We are pleased with where we are today. When we think about that first quarter, we saw both of our key indicators of success really starting to tick up. The first being number of physicians who are prescribing the product. We had roughly 1,028 physicians who had prescribed the product. That's a 28% increase, and that really reflects diversification in our prescriber base, moving away from USRC and adding in IRC and DCI and DaVita physicians. We also saw an increase in patients, a 60% increase in patients that are on Vafseo quarter-over-quarter. A significant uptake. Again, a lot of that driven by USRC, IRC and DCI, to a lesser extent, DaVita.
A majority of those 60% increase in patients came in the month of March.
Okay.
Really excited that those patients will be generating more months of revenue in the second quarter and moving forward. That was particularly strong as well. As we think about the rest of the year, really DaVita is the 200,000 patient gorilla in the marketplace. While we have access to DaVita, they're piloting and implementing TIW protocols across many of their clinics. It's a little slower than we'd want.
It's really going to be a second half story with DaVita, and we're looking forward to seeing that growth materialize.
Excellent. I think that two major dynamic here is one is how many patient or DO can access Vafseo. The other one is when they get access, how they're going to stay on treatment or do the refill. On the first part, you mentioned the second half of DaVita is the gorilla there, you want to ramp up there. How should we think about the cadence of the ramp up? I know you have some pilot program did with them, then they're happy. What is the ramp up compared to USRC, which is I think you're the biggest advocator there, then compared to other mid-size or smaller than DaVita?
Yeah, it's a great question. When we think about the mid-size USRC, IRC, DCI, their clinical advocacy extremely strong. They've taken a top-down approach. They're sending lists of patients across broad profile out to physicians, really asking physicians to consider Vafseo for those patients with anemia.
All patients who have reimbursement, not just high-dose ESA, not just older patients-
Everyone. Yeah
...all patients. We're really pleased with that. DaVita's taking a little bit of a different approach, a more cautious approach. They're taking the approach of we're going to make it available, and we're going to let the physicians decide which patients. It's coming out to be more like I'll call it a typical launch trajectory-
Right.
...where you have your early adopters that are going in, and then you have folks that are in the wait and see mode on the other side.
Right.
That DaVita piece, we expect to come. They continue to make progress on their TIW rollout.
Which is great. Again, a little later than we would've thought.
That second half growth story is there with DaVita.
Okay, got it. In terms of the refill dynamic, seems the TIW is really a lot of the deal want to follow, and then the compliance probably also will be slightly better. Tell us a little bit more about the refill rate here. I know it's still early days, right? A lot of patients still have that first refill, second. How you think about how we see this churn on those patient already used the Vafseo?
Yeah, I always start with dialysis for folks that may not be aware. In dialysis, you have an extremely high mortality rate. Roughly 20% of patients unfortunately die each year due to dialysis and CKD. That 20%, plus you add in folks that have the ability to get to a transplant, your underlying churn is about 2%-4% per month of churn, right? When we think about refill rates, et cetera, it's hard for us to glean from our data which are transplant patients, which happen to discontinue due to death, and which are discontinuing due to the product. With three times weekly dosing, we've seen a significant improvement in what we call first refill. We're somewhere between 85%-90% of folks are receiving a first refill of their prescription for Vafseo when treated TIW. Again, significant improvement over QD.
Happy with that. You mentioned it, Roger. Early days, when we look at that cohort of patients that are on their second refill, third refill, or fourth refill, a pretty small population we're monitoring closely, but it's not big enough yet to give us a conclusion around where that's heading. With the early data we're seeing, we're pleased by it. Our long-term goal is to be in that standard refill rate, somewhere in that kind of 80% range, 70%-80% range. The early data is encouraging in that regard. The other piece you had mentioned was folks coming back to and restarts.
Restarts, yeah.
Restarts are so important. At USRC, remember, they're the only ones that unfortunately suffered from the larger discontinuations because they were so early to the game with QD. IRC and DCI are TIW from the go.
Yeah.
They won't have this issue come through, but we are seeing roughly 20% of the patients that discontinued therapy at USRC are back on Vafseo today.
Mm-hmm. Got it. Yeah. Okay. That's encouraging. I just want to emphasize this. Patient discontinue or the compliance is not as high as you want to see, 70%-80%, is not because they don't like the drug, right? Majority of them are okay with the drug. They may not experience this type of the mechanism. Maybe the hemoglobin drop a little bit. They are worried. My point is, will this TIW and then education and experience will significantly improve to the rate you want to talk? What are the other tactics or strategy you can implement in order to get the compliance and the refill rate?
A number of things. Thank you for the question. Dialysis is a complex care center. You have physicians who are rounding at dialysis clinics. You have anemia managers. Sometimes those anemia managers are within the clinic. Sometimes they're actually centralized, and they're managing anemia with the staff in the clinic. If any member of the care team is uncertain or unaware or misinformed about Vafseo, that can fall down because it is really a team approach to managing anemia.
You had mentioned hemoglobin instability. We see through the mechanism of action of Vafseo that folks, a starting dose being 300 mg daily, they're going to need to titrate up. It's a titratable drug.
If people don't realize it's a titratable drug, they're going to see a drop in hemoglobin, they're going to say, "Oh, no. I need to immediately switch back."
Yeah.
Making sure those programs, whether it be peer-to-peer programs, our medical team educating around dosing, it's critical to success to make sure we get every member of that team.
Yeah. Got it. By the way, I do want to highlight one thing is the team. I think CEO John, then Nick, Erik, you have a significant experience in this renal space. I think you must have been Knowing this launch in renal space is not easy. I think the experience the team has, it probably gives people a lot of confidence that you are the right team to launch this.
Thank you.
Okay, good. We move beyond 2026 because that's the time point you will out of the TDAPA agreement. Two things people want to ask about the durability and then how the trajectory afterwards. Two things I think dynamic is: one is the pricing, two is what is the patient segment you can potentially penetrate, because right now its access is mostly the Medicare. You do have some other fee-for-service beyond the Medicare Advantage. Tell me maybe from the pricing, start from pricing, how we think about this.
Yeah, we've said a few times before that we're going to see a drop in pricing. We're going to be ESA-like pricing, which means our price is going to come down post-TDAPA. We have a variety of different size players out there. Large players, it'll be lower pricing than smaller players. I would use that ESA pricing-like system out there to gauge price as we move forward post-TDAPA.
Mm-hmm. Okay. Then in terms of access on the reimbursement side, you are open to everyone or some of them are a little bit more amenable to those novel mechanism?
Yeah. I actually think there's a tremendous upside in the patients available to us. Today, you have some Medicare Advantage plans covering it, some fee for service. Roughly between 50%-60% of patients have access. Post-TDAPA, every single patient has access.
Reimbursement is off the table for the most part. Even more encouraging is physicians don't have to go through a formulary exception process. There's an extra step in there that they have to do. They have to enter that patient in the system. They have to wait for approval. All the while, the physician is thinking about many things besides anemia, not having to go through a special process makes those patients more addressable than they are today.
When you think about patient populations, we are very, very lucky to have a broad label. It doesn't carve out particular patient types we go after. In those DOs that are top-down, again, as we said earlier, they're putting every patient on-
Yeah
...fits the label or is eligible. In the folks that are not top-down, and specifically DaVita, you may see them start with those most in need. Unstable hemoglobin.
High-dose ESA. Frankly, the product profile works really well in the QD population who is getting their dialysis at home.
An oral therapy for that population makes a lot of sense. You may see them picking and choosing there before they adopt more broadly for stable hemoglobin patients.
Just from the modeling perspective, we build pretty sophisticated model. I think your internal model probably even more so. How should we think about the different maybe peak or high penetration, which segment you think you have more confidence versus the other one may take a bit longer time?
Yeah, really the goal is from a clinical advocacy to get the clinical decision makers within the DO to help advocate for the product with their physicians. In those scenarios, it isn't based on patient population, it's based on do you have coverage, and do they believe in the clinical profile of the product broadly.
In those that are not doing that, I look forward to those patients that are most in need. Remember, high-dose ESA, hyporesponders, those with an unstable hemoglobin, they're a significant percentage of the population.
I don't think we're niching ourselves in any way by focusing on a particular segment.
Okay, good.
I think we were pleasantly surprised by that too, out of the gate. We expected maybe what you're getting at-
That's right. Yeah.
...certain niche populations would adopt first, but it was more broad than that.
Yeah, to echo Thank you, Erik, and then to echo Nick's earlier point, it's a broad label. It's not like you have any restriction on the certain population, but just in reality in which one may adopt earlier, but in the real world, actually, they are not discriminate any of the population. Okay, and then speaking to the label, you do have some other new clinical data supplement or even enrich the label, so VOICE and then the VOCAL. Walk us through why those data point will be meaningful and then how this will impact the launch, particularly after TDAPA .
No, thanks for the question. It's always been a key element of our strategy to continue to generate data, post-approval, to continue to demonstrate ways that the product is beneficial to patients. We have two studies we can talk about. I'll start with the VOCAL study. That's being conducted in DaVita clinics.
Correct.
It's TIW dosing, where we're looking at Vafseo versus the standard of care, evaluating safety and efficacy. Notably, there is a sub-study as part of this trial where we're looking at the characteristics of red blood cells. We know that red blood cell quality is important when you look at disease severity for anemia caused by CKD in patients. We'll be looking at a number of measurements there, which should be really interesting to dig into. That data will be available in the fourth quarter of this year. The other trial is the VOICE trial. This is a collaborative clinical trial we're conducting with U.S. Renal Care-
...led by Dr. Geoff Block there. 2,100 patients we fully enrolled in June, so we'll have data in the first quarter of 2027. Looking at two key things here, one is all-cause mortality, the other is hospitalization rates. I think the hospitalization rates component is particularly interesting. For those who aren't aware, when dialysis patients go to the hospital, the DOs share in a portion of those costs. If we can show that the hospitalization rates are lower with Vafseo, clearly there's an economic value to the DOs. Of course, most importantly, it's best for patients.
I think that data too is going to be confirmatory. When we think about the data that we have, at ADC in the winter, we presented this health economic data that showed a 7.7% reduction in hospitalization events, a 16% reduction in hospitalization days. One, good for patients to keep them out of the hospital. Two, when you look at the dollar savings per year, $3,700 per year savings. If you look at someone like DaVita that has 200,000 patients, even if only 50% are in these value-based care programs, that's 100,000 patients that could receive, to DaVita, $3,700 of value. That's a significant opportunity. The VOICE study confirmatory to that at USRC.
Excellent. All right. I think we talked a lot about the dynamics, clinical, and then the strategy. You on the Vafseo, you have another product, I think, Erik, you mentioned earlier, Auryxia. I always think this is like a Christmas gift to you because you have been out of the LOE for a long time.
Yeah.
It's a long Christmas. Seems now we have a few more generics coming. I know you're internally pretty conservative.
Yeah.
Same we are. It's like we don't want to make this. We don't assuming nothing going to enroll. How should we think about, my question is actually, how much the dependence you need for your current financial or balance sheet to rely on this Auryxia versus the Vafseo is catching on to get to generate the revenue?
Yeah, you're absolutely right about this being a gift. We have seen the Auryxia erosion coming for some time, and it did take longer to materialize than we had planned. We generated $180 million in Auryxia revenue last year, which was great.
Yeah.
Yeah. To the core of your question, we haven't guided on run rates specifically. We do remain conservative when we forecast Auryxia. We take into account those revenue forecasts when we budget our expenses from a forecasting perspective.
Got it. In terms of the budgeting, because we are exiting the TDAPA, and then you're moving to broad access, how do you think about the G&A or SG&A, and then any guidance around that in the near term and then longer term?
A couple of points. One, Auryxia does not require sales force effort. That is really working on its own. The sales and marketing activities are pretty much exclusively focused on Vafseo.
The good news is, at this point in the launch, we're past things like that initial spend for market awareness of the product, right? You continue to invest in these things, but that bolus is behind us. We can in fact be more targeted in the work that we do on the sales and marketing side at this point in the launch. We don't expect increases from an SG&A standpoint, and think that the infrastructure that is in place now is highly leverageable.
Okay. You don't think you need to significantly or meaningfully increase the infrastructure even outside of the TDAPA?
That's correct.
Okay. Got it. All right. Good. Maybe we use the rest of the time talk about your pipeline.
Yeah.
Which honestly, we think is very interesting, and then obviously it's not in the stock.
Yeah.
Too much in the investors expectation, but this can be a significant upside potential from here. You have quite a few, right?
Yeah.
Maybe just walk us through each one, and then what's the strategic priority there, and then maybe a couple point in the question afterwards.
Yeah. We believe there's a lot of potential here, too, and stepping back strategically, it just makes sense that we're in these areas given our expertise in kidney disease. Yeah, maybe I'll focus more on praliciguat and AKB-097 in the interest of time. Praliciguat, as I mentioned, it's an sGC stimulator, so by definition, it is stimulating soluble guanylate cyclase, which binds with nitric oxide, which creates cGMP, which increases vasodilation, blood flow, reduces inflammation, reduces fibrosis. We know that the nitric oxide pathway is dysregulated in a range of kidney diseases, including FSGS. Our first trial with this drug is a phase II randomized, placebo-controlled, double-blind trial evaluating praliciguat in FSGS. We're planning to enroll up to 60 patients. We'll look at uPCR levels. There will be a crossover. Placebo patients will cross over to drug after 24 weeks.
Once that final placebo patient has crossed over, we'll be able to unblind the data. Yeah. FSGS, about 40,000 patients, high unmet need, characterized by scarring of the filtering units within the kidney. Yeah, the enrollment has been going well. We're really pleased with it, and look forward to continuing to update the Street as we progress.
Excellent. Maybe just on the expectation, praliciguat, you are running a phase II. We do know FSGS is active out there.
Yeah.
How do you think about the profile you want to achieve-
Yeah.
...before you can move forward, and then how you think the competitive landscape, and then what you want to be landed at?
Yeah. We'd like to see a statistically significant difference in terms of the reduction in uPCR levels between us and the treated group and the placebo group. Also like to see an increased proportion of partial and complete responders versus placebo. Yeah. There are other entrants in this space. FSGS is a heterogeneous disease. We think there's going to be room for multiple treatment options. From an MOA perspective, we're the only sGC stimulator attacking FSGS at this time.
Okay, good. Yeah, look forward to that data. Data will be sometime next year?
We haven't guided specifically on the timing of that data. We'd like to see how the enrollment continues to progress. We'll be able to get more granular on that timing.
Excellent. Okay, good. You did acquire a complement inhibitor, and then which is differentiated in terms of the targeting, the tissue targeted. Walk us through the mechanism and then why it's tissue targeted, and then we know we have a lot of complement inhibitor out there. Why-
Yeah.
...do you think this can be very differentiated here?
Yeah. We're really excited about this asset. As you mentioned, it's a differentiated complement inhibitor in that it targets the complement system locally versus systemically. Traditional approaches are more systemic in their nature. Just by way of background, there are a number of complement-mediated diseases, many of which are in the kidney. What happens here is the complement system becomes dysregulated. The complement system is part of the immune system. The way that the complement inhibitors work is by binding to the complement proteins and halting that cascade of cell destruction. AKB-097 is a fusion protein. It binds with high affinity to C3d. We know that C3d is present at sites of complement activation, and so that is part of the MOA that allows for that targeting.
Patients are going to be on these drugs for a long time, the less that you can be stressing the immune system, the better. We think there's also advantages from a convenience standpoint as well when we look at how we think about dosing of this product down the line. The trial that we are evaluating AKB-097 in, as I mentioned, is a basket trial.
We plan to start that in the second half of the year. We're looking at three rare renal diseases initially, C3G, lupus nephritis, and IgA nephropathy.
We'll be looking at proteinuria levels as you might expect, and we'll have data from that trial in 2027.
Okay. It is a basket trial, I think they all belong to rare kidney disease and some of them are a little bit more than the others.
Yeah.
How should we think about the data you're going to present in 2027, and what you want to achieve, and then how you're going to select the indication from there if you want to select one-
Yeah.
...from there.
Yeah. We'll be looking for a clinically meaningful reduction in proteinuria.
We're looking at some pretty interesting biomarkers. One is C5b-9, that measures the levels of complement activity within the kidney.
Yes.
That'll help us assess how the drug's performing there. From a market standpoint, any and all of these are very interesting and attractive places for us to be.
Some more competitive than others. We see an unmet need, kind of across the board in certainly places where we can play. I think one of the benefits of this type of trial design is that it does give you that flexibility, right, to, should you see interesting data from a particular indication, move forward, right? You don't need to wait for that trial to fully be completed before you go to the next move.
Okay. The expectation is that when you have sufficient data from the basket trial for one or more indication, then you can decide to the next step.
That's right.
okay.
We have that optionality.
Yeah, okay.
Which I think is great.
Got it. All right, good. Maybe just one more pipeline throwing in the ring here.
Yeah.
AKB-9090, so what does that really try to address? I asked earlier, so how are you going to prioritize all of those pipelines?
Yeah.
Seems you want to go in parallel, and then this is the number three or the third lead kind of pipeline.
Yeah. This one's a little bit earlier. AKB-9090, as mentioned, HIF-PHI. Acute kidney injury, if you look at the number of cardiac surgery-related procedures over the course of a year in the U.S., there's about 400,000 of those. Our research indicates that up to 30% of those cases involve AKI. This is a huge problem. There's no drug specifically approved for cardiac surgery-associated AKI. The phase I, as you'd imagine, is more focused on safety. SAD, MAD, PK, PD. We will be able to evaluate, I think to your question, EPO, right?
We can see whether there's target engagement in the kidney. Is HIF being stabilized and EPO being produced? Again, that's in a phase I. A bit earlier, but we'll have that top-line data in the first quarter of next year.
Excellent. Last 30 seconds, Erik, if you want to wrap this up and then maybe financial-wise, guidance, anything you want to highlight?
Yeah. From a financial perspective, as we guided in our Q1 earnings, based on our operating plan, we have at least two years of cash one way. In terms of key catalysts and milestones over the next 12- 18 months, there's a fair amount.
Starting with Vafseo, we'll of course have the continued quarterly revenue updates on the product. We have the VOCAL data in Q4 of this year we mentioned. Then the VOICE data in Q1 of next year. Turning over to the pipeline, for AKB-097, starting that basket trial in the second half of this year with initial data to come in 2027. As we discussed for praliciguat, as we continue to advance enrollment, we'll have more granular timing guidance on that data. Rounding out the pipeline for AKB-9090, we'll have that phase I top-line data on the AKI program in the first quarter of next year.
Excellent. Thank you, Erik. Thank you, Nick. Thank you everyone listening and watching.
Great. Thank you.