Okay, thank you for joining us for our fireside chat with Ardelyx. We have with us here today from management, we have Justin Renz, CFO, and Caitlin Lowie from IR. So to begin, for those that are not as familiar with Ardelyx, can you give us a brief overview of your story and what your vision is over the next several years for the company?
Great. Thank you. Good morning, and thank you for having us at this beautiful healthcare conference. As Louise said, I'm Justin Renz, Chief Financial and Operations Officer. Ardelyx is a company that was founded with the vision of being, having first-in-class medicines for novel therapies and unmet medical needs. And so, we've had the privilege of now doing this twice, with two approved therapies, one called IBSRELA, which is approved for irritable bowel syndrome with constipation, and XPHOZAH, which is for end-stage renal disease patients on dialysis for phosphate-lowering therapy. IBSRELA was approved in 2019 . We launched it in 2022 , and that is been doing very well, and we'll get into that. And XPHOZAH was approved last November, and we've been selling that this year.
Sales are going very well for that, and we'll get into the guidance and range and things of that nature. We have some early-stage compounds that we're excited about, and so we look forward to sharing that information with you, probably early part of next year. And at the same time, because of our ability to, develop, commercialize, these assets, we believe that we will hopefully be the partner of choice. So we've begun explorations into partnering and in-licensing to try to build out our pipeline as we seek to grow, you know, a pharmaceutical company, if you will, that has, again, as a general core, novel unmet medicines in areas of unmet need with, you know, again, obviously, long IP and build a business.
Okay, great. So you have two approved products. Can you give us an overview on each product and your commercialization strategy behind both of them?
Sure. So IBSRELA is for IBS-C, so that's constipation. That market was really created from a pharmaceutical perspective by the work done by Ironwood and AbbVie. And so we are second-line therapy, so our drug is approved in adults. So again, it's only for 18 years and older, and it is an Na+/ H+ or a sodium hydrogen exchanger, so Na+/ H+ inhibitor. And so our drug works. It quite effectively. It's one small pill twice a day, 50mg . We did $80 million in revenue in 2023. This year, we've guided that we will do between $140 million and $150 million in net revenue sales. We're very encouraged about that. We've affirmed that guidance now several times. So that's going quite well, and we're optimistic.
We're sharing our Q3 results, you know, late in October. XPHOZAH is a phosphate absorption inhibitor, that is, again, for end-stage renal disease patients on dialysis. That was approved again last year. We've been selling that quite well. In our second full quarter of launch, Q2 of this year, we did over $37 million in revenue. We're not at the stage yet to give guidance yet, but we're very pleased with that. Clearly, there's unmet need. Same general idea, one small pill twice a day, primary dosage strength is 30mg , and it works quite well. So it's indicated for patients, either, they're, again, end-stage renal disease patients on dialysis to help them with lowering phosphate.
Many of them are on binders, so our drug can work for you, and it's indicated either if the binder you can't tolerate it or it's not working for you, or in addition to the binder to help supplement in that lowering of the phosphate in your blood.
Okay.
I think if I would just add to your question about our commercialization strategy, because it's quite unique in that both of our products are able to deploy a similar strategy. They're both first-in-class products that are in markets that have established patient bases, but only a single mechanism available to them. So we're able to identify patients who are not properly treated on the, you know, current standard of care or the other class. They now have a new option where previously they had no other therapeutic options. So it really allows us to take a targeted approach, going to physicians that are high prescribers, that see these patients every single day and allow us to capture share within this identified patient population. In both cases, there's recognized to be high unmet need among patients.
We have a very targeted approach to high-prescribing physicians, going after patients who need a new option, and this first-in-class medicine is a great option for them.
We expanded our footprint, so in the course of 2024 , we've grown our IBSRELA sales force to up to 124 reps in the lower 48, and XPHOZAH, we have 60 area business directors. Again, to Caitlin's point, we're a very specific target approach.
Okay, great. So maybe we'll dig a little bit more into each product. So for IBSRELA, can you talk about your peak sales projection, how you plan to get there? And then there's been some quarterly fluctuation in gross-to-net, so is that going to stabilize over time?
Yes, great. Excellent question. We'll do the first one is on peak sales. So we announced earlier this year that we believe that we can achieve peak sales of over $1 billion prior to losing patent exclusivity. And we get there because there's, d epending on how you keep score, between 5 and 6 million prescriptions written each year for IBS-C indicated drugs. So, if we achieve over 10% market share of that at our current price point, which is $1,760 per month WAC, with our gross-to-net, that math will get us to over $1 billion.
Our g eneral go-to-market strategy, again, as Caitlin mentioned, is a very targeted approach, and we have a dedicated Ardelyx Assist program to help the patient get the prior authorization approved that they need, because, for IBS-C, they've failed or no longer can take a prior therapy, such as LINZESS or Trulance or something to that effect. So, on our gross-to-net front, we will help you if the patient's commercial copay, commercial healthcare plan will allow, we will buy down, if you will, point of sale, their copay. And so in the first quarter of the calendar year, many folks' plans reset, and so your deductible is higher, perhaps in January, than what it would be later in the year.
So what we've seen in our two years so far is that the first quarter has our most unfavourable gross to net because the commercial copay aspect, where we will pay for you at the point of sale, your copay, the claims are higher, and the amount per claim is higher. So, for example, in the first quarter of this year, our gross to net deduction was around 33.5%. It improved to the high 29% range in Q2, and we expect to be slightly better the rest of the year. For our historical reference in 2023, our gross to net was 33.8% or so in Q1 of 2023 and declined to around 26% in Q4.
Okay, great. XPHOZAH, a controversial product that you have. Your sales have been great, and I'm wondering if you need any reimbursement from the government agencies in order to make this product work for you.
Sure. So, the end-stage renal disease or oral-only medicines currently are slated to go into what's called the capitated payment system, or colloquially, the bundle, on January first of 2025. And so, we, as you would hope and expect, have Medicare, Medicaid, commercial, VA, TRICARE, and many other different types of patients with different payment plans and mechanisms. Medicare is, of course, an important part of our business. XPHOZAH is an excellent drug. It's already proven to be quite helpful for patients, and that's, again, I think the reason why we've had such success in the first couple quarters of launch, because there's clear unmet need, and our drug is there. Our philosophy for both IBSRELA and XPHOZAH is to make our drug available for as many patients as possible.
So we will do our part through Ardelyx Assist to help adjudicate the patient's individual situation and make the drug available to them. So should Medicare Part D pharmacy benefit unfortunately be transitioned into Medicare Part B, as in boy, on January first, because our different ways of trying to get this alleviated for Medicare Part D patients, which we'll get to in a minute, happens, there still is a meaningful business outside of Medicare patients. We, of course, will do everything we can to help those patients with access, should this come to be next year, because there is still a meaningful business amongst the commercial payers, the Medicaid payers, and other.
Okay, what percentage of the payers are outside of Medicare?
Yeah, historically, it's been around 60% that are Medicare, so plus or minus, you know, 5%, and commercial, VA, TRICARE, and Medicaid is somewhere around 40%, plus or minus 5%. So that's kind of our general metrics that we've been sticking to, kind of a 60/40 Medicare, non-Medicare.
Okay. And any, could you venture at least to give us some sort of sales range outside of Medicare, what that could be for you?
Not yet. We're, as you might imagine, doing a lot of planning and modelling and forecasting work. We're just not quite ready to share that yet.
Okay. And also, there's been some recent movement on the legislative front. Can you talk about those and how that might impact your, your case?
Yes, absolutely, so last July of 2023, Buddy Carter initiated Bill H.R. 5074, the Kidney Patient Act, and we have gotten tremendous bicameral and bipartisan support, so we currently have 24 co-sponsors in the House and several co-sponsors in the Senate under Marsha Blackburn's leadership from Tennessee, and so that currently is in Congress. We have the support from both parties, as I mentioned. The Ways and Means and Energy and Commerce, the two subcommittees in the House that look over healthcare packages, both approved them, so it essentially got out of committee, and so it's essentially on the floor of the House, if you will, we are hopeful that there is. It just needs a vehicle to pass, so we have the appropriate support from both parties.
It is bipartisan, which is fantastic, and we think it's the right thing for patients, again, to make this available for sure for Medicare patients besides commercial, TRICARE, VA, and Medicaid, and so we need a healthcare package, an omnibus package, a minibus package, maybe there'll be a lame duck session. Continuing resolution, it's hard to say because there's a lot that goes in and out, and so we just need to find the appropriate vehicle, but our bill has been one of the most researched and read bills available, so we believe we've done everything we can. We have the appropriate levels of support. It just needs the inertia of Congress to act and pass the bill.
Okay. So I assume nothing will be completed before everything gets enacted on the Medicare front, right? So will there be any sort of headwind to sales as we look out into next year?
Yeah, great question. So again, there is unmet need for sure in this patient population. So our team is doing everything they can to initiate new starts and, again, refills. So Q3, again, we'll report in late October. You know, so far so good. We're, again, there's such a large patient population. We have not hit that headwind yet. It is possible, of course, by the fourth quarter, should we get close, it appears this, there may be some hesitancy on the part of the nephrologist.
Okay.
But part of our efforts, though, are to really try to help understand that by you're not applying for TDAPA and remaining outside of the capitated system, that we can continue to make XPHOZAH available for patients. So as much as possible, continuing to emphasize that the product will be available and that there shouldn't be that hesitation. So, we recognize that there is some uncertainty and, you know, we have to continue to communicate, but understanding now that we're trying to remove those barriers as much as possible is a very aggressive activity of our field-based team.
Okay. So given the uncertainty, would you still consider giving financial guidance for XPHOZAH in 2025? Is that on the table still?
In early 2025, should all these efforts come and we have resolution favourably or unfavourably, I think it's in our best interest to help the investment community in early 2025 when we know the game that we're playing in, so to speak.
Okay. And you've also talked about other regulatory or commercial strategies to get beyond this Medicare issue that you're having. When will we hear more about that? Is that still something that you think about? And what could those be? What are some,
Sure.
without disclosing exactly?
Yeah. Well, again, there is litigation that is underway. So in July, we did challenge the CMS that this overreaches their authority to include oral-only therapies for these patients. If you think about the regulations around dialysis, as currently written, it's for medicines that are furnished during dialysis. Well, our drug is not at all furnished during dialysis. In fact, it's contraindicated because if when you're dialyzed, you would not want to take tenapanor, which is the generic name, of course, for XPHOZAH, before you're dialyzed, in case you have to, you know, need to use the restroom or not, you'd have to get unplugged and things of that nature. So it is truly contraindicated for days of dialysis. You take it after dialysis, before your first meal, and then again at dinner. And so it truly shouldn't be part of that system.
So that is our argument to CMS, that it should not be included in that. So that litigation is up front. It's been assigned to the district court. We filed in the district court, Washington, D.C., and we wait to see how this plays out. It's currently progressing as we expected. It's an orderly, you know, slow process, as you might imagine, with the government. Well, again, we're hopeful that we need to take action by the end of the year because January first is coming.
Okay, great. And you have a program called Ardelyx Assist. Can you tell us more about that and how that's helping the uptake and adoption of your products?
Fantastic question. So we're very proud of Ardelyx Assist. It's our homegrown system where we do everything we can to help triangulate around the physician, the patient, their coverage, and how we can best get the drug to them, adjudicating all their information. And so we basically ask the nephrologist or the gastroenterologist, the prescriber, to reach out to Ardelyx Assist, and with our electronic network system, we can see the coverages they have. And then both of our drugs are primarily specialty pharmacy, so it's mail order to the person's house. So we'll help adjudicate their coverage and make the drug available to them. Should they be eligible, we have a patient assistance program where. Because we believe every patient deserves our drug, and so if they meet the criteria, we will give it to them under our patient assistance program.
I think to your question about how that's helping to drive adoption, it allows us to connect all the pieces together so that we don't lose patients within that prior authorization and distribution funnel. We help to ensure that the physician knows they have to complete the prior authorization and will help resubmit it if it needs to be done. It's automatically funnelling a patient into a system to be, you know, considered for our affordability and access program, and then it automatically ships the product to them. It's really our way of controlling the patient experience so that we can prevent as much loss of patient. Once a physician has determined that a patient is clinically, would be clinically benefited by our product, we want to convert them into a full prescription.
As you might imagine, whether you're a nephrologist or a gastroenterologist with a new therapy, and again, these are patients that have unmet need, we want to make the drug available to them. It'd be very frustrating for the prescriber to write the prescription and then the patient not get it. That is, and it only has to happen a few times, and what the prescriber might say, "This drug is too hard to get for my patient. I got to do something else," and so it's so important for us to do everything we can, as Caitlin said, to make sure we can do everything in our powers to help the patient get the medicine they deserve.
And I think recognizing that our products are, as Justin mentioned, second line, they're only accessible after a patient has not been fully, you know, serviced on another product. It made it more important for us to invest there. You know, we, you know, as Justin spoke to our gross to net, we have a very favourable gross to net deduction, so we're able to invest in this other service that we believe provides a more direct benefit to the patient and the physician.
Yeah.
Great. Can I ask you about your international expansion strategy? I know you already have several partnerships in place. Some of those are fully monetized, some of them are not yet. So can you walk through those and where, what your economics are and what remains, that you can collect?
Sure. We'll go west to east.
Okay.
We have partnered Knight for IBS-C in Canada. They are doing well. We get a royalty check from them on a quarterly basis. They have not pursued hyperphosphatemia in Canada. We're partnered with Fosun in China for both indications. They have filed for hyperphosphatemia. That is due to be ruled upon, if you will, hopefully approved by the end of the year. So we've guided that we expect an answer for hyperphosphatemia by the end of the year. We have commercial milestones as well as sales milestones, and then we would get a royalty stream of double digits in nature or higher, depending on how well they sell. So we're optimistic about that. We have partnered for hyperphosphatemia in Japan with Kyowa Kirin Corp.
A couple of years ago, as part of our financing structure and planning, and we monetized the preponderance of the commercial milestones as well as the royalties. We do get a small royalty check, and that's. I think that's in the public domain, but we then monetize that tail with HealthCare Royalty Partners. So we, you know, if you want to do an aggregate, we essentially monetize the Japanese hyperphosphatemia market for us for $85 million, and now that money flows through essentially from Kyowa Kirin into HealthCare Royalty Partners. It is unpartnered for IBS-C in Japan, so that is an opportunity for us for sure. So LINZESS, the second biggest market after the United States, per my understanding, is Japan.
So there's true opportunity for IBS-C in Japan, and so we've been looking into that for the past year, trying to find the right partner and the right economics. And Europe has definitely an opportunity, certainly for hyperphosphatemia, and so we've been beginning those discussions with partnering opportunities in Europe.
When do you think we'll hear something on Europe, and how could you size the potential possibility for you there?
Clearly smaller than the United States in terms of size. In terms of timing, I think it's a corporate goal that we'd like to do something, you know, I can't say when, so it has to make sense, right? We don't ever want to be a company that does a deal just to do a deal. So with the right partner, with the right infrastructure, and the right time, we will do that.
I think we're still also understanding what are the clinical needs that would be associated with it. So I could imagine we may even be able to talk about some of the, you know, preparation steps as we think about expansion as well. So, you know, when that could be, we can't really say and what the markets look like, but we recognize that there's, you know, certainly patients that are out there. We have both products indicated and approved in the US with very strong labels, which are kind of helping as we go to have those discussions. So it's certainly a priority and an area of opportunity.
Okay, great. I wanted to open it up to the audience, see if anybody had any questions for the company. Sarah?
You talked about significant barriers, where you, like, emphasized that you want your product to be available and you recognize there's some uncertainty. So those barriers, what are they? How we overcome them, and is that where the Ardelyx Assist comes in, or is there something else involved in there?
Is that related to XPHOZAH? Or what?
So I think as we think about some of the barriers, I mean, this, you know, decision, you know, as we approach January first, whether or not, you know, XPHOZAH will lose Medicare coverage when it moves from Part D to Part B. You know, that is certainly a situation where we have as many opportunities as we can. We've got the litigation, we've got the legislation that will hopefully ensure that Medicare patients continue to have access. You know, our choice not to apply for TDAPA and to keep XPHOZAH out of that capitated system really allowed us to protect, especially the non-Medicare segment that should not be affected by the bundle, but given the dynamics of that environment, would have been. We really believe and call on Congress to pass the legislation to protect all patients, but also Medicare patients, especially.
Hopefully, the litigation will help, but ultimately, our decision to continue to make XPHOZAH available outside of the bundle is how we're able to create the opportunities commercially. We haven't gone into a tremendous amount of detail, and we'll hopefully be able to do that in the future. But, you know, it's a first-in-class drug. It's bringing benefits to patients. So we are hyper-focused on creating opportunities to make it accessible. And to your point about our Ardelyx Assist, absolutely, that is a program that we have in place that for patients who are facing affordability or access challenges, but they're deemed clinically meaningful for the product, then, you know, they can apply through that program, and we can ensure the patients continue to have access.
Yeah. The key will be to keeping the nephrologist or the prescriber as the focal point. And so our team members will be focused on those high-prescribing nephrologists or other, similar APP types, that we will give them the attention they need and tell them: "Hey, reach out to our Ardelyx Assist for your patients that still need help with their phosphate levels, and we will do everything we can to make our drug available to them.
Okay. What's your capital allocation strategy, and how do you prioritize R&D and then potentially business development to expand your pipeline?
Sure. So we finished the first quarter with around $203 million in cash, and we finished the second quarter with approximately $186 million in cash. And so that $17 million was also very similar to our GAAP net loss. And why I say that is because our investment in building our supply chain was around $12 to 13 million in Q2, and our operating loss, if you will, was around $4 million, and that also held true. Our non-GAAP, if you will, loss in Q2 was around $4 to 5 million because we had $12 million of non-cash charges. So my point is our operating burn in Q2 was around $4 to 5 million. Very small. We're getting very close to break even, so our capital needs for the day-to-day business are low.
So our capital is gonna be in looking to build a great company. So we've begun in some early development with preclinical assets that we have that we're excited to share about next year. We're gonna look to in-license. So again, we are being, again, very disciplined. We're never gonna do a deal just to do a deal, but there are a number of very good clinical candidates out there that we'd like to add and grow to our pipeline. We still have, of course, potential to expand the label on XPHOZAH and IBSRELA for either going into pediatric population or, you know, to the extent that we can add complementary assets, we will. The goal is to build a good company. We may need additional capital to do a larger deal, which we would. If we could, I think our access to capital is good.
But again, in general, we're well-resourced to do what we need to do.
Okay. And historically, you've had a few products in your pipeline. You haven't talked about them in a while. Are they still products that you consider developing, or are you moving on?
We had two specific clinical-stage assets that, of course, we still have all the rights to. We took a really careful, critical eye to it because, again, to my most recent point, we don't want to do something just to do something. So we try to evaluate the market opportunity, what the indication will look like, you know, when the approval comes in, and what the competing landscape is, what reimbursement might be, how it will play out, and for both of those two products, we didn't think it was a good use of shareholder resources and internal resources to further develop them because we didn't think the commercial opportunity at the time would justify the effort.
Okay, great. Well, we're almost out of time here, so I wanted to ask both you and Caitlin, is there anything that I didn't ask you about that you think is important to highlight to investors?
I mean, I think where we think about the future of the business is the balanced portfolio. So, you know, we do get a lot of questions related to XPHOZAH, but on the other side, IBSRELA is doing incredibly well. You know, the reason why we expanded the sales force is because we recognize that this can be a billion-dollar drug, and we're going to achieve that because of how responsive the market is, how well it's performing with patients. And so our ability to continue to nurture and grow that business is really kind of the base of our business. It's very strong, and it's continuing to grow. We have a lot of confidence in that. You know, as we approach, you know, XPHOZAH, there is a decision, and there will be, you know, a different environment, you know, in January.
However, that product, there will be a business there, no matter what, and as we define kind of what that means and understand the different scenarios that are playing out, that will come to life, but you know, we're well-financed. We have a very strong commercial organization that can bring in new assets that we can move through the entire, you know, entity that from, you know, development through approval, so we have the ability to grow the business. We have a strong balance sheet that will help us both grow expansion of our current products, as well as bring in other assets, so I think that we really want to emphasize that the core business and the portfolio that we have is very rich, and you know, we can grow from there as we move through some of these uncertainties in the near future. Justin?
I think Caitlin said it best, so yes, I think IBSRELA is an excellent first product we have. That is our anchor, and.