Thanks, everybody, for coming to our Leerink Global Healthcare Conference. My name is Roanna Ruiz, and I'm one of the Senior Biotech Analysts here at Leerink. It's my pleasure to introduce the CEO of CorMedix, Joe Todisco. Thanks for joining us.
Thank you, Roanna.
Yeah, great to have you. I think the plan for today, I'll start with some bigger picture questions and then drill down in more specifics. Just for investors who may be new or revisiting the CorMedix story, could you summarize the company's mission and differentiation in infection prevention, and particularly as it relates to your lead product, DefenCath?
Sure, thanks. I guess I'll just start by describing a little bit about who we are. We're currently a small-cap, now commercial-stage biotech company. We just launched our lead drug, DefenCath, in the middle of last year. The core technology that underlies our main product is our taurolidine platform, which is a proprietary new chemical entity, amino acid derivative that has broad-spectrum antimicrobial capabilities. It's got broad activity against Gram-positive and Gram-negative bacteria, as well as fungus. It is not an antibiotic. It's definitively a unique molecule. DefenCath itself is a combination of our molecule taurolidine and heparin sodium. DefenCath is what's called a catheter lock solution. It sits in a catheter in between the time that a catheter is being accessed. Many patients with different diseases have to wear a catheter for vascular access.
The initial indication for our drug is in reduction of catheter-related bloodstream infections in catheterized hemodialysis patients. Hemodialysis patients, about 80% of them start with a catheter for vascular access. About 25% of them remain on a catheter long-term for vascular access. They have incredibly high infection rates, upward of 25% to a third of patients will encounter a bloodstream infection in the HD space. Most of those infections happen fairly quickly. About half of patients will get an infection the first 90 days they have a catheter instilled. They have incredibly high mortality rates. These infections are absolutely a critical unmet medical need. They happen often. They happen fast. They kill at an alarming rate. The way that DefenCath works, it sits in the catheter in between dialysis sessions. Typical hemodialysis patient receives dialysis three times a week, 52 weeks a year.
Our product works by inhibiting biofilm buildup in the catheter, which has led to, in clinical trials, we demonstrated a 71% reduction in infections compared to locking with heparin alone, which is the standard of care.
Yep, got it. That's really helpful. I think lately you've recently achieved break-even fiscal status in 2024. I was curious, could you talk about this financial milestone for the company? How does that tee up other priorities going into 2025?
I think us getting to break even before the end of the first year of launch was an incredible milestone for the company, something we're very proud of. I know that we had talked quite a lot about getting to break even on a run rate basis. We actually achieved EBITDA profitability for the full fourth quarter, which is essentially only six months of our full launch, which really kicked in in July of this year. Now, in the outpatient hemodialysis space, there are two settings of care where our drug is currently being utilized. One is in outpatient hemodialysis. The other is in inpatient hospitals. The thrust of the market opportunity is on the outpatient side. About 90% of the market opportunity is outpatient. You have a large amount of consolidation amongst those customers.
There are five dialysis operators representing about 90% of the market, two large guys, DaVita and Fresenius, and then three smaller guys, U.S. Renal Care, DCI, and Satellite Healthcare. We have talked quite a lot about the last year. U.S. Renal Care was our initial anchor customer. They have been kind of the driving force behind our initial growth. What we need to focus on now is onboarding new customers and then continuing to grow patients with existing customers.
Yep, got it. I know you recently gave cash position and OpEx guidance. Maybe could you just give us a highlight about that and how you balance investing across just executing on the DefenCath launch and just thinking about pipeline opportunities down the road?
Sure. We have been a fairly SG&A-light organization even through launch. We were running only at about $15-$16 million a quarter of OpEx over the past year. We have given guidance for this year that is just a tick above that, probably about $17-$19 million a quarter throughout the year. Most of that delta, that increase, is for investment in new clinical initiatives. We have announced essentially four clinical initiatives, one of which is already underway, which is our real-world evidence study with U.S. Renal Care. We see that as an important value driver for us long-term, the data that we want to capture in a real-world setting beyond just infection rate. We want to show that we have an impact on hospitalizations, that we have an impact on antibiotic use, on other downstream sequelae from CRBSI.
That's data that will be important for us as we make our case to Medicare Advantage plans down the road. Now, from a broadening the label standpoint, we've announced we'll be kicking off a clinical initiative in total parenteral nutrition, showing that the product works for reducing bloodstream infections in that patient population. We expect first patient in in the first half of this year, likely in the second quarter. Pediatric study, which is an FDA requirement, is a much smaller study, only about 35 patients, should also kick off later this year. Lastly, we've stood up an expanded access platform for patients that are at risk for infection but are in some smaller niche disease states where we're not going to be funding clinical expansion.
Yep, got it. I did want to take a step back a little bit. For some of the audience that might be newer to DefenCath, could you just remind us, is it a device? Is it a therapy? How is it exactly used in patients?
No, thank you. We get that question. We get that question quite often. Because in other parts of the world, the catheter lock solution is considered a medical device. Outside in Western Europe, historically, that's been the case. The FDA considers it to be a drug product, and taurolidine would need to be a new chemical entity. It is absolutely classified as a therapeutic agent. In the U.S., we had to undergo a full NDA development program and submission process.
Great. Sounds good. Just coming off of your preliminary Q4 announcement, it looks like there's a lot of good strength in DefenCath revenues. Could you talk about the adoption there? I think it's mostly the outpatient setting you said earlier. What are some of the factors that fueled this growth?
Sure. We actually pre-announced our fourth quarter earnings back in January. I'm going to be a little bit careful and not kind of get ahead of anything that wasn't in our pre-announcement because we're going to be reporting earnings in the next couple of weeks. You're right. A lot of the initial uptake has been in the outpatient segment, much of it driven by our anchor customer, which is U.S. Renal Care, who kind of came on board in early July. We started to see adoption from two of the other mid-sized players, DCI and ARC, in November and kind of pushed us through the back part of the year. Really pleased with where we ended up at the end of the year. One of the things that I guess I wasn't pleased with was the uptake on the inpatient side.
We kind of launched middle of the year with a field team that was combined and calling on both therapeutic segments, using the same team to cover both outpatient renal as well as inpatient hospitals. That team was largely experienced in the outpatient renal setting. As we moved through the third quarter, we took a couple of learnings, one of which was we did not need as large a field presence to drive uptake on the outpatient side because much of it is done off contract and done at the decisions made at the parent level. We shrunk the outpatient team down a little bit and combined it with our market access unit and have decided to stand up a completely independent inpatient team. We have partnered with Syneos Health on staffing that organization.
There'll be dedicated, essentially, CorMedix employees that we're utilizing Syneos for some operations logistics support. There'll be dedicated CorMedix employees, with the goal of standing that team up by the end of the first quarter.
Got it. Great. In terms of sustainability of demand, are there any sort of qualitative comments you can make in terms of what you're hoping to see in 2025?
I think the big variable, which everyone's going to want an update on on our next earnings call, is where are we at with we've announced previously that we signed one of the two LDOs to a contract. That LDO's target was to put it in about 10% of their catheterized patients. We'll be updating investors on where we stand with that customer as well as with the other that we don't have or didn't announce a contract with when we get to our earnings call in a few weeks. That's the largest, I guess, variable in our value prop, so to speak, to the extent that we do materially more, materially less than what's forecasted. That's what everyone wants to understand.
Yep, got it. Just to double-click on, you mentioned just leading more into the inpatient hospital setting. What are some of the goals of that expanded field rep team?
I think first, I just want to say from an inpatient standpoint, while it's a smaller market opportunity in terms of it might only be 10% of the market volume for hemodialysis, first, we think there's better price durability in the hospital segment. The dollar value might be a little bit higher. We do think it's important from a longer-term standpoint. Certainly, as we talk about TPN, a third of TPN is used in the inpatient setting. Beyond TPN, there's going to be a number of other, we think, opportunities for spillover on a long-term basis. Having a robust and credible inpatient team is vital to kind of the future, I view, the future uptake of DefenCath.
Yep, got it. I think there's a question in the audience.
Just on the fact that the product's a drug as opposed to being a device, how does that change your ability to defend your IP and put moats around your product relative to other catheter products in the marketplace?
I wouldn't say it changes the landscape for having IP. What it creates, though, when you're a drug product, there is a clearly defined IP process for patent challenges. You have this ANDA, they call it a paragraph four certification. Our product first has 10 years of market exclusivity. A generic can't be approved for a decade. We also have an Orange Book listed formulation patent that goes to 2042. There are statutory procedures by a generic filer that they have to fulfill in order to challenge that patent. They can't file an ANDA until a certain point in time. It has to be one year prior to the NCE exclusivity date, which would be year five. Then there's a litigation process.
Now, we feel pretty good about the validity of our patent claims and the difficulty that a generic would have in designing around that patent. We feel good about the IP protection that we have. I think it affords investors a lot of clarity and visibility. As opposed to on the device side, I do not believe that that type of pathway exists.
Thank you.
You're welcome.
Great. I'll keep rolling if there are no other questions. I think you mentioned being able to having some partnerships, accessing the VA and federal facilities, things like that. Can you talk about the eligible patient population there? What does that look like?
It's a little bit tough to tease out data on the Veterans Administration. We do know there's about 55,000 ESRD patients that are receiving hemodialysis or that are veterans receiving hemodialysis. Our estimate is about 25% of those have a catheter, so may be eligible. Now, all of them are getting dialysis at a VA site. Some may be going to other third-party sites. They could be going to a U.S. Renal Care or a DaVita site. We think that at least somewhere between 30%-50% of them are getting dialysis at a Veterans Administration facility. There's about 171 VA hospitals nationwide, and about 71 of those perform outpatient hemodialysis services. We do think the opportunity is material enough. The collaboration we have with WSI is not a large commitment on our part from an SG&A standpoint, spend standpoint.
We think that the approach is the right one to take to see what type of traction we can get. We were added to VA formulary. We were approved at the VA non-formulary. We do not have a tremendous obstacle to uptake.
Got it. Great. Thinking about your recent contracts with large and medium dialysis organizations, can you talk about how that helps boost scalability for DefenCath and any lessons that you're learning right now that you can lean into more going into the future?
Sure. Look, I think having a contract in place is essential to getting any utilization within the organization. Unless the parent organization is kind of signed off on from a formulary standpoint, you ultimately end up getting blocked off regardless of whether treating physicians want to use the product. That is critical. Look, I think I've taken a lot of learnings over the last year that I could apply both to our ongoing launch going forward as well as to any future launch we did in the renal space that had TDAPA, so to speak. That would not be DefenCath, but some other type of product. It is a very unique niche space. I guess I'll leave it at that.
That makes sense. I know things are still going on in real time, but I was curious, how are your payer negotiations progressing with both Medicare and Medicaid? What % of the dialysis market do you expect to penetrate over time, like this year, next year, et cetera?
I guess I'm going to start with the first part of that question. We don't, at this point in time, while 80% of our patients are Medicare, so 80% of our patients are Medicare, half of those are fee for service, half of them are Medicare Advantage, almost all of them are eligible for TDAPA for the most part. That doesn't require any proactive negotiation on our part. What we are very cognizant of is over time, we expect two things. One, the trend is that patients are shifting more and more away from Medicare fee for service into Medicare Advantage. Medicare Advantage, a couple of years ago, was only 25% of ESRD patient states, 50%. We think in a few years, it's going to be 70%. We see that as a huge opportunity for DefenCath because the Medicare Advantage plan is the payer.
Unlike fee for service, the way that the privatized MA plan, whether it's United or Humana or Aetna, look at things, they're seeing both sides of the ledger. We have a unique product opportunity that can not only improve patient outcomes, but can ultimately save the payer money by reducing other costs that they have to pay out of pocket for treating infections, hospitalizations, antibiotic use. I mentioned before our real-world evidence study that we're running in combination with U.S. Renal Care. A lot of the data that we're hoping to generate out of that study would be used for discussions with MA plans that we intend to start later this year. We do have relationships. We are in communication, but we're not at that point yet where we're aggressively pushing to put a contract in place. This would also be somewhat of an N-of-1 situation.
I don't know that there's been another TDAPA product like ours that has, I'd say, prophylactic utility that could potentially be a cost offset for the MA plans.
Got it. Maybe taking a step back for a second, for new investors, can you just remind us what is TDAPA? How does it play in this description you gave?
Sure. TDAPA is a transitional drug add-on payment. Most patients, or all patients that are Medicare that have end-stage renal disease, the dialysis operator gets paid a fixed bundled payment for the provision of dialysis services. Now, to incentivize the utilization of innovative drugs like ours, CMS has created a transitional drug add-on payment adjustment, which has two phases. The first phase is two years where the dialysis operator gets reimbursed at ASP, which is, in theory, the most profitable period of time for the provider. There are three more years after that with a calculation and a bundled adjustment. It is a continually evolving program, I would say. By statute, it somewhat applies to fee for service, but the Medicare Advantage plans are following. They are obligated to follow TDAPA. Long-term, we see that as the opportunity to enter into direct negotiations with Medicare Advantage.
Yep. Yep. I just want to give you a chance also to highlight the real-world evidence study as well. How does that play into the long-term trajectory of DefenCath, thinking about TDAPA changes and things like that?
As I said before, I think it's going to be essential for whatever we do with Medicare Advantage. This is critical data that doesn't just our clinical study is great. We demonstrated a 71% reduction head-to-head against heparin. When you're designing a phase three study, you've got your primary and secondary endpoint. With a real-world evidence study, we can track a large number of other metrics and outcomes and really show in potentially thousands of patients. It's going to be more than 2,000. We're already over 2,000 patients for which we're tracking data. We're hoping to show a significant impact on a number of scores.
Yep. Got it. Also looking forward to the future and label expansion for DefenCath. I know you mentioned TPN in the beginning. Just to level set here, what's the unmet need and population size, et cetera, for TPN?
TPN patients, patients that are receiving continuous IV nutrition therapy, it's often through a catheter. Very similar to hemodialysis, these catheters have incredibly high infection rates, upwards of 25%, and mortality rates that are similar to HD. Again, another critical unmet medical need. I think the size of the market is somewhere around 5 million infusions a year, which is about 10 million vials of DefenCath. It's a meaningful market opportunity. We've guided investors. We think peak sales in this space can be in the $150 million-$200 million range for DefenCath. We're hopeful. As I said, we expect first patient in the second quarter. We believe we can run the study end-to-end in around 18 months. It's a little ambitious, but that's what we're pushing for with the hope of commercializing by the end of 2027.
Got it. In terms of enrollment cadence, what's your expectation for TPN patients in particular?
Look, I don't want to give specific numbers, but we had a lot of inbound interest immediately upon approval for doctors looking to get DefenCath for their TPN patients under an expanded access. We somewhat had to hold that off while we stood up the clinical study because we want to bring those patients into a clinical study. We have stood up expanded access, and we will be putting or making available to pediatric TPN patients through expanded access.
Yep. Speaking of pediatric, I know you're also looking at pediatric hemodialysis a little bit more broadly. I think the trial's going to start later this year, if I remember. Just thinking about that patient pool, what safety and efficacy benchmarks do you want to see with DefenCath there?
The primary endpoint is a safety endpoint. The secondary is going to be incidence of reduction in CRBSI. Now, you're only talking about 36 patients. The pediatric study is an FDA obligation. This is part of the commitment you give the agency when you get your FDA approval. The overall market size for pediatric isn't tremendously large. We do think patient enrollment is going to be very slow because, one, the patient population is small. Two, parents aren't always or may be reluctant to enroll their children in a study like this. We should have the study kicked off by the end of the year, again, 36 patients, and with the primary endpoint being safety.
Got it. If I zoom out a little bit and think about profitability trends for DefenCath in the long term, how are you balancing executing on the launch for DefenCath now with internal R&D and these new indications coming up in the pipe, like TPN, pediatric, et cetera?
As I mentioned before, we've run the business fairly SG&A light, historically $15-$16 million a quarter. The R&D commitment, we're increasing this year into the, call it $18-$19 million range a quarter. A lot of that is R&D. The R&D initiatives we're kicking off aren't huge spends comparative to other disease states. I think the largest study obligation we have is going to be TPN, which is going to be about $12-$14 million over two years. Expanded access is less than $1 million. Real-world evidence is less than $1 million a year. Pediatric is probably going to be about $4-$6 million over maybe four or five years. It is going to be a longer, slower spend. Still, we feel that we run the business pretty SG&A light, even with the R&D increase.
Got it. Yeah. Sounds good. I know you've also alluded to oncology, ICU, critical care. Where does DefenCath fit in those worlds?
We're in the process of doing some feasibility analysis around oncology. As you know, oncology is not a disease state. It's an umbrella that encompasses multiple different disease states. Our goal would be to design what we would call an all-comers study, multiple different types of cancer, with the common thread being they have either a catheter or a port. We'd like to actually cover both. You have catheters being accessed in completely different intervals. It's a little bit of a challenge, but we do see tremendous long-term value in being able to demonstrate efficacy in that disease state specifically. I know I've, in the past, put timing around, hey, I wanted to get a proposal in front of FDA for oncology by X date.
What I want to make sure is we get the right proposal in front of FDA that designs this study in a way that best sets us up for success.
Got it. We also learned in some of our doc checks that some hospitals are penalized by CMS for high infection rates or re-hospitalizations. Where does DefenCath fit and be the solution?
I think it's part of the value prop as we go to P&T committee on the inpatient side. I think the biggest penalty that they receive, one, yes, there's quality scores. When they have a readmission for a blood stream infection within 30 days, they don't get reimbursed for that continued treatment. You could consider it a financial penalty, but they end up eating a large amount of cost. Inpatient infection rates are incredibly high. If you get an infection, the readmission rate is even higher. I think it's somewhere between 70-90% of patients get readmitted within 30 days for recurring infections. I think that's an area where absolutely we can have an impact.
Got it. In terms of just hearing things from your field force, what's the feedback been on DefenCath use so far, and how are hospitals and clinics responding?
I think obviously most of our use has been on the outpatient side. I think feedback from an administration standpoint has been kind of seamless because you're either locking the catheter with heparin or saline, or you're locking it with DefenCath. There's really no change to workflow. From an operation standpoint, we haven't had any problematic feedback. What we're hoping to start seeing, and it's a little bit still premature because we're only six months into rollout, and this is a large part of what we want to see in the real-world evidence study, is we want to see an impact on infection rates. That's what we want to show. We want to show that we're not just impacting infection rates, that we're reducing hospitalizations, that we're reducing other comorbidities. That's what we're hoping to show.
Yep. Got it. Maybe I'll pause here, see if anybody in the audience has extra questions. Okay. I'll keep rolling. We have a couple of minutes left. Tying it all together, you've got a lot going on. You've got launching DefenCath now, leaning into the inpatient setting, VA rollout, et cetera, TPN trial ramping up. What milestones should investors follow from here on out, and what do you think is most interesting for them to look at?
Obviously, as we talked about before, I think the biggest future potential value driver for us are the LDO contracts and how we convert on those and generate kind of patient acquisition through those agreements. Beyond that, we are, over time, looking to develop beyond being a single product company. So we've got the R&D initiatives we have in place, but we're also open and looking for business development opportunities, looking at things that are commercial, fit with our infrastructure, kind of our infrastructure light mindset. There are two areas where we've got core competency. One is in the renal space, specifically around TDAPA. The other in the hospital, in the hospital segment. We can be a little bit therapeutic area agnostic on the hospital side because it's more about the therapeutic setting of care than it is specific disease state.
Those are areas that we're actively outlooking.
Yep. Got it. One last question before I close out. Just what do you think investors least understand or least appreciate right now in the story for CorMedix, and what should they look for going in the next couple of years?
I think when you're thinking about DefenCath, you have to look at it on a longer-term basis and not quarter to quarter. I think what's least understood is also I think what's most worried about, which is the TDAPA structure. It's a little bit unique. It's not something that applies across, I think, any other disease state. I don't think it's something that, from a long-term standpoint, gives me a lot of anxiety because we're focused on growing the label, growing the opportunity, and over time, shifting to much more of a kind of volume play, making this we'd like to see this to become the standard of care. That's ultimately the goal, whether that's in hemodialysis or hemodialysis plus TPN. From a long-term basis, this should be the standard for which people lock catheters.
Yeah. Got it. I'll squeeze in one more. Since you were talking about becoming the standard of care, what steps need to happen for DefenCath to be there?
I think time is a big part of it. One of the things, the obstacles when you're launching a drug that you come across is you will come across a large number of physicians that'll say, "Oh, this sounds fantastic. Let's talk in a year after you've been on the market for a year. I'd love to see some real-world results." That is exactly why we're running the real-world evidence study. We want to have a much more longer-term mindset in terms of what DefenCath can be. Part of that is generating data that shows the impact.
Got it. Super interesting. We're looking forward to more updates. Thanks again, Joe, for joining us.
Appreciate it. Thank you.