Hi, good morning. My name is Serge Bélanger. I'm one of the healthcare analysts at Needham. I want to welcome everybody this morning to Needham's 25th Annual Healthcare Conference. For our next fireside chat session, we have KalVista Pharmaceuticals with us, a company we've covered for a long time that made the transition to the commercial side just last year. From the company, we have the CEO, Ben Palleiko, with us. I'll hand it over to Ben for a quick overview of the company, for those who aren't familiar with KalVista, then we'll proceed to some Q&A. Ben, welcome, and I'll hand it over to you.
Thanks, Serge. Nice to see you. Thank you for inviting us. As always, we always enjoy doing this conference every year, and thank you to Needham and Company for all the support over, like you said, a long time, actually. Real quick, KalVista, we are a commercial pharmaceuticals company. Last July, we received FDA approval for the first and only oral on-demand therapy for treatment of HAE attacks. It's called EKTERLY. We launched it immediately after the approval. We had, I think everyone would agree, a very successful start to the commercialization effort last year. Put up just under $50 million of revenues in the slightly less than six-month period into last year. At this point, have something over 20% of all patients in the space who've received prescriptions for EKTERLY. The uptake has been really strong, really consistent.
I feel like we'll talk about that more in a minute or two. We also followed that up with a launch in Europe last year. We launched in Germany in October. That launch, much smaller company, so the results are different, but the baseline trends have looked very similar. A lot of high demand from patients in the space. A lot of very high satisfaction amongst both physicians and patients to date. The German launch, at a fundamental level, looks very similar to the U.S. so far. Most recently, just about three weeks ago or so now, we actually finalized pricing in Japan and, through a partner, Kaken Pharmaceutical in Japan, launched EKTERLY in Japan also, really to talk too much about that one, except to say that the initial uptake has been quite good.
Probably more favorable than we expected, which I think has been a little bit of a routine here. We haven't gotten a lot of feedback from there yet, but the little we have heard has, again, been just overwhelmingly favorable. This really represents a meaningful advance for people living with HAE around the world, it seems.
Yeah. Great. Well, like you said, feel it's been a strong initial EKTERLY launch thus far. What do you think has been behind this strong uptake and usage by patients?
Yeah. I mean, 10 years of hard work is the quick summary. It's worth noting that, and actually this is really important. First of all, EKTERLY is just fundamentally a better option for people. There's a lot of therapies out there for HAE and for both prophylaxis and on-demand usage. They all, despite the fact they have good clinical efficacy, just have a lot of challenges. They don't fit well with patients' lives. Until EKTERLY came along, the only therapies approved for on-demand use in HAE were either IV delivered, which is very burdensome, or only slightly less burdensome, subcutaneous. The market leader was a drug called FIRAZYR. When generic, it's called, generically, the name is icatibant. Very efficacious, but has a lot of challenges with carrying it, with using it, with storing it.
Causes a pretty significant injection site reaction, fair bit of pain when people give the injections. Until EKTERLY came along, really, people knew they should treat more attacks and knew they should treat them early, but just simply didn't. That has been why, to a large extent, people have just suffered needlessly, endured a lot of pain, a lot of disabilities, just a lot of challenges in living with HAE. Despite the fact, if you look at from a clinical data standpoint, it looks like these drugs work pretty well. What then led to the, however, really, to the launch results to date, is what I've described as what we believe is the largest clinical trial program ever conducted in HAE. We went out with a drug that we think is superior across the board as an option for patients.
It just sort of is a dominant choice, right, because of all the advantages it offers? We backed that up with clinical trial data that showed, again, we've treated more attacks in a clinical trial setting than anyone ever has before, something like 2,700. We've treated to ages 12 and up. We actually now treated ages all the way down to age two. We've generated a couple hundred attacks in the two-11 population at this point in the cycle. We've treated it in all attack severities, all attack locations, all attack types. We've treated what I think is more laryngeal attacks in a clinical trial setting than anyone ever has. We've treated with all approved forms of prophylaxis. Because we've treated so many attacks, in each of those instances, we have statistically significant data that shows people that EKTERLY works for them.
It works very consistently, and it works no matter what your situation is. If you have a lot of abdominal attacks or a lot of laryngeal attacks, or if you're using any of the prophylaxis therapies or whatever your situation is, we went out with data that we've been talking about for the last several years, really, that showed that EKTERLY can work for people. When we got to market in July, we came with this incredibly robust data set. We'd educated physicians, and so even from the start, they were pretty comfortable prescribing EKTERLY. As we've rolled out, they've only become more so. Actually, and I'll stop here, except to say that what's been really nice this week is I've actually been on the road with our field team, going out and meeting some of the key physicians in the space.
It's been really nice to hear their commentary, which ties in with all that I just said. Their early experience has been great. The patient reviews have been really favorable. The drug has exceeded their expectations in pretty much all regards, and so I think that's why the launch has gone so well, and that's why we think the commercialization effort's going to continue to go well for the foreseeable future.
Like you mentioned, I think you launched in July. The product generated about $50 million in sales in less than six months in 2025.
Yep.
During your 4Q update in late March, you provided some updated launch metrics. Maybe just highlight those to highlight how the growth appears to be continuing in the early part of 2026.
Yeah. We said early on that we expected growth, by which we mean patient start forms, to be consistent and pretty linear, and to an extreme degree, actually, it has been consistent and linear to date. There's some noise, like everything, but it's been quite comforting on a fundamental level just to see the linearity of the launch. That will change over time, right? You don't grow at 7% per quarter of the patient population forever. To date, we have. Well, in March, we gave updates through February, so this isn't the full quarter, but we had just around 1,700 start forms at that point, which is just a little bit over 20% of what we believe is the addressable market.
We had right around, I think 724 physicians who prescribed EKTERLY at that point, which is there's about 22,000, maybe 2,500 physicians total who have ever prescribed. You're talking about basically 1/3 of the physicians. If you look at the highest-tier prescribers, the folks who do most of the prescriptions, the vast majority of prescriptions, we're in an even higher percentage there. We're probably at 2/3 or more of the higher-tier physicians who prescribed, most of whom have prescribed multiple times at this point. The fundamentals look really favorable, and the growth rate has been consistent, and we expect that, like I said, to go on.
Yeah. I don't want you to spill the beans on 1Q just yet.
Yeah
In terms of seasonality from 4Q to 1Q.
Yeah
Anything stand out for a product like EKTERLY?
We do think the end of the year sees pull forward. We think payers, not payers, I'm sorry, we think specialty pharmacies pulled forward, to some degree. We think patients probably pulled forward to some degree because, again, Q1 is when a lot of them tend to go through annual re-auths on their prophylaxis therapies. There's some dynamics that occur in Q1, deductibles reset, things like that. So we do believe that there was some stocking, if you will, kind of across the board with all the constituencies in the fourth quarter. First quarter, everyone has said this, I don't think we stand out at all. Everyone's talked about the fact that you had some really significant weather events.
Factually, we talked about it a few weeks ago, like you said, that there were days in the first quarter when we had zero starts because the physicians' offices were closed. That also means that prior authorizations aren't being processed, and so your commercial conversions also get impacted in those kind of days as well. Some of that you probably make up, but some of it you may not make it all up right away.
Yeah.
There's nothing that's fundamentally shifted in the first quarter, but we have said to people, the first quarter's a little tricky, and for us, every quarter at the moment is the first quarter we've done this. We try to just remind people that we're still sorting this out, and that even if we are really comfortable with the fundamentals, there are going to be some timing issues that get in the way, and these things are going to have a little bit of noise attached to them.
Yeah. I think since the launch, you've highlighted the actual usage has overindexed in some of these higher demand, higher attack frequency patients. Was that by design from a KalVista approach, or was it from the KOLs and early adopters that wanted to use this in their higher demand patients?
I don't know that we intentionally targeted those folks. Our view is that EKTERLY should be for everybody, right? This is just simply a better option for people. We do believe that this market overwhelmingly should transition to oral therapies over the next few years. We're looking to make this available to everyone living with HAE, not just a subset of the population. Factually, when we got started, physicians were certainly, when they would think about who they were going to call first, they were always talking about these high-end, unmet need people, that they were going to be the first ones they're going to reach out to.
There's been, to some physicians, actually, in fact, I had a couple meetings over the last few days where I heard this from people, a shocking degree of outreach by patients who are MyCharting, right, MyChart messaging their physicians and saying, "I want to come in and talk about EKTERLY." We certainly saw that as well in the early days of launch, these folks with high disease burden who wanted to get access to EKTERLY and were very proactive about it. We certainly have overindexed on them, to your point. They're probably something less than 20% of the patient population, maybe 15% or 20% of the folks, we're something like double that right now in terms of their percentage of the start forms.
We continue to get them at an overachievement kind of rate. We think about 1/3 of them at this point are in the process of switching to EKTERLY. We have a long way to go. There's a lot of them left to come.
Yeah.
Certainly, they have been the earliest adopters, if you will, as we got the launch started.
EKTERLY is the second orally administered product for HAE.
Yep.
ORLADEYO was the first one on the prophylactic side.
Yep
... despite some efficacy shortcomings with that product, it still became the de facto prophylactic standard of care for new patients. Based on your patient start, it seems like six months in, EKTERLY is already in that position. Do you agree, or that it's become the standard of care for new patients, and as an on-demand product?
Yeah. This is one, probably a little bit of humility is called for on my part, right? So, again, we believe in EKTERLY . I think it would be a bold assertion to call it the standard of care at this point, nine months into the launch. But I will say that EKTERLY offers people an opportunity to treat their disease the way they should with no trade-offs, right? It doesn't have efficacy challenges. It doesn't have safety profile challenges. It just simply gives them a better way to treat their disease the way they should. Guidelines say that people should consider treating all attacks, and when they treat attacks, they should treat them early. They weren't doing either of those things. For the first time, now people can do that.
Again, as I've been on the road here, I've heard some wonderful anecdotes from people that just confirm that that's what's going on. People are absolutely doing both of those things, which makes them better off. Certainly would the vision of KalVista be that EKTERLY turns into the brand name in the space, the trusted therapy, that becomes the foundational one for your disease? Absolutely. I think that's where we want to get to. Again, I think I'd like a little bit further along before I start making that be the baseline pitch here.
Yeah, maybe let's talk about the payer coverage for the product. I think initially there's a lot getting covered via medical exception, but I think the company's in the process of securing formulary coverage.
Yep.
Just an update on that front.
Sure. Right. The difference in rare disease, probably generally, and certainly HAE specifically, is when you launch, it's impossible effectively to get formulary access really early. The large payers in particular uniformly make you get through six months at least before they'll start to engage with you. When we launched EKTERLY, in the early days, it was effectively 100% medical exception process to get coverage. We were good at that. We've got an extraordinarily experienced team. I think we understand how to work through that process, and so our conversion rates have been very good, and compared to where we expect them to be, and I think compared to what I think people would find to be acceptable.
Nowadays, we did pick up some formularies over the course of last year, and so I'm not sure I have the current number, but we're still primarily medical exception coverage. There are, as you know, though, the ones I talked about, the large PBMs that make you wait six months. They really were only willing to start to engage with us starting last month. We're starting to work our way through the process with all of them. I don't want to get too in depth in this. Just you never know who's going to listen to the thing. That takes a quarter or two to work its way through.
Once we work that through, I think we'll all find ourselves in a satisfactory position, and from their perspective, in terms of how the cost works, and from our perspective, in terms of just getting meaningfully improved access and a faster pathway to access for patients that I think will benefit everybody.
In terms of patient journey right now, they still go through your Quickstart program.
Mm-hmm
For initial access, and then either medical exception or the formulary coverage is the path?
Yep, exactly. Basically, whenever someone wants to switch to EKTERLY, they get a Quickstart form. We call it a start form, and then they get a prescription for EKTERLY. This is important because people don't always have as much experience with the on-demand prescriptions. Typically, those prescriptions are in for a year, and they're typically written with an initial fill, which, on average, is right around two cartons nowadays. It could be more, it could be less. It's typically written as the patient can refill as needed. Once they have the prescription, and it's been approved, they don't have to keep calling the physician to get a refill. They just call and get them whenever they want.
When that goes to our patient hub, once we verify that someone has insurance, we immediately send them a carton of Quickstart, and the idea is let them start to get some experience, let them treat an attack with EKTERLY, let them get used to it. Because it is different, if you've been injecting yourself your whole life, taking EKTERLY is a different thing. It takes a little bit of time to get used to it. What happens is we work on the commercial approval. As soon as that approval is received, the patient will get their first commercial shipment of EKTERLY. They don't actually have to use the Quickstart to get the commercial shipment. It could happen whatever immediately. They will get their first shipment of whatever size the physician has designated.
Like I said, once they need a refill, they don't have to deal with the physician or the insurance company again. They just call their pharmacy and get a refill sent to them. It's very straightforward for people once you get through this initial approval phase.
In terms of expectations for formulary coverage, I know you're still in negotiations, but do you expect to be on par with the generic FIRAZYR and the same prior auths, and process, and things like that?
Yeah. HAE is 100% prior authorization is required. Everybody has to have a prior authorization.
Yeah.
There's nothing different about that. No matter what your therapy is, you have to have a prior auth in HAE. That'll always be the case. In the long run, we expect that we'll be primarily formulary access. In the long run, even then you end up with 20% or 25% of people are always on medical exception. It'll always be a portion of the patient universe that actually is getting their approvals through the medical exception pathway. No, it would be wonderful to be on par with the icatibant, but that would probably require icatibant pricing. In general, I think what we'll expect is there'll be some type of.
I don't know if this would be in all cases, but we would generally expect a step through generic icatibant, which we've long said is completely reasonable and which we don't think is burdensome on either the company or the patient population. By our estimates, something like 80% of people with HAE have either previously been or are currently on generic icatibant anyway. Moving them through generic icatibant, we believe, is a pretty straightforward process, and that won't represent a burden. We've always operated with that assumption. I'm sure I've been talking to you about this since the launch. We'll see how it plays out, but I think that's our baseline expectation.
Thus far, there's been no issues with refills and number of refills. Like we said, will you over-index with some of these higher-need patients?
Yeah.
There hasn't been an issue thus far?
No, it's a small enough indication that payers tend to have awareness of these folks on more of an individual level. If someone has really high burden disease and used a lot of whatever their prior therapy was, a lot of FIRAZYR or something, yeah, they'll know that. If these people are coming through with high refill rates, they tend to be aware, and that tends to not run into excessive challenges. Over time, we do expect that the way that payers will look to manage this is more from a quantity limits perspective. You get some kind of baseline amount every month, and if you need more, you have to go through some process. It hasn't happened yet with anybody. We just expect it to occur over time.
I certainly wouldn't expect in the first year or so, because again, everyone's still trying to get enough experience with usage rates to really be able to draw some conclusions. That's an item further down the road, but it certainly hasn't been a topic yet.
Okay. Maybe it's worth going over just the overall HAE market. There's been a lot of activity recently, mostly on the prophylactic side.
Absolutely
Obviously you are a new entrant on the on-demand segment.
Yep.
An idea of the overall units and how stable or how much growth you expect to see from the market.
Sure. Yeah. Right, so yeah, just a little baseline data. In the U.S., which is all the only data here, about 70% of people use prophylaxis and on-demand generally. About 30% of people treat only with on-demand. Those numbers have been quite stable for many years now. The key part there is even people on prophylaxis typically have breakthrough attacks at some rate, and so virtually everyone with HAE gets a prescription for an on-demand therapy. It's almost 100% of people have prescriptions. Prophylaxis, to your point, has been extraordinarily active in the past few years. There are, I believe nowadays, I think it's seven approved therapies for HAE. This is for a 9,000-person disease, by the way. There are seven approved therapies. I think there's another four coming.
Mm-hmm.
It's an area that's got a lot of attention. They're all quite efficacious. I'm not sure any of them really differentiate on the basis of efficacy. I think they all have very high rates of attack rate reduction. Where they mostly differentiate nowadays is on dosing profiles. You've got one oral, you've got some longer-acting injectables that have come out. Last year there were two launches, ANDEMBRY, which is a once-a-month prophylaxis, and DANZALERA, which is a once-a-month or perhaps once every two months prophylaxis as well. People have been extending out their dosing schedules primarily. There's another oral prophylaxis on the way. There's multiple, more potentially long-acting injectables on the way as well. Over the next few years, that market's going to get even more crowded.
Again, with therapies that are probably, from an efficacy standpoint, similar, but just giving people different choices with regard to the dosing. On-demand's a little different. Again, EKTERLY is the first and only oral on-demand in the space right now. Until it came along, the primary therapies were either IV delivered or injectable. Icatibant, the brand name is FIRAZYR, was the market leader, primarily because it's subcutaneous. Again, they're also all efficacious. They have similar burdens though with any injectable or IV therapy. There is one other potential oral entrant that we expect to come next year.
Mm-hmm.
They had phase III data at the end of last year. We'll see where they come out. I don't know that anyone view them as meaningfully differentiated from EKTERLY. We think more options are always better for patients. We support choice. We think certain people will like certain things for different reasons, but we think EKTERLY is going to be a very well-established brand over the next year and will probably be on its way to becoming the patient's first choice in most cases.
Yeah. Based on the data they provided when they reported the phase III data, do you feel it's more or less the same as EKTERLY, or does it differentiate?
I think my view and their view are probably somewhat different in this regard. There's certainly some numbers that were different. I think the question is, are they better? Based upon my interactions, well, and our team's interactions, and even just the last couple of days, seeing physicians' interactions, I don't think that anyone views them as having a meaningful differentiation. Again, it's a different mechanism. Some people will like it more or less. There's a place for this therapy.
Yeah.
I don't know that I see a pathway to it becoming a predominant option. Again, more choices is always good for people.
Yeah. In terms of the prescriber base in HAE, what does it look like, and with your current sales force, how much of it are you able to address?
Yeah. The short answer is all of it, to the last part of the question. When we launched, we believed there were around 2,000 physicians in HAE who wrote prescriptions. Since then, we've actually found there's about 500 more, so we think it's right around 2,500 now. It's a pretty concentrated space, though. A couple of hundred physicians write over half the scripts. Something less than 1,000 physicians write around 80% of the scripts. You've got a long tail with a lot of physicians who have a patient or two. Prior to launch, our team, which I should just point out is just an exceptional group of folks, there's 32 of them in the field, they actually called on effectively all 2,000 physicians.
Yeah.
Maybe there were a few tier threes left out, but it was the overwhelming majority, way over 90% they called on beforehand. They've continued to provide really effective coverage. I think we designed the territory as well. Again, I think we've got a team that kind of knows what they're doing. Obviously, you spend most of your time with the tier ones, tier twos. Their rates of prescribing are very high. Last data I saw, they were something over 2/3 or maybe even 3/4 at this point have prescribed in some of these tiers. I think of the top 30 physicians we've identified, I think at this point, all of them have prescribed, and in fact, I think most of them have actually prescribed multiple times.
Yeah.
The penetration's been really good. It continues to broaden out really nicely. We expect that to continue.
Based on the last updated launch metrics, there were over 700 prescribers as of the end of February, so that must account for the bulk of the opportunity, the TAM.
I haven't done that calculation, but those folks are going to be the majority of prescriptions.
Yeah.
Probably a supermajority of prescriptions, I would agree with you.
Okay. Let's spend some time on the ex-U.S. launch.
Mm-hmm.
We've seen a number of approvals. I think the first one was Germany.
Mm-hmm.
Has the U.K. been off the.
U.K.'s approved.
Approved? Okay. Europe is going to continue rolling in, so maybe just highlight what you're seeing so far in Europe.
Sure.
How it compares.
Yeah. Right. We got seven approvals last year. U.S., U.K., European Union, so a territory, Japan, Switzerland, Singapore, and Australia, those seven approvals last year. We have launched to date in the U.S., Germany, and Japan, like I said earlier. Germany is a pretty straightforward country to launch in. You're allowed to launch roughly 30 days after approval. We did that. Initial uptake, like I said before, has been quite strong, same baseline dynamics as you see in the U.S. In fact, again, we think those dynamics are going to play out everywhere. People value the oral option. They like the data. Physicians are really pleased with it. Early responses have been really favorable. That's going certainly at least as well, in fact, I guess I would say better than we initially expected.
It had gone so well. We actually had to do a reforecast in January just to kind of know the numbers we went past so quickly. Rest of Europe takes longer because you have to work through pricing in those territories before we launch. We're in those conversations. They'll play out starting out over the course of the spring and then through the rest of the year, and actually, some of them even go in the next year. Factually, there will probably be some territories where it becomes more challenging for us to launch given the economics. Right? We've talked about the fact that pricing in Europe is uniformly meaningfully lower than the U.S., and in some countries, it actually just may not make economic sense for us as a company to do that. We haven't made any decisions yet, but that's just how it works in Europe.
Yeah. When we think about the ex-U.S. opportunity for EKTERLY, it's kind of the standard 15%-20% of what the U.S. can be?
Yeah.
Is that how we should think about it?
Yes.
Okay.
We've very consistently said that. Yeah. Obviously in these days it's less.
Yeah.
That's a reasonable long-run expectation, yeah.
just takes longer to ramp up for the reasons you mentioned about the price and negotiations and things like that.
Yeah. The rest of the world is overwhelmingly on-demand only.
Yeah
Almost 90% on-demand only, but the pricing is so much lower, and in some countries it's very, very low.
Yeah. You worked on an ODT formulation for kind of a pediatric.
Mm-hmm
Label expansion or I guess it'd be a separate product.
Yeah.
It'd be an NDA, not an SNDA, right?
Yeah. I love this indication. This is just a bit of a story, but I like it so much, I'm going to make you listen. We got the initial approval in ages 12 and up.
Yeah
It was great. We always knew we wanted to extend into pediatrics, because there's some amount of unmet need. Truthfully, I don't think anybody realized how much unmet need there was. When we started our clinical trial, we thought it was going to take us three years to get that study completed. We thought it'd take two years to enroll 36 pediatrics ages 2-11. We had hoped that whole group, we would get a total of 12 attacks, and then over the next year, and then we'd wrap it up in three years. That was the goal. Well, it turned out when we went out with, we actually enrolled all those 36 kids and actually a few more, in less than a year. We enrolled a full year ahead of schedule.
What we discovered as we went through the study was the reason we wanted 12 attacks was because it was believed for a long time that young kids don't attack at very significant rates. Our baseline assumption was these kids are having a few, a couple attacks a year. That's what we'd heard forever. We designed the trial that way. We really thought we'd end up with a very small number of attacks. What we found was that over the next year of the study, we actually, as of the last update I got, which was a few weeks ago now, we had something like 230 attacks in these kids. They're attacking at these astonishing rates.
Wow.
What we found out was actually that their attack rates aren't a whole lot less than they are in adults. On average, they were having right around 0.7 attacks a month, which is maybe half, two-thirds of what the overall population rate is. It's dramatically higher than anybody ever thought it was. What we learned was that it wasn't that these kids aren't having attacks, it's that these attacks aren't being treated. Because the only approved options in the on-demand space, well, the only approved option period is an IV therapy.
Yeah.
Nobody wants to give their kid an IV unless it's obviously a really extreme form attack. The learning here, the shocking learning, and we presented this data at the end of last year, our Chief Medical Officer has showed it at a couple conferences now, and it's been just remarkably well-received, is that actually these kids are having enormous numbers of attacks. Actually, really, to a degree we didn't even appreciate, it just really offers a tremendous opportunity for these kids to get better care than they have historically. Kids that age are 10% or 12% of the population, so it's not an enormous uptick in terms of revenue opportunity. In terms of an unmet need and a quality-of-life opportunity, it's a dramatic enhancement.
That's why I like to tell that story, because it just really shows that sometimes you go into something with fundamental thoughts, and you realize you can have much more impact than you expected you could.
Okay. You'll be filing the NDA, is it in the third quarter?
Yep
Sometime this year, right?
Yep. The plan is to file the NDA in the third quarter, and then, depending on the review type, get it ideally approved earlier mid next year.
Once it's approved, do you expect a portion of the adult patients to switch to an ODT formulation? I don't know if there's an advantage.
Well, yeah. We actually thought about doing an ODT formulation for adults as well. I should probably explain. For kids, the tablet is a 300 mg capsule, so it's around the size of an Advil capsule, if you know how big that is.
Okay.
It's not big, and it's got a very slippery coating, so it's easy to take even without water. For kids, that's a little bit more of a challenge for young children. We actually developed an oral dissolving tablet for kids that you can just put it in their mouth, and it dissolves in 10 or 15 seconds. You don't even have to swallow it. It just goes straight down with your saliva. It doesn't really have any taste. It doesn't have any noticeable attributes for children that would dissuade them from using it. It's pretty useful. We actually thought about doing that for adults as well.
Yeah
They might prefer a tablet. What we found when we went out and did our research was actually patients didn't see any need for it. Again, in 2,700 clinical trial attacks, and then I don't even know how many more since we launched, I've never heard of anybody having trouble swallowing the tablet. There just didn't seem to be a significant need. It wasn't clear to us what value it would provide to people that would be incremental. They've been so happy with what they had so far. We decided we'll stick with the ODT formulation for the pedes. I guess if some adult really wanted a ODT, I presume they could.
It could be available, yeah.
We're not going to pursue it as a label.
Got it. Makes sense. I think during the last quarterly update, you talked about short-term prophylaxis as an additional opportunity.
Yeah.
How significant could that be? Is it incremental?
Yeah
A lot of work for that?
Yeah, it's incremental. It is value. Again, several of the conversations I had the last few days, actually, physicians brought it up unbidden by me as something they think about. It is a challenge for people in the space nowadays because, again, trauma can be a trigger for attacks. There is commonly a concern that if you're going in for any kind of procedures, last night we talked about dental. Dental procedures are commonly talked about, but things like colonoscopies can cause attacks. All these things can be triggers. There have been some short-term prophylaxis ideas, methods used in the past. We've had some people using EKTERLY for short-term prophylaxis already, right? Obviously, it's just a better way to do it.
We have also decided we'd like to generate some additional data to help physicians talk about the schedule for it and just give them some more confidence in it. We are kicking off a short-term prophylaxis study this year. It's pretty short. It's small, it's open-label, it's short. It isn't to support a label change, it's just to provide additional data to people to help them understand how to go about it. That'll have some data next year.
Okay. I think we only have a few minutes left, so I'll ask you to just give us an overview of financials, and then we'll wrap it up.
Yeah. This is one, not being the CFO anymore, it caused me to have to think about this. We gave the fourth quarter update a couple weeks ago. We ended the fiscal year with right around $300 million in cash. We have said we're funded to profitability.
Mm-hmm.
We've said that we expect that we can reach cash flow breakeven within the first few years of launch. Folks that know us know we've always been really careful consumers of shareholder capital. We continue to do that now. We're thoughtful on the expenditures always, and so that's one thing that won't change as we continue to move forward.
All right. As we've discussed, the launch of EKTERLY has been really strong. Despite that, there's still kind of a meaningful short interest in the stock. Just curious what you believe investors or even analysts may be missing about the story at this point.
Yeah. Based upon the conversations we have, I don't know that I'd say anybody's missing any part of the story. I think we don't get a lot of concerns over the story. I think the truth is all they're missing is more data.
Yeah.
I think factually, we've reported a partial quarter and now one full quarter, and I think people just want to see a little more evidence of sustained traction. We understand. That's a perfectly rational response. We'll report the first quarter sometime next month, and I think people will continue to assess based upon that. Hopefully, that gives them a little bit more of an update they can use to extrapolate from. Again, we remain very comfortable in the fundamentals. We think EKTERLY can become the foundational therapy for HAE, and so we look forward to continuing to bring this to as many people globally as we can, as fast as we can, and really helping these people to live the better lives that they've hoped for, and until EKTERLY came along, just didn't have the opportunity to do.
All right. Well, Ben, thanks for spending time with us and participating in the Needham Conference. We appreciate it.
Always great to see you again, Serge. Thanks.