Conference Transcript - PHVS 2026-09-15

Max Skor, Analyst — Morgan Stanley

Great. Thank you very much, everyone, for attending. I'm Max Skor, a biotech analyst with Morgan Stanley. And before we get started, for important disclosures, please see the Morgan Stanley Research Disclosure website at www.morganstanley.com forward slash research disclosures. And with that, I'm very happy to have the Favaris team with me today, Bernd Mody, CEO, and Maggie Beller, head of IR. Thank you very much for joining us.

Bernd Mody, CEO

Thank you, Max. Good to be here.

Max Skor, Analyst — Morgan Stanley

Great. So maybe we start off, I think this is the third year in a row you've attended. We've sat here at the Fireside Chat. This is a momentous time in the story of Favaris. If you can just maybe step back and give us a brief overview of just how you got here and what are the key steps going forward. Sure.

Bernd Mody, CEO

So actually our journey, or Nelson, my personal journey in HAE started 23 years ago. not with Vavars, but with the company that developed Icadiband. And I worked there together with Jochen Knolle, who's the inventor of Icadiband. And then, as we all know, Shire took over in 2008, and it then became, still is, the most widely used acute therapy for subcutaneous injection. But already back then, we knew that patients were looking for better treatments, And already then we were just starting to think about the vision for an oral therapy. And then later, a couple of years later in 2015, starting in operations 2016, then Jochen and I started Favars with the idea to develop an orally available beta-receptor antagonist, really from chemistry, square ones, really from scratch. And we're really excited to what we see today with what came out of that is the CRICT event. and the properties of that molecule makes it very different from ecatabans. It's a B2 receptor antagonist, but it's, to our knowledge, the first and only orally available B2 receptor antagonist. And it also has the properties that make it a half-life and so on that makes it suitable for prophylactic ecatabans. So looking at the therapeutic area over more than two decades, you see the big unmet need for an efficacious oral therapy option, and there's still nothing approved out there, and we are, of course, hoping that the Crickipan is going to be the first orally available V2 receptor antagonist in oral therapy with a high level of efficacy for people living with HAE.

Max Skor, Analyst — Morgan Stanley

Great. Thank you very much for that introduction. So when we think about the HAE market, we have the on-demand market, which you also have a formulation for of Ducryctoban, and then the prophylactic. Let's start with the prophylactic, because you just reported impressive Phase 3, Chapter 3 data. Maybe give us a high-level overview of the overall efficacy profile, and then we can dive into specifics.

Bernd Mody, CEO

Yeah. So, yeah, you saw the data. We had a remarkable 87% efficacy in the Type 1, Type 2 population of hereditary angedema. And also the study included, as also part of the overall protocol, patients with normal CU1 mutations, other mutations that, and so the primary endpoint included a small number of such patients, and that total primary endpoint, then efficacy reduction, the attack reduction was 83%. So that really means that we are in the area of what we can call injectable-like efficacy. And if you look at the other injectables in the prophylactic space, therapies out there, there all seems to be almost like a ceiling, more or less, maybe not coincidence, but there seems to be like there is a maximum level of efficacy at around 87 percent. And we're very happy to see that with the Crick-de-Ban, and it really puts us in a very differentiated position. Because as I said, we see a huge amount of need for an oral therapy that really provides that level of efficacy.

Max Skor, Analyst — Morgan Stanley

And maybe if we can dive into the key secondary endpoints, maybe you can talk about the baseline characteristics and how that compares to competitors in the space. Specifically, I imagine everyone's looking at Orlodeo and how that phase three lined up versus chapter three.

Bernd Mody, CEO

Yeah, the baseline characteristics are very similar to other therapies. I mean, they're all in terms of attack frequency. They range from, I think, the lowest is the texiro at 1.7, and the highest is Orladeo, indeed. And we were a little over 2.1, I think it was, in our case. So it's all very, very, very similar. These are fairly severe patients. Yeah, yeah. So I think that's...

Maggie Beller, Head of Investor Relations

I'll add on that those were our placebo rates in the full 24-week study for the baseline characteristics leading into the study, our average attack rate was about 3.6. So that is a more severe patient population. So we were incredibly impressed to see the 87% attack reduction given the severity of the patients that we were treating.

Max Skor, Analyst — Morgan Stanley

And then I guess after the results came out, there were some questions around the safety profile. We actually hosted a KOL a couple days later. The KOL was comfortable with the enzyme elevations given the confounding factors such as androgen exposure, fatty liver, but could you comment on those?

Maggie Beller, Head of Investor Relations

Yes, happy to. So in our study, we did see two grade three level two liver enzyme elevations. As you mentioned, Max, one of those patients did have fatty liver disease. That was also a normal C1 patient. So that person ended up discontinuing the study drug but stayed on the study, so we were able to continue their lab assessments. The second patient had previously used androgens. They actually stayed on study and completed the study and then rolled over onto the open-label extension. Both of those patients, neither were symptomatic and neither saw elevated bilirubin, so we were pleased to see that.

Max Skor, Analyst — Morgan Stanley

Okay. That's very helpful. And then there was one patient, I believe, laryngeal, attacked. Any commentary around that?

Maggie Beller, Head of Investor Relations

Absolutely. So that was our grade 4 finding, which is the same as the serious adverse event in the safety table. This was, once again, a person with normal C1. They had a plasminogen mutation. This person, unfortunately, leading into the study, had experienced severe laryngeal attacks. So as many as 19 laryngeal attacks in the three months leading up to the study. These attacks required constant intubation. So this was somebody who had been intubated multiple times. Upon start of the study, this person experienced a 90% decrease in their attack frequency. So really impressive efficacy findings there. At one point, they had a laryngeal dyskinesia, which just means that they had difficulty breathing. that resulted in them being hospitalized and being intubated. While intubated, they were scoped, and it was confirmed that this was not, there was no angioedema associated, so this was not a laryngeal attack. It was just a result of the frequent intubation prior to the study start. This person stayed on study drug. They completed the study. In fact, afterwards, we reached out to the PI just to see how the person was feeling. They had reported that they were happy with their Ducroctoban treatment and actually were able to go back to work. So not only did they experience the improved efficacy, but also improved quality of life. So we view that as a real success story with Ducroctoban.

Bernd Mody, CEO

That's great. Yeah, maybe I'll also add to that. I mean, it's hard to imagine for any of us here what it's like to have 19 laryngeal attacks and suffer through that. And so this is, as Megan said, is a normal C1 patient. And so part of our approach here, and also as we designed the study, was to include, as I mentioned, normal C1 patients. And this is not an insignificant portion of the patient population, and also because of the mechanism of B2 receptor antagonist. That is really the only mechanism that has real efficacy for this patient population.

Max Skor, Analyst — Morgan Stanley

Can you talk about that opportunity?

Bernd Mody, CEO

Yeah, so it's estimated now that approximately 20% of the HAE population is the normal C1 mutations. So that follows a different pathway. It doesn't go through the calicrine pathway. So it's already at the bottom of the receptor level, then you can really most effectively treat that condition. They do get therapy today with the existing therapies. that that is because the more generalized label for those indications, hereditary angioedema, because the normal C1 is also sometimes called type 3. It's one form of hereditary angioedema. But with our data now, we hope to have to get that also included in our label and also finally a real efficacious therapy for these types of patients.

Max Skor, Analyst — Morgan Stanley

And are these patients generally managed with on-demand or prophylactic treatment?

Bernd Mody, CEO

I think for the most part, it's on-demand. today. Many use catavent. They also use stride and the other drugs. They do get the other drugs, also prophylactic and prescribed, but not with modest efficacy or very low efficacy.

Max Skor, Analyst — Morgan Stanley

And then maybe just going back to the safety profile, how does it compare to Orlodeo? I think on the Orlodeo label, something's called out at higher doses, cardiac implications. Any commentary around how ducryptoband compares to that?

Maggie Beller, Head of Investor Relations

Thanks for the question. That is something that we're really proud of. We didn't see any additional cardiovascular signs, and in fact, we saw one of the major pushbacks that we get for the other approved oral is GI side effects. We did not see an increase in GI risk with ducryptoband treatment, and the placebo and ducryptoband arms are balanced with respect to GI as well. So So not only do we have a thorough QT waiver, we've put out a lot of cardio safety data, and we were happy to see in clinical studies that there's no additional cardio risk with tucuritaban.

Max Skor, Analyst — Morgan Stanley

Now, going down the path of potential approval in prophylactic, we'll touch on On Demand in a bit, but what have you learned from the Oralodeo experience so far? How are you going to approach marketing, talking to physicians? How should we think about the launch curve?

Bernd Mody, CEO

Yes, so Orladea, as you know, had a pretty strong uptake, which is not a surprise to us because it reflects the fundamental need for an oral therapy. So patients desire to get away from injections. And I think what we've seen now since the launch of Orladea is that there is a very high churn rate. There's very many patients drop off after trying it. And I think also in the call that you mentioned, Karyon Colley mentioned that you also wrote about that it said that 50% of his patients discontinue Orladeo within six months. And that is, then go back to the injectables, and that's really simply because of the lower efficacy. So that patient segment, patients who have tried Orladeo, and the reason for trying it is, of course, the desire for an oral, and then they go back to the injectables because it didn't do so well for them. With the Cricitibent now, they have the opportunity then to try again. So I think that the propensity for that type of patient to try the Cricitibent would be very high. So that's a very clear segment. We've estimated some numbers. I think Brian Christ has also mentioned numbers ranging from up to 1,900 patients that have tried Orlodeo and dropped off. So that's very clear. Then there is the patient population that, of course, are aware of that. They're still waiting for an oral, but they don't want to take that risk of starting to having attacks again, so they stay on the injectable. They're waiting for the cryptoband to concession. That's another segment that's very logical for us to approach. And then the first-line population, newly diagnosed, it's very logical to start with an oral as a first-line therapy, and that there are 152 to 50 patients every year that come into the mix, and that kind of accumulates over time. And what we hope to see, of course, is that when somebody starts with the Crixiband, that they would be satisfied, and then the search is over, and there's no need to switch. So then with that building patient population, also from the new patients, it kind of accumulates over time. And the chief commercial officer likes to describe it as a pancake effect, but it's basically accumulation of patients coming in and newly diagnosed.

Max Skor, Analyst — Morgan Stanley

So maybe that leads into the on-demand opportunity, but stepping back and thinking about the prophylactic versus on-demand, having dual formulations of the same drug, how are you thinking about the market opportunities for each and then on-demand launching first, how does that help you define the prophylactic market going forward?

Bernd Mody, CEO

Yeah, I mean, we do think that the on-demand market is a viable market. Also in the future, there are certain patients that prefer to treat when the attack happens, especially if you have something that's very reliable and simple. And again, also in the on-demand segment, same phenomenon. None there is an oral therapy, but not the optimal efficacy. So I think it's similar to the prophylactic setting. We hope and expect that the corrective band will also take over a lot of those patients. And also their patient population is still on the cativant. and I think that's 65% or something more, actually patients that really are still on the cataband and what they like about the cataband is the reliability, the efficacy, the trust in the mechanism. But if you have a world that works as good or even potentially better, then that's a very logical switch for that patient population as well. So it's very similar to the dynamics. And I think there is a segment of people living with HIV that may want to stay on the mask. We think that's also why we're happy that we have both options for the patients.

Max Skor, Analyst — Morgan Stanley

And so in regards to the actual segment breakdown, do you see prophylactic growing or on-demand growing? How does that look?

Bernd Mody, CEO

The different growth factors. I mean, overall, the prophylactic segment continues to grow. I think that you see that is clearly the trend. I think that's also in the treatment guidelines that patients really should be on prophylactic and not to have attacks. So I think that's clearly the trend. I mean, I think we've seen that over the years, and I think that continues. But as I said, I think there's still a remaining segment that also where the on-demand therapy option also could make sense. And I think also patients that are also very relevant.

Max Skor, Analyst — Morgan Stanley

Yes, it sounds as, based on our conversations with KOLs, it sounds like all prophylactic patients also have an on-demand that they carry with them for breakthrough attacks. How does having two formulations of the same drug potentially address that issue?

Bernd Mody, CEO

Yes, so the first two I think we're going to describe is potential combo use, and I think that the treatment modalities in HAE or in angioedema is really exposure-driven, So it's all about maintaining a certain therapeutic exposure. And so unlike some other diseases where you have to approach it from a different side. So a breakthrough attack typically occurs when that exposure and the efficacy is not there. So you're basically topping up with the same drug, which is a combo use from a medical perspective or mechanistically is fine. And we see that as also a potential for the correct event. Okay.

Maggie Beller, Head of Investor Relations

I'll also add that right now in clinical practice, people use the same mechanism to rescue their attacks when they have breakthrough attacks. So right now, if you're on Texiro or Orlodea, which are calocrine inhibitors, and you have a breakthrough attack, you treat with Ectoralee, which is also a calocrine inhibitor. That is a well-understood additive process. So for us, we're using that same philosophy of mechanism plus mechanism. them.

Max Skor, Analyst — Morgan Stanley

Do you have any, I'm going to ask you to put some numbers or percentages around that based on what we've learned from the Ectorly launch or what you've learned from talking to KOLs?

Maggie Beller, Head of Investor Relations

I think right now we're seeing that people on Orlodeo tend to have about six to 12 breakthrough attacks a year. So those are treated in a number of different fashions. Many of them are with Ectorly. And then for Texira, we're seeing that although you see in the clinic, there's 87% attack reduction. As people have been moving from the two-week treatment regimen to the four-week treatment regimen, there may be more breakthrough attacks there. We haven't seen exact numbers around how many ectorily treated attacks are prophylactic breakthroughs versus individual patients who are just treating attacks as they happen and aren't on background prophylaxis.

Max Skor, Analyst — Morgan Stanley

Okay, maybe we can pivot over to the on-demand opportunity here. You have a PDUFA date coming up. Maybe just stepping back, give us a quick overview of the efforts that you've already accomplished and what's still ahead.

Bernd Mody, CEO

Yeah, so the launch preparations are full underway and fully on track, and the team is growing. There's a tremendous growth in the organization. A lot of people coming on board and building the commercial team. We're also very happy that a lot of various good talents that people live with have prior experience with HAE and come from companies and have other therapies in HAE and work on those therapies. Our head of patient services launched for Azure, for example, and I think it's almost 40% a little bit more about team members building from the top down have prior experience in the area. so that's very strong and also there's a lot of excitement about Favar so also from other skilled talent from other companies joining us so that's going really well and fully on track and then getting closer to the remaining phase in building the sales force and then to get ready for the launch How should we think about the sales force? Yeah it's relatively compact compared to many other therapeutic areas. I mean, I think that what we are building is very similar to what others have as well. I mean, to have like 35 people in the field and then the overall commercial organization, I think, is going to be around 70, then adding a few more, maybe a total of about 90 people by the time we launched Profeet, which is also, HIE is an area that's also suitable for a smaller company like us to commercialize on their own and achieve forward integration.

Max Skor, Analyst — Morgan Stanley

And which, so in regards, leading up to the April 2027 PDUFA date, what interactions do you expect to have with regulators? How is everything going, getting commercial supply up and running? And then we can potentially talk about expected label, price, economics.

Bernd Mody, CEO

Yeah, so the review is ongoing, and then it will be coming soon also for mid-review meetings, and so that's underway. And we also, on the CMC and drug supply, that's also getting ready for the launch. It's also on track. And then on pricing, that's something we're evaluating now. I think that fundamentally we'll be looking to price for value. I think that what we've also seen in other launches in HAE that there hasn't been any disruptive pricing strategies by anyone. And I think with the profile of the correct event, I think we have the potential to really illustrate that value that can provide an attractive pricing. I think a key point also in On Demand is our data in On Demand is significantly differentiated when it comes to single-dose attack resolution, which is a very important aspect clinically and also in real life for patients, trusting that what they take is going to help them and they don't have to worry about you to take a second dose or not and to get the complete symptom resolution. I think that's a clear differentiator versus act early. And then another point is also the endpoint and data point that we have in our protocol, which is at the beginning of the whole attacks process, is the so-called end of progression. And that is also a clinically meaningful endpoint, And so subject to agreement with the regulators, we may be able to get that into the label as well. And that shows that the end of progression means that this is when a patient really feels that things are under control, it's not going to get worse. And we see that in 17 minutes with the correct amount.

Maggie Beller, Head of Investor Relations

I'll highlight that we had 12 endpoints in our study, all of which hit statistical significance in a hierarchical fashion. So I'm not sure we're going to be able to get all 12 into the label. That would be a very large packet insert. But as Bernd highlighted, the key differentiating factors are the rapid onset of action, the complete symptom resolution, and the single-dose durability. If we're able to have a label that's appropriately demonstrating those three factors, I think our commercial team will have a successful launch.

Max Skor, Analyst — Morgan Stanley

And so since we have an oral and on-demand, we have an oral and prophylactic, specifically an on-demand, how should we think about the launch curve? Are people going, I imagine people are going to immediately comp it to ectorlies in early stages. How are you thinking about that?

Bernd Mody, CEO

Yeah, so I think that the level of awareness in the community that the cryptoband is coming is very high. and so that speaks in our favor. I think also part of our strategy is also to the extent within compliance guardrails to identify patients that can be interested in the curriculum on the launch and we have this so-called opt-in program where patients consent to receive information and we are already, I think, up to, like, almost 2,000 patients, targeting almost 3,000 patients by the time we get to launch. So I think that that puts us in a strong position, and that kind of accelerates further as we then progress further to the prophylactic launch because then we already have a good patient population as a basis that could potentially also then migrate over to the prophylactic. So I think that gives us a very unique advantage in the real high-value launch, which is prophylactic as a new market entrant, because we have this sort of stepping stone with the on-demand launch. It's all very integrated.

Max Skor, Analyst — Morgan Stanley

And then in regards to the opportunity in Europe, how are you sequencing the potential launches? I believe the European package is under review now. Any commentary around that?

Bernd Mody, CEO

Yes, that's under evaluation. And so I think that there are also other factors that play a role in the assessment of the European opportunity. I mean, we'd like to, I mean, fundamentally, we would like to see the correct events serve the patient's needs on a global scale. I mean, I think that many patients participated in our trials globally, and we'd like to see whatever we can do to achieve that. I think the ex-U.S. has its own different challenges. I think especially in H.A.E., the commercial opportunities, the kind of difference between the U.S. market and the rest of the world is, for most therapies, pretty significant. But in H.A.E., it's even more so because of the pricing structure for historical reasons. Because a lot of the therapies, the first therapies in H.A.E. started in Europe. So that's something we are evaluating, and more to come on that, but I think it's fair to say that there are definitely opportunities there, and also not only in Europe, I mean in Japan, and potentially in China and other territories, could also have significant patient Should we start thinking about contributions coming ex-US in 27 or 28?

Max Skor, Analyst — Morgan Stanley

How should we think about that?

Bernd Mody, CEO

That's a little bit too early to guide on, so we'll provide more guidance on that once we have. Okay.

Max Skor, Analyst — Morgan Stanley

Is there a U.S. Expanded Access Program? Any color on that and how that's going?

Bernd Mody, CEO

Yeah, that's in the sort of the medical area. That's something that we set up, and there's a lot of interest for that, so that's also underway.

Max Skor, Analyst — Morgan Stanley

And are you getting any feedback from physicians? I imagine this is a good example for physicians who maybe weren't involved in the clinical trials to get some experience with Ducryctoban.

Bernd Mody, CEO

Yeah, that's sort of still early stages there to really get some meaningful feedback there. But as I said, the program has a lot of interest, and we're really happy about that to be able to provide that.

Maggie Beller, Head of Investor Relations

We're careful on giving guidance around that because it could be considered preapproval promotion since it's a medically driven program. will say that, as Bernd said, we obviously opened it based off of interest that we received from physicians and KOLs. All of that is reactive in response to access challenges that people have. So that does give people the ability to treat their on-demand techs with do correct demand prior to us getting a formal approval, but it's a medically driven program.

Max Skor, Analyst — Morgan Stanley

And then maybe we have about six minutes left. Let's touch on, you have another clinical trial, CREAT currently ongoing? How is that going? When can we expect data? And what does that opportunity look like?

Bernd Mody, CEO

Yeah, so that's the CREAT trial, just to remind that that is a foreign indication called acquired angiotema. So that is another form of angiotema that's due to underlying disease. So basically it costs the C1 deficiency with the same angiotema symptoms that you see in hereditary angiotema. so basically follows the same pathway. That's about 10% of the patient population right now. There's nothing approved for that, and in our discussions with the FDA, we have received encouragement to pursue that patient population, and we see the potential for underdiagnosed situation there because, as I said, it's because of underlying disease, and these are patients that are referred by the other treating physicians when they're able to figure out what's going on with these attacks that these patients have. So with the potential then with an approved approval in that indication, then that could uncover the broader opportunity. And our plan with that trial, which is ongoing and also on track, and we've got the top-line readout in Q1. next year, and our plan is then to combine the data of that study with the NDA filing for PROFI, and with that, we then may have a potential upside subject to regulatory agreement. The fundamental goal is to, with that data together with the other data, to get the broad label for bradykinin-mediated angioidema, not just type HAE 1 and 2 and 3. And that would be differentiated from any offer. That's another way of differentiating. It could have benefits for prescribers. It makes life easier. It could have benefits on the payer side. And so basically you have a therapy that covers all forms of angioidema. That makes it that there's no other company that really has that.

Max Skor, Analyst — Morgan Stanley

Okay.

Bernd Mody, CEO

Two more questions. first cash runway can you talk about your financials yeah I'll leave it there yeah so the guidance on cash is mid 28 that is a relates to what we refer to as the base case and the key core focus of the company which is to launch the quick demand in the US both in Prophi and on demand and we are now of course looking at the other different points of potential other capital raises, and we haven't made any decisions there yet. We've taken sort of an opportunistic view when it comes to the potential equity. We also have other forms of financing available to us. I also get closer to the commercial profile. And with the financing that we did a couple of months ago, So that gave us that financial flexibility, so we have a lot of options to take the next step in our capital formation.

Max Skor, Analyst — Morgan Stanley

Then maybe in the last two minutes, over the next six to 12 months, what are we going to hear from Favaris outside of potential approval, launch expectations? Should we expect more data? We talked about the CREAT study, longer-term data, LOE, et cetera.

Maggie Beller, Head of Investor Relations

We've submitted a, or we plan to submit a late-breaker abstract for the college meeting, that's ACAAI, that's in the first week of November, where that will be our first opportunity to present our Chapter 3 data to the medical community. So we're excited about that. Our manuscript is shelled out and has data starting to be dropped in it right now. We hope to publish that for Chapter 3 in the first quarter of next year. Also, as Bernt mentioned, as part of our NDA package, we'll be cutting our open label extension data. So when that data cut occurs, we'll either be including that as a poster or a manuscript. So we'll decide on how we're going to disclose that open label extension data. Bernt spoke about create part one data. We have hopefully an approval with a PDUFA target date of April 23rd. and then, of course, any sort of feedback that we get from the FDA regarding our NDA submission for prophylaxis.

Max Skor, Analyst — Morgan Stanley

Okay, and mid-cycle for the on-demand, should we expect an update around that?

Maggie Beller, Head of Investor Relations

Yeah, we're probably not going to give incremental updates on our ongoing interactions with regulators, but as we've disclosed, conversations are happening, questions are being addressed, so things are going along as planned.

Max Skor, Analyst — Morgan Stanley

Okay, great. Well, it sounds like everything's on track, and thank you very much for attending today. Really appreciate it.

Bernd Mody, CEO

Yeah, thanks, Max.

Max Skor, Analyst — Morgan Stanley

Great to be here.

Maggie Beller, Head of Investor Relations

Thanks, Max.