Hi, good morning, everybody. Welcome to day three of Cantor's Global Healthcare Conference. I'm Kristen Kluska, one of the analysts. I'm joined by my colleague Ayan Hussein on stage. We're very happy to have the Egetis Therapeutics team on stage. This is a company we recently launched coverage on over the summer. We're very excited. Joining me is Nicklas Westerholm, the CEO, and Anny Bedard, the President of North America. Thank you both so much for making the trip here. We really appreciate it.
Thank you. Thank you for having us. It's a privilege.
Yes, of course.
Thank you.
So maybe just to kick our discussion off, do you mind providing us with a high-level overview of the company?
Yeah, sure, and I'll keep this very short, and I think it resonates, Kristen, what you had in your initiation report, actually. Egetis Therapeutics is a company that's somewhat been flying under the radar, and especially for the U.S. investors, I guess. This is driven by a couple of factors. One, the company actually came about only back end of 2020, so we've only been in our existence for six years. Focus initially, since we're based in Stockholm with the headquarters there, listed at Nasdaq, has been drug development and the exposure there. Over the last couple of years, I think we have started to more invest in the U.S. with presence here, but also engaging with analyst and investor community. The focus of the company is very simple, late-stage development in the rare disease space with the ability to commercialize ourselves in U.S. and Europe.
Our lead candidate has been and is still tiratricol, with the brand name Emcitate in Europe for the treatment of the ultra-orphan condition, MCT8 deficiency. That's Egetis in a nutshell.
Okay. Thank you so much for that. MCT8 deficiency, a lot of our audience here may be new to the condition. Can you walk us through how these thyroid hormones typically move and interact throughout the body? What then happens in this syndrome where levels become too low in the brain and too high elsewhere in the body?
Sure. That's not surprising that people haven't heard about MCT8 deficiency. It is definitely an ultra-orphan condition, a very much ultra-rare disease. MCT8 deficiency is a thyroid hormone signaling disorder. MCT8 is monocarboxylate transporter 8, so it's a mutation on the transporter. It's an X-linked disorder, which means that it mainly affects men, and the estimated incidence levels are roughly one in 70,000 males that are born with this condition. As you mentioned yourself, Kristen, it actually comes with very interesting disease characteristics with two clinical phenotypes, hypothyroidism and hyperthyroidism. So in the same patient, you have simultaneously too high thyroid hormone levels in the peripheral system and too low thyroid hormone levels in the central nervous system, which actually gives a very intriguing phenotype for the overall patient. We have thyrotoxicosis as well as lack of neurocognitive development within one patient.
That is how one could summarize MCT8 deficiency in short.
Okay. Thank you. When you first sought out clinical development for this indication, how many patients were identified in the U.S. and EU5, and how has this changed since you've really progressed development, had more boots on the ground, and what is your expectation of how it could grow over time?
Well, that is the billion-dollar question, right?
Yes.
As for many rare diseases, it's difficult to predict exactly how many patients are out there. But starting off, when we started to work actively on this drug, which was back end of 2020, as I mentioned before, we knew about 20 patients diagnosed with MCT8 deficiency in the U.S. and somewhat higher in Europe. The reason why I can't give you a number in Europe is that we are very much driven by the data regulation protection.
We're not allowed to know the exact patient with name and identity. But very few patients were known at that point in time, which is not unreasonable, recognizing that it's a relatively new disease. The transporter protein MCT8 was discovered in 2004. We are the only company ever worked within the prospective clinical trials. That, in combination with fairly low disease awareness, leads to that not at that point in many time, many patients was diagnosed. Having said that, though, with the team in place now, in total have 60 FTEs, or 60 employees within the company, where 1/3 is based in the U.S. We have really gained traction. We started to invest incrementally over the last, I would say, 9- 12 months, especially in the U.S. So today, we have 60 patients, 6-0 patients under EAP program in the U.S. alone.
We know that more than 100 patients are diagnosed with this condition. We continue to drive disease awareness to further identify patients. I think the 60 patients in U.S. is key, of course, at the point of a potential approval and launch to secure both continuity of care, but also realize the revenue short term. I think, I don't want to use the word unprecedented, but it is an unusually large number of patients being on an EAP for being an ultra-orphan condition.
Okay. Thank you. I know that patient identification and diagnosis, of course, is going to be a key priority for you looking forward. What are some of the initiatives that you believe could improve diagnosis rates, especially if a treatment ultimately becomes available for patients?
Very much, of course, looking forward to the PDUFA date on the 28th of September. As I mentioned, I'll hand over to Anny Bedard, is that short term, the focus of the U.S. organization is really continuity of care for these 60+ patients on an EAP program. Making sure that they transition over to commercial product. Of course, ensuring the patient continuity there for treatment, but also to realize revenues as soon as possible. We have several activities and initiatives to further focus on patient identification. I'll hand over to Anny to elaborate a bit further on that.
Yes. So definitely, patient identification is critical, and it is at the core of our initiatives. In addition to our teams in the field from a medical and commercial side, we also are focusing on maximizing the efforts. One key area that we are focusing on is through analytics. Developing analytics algorithms in order to identify the patients who might have a potential to have MCT8 deficiency in larger patient population. So analytics is one of the key approach that we are focusing on. We are also focusing on partnerships with commercial genetic laboratories in order to tap into their database and any new patient that is identified to be flagged. We are working also with organizations who have registries, organizations particularly who are focusing on conditions that are often misdiagnosed or confused with MCT8 deficiency, and we can find patients there.
Of course, we are tapping the standard social media, patient advocacy organization. So we are tapping into different channels in order to maximize patient identification.
Okay, thanks. Let us talk about some of the data and the regulatory process. So you have one of the most robust packages in terms of trials conducted and endpoints for a rare disease. Which metrics are going to carry the most weight for treating physicians and patients, and what did you see with MCT8?
Sure, and thank you for acknowledging that, Kristen, because I agree. We have a very robust and comprehensive data package for being an ultra-orphan condition. Just for the audience, we have three prospective clinical trials that we have been executing on over the last couple of years. It is the Triac Trial I with 46 patients, Triac Trial II with 22 patients, and the ReTriAct study with 15 patients, including placebo control. So that is the prospective clinical studies we have. Then we also have a robust set of real-world evidence studies as well, which is the EMC Cohort Study, six years follow-up on patients being treated. A survival study where high level results has been published in an abstract, which is of course very interesting, and that was what we got the Breakthrough Therapy designation last year granted based on.
Also we have data from the EAP program in the U.S. that was forming a part of the dossier as well. If you take a step back then and what we have seen and what carries most weight, which was your question, Kristen, was, of course, the hallmark of the disease is elevated T3 levels, right? That's something we have seen across the board, across all the studies. The ability to normalize T3 levels very fast and also durable up to six years. Then that translates into some very important improvements in the peripheral thyrotoxicosis side. So we have clinical benefits on heart rate, blood pressure, improving in weight, et cetera. That then also theoretically, as we have seen in the survival study abstract, translate into improved overall survival.
Because I think we need to put this disease in context, that the median life expectancy is 35 years for untreated patients, together with 30% of the patients dying in early childhood. The ability to then extend and improve life, of course, is important.
Yeah. Thank you for that. So you now have a few quarters under your belt in Europe, particularly in Germany, where you recently concluded the pricing negotiations. What's been the experience so far, and how receptive has the community in Germany been?
Well, I think the product has been in the markets in Germany since 1st of May. That's when we launched it, right? We are roughly a year and a bit into the launch. What we initially saw was that a very quick conversion. If I draw parallels to the EAP program, we had a managed access program available in Europe. We saw the conversion of all those patients, or transition into commercial product within the first three months, and that was a very strong sign for us. In addition, we of course have had new patients being initiated to treatment that was diagnosed. More importantly, I would say for the long-term prospects, that hopefully also translates into the U.S., that when a drug gets approved in an ultra-orphan condition, the interest from physicians are actually increasing.
What we have seen is that the number of diagnosed patients and identified patients in Germany has increased by 100% since the point of launch, and I think that is a very important KPI. Not all of them are on treatment. It's a very important KPI for the mid to long-term prospects.
Okay, thanks. I can respect that you are in active review in the U.S. today with the PDUFA a little over two weeks away. What's the latest you're able to share with us about how this is going?
Well, that's the billion-dollar question, right? Isn't it, Kristen? Joke aside, I think this is, of course, nothing I can comment on.
Of course
Since we are in an active dialogue as part of the NDA review process. I will try to triangulate it a bit for the audience and to get some further insight. We are a publicly listed company. If our plans would have changed, i.e., we are working towards a PDUFA date on the 28th of September . If that would have materially changed, we would have to announce it because that would have been material information.
Yeah.
We haven't announced anything. What I can say, though, is that it's been a collaborative discussion with the agency so far. Very much so. We have, of course, if you look at the generic stipulated timelines for an NDA review under Priority Review, one, it's reasonable to assume that we have concluded the mid-cycle review meeting, the late-cycle review meeting. I am sure you appreciate that based on the stipulated timelines 30 days ahead of a PDUFA date, label negotiation starts. It's not unreasonable to think that that's where we are in the process.
Okay. We're really excited. Wishing you all the best in the 17 days from now or whatever it is. But appreciate, appreciate those high-level comments. What is the latest in terms of the review for the manufacturing process, and can you also remind us what your IP portfolio is?
Yeah. Again, I cannot give you any details, right?
I got to try and ask.
Yeah, points for trying, right? I would have done the same if I were you. But joke aside, I think, again, if something materially would have diverted from the stipulated timelines, we would have to communicate that. So the interpretation is that we are on track when working together with the FDA towards the PDUFA date on the 28th of September , 17 days ahead.
Yes.
When it comes to IP, and this is an important aspect and something we're really proud of. Initially, when we started development on this molecule, we were very much driven from an exclusivity perspective of the Orphan Drug Exclusivity, which of course, is a strong protection, 10 years in Europe and seven years in the U.S. Obviously, with the formulation development we have done over the last couple of years, we've been able to submit a patent application to the USPTO last year, and that patent was granted early spring this year. That patent encompasses composition of the product, including excipients and dosing together with method of use. And this is a patent that extends to 2045 and it's Orange Book listable as well. So I think that adds another layer to the protection from an IP perspective.
Okay, thanks. If you are approved, how should we be thinking about the current EAP and the process to move patients to commercial therapy? I would imagine it could be more rapid than if you were just diagnosing those patients and adding them at that point.
Sure. As I mentioned as part of the introduction, this is one of the key priorities for our U.S. business, and I invite Anny Bedard to comment because I know you have been incredibly busy with focusing just on this topic.
Yeah. So definitely the patients who are on the EAP are critical for us, and that's the main focus. We've been actively working with the centers who are managing these patients to make sure that everyone is ready in the eventuality that we get an approval in 17 days. That everyone knows what to do in order to ensure the continuity of care for these patients. We've been also putting in place the patient service program that will facilitate that and working proactively with the payers as well. That will be the main focus for us and to do this transition as quickly as possible. Then we also have some patients that are identified, diagnosed, but not treated, as Nick mentioned. And that will be the second immediate priority for us in order to secure the foundation for the launch.
Then build on the activities that we have for diagnosing new patients and making sure that we continue to feed the pipeline, if I can say.
Okay. Then collectively, between your own footprint and the global partnerships, how should investors be thinking about the total opportunity for MCT8 in MCT8 deficiency?
Well, as I mentioned in the outset, the ambition with the company was to build a sustainable rare disease company with the ability to commercialize ourselves in U.S. and Europe, and we have established a footprint there. We today have 60 employees covering Europe and U.S. in total. Obviously, we are broadening access, and this is important, providing access to patients worldwide. Initially, we have now signed partnerships for Central Eastern Europe together with Turkey, with Er-Kim. And then we have a partnership also with Taiba Rare for the Gulf region. We most recently signed a supply agreement for New Zealand and Australia. So we are really broadening access, right?
Having said that, though, there's plenty of more things to do. We have the development and commercialization agreement in Japan, which is very exciting, where we are looking forward to a submission of a MAA to the Japanese PMDA early next year.
That to a side, we are continuing to evaluate additional partnerships for other countries as well as regulatory submissions in the likes of Canada, U.K., partnerships in LATAM, amongst others. So it's more to come there. But if you take a step back in considering the overall opportunity, I think, of course, the value here is very much driven from the U.S. And that's why it's our key priority going forward, of course, in parallel with providing access to the drug worldwide. But the key priority is the U.S. market. We believe that if you think about the estimated incidence levels that are out there in the literature, and as for any rare disease, one can't guarantee that, but that cites one in 70,000 males are born with this condition, median life expectancy untreated 35 years.
So if you do the math there, one will come to a number around 1,000 patients in the U.S. alone, then one can argue how many of these will we find. And as Anny mentioned, we only know more than 100 patients today, whereas 60 of them are already being treated. Some good other proxies there is Germany, as I mentioned, after the drug got approved. We actually doubled the amount of diagnosed patients that we are aware of within a year. So the opportunity is there, and it is substantial for sure.
Thank you. All right. Moving on to your second indication, RTH-beta. This is a disorder that similarly involves this paradoxical coexistence of thyroid hormone excess and the deficiency. What are the key manifestations of this indication, and what is it about the underlying biology of the drug that makes it suitable here?
Well, it's equally interesting, the RTH-beta opportunity here for us as a company, and that's driven by a number of factors. This is a mutation on the receptor rather than the transporter. What has materialized over the last couple of years was initially a natural history paper that was published two years ago, which now actually illustrates that patients with RTH-beta also have a reduced median life expectancy of around 11 years. You also have a lot higher risk for MACE. You have a sixfold higher risk for heart failure, et cetera. The physician's appreciation for a need of treatment for this condition has materialized substantially. If you think about the hallmark of this disease, in contrast to MCT8 deficiency, where it's elevated T3 levels, here you have both elevated T3 and free T4 levels.
Here we also have a managed access program in place with more than 50 patients, 50 patients, being treated with tiratricol, our drug. What we have seen there was a publication out in November last year, Moran et al., illustrating eight patients, all of them being normalized both from a T4 and T3 perspective. That then translates into benefits on the cardiovascular system. This is a very much more heterogeneous disease. You also have patients that are asymptomatic. The prevalence, it's not quite a lot more common than MCT8 deficiency. It's still a rare disease, but it's more common. The estimates here are 1 in 20,000 or 1 in 40,000 in between are affected by this, both genders. Again, substantially more potential patients out there.
What we see from a symptom perspective is that early stage in life is more ADHD type of symptoms, whereas the older patients then, where you have had incremental T4 and T3 stress on your system over a number of years, you start to see the cardiovascular elements materialize, like blood pressure, heart rate, et cetera. That then translates into higher risk factor from a morbidity and mortality perspective. Here we are very excited to progress this as a next opportunity.
What's interesting here is that tiratricol is included in the guidelines. Why is that the case, and what evidence is there in the patient population?
No, this is quite intriguing, and I think for the audience, you are referring to the European Thyroid Association guidelines that was issued back in 2024. This is quite interesting. So this was before MCT8 got even approved for MCT8 deficiency. Interesting enough is that exactly as you said, these guidelines stipulate that both patients diagnosed with MCT8 deficiency as well as RTH-beta should be treated with tiratricol as the first-line treatment. Which is, in my humble opinion here, somewhat unprecedented that they recommend at that point in time, unapproved drug for the treatment.
Yeah. Agreed.
The evidence, coming back to what I just mentioned, is that, one, we see improvements in the 50 patients, or we, the physicians, see improvement in the 50 patients on there, plus patients in the managed access program. Together with what we have seen in the public domain that was published, as I said, Moran et al., where we will be able to normalize T4 and T3 levels for all these eight patients that were included in the cohort. So very exciting now to move forward with this as an indication expansion opportunity, which from a commercial standpoint, more or less according to our own assumptions, is on par with MCT8 deficiency.
We understand that you are still working on the trial design aspects, and you intend to meet with the agency. Just from a high level, can you tell us some of the endpoints and then the key measures that would be most important?
Yeah, sure. I think just again for the audience here, we have called out that we are diligently working with finalizing the clinical development program and the target product profile. We actually held an advisory board last weekend at the European Thyroid Association, including key opinion leaders both from the U.S. and from Europe. We focused on clinical trial design. It is very evident, of course, that we are focusing on one study. Pivotal study obviously needs to be aligned with FDA and the EMA, but one study to get the drug approved also in this setting, RTH-beta. Without going into any details, it is reasonable to assume that a key endpoint would be T4, T3, as we have seen for MCT8 deficiency. A key endpoint there was T3, obviously. That is something that resonates with the regulatory agencies.
And then, of course, complementing that with objective measures in the cardiovascular area. But we'll keep the market posted the closer we get to regulatory interactions on this.
Very excited to hear more on this.
The ambition, sorry, I should mention that, is to start the pivotal study already next year.
Great. Thank you for that. Just the last few minutes or so, what do you think is the most misunderstood or undervalued component of Egetis valuation, and why should the investors consider looking at the company now?
Well, I tend to refrain to comment on valuation of a company, right? Because it's the market that values a company. But I can talk a bit about the future prospects, right? What we see only with MCT8 in MCT8 deficiency is a substantial commercial opportunity, right? Obviously, we don't give guidance on price points, but you have analogs out there that illustrates annual treatment costs in the U.S. between $750,000 to up to $1 million. It's reasonable to assume that MCT8 potentially could have those annual treatment costs as well. If you think about the number of patients, as Anny mentioned, it's very clear. Already today, we have 60 patients, 60 patients, being treated under the EAP program with quite a few other patients diagnosed, in total over 100.
So just the short-term prospects by transitioning from EAP to commercial product from a revenue perspective is very attractive, especially for a company like ours, where we are running with a very tight cost base. So the future prospects are good. Then, of course, also as Anny mentioned, further building on this, identifying further patients. We're not just seeing a good launch. We're seeing mid to long-term growth prospects, definitely only in MCT8 deficiency. Then if you add on top of that the RTH-beta opportunity, it's very attractive.
And we would totally agree with that. So thank you so much, and we appreciate you guys being here and your support.
Thank you.
All the best in the next few weeks.
Yeah, fingers crossed, right?
Very exciting.
Thank you.