Egetis Therapeutics AB (publ) (EGTX) Earnings Call Transcript & Summary

Thank you all for being here. I'm James Machen from the Morgan Stanley team. I'm delighted to have Nicklas from Agetis Therapeutics here today. We'll walk through a few of the ins and outs around the business over the next 30 minutes. I think starting point, maybe for the benefit of those that haven't come across the company before, maybe a short intro to yourself and Agetis and the difference you're looking to make for patients.

Thank you very much, James, and a privilege to be here. So for those who haven't met before, my name is Nicklas Westerholm. I'm the CEO of Agetis Therapeutics and has been so since the formation of the company. Short background, chemistry by trade, started off my career in AstraZeneca where I spent close to 20 years of my professional career across a number of different business areas, mainly internationally, but late-stage development for 10 years, global supply chain and manufacturing for a couple of years, privileged to work in finance and investor relations as well at that headquarters in London, responsible for AstraZeneca's business in Japan, and the last role I had within AC was late-stage development responsible globally in the cardiovascular metabolic disease. So shortly about the company, and rightly so, James, as you mentioned, Agetis Therapeutics is probably a company, especially here in the U.S., that's been somewhat flying under the radar, driven by a number of things. One, it's actually recently created, as I mentioned before. The company came about only back end of 2020, so we've been in our current shape and form for close to six years. The purpose of the company has been very clear from the outset. It's focusing on rare diseases only. Late-stage development is our sweet spot, the ambition and ability to commercialize ourselves in Europe and the U.S. and provide access in the rest of the world through partnerships. So that's.

in a nutshell. Okay, great. Let's maybe turn to the lead asset within the business. Clearly some news flow coming over the coming weeks, which we'll get to. assets for an indication called MCT8 deficiency. Let's start there. So what's the disease, what's the unmet need, and how does this present with inpatients? Sure. And again, for the audience, I appreciate that MCT8 deficiency for many is unheard of. And that's not a surprise. It's an ultra-orphan condition where the gene was only discovered back in 2004. So it's actually relatively, within the context of life science, a new disease. So MCT8 deficiency is a mutation in a thyroid hormone transporter. So this is a thyroid hormone signaling disorder. MCT8 actually stands for monocarboxylate transporter number 8. It's an X-linked disorder, which means that it affects mainly males. Incidence levels are estimated to be around 1 in 70,000. It's a really severe and debilitating condition, and the manifestation translates into two severe clinical phenotypes in actually one patient. So in different compartments in the body, you have either too high or too low thyroid hormone levels. So in the peripheral system, you have too high thyroid hormone levels, i.e., thyrotoxicosis, that translates into severe stress on the cardiac system. In the central nervous system, you get more or less null or very limiting exposure to the thyroid hormone, which leads to lack of neurocognitive development. So you have a very intriguing patient phenotype divided into two specific phenotypes in one patient. As I mentioned before, this is an ultra-rare condition with 1 in 70,000 males being affected by this. Up until now, no treatment has ever been approved for this condition. Our product MCT8 or the pro-drug active substance tiratricol was the first and only approved treatment in the European Union last year on the 12th of February, and here in the U.S. we are, of course, very, very excited about our upcoming PDUFA date on the 28th of September this year, so only 12 days away. Indeed, indeed. And I guess therapy has priority review, has breakthrough, has fast track, has orphan designation, so clearly well supported by the agencies to date. The focus will be on the PDUFA, the approval over the coming weeks. Can you provide a short overview of the data that supported that approval and the work that you've done to date to get to this point?

Yes, and to put this into context as well, the severe thyrotoxicosis for these patients leads to a median life expectancy of 35 years, as well as 30% of the patients die in early childhood. So I think that's important to put it in context with the clinical data we have generated. So we have, in my humble opinion, an unusually large and robust set of data generated over a number of years, being an ultra-orphan condition. So we are actually three prospective clinical trials, and we also have three real-world evidence studies that has formed a part of the NDA, the New Drug Application here in the U.S. And what we have seen across the board, the hallmark of this disease is elevated T3 levels. What we have seen across the board, the prospective, as well as the real-world evidence studies, has been our ability to normalize T3 levels in the peripheral system, that then translates into clinical benefits on the cardiovascular system, such as blood pressure, heart rate, et cetera. And why is that important? I mentioned median life expectancy of 35 years. The main course of mortality in this population is cardiovascular driven by sudden cardiac death. So we see substantial improvements in all these clinical parameters I referred to. And most lately, even though it's not fully published, it has been an abstract out in 2024, which we could refer to as the survival study, which is by far the largest patient cohort ever gathered information around for this condition. It included 612 patients in total, where it illustrates also the ability for a survival benefit, substantial survival benefit in patients treated with our drug versus untreated patients. I think all in all, we have a very robust clinical data package. It has been recognized by the agency from a breakthrough therapy designation perspective, fast-track priority review, et cetera. And we are, of course, very much again looking forward to the PDUFA data in the coming weeks. Indeed.

Indeed, indeed. You mentioned no other therapeutic options. Is there anything else emerging for the indication? And how do you think MCT8 will really differentiate and make that difference for patients?

No, and I think this is an important point, right? So today there are no other active drugs, sponsored drugs in clinical development. So I think that's important to recognize. Nothing is out there in clinical development by a sponsor. We know there are a few investigator-initiated studies locally in certain countries, but nothing in the context of potential competition in the mid- to long-term distance is something we see in the future. So I think from a caregiver, from a patient perspective, obviously just bringing a treatment to provide some hope for these devastated patients, but also the families and the caregivers is very much appreciated. We have had up until the approval in the European Union, but also now here in the U.S. have had a very comprehensive managed access program or EAP program with more than several hundreds of patients treated globally worldwide without drugs even before approval. I think all in all it's good. There's very little competition out there and there's no other treatment available. With the exception, of course, in general, if you have some symptomatic treatment for, if you have a seizure, you get an epileptic treatment, anti-epileptic treatment.

Great, makes sense. Focus will then clearly, and we'll go through hopefully the approval, focus will turn then to the commercial launch. Can you frame a little bit around how prepared you are for that, how quickly can we expect the product being brought to patients? And what's the footprint that you've put in place?

in the U.S. so far? Sure. And I think with, of course, the progress on the regulatory front in the U.S., being a small Swedish biotech, a biotech company, not having unlimited means. We have been very careful until we have clarity on the regulatory milestones and potential PDUFA date. But obviously, with the visibility we gained towards the back end of last year, to based on a very successful pre-NDA meeting, we started to invest exponentially, I would say, in the U.S. commercial and medical affairs infrastructure. So today we have 23 employees in place in the United States alone, only focusing on commercial and medical affairs. Key focus is really in two buckets, right? One is the medical affairs side of things together with the key account managers, to make sure that we launch readiness, educate the physicians, make sure that we have patient support programs in place to ensure a smooth transition from EAP to commercial product. The other bucket, of course, is access and pricing. So those are the key areas we've been investing in. And so today, as an example, we have four key account managers up and running in the different territories. We have four regional medical affairs directors in their respective territories to be out and about and really preparing for launch. I think, in our case, in essence, last week or two weeks ago in Boston, we had with the whole team a dry run, launch readiness meeting where we went through everything from patient flow, diagnostic journey, product flow, et cetera.

my humble opinion, we're in good shape. Okay, great. How do you then, so clearly field force is ready, clearly you have the patients that are on the EAP already, clearly also a number of other patients that you're aware of. Any commentary on how we can expect that uptake over time and and how you and and also any learnings from Europe as to how that has translated to more patients starting to be identified post the launch?

Thank you, James. That's a really good and important question. I think I'm sure everybody appreciates that short-term, and when I refer to short-term, it's zero to six months after launch. The key focus there would be to have the 60 patients we today have on an EAP program, to facilitate a smooth transition and continuity of care to commercial product. These 60 patients are spread across 17 EAP sites. So obviously it's a decent site to manage within a small team like we have. So the key focus short term is really to facilitate that transition to commercial products with the caveat, of course, that the product has to prove. And that, of course, then will realize short-term revenues. And the expectation, without giving any guidance, if you look at other companies out there that's going through the same journey in the ultra-rare disease space, one would envisage that within roughly six months all these patients, the 60 patients, have been transitioned over to commercial products. That to a side, we have another 50 plus patients also identified in the United States, but are treatment naive today, so not on the EAP program. That of course also have priority to now cater, go broader than the 17 EAP sites to cater to their needs as well and ensure that the NHCA treatment once the product is approved. And then last but not least is to further work on, as we have done in Europe, driving further disease awareness and facilitate further patient identification. Because coming back to the incidence numbers, 1 in 70,000 males are born with this condition. You need to account for median life expectancy of 35 years. That gives you a ballpark theoretical figure of the number of patients in the U.S. that amounts to roughly 1,000 patients. Then it's all about finding these patients.

Okay, great. Final pass on the U.S. I believe, and correct me if I'm wrong, eligible for a pediatric voucher. Sorry. How does that play into your plans? And clearly there's been some good value in those recently, given the recent sale transactions that we've seen. For sure.

That's a very important part of our future financing plans. Of course, we just recently, a couple of weeks ago, reported our quarter two results. And we reported roughly $40 million cash at hand. Of course, then with us being granted a rare pediatric disease designation, that makes us eligible for a priority review voucher. It's not unreasonable, James, as you said, that a company like ours will most likely monetize that as soon as possible. Worthwhile noting is that 50% of the net proceeds of monetization of a priority review voucher for our company goes to the founders of the company, so 50% to Agetis as an entity. But as you said, if you have sales values of, let's say, $180 million or $200 million, half of that to us, that will fund the reminder of the launch with even further investments during 2027 in the organization in the U.S. and takes us to profitability.

Great. Let's transition from U.S. to outside the U.S. You cited the German launch previously. Any updates on where you stand now with that launch and what really should we focused on as we think about other markets across Europe over the coming months? Sure. So here we need to be very clear, because we have to navigate this carefully, of course, when it comes to U.S. and Europe and launch timelines in relation.