Welcome to Egetis press conference regarding the FDA approval of MCT8. During the questions and answers session, participants on the telco are able to ask questions by dialing pound key five on their telephone keypad. Now, I will hand the conference over to CEO Nicklas Westerholm. Please go ahead.
Thank you very much, operator. Thank you everyone for joining us today. For those I haven't had the privilege to meet before, my name is Nicklas Westerholm, and I am the CEO of Egetis Therapeutics. Today is truly an historic and transformative day for Egetis with the approval of EMCITATE for patients with MCT8 deficiency in the U.S. But with me also today, I have our Chief Medical Officer, Tiago Nunes, who will remind us about the severe condition of MCT8 deficiency. We also have Christian Sonesson, Vice President Product Strategy and Development, who will elaborate about our robust data package that form the basis on the NDA and the USPI, the so-called label. Last but not least, we have Anny Bedard, President of our Egetis in North America, who will elaborate on our launch readiness. Next slide, please.
We will also leave ample time for questions and answers at the end. Late last night, we announced that the U.S. Food and Drug Administration has approved EMCITATE for the treatment of peripheral thyrotoxicosis in adults and pediatric patients with MCT8 deficiency. This approval represent the first and only FDA-approved treatment for this rare, severe, life-shortening genetic disorder and marks an important milestone for patients, families, clinicians, and the broader MCT8 community. For families affected by this condition, the journey to diagnosis and care is often difficult and uncertain. Today changes that. For Egetis, this approval represents far more than a regulatory milestone. It is the culmination of a comprehensive development program that has evaluated EMCITATE across multiple clinical studies and patient populations. It is a validation of years of research and development together with our key collaborator, the Erasmus University Medical Center, or EMC, from Rotterdam.
This also marks the beginning of a new chapter for us as a company, as we transition fully into a commercial stage, rare disease company in the world's largest pharmaceutical market. Our focus now turns from approval to commercialization. We have been preparing for this moment and have established a commercial and medical affairs infrastructure necessary to support patients and healthcare providers from day one. Our goal is simple, to help eligible patients to gain access to EMCITATE as quickly and seamlessly as possible following approval. We are also pleased that the FDA granted Egetis a Rare Pediatric Disease Priority Review Voucher in connection with the approval. The voucher represents a very important financial asset for the company and will provide additional flexibility as we continue to execute our mid to long-term growth strategy, building a sustainable rare disease company.
As outlined in today's press release, we expect to evaluate opportunities for the monetization of the voucher during the fourth quarter of this year. Today's achievement also reinforces the strength of Egetis' rare disease strategy. We remain focused on developing and commercializing therapies that address significant unmet medical need for small patient populations, where meaningful innovation can have a profound impact on patients' lives. With that, I will now hand over the call to our Chief Medical Officer, Tiago Nunes, who will discuss the devastating condition of MCT8 deficiency.
Thank you, Nicklas. MCT8 deficiency is a rare, severely debilitating, life-shortening condition. It is caused by mutations in the SLC16A2 gene located on the X chromosome, hence its predominance in males. In MCT8 deficiency, impaired transport of thyroid hormone leads to low T3 levels in the brain and high circulating T3 levels. This disease is characterized by impairment in neurodevelopment and systemic metabolic and cardiovascular disturbances with increased morbidity and mortality. There is a median overall survival of 35 years of age, and it is expected that one in three children with MCT8 deficiency will not survive to adulthood. Cellular differences in MCT8 dependence lead to simultaneous thyroid hormone insufficiency in the CNS, central nervous system, and persistent peripheral thyrotoxicosis. This is a clinical state in which tissues are exposed to and respond to excessive thyroid hormone.
Thyroid hormone insufficiency in the central nervous system occurs because T3 cannot enter due to MCT8 dysfunction. Entry of T3 via alternative transporters leads to peripheral thyrotoxicosis in the periphery in organs like the heart and the muscle. In MCT8 deficiency, clinical presentation results from thyroid hormone insufficiency in the central nervous system and persistent peripheral thyrotoxicosis. These patients present high T3 levels, the active hormone, low to normal T4 levels, the storage hormone and TSH, which is the control hormone, which is normal or slightly elevated. SLC16A2 genetic testing confirms the diagnosis. Impairment in neurodevelopment, hyperphagia, and failure to thrive are characteristic. Untreated persistent peripheral thyrotoxicosis that may include tachycardia, cardiac arrhythmias, and hypertension are associated with elevated risk of major cardiovascular events and cardiovascular abnormalities. Sudden death has been reported as frequent cause of mortality and is likely linked to underlying cardiac abnormalities.
The treatment of MCT8 deficiency is multidisciplinary, and one of the main focus of treatment is the management of failure to thrive. Most affected patients are underweight with body weight for age showing progressive decline over time. Undernourishment can weaken the immune system, leading to difficulties fighting infections. Failure to gain weight is associated with poor prognosis and increased risk of death in this population. Up to recently, there was no approved drug for the management of MCT8 deficiency. The approval of EMCITATE for the treatment of peripheral thyrotoxicosis in patients with MCT8 deficiency has changed that. EMCITATE is a thyroid hormone receptor agonist that enters MCT8-dependent cells independent of MCT8 transporter. EMCITATE replaces T3 in MCT8-dependent tissues and normalizes thyroid hormone activity across tissues. Clinical trials show that MCT8 reduces serum T3 levels and improves clinical manifestations of thyrotoxicosis, which are associated with increased morbidity and cardiovascular mortality.
I now want to present and introduce Christian Sonesson, Vice President, Product Strategy and Development, to take a closer look at the clinical data supporting EMCITATE treatment in MCT8 deficiency.
Thank you, Tiago. Next slide, please. Before I turn to the label itself, let me start with the clinical evidence submitted in the NDA. This package rests on two pillars. First, three clinical studies. Triac Trial I, which established the core effect of EMCITATE in lowering T3 and improving the clinical markers of peripheral thyrotoxicosis. ReTRIACt, the randomized withdrawal study that delivered confirmatory placebo-controlled evidence that this T3 lowering effect is directly attributable to EMCITATE. Triac Trial II, which added safety data at high doses in the youngest patients, together with three independent sources of real-world evidence. The Erasmus University Medical Center Cohort Study following patients for up to six years, confirming that the effect on T3 and clinical markers of peripheral thyrotoxicosis are durable over the long term. The EMC Survival Study, which examined treated versus untreated patients in a cohort of more than 600 individuals.
The U.S. Expanded Access Program showed that the real-world experience in the U.S. was consistent with everything we had seen in the clinical trials. From this clinical package, really two things stand out. First, the robustness of the data behind EMCITATE, which is unusually complete for an ultra-rare genetic disease. Second, the consistency of the results across different designs, different populations, and different settings. Building on what you heard Tiago just describe, the biological plausibility of the treatment effects seen in our clinical data has been key in the NDA package. Peripheral thyrotoxicosis is a well-characterized condition. Its link to cardiovascular and metabolic harm is extensively documented, and correcting the thyroid hormone excess is known to reverse it. With the EMCITATE lowering the elevated T3, we see the expected clinical benefits also in patients with MCT8 deficiency. The clinical data in the NDA package don't stand alone.
They sit on top of well-established biology. Every data we have move in the same direction, and that coherence is what underpins our confidence in the clinical package. Next slide, please. Turning to the label itself. Key here is the broad indication statement covering all patients with MCT8 deficiency, saying that EMCITATE is a thyroid hormone receptor agonist indicated for the treatment of peripheral thyrotoxicosis in adults and pediatric patients with MCT8 deficiency. With this approval, EMCITATE becomes the first and only approved therapy for these patients in the United States. The remainder of this slide gives a brief overview of the highlights of the USPI. The class black box warning, how the medicine is dosed and individualized, the dosage forms, the warnings and precautions, the contraindication, most common adverse reactions, and drug interactions. I won't go through each of these in details here.
The full approved prescribing information is the definitive source, and it's linked on this slide for anyone who would like to see the complete safety and usage information. Next slide, please. Here we see how the clinical data package translates into Section 14 of the USPI. In other words, which studies FDA considered adequate and well-controlled to provide the primary support of effectiveness. Two studies are included. ReTRIACt is Study 1, the randomized withdrawal study. It gives us two findings. First, the rate of T3 change, where total T3 rose 1.5 x faster on placebo than on EMCITATE during the randomized treatment period, with a P value of 0.034. Second, the need for rescue, where four out of eight patients on placebo met the rescue criterion of total T3 above the upper limit of normal, versus one out of seven on EMCITATE.
I would note the asterisks here that that single case was a participant who discontinued prematurely for reasons not related to drug and was counted as having met the criteria. What is also worth emphasizing is the consistency. Every patient randomized to placebo had a larger increase in total T3 than any participant who continued EMCITATE. Triac Trial I is Study 2, a 12-month open-label study where we saw a reduction in mean total T3 from 323 ng/dL- 118 ng/dL over a year. A change of minus 205 with a P value below 0.0001. Alongside that, key clinical markers of thyrotoxicosis improved. Systolic blood pressure down, heart rate down, and body weight for age Z-score up.
All three secondary endpoints moved in the direction of reduced peripheral thyrotoxicosis, which is positive to have acknowledged in labeling, given the importance of improvement on thyrotoxicosis in this patient population, as previously covered by Tiago. The EMC Survival Study was not included in labeling. It is important to remember that this study is a retrospective, real-world cohort study, and the scientific and regulatory perspective on rigor is slightly different. Ultimately, FDA considered that given the limitations of the retrospective real-world design, the study did not meet the regulatory requirements for an adequate and well-controlled study. But certainly, from a scientific perspective, we are confident that the EMC Survival Study is unprecedented, and the data have shown to be robust across a multitude of different sensitivity analyses.
Instead, in the short term, we look forward to seeing the EMC Survival Study published so that the scientific and clinical community can be informed about the study. With that, I leave the word to Anny Bedard, our President of North America, to cover the U.S. launch readiness.
Thank you very much, Christian. Today marks an important milestone for the MCT8 deficiency community and for Egetis. For this community, EMCITATE is the first and only approved treatment in the United States, and for Egetis, it marks our transition from a development stage company to a commercial rare disease organization. This is a pivotal moment with our focus now shifting to execution, enabling access, and supporting physicians and families to treatment initiation. Next slide, please. Our commercial readiness is grounded in real-world experience, extensive pre-approval preparation, and stakeholder insight. We identified more than 100 genetically confirmed patients, of which more than 60 have been treated with EMCITATE across 17 sites in our early Expanded Access Program. Evidence of an identified patient base, confidence in therapeutic effectiveness, early adoption, and physician motivation to prescribe following approval.
Our work with patient advocacy organization has raised the patient voice, built disease awareness, and shaped the caregiver education and support we're rolling out at launch. The community is informed and ready to act, which we expect will translate into faster identification and referral of patients. We engaged payer well ahead of approval, including the three largest pharmacy benefit managers, representing more than 80% of U.S. prescription claims and more than a third of state Medicaid plans. Feedback has been constructive, with payers recognizing the burden of the disease and the importance of appropriate access for eligible patients. We're confident in the team we've built. Their rare disease expertise, multi-launch experience, and discipline execution gives us conviction in our ability to deliver. Next slide. More than 65% of early access patients are managed across six centers, which gives us a concentrated place to start.
Supporting continuity of care is our top priority. Our team is prepared to work with physicians, and where they determine continued treatment is appropriate, help patients transition from early access to commercial supply. The same preparation applies to the remaining early access site. Every investigator and care team has been engaged multiple times by our medical team ahead of approval, and account-level activation plans are ready to go. Next slide. Persistent peripheral thyrotoxicosis is a central feature of MCT8 deficiency and is associated with systemic burden, medical fragility, and increased mortality risk. Until now, no approved treatment has been available. The price of EMCITATE balances four principles: access, affordability, the value of the medicine, and sustaining innovation.
It reflects the severity and life-shortening nature of MCT8 deficiency, the absence of approved alternatives, EMCITATE's potential to address the modifiable systemic driver of morbidity and mortality, and the need to reliably supply and support an ultra-rare patient population. We've set the wholesaler acquisition cost at $74,026 per pack of 60 scorable tablets of 350 mcg. Based on the average daily dose of approximately 740 mcg observed in Triac Trial I, and as described in the U.S. Prescribing Information, this equates to an annualized wholesaler acquisition cost of approximately $950,000. Broad and equitable access remains our commitment, and our work with payers will continue through launch and beyond. This gives Egetis a strong foundation for commercialization and sustainable growth. Next slide. To support access to therapy, we built Egetis RareLink, a fit-for-purpose patient support program developed in partnership with PANTHERx Rare Pharmacy.
Egetis RareLink brings treatment, education, access support, and specialty pharmacy coordination into one connected experience. The program offers personalized support through a patient support liaison, dedicated care managers who help coordinate insurance requirements and financial support options, and resources for families and providers. Our goal is to help families and providers move through the access process efficiently. Next slide. Commercial product is expected to be available in approximately 8- 10 weeks. In the meantime, prescription, enrollment, and access preparation begins now. Through Egetis RareLink, we will support physicians through that process so the necessary steps are completed before product availability, allowing eligible patients to move toward treatment efficiently once supply is available. As stated, this is an exciting and defining moment for the patients, for the MCT8 deficiency community, and for Egetis.
From here, it's execution, disciplined delivery, appropriate patient access, and establishing EMCITATE as the standard of care for peripheral thyrotoxicosis in MCT8 deficiency. We're ready. With that, I'll turn it back to Nick.
Thank you very much, Anny. Let me now close on how we see the value drivers for the company. EMCITATE addresses a devastating rare disease with severe lifelong morbidity, shortening life expectancy, and substantial caregiver burden. It's now the first and only approved therapy in the United States for patients with MCT8 deficiency, following the EU approval in February last year. This truly marks the transition for Egetis into a fully commercial stage rare disease company. As you have heard from Anny, we're ready to launch in the U.S., the world's largest pharmaceutical market. Around 60 U.S. patients are already on expanded access, and the identified 100-plus patient pool keeps growing. We see EMCITATE's clinical profile and disease severity supporting orphan premium pricing. For exclusivity, we have seven years Orphan Drug Exclusivity in the U.S., 10 years in Europe and Japan, with patents expected to protect to roughly 2045.
Furthermore, we see that indication expansion into RTHß represents a meaningful additional growth opportunity in a larger, non-overlapping patient population with cardiovascular morbidity, where the ambition is to start a pivotal study next year. Last but not least, I would like to acknowledge and thank the people who made this achievement possible. We're deeply grateful to the patients, caregivers, investigators, clinicians, and advocacy organizations whose partnership and determination made this achievement possible. Finally, I would like to thank everyone at Egetis. This approval reflects years of dedication, persistence, and unwavering focus on our mission to bring innovative therapies to patients with rare diseases. With that, thank you, and we're now happy to take questions. Operator, over to you.
If you wish to ask a question, please dial pound key five on your telephone keypad to enter the queue. The next question comes from Kristen Kluska from Cantor Fitzgerald. Please go ahead.
Hi, everybody. Good morning, and congratulations on this approval. You should be very proud of all the work you've done for this rare disease community. A few questions from me. First, how should we be thinking about gross to net pricing? Then second, I know you mentioned that there were over 100 patients identified in the U.S. I believe when you really first started this mission, there was far fewer that you had identified in the U.S. Hello? Hello?
Yeah, we hear you, Kristen.
Hello?
We can hear you, Kristen.
Can you hear me?
Yes, we can.
Oh, okay. Perfect. Okay, good morning. Congratulations on this approval. Should feel very proud of all the work you have done for this patient community.
Yeah, yeah. Everything is working.
Okay, great. Just two questions for me. First, how should we be thinking about gross to net pricing? Then second, of the identified pool of 100+ patients, I believe that this number has grown substantially since you started working in the U.S. Can you give us a sense of the most successful ways you've been able to identify these patients and how you plan to use that going forward to further that pool?
Nick, would you want me to take that answer?
If you go out.
Can you hear me, Kristen?
We seem to have a problem with the speaker line. They are logging on again.
Yes, I can hear you.
Carl is logging on. Now they are back again. Can anybody hear us? Yes.
Now we can hear you.
We can hear you, Nicklas. Maybe I can try and dial out from the system to Carl, and we can continue on the phone if they have problem with the internet in the room, or if you can try to reconnect.
I can. Yeah.
Maybe you can start, Anny.
Yes, I'll answer that. Apologies for those technical challenges. In terms of the gross to net, at this moment, we're not giving any specific guidance regarding the gross to net, but we do expect it to reflect the payer mix that we have, and that does include a meaningful proportion of Medicaid patients, which includes, in our assumption, the customary government rebates, discount, and also other channel deductions that we're taking in consideration. I guess here the question comes really mostly based on the high proportion of pediatric patients that we have, and hence significant number of patients that will include government rebates. That's the information that we can share at this moment. We'll continue to monitor that as well as we refine the information that we get into our exact payer mix.
I apologize. We're back here from the Stockholm office. We have seemed to have some technology issues. Thank you, Anny, for responding to the question, which we didn't hear. Kristen, thank you also for your acknowledgement on the progress we made so far. I hope and assume that you got answers to the two questions you posed, which again, we didn't have an opportunity to hear.
The first question was regarding our expectation in gross to net. The second question was regarding the number of patients that we've achieved in terms of identifying to date and what has been the most successful and how we can continue to use that moving forward. We've been using multiple approaches to help identify patients. Prior to our teams being all on board, conferences was one of the key initiatives where patients have been identified. Since we've built our team and have been actively present, that has contributed significantly to increasing the number of new patients that are identified as we increase awareness. We're also leveraging other mechanisms such as data analytics, and looking at building some algorithms that can help us better identify patients in populations that have characteristics looking similar to MCT8 deficiency.
We've been using different channels, and we will continue to leverage these. Most of them have been very effective.
Thank you, Anny and Kristen. I know we discussed this before, but I'm sure you appreciate this, that as for many rare diseases where you haven't had an approved therapy, usually the patient finding starts to rapidly evolve post an approved therapy. The estimated incidence levels in this disease, roughly one in 70,000 males are born with this condition. When they account for a median life expectancy in considering the U.S. population, you're looking at the patient number of roughly 1,000 patients in the U.S. Well, thank you, Anny, for managing this, and I think we should now move to the next question.
The next question comes from Chiara Montironi from Van Lanschot Kempen. Please go ahead.
Good morning, team. Congratulations on the fantastic update. I have a couple of questions. The first one will be just to make sure that the label also includes infants, and that there is not a cut-off for age. I was wondering, in terms of funding, do you believe you can fully cover the launch and also a potential registrational trial for RTHß, given this pricing and the PRV?
No, thank you, Chiara, and great questions as always. Let us be very clear here. There is no age restrictions whatsoever in the label. Very important. The FDA approval gives us eligibility to treat all patients with MCT8 deficiency, regardless of age. I think that is very important, and on par with the label we see in the European Union. The second question was around RTHß, cash runway, et cetera. Monetization of the PRV is of course very important for us. That is something we will progress now in quarter four. I am sure you appreciate that 50% of the net proceeds is eligible for the company. You are seeing that the latest sales prices of a PRV during 2026 has been in the range of $180 million- $220 million per voucher.
So obviously this adds incredibly important financial flexibility and will most definitely support the upcoming launch during 2026 until it becomes cash positive. When it comes to RTHß, it is difficult to give you a straight answer there now because it is still work in process. Our Chief Medical Officer sitting next to me here on my right-hand side is working very diligently on finalizing the clinical trials design for a pivotal study. I am sure you appreciate that until the study design has been finalized, including sample size, it is difficult to cost that. So we have to get back to you, to the market on that specific topic. But thank you very much for your questions. Operator, next question, please.
The—
Oh, sorry.
The next question comes from Clémence Thiers from Stifel. Please go ahead.
Hi, team. First, congratulations on the approval, and thanks for taking our question. Just for following up from some of the earlier question. I understand you're working hard to turn the existing patient pool into commercial patient as quickly as possible. With your 60 patient receiving through the EAP, how quickly do you think those patient can transition onto commercial MCT8? Then how should we think about timing of bringing the other treatment naive patient onto therapy? That would be the first. The second, on reimbursement, you've obviously been engaging with commercial payers and Medicaid. Is there anything that you can share on the feedback you received so far, and how you're thinking about access and coverage in the early post-launch? Thank you very much.
Thank you very much, Clémence, and I'll give a couple of overview remarks on this, then I'll hand over to Anny. But I think you're absolutely right. The 60 patients on EAP is the highest priority short term, to ensure continuity of care. Very important, of course, to ensure that, but also to realize revenue sooner rather than later. With that, Anny, I don't know if you want to provide some more granularity on the transition of EAP patients into commercial product, as well as how we view the additional patient pool of identified patients and on commercial product. Anny, over to you.
Yeah, thank you. Absolutely, want to reemphasize that the continuity of care is our top priority, and we've been preparing very well for that transition. We've been engaging, as I mentioned earlier, extensively with all the sites to facilitate this transition. We'll be working with the physicians and with caregivers through our Egetis RareLink in order to make sure that all these families are ready, in terms of the access until product is available. In terms of the speed of getting access, we expect that this will be a progressive uptake. Typically, we know that in general, for a rare disease launch, it may take 6- 12 months for coverage to policies to be fully established. However, in the meantime, we will work with each patient case by case through our support with the Egetis RareLink in order to provide them access.
That's how we're planning to operate. In terms of the patients that were not part of the EAP program, this will follow the similar process, to get them to access as quickly as possible.
Thank you, Anny, and I think, again, reiterate the unmet medical need here. We heard from Tiago about the devastating condition with the shortened and median life expectancy. I think the sense of urgency is there, both from physicians but also caregivers. Thank you so much, Clémence. I think that we'll—
Thank you.
—move to the last question of today from Arvid.
The next question comes from Arvid Necander from DNB Carnegie. Please go ahead.
Good morning, everyone, and congratulations on this great milestone today. I have a question on the claims you now can make and the pricing implications. I guess overall this seems like a really clean label and broadly in line with what we saw in Europe. Just wondering, how do you expect the lack of mortality claims to influence pricing here? Do you believe that the analogs you have put forward in the past are still relevant? I guess more broadly, within that sort of range that the analogs provide us with, have you gained more confidence on where it should be in that pricing interval now that you know which claims you can make? It would just be interesting to hear your thoughts here. Then just a quick last one, if I may.
You said a little bit about the payer mix and that there will be a meaningful part in the Medicaid population. Roughly, what is the split between commercial and Medicare/Medicaid here? Thanks.
Thank you, Arvid, and good questions as always. Let's start with the price, and as I assume you heard from this call from Anny, we have set the wholesaler acquisition cost at $74,026 per pack, 60 tablets per pack. That translates into an annualized wholesaler acquisition cost of approximately $950,000 per patient per annum. That's a price we have set. Very important to recognize that. When it comes to the other components, we truly believe that this is an orphan premium price, even without the mortality data in the label. We also see that moving forward, as Christian mentioned, we're very much looking forward to the EMC Survival Study being published and for the medical community to actually fully appreciate what's in that study.
But I'll hand over to Anny to further elaborate on the label, if any comments, but again, re-emphasizing that there's no restrictions on the label from an age perspective, and all patients with MCT8 deficiency should be eligible. Anny, over to you.
Yeah. In terms of the pricing, I think you've captured everything. As it relates to your second question about the Medicaid/Medicare split, right now we're looking at a disease that is much more pediatric. So we expect that we'll get a significantly larger proportion of Medicaid patients. The exact split is still going to be refined as we learn more about the patients who start therapy, b ut definitely a greater proportion of Medicaid patients.
Great.
Thank you very much.
Thanks for that. Just a final follow-up, if I may. Sorry, I was a bit late to the call, so I didn't get the gross pricing number, but that's very transparent. Is there anything leading you to believe that the discounting wouldn't be within the sort of typical 20%-30% gross to net discounting? Can you comment anything on that?
No, I think, Arvid, it's reasonable to believe that the part of the payer mix that is related to Medicaid will have the usual rebates, around 23-ish percent. When it comes to commercial payers, we don't see any discount whatsoever.
Great. That's very helpful. Thank you so much, and congratulations again.
Thank you so much. Thank you, everybody, for participating today. We have to apologize for the technical issues we had at this end during the initial part of the question and answer session. But again, today truly marks a historical moment for Egetis and patients with MCT8 deficiency in the U.S. I wish you all a great rest of the day. Thank you.