Welcome to Egetis Therapeutics' webcast. During the questions and answers session, participants are able to ask questions by dialing #5 on their telephone keypad. If you are listening to the presentation via webcast, you can ask written questions using the form below. Now, I will hand the conference over to CEO Nicklas Westerholm. Please go ahead.
Welcome to Egetis Therapeutics' webcast. My name is Nicklas Westerholm, CEO of Egetis Therapeutics. With me today, I have also Cristian Fabbri, Vice President, Product Strategy and Development, Yilmaz Mahshid, Chief Financial Officer, as well as Karl Hård, Head of Investor Relations. This morning, we issued a press release announcing that Egetis has been granted a rolling New Drug Application, a so-called NDA, review by the U.S. FDA for MCT8, for the treatment of MCT8 deficiency based on currently available clinical data. This is great news, and the result of a very successful pre-NDA meeting we had with the FDA earlier this week on the back of the granted Breakthrough Therapy Designation in July.
We are strongly encouraged by the dialogue with the FDA on MCT8, and we appreciate the agency's collaborative spirit as we work to deliver the first therapy to patients with MCT8 deficiency as efficiently as possible in the U.S. The objective of the pre-NDA meeting was to seek FDA advice and agreement on the overall content to support our NDA submission for MCT8, with a special focus and emphasis on the clinical data package. We have now reached an agreement with the FDA that we can commence a rolling NDA submission in December 2025, targeting a complete NDA submission in early 2026 and the completion of the FDA review process in the third quarter of 2026, as we expect a priority review. This is, of course, a substantial step towards an approval in the United States.
The Breakthrough Therapy Designation was granted in July this year based on FDA's review of our independent analysis of the survival data from the international cohort study conducted by the Erasmus Medical Center, which we called the EMC Survival Study. We will start the rolling submission in December with the non-clinical and quality modules of the NDA dossier and complete the NDA with a clinical module in early 2026. Importantly, the NDA for MCT8 for the treatment of MCT8 deficiency will be based on currently available clinical data. Considering that MCT8 deficiency is an ultra-rare disease, we have an unusually large and robust clinical data set from six different sources.
This includes TriTrial1 with 46 patients, TriTrial2 with 22 patients, where data in the NDA will include long-term safety data up to four years of treatment in younger children, the retried study with 15 patients, the EMC cohort study with 67 patients, and the EMC survival study with over 600 patients, as well as the U.S. Expanded Access program, where we currently today have around 30 patients. Importantly, the ongoing retried study is no longer considered as pivotal for the New Drug Application, which it was when the study was designed originally. The study will be closed as soon as possible, and no further patients will be added. The FDA has recommended that the statistical analysis plan, the so-called SAP for the trial, will be revised.
While I can't comment on any specific details about the retried study or the SAP, what I can say is the revised SAP will reflect the fact that the retried study is now a complementary study to the survival data and other clinical components in the NDA rather than being the pivotal study. We estimate top-line results from the retried study will be communicated during the fourth quarter of 2025. Data accrued to date will be included in the NDA. In summary, we are very pleased with the agreement we have with the FDA and the clear path to an NDA for MCT8 in the U.S. based on the currently available clinical data already in the coming month. With that, I would like to open up for questions. Operator, please go ahead.
If you wish to ask a question, please dial #5 on your telephone keypad to enter the queue. The next question comes from Chiara Montironi from Van Lanschot Kempen. Please go ahead.
Hello, team. Congratulations with the update and thanks for taking my question. As MCT8 oversight has transitioned in the past from the Rare Division to the Endocrinology Division, can you share your thoughts on how this is affecting the regulatory process? Do you believe that this new division facilitated the possibility of a rolling submission?
Yes, of course, and good morning, Chiara. Good to speak to you again. No, I think it's a valid question. As you pointed out, previously our application or IND has been situated with the Rare Disease Division and was during 2024 moved to the Endocrinology Division, which in our humble opinion has been very positive. As I mentioned also during my initial opening remarks here, the dialogue with the FDA has been very open, very positive, and we are strongly encouraged by the agency's collaborative spirit. I think there is a strong appreciation from the agency and the Division of Endocrinology that the unmet medical need for patients with MCT8 deficiency is high, that we have generated an unusually large and robust clinical data set to date, and an appreciation of the disease itself being an ultra-orphan condition.
Overall for us, that has been very positive, which was reflected in, I would say, the outcome of the pre-NDA meeting we held earlier this week.
Yes, really clear. If I may, I have another question. I actually wanted to ask about powering, but I will ask it another time. When are we going to be able to see the survival study published by Erasmus? I know it's in their hands, but I was wondering maybe you have some timelines?
That's also a very good question. As they rightly pointed out, it's not in our hands. The publication and manuscript related to the survival study carried out by Erasmus Medical Center based on data from the international consortium will be published in the not-too-distant future. I don't have the insights, and I can't comment any further than that, but hopefully in the not-too-distant future.
Okay, thank you so much.
Thank you, Chiara.
The next question comes from Oscar Haffen Lamm from Stifel. Please go ahead.
Hi, team. Congrats first of all on the positive updates. I was just wondering, in your discussions with the U.S. Food and Drug Administration, have you already discussed the potential label for MCT8? In particular, if the survival data will end up in the label, which obviously would be important from a pricing perspective.
Sure, that's a really good question. Thank you, Oscar. Of course, we have had, as you always do, initial discussions around potential label. However, as standard practice, when you are in the process we are, i.e., leading up to an NDA submission and subsequently a review, this will be discussed further in detail during the review process. From a company perspective, the ambition is, of course, to have as broad a label as possible, including all the possible claims based on the clinical data we have generated to date, including survival benefits. This will be more detailed discussions with the agency during the NDA review process.
Okay, got it. Thank you.
Thank you.
There are no more questions at this time. I hand the conference back to the speakers for written questions and closing comments.
We have some written questions, and the first one comes from Frederic Tour. The question is, given that the retried study is complementary, does it mean that the statistical results are a bit more flexible? I can't comment in detail, Frederic. Thank you for your written question. As mentioned in my opening remarks, while I can't comment on any specific details about the retried study nor the statistical analysis plan, what I can say is the revised statistical analysis plan will reflect the fact that the retried study now is considered to be complementary to the survival data and other clinical components in the New Drug Application rather than a pivotal study. There is a follow-up question from Frederic Tour. You mentioned that there is a collaborative spirit at the U.S. Food and Drug Administration when it comes to the Priority Review Voucher program and its potential end.
How much does this impact the FDA's plans to take a decision before the end of September? This is Karl Hård. I can comment on this. At the moment, the sunset clause for the Priority Review Voucher program is, as you said, end of September 2026. We know from before that the FDA is forthcoming in trying to be helpful. However, it's also important to note that there is a bill in the House, number HR 1262, which is called Give Kids a Chance, which has passed the first committee with a vote of 47-0 to be actually pushed forward for a vote in the whole House. It has currently over 310 co-sponsors, both from Republicans and Democrats. That looks very promising that that will also be going forward. If that becomes law, then it would mean that the program would be prolonged until the end of September 2029.
That still needs to happen then. Thank you so much, Karl, for that update. With that, we'd like to take the opportunity to thank the participants joining the call today. In summary, we are incredibly pleased with the discussions and the agreement with the FDA and with the clear path forward to a New Drug Application for MCT8 in the United States based on currently available clinical data already in the coming couple of months. We look forward to keep the market updated as we progress. Thank you very much, and I wish you all a good day.