Egetis to commence a rolling NDA submission in December as granted by the FDA for Emcitate® (tiratricol)

·         Egetis received FDA Breakthrough Therapy Designation for Emcitate® (tiratricol) for MCT8 deficiency
·         At the successful pre-NDA meeting on October 21, FDA has agreed that Egetis can submit the NDA for Emcitate® (tiratricol) based on currently available data
·         Egetis successfully carried out an oversubscribed directed share issue amounting to SEK 183 million
·         Egetis completed the ReTRIACt study of Emcitate® (tiratricol) in MCT8 deficiency and announced positive results
·         Egetis and taiba rare signed exclusive distribution and early access agreement to enable named patient sales of Emcitate® (tiratricol) in the Gulf Region

Financial overview July-September
·         Quarterly revenue MSEK 17.4 (9.4)
·         Quarterly loss MSEK -82.4 (-86.2)
·         Cash at the end of the quarter amounted to MSEK 145.7 (129.9)
·         Cash flow for the quarter was MSEK -57.2 (-63.2)
·         Earnings per share before/after dilution SEK -0.2 (‑0.3)

Financial overview January-September
·         Revenue for the period MSEK 44.6 (35.3)
·         Net loss for the period MSEK -222.8 (-233.1)
·         Cash at the end of the period amounted to MSEK 145.7 (129.9)
·         Cash flow for the period was MSEK -200.6 (-176.2)
·         Earnings per share before/after dilution SEK -0.6 (‑0.8)
 
Significant events during the quarter
·         Egetis received FDA Breakthrough Therapy Designation for tiratricol for MCT8 deficiency, based on the Agency’s review of Egetis’ analysis of the survival data set from the international real-world cohort study (EMC Survival Study) by the Erasmus University Medical Center
·         Egetis submitted a pre-NDA meeting request to the FDA to discuss the contents and timing of the US NDA submission for tiratricol, including the role and position of the ReTRIACt study

Significant events after the quarter
·         Egetis successfully carried out an oversubscribed directed share issue amounting to SEK 183 million (gross)
·         Egetis and taiba rare signed exclusive distribution and early access agreement to enable Named Patient Sales of Emcitate® (tiratricol) in the Gulf Region
·         Egetis to commence a rolling NDA submission in December as granted by the FDA for Emcitate® (tiratricol)
·         Egetis announced positive results from the ReTRIACt study of Emcitate® (tiratricol) in MCT8 deficiency

Comments from the CEO

On July 15 the US Food and Drug Administration (FDA) granted a Breakthrough Therapy Designation (BTD) for tiratricol for the treatment of monocarboxylate transporter 8 (MCT8) deficiency. The BTD was based on the Agency’s review of Egetis’ analysis of survival data from the international cohort study (EMC Survival Study) conducted in over 600 patients with MCT8 deficiency by Erasmus University Medical Center (EMC) in Rotterdam, the Netherlands. Subsequently, in August, we submitted a pre- New Drug Application (NDA) meeting request to the FDA and were granted a face-to-face meeting with the FDA in October.
 
Egetis to commence a rolling NDA submission for Emcitate® (tiratricol) as granted by the FDA
On October 21 we held a successful pre-NDA meeting with the FDA. The objective of the meeting was to seek FDA advice and agreement on the overall content to support the NDA submission, with a special focus on the clinical data package, including the role and position of the ReTRIACt study.
We agreed with the FDA to submit a rolling NDA for Emcitate® (tiratricol), commencing in December 2025, targeting a complete NDA submission in early 2026 and anticipating completion of FDA’s review process in the third quarter of 2026, if Priority Review is granted. As Emcitate® (tiratricol) has Breakthrough Therapy Designation, Egetis will request a Priority Review. Acceptance of the NDA for filing will be subject to FDA’s review of the complete submission.
Following the discussion with the FDA, the ReTRIACt trial was closed and data accrued will be included in the NDA, which will be based on currently available clinical data from Triac Trial I, Triac Trial II, ReTRIACt, EMC Cohort Study, EMC Survival Study and the US Expanded Access Program.
 
Positive ReTRIACt study results
The ReTRIACt study was a double-blind, placebo-controlled, randomized, withdrawal study in patients with MCT8 deficiency. Males 4 years of age and above who were on stable maintenance treatment with tiratricol, were randomized to continue tiratricol or receive placebo for 30 days or until reaching the rescue criterion, based on their serum thyroid hormone T3 concentrations.
As a result of the pre-NDA meeting Egetis held with the FDA in October 2025, data from the ReTRIACt study will complement the existing data from Triac Trial I, Triac Trial II, EMC Cohort Study, EMC Survival Study and the US Expanded Access Program in the NDA submission.
Furthermore, after discussions with the FDA, the Statistical Analysis Plan (SAP) of the ReTRIACt study was revised, and the study was closed. A Primary Endpoint 1 (PE1), evaluating rate of change in serum T3 during the double-blind Randomized Treatment Period, was added to the previously described Primary Endpoint 2 (PE2), based on the T3 rescue criterion.
 The Primary Endpoint 1 (PE1) shows a statistically significant difference between placebo and tiratricol (ratio of T3 rate of change (placebo/tiratricol): 1.494; 95% Confidence Interval: [1.035, 2.155]; p=0.034). The results show a complete separation of the two groups with all 8 patients randomized to placebo having an increase in serum T3 during the randomized treatment period larger than all 7 patients randomized to continue tiratricol. In the placebo group, 4 patients met the rescue criterion during the randomized treatment period compared to 0 patients randomized to tiratricol (p=0.070 on observed cases of rescue). One patient randomized to tiratricol discontinued (not drug related) during the randomized treatment period, which per the SAP was imputed as rescue in PE2, leading to a 4 (placebo) versus 1 (tiratricol) comparison for PE2 (p=0.182). All patients randomized to placebo showed an expected decrease in serum T3 levels following reinitiation of tiratricol in the follow-up period.
For more information on the ReTRIACt study, please see https://clinicaltrials.gov/study/NCT05579327
 
Commercialization of Emcitate® in EU
On May 1 this year, we initiated the launch of Emcitate® in the first country, Germany. All patients who participated in our Managed Access Program in Germany have now been transitioned to the commercial product. In parallel, new MCT8 patients continue to be identified, some of whom have already started Emcitate® therapy.
In October, pricing and reimbursement negotiations started as part of the German AMNOG process and according to standard AMNOG timelines we expect the process to conclude in the second quarter of 2026.
The pricing and reimbursement process in France was initiated in April 2025. In September 2025 our first reimbursement submission in France was rejected by the French health authorities (HAS). The objections raised by HAS included the lack of randomized controlled data. This will be addressed in a resubmission, including new data from the randomized controlled ReTRIACt trial, which we plan to resubmit in early 2026. In the meantime, patients in France continue to benefit from treatment through a managed access program.
In November we initiated the pricing and reimbursement process in Italy and preparations are progressing to submit a pricing and reimbursement dossier in Spain. In parallel, we have successfully realized alternative funding mechanisms in some other EU countries.
As previously communicated, treatment with tiratricol for MCT8 deficiency is already approved in EU and included in the clinical guidelines from the European Thyroid Association, published in 2024.
 
Markets outside Europe and the US
In October, we entered into an exclusive distribution and early access agreement with Taiba Middle East FZ LLC (“taiba rare”) to supply Emcitate® (tiratricol), for the treatment of MCT8 deficiency, in Saudi Arabia, United Arab Emirates, Qatar, Oman, and Bahrain with the ambition to extend to other countries in the Middle East.
As previously communicated, in June this year we signed a distribution agreement with Er-Kim for Emcitate® in Türkiye. This second distribution agreement with taiba rare highlights our continued focus on reaching patients with urgent unmet needs, wherever they are.
In Japan we have a license agreement with Fujimoto Pharmaceutical Corporation to develop and commercialize tiratricol. Discussions continue with the Japanese pharmaceutical regulatory authority PMDA regarding the regulatory development pathway required to obtain approval of tiratricol in Japan where a pre-NDA meeting is scheduled for December.
 
 Expanded Access Program for tiratricol in the US
At the request of the FDA, Egetis has implemented an Expanded Access Program (EAP) in the US. Currently, 15 hospitals are included in the EAP program. The EAP facilitates physicians’ access to tiratricol for their patients with MCT8 deficiency who are not eligible for a clinical trial until the product receives marketing authorization, as well as providing continued treatment to patients who completed the ReTRIACt study.
For more information on the EAP program, please see https://clinicaltrials.gov/study/NCT05911399
 
Cash position
We report a cash position of approximately SEK 146 million as of September 30, 2025. Post period, on October 2, 2025, we successfully carried out a Directed Share Issue amounting to SEK 183 million, before deducting transaction costs. The Directed Issue was oversubscribed and included participation from new and existing international and Swedish institutional investors, including US biotech investors Frazier Life Sciences, Invus, Petrichor and Woodline Partners LP, as well as Swedish investors The Fourth Swedish National Pension Fund, Cidro Förvaltning AB (Peter Lindell), Linc AB and other institutional investors.
 
Outlook
2025 has been a year with several important milestones for Egetis. Our team continues to focus on the following key priorities:
 
·         Optimize pricing and reimbursement processes and launch in Europe
 
·         Initiate the rolling submission of the NDA for tiratricol in the USA
 
·         Preparatory launch activities in the USA including buildup of a commercial and medical affairs infrastructure
 
Nicklas Westerholm, CEO