Egetis Therapeutics

Egetis submitted the U.S. NDA for Emcitate® (tiratricol) for treatment of MCT8 deficiency

·         Emcitate® revenue during 2025 amounted to MSEK 62.3, a 40 % increase in constant exchange rates from 2024.

Financial overview October-December
·         Quarterly revenue MSEK 17.9 (10.8)
·         Quarterly loss MSEK -119.7 (-110.5)
·         Cash at the end of the quarter amounted to MSEK 215.8 (351.0)
·         Cash flow for the quarter was MSEK 70.8 (218.1)
·         Earnings per share before/after dilution SEK -0.3 (‑0.3)

Financial overview January-December
·         Revenue for the period MSEK 62.4 (46,1)
·         Net loss for the period MSEK -342.5 (-343,6)
·         Cash at the end of the period amounted to MSEK 215.8 (351.0)
·         Cash flow for the period was MSEK -129.8 (41.8)
·         Earnings per share before/after dilution SEK -0.9 (‑1.1)

Significant events during the quarter
·         As an outcome of a successful pre-New Drug Application (NDA) meeting with the FDA, Egetis was granted a rolling NDA for Emcitate® based on currently available clinical data
·         Egetis announced positive results from the ReTRIACt study of Emcitate® in MCT8 deficiency
·         Egetis initiated the U.S. rolling NDA submission in December for Emcitate® for the treatment of MCT8 deficiency
·         Egetis and taiba rare signed exclusive distribution and early access agreement to enable Named Patient Sales of Emcitate® (tiratricol) in the Gulf Region
·         Egetis and Er-Kim expanded partnership to broaden access to Emcitate® across Central, Eastern, and Southeastern Europe
·         Egetis successfully carried out an oversubscribed directed share issue amounting to SEK 183 million (gross)
·         New publication highlighted meaningful clinical benefits of Emcitate® in treating Resistance to Thyroid Hormone Beta

Significant events after the quarter
·         Egetis completed U.S. rolling NDA submission for Emcitate® for treatment of MCT8 deficiency
·         FDA’s rare pediatric disease priority review program extended until 2029
·         Fujimoto had a pre-application consultation for drugs with Japan’s PMDA. The NDA in Japan is expected to utilize existing data generated from the global clinical development program

Comments from the CEO
The year 2025 was historic for Egetis. In February Emcitate® (tiratricol) was approved in the EU, as well as Iceland, Norway, and Liechtenstein, as the first and only treatment for patients with monocarboxylate transporter 8 (MCT8) deficiency. We initiated the launch of Emcitate® in the first country, Germany, in May 2025. Emcitate revenue during the year amounted to MSEK 62.3, which is an increase of 40 % in constant exchange rates from 2024 (MSEK 46.1).
 
Positive pre-NDA Meeting and Rolling NDA Submission
As previously reported, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation (BTD) for Emcitate® for the treatment of MCT8 deficiency in July, and subsequently granted a pre-New Drug Application (NDA) meeting. On October 21, 2025, we held a successful pre-NDA meeting with the FDA, with the purpose to obtain FDA’s advice and agreement regarding the overall NDA package, with specific focus on the clinical data set, including the role and positioning of the ReTRIACt study. As a result of discussions at the October 2025 pre-NDA meeting, data from the ReTRIACt study, which have been previously reported, complement data from Triac Trial I, Triac Trial II, the EMC Cohort Study, the EMC Survival Study, and the U.S. Expanded Access Program as part of the NDA submission, forming part of the robust clinical data package in this rare and debilitating condition.
We agreed with the FDA to submit a rolling NDA for Emcitate®, which we initiated on December 19, 2025 with the submission of the preclinical and CMC (chemistry, manufacturing and controls) sections and completed the NDA on January 29, 2026 by submitting the clinical sections.
The FDA is expected to validate within 60 days whether the NDA is complete. Since Emcitate® has both Fast Track and Breakthrough Therapy status, Egetis has requested Priority Review, which if granted would result in FDA’s review being completed within six months after the validation period, and a regulatory decision in September 2026.
 
The ReTRIACt Study
In November 2025, Egetis announced positive results from the ReTRIACt study of Emcitate® in MCT8 deficiency. The ReTRIACt study was a double-blind, placebo-controlled, randomized withdrawal study in patients with MCT8 deficiency and is the last study to be reported for Emcitate® in MCT8 deficiency.
 
Commercialization of Emcitate® in the EU
On May 1, 2025, we initiated the launch of Emcitate® in the first country, Germany. All patients in our Managed Access Program in Germany were transitioned to commercial product during Q3. In parallel, new MCT8 deficiency patients continue to be identified, several of whom have already started treatment with Emcitate®.
In October, we initiated price negotiations within the German reimbursement process (AMNOG), and according to standard AMNOG timelines, we expect the process to conclude in Q2 2026.
The pricing and reimbursement process in France began in April 2025. In September 2025, the French HTA authority HAS rejected our initial reimbursement application. Their concerns included the absence of randomized controlled data. This will be addressed in a resubmission, including data from the randomized controlled ReTRIACt study. Meanwhile, patients in France continue to receive treatment through a Managed Access Program.
In November, we initiated the pricing and reimbursement process in Italy, and preparations are ongoing to submit a pricing and reimbursement dossier in Spain. In parallel, we have successfully implemented alternative reimbursement solutions in certain other EU countries.
As previously communicated, treatment with tiratricol for MCT8 deficiency is already included in the European Thyroid Association’s clinical guidelines published in 2024.
 
Markets Outside Europe and the U.S.
In October, we entered into an exclusive distribution and early access agreement with Taiba Middle East FZ LLC (“taiba rare”) to provide Emcitate® in Saudi Arabia, the United Arab Emirates, Qatar, Oman, and Bahrain, with the ambition to expand to additional Middle Eastern countries.
In December, we entered into an expanded exclusive distribution agreement with Er-Kim to provide Emcitate® for MCT8 deficiency in Albania, Bosnia and Herzegovina, Bulgaria, Cyprus, Estonia, Greece, Kosovo, Croatia, Latvia, Lithuania, Montenegro, North Macedonia, Poland, Serbia, Slovenia, and Hungary. This follows the initial agreement signed in June 2025 for Turkey, and marks another important milestone in our growing partnership.
In Japan, we have a licensing agreement with Fujimoto Pharmaceutical Corporation for the development and commercialization of tiratricol. In December, Fujimoto completed a Pre-application consultation for drugs with Japan’s PMDA. The NDA in Japan is expected to utilize existing data generated from the global clinical development program
 
Expanded Access Program (EAP) in the U.S.
At FDA’s request, Egetis has implemented an Expanded Access Program (EAP) in the U.S. Currently, 17 hospitals are included. The EAP allows physicians to access tiratricol for patients not eligible for clinical trials prior to marketing approval, as well as for continued treatment of patients who completed the ReTRIACt and Triac Trial II studies.
 
Emcitate® (tiratricol) in the Treatment of Resistance to Thyroid Hormone Beta (RTH-beta)
In December, a scientific article was published describing significant clinical benefits of treatment with Emcitate® (tiratricol) in patients with resistance to thyroid hormone beta (RTH-beta).
RTH-beta is a rare congenital genetic disorder caused by mutations in one of the two subtypes of thyroid hormone receptors (TR), leading to impaired hormonal signaling in affected tissues. Clinical manifestations include a combination of symptoms of thyrotoxicosis and hypothyroidism across different tissues, such as goiter, liver steatosis and dyslipidemia, impaired hearing and color vision, neurocognitive deficits, and cardiovascular strain. Prevalence is estimated at between 1 in 20,000–40,000 births. Currently, no approved treatment exists for patients with RTH-beta.
Now with Emcitate® NDA submission completed for MCT8 deficiency, Egetis will evaluate subsequent clinical development for Emcitate in new indications, such as RTH-beta.
 
 Directed Share Issue
On October 2, 2025, we successfully completed a directed share issue of SEK 183 million, before transaction costs. The directed share issue was oversubscribed and included both new and existing international and Swedish institutional investors, including the U.S. biotech investors Frazier Life Sciences, Invus, Petrichor and Woodline Partners LP, as well as Swedish investors AP4 (Fjärde AP-fonden), Cidro Förvaltning AB (Peter Lindell), Linc AB and other institutional investors.
 
Cash Position
We report cash of approximately SEK 216 million as of December 31, 2025.
 
Outlook
The year 2026 will bring several important milestones for Egetis. Our team is focused on the following key priorities:
 
·         Work closely with FDA during the review of the Emcitate® NDA, with the goal of approval in the second half of 2026
·         Preparing U.S. launch activities, including building commercial and medical infrastructure
·         Successfully launching Emcitate® in the U.S. in the fourth quarter
·         Optimizing pricing and reimbursement, and continuing the rollout of Emcitate® in Europe
 
Nicklas Westerholm, CEO