European Commission has approved Egetis’ Emcitate® (tiratricol) as the first and only treatment for patients with MCT8 deficiency

·         Egetis expects to launch Emcitate in the first country, Germany, during the second quarter of 2025.
·         Egetis successfully carried out directed share issuances amounting to SEK 300 million (gross).
·         In the ReTRIACt study, which is pivotal for the New Drug Application in the USA, 11 patients have completed the randomized phase.

Financial overview October-December
·         Quarterly revenue MSEK 10.8 (32.6)
The corresponding quarter 2023 included MSEK 14.5 license income from Japan
·         Quarterly loss MSEK -110.5 (-86.3)
·         Cash flow for the quarter was MSEK 218.1 (223.6)
·         Cash at the end of the quarter amounted to MSEK 351.0 (303.3)
·         Earnings per share before/after dilution SEK -0.3 (‑0.3)

Financial overview January-December
·         Revenue for the period MSEK 46.1 (57.6)
The corresponding period 2023 included MSEK 14.5 license income from Japan
·         Net loss for the period MSEK -343.6 (-326.9)
·         Cash flow for the period was MSEK 41.8 (180.4)
·         Cash at the end of the period amounted to MSEK 351.0 (303.3)
·         Earnings per share before/after dilution SEK -1.1 (‑1.3)

Significant events during the quarter
·         Egetis received a positive CHMP opinion for Emcitate for the treatment of MCT8 deficiency.
·         Egetis successfully carried out directed share issuances, amounting to SEK 300 million (gross). The Directed Issue was oversubscribed and included both existing and new international and Swedish institutional investors. It was led by US healthcare investor Frazier Life Sciences with a USD 10 million investment.
·         Egetis decided to evaluate resistance to thyroid hormone beta (RTH-beta) as the next potential indication for Emcitate.
·         Egetis hosted an investor day in Stockholm. Prof. E. Visser gave a presentation on MCT8 deficiency, and Prof. A. Rees presented on RTH-beta. Members of Egetis' management team highlighted the progress made toward market approvals for Emcitate, as well as the Company's strategic objectives. (Link to the Investor Day)

Significant events after the quarter
·         European Commission approved Emcitate as the first and only treatment for MCT8 deficiency.
·         In the ReTRIACt study, which is pivotal for the New Drug Application in the USA, so far 19 patients have been included of which 11 patients have completed the randomized phase.
·         Emcitate is being prescribed via Managed Access Programs to approximately 230 patients.

Comments from the CEO

On February 13, 2025, the European Commission (EC) approved Emcitate® (tiratricol) as the first and only treatment for patients with monocarboxylate transporter 8 (MCT8) deficiency in all 27 European Union member states, as well as Iceland, Norway, and Liechtenstein. I am very proud of the approval of Emcitate, which represents the single most important milestone in Egetis’ history and a major step forward in building a sustainable rare disease company. We are delighted to bring this much needed new treatment to patients in the EU.
I would like to thank all patients, parents, caregivers and investigators who have taken part in the comprehensive development program for Emcitate and all Egetis employees and collaborators for their dedicated and hard work, in particular the group of Prof. Dr. Edward Visser at the Erasmus University Medical Center, Rotterdam, The Netherlands.
We look forward to initiating pricing and reimbursement processes and discussions in Europe and expect to launch Emcitate in the first country, Germany, during the second quarter of 2025.
 
Update on the ReTRIACt study
Following an agreement with the FDA, Egetis is conducting a pivotal, randomized, placebo-controlled study (ReTRIACt) in at least 16 evaluable patients with MCT8 deficiency to support the submission of a New Drug Application (NDA) in the USA.
To increase the recruitment capacity in the study four additional clinical study sites have been opened in the USA in 2024: one each in Texas, Georgia, North Carolina and Florida. So far, 19 patients have been included, of which 11 patients have completed the randomized phase, 3 patients are in the run-in period and additional patients are being evaluated for screening.
As previously communicated, we will update the market as soon as recruitment of the ReTRIACt trial is closed. At that time, we will also provide information on when to expect topline results and when we plan to submit the NDA application.
More information about the ReTRIACt study is available on clinicaltrials.gov under the code NCT05579327.
 
Egetis participated in a TV-show in the USA about MCT8 deficiency
On February 24, Egetis participated in Behind the Mystery™, a TV show in the U.S. that airs during the morning program The Balancing Act®. This episode, sponsored by Egetis, aimed to raise awareness about MCT8 deficiency in connection with Rare Disease Day on February 28. The episode on MCT8 deficiency will also air on March 3, 2025. A replay can be viewed at TheBalancingAct.com/rare.
MCT8 deficiency is a rare genetic disease first described in 2004, and in the EU Emcitate is now the first and only approved therapy. Consequently, the general awareness of the disease and the diagnosis are very low, even among specialist physicians, and a large portion of patients remain misdiagnosed. Our medical affairs activities are focused on improving awareness of the disease and improving knowledge about its diagnosis, by participation and dialogues at scientific conferences, partnering with genetic testing companies, engaging with Key Opinion Leaders, advisory committees, and interactions with patient groups. During 2024, Egetis participated at over 30 scientific conferences on topics such as endocrinology, pediatrics, and neurology, where MCT8 deficiency has been presented.
For more information about MCT8 deficiency, please see https://www.mct8deficiency.com/
 
Managed access program for tiratricol
There is continued significant and growing interest from physicians worldwide in treating patients with MCT8 deficiency with Emcitate, which is already being prescribed as part of Managed Access Programs to patients in over 25 countries. Currently approximately 230 patients are being treated with Emcitate.
At the request of the FDA, Egetis has implemented an Expanded Access Program (EAP) in the USA. Currently, 12 sites are open to enroll patients in the EAP and an additional 8 hospitals are in the process of joining the program. The EAP program facilitates physicians in accessing tiratricol for their MCT8 deficiency patients who are ineligible for a clinical trial until the product receives market authorization. The program is also important for patients in the ReTRIACt study, so that they have the option to continue treatment with tiratricol after completing the study.
 
Egetis decided to evaluate RTHβ as the next indication for Emcitate
In December 2025, we announced that the Company has selected resistance to thyroid hormone beta (RTHβ) as the next potential indication for Emcitate. The company plans to support an investigator-initiated Phase 2 multicenter study for patients with RTHβ in collaboration with academic research groups.
RTHβ is a rare genetic disorder with a significant unmet medical need and currently has no approved treatments. The condition affects 1–2 individuals (both males and females) per 40,000 live births and is caused by mutations in the thyroid hormone receptor beta gene. Thyroid hormone is essential for development and metabolism in virtually all tissues and acts by binding to nuclear thyroid hormone receptors, leading to the transcription of hormone-sensitive genes.
Recent studies have shown that patients with RTHβ have reduced survival and an increased risk of cardiovascular disease (Okosieme et al. 2023, Campi et al. 2024).
RTHβ is a distinct indication, with no overlap in the patient population with MCT8 deficiency. In 2022, Egetis received Orphan Drug Designation (ODD) for RTHβ for Emcitate in both the U.S. and the EU.
 
Egetis’ Investor Day December 18, 2024
On December 18, 2024, Egetis hosted an Investor Day in Stockholm. During the event, Professor Edward Visser from Erasmus University Medical Center gave a presentation on MCT8 deficiency and the unmet medical need, while Professor Aled Rees from Cardiff University spoke about RTHβ and the significant unmet medical need in this condition.
Members of Egetis’ leadership team highlighted the progress made toward marketing approvals for Emcitate, including a status update on the ReTRIACt study, as well as plans for preparatory activities and commercialization, focusing on disease awareness, market access, and value proposition. Additionally, the Company’s strategic short-term goals and long-term ambitions to build a sustainable orphan drug company were presented. (Link to Investor Day)
 
Successful Directed Share Issue
In October 2024 we carried out a directed share issue at a market price of SEK 4.50 per share, raising net proceeds of SEK 281 million. This new financing reflects investors’ continued confidence in our assets, our team, and our work.
The directed share issue was oversubscribed and included both existing and new international and Swedish institutional investors. The issuance was led by the U.S.-based life sciences investor Frazier Life Sciences with an investment of USD 10 million and was supported by international specialist investor Invus (USA/France), as well as Platinum Asset Management (Australia), Fjärde AP-fonden, Handelsbanken Fonder AB through the investment fund Hälsovård Tema, Unionen, HealthInvest Partners AB, and Cidro Förvaltning AB.
 
Cash
We report cash of approximately SEK 351 million as of December 31, 2024. Currently, the Company has an ongoing dialogue with BlackRock regarding the conditions and a prolongation of the Tranche B (EUR 15 million) draw down window of the loan facility.
 
Outlook
2025 is a year marked by several important milestones for Egetis. Our team continues to focus on delivering four key priorities:
1.        Optimize pricing- and reimbursement processes and launch in EU4;
2.        Complete the ReTRIACt study, which is pivotal in the USA, as soon as possible;
3.        Submit the NDA for Emcitate in the USA;
4.        Preparatory launch activities in the USA.
 
Nicklas Westerholm, CEO