Egetis plans to launch Emcitate® in the first country, Germany, on May 1, 2025

• The European Commission approved Emcitate® (tiratricol) as the first and only treatment for patients with MCT8 deficiency.
• In the ReTRIACt study, which is pivotal for the marketing authorization application in the USA, 12 patients have completed the randomized phase, 1 patient is currently in the randomized phase, 1 patient is in the run-in period and 2 patients are planned for screening.
• In the MENAT region (Middle East, North Africa, and Turkey), discussions are ongoing with potential distribution partners to enable patient access to Emcitate.

Financial overview January-March

• Quarterly revenue MSEK 12.7 (12.1)
• Quarterly loss MSEK -62.9 (-75.0)
• Cash flow for the quarter was MSEK -74.2 (-56.0)
• Cash at the end of the quarter amounted to MSEK 272.8 (251.7)
• Earnings per share before/after dilution SEK -0.2 (‑0.3)

Significant events during the quarter

• The European Commission approved Emcitate as the first and only treatment for patients with MCT8 deficiency.
• Egetis participated in the TV program Behind the Mystery, which aired in the USA, to raise awareness about MCT8 deficiency.
• Egetis organized a virtual KOL (Key Opinion Leader) event on MCT8 deficiency.

Significant events after the quarter

• In the ReTRIACt study, which is pivotal for the New Drug Application in the USA, 12 patients have completed the randomized phase, 1 patient is currently in the randomized phase, 1 patient is in the run-in period and 2 patients are planned for screening.
• Emcitate is being prescribed via Managed Access Programs to over 230 patients.

Comments from the CEO
I am very proud of the EU approval of Emcitate® (tiratricol), and I am grateful for the hard work of Egetis employees and our partners over the years, during which we have invested more than EUR 100 million in the development of the first and only approved treatment for patients with MCT8 deficiency. A special thanks goes to Professor Edward Visser and his team at Erasmus University Medical Center in Rotterdam, the Netherlands. I am pleased that we can now offer this new treatment to patients in Europe.

On February 13, 2025, the European Commission approved Emcitate in all 27 EU member states as well as Iceland, Norway, and Liechtenstein. We are initially focusing our launch efforts on the EU4 countries (Germany, France, Spain, and Italy), where we have compiled pricing and reimbursement dossiers for Germany and France. The French dossier was submitted to Haute Autorité de Santé (HAS) during the first week of April. The German dossier is being submitted today (April 30, 2025) to the Gemeinsamer Bundesausschuss (GBA), and the launch of Emcitate in Germany is planned for tomorrow (May 1, 2025). The German AMNOG process is highly structured and takes exactly one year before the final reimbursement price in Germany is determined (see page 19 of Egetis’ 2024 Annual Report for an overview of the AMNOG process). In France, it may take 1–2 years before a reimbursed price is fully negotiated. Preparations are underway to initiate pricing and reimbursement processes in Spain and Italy.

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 a New Drug Application (NDA) in the United States.
To increase recruitment capacity for the study, we opened four additional study sites in the U.S. during 2024—one each in Texas, Georgia, North Carolina, and Florida. To date, 12 patients have completed the randomized phase, 1 patient is currently in the randomized phase, and 1 patient is in the initiation phase. Two patients are planned for screening and additional patients are being evaluated.
For more information about the ReTRIACt study, see:
https://clinicaltrials.gov/study/NCT05579327

Markets Outside Europe and the U.S.
Egetis has a license agreement with Fujimoto Pharmaceutical Corporation for the development and commercialization of Emcitate in Japan. Discussions are currently ongoing with the Japanese regulatory authority, the PMDA, regarding the regulatory development pathway required to obtain approval for Emcitate in Japan.
In the MENAT region (Middle East, North Africa, and Turkey), discussions are underway with potential distribution partners to enable patient access to Emcitate.

Egetis featured in U.S. TV program on MCT8 deficiency
On February 24, Egetis appeared in Behind the Mystery™, a U.S. television program aired during the morning show The Balancing Act®. This episode, sponsored by Egetis, aimed to raise awareness of MCT8 deficiency in connection with Rare Disease Day on February 28. The episode on MCT8 deficiency also aired again on March 3. A replay is available at: https://www.mct8deficiency.eu/behind-the-mystery/

MCT8 deficiency is a rare genetic disorder first described in 2004. The Company’s medical affairs activities focus on raising awareness of the disease and improving diagnostic understanding through participation and dialogue at scientific conferences, partnerships with genetic testing companies, engagement with leading experts, and interactions with patient organizations. So far in 2025, Egetis has participated in six scientific conferences in fields such as endocrinology, pediatrics, and neurology, in which MCT8 deficiency has been highlighted.

For more information about MCT8 deficiency, visit: www.mct8deficiency.com

Egetis Hosted a Virtual KOL Event on MCT8 Deficiency
On March 4, Egetis hosted a virtual Key Opinion Leader (KOL) event for analysts and investors. Professor Edward Visser from Erasmus University Medical Center in Rotterdam, the Netherlands, presented on the medical need in MCT8 deficiency and shared published data on the effects of Emcitate in the treatment of patients with MCT8 deficiency.
For a link to the KOL event information, visit: https://egetis.events.inderes.com/kol-event-2025

Managed access program for Emcitate
There continues to be significant and growing interest from physicians worldwide in treating patients with MCT8 deficiency using Emcitate, which is already being prescribed to patients in over 25 countries as part of our Managed Access Program. Currently, more than 230 patients are being treated with Emcitate.

At the request of the FDA, Egetis has implemented an Expanded Access Program (EAP) in the United States. At present, 13 hospitals are ready to receive patients under the EAP. The EAP facilitates access to tiratricol for physicians treating patients with MCT8 deficiency who are not eligible for a clinical trial, until the product receives marketing authorization. It also provides continued treatment for patients who have completed the ReTRIACt study.

For more information about the EAP program, see:
https://clinicaltrials.gov/study/NCT05911399

Cash
We report cash of approximately SEK 273 million as of March 31, 2025. 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 Europe;
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