Egetis Therapeutics AB: Interim report Q1 2025

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

For further information, please contact

Nicklas Westerholm, CEO
[e-mail]
[phone]

Yilmaz Mahshid, CFO
[e-mail]
[phone]

Karl Hård, Head of Investor Relations & Business Development
[e-mail]
[phone]

This information is information that Egetis Therapeutics is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact persons set out above, at 2025-04-30 07:00 CEST.

About Egetis Therapeutics

Egetis Therapeutics is an innovative and integrated pharmaceutical company, focusing on projects in late-stage development for commercialization for treatments of serious diseases with significant unmet medical needs in the orphan drug segment.

On February 13, 2025, the European Commission approved Emcitate® (tiratricol) as the first and only treatment for MCT8 deficiency in EU.

The Company's lead drug candidate Emcitate® (tiratricol) is under development for the treatment of patients with monocarboxylate transporter 8 (MCT8) deficiency, a highly debilitating rare disease with no available treatment. In previous studies (Triac Trial I and a long-term real-life study) tiratricol has shown highly significant and clinically relevant results on serum thyroid hormone T3 concentrations and secondary clinical endpoints. Triac Trial II investigated a potential treatment effect on neurocognitive development in young children under 30 months with MCT8 deficiency. The study did not show a statistically significant improvement compared to historical controls.

After a dialogue with the FDA, Egetis is conducting a randomized, placebo-controlled pivotal study in at least 16 evaluable patients to verify the results on T3 levels seen in previous clinical trials and publications. As previously communicated, the Company will update the market as soon as recruitment closes, and at that time, the Company will also provide information on when to expect topline results and when the Company plans to submit the NDA application.

Tiratricol holds Orphan Drug Designation (ODD) for MCT8 deficiency and resistance to thyroid hormone type beta (RTH-beta) in the US and the EU. MCT8 deficiency and RTH-beta are two distinct indications, with no overlap in patient populations. Tiratricol has been granted Rare Pediatric Disease Designation (RPDD) which gives Egetis the opportunity to receive a Priority Review Voucher (PRV) in the US, after approval. This voucher can be transferred or sold to another sponsor.

The drug candidate Aladote® (calmangafodipir) is a first in class drug candidate developed to reduce the risk of acute liver injury associated with paracetamol (acetaminophen) overdose. A proof of principle study has been successfully completed. The design of a pivotal Phase IIb/III study (Albatross), with the purpose of applying for market approval in the US and Europe, has been finalized following interactions with the FDA, EMA and MHRA. The development program for calmangafodipir has been parked until Emcitate marketing authorization submissions for MCT8 deficiency have been completed in the EU and the USA. Aladote has been granted ODD in the US and in the EU.

Egetis Therapeutics (Nasdaq Stockholm: EGTX) is listed on the Nasdaq Stockholm main market. For more information, see www.egetis.com