Egetis highlights the positive CHMP opinion for Emcitate®, recent major milestones, and gives a corporate update at Investor Day in Stockholm today

Stockholm, Sweden, December 18, 2024. Egetis Therapeutics AB (publ) (“Egetis” or the “Company”) (Nasdaq Stockholm: EGTX) is today hosting an Investor Day in Stockholm, Sweden. The event will feature presentations by Professor Edward Visser, Erasmus MC, Rotterdam, The Netherlands on the recent advances with tiratricol in monocarboxylate transporter 8 (MCT8) deficiency and Professor Aled Rees, Cardiff University, UK, on resistance to thyroid hormone beta (RTH-beta), the unmet medical need and the potential opportunity for Emcitate® in this disease. In addition, members of Egetis’ management team will highlight the significant progress made by Egetis towards marketing approvals of Emcitate® (tiratricol), including the positive CHMP opinion for Emcitate®, an update of the status of the ReTRIACt trial, and the prelaunch activities and commercialization plans in the EU. Nicklas Westerholm, CEO of Egetis, will also present the Company’s near-term strategic objectives and long-term ambitions.

Please follow this link to attend the webcast (no preregistration required):

https://www.redeye.se/events/1061014/egetis-therapeutics-investor-day

Agenda

Nicklas Westerholm, CEO of Egetis, commented: “The recommendation from the European Medicines Agency to grant marketing authorization for Emcitate® is the single most important milestone in Egetis’ history and a major step forward in building a sustainable rare disease company. I’m delighted to share our progress and plans with the investment community today and look forward to bringing the first approved medicine to MCT8 deficiency patients in the EU, pending the European Commission grants the marketing authorization.”

On December 12, 2024, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion, recommending the granting of a marketing authorisation for Emcitate®, intended for the treatment of MCT8 deficiency (1). The European Commission, which grants central marketing authorisations in the European Union (EU), will review the CHMP recommendation and is expected to make a final decision within 67 days. If approved, Emcitate® will become the first approved drug which addresses MCT8 deficiency.

During the Investor Day the Company will also give an update on the progress made in the ReTRIACt trial, which is pivotal for the New Drug Application (NDA) in the US. So far, 18 patients have been included, of which 8 patients have completed the randomized phase, 1 patient is in the randomized phase and 4 patients are in the run-in period. In January, 4 additional patients are planned for screening.

There is a continued large and increasing interest from physicians to treat patients suffering from MCT8 deficiency with Emcitate® (tiratricol), and it is already prescribed as part of different Managed Access Programs to patients in over 25 countries. In total, around 230 patients are now being treated, and more and more patients are gaining access to treatment.

Egetis’ strategy to build a sustainable rare-disease company also explores opportunities to extend the use of Emcitate® into other indications, like RTH-beta, which is a separate condition, with a non-overlapping patient population to MCT8 deficiency.

(1) https://www.egetis.com/mfn_news/egetis-receives-positive-chmp-opinion-for-emcitate-tiratricol-for-the-treatment-of-mct8-deficiency/