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Egetis submits marketing authorisation application for Emcitate for treatment of MCT8 deficiency to the European Medicines Agency

·        Submission based on efficacy data from two studies in a total of 86 MCT8 deficiency patients with up to 6 years of treatment with Emcitate
·        To date an accumulated 500 patient years of Emcitate treatment have been gathered in patients with MCT8 deficiency
·        If approved, Emcitate would become the first treatment for MCT8 deficiency

Stockholm, Sweden, October 9, 2023. Egetis Therapeutics AB (publ) (“Egetis” or the “Company”) (Nasdaq Stockholm: EGTX), today announced that the Company has submitted a marketing authorisation application (MAA) to the European Medicines Agency (EMA) for Emcitate (tiratricol) for the treatment of MCT8 deficiency. This is an important step towards bringing the first approved treatment for MCT8 deficiency to patients and a transformative milestone for the Company.

As agreed with the EMA, the MAA submission is based on results from Triac Trial I (Groeneweg et al. 2019), which showed statistically significant and clinically relevant treatment effects on key aspects of the disease, including reduction in serum thyroid hormone T3 concentrations, as well as improvements in bodyweight, heart rate and blood pressure. The submission is supported by real-world, retrospective data collected in a multi-centre cohort study in which patients were treated with Emcitate for up to six years (van Geest et al. 2022).

Nicklas Westerholm, CEO of Egetis, commented: “MCT8 deficiency is a severely debilitating ultra-rare disease without any approved treatments. We are delighted to have reached this transformative milestone for the Company in our efforts to bring the first approved treatment of MCT8 deficiency to patients. We look forward to engaging with the EMA on our submission.”
Emcitate has been granted Orphan Drug Designation by the EMA for MCT8 deficiency, and will following approval by the European Commission be eligible for 10 years of market exclusivity within the EU. The median review time for marketing authorisation applications in the EU is around 13-14 months.

As agreed with the US FDA, Egetis is conducting a randomized, placebo-controlled pivotal study (ReTRIACt) in 16 evaluable patients to verify the results on thyroid hormone T3 levels seen in previous clinical trials and publications. The study started recruiting in July this year and patient recruitment is proceeding according to plan. Topline results are expected during the first half of 2024 and Egetis intends to submit a new drug application (NDA) in the USA for Emcitate in mid-2024 under the Fast-Track Designation granted by the FDA.

In addition to the completed and ongoing clinical trials with Emcitate, more than 180 patients in over 25 countries, are currently being treated with Emcitate as part of the Company’s Expanded Access/ Named Patient Use/ Compassionate Use programs.

Links to clinical trials:
Triac Trial I

Groeneweg, S. et al. Lancet Diabetes Endocrinol. 2019; 7(9):695-706
Van Geest, F.S. et al. J. Clin. Endocrinol. Metab. 2022; 107(3):e1136-e1147