Egetis Therapeutics asks for hugs to increase disease awareness of MCT8 deficiency

About Egetis Therapeutics

Egetis Therapeutics is an innovative and integrated pharmaceutical drug development company, focusing on projects in late-stage development for treatment of serious diseases with significant unmet medical needs in the orphan drug segment. The drug candidate Emcitate is developed as the first potential treatment for patients with MCT8 deficiency, a rare disease with high unmet medical need and no available treatment. Triac Trial I (Phase IIb) and a long-term real-life study have been completed with clinically relevant and highly significant results on serum T3 concentrations and secondary clinical endpoints. Triac Trial II is an ongoing study in very young MCT8 deficiency patients (<30 months of age) investigating neurocognitive effects of early intervention with Emcitate. Results are expected in Q1 2024. Egetis intends to submit a marketing authorization application for Emcitate to the European Medicines Agency in H1 2023 based on existing clinical data. As a result of fruitful FDA interactions, Egetis will conduct a randomized, placebo-controlled study in 16 patients to verify the results on T3 levels seen in previous clinical trials and publications. Egetis intends to submit an NDA in the US for Emcitate in mid-2023 under the Fast Track Designation granted by the FDA. Emcitate holds Orphan Drug Designation (ODD) in the US and EU and has been granted Rare Pediatric Disease Designation.
The drug candidate Aladote is a first in class drug candidate developed to reduce the risk of acute liver injury associated with paracetamol (acetaminophen) poisoning. A proof of principle study has been successfully completed and the design of the upcoming pivotal Phase IIb/III study for Aladote has been finalized after completed interactions with FDA, EMA and MHRA. Aladote has been granted ODD in the US and an application for ODD was submitted in Europe in Q1 2021. There is an ongoing dialogue with EMA on the appropriate indication for an ODD in the EU.
Egetis Therapeutics (STO: EGTX) is listed on the Nasdaq Stockholm main market. For more information, see www.egetis.com

About MCT8 Deficiency

Monocarboxylate transporter 8 (MCT8) deficiency is a rare genetic disease with high unmet medical need and no available treatment, affecting 1:70,000 males. Thyroid hormone is crucial for the development and metabolic state of virtually all tissues. Thyroid hormone transport across the plasma membrane is required for the hormone’s metabolism and intracellular action and is facilitated by thyroid hormone transporters, including MCT8. Mutations in the gene for MCT8, located on the X-chromosome, cause MCT8 deficiency, also called Allan-Herndon-Dudley syndrome (AHDS) in affected males. The resulting dysfunction of MCT8 leads to impaired transport of thyroid hormone into certain cells and across the blood-brain-barrier and disruption of normal thyroid hormone regulation. This leads to a complex pattern of symptoms with neurological developmental delay and intellectual disability, accompanied by strongly elevated circulating thyroid hormone concentrations which are toxic for tissues including the heart, muscle, liver and kidney and results in symptoms such as failure to thrive, cardiovascular stress, insomnia and muscle wasting. Most patients will never develop the ability to walk or even sit independently. At present there is no approved therapy available for the treatment of patients with MCT8 deficiency.