About Resistance to thyroid hormone (RTH-β)

RTH-β (Resistance to thyroid hormone) is a rare genetic disorder with high unmet medical need and no approved treatment, affecting 1:20,000-40,000 individuals. Thyroid hormone is crucial for the development and metabolic state of virtually all tissues and acts through binding to a nuclear receptor resulting in transcription of a range of hormone responsive genes. There are two subtypes of thyroid hormone receptors in the body (alpha and beta), preferentially expressed in different tissues. RTH-β is caused by mutations in the thyroid hormone receptor beta and leads to impaired thyroid hormone signaling in tissues dependent on this receptor subtype. Clinical manifestations of RTH-β include a mix of symptoms of thyrotoxicosis and hypothyroidism in different tissues, including goiter, hepatic steatosis and dyslipidemia, impaired hearing and color vision, neurocognitive dysfunction and cardiovascular stress. RTH-β affects both genders equally and normally displays an autosomal dominant inheritance pattern. Homozygous disease is extremely rare and results in pronounced symptoms normally leading to death during early childhood.

About Egetis Therapeutics AB

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 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