For further information, please contact:
nicklas.westerholm@egetis.com
+46 (0) 733 542 062
Yilmaz Mahshid, CFO
yilmaz.mahshid@egetis.com
+46 (0) 722 316 800
Karl Hård, Head of Investor Relations and Communications
karl.hard@egetis.com
+46 (0) 733 011 944
About
The Company’s lead candidate Emcitate 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) Emcitate has shown highly significant and clinically relevant results on serum T3 levels and secondary clinical endpoints. As a result of fruitful regulatory interaction Egetis intends to submit a marketing authorisation application (MAA) for Emcitate to the
In the US, after discussions with the FDA, Egetis will conduct a small 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 a new drug application (NDA) in the US for Emcitate in mid-2023 under the Fast-Track Designation granted by FDA.
Emcitate is currently being investigated in Triac Trial II, a Phase II/III study in very young MCT8 deficiency patients (<30 months of age) exploring potential disease modifying effects of early intervention from a neurocognitive and neurodevelopmental perspective. The recruitment target of 16 patients was reached in the beginning of
Emcitate holds Orphan Drug Designation (ODD) for MCT8 deficiency and resistance to thyroid hormone type beta (RTH-β) in the US and the EU. Emcitate has been granted Rare Pediatric Disease Designation (RPD) which gives Egetis the opportunity to receive a Priority Review Voucher (PRV) in the US, after approval.
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 with the purpose of applying for market approval in the US and
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