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Egetis is an innovative and integrated pharmaceutical company, focusing on projects in late-stage development for commercialization for treatments of serious diseases with significant unmet medical needs in the orphan drug segment

First patient dosed in Phase IIb/III study with Emcitate®

December 7, 2020

Stockholm, December 07, 2020. PledPharma AB (publ) (STO: PLED) announced today that the first patient has been dosed in the pivotal Phase IIb/III early intervention study in young patients with the drug candidate Emcitate®, a novel therapy developed to treat MCT8 deficiency, a genetic disturbance in thyroid hormone signalling with life-long severe disability.

Emcitate is developed for the treatment of MCT8 deficiency, a genetic disturbance in thyroid hormone trafficking with detrimental natural history and no currently available therapy. The pivotal Phase IIb/III study is an international, open label, multi-centre study in very young children with MCT8 deficiency, run at 10 centres in both Europe and North America. 12-22 children 0-30 months of age is planned to be included in the study, the design of which has been discussed and anchored with the regulatory authorities European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA).
The efficacy endpoints in the study are improvement of neurocognitive development, achievement of motor milestones, and a normalisation of thyroid hormone function tests and markers of thyrotoxicosis. The recruitment of patients is projected to be completed in H2 2021, with interim results in 2022 to pave the way for regulatory submission, market approvals and commercial launch.
“We see a great potential for Emcitate to be beneficial to patients with MCT8 deficiency where there is no available treatment today. The start of this study confirms our commitment to build a specialized late-stage orphan drug development company, which will be renamed Egetis Therapeutics, that was formed when PledPharma recently acquired Rare Thyroid Therapeutics.,” said Nicklas Westerholm, CEO of PledPharma.



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