Stockholm, Sweden, July 24, 2023. Egetis Therapeutics AB (publ) (Nasdaq Stockholm: EGTX) today announced that the first patient has been included and a second site has been activated in the pivotal ReTRIACt clinical trial for the new drug application (NDA) in the USA for Emcitate. The trial will be conducted across three centers: at Erasmus Medical Center, Rotterdam, the Netherlands, Children’s Hospital of Philadelphia, Philadelphia, PA, USA, and Addenbrooke’s Hospital, Cambridge, UK. As previously reported, the Company expects topline results from the ReTRIACt study during the first half of 2024 and estimates a subsequent NDA submission in the USA in mid-2024, under the fast-track designation.
Nicklas Westerholm, CEO of Egetis, commented: “I am delighted that the first patient has been included and that we now have two active sites in the ReTRIACt trial for Emcitate, which is pivotal for the New Drug Application in the USA. These are important milestones for the Company, and I would like to thank the investigators, patients, and parents/guardians for their commitments to participate in this trial, which is crucial for bringing the first possible treatment for MCT8 deficiency to patients in the USA. We continue to work diligently with the trial sites to facilitate a smooth and efficient execution of the ReTRIACt trial. For the EU, we are on track towards submitting the Marketing Authorisation Application for Emcitate in the early autumn of 2023, as previously communicated.”
About the ReTRIACt trial
The ReTRIACt trial (clinicaltrial.gov identifier NCT05579327) is a double-blind, randomized Phase 3 multicenter placebo-controlled study in at least 16 evaluable male participants diagnosed with MCT8 deficiency. The study protocol starts with an open-label treatment period in which a stable maintenance dose of tiratricol, essential for progression into the Randomized Treatment Period, will be established. The duration of the initial open-label treatment period will vary depending on whether the participant is currently receiving treatment with tiratricol at the time of enrollment in the study (Cohort A), or if they are considered to be tiratricol treatment-naïve (Cohort B). Participants are considered to be tiratricol-naïve if they have never previously been administered tiratricol, or have previously received tiratricol but are not receiving tiratricol at the time of enrollment. Participants, from 4 years of age and having demonstrated stable maintenance treatment with Emcitate, will be randomized to receive placebo or Emcitate for 30 days or until reaching rescue criterion (serum total triiodothyronine [T3] above upper limit of normal [ULN] of the participant's normal range, for a sample collected during the 30-day Randomized Treatment Period). The research hypothesis to be tested is that, for participants in the placebo group, removal of Emcitate will lead to an increase of serum total T3 concentration above the ULN and requirement of rescue treatment with Emcitate, compared to those who continue to receive tiratricol.
