Stockholm, Sweden, October 17, 2022. Egetis Therapeutics AB (publ) (NASDAQ Stockholm: EGTX) today announced the detailed design for the small, randomized, placebo-controlled trial for the new drug application (NDA) in the United States for its leading candidate drug Emcitate, which is being developed for the treatment of monocarboxylate transporter 8 (MCT8) deficiency.
Activities are ongoing to support a submission of a marketing authorisation application (MAA) to the European Medicines Agency (EMA) in the first half of 2023, based on already available clinical data. The NDA to the US FDA is targeted for mid-2023 under the Fast Track Designation granted by the FDA. As previously communicated, the NDA requires a small, randomized, placebo-controlled trial with 16 patients, and up to 30 days treatment duration with either Emcitate or placebo, to be conducted to verify the results on thyroid hormone T3 levels seen in previous clinical trials and publications. An application for this clinical trial has been agreed with the US FDA and the detailed design of the trial is now available on www.clinicaltrials.gov with identifier NCT05579327. The study will commence during the next month.
Nicklas Westerholm, CEO of Egetis, said: ”I am delighted to report that we progress towards submitting the MAA for Emcitate in the first half of 2023 and the NDA mid-2023 under the Fast Track Designation granted by the FDA. For the United States we will initiate the small, randomized, placebo-controlled trial with 16 patients, which is required for the new drug application, as soon as possible now when the detailed design has been agreed with the FDA. At the same time, we are continuing the step-wise build up of a commercial infrastructure in Europe and the US.”
About the Emcitate randomized placebo-controlled study for the NDA in the United States (ReTRIACt)
This is a double-blind, randomized Phase 3 multicenter placebo-controlled study in at least 16 male participants diagnosed with MCT8 deficiency. 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 tri-iodothyronine [T3] > upper limit of normal [ULN] of the participant's normal range, for a sample collected during the 30-day Randomized Treatment Period). The primary endpoint is the proportion of participants in the placebo group, compared to those who continue to receive Emcitate, for which removal of Emcitate will lead to an increase of serum total T3 concentration above the ULN.
