Stockholm, Sweden, April 06, 2022. Egetis Therapeutics AB (publ) (Nasdaq Stockholm: EGTX) today announced that the recruitment target of 16 patients has been met in the Triac Trial II clinical study with Emcitate® (tiratricol) in patients with MCT8 deficiency.
Triac Trial II (clinicaltrials.gov identifier NCT02396459) is an ongoing international, open label, multi-center study in children with MCT8 deficiency, conducted in both Europe and North America, investigating neurocognitive effects of early intervention with Emcitate in very young (<30 months of age) patients. The first patient was dosed in December 2020. The primary endpoint is to evaluate the effects of Emcitate after 96 weeks of treatment as measured by the Gross Motor Function Measure (GMFM)-88 assessment and the Bayley Scales of Infant Development (BSID-III) Gross Motor Skill Domain score. The study will also evaluate the effect of Emcitate on specific motor milestones, such has holding head and sitting independently, as well as the effect on clinical and biochemical thyrotoxic features.
The recruitment target of 16 patients has been met in the Triac Trial II clinical study but to include additional patients who has already been identified but not yet screened, the trial will remain open for recruitment a few additional weeks. Results of Triac Trial II are expected in the first quarter of 2024 and are expected to be submitted post-approval to regulatory authorities.
Data from preclinical studies suggest that Emcitate restores abnormal neuronal development and myelination in animal models of MCT8 deficiency if given in early postnatal life. In the Triac Trial I study, effects on neurocognitive development were included as exploratory endpoints (in a subset of patients) andthe results indicate a potential for Emcitate to positively influence neurocognitive development when treatment is introduced early, with the largest increase in gross motor function seen in patients where treatment was started before the age of 4 years.
Nicklas Westerholm, CEO of Egetis, commented: “We are very excited to have reached this important recruitment milestone for Triac Trial II. Although Triac Trial II is not required for regulatory approvals for Emcitate it remains an important study for establishing neurodevelopmental effects of early intervention with Emcitate. We would like to thank participating families, study staff and investigators for taking part in Triac Trial II.”
As a result of fruitful regulatory interactions, Egetis intends to submit a marketing authorisation application (MAA) for Emcitate to the European Medicines Agency (EMA) in the first half of 2023 based on existing clinical data.
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 has been granted Rare Pediatric Disease Designation (RPD) which gives Egetis the opportunity to receive a Priority Review Voucher (PRV) in the US.
For further information, please contact:
Nicklas Westerholm, CEO
Tel. +46 (0) 733 542 062
[email protected]
Karl Hård, Head of Investor Relations and Communications
Tel. +46 (0) 733 011 944
[email protected]
The information was submitted for publication, through the agency of the contact persons set out above, on April 06, at 8:00 CET.
