Egetis Therapeutics has announced promising new data indicating that tiratricol (Emcitate®) treatment is associated with significant survival benefits for patients with MCT8 deficiency. The findings, presented at the European Thyroid Association Annual Meeting, reveal an approximately three times lower risk of mortality compared to untreated patients. This development coincides with the European Thyroid Association's recommendation of tiratricol as a long-term therapy for all MCT8 deficiency patients in their new guidelines, marking a significant step forward in the treatment of this rare genetic disorder.
Tiratricol's Impact on MCT8 Deficiency
MCT8 deficiency, also known as Allan-Herndon-Dudley syndrome, is an X-linked disorder affecting thyroid hormone transport in the brain. This deficiency leads to severe neurological impairment and reduced life expectancy. There are currently no approved therapies, and the standard of care is largely supportive.
The new data presented by Dr. F. van der Most at the European Thyroid Association demonstrated a significant survival advantage for patients treated with tiratricol. The study compared mortality rates between treated and untreated MCT8 deficiency patients, revealing the threefold reduction in mortality risk. This finding underscores the potential of tiratricol to address the critical unmet need in managing this condition.
Regulatory Progress and Clinical Trials
Egetis Therapeutics is actively pursuing regulatory approval for tiratricol. The Marketing Authorisation Application (MAA) review by the European Medicines Agency (EMA) is ongoing, with the company responding to the Day 180 List of Outstanding Issues. The ReTRIACt study, a pivotal, randomized, placebo-controlled trial in the USA, has enrolled 17 patients, with 8 having completed the randomized phase. This study is crucial for the submission of a New Drug Application (NDA) to the FDA.
Nicklas Westerholm, CEO of Egetis, stated, "The review of the Company's MAA application with the EMA is progressing according to the EMA's stipulated timelines, and we are responding to additional questions along the path toward a potential market approval."
Managed Access Programs and Expanded Access Program
Tiratricol is currently available to over 220 patients through Managed Access Programs in over 25 countries. In the USA, an Expanded Access Program (EAP) has been implemented at the request of the FDA to provide access to tiratricol for MCT8 deficiency patients ineligible for clinical trials until market authorization is obtained.
Additional Positive Outcomes
New post-hoc analysis of Triac Trial I data indicates improvements reported by caregivers, including improved interaction (22/39), greater alertness (19/39), improved motor skills (12/39), improved head control (7/39), and improved sleep (8/39) with tiratricol treatment.
Financial Position
Egetis reported cash reserves of approximately SEK 130 million as of September 30, 2024, with an additional SEK 282 million received post-period from directed share issuances.
