Egetis Therapeutics AB has announced the submission of a patent application to the United States Patent and Trademark Office (USPTO) for "Processes of Preparation" of tiratricol. This patent, if granted, would represent a significant advancement in the production of the investigational drug.
Tiratricol is an endogenously available metabolite of thyroid hormone, exhibiting similar bioactive properties to T3. It can enter cells independently of the monocarboxylate transporter 8 (MCT8), which is particularly relevant in treating MCT8 deficiency, where this transport mechanism is impaired. Clinical trials are ongoing to evaluate tiratricol's efficacy in treating MCT8 deficiency, and a marketing authorisation application (MAA) was submitted to the European Medicines Agency (EMA) in October 2023.
Significance of the Patent
The newly patented processes and compounds are designed to improve the synthetic routes for tiratricol, addressing the increasing need for efficient production methods as clinical development progresses. Egetis currently holds Orphan Drug Designation (ODD) for tiratricol (Emcitate®) in both the US and EU for MCT8 deficiency, granting marketing exclusivities of 7 and 10 years, respectively, from regulatory approval dates. The new patent, if approved, would add an exclusivity term of 20 years from the application filing date.
Ongoing Clinical Development
Egetis is currently conducting a randomized, placebo-controlled pivotal study involving 16 patients to confirm previous findings on T3 levels. This study follows discussions with the FDA and aims to support a future New Drug Application (NDA) filing. The company plans to update the market upon completion of recruitment, including the expected timing for top-line results and subsequent NDA submission.
About Tiratricol and MCT8 Deficiency
Tiratricol (Emcitate®) is being developed for patients with monocarboxylate transporter 8 (MCT8) deficiency, a rare and debilitating disease for which there is currently no approved treatment. Prior studies have demonstrated clinically relevant results on serum thyroid hormone T3 levels and other clinical endpoints. Tiratricol also holds Orphan Drug Designation for resistance to thyroid hormone type beta (RTH-beta), a separate indication with a distinct patient population. Additionally, Emcitate® has been granted Rare Pediatric Disease Designation (RPDD), potentially entitling Egetis to a Priority Review Voucher (PRV) upon approval, which can be sold or transferred.
