Egetis reports progress towards US NDA submission for tiratricol

·         FDA awarded tiratricol Breakthrough Therapy Designation (BTD) in July 2025, based on the Agency’s review of Egetis’ analysis of the survival data set from the international real-world cohort study by the Erasmus University Medical Center
·         There are 15 evaluable patients in the ReTRIACt study
·         In light of the above, Egetis has submitted a pre-NDA meeting request to the FDA to discuss the contents and timing of the US NDA submission for tiratricol, including the role and position of the ReTRIACt study
·         Egetis initiated the launch of Emcitate® in Germany on May 1, 2025
·         Egetis announced exclusive distribution agreement with Er-Kim for Emcitate® in Türkiye
 
Financial overview April-June
·         Quarterly revenue MSEK 14.5 (13.9)
·         Quarterly loss MSEK -77.6 (-71.9)
·         Cash at the end of the quarter amounted to MSEK 202.6 (192.6)
·         Cash flow for the quarter was MSEK -69.2 (-57.1)
·         Earnings per share before/after dilution SEK -0.2 (‑0.2)

Financial overview January-June
·         Revenue for the period MSEK 27.1 (25.9)
·         Net loss for the period MSEK -140.5 (-146.9)
·         Cash at the end of the period amounted to MSEK 202.6 (192.6)
·         Cash flow for the period was MSEK -143.4 (-113.1)
·         Earnings per share before/after dilution SEK -0.4 (‑0.5)
 
Significant events during the quarter
·         Egetis initiated the launch of Emcitate® in Germany on May 1, 2025
·         Egetis announced exclusive distribution agreement with Er-Kim for Emcitate® in Türkiye

Significant events after the quarter
·         Egetis received FDA Breakthrough Therapy Designation for tiratricol for MCT8 deficiency
·         There are 15 evaluable patients in the ReTRIACt study
·         Egetis submitted a pre-NDA meeting request to the FDA to discuss the contents and timing of the US NDA submission for tiratricol, including the role and position of the ReTRIACt study

Comments from the CEO
FDA’s Breakthrough Therapy Designation (BTD) for tiratricol for the treatment of monocarboxylate transporter 8 (MCT8) deficiency, which was granted on July 15, was based on the Agency’s review of Egetis’ analysis of survival data from the international cohort study conducted by Erasmus University Medical Center in Rotterdam, the Netherlands (van der Most et al. 2024). BTD is a process intended to expedite the development and review of drugs that are designed to treat a serious condition and where preliminary clinical data show that the drug may offer a substantial improvement over available treatments on a clinically relevant endpoint. Receiving a BTD this late in a clinical development program is very encouraging for the forthcoming NDA process, as these designations are typically awarded at an early stage in development.
In August we submitted a pre-NDA meeting request to the FDA and look forward to discussing the NDA submission for tiratricol with the Agency, with the aim of making this potential treatment available to patients in the US as soon as possible.
 
Update on the ReTRIACt study
Following a previous agreement with the FDA, we are conducting a randomized, placebo-controlled study (ReTRIACt) in patients with MCT8 deficiency to support the US NDA submission.
We currently have 15 evaluable patients in the ReTRIACt study. This study, together with the positive survival results acknowledged in the FDA-granted BTD, forms the basis for the planned NDA submission.
For more information on the ReTRIACt study, see https://clinicaltrials.gov/study/NCT05579327
 
Commercialization of Emcitate® in EU
On May 1 this year, we initiated the launch of Emcitate® in the first country, Germany. Almost all patients who participated in our Managed Access Program in Germany have been transitioned to the commercial product. At the same time, we have continued identifying new MCT8 patients, mainly by participating in scientific congresses and other disease awareness activities.
The German AMNOG process is highly structured and takes exactly one year before the final reimbursement price in Germany is determined by May 1, 2026. Our interaction with the AMNOG process is progressing according to plan.
The pricing and reimbursement process in France was initiated in April and preparations are progressing to initiate pricing and reimbursement processes in Italy and Spain. In parallel, we are progressing alternative funding mechanisms in some other EU countries.
As previously communicated, treatment with tiratricol for MCT8 deficiency is already included in the clinical guidelines from the European Thyroid Association.
 
Markets outside Europe and the US
Egetis has a license agreement with Fujimoto Pharmaceutical Corporation to develop and commercialize tiratricol in Japan. Discussions are currently ongoing with the Japanese pharmaceutical regulatory authority PMDA regarding the regulatory development pathway required to obtain approval of tiratricol in Japan.
In June, we signed an exclusive distribution agreement with the pharmaceutical company Er-Kim, headquartered in Istanbul, for Emcitate® in Türkiye.
Discussions are also ongoing with potential partners for an exclusive distribution agreement for the MENA (Middle East and North Africa) region.
 
Expanded Access Program for tiratricol in the US
At the request of the FDA, Egetis has implemented an Expanded Access Program (EAP) in the US. Currently, 14 hospitals are included in the EAP program. The EAP facilitates physicians’ access to tiratricol for their patients with MCT8 deficiency who are not eligible for a clinical trial until the product receives marketing authorization, as well as providing continued treatment to patients who have completed the ReTRIACt study.
For more information on the EAP program, see https://clinicaltrials.gov/study/NCT05911399
 
Cash position
We report a cash position of approximately SEK 203 million as of June 30, 2025. Discussions are still ongoing between the Company and BlackRock regarding the terms and a possible extension of the drawdown window for Tranche B of the loan facility of EUR 15 million.
 
Outlook
2025 is a year with several important milestones for Egetis. Our team continues to focus on delivering four key priorities:
 
·         Optimize pricing and reimbursement processes and launch in Europe
 
·         Pre-NDA meeting to discuss the NDA submission for tiratricol with the FDA and complete the ReTRIACt study
 
·         Initiate the submission of the NDA for tiratricol in the USA
 
·         Preparatory launch activities in the USA
 
Nicklas Westerholm, CEO