Egetis accelerates pre-launch activities for Emcitate® (tiratricol) in the EU

• Several pre-launch activities have commenced to increase disease awareness of MCT8 deficiency and its impact on patients, caregivers, and the healthcare system
• In the ReTRIACt study, which is pivotal for the New Drug Application in the USA, 13 patients have been included
• In Triac Trial II, which examines the neurocognitive effects of early intervention with tiratricol, top-line results are expected in mid 2024 after patients have completed the initial 96 weeks of treatment
• The Company is currently preparing responses to the 120-day list of questions from the EMA, which were received in February 2024, and plan to submit responses in August 2024, as agreed with the EMA

Financial overview January-March

• Quarterly revenue MSEK 12.1 (6.8)
• Quarterly loss MSEK -75.0 (-74.9)
• Cash at the end of the quarter amounted to MSEK 251.7 (243.5)
• Cash flow for the quarter was MSEK -56.0 (115.8)
• Earnings per share before/after dilution SEK -0.3 ( -0.3)

Significant events during the quarter

• Several pre-launch activities have commenced to increase disease awareness of MCT8 deficiency and its impact on patients, caregivers, and the healthcare system

Emcitate® (tiratricol)

• The Company is currently preparing responses to the 120-day list of questions from the EMA, which were received in February 2024, and plan to submit responses in August 2024, as agreed with the EMA
• In the ReTRIACt study, which is pivotal for the US NDA submission, 13 patients have been included so far, whereof 6 patients have completed the randomized phase. Recruitment will continue until at least 16 patients have completed the randomized phase
• The collaboration with Fujimoto is off to a good start and the development plan for tiratricol in Japan is being drafted ahead of regulatory interactions with the Japanese Pharmaceuticals and Medical Devices Agency (PMDA)

Comments from the CEO
As we are coming closer to an expected launch in the EU of Emcitate® (tiratricol) for treatment of MCT8 deficiency, we have continued to invest in pre-launch activities to increase disease awareness and the impact the disease has on patients, caregivers, and the healthcare system. We are investing in a stepwise fashion, in line with the clinical and regulatory progress we make.
 
Egetis works towards increased disease awareness of MCT8 deficiency and its impact on patients, caregivers and the healthcare system
MCT8 deficiency is an ultra-rare genetic disease first described in 2004, and there are currently no approved therapies for this disease. Consequently, the general awareness of the disease and the diagnosis are very low, even among specialist physicians, and a large portion of patients remain misdiagnosed. Our medical affairs activities are focused on improving awareness of the disease and its diagnosis, by participation and dialogues at scientific conferences, partnering with genetic testing companies, engaging with Key Opinion Leaders, advisory committees, and interactions with patient groups.
Recently we completed a Caregiver study, which evaluated the impact of MCT8 deficiency on caregivers of MCT8 deficiency patients.
In 2024 Egetis has so far participated at five scientific conferences relevant to MCT8 deficiency. At the American College of Medical Genetics Meeting in March, Egetis and collaborators presented a poster entitled Phenotypic spectrum of individuals with SLC16A2 variants: MCT8 deficiency (Allan-Herndon-Dudley syndrome), which was ranked among the top 20 posters at the conference.  
More information about MCT8 deficiency is available at www.mct8deficiency.com.
 
Two new hospitals have been included in the ReTRIACt study, which is pivotal in the USA
Following an agreement with the FDA, Egetis is conducting a pivotal, randomized, placebo-controlled study (ReTRIACt) in 16 evaluable patients to verify the results from previous clinical trials and publications regarding the normalization of thyroid hormone T3 levels, to support the submission of a New Drug Application (NDA) in the USA.
The first patients were included in the ReTRIACt study in July 2023, at Erasmus Medical Center in Rotterdam, The Netherlands, and Children’s Hospital of Philadelphia, USA. During late 2023 and early 2024 two additional hospitals have been included in the study: Addenbrooke’s Hospital in Cambridge, UK, and Saint Louis University Hospital in St. Louis, Missouri, USA. So far 13 patients have been included, whereof 6 patients have completed the randomized phase. Recruitment will continue until at least 16 patients have completed the randomized phase.
As previously communicated, we will update the market as soon as recruitment is closed. At that time, we will also provide information on when to expect topline results and when we plan to submit the NDA application.
More information about the ReTRIACt study is available on clinicaltrials.gov under the code NCT05579327.
 
Triac Trial II with tiratricol
Triac Trial II is an ongoing international, open-label, multicenter study in young patients (<30 months old) with MCT8 deficiency. The recruitment target was achieved in the second quarter of 2022, with 22 patients included. The study is being conducted in Europe and the USA and examines the neurocognitive effects of early intervention with tiratricol, as well as the effect on clinical and biochemical aspects of thyrotoxicosis. Patients are initially treated with tiratricol for 96 weeks and then followed for an additional two years. Results from the study, after 96 weeks of treatment, are expected in mid-2024. The design of the Triac Trial II study is available on clinicaltrials.gov under the code NCT02396459.
 
Egetis marketing authorisation application in the EU for tiratricol for the treatment of MCT8 deficiency
On October 26, 2023, the European Medicines Agency (EMA) validated the Marketing Authorisation Application (MAA) for Tiratricol for the treatment of MCT8 deficiency. This started the formal review of the MAA dossier by the Committee for Medicinal Products for Human Use (CHMP) at the EMA. The Company is currently preparing responses to the 120-day list of questions from the EMA, which were received in February 2024, and plan to submit responses in August 2024, as agreed with the EMA.
 
Tiratricol is available to qualifying patients through Egetis' Expanded Access Program in the USA
At the request of the FDA, Egetis has implemented an Expanded Access Program (EAP) in the USA.  Currently, 5 sites are now open to enroll patients in the EAP and an additional 12 hospitals are in the process of joining the program. The EAP program for tiratricol facilitates physicians in accessing the medication for their MCT8 deficiency patients who are ineligible for a clinical trial until the product receives market authorization. The program is also important for patients in the ReTRIACt study so that they can continue treatment with tiratricol after completing the study.
There is continued significant and growing interest from physicians worldwide in treating patients with MCT8 deficiency with tiratricol, which is already being prescribed as part of Managed Access Programs to patients in over 25 countries. Currently over 200 patients are being treated with tiratricol, and more patients are gaining access to treatment.
 
The collaboration with Fujimoto to develop and commercialize tiratricol in Japan is off to a good start
Egetis entered into an exclusive licensing agreement with Fujimoto Pharmaceutical Corporation in November 2023 to develop and commercialize tiratricol in Japan. The collaboration with Fujimoto is off to a good start and the development plan for tiratricol in Japan is being drafted, led by Fujimoto,  ahead of regulatory interactions with the Japanese Pharmaceuticals and Medical Devices Agency (PMDA).
 
The pivotal Albatross study for Aladote® (calmangafodipir)
The pivotal phase IIb/III study (Albatross) with Aladote to reduce the risk of acute liver injury associated with paracetamol (acetaminophen) overdose has been designed in consultation with the regulatory authorities FDA, EMA, and MHRA. There have been no activities related to calmangafodipir during the quarter. As previously communicated, the start of the study has been postponed until tiratricol marketing authorization submissions for MCT8 deficiency have been completed.
 
Cash
We report cash of approximately SEK 252 million as of March 31, 2024. In addition, we have access to a debt financing totaling EUR 15 million, which will be available provided that the Company meets certain conditions, including those related to the Phase III ReTRIACt study for tiratricol.
 
Outlook
2024 is a year marked by several important milestones for Egetis. Our team continues to focus on delivering five key priorities.:
1.         Complete the ReTRIACt study, which is pivotal in the USA, as soon as possible;
2.         Potential positive opinion from EMA for tiratricol for MCT8 deficiency;
3.         Results from the Triac Trial II study in mid-2024;
4.         Preparatory launch activities in Europe;
5.         Preparing the NDA for tiratricol in the USA.

Nicklas Westerholm, CEO