New study reports tiratricol (Emcitate®) treatment in patients with MCT8 deficiency is associated with survival benefits
- An Abstract published ahead of the European Thyroid Association's Annual Meeting reports that treatment with tiratricol (Emcitate®) in pediatric and adult patients with MCT8 deficiency is associated with an approximately three times lower risk of mortality.
- European Thyroid Association recommended tiratricol as long-term therapy for all patients with MCT8 deficiency in new guidelines.
- In the ReTRIACt study, which is pivotal for the New Drug Application in the USA, 6 patients have completed the study, and Egetis estimates that 2 patients will be randomized and a further 6 to commence screening during the next 6 weeks. To increase the recruitment capacity in the ReTRIACt study, the Company has opened three additional study centers in the USA.
- The non-pivotal Triac Trial II did not meet its primary neurodevelopmental endpoints in the studied subpopulation, despite numerical improvements versus baseline. The study confirmed the significant and durable reduction of endogenous T3 concentrations and the well-tolerated safety profile of tiratricol seen in previous clinical studies.
- Tiratricol (Emcitate®) is being prescribed via Managed Access Programs to approximately 220 patients.
- The Company submitted responses on August 15, 2024, to the Day 120 List of Questions which form part of the EMA review process of the Marketing Authorisation Application in the EU.
Financial overview April-June
· Quarterly revenue MSEK 13.9 (5.9)
· Quarterly loss MSEK -71.9 (-79.5)
· Cash at the end of the quarter amounted to MSEK 192.6 (179.2)
· Cash flow for the quarter was MSEK -57.1 (-64.8)
· Earnings per share before/after dilution SEK -0.2 (-0.3)
Financial overview January-June
· Revenue for the period MSEK 25.9 (12.7)
· Net loss for the period MSEK -146.9 (-154.4)
· Cash at the end of the period amounted to MSEK 192.6 (179.2)
· Cash flow for the period was MSEK -113.1 (51.0)
· Earnings per share before/after dilution SEK -0.5 (-0.6)
Significant events during the quarter
· Egetis awarded Promising Innovative Medicine designation in the UK for tiratricol (Emcitate®) for treatment of MCT8 deficiency.
· Tiratricol (Emcitate®) is being prescribed via Managed Access Programs to approximately 220 patients.
· Egetis announced topline results of the Phase 2 Triac Trial II with tiratricol for MCT8 deficiency, given to young boys <30 months of age during two years.
Significant events after the quarter
· European Thyroid Association recommended tiratricol as long-term therapy for all patients with MCT8 deficiency in new guidelines.
· Egetis submitted responses to the European Medicines Agency's Day 120 List of Questions for the Marketing Authorisation Application for tiratricol.
· An Abstract published ahead of the European Thyroid Association's Annual Meeting shows that treatment with tiratricol (Emcitate®) in pediatric and adult patients with MCT8 deficiency is associated with an approximately three times lower risk of mortality.
Comments from the CEO
The last few months have been eventful for Egetis. We are pleased by the important recommendations from the European Thyroid Association (ETA) to use tiratricol as long-term therapy for all patients with MCT8 deficiency. We are also looking forward to the new survival data which will be presented at the ETA Annual Meeting in September.
While Triac Trial II did not meet the neurodevelopmental endpoints in the studied subpopulation, it confirmed the well-tolerated safety profile seen in previous clinical studies, despite higher dosing per kg body weight compared to previous studies.
Tiratricol (Emcitate®) treatment in patients with MCT8 deficiency is associated with three times lower risk of mortality
Treatment with the investigational drug tiratricol (Emcitate®) in pediatric and adult patients with MCT8 deficiency is associated with an approximately three times lower risk of mortality compared to MCT8 deficiency patients not treated with tiratricol. These new encouraging data are presented in an Abstract by Dr Floor van der Most, and co-authors, Erasmus Medical Center, Rotterdam, The Netherlands, published ahead of the 46th Annual Meeting of the European Thyroid Association, to be held in Athens, Greece, on September 7-10, 2024.
European Thyroid Association recommended tiratricol as long-term therapy for all patients with MCT8 deficiency in new guidelines
Early July, The European Thyroid Association (ETA) published new guidelines recommending the use of tiratricol as long-term therapy for all patients with MCT8 deficiency, and for certain patients with Resistance to Thyroid Hormone (RTH)-beta, as further outlined in the guidelines.
These inaugural 2024 European Thyroid Association Guidelines on diagnosis and management of genetic disorders of thyroid hormone transport, metabolism and action were commissioned by the Executive Committee of the ETA and developed by an independent team of experts.
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 submitted responses on August 15, 2024, to the Day 120 List of Questions from the EMA.
Egetis continues to work 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.
In 2024 Egetis has so far participated at 17 scientific conferences relevant to MCT8 deficiency.
More information about MCT8 deficiency is available at www.mct8deficiency.com.
Three new clinical study sites have been added to 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 at least 16 evaluable patients with MCT8 deficiency 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 were included in the study: Addenbrooke's Hospital in Cambridge, UK, and Saint Louis University Hospital in St. Louis, Missouri, USA. To increase the recruitment capacity in the ReTRIACt study we have opened three additional clinical study sites in the USA: one each in Texas, Georgia and North Carolina. So far, 13 patients have been included, whereof 6 patients have completed the study, and Egetis estimates that 2 patients will be randomized and a further 6 to commence screening during the next 6 weeks.
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 international, open-label, multicenter study in young patients (<30 months old) with MCT8 deficiency with 22 patients included. The study is conducted in Europe and the USA and examined the neurodevelopmental effects of early intervention with tiratricol, as well as the effect on clinical and biochemical aspects of thyrotoxicosis. Patients were initially treated with tiratricol for 96 weeks and will be followed for an additional two years. Top-line results from the study, after 96 weeks of treatment, were announced on June 19, 2024. The trial did not meet its primary endpoints, which were assessed by changes in the Gross Motor Function Measure (GMFM)-88 total score and the Bayley Scales for Infant and toddler Development (BSID)-III Gross Motor Skill domain, compared to natural history scores from the Triac Trial I. Among key secondary endpoints, total serum thyroid hormone T3 concentrations were reduced significantly and durably in all patients, thereby verifying tiratricol's ability to alleviate thyrotoxicosis in MCT8 deficiency patients.
As agreed with the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA), the Triac Trial II study is complementary to the already demonstrated treatment effects on T3 concentrations and chronic thyrotoxicosis in patients of all ages, which the already submitted and validated marketing authorization application for Emcitate® (tiratricol) for the treatment of MCT8 deficiency in the EU is based on.
The trial confirmed the well-tolerated safety profile seen in previous clinical studies, despite higher dosing per kg body weight compared to previous studies. The design of the Triac Trial II study is available on clinicaltrials.gov under the code NCT02396459.
Egetis awarded Promising Innovative Medicine designation in the UK for tiratricol (Emcitate®) for treatment of MCT8 deficiency
A Promising Innovative Medicine Designation (PIM) designation is a recognition of tiratricol as a promising candidate drug in the UK for the treatment of patients with MCT8 deficiency. MCT8 deficiency is a debilitating condition, and we are delighted that the MHRA has recognized the potential of Emcitate® (tiratricol) for patients living with this disease. This is testament to our commitment to supporting patients with MCT8 deficiency in the UK.
Managed access program for tiratricol
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 approximately 220 patients are being treated with tiratricol, and more patients are gaining access to treatment.
At the request of the FDA, Egetis has implemented an Expanded Access Program (EAP) in the USA. Currently, 8 sites are open to enroll patients in the EAP and an additional 10 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.
Cash
We report cash of approximately SEK 193 million as of June 30, 2024. In addition, we have access to 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 four 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. Preparatory launch activities in Europe;
4. Preparing the NDA for tiratricol in the USA.
Nicklas Westerholm, CEO
For further information, please contact
Nicklas Westerholm, CEO
nicklas.westerholm@egetis.com
+46 (0) 733 542 062
Yilmaz Mahshid, CFO
yilmaz.mahshid@egetis.com
+46 (0) 722 316 800
Karl Hård, Head of Investor Relations & Business Development
karl.hard@egetis.com
+46 (0) 733 011 944
This information is information that Egetis Therapeutics is obliged to make public pursuant to the EU Market Abuse Regulation and the Securities Markets Act. The information was submitted for publication, through the agency of the contact persons set out above, at 2024-08-22 07:00 CEST.
About Egetis Therapeutics
Egetis Therapeutics is an innovative and integrated pharmaceutical company, focusing on projects in late-stage development for commercialization for treatments of serious diseases with significant unmet medical needs in the orphan drug segment.
The Company's lead drug candidate Emcitate® (tiratricol) is under development for the treatment of patients with monocarboxylate transporter 8 (MCT8) deficiency, a highly debilitating rare disease with no available treatment. In previous studies (Triac Trial I and a long-term real-life study) Emcitate has shown highly significant and clinically relevant results on serum thyroid hormone T3 levels and secondary clinical endpoints. Egetis submitted a marketing authorisation application (MAA) for Emcitate to the European Medicines Agency (EMA) in October 2023.
After a dialogue with the FDA, Egetis is conducting a randomized, placebo-controlled pivotal study in 16 evaluable patients to verify the results on T3 levels seen in previous clinical trials and publications. Egetis will update the market as soon as recruitment has been completed and at that point inform about the timing of availability of top-line results, and the expected timing of the subsequent NDA filing.
Emcitate holds Orphan Drug Designation (ODD) for MCT8 deficiency and resistance to thyroid hormone type beta (RTH-beta) in the US and the EU. MCT8 deficiency and RTH-beta are two distinct indications, with no overlap in patient populations. Emcitate has been granted Rare Pediatric Disease Designation (RPDD) which gives Egetis the opportunity to receive a Priority Review Voucher (PRV) in the US, after approval. This voucher can be transferred or sold to another sponsor.
The drug candidate Aladote® (calmangafodipir) is a first in class drug candidate developed to reduce the risk of acute liver injury associated with paracetamol (acetaminophen) overdose. A proof of principle study has been successfully completed. The design of a pivotal Phase IIb/III study (Albatross), with the purpose of applying for market approval in the US and Europe, has been finalized following interactions with the FDA, EMA and MHRA. The study start has been postponed until Emcitate marketing authorization submissions for MCT8 deficiency have been completed. Aladote has been granted ODD in the US and in the EU.
Egetis Therapeutics (STO: EGTX) is listed on the Nasdaq Stockholm main market. For more information, see www.egetis.com
