Egetis Therapeutics

Egetis successfully carried out directed share issuances amounting to SEK 300 million

·        The Company’s MAA review for tiratricol (Emcitate®) at the EMA remains on track according to EMA’s stipulated timelines.
·        New data shows tiratricol (Emcitate®) treatment in patients with MCT8 deficiency is associated with survival benefits.
·        The 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, 17 patients have been included, whereof 8 patients have completed the randomized phase.
·        Tiratricol (Emcitate®) is being prescribed via Managed Access Programs to over 220 patients.

Egetis will host a conference call today at 10:00 am CET to discuss the third quarter 2024 financial results and recent corporate progress.
If you wish to participate via webcast, please use the link below. You can ask written questions via the webcast.
https://ir.financialhearings.com/egetis-therapeutics-q3-report-2024/register  
If you wish to participate via teleconference, please register via the link below. After registration you will be provided with phone numbers and a conference ID to access the conference. You can ask questions verbally via the teleconference. 
https://conference.financialhearings.com/teleconference/?id=5003940
The conference call will be made available on the Company’s website after the call.

Financial overview July-September
·        Quarterly revenue MSEK 9.4 (12.2)
·        Quarterly loss MSEK -86.2 (-86.2)
·        Cash flow for the quarter was MSEK -63.2 (-94.2)
·        Cash at the end of the quarter amounted to MSEK 129.9 (85.0)
·        Earnings per share before/after dilution SEK -0.3 (‑0.3)

Financial overview January-September
·        Revenue for the period MSEK 35.3 (25.0)
·        Net loss for the period MSEK -233.1 (-240.7)
·        Cash flow for the period was MSEK -176.2 (-43.2)
·        Cash at the end of the period amounted to MSEK 129.9 (85.0)
·        Earnings per share before/after dilution SEK -0.8 (‑1.0)

Significant events during the quarter
·        New data shows tiratricol treatment in patients with MCT8 deficiency is associated with survival benefits.
·        The 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.
·        New post-hoc analysis reports positive effects of tiratricol on patient-centered outcome measures in patients with MCT8 deficiency in Triac Trial I.
·        Egetis submitted a patent application to the United States Patent and Trademark Office for “Processes of Preparation” of tiratricol (Emcitate®).
·        In the ReTRIACt study, which is pivotal for the New Drug Application in the USA, 17 patients have been included, whereof 8 patients have completed the randomized phase.
·        Tiratricol is being prescribed via Managed Access Programs to over 220 patients.

Significant events after the quarter
·        On October 17, 2024, Egetis received the Day 180 List of Outstanding Issues (LoOI) to its MAA for tiratricol (Emcitate®) and plans to respond to these by November 12, according to EMA’s published procedural timetables.
·        Egetis successfully carried out directed share issuances, at an ‘at market’ price of SEK 4.50, amounting to SEK 300 million (gross). The Directed Issue was oversubscribed and included both existing and new international and Swedish institutional investors. It was led by US healthcare investor Frazier Life Sciences with a USD 10 million investment.
·        Peder Walberg resigned from the Board of Directors, but he will continue to support the Company as a major shareholder and as a consultant for ongoing operational work.

Comments from the CEO
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. I’m pleased and proud that we could successfully carry out directed share issuances of shares at an ‘at market’ subscription price of SEK 4.50 per share, through which the Company received SEK 300 million (approximately USD 30 million) before transaction costs, as announced on September 30, 2024. This new funding indicates investors' continued confidence in our product candidate, our team, and our work.
The Directed Issue was oversubscribed and included both existing and new international and Swedish institutional investors. It was led by US healthcare investor Frazier Life Sciences with a USD 10 million investment and supported by the international healthcare specialist Invus (USA/France), as well as Platinum Asset Management (Australia), The Fourth Swedish National Pension Fund, Handelsbanken Fonder AB through the investment fund Hälsovård Tema (Sweden), Unionen (Sweden), HealthInvest Partners AB (Sweden) and Cidro Förvaltning AB (Sweden).
 
Egetis marketing authorisation application in the EU for tiratricol for the treatment of MCT8 deficiency
In October 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. On October 17, 2024, Egetis received the Day 180 List of Outstanding Issues (LoOI) and plans to respond to these by November 12, according to EMA’s published procedural timetables.
 
Tiratricol treatment in patients with MCT8 deficiency is associated with three times lower risk of mortality
Treatment with tiratricol 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, according to data presented in an oral presentation by Dr F. van der Most, Erasmus Medical Center, Rotterdam, The Netherlands, at the Annual Meeting of the European Thyroid Association, on September 9, 2024. More information is available at https://www.egetis.com/mfn_news/new-data-shows-tiratricol-emcitate-treatment-in-patients-with-mct8-deficiency-is-associated-with-survival-benefits/
 
The 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 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 improve 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. So far in 2024, Egetis has participated at 29 scientific conferences on topics such as endocrinology, pediatrics, and neurology, where MCT8 deficiency has been presented.
More information about MCT8 deficiency is available at https://www.mct8deficiency.com/
 
Update on the ReTRIACt study
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 support the submission of a New Drug Application (NDA) in the USA.
To increase the recruitment capacity in the study three additional clinical study sites have been opened in the USA in 2024: one each in Texas, Georgia and North Carolina. So far, 17 patients have been included, whereof 8 patients have completed the randomized phase. This includes the 2 patients we expected to be randomized in the Q2 report. We now have 4 patients in the run-in period and further recruitment continues with full focus.
As previously communicated, we will update the market as soon as recruitment of the ReTRIACt trial 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.
 
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 over 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, 10 sites are open to enroll patients in the EAP and an additional 8 hospitals are in the process of joining the program. The EAP program facilitates physicians in accessing tiratricol 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.
 
New post-hoc analysis reports positive effects of tiratricol on patient-centered outcome measures in patients with MCT8 deficiency in Triac Trial I
New data were presented by Dr M. Freund on September 9, 2024, at the Annual Meeting of the European Thyroid Association. There were improvements upon tiratricol treatment reported by caregivers related to improved interaction (22/39), greater alertness (19/39), improved motor skills (12/39), improved head control (7/39), and improved sleep (8/39). For 1 patient, also negative changes were reported, specifically increased constipation and higher unsettledness. Compared to the baseline visit, excessive sweating was much less reported (48.6% vs. 8.1%) and less salivary flow was observed (30.6% vs. 22.2%) by the caregivers at the end study visit. Seizures and continence were reportedly unchanged. All parents (40/40) preferred to continue tiratricol treatment.
 
Patent application for “Processes of Preparation” of tiratricol
On September 19, 2024, we announced that we have submitted a patent application to the United States Patent and Trademark Office (USPTO) for “Processes of Preparation” of tiratricol. If granted, this would strengthen our patent portfolio. Generally, the exclusivity term of a new patent is 20 years from the date on which the application for the patent was filed in the United States.
In addition, we have Orphan Drug Designation (ODD) for tiratricol (Emcitate®) for MCT8 deficiency in the US and the EU, which provides marketing exclusivities of 7 and 10 years, respectively, from the dates of regulatory approvals.
 
Cash
We report cash of approximately SEK 130 million as of September 30, 2024. Post period we have received an additional SEK 282 million from the Directed Share Issuances announced on September 30, 2024, after deducting transaction costs. Currently, the Company has an ongoing dialogue with BlackRock regarding the conditions and a prolongation of the Tranche B (EUR 15 million) draw down window.
 
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