Selskabsmeddelelse

Interim report Q2 2023

Egetis recruits first patients in the pivotal ReTRIACt trial for the US NDA for Emcitate

· Egetis on track to submit Emcitate marketing authorisation application in EU on the by EMA predetermined date of October 9, 2023

Financial overview April-June
·        Quarterly Revenue MSEK 5.9 (4.7)
·        Quarterly loss MSEK -79.5 (-33.2)
·        Cash balances at the end of the quarter amounted to MSEK 179.2 (233.2)
·        Cash flow for the quarter was MSEK -64.8 (124.3)
·        Earnings per share before/after dilution SEK -0.3 (‑0.2)

Financial overview January-June
·        Revenue for the period MSEK 12.7 (11.8)
·        Net loss for the period MSEK -154.4 (-62.1)
·        Cash balances at the end of the period amounted to MSEK 179.2 (233.2)
·        Cash flow for the period MSEK 51.0 (86.5)
·        Earnings per share before/after dilution SEK -0.6 (‑0.3)

Significant events during April-June
· Announced termination of discussions regarding a potential acquisition of the Company
· Board directors in Egetis acquired shares

Emcitate
Announced site activation in the pivotal ReTRIACt trial for Emcitate and updated timeline for the US NDA submission

Significant events after the period

Emcitate
· Announced first patient included and second site activated in the ReTRIACt trial, which is pivotal for the US NDA submission

Aladote
· Announced that in-house development of Aladote will be parked, until Emcitate submissions have been completed

Comments from the CEO
I am delighted that the first patients have been included and that we now have two active sites in the ReTRIACt trial for Emcitate, which is pivotal for the New Drug Application (NDA) in the USA. I am also pleased that we are nearing our first marketing application for Emcitate in the EU, which is based on existing clinical data. These are important and transformative milestones for the Company.

The Emcitate project is progressing with focus on applications for market approvals in the EU in 2023 and the US in 2024
We are on track to submit a marketing authorisation application (MAA) for Emcitate to the EMA on October 9, 2023, based on existing clinical data.
As previously communicated, for the US NDA Egetis is conducting a confirmatory pivotal randomized placebo-controlled trial in 16 patients to verify the results of previous clinical trials and publications regarding thyroid hormone T3 levels.
We currently have two active sites in this pivotal ReTRIACt study for the NDA in the USA for Emcitate and the first patients have been recruited. The Company expects topline results from the ReTRIACt study during the first half of 2024 and estimates subsequent NDA submission in the USA in mid-2024, under the fast-track designation. The updated timelines announced in June are due to the substantial delay in the study start, and an anticipated higher number of treatment naïve patients, which implies a longer trial duration per patient, expected to be recruited in the trial compared to the original assumptions. In addition, there is lower-than-expected recruitment capacities per month at the participating sites.
The Company is working diligently with the ReTRIACt study sites to facilitate a smooth and efficient execution of the trial and more than 30 eligible patients have been identified across the three participating sites.
The design of the study is available on clinicaltrials.gov under the code NCT05579327.

Egetis aligns the build-up of a commercial organization in the US with the updated NDA submission timelines for Emcitate
As a consequence of the delay with the expected submission of Emcitate in the US, the build-up of the commercial infrastructure in the US will be aligned with the updated NDA submission timelines and all resources will be focused on the Emcitate ReTRIACt study and the upcoming EU submission.

Implementation of the Expanded Access Program for Emcitate in the USA
There is continued large and increasing interest from physicians all over the world to treat patients suffering from MCT8-deficiency with Emcitate, which is already prescribed on an individual license to patients in over 25 countries. In total, over 180 patients are now being treated with Emcitate, and we see more and more patients gaining access to treatment. This underlines the great medical need for a treatment for these patients.
On the request of the FDA Egetis submitted in the fourth quarter of 2022 an ‘Expanded Access Program’ in the USA, which is now being implemented. Our Expanded Access Program for Emcitate reduces the administrative burden for treating physicians in the US, should they wish to prescribe Emcitate to MCT8 patients under their care, until the product gains market approval. This program is also important for those patients finishing the ReTRIACt trial, enabling them to continue Emcitate treatment after the trial has ended.

Egetis continues to raise awareness of MCT8 deficiency among medical specialists and other key stakeholders
During the second quarter of 2023 Egetis participated at six international scientific and medical conferences. There is great interest among pediatric neurologists and pediatric endocrinologists to learn more about MCT8 deficiency, and general awareness of the disease is still limited. More information about MCT8 deficiency can be found at www.mct8deficiency.com

The Triac Trial II study with Emcitate
Triac Trial II is an ongoing international, open-label, multicentre study that investigates the effect of treatment with Emcitate on neurocognitive endpoints in young boys (≤30 months) with MCT8 deficiency. Patients will initially be treated for 96 weeks with Emcitate, after which they will be followed for an additional two years.
The recruitment target for Triac Trial II was achieved in the second quarter of 2022 where 22 patients have been included. Results from the study are expected in mid-2024. The design of the Triac Trial II study is available on clinicaltrials.gov under the code NCT02396459.

The pivotal study Albatross for Aladote in the US, EU and UK
There is a significant medical need for the approximately 25% of patients who reach hospital more than eight hours after paracetamol overdose. These patients have an increased risk of acute liver failure and need additional treatment options beyond the currently available N-acetylcysteine (NAC).
The design of the pivotal Phase Iib/III study, which is called Albatross, has been agreed with the FDA, EMA and MHRA. The start of the study is now planned after completion of the Emcitate regulatory submissions in EU and the US.

Cash position
In January 2023 we raised net proceeds of SEK 196 million, after issuance costs, in a directed share issue. We reported a cash position of approximately SEK 179 million as of June 30, 2023.

Looking ahead
Egetis is an innovative and integrated pharmaceutical company, focused on projects in late clinical development phase for commercialization within the orphan drug segment for the treatment of serious and rare diseases with significant unmet medical needs.
Our efforts are now focussed on the efficient execution of the pivotal ReTRIACt study, needed for the US NDA, and on the submission of the marketing application in the EU. I look forward to informing you about the future development of Egetis during this transformative year for the Company.

Nicklas Westerholm, CEO

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 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. As a result of regulatory interaction Egetis intends to submit a marketing authorisation application (MAA) for Emcitate to the European Medicines Agency (EMA) on October 9, 2023, based on existing clinical data.
After a dialogue with the FDA, Egetis is conducting a small randomized, placebo-controlled pivotal study in 16 patients to verify the results on T3 levels seen in previous clinical trials and publications. Egetis intends to submit a new drug application (NDA) in the US for Emcitate in mid 2024 under the Fast-Track Designation granted by FDA.
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 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 and the design of the upcoming pivotal Phase Iib/III study with the purpose of applying for market approval in the US and Europe for Aladote has been finalized after completed interactions with FDA, EMA and MHRA and study start is planned after Emcitate submissions 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