Egetis Therapeutics AB: Year-End report January-December 2023

Egetis achieved several significant milestones during the fourth quarter of 2023

• Submitted marketing authorisation application for Emcitate® (tiratricol) for the treatment of MCT8 deficiency to the European Medicines Agency EMA
• Secured MSEK 462 in combined financing, consisting of a private placement of MSEK 172, at a premium, and a debt financing of MSEK 290
• Entered into an exclusive licensing agreement with Fujimoto to develop and commercialize Emcitate® in Japan
• The ReTRIACt study is progressing with 10 patients included

Financial overview October-December

• Quarterly revenue MSEK 32.6 (5.7), of which MSEK 14.5 (-) was a milestone payment for a licensing agreement in Japan
• Quarterly loss MSEK -86.3 (-77.8)
• Cash at the end of the quarter amounted to MSEK 303.3 (127.7)
• Cash flow for the quarter was MSEK 223.6 (-62.8)
• Earnings per share before/after dilution SEK -0.3 (-0.4)

Financial overview January-December

• Revenue for the period MSEK 57.6 (22.6), of which MSEK 14.5 (-) was a milestone payment for a licensing agreement in Japan
• Net loss for the period MSEK -326.9 (-193.8)
• Cash at the end of the period amounted to MSEK 303.3 (127.7)
• Cash flow for the period MSEK 180.4 (-19.5)
• Earnings per share before/after dilution SEK -1.3 (-1.0)

Significant events during the quarter

• Secured approximately MSEK 462 in a combined financing comprising a MSEK 172 equity private placement, at a premium, and MSEK 290 debt financing (MEUR 25)
• Delivered a drawdown notice to BlackRock for the drawdown of Tranche A (MEUR 10) of the Debt Financing
• Recruited Desiree Luthman as Vice President Global Regulatory Affairs and Laetitia Szaller as Legal Counsel and Head of Compliance
• Organized an Investor Day on December 19, 2023 (a link to the Investor Day can be found here)

Emcitate® (tiratricol)

• Submitted marketing authorisation application (MAA) for Emcitate for the treatment of MCT8 deficiency to the EMA, which was subsequently verified on October 26
• Entered into an exclusive licensing agreement with Fujimoto to develop and commercialize Emcitate in Japan
• Provided updates on the Expanded Access Program for Emcitate in the USA

Significant events after the quarter

• In total, 10 (out of a target of 16 evaluable) patients have been included for the ReTRIACt study, which is pivotal for the US submission

Comments from the CEO

2023 was a year of strong progress for Egetis and to make our lead investigational drug Emcitate (tiratricol) available to patients in need. While the first half of the year saw an unsolicited approach from external parties regarding a potential acquisition of the Company, the second half of 2023 provided several important milestones. In July, the first patients were included in the ReTRIACt study, which is pivotal in the USA. In October, the Company submitted a marketing authorisation application (MAA) for Emcitate for the treatment of MCT8 deficiency to the European Medicines Agency (EMA), and shortly thereafter, Egetis secured approximately 462 million SEK in combined financing, consisting of a directed new share issue of 172 million SEK (gross), at a premium to the closing price of the day, and a debt financing of approximately 290 million SEK (MEUR 25). Our largest shareholder is now specialist investor Frazier Life Sciences from California, USA, which has a track record of partnerships with scientifically driven companies in the healthcare sector.
In November, the Company entered into an exclusive licensing agreement with Fujimoto to develop and commercialize Emcitate in Japan.

Egetis submitted marketing authorisation application for Emcitate for the treatment of MCT8 deficiency to the EMA
On October 9, we submitted our MAA for Emcitate for the treatment of MCT8 deficiency to the EMA. On October 27, we announced that the MAA had been validated and is under formal review by the Committee for Medicinal Products for Human Use (CHMP) at the EMA. The average duration of the review process for MAAs at EMA is approximately 13-14 months.

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 confirmatory, 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, at Erasmus Medical Center in Rotterdam, The Netherlands, and Children's Hospital of Philadelphia, USA. Recently, two additional hospitals have been included in the study. Addenbrooke's Hospital in Cambridge, UK, began recruiting patients in mid-December, and Saint Louis University Hospital in St. Louis, Missouri, USA, started recruiting patients in January 2024. As of February 22, 2024, a total of 10 patients had been included in the study. As previously communicated, the Company will update the market once recruitment is completed, and at that time provide information on when to expect topline results and subsequently when the NDA is expected to be submitted.
More information about the ReTRIACt study is available on clinicaltrials.gov under the code NCT05579327.

Egetis entered into an exclusive licensing agreement with Fujimoto to develop and commercialize Emcitate in Japan
Egetis entered into an exclusive licensing agreement with Fujimoto Pharmaceutical Corporation in November to develop and commercialize Emcitate in Japan. According to the terms of the agreement, Fujimoto has been granted exclusive rights for the development and commercialization of Emcitate for the treatment of MCT8 deficiency in Japan. Fujimoto will pay upfront, development, and regulatory milestones totaling JPY 600 million (approximately SEK 45 million). Egetis will supply the product in semi-finished form and receive approximately one-third of the applicable income from Fujimoto. Fujimoto will also finance the development program required for Emcitate in Japan, which will be clarified after discussions with the Pharmaceuticals and Medical Devices Agency (PMDA). As the future holder of the marketing authorization in Japan, Fujimoto will be responsible for regulatory interactions with PMDA.

Egetis continued to increase awareness of MCT8 deficiency
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. Egetis' medical affairs activities are focused on improving awareness of the disease and its diagnosis, by participation and dialogues at scientific conferences, creating patient identification partnerships with genetic testing companies, engaging with Key Opinion Leaders, advisory committees, and interactions with patient groups. This has resulted in an additional 50 patients being identified with MCT8 deficiency in the USA who were previously undiagnosed. More information about MCT8 deficiency is available at www.mct8deficiency.com.

Emcitate 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, 12 hospitals have requested participation in this program. The EAP program for Emcitate 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 who complete the ReTRIACt study so that they can continue treatment with Emcitate after completing the study.
There is continued significant and growing interest from physicians worldwide in treating patients with MCT8 deficiency with Emcitate, which is already being prescribed as part of Managed Access Programs to patients in over 25 countries. Currently around 200 patients are being treated with Emcitate, and more patients are gaining access to treatment, a true validation of the significant medical need for these patients.

Triac Trial II with Emcitate
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 Emcitate, as well as the effect on clinical and biochemical aspects of thyrotoxicosis. Patients are initially treated with Emcitate for 96 weeks and then followed for an additional two years. 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 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. The start of the study has been postponed until Emcitate marketing authorization submissions for MCT8 deficiency have been completed.

Egetis continues to attract and recruit highly experienced colleagues
In November Desiree Luthman was recruited as Vice President Global Regulatory Affairs. Desiree has over 25 years of experience in the pharmaceutical industry in Global Regulatory Affairs and has successfully contributed to obtaining both FDA and EMA approvals for new drugs. Before joining Egetis, Desiree was Senior Vice President Regulatory Affairs at Passage Bio, a US gene therapy company. Previously, she held senior positions at Verona Pharma, Sanofi, BMS, Celgene, Xytis, and AstraZeneca.
In December Laetitia Szaller was recruited as General Counsel and Head of Compliance. Laetitia has over 17 years of experience in the pharmaceutical sector. She holds dual qualifications to practice law in both the UK and Belgium. Before joining Egetis, Laetitia was General Counsel and VP Business Development at AM-Pharma in the Netherlands and was a member of the company's management team. Prior to that, Laetitia worked at UCB, Abbott, and Zoetis.

An Investor Day was held on December 19, 2023
On December 19, 2023, Egetis hosted an investor day in Stockholm. The investor day included presentations by Dr. Andrew Bauer from Children's Hospital of Philadelphia on MCT8 deficiency and the unmet medical need, as well as by Dr. Carla Moran from University College Dublin on a separate indication called resistance to thyroid hormone beta (RTH-beta) and the unmet medical need in this disease. Members of Egetis' management team highlighted the significant progress made by Egetis towards potential market approvals for Emcitate, including a status update on the ReTRIACt study, as well as plans for preparatory activities and commercialization focusing on disease awareness, market access, and value proposition. Furthermore, the Company's strategic goals in the short and long term were presented. (Link to the Investor Day)

Cash
We reported cash of approximately SEK 303 million as of December 31, 2023. In addition, we have access to an additional 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 Emcitate.

Outlook
I look forward to 2024 to be a transformational year for Egetis. Our employees are focused on delivering five key milestones:

1. Complete the ReTRIACt study, which is pivotal in the USA, as soon as possible
2. Results from the Triac Trial II study in mid-2024
3. Thriving towards a potential positive opinion from EMA for Emcitate for MCT8 deficiency
4. Preparatory launch activities in Europe
5. Preparing the NDA for Emcitate in the USA

Nicklas Westerholm, CEO

For further information, please contact

Nicklas Westerholm, CEO
[e-mail]
[phone]

Yilmaz Mahshid, CFO
[e-mail]
[phone]

Karl Hård, Head of Investor Relations & Business Development
[e-mail]
[phone]

This information is information that Egetis Therapeutics is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact persons set out above, at 2024-02-22 07:00 CET.

About Egetis Therapeutics

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. 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 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 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