Significant progress towards Emcitate® marketing applications in the US and Europe in 2023

Financial overview October – December
· Quarterly net revenue MSEK 3.5 (5.3)
· Quarterly loss MSEK -32.1 (-74.3)
· Cash and cash equivalents MSEK 144.0 (287.9)
· Cash flow for the period MSEK -29.6 (129.8)
· Loss per share before/after dilution SEK -0.2 (-0.7)

Financial overview January – December
· Net revenue for the period MSEK 38.5 (40.7)
· Loss for the period MSEK -104.5 (-178.0)
· Cash and cash equivalents MSEK 144.0 (287.9)
· Cash flow for the period MSEK -145.0 (34.2)
· Loss per share before/after dilution SEK -0.6 (-2.6)

Significant events during the period October – December
· Receives FDA Fast Track Designation for Emcitate for MCT8 deficiency.
· New real-world data published confirms long-term efficacy and safety of Emcitate in MCT8 deficiency patients up to six years.
· Intends to submit a marketing authorisation application (MAA) for Emcitate in Europe based on existing clinical data. Having all clinical data required for regulatory submission already available significantly reduces the remaining risk for Emcitate.
· Preparation for the pivotal Phase IIb/III study for Aladote continues, targeting study start 2022, pending the COVID-19 situation.
· Gets Notice of Allowance for a new US patent for a combination therapy with Aladote and N-acetylcysteine.

Significant events after the reporting period
· Fruitful regulatory interactions clarify the regulatory path forward for Emcitate.
· Targeting Emcitate EU MAA submission H1 2023.
· Targeting Emcitate US NDA submission mid-2023 under the Fast Track Designation.
· FDA acknowledges that effects on T3 levels and the manifestations of chronic thyrotoxicosis could provide a basis for Emcitate approval.
· For the US submission, a 30-day, placebo-controlled study in 16 patients will be conducted to verify the results on T3 levels seen in previous clinical trials.
· The outcome from the regulatory interactions increases the likelihood of success for Emcitate and the probability to receive a Rare Pediatric Disease Priority Review Voucher (PRV) in the US.
· Gets Notice of Intent to Grant for a new European patent for a combination therapy with Aladote and N-acetylcysteine.
· Receives a conditional acceptance from the FDA for the use of the brand name Emcitate in the US.

Comments from the CEO

Last year was a transformative busy year for Egetis, in which we achieved important milestones in our exciting Emcitate program. I’m proud of our progress to become a nimble and highly experienced rare disease company, well positioned for the future.

Fast Track Designation granted for Emcitate
In early October, the U.S. Food and Drug Administration (FDA) granted Emcitate Fast Track Designation for the treatment of MCT8 deficiency. This designation is an acknowledgement from the FDA of the importance of Emcitate to address the significant unmet medical need for patients from this devastating disease. With a Fast Track Designation comes opportunities to expedite both the new drug application (NDA) submission and FDA’s review which could enable an earlier regulatory approval of Emcitate.

New data confirms long-term efficacy and safety of Emcitate in MCT8 deficiency patients
In October, an investigator-initiated real-life cohort study at 33 sites conducted by the Erasmus Medical Center, Rotterdam, The Netherlands reported long-term treatment effects in 67 subjects, up to 6 years, with Emcitate. The study was published in the Journal of Clinical Endocrinology & Metabolism.
This long-term data confirms the positive results from the previous Triac Trial I and verifies that the beneficial effects are maintained over time, up to six years. The consistent efficacy seen across several key clinical and biochemical parameters regardless of age, further supports the use of Emcitate in the treatment of MCT8 deficiency.  

Fruitful interactions with regulatory agencies de-risk the Emcitate program
Based on the new long-term data, we had further positive interactions with the regulatory agencies in the US and Europe. In December, the European Medicines Agency (EMA) concluded that the clinical data from the Triac Trial I, together with the published data from long-term treatment will suffice for a regulatory submission of a Marketing Authorisation Application (MAA) to the EMA for the treatment of MCT8 deficiency. We plan to submit the MAA in the first half of 2023. Having all clinical data required for regulatory submission in EU already at hand significantly reduces the remaining risk for Emcitate.
After the period, in January 2022, we announced our intention to submit the NDA in the US for Emcitate in mid-2023 under the Fast Track Designation granted by the FDA. This followed recent positive regulatory interactions, in which the FDA acknowledges that treatment effects on T3 levels and the decrease in chronic thyrotoxicosis in MCT8 deficiency could provide a basis for marketing approval in the US.
We have agreed with the FDA to perform a small, randomized study in 16 patients for up to 30 days to verify our T3 results, seen in previous clinical trials and publications. It is well-established that the T3 levels in untreated MCT8 patients are significantly elevated, and we have previously shown that Emcitate is able to rapidly and durably normalize these levels. The primary source of patients for this study will be through our existing named patient program.
The outcome of these regulatory interactions have created a major step towards marketing applications in EU and the US, making Emcitate available to patients who suffer from MCT8 deficiency, increasing the likelihood of success for Emcitate and the probability for Egetis to receive a US Rare Pediatric Disease Priority Review Voucher (PRV).

Triac Trial II
The ongoing Triac Trial II remains important to further establish the effects of early intervention on the neurocognitive development aspects of the disease, previously seen in young patients in the Triac Trial I. Patients continue to enter the study despite the challenging COVID-19 situation. Results from the Triac Trial II are expected in the first quarter of 2024 and is expected to be submitted post-approval to regulatory authorities shortly thereafter.

Emcitate supplied on compassionate use and named patient basis
Parallel to our clinical program with Emcitate, there is a continued interest from physicians across the globe to use Emcitate for patients that suffer from MCT8 deficiency.
More than 140 patients in more than 25 countries are being treated with Emcitate on a named patient or compassionate use basis demonstrating the significant unmet medical need in this patient population and verifies the interest to treat patients that suffer from MCT8 deficiency.

Launch of campaign to raise awareness of MCT8 deficiency
We are committed to help transforming and extending the lives of patients with rare diseases such as MCT8 deficiency. One important pillar is raising disease awareness, and in September we launched an awareness campaign, including the website www.mct8deficiency.com. In addition to other disease educational activities, e.g., at scientific and medical conferences targeting health care professionals, the website will be used for educational purposes through the expanding network of key opinion leaders, physicians and patient advocacy groups focused on MCT8 deficiency.
These activities aim to shorten the time to diagnosis of MCT8 deficiency and enable earlier treatment, to help relieve the heavy burden of disease that MCT8 deficiency places on the affected individuals, their families and the caregivers they are heavily dependent on.

Preparations for the Aladote pivotal Phase IIb/III study are ongoing
Preparations for the planned Phase IIb/III study with Aladote are ongoing. The COVID-19 pandemic is still making it challenging to start a clinical study in an emergency/intensive care setting. Therefore, pending how the situation evolves, we expect study start will likely take place later this year.
We remain committed to the continued development of Aladote, which has the potential to be the first approved drug to benefit patients with an increased risk of liver injury, who are not adequately treated with standard of care N-acetylcysteine (NAC) after a paracetamol overdose. Aladote has been granted ODD in the US and we have an ongoing dialogue with EMA on the appropriate indication for an ODD in the EU.
In December 2021 and January 2022, we received Notices of Intent to Grant for new patents in the US and Europe, respectively. These patents further strengthen our robust calmangafodipir patent portfolio that includes a composition-of-matter patent with protection until year 2032 in US and EU.

Organization
In 2021, we have significantly strengthened our organization with the hiring of Dr Kristina Sjöblom Nygren as new CMO, and of Dr Yilmaz Mahshid as new CFO. We plan more key recruitments in 2022 as we continue to grow and add competence to the Company.
In 2021, Dr Thomas Lönngren (Chairman) and Mats Blom joined our board of directors.

Cash position
We reported a cash position of approximately 144 million SEK on December 31, 2021.

COVID-19
We are still being affected by the ongoing COVID-19 pandemic. We continue to carefully monitor this development and take every precaution to ensure that patients, healthcare staff, our organization and those working on our trials are safe and well, and that our operations continue according to plan.

Looking ahead
Our pipeline focus to provide treatment for patients suffering from rare and serious diseases remains firm as we shape the future of Egetis.
We plan to commercialize Emcitate in the US and Europe ourselves and will stepwise initiate the establishment of a small and focused organization in 2022 and 2023, to ensure the successful commercialization. Given the ultra-rare disease setting and uniqueness of Emcitate we plan for a commercial team of less than 50 FTEs at the time of launch.
We believe our core expertise could provide a platform to potentially be leveraged for additional late-stage orphan drug projects.
I look forward to relaying news to you around the projects and the progress of Egetis during the year ahead.
Finally, I extend my sincere thanks to all employees at Egetis for their committed work during 2021, the board of directors for governance and helpful counsel, our shareholders for their continued support and to all patients and physicians participating in the development of our product candidates.

Nicklas Westerholm, CEO