Interim report January-September 2022

Egetis continues the build-up of an organization in the US and Europe for the commercialization of Emcitate® in 2024

Financial overview July-September

  • Quarterly revenues MSEK 5.1 (6.2)
  • Quarterly loss MSEK -53.9(-18.8)
  • Cash balances at the end of the quarter amounted to MSEK 190.1 (173.2)
  • Cash flow for the period MSEK -43.2(-34.5)
  • Loss per share before/after dilution SEK -0.3(-0.1)

Financial overview January-September

  • Revenues for the period MSEK 16.9 (35.0)
  • Loss for the period MSEK -115.9(-72.5)
  • Cash balances at the end of the period amounted to MSEK 190.1 (173.2)
  • Cash flow for the period MSEK 43.3 (-115.4)
  • Loss per share before/after dilution SEK -0.6(-0.4)

Significant events during the period July- September

  • Recruited Sara Melton as President of Egetis North America
  • Established a wholly-owned subsidiary in the United States, Egetis Therapeutics US Inc.

Emcitate

  • Egetis participated at the Society for the Study of Inborn Errors of Metabolism Annual Symposium, Annual Meeting of the European Thyroid Association and Annual Meeting of the European Society of Paediatric Endocrinology

Aladote

  • Received Orphan Drug Designation in the EU for Aladote for the prevention of acute liver failure

Significant events after the reporting period

  • Hosted a Capital Markets Day on October 13 in Stockholm, where an overview of the Company's strategy and project portfolio was presented (a video from the day can be foundhere)

Emcitate

  • Announced the design of a randomized, placebo- controlled study in 16 patients, to verify results of T3 levels from previous clinical studies and publications for a New Drug Application in the US. First patient in the study is expected in Q4 2022
  • Egetis participated at the International Child Neurology Congress and Annual Meeting of the American Thyroid Association
  • FDA has requested that Egetis applies for an
    'Expanded Access Program', to increase the availability of Emcitate for patients with MCT8 deficiency

Financial overview

2022

2021**

2022

2021**

2021

Jul-Sep

Jul-Sep

Jan-Sep

Jan-Sep

Jan-Dec

Net revenues, MSEK

5.1

6.2

16.9

35.0

38.2

Result after tax, MSEK

-53.9

-18.8

-115.9

-72.5

-104.5

Cash flow, MSEK

-43.2

-34.5

43.3

-115.4

-145.0

Cash, MSEK

190.1

173.2

190.1

173.2

144.0

Equity ratio %

94%

93%

94%

93%

93%

Earnings per share, SEK*

-0.3

-0.1

-0.6

-0.4

-0.6

Earnings per share after dilution, SEK*

-0.3

-0.1

-0.6

-0.4

-0.6

Average number of employees

14

11

14

11

11

*) The comparative figures in the table have been adjusted for the share issue in May 2022. **) Restated, see note 4.

Egetis Therapeutics interim report January-September 2022 1

Comments from the CEO

The third quarter of this year has been characterized by the continued stepwise build-up of Egetis' commercial infrastructure in preparation for an expected approval of Emcitate in the US and Europe in 2024.

The Emcitate project is progressing according to plan for the application for market approval in the US and Europe in 2023

Egetis intends to submit a marketing authorization application for Emcitate to the EMA in the first half of 2023, based on existing clinical data, after the required stability data has been obtained for the commercial product of Emcitate.

As previously communicated, Egetis will conduct a confirmatory randomized placebo-controlled trial in 16 patients to verify the results of previous clinical trials and publications regarding thyroid hormone T3 levels. The company has agreed the protocol for this study with the FDA, and the study is expected to start in the fourth quarter of 2022. The design of the study (ReTRIACt) is now available on clinicaltrials.gov under the code NCT05579327. Egetis intends to apply for market approval for Emcitate in the US in mid-2023, under the 'Fast Track Designation' granted by the FDA.

The Triac Trial II study with Emcitate

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 and are planned to be submitted to regulatory authorities after market approvals have been obtained. The design of the Triac Trial II study is available on clinicaltrials.gov under the code NCT02396459.

FDA has requested an 'Expanded Access Program' for Emcitate

There is continued great interest from physicians all over the world to treat patients suffering from MCT8- deficiency with Emcitate, which is prescribed on an individual license to patients in over 25 countries. In total, more than 160 patients are 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.

FDA has requested Egetis to apply for a so-called 'Expanded Access Program' in the USA. The company welcomes this request as it would ease the workload for both physicians and the FDA, thereby increasing the availability of Emcitate for MCT8-deficiency patients, before the product receives market approval.

Egetis continues the build-up of an organization in the US and Europe for the commercialization of Emcitate in 2024

The US is a key market for patients suffering from MCT8 deficiency. In June we announced that Sara Melton has been recruited as President of Egetis in North America. Sara is part of the Company's leadership team and has over 20 years of commercial leadership experience in biotechnology, pharmaceutical and medical technology companies, including rare diseases. She will be responsible for establishing and running a successful organization for Egetis and the launch of its products in the US and Canada.

After the period, we have recruited two key roles in the US. John Walsh, MD, has joined as VP Medical Affairs, North America, and Kate Sulham has joined as VP Pricing and Market Access, North America. John has previously worked at e.g. Biogen and EMD Merck- Serono. Kate has experience from e.g. The Medicines Company and Boston Healthcare Associates.

We have also established a wholly owned subsidiary in the United States, Egetis Therapeutics US Inc., incorporated in the state of Delaware.

Egetis continues to raise awareness of MCT8 deficiency among medical specialists and other key people in the healthcare sector

During the period, Egetis participated with exhibition stands at the Society for the Study of Inborn Errors of Metabolism Annual Symposium, the Annual Meeting of the European Thyroid Association and the European Society of Pediatric Endocrinology, and after the period at the International Child Neurology Congress and the American Thyroid Association. There is great interest among pediatric neurologists and pediatric endocrinologists in MCT8 deficiency, but awareness of the disease is still limited.

Egetis Therapeutics interim report January-September 2022 2

The pivotal study Albatross for Aladote in the US, EU and UK

The European Commission granted in August orphan drug designation for Aladote for the prevention of acute liver failure. Orphan drug designation in the EU follows the already granted orphan drug designation for Aladote for the treatment of paracetamol overdose obtained by the FDA in 2019.

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 Albatross, with the aim of applying for market approval in the USA, EU and the UK has been completed and the start of the study is planned early in 2023.

Cash position

We reported a cash position of approximately SEK 190 million as of September 30, 2022.

Looking ahead

Egetis is an innovative and integrated pharmaceutical company, focused on projects in late clinical development phase for commercialization within the orphan drug area for the treatment of serious and rare diseases with significant medical needs. We continue to be focused on developing our drug candidates Emcitate and Aladote for all the patients who have a great need for these preparations. It was a pleasure to notice the great interest in our Capital Markets Day in October. In total, around 300 people either viewed it live or streamed it afterwards. A recording is available on our homepage and via this link. I look forward to informing you about the future development of Egetis.

Nicklas Westerholm, CEO

Egetis Therapeutics interim report January-September 2022 3

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 fruitful regulatory interaction Egetis intends to submit a marketing authorisation application (MAA) for Emcitate to the European Medicines Agency (EMA) in the first half of 2023 based on existing clinical data.

In the US, after discussions with the FDA, Egetis will conduct a small randomized, placebo-controlled 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-2023 under the Fast-Track Designation granted by FDA.

Emcitate is currently being investigated in the Triac Trial II, a Phase II/III study in very young MCT8 deficiency patients (<30 months of age) exploring potential disease modifying effects of early

intervention from a neurocognitive and neurodevelopmental perspective. The recruitment target was achieved in the second quarter 2022 and 22 patients have been included in the study. Results are expected in mid 2024 and are expected to be submitted post-approval to regulatory authorities shortly thereafter.

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.

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 for early 2023. 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.

Pipeline overview

Egetis Therapeutics interim report January-September 2022 4

Project updates

Emcitate

Events during the quarter

  • Egetis participated at the Society for the Study of Inborn Errors of Metabolism Annual Symposium, Annual Meeting of the European Thyroid Association and Annual Meeting of the European Society of Paediatric Endocrinology

Events after the reporting period

  • Announced the design of a randomized, placebo controlled study in 16 patients, to verify results of

About Emcitate

Emcitate is Egetis' lead drug candidate in clinical development. It addresses monocarboxylate transporter 8 (MCT8) deficiency, also known as Allan- Herndon-Dudley Syndrome (AHDS), a rare genetic disease that affects 1 in 70,000 men with high unmet medical need with no available treatment.

Thyroid hormones are crucial for the development and metabolic state of virtually all tissues. Thyroid hormone transport across the plasma membrane is required for the hormones' metabolism and intracellular action and is facilitated by thyroid hormone transporters, including MCT8. Mutations in the gene for MCT8 cause MCT8 deficiency. The gene is located on the X chromosome and therefore mainly affects men, as men only have one X chromosome.

The resulting dysfunction of MCT8 leads to impaired transport of thyroid hormone into certain cells and across the blood-brain-barrier and disruption of normal thyroid hormone regulation. Patients with MCT8 deficiency therefore have low concentrations of thyroid hormone in the central nervous system, which signals that the body should produce more thyroid hormone. This leads to increased levels of thyroid hormone in peripheral tissues, also called thyrotoxicosis. This leads to a complex pattern of symptoms with neurological developmental delay and intellectual disability, accompanied by severely elevated circulating thyroid hormone concentrations which are toxic for tissues including the heart, muscle, liver and kidney and results in symptoms such as failure to thrive, cardiovascular stress, insomnia and muscle wasting.

T3 levels from previous clinical studies and publications for a New Drug Application in the US. First patient in the study is expected in Q4 2022

  • Egetis participated at the International Child Neurology Congress and Annual Meeting of the American Thyroid Association
  • FDA has requested that Egetis applies for an
    'Expanded Access Program', to increase the availability of Emcitate for patients with MCT8 deficiency

Most patients will never develop the ability to walk or even sit independently. At present there is no approved therapy available for the treatment of MCT8 deficiency.

Emcitate was granted Orphan Drug Designation in the EU in 2017 and the US in 2019. Emcitate received US Rare Paediatric Disease Designation (RPDD) in 2020. Upon approval of the NDA, sponsors holding a RPDD and meeting the criteria specified can apply to receive a Priority Review Voucher (PRV). A PRV provides accelerated FDA review of a subsequent new drug application for any drug candidate, in any indication, shortening time to market in the US. The voucher may also be sold or transferred to another sponsor. In 2022, four PRVs have been sold so far: one for $100 million and three for $110 million.

A Phase IIb clinical trial (Triac Trial I) in MCT8 deficiency has been completed which showed significant and clinically relevant treatment effects on key aspects of the disease. In October 2021, strong data from long-term treatment in patients with MCT8 deficiency up to 6 years, with Emcitate was published in the Journal of Clinical Endocrinology & Metabolism. The data comes from an investigator-initiatedreal-life cohort study at 33 sites conducted by the Erasmus Medical Center, Rotterdam, The Netherlands, where the efficacy and safety of Emcitate was investigated in 67 patients with MCT8 deficiency.

Based on the new long-term data, Egetis had further positive interactions with the regulatory agencies in the US and Europe. In December 2021, the EMA concluded that the clinical data from the Triac Trial I, together with the published data from long-term

Egetis Therapeutics interim report January-September 2022 5

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Egetis Therapeutics AB (publ) published this content on 08 November 2022 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 08 November 2022 07:13:08 UTC.