Interim report January-September 2022
November 8, 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.
· 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
· 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 found here)
· 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
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.
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.
We reported a cash position of approximately SEK 190 million as of September 30, 2022.
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