Emcitate® project progressing with focus on applications for market approvals in the US and EU in 2023

Financial overview October-December
·       Quarterly revenues MSEK 5,7 (3,2)
·       Quarterly loss MSEK -77,8 (-32,1)
·       Cash balances at the end of the quarter amounted to MSEK 127,7 (144,0)
·       Cash flow for the period MSEK -62,8 (-29,6)
·       Loss per share before/after dilution SEK -0.4 (-0.2)

Financial overview January-December
·       Revenues for the period MSEK 22,6 (38,2)
·       Loss for the period MSEK -193,8 (-104,5)
·       Cash balances at the end of the period amounted to MSEK 127,7 (144,0)
·       Cash flow for the period MSEK -19,5 (-145,0)
·       Loss per share before/after dilution SEK -1.0 (-0.6)

Significant events during the period October-December
·       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)
·       Continued to strengthen the commercial and medical affairs organizations for the expected launch of Emcitate in 2024
·       Recruited Anny Bedard as President of Egetis North America

Emcitate
·       Announced detailed design for small, randomized, placebo-controlled trial for Emcitate for US NDA submission
·       Submitted an Expanded Access Program for Emcitate in the USA

Significant events after the reporting period
·       Received MSEK 210 (gross) through an oversubscribed directed share issue
·       Recruited Katayoun Welin-Berger as Vice President Operations, responsible for designing and managing the supply chains for development candidates and commercial products. Katayoun will join Egetis from Calliditas Therapeutics in March 2023

Comments from the CEO
I am very pleased with our progress during the last quarter of 2022, which enabled our directed share issue in the beginning of 2023. The Directed Issue was oversubscribed and Egetis received SEK 210 million before transaction costs. New investors comprise institutional international and Swedish sector specialist investors, including AXA Investment Managers, Handelsbanken Fonder AB through the investment fund Hälsovård Tema, and Medical Strategy GmbH, as well as existing investors including The Fourth Swedish National Pension Fund (AP4), Linc AB and Unionen. The net proceeds from the Directed Issue will primarily finance continued build-up of the Company’s commercial infrastructure in Europe and the US and pre-launch activities for the planned commercialization of Emcitate in 2024, as well as general corporate purposes and financial flexibility.
 
Successful Capital Markets Day
On October 13, 2022, we hosted a Capital Markets Day in Stockholm, and presented an overview of the Company’s strategy and project portfolio. The event was very successful, with positive feedback from analysts, investors and other stakeholders. In total, around 350 people either viewed it live or have streamed it afterwards.  A recording is available on the Company’s homepage and via this link.
 
The Emcitate project is progressing with focus on applications for market approvals in the US and EU in 2023
Egetis intends to submit a marketing authorization application for Emcitate to the EMA in the second quarter of 2023, based on existing clinical data, after sufficient 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 first patient in the study is expected to be enrolled in the coming weeks. The delay from the anticipated start by end of 2022 is due to Covid-related administrative backlog at the American hospital participating in the study. The design of the study, which is called ReTRIACt, is available on clinicaltrials.gov under the code NCT05579327.
Egetis intends to apply for market approval for Emcitate in the US in the second half of 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. This study 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. 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.
 
Egetis has submitted an 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 170 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. By implementing our Expanded Access Program for Emcitate the administrative burden for treating physicians in the US will be reduced, should they wish to prescribe Emcitate to MCT8 patients under their care.
 
Egetis continues the step-wise build-up of an organization in the US and Europe for the commercialization of Emcitate in 2024
In December we announced that Anny Bedard has been recruited as President of Egetis in North America and as a member of the Company’s leadership team. Anny has over 25 years of international experience in both established and entrepreneurial bio-pharmaceutical companies and 15 years of experience with rare diseases. Ms. Bedard will be responsible for establishing and maintaining a successful presence for Egetis and its products in the United States and Canada.
In November, we announced the recruitments of three General Managers (GMs) in Europe. Nigel Nicholls will take on the position as GM for the UK and Northern European Cluster (Ireland, Nordics and Baltics). He will join Egetis in April 2023 and be based in the UK. Raymond Francot has taken on the position as GM for Germany, Austria, Switzerland and Central and Eastern European Cluster. He joined Egetis in January 2023 and is based in Zurich, Switzerland. Sylvain Forget has taken on the position as GM for France and Southern European Cluster (Portugal, Spain, Italy and Greece). He joined Egetis in January 2023 and is based in Paris, France.
These key recruitments bring substantial experience and proven track records in successful launch preparations and commercialization of drugs in ultra-orphan diseases, including products such as Spinraza®, Brineura® and Tecfidera®, as well as experience from several successful rare-disease companies, such as BioMarin, Global Blood Therapeutics, SOBI and Vertex.
 
Egetis continues to raise awareness of MCT8 deficiency among medical specialists and other key stakeholders
During the second half of 2022 Egetis participated with exhibition booths at seven international scientific and medical conferences. There is great interest among pediatric neurologists and pediatric endocrinologists to learn more about MCT8 deficiency, but general awareness of the disease is still limited. To increase awareness of the disease a new patient video has been created, which can be accessed here (www.mct8deficiency.com).
 
Developing a compelling clinical and economic value proposition
During the period, we have continued to assess the impact of MCT8 deficiency on patients and caregivers. To this end we have completed a so called Vignette study, involving treating physicians to derive utility values for a defined range of MCT8 deficiency health states, suitable for cost-effectiveness analysis. In addition, we have carried out a Caregiver study, to generate burden of disease data (costs and Quality of Life) from caregivers to support our value proposition and ensuring broad access.
 
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 planned during 2023.
 
Cash position
We reported a cash position of approximately SEK 128 million as of December 31, 2022. Post period we raised net proceeds of SEK 196 million, after issuance costs, in a directed share issue.

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. With the recently strengthened balance sheet we can now continue to be focused on developing our drug candidates Emcitate and Aladote for all the patients who have a great need for these therapies I look forward to informing you about the future development of Egetis during this upcoming transformative year for the Company.
 
Nicklas Westerholm, CEO