Interim report January-March 2023
April 26, 2023
Emcitate® project progressing with focus on applications for market approvals in the EU and USA in 2023
Financial overview January-March
· Quarterly net revenue MSEK 6,8 (7,1)
· Quarterly loss MSEK -74,9 (-28,8)
· Cash flow for the period MSEK 115,8 (-37,8)
· Cash balances at the end of the quarter amounted to MSEK 243,5 (106,8)
· Loss per share before/after dilution SEK -0.3 (-0.2)
Significant events during the period
· Received MSEK 196 (net) through an oversubscribed directed share issue
· In light of existing rumors in the market the Board of Directors of Egetis made the following statement: Egetis has ongoing discussions with certain external parties regarding a potential acquisition of the company. There can be no certainty that a public take-over offer will be made, nor as to the terms of any such offer
· Recruited Katayoun Welin-Berger as Vice President Operations, responsible for designing and managing the supply chains for development candidates and commercial products. Katayoun joined from Calliditas Therapeutics in March
· Continued to strengthen the commercial organization with two general managers (GM): In January Raymond Francot joined as GM for Germany, Austria, Switzerland, and Central and Eastern European Cluster, and Sylvain Forget joined as GM for France and Southern European Cluster (Portugal, Spain, Italy, and Greece)
· Received approvals for the pivotal ReTRIACt study from the Institutional Review Boards at Children’s Hospital of Philadelphia, USA, and Erasmus Medical Center in Rotterdam, The Netherlands
Significant events after the period
· Based on regulatory feedback on the documentation for the ReTRIACt study, certain clinical trial documentation has been updated, which necessitates renewed approvals from the ethics committees at the three participating hospitals, and if necessary, regulatory authorities. This work has started and is expected to result in the first patient being treated in the second quarter. In parallel, we have commenced the work to identify possible study participants, and so far we have identified >30 patients at the various trial sites. Therefore, our overall assessment is that this administrative delay of the start of the study will not significantly affect the timing of the submission of a marketing application in the USA
· Egetis plans to submit an NDA in the USA in the fourth quarter of 2023 under the ’Fast Track Designation’ granted by the FDA. A marketing application in Europe is expected, as previously communicated, to be submitted during Q2 2023 and is not dependent on the ReTRIACt study
Comments from the CEO
The ongoing quarter is very exciting and transformative for Egetis. We are approaching our first application for market approval in Europe for our lead drug candidate Emcitate.
I am also very pleased with our Directed Share Issue in the beginning of 2023. The Directed Issue was oversubscribed and Egetis received SEK 196 million after 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.
The Emcitate project is progressing with focus on applications for market approvals in the US and EU in 2023
We work intensively 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.
Based on regulatory feedback on the documentation for the ReTRIACt study, certain clinical trial documentation has been updated, which necessitates renewed approvals from the ethics committees at the three participating hospitals, and if necessary, regulatory authorities. This work has started and is expected to result in the first patient being treated in the second quarter. In parallel, we have commenced the work to identify possible study participants, and so far we have identified >30 patients at the various trial sites. Therefore, our overall assessment is that this administrative delay of the start of the study will not significantly affect the timing of the submission of a marketing application in the USA. Egetis plans to submit an NDA in the USA in the fourth quarter of 2023 under the ’Fast Track Designation’ granted by the FDA. The design of the study is available on clinicaltrials.gov under the code NCT05579327.
As previously communicated, a marketing application in Europe is expected to be submitted during Q2 2023 and is not dependent on the ReTRIACt study.
Implementation ongoing for the 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, around 180 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, which is now being implemented. Our Expanded Access Program for Emcitate reduces the administrative burden for treating physicians in the US, should they wish to prescribe Emcitate to MCT8 patients under their care, until the product gains market approval. This program will also be important for those patients finishing the ReTRIACt trial, enabling them to continue Emcitate treatment after the trial has ended.
Egetis continues the step-wise build-up of an organization in the US and Europe for the commercialization of Emcitate in 2024
The commercial organization was strengthened during the first quarter with three General Managers (GMs). In January Raymond Francot joined as GM for Germany, Austria, Switzerland and Central and Eastern European Cluster, and Sylvain Forget joined as GM for France and Southern European Cluster (Portugal, Spain, Italy and Greece). In April Nigel Nicholls joined as GM for the UK and Northern European Cluster (Ireland, Nordics and Baltics).
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 first quarter of 2023 Egetis participated at three international scientific and medical conferences. There is great interest among pediatric neurologists and pediatric endocrinologists to learn more about MCT8 deficiency, and 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).
The Triac Trial II study with Emcitate
Triac Trial II is an ongoing international, open-label, multicentre study that 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.
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.
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.
During the period we raised net proceeds of SEK 196 million, after issuance costs, in a directed share issue. We reported a cash position of approximately SEK 244 million as of March 31, 2023.
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 transformative year for the Company.
Nicklas Westerholm, CEO