Interim report January-March 2022
April 26, 2022
Significant progress towards Emcitate® marketing applications in the US and Europe in 2023
Financial overview January-March
· Quarterly net revenue MSEK 7.1 (3.8)
· Quarterly loss MSEK -28.8 (-19.3)
· Cash and cash equivalents MSEK 106.8 (249.8)
· Cash flow for the period MSEK -37.8 (-38.4)
· Loss per share before/after dilution SEK -0.2 (-0.1)
Significant events during the period January–March
· Fruitful regulatory interactions clarify the regulatory path forward for Emcitate.
· Targeting Emcitate EU MAA submission the first half of 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 and publications.
· 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.
· Receives a conditional acceptance from the FDA for the use of the brand name Emcitate in the US.
· Receives orphan drug designation (ODD) for Emcitate for RTH-β in the US and a positive opinion from EMA on ODD in the EU.
· Receives a ‘Notice of Intent to Grant’ for a new European patent for a combination therapy with Aladote and N-acetylcysteine.
· Karl Hård joined the Company on February 1 as Head of Investor Relations and Communications, and member of the Company’s leadership team.
Significant events after the reporting period
· The recruitment target was achieved in the Triac Trial II study with Emcitate.
· A SEK 180 million fully guaranteed preferential rights issue was approved at an extraordinary general meeting on April 13, 2022.
· Receives ODD for Emcitate for RTH-β by the European Commission.
Comments from the CEO
Egetis has had a strong start to 2022. We have clarified the continued development program for our leading drug candidate Emcitate and plan to submit a marketing application in the EU in the first half of 2023 and in the US in mid-2023. Despite challenging times on capital markets, and in particular in the biotech sector, we announced in March a fully guaranteed preferential rights issue of approximately SEK 180 million. The purpose of the rights issue is to finance the preparations for regulatory submissions for market approval in the EU and the USA for Emcitate, to initiate the establishment of a commercial infrastructure in Europe and the USA and pre-launch activities. We have further strengthened the shareholder base with specialist investors through Linc AB.
Emcitate's development program clarified – application for market approval is expected in 2023
In January 2022, we announced that we intend to submit a New Drug Application (NDA) in the United States for Emcitate in mid-2023 under the 'Fast Track Designation' granted by the US Food and Drug Administration (FDA). After positive interactions, the FDA confirmed that a treatment effect on T3 levels and chronic thyrotoxicosis in MCT8 deficiency could form the basis for market approval in the United States. To supplement the existing clinical data in an upcoming NDA in the USA, we have agreed with the FDA to conduct a small, randomized, controlled trial in 16 patients for up to 30 days to verify the T3 results we have seen in previous clinical trials and publications. It is well established that T3 levels in untreated MCT8 patients are significantly elevated, and we have previously shown that Emcitate can quickly and durably normalize these levels.
In December 2021, following discussions with the European Medicines Agency (EMA), Egetis concluded that clinical data from the Triac Trial I study, together with data from long-term treatment, will be sufficient for a Marketing Authorisation Application (MAA) in the EU for the treatment of MCT8 deficiency. In February this year, we announced that after completing all parts of the regulatory dossier, we plan to submit an MAA in the first half of 2023. As the requirements for necessary clinical data have already been met, the remaining risk of Emcitate is reduced considerably.
The result of these regulatory interactions is a major step towards the application for market approval in the EU and the US, which would make Emcitate the first approved treatment for patients suffering from MCT8 deficiency and increase the likelihood of success for Emcitate, whereby Egetis can also receive a Priority Review Voucher (PRV) in the United States.
The recruitment target has been met in the Triac Trial II study with Emcitate
After the end of the period, in early April, we announced that the recruitment target of 16 patients has been met in the Triac Trial II study with Emcitate in patients with MCT8 deficiency. Triac Trial II is an ongoing study conducted in Europe and North America examining the neurocognitive effects of early intervention with Emcitate in very young patients (<30 months old). To allow for the inclusion of patients who have already been identified but have not yet completed the screening procedure, the study will be open until April/May 2022. Results from Triac Trial II are expected in the first quarter of 2024 and are scheduled to be submitted to regulatory authorities after market approval.
Global interest in Emcitate
There is a continuing strong interest from physicians around the world in treating patients suffering from MCT8 deficiency with Emcitate, which is prescribed on a named patient basis in over 25 countries. In total, over 150 patients are now being treated with Emcitate, and we are seeing more and more patients gain access to treatment. This shows how important the treatment is for these patients who have a great medical need.
Initiatives to raise disease awareness of MCT8 deficiency
In late 2021, we launched initiatives to raise disease awareness of MCT8 deficiency, including the global 'Cuddly Toy' campaign to raise awareness among healthcare professionals and support diagnosis. This online campaign features a series of stuffed animals with floppy heads, synonymous with the inability of affected boys to hold their heads up. In connection with 'Rare Disease Day' at the end of February 2022, we started another campaign, called #MCT8Hugs, which is now available on major social media platforms. We have also started a so-called Vignette study among physicians, to measure health-related quality of life among MCT8 patients, and a Caregiver study. The results from the studies will be used to increase awareness of MCT8 deficiency and be part of the work to get national stakeholders to prioritize funding for MCT8 deficiency treatment.
Orphan drug designation to Emcitate for RTH-β
In February, the US FDA granted orphan drug designation to Emcitate for the treatment of resistance to thyroid hormone type beta (RTH-β), and in March the EMA issued a positive opinion on orphan drug designation for RTH-β, which has been adopted by the European Commission in April. RTH-β is a further indication, without overlap in patient populations, to the previously obtained ODD for MCT8 deficiency. The ODD for RTH-β is a direct result of our work to extend the indications for the Emcitate program to related but distinct conditions. RTH-β is a rare genetic disease caused by mutations in one of the body's two types of thyroid hormone receptors and leads to decreased thyroid hormone signaling in tissues that depend on thyroid hormone receptor beta. The disease affects 1 in 20,000–40,000 individuals. We will continue to evaluate the development of Emcitate towards market approval also for this disease, which could increase the value and extend the market exclusivity for Emcitate.
Brand name Emcitate accepted
In January, we received conditional acceptance from the FDA for the use of the Emcitate brand name in the United States. The Company has previously received a corresponding acceptance from the EMA for the use of Emcitate in the EU. This is the best possible result for securing a global brand name, and the final approval process for the use of the Emcitate brand will be linked to the regulatory application in each market.
Preparations for the pivotal Phase IIb/III-study with Aladote are ongoing
We remain focused on the continued development of Aladote, which has the potential to become the first approved drug for patients at increased risk of liver injury after overdosing on paracetamol and for whom standard N-acetylcysteine (NAC) treatment is not effective. Preparations for the planned Phase IIb/III study with Aladote are ongoing. The COVID-19 pandemic has made it difficult to start a clinical study that is carried out in emergency and intensive care clinics, but the situation is now developing for the better and we expect the study to start later in 2022.
In January, the European Patent Office (EPO) issued a so-called ‘Notice of Intent to Grant’ for a new patent that includes a combination treatment with Aladote and NAC. The new patent further enhances the unique position of the Aladote program and provides patent protection until the year 2037 in Europe, before a potential extension.
Cash position and fully guaranteed preferential rights issue
We reported a cash position of approximately SEK 107 million as of March 31, 2022. On March 21, the Board decided on a new issue of shares corresponding to approximately SEK 180 million with preferential rights for the Company's existing shareholders. The fully guaranteed rights issue was approved at an Extraordinary General Meeting on April 13, 2022. The primary purpose of the rights issue is to finance preparations for the EU and US market approval application process, initiate the establishment of a European and US commercial infrastructure for Emcitate and launch preparation activities.
I am grateful for continued support from our current shareholders, especially the specialized life science investor Flerie Invest AB for their commitment to increase their shareholding. In addition, we further strengthen the shareholder base with specialist investors through Linc AB, and I welcome them as a new shareholder.
Egetis 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. We are fully focused on developing our drug candidates Emcitate and Aladote in 2022 for all those patients who have a great need for these drugs. In 2022, we will also initiate the establishment of a commercial infrastructure in Europe and the United States. I look forward to informing you about the future progress at Egetis.
Nicklas Westerholm, CEO