Patient recruitment in the pivotal study with Emcitate progressing according to plan
April 22, 2021
January March • Quarterly net sales MSEK 3.8 (11.7) • Quarterly result MSEK -19.3 (-42.8) • Cash and cash equivalents MSEK 249.8 (221.1) • Cash flow for the period MSEK-38.4 (-37.2) • Loss per share before/after dilution SEK -0.1 (-0.8)
Comments from the CEO
The first three months of the new year have indeed given a good start to Egetis Therapeutics, our new focused orphan drug development company with two important assets Emcitate and Aladote – both in late-stage clinical development. During this intense period, we have integrated Rare Thyroid Therapeutics, strengthened and further adapted the organization to the new strategic direction and thereby built a solid foundation for Egetis’ future success as a sustainable orphan drug company dedicated to development and commercialization of therapies for rare diseases. Our goal is to offer medicines to patients with serious and rare diseases lacking adequate medical treatments and thereby create value for patients, shareholders and society.
Recruitment to the Phase IIb/III study TRIAC II with Emcitate is progressing well according to plan
Emcitate, which has been granted Orphan Drug Designation (ODD) in both EU and the US and received a US Rare Pediatric Disease designation (RPD) in November 2020, is being developed for the treatment of MCT8 deficiency, a rare congenital disorder of thyroid hormone trafficking with detrimental natural history and no currently available therapy. Approximately 1 in 70,000 males are affected.
The first patient in the pivotal Phase IIb/III early intervention trial TRIAC II was dosed in December 2020, and the recruitment is progressing well and is expected to be completed in Q4 2021 as per plan. Interim results are targeted to be available in H2 2022 and expected to pave the way for regulatory approvals and commercial launch. TRIAC II is an international, open label, multi-center study in children younger than 30 months with MCT8 deficiency, conducted in both Europe and North America.
We also see an increased interest from physicians across the globe to treat patients that suffer from MCT8 deficiency with Emcitate. Emcitate is supplied on a named patient basis, following individual regulatory approval from the national regulatory agency. Named patient access is a mechanism to allow for early access to important and life-saving medicines in situations with high unmet medical needs and where no available treatment alternatives exist or are suitable. Already more than 100 patients with MCT8 deficiency in several countries have been granted such named patient approval and are being treated with Emcitate, underlining the significant unmet medical need in this patient population.
Preparations for the Aladote pivotal Phase IIb/III study are ongoing
Preparations for the planned Phase IIb/III study with Aladote are ongoing in the US, UK and EU together with the selected CRO. The Covid-19 pandemic makes it very challenging to start a clinical study in an emergency/intensive care setting. Therefore, pending how the situation evolves, we expect study start will likely take place in H2 2021.
We remain committed to the continued development of Aladote, which has the potential to be the first approved drug to benefit patients with an increased risk of liver injury, who are not adequately treated with NAC after a paracetamol overdose. Aladote has been granted ODD in the US, and an application for an ODD in the EU was submitted to the EMA in March.
We continue to see a strong interest in the scientific community for Aladote, which was presented by Professor James Dear from the University of Edinburgh, UK at two scientific conferences in March and April as a novel emerging treatment of paracetamol overdose.
After discussion with our partner Solasia, Egetis Therapeutics has decided to park the development of PledOx following the POLAR results. Solasia will further evaluate PledOx through a pre-clinical program in taxane-induced peripheral neuropathy.
To continue the development of our clinical portfolio, we reported a cash position of approximately 250 million SEK on March 31, 2021, which is planned to finance the development of Emcitate and Aladote towards market approval.
Strengthened organization and board
We continue to strengthen the company, in order to adapt to our new strategic direction, prepare for the next steps of our clinical programs and ultimately launch of our innovative drug candidates. In February, Kristina Sjöblom Nygren, MD, was appointed Chief Medical Officer (CMO), effective May 1, and will be member of the company’s leadership team. Kristina has more than 20 years’ experience in the pharmaceutical industry from both large pharmaceutical companies and smaller biotech companies. She has an extensive experience in late-stage development and regulatory interactions in the rare disease and orphan drug segment in particular and will be instrumental in the execution of our development programs.
After the period, I was very pleased to announce that Yilmaz Mahshid, PhD, will join the company as Chief Financial Officer (CFO), starting in the second quarter this year. I worked with Yilmaz for three years at PledPharma and am very happy to renew this fruitful collaboration.
On the Board level, the nomination committee has nominated Dr Thomas Lönngren (chairman) and Mats Blom to the Board of Directors. The decision is expected to be taken at the Annual General Meeting on April 29. We are grateful to be to able attract Thomas and Mats to the Board as they will add valuable experience, knowledge and expertise to Egetis when building the company with focus on orphan late-stage development, registration and commercialization for the future. Among other things, Thomas’s ten-year mandate as Head of the European Medicines Agency (EMA) will provide an extra edge and understanding of the regulatory work in the complex life science sector.
Our focus on our clinical candidates with their opportunity to provide treatment for patients suffering from rare and serious diseases is firm as we shape the future of Egetis, our exciting company focusing on the orphan drug and rare disease segment. We continue to carefully monitor the impact of the Covid-19 pandemic and take every precaution to ensure that staff, collaborators, and study participants are safe and stay well, while progressing our clinical studies with high data quality.
I believe we are well positioned to deliver on our projects Emcitate and Aladote and their respective pivotal studies. I look forward to relaying news to you around the clinical studies and the progress of Egetis Therapeutics.
Nicklas Westerholm, CEO
Egetis Therapeutics AB (publ.) Stockholm