Regulatory press release

Egetis Therapeutics presents the fourth quarter and the year-end report 2020

October – December
·        Quarterly net sales MSEK 5.3 (17.1)
·        Quarterly result MSEK -75.4 (-23.0)
·        Cash and cash equivalents MSEK 287.9 (255.1)
·        Cash flow for the period MSEK 129.8 (-29.1)
·        Loss per share before/after dilution SEK -0.7 (-0.4)

Significant events during the period October-December
·        Egetis Therapeutics acquired all outstanding shares in Rare Thyroid Therapeutics International AB (RTT) on November 3, 2020. The purchase price for the shares in RTT consisted of a cash component of 60 MSEK, funded from own cash-in-hand, and a share purchase price consisting of 63,773,345 new shares in Egetis Therapeutics. These new shares were issued at a price of 5.25 SEK per share, amounting to a total of 334 810 061,25 SEK. Further details of the acquisition are disclosed in note 7.
·        Egetis Therapeutics completed an oversubscribed rights issue with an overallotment option of 47,761,894 shares in total, raising gross proceeds of SEK 251 MSEK.
·        Peder Walberg was elected as a board member at the EGM on October 28, 2020.
·        The Company name was changed to Egetis Therapeutics AB at the EGM on December 11, 2020.
·        US Food and Drug Administration (FDA) granted the company’s application for Rare Pediatric Disease designation (RPD) for Emcitate in the treatment of MCT8 deficiency.
·        The first patient was dosed in the pivotal Phase IIb/III early intervention study in young patients with the drug candidate Emcitate.
·        The design of the pivotal Phase IIb/III study for Aladote was completed following interactions with the FDA, the European Medicines Agency (EMA) and the Medicines & Healthcare products Regulatory Agency (MHRA) in the UK.
·        On December 15, the company announced that PledOx did not meet the efficacy endpoint in the prematurely closed Phase III POLAR program.
January – December
·        Net sales for the period MSEK 40.7 (82.6)
·        Loss for the period MSEK -179.1 (-61.4)
·        Cash and cash equivalents MSEK 287.9 (255.1)
·        Cash flow for the period MSEK 34.2 (24.1)
·        Loss per share before/after dilution SEK -2.7 (-1.2)

Significant events during the period January- December
·        Egetis Therapeutics acquired all outstanding shares in RTT, see note 7.
·        Rights issue of 47,761,894 shares, raising gross proceeds of 251 MSEK.
·        Name change to Egetis Therapeutics AB (publ.).

Emcitate®
·        US FDA granted the company’s application for Rare Pediatric Disease designation (RPD) for Emcitate in the treatment of MCT8 deficiency.
·        The first patient was dosed in the pivotal Phase IIb/III early intervention study in young patients with the drug candidate Emcitate.

Aladote®
·        The design of the pivotal Phase IIb/III study for Aladote, was completed following interactions with the FDA, EMA and MHRA. If successful, it is considered to be sufficient for a marketing authorization application in both US and EU.

PledOx®
·        In Q2 the company decided to prematurely close the POLAR Phase III program. The decision was taken after a recommendation from the independent Drug Safety Monitoring Board (DSMB) and followed the clinical holds issued by FDA and French Regulatory Authority (ANSM) earlier in the year.
·        On December 15, the company announced that PledOx did not meet the efficacy endpoint in the prematurely closed Phase III POLAR program.

Significant events after the reporting period
·        The company’s Chief Medical Officer (CMO) has decided to leave the company to pursue other opportunities.
·        The company announced that Aladote will be presented as a novel emerging treatment of paracetamol overdose at two upcoming scientific conferences in March and April.
·        The company has appointed Kristina Sjöblom Nygren, MD, as CMO, effective May 1, and will be member of the company’s leadership team. The recruitment expands the orphan drug experience and expertise also to the clinical and medical function, further strengthening the company’s alignment and strategic focus on this segment.

Comments from the CEO

As we now put 2020 behind us, we can look back on an eventful year, where Covid-19 pandemic made a deep and lasting impact on all of us. This was also a year when PledPharma through the acquisition of Rare Thyroid Therapeutics (RTT) created Egetis Therapeutics – our new focused orphan drug development company with two important assets - Emcitate and Aladote - in late-stage development. The acquisition was completed in early November, and the name change became official in December.
The acquisition of RTT, with its focus on rare thyroid hormone signalling disorders represents an important step in building a company with a strategic focus on the attractive orphan drug segment. The teams from RTT and PledPharma with complementing experience in orphan drugs, late-stage development and commercialisation set a solid foundation for Egetis 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.
Both Emcitate and Aladote have a clear path to market approval in the EU and the US in the coming three years, approximately. As part of the new strategic direction, Egetis intend to set up a niche marketing organization to launch our exciting assets, creating a foothold in the attractive orphan drug market. As a first step in this important transition, we appointed a Vice President for Commercial Operations in November.

First patient dosed in the Phase IIb/III study with Emcitate
Emcitate is being developed for the treatment of MCT8 deficiency, which is 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. A successful Phase IIb trial was completed in 2018.
The first patient in the pivotal Phase IIb/III early intervention trial was dosed in December. The study is an international, open label, multi-center study in children younger than 30 months with MCT8 deficiency, conducted in both Europe and North America. Interim results are targeted to be available in 2022 and expected to pave the way for regulatory approvals and commercial launch.
Emcitate has been granted Orphan Drug Designation (ODD) in both EU and the US. In addition, the US Food and Drug Administration (FDA) granted it Rare Pediatric Disease (RPD) designation in November. Upon approval of a new drug application (NDA), sponsors holding a RPD and meeting the criteria specified can apply to receive a US Rare Pediatric Disease Priority Review Voucher (PRV). A PRV allows accelerated FDA review of a new drug application for any drug candidate, in any indication, thus shortening time to market for a new entity in the US. The voucher may also be sold or transferred to another sponsor.

Study design of Aladote pivotal study completed
In October, we announced that the study design for the pivotal Phase IIb/III study with Aladote has been finalized following valuable interactions with the US FDA, the EMA and the MHRA during Q3 2020. Preparations for the planned Phase IIb/III study are ongoing in US, UK and EU together with the CRO selected to conduct the study. Due to the ongoing Covid-19 pandemic, it is 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 the EU is planned to be submitted to the EMA during Q1 2021.

PledOx® POLAR program
The Phase III POLAR program for the drug candidate PledOx was prematurely closed in Q2 2020. In December, we announced together with our Japanese partner Solasia that PledOx did not meet the efficacy endpoint in the POLAR program. Based on continued evaluation of the results, the strategic next steps for PledOx will be determined together with Solasia.

Cash position
The successfully completed oversubscribed rights issue in November generated gross proceeds of SEK 251 MSEK. and strengthened the institutional investor base through allocation of the overallotment option to Fourth Swedish National Pension Fund (“AP4”), NYIP (Nyenburgh Holding BV) and Nordic Cross.
To continue the development of our clinical portfolio, we reported a cash position of approximately 288 million SEK on December 31, 2020 which is planned to finance the development of Emcitate and Aladote towards to market approval.

Looking ahead
Our focus on our clinical development programs and the 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. After a smooth transition into the new company, Egetis Therapeutics, we are now well positioned to deliver on our projects, Emcitate and Aladote and their respective pivotal studies. We recently announced that Kristina Sjöblom Nygren, MD, has been appointed Chief Medical Officer (CMO). The recruitment expands the orphan drug experience and expertise also to the clinical and medical function, further strengthening the company’s alignment and strategic focus on this segment. I look forward to relaying news to you around the clinical studies and the future development of Egetis Therapeutics.

Nicklas Westerholm, CEO
Egetis Therapeutics AB (publ.) Stockholm

About Egetis Therapeutics

Egetis Therapeutics is an innovative, unique, and integrated pharmaceutical drug development company, focusing on projects in late-stage development for treatment of serious rare/niche diseases with significant unmet medical needs in the orphan drug segment. The drug candidate Emcitate is developed as the first potential treatment for patients with MCT8 deficiency, a rare disease with high unmet medical need and no available treatment. A Phase IIb clinical trial has been completed with significant and clinically relevant effects. A pivotal Phase IIb/III early intervention study has been initiated in Dec 2020 with the first patient dosed and interim results are expected in 2022. Emcitate holds Orphan Drug Designation in the US and EU and was granted Rare Pediatric Disease Designation by the US FDA in November 2020. The drug candidate Aladote is a first in class drug candidate developed to reduce the risk of acute liver injury associated with paracetamol poisoning. A proof of principle study has been successfully completed and the design of the upcoming pivotal Phase IIb/III study for Aladote has been finalized after completed interactions with FDA, EMA and MHRA. Aladote has been granted Orphan Drug Designation in the US. Results from the PledOx POLAR program in Dec 2020 shows that PledOx did not meet the efficacy endpoint. Based on further evaluation of the results from the POLAR studies, the strategic next steps for PledOx will be determined together with our partner Solasia.

Egetis Therapeutics (STO: EGTX) is listed on the Nasdaq Stockholm main market. For more information, see http:// www.egetis.com/

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