Interim Report January 1-September 30, 2020

January – September
·       Net sales for the period amounted to MSEK 35.8 (65.5)
·       Loss for the period MSEK -103.7 (-38.4)
·       Cash and cash equivalents MSEK 159.4 (286.7)
·       Cash flow for the period MSEK -95.6 (52,9)
·       Loss per share before/after dilution SEK -1.9 (-0.8)
 
Significant events during the period January- September
Aladote®
·       PledPharma has planned for one pivotal phase II/III study which is expected to be sufficient for a marketing authorization application in both US and EU
PledOx®
·       In March PledPharma decided to prematurely stop 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 ANSM earlier this year.
·       PledPharma has finalized the data collection from the  Phase III POLAR program during the Q3 2020 with the aim of communicating the primary resultsduring the fourth quarter 2020.The totality of data generated will enable a thorough efficacy and safety evaluation and an assessment of the benefit/risk of PledOx. This evaluation will determine if further activities to find a path forward for PledOx to treat nerve damage associated with chemotherapy is motivated.
·       Positive pre-clinical results with PledOx were presented at the Peripheral Nerve Society’s annual meeting and were published in the scientific journal Antioxidants.
 
July – September
·       Quarterly net sales MSEK 2.6 (6.2)
·       Quarterly result MSEK till -24.7 (-31.9)
·       Cash and cash equivalents MSEK 159.4 (286.7)
·       Cash flow for the period MSEK -24,6 (35.9)
·       Loss per share before/after dilution SEK -0.5 (-0.6)
 
Significant events after the reporting period
·       PledPharma acquired all outstanding shares in Rare Thyroid Therapeutics International AB (RTT) on November 3, 2020. The purchase price for the shares in RTT consists 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 PledPharma. These new shares were issued at a price of 5.25 SEK per share, amounting to a total of 334 810 061,25 SEK. In addition, the sellers of RTT are entitled to earnout payments based on the future net sales of Emcitateâ as well as an earnout payable in connection with a potential sale of a so-called US Rare Pediatric Disease Priority Review Voucher. The acquisition is disclosed in note 7.

·       The Extra General Meeting (EGM) on October 28, 2020 approved issuing no more than 38,238,085 shares with pre-emption rights for existing shareholders and, in the case of oversubscription, resolved to issue an additional maximum of 9,523,809 shares as an overallotment option.

·       The design of the pivotal Phase IIb/III study for Aladote has been completed following interactions with the FDA, EMA and MHRA. The first patient is planned to be randomized in the study in H1 2021, pending approval of clinical trial applications and no unexpected disruption due to COVID 19.

·       Peder Walberg was elected as a board member at the EGM on October 28, 2020.

·       Notice of EGM issued for December 11, 2020 to resolve for a company name change to Egetis Therapeutics.

Comments from the CEO
 
PledPharma’s acquisition of Rare Thyroid Therapeutics creates a new focused orphan drug development company
In Q3, we continued to work intensely with our clinical portfolio while also refining PledPharma’s future strategy, which ultimately led up to one of the most fundamental events in the history of the company with the acquisition of Rare Thyroid Therapeutics (RTT),  a privately held company focused on rare thyroid hormone signalling disorders. The acquisition creates a new specialized late-stage orphan drug development company with core expertise in clinical development, registration and commercialization.

The new company, which will be called Egetis Therapeutics, will initially have two orphan drug assets in late stage development: Emcitate and Aladote. These products have a clear path to launch in EU and US in approximately three years. Egetis intends to set up a niche marketing organization to address the attractive orphan drug market.
 
At the same time, we announced a fully underwritten rights issue of approximately SEK 200 million (plus an over-allotment option of approximately SEK 50 million) which will finance the development of Emcitate and Aladote up to market approval.
Emcitate is 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 has been completed. A pivotal Phase IIb/III early intervention trial in very young patients is planned to start in Q4 2020. Interim results are planned to be available in 2022 to pave the way for regulatory approvals and commercial launch. Emcitate has Orphan Drug Designation (ODD) in both EU and the US. We believe that Emcitate holds potential to become the first approved therapy for the treatment of MCT8 deficiency.

The acquisition of RTT is an important step towards building a company with a strategic focus on the attractive orphan drug patient segment. The RTT team, with long experience within orphan drugs, will complement PledPharma’ s late stage development focused organisation, building a sustainable orphan drug company dedicated to development and commercialisation of therapies for rare diseases. We look forward to building a new Swedish development company with the capacity to bring our own projects to market. The goal is to offer medicines to patients with serious and rare diseases lacking adequate medical treatments and create value for patients and shareholders.
 
Aladote registrational study
Aladote represents PledPharma’s internal orphan drug candidate. In October, we announced that the study design for the pivotal Phase IIb/III study with Aladote has been completed after interactions during Q2-Q3 2020 with the US Food and Drug Administration (FDA), the European Medicines Agency (EMA) and the Medicines & Healthcare products Regulatory Agency (MHRA) in the UK.

Following successful clinical trial applications and subsequent study conduct, marketing authorization applications in the US, EU and UK are planned for 2023.

Aladote has ODD status in the US and an application is pending also in the EU (post-Brexit). A proof of principle study has been successfully completed, establishing safety and tolerability, suggesting that Aladote may reduce liver injury after paracetamol overdose.

The Phase IIb/III study is targeting patients with increased risk of liver injury, arriving late, more than 8 hours after a paracetamol overdose, to hospital. In these patients, the current standard of care, N-acetylcystein (NAC), is no longer effective. The study consists of two parts, with an interim analysis carried out by an independent Drug Safety Monitoring Board (DSMB). The total planned number of patients are 225, and first patient is planned to be randomized in the study in H1 2021, pending approval of clinical trial applications and no unexpected disruption due to Covid-19.

We are very pleased to have completed the interactions with FDA, EMA and MHRA and look forward to initiating the study. We are committed to the continued development of Aladote and believe it 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.

PledOx POLAR program
The phase III POLAR program for the drug candidate PledOx was prematurely stopped in Q2 2020. Results are expected to be announced in Q4 2020 and will enable a thorough evaluation of the safety and efficacy, providing an assessment of the benefit/risk of PledOx in patients with chemotherapy induced peripheral neuropathy (CIPN). This evaluation will determine if further development of PledOx via strategic partnership/s are motivated.
  
Robust cash position
In order to continue the development of our clinical portfolio, we have a robust cash position of approximately 159 million SEK in cash reported on September 30, 2020, together with the planned underwritten rights issue of approximately SEK 200 million.

Our focus on our clinical development programs and participating subjects remain firm while we build the future of the new company, Egetis Therapeutics. 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.

I look forward to relaying news to you around our planned studies for Aladote and Emcitate, as well as the upcoming results from the POLAR program.

Nicklas Westerholm, CEO
PledPharma AB (publ), Stockholm