Genkyotex Provides Q4 Business Update and Reports Cash Position

Regulatory News:

Genkyotex (Euronext Paris & Brussels: FR0013399474 – GKTX) (Paris:GKTX) (Brussels:GKTX), a biopharmaceutical company and the leader in NOX therapies, today reported cash and cash equivalents of €1.2 million as of December 31, 2020. The existing cash and cash equivalents provide cash runway to the end of February 2021. After this date Genkyotex will be required to fund the ongoing operations with new capital. Calliditas Therapeutics, the majority shareholder of Genkyotex, has expressed its intent to participate in such future financings on a pro rata basis. Until such financing has occurred, Calliditas will support Genkyotex by way of debt financing to enable the Company to meet its liabilities as they fall due and to carry on its normal business without any significant curtailment of operations.

Business update

On August 13, 2020, the Company announced the agreement by Calliditas to acquire 7,236,515 ordinary shares of Genkyotex, representing 62.7% of the share capital and voting rights from the Company’s largest shareholders and management team.

On December 17, 2020, the Company announced the results of the tender offer, according to the results published on December 16, 2020 by the Autorité des marchés financiers (AMF), a total of 2,885,161 shares were tendered to the public offering. Following the close of the public offer, Calliditas held 10,121,676 shares of the Company representing 86.24% of the share capital and voting rights of the Company.

Clinical highlights

• Phase 1 trial with setanaxib at higher doses: in January 2021, the Company reported positive data from its Phase 1 clinical trial to evaluate the safety and pharmacokinetics of setanaxib at dosages up to 1,600 mg/day.

• Development strategy for setanaxib in primary biliary cholangitis (PBC): based on the Phase 1 trial data and interactions with US and European regulators in 2020, the Company recently announced that it plans to launch a pivotal and potentially registrational Phase 2/3 trial of setanaxib in patients with primary biliary cholangitis (PBC) in the second half of 2021. The final design and protocol details are subject to feedback from the US Food and Drug Administration (FDA).

• Phase 2 proof-of-concept study in patients with head and neck cancer: the Company recently announced that it plans to initiate a Phase 2 proof-of-concept study in patients with head and neck cancer before the end of the year. The trial will evaluate administration of setanaxib, targeting cancer associated fibroblasts (CAFs), in conjunction with immunotherapy.

• Orphan drug designation (ODD) for setanaxib in primary biliary cholangitis (PBC): setanaxib was granted orphan drug designation for the treatment of primary biliary cholangitis (PBC) by the US Food and Drug Administration (FDA) in October and by the European Commission in December, following the positive opinion from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA).

• Phase 2 trial of setanaxib in idiopathic pulmonary fibrosis (IPF): the Company announced on September 14, 2020, the enrollment of the 1^st patient in a Phase 2 trial of setanaxib in IPF. This study is conducted in accordance with a protocol approved by the U.S. Food and Drug Administration (FDA) and the relevant Institutional review board (IRB). This investigator-initiated study is being led by Professor Victor Thannickal of the University of Alabama at Birmingham and includes a consortium of five research centers of excellence in the United States. It is fully funded by an $8.9 million grant awarded to Professor Thannickal’s team by the U.S. National Institutes of Health (NIH). The aim of the study is to evaluate the safety and efficacy of setanaxib dosed at 800 mg/day (400 mg BID) in 60 IPF patients receiving standard treatment (pirfenidone or nintedanib) over a period of 24 weeks.

• Phase 2 trial of setanaxib in diabetic kidney disease (DKD): following the Company’s Phase 2 trial with setanaxib in PBC, the DKD trial protocol was amended to increase the dose to 400 mg BID. To date, 29 patients have completed the full 48-week treatment and no safety signals have been identified. The DKD trial is being conducted primarily in Australia, with work ongoing to activate centers in New Zealand, Denmark, and Germany. In the context of the COVID-19 pandemic, investigators have taken steps to minimize patient visits to investigation centers, in accordance with applicable rules and recommendations, but investigators cannot exclude a possible slowdown in new patient enrollment in the study.

Q4 2020 Financial highlights