Positive Phase 1 results in high-dose setanaxib trial

Positive Phase 1 results in high-dose setanaxib trial

Jan 18, 2021

Genkyotex SA, a subsidiary of Calliditas Therapeutics AB (publ) (“Calliditas”) (Nasdaq OMX – CALTX; NASDAQ – CALT), today announced positive Phase 1 data demonstrating a favorable safety and pharmacokinetic profile of high-dose setanaxib, Genkyotex’s lead asset.

The Phase 1 study demonstrated that setanaxib is well tolerated at the doses tested, with no safety signal or dose-limiting toxicity being identified.  The results provide an opportunity for the company to pursue a pivotal Phase 2/3 clinical trial in patients with primary biliary cholangitis (PBC), based on interactions with the FDA.  The study assessed the safety and pharmacokinetics of oral setanaxib at selected doses in 46 healthy adult male and female subjects.  The trial consisted of a single ascending dose (SAD) part and a multiple ascending dose (MAD) part with dosing up to 1600mg/day.

Previously, doses of up to 800 mg/day were evaluated in a 24-week Phase 2 trial in PBC patients. In that trial, setanaxib dosed at 800 mg/day achieved reductions in markers of cholestasis, including alkaline phosphatase, and in multiple non-invasive markers of liver fibrogenesis, including liver stiffness and PRO-C3 and C3M. Significant improvement in fatigue was also achieved. All doses tested in that trial were well-tolerated, with no safety signal compared to placebo.

“This result provides a foundation for a clinical development program using higher doses of setanaxib across a variety of orphan indications.  This includes the potential to launch a pivotal phase 2/3 trial with a clearly differentiated drug candidate in PBC but will also allow us to further explore a select number of kidney related orphan diseases in which inflammation and fibrosis play a part.”, said CEO Renée Aguiar-Lucander.

Calliditas acquired 62.7% of the share capital and voting rights of Genkyotex, in November 2020, before submitting a simplified public mandatory offer to the shareholders of Genkyotex on 26 November 2020. Calliditas controls 86.2% of the share capital and total number of votes of Genkyotex.

For further information, please contact:

Marie Galay, IR Manager, Calliditas

Tel.: +44 79 55 98 12 45, email: marie.galay@calliditas.com