Clinical development

Positive results from clinical phase 1 program in 2020

In spring 2019, the first clinical (first-in-human) study of ROSgard was initiated. A clinical phase 1 program including three separate studies has been successfully completed and demonstrates a favorable safety profile and appropriate pharmacokinetics of ROSgard for the future treatment of acute kidney injures (AKI). All studies were conducted in Sweden.

The following studies were completed with positive results:

  • ROS-01: A single ascending dose (SAD) study in healthy subjects where ROSgard was administered in the dose range of 0.08 to 2.6 mg/kg
  • ROS-02: A multiple ascending dose (MAD) study in healthy subjects where ROSgard was administered in the dose range of 0.43 to 1.3 mg/kg
  • ROS-03: A study in subjects with various degree of renal impairment (eGFR≥15 till ≤90 mL/min/1.73 m2) where ROSgard was administered at a single dose of 0.43 mg/kg.

In summary, the results of the phase 1 program demonstrates that ROSgard has a favorable safety profile and pharmacokinetic characteristics and provide a solid foundation for future studies in patients undergoing open chest cardiac surgery (i.e. with use of heart-lung machine).

On the basis of available phase 1 results, the first trial in patients undergoing open-chest cardiac surgery is planned in Germany (phase 1b). The clinical trial application has been approved by the German health authorities (BfArM) and recruitment of patients is planned to commence in February 2021.

In parallel, preparations are ongoing for a planned phase 2 program to be conducted both in the US and in EU. The primary purpose of a phase 2 study is to demonstrate a clinically relevant treatment effect in the target patient population, i.e. patients at increased risk for developing acute kidney injury (AKI) after open chest cardiac surgery. The design of the phase 2 study will be completed based on regulatory feedback be the US Food and Drug Administration (FDA) and leading global clinical experts and key opinion leaders in the field.