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).

In summary, the phase 1 results demonstrate that ROSgard is safe and generally well tolerated and provide a solid foundation for continued clinical development in patients undergoing open-chest cardiac surgery with use of cardio-pulmonary bypass.

  • ROS-04: On the basis of phase 1 results the first study in patients undergoing cardiac surgery is currently ongoing (phase 1 b). The study is conducted at a larger site for cardiothoracic surgery in Germany. In total 12 subjects are planned to be enrolled (8 on ROSgard and 4 on placebo). The recruitment is expected to be completed by the end of June 2021 with top-line results available towards the end of summer 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.