Clinical development

In early 2022, a global Phase 2 study of RMC-035 was launched

In spring 2019, the first clinical (first-in-human) study of RMC-035 (ROSgard) was initiated. A clinical phase 1 program including four separate studies has been successfully completed and demonstrates a favorable safety profile and appropriate pharmacokinetics of RMC-035 for the future treatment of acute kidney injures (AKI).

In April 2022, the first patient in a global phase 2 study (AKITA) was subsequently dosed with RMC-035. The study is performed in patients who undergo open cardiac surgery and thus run the risk of developing acute kidney injuries.

During fall 2022, the clinical program was expanded to a new indication with the start of a phase 1b study with RMC-035 in kidney transplantation.

Clinical studies with RMC-035

Early phase 1-studies

The following studies were completed with positive results:

      • ROS-01: A single ascending dose (SAD) study in healthy subjects where RMC-035 was administered in the dose range of 0.08 to 2.6 mg/kg. (Study conducted in Sweden)
      • ROS-02: A multiple ascending dose (MAD) study in healthy subjects where RMC-035 was administered in the dose range of 0.43 to 1.3 mg/kg. (Study conducted in Sweden)
      • ROS-03: A study in subjects with various degree of renal impairment (eGFR≥15 till ≤90 mL/min/1.73 m2) where RMC-035 was administered at a single dose of 0.43 mg/kg. (Study conducted in Sweden)

    In summary, the results of the phase 1 program demonstrates that RMC-035 has a favorable safety profile and pharmacokinetic characteristics and provide a solid foundation for continued clinical development.

    Clinical studies in cardiac surgery


    On the basis of existing phase 1 results the first study in patients undergoing cardiac surgery (phase 1 b) was initiated in early 2021. The study was conducted at a larger site for cardiothoracic surgery in Germany. The main purpose of the phase 1b study was to evaluate the investigational drug’s safety and tolerability profile and its pharmacokinetic properties in 12 patients who underwent extensive open-chest heart surgery and had additional risk factors for acute kidney injury (AKI). The study results showed that RMC-035 was well tolerated and no side effects attributed to the investigational drug were reported. Pharmacokinetic analyses showed desirable blood concentrations of RMC-035, providing support for the dosing regimen intended to be used in the planned phase 2 study.

    In addition, explorative analyses of several well-established biomarkers indicate that RMC-035 potentially has the ability to protect kidney cells from injury associated with heart surgery.

    AKITA study

    In early 2022, a global Phase 2 study of RMC-035, the AKITA study, was launched. The primary purpose of the 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. A number of important secondary endpoints, such as changes in renal function during hospitalization and up to three months after surgery, will be analysed. The study design will facilitate an informed decision about optimal Phase 3 design. The design of the phase-2 study has been anchored with leading global experts in the field.

    The AKITA study is being conducted in both North America and Europe and includes approximately 270 patients. The phase 2 study is randomized, double-blind and placebo-controlled and enables, in the event of a positive outcome, the start of a pivotal phase 3 study.

    Clinical studies in kidney transplantation


    Based on available preclinical and clinical results, we have chosen to expand the clinical program for RMC-035 to kidney transplantation. The recipient of a donated kidney from a deceased donor will be treated with RMC-035 in order to reduce the injury caused by the transplant and hence improve kidney function and ultimately prolong the survival of the donated kidney.

    ROS-06, was initiated in August 2022 and is an unblinded study without control group and includes 8-12 patients undergoing kidney transplantation at Karolinska University Hospital in Huddinge. Initially, eight patients are included in two dose groups and after an interim analysis of the pharmacokinetics of the drug candidate, a decision can be made to possibly move on to an optional third dose group. The primary endpoints of the study include the pharmacokinetic profile of RMC-035, and the results will form the basis of a potential subsequent Phase 2 study (efficacy study) with the goal of improving kidney function in renal transplant patients with deceased donor.