Guard Therapeutics announced that the United States Food and Drug Administration (FDA) has provided positive feedback regarding the continued development of the company's leading drug candidate RMC-035 in open-heart surgery. The next developmental step includes a phase 2b study with the aim of identifying an optimal dosage of RMC-035. The phase 2b study is also expected to enable the most efficient design of a subsequent pivotal phase 3 study.

The advisory meeting with the FDA was based on the results of the recently completed randomized, double-blind, and placebo-controlled phase 2 study AKITA with RMC-035. The study included a total of 177 patients who underwent open-heart surgery with an increased risk of developing kidney injuries. The study results showed a clinically relevant and statistically significant improvement in kidney function compared to placebo at 90 days after surgery, both as measured by change in kidney function (estimated glomerular filtration rate, eGFR) over time and the number of kidney-related events according to a composite outcome measure (major adverse kidney events, MAKE), including death, dialysis treatment or at least a 25% loss of eGFR compared to baseline.

In line with standard drug development practice, the next step is to conduct a phase 2b study aimed at identifying an optimal dosage for RMC-035 in a subsequent pivotal study. The phase 2b study will be randomized, double-blind and placebo controlled, and, like the AKITA study, include patients undergoing open-heart surgery who are at increased risk of developing kidney injuries in connection with the procedure. The study is expected to include a total of approximately 160 patients divided into two different dose arms of RMC-035 (60 mg and 30 mg) as well as a control arm (placebo).

The primary endpoint is change in eGFR from study start to 90 days after surgery, which corresponds to the planned follow-up period of the study. Patient recruitment is expected to begin in the third quarter of 2024 and continue for approximately one year. Study results are expected to be available approximately 6 months after completion of patient recruitment.

RMC-035 has previously been awarded a Fast Track Designation by the FDA. This designation means opportunities for faster development and a shorter registration process for RMC-035, as well as the opportunity for more frequent guidance from the FDA. Based on the current dialogue with the FDA, and assuming positive results in the planned phase 2b study, the company plans to subsequently conduct a single pivotal phase 3 study with an endpoint reflective of an irreversible loss of kidney function, and to discuss the potential for a study design that allows for accelerated approval.