Guard Therapeutics has announced the publication of Phase 2 clinical study (AKITA) results in eClinicalMedicine, a Lancet Discovery Science journal, highlighting the kidney-protective effects of RMC-035 in patients undergoing open-heart surgery. The study's findings support the ongoing clinical development program for RMC-035 and the design of the recently initiated Phase 2b POINTER study.
The AKITA study demonstrated that RMC-035 had a statistically significant and clinically relevant kidney-protective effect compared to placebo in a stable phase after surgery. This was evidenced by improvements in kidney function, as measured by estimated glomerular filtration rate (eGFR) at 90 days post-surgery. The study also showed a reduction in Major Adverse Kidney Events (MAKE) at the same time point, an endpoint expected to be the primary efficacy measure in a future pivotal Phase 3 trial.
Key Findings from the AKITA Study
The publication, titled Efficacy of therapeutic alpha-1-microglobulin in reducing kidney injury after cardiac surgery: a randomized placebo-controlled Phase 2 study, details the results of the trial. According to Professor Alexander Zarbock from the University Hospital of Münster, Germany, the global principal investigator for the AKITA study, the results indicate "a potential breakthrough for the treatment of kidney injury associated with open-heart surgery." He emphasized the concordance between improved renal function and the reduction of MAKE, reflecting different ways of assessing the kidney-protective treatment effect.
The study enrolled 177 patients and demonstrated a statistically significant and clinically relevant favorable effect of RMC-035 on long-term kidney outcomes in this patient population.
RMC-035: A Novel Therapeutic Approach
RMC-035 represents a first-in-class drug, a recombinant and modified variant of the endogenous protein alpha-1-microglobulin. It protects cells and their mitochondria from damage caused by oxygen deprivation and elevated levels of the oxygen-binding and toxic protein heme. Preclinical studies have shown favorable treatment effects of RMC-035 in several disease models. The drug has a natural affinity for the kidneys and is being developed as an intravenous kidney-protective treatment for patients at high risk of developing acute kidney injury (AKI).
Ongoing Clinical Development
The next step in the clinical program for RMC-035 is the Phase 2b POINTER study, which aims to optimize the dose and identify the exact target patient population before a pivotal Phase 3 study. Patient recruitment for the POINTER study has recently begun and is expected to take approximately one year. Overall study results are anticipated to be available about 6 months after the completion of patient recruitment.
RMC-035 has received Investigational New Drug (IND) clearance from the U.S. Food and Drug Administration (FDA) for the treatment of AKI in open-heart surgery. The FDA has also granted Fast Track Designation to RMC-035 to reduce the risk of irreversible loss of kidney function, the need for dialysis treatment, or death after open-heart surgery in patients at elevated risk of AKI.
