Rectify Pharmaceuticals presented data on RTY-822, a positive functional modulator (PFM) targeting the ABCC6 membrane transporter, at the 2025 European Renal Association Congress. Preclinical studies demonstrated RTY-822 improved kidney function and reduced vascular calcification in animal models of chronic kidney disease (CKD). The PFM works by increasing ABCC6 protein expression and plasma inorganic pyrophosphate (PPi) levels, mitigating the effects of reduced ABCC6 activity associated with CKD.
This research is potentially groundbreaking for CKD patients, who currently face limited treatment options and often experience progressive decline in kidney function leading to end-stage renal disease. The data suggests RTY-822 could offer a novel therapeutic approach, not only slowing CKD progression but also addressing the associated vascular calcification that contributes significantly to cardiovascular complications and mortality in this population. This dual action represents a substantial advance over existing treatments, which typically focus on individual symptoms rather than the underlying disease mechanisms.
In preclinical models, RTY-822 demonstrated statistically significant improvements in multiple markers of kidney function, including serum creatinine, urea, cystatin C, and estimated glomerular filtration rate (eGFR). Importantly, the treatment also reduced vascular calcification in both the aorta and femoral arteries. These findings were consistent across different CKD models, strengthening the evidence for RTY-822’s efficacy. Additionally, the observed reduction in kidney injury biomarkers like KIM-1 and NGAL suggests the treatment may protect against further kidney damage.
The promising preclinical results for RTY-822 suggest the potential for a new class of CKD therapies targeting ABCC6. Further research, including clinical trials, will be crucial to validate these findings in humans and assess the long-term safety and efficacy of this promising PFM. If successful, RTY-822 could significantly alter the CKD treatment landscape, offering patients a much-needed therapeutic option to slow disease progression and reduce cardiovascular risk.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
