Rectify Pharma Presents Translational Data Demonstrating Improvements in Kidney Function and Reductions in Vascular Calcification at ERA 2025
Potent, selective ABCC6-targeting positive functional modulator (PFM) improved multiple markers of renal function in animal models of chronic kidney disease (CKD)
PFM improved inorganic pyrophosphate (PPi) efflux via on-target engagement of the ABCC6 membrane transporter and reduced vascular calcification in animal models of CKD
BOSTON, June 06, 2025 (GLOBE NEWSWIRE) -- Rectify Pharmaceuticals, Inc., (“Rectify”) a biotechnology company pioneering positive functional modulators (PFMs) that restore and enhance membrane protein function, today showcased new data from its cardiorenal program with its ABCC6-targeting PFM, RTY-822. The results were presented in an oral presentation during the European Renal Association (ERA) 2025 Congress, taking place in Vienna, Austria, June 4 – 7, 2025.
“These translational results demonstrate that Rectify’s PFM reduces vascular calcification and kidney injury and improves kidney function through enhancement of ABCC6 function,” said Rajesh Devraj, Ph.D., President and Chief Executive Officer of Rectify. “We believe the strength of these data underscores the potential of our approach to offer a novel and differentiated therapeutic strategy to treat renal dysfunction and injury, as well as address vascular calcification driving cardiovascular disease. We are committed to continuing to advance meaningful therapies for patients with CKD and serious cardiovascular conditions.”
Robert Hughes, Ph.D., Chief Scientific Officer of Rectify, added, “Enhancing the function of ABCC6 holds the potential to address key drivers of vascular calcification and tubular dysfunction seen in CKD. With demonstrated on-target engagement, RTY-822 shows significant promise to reduce CKD-associated calcium deposits in arterial walls, a well-established contributor to cardiovascular morbidity and mortality seen in patients with CKD, and remarkably, also improves glomerular function and tubular injury. The translational readiness of this program makes it a compelling de-risked asset with the potential to address CKD and multiple cardiovascular indications.”
Title: A Positive Functional Modulator of ABCC6 Decreases Vascular Calcification and Improves Kidney Function in a Rat Adenine Diet Model of Chronic Kidney Disease
Abstract Number: 1723
Presenter: John Miller, Ph.D.
Session: Chronic Kidney Disease, Focused Oral Room 4
Date and Time: Friday, June 6, 2025, 8:51 a.m. – 8:57 a.m. CEST
In CKD, reduced ABCC6 activity or expression may lead to lower PPi levels, contributing to pathological calcification in blood vessels and kidneys. Restoring ABCC6 function or enhancing its expression offers a new and compelling therapeutic strategy to prevent the progression of CKD towards end-stage renal disease in addition to reducing large vessel calcification, a major driver of cardiovascular complications in CKD.
Key findings
- RTY-822, an orally administered PFM targeting ABCC6 increased both ABCC6 protein expression and plasma PPi levels in primary human hepatocytes, supporting its proposed mechanism of action
- In a rat model of CKD and vascular calcification induced by an adenine-enriched diet with calcitriol supplementation:
- RTY-822 treatment led to statistically significant reductions in vascular calcification in the aorta and the femoral arteries
- After a six-week treatment course, RTY-822 improved glomerular and tubular kidney functions, with statistically significant reductions in serum creatinine, urea, and cystatin C, and an improved estimated glomerular filtration rate (eGFR)
- Calcium accumulation in the kidney was also significantly reduced
- Kidney injury biomarkers were reduced, including a significant decrease in plasma KIM-1 and urinary Lipocalin-2 (NGAL) levels
- In rats fed an adenine-rich diet without calcitriol supplementation, treatment with RTY-822 resulted in:
- Improved glomerular and tubular kidney functions
- Reduced serum creatinine and cystatin C levels
- Increased eGFR
- Reduced urinary NGAL levels, a biomarker of acute kidney injury
- Improved glomerular and tubular kidney functions
The presentation is available on the Rectify website at https://rectifypharma.com/publications/.
About Rectify Pharmaceuticals, Inc. (“Rectify”)
Rectify is advancing Positive Functional Modulators (PFMs), a novel class of oral, small molecules that restore and enhance membrane protein function to address the underlying cause of serious diseases. Rectify’s PFMs have potential to modulate the activity of wild-type and mutated membrane-bound proteins, a historically difficult challenge with a small molecule approach. The Company’s breakthrough product platform enables efficient and rapid discovery of first- and best-in-class small molecule therapies with the potential to address membrane protein dysfunction for treatment of rare and common diseases, including liver, cardio-renal-metabolic, and neurodegenerative diseases. Rectify was founded and seeded by Atlas Venture who co-led the $100M Series A round with Omega Funds and were joined by Forbion and Longwood Fund.
For more information, please visit www.rectifypharma.com or follow us on X and LinkedIn.
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