Benzinga | May 3, 2021 08:07AM EDT

UPDATE: Regulus Therapeutics Says 'Mean PC2 levels increased compared to baseline levels (>20%) however the results did not reach statistical significance.'

A statistically significant increase in the PC1 biomarker was observed in the first cohort of this study. The mean increase at Day 71 (n=8) compared to baseline was greater than 50% and all patients had double digit increases in PC1 levels with an overall trend showing increasing levels of both PC1 and PC2 over time. Mean PC2 levels increased compared to baseline levels (>20%) however the results did not reach statistical significance. Importantly, at the time of the analysis, mutational status was not known and may further contribute to understanding differences in response rates. Approximately 85% of patients with ADPKD are reported to have a mutation in the PKD1 gene, while the remaining 15% have a mutation in the PKD2 gene. Measured levels of these biomarkers (PC1 and PC2) inversely correlate with disease severity and are believed to be directly linked to the underlying genetic drivers of the disease. Regulus believes these initial data demonstrate that RGLS4326 engages the target miR-17 leading to de-repression of the PKD1 and PKD2 genes and the resultant increases in measured polycystin levels. In addition to polycystin changes, one other notable improvement was observed in NGAL levels for one patient in the first cohort. As anticipated, NGAL levels for nearly all patients in this study were within the normal range. However, one patient had levels that were approximately twice the normal range at baseline and that individual saw NGAL levels drop to within the normal range by the end of study. Further analyses of the data are ongoing to better understand drivers of response and correlations with baseline characteristics and other parameters. Data from this first cohort is planned to be submitted for presentation at Kidney Week, the American Society of Nephrology annual meeting being held in November 2021.