Benzinga | May 3, 2021 09:26AM EDT

Regulus Therapeutics' RGLS4326 Reports Mixed Bag On Polycystic Kidney Disease-Associated Biomarker Study

* Regulus Therapeutics Inc (NASDAQ: RGLS) has announced top-line results from the first cohort of patients (n=9) with Autosomal Dominant Polycystic Kidney Disease (ADPKD) in its ongoing Phase 1b trial of RGLS4326.

* The study evaluates the safety, pharmacokinetics, and effects on pharmacodynamic biomarkers of multiple doses of RGLS4326.

* RGLS4326 was well tolerated by all nine patients, with no serious adverse events reported. All reported adverse events were mild and generally transient in nature.

* 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 a double-digit rise in Polycystin 1 (PC1) levels with an overall trend showing increasing levels of both PC1 and Polycystin 2 (PC2) over time.

* Mean PC2 levels increased compared to baseline levels (more than 20%). However, the results did not reach statistical significance.

* 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.

* Another notable improvement was observed in neutrophil gelatinase-associated lipocalin (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 approximately twice the normal range at baseline, and that individual saw NGAL levels drop to within the normal range by the end of the study.

* Data from this first cohort is planned to be submitted for presentation at Kidney Week, the American Society of Nephrology annual meeting held in November 2021.

* Regulus will host a conference call and webcast at 8:30 a.m. Eastern Time today.

* Price Action: RGLS shares are down 2.88% at $1.35 in the premarket on the last check Monday.