Regulus Therapeutics Inc. ( RGLS ) released topline results from all patients in the fourth cohort in its Phase 1b MAD study of farabursen (RGLS8429) autosomal dominant polycystic kidney disease (ADPKD) on Thursday.

In the fourth cohort, 26 subjects received a fixed dose of 300 mg of farabursen every other week for three months.

ADPKD is caused by mutations in the PKD1 or PKD2 genes, resulting in end-stage renal disease.

The disease is characterized by the development of multiple fluid-filled cysts, primarily in the kidneys.

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The full cohort of 26 patients demonstrated a similar mechanistic response based on urinary PC1 and PC2 levels (which inversely correlate with disease severity) and mean halting of htTKV (which inversely correlates with kidney function) growth over the four-month study period.

Cohort 4 data highlights:

Increases in urinary PC1 and PC2 levels were similar to cohort 3 and reached a similar level of statistical significance compared to placebo (PC1 p=0.026; PC2 p=0.014).

The mean htTKV growth rate over 4 months was 0.05%, while placebo subjects in the trial experienced a mean growth rate of 2.58%.

Changes in htTKV were highly correlated with changes in renal cyst volume, suggesting farabursen directly impacts disease progression by limiting abnormal cyst growth.

An exploratory analysis compared the combination of patients treated with 3 mg/kg or 300 mg fixed dose faburden (n=35) to a combined historical control group of placebo-treated patients from prior pivotal ADPKD trials (n=550).

Farabursen-treated patients experienced a mean reduction in htTKV growth rate (-0.14%) compared to a mean increase of (+1.87%) in placebo-treated patients (p=0.0056).

Farabursen at 300 mg demonstrated a favorable safety and tolerability profile in this study, consistent with earlier cohorts.

RGLS Price Action: Regulus Therapeutics ( RGLS ) stock is up 51.94% at $1.96 at publication Thursday.

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