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Regulus Therapeutics Reports Positive Phase 1b Results for ADPKD Treatment

Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical pioneer focusing on the development of innovative treatments that target microRNAs, has announced significant positive outcomes from its Phase 1b Multiple-Ascending Dose (MAD) clinical trial involving Farabursen (RGLS8429). This treatment is specifically designed for patients suffering from Autosomal Dominant Polycystic Kidney Disease (ADPKD).

Overview of the Trial

The Phase 1b MAD study is a double-blind, placebo-controlled clinical trial aimed at assessing the safety, tolerability, pharmacokinetics, and pharmacodynamics of Farabursen in adults diagnosed with ADPKD. Conducted with three different weight-based dosing levels as well as a fixed dose of 300 mg, this assessment involved both immediate impacts and follow-up evaluations.

Primary focus points of the trial included monitoring changes in urinary levels of polycystin (PC) 1 and PC2 proteins, along with an exploratory evaluation of kidney volume growth rate over time. These biomarkers directly correlate with the severity of the disease and its impact on renal function, making them critical in understanding Farabursen’s efficacy.

Promising Results from the Fourth Cohort

In the most recent analysis, which focused on 26 subjects receiving a fixed dose of 300 mg of Farabursen bi-weekly for three months, significant improvements in biomarker levels and kidney growth stasis were recorded. Consistent with prior findings, increased urinary levels of PC1 and PC2 proteins indicated positive mechanistic responses when compared to control subjects receiving placebo, with p-values reflecting a significance of less than 0.05.

Moreover, the study observed that patients treated with Farabursen had a mean halt in growth of height-adjusted total kidney volume (htTKV) over a period of four months, a substantial finding given that placebo subjects exhibited notable kidney growth.

Comparative exploratory analyses indicated that patients treated with either 3 mg/kg or 300 mg of Farabursen experienced a reduction in htTKV growth, against a backdrop of past placebo-controlled cohorts, establishing a compelling argument for the medication’s efficacy.

Future of Farabursen

Following the promising data generated from this cohort, Regulus Therapeutics is optimistic about advancing its clinical pathway. The company is on track to initiate a pivotal Phase 3 trial scheduled to commence in the third quarter of 2025. This trial aims to substantiate the observed effects further and potentially lead to an accelerated approval pathway through regulatory bodies.

Dr. Preston Klassen, President and Head of Research & Development at Regulus, expressed his enthusiasm regarding the results, indicating that the 300 mg fixed dose appears capable of achieving optimal target exposure for patients. He pointed out that the halted kidney growth observed after a brief treatment period presents a substantial breakthrough in managing ADPKD.

In tandem, Jay Hagan, CEO of Regulus Therapeutics, acknowledged the contributions of the patients and medical professionals involved in the trial, emphasizing the hope that Farabursen presents in a landscape with limited treatment options for ADPKD.

About ADPKD and Farabursen

ADPKD is a genetic disorder known for causing multiple cysts to develop in the kidneys, leading to serious complications such as end-stage renal disease in many cases. Current estimates suggest that approximately 160,000 patients are diagnosed with ADPKD in the United States, while global prevalence ranges between 4 to 7 million individuals.

Farabursen aims to inhibit the miR-17 pathway, resulting in improved kidney function and size, as evidenced by preliminary results from various cohorts within the clinical studies held to date.

For those wishing to learn more about the MAD clinical trial, further details are available on clinicaltrials.gov (NCT05521191).

In summary, the recent findings from Regulus Therapeutics signify hope for ADPKD sufferers and suggest advancing potential treatments that could help manage this challenging condition more effectively.