SAN DIEGO - Regulus (NASDAQ:RGLS) Therapeutics Inc. (NASDAQ:RGLS), a biopharmaceutical company, has announced positive topline results from a Phase 1b trial of RGLS8429, a treatment candidate for autosomal dominant polycystic kidney disease (ADPKD). The study demonstrated a dose response in urinary biomarkers and a reduction in kidney volume in a majority of patients at the 3 mg/kg dose level.
The randomized, double-blind, placebo-controlled trial evaluated the safety, tolerability, and pharmacokinetics of RGLS8429 across various doses. Notably, the 3 mg/kg dose showed statistical significance in the change from baseline in urinary polycystins 1 and 2 (PC1 and PC2), which are inversely correlated with disease severity.
Exploratory magnetic resonance imaging (MRI) analyses indicated a reduction in total kidney volume (htTKV) in 70% of patients treated with the 3 mg/kg dose. The company has commenced enrollment for the fourth cohort of the study, which will involve an open-label fixed dose of 300 mg of RGLS8429 administered every other week for three months.
Preston Klassen, M.D., President and Head of Research & Development at Regulus, expressed satisfaction with the consistent mechanistic response and safety profile observed. The data may support a potential Phase 2/3 study under an Accelerated Approval regulatory pathway with the FDA, which the company plans to discuss.
Jay Hagan, CEO of Regulus, reinforced the company's confidence in the treatment's ability to address the genetic cause of ADPKD. Regulus anticipates an End-of-Phase 1 meeting with the FDA in the fourth quarter of this year and expects to provide an update from the final cohort by year-end.
ADPKD is one of the most common monogenic disorders and a leading cause of end-stage renal disease. The disease results in the development of multiple cysts in the kidneys and can lead to renal failure.
RGLS8429, an oligonucleotide designed to inhibit miR-17 and target the kidney, has shown improvements in kidney function and disease severity in preclinical models. The Phase 1b study follows a favorable safety and pharmacokinetics profile established in a prior Phase 1 SAD study completed in September 2022.
The information reported is based on a press release statement from Regulus Therapeutics Inc .
In other recent news, Regulus Therapeutics Inc., a biopharmaceutical company, has shared encouraging top-line results from a key study of its investigational drug RGLS8429, designed to treat autosomal dominant polycystic kidney disease (ADPKD). The findings come from the third patient cohort in the Phase 1b multiple-ascending dose study, which is assessing the safety, tolerability, and pharmacokinetics of the drug.
The treatment was well-tolerated with no significant safety concerns reported. The study demonstrated a dose-response relationship, with the 3 mg/kg dose showing more consistent increases in urinary levels of polycystins 1 and 2 (PC1 and PC2), proteins that inversely correlate with disease severity.
A statistically significant change from baseline in PC1 and PC2 levels was observed at the 3 mg/kg dose compared to placebo. Preliminary imaging analysis suggested that RGLS8429 may reduce kidney volume after three months of dosing, with 70% of subjects showing reductions at the 3 mg/kg level. The company has initiated enrollment for a fourth cohort, which will involve up to 30 patients receiving a fixed dose of 300 mg of RGLS8429 every other week for three months.
InvestingPro Insights
As Regulus Therapeutics Inc. (NASDAQ:RGLS) advances its clinical trial for RGLS8429, investors may find it prudent to consider several financial metrics and analyst insights that could impact the company's stock performance. According to InvestingPro data, Regulus has a market capitalization of $164.32 million, indicating a modest size within the biopharmaceutical industry. The company's Price to Book ratio, as of the last twelve months ending Q1 2024, stands at 1.53, which may suggest that the stock is reasonably valued in terms of its net assets.
Investors should note that Regulus has been facing challenges with profitability, as underscored by a negative P/E ratio of -5.24 for the same period. Additionally, the company's gross profit margin has been weak, with a reported gross profit of -$22.27 million, which aligns with one of the InvestingPro Tips highlighting concerns over the company's ability to generate earnings.
However, it's not all bleak for Regulus. The company has seen a strong return over the last month, with a 24.26% total price return, and an even more impressive six-month price total return of 91.6%. This suggests a potential turnaround in investor sentiment, which may be buoyed by the positive clinical trial results. Moreover, one of the InvestingPro Tips indicates that analysts have revised their earnings upwards for the upcoming period, reflecting a more optimistic outlook on the company's financial performance.
For those considering an investment in Regulus, the InvestingPro platform offers additional insights and tips that could further inform their decision. There are 12 more InvestingPro Tips available for RGLS, which investors can access by visiting InvestingPro. To take advantage of these insights, users can use the coupon code PRONEWS24 to get an additional 10% off a yearly or biyearly Pro and Pro+ subscription.
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