Regulus Therapeutics Announces Completion of Enrollment in First Cohort of Phase 1b Multiple-Ascending Dose (MAD) Clinical Trial of RGLS8429 for the Treatment of Patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Rhea-AI Summary

Regulus Therapeutics Inc. (Nasdaq: RGLS) has successfully completed enrollment in the first cohort of its Phase 1b multiple ascending dose (MAD) clinical trial for RGLS8429, aimed at treating Autosomal Dominant Polycystic Kidney Disease (ADPKD). This trial evaluates the drug's safety, tolerability, and pharmacokinetics in adults diagnosed with ADPKD. Topline data is anticipated by Q3 2023. The first cohort will receive RGLS8429 at a dose of 1 mg/kg every other week for three months. Following safety reviews, a second cohort will commence with a 2 mg/kg dosing regime. RGLS8429, designed to inhibit miR-17, has shown promising preclinical results, indicating improvements in kidney function and other disease metrics. ADPKD affects roughly 160,000 people in the U.S. and 4 to 7 million globally, making this research particularly significant.

Positive

• Completion of enrollment in Phase 1b MAD trial for RGLS8429 is a significant milestone.
• Topline data from the trial is expected by the end of Q3 2023.
• RGLS8429 has demonstrated favorable safety and pharmacokinetic profiles in previous studies.

Negative

• N/A

SAN DIEGO, April 12, 2023 /PRNewswire/ -- Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), today announced it has completed enrollment in the first cohort of patients in the Phase 1b MAD study of RGSL8429 for the treatment of ADPKD.

"Completion of enrollment in the first cohort of the Phase 1b MAD study is an important milestone in the development of this novel treatment for ADPKD," said Jay Hagan, CEO of Regulus. "Topline data from the first cohort are expected to be available around the end of the third quarter of this year."

The Phase 1b MAD study is a double-blind, placebo-controlled trial evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics (PK/PD) of RGLS8429 in adult patients with ADPKD. The study will evaluate the safety and PK/PD of RGLS8429 treatment across three different dose levels, including measuring changes in polycystins, height-adjusted total kidney volume (htTKV), and overall kidney function. The first cohort is being dosed at 1 mg/kg of RGLS8429 or placebo every other week for three months. The Company expects to begin dosing the second cohort following a review of all available cohort 1 safety data, which is planned for May 2023. Cohort 2 patients will receive 2 mg/kg of RGLS8429 or placebo every other week for three months.

More information about the MAD clinical trial is available at clinicaltrials.gov (NCT05521191).

About ADPKD
Autosomal Dominant Polycystic Kidney Disease (ADPKD), caused by mutations in the PKD1 or PKD2 genes, is among the most common human monogenic disorders and a leading cause of end-stage renal disease. The disease is characterized by the development of multiple fluid filled cysts primarily in the kidneys, and to a lesser extent in the liver and other organs. Excessive kidney cyst cell proliferation, a central pathological feature, ultimately leads to end-stage renal disease in approximately 50% of ADPKD patients by age 60. Approximately 160,000 individuals are diagnosed with the disease in the United States alone, with an estimated global prevalence of 4 to 7 million.

About RGLS8429
RGLS8429 is a novel, next generation oligonucleotide for the treatment of ADPKD designed to inhibit miR-17 and to preferentially target the kidney. Administration of RGLS8429 has shown robust data in preclinical models, where clear improvements in kidney function, size, and other measures of disease severity have been demonstrated along with a superior pharmacologic profile in preclinical studies compared to Regulus' first-generation compound, RGLS4326. Regulus announced completion of the Phase 1 SAD study in September 2022 and dosed the first patient in the Phase 1b MAD study in early November. The Phase 1 SAD study demonstrated that RGLS8429 has a favorable safety and PK profile. RGLS8429 was well-tolerated with no serious adverse events reported and plasma exposure was approximately linear across the four doses tested and is similar to the PK data from the first-generation compound.

About Regulus
Regulus Therapeutics Inc. (Nasdaq: RGLS) is a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs. Regulus has leveraged its oligonucleotide drug discovery and development expertise to develop a pipeline complemented by a rich intellectual property estate in the microRNA field. Regulus maintains its corporate headquarters in San Diego, CA.

Forward-Looking Statements
Statements contained in this presentation regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements associated with the Company's RGLS8429 program, including the expected timing for initiating clinical studies, the expected timing for reporting topline data, and the timing and future occurrence of other preclinical and clinical activities. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "intends," "will," "goal," "potential" and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Regulus' current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics and in the endeavor of building a business around such drugs, and the risk additional toxicology data may be negative. In addition, while Regulus expects the COVID-19 pandemic to adversely affect its business operations and financial results, the extent of the impact on Regulus' ability to achieve its preclinical and clinical development objectives and the value of and market for its common stock, will depend on future developments that are highly uncertain and cannot be predicted with confidence at this time, such as the ultimate duration of the pandemic, travel restrictions, quarantines, social distancing and business closure requirements in the U.S. and in other countries, and the effectiveness of actions taken globally to contain and treat the disease. These and other risks are described in additional detail in Regulus' filings with the Securities and Exchange Commission, including under the "Risk Factors" heading of Regulus' most recently filed annual report on Form 10-K. All forward-looking statements contained in this press release speak only as of the date on which they were made. Regulus undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

 

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SOURCE Regulus Therapeutics Inc.

FAQ

What is the focus of the Phase 1b MAD study for RGLS8429?

The study focuses on evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of RGLS8429 in adults with ADPKD.

When can we expect topline data from the RGLS8429 trial?

Topline data from the first cohort of the trial is expected to be available by the end of Q3 2023.

What is Autosomal Dominant Polycystic Kidney Disease (ADPKD)?

ADPKD is a common genetic disorder characterized by the growth of numerous cysts in the kidneys, potentially leading to kidney failure.

How many patients are affected by ADPKD in the U.S.?

Approximately 160,000 individuals are diagnosed with ADPKD in the United States.

What are the dosing schedules for the RGLS8429 trial?

The first cohort receives RGLS8429 at a dose of 1 mg/kg every other week for three months, while the second cohort will receive 2 mg/kg following safety review.