Targeting Autosomal Dominant Polycystic Kidney Disease With Novel Approaches

Approximately 160,000 individuals in the United States are diagnosed with polycystic kidney disease, and the global prevalence of the condition is estimated at between 4 million and 7 million people.

The disease, formally known as autosomal dominant polycystic kidney disease (ADPKD), 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 the age of 60, and the condition is seen as a leading inheritable cause of end-stage renal disease requiring dialysis or transplant to survive.

There is currently no known cure for the disease itself and there is only one approved drug in the world for treating adults with APDKD, and one San Diego-based pharmaceutical company reports developing a potential new approach to add to the mix. 

Showing Promise?

Regulus Therapeutics Inc. RGLS is developing RGLS8429, a novel oligonucleotide designed to preferentially target the kidney. Like its first-generation compound, the drug showed what the company called “clear improvements in kidney function, size and other measures of disease severity” in preclinical studies of the disease.

Regulus completed a Phase 1a single ascending dose (SAD) clinical trial of RGLS8429 with positive topline safety and pharmacokinetic (PK) data. This led to the initiation of a Phase 1b multiple ascending dose (MAD) trial of RGLS8429 in patients with ADPKD.

In November 2022 Regulus announced dosing of the first patient in the Phase 1b trial. Topline data from the first cohort of patients is expected mid-2023. 

“The safety and PK data generated in our Phase 1a trial are highly encouraging and allows us the flexibility to use a range of dose levels as we explore the safety and efficacy of RGLS8429 in the Phase 1b MAD study,” Jay Hagan, president and CEO of Regulus Therapeutics, said of the initial trial outcomes.

In describing the safety data from the Phase 1a trial which involved 32 subjects, there were nine adverse events, all of which were described as mild except one involving a sinus infection, which was graded moderate in severity. The first planned dose level to be tested in ADPKD patients in the Phase 1b MAD trial is 1 mg/kg dosed every other week for three months with topline data from the first cohort of patients expected in the first half of 2023.

Other Pharmas Looking Too

Currently the only drug approved to treat the disease is tolvaptan, which is branded under the name Jynarque and manufactured by the Japanese group Otsuka Co. Holdings Ltd. OTSKY. That drug was approved for use in the U.S. in 2018 but comes with a black-box warning for potential liver toxicity.  

There may be as many as 22 companies involved in researching ADPKD treatments, according to a report from Research and Markets, including Abbott Laboratories ABT

Regulus Therapeutics is a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs implicated in genetic kidney disease with their lead program targeting miR-17 for the treatment of ADPKD. 

Click here for more information on Regulus Therapeutics.

Featured photo by Robina Weermeijer on Unsplash

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