Regulus Back on Track with RG-012 Clinical Development for Alport Syndrome

Regulus Therapeutics has completed dosing of its Alport syndrome drug candidate RG-012 in a Phase 1 trial testing several ascending doses of the treatment in healthy volunteers.

The treatment was found safe and well-tolerated, and no serious adverse events were seen during the trial.

The company also announced it had selected a dose of RG-012 for the planned Phase 2 HERA study (NCT02855268).

Preliminary data from the Phase 1 study also suggested that the behavior of the drug in the body — measurements researchers call pharmacokinetics — did not deviate from expectations. The concentration of RG-012 in the blood increased with increasing dose, researchers said.

The drug, developed in partnership with Genzyme, blocks a microRNA called miR-21, which researchers believe is involved in the development of kidney fibrosis and tissue damage.

Preclinical studies suggested the drug was effective in animal models of Alport syndrome and the company plans to test the treatment in Alport patients for the first time in the HERA study.

Since European health authorities expressed concern over the initial design of the HERA trial, Regulus launched the Phase 1 multiple ascending dose study in healthy volunteers to gather additional data before testing the drug in patients.

The company is also recruiting patients for an observational study, called ATHENA (NCT02136862), intended to study the natural course of Alport disease.

The company expects the HERA trial, as well as a kidney biopsy study, to begin mid-2017. The Phase 2 trial will include 30 Alport patients with a rapid decline in kidney function. They will be randomized to receive either RG-012 or placebo treatment for 48 weeks, after which researchers will evaluate the safety and effectiveness of the drug in patients.

The Phase 1/2 kidney biopsy study intends to further study the pharmacokinetics of RG-012 in Alport patients.

Data from the kidney biopsy study is anticipated by the end of 2017, and interim data from the HERA trial is expected by mid-2018.

“Our priorities remain on our most promising programs allowing us to achieve anticipated milestones,” Jay Hagan, recently appointed president and CEO of Regulus, said in a press release.

“We continue to advance our clinical and preclinical pipeline, and importantly, are on track to commence the Phase 2 HERA and renal biopsy studies for RG-012 as planned.”