Regulus Therapeutics, a pharmaceutical company dedicated to the development of microRNA treatments, announced that the design of the Phase 2 clinical program to test its product RG-012 as a potential therapy for Alport syndrome has been subject to changes aimed at speeding up patient enrollment and improving result analysis so that preliminary data would be known later in 2017.
Based on these changes, the HERA Phase 2 trial (NCT02855268), which is evaluating the safety and effectiveness of RG-012 in patients with Alport syndrome, will now include 40 patients (10 more than originally planned) to strengthen result analyses. Patients will be randomized to receive either RG-012 or placebo treatment once every other week for 48 weeks.
Researchers will then assess the safety and efficacy of RG-012, and will also conduct a Phase 1/2 kidney biopsy study to assess the drug’s pharmacokinetics (how it is processed in the body), its targets and its effects in genetic biomarkers in Alport syndrome patients.
Results from the biopsy study are expected by the end of 2017 and results from the HERA trial are expected to be released in mid-2018.
“MicroRNA therapeutics have the potential to become an important new class of drugs with broad therapeutic application,” Jay Hagan, Regulus Therapeutics’ president and CEO, said in a press release issued by the California-based company. “Regulus’ focus will be in diseases with significant unmet medical need in organs to which we have been able to preferentially deliver oligonucleotide therapeutics effectively, such as the liver and kidney.”
RG-012 acts by inhibiting a microRNA called miR-21, which researchers believe participates in the development of kidney fibrosis and tissue damage.
Human DNA contains the information for the production of proteins necessary for cell function stored in the genes, but it also includes information for the expression of molecules that regulate protein synthesis, such as microRNAs.
MicroRNAs, or simply miRs, are able to bind to an mRNA molecule (messenger RNA; they carry coding information for the production of the corresponding protein) and interfere with the production of that protein. One miR can bind to many different mRNA molecules, which makes miRs powerful regulators of gene expression.