Regulus’ RG-012 gets orphan medicinal product designation in Europe to treat alport syndrome

In July 2014, the company has secured orphan drug designation from the US Food and Drug Administration (FDA) for RG-012 to treat Alport syndrome.

Currently, the company is enrolling patients in a natural history of disease study called ATHENA to gather information about the changes in renal function over time in these patients.

Data from the ATHENA trial will provide the clinical basis for the design of a Phase II trial to monitor the therapeutic effect of RG-012 on the decline in renal function and time to end-stage renal disease in patients with Alport syndrome.

Regulus chief medical officer Paul Grint said: "We are pleased to have received orphan medicinal product designation in the European Union for RG-012, a key microRNA therapeutic program under our ‘Clinical Map Initiative’.

"Alport syndrome is a life threatening disease and patients have very limited treatment options because there is currently no approved therapy.

"We believe that RG-012 represents an opportunity to make a significant impact in the lives of patients with Alport syndrome and we look forward to advancing this program into the clinic."

As part of the ‘Clinical Map Initiative’, the company plans to start a Phase I trial evaluating RG-012 in healthy volunteers in the first half of 2015 and a Phase II proof-of-concept trial thereafter.

The company is focused on the discovery and development of new medicines targeting microRNAs, which are small RNA molecules, typically 20 to 25 nucleotides in length that do not encode proteins but instead regulate gene expression.