Current Advances

Regulus announces first demonstration of proof-of-concept in patients with hepatitis C virus (HCV)

Regulus reported today that a single 2 mg/kg dose of RG-101, an anti-miR targeting microRNA-122 ("miR-122") in patients with hepatitis C virus (HCV), demonstrated a mean viral load reduction of 4.1 log10 at Day 29 in 14 patients with consistent effects across multiple HCV genotypes.

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RG-101 is the first drug to enter clinical development at Regulus, and the first drug to enter clinical development that incorporates Isis' antisense technology to target a microRNA. RG-101 was discovered using the Isis antisense technology platform and incorporates a galNac-conjugate, which confers the ability to distribute more drug to its target tissue, the liver.

RG-101 is the most advanced program in Regulus' pipeline and the data reported today is the first demonstration of activity in patients with HCV. The target of RG-101, miR-122, is the most abundant microRNA in the liver. miR-122 is a critical host factor for survival and replication of all known HCV genotypes. The duration of action observed for RG-101 supports the potential for a once-a-month dosing regimen.

Regulus was cofounded by Isis and Alnylam to focus on the discovery, development and commercialization of microRNA-targeted therapeutics. Because microRNAs may act as master regulators of the genome, affecting the expression of microRNAs provides a broad therapeutic opportunity. Regulus is addressing therapeutic opportunities that arise from alterations in microRNA expression.

Regulus' initial public offering was in 2012. Isis is currently the top Regulus shareholder.

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