Arrowhead Pharmaceuticals Regains Full Rights to NASH-Targeting RNAi Candidate ARO-PNPLA3 from Johnson & Johnson

On February 15, Arrowhead Pharmaceuticals announced that it has regained full rights to ARO-PNPLA3 (formerly known as JNJ-75220795). Arrowhead stated that following a strategic R&D portfolio review, Johnson & Johnson decided to return all rights to ARO-PNPLA3 to Arrowhead.

ARO-PNPLA3 is an investigational RNA interference (RNAi) therapy developed using Arrowhead's proprietary TRiMTM platform. It aims to reduce the liver expression of patatin-like phospholipase domain-containing protein 3 (PNPLA3) for the treatment of non-alcoholic steatohepatitis (NASH). PNPLA3, as a driver of liver fat accumulation and damage in patients carrying the common I148M mutation, has strong genetic and preclinical validation.

Due to the lack of FDA-approved treatments, NASH remains an area of serious unmet medical need. The I148M genetic variant in the PNPLA3 gene is associated with the underlying pathophysiology and is a known risk factor for hepatic steatosis, steatohepatitis, elevated plasma liver enzyme levels, liver fibrosis, and cirrhosis. The ongoing Phase I clinical study (NCT04844450) is a double-blind, placebo-controlled, randomized, single- and multiple-ascending dose study designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of subcutaneous administration of ARO-PNPLA3. Preliminary data show that, following a single dose, there was an average reduction of up to 40% in liver fat in homozygous patients with the I148M mutation in a dose-dependent manner, with no clinically significant changes or trends in any safety parameters, and no serious or severe adverse events (AEs) or treatment discontinuations due to adverse events were reported.

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