Arrowhead Pharmaceuticals Hosts R&D Day on Emerging Pipeline of Pulmonary Targeted RNAi Therapeutics
Arrowhead Pharmaceuticals (NASDAQ: ARWR) is hosting a pulmonary R&D Day in New York City to unveil its pipeline of RNA interference therapeutics targeting pulmonary diseases. Key presentations include ARO-MUC5AC, ARO-RAGE, and ARO-MMP7, aimed at treating muco-obstructive and inflammatory conditions. ARO-RAGE shows over 90% mRNA reduction in preclinical studies, while ARO-MUC5AC promises significant silencing of pathogenic expression. ARO-MMP7 targets idiopathic pulmonary fibrosis. The event will enhance confidence in upcoming clinical trials.
- ARO-RAGE shows over 90% reduction in lung RAGE mRNA and promising preclinical results.
- ARO-MUC5AC demonstrates up to 90% silencing of MUC5AC expression in preclinical studies.
- ARO-MMP7 targets key mechanisms in idiopathic pulmonary fibrosis (IPF) with positive outcomes in preclinical models.
- Previous clinical candidate ARO-ENaC faced adverse findings leading to a voluntary pause in 2021.
Presentations will begin at
Arrowhead will describe the development of investigational candidates:
- ARO-MUC5AC, designed to reduce expression of mucin 5AC (MUC5AC) as a potential treatment for various muco-obstructive pulmonary diseases
- ARO-RAGE, designed to reduce expression of the receptor for advanced glycation end products (RAGE) as a potential treatment for various obstructive inflammatory pulmonary diseases
- ARO-MMP7, Arrowhead’s newest and previously undisclosed candidate designed to the reduce expression of matrix metalloproteinase 7 (MMP7) as a potential treatment for idiopathic pulmonary fibrosis (IPF)
The company will also discuss learnings from its first pulmonary clinical candidate, ARO-ENaC, that led to enhanced candidate and platform designs that have the potential to offer improved potency, duration, and the flexibility of subcutaneous as well as inhaled delivery.
“Today we’re discussing ARO-MUC5AC, ARO-RAGE, and ARO-MMP7, three of our pulmonary candidates. These are each potentially important new investigational medicines that seek treat muco-obstructive and inflammatory lung diseases and IPF in fundamentally new ways,” said
Select R&D Day Highlights
ARO-RAGE
ARO-RAGE is an investigational medicine that aims to be a better anti-inflammatory therapy with broader anti-inflammatory effects than current biologics and with a more convenient inhaled mode of administration. Preclinical studies have yielded highly positive results. Single inhaled doses of ARO-RAGE in rats and primates led to reductions of greater than
ARO-MUC5AC
ARO-MUC5AC is the first investigational medicine to directly silence pathologic MUC5AC expression and potentially address muco-obstructive disease, characterized by mucus hypersecretion, in a fundamentally different way than current therapies. Preclinical results have shown deep silencing of up to 70
ARO-MMP7
ARO-MMP7 is Arrowhead’s previously undisclosed investigational medicine being developed as a potential treatment for IPF. MMP7 plays multiple roles in IPF pathogenesis, including promoting inflammation and aberrant epithelial repair and fibrosis. Silencing MMP7 expression in a rat IPF model reduced inflammatory cell infiltration, limited lung fibrosis, and preserved pulmonary function. Additional data on this program will be presented at the
Non-clinical Toxicology
Arrowhead’s first clinical candidate, ARO-ENaC, using the lung targeted TRiMTM platform was voluntarily paused in 2021 based on non-clinical findings showing adverse local lung effects seen in chronic rat (6-month) and primate (9-month) GLP toxicology studies. The company believes that the mechanism underlying ARO-ENaC chronic toxicology findings are consistent with lung macrophage overload, which has been widely studied for over 40 years and extensively described in the literature. Arrowhead believes that a lower cumulative dose in future chronic toxicology studies decreases the risk of adverse findings. For example, the planned 6-month rat ARO-MUC5AC and ARO-RAGE dose levels and dose intervals yield a cumulative dose well below the threshold where adverse findings were seen in the ARO-ENaC 6-month rat study.
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