Arrowhead Pharmaceuticals Stock Falls After Early-Stage Data From RNA-Based Therapeutic For Kidney Diseases

On Monday, Arrowhead Pharmaceuticals, Inc. (NASDAQ:ARWR) released topline results from Part 2 of a Phase 1/2 study of ARO-C3, the company’s investigational RNA interference (RNAi) therapeutic designed to reduce liver production of complement component 3 (C3) as a potential therapy for various complement-mediated diseases.

ARO-C3 is designed to reduce hepatocyte production of complement C3 as a potential treatment for various complement-mediated renal diseases.

The dysregulated activity of the complement system can contribute to tissue injury and the progression of the disease. By silencing C3, investigational ARO-C3 has the potential to treat complement-mediated renal diseases by modulating the activation of the complement cascade.

Also Read: Veren Soars On Whitecap Merger Deal: Details

The company plans to present additional results at a medical meeting in 2025.

Select Phase 1/2 Study Results

Pharmacodynamic effects: Maximum mean reduction in C3 of 89% and mean sustained reduction of greater than 87% from baseline through week 24. Maximum mean reduction in serum AH50 (alternative pathway hemolytic assay) of 85% and mean sustained reduction greater than 76%. Maximum mean reduction in Wieslab AP (alternative pathway) of 100% and mean sustained reduction greater than 89%. Duration of effect supportive of once every three months or less frequent subcutaneous dosing in later-stage studies Effects on proteinuria (too much protein in the urine): The mean reduction in spot UPCR was 41%, and the maximum individual reduction was 89%.

In January, the U.S. Food and Drug Administration accepted Arrowhead Pharmaceuticals’ New Drug Application for investigational plozasiran for familial chylomicronemia syndrome, a severe and rare genetic disease. The FDA provided a Prescription Drug User Fee Act (PDUFA) action date of November 18, 2025, and indicated it is not currently planning to hold an advisory committee meeting.

In February, Arrowhead Pharmaceuticals closed a global licensing and collaboration agreement with Sarepta Therapeutics Inc. Arrowhead receives a $500 million upfront payment and $325 million through Sarepta stock.

Price Action: ARWR stock is down 8.72% at $15.49 at the last check Monday.

Read Next:

Pfizer’s COVID-19 Shot Partner BioNTech Reports Q4 Profit, But 2025 Guidance Falls Short On Expectations

Photo via Shutterstock.

Up Next: Transform your trading with Benzinga Edge's one-of-a-kind market trade ideas and tools. Click now to access unique insights that can set you ahead in today's competitive market.

Get the latest stock analysis from Benzinga?

Story Continues

ARROWHEAD PHARMA (ARWR): Free Stock Analysis Report

This article Arrowhead Pharmaceuticals Stock Falls After Early-Stage Data From RNA-Based Therapeutic For Kidney Diseases originally appeared on Benzinga.com

© 2025 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.

View Comments