Why This Interim Liver Study Is Huge for Arrowhead Pharma

By Chris Lange Updated Aug 6, 1:40PM EDT · Published Sep 16, 9:50AM EDT

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Why This Interim Liver Study Is Huge for Arrowhead Pharma — © Thinkstock

Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) has announced positive interim 24-week liver biopsy results in four subjects from its midstage study of ARO-AAT. Shares rallied on the news early Wednesday.

ARO-AAT is the firm’s second-generation investigational RNA interference (RNAi) therapeutic being developed as a treatment for the rare genetic liver disease associated with alpha-1 antitrypsin deficiency.

The results demonstrated clear evidence of a meaningful pharmacodynamic effect by ARO-AAT, leading to improvements in relevant biomarkers, including substantial reductions in intra-hepatic mutant AAT protein (Z-AAT), both Z-AAT monomer and Z-AAT polymer; improvements in liver stiffness based on FibroScan; and, a decrease in alanine aminotransferase (ALT) and gamma-glutamyl transferase (GGT), both serum biomarkers of liver injury.

After 24 weeks of treatment with ARO-AAT in the study, serum and total intra-hepatic Z-AAT decreased in all four patients by up to 93% and 95%, respectively. Three of four patients demonstrated reductions from baseline in intra-hepatic Z-AAT polymer, with a maximum reduction of 97%. All four patients showed reductions in ALT and GGT, with maximum reductions of 66% and 58%, respectively. All patients demonstrated improved transient elastography FibroScan values, with three of four patients exhibiting greater than 20% reductions.

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Management noted that these are very exciting results so far and they provide the firm with increased confidence in the potential of this program. Based on these results, Arrowhead is actively assessing its clinical and regulatory path forward, including meeting with the U.S. Food and Drug Administration and other regulatory agencies, to identify areas in which the program could potentially be streamlined and accelerated.

Arrowhead Pharmaceuticals stock traded up more than 30% early Wednesday to $44.00, in a 52-week range of $19.51 to $73.72. The consensus price target is $64.27.

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About the Author Chris Lange →

Chris Lange is a writer for 24/7 Wall St., based in Houston. He has covered financial markets over the past decade with an emphasis on healthcare, tech, and IPOs. During this time, he has published thousands of articles with insightful analysis across these complex fields. Currently, Lange's focus is on military and geopolitical topics.

Lange's work has been quoted or mentioned in Forbes, The New York Times, Business Insider, USA Today, MSN, Yahoo, The Verge, Vice, The Intelligencer, Quartz, Nasdaq, The Motley Fool, Fox Business, International Business Times, The Street, Seeking Alpha, Barron’s, Benzinga, and many other major publications.

A graduate of Southwestern University in Georgetown, Texas, Lange majored in business with a particular focus on investments. He has previous experience in the banking industry and startups.