Benzinga - Arrowhead Pharmaceuticals Inc (NASDAQ: ARWR) shares are down 20% Monday after the company announced topline results from the SEQUOIA Phase 2 study of fazirsiran in patients with alpha-1 antitrypsin deficiency-associated liver disease.
What To Know: Fazirsiran is a potential first-in-class investigational RNA interference therapy designed to reduce the production of mutant alpha-1 antitrypsin protein as a potential treatment for rare genetic liver disease.
Patients receiving 25 mg, 100 mg, or 200 mg of fazirsiran demonstrated a dose dependent mean reduction in serum mutant alpha-1 antitrypsin protein concentration at week 48 of 74%, 89%, and 94%, respectively. All three doses led to a dramatic reduction in total liver Z-AAT with a median reduction of 94%.
"Currently there is no treatment for liver disease from alpha-1 antitrypsin deficiency. The results presented today from the SEQUOIA study are highly encouraging to physicians and patients in need of a safe and effective therapy for this rare genetic condition," said Virginia Clark, M.D., M.S., University of Florida, Division of Gastroenterology, Hepatology, and Nutrition.
This month, the company plans to initiate a randomized, double-blind, placebo-controlled, Phase 3 study to evaluate the efficacy and safety of fazirsiran in the treatment of alpha-1 antitrypsin deficiency–associated liver disease with METAVIR stage F2 to F4 fibrosis.
Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them.
ARWR Price Action: Arrowhead has a 52-week high of $61.25 and a 52-week low of $26.81.
The stock was down 20.1% at $29.88 at time of publication, according to Benzinga Pro.
Photo: Gerd Altmann from Pixabay.
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