Update on New Liver-related Alpha-1 Therapeutic Development

Published on April 23rd, 2018

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Promising results for three drugs for the treatment of three rare liver diseases were presented at the International Liver Congress 2018, which took place April 11-15 at the Paris Expo Porte de Versailles, Paris, France.

Every year in April, scientific and medical experts from a broad range of fields including hepatology, gastroenterology, internal medicine, cell biology, transplant surgery, infectious diseases, microbiology and virology, pharmacology, pathology and radiology, and imaging come together from around the world to learn about the latest in liver research.

Specialists share recent data, present studies and findings, and discuss the hottest topics on liver disease. The annual Congress attracts around 10,000 delegates and 250 media representatives from all over the world making this a truly international networking opportunity.

Preliminary findings demonstrated positive preclinical safety and efficacy of an ARO-AAT in Alpha-1 Antitrypsin Deficiency (Alpha-1) – pointing to the developing potential of this new therapeutic strategy in patients with few treatment options.

Alpha-1 is an autosomal, co-dominant genetic disorder in which the PiZ mutation results in the misfolded protein (Z-AAT) that accumulates in hepatocytes and can lead to fibrosis, cirrhosis and hepatocellular carcinoma.

The only current treatment option for Alpha-1-related liver disease is liver transplant. ARO-AAT is a second-generation, subcutaneously administered RNAi therapeutic that replaces ARC-AAT, a first-generation intravenously administered RNAi therapeutic that previously demonstrated proof of concept in the PiZ mouse model expressing human Z-AAT, and achieved deep knockdown in healthy volunteers and patients.

“We are very pleased to see Arrowhead’s hard work and persistence yielding results. The efficacy of ARO-AAT in animal models is encouraging, and we look forward to the results of the Phase 1 trials,” expressed Jean-Marc Quach, president and CEO of The Alpha-1 Project (TAP), a wholly-owned for profit subsidiary of the Alpha-1 Foundation (A1F) singularly focused on providing critical funding and advice to speed the commercialization of therapies for the elimination of Chronic Obstructive Pulmonary Disease (COPD) and liver disease caused by Alpha-1.

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Also at the International Liver Congress in Paris, it was announced that sebelipase alfa, approved for treatment of lysosomal acid lipase (LAL) deficiency in 2015, showed sustained improvements and long-term tolerability in a diverse patient population. Other findings were also positive with givosiran, which substantially reduced the annualized attack rate in patients with acute intermittent porphyria (AIP).

“Rare diseases are a greater challenge than you might expect, as apart from the difficulties in reaching a full diagnosis, there are often no effective treatments available,” said Prof. Marco Marzioni from the University Hospital of Ancona, Italy, and EASL Governing Board Member.

SOURCES: Drug, Discovery and DevelopmentInternational Liver Congress 2018 the Alpha-1 Foundation

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