Local Pharmaceutical Developer, Hoping For Another Success, Applies to FDA for Trial Testing of Gene-Silencing Drug

Published : Tuesday, March 19, 2019 | 5:30 AM

Pasadena-based Arrowhead Pharmaceuticals, just five months after it announced a license and collaboration agreement a Johnson & Johnson with a potential value of over $3.7 billion, said Monday it has applied to the U.S. Food and Drug Administration (FDA) to test its potentially curative gene-silencing drug for hepatitis-B infection.

Specifically, the company announced its submission of an Investigational New Drug application for the subcutaneously administered RNA interference (RNAi) therapeutic ARO-AAT.

“We look forward to initiating this trial and moving closer to potentially providing patients, physicians, and the alpha-1 community with a much-needed therapy for alpha-1 liver disease,” said Dr. Bruce Given, Arrowhead’s chief operating officer and head of research and development.

Arrowhead Pharmaceuticals is developing ARO-AAT as a treatment for a rare genetic liver disease associated with alpha-1 antitrypsin deficiency (AATD), a company announcement Monday said.

AATD, characterized by the lack of a protein made by the liver that’s released into the bloodstream, is a genetic disease that can affect the liver or lung.

Pending regulatory review, Arrowhead Pharmaceuticals said it intends to initiate the adaptive design Phase 2/3 study of ARO-AAT in patients with AATD-associated liver disease.

The primary objectives of the study are to evaluate the drug’s safety and pharmacodynamic dose response and to evaluate its efficacy.

Arrowhead intends to initiate the study at various sites in the U.S. in the second quarter of 2019, followed by various international sites in Europe. The company plans to provide additional study details as soon as the study begins.

Arrowhead Pharmaceuticals, whose corporate offices are at 225 South Lake Avenue, Suite 1050 in Pasadena, develops medicines that treat intractable diseases by silencing the genes that cause them.

Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference (RNAi) mechanism to induce rapid, deep, and durable knockdown of target genes. The mechanism, present in living cells, inhibits the expression of a specific gene, thereby affecting the production of a specific protein – a natural pathway of gene silencing.
For more information, visit www.arrowheadpharma.com.

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