Arrowhead Pharmaceuticals Files for Regulatory Clearance to Begin Phase 1 Study of ARO-AAT

PASADENA, CA, USA I December 20, 2017 I Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that it filed a regulatory submission to begin a first-in-human study of ARO-AAT, a second-generation investigational medicine for the treatment of alpha-1 liver disease that leverages Arrowhead's subcutaneously administered Targeted RNAi Molecule (TRiMTM) technology. Pending approval, Arrowhead intends to proceed with AROAAT1001, a Phase 1 single and multiple ascending dose study to evaluate the safety, tolerability, pharmacokinetics, and effect of ARO-AAT on serum alpha-1 antitrypsin levels in healthy adult volunteers.

Chris Anzalone, Ph.D., president and CEO of Arrowhead Pharmaceuticals, said: "ARO-AAT is our second candidate targeting alpha-1 liver disease and represents the first of five planned regulatory submissions over the next twelve months for product candidates built on our TRiMTM platform. We have moved very rapidly with this program and are thrilled to now submit the regulatory filing ahead of schedule."

The application for approval of the clinical trial was submitted to the New Zealand Medicines and Medical Devices Safety Authority (MEDSAFE) for review by the Standing Committee on Therapeutic Trials (SCOTT) as well as the local Ethics Committee.

About Arrowhead Pharmaceuticals

Arrowhead Pharmaceuticals 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 mechanism to induce rapid, deep, and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead's RNAi-based therapeutics leverage this natural pathway of gene silencing.

SOURCE: Arrowhead Pharmaceuticals

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