Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that it filed a regulatory submission to begin a Phase 1/2 study of ARO-HBV, which is being developed as a potentially curative therapy for patients with chronic hepatitis B virus (HBV) infection. Pending approval, Arrowhead intends to proceed with AROHBV1001, a Phase 1/2 single-dose escalation study to evaluate the safety, tolerability, and pharmacokinetic effects of ARO-HBV in healthy adult volunteers, as well as a multiple-dose escalation study to evaluate the safety, tolerability, and pharmacodynamic effects in HBV patients.

Chris Anzalone, Ph.D., president and CEO of Arrowhead Pharmaceuticals, said: "ARO-HBV is designed to silence all HBV gene products, including transcripts from both cccDNA and integrated DNA. We believe ARO-HBV, as a key component of combination therapies, may allow the body's natural immune defenses to control the virus and lead to a functional cure. The clinical data that we recently presented at HEP DART 2017 indicate that our prior generation compound, ARC-520, achieved a Sustained Host Response in half of the previously treatment-naïve patients chronically treated in that study, which we think bodes very well for ARO-HBV. The regulatory filing announced today is substantially ahead of schedule and we are excited to move into the clinic with multiple new compounds that leverage our Targeted RNAi Molecule, or TRiM TM, platform during 2018."

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.

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Safe Harbor Statement under the Private Securities Litigation Reform Act:

This news release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. These statements are based upon our current expectations and speak only as of the date hereof. Our actual results may differ materially and adversely from those expressed in any forward-looking statements as a result of various factors and uncertainties, including the safety and efficacy of our product candidates, the duration and impact of regulatory delays in our clinical programs, our ability to finance our operations, the future success of our scientific studies, our ability to successfully develop drug candidates, the timing for starting and completing clinical trials, rapid technological change in our markets, and the enforcement of our intellectual property rights. Our most recent Annual Report on Form 10-K and subsequent Quarterly Reports on Form 10-Q discuss some of the important risk factors that may affect our business, results of operations and financial condition. We assume no obligation to update or revise forward-looking statements to reflect new events or circumstances.

Source: Arrowhead Pharmaceuticals, Inc.

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