Arrowhead Pharmaceuticals Files for Regulatory Clearance to Begin Phase 1 Study of ARO-APOC3 for Treatment of Hypertriglyceridemia

Chris Anzalone, Ph.D., president and CEO of Arrowhead Pharmaceuticals, said: “ARO-APOC3 will be our fifth product candidate leveraging the Targeted RNAi Molecule, or TRiM^TM, platform to enter clinical studies. Addressing hypertriglyceridemia through reduction of liver-produced apoC-III offers multiple potential development opportunities, including both orphan indications, such as FCS, and large market indications. In AROAPOC31001, our first-in-human study, we intend to evaluate ARO-APOC3 in healthy volunteers and various patient populations with elevated triglycerides, which will help inform our strategy around the ideal development and regulatory paths to pursue.”

Pending approval, Arrowhead intends to proceed with AROAPOC31001 (NCT03783377), a Phase 1 single and multiple dose-escalating study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of ARO-APOC3 in adult healthy volunteers, hypertriglyceridemic patients, and patients with familial chylomicronemia syndrome (FCS). The study is designed to enroll up to 63 subjects.

The application for approval of the clinical trial is being submitted to a local Ethics Committee and to the New Zealand Medicines and Medical Devices Safety Authority for review by the Standing Committee on Therapeutic Trials.

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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Source: Arrowhead Pharmaceuticals, Inc.

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