Arrowhead Pharmaceuticals Files IND to Begin Phase 2b Study of ARO-APOC3 in Patients with Severe Hypertriglyceridemia

PASADENA, CA, USA I March 01, 2021 I Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that it has submitted an Investigational New Drug Application (IND) to the U.S. Food and Drug Administration (FDA) for ARO-APOC3, the company’s investigational RNA interference (RNAi) therapeutic being developed as a treatment for patients with hypertriglyceridemia.

Javier San Martin, M.D., chief medical officer at Arrowhead, said: “There is a clear need for new therapies that can reduce triglycerides across multiple patient populations, including those with the most severe elevations that can lead to recurrent bouts of pancreatitis. In the initial clinical study of ARO-APOC3, which we presented at the American Heart Association meeting last fall, triglycerides were lowered by up to 92% after just two doses. Based on these highly promising results, we intend to conduct Phase 2b and Phase 3 clinical studies across a wide spectrum of patient populations, pending review by regulators.”

Following the FDA’s review of the IND, the company intends to initiate a Phase 2b dose-finding clinical study (ARO-APOC3-2001) in patients with severe hypertriglyceridemia (sHTG). Additional clinical studies planned in 2021 include a Phase 2b dose-finding clinical study in patients with mixed dyslipidemia and a Phase 3 clinical study in patients with familial chylomicronemia syndrome (FCS).

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.

For more information, please visit www.arrowheadpharma.com, or follow us on Twitter @ArrowheadPharma. To be added to the Company's email list and receive news directly, please visit http://ir.arrowheadpharma.com/email-alerts.

SOURCE: Arrowhead Pharmaceuticals

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