RARE Daily

Arrowhead Raises $450 Million to Advance RNAi-based Therapeutics Pipeline

January 8, 2024

Rare Daily Staff

Arrowhead Pharmaceuticals raised $450 million in a public offering to advance its RNAi-based genetic medicines.

The company sold 15.8 million shares of its common stock at $28.50 per share, for gross proceeds of approximately $450.0 million, before deducting underwriting discounts, commissions and other offering expenses payable by the company.

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.

The company’s lead therapeutic candidate, ARO-APOC3, is being investigated in the phase 3 PALISADE clinical study for reducing triglycerides in adult patients with familial chylomicronemia syndrome (FCS), a rare genetic disorder that causes severely elevated triglyceride levels, which can result in acute and potentially fatal pancreatitis. There are currently no FDA approved therapies to treat FCS.

ARO-APOC3 is an investigational RNAi therapeutic targeting apolipoprotein C-III (APOC3) is also being developed as a treatment for patients with severe hypertriglyceridemia (SHTG), and mixed dyslipidemia (MD). ARO-APOC3 was previously granted Orphan Drug and Fast Track designations by the U.S. Food and Drug Administration and Orphan Drug designation by the European Union.

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