Arrowhead Pharma, Horizon Thera partner on RNA gout drug

June 21, 2021

Arrowhead Pharmaceuticals and Horizon Therapeutics announced a global collaboration and license agreement for Arrowhead's investigational RNA interference therapeutic being developed as a potential treatment for people with uncontrolled gout — a deal that could eventually be worth over $700 million.

The deal centers around California-based Arrowhead's ARO-XDH, a previously undisclosed discovery-stage RNA interference (RNAi) therapeutic that is designed to target the primary source of serum uric acid. High levels of this acid, found in gout patients, can lead to serious long-term or even permanent damage to the bones, joints and organs. While approximately a third of gout patients in the U.S. are treated with oral urate-lowering therapies, a meaningful portion of these patients do not respond to treatment and continue to experience painful and debilitating gout symptoms.

The deal with add to Horizon's gout treatment arsenal. The drugmaker's best-selling Krystexxa, approved in 2010 to treat chronic gout in adults refractory to conventional therapy, is expected to achieve peak net sales of $1 billion.

Under the terms of the deal, Arrowhead will conduct all activities through preclinical stages of development of the siRNA therapeutic. Dubin-based Horizon will receive a worldwide exclusive license to the therapeutic and will be responsible for clinical development and commercialization. Arrowhead will receive $40 million as an upfront payment from Horizon and is eligible to receive up to $660 million in potential development, regulatory and commercial milestones, and is further eligible to receive royalties on net product sales.

Read the press release