Pharma Focus Asia

Arrowhead Pharmaceuticals and Horizon Therapeutics Announce Collaboration to Develop RNAi Therapeutic for Uncontrolled Gout

Tuesday, June 22, 2021

Arrowhead Pharmaceuticals Inc. and Horizon Therapeutics plc today announced a global collaboration and license agreement for ARO-XDH, a previously undisclosed discovery-stage investigational RNA interference (RNAi) therapeutic being developed by Arrowhead as a potential treatment for people with uncontrolled gout.

Gout is a serious and painful form of arthritis that is caused by excess uric acid in the blood. In the United States, there are more than nine million gout patients and approximately a third of those patients are treated with oral urate-lowering therapies. However, a meaningful portion of treated patients do not respond sufficiently to treatment and therefore continue to experience painful and debilitating gout symptoms. XDH represents a clinically validated target that is the primary source of serum uric acid (sUA). High levels of sUA, if left untreated or undertreated, can potentially lead to serious long-term or even permanent damage to the bones, joints and organs.

Under the terms of the agreement, Arrowhead will conduct all activities through preclinical stages of development of the siRNA therapeutic. Horizon will receive a worldwide exclusive license to the therapeutic and will be wholly 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 in the low- to mid-teens range on net product sales.

“We believe Horizon’s expertise in the clinical development and commercialization of therapies for gout makes them an ideal partner. We look forward to working closely with Horizon as we advance this potential new therapy for patients in need,” said Christopher Anzalone, Ph.D., Arrowhead’s president and CEO. “This collaboration with Horizon also represents further expansion of our rapidly growing pipeline of investigational RNAi therapeutics that utilize Arrowhead’s proprietary Targeted RNAi Molecule (TRiMTM) platform.”

“As a commercial and clinical leader in gout, we understand the disease and its impact, and that there is still a tremendous unmet treatment need,” said Tim Walbert, chairman, president and CEO, Horizon. “The collaboration with Arrowhead adds an additional program to our research and development focus to discover and develop new, innovative medicines that continue to improve gout treatment across the continuum of care.”

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