Horizon Bets $700 Million on Arrowhead's Gout RNAi Drug

Horizon_Artur Widak/NurPhoto via Getty

Artur Widak/NurPhoto via Getty Images

Arrowhead Pharmaceuticals and Horizon Therapeutics announced Monday they have both entered into a research and development collaboration to advance an RNA interference (RNAi) therapeutic for uncontrolled gout.

Under terms of the collaboration, Arrowhead Pharmaceuticals is set to receive an upfront payment of $40 million. In exchange, Horizon Therapeutics will receive a global exclusive license for a short interfering RNA (siRNA) treatment targeting xanthine dehydrogenase (XDH) currently being investigated for the treatment of uncontrolled gout. 

The agreement sees Arrowhead Pharmaceuticals conducting its activities through preclinical development stages of the investigational siRNA therapy. In turn, Horizon Therapeutics will have the sole responsibility for the clinical development and commercialization of the medicine. 

In addition to the upfront $40 million payment, Arrowhead Pharmaceuticals will also be eligible for up to $660 million in future development, regulatory and commercial milestones. Additionally, the company should receive royalties on net product sales in the low- to mid-teens range.

“We believe Horizon’s expertise in the clinical development and commercialization of therapies for gout makes them an ideal partner,” said Arrowhead’s President and Chief Executive Officer, Christopher Anzalone, Ph.D., in a statement. “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.”

The collaboration comes when a meaningful proportion of patients with gout in the United States continue to report debilitating symptoms, even despite the use of currently approved therapies. In addition, the XDH domain represents a clinically validated target, according to the two companies, as this is the primary source of serum uric acid. Left untreated or undertreated, high serum uric acid levels can cause long-term or permanent bone, joint, and organ damage.

“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,” according to Horizon’s President and CEO, Tim Walbert, who also serves as Chairman of the company. “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.”

Arrowhead enters this new collaboration under an existing partnership with Johnson & Johnson for ARO-JNJ1, an investigational RNAi candidate for hepatitis B infection. In May, Arrowhead earned a $10 million option exercise fee after J&J announced its intent to exercise its option right for the candidate. Exercising this option, J&J received all licensing, development and commercialization rights for ARO-JNJ1.

Horizon Therapeutics also previously agreed to a collaboration with privately-held clinical stage biotech company HemoShear Therapeutics. In April, HemoShear announced a second drug target for gout under the partnership, earning the company two milestone payments. Within two years, HemoShear has already produced and validated two novel targets for gout. The company is eligible to receive more than $500 million in milestone payments, in addition to royalties on future product sales.

 

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