Arrowhead Pharmaceuticals has signed a worldwide licensing and collaboration agreement with Sarepta Therapeutics for the development of its clinical and preclinical programmes.
The agreement covers multiple clinical and preclinical programmes in rare, genetic diseases of the muscle, central nervous system (CNS), and lungs.
Under the terms of the agreement, Arrowhead will receive $825m, including $500m cash and $325m as an equity investment priced at a 35% premium.
Arrowhead will also receive $250m in equal instalments over five years and is eligible to receive an additional $300m in near-term payments, in the next 12 months.
During the five years, Sarepta can select up to six new central nervous system (CNS) or muscle targets for which Arrowhead will perform discovery and preclinical development.
Sarepta can select up to six new targets for Arrowhead to conduct discovery and preclinical development activities in precision genetic medicine for rare diseases.
Arrowhead will leverage its unique and differentiated Targeted RNAi Molecule (TRiM) platform and develop the programmes in areas complementary to Sarepta.
In addition, Arrowhead is eligible to receive royalties on commercial sales and up to $10bn in future potential milestone payments, and tiered royalties on commercial sales.
The transaction is expected to be completed in early 2025, subject to certain customary conditions, including the expiration of the waiting period under the Hart-Scott-Rodino Act.
Upon closing, Sarepta president and CEO Doug Ingram will be appointed as a member of Arrowhead Board of Directors.
Arrowhead president and CEO Christopher Anzalone said: “We estimate that this transaction extends Arrowhead’s cash runway into 2028 and potentially through multiple new drug launches, including wholly owned and partnered programmes.
“We now turn our focus as a company to launching investigational plozasiran for the treatment of familial chylomicronemia syndrome potentially in 2025, pending FDA review and approval, which would be our first commercial product.
“We are also prioritising further development of additional pipeline programs which would be complementary to plozasiran from a clinical, regulatory, and commercial perspective.”
The license and collaboration agreement includes several clinical and preclinical-stage programmes, the discovery of new drugs and their manufacturing.
The clinical-stage programmes include ARO-DUX4 for muscular dystrophy type 1, ARO-DM1 for myotonic dystrophy, ARO-MMP7 for matrix metalloproteinase and ARO-ATXN2 for spinocerebellar ataxia 2 (SCA2).
The preclinical programmes include ARO-HTT for Huntington’s disease patients, ARO-ATXN1 for with spinocerebellar ataxia 1 (SCA1) patients, and ARO-ATXN3 for with spinocerebellar ataxia 3 (SCA3).
Arrowhead will manufacture clinical drug supply for all programs emerging from the license and collaboration, and commercial drug product for the four pre-clinical programmes.
Gibson, Dunn & Crutcher served as a legal advisor to Arrowhead on the transaction.
Anzalone added: “The agreement marks the start of a synergistic relationship with Arrowhead’s leading siRNA technology and Sarepta’s proven success in bringing rare disease treatments to patients.
“Together, we will expedite the development of Arrowhead’s promising scientific approach and bring best-in-class treatments to patients with devastating rare diseases where treatment options are limited or do not exist.
“I am honoured to serve on the Arrowhead Board of Directors and to help Arrowhead advance its extraordinary science for the benefit of patients around the world.”
