Arrowhead Pharmaceuticals Partners with Sarepta to Advance Genetic Medicine and Boost Pipeline

Arrowhead Pharmaceuticals and Sarepta Therapeutics Forge Strategic Partnership in Genetic Medicine

Arrowhead Pharmaceuticals, Inc. has entered into a transformative global licensing and collaboration agreement with Sarepta Therapeutics, Inc. This partnership is designed to enhance Sarepta's pipeline in precision genetic medicine, focusing on rare diseases that affect muscles, the central nervous system (CNS), and lungs. Under the terms of the agreement, Sarepta obtains exclusive global rights to a range of clinical, preclinical, and discovery-stage programs that leverage Arrowhead’s proprietary Targeted RNAi Molecule (TRiM™) platform for effective siRNA delivery and gene suppression. The collaboration is expected to significantly boost Sarepta's mid- and early-stage therapies, further establishing its leadership in genetic conditions such as Duchenne muscular dystrophy and limb-girdle muscular dystrophies.

Key clinical-stage programs included in this collaboration are ARO-DUX4, targeting facioscapulohumeral muscular dystrophy (FSHD), ARO-DM1 for myotonic dystrophy type 1 (DM1), ARO-MMP7 aimed at idiopathic pulmonary fibrosis (IPF), and ARO-ATXN2 for spinocerebellar ataxia 2 (SCA2). Additionally, preclinical initiatives focus on ataxin proteins linked to other genetic disorders, including Huntington’s disease. The partnership also opens the door for Sarepta to identify up to six new targets for Arrowhead to develop, which may lead to further innovations in the treatment of muscle, cardiac, and CNS disorders. This strategic move aligns with the ongoing success of Sarepta’s product Elevidys and reinforces Arrowhead’s commitment to advancing therapeutic solutions in the genetic medicine landscape.

The collaboration not only strengthens the capabilities of both companies but also signals a pivotal shift for Arrowhead as it transitions from a developmental stage to a commercial entity. Arrowhead’s CEO, Christopher Anzalone, emphasizes the importance of this partnership in expanding the company’s cash runway into 2028, which supports multiple drug launches. Moreover, the agreement includes substantial financial backing for Arrowhead, comprising $825 million, encompassing $500 million in cash and $325 million in equity investment. This strategic infusion of capital, along with the addition of Doug Ingram, Sarepta's president and CEO, to Arrowhead’s Board of Directors, enhances the company’s clinical and regulatory expertise, paving the way for successful commercialization of its innovative therapeutic candidates.

In addition to this collaboration, Arrowhead is on track to submit its first New Drug Application (NDA) for plozasiran, an investigational product for familial chylomicronemia syndrome, with a potential launch anticipated in 2025, pending FDA approval. The company also solidifies its financial future through a $500 million senior secured credit facility agreement with Sixth Street, which further strengthens its position for future growth. Overall, this partnership marks a significant milestone for Arrowhead Pharmaceuticals, setting the stage for impactful advancements in the treatment of rare genetic diseases.