Sarepta, Arrowhead Strike Global Collaboration Deal to Develop Treatments for Rare Genetic Diseases Affecting Muscles

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Sarepta Therapeutics has entered a global licensing and collaboration agreement with Arrowhead Pharmaceuticals to develop RNA interference (RNAi)-based treatments for rare genetic diseases affecting muscles, the central nervous system (CNS), and lungs. According to the company, the agreement will grant exclusive rights to a number of Arrowhead’s clinical-stage programs, including ARO-DUX4 for facioscapulohumeral muscular dystrophy, ARO-DM1 for myotonic dystrophy type 1, ARO-MMP7 for idiopathic pulmonary fibrosis, and ARO-ATXN2 for spinocerebellar ataxia 2. All programs will leverage Arrowhead’s TRiM program platform for precise siRNA delivery.¹

“With the launch of Elevidys going exceedingly well, this broad siRNA collaboration with Arrowhead provides a synergistic platform to complement Sarepta’s gene therapy and gene editing engine. Through a strategic deployment of capital, we are able to access Arrowhead’s leading RNAi platform and will work to rapidly advance new treatments for devastating genetic diseases where there is significant unmet need. The agreement affords multiple potential blockbuster opportunities, serves our strategic priorities for the remainder of the decade and beyond, and diversifies our business model across one-time therapies and chronic treatments allowing for long-term growth and success. Given the strength of our performance and ability to generate substantial cash to invest in our business over the next several years, Sarepta’s Board of Directors has approved a $500 million share repurchase program as part of our overall capital allocation strategy,” said Doug Ingram, president, CEO, Sarepta, in a press release. “We look forward to embarking on this partnership with Arrowhead, having been impressed with their scientific capabilities in developing a potentially best-in-class approach to siRNA and the quality of the team they have built. Over the course of the next 12-18 months, we expect to share multiple data readouts from across our pipeline.

Under terms of the deal, Arrowhead will receive an upfront payment of $500 million, with Sarepta offering an equity investment of $325 million in Arrowhead common stock, priced at a 35% premium to the 30-day volume weighted average price prior to the announcement of the agreement. Additionally, Arrowhead will achieve $250 million to be paid in annual installments of $50 million over five years, in addition to being eligible for future milestone payments and royalties. Further, Arrowhead will be responsible for Phase I/II trials, which are currently in progress. Upon completion of ND-enabling activities, preclinical assets will transition to Sarepta.¹

“We welcome the Sarepta team as new Arrowhead collaboration partners who bring a wealth of clinical, regulatory, and commercial expertise in key areas outside of our cardiometabolic focus. We see our TRiM platform as a broad and elegant solution for delivery of siRNA to multiple cell types throughout the body. We also have a very efficient drug discovery engine that continues to generate many promising programs, and we have great confidence in Sarepta’s ability to take the next steps to advance and commercialize multiple Arrowhead-discovered drug candidates, which we believe have the potential to be best-in-class,” said Chris Anzalone, PhD, president, CEO, Arrowhead, in the press release. “At the close of this agreement, Doug Ingram will be appointed to the Arrowhead board of directors. He has led Sarepta as they advanced multiple investigational medicines through the clinical and regulatory process, built a commercial organization from the ground up, launched multiple drugs, and moved the company toward profitability. His experience and guidance will be valuable as Arrowhead seeks the same transition.”

In addition, Sarepta and Arrowhead have entered into a discovery collaboration for up to six additional muscle, cardiac, and/or CNS targets, using Arrowhead’s novel delivery technologies. As part of the collaboration, Sarepta has an exclusive license to Arrowhead’s technology to develop therapeutics against a broad range of skeletal muscle gene targets.¹

Reference

1. Sarepta Therapeutics Announces Global Licensing and Collaboration Agreement with Arrowhead Pharmaceuticals for Multiple Clinical and Preclinical siRNA Programs. Sarepta. November 26, 2024. Accessed November 27, 2024. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-global-licensing-and