Arrowhead Pharmaceuticals and Sarepta Therapeutics have formed a global licensing and collaboration agreement.
The agreement covers multiple clinical and preclinical programs in rare, genetic diseases of the muscle, central nervous system (CNS), and the lungs, as well as allows Sarepta to select up to six new targets for Arrowhead to conduct discovery and preclinical development activities in areas complementary to Sarepta’s leadership in precision genetic medicine for rare diseases, which can utilize Arrowhead’s proprietary and differentiated Targeted RNAi Molecule (TRiM) platform.
Arrowhead will receive $825 million, consisting of $500 million cash and $325 million as an equity investment priced at a 35% premium. Arrowhead also receives $250 million to be paid in equal installments over five years and is eligible to receive an additional $300 million in near-term payments, which Arrowhead is on track to achieve during the next 12 months. Additionally, Arrowhead is eligible to receive royalties on commercial sales and up to approximately $10 billion in future potential milestone payments.
Christopher Anzalone, Ph.D., president and CEO at Arrowhead, said: “For Arrowhead, this strategic transaction accomplishes several important goals that we see as critical to our success as we move from a development stage company operating in several therapeutic areas to a more focused commercial stage company over the coming years. It provides us with a transformational amount of capital immediately and the potential for significant non-dilutive cash throughout the duration of the collaboration.
“We estimate that this transaction extends Arrowhead’s cash runway into 2028 and potentially through multiple new drug launches, including wholly owned and partnered programs. 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 prioritizing further development of additional pipeline programs which would be complementary to plozasiran from a clinical, regulatory, and commercial perspective.
“With this agreement we also 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 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.”
At the close of the agreement, Doug Ingram, president and CEO of Sarepta, will be appointed to the Arrowhead Board of Directors.
Mr. Ingram 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 honored to serve on the Arrowhead Board of Directors and to help Arrowhead advance its extraordinary science for the benefit of patients around the world.”
