Arrowhead and Sarepta Partner on Rare Genetic Disease Treatments

Arrowhead Pharmaceuticals has established a global licensing and partnership agreement with Sarepta Therapeutics to advance treatments for rare genetic disorders. This collaboration will focus on clinical and preclinical programs targeting rare conditions that impact the muscles, lungs, and central nervous system.

Arrowhead will receive a significant financial package from Sarepta, comprising $500 million in cash and a $325 million equity investment at a 35% premium. Additionally, Arrowhead is set to receive $250 million distributed in equal installments over five years and could also earn an additional $300 million as specific cohorts are enrolled in a Phase I/II trial. Beyond these payments, Arrowhead stands to gain royalties from commercial sales and a potential $10 billion in future milestone payments.

As part of the agreement, Sarepta can propose up to six new targets for which Arrowhead will conduct discovery and preclinical development using its Targeted RNAi Molecule (TRiM) platform. Sarepta will then receive an exclusive license for these programs and oversee their clinical development and commercialization. This arrangement could yield development milestone payments to Arrowhead ranging from $110 million to $410 million per program, along with sales milestone payments between $500 million and $700 million per program. Arrowhead is also entitled to receive tiered royalties on commercial sales.

The partnership also brings critical personnel changes, with Sarepta’s president and CEO, Doug Ingram, set to join Arrowhead’s board of directors. Arrowhead will be responsible for manufacturing clinical drug supply for all programs under this agreement and for commercial drug products for the four programs that are currently under study.

The completion of this transaction is projected for early 2025, pending the expiration or termination of the waiting period required by the Hart-Scott-Rodino Antitrust Improvements Act of 1976 and other customary conditions.

Christopher Anzalone, CEO and president of Arrowhead Pharmaceuticals, commented on the deal, noting that this transaction is expected to extend Arrowhead's financial viability into 2028 and potentially support multiple new drug launches, including both fully owned and partnered programs. He also highlighted the company's focus on preparing to launch investigational plozasiran for the treatment of familial chylomicronaemia syndrome, potentially in 2025, pending FDA review and approval. This product could become Arrowhead’s first commercial product.

In September 2024, the U.S. Food and Drug Administration granted breakthrough therapy status to Arrowhead's plozasiran, further advancing the company’s efforts in addressing rare genetic diseases through innovative treatments.