Sarepta Therapeutics and Arrowhead Pharmaceuticals Enter $10 Billion Collaboration for Rare Disease Treatments

Agreement Overview:
Sarepta Therapeutics and Arrowhead Pharmaceuticals have entered a global licensing and collaboration agreement for multiple clinical and preclinical programs in rare, genetic diseases of the muscle, central nervous system (CNS), and the lungs[1][2].

Financial Terms:

Immediate Payment:
Arrowhead will receive $825 million upon closing, consisting of $500 million cash and $325 million as an equity investment at a 35% premium[2].

Future Payments:
Arrowhead will receive an additional $250 million over five years and is eligible for up to $10 billion in future potential milestone payments and royalties on sales[2].

Programs Included:
The agreement covers seven programs, including ARO-HTT for Huntington’s disease, ARO-ATXN1 for spinocerebellar ataxia 1 (SCA1), and ARO-ATXN3 for spinocerebellar ataxia 3 (SCA3), among others[2].

Discovery and Development:
Sarepta can select up to six new targets for Arrowhead to conduct discovery and preclinical development activities using Arrowhead’s proprietary Targeted RNAi Molecule (TRiM™) platform[1][2].

Closing and Timeline:
The transaction is expected to close in early 2025, subject to customary conditions and regulatory approvals[2].

Strategic Impact:
This collaboration aims to expedite the development of treatments for rare diseases, leveraging Arrowhead’s siRNA technology and Sarepta’s expertise in precision genetic medicine[2][4].

Commercial Potential:
The agreement has the potential to generate significant non-dilutive cash for Arrowhead and extend its cash runway into 2028, supporting multiple new drug launches[1][2].