Regeneron, Alnylam Ink RNAi Alliance

To discover, develop and commercialize new RNA interference therapeutics for a broad range of diseases

Regeneron Pharmaceuticals, Inc. and Alnylam Pharmaceuticals, Inc. have entered a collaboration to discover, develop and commercialize new RNA interference (RNAi) therapeutics for a broad range of diseases by addressing disease targets expressed in the eye and central nervous system (CNS), and a select number of targets expressed in the liver. The collaboration will leverage both companies' scientific and technological expertise and will build on Alnylam's recent preclinical data showing potent and highly durable delivery of RNAi therapeutics to achieve target gene silencing in the eye and CNS. The collaboration will also leverage Regeneron's indus VelociSuite technologies and capabilities from the Regeneron Genetics Center (RGC).
 
Alnylam will work exclusively with Regeneron to discover RNAi therapeutics for eye and CNS diseases. Regeneron will lead development and commercialization for all programs targeting eye diseases, with Alnylam entitled to potential milestone and royalty payments. The companies will jointly advance and alternate leadership on CNS programs, with the lead party retaining global development and commercial responsibility. For CNS programs, both companies will have the option at candidate selection to participate equally in potential future profits of programs led by the other party.
 
The collaboration also includes a select number of RNAi therapeutic programs designed to target genes expressed in the liver, which can influence a wide variety of diseases. These programs include a planned joint effort evaluating anti-C5 antibody-siRNA combinations for C5 complement-mediated diseases including evaluating the combination of Regeneron's pozelimab (REGN3918), currently in Phase I development, with Alnylam's cemdisiran, currently in Phase II development. Alnylam will retain control of cemdisiran monotherapy development, and Regeneron will lead combination development. The parties will share investment and potential future profits on the monotherapy program, and Alnylam will receive royalties on any potential combination product sales.

Alnylam will receive $400 million upfront and Regeneron will purchase $400 million of Alnylam equity. Alnylam is eligible to receive up to an additional $200 million in milestone payments. The companies plan to advance programs directed to 30 targets and introduce many into clinical development during the initial five-year discovery period, which includes an option to extend.

"This collaboration couples proven and emerging RNAi technology, which holds important promise in many diseases, with Regeneron's world-leading genetics research and target discovery engine," said George D. Yancopoulos, M.D., Ph.D., president and chief scientific officer of Regeneron. "This collaboration enables us to reach targets inside the cell, complementing our expertise in antibodies, which are ideal for extracellular targets and those on the cell surface. Through the RGC and our other research groups, we are already identifying additional targets that may be well-suited for RNAi-based drug development, particularly in the eye and CNS."

"We are thrilled to collaborate with Regeneron, a like-minded science-based organization, to significantly accelerate our efforts to bring RNAi therapeutics to patients," said John Maraganore, Ph.D., chief executive officer of Alnylam. "Importantly, the alliance structure enables Alnylam to continue to build its industry-leading pipeline of RNAi therapeutics while retaining significant product rights."

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