United States-based Alnylam Pharmaceuticals has collaborated with United States-based Regeneron Pharmaceuticals.

It was reported yesterday that the collaboration is aimed at discovering, developing and commercialising new RNA interference therapeutics for a broad range of diseases by addressing disease targets expressed in the eye and central nervous system, in addition to a select number of targets expressed in the liver. It will leverage both companies' scientific and technological expertise and will build on Alnylam's recent preclinical data indicating potent and highly durable delivery of RNAi therapeutics to achieve target gene silencing in the eye and central nervous system. The collaboration will also benefit from Regeneron's VelociSuite technologies and capabilities from the Regeneron Genetics Center.

According to the terms of the collaboration, Alnylam will work exclusively with Regeneron to discover RNAi therapeutics for eye and central nervous system diseases. Regeneron will lead development and commercialisation for all programs targeting eye diseases, with Alnylam entitled to potential milestone and royalty payments. Both firms will jointly advance and alternate leadership on central nervous system programs, with the lead party retaining global development and commercial responsibility.

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 throughout the body. 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 1 development, with Alnylam's cemdisiran, currently in Phase 2 development.

Alnylam will retain control of cemdisiran monotherapy development, and Regeneron will lead combination development. The parties will equally share investment and potential future profits on the monotherapy program, and Alnylam will receive royalties on any potential combination product sales. For all other alliance liver programs, the parties will alternate leadership and participate equally in potential profits.

Regeneron has agreed to make a USD400 million upfront payment to Alnylam and to purchase USD400 million of Alnylam equity at a price per share of USD90.00 (4.44 million common shares), based on the volume-weighted average price over the last fifteen-trading-day period. Alnylam is eligible to receive up to an additional USD200 million in milestone payments upon achievement of certain criteria during early clinical development for the eye and CNS programs.