Regeneron, Alnylam Form $1-Billion Pact for RNAi Therapeutics

By Patricia Van Arnum - DCAT Editorial Director

April 11, 2019

Regeneron Pharmaceuticals, a Tarrytown, New York-based biopharmaceutical company, and Alnylam Pharmaceuticals, a Cambridge, Massachusetts-based company developing RNA interference (RNAi) therapeutics, have formed a collaboration, worth up to $1 billion, to discover, develop, and commercialize RNAi therapeutics for a broad range of diseases of the eye, central nervous system (CNS), and liver.

Under the terms of the alliance, 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.

Regeneron has agreed to make a $400-million upfront payment to Alnylam and to purchase $400 million of Alnylam equity at a price per share of $90.00 (4.44 million common shares). Alnylam is eligible to receive up to an additional $200 million in milestone payments upon achievement of certain criteria during early clinical development for the eye and CNS programs. 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. For each program, Regeneron will provide Alnylam with $2.5 million in funding at program initiation and an additional $2.5 million at lead candidate identification, translating to the potential for approximately $30 million in annual discovery funding to Alnylam.

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, 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 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. The companies will continue their previously-announced collaboration to identify RNAi therapeutics for the chronic liver disease nonalcoholic steatohepatitis (NASH) based on novel findings from the Regeneron Genetics Center. Alnylam retains broad global rights to all of its other unpartnered liver-directed clinical and preclinical pipeline programs.

Alnylam, alongside multiple other life-sciences companies, is also a member of Regeneron's pre-competitive consortium to sequence the DNA of 500,000 individuals in the UK Biobank health resource and subsequently make the data publicly available to the global research community.

The alliance and equity-related agreements are subject to customary closing conditions and clearances, including clearance under the Hart-Scott Rodino Antitrust Improvements Act.

Source: Regeneron Pharmaceuticals and Alnylam Pharmaceuticals