Sanofi, Alnylam ink strategic restructuring of RNAi therapeutics rare disease alliance

Under the agreement:
Sanofi will obtain global development and commercialization rights to fitusiran, an investigational RNAi therapeutic, currently in development for the treatment of people with haemophilia A and B. Global commercialization of fitusiran, upon approval, will be done by Sanofi Genzyme, the speciality care global business unit of Sanofi. Alnylam will receive royalties based on net sales of fitusiran products.

Alnylam will obtain global development and commercialization rights to its investigational RNAi therapeutic programs for the treatment of ATTR amyloidosis, including patisiran and ALN-TTRsc02. Sanofi will receive royalties based on net sales of these ATTR amyloidosis products.

With respect to other products falling under the RNAi therapeutics alliance, the material terms of the 2014 Alnylam-Sanofi Genzyme alliance remain unchanged. 

“The restructured alliance reflects Sanofi Genzyme’s sustained interest in the strong potential of Alnylam’s portfolio of genetic medicines. The new structure simplifies operations, providing both parties the agility needed to make these medicines available to patients as quickly as possible once approved,” said Bill Sibold, executive vice president and head of Sanofi Genzyme. “This restructuring will enable both parties to maximize the value of each asset and allows us to maintain shared economics across the alliance programme.”

Fitusiran complements Sanofi Genzyme’s rare hematology portfolio, and creates a focus on bringing an innovative product to market globally, upon approval, for people living with hemophilia, one of the most common rare diseases.

“This strategic restructuring enables streamlined development and an optimized approach to bringing innovative medicines to patients with ATTR amyloidosis and hemophilia around the world, maximizing the commercial opportunities for these programmes,” said John Maraganore, Ph.D., chief executive officer of Alnylam. “For Alnylam, this provides strategic clarity and operational alignment with regard to the development and commercialization of patisiran and ALN-TTRsc02. This will allow us to develop both products in a comprehensive manner, potentially addressing the full spectrum of transthyretin-mediated amyloidosis disease treatment and prevention. At the same time, we will continue to support and benefit – via royalties – from the fitusiran opportunity through Sanofi’s significant development and commercial leadership.”

This restructuring provides Alnylam with the opportunity to consolidate its ATTR amyloidosis business to maximize its value, and the opportunity for near-term acceleration of product revenue growth based on newly obtained rights to commercialize patisiran around the world, once approved. In addition, it enables Alnylam to build a global presence and commercial infrastructure that can be leveraged for ALN-TTRsc02 and additional programs, including givosiran, an investigational RNAi therapeutic for the treatment of acute hepatic porphyrias, and cemdisiran, an investigational RNAi therapeutic for the treatment of complement-mediated diseases – where Alnylam has retained global ownership.

Terms of the agreements: 
Fitusiran: The restructuring will enable Sanofi to assume full responsibility for development and commercialization of fitusiran, including costs. However, during the anticipated transition period Alnylam will fund such costs. Alnylam intends to substantially complete the transition of fitusiran to Sanofi by mid-2018. Sanofi will pay Alnylam a milestone of $50 million following dosing of the first patient in the ATLAS phase 3 programme for fitusiran. 

Patisiran and ALN-TTRsc02: Alnylam will fund all development and commercialization costs for patisiran and ALN-TTRsc02 going forward. There will be no additional milestones due to either company with respect to patisiran or ALN-TTRsc02.

Sanofi intends to substantially complete the transition of its patisiran activities in regions outside the United States, Canada, and Western Europe, consistent with the original scope of its license rights to patisiran, by mid-2018.

Sanofi Genzyme and Alnylam will be eligible to receive tiered royalties of 15 to 30 percent on global net sales of ALN-TTRsc02 and fitusiran, respectively, upon approval and commercialization. Previously, these programs were subject to co-development and co-commercialization terms in the United States, Canada and Western Europe.

For patisiran, Sanofi Genzyme will be eligible to receive royalties, increasing over time  to up to 25 per cent, on sales in territories excluding the United States, Canada, and Western Europe.

Sanofi continues to have the right to opt into other Alnylam rare genetic disease programs for development and commercialization in territories outside of the United States, Canada and Western Europe, as well as one right to a global license.

The transaction is subject to customary closing conditions and clearances, including clearance under the Hart-Scott Rodino Antitrust Improvements Act.

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