Alnylam Pharma (ALNY) Announces Sanofi Genzyme Will Opt-in to co-Develop, co-Commercialize Fitusiran in Hemophilia, Rare Bleeding Disorders

November 14, 2016 8:04 AM EST

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Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY) announced that, pursuant to the companies’ global alliance signed in January 2014, Sanofi Genzyme elected to opt in to co-develop (through Sanofi R&D) and co-commercialize fitusiran, an investigational RNAi therapeutic for the treatment of hemophilia and rare bleeding disorders (RBD), in the United States, Canada and Western Europe. This expanded right is in addition to their previously exercised opt-in decision to develop and commercialize fitusiran in their rest of world territories. The opt in decision was based on recent promising interim clinical results from a Phase 1 study of fitusiran presented at the World Federation of Hemophilia (WFH) in late July and additional data that will be presented at the American Society of Hematology (ASH) meeting in December. Alnylam is on track to initiate the fitusiran Phase 3 program in early 2017.

“This marks another milestone for this landmark collaboration and an important step forward on the path to bringing RNAi therapeutics to patients. This decision allows us to broaden the global reach and accelerate the commercial development of fitusiran, a potentially transformative approach to the treatment of hemophilia and rare bleeding disorders, with a well-established partner in Sanofi Genzyme,” said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. “We look forward to continued collaboration with Sanofi Genzyme to advance fitusiran as Alnylam transitions toward the commercial stage with our late-stage pipeline."

“We are pleased to collaborate with Alnylam to develop this important and innovative potential new treatment option for people living with hemophilia across the world. We believe that significant unmet need still exists, particularly for patients with inhibitors, and we are excited by the promising early clinical data coming from the fitusiran program,” said David Meeker, M.D., Executive Vice President and Head of Sanofi Genzyme. “This expanded collaboration with Alnylam supports our deep and lasting commitment to patients with rare diseases. We look forward to sharing our operational, regulatory and commercial experience with Alnylam as we advance this investigational product.”

Alnylam and Sanofi Genzyme will now co-develop and co-commercialize fitusiran in the Co-Commercialization Territory (United States, Canada and Western Europe), while Sanofi Genzyme will retain exclusive rights to develop and commercialize the product in the Sanofi Genzyme Territory (rest of world). Certain development and sales and marketing costs for fitusiran will be shared 50/50 between Sanofi Genzyme and Alnylam. In addition, Sanofi Genzyme will be required to make payments totaling up to $75 million upon the achievement of development and regulatory milestones for fitusiran. Upon the initiation of the first global Phase 3 clinical trial for fitusiran, Alnylam will earn a milestone payment of $25 million. Sanofi Genzyme also will be required to pay tiered double-digit royalties up to twenty percent on annual fitusiran net sales in the Sanofi Genzyme Territory. The companies will share profits equally in the Co-Commercialization Territory, where Alnylam expects to book product sales.

Sanofi Genzyme has elected not to opt in for ALN-AS1, an investigational RNAi therapeutic for acute hepatic porphyrias. The clinical dataset informing the Sanofi Genzyme decision consisted of Part A and Part B results from the ongoing ALN-AS1 Phase 1 study, recently presented at the Society for the Study of Inborn Errors of Metabolism (SSIEM) meeting in September. Based on this decision, Alnylam intends to develop and commercialize ALN-AS1 globally upon product approval. Alnylam will present initial results from Part C of the ongoing Phase 1 study at the ASH meeting in December.

Alliance Background

In January 2014, Alnylam and Sanofi Genzyme, the specialty care global business unit of Sanofi, formed an alliance to accelerate and expand the development and commercialization of RNAi therapeutics across the world. The alliance is structured as a multi-product geographic alliance in the field of rare diseases. Alnylam retains product rights in the United States, Canada and Western Europe, while Sanofi Genzyme obtained the right to access certain programs in Alnylam's current and future Genetic Medicines pipeline in the rest of the world through the end of 2019, together with certain broader co-development/co-commercialization rights and global rights for certain products.

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