Genzyme and Alnylam expand rare genetic diseases collaboration

Genzyme becomes major shareholder in the company through a US$700m equity investment

Genzyme, the biopharmaceuticals division of Sanofi, has taken a 12% stake in Alnylam Pharmaceuticals for US$700m and has expanded its agreement with the company to develop and commercialise treatments for rare genetic diseases.

John Maraganore, Chief Executive of Alnylam, said the new relationship with Genzyme will be 'transformational' for the company.

'It is a game changer for both the advancement of RNAi therapeutics as a new class of genetic medicines to patients around the world, and for our commitment to build a leading, independent biopharmaceutical company that delivers value to our shareholders,' he said.

The companies originally agreed to work together in 2012 to develop Alnylam's leading product, patisiran, for the treatment of transthyretin (TTR)-familial amyloid polyneuropathy, a rare life-threatening disease that damages the nervous system. This agreement gave Genzyme rights in Japan and the broader Asia-Pacific region where the disease is most common. But under the expanded partnership, Genzyme will have rights to sell the drug, if it is approved, in all regions outside North America and Western Europe, where Alnylam will retain exclusive rights.

This collaboration is an important building block for our future

In addition, Genzyme will now also obtain rights to commercialise worldwide three products in Alnylam's pipeline.

First, the two firms will co-develop and co-commercialise ALN-TTRsc, a product currently in Phase II development for the treatment of familial amyloid cardiomyopathy, in North America and Western Europe, while Genzyme commercialises the product in the rest of world.

Second, Genzyme will have the rights to two additional products after the completion of early clinical trials and will be able to choose between full global rights or co-commercialisation rights, depending on the product.

Third, Genzyme will have the option up until 2020, with the possibility of extension to the end of 2021, to develop and commercialise outside North America and Western Europe all products being developed to treat rare genetic diseases from Alnylam's pipeline.In addition, Alnylam will receive R&D funding, starting on 1 January 2015, for programmes where Genzyme has elected to opt-in for development and commercialisation. The company is also eligible to receive milestones and royalties.

'This collaboration is an important building block for our future. It strengthens our pipeline and provides us with the opportunity to meet the needs of patients with rare diseases around the world through our well-established global organisation,' said David Meeker, Genzyme's President and CEO.