Genzyme and Alnylam expand collaboration on rare genetic diseases

Genzyme, a Sanofi company (EURONEXT: SAN and NYSE: SNY), and Alnylam Pharmaceuticals, Inc. (NASDAQ: ALNY) announced today that they have significantly expanded their strategic agreement to develop and commercialize treatments for rare genetic diseases. Genzyme will have significant rights to Alnylam’s portfolio of clinical and pre-clinical stage drug candidates. Alnylam will retain most product rights in North America and Western Europe, and will have significantly expanded development and commercial opportunities for its genetic medicine pipeline through Genzyme’s established global infrastructure in rare diseases.

“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 wellestablished global organization,” said David Meeker, MD, Genzyme’s President and CEO. “This transaction also powerfully underscores Sanofi’s commitment to investing in Genzyme as one of the company’s key growth drivers. Our partnership with Alnylam has been highly collaborative, and their world-class RNAi technology holds the promise to provide a platform for sustained drug development for rare genetic diseases for years to come.”

“This new relationship with Genzyme is transformational for Alnylam. 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,” said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. “In this new alliance, Alnylam benefits enormously from Genzyme’s proven global capabilities, enabling us to accelerate and expand market access for our ‘Alnylam 5×15’ products.”

In 2012, Alnylam and Genzyme formed an exclusive alliance to develop and commercialize Alnylam’s lead product, patisiran, which is in Phase 3 development for the treatment of transthyretin (TTR)-familial amyloid polyneuropathy, a rare life-threatening disease that damages the nervous system.

The expanded relationship between Genzyme and Alnylam includes the following components:

First, Genzyme will obtain expanded rights to patisiran. Under the original agreement from 2012, Genzyme had rights to commercialize patisiran in Japan and the broader Asia-Pacific region. This disease has a disproportionately high prevalence in these territories. Under the expanded agreement, Genzyme will now commercialize patisiran in all territories outside of North America and Western Europe, which are retained by Alnylam for their commercialization.

Second, Genzyme will obtain rights to commercialize worldwide three products in Alnylam’s pipeline. Specifically, (1) Genzyme and Alnylam will co-develop and co-commercialize ALN-TTRsc, a product 2/4 currently in Phase 2 development for the treatment of familial amyloid cardiomyopathy, in North America and Western Europe, while Genzyme commercializes the product in the rest of world; (2) 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-commercialization rights, depending on the product.

Third, Genzyme will have the option up until 2020, with the possibility of extension through the end of 2021,to develop and commercialize outside of North America and Western Europe all products being developed to treat rare genetic diseases from Alnylam’s pipeline. Alnylam retains its rights to co-develop and co-commercialize its genetic medicine pipeline in North America and Western Europe.

Finally, Genzyme will become a major Alnylam shareholder with a stake of approximately 12% percent through a $700 million investment at a price of approximately $80/share, which represents a 27% premium as compared to the average share price over the last 30 days. In addition, Alnylam will receive R&D funding, starting on January 1, 2015, for programs where Genzyme has elected to opt-in for development and commercialization. Further, Alnylam is eligible to receive milestones and royalties.

This transaction has been approved by the boards of both companies, and is subject to customary closing conditions and clearances under the Hart-Scott Rodino Antitrust Improvements Act.

Conference Call Information

Alnylam and Genzyme management will discuss this new alliance in a conference call on January 13, 2014 at 9:00 am ET (6:00 am PT). A slide presentation will also be available on the News & Investors page of the company’s website, www.alnylam.com, to accompany the conference call. To access the call, please dial 877-312-7507 (domestic) or 631-813-4828 (international) five minutes prior to the start time and refer to conference ID 31887205. A replay of the call will be available beginning at 12 :00 pm ET (9 :00 am PT) on January 13, 2014. To access the replay, please dial 855-859-2056 (domestic) or 404-537-3406 (international), and refer to conference ID 31887205.