- Sanofi and Alnylam Pharmaceuticals, Inc. are restructuring their 2012/2014 deal for Alnylam's pipeline drugs, giving Alnylam back development and commercialization rights to RNAi therapeutics for the treatment of ATTR amyloidosis, including patisiran and ALN-TTRsc02. Patisiran is pending approval in the U.S. and Europe.
- Sanofi will get royalties on net sales of the amyloidosis products, as well as global development and commercialization rights to the RNAi therapeutic fitusiran, in late stage clinical development for hemophilia A and B. Sanofi Genzyme will be responsible for sales and marketing, and Alnylam will be paid royalties. The rest of the deal remains unchanged.
- Alnylam will use the opportunity to consolidate its amyloidosis portfolio, as well as gain revenue from product sales and royalties post-approval.
The announcement from Sanofi and Alnylam is a major strategic restructuring of an existing deal between the two companies, described as a step to "streamline and optimize" the development of the rare genetic diseases products.
“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 programs,” said John Maraganore, CEO, Alnylam.
In a breakout session at the J.P. Morgan 36th Annual Healthcare Conference in San Francisco this week, Maraganore discussed the restructuring in more detail:
"It was a mutual initiation. Sanofi has been interested in more global rights from our pipeline… so they were quite interested in finding ways that they could get a more global footprint out of our rare disease pipeline in general," he said, according to a transcript.
At the same time, Alnylam was very interested to resolve … a complicated business arrangement on ALN-TTRsc02 versus patisiran, where in one asset we had 100% North America and Western Europe and the other asset we had a 50-50 relationship in North America and Western Europe.
Frankly, we were both converging toward this kind of discussion. Yvonne [Greenstreet, Chief Operating Officer] and I met Olivier [Brandicourt] and his team, Bill Sibold, in early December of the year. In the course of one hour… we crafted this deal to essentially provide them the lead on fitusiran and ALN-TTRsc02."
Maury Raycroft, an equity analyst at Jefferies, cast the deal as positive in a note.
"Restructuring improves ALN-TTRsc02 economics, with [the] launch now accretive to Alnylam, removing an overhang. Investors… have been concerned [about] ALN-TTRsc02 eroding Alnylam's patisiran economics, introducing some risk to long-term value. Alnylam is now able to focus on advancing ALN-TTRsc02 aggressively, building out the hATTR market, and incentivizing high conversion and good competitive positioning."
The compound is expected to move into pivotal studies late this year, in studies including pre-symptomatic patients. A patisiran launch is expected in the U.S. in mid-2018, and in Europe in late 2018.
Sanofi company Genzyme and Alnylam struck the original deal to develop and commercialize patisiran for transthyretin (TTR)-familial amyloid polyneuropathy, in 2012. The pact was expanded in January 2014 to all territories worldwide outside Western Europe and North America for patisiran. Sanofi also gained North American and Western European rights to commercialize Alnylam's ALN-TTRsc02, in development for familial amyloid cardiomyopathy, and to two additional products in Alnylam's pipeline.
Finally, the deal gave Sanofi the option, until 2020, to develop and commercialize all of Alnylam's rare genetic disease products outside North America and Western Europe.