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Sanofi strikes $700m rare diseases deal

Company's Genzyme division will obtain significant rights to Alnylam’s pipeline of orphan drugs
Sanofi reception

Sanofi has agreed a major expansion to its rare diseases partnership with genetic medicine specialist Alnylam.

The new deal will see Sanofi's rare disease-focused biotech arm Genzyme become a major stakeholder in Alnylam through a $700m investment and obtain certain global rights to three products in Alnylam's pipeline.

The only one of these treatments named so far is ALN-TTRsc, which is an RNAi therapeutic in phase II development for the treatment of familial amyloid cardiomyopathy – a genetic condition where the heart muscle is damaged by a build up of amyloid deposits.

Alnylam and Genzyme will co-commercialise the drug in North America and Western Europe while Genzyme will hold sole rights in the rest of the world.

Genzyme will be able to select the two other treatment prospects from Alnylam's pipeline to either obtain either full global co-commercialisation rights after the completion of early clinical trials.

This pipeline features a series of RNAi therapeutics designed to 'silence' mutated genes in rare genetic diseases in order to stop the disease-causing proteins from being formed. These cover conditions such as haemophilia, hypercholesterolemia and the liver disease hepatic porphyrias.

As mentioned in its 'Alnylam 5x15' guidance from 2011, the company had originally expected to have five genetic medicine programmes in clinical trials by the end of 2015, but last week it announced this number is to rise to six or seven, including two phase III programmes.

In addition to granting Genzyme licence options on these new drugs, the revised collaboration with Alnylam expands the 2012 deal agreed between the two companies to develop and commercialise the RNAi therapeutic patisiran (formerly ALN-TTR02).

Originally, the deal only covered parts of Asia, due to the high prevalence of transthyretin (TTR)-familial amyloid polyneuropathy, the rare nervous system for which patisiran is being investigated as a treatment.

However, the new deal will see Genzyme obtain rights to the drug in all territories outside of North America and Western Europe, with these two markets retained by Alnylam.

The final part of the new agreement gives Genzyme the option up until 2020 to develop and commercialise outside of North American and Eastern Europe all products being developed to treat rare genetic diseases from Alnylam's pipeline.

Genzyme's CEO David Meeker was confident in the deal's ability to deliver new orphan treatments with rare diseases becoming a major growth market for the pharma industry.

“This transaction also powerfully underscores Sanofi's commitment to investing in Genzyme as one of the company's key growth drivers,” he said.

“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.”

Article by
Thomas Meek

13th January 2014

From: Research

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