Please login to the form below

Not currently logged in

Genzyme enters $22.5m RNAi deal with Alnylam in Asia

Programme targets condition affecting heart and nervous system

Sanofi's Genzyme division will make an upfront payment of $22.5m to Alnylam Pharmaceuticals under a newly-signed deal that will see the firms develop and commercialise RNAi therapeutics in Asia.

The investigational drugs will target transthyretin for the treatment of transthyretin-mediated amyloidosis (ATTR), a rare and hereditary condition affecting the nervous system and heart, which severely reduces someone's life expectancy.

Genzyme and Alnylam will develop the ALN-TTR programme to target variations of ATTR, including familial amyloidotic polyneuropathy (FAM), which can occur in patients carrying the V30M TTR mutation.

Other ATTR-related conditions also touted as potential indications include familial amyloidotic cardiomyopathy (FAC) and senile systemic amyloidosis (SSA).

According to Alnylam, ATTR is an orphan condition, with FAP thought to affect about 10,000 people worldwide and FAC though to affect at least 40,000 people.

However, despite the significant morbidity and mortality of the conditions, there is a considerable unmet medical need, with liver transplantation and Pfizer's Vyndaqel (tafamidis) often the only two treatment options.

Specific drugs in development as part of the ALN-TTR programme include ALN-TTR02, which is currently in phase II development following promising results in phase I where the drug demonstrated it was able to suppress transthyretin. A phase III trial is expected to begin by the end of 2013.

In addition, ALN-TTRsc, a subcutaneously administered RNAi therapeutic is in late stage pre-clinical development. 

“We are encouraged by Alnylam's progress with their ALN-TTR programme and are excited by the potential for this innovative drug candidate to make a difference in the lives of patients with ATTR,” said David Meeker, president and CEO of Genzyme.

“The results to date demonstrate impressive clinical activity and support advancement of this promising therapeutic into pivotal studies and toward the market.”

As well as the upfront payment, Genzyme will also pay Alnylam development milestones and tiered royalties.

The Genzyme-Alnylam deal applies only to Japan and other Asian countries, with Alnylam harbouring alternative intentions in the US, Europe and rest of the world.

23rd October 2012

From: Research



Featured jobs

Subscribe to our email news alerts


Add my company

Welcome to HAVAS LYNX, a leading global healthcare communications group....

Latest intelligence

NEW EU DATA PROTECTION REGULATION POISED TO CHANGE DATA PRIVACY LANDSCAPE The bold digital clock featured on the EUGDPR (EU General Data Protection Regulation)1 website indicates – to the second...
Health knowledge: Why is it so important?
Health knowledge plays an important role in population health, but by itself is rarely enough to prompt a change in the behaviours that cause the risks....
Personas: a top tool for personalising pharma marketing campaigns
Healthcare customers are calling for greater personalisation in care from providers. Here, we explain why personas are the top tool for ensuring your brand meets this need efficiently and effectively....