Skip to NavigationSkip to content

Regeneron-Alnylam team up to treat NASH

Published on 22/03/18 at 10:18am

Coming on the back of a Regeneron research piece published in the New England Journal of Medicine, the company announced a link up with Alnylam to work on the discovery it had made in the potential development of a treatment for non-alcoholic steatohepatitis (NASH).

The research discovered that a variant of the HSD17B13 gene, which was found to be associated with a reduced risk of chronic liver disease.

This opens up the possibility of using Alnylam’s gene silencing technology to specifically target the gene and silence its function; a naturally occurring genetic variation, uncovered by Regeneron’s researchers, found that people who exhibited it were protected from NASH disease progression.

If the collaboration can discover an RNAi therapeutic that is able to successfully achieve this, the two companies could be onto a huge breakthrough in the space.

As yet, there are no treatments for NASH development, beyond mediating symptoms. With 3-12% of the US population suffering from the disease, and this is only expected to grow in years to come, it represents a huge potential market.

The prevalence of NASH is linked to rising obesity levels and, as such rates are growing worldwide, it means the importance of the development of a treatment is incredibly important – over 38,000 people died due to chronic liver disease and cirrhosis in the US during 2014.

“Our Regeneron Genetics Center is delivering new targets that will require new approaches beyond our biologics capabilities. Since we are committed to following the science, we are pleased to join together with an equally science-minded company with a novel RNAi therapeutic approach that appears well-suited to impact this particular target,” said George D. Yancopoulos, President and Chief Scientific Officer of Regeneron.

John Maraganore, Chief Executive Officer at Alnylam, said, “As we transition Alnylam toward commercialisation in rare diseases, the prospect of collaborating with a scientific leader like Regeneron on innovative medicines for more prevalent diseases like NASH makes perfect strategic sense. We believe the exquisite specificity afforded by the RNAi mechanism of action and our industry-leading, proprietary GalNAc-conjugate approach for delivery to the liver is an unparalleled combination for developing an RNAi therapeutic toward genetically-validated targets in NASH.”

Some of that exquisite specificity has already been used to develop the biotech’s lead drug, patisiran, which is expected to gain approval by the FDA in treating hereditary ATTR amyloidosis by 11 August.

It is predicted that the biotech will be rewarded for its work in RNAi therapeutics by achieving peak sales of anywhere from $1.5 billion to as high as $2.9 billion for the drug.

Ben Hargreaves

Mission Statement
Pharmafile.com is a leading portal for the pharmaceutical industry, providing industry professionals with pharma news, pharma events, pharma service company listings and pharma jobs,
Site content is produced by our editorial team exclusively for Pharmafile.com and our industry newspaper Pharmafocus. Service company profiles and listings are taken from our pharmaceutical industry directory, Pharmafile, and presented in a unique Find and Compare format to ensure the most relevant matches