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Msg  35093 of 35106  at  7/27/2022 7:14:48 PM  by


RNAi for common diseases? Endpoints

July 27, 2022 09:10 AM EDTUpdated 01:03 PM R&DDiscovery
RNAi for common diseases? Alnylam plans to forge a path forward, starting with new obesity gene target
Lei Lei Wu
News Reporter
Just over a month after it scored an approval for a next-gen RNAi therapy for hATTR amyloidosis, Alnylam has unveiled a bit more on where it plans to go next.

The Cambridge, MA-based biopharma announced Wednesday morning that it would be putting its RNAi technology toward some much, much broader indications — cardiometabolic diseases, which implicates a broad range of conditions, such as heart disease, diabetes and stroke, that collectively are the leading cause of death in the world.

Alnylam already has a program for one cardiometabolic disease, NASH, in which fatty tissue builds up in the liver and causes damage and inflammation. The biopharma also has a program for hypertension, or high blood pressure, which is associated with many cardiometabolic conditions.


Specifically, this new program plans on targeting the INHBE gene. The target was derived from a Alnylam-led study that used UK Biobank data, and arose from a larger UK Biobank-Biopharma collaboration that also includes Regeneron, AbbVie, AstraZeneca, Biogen and Pfizer, in which the biopharmas will sequence the exomes of all UK Biobank volunteers.

While Alnylam did not specify a disease area, the paper and subsequent press release both point toward type 2 diabetes and coronary heart disease as potential indications for this new program.

In the Nature Communications study, the researchers found that rare mutations in INHBE in liver cytokines were associated with lowering a surrogate measure (that combines waist-to-hip ratio and BMI) for abdominal fat, suggesting INHBE as a potential target. In the paper, the researchers write, “Importantly, by reducing abdominal fat, drugs targeting INHBE would have a distinct biological mechanism to existing drugs for CHD and T2D and may complement current therapies.”

Paul Nioi, who leads Alnylam’s discovery and translational research program, echoed that idea in a prepared statement:

There is a well-established causal link between increased waist-to-hip ratio and a person’s risk of cardiometabolic conditions. By exploring the genetic determinants of waist-to-hip ratio in this study, important insights into the mechanisms that contribute to body fat distribution were uncovered helping identify potential therapeutic targets for abdominal obesity, like INHBE. The results of this exome-wide analysis suggest that targeting INHBE is predicted to have broad beneficial effects on all facets of metabolic syndrome with potential reductions in the risk of type 2 diabetes and coronary heart disease. We are currently testing this hypothesis, with the goal of pursuing a development candidate targeting INHBE in the near future.

The program continues to push Alnylam’s RNAi technology to increasingly common conditions, a direction it has been headed more recently, despite starting in the rare disease space with hATTR amyloidosis.

In other news on its amyloidosis program, the biopharma awaits data on the use of Onpattro in ATTR with cardiomyopathy, a more common subtype of the disease. In that indication, Pfizer’s Vyndaqel/Vyndamax made $2 billion in 2021, making it Pfizer’s top-selling rare disease drug.

This story was updated to clarify that Alnylam is waiting for data on Onpattro, not an FDA decision.

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