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After decades of determined research, RNAi drugs have their day in the sun

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L-R: Dietrich Stephan, Barbara Ryan, Pushkal Garg, Stephane Bancel, Christopher Anzalone

The announcement during last week’s J.P. Morgan Healthcare Conference of a deal between Novartis and England’s National Health Service to supply a cholesterol-lowering drug dosed only twice per year marked a significant event in the history of a class of drugs that only entered the market two years ago, but whose development goes back decades.

Biotech Showcase – an event in San Francisco that coincides with the annual JPM conference – featured a panel discussion Tuesday on the coming of age of RNA-interference, or RNAi drugs, including where the field could be headed over the next decade. Moderated by Piper Jaffray analyst Edward Tenthoff, the panel included Neubase Therapeutics CEO Dietrich Stephan, biopharma consultant Barbara Ryan, Alnylam Pharmaceuticals Chief Medical Officer Pushkal Garg, Moderna CEO Stephane Bancel and Arrowhead Pharmaceuticals CEO Christopher Anzalone.

Alnylam has the distinction of receiving the first two Food and Drug Administration approvals for RNAi medicines: Onpattro (patisiran), for hATTR amyloidosis, in August 2018; followed by the November 2019 approval of Givlaari (givosiran), for acute hepatic porphyria.

A third approval is likely in the near term for inclisiran, developed by The Medicines Company, in partnership with Alnylam. Novartis bought The Medicines Company in November for $9.7 billion, inheriting both inclisiran and the NHS deal. On Monday, the two announced a population health initiative that will provide the drug to patients and also launch a clinical trial in those who are at risk of developing heart disease.

During the Biotech Showcase discussion, panelists pointed out that the successes happening today were the result of dogged determination over the course of decades.

“What’s striking here is that all of you have ave been active here for decades and haven’t given up,” Ryan said.

Looking ahead, panelists foresaw a rapid expansion of pipelines over the next several years.

“Molecular therapies are wonderful because once you figure how to make the first one then the ability to scale is just incredible,” Bancel said. “Still I see a lot of people in pharma because it’s so foreign to them not appreciating that this is a platform.”

Garg said he foresees the sector going into more complex diseases as well, such as neurodegenerative diseases and hepatitis.

“It’s a very bright future – it’s hard to think about what it’ll look like,” he said.

In an interview, Christine Siu, chief financial officer of Eidos Therapeutics – one of the portfolio companies of BridgeBio – said she saw RNAi therapies as part of a larger armamentarium. Several of BridgeBio’s companies are developing therapies that would potentially compete with RNAi medicines, including Eidos, which has a drug in development for TTR amyloidosis and thus could face competition from Onpattro. Whereas Onpattro works by knocking down TTR – which stands for transthyretin – Eidos’ AG10 works by stabilizing it. Siu said the company views stabilization as a better mechanism than knocking it down because some TTR mutations actually improve patient outcomes.

“I think RNAis are very valuable and useful tools in therapeutic toolkit,” she said. “They can be great for some diseases – certainly they have shown that. It’ snot necessarily going to be a one stop shop for every disease.”

Photo: Alaric DeArment, MedCity News

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