Poised to launch its first marketing effort, Alnylam makes a big leap into CNS diseases and Alzheimer’s

With its lead RNAi drug poised for an historic approval, Alnylam $ALNY is now laying plans to create a pipeline of CNS drugs that will target some of the toughest diseases that face researchers today — including Alzheimer’s, the Night King of diseases.

Kevin Fitzgerald

Alnylam researchers are following up on a rodent study where they say they were able to deliver a small interfering RNA drug — or siRNA —into the brain and spinal cord area. Using intrathecal injection — circumventing the blood-brain barrier — the biotech says that they were able to durably silence a disease target gene transcript, offering proof of principle evidence that they could succeed where a long queue of contenders have failed.

Using new technology, Alnylam says the plan now is to select its first CNS candidate later this year, with a growing effort to go after a string of tough-to-beat diseases, including Alzheimer’s, Huntington’s, and Parkinson’s.

“We have now applied our learnings, including additional chemistry advances, to enable delivery of siRNAs beyond the liver to the CNS, where there are a large number of unmet needs well suited for RNAi therapeutics. As we begin to advance our CNS pipeline, initial efforts are focused on genetically validated CNS targets, use of biomarkers for initial proof-of-concept, and disease settings with high unmet need and a definable path to regulatory approval and patient access,” said Kevin Fitzgerald, the senior VP of research at Alnylam. 

Alzheimer’s in particular has proven to be the toughest target in biopharma. Merck’s recent failure with a BACE1 inhibitor led the pharma giant to conclude that that approach — eliminating the flow of amyloid beta into the brain — will likely not work in symptomatic patients. Their researchers also conceded that the whole amyloid beta theory could be nothing but a fiction that drove billions of dollars of misspent research investments.

The focus now has shifted more toward pre-symptomatic patients as well as combination therapies going after more than one target at once. And the FDA has indicated its willingness to provide new guidelines that focus more on biomarkers of the disease to prove a drug’s ability to drive improved cognition for patients.

While a host of major players like Pfizer and AstraZeneca and GSK have retreated from the field in the wake of multiple failures, leaving few or no projects in their wake, new players like Celgene and the startup Denali have been getting into the Alzheimer’s arena. You can now include one more big player which plans to make a difference.

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