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Msg  35107 of 35122  at  12/16/2022 3:01:13 PM  by


Endpoints on Alnylam's R&D update

Alnylam's R&D update includes 3 halts, Alzheimer's timeline and another NASH
Kyle LaHucik
Associate Editor
Alnylam Pharmaceuticals touted its high probability of success in bringing assets from Phase I to Phase III — about 62% compared to an industry average of 5% to 10% — in an R&D update Thursday.

But, even the RNAi drug developer with five medicines now under its wings will come out with the scissors and scrap studies it finds lacking, as the Boston-area biotech outlined in an investor presentation. That includes cemdisiran for kidney disease IgA nephropathy, an early-stage drug for gout and lumasiran for recurrent renal stones.

Akshay Vaishnaw
“In spite of this enviable historical POS rate, it’s critical that we remain disciplined in making all data-driven pipeline decisions,” president Akshay Vaishnaw said during the virtual presentation. Regarding the drug ALN-XDH for gout, he said the asset hadn’t shown “transformative potential.”

In dropping the lumasiran study, Alnylam cited “pre-randomization screening showed urinary oxalate not elevated to degree suggested by literature and expert community.”


Moving forward, Alnylam further outlined its five-year 2025 vision, which CEO Yvonne Greenstreet said the biotech is “making really good progress” on, with the goal of treating 500,000 patients, launching four or more INDs per year and bringing to market a sixth drug. An expanded label for Onpattro is expected in late 2023 for cardiomyopathy of ATTR amyloidosis.

Yvonne Greenstreet
On tap for early next year are topline results from Alnylam’s Phase I study of an amyloid-focused Alzheimer’s drug. The drug developer claims its ALN-APP is the first RNAi therapeutic for a central nervous system disease. The early-stage clinical trial began in February and was originally slated for a year-end readout.

Eyes will likely be affixed to whether Alnylam can prove its RNAi approach to the memory-robbing disease, especially as the topline data are expected shortly after Eisai and Biogen hear from the FDA whether their Alzheimer’s drug lands an accelerated OK.

Meanwhile, in nonalcoholic fatty liver disease, Alnylam is ready to send its next drug into the clinic for an indication that has tripped up nearly every biopharma in the industry that has waded into the arena. Through the help of partner Regeneron, the duo will start a Phase I early next year for ALN-PNP, adding to their clinical roster in the area, as ALN-HSD begins mid-stage testing.

An RNAi for treating type 2 diabetes will also enter the clinic in the beginning months of 2023.

Kyle LaHucik
Associate Editor

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