Alnylam says kidney disease therapy led to 37% drop in key indicator
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Alnylam Pharmaceuticals (NASDAQ:ALNY), a biotech focused on RNA interference, announced Thursday that its experimental RNAi therapy cemdisiran caused a 37% mean reduction of an important indicator of disease progression in immunoglobulin A nephropathy ((IgAN)).
Alnylam (ALNY) has partnered with Regeneron (REGN) to develop cemdisiran for IgAN, an inflammatory condition affecting the kidney, often leading to kidney failure.

Sharing topline results from a 31-patient Phase 2 trial, Alnylam (ALNY) said that at week 32, cemdisiran resulted in a 37% mean reduction of the 24-hour urine protein to creatinine ratio relative to placebo, the primary endpoint of the trial, and a key indicator of the disease progression.
While the results of secondary endpoints were consistent with the treatment benefit of cemdisiran, 12 of 22 (55%) patients in the cemdisiran group experienced treatment-emergent AEs which were related to the study drug, the company said.
In comparison, only two of nine (22%) patients on placebo witnessed treatment-emergent AEs. However, there were no discontinuations from the study drug due to AEs.
Given the acceptable safety profile and significant unmet need in IgAN, Alnylam (ALNY) and Regeneron (REGN) are planning to advance cemdisiran for Phase 3 development. 
Read more about late-stage data for another kidney disease drug developed by Alnylam (ALNY) for advanced primary hyperoxaluria type 1.