Alnylam's Lumasiran Shows Clinical Benefit In Rare Kidney Disease

Alnylam Pharmaceuticals Inc ALNY announced new results from the six-month primary analysis of the ILLUMINATE-C Phase 3 open-label study of lumasiran for primary hyperoxaluria type 1 (PH1).
PH1 is a rare disorder that mainly affects the kidneys. It results from the buildup of a substance called oxalate, typically filtered through the kidneys and excreted in the urine.
Treatment with lumasiran resulted in substantial reductions in plasma oxalate (POx) at six months.
Read Next: See Why Did FDA Extend Review Period For Alnylam's Vutrisiran In Amyloidosis.
New exploratory analyses show that lumasiran treatment improved cardiac measures, nephrocalcinosis, and kidney stone events.
In addition, some patients treated with lumasiran experienced improvements in their most burdensome symptoms, including fatigue, nausea, and bone pain, with no patients reporting worsening of those symptoms.
ILLUMINATE-C enrolled 21 patients, including six who were not on dialysis at the study start (Cohort A) and 15 on hemodialysis (Cohort B).
The most common adverse event related to lumasiran was the injection-site reaction (ISR) reported in 5 of 21 patients. All ISRs were mild and transient, and the most common symptoms included erythema, discoloration, and hematoma.
A separate analysis presented demonstrated that the recommended weight-based dosing regimens of lumasiran showed equivalent efficacy in patients of all ages and degrees of kidney function, including patients on hemodialysis.
Price Action: ALNY shares are trading 2.55% lower at $126.27 during the market session on the last check Tuesday.

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