- Alnylam Pharmaceuticals Inc (NASDAQ: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.
