Initial results of Phase 3 trial of lumasiran in kids are promising

Alnylam Pharmaceuticals, Inc. announced positive preliminary results from the company's ILLUMINATE-A Phase 3 study of OXLUMO® (lumasiran), which is an RNAi therapeutic targeting hydroxyacid oxidase 1 – the gene encoding glycolate oxidase, to treat primary hyperoxaluria type 1, an ultra-rare genetic disease characterized by oxalate overproduction in the liver.¹ The data were released at the Pediatric Academic Societies virtual meeting.

The trial was a 6 month, randomized, double-blind, placebo-controlled, global, multicenter Phase 3 study that studied efficacy and safety of lumasiran in 39 patients aged 6 years and older who had a documented diagnosis of primary hyperoxaluria type 1. Patients were randomized 2:1 to either receive 3 monthly doses of the drug or placebo followed by quarterly doses at 3 mg/kg. Twenty-four of the participants had valid renal ultrasounds at baseline and 11 had improvement in the nephrocalcinosis grade relative to baseline and 4 remained stable. Only 3 children experienced worsening in the grade. In the 14 children with baseline nephrocalcinosis and available ultrasounds, 11 showed improvement when compared to the baseline and 8 of those children saw improvement in both kidneys. Common adverse effects included injection site reaction, erythema, pain, pruritus, and swelling.

Alnylam Pharmaceuticals, Inc. indicated that further data would be available later in the year.

Reference

1. Alnylam Pharmaceuticals, Inc. Alnylam announces positive early results on clinical outcome measures from ILLUMINATE-A phase 3 study of OXLUMO® (lumasiran). Published May 3, 2021. Accessed May 3, 2021. https://www.businesswire.com/news/home/20210503005235/en/