Alnylam Pharmaceuticals announced positive topline results from the phase 3 ILLUMINATE-A trial of lumasiran for the treatment of primary hyperoxaluria type 1 (PH1).

Lumasiran is a subcutaneously administered, investigational RNAi therapeutic that inhibits oxalate production by targeting hydroxyacid oxidase 1 (HAO1), which depletes glycolate oxidase (GO). In ILLUMINATE-A, the safety and efficacy of lumasiran was evaluated in 30 patients aged ≥6 years with PH1. Patients were randomized 2:1 to receive either lumasiran 3mg/kg monthly for 3 months followed by quarterly maintenance doses, or placebo. The primary end point was the percent change from baseline in 24-hour urinary oxalate excretion averaged across 3 to 6 months. 

Findings from the study showed that lumasiran achieved statistically significant results for the primary end point at 6 months (P <.0001) and for all 6 hierarchically-tested secondary end points (P ≤.001), including the proportion of lumasiran patients that achieved near-normalization or normalization of urinary oxalate levels, compared with placebo. With regard to safety, lumasiran was found to be well tolerated and was not associated with any serious or severe adverse events.

“The results from ILLUMINATE-A demonstrate that lumasiran can significantly reduce the hepatic production of oxalate, which we believe can thereby address the underlying pathophysiology of PH1,” said Akshay Vaishnaw, MD, PhD, President of R&D at Alnylam. “Further, we are encouraged by the safety and tolerability profile of lumasiran and believe this investigational medicine has the potential to have a meaningful clinical impact on patients living with PH1.” Full study results will be presented at an oral plenary session at OxalEurope International Congress in Amsterdam, Netherlands.


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Based on these results, Alnylam plans to submit a New Drug Application (NDA) for lumasiran in early 2020. Additionally, the Company is currently investigating lumasiran in two phase 3 trials, ILLUMINATE-B and ILLUMINATE-C, with results expected in mid-2020 and 2021, respectively.  

The Food and Drug Administration (FDA) previously granted Orphan Drug and Breakthrough Therapy designations to lumasiran for the treatment of PH1. 

For more information visit alnylam.com.