Phase 1/2 Results for Lumasiran Presented at ASN Kidney Week

Phase 1/2 Results for Lumasiran Presented at ASN Kidney Week

We presented positive preliminary data from the ongoing Phase 1/2 study with lumasiran (formerly known as ALN-GO1), an investigational RNAi therapeutic targeting glycolate oxidase (GO) for the treatment of Primary Hyperoxaluria Type 1 (PH1) at the American Society of Nephrology (ASN) Kidney Week 2017 Annual Meeting in New Orleans.

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In the first cohort, patients treated with lumasiran (N=3) experienced >50% decreases in urinary oxalate excretion relative to baseline. One placebo patient subsequently given lumasiran experienced a similar reduction in urinary oxalate, and the mean maximal reduction for all patients in this initial low dose cohort was 66%.

Patients in the first cohort have been monitored for up to seven months after receiving their first dose of lumasiran with no drug-related serious adverse events (SAEs) or discontinuations from study. In all patients, lumasiran was generally well tolerated and the only drug-related adverse event (AE) reported was a mild and transient injection site reaction.

These initial results support the hypothesis that GO inhibition has the potential to reduce and possibly normalize levels of hepatic oxalate production, thus potentially halting PH1 disease progression and fundamentally transforming the management of patients afflicted by this condition.



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