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Abstract: FR-PO1054

A Phase 1/2 Trial of ALN-GO1: An Investigational RNAi Therapeutic for Primary Hyperoxaluria Type 1

Session Information

Category: Developmental Biology and Inherited Kidney Diseases

  • 403 Pediatric Nephrology


  • Frishberg, Yaacov, Shaare Zedek Medical Center, Jerusalem, Israel
  • van't Hoff, William, Great Ormond Street Hospital, London, United Kingdom
  • Hulton, Sally, Birmingham Childrens' Hospital, Birmingham, United Kingdom
  • Haslett, Patrick, Alnylam Pharmaceuticals, Cambridge, Massachusetts, United States
  • Erbe, David V, Alnylam Pharmaceuticals, Cambridge, Massachusetts, United States
  • Mcgregor, Tracy, Alnylam Pharmaceuticals, Cambridge, Massachusetts, United States
  • Deschênes, Georges, Hospital Robert Debre, Paris, France

In Primary Hyperoxaluria Type 1 (PH1) alanine-glyoxylate aminotransferase deficiency leads to excessive hepatic oxalate production. This results in nephrocalcinosis, recurrent calcium oxalate stones, and progressive renal impairment, ultimately with multi-organ damage from systemic oxalosis. ALN-GO1 is an investigational RNAi therapeutic which suppresses hepatic glycolate oxidase, decreasing the conversion of glycolate to glyoxylate, a required substrate for oxalate production. Previously released study data from healthy adult volunteers showed that single dose ALN-GO1 was well tolerated with dose-dependent elevations in plasma glycolate. Here we report initial outcomes of patients with PH1 in the Phase 2 portion of the study.


This randomized, placebo controlled, single blind, multicenter trial is ongoing ( NCT02706886). To be eligible, patients with PH1 must have urinary oxalate ≥0.7 mmol/24h/1.73m2 and eGFR >45 ml/min/1.73m2. One of four patients in each cohort is randomized to 3 doses of placebo prior to dosing with ALN-GO1. The first cohort received 1 mg/kg ALN-GO1 subcutaneously every 28 days x 3 doses, with the second cohort receiving 3 mg/kg ALN-GO1. The primary endpoint is safety, and secondary endpoints include change in 24 hour urinary oxalate excretion from baseline.


ALN-GO1 has demonstrated acceptable safety and tolerability to date with no treatment related serious adverse events or discontinuations from the study. Preliminary results reveal that all three patients randomized to ALN-GO1 in the first cohort experienced >50% decrease in urinary oxalate excretion from baseline. Two of the three patients achieved levels within the normal range.The patient randomized to placebo in the first cohort shows a similar trend after initial dosing with ALN-GO1. Data from the second cohort are forthcoming.


ALN-GO1 is an investigational RNAi therapy which has been well tolerated while showing promising activity in lowering urinary oxalate excretion in patients with PH1. These preliminary data support continued development of ALN-GO1 as a potential therapeutic approach to alleviating the pathologic overproduction of oxalate in this devastating disease.


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