US-based biopharmaceutical company Alnylam has reported positive initial data from its ongoing Phase I/II study of ALN-GO1 to treat Primary Hyperoxaluria Type 1 (PH1).
ALN-GO1 is an RNA interference (RNAi) therapeutic product developed to target glycolate oxidase (GO) for the treatment of PH1.
RNAi is a natural process of gene silencing that takes place in organisms such as plants and mammals.
RNAi therapeutic medicines are a result of the usage of this natural biological process of RNAi occurring in cells of living beings.
The Phase I/II trial has been designed as a randomised, single-blind, placebo-controlled study and is being conducted in two parts.
Part A has been conducted as a single-dose study enrolling 32 healthy adult subjects, while Part B will be conducted as a multi-dose study, which is planning to enrol 20 patients suffering from PH1.
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The trial is primarily focused on testing the safety and tolerability of single and multiple subcutaneous doses of ALN-GO1.
The secondary goals of the trial are to determine the pharmacokinetics profile and clinical activity for ALN-GO1, determined by its effects on plasma glycolate and urinary oxalate levels in normal healthy volunteers and PH1 patients.
Data of Part A of the study suggested that a single, subcutaneously administered dosage of ALN-GO1 was able to achieve dose-dependent increases in plasma and urine glycolate.
It was also well tolerated by subjects throughout the study.
Alnylam chief medical officer and research and development executive vice-president Akshay Vaishnaw said: “We believe ALN-GO1 has the potential to be a transformative therapy for patients with PH1, a potentially fatal and ultra-rare orphan disease that primarily afflicts children.
“The current treatment approach for patients suffering from this condition is routine dialysis and, ultimately, a dual liver and kidney transplant, as no approved pharmaceutical options currently exist.”
The company is now planning to proceed with Part B of the ongoing Phase I/II study of ALN-GO1.