Alnylam reports positive initial data from Phase I/II study of ALN-GO1 to treat PH1

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.

"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 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.

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.