Aeglea BioTherapeutics has reported that the Phase III PEACE clinical trial of its therapy pegzilarginase to treat individuals with Arginase 1 Deficiency (ARG1-D), met the primary goal.
A new recombinant human enzyme, pegzilarginase is claimed to degrade the amino acid arginine.
The single, international, randomised, placebo-controlled, double-blind trial analysed the impact of weekly infusions of pegzilarginase compared with placebo in subjects with ARG1-D for more than 24 weeks.
It enrolled a total of 32 subjects of age two years and above in the US, Canada and Europe.
The decline in plasma arginine from baseline was the trial’s primary goal.
Safety, pharmacokinetics and clinical outcome analysis focusing on various mobility evaluations comprised the secondary goals.
Findings showed that treatment with pegzilarginase offered a highly statistically significant 80% decline in mean plasma arginine, meeting the primary goal.
In addition, 90.5% of the subjects in the pegzilarginase arm attained normal plasma arginine levels of 40-115µM versus none in the placebo group.
A positive trend in mobility analysis was reported with the least-squares mean Gross Motor Function Measure Part E (GMFM-E) score increasing by 4.2 units in the pegzilarginase arm while it declined by 0.4 units in subjects who received placebo.
GMFM-E is a vital clinical assessment of the mobility of the patient, which includes the ability to walk, run and jump.
Furthermore, pegzilarginase was demonstrated to be well-tolerated in the trial with the safety profile of the drug in line with previously reported data.
Aeglea BioTherapeutics president and CEO Anthony Quinn said: “The dramatic reduction in plasma arginine levels and the positive trend in GMFM-E are very encouraging and represent an important step in our mission to bring a transformative therapy to this underserved patient community.
“We believe that the announcement demonstrates validation of our scientific platform, overall pipeline and potential to address other rare metabolic diseases.”
Based on the trial data, the company intends to submit a Biologics Licence Application for the therapy to the US Food and Drug Administration in the first half of next year.
Along with Immedica Pharma, the company’s commercial partner in Europe and certain countries in the Middle East, it plans to apply for marketing authorisation in those regions.
In September 2017, Aeglea commenced dosing subjects in the repeat dose part of its Phase I/II trial of AEB1102 (pegzilarginase) for the treatment of ARG1-D.
A rare and progressive disease, ARG1-D is characterised by arginine build-up.