Aeglea BioTherapeutics has begun dosing patients in the repeat dose part of its Phase I/II clinical trial of AEB1102 (pegzilarginase) to treat arginase 1 deficiency.
AEB1102 is an engineered human arginase 1 enzyme being developed for degradation of the amino acid arginine for the treatment of arginine excess.
The drug candidate is expected to serve as an enzyme replacement therapy to restore arginase 1 function in patients by bringing increased blood arginine levels to normal physiological range.
Designed to include a minimum of ten adult and paediatric patients, the multi-centre, single arm, open label trial will assess the safety and tolerability of intravenous pegzilarginase as the primary endpoint.
The trial will also investigate the pharmacokinetic and pharmacodynamic effects such as plasma arginine levels after administration of repeated doses of pegzilarginase.
Aeglea BioTherapeutics chief medical officer James Wooldridge said: “The initiation of the repeat dose part of the study is important given the encouraging initial results seen with single doses of pegzilarginase, as it will provide important insights into the effects of longer-term arginine reduction in patients with this serious and progressive disease.
“Additionally, we are continuing our discussions with the FDA about including pediatric patients in the trial, and have initiated activities in Europe and Canada to reach more patients with this very rare disease.”
In the initial Phase I part of the trial, single-ascending doses of pegzilarginase showed favourable tolerability and decreased arginine levels in the blood.
Top-line results from the Phase I/II trial are expected to be available in the second half of next year.