Aeglea Biotherapeutics has just announced a new collaboration with Immedica to bring pegzilarginase to other countries outside of the United States. They have created a license and supply agreement to aid in the delivery of the therapy as it becomes approved and commercialized across the world.
Immedica has a history of delivering rare disease therapeutics to the Middle East and Europe, and Aeglea is excited about the potential of this partnership. This collaboration provides Immedica commercialization rights to several countries in the Middle East and Europe. Aeglea will remain responsible for clinical development and manufacturing. Additionally, they will retain the rights to commercialization in the United States.
Pegzilarginase is a recombinant human arginase 1 enzyme developed to treat Arginase 1 Deficiency (ARG1-D) by lowering the levels of arginine in the body. ARG1-D is progressive in nature and therefore delivering this therapy to patients in need as soon as possible is critical.
Arginase 1 Deficiency
ARG1-D is a rare disease where arginine, an amino acid, accumulates in toxic levels in the body. This can lead to seizures, intellectual disability, developmental delays, spasticity-related mobility issues, and early mortality.
The condition presents in childhood.
Pegzilarginase is a recombinant human enzyme.
Pegzilarginase is currently being examined in a Phase 3 clinical trial called PEACE. Final results from this investigation are expected in the last quarter of this year. This investigation is examining treatment compared to placebo for 24 weeks. The primary endpoint is plasma arginine reduction.
A Phase 1/2 open label extension investigation has demonstrated that Pegzilarginase can effectively lower arginine levels in the body. In this trial, patients were treated for 56 weeks. All 13 of the patients in the trial achieved arginine levels in the plasma of <200µM which was within the target range. Additionally, 85% of participants had notable improvements in assessments of mobility.
This therapy has had a positive safety profile throughout its trials. The most common AEs have been hyperammonemia and hypersensitivity. Both of these were infrequently reported and easily managed. Further, most AE’s decreased the longer patients were taking the therapy.
This therapy has already been provided Rare Pediatric Disease Designation, Orphan Drug Designation, Breakthrough Therapy Designation, and Fast Track Designations from the FDA. Additionally, the EMA has granted Orphan Drug Designation to the therapeutic.
You can read more about this collaboration and investigative therapy here.