Aeglea BioTherapeutics and Immedica Announce Commercialization Agreement for Pegzilarginase for the Treatment of Arginase 1 Deficiency in Europe and Middle East
Published: Mar 22, 2021

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AUSTIN, Texas and STOCKHOLM, March 22, 2021 /PRNewswire/ — Aeglea Biotherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics as innovative solutions for rare metabolic diseases, and Immedica Pharma AB (Immedica) today announced the license and supply agreement for pegzilarginase, a novel, recombinant human arginase 1 enzyme that has been shown to lower toxic levels of the amino acid arginine in patients with Arginase 1 Deficiency (ARG1-D). People living with ARG1-D, a rare, progressive disease characterized by high levels of arginine, may experience severe spasticity-related mobility limitations, seizures, developmental delay, intellectual disability and early mortality.

“With a proven track record of bringing important products to the rare disease community in Europe and the Middle East, Immedica represents an ideal partner as we plan for the potential approval and commercialization of pegzilarginase in key markets beyond the U.S.,” said Anthony G. Quinn, M.B. Ch.B., Ph.D., president and chief executive officer of Aeglea. “There is an urgent need to deliver new therapies for Arginase 1 Deficiency due to its progressive and devastating nature. We are pleased to have a partner who believes in the potential of this program and shares our excitement for bringing pegzilarginase to market to further our mission of improving the lives of families affected by Arginase 1 Deficiency.”

“We are excited to enter this partnership with Aeglea and expand our growing portfolio to include pegzilarginase, which fits strategically very well within our existing rare disease and urea cycle disorder portfolio and commercial infrastructure,” says Anders Edvell, CEO of Immedica. “This agreement strengthens our position as a partner of choice for rare disease collaborations.”

Under the terms of the agreement, Immedica will make an upfront payment of $21.5 million to Aeglea. Additionally, Aeglea will be eligible to receive up to approximately $130 million in regulatory and commercial milestones as well as mid-twenties percentage royalties on net sales. Immedica receives commercialization rights in Europe and several Middle East countries. Aeglea will continue to be responsible for certain clinical development activities and the manufacturing of pegzilarginase, and retains commercialization rights in the U.S. and rest of the world.

Pegzilarginase is currently being investigated as a treatment for ARG1-D in the PEACE study, a pivotal Phase 3 clinical trial with topline data expected in the fourth quarter of 2021. In Phase 1/2 and ongoing open-label extension studies, pegzilarginase has been shown to lower toxic levels of arginine in patients with ARG1-D. After a 56 week treatment period, all 13 patients achieved plasma arginine levels within the target range (<200µM) and 11 patients (85%) were considered clinical responders based on improvements in mobility assessments. Pegzilarginase was also shown to have a favorable safety profile with the most common treatment-related serious adverse events being hypersensitivity and hyperammonemia, both of which were infrequent, expected and managed with standard of care. Most treatment-related adverse events were mild and decreased in frequency over time.