Immedica to present new pegzilarginase data at the SSIEM Annual Symposium 2023

Report this content

Stockholm, August 28, 2023 Immedica today announces the presentation of new data at the SSIEM (Society for the Study of Inborn Errors of Metabolism) annual symposium taking place in Jerusalem, Israel. The presentation highlights important long-term patient-level data on pegzilarginase, a potential first disease modifying treatment for arginase 1 deficiency (ARG1-D).

Anders Edvell, Immedica CEO commented: “We are very pleased to be able to present this new patient-level data that demonstrates long-term clinical outcomes of pegzilarginase for the treatment of ARG1-D, a disease where the unmet medical need is huge”.

Oral presentation details

Presentation title: “Pegzilarginase demonstrates long-term, clinically meaningful improvements in functional mobility in ARG1-D: patient-level analysis from the Phase 3 PEACE trial”.

Authors: Serena Gasperini1, Rossana Sanchez Russo2, Gillian Bubb3, Linda Neuman3, Mattias Rudebeck4,5, Anais Brassier6, Gregory M Enns7

Date and time: August 30, 8:30 am IDT.

Presenter: Dr Serena Gasperini, PEACE study Italian principal investigator.


About pegzilarginase
Pegzilarginase is a novel recombinant human enzyme and has been shown to rapidly and sustainably lower levels of the amino acid arginine and its toxic metabolites in plasma accompanied by improvements in clinical outcomes. Pegzilarginase has been developed for the treatment of people with the urea cycle disorder arginase 1 deficiency (ARG1-D).

Pegzilarginase is currently not approved in any market or indication. It is currently under review by the European Medicinal Agency, EMA.


About ARG1-D
ARG1-D is one of the eight urea cycle disorder (UCD) subtypes. It shares overlapping features with other UCDs and the most prominent is the impairment in excreting nitrogen. However, in ARG1-D, hyperammonemia is generally less severe and instead these patients show spasticity, which other subtypes do not. The principal defect in ARG1-D leads to accumulation of plasma arginine and its toxic metabolites, which occurs in almost all patients with this disorder. Patients are often diagnosed in late infancy or early childhood and the symptoms include spasticity, seizures, developmental delay, intellectual disability, and early mortality.

About Immedica

Immedica is a pharmaceutical company, headquartered in Stockholm, Sweden, focused on the commercialization of medicines for rare diseases and specialty care products. Immedica’s capabilities cover marketing and sales, compliance, pharmacovigilance, quality assurance, regulatory, medical affairs and market access, as well as a global distribution network serving patients in more than 50 countries. Immedica is fully dedicated to helping those living with diseases which have a large unmet medical need. Immedica’s therapeutic areas are within genetic & metabolic diseases, hematology & oncology and specialty care.


Immedica was founded in 2018 by the investment company Impilo and Buy-in-Management. Today Immedica employs more than 90 people across Europe and the Middle East.

For more information visit www.immedica.com

Immedica contact:
Linda Holmström
Head of Communication
linda.holmstrom@immedica.com
+ 46 708 73 40 95

Immedica Pharma AB
Solnavägen 3H
SE-113 63 Stockholm