verenafusp alfa (GNR-055) / Generium - LARVOL DELTA

Safety, tolerability, and pharmacokinetics of verenafusp alfa in healthy volunteers: results of an open-label multicohort phase I study (PubMed, Ter Arkh) - "Single intravenous administration of verenafusp alfa at doses ranging from 0.3 to 3 mg/kg demonstrated a favorable safety profile and good tolerability in healthy volunteers. The drug's pharmacokinetics was nonlinear, with a dose-dependent increase in Cmax and AUC with the dose increment. Volume of distribution volume lowered with increase of the dose."

Journal • P1 data • PK/PD data • Hunter Syndrome • IR

A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II (clinicaltrials.gov) - P2/3 | N=32 | Recruiting | Sponsor: AO GENERIUM | Trial primary completion date: Feb 2025 ➔ Jun 2026

Trial primary completion date • Hunter Syndrome • Metabolic Disorders

A Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy Volunteers (clinicaltrials.gov) - P1 | N=18 | Completed | Sponsor: AO GENERIUM

New P1 trial • Hunter Syndrome • Metabolic Disorders

A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II (clinicaltrials.gov) - P2/3 | N=32 | Recruiting | Sponsor: AO GENERIUM | Trial completion date: Mar 2025 ➔ Mar 2028

Trial completion date • Hunter Syndrome • Metabolic Disorders

A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II (clinicaltrials.gov) - P2/3; N=32; Recruiting; Sponsor: AO GENERIUM

New P2/3 trial • Hunter Syndrome • Metabolic Disorders • MRI

A Multi-cohort Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy Volunteers (clinicaltrials.gov) - P1; N=20; Completed; Sponsor: AO GENERIUM; Recruiting ➔ Completed

Clinical • Trial completion • Hunter Syndrome • Metabolic Disorders

A Multi-cohort Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy Volunteers (clinicaltrials.gov) - P1; N=20; Recruiting; Sponsor: AO GENERIUM

Clinical • New P1 trial • Hunter Syndrome • Metabolic Disorders