R-troloxamide quinone (EPI-589) / Sumitomo Pharma, PTC Therap - LARVOL DELTA

EPIC-ALS: A phase 2 trial of EPI589 in ALS (ALS-MND 2024) - No abstract available

P2 data

Characterization and expression of highly active recombinant human Glucuronyl C5-epimerase in mammalian cells. (PubMed, Glycobiology) - "A high-yield expression strategy was established using the lentiviral-based transduction system to produce recombinant HSepi (HSepi589) with a specific activity of up to 1.6 IU/mg. Together, this study contributes to the preparation of highly active HSepi for the enzymatic synthesis of heparins by providing additional insights into the catalytic activity of HSepi."

Journal • Thrombosis

An Exploratory Trial of EPI-589 in Amyotrophic Lateral Sclerosis (EPIC-ALS): Protocol for a Multicenter, Open-Labeled, 24-Week, Single-Group Study. (PubMed, JMIR Res Protoc) - "This study can provide useful data to understand the characteristics of EPI-589."

Journal • Amyotrophic Lateral Sclerosis • CNS Disorders • Metabolic Disorders • NEFH • NEFL

Phase 1 Study to Investigate the Safety, Tolerability, and Pharmacokinetics of EPI-589 in Healthy Participants. (PubMed, Clin Pharmacol Drug Dev) - "At both doses studied (500 mg twice daily and 750 mg twice daily), C t , and AUC were generally comparable between day 1 and day 7 and between the Japanese and White participants. EPI-589 was well tolerated as a single daily dose up to 1000 mg and as twice-daily doses up to 750 mg, with a linear pharmacokinetic profile."

Journal • P1 data • PK/PD data • Amyotrophic Lateral Sclerosis • CNS Disorders • Metabolic Disorders

Safety and Biomarker Study of EPI-589 in Parkinson's Disease (clinicaltrials.gov) - P2a; N=44; Completed; Sponsor: PTC Therapeutics; Active, not recruiting ➔ Completed

Biomarker • Clinical • Trial completion • Alzheimer's Disease • CNS Disorders • Gene Therapies • Movement Disorders • Parkinson's Disease

Safety and Biomarker Study of EPI-589 in Parkinson's Disease (clinicaltrials.gov) - P2a; N=40; Recruiting; Sponsor: Edison Pharmaceuticals Inc; Trial completion date: Jul 2018 ➔ Dec 2018; Trial primary completion date: Apr 2018 ➔ Dec 2018

Trial completion date • Trial primary completion date • Biosimilar • CNS Disorders • Gene Therapies • Genetic Disorders • Parkinson's Disease

Safety and Biomarker Study of EPI-589 in Parkinson's Disease (clinicaltrials.gov) - P2a; N=44; Active, not recruiting; Sponsor: Edison Pharmaceuticals Inc; Trial completion date: Dec 2018 ➔ Dec 2019; Trial primary completion date: Dec 2018 ➔ Dec 2019

Biomarker • Clinical • Trial completion date • Trial primary completion date