sodium phenylbutyrate liquid (TIK 201) / Tikvah Therap - LARVOL DELTA

CENTAUR: AMX0035 in Patients With Amyotrophic Lateral Sclerosis (ALS) (clinicaltrials.gov) - P2; N=137; Completed; Sponsor: Amylyx Pharmaceuticals Inc.; Phase classification: P2/3 ➔ P2

Phase classification • Amyotrophic Lateral Sclerosis • CNS Disorders • Proteinopathy

PEGASUS: Study to Assess the Safety and Biological Activity of AMX0035 for the Treatment of Alzheimer's Disease (clinicaltrials.gov) - P2; N=100; Active, not recruiting; Sponsor: Amylyx Pharmaceuticals Inc.; Trial completion date: Dec 2020 ➔ Aug 2021; Trial primary completion date: Oct 2020 ➔ Apr 2021

Clinical • Trial completion date • Trial primary completion date • Alzheimer's Disease • CNS Disorders • MRI

CENTAUR: AMX0035 in Patients With Amyotrophic Lateral Sclerosis (ALS) (clinicaltrials.gov) - P2/3; N=137; Completed; Sponsor: Amylyx Pharmaceuticals Inc.; Active, not recruiting ➔ Completed; Phase classification: P2 ➔ P2/3

Clinical • Phase classification • Trial completion • Amyotrophic Lateral Sclerosis • CNS Disorders • Complement-mediated Rare Disorders • Pain • Proteinopathy

Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders (clinicaltrials.gov) - P4; N=30; Not yet recruiting; Sponsor: Horizon Therapeutics, LLC

New P4 trial • Biosimilar

Sodium Phenylbutyrate and Edaravone Abrogate Chronic Restraint Stress-Induced Behavioral Deficits: Implication of Oxido-Nitrosative, Endoplasmic Reticulum Stress Cascade, and Neuroinflammation. (PubMed) - Cell Mol Neurobiol - "We found significant up-regulation of ER stress-related genes in both HC and PFC regions, which were suppressed by SPB and edaravone treatment in CRS mice. Our study provides evidence that SPB and edaravone exerted neuroprotective effects on CRS-induced cognitive deficits and anxiety- and depressive-like behavior, which is possibly coupled with inhibition of oxido-nitrosative stress, neuroinflammation, and ER stress cascade."

Journal • Alzheimer's Disease • Biosimilar • Depression • Immunology

Comparative Efficacy of Phenylbutyrate vs. Benzoate in Urea Cycle Disorders (clinicaltrials.gov) - P4; N=9; Recruiting; Sponsor: Baylor College of Medicine; Initiation date: Mar 2014 ->Sep 2014

Trial initiation date • Biosimilar • Fibrosis • Hemophilia

Switch from Sodium Phenylbutyrate to Glycerol Phenylbutyrate Improved Metabolic Stability in an Adolescent with Ornithine Transcarbamylase Deficiency. (PubMed) - JIMD Rep - "He had an extensive history of hospitalizations for hyperammonemic crises throughout childhood and early adolescence, which continued after the addition of sodium phenylbutyrate in 2009. In December 2013 he was switched to glycerol phenylbutyrate, and his metabolic stability was greatly improved over the following 7 months prior to liver transplant."

Journal • Biosimilar

Self-reported treatment-associated symptoms among patients with urea cycle disorders participating in glycerol phenylbutyrate clinical trials. (PubMed) - "The reduction in symptoms following 3months of open-label GPB dosing was similar in pediatric and adult patients and may be related to chemical structure and intrinsic characteristics of the product rather than its effect on ammonia control."

Journal • Biosimilar • Pain

PEGASUS: Study to Assess the Safety and Biological Activity of AMX0035 for the Treatment of Alzheimer's Disease (clinicaltrials.gov) - P2; N=100; Active, not recruiting; Sponsor: Amylyx Pharmaceuticals Inc.; Recruiting ➔ Active, not recruiting; Trial completion date: Sep 2020 ➔ Dec 2020; Trial primary completion date: Jul 2020 ➔ Oct 2020

Clinical • Enrollment closed • Trial completion date • Trial primary completion date • Alzheimer's Disease • CNS Disorders • Tauopathies And Synucleinopathies • MRI

TIGEM2-PDH: Use of Phenylbutyrate Therapy for Patients With Pyruvate Dehydrogenase Complex Deficiency. (clinicaltrials.gov) - P1/2; N=10; Recruiting; Sponsor: Fondazione Telethon; Trial completion date: Oct 2019 ➔ Dec 2020; Trial primary completion date: Oct 2019 ➔ Dec 2020

Clinical • Trial completion date • Trial primary completion date

Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders (clinicaltrials.gov) - P4; N=18; Recruiting; Sponsor: Horizon Therapeutics, LLC; N=30 ➔ 18; Trial completion date: Mar 2020 ➔ Mar 2023; Trial primary completion date: Dec 2019 ➔ Sep 2022

Clinical • Enrollment change • Trial completion date • Trial primary completion date

Phenylbutyrate and Valganciclovir in Treating Patients With Relapsed or Refractory Epstein-Barr Virus-Positive Cancer (clinicaltrials.gov) - P2; N=0; Withdrawn; Sponsor: University of California, San Diego; N=14 ➔ 0; Active, not recruiting ➔ Withdrawn

Clinical • Enrollment change • Trial withdrawal

PEGASUS: Study to Assess the Safety and Biological Activity of AMX0035 for the Treatment of Alzheimer's Disease (clinicaltrials.gov) - P2; N=100; Recruiting; Sponsor: Amylyx Pharmaceuticals Inc.; N=50 ➔ 100

Clinical • Enrollment change

CENTAUR: AMX0035 in Patients With Amyotrophic Lateral Sclerosis (ALS) (clinicaltrials.gov) - P2; N=132; Active, not recruiting; Sponsor: Amylyx Pharmaceuticals Inc.; Recruiting ➔ Active, not recruiting

Clinical • Enrollment closed

CENTAUR: AMX0035 in Patients With Amyotrophic Lateral Sclerosis (ALS) (clinicaltrials.gov) - P2; N=132; Recruiting; Sponsor: Amylyx Pharmaceuticals Inc.; Trial completion date: May 2019 ➔ Dec 2019; Trial primary completion date: Dec 2018 ➔ Oct 2019

Clinical • Trial completion date • Trial primary completion date