utreloxastat (PTC857) / PTC Therap - LARVOL DELTA

CARDINALS: Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALS (clinicaltrials.gov) - P2 | N=307 | Terminated | Sponsor: PTC Therapeutics | Trial completion date: Mar 2027 ➔ Jan 2025 | Active, not recruiting ➔ Terminated; Company decision

Trial completion date • Trial termination • Amyotrophic Lateral Sclerosis • CNS Disorders

PTC Therapeutics Announces Topline Results of CardinALS Trial of Utreloxastat in ALS Patients (PRNewswire) - P2 | N=307 | CardinALS (NCT05349721) | Sponsor: PTC Therapeutics | "PTC Therapeutics...announced that the global Phase 2 placebo-controlled CardinALS study did not meet its primary endpoint of slowing disease progression on the composite ALSFRS-R and mortality analysis. While there was modest numerical benefit recorded on the primary endpoint and correlation of favorable clinical effect with lowering of plasma neurofilament light chain (NfL), a biomarker of neuronal damage, statistical significance was not achieved (p= 0.52). In addition, significance was not achieved on the secondary efficacy endpoints....Utreloxastat was demonstrated to be safe and well tolerated in the CardinALS trial. However, due to the lack of efficacy and biomarker signal, further development is not planned at this time."

Biomarker • Discontinued • P2 data • Amyotrophic Lateral Sclerosis

CARDINALS: A phase 2, randomized, double-blind, placebocontrolled study to evaluate the efficacy and safety of Utreloxastat (PTC857) in ALS (ALS-MND 2024) - No abstract available

Clinical • Late-breaking abstract • P2 data

CardinALS: a phase 2, randomized, double-blind, placebo-controlled study of utreloxastat (PTC857) in patients with amyotrophic lateral sclerosis (ALS-MND 2024) - P2 | " Utreloxastat is an oral small molecule with a novel mechanism of action in development for the treatment of ALS. CardinALS, a Phase 2, global clinical trial is fully enrolled, with demographics consistent with contemporary late-stage clinical trials in ALS, and with top-line results expected in the fourth quarter of 2024."

Clinical • P2 data • Amyotrophic Lateral Sclerosis • CNS Disorders

CARDINALS: Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALS (clinicaltrials.gov) - P2 | N=307 | Active, not recruiting | Sponsor: PTC Therapeutics | Trial completion date: Feb 2025 ➔ Mar 2027

Trial completion date • Amyotrophic Lateral Sclerosis • CNS Disorders

CARDINALS: A Phase 2, Randomized, Double-blind, Placebo-controlled, Parallel-group Study to Evaluate the Efficacy and Safety of Utreloxastat (PTC857) in Patients with Amyotrophic Lateral Sclerosis (AAN 2024) - P2 | "Utreloxastat is currently under evaluation in CARDINALS, a clinical trial in patients with ALS."

Clinical • P2 data • Amyotrophic Lateral Sclerosis • CNS Disorders

CARDINALS: Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALS (clinicaltrials.gov) - P2 | N=258 | Active, not recruiting | Sponsor: PTC Therapeutics | Recruiting ➔ Active, not recruiting

Biomarker • Enrollment closed • Amyotrophic Lateral Sclerosis • CNS Disorders

PTC Therapeutics Provides an Update on Commercial Progress and R&D Pipeline at 42nd Annual J.P. Morgan Healthcare Conference (BioSpace) - "Topline results from the CardinALS trial of utreloxastat in ALS are expected in the fourth quarter."

P2 data • Amyotrophic Lateral Sclerosis • CNS Disorders

CARDINALS: a phase 2 randomized double-blind placebocontrolled parallel-group study to evaluate the efficacy and safety of utreloxastat (PTC857) in patients with ALS (ALS-MND 2023) - P2 | "Utreloxastat is an oral small molecule with a novel mechanism of action under development for the treatment of ALS and is currently under evaluation in CARDINALS, a phase II, global clinical trial of ALS patients. mjohnston@ptcbio.com"

Clinical • P2 data • Amyotrophic Lateral Sclerosis • CNS Disorders

First-in-Human Studies of Pharmacokinetics and Safety of Utreloxastat (PTC857), a Novel 15-Lipooxygenase Inhibitor for the Treatment of Amyotrophic Lateral Sclerosis. (PubMed, Clin Pharmacol Drug Dev) - "Utreloxastat showed no marked safety signal following single doses up to 1000 mg and multiple doses over 14 days of 500 mg once daily or 250 mg twice daily. The results support further development of utreloxastat for the treatment of patients with amyotrophic lateral sclerosis at a 250-mg twice-daily dose administered with food."

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

CARDINALS: Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALS (clinicaltrials.gov) - P2 | N=258 | Recruiting | Sponsor: PTC Therapeutics | Not yet recruiting ➔ Recruiting

Biomarker • Enrollment open • Amyotrophic Lateral Sclerosis • CNS Disorders

CARDINALS: Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALS (clinicaltrials.gov) - P2 | N=258 | Not yet recruiting | Sponsor: PTC Therapeutics

New P2 trial • Amyotrophic Lateral Sclerosis • CNS Disorders

PTC Therapeutics Provides an Update on Commercial Progress and R&D Pipeline at 40th Annual J.P. Morgan Healthcare Conference (Yahoo Finance) - “2022 Potential Key Value-Creating Milestones:...Results for MIT-E, the registration-directed study of vatiquinone in mitochondrial disease associated seizures, are expected in the fourth quarter of 2022...From the splicing platform, the PIVOT-HD Phase 2 study of PTC518 in Huntington's disease patients is planned to initiate in the first quarter of 2022. From the Bio-e platform, the registration-directed CardinALS study of PTC857 in amyotrophic lateral sclerosis (ALS) patients is expected to be initiated in the second quarter of 2022.”

New P2 trial • New trial • P2/3 data • Amyotrophic Lateral Sclerosis • CNS Disorders • Epilepsy • Huntington's Disease

PTC Therapeutics Inc (PTCT) Q3 2021 Earnings Call Transcript (The Motley Fool) - "As a reminder, PTC 857 inhibits the enzyme 15-Lipoxygenase....In a series of preclinical studies, we have demonstrated that PTC 857 is a potent protector of neuronal cell death and neuropathology in a number of in vitro and in vivo neurodegenerative disease test systems. We plan to initiate the Phase two study in the first quarter of 2022, and we'll provide additional details on the study design once it is finalized....We have selected ALS as the first indication and are aiming to initiate the Phase two trial for PTC 857 in the first quarter of next year...."

New P2 trial • Preclinical • Trial initiation date • Amyotrophic Lateral Sclerosis • CNS Disorders • Parkinson's Disease

PTC Therapeutics Provides a Corporate Update and Reports Second Quarter 2021 Financial Results (PRNewswire) - “A Phase 1 healthy volunteer trial of the second Bio-e compound, PTC857, was recently completed and results demonstrated predictable pharmacology and no reported tolerability findings. This allows dose selection necessary to move forward to Phase 2, which we plan to initiate for amyotrophic lateral sclerosis (ALS) in the first quarter of 2022. Results from additional cohorts are expected from the Phase 1 healthy volunteer trial of PTC518 for Huntington's disease in the third quarter of this year. Phase 2 planning is already underway and is expected to be initiated by the end of 2021.”

New P2 trial • P1 data • Amyotrophic Lateral Sclerosis • CNS Disorders • Huntington's Disease

PTC Therapeutics Reports Third Quarter 2020 Financial Results and Provides a Corporate Update (PRNewswire) - "Third Quarter Clinical Updates: A registration-directed Phase 2/3 placebo-controlled trial to evaluate vatiquinone (PTC743) in children with mitochondrial epilepsy began enrollment in October 2020....The Phase 1 single ascending dose study for PTC857 has been completed and dosing in the multiple ascending dose study is expected to be completed by the end of 2020."

Enrollment status • Trial status • CNS Disorders • Epilepsy • Parkinson's Disease

PTC Therapeutics Announces First Patient Dosed in Phase 1 Trial for PTC857 (PRNewswire) - "PTC Therapeutics, Inc....today announced that the first subject in the Phase 1 clinical trial evaluating PTC857 in healthy volunteers has been dosed....The Phase 1 study will evaluate PTC857 in healthy human volunteers with single and multiple ascending doses that will inform safety and pharmacology, and the design for the Phase 2 program."

Trial status • CNS Disorders • Parkinson's Disease

PTC Therapeutics to host first deep dive webinar on Bio-e platform (PRNewswire) - "PTC Therapeutics, Inc....today announced that it will hold a series of virtual deep dive webinars....The Bio-e platform deep dive will provide an overview of the Bio-e platform and its novel scientific approach to treating central nervous system (CNS) and non-CNS diseases. Bio-e platform technology will be reviewed in detail and prior clinical results for its lead compound PTC743 will be highlighted. Expectations for upcoming trials will be outlined, including potential registrational trials for PTC743 in refractory mitochondrial epilepsy and Friedreich ataxia (FA) and a Phase 1 trial for a second compound, PTC857, targeting GBA Parkinson's disease as its first indication."

Clinical data • Live event • New P1 trial • New trial • CNS Disorders • Epilepsy • Friedreich ataxia • Parkinson's Disease