PNT001 / Pinteon - LARVOL DELTA

A Phase 1 Single-Ascending-Dose Trial in Healthy Volunteers to Evaluate the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of Intravenous PNT001, a Novel Mid-domain Tau Antibody Targeting cis-pT231 Tau. (PubMed, J Prev Alzheimers Dis) - "Single doses of PNT001 were safe and well-tolerated at all dose levels studied, including those doses expected to produce therapeutic benefit. These results support multiple ascending dose trials in patients with neurodegenerative tauopathies for this novel mid-domain tau antibody."

Clinical • Journal • P1 data • PK/PD data • CNS Disorders • PSCA

Exploring the Therapeutic Potential of Phosphorylated Cis-Tau Antibody in a Pig Model of Traumatic Brain Injury. (PubMed, Biomedicines) - "These findings demonstrate a subtle reduction in the areas of white matter injury and biomarkers of neurological injury after treatment with PNT001 following TBI. These findings support additional studies for PNT001 as well as the potential use of this agent in clinical trials in the near future."

Journal • Preclinical • CNS Disorders • Vascular Neurology • GFAP

Preclinical characterization and IND-enabling safety studies for PNT001, an antibody that recognizes cis-pT231 tau. (PubMed, Alzheimers Dement) - "The data support clinical development of PNT001 in human tauopathies."

Journal • Preclinical

Safety and Tolerability of PNT001 in Patients With Acute Traumatic Brain Injury (TBI) (clinicaltrials.gov) - P1 | N=1 | Terminated | Sponsor: Pinteon Therapeutics, Inc | N=64 ➔ 1 | Recruiting ➔ Terminated; The study was terminated due to administrative, non-safety related reasons.

Enrollment change • Trial termination • CNS Disorders • Vascular Neurology

FINAL DATA FROM PHASE 1 SAD TRIAL OF PNT001, A MONOCLONAL ANTIBODY UNIQUELY RECOGNIZING CIS-PT231 TAU FOR TREATMENT OF TAUOPATHIES (ADPD 2022) - "PNT001 at all dose levels was well tolerated, including those doses expected to produce target engagement, supporting multiple ascending dose trials in patients with neurodegenerative tauopathies."

P1 data • CNS Disorders • Immunology • Inflammation

PNT001, A SELECTIVE CIS-PT231 TAU ANTIBODY, TARGETS BRAIN TAU SEEDS IN ALZHEIMER'S DISEASE, PROGRESSIVE SUPRANUCLEAR PALSY, AND A MOUSE MODEL OF TAUOPATHY (ADPD 2022) - "PNT001 targets tau seeds that occur in both 3R/4R and 4R tauopathies of AD and PSP. This further indicates cistau is included in seed-competent forms of tau that can be targeted with antibody therapies."

Preclinical • Alzheimer's Disease • CNS Disorders • Movement Disorders • Progressive Supranuclear Palsy

Final Data From a Phase 1 Single-Ascending-Dose Trial of PNT001, A Monoclonal Antibody Uniquely Recognizing cis-pT231 Tau for Treatment of Tauopathies (CTAD 2021) - "PNT001 was well tolerated in human volunteers at CSF drug concentrations expected to produce target engagement supporting multiple ascending dose studies in patients with neurodegenerative tauopathies like Alzheimer’s disease."

Late-breaking abstract • P1 data • Alzheimer's Disease • CNS Disorders • Epilepsy • Immunology • Inflammation

Safety and Tolerability of PNT001 in Patients With Acute Traumatic Brain Injury (TBI) (clinicaltrials.gov) - P1; N=64; Recruiting; Sponsor: Pinteon Therapeutics, Inc; Trial completion date: Jul 2022 ➔ Jan 2023; Trial primary completion date: Jul 2022 ➔ Jan 2023

Clinical • Trial completion date • Trial primary completion date • CNS Disorders • Vascular Neurology

Pinteon and Lonza Extend Collaboration for Manufacturing Antibody Therapeutic (Pharmtech) - “Pinteon Therapeutics, a biotechnology company, and Lonza announced on June 24, 2021 that they have expanded their manufacturing agreement to enable future clinical production of Pinteon's lead candidate, PNT001, which has shown promising results in a Phase I clinical study. This extension follows the multi-year manufacturing relationship between the companies established in Lonza's Slough, UK, site in 2018, which was to provide drug substance (DS) manufacturing for Phase I clinical supply. Starting in the fourth quarter of 2021."

Licensing / partnership • Alzheimer's Disease • CNS Disorders • Tauopathies And Synucleinopathies

Pinteon Therapeutics Encouraged by Publication of Preclinical Study Supporting PNT001 for the Treatment of Tauopathies in Science Translational Medicine (Businesswire) - “Pinteon Therapeutics…publication of a preclinical study supporting PNT001 for the treatment of vascular dementia and tauopathies in Science Translational Medicine…showed that a monoclonal antibody targeting cis-pT231 reduced neurodegeneration and cognitive impairment in a mouse model of vascular dementia and in a model of human tau overexpression…‘we believe this study provides further evidence for the ability of PNT001 to treat the cognitive impairments seen in tauopathies like Alzheimer’s disease and other neurodegenerative disorders. We are excited by these findings as we look to begin a Phase 1b/Phase 2 study of PNT001 in patients with Alzheimer’s disease later this year’…The company expects to initiate a Phase 1b/Phase 2 study of PNT001 in patients with Alzheimer’s disease by the end of 2021.”

New P1/2 trial • Preclinical • Alzheimer's Disease • CNS Disorders • Dementia • Tauopathies And Synucleinopathies

Safety and Tolerability of PNT001 in Patients With Acute Traumatic Brain Injury (TBI) (clinicaltrials.gov) - P1; N=64; Recruiting; Sponsor: Pinteon Therapeutics, Inc; Not yet recruiting ➔ Recruiting

Enrollment open • CNS Disorders • Vascular Neurology

Safety and Tolerability of PNT001 in Healthy Adults (clinicaltrials.gov) - P1; N=49; Completed; Sponsor: Pinteon Therapeutics, Inc; Active, not recruiting ➔ Completed

Clinical • Trial completion

[VIRTUAL] A PHASE 1 SINGLE ASCENDING DOSE STUDY OF PNT001, A MONOCLONAL ANTIBODY RECOGNIZING CIS-PT231 TAU FOR TREATMENT OF TAUPATHIES (ADPD 2021) - "Well-tolerated doses provided CSF concentrations of PNT001 several fold in excess of the Kd for PNT001-antigen binding.  Conclusions PNT001 was well tolerated in human volunteers at doses that provide a potentially therapeutic CSF drug concentration. These results support further multiple ascending dose studies in patients with tauopathy."

P1 data

[VIRTUAL] EVALUATION OF PNT001, A MONOCLONAL ANTIBODY DIRECTED TOWARD CIS-PT231 TAU, IN HUMAN TISSUE AND THE TG4510 TAU TRANSGENIC MOUSE (ADPD 2021) - "PNT001 selectively recognizes cis-pT231 tau in PSP, AD and CTE, reduces seeding activity from AD brain, and improves biochemical and functional endpoints in an aggressive model of pathologic tau overexpression. These data support clinical entry into PSP, AD, TBI and other tauopathies."

Alzheimer's Disease • CNS Disorders

Safety and Tolerability of PNT001 in Patients With Acute Traumatic Brain Injury (TBI) (clinicaltrials.gov) - P1; N=64; Not yet recruiting; Sponsor: Pinteon Therapeutics, Inc

Clinical • New P1 trial • CNS Disorders • Vascular Neurology

Safety and Tolerability of PNT001 in Healthy Adults (clinicaltrials.gov) - P1; N=48; Active, not recruiting; Sponsor: Pinteon Therapeutics, Inc; Recruiting ➔ Active, not recruiting

Clinical • Enrollment closed

[VIRTUAL] EVALUATION OF PNT001, A MONOCLONAL ANTIBODY DIRECTED TOWARD CIS-PT231 TAU, IN HUMAN TISSUE AND THE TG4510 TAU TRANSGENIC MOUSE (AAT-ADPD 2020) - "Conclusions Our studies demonstrate that PNT001 selectively recognizes cis-pT231 tau in PSP, AD, CTE and Pick’s disease and is able to improve biochemical and functional endpoints in an aggressive model of pathologic tau overexpression. These data suggest support clinical entry into PSP, AD and other tauopathies."

Preclinical