MedPath

Vesper Bio's VES001 Shows Promise in Phase I Trial for Frontotemporal Dementia

• Vesper Bio's VES001 demonstrated a favorable safety and tolerability profile in a Phase I trial involving 78 healthy subjects. • The study confirmed that VES001 effectively distributes to plasma and the central nervous system after oral administration. • VES001 significantly engaged its target, increasing progranulin levels and showing potential for normalizing concentrations in patients. • Based on Phase I results, Vesper Bio plans to initiate a Phase IIa proof-of-concept study in Q4 2024.

Vesper Bio has announced positive data from its Phase I clinical trial of VES001, a novel treatment for frontotemporal dementia (FTD) targeting progranulin gene mutations (GRN). The trial, involving 78 healthy volunteers, assessed the safety, tolerability, pharmacokinetics, and target engagement of VES001.
The study met all primary endpoints, with data from the multiple ascending dose (MAD) stage confirming earlier single ascending dose (SAD) findings. VES001 exhibited a high safety and tolerability profile, with no serious or treatment-emergent adverse events reported.

Pharmacokinetics and Target Engagement

Pharmacokinetic data indicated that VES001 is effectively distributed to both plasma and the central nervous system following oral administration. Furthermore, the treatment demonstrated significant target engagement, increasing progranulin levels in participants receiving daily doses. The data suggest that VES001 could substantially normalize progranulin concentrations in patients after seven days of dosing.

Mechanism of Action

VES001 is a brain-penetrant, small-molecule sortilin inhibitor designed to modify the course of FTD. It functions by inhibiting the sortilin receptor, which competes with progranulin receptors and contributes to neuronal damage when progranulin binds to it. By preventing this binding, VES001 aims to maintain and normalize progranulin levels, offering a convenient, orally administered treatment option.

Next Steps

Based on these promising Phase I results, Vesper Bio has submitted a clinical trial application (CTA) to initiate a Phase IIa proof-of-concept study. Patient dosing is expected to begin in the fourth quarter of 2024.

Management Commentary

"At Vesper, we are motivated by our patients and their relatives, who inspire our mission to develop innovative therapies that can help fight this awful disease," said Vesper Bio CEO Paul Little. "These promising clinical data coupled with VES001’s patient-friendly profile bring us one step closer to transforming patient outcomes in frontotemporal dementia."
Subscribe Icon

Stay Updated with Our Daily Newsletter

Get the latest pharmaceutical insights, research highlights, and industry updates delivered to your inbox every day.

Related Topics

Vesper Bio Initiates Phase Ib/IIa Trial of VES001 for Frontotemporal Dementia (FTD) with GRN Mutations

Vesper Bio has commenced a Phase Ib/IIa clinical trial, named SORT-IN-2, to assess VES001 in asymptomatic patients with GRN mutations causing frontotemporal dementia (FTD).
VES001, an orally administered drug, aims to normalize progranulin levels by inhibiting sortilin, a protein that degrades progranulin, which is deficient in FTD(GRN) patients.

Tenaya Therapeutics Doses First Patient in Phase 1b Trial of TN-401 Gene Therapy for ARVC

Tenaya Therapeutics has dosed the first patient in the RIDGE-1 Phase 1b trial evaluating TN-401 for PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC).
TN-401 is an AAV9-based gene therapy designed to deliver a functional copy of the PKP2 gene directly to heart cells, addressing the underlying genetic cause of ARVC.

VQ-101 Shows Promise in Phase 1 Trial for GBA-Parkinson's Disease

VQ-101, an oral GCase activator, demonstrated over 75% target engagement in healthy volunteers in a Phase 1a trial, exceeding the initial goal of 50% activation.
The Phase 1a trial showed VQ-101 achieved significant cerebrospinal fluid levels, indicating full central nervous system penetration, with mild to moderate adverse events.

Vesper Bio's VES001 Shows Promise in Phase 1 Trial for Frontotemporal Dementia

Vesper Bio announced positive results from its Phase 1 clinical trial of VES001, an oral drug for frontotemporal dementia (FTD) linked to GRN mutations.
The trial demonstrated excellent safety and tolerability of VES001 in healthy volunteers, with normalization of progranulin levels observed.

ProMIS Neurosciences' PMN310 Shows Promise in Phase 1a Alzheimer's Trial

PMN310, a monoclonal antibody targeting toxic amyloid beta oligomers, was well-tolerated in a Phase 1a trial with healthy volunteers.
The trial demonstrated that PMN310 crosses the blood-brain barrier, reaching cerebrospinal fluid concentrations suitable for target engagement.

FDA Lifts Partial Clinical Hold on Vigil Neuroscience's VG-3927 Phase I Trial, Expands to Alzheimer's Patients

The FDA has lifted the partial clinical hold on Vigil Neuroscience's Phase I trial of VG-3927, a treatment for neurodegenerative diseases, following a complete response from the company.
Interim data from the trial indicated VG-3927's safety, tolerability, and potential for once-daily dosing, alongside a sustained decline in soluble TREM2 levels in cerebrospinal fluid.

Basking Biosciences Initiates Phase II Trial of BB-031 for Acute Ischemic Stroke

Basking Biosciences has dosed the first subject in its Phase II RAISE trial evaluating BB-031 for acute ischemic stroke (AIS).
The trial is a multicenter, randomized, placebo-controlled, double-blinded study assessing safety, tolerability, and preliminary efficacy of BB-031.

Passage Bio Advances in Clinical Trial for Genetic FTD Treatment

Passage Bio reports promising progress in its phase 1/2 clinical trial for a genetic form of FTD, with updated interim data showing elevated progranulin levels in patients. The company plans to dose a second cohort in 2024 and expects further data by 2025.

ViGeneron Doses First Patient in Phase 1b Clinical Trial of VG901 for Retinitis Pigmentosa Treatment

ViGeneron GmbH has dosed the first patient in its Phase 1b clinical trial for VG901, a gene therapy aimed at treating retinitis pigmentosa caused by CNGA1 gene mutations. The trial evaluates the safety and efficacy of intravitreal VG901 injections, with results expected by the end of 2025.

Passage Bio Reports Preliminary Data from FTD-GRN Gene Therapy Trial

Passage Bio has released initial data from its Phase I/II upliFT-D clinical trial, showing promising results for its PBFT02 gene therapy in treating frontotemporal dementia with granulin mutations. The therapy, delivered via an AAV1 viral vector, significantly increased cerebrospinal fluid progranulin levels in patients, with levels maintained for up to six months post-treatment. The trial also found the therapy to be well-tolerated with an improved steroid regimen for immunosuppression.

Reference News

[1]
Vesper Bio reports data from Phase I frontotemporal dementia treatment trial
clinicaltrialsarena.com · Sep 5, 2024

Vesper Bio reported Phase I study data for VES001, a potential FTD treatment targeting GRN mutations, showing safety, to...

© Copyright 2025. All Rights Reserved by MedPath
HomeTerms & ConditionsPrivacy Policy