VES001 / Vesper Bio - LARVOL DELTA

SORTILIN INHIBITION ELEVATES PROGRANULIN LEVELS WITH THERAPEUTIC POTENTIAL IN PARKINSON'S DISEASE (ADPD 2026) - "VES002 and VES001, the clinical candidate for frontotemporal dementia (FTD), have been tested in a viral alpha-synuclein overexpression mouse model of PD (AAV-A53T-aSyn)... Orally delivered sortilin inhibitors hold promise as a novel therapeutic approach to attenuate neuroinflammation and neurodegeneration for different neurodegenerative diseases, including PD."

Alzheimer's Disease • CNS Disorders • Dementia • Frontotemporal Lobar Degeneration • Inflammation • Movement Disorders • Parkinson's Disease • SORT1

PHASE IB/2A DATA: SORTILIN INHIBITION WITH VES001 IN HUMAN GRN MUTATION CARRIERS WAS WELL-TOLERATED AND ELEVATED PLASMA AND CSF PROGRANULIN (ADPD 2026) - P1 | "Sortilin inhibition with the novel oral small molecule, VES001, increases PGRN in different compartments and holds promise as a novel therapeutic approach to attenuate neuroinflammation and neurodegeneration for different neurodegenerative diseases, including FTD-GRN."

Alzheimer's Disease • CNS Disorders • Dementia • Frontotemporal Lobar Degeneration • Inflammation • GRN • NGFR • SORT1

PHASE 1B/2A DATA ON SORTILIN INHIBITION WITH VES001 IN HUMAN GRN MUTATION CARRIERS – PRIMARY AND EXPLORATORY BIOMARKER READOUT (ADPD 2026) - P1 | "Sortilin inhibition with the novel oral small molecule, VES001 increases PGRN in different compartments and holds promise as a novel therapeutic approach to attenuate neuroinflammation and neurodegeneration for different neurodegenerative diseases, including FTD-GRN."

Biomarker • P1/2 data • Alzheimer's Disease • CNS Disorders • Dementia • Frontotemporal Lobar Degeneration • Inflammation • GRN • SORT1 • TARDBP

Phase 1B/2A data from SORT-IN-2: Sortilin inhibition with VES001 in GRN mutation carriers was well-tolerated and elevated plasma and CSF progranulin (CTAD 2025) - No abstract available

Late-breaking abstract • P1/2 data • GRN • SORT1

SORT-IN-2: Study in Asymptomatic GRN-FTD Patients to Investigate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VES001 (clinicaltrials.gov) - P1 | N=6 | Completed | Sponsor: Mads Kjolby | Active, not recruiting ➔ Completed

Trial completion • Alzheimer's Disease • CNS Disorders • Dementia • Frontotemporal Lobar Degeneration

SORT-IN-2: Study in Asymptomatic GRN-FTD Patients to Investigate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VES001 (clinicaltrials.gov) - P1 | N=6 | Active, not recruiting | Sponsor: Mads Kjolby | Enrolling by invitation ➔ Active, not recruiting

Enrollment closed • Alzheimer's Disease • CNS Disorders • Dementia • Frontotemporal Lobar Degeneration

SORTILIN INHIBITION WITH VES001 AND ELEVATION OF PROGRANULIN AS A NOVEL THERAPEUTIC APPROACH IN FTD -GRN AND OTHER NEURODEGENERATIVE DISEASES (ADPD 2025) - "Sortilin inhibition with oral small molecule VES001 increases PGRN in different compartments and holds pr omise as a novel therapeutic approach to attenuate neuroinflammation and neurodegeneration for different neurodegenerative diseases, including FTD -GRN."

Alzheimer's Disease • CNS Disorders • Dementia • Frontotemporal Lobar Degeneration • Inflammation • GRN • NGFR • SORT1

PHASE IA CLINICAL DATA ON SORTILIN INHIBITION WITH VES001 IN HEALTHY VOLUNTEERS WAS WELL-TOLERATED AND SHOWED PROGRANULIN ELEVATION IN PLASMA/CSF (ADPD 2025) - P1 | "No severe adverse events were associated with treatment. Conclusions Sortilin inhibition with novel oral small molecule, VES001, increases PGRN in different compartments and holds promise as a novel therapeutic approach to attenuate neuroinflammation and neurodegeneration for different neurodegenerative diseases, including FTD-GRN."

Clinical data • P1 data • Alzheimer's Disease • CNS Disorders • Dementia • Frontotemporal Lobar Degeneration • Inflammation • GRN • NGFR • SORT1 • TARDBP

SORT-IN-2: Study in Asymptomatic GRN-FTD Patients to Investigate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VES001 (clinicaltrials.gov) - P1 | N=6 | Enrolling by invitation | Sponsor: Mads Kjolby | Recruiting ➔ Enrolling by invitation

Enrollment status • Alzheimer's Disease • CNS Disorders • Dementia • Frontotemporal Lobar Degeneration

Vesper Bio initiates Phase Ib/IIa proof of concept study of VES001 in asymptomatic patients with gene mutations that cause frontotemporal dementia (FTD) (PRNewswire) - "Vesper Bio ApS...announces the initiation of a Phase Ib/IIa Proof of Concept (POC) study of its lead candidate VES001 for frontotemporal dementia. The SORT-IN-2 study will evaluate VES001 in patients with mutations in their progranulin-coding gene (GRN), which are causal for FTD(GRN)....SORT-IN-2 is an open-label, dual centre study assessing the clinical efficacy of VES001 in patients with GRN mutations who are currently asymptomatic. It will be performed at one clinical centre in the Netherlands and another in the United Kingdom. The first patient has now been enrolled and Vesper expects to have completed enrolment and dosing by mid-2025."

Trial status • Dementia • Frontotemporal Lobar Degeneration

SORT-IN-2: Study in Asymptomatic GRN-FTD Patients to Investigate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VES001 (clinicaltrials.gov) - P1 | N=6 | Recruiting | Sponsor: Mads Kjolby | Not yet recruiting ➔ Recruiting

Enrollment open • Alzheimer's Disease • CNS Disorders • Dementia • Frontotemporal Lobar Degeneration

SORT-IN-2: Study of How VES001 Works and How Safe it is to Participants With GRN-frontotemporal Dementia Showing no Symptoms (clinicaltrials.gov) - P1 | N=6 | Not yet recruiting | Sponsor: Mads Kjolby

New P1 trial • Alzheimer's Disease • CNS Disorders • Dementia • Frontotemporal Lobar Degeneration

Vesper Bio announces successful Phase I study for potentially disease-modifying treatment for frontotemporal dementia (PRNewswire) - P1 | N=78 | NCT06226064 | Sponsor: Vesper Biotechnologies ApS | "Vesper Bio ApS...announces the successful completion of the Phase I study for its lead candidate, VES001, a potentially disease-modifying treatment for frontotemporal dementia patients with mutations in the progranulin gene (FTD(GRN))....High levels of safety and tolerability of VES001 were observed across the doses tested, with no serious or treatment-emergent adverse events reported. The data demonstrate that following oral dosing, VES001 exhibits excellent pharmacokinetics and distribution to plasma and the targeted Central Nervous System compartment....Vesper Bio has completed a clinical trial application (CTA) seeking to progress VES001 into a Phase IIa proof-of-concept study in a relevant patient population, with dosing expected to start in Q4 2024."

New P2 trial • P1 data • CNS Disorders • Dementia

SORT-IN-1: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VES001 in Healthy Participants (clinicaltrials.gov) - P1 | N=78 | Completed | Sponsor: Vesper Biotechnologies ApS | Recruiting ➔ Completed

Trial completion • Alzheimer's Disease • CNS Disorders • Dementia

Vesper Bio announces completion of single ascending dose stage in trial of its lead candidate VES001, a potentially disease-modifying treatment for fronto-temporal dementia (PRNewswire) - P1 | N=78 | SORT-IN-1 (NCT06226064) | Sponsor: Vesper Biotechnologies ApS | "Vesper Bio ApS...today announces completion of the single ascending dose stage of its first-in-human trial of VES001 in healthy volunteers....Importantly, volunteers who received VES001 experienced significant and robust increases in levels of progranulin, demonstrating target engagement."

P1 data • Trial status • CNS Disorders • Dementia • Frontotemporal Lobar Degeneration

SORTILIN INHIBITION WITH VES001 AND ELEVATION OF PROGRANULIN AS A NOVEL THERAPEUTIC APPROACH IN FTD-GRN AND OTHER NEURODEGENERATIVE DISEASES (ADPD 2024) - "Sortilin inhibition with oral small molecule VES001 increases progranulin in different compartments and holds promise as a novel therapeutic approach to attenuate neuroinflammation and neurodegeneration for different neurodegenerative diseases, including FTD-GRN."

Alzheimer's Disease • CNS Disorders • Dementia • Frontotemporal Lobar Degeneration • Inflammation • GRN • NGFR • SORT1 • TARDBP

SORT-IN-1: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VES001 in Healthy Participants (clinicaltrials.gov) - P1 | N=78 | Recruiting | Sponsor: Vesper Biotechnologies ApS

New P1 trial • Alzheimer's Disease • CNS Disorders • Dementia