VB-08 / Vincere Biosci - LARVOL DELTA

ADVANCING A FIRST-IN-CLASS CNS-PENETRANT USP30 INHIBITOR TO INCREASE MITOPHAGY AS DISEASE MODIFYING THERAPY FOR PARKINSON'S DISEASE (ADPD 2026) - "Preclinical studies evaluated (i) cellular activity in human neural models of mitochondrial stress, (ii) in vivo engagement and enhancement of mitophagy in mitoQC reporter mice, (iii) safety, pharmacokinetics, and tolerability in IND-enabling studies. Vincere's first-in-class, oral, CNS-penetrant USP30 inhibitor restores mitochondrial quality control and has potential to be the first disease-modifying therapy targeting mitophagy in Parkinson's disease."

First-in-human • Alzheimer's Disease • CNS Disorders • Metabolic Disorders • Movement Disorders • Parkinson's Disease • Targeted Protein Degradation

ENCHANCEMENT OF MITOPHAGY VIA USP30 INHIBITION AS A THERAPEUTIC STRATEGY FOR ACUTE KIDNEY INJURY (ISN-WCN 2024) - "These findings highlight Vincere's USP30 inhibitor molecules potential in AKI treatment and as a prophylactic measure against CKD progression. The mitophagy-enhancing ability, coupled with selectivity and favorable pharmacological profile present a promising therapeutic approach in AKI management and CKD prevention."

Acute Kidney Injury • Chronic Kidney Disease • Nephrology • Renal Disease • Targeted Protein Degradation

VB-08: A SMALL MOLECULE INHIBITOR OF USP30 THAT ENHANCES MITOPHAGY (ADPD 2023) - "Here, we report a novel USP30 inhibitor, VB - 08... VB-08 presents a useful tool to test the effect of increased mitophag y in rodent models of neurodegeneration and sets the stage for translational development of a new and promising class of compounds for PD."

CNS Disorders • Movement Disorders • Parkinson's Disease • Targeted Protein Degradation