Vertero Initiates Phase 1 Study of VT-5006, A Selective Small Molecule Therapy for Parkinson’s Disease
VT-5006 is a first-in-class small molecule designed to precisely engage a validated peripheral target to delay onset and slow progression of Parkinson’s disease
WOBURN, Mass., Nov. 24, 2025 (GLOBE NEWSWIRE) -- Vertero Therapeutics, a clinical-stage biotechnology company breaking barriers in neurodegenerative disease treatment, today announced that it has initiated a Phase 1 study of its lead candidate, VT-5006, a first-in-class, gut-selective small molecule for the treatment of Parkinson’s disease (PD).
VT-5006 is a novel, oral therapeutic developed by Vertero that acts on CsgA, a microbial amyloid present in the GI wall that drives aggregation of alpha-synuclein (⍺Syn) and inflammation, which propagates to the brain and provokes PD progression. By targeting CsgA, VT-5006 aims to reduce the load of aggregation and dampen inflammation — potentially both alleviating symptoms and slowing disease progression to preserve functionality for affected individuals.
“We have reached an important milestone for Vertero as we transition into clinical development for VT-5006. This program reflects our strategy to change the way we approach neurodegenerative diseases by targeting causative triggers in peripheral systems that fuel these diseases,” said Becca Senter, Ph.D., Chief Scientific Officer. “We believe VT-5006 could effectively intervene upstream in the peripheral system to delay onset, slow progression and preserve quality of life for people with Parkinson’s disease, and we look forward to the insights this study will generate to support our program.”
The Phase 1a portion of the study is enrolling healthy volunteers to receive VT-5006 either as a single dose or once daily for seven days. Primary endpoints include safety, tolerability and pharmacokinetics of VT-5006 at sequentially-ascending doses compared to placebo. The Phase 1b portion will include adults who have been diagnosed with Parkinson’s disease in the last 10 years, who are at early or moderate stage of disease (Hoehn and Yahr (H&Y) scale < 3), and who are CsgA-positive. The trial is informed by promising preclinical studies which demonstrated reduced pathology and inflammation in the brain, improved motor function, and slowed disease progression, with a strong safety profile.
“Parkinson’s carries a considerable burden, not only for the affected individual but for caregivers and loved ones. Current therapies are limited primarily to symptom management, so there remains a persistent and urgent need for novel approaches to meaningfully address the disease and slow progression,” said Philip Kremer, MD, PhD, Research Director Neurology, Centre for Human Drug Research (CHDR), and principal investigator for this study. “As the healthcare community comes to understand the causative connection between peripheral triggers and neurodegenerative diseases, the insights from this trial will help further refine a strong clinical development program to demonstrate how this selective small molecule can alter the trajectory of Parkinson’s disease.”
For more information on VT-5006, please visit Vertero’s Science page.
About Vertero
Vertero Therapeutics is a clinical-stage company pioneering science beyond the brain to treat neurodegenerative diseases at their source. Vertero’s development pipeline of differentiated therapies targets the peripheral nervous system to delay onset and slow progression of challenging conditions such as Parkinson’s disease. The company's lead program currently in a Phase 1 clinical trial is VT-5006, designed to precisely treat a validated target in the gut that feeds the protein aggregation and inflammation implicated in Parkinson’s disease. The company also has an asset targeting bile acid dysregulation in early development for undisclosed indications. For more information, visit www.vertero.com or LinkedIn.
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