Targeting sortilin to rebalance progranulin levels thought to hold promise in CNS diseases, including Parkinson’s.
Danish biotech Vesper Bio revealed it has been awarded a grant worth approximately $873,000 by The Michael J Fox Foundation for Parkinson’s Research (MJFF). The Copenhagen-based company specializes in sortilin receptor biology will use the funding to facilitate the assessment of sortilin inhibitors in the treatment of Parkinson’s disease.
The research initiative, set to kick off this month, falls under the MJFF’s Parkinson’s Disease Therapeutics preclinical pipeline program, which supports promising therapeutic developments with the potential to prevent, stop, or delay Parkinson’s disease progression or alleviate daily symptoms.
Sortilin receptor biology is linked to diseases of the central nervous system, eye, cardiovascular system, dyslipidaemias, and oncology. Vesper’s approach involves targeting sortilin actions specifically at the cell membrane, differentiating it from methods that modulate intracellular trafficking of proteins.
The company’s lead program focuses on using a sortilin inhibitor to rebalance levels of progranulin, a protein crucial for regulating cell growth, survival, repair, and inflammation avoidance. Low progranulin levels are believed to contribute to cell dysfunction and damage in various neurological conditions.
“Vesper has developed orally administered, small molecule, sortilin inhibitors that elevate central progranulin levels and these hold promise for treating certain CNS diseases, including Parkinson’s disease,” said Dr Anders Nykjaer, Chief Scientific Officer of Vesper.
Vesper’s lead compound, VES001, is an orally administered treatment designed to address progranulin deficiency. With the ability to cross the blood-brain barrier, VES001 is particularly suitable for patients experiencing a rapid decline in mental function. Progranulin mutant alleles have implications in Frontotemporal Dementia (FTD) and Alzheimer’s disease, and high extracellular progranulin levels have been shown to have protective effects in models of ALS, Parkinson’s, stroke, arthritis, and atherosclerosis.
“MJFF greatly values research into the biological underpinnings of Parkinson’s disease and leveraging that insight for new treatment ideas,” said Dr Jessica Tome Garcia, Associate Director, Translational Research at MJFF. “We are proud to fund the work of researchers at Vesper Bio as they investigate new ways to fulfil the unmet needs of people with Parkinson’s.”
Vesper is currently advancing the development of VES001 for FTD through ongoing Phase I studies, including a Phase Ib Proof-of-Concept trial. Additionally, preparations are underway for Phase I trials of VES002, targeting a second, undisclosed, central nervous system indication.