MJFF grant supports work into oral therapy to protect nerve cells

Vesper Bio aiming to modify disease by raising progranulin protein levels

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by Andrea Lobo |

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The Michael J. Fox Foundation for Parkinson’s Research (MJFF) has awarded an $873,000 grant to Vesper Bio to advance the preclinical development of small-molecule therapies aiming to increase progranulin levels in people with Parkinson’s disease.

Low levels of progranulin, a protein critical for immune activity and nerve cell survival, have been associated with the severity of Parkinson’s and other neurological conditions.

The project, expected to start this month, will assess Vesper compounds that inhibit sortilin, a receptor protein at the surface of cells in the nervous system that works to regulate progranulin levels. Sortilin suppression would raise progranulin levels, potentially offering a therapeutic avenue for neurodegenerative diseases like Parkinson’s.

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“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,” Anders Nykjaer, MD, PhD, Vesper Bio’s chief scientific officer, said in a company press release.

Vesper reports that its oral sortilin inhibitors can cross the blood-brain barrier, a highly selective membrane that prevents blood-borne threats like viruses and bacteria from reaching the central nervous system (CNS; the brain and spinal cord). This protective barrier also prevents the entry of large molecules, making it more difficult for intended therapies to reach the brain.

By normalizing progranulin levels, Vesper believes its compounds will have a disease-modifying effect by protecting and preserving a patient’s nerve cells.

The company’s lead compound, VES001, currently is being developed to treat people with frontotemporal dementia, a progressive brain disease largely marked by changes in behavior, cognition, and speech.

VS001’s safety, tolerability, and pharmacological properties are under evaluation in a Phase 1 clinical trial (NCT06226064) in healthy adults, testing single and multiple treatment doses at a single site in the Netherlands.

This study will be followed by a Phase 1b proof-of-concept trial of the treatment’s preliminary efficacy in asymptomatic people with mutations in the GRN gene, which provides instructions to produce progranulin. These mutations reduce the amount of progranulin produced and are linked to genetic cases of frontotemporal degeneration.

The company also is developing VES002, a treatment candidate for a yet undisclosed central nervous system indication.

Grant money was awarded under the MJFF’s Parkinson’s Disease Therapeutics Pipeline Program, helping to advance preclinical and clinical testing of promising approaches to ease disease symptoms or prevent, stop, or delay Parkinson’s progression.

“MJFF greatly values research into the biological underpinnings of Parkinson’s disease and leveraging that insight for new treatment ideas. We are proud to fund the work of researchers at Vesper Bio as they investigate new ways to [fulfill] the unmet needs of people with Parkinson’s,” said Jessica Tome Garcia, PhD, the foundation’s associate director of translational research.