Michael J Fox Foundation awards Vesper Bio over $870,000 for Parkinson’s treatment

26 Jan 2024
www.pmlive.com
Phase 1
Michael J Fox Foundation awards Vesper Bio over $870,000 for Parkinson’s treatment
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Source: PMLiVE
The Michael J Fox Foundation (MJFF) for Parkinson’s disease (PD) has awarded Vesper Bio a grant totalling $873,000 to assess sortilin inhibitors in the treatment of PD.
Beginning in January 2024, the project received the award under the MJFF’s PD Therapeutics Pipeline Program to advance preclinical testing of therapeutic developments to address unmet medical needs of the neurological condition.
Affecting nearly one million people in the US, PD is a neurological condition that gradually damages parts of the brain.
Vesper is currently expediting the development of VES001 for GRN-related frontotemporal dementia (FTD), an uncommon type of dementia that affects the frontal and temporal lobes of the brain and can cause issues with behaviour and language, through ongoing phase 1 studies.
Patients living with FTD may develop very similar symptoms to PD, otherwise known as FTD with parkinsonism or FTDP.
Progranulin is a protein that the body uses to regulate cell growth, survival, repair and avoid inflammation. When levels of the protein are low, cell dysfunction can occur and cause a range of neurological damage.
Vesper’s current lead compound, VES001, is a patient-friendly, first-in-class brain-penetrant treatment that is given orally. It works by targeting the progranulin deficiency and is able to cross the blood-brain barrier.
The clinical-stage biotech company suggests that its compounds will have a disease-modifying effect when normalising progranulin levels to protect and preserve remaining cells.
The studies will include a phase 1b proof of concept in GRN mutation carriers ahead of potentially registrational phase 2/3 trials, as well as a phase 1 readiness of VES002, a treatment focused on a second central nervous system indication.
Anders Nykjaer, chief scientific officer, Vesper Bio, commented: "This grant…  will support our research as we look to further demonstrate the efficacy of this novel therapy against this terrible disease."
Jessica Tome Garcia, associate director, translational research at MJFF, said the organisation is “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”.
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