MJFF grants $2.7M to push new Parkinson’s drug toward human trials
Pipeline funding will allow lab testing of therapy targeting disease causes
Written by |
- The Michael J. Fox Foundation for Parkinson's Research has awarded $2.7 million to X-tosis to help advance its novel treatment XTS001 toward clinical testing.Â
- The drug targets a key driver of Parkinson's neurodegeneration, aiming to shift treatment from symptomatic relief to addressing the disease's causes.
- The funding, from the Therapeutics Pipeline Program, will support preclinical studies to gather safety and efficacy data, with a goal of advancing XTS001 into human trials.
The Michael J. Fox Foundation for Parkinson’s Research (MJFF) has granted $2.74 million to X-tosis to support the preclinical development of XTS001, the company’s lead candidate for the treatment of Parkinson’s disease.
The funding, granted through MJFF’s Parkinson’s Disease Therapeutics Pipeline Program, will support laboratory research into XTS001. The goal, according to a company press release announcing the award, is to gather initial safety and efficacy data to support investigational new drug (IND) studies that advance the experimental therapy into human trials.
Parkinson’s is caused by the gradual loss of dopaminergic neurons — nerve cells in the brain that produce dopamine, a signaling molecule involved in motor control — that ultimately leads to the disease’s symptoms. Although the mechanisms driving neuronal loss aren’t fully understood, problems with mitochondria, the energy-producing structures in cells, are thought to play a role.
“This award from The Michael J. Fox Foundation is more than funding; it is support for further investigation of our scientific approach that mitochondrial dysfunction is a central addressable driver of Parkinson’s disease,” said Erin Henderson, X-tosis’ CEO. “We believe XTS-001 represents a first-in-class opportunity to intervene upstream in the neurodegenerative cascade, potentially transforming the treatment paradigm from symptomatic management to targeting disease biology.”
XTS001, previously VBIT-4, is an oral, brain-penetrant small molecule designed to selectively inhibit the clumping, or oligomerization, of voltage-dependent anion channel 1 (VDAC1) — an ion channel essential for mitochondrial function. By preventing VDAC1 oligomerization, XTS001 is expected to restore mitochondrial function and prevent neuroinflammation and neuronal loss.
MJFF grant supporting research into brain-penetrating therapy XTS001
The researchers believe that stopping the loss of neurons, the nerve cells that control movement, could help in preventing, or at least slowing, the development of Parkinson’s.
We aim to shift treatment from symptomatic relief to targeting underlying disease biology.
According to the company, preclinical studies in animal models of Parkinson’s showed that VDAC1 oligomerization inhibitors reduced the loss of dopaminergic neurons, restored dopamine levels, and protected against other disease-associated events by preserving mitochondrial function.
“By preserving mitochondrial integrity upstream in the neurodegeneration cascade, we aim to shift treatment from symptomatic relief to targeting underlying disease biology,” said Yotam Nisemblat, chief scientific officer at X-tosis, who is serving as principal investigator on the project.
Nisemblat called the award “a major milestone for X-tosis,” saying it “represents a strong validation of our … approach.”
“We are grateful for MJFF’s support to advance confirmatory studies, biomarker development, and IND-enabling work,” Nisemblat added.
X-tosis is also developing XTS001 for other neurodegenerative conditions marked by mitochondrial dysfunction, such as Alzheimer’s disease. For that condition, the molecule was shown to prevent neuronal loss, neuroinflammation, and cognitive decline in a mouse model.
Leave a comment
Fill in the required fields to post. Your email address will not be published.