MJFF backs protein-targeting Parkinson’s therapy with $3.4M grant

The Michael J. Fox Foundation for Parkinson’s Research (MJFF) awarded Lysoway Therapeutics $3.4 million in funding to support the development of an investigational therapy targeting the TMEM175 protein.

The grant is part of the foundation’s Parkinson’s Disease Therapeutics Pipeline Program, which aims to accelerate the development of potential disease-modifying therapies that can prevent, halt, or delay disease progression, as well as therapies that target the disease’s symptoms.

Under the now-funded project, researchers will assess whether a small-molecule, brain-penetrant agonist (activator) of TMEM175 can affect the function of lysosomes, cellular structures that help clear waste and recycle damaged molecules.

“The Therapeutics Pipeline Program is designed to support the evaluation of promising therapeutic approaches grounded in Parkinson’s biology,” Jessica Tome Garcia, PhD, lead scientific program manager, translational research at MJFF, said in a company press release. “We are interested in better understanding targets like TMEM175 and their role in lysosomal function, and how these pathways may inform future therapeutic strategies.”

Yongchang Qiu, PhD, co-principal investigator and Lysoway’s founder and CEO, said the grant will help advance the company’s lead TMEM175 agonist candidate, establish target engagement biomarkers, and initiate investigational new drug (IND)-enabling studies. IND studies are preclinical tests and safety evaluations before a drug can be tested in humans.

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Keeping lysosomes working

Parkinson’s is caused by the progressive loss of dopaminergic neurons (nerve cells that produce dopamine, a signaling molecule involved in motor control). A hallmark of Parkinson’s is the formation of toxic clumps of misfolded alpha-synuclein in nerve cells, believed to contribute to neuronal dysfunction and death. As neurons die, Parkinson’s symptoms appear and gradually worsen.

Healthy cells use lysosomes to clear away misfolded proteins like alpha-synuclein before they can accumulate, a recycling process known as autophagy. Lysosomes usually function in an acidic, or low pH, environment. If pH equilibrium is disrupted, lysosomes cannot function properly, leading to the toxic accumulation of proteins.

TMEM175 is an ion channel protein important for maintaining lysosomal acidity, and mutations in the TMEM175 gene have been identified as a risk factor for Parkinson’s disease.

Lysoway has developed a brain-penetrant, small-molecule TMEM175 agonist to restore protein function, restore lysosomes’ pH and function, and enhance clearance of disease-causing alpha-synuclein clumps.

“TMEM175 is genetically linked to Parkinson’s disease risk and plays a critical role in maintaining lysosomal pH, autophagic capacity, and cellular resilience,” Valerie Cullen, PhD, principal investigator and senior vice president of research and translation at Lysoway. “Our lead development candidate is both orally bioavailable and highly brain-penetrant, addressing key challenges historically associated with targeting lysosomal ion channels for neurodegenerative diseases.”

The company received a previous MJFF grant of $2.93 million to advance a potential therapy targeting TRPML1, a protein that regulates calcium signaling, a key pathway in autophagy.

“The advancement of a second development candidate targeting a lysosomal ion channel further highlights the capabilities of our structure-based discovery platform and our ability to translate complex lysosomal biology into small molecules approaches for therapeutic investigation,” Qiu said.