According to a story from PR Newswire, the biotech company Mission Therapeutics was just awarded $5.2 million in funding to support MTX325, the company’s investigational Parkinson’s disease therapy. Intended to treat patients in the early stages of the illness, the funds came from two nonprofit organizations: Parkinson’s UK and The Michael J. Fox Foundation for Parkinson’s Research.
About MTX325
MTX325 is currently in the midst of phase 1 trials. The funds will support dosing of the drug for 28 days during the program. The dosing portion of the trial is expected to begin in early 2025. The drug is classified as an inhibitor of USP30, a mechanism that is believed to protect neurons responsible for the production of dopamine through improving mitochondrial quality and functioning. This process is called mitophagy, in which deficient mitochondria are identified and then removed. USP30, a deubiquitylating enzyme (DUB), has been found to impede regular mitophagy.
The trial initiated in March of this year with an ascending, single dose stage utilizing healthy human volunteers. This initial phase produced good results, which demonstrated sufficient penetration of the central nervous system (CNS) and a promising overall safety profile.
The monetary support from two of the most prominent nonprofit organizations in the Parkinson’s disease space is a strong signal of optimism and support of the drug’s mechanism of action. Currently available therapies for Parkinson’s have limited effectiveness, so the patient community will likely be watching the progress of this drug’s development closely.
A continuously growing body of research is implicating dysfunctional mitochondria as contributing to a number of diseases, such as Duchenne muscular dystrophy, idiopathic pulmonary fibrosis, and kidney disease.
About Parkinson’s Disease
Parkinson’s disease is a type of long term, progressive, degenerative illness that affects the central nervous system. Symptoms tend to develop over a period of years and primarily affect the movement ability and mental state of the patient. The cause of Parkinson’s disease remains a mystery, although there are a number of risk factors that have been identified. These factors include head injuries, pesticide exposure, and certain genetic variants and mutations. About 15% of patients have a close relative with the disease, suggesting some genetic connection. Symptoms include slowed movements, poor coordination, trouble walking, shaking, stiffness, abnormal posture, depression, anxiety, inhibited thinking, hallucinations, and dementia. Treatment may involve a number of medications, rehabilitation, and surgical operations. Survival rate varies, but most patients survive around a decade after getting diagnosed. To learn more about Parkinson’s disease, click here.
