Mission Therapeutics ("Mission"), a drug discovery and development company focused on treating mitochondrial diseases, fibrosis and neurodegenerative disorders by selectively inhibiting deubiquitylating enzymes (DUBs), announced today that it has been awarded a Therapeutic Pipeline Program grant of approximately 0.5M USD from The Michael J. Fox Foundation for Parkinson's Research (MJFF).
Following a previous grant awarded in 2017, this research grant will support testing of Mission's lead CNS-penetrant USP30 DUB inhibitors in an in vivo model of Familial Parkinson's Disease (PD), as well as ongoing preclinical development towards candidate selection.
Parkinson's disease is a chronic, degenerative neurological disorder that affects one in 100 people over age 60. Estimates of the number of people living with the disease vary due to high rates of misdiagnosis, but research indicates that it is at least one million people in the United States and more than five million worldwide.
USP30 is a mitochondrial-associated DUB that negatively regulates mitophagy ? a process in which dysfunctional mitochondria are tagged with ubiquitin and selectively degraded and cleared from the cell. In some forms of familial PD, the protein that adds the ubiquitin, Parkin, is impaired, leading to reduced mitochondrial quality control. This can lead to degeneration of highly active substantia nigra neurons in the brain, which results in Early Onset Parkinson's Disease (EOPD). It is believed that inhibition of USP30 will improve mitochondrial quality control, resulting in neuroprotection.
The MJFF grant will fund a set of studies to measure the effect of Mission's lead CNS USP30 inhibitor on dopaminergic neuron protection. They will also assess indicators of mitochondrial quality control loss that are commonly observed in EOPD patients, such as elevated mitochondrial DNA mutations and inflammation.
Shalini Padmanabhan, PhD, Director of Research Programs at MJFF, said: "The number of people living with Parkinson's continues to grow, but there are currently no treatments capable of stopping or reversing progression of this debilitating disease. Mission's CNS-penetrant USP30 DUB inhibitors may offer an opportunity to make a meaningful impact on the lives of PD patients by improving the health of cells in the brain. We are proud to support a project aiming to meet this critical unmet need."
Dr Paul Thompson, Mission's Chief Scientific Officer, added: "Receiving funding from the Michael J. Fox Foundation is a great accolade, recognizing the importance of USP30 as a potential therapeutic target for PD, and the quality of Mission's chemistry. We are also pleased to contribute to further validation of non-clinical in vivo models of PD, an area which requires ongoing collaboration and support. We hope this will be the first of many Mission DUB inhibitor programs that can address specific disease-driving pathologies in both Parkinson's Disease and other neurodegenerative disorders."
It is anticipated that USP30 inhibitor mechanisms will apply to idiopathic PD, neurodegenerative disorders, and other diseases more broadly. Mission's first USP30 inhibitor, a non-CNS penetrant molecule, is scheduled to enter the clinic in early 2022 for kidney and liver disease.
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About Mission Therapeutics
Mission Therapeutics is an early-stage drug development company targeting the ubiquitin pathway for the treatment of kidney disease, neurodegenerative disease, rare mitochondrial diseases and fibrosis. The Company has built a leading platform for the discovery and development of first-in-class, small molecule drugs that selectively target deubiquitylating enzymes (DUBs) ? an emerging drug class that is attracting significant commercial interest in the area of protein homeostasis.
Mission has strong links with key academic and research centers, including Prof. Steve Jackson's Cancer Research UK Laboratories at the University of Cambridge Gurdon Institute, and leading UK centres in neurodegenerative diseases. The Company also has secured major industry partnerships, including its collaboration with AbbVie in November 2018, for the research and preclinical development of specified DUB inhibitors for the treatment of Alzheimer's Disease and Parkinson's Disease. The Company is managed by a team with broad international, commercial and clinical-science experience.
To date the Company has received £73 million / $101 million in funding and its investors comprise blue chip institutional and corporate investors including: Pfizer Venture Investments, Sofinnova Partners, Roche Venture Fund, SR One, IP Group and Rosetta Capital. Mission Therapeutics was founded in 2011 and is based at the Babraham Research Campus, Cambridge, UK.
About The Michael J. Fox Foundation for Parkinson's Research
As the world's largest nonprofit funder of Parkinson's research, The Michael J. Fox Foundation is dedicated to accelerating a cure for Parkinson's disease and improved therapies for those living with the condition today. The Foundation pursues its goals through an aggressively funded, highly targeted research program coupled with active global engagement of scientists, Parkinson's patients, business leaders, clinical trial participants, donors and volunteers.
In addition to funding $1 billion in research to date, the Foundation has fundamentally altered the trajectory of progress toward a cure. Operating at the hub of worldwide Parkinson's research, the Foundation forges groundbreaking collaborations with industry leaders, academic scientists and government research funders; increases the flow of participants into Parkinson's disease clinical trials with its online tool, Fox Trial Finder; promotes Parkinson's awareness through high-profile advocacy, events and outreach; and coordinates the grassroots involvement of thousands of Team Fox members around the world.
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