Mission Therapeutics’ Highlights Positive Parkinson’s Disease USP30 Target and Inhibitor Data at the Society for Neuroscience 2017 Annual Meeting

CAMBRIDGE, UK –15 November 2017 – Mission Therapeutics, the leading drug discovery and development company focused on selectively targeting deubiquitylating enzymes (DUBs) to treat neurodegenerative diseases, fibrosis, and other diseases with high unmet medical need, is presenting two posters with new research and preclinical data from its USP30 inhibitor Parkinson’s disease programmes today at Neuroscience 2017, the 47th Annual Meeting of the Society for Neuroscience, in Washington, DC. The meeting is the world’s largest neuroscience conference.

USP30 is a mitochondrial-associated DUB that has emerged as a promising new target in Parkinson’s disease. It has been implicated in the control of mitophagy, a cellular mitochondrial quality control mechanism. This process regulates the selective clearance of poorly functioning mitochondria by modifying levels of a protein called ubiquitin.

Failure of mitochondrial quality control results in the accumulation of dysfunctional energy-producing mitochondria, which causes oxidative stress. This stress eventually leads to a pathological mechanism that can result in Parkinson’s disease, and involves the degeneration of the highly-active substantia nigra neurons in the brain. Mission Therapeutics is developing potent and selective inhibitors against USP30 which can help improve mitochondrial quality control, with the aim of halting or slowing down the development of Parkinson’s.

Dr Anker Lundemose, Chief Executive Officer of Mission Therapeutics, said:

“DUBs are playing an emerging role as targets across a number of serious diseases, as highlighted in our team’s recently-published Nature Reviews Drug Discovery paper. The data from the studies presented at this conference further enhances our understanding of the cellular mechanisms of USP30 and the significance of inhibiting this particular DUB in Parkinson’s disease. Our research is also providing invaluable information that is helping to shape our preclinical development strategy.”

Details of Poster Presentations:

Poster# 759.03/Title: USP30 inhibitors for Parkinson’s Disease

Poster# 759.04/Title: Targeting USP30 in Parkinson’s iPSC-derived dopamine neurons

Session Title: The Pathogenesis Mechanisms of Mitochondria in Parkinson’s Disease

Session Number: 759

Session Time: Wednesday November 15th, 2017 1.00 pm – 5:00 pm ET

The meeting will be attended by two scientists from Mission Therapeutics’ CNS team: Dr Paul Thompson, Medical Director, CNS Translational Medicine and Dr Marc Watson. Principal Scientist, CNS.



Mission Therapeutics Ltd

Anker Lundemose MD PhD

Chief Executive Officer

Tel: +44 (0) 1223 497199

Instinctif Partners

Melanie Toyne-Sewell / Eileen Paul / Priya Kalia

Tel: +44 (0) 20 7457 2020


Westwicke Partners (U.S.)

Chris Brinzey

Tel: +1 339-970-2843




About Mission Therapeutics

Mission Therapeutics is an early-stage drug development company targeting the ubiquitin pathway for the treatment of neurodegenerative disease, fibrosis, inflammation, cancer and other diseases of unmet need. 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. Jackson’s Cancer Research UK Laboratories at the University of Cambridge Gurdon Institute, and leading UK centres in neurodegenerative diseases. The Company is managed by a team with broad international, commercial and clinical-science experience.

In February 2016, the Company completed an $86m financing that was led by Imperial Innovations and Woodford Patient Capital Trust and included participation from existing investors Sofinnova Partners, Roche Venture Fund, Pfizer Venture Investments and SR One. Mission Therapeutics was founded in 2011 and is based at the Babraham Research Campus, Cambridge, UK.