Collaborative team to progress novel USP30 DUB inhibitors to develop potential therapies for Parkinson’s disease

 CAMBRIDGE, UK – 12 October 2021 – 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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 FOR MORE INFORMATION: 

Mission Therapeutics Ltd Anker Lundemose MD PhD Chief Executive Officer Tel: +44 (0)1223 607 340

Instinctif Partners Melanie Toyne-Sewell / Katie Duffell Tel: +44 (0) 20 7457 2013 missiontherapeutics@instinctif.com

NOTES TO EDITORS:

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.

For more information, please visit our website, www.missiontherapeutics.com, or follow us on Twitter or LinkedIn.

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.

For more information, visit us at www.michaeljfox.org, on Facebook, Twitter, Instagram and LinkedIn.

Mission receives $20 million milestone payment

CAMBRIDGE, UK – 9 August 2021 – Mission Therapeutics (“Mission”), a drug discovery and development company focused on targeting the ubiquitin pathway, and AbbVie (“AbbVie”)(NYSE: ABBV) today announced progression of selected deubiquitylating enzyme (DUB) targets into the next phase of research in their neurodegenerative disease collaboration.

In the second major milestone of the Companies’ research and preclinical development collaboration, AbbVie has nominated two DUB targets to progress to the next stage of drug discovery. This selection follows supportive data from in vitro and in vivo Alzheimer’s and Parkinson’s disease models. As a result of the nomination, Mission will receive a milestone payment of $20 million from AbbVie.

Alzheimer’s and Parkinson’s diseases are the most common neurological disorders worldwide, with over 50 million people living with dementia and Alzheimer’s disease, and 10 million more with Parkinson’s. While there are treatments to help reduce symptoms, there are no treatments available to help stop or reverse progression.

Both Alzheimer’s and Parkinson’s are associated with the accumulation of misfolded, proteins in the brain, which are believed to cause impaired function and death of nerve cells. DUBs can help to maintain cell health by regulating the degradation of these proteins.

Under the terms of the agreement, first announced in 2018, Mission and AbbVie are collaborating to identify novel DUB targets and discover associated inhibitor compounds for neurodegenerative diseases. AbbVie has the option to gain exclusive rights to develop and commercialize inhibitors of selected DUB targets and will pay Mission success-based milestones as well as royalty payments for each commercialized product.

Commenting on the development, Dr Anker Lundemose, Mission’s CEO, said:

“We are delighted to have reached this next major milestone in our collaboration with AbbVie and receive the $20 million milestone – this represents a big step forward for Mission and further highlights DUBs as targets for drug development. The successful and timely progression of two DUB targets into the drug discovery phase is further validation of our platform. We look forward to the continuation of this great neurodegenerative disease collaboration.”

Dr Eric Karran, Vice President, Neuroscience Discovery Research, AbbVie commented:

“There is an urgent need for treatments that can make tangible and lasting improvements to the lives of Alzheimer’s and Parkinson’s patients. Our collaboration with Mission has the potential to identify novel therapeutic options for neurodegenerative disorders. We have had a great experience working with Mission and are pleased to be able to continue to draw on their valued expertise as we enter the next phase of drug development.”

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FOR MORE INFORMATION:

Mission Therapeutics Ltd Anker Lundemose MD PhD Chief Executive Officer Tel: +44 (0)1223 607 340  AbbVie Media Frank Benenati +1 (224) 688-4169 frank.benenati@abbvie.com

Instinctif Partners Melanie Toyne-Sewell / Katie Duffell Tel: +44 (0) 20 7457 2013 missiontherapeutics@instinctif.com

 

 NOTES TO EDITORS:

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.

For more information, please visit our website, www.missiontherapeutics.com, or follow us on Twitter or LinkedIn.

About AbbVie

AbbVie’s mission is to discover and deliver innovative medicines that solve serious health issues today and address the medical challenges of tomorrow. We strive to have a remarkable impact on people’s lives across several key therapeutic areas: immunology, oncology, neuroscience, eye care, virology, women’s health and gastroenterology, in addition to products and services across its Allergan Aesthetics portfolio. For more information about AbbVie, please visit us at www.abbvie.com. Follow @abbvie on Twitter, Facebook, Instagram, YouTube and LinkedIn.

 

CAMBRIDGE, UK – 2 June 2021 – Mission Therapeutics (“Mission”), a drug discovery and development company focused on selectively inhibiting deubiquitylating enzymes (DUBs), has announced today that Paul Thompson, Chief Scientific Officer (CSO), will present at the upcoming Next Wave in Neurodegeneration Investor event (3 June), as part of the UBS Innovation Series.

Paul will present on Mission’s world-leading DUB platform and pipeline, including the potential of the Company’s lead in-house target, USP30, in treating neurodegenerative diseases, and will provide Mission’s perspective on the current research and development (R&D) landscape in this field. Paul’s 30 minute presentation will be followed by a Q&A session.

The Next Wave in Neurodegeneration Investor event brings together investors and innovative private companies engaged in R&D with a focus on neurodegenerative diseases. Executives will share an overview of the next-generation approaches companies are adopting to treat diseases such as Amyotrophic Lateral Sclerosis (ALS), Huntington’s, and Parkinson’s, and will discuss any successes and failures in their effort to achieve neurodegenerative modification.

– ENDS –

FOR MORE INFORMATION:

Mission Therapeutics Ltd
Anker Lundemose MD PhD
Chief Executive Officer
Tel: +44 (0)1223 607 340

Instinctif Partners
Melanie Toyne-Sewell / Katie Duffell
Tel: +44 (0) 20 7457 2013
missiontherapeutics@instinctif.com

NOTES TO EDITORS:

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.

For more information, please visit our website, www.missiontherapeutics.com, or follow us on Twitter or LinkedIn.

CAMBRIDGE, UK– 26 April 2021 – Mission Therapeutics (“Mission”), a drug discovery and development company focused on selectively inhibiting deubiquitylating enzymes (DUBs), has announced today that Paul Thompson, Chief Scientific Officer, will present at the Mitochondria-Targeted Drug Development Digital Summit (27-29 April).

Paul will present a seminar titled: USP30 Inhibitors: Releasing the Brakes to Improve Mitochondrial Quality Control During Disease, on 28 April at 14:15 EST / 20.15 CEST. He will explore multiple disease pathologies and detail how they can be prevented by inhibiting Mission Therapeutics’ in-house DUB target, USP30.

The Mitochondria-Targeted Drug Development Digital Summit is an industry-led meeting focused on end-to-end mitochondria targeting. The virtual event will be attended by over 80 senior CSOs, CEOs, research scientists, academics and investors, who share a commitment to tackling mitochondrial and age-related diseases. Over the three days, a series of presentations, workshops and networking sessions will focus on success stories, common obstacles, and opportunities in the field of mitochondria-targeted drug development.

– ENDS –

FOR MORE INFORMATION:

Mission Therapeutics Ltd
Anker Lundemose MD PhD
Chief Executive Officer
Tel: +44 (0)1223 607 340

Instinctif Partners
Melanie Toyne-Sewell / Katie Duffell
Tel: +44 (0) 20 7457 2013
missiontherapeutics@instinctif.com

NOTES TO EDITORS:

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.

For more information, please visit our website, www.missiontherapeutics.com, or follow us on Twitter or LinkedIn.