CAMBRIDGE, UK – 31 March 2022 – Mission Therapeutics (“Mission”), a drug discovery and development company focused on protein homeostasis by selectively inhibiting deubiquitylating enzymes (DUBs), today announced it has been granted approval to take its lead USP30 DUB inhibitor, MTX652, into clinical study.

Mitochondria are essential for energy production and cellular health. When they become damaged or stop working properly, they are labelled with ubiquitin, marking them for degradation. The mitochondrial-associated DUB, USP30, removes this ubiquitin and thereby inhibits the degradation, which can affect cell health. Mission has developed MTX652 to inhibit USP30 with the aim to enable appropriate degradation of dysfunctional mitochondria to preserve and improve cellular health.

Specific kidney cells are rich in mitochondria and highly vulnerable to injury if those mitochondria are not working correctly. Mitochondrial dysfunction is strongly implicated in kidney injury and chronic kidney disease (CKD). In addition to kidney diseases, mitochondrial quality is implicated in a range of other poorly treated conditions such as idiopathic pulmonary fibrosis, muscular dystrophy and primary mitochondrial disease. Mission is also investigating the potential of USP30 inhibitors to treat these diseases.

Following candidate nomination last year, MTX652 has successfully advanced through preclinical regulatory toxicology studies and is ready to be progressed into the clinic. The planned initial clinical trial will evaluate the safety, tolerability and pharmacokinetics of MTX652 in over 60 participants given either a single or multiple doses. The trial is due to start in April 2022.

Dr Suhail Nurbhai, CMO of Mission Therapeutics, commented: “We are delighted that our lead programme is entering the clinic. Mission has built a groundbreaking platform for the discovery and development of first-in-class small molecule drugs that selectively target DUBs. MTX652 entering the clinic is a major milestone for the Company and a tremendous achievement. We are excited to be taking this important next step.”

Kidney diseases are a major global health problem and are reported by the US CDC as a leading cause of death in the US. CKD affects an estimated 780 million people around the world. The attributable UK NHS costs in 2009/10 were around £1.5 billion and the estimated US Medicare costs for CKD are greater than US$87 billion. Acute kidney injury (AKI) and CKD are increasingly viewed as a continuum of kidney disease and increase risk to patients of damage to non-renal organs, cardiovascular disease, and early death. Preventing these conditions is therefore an important unmet medical need.

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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.

CAMBRIDGE, UK – 22 March 2022 – Mission Therapeutics (“Mission”), a drug discovery and development company focused on protein homeostasis by selectively inhibiting deubiquitylating enzymes (DUBs), today announced that Dr Paul Wallace, Chief Business Officer, will attend the sixteenth annual BIO-Europe Spring conference (28-31 March).

BIO-Europe Spring is one of the most international gatherings in the biopharma industry, bringing together the life science community for global dealmaking. Dr Paul Wallace will join participants from other leading companies in the sector, attending meetings, panel discussions, and showcases, and is available for one-to-one meetings. The event is being held virtually.

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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.

CAMBRIDGE, UK – 7 March 2022 – Mission Therapeutics (“Mission”), a drug discovery and development company focused on protein homeostasis by selectively inhibiting deubiquitylating enzymes (DUBs), today announced that its VP of Translational Science, Dr Andy Pearce, will attend and present at the Babraham Research Campus Signalling Event on 9 March 2022.

In his presentation, titled ‘Targeting Deubiquitinase Enzymes for Treatment of a Broad Spectrum of Diseases’, Andy will discuss the Company, discovery platform, cross disease interest, and data from its lead program, USP30. The presentation at 13.35 will include time for questions and Andy will be available for one-to-one meetings.

Mission Therapeutics has built a world-leading proprietary DUB platform for the discovery and development of first-in-class drugs that selectively target specific disease-associated DUBs. As the VP of Translational Science, Dr Pearce leads the Translational Science department and drug discovery projects. He previously led projects and research groups for over 15 years at Novartis and GSK.

The Signalling Event will focus on the range of R&D ongoing across the Babraham campus, as well as highlighting scientific services and capabilities available on site.

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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.

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.