Mission Therapeutics announces US FDA approval to initiate Phase II clinical trial of its lead asset MTX652 in Acute Kidney Injury

  • Mission gains clearance from US FDA for Phase II trial of MTX652 after it receives official approval of its Investigational New Drug (IND) application
  • Phase II trial expected to begin Q1 2024 in up to 160 patients with acute kidney injury (AKI) following cardiac surgery

Cambridge, UK – December 14, 2023 – Mission Therapeutics (“Mission” or the “Company”), a clinical-stage biotech developing first-in-class therapeutics targeting mitophagy, today announces that the US Food and Drug Administration (FDA) has approved its Investigational New Drug (IND) application for its lead asset MTX652, allowing Mission to proceed with its planned Phase II clinical trial in the US.

Up to 160 adults with increased risk for Acute Kidney Injury (AKI) following cardiac surgery are planned to be recruited for the trial from multiple sites in North America and Europe. The double-blind, placebo-controlled trial is expected to begin early next year and is intended to show that MTX652 protects this high-risk group of patients from AKI by assessing standard markers of renal function and renal injury over time.

Dr Anker Lundemose, Chief Executive Officer of Mission Therapeutics, said: “The FDA’s approval of our Phase II clinical study for our lead asset MTX652 marks a major milestone for Mission. We now have two USP30 inhibitors advancing through clinical trials, MTX652 for acute kidney injury and MTX325 for Parkinson’s Disease, validating our unique approach and the breadth of our assets.

Dr Suhail Nurbhai, Chief Medical Officer of Mission Therapeutics, said: Recent reports suggest that up to 50% of high-risk patients suffer acute kidney injury following heart surgery[i] [ii]. There are no approved drug treatments and the immediate and longer-term consequences can be very serious, including continued decline of renal function and/or the requirement for renal replacement therapy. We believe MTX652 has the potential to alleviate these outcomes and to meet this serious and important unmet medical need. We are delighted to have received this approval and look forward to starting this trial in 2024.

Dr Paul Thompson, Chief Scientific Officer of Mission Therapeutics, commented:In preclinical experiments, MTX652 demonstrated significant protective effects in multiple models of kidney injury, and this breadth of effect is very encouraging in indicating potential clinical benefit. We are very pleased with this strong preclinical foundation, which supports our progress into clinical efficacy trials.”

The FDA’s decision follows completion of a Phase I First Time in Human (FTIH) study of MTX652 earlier in 2023, in which over 80 healthy volunteers received MTX652. The trial successfully demonstrated MTX652 was safe and well tolerated up to a single dose of 200mg a day and multiple doses of 100mg daily for 14 days, with an excellent pharmacokinetic profile.

About MTX652 and USP30

MTX652 is a potent and selective compound designed to improve mitochondrial quality and function by enhancing mitophagy through inhibition of USP30, a deubiquitylating enzyme localised to mitochondria which is a negative regulator of mitophagy. It thereby promotes cellular health by enhancing this key mechanism, which cells use to clear dysfunctional mitochondria. MTX652 has the potential to reduce the impact of impaired mitochondria associated with Ischaemic Reperfusion Injury (IRI) in the heart and kidneys in patients who have undergone heart surgery.

ENDS 

For further information, please contact:

Mission Therapeutics Ltd:

Anker Lundemose MD PhD

Chief Executive Officer

Tel: +44 (0)1223 607 340

Optimum Strategic Communications:

Mary Clark, Stephen Adams, Vici Rabbetts

Email: mission@optimumcomms.com

Tel: +44 (0) 208 078 4357

About Mission Therapeutics

Mission Therapeutics is a world leader in discovering and developing novel therapeutics which promote the removal of dysfunctional mitochondria, promoting cell health and function. Mitochondria are energy producing organelles which require lifetime quality control through a ubiquitin-mediated clearance mechanism known as mitophagy. In certain situations, such as cellular stress, cell injury, and/or defects of the mitophagy process, the mitochondria can become dysfunctional and damaging to the cell, leading to reduced energy production, oxidative stress, inflammation and potentially cell death. Dysfunctional mitochondria are significant drivers of disease pathophysiology in acute kidney injury (AKI), Parkinson’s Disease (PD), heart failure, Duchenne’s Muscular Dystrophy, IPF, mitochondrial diseases and Alzheimer’s.

USP30 is a deubiquitylating enzyme that constantly removes ubiquitin from mitochondria, providing a potential brake on clearance of dysfunctional mitochondria. Mission is currently developing two small molecule drugs, MTX652 (peripheral) and MTX325 (targeting the CNS) which, through inhibition of the mitochondrial DUB enzyme USP30, will promote clearance of dysfunctional mitochondria – consequently improving overall cellular health. Mission’s USP30 inhibitors MTX652 and MTX325 could potentially be used to treat any disease or condition driven by mitochondrial dysfunction.

Mission is backed by blue chip investors including Pfizer Venture Investments, Sofinnova Partners, Roche Venture Fund, SR One, IP Group and Rosetta Capital.

[i] https://guardtherapeutics.com/en/press-releases/guard-therapeutics-reports-robust-efficacy-of-rmc-035-in-phase-2-akita-and-advances-clinical-development-program

[ii] https://pubmed.ncbi.nlm.nih.gov/34474590/

Mission Therapeutics granted MHRA Clinical Trial Authorisation (CTA) for MTX325 for the treatment of Parkinson’s Disease

  • Company on-track to dose first participant in multi-part, adaptive Phase 1, first-in-human study in healthy volunteers and Parkinson’s Disease (PD) patients in the first quarter of 2024
  • CTA comes as Nature Reviews Drug Discovery hails Mission’s mitophagy approach as “an appealing disease-modifying therapeutic strategy” for PD

Cambridge, UK – 5 December 2023 – Mission Therapeutics (“Mission” or the “Company”), a clinical-stage biotech developing first-in-class therapeutics targeting mitophagy, today announces that the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) has granted clinical trial authorisation (CTA) for Mission to commence its multi-part, adaptive Phase 1, first-in-human study evaluating the safety and tolerability of MTX325, intended for the treatment of Parkinson’s Disease (PD).

“The MHRA’s authorisation marks a major step forward in our mission to develop MTX325 as a disease-modifying therapy for Parkinson’s Disease,” said Anker Lundemose, Chief Executive Officer, Mission Therapeutics. “Having recently published data[i] showing that MTX325 produced a similar beneficial effect to knockout of USP30 in a mouse model of PD, bringing MTX325 into the clinic – with its potential to preserve vital dopamine-producing neurons in the brain – demonstrates our commitment to pushing the boundaries of research in diseases with limited treatment options. Following successful completion of the Phase 1 clinical study with our peripheral compound, MTX652, earlier this year, we now have two compounds targeting mitophagy in clinical phase testing and this approval represents significant further validation of our unique approach to target unmet medical needs.”

Dr Paul Thompson, Chief Scientific Officer, Mission Therapeutics, said: “There are currently no approved treatments for Parkinson’s Disease which modify the underlying pathology of this common and devastating degenerative condition. By enhancing mitophagy, MTX325 is designed to address the loss of dopamine-producing neurons resulting from the build-up of dysfunctional mitochondria in these brain cells. Mitophagy is the natural ‘quality control system’ cells use to clear out dysfunctional mitochondria and we believe that increasing the removal of these dysfunctional mitochondria will improve the health of dopaminergic neurons and thereby slow the progression of PD.”

Dr Suhail Nurbhai, Chief Medical Officer, Mission Therapeutics, commented “We are delighted to have approval to start clinical testing with MTX325. This Phase 1 trial is intended to confirm the safety and tolerability of MTX325, in both healthy volunteers and patients with Parkinson’s Disease, and help us determine appropriate doses for future efficacy testing. We look forward to starting the trial in the coming months, and providing preliminary first-in-human data later in 2024.”

 

Up to 160 adults, both healthy people and those with PD, will be recruited for the trial, which is planned to take place at sites across the UK.

 

The CTA comes as the journal Nature Reviews Drug Discovery identified Mission’s recent work with scientists at Cambridge University and Harvard University – investigating the potential of MTX325 in PD mouse modelsi – as a ‘Research Highlight’. NRDD commented: “Mitochondrial dysfunction and reduced mitophagy are strongly implicated in the pathogenesis of dopaminergic neurodegeneration in both sporadic and familial Parkinson’s Disease (PD). Restoring mitophagy to accelerate the removal of damaged mitochondria is therefore an appealing disease-modifying therapeutic strategy.”[ii]

 

About MTX325 and USP30

MTX325 is a potent selective central nervous system-penetrant compound designed to improve mitochondrial quality and function by enhancing mitophagy. MTX325 inhibits USP30, a deubiquitylating enzyme localised to mitochondria which is a negative regulator of mitophagy. Data from an in vivo model of Parkinson’s, where USP30 was deleted through gene knockout, validate USP30 as a potential target in PD. Researchers found MTX325 produced a similar effect to gene knockout of USP30 in the same PD mouse model, further validating the approach of USP30 inhibition in PD. See the paper in Nature Communications here: https://www.nature.com/articles/s41467-023-42876-1

– ENDS –

For further information, please contact:

 

Mission Therapeutics Ltd

Anker Lundemose MD PhD

Chief Executive Officer

Tel: +44 (0) 122 360 7340

 

Optimum Strategic Communications:

Mary Clark, Stephen Adams, Vici Rabbetts, Vareen Outhonesack

Tel: +44 (0) 208 078 4357

E-mail: mission@optimumcomms.com

 

About Mission Therapeutics

Mission Therapeutics is a world leader in discovering and developing novel therapeutics which promote the removal of dysfunctional mitochondria, promoting cell health and function. Mitochondria are energy producing organelles which require lifetime quality control through a ubiquitin-mediated clearance mechanism known as mitophagy. In certain situations, such as cellular stress, cell injury, and/or defects of the mitophagy process, the mitochondria can become dysfunctional and damaging to the cell, leading to reduced energy production, oxidative stress, inflammation and potentially cell death. Dysfunctional mitochondria are significant drivers of disease pathophysiology in acute kidney injury (AKI), Parkinson’s Disease (PD), heart failure, Duchenne’s Muscular Dystrophy, IPF, mitochondrial diseases and Alzheimer’s.

 

USP30 is a deubiquitylating enzyme that constantly removes ubiquitin from mitochondria, providing a potential brake on clearance of dysfunctional mitochondria. Mission is currently developing two small molecule drugs, MTX652 (peripheral) and MTX325 (targeting the CNS) which, through inhibition of the mitochondrial DUB enzyme USP30, will promote clearance of dysfunctional mitochondria – consequently improving overall cellular health. Mission’s USP30 inhibitors MTX652 and MTX325 could potentially be used to treat any disease or condition driven by mitochondrial dysfunction.

 

Mission is backed by blue chip investors including Pfizer Venture Investments, Sofinnova Partners, Roche Venture Fund, SR One, IP Group and Rosetta Capital.

[i] https://www.nature.com/articles/s41467-023-42876-1

[ii]USP30 inhibition protects dopaminergic neurons (nature.com)

Mission Therapeutics announces publication in Nature Communications outlining potential of experimental drug MTX325 as a disease-modifying therapy for Parkinson’s Disease

International research collaboration between Harvard Medical School, Cambridge University and Mission Therapeutics

CAMBRIDGE, UK, 13 November, 2023 – Mission Therapeutics (“Mission”), a clinical-stage biotech company developing first-in-class therapeutics targeting mitophagy, today announces the publication of a peer-reviewed article titledKnockout or inhibition of USP30 protects dopaminergic neurons in a Parkinson’s Disease (PD) mouse model’ in the journal Nature Communications, which can be freely accessed here. The research was the result of collaborative work between Cambridge University, Harvard Medical School, and Mission Therapeutics.

Mission Therapeutics is a global leader in discovering and developing innovative therapeutics that promote mitophagy. This quality control process enables the removal of dysfunctional mitochondria, thereby improving cell health and function. Parkinson’s Disease is highly associated with mitochondrial dysfunction, making it a key pathophysiological driver. Mission believes that by improving mitophagy, it can reduce the burden of dysfunctional mitochondria and thus slow or prevent the progression of Parkinson’s Disease, which is a major unmet need for patients with this condition.

Dr Paul Thompson, Chief Scientific Officer at Mission Therapeutics, said: “It is well recognized that mitochondrial dysfunction is a key driver of Parkinson’s Disease mechanisms, in particular, playing an important role in the degeneration of brain cells that produce dopamine. By inhibiting the enzyme USP30, Mission’s experimental drug MTX325 helps promote mitochondrial quality control by increasing the removal of dysfunctional mitochondria. This is likely to have a positive impact on dopaminergic neurons undergoing chronic degenerative processes, which result in functional impairment. This paper strongly supports further study of USP30 inhibition as a potential disease-modifying therapy for Parkinson’s Disease.”

Dr Anker Lundemose, Chief Executive Officer at Mission Therapeutics, said: “The exciting findings of this Nature Communications paper are a tremendous boost to Mission’s Parkinson’s Disease programme. We look forward to starting our first in-human trial of MTX325 early next year.”

The Nature Communications paper outlines how Mission’s drug MTX325 has potential as a novel, disease-modifying treatment for Parkinson’s, by enhancing mitophagy and clearing dysfunctional mitochondria. Dysfunctional mitochondria are usually tagged for removal via mitophagy with a protein ‘flag’ called ubiquitin. However, the enzyme USP30 removes these flags, inhibiting normal mitophagy, leading to a build-up of dysfunctional mitochondria in cells. By inhibiting USP30, MTX325 helps restore normal mitophagy and thus cellular health.

The Nature Comms paper provides key experimental evidence to support USP30 as a valid target in PD through both in vitro mechanisms as well as in vivo PD models. Using both a USP30 knockout mouse model and a pharmacological strategy with Mission’s experimental USP30 inhibitor drug MTX325, we found that USP30 inhibition led to protection against loss of dopamine and dopaminergic neurons induced by alpha-synuclein in vivo and reduced potential biomarkers of disease including phosphorylated alpha-synuclein and glial cell activation.

These new findings suggest that maintaining healthy mitochondrial function by blocking USP30 may slow or even stop the progression of pathology which drives PD.

Prof Gabriel Balmus, Cambridge University, said: “Our study has shown that it is possible to enhance the removal of damaged mitochondria through a process called mitophagy. We demonstrated this by inhibiting the activity of USP30 in both mice and human cells, which resulted in the increased removal of damaged mitochondria and the subsequent protection of dopaminergic neurons against the harmful effects of Parkinson’s Disease, which would typically result in the neurons’ death. This research provides compelling evidence that USP30 is a promising therapeutic target for Parkinson’s disease, where there is a pressing need for disease-modifying treatments.”

Prof David K. Simon, Harvard Medical School, said: “Our study of USP30 KO mice in a synuclein-driven PD model clearly shows an advantage of removing USP30 for dopaminergic neuron protection and maintenance of normal motor function. It has been our pleasure to collaborate with Cambridge and Mission Therapeutics on these studies, and we are keen to see the outcomes of early MTX325 clinical investigations.”

Mission is planning to initiate a MTX325 Phase I trial in humans in early 2024.

ENDS

For further information, please contact:

Mission Therapeutics Ltd:

Anker Lundemose MD PhD

Chief Executive Officer

Tel: +44 (0)1223 607 340

Optimum Strategic Communications:

Mary Clark, Stephen Adams, Vici Rabbetts

Email: mission@optimumcomms.com

Tel: +44 (0) 208 078 4357

About Mission Therapeutics

Mission Therapeutics is a world leader in discovering and developing novel therapeutics which promote the removal of dysfunctional mitochondria, promoting cell health and function. Mitochondria are energy producing organelles which require lifetime quality control through a ubiquitin-mediated clearance mechanism known as mitophagy. In certain situations, such as cellular stress, cell injury, and/or defects of the mitophagy process, the mitochondria can become dysfunctional and damaging to the cell, leading to reduced energy production, oxidative stress, inflammation and potentially cell death. Dysfunctional mitochondria are significant drivers of disease pathophysiology in acute kidney injury (AKI), Parkinson’s Disease (PD), heart failure, Duchenne’s Muscular Dystrophy, IPF, mitochondrial diseases and Alzheimer’s.

USP30 is a deubiquitylating enzyme that constantly removes ubiquitin from mitochondria, providing a potential brake on clearance of dysfunctional mitochondria. Mission is currently developing two small molecule drugs, MTX652 (peripheral) and MTX325 (targeting the CNS) which, through inhibition of the mitochondrial DUB enzyme USP30, will promote clearance of dysfunctional mitochondria – consequently improving overall cellular health. Mission’s USP30 inhibitors MTX652 and MTX325 could potentially be used to treat any disease or condition driven by mitochondrial dysfunction.

Mission has completed its first Phase I clinical study with MTX652, demonstrating a highly encouraging safety, tolerability and pharmacokinetic profile. A Phase II trial of MTX652 in patients at risk of acute kidney injury following cardiac surgery is planned to start in Q1 2024.

A Phase I trial of MTX325 in healthy volunteers and patients with PD is planned to start in Q1 2024. Mission is backed by blue-chip investors including Pfizer Venture Investments, Sofinnova Partners, Roche Venture Fund, SR One, IP Group and Rosetta Capital.

Mission Therapeutics to Present Abstract at American Society of Nephrology (ASN) Kidney Week 2023

CAMBRIDGE, UK, 2 November 2023 – Mission Therapeutics (“Mission”), a clinical-stage biotech company developing first-in-class therapeutics targeting mitophagy, today announces that it will present an oral abstract on its experimental drug candidate MTX652 at the American Society of Nephrology’s Kidney Week in Philadelphia, from 2-5 November 2023.

Mission Therapeutics is a world leader in discovering and developing novel therapeutics which promote the removal of dysfunctional mitochondria to maintain cell health and function. MTX652 targets USP30, a deubiquitylating (DUB) enzyme uniquely localized to the mitochondria that removes ubiquitin groups and is a negative regulator of the PINK1/Parkin pathway and mitophagy. Mitophagy is a quality control system that ensures dysfunctional mitochondria are removed.

Pau Aceves, MS, MSc, PharmD., Head of Clinical Pharmacology and Pharmacometrics, Mission Therapeutics, commented: We look forward to presenting our latest Phase 1 and Modelling & Simulation results on MTX652 for the treatment of acute kidney injury (AKI). The American Society of Nephrology is a major organization working toward a goal of a world without kidney diseases and we are excited to have the opportunity to share our progress at this important conference.

Details of the presentation are as follow:

Presentation Title: MTX652, a Novel Selective USP30 Inhibitor for the Treatment of AKI: Phase 1 Results in Healthy Subjects and Model-Driven Human Efficacious Dose Projections

Session Name: Onconephrology and Precision Pharmacology
Session Date: Thursday, November 2nd, 2023
Presentation Time: 4:48 PM – 4:57 PM

Presenter: Pau Aceves, MS, MSc, PharmD., Head of Clinical Pharmacology and Pharmacometrics

Location: Room 107, Pennsylvania Convention Center

Mission has completed its first Phase I clinical study with MTX652, in which it demonstrated a highly encouraging safety, tolerability and pharmacokinetic profile. A Phase II trial of MTX652 in patients at risk of acute kidney injury following cardiac surgery is planned to start in Q1 2024.

– ENDS –

For further information, please contact:

Mission Therapeutics Ltd:

Anker Lundemose MD PhD

Chief Executive Officer

Tel: +44 (0)1223 607 340

Optimum Strategic Communications:

Mary Clark, Stephen Adams, Vici Rabbetts

Email: mission@optimumcomms.com

Tel: +44 (0) 208 078 4357

About Mission Therapeutics

Mission Therapeutics is a world leader in discovering and developing novel therapeutics which promote the removal of dysfunctional mitochondria, promoting cell health and function. Mitochondria are energy producing organelles which require lifetime quality control through a ubiquitin-mediated clearance mechanism known as mitophagy. In certain situations, such as cellular stress, cell injury, and/or defects of the mitophagy process, the mitochondria can become dysfunctional and damaging to the cell, leading to reduced energy production, oxidative stress, inflammation and potentially cell death. Dysfunctional mitochondria are significant drivers of disease pathophysiology in acute kidney injury (AKI), Parkinson’s Disease (PD), heart failure, Duchenne’s Muscular Dystrophy, IPF, mitochondrial diseases and Alzheimer’s.

USP30 is a deubiquitylating enzyme that constantly removes ubiquitin from mitochondria, providing a potential brake on clearance of dysfunctional mitochondria. Mission is currently developing two small molecule drugs, MTX652 (peripheral) and MTX325 (targeting the CNS) which, through inhibition of the mitochondrial DUB enzyme USP30, will promote clearance of dysfunctional mitochondria – consequently improving overall cellular health. Mission’s USP30 inhibitors MTX652 and MTX325 could potentially be used to treat any disease or condition driven by mitochondrial dysfunction.

A Phase I trial of MTX325 in healthy volunteers and patients with PD is planned to start in Q1 2024.

Mission is backed by blue chip investors including Pfizer Venture Investments, Sofinnova Partners, Roche Venture Fund, SR One, IP Group and Rosetta Capital.

Mission Therapeutics to Present at Michael J. Fox Foundation’s 15th Annual Parkinson’s Disease Therapeutics Conference

CAMBRIDGE, UK, 10 October 2023 Mission Therapeutics (“Mission”), a clinical-stage biotech company developing first-in-class therapeutics targeting mitophagy, today announces that its Chief Scientific Officer, Dr Paul Thompson, will give an oral presentation on its experimental drug candidate MTX325 at the 15th Annual Michael J. Fox Foundation’s Parkinson’s Disease Therapeutics Conference on October 19, 2023, in New York.

Dr Thompson will participate in the session titled ‘Advances in Emerging Targets and Therapeutic Development’ where he will discuss Mission’s data demonstrating development of USP30 inhibitors for the treatment of Parkinson’s Disease (PD).

As the world’s largest nonprofit funder of Parkinson’s research, The Michael J. Fox Foundation (MJFF) is dedicated to accelerating a cure for Parkinson’s Disease and improved therapies for those living with the condition today. The annual Michael J. Fox Foundation’s Parkinson’s Disease Therapeutics Conference brings together 300 research and business development professionals from both academia and industry and showcases the most exciting and innovative research from MJFF’s research portfolio, and serves as a unique platform for field leaders to share new and unpublished results.

Dr Paul Thompson, Ph.D., Chief Scientific Officer of Mission Therapeutics, commented:We look forward to presenting our recent findings on USP30 inhibition for the treatment of Parkinson’s Disease at this prestigious conference. The Michael J. Fox Foundation shares our mission of unlocking the complex biology of Parkinson’s Disease to slow disease progression and improve patient lives with the development of the next generation of treatments.”

Mission Therapeutics is a world leader in discovering and developing novel therapeutics which promote the removal of dysfunctional mitochondria to maintain cell health and function. A major unmet need for Parkinson’s Disease patients is a treatment that slows or prevents the progression of disease. Mitochondrial dysfunction is considered a key pathophysiological driver of Parkinson’s Disease (PD) and several disease-associated gene mutations affect proteins involved in a dysfunctional mitochondria ubiquitin-dependent clearance mechanism known as mitophagy, including PINK1 and the ubiquitin E3 ligase, Parkin.

USP30 is a deubiquitylating (DUB) enzyme uniquely localized to the mitochondria that removes ubiquitin groups and is a negative regulator of the PINK1/Parkin pathway and mitophagy. Inhibiting USP30 has therefore been proposed as a therapeutic mechanism for protecting vulnerable dopamine-producing neurons in PD. Mission Therapeutics is currently developing two DUB inhibitors: MTX325 (targeting the CNS) and MTX652 (peripheral) which can potentially be used to treat any disease driven by mitochondrial dysfunction.

– ENDS –

For further information, please contact:

Mission Therapeutics Ltd:

Anker Lundemose MD PhD

Chief Executive Officer

Tel: +44 (0)1223 607 340

Optimum Strategic Communications:

Mary Clark, Stephen Adams, Vici Rabbetts

Email: mission@optimumcomms.com

Tel: +44 (0) 208 078 4357

 

About Mission Therapeutics

Mission Therapeutics is a world leader in discovering and developing novel therapeutics which promote the removal of dysfunctional mitochondria, promoting cell health and function. Mitochondria are energy producing organelles which require lifetime quality control through a ubiquitin-mediated clearance mechanism known as mitophagy. In certain situations, such as cellular stress, cell injury, and/or defects of the mitophagy process, the mitochondria can become dysfunctional and damaging to the cell, leading to reduced energy production, oxidative stress, inflammation and potentially cell death. Dysfunctional mitochondria are significant drivers of disease pathophysiology in acute kidney injury (AKI), Parkinson’s Disease (PD), heart failure, Duchenne’s Muscular Dystrophy, IPF, mitochondrial diseases and Alzheimer’s.

USP30 is a deubiquitylating enzyme that constantly removes ubiquitin from mitochondria, providing a potential brake on clearance of dysfunctional mitochondria. Mission is currently developing two small molecule drugs, MTX652 (peripheral) and MTX325 (targeting the CNS) which, through inhibition of the mitochondrial DUB enzyme USP30, will promote clearance of dysfunctional mitochondria – consequently improving overall cellular health. Mission’s USP30 inhibitors MTX652 and MTX325 could potentially be used to treat any disease or condition driven by mitochondrial dysfunction.

Mission has completed its first Phase I clinical study with MTX652, demonstrating a highly encouraging safety, tolerability and pharmacokinetic profile. A Phase II trial of MTX652 in patients at risk of acute kidney injury following cardiac surgery is planned to start in Q1 2024.

A Phase I trial of MTX325 in healthy volunteers and patients with PD is planned to start in Q1 2024.

Mission is backed by blue chip investors including Pfizer Venture Investments, Sofinnova Partners, Roche Venture Fund, SR One, IP Group and Rosetta Capital.

 

 

Mission Therapeutics founder to receive Knighthood in Birthday Honours List

Professor Steve Jackson earns KBE for services to innovation and research

CAMBRIDGE, UK – 17 June 2023 – Mission Therapeutics (“Mission”), a drug discovery and development company focused on protein homeostasis by selectively inhibiting deubiquitylating enzymes (DUBs), sends its congratulations to the company’s founder, Professor Steve Jackson, who has been made a Knight Bachelor as part of His Majesty The King’s Birthday Honours List.

Buckingham Palace confirmed the award was for services to innovation and research when the full list was announced on 17 June 2023.

Professor Jackson was appointed Frederick James Quick Professor of Biology at the University of Cambridge in 1995. He founded Mission Therapeutics in 2011 in order to advance and explore new therapeutic opportunities through his research in protein ubiquitylation and deubiquitylation. He has served as Chairman of the Scientific Advisory Board since the Company’s founding.

Professor Jackson also serves as Senior Group leader at Cancer Research UK Cambridge Institute, having previously been Head of Laboratories at the University’s Gurdon Institute. His team was responsible for the discovery of the mechanism that led to the creation of Olaparib, the world’s first marketed DNA-repair enzyme inhibitor, which has now been administered to over 60,000 patients globally.

Anker Lundemose, CEO of Mission Therapeutics, said: “The team at Mission are delighted for Steve and wish him many congratulations. His pioneering research and discoveries in the DUB field are the inspiration for Mission. His continued guidance on the Company’s development has been invaluable. The team at Mission are very proud of his achievements and this recognition in the Birthday Honours list is richly deserved.”

– ENDS –

FOR MORE INFORMATION PLEASE CONTACT:

Mission Therapeutics Ltd

Anker Lundemose MD PhD

Chief Executive Officer

Tel: +44 (0)1223 607 340

Instinctif Partners

Melanie Toyne-Sewell / Adam Loudon

Tel: +44 (0) 20 7457 2013

missiontherapeutics@instinctif.com

 NOTES TO EDITORS:

About Mission Therapeutics

Mission Therapeutics is a clinical-stage drug development company targeting the ubiquitin pathway for the treatment of kidney disease, heart disease and neurodegenerative disease. 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 $112 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.

Mission Therapeutics to Present at the Targeted Protein Degradation Europe Conference 2023

Presentation at 9am Thursday, March 30th 2023

CAMBRIDGE, UK– 6 March 2023 – Mission Therapeutics (“Mission”), a drug discovery and development company focused on protein homeostasis by selectively inhibiting deubiquitylating enzymes (DUBs), announces that Dr Nick Edmunds, Chief Technology Officer at Mission, will be presenting at the Targeted Protein Degradation Europe conference 2023.

Mission Therapeutics’ presentation will take place 9am Thursday, March 30th, 2023, at The Tower Hotel, St Katharine’s Way, London, E1W 1LD. The presentation will cover the Development of a DUB Inhibitor Platform & Emerging Data from a USP30 Inhibitor, based on Mission’s recent research. It has been highlighted as one of 5 key talks not to miss by the conference organisers.

The event, which will take place in London, 28th-30th March, is the Leading Forum Showcasing Key Stakeholders & Expert Minds from the European Degradation Community. The conference is returning to London for its third year to discuss key topics in what is expected to be a landmark year for the degradation community.

Prospective attendees can register for the event here: https://tpd-europe.com/

If you would like to meet with Nick and the team at TPD Europe, please contact info@missiontherapeutics.com.

– 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 / Agnes Stephens

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 $103 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.

Mission Therapeutics Successfully Completes First Clinical Assessment for Lead DUB Program, MTX652

  • Safety, tolerability and pharmacokinetic endpoints successfully met
  • Follow-on clinical trials to be initiated in 2023

CAMBRIDGE, UK – 5 January 2023 – Mission Therapeutics (“Mission”), a drug discovery and development company focused on protein homeostasis by selectively inhibiting deubiquitylating enzymes (DUBs), today announced the successful completion  of its first Phase I clinical assessment for lead USP30 DUB inhibitor, MTX652.

The Phase I First Time In Human (FTIH) study commenced in May 2022, following clinical trial approval in March. Involving over 80 healthy volunteers, the study was led by Principal Investigator Dr Annelize Koch, Senior Medical Director at Simbec-Orion.

The trial successfully achieved its key goals of demonstrating the safety, tolerability and pharmacokinetics of MTX652, given as single- and multiple-ascending doses of oral solution or suspension to the healthy subjects. MTX652 was shown to be well tolerated and safe up to a single dose of 200mg and multiple doses of 100mg once daily for 14 days. It also demonstrated an excellent pharmacokinetic profile with dose proportionality, and time independent exposure.

Following the successful completion of the single and multiple ascending dose cohorts, Mission is moving forward with further assessment of MTX652 in elderly subjects and using other dose forms. The Company is also preparing plans to initiate further clinical trials later this year to demonstrate the beneficial effects of MTX652 in patients with muscular, cardiac and kidney pathologies.

Dr Suhail Nurbhai, CMO of Mission Therapeutics commented:

“We are delighted with the successful completion of our first Phase I clinical assessment. These MTX652 results represent a major milestone for Mission and, coupled with the results from our broad translational pharmacology program, give us a great deal of optimism that we may eventually be able to help patients with a range of poorly-treated diseases. Importantly, it also helps to validate our unique discovery platform.

“We are now in the process of planning for our next stage of clinical development and are looking forward to MTX652 entering further clinical trials later this year.”

About Mitochondria and USP30

Mitochondria are essential for energy production and cellular health, and when they become dysfunctional or damaged, they are labelled with ubiquitin, marking them for degradation. USP30, a specific mitochondrial-associated DUB, removes this ubiquitin, thereby inhibiting the degradation of damaged mitochondria, which could affect cellular health and eventually lead to the development of various medical conditions.

Mission’s MTX652 potently and specifically inhibits USP30 with the aim of enabling appropriate degradation of dysfunctional mitochondria to preserve overall mitochondrial quality and improve cellular health.
Mitochondrial dysfunction is increasingly implicated in many poorly treated pathologic conditions including muscular dystrophy, heart failure, cardiomyopathy, kidney diseases, idiopathic pulmonary fibrosis, rare mitochondrial diseases, and neurodegenerative diseases such as Parkinson’s Disease. Inhibition of USP30 is therefore a promising therapeutic approach for treatment of these conditions and Mission is investigating its USP30 inhibitor compounds as potential therapeutic interventions for a breadth of diseases.

– 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 / Agnes Stephens / Jonjo Cordey

Email: missiontherapeutics@instinctif.com
Tel: +44 (0) 207 457 2002

 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 $103 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.

Mission Therapeutics to Present at the 41st Annual J.P. Morgan Healthcare Conference

Presentation at 5pm Wednesday January 11, 2023

CAMBRIDGE, UK– 3 January 2023 – Mission Therapeutics (“Mission”), a drug discovery and development company focused on protein homeostasis by selectively inhibiting deubiquitylating enzymes (DUBs), announces that CEO, Dr Anker Lundemose, will present at the upcoming J.P Morgan Healthcare Conference.

Mission Therapeutics’ presentation will take place at 5pm Wednesday January 11, 2023 in the Golden Gate Room at the Westin St Francis Hotel.

The event, which will take place in San Francisco on 9-12 Jan 2023, is the largest healthcare investment symposium in the industry, and aims to connect global industry leaders, emerging fast-growth companies, innovative technology creators and members of the investment community.

If you would like to meet up with Anker and the team, please contact info@missiontherapeutics.com.

– 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 / Agnes Stephens

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 $103 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.

New Data from Harvard Medical School Highlights USP30 Reduction as a Potential Strategy for Treatment of Parkinson’s Disease

CAMBRIDGE, UK – 15 November 2022 – Mission Therapeutics (“Mission”), a drug discovery and development company focused on protein homeostasis by selectively inhibiting deubiquitylating enzymes (DUBs), welcomes the presentation of new data from the Beth Israel Deaconess Medical Center (BIDMC) in Boston, which suggests that manipulating USP30 is a potential strategy for treating Parkinson’s disease.

The data, which was presented yesterday at the 2022 Society for Neuroscience (SfN) conference in San Diego, showed that deletion of DUB USP30 significantly reduced pathological changes associated with Parkinson’s disease.

Mission is already investigating USP30 as a therapeutic target for the treatment of a range of diseases. These include renal diseases such as acute kidney injury (AKI) and chronic kidney disease (CKD), congestive heart failure/cardiomyopathy, as well as other indications, such as idiopathic pulmonary fibrosis (IPF), neurodegenerative diseases, including Parkinson’s disease, and rare genetic mitochondrial diseases.

Mitochondria play a critical role in cellular health. The Ubiquitin E3 Ligase Parkin protein marks damaged or dysfunctional mitochondria for degradation, known as mitophagy. USP30 counteracts Parkin and can therefore prevent the degradation of potentially aberrant mitochondria. Inhibition of USP30 therefore promotes mitophagy and increases clearance of impaired mitochondria, potentially improving cellular health and longevity.

There is accumulating evidence that mitochondrial dysfunction plays a role in the progression of dopaminergic neurodegeneration in Parkinson’s disease patients and model systems. The study presented yesterday by T. Fang, S. Eleuteri and D.K. Simon aimed to investigate USP30 as an active agent in the development of Parkinson’s disease.

The team demonstrated that USP30 knockout enhanced mitophagy and decreased alpha-synucleinopathy and dopaminergic neuronal loss, key pathological hallmarks of Parkinson’s.

Commenting on the new data, Dr Paul Thompson, CSO of Mission Therapeutics, said:

“USP30 is one of Mission’s key therapeutic targets, under investigation for the treatment of a number of diseases, including renal diseases, IPF, heart diseases and neurodegenerative diseases. This year, our lead USP30 DUB program, MTX652, entered the clinic for the treatment of kidney disease. The new, independent data presented yesterday from Harvard Medical School provides further evidence that inhibition of USP30 has potential as a new strategy to treat Parkinson’s disease.”

– 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 / Jonjo CordeyEmail: missiontherapeutics@instinctif.com
Tel: +44 (0) 20 7457 2013

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.