Enterprise Therapeutics’ First-in-Class TMEM16A potentiator program for treatment of cystic fibrosis and other respiratory diseases acquired by Roche

  • Enterprise’s novel TMEM16A potentiator portfolio includes ETD002, a first in class compound which is currently in Phase 1
  • TMEM16A potentiation is a novel therapeutic approach applicable to all cystic fibrosis patients, independent of CFTR genotype, and may provide benefit in other respiratory diseases
  • Enterprise’s shareholders received an upfront payment of £75 million

Brighton, UK, 07 October 2020: Enterprise Therapeutics Ltd (Enterprise), a biopharmaceutical company dedicated to the discovery and development of novel therapies to improve the lives of patients suffering with respiratory disease, today announced its novel TMEM16A potentiator portfolio has been fully acquired by Roche and will be developed by Genentech, a member of the Roche Group. The portfolio includes ETD002 which recently entered Phase 1 trials.

Enterprise’s shareholders received an upfront payment of £75 million and are eligible to receive additional contingent payments, to be made based on the achievement of certain predetermined milestones.

The TMEM16A portfolio is focused toward treating all people with cystic fibrosis (CF), with potential to benefit people with other severe respiratory diseases characterised by excessive mucus congestion.

 Dr John Ford, CEO, Enterprise Therapeutics, said: “Roche and Genentech have a proven track record of bringing new medicines to people with respiratory diseases, and have recognised the opportunity that our TMEM16A potentiator portfolio presents. I am very proud of the team at Enterprise for identifying and developing this innovative approach to treat patients, with ETD002 the first of our compounds to reach clinical stage. TMEM16A potentiation has the potential to significantly increase the quality of life for people living with cystic fibrosis, for many of whom existing therapies are not effective.”

 Dr James Sabry, MD, PhD, Global Head of Pharma Partnering, Roche, commented: “We are excited to add Enterprise’s TMEM16A potentiator program to our existing respiratory portfolio. We have deep capabilities in this area and look forward to a robust program focused on helping cystic fibrosis patients and patients suffering from other muco-obstructive disorders as quickly as possible.”

CF is estimated to affect 75,000 people globally. One of the main causes of difficulty in breathing and increased risk of infection is mucus congestion in the lungs. The ETD002 compound targets the underlying mechanisms of mucus congestion, and is expected to restore lung function, reduce the frequency of lung infections and improve patient quality of life. CF is caused by loss of function mutations in the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) gene, that normally produces an anion channel highly expressed by the airway epithelium. Increasing anion conductance via CFTR modulation is a clinically validated approach for treating CF, however it is not currently available or effective for all people with CF.

In pre-clinical models, Enterprise has demonstrated that ETD002 enhances the activity of TMEM16A, an alternative anion channel present in airway epithelial cells, and by doing so increases anion and fluid flow into the airways, thinning the mucus and increasing its clearance. As TMEM16A potentiation is independent of the mutational status of CFTR, this makes the approach potentially applicable to all people with CF, and perhaps patients with non-CF muco-obstructive lung disease.

Moelis acted as financial advisor and Goodwin Procter acted as legal counsel to Enterprise Therapeutics.

Enterprise Therapeutics doses first subjects in Phase 1 trial for First-in-Class cystic fibrosis therapy ETD002

  • ETD002 is a novel TMEM16A chloride channel potentiator
  • Therapy applicable to all cystic fibrosis patients, independent of genotype

Brighton, UK, 17 August 2020: Enterprise Therapeutics Ltd (Enterprise), a biopharmaceutical company dedicated to the discovery and development of novel therapies to improve the lives of patients suffering with respiratory disease, today announced it has successfully dosed the first subjects in a Phase 1 trial for its novel inhaled cystic fibrosis therapy, ETD002. The first-in-man safety study is being conducted in healthy participants with ETD002, a TMEM16A potentiator aimed at treating all people with cystic fibrosis (CF).

CF is estimated to affect 75,000 people globally. One of the main causes of difficulty in breathing and increased risk of infection in CF is mucus congestion in the lungs. Enterprise’s proprietary compound ETD002 targets the underlying mechanisms of mucus congestion, and is expected to restore lung function, reduce the frequency of lung infections and improve patient quality of life. CF is caused by loss of function mutations in the cystic fibrosis transmembrane conductance regulator (CFTR), an anion channel highly expressed by the airway epithelium. Increasing anion conductance via CFTR modulation is a clinically validated approach for treating CF, however it is not currently available or effective for all people with CF.

In pre-clinical models, Enterprise has demonstrated that ETD002 enhances the activity of TMEM16A, an alternative anion channel present in airway epithelial cells, and by doing so increases anion and fluid flow into the airways, thinning the mucus and increasing its clearance. As TMEM16A potentiation is independent of the mutational status of CFTR, this makes the approach applicable to all people with CF, and potentially patients with non-CF lung disease. Additionally, ETD002 is expected to deliver benefit as a monotherapy and in combination with other therapies, including CFTR repair.

Dr John Ford, CEO, Enterprise Therapeutics, said: “Considerable efforts to identify and develop this innovative compound have resulted in a TMEM16A potentiator that has the potential to significantly increase the quality of life for people living with CF, for many of whom existing therapies are not effective. We are excited to have begun the clinical stage of development for ETD002.”

 Dr David Morris, CMO, Enterprise Therapeutics, said: “Although CFTR modulators have successfully demonstrated improved clinical outcomes in those genetically suited to these therapies, we are hopeful that TMEM16A potentiation via ETD002 will provide clinical benefit to the many people with CF who do not share these CFTR mutations. We look forward to generating our first data in human volunteers over the next few months and are grateful to the subjects and investigators who are helping us to advance this novel treatment for individuals with CF.”

This work is in part funded by a Therapeutics Development Award from the Cystic Fibrosis Foundation to Enterprise Therapeutics.

Enterprise Therapeutics appoints Dr David Morris MD as Chief Medical Officer

Dr Morris will lead Enterprise’s therapeutics development strategy and drive the Company’s two lead respiratory programmes through the clinic

Brighton, UK, 10 February 2020: Enterprise Therapeutics Ltd (Enterprise), a biopharmaceutical company dedicated to the discovery and development of novel therapies to improve the lives of patients suffering with respiratory disease, today announced it has appointed Dr David Morris MD as Chief Medical Officer to lead the Company’s respiratory programmes. These programmes include clinical development for ETD002, a first-in-class TMEM16A potentiator and ETD001, a novel ENaC blocker, therapies aimed at treating all cystic fibrosis patients.

Enterprise Therapeutics is developing novel disease-modifying therapies which target the underlying mechanisms of mucus congestion, to enhance the clearance of mucus from the airways, restore lung function, and ultimately to reduce morbidity and mortality in chronic respiratory diseases including cystic fibrosis (CF).

Dr Morris joins Enterprise from the Novartis Venture Fund, an investor in Enterprise, where he is currently a Managing Director, and where he will maintain an appointment as an Operating Partner. Prior to his career in venture capital David held various leadership roles in the Novartis Pharmaceuticals development organisation, including Development Franchise Head of Respiratory, Development Franchise Head of Primary Care, and Global Head of Clinical Operations, Analytics and Regions, where he was responsible for all aspects of global clinical trials operations, monitoring, and reporting. In addition, David has also held Director level roles in respiratory discovery research and translational medicine at Roche.

Dr Morris received his Bachelors and Medical Degrees with distinction from the University of Rochester.  His clinical training in internal medicine and pulmonary and critical care medicine were at Massachusetts General Hospital and the University of California, San Francisco.  Before joining industry, he held faculty appointments and led basic and translational research programs at University of California, San Francisco and Yale University School of Medicine.

Dr John Ford, CEO, Enterprise Therapeutics, said: “The knowledge and experience David has gained through his successful career in biopharmaceutical discovery and development, as well as his stellar sector expertise in respiratory biology, will be an invaluable asset as we work to define our strategy to progress our respiratory programmes which will initially focus on CF.”

Dr David Morris, CMO, Enterprise Therapeutics, said: “Enterprise’s programmes show great potential to deliver disease-modifying, clinically effective candidates for a significant number of patients with respiratory diseases, including all CF patients regardless of underlying mutations. I am excited to become more closely involved with the team moving forward, at this exciting point in the Company’s development.”

Enterprise Therapeutics publishes paper on novel therapeutic approach for treatment of all cystic fibrosis patients

  • “TMEM16A Potentiation: A Novel Therapeutic Approach for the Treatment of Cystic Fibrosis” published in American Journal of Respiratory and Critical Care Medicine
  • Paper demonstrates first in class TMEM16A chloride channel potentiators, to accelerate mucociliary clearance
  • Co-authored by researchers from University of Sussex, University of North Carolina and University of Miami

Brighton, UK, 08 January 2020: Enterprise Therapeutics Ltd (Enterprise), a biopharmaceutical company dedicated to the discovery and development of novel therapies to improve the lives of patients suffering with respiratory disease, today announced the publication of its first peer-reviewed paper. The open access paper, published in the American Journal of Respiratory and Critical Care Medicine1, describes TMEM16A potentiation via ETX001 as a novel approach for the treatment of cystic fibrosis (CF). The research was conducted in collaboration with University of Sussex, University of North Carolina and University of Miami.

The paper, entitled “TMEM16A Potentiation: A Novel Therapeutic Approach for the Treatment of Cystic Fibrosis”, demonstrates the ability of Enterprise’s proprietary compound, ETX001, to enhance the activity of TMEM16A in human bronchial epithelial cells from CF patients, increasing epithelial fluid secretion and mucus clearance providing the first pre-clinical proof of principle for this approach.

CF is estimated to affect 75,000 patients globally and is caused by loss of function mutations in the anion channel, cystic fibrosis transmembrane conductance regulator (CFTR). Increasing anion conductance via CFTR modulation is a clinically validated approach for treating CF, however it does not treat ≥10% of patients with a combination of nonsense and other rare mutations. In addition, many patients eligible for CFTR repair therapy do not benefit from these therapies. TMEM16A potentiation offers a non-CFTR mediated approach for the treatment of CF and can be delivered as a monotherapy or in combination with other therapies such as CFTR repair.

Dr Henry Danahay, Head of Biology, Enterprise Therapeutics, and lead author of the paper, said:

“We have successfully demonstrated the positive effects of ETX001 on both airway fluid secretion and mucus clearance in cells from CF patients. Given the percentage of the population of CF patients who are not genetically matched to existing CFTR repair therapies, this paper builds a strong case for testing TMEM16A potentiation in the clinic.”

Authors on the paper include Henry L Danahay (Enterprise Therapeutics), Sarah Lilley (Sussex Drug Discovery Centre, University of Sussex), Roy Fox (Sussex Drug Discovery Centre, University of Sussex), Holly Charlton (Sussex Drug Discovery Centre, University of Sussex), Juan Sabater (Mount Sinai Medical Centre, University of Miami), Brian Button (Department of Biochemistry & Biophysics, UNC Chapel Hill, North Carolina), Clive McCarthy (Enterprise Therapeutics), Stephen P Collingwood (Enterprise Therapeutics), and Martin Gosling (Enterprise Therapeutics, and Sussex Drug Discovery Centre, University of Sussex).

This work was in part funded by a Therapeutics Development Award from the Cystic Fibrosis Foundation to Enterprise Therapeutics.

1https://www.atsjournals.org/doi/abs/10.1164/rccm.201908-1641OC

Enterprise Therapeutics awarded up to £5.7M ($7M) from Cystic Fibrosis Foundation to Support Clinical Development of Novel Chloride Channel Modulator

  • Support for clinical development up to end of Phase 2 for ETD002, first-in-class TMEM16A potentiator for the treatment of cystic fibrosis
  • Milestone Funding through Cystic Fibrosis Foundation’s Therapeutics Development Award programme

Brighton, UK, 15 October 2019: Enterprise Therapeutics Ltd (Enterprise), a biopharmaceutical company dedicated to the discovery and development of novel therapies to improve the lives of patients suffering with respiratory disease, today announced it has been awarded up to £5.7M ($7M) funding under the Therapeutics Development Award programme from the Cystic Fibrosis Foundation. The milestone-related funding will support clinical development up to the end of Phase 2 for ETD002 – a first-in-class TMEM16A potentiator that has the potential to treat all persons with cystic fibrosis (CF), independent of their cystic fibrosis transmembrane conductance regulator (CFTR) genotype.

Enterprise Therapeutics is developing novel disease-modifying therapies which target underlying mechanisms of mucus congestion, enhancing the clearance of mucus from the airways, thereby restoring lung function and to reduce morbidity and mortality in CF. The Company’s first-in-class ETD002 programme is targeting the calcium-activated chloride ion channel TMEM16A. By enhancing the activity of TMEM16A, there is increased anion and fluid flow into the airways, thinning the mucus and increasing its clearance from the airways.

CF is a devastating and life-limiting genetic disease. CF patients have a significant reduction in the hydration of their airway mucus, leading to failed clearance, a high incidence of infections, and rapid decline in lung function.

Dr John Ford, CEO, Enterprise Therapeutics, said:

“We are delighted to receive such a significant award from the CF Foundation. This funding will enable critical clinical research to be undertaken on our novel TMEM16A programme, and highlights the potential of chloride channel modulation via alternative channels to deliver innovative and effective treatments for all CF patients.”

Enterprise Therapeutics strengthens leadership team, appoints Amit D. Munshi as Non-Executive Chairman

Appointment brings significant commercial pharma experience as Enterprise Therapeutics’ two lead respiratory programmes are progressed towards the clinic

Brighton, UK, 25 March 2019: Enterprise Therapeutics Ltd (Enterprise), a biopharmaceutical company dedicated to the discovery and development of novel therapies to improve the lives of patients suffering with respiratory disease, today announced the appointment of Amit D. Munshi as Non-Executive Chairman of the Board of Directors. Amit’s appointment is significant for Enterprise, strengthening the ability of the leadership team as it transitions to a clinical stage development company, addressing areas of critical unmet needs in respiratory disease.

Mr. Munshi has more than 28 years of global biopharmaceutical industry experience in executive management, business development, product development and portfolio management, and has served on several private and public company boards. He is Director, President and Chief Executive Officer of Arena Pharmaceuticals, Inc., a Nasdaq-listed biopharmaceutical company which has a proprietary pipeline in mid-late stage clinical development. Previously, Mr. Munshi served as President and Chief Executive Officer of Epirus Biopharmaceuticals, Inc. and Percivia LLC. Prior to Epirus and Percivia, he was a co-founder and Chief Business Officer of Kythera Biopharmaceuticals, Inc., and held multiple leadership positions at Amgen, Inc. Mr. Munshi holds a B.S. in Economics and a B.A. in History from the University of California, Riverside, and an M.B.A. from the Peter F. Drucker School of Management at Claremont Graduate University.

Dr John Ford, CEO, Enterprise Therapeutics, said: “We are delighted that Amit has chosen to join as Chairman at this exciting time for the Company, as we prepare to take our two lead programmes into the clinic in the next 12 months. Amit brings a wealth of experience in product development, portfolio management and commercial strategy in both the US and EU that will be critical in driving the Company through the next phase of its development.”

Amit D. Munshi, Chairman, Enterprise Therapeutics, said: “Enterprise is led by an impressive management team with significant expertise and experience, and is backed by a high-profile syndicate of investors. I believe the Company and its therapeutic programmes have real potential to make a difference to the lives of respiratory disease patients.”

Enterprise Therapeutics is developing novel disease-modifying therapies which target underlying mechanisms of mucus congestion, enhancing the clearance of mucus from the airways, thereby restoring lung function and reducing morbidity and mortality in respiratory diseases. Enterprise’s programmes differentiate from the current standard of care as they aim to deliver disease-modifying, clinically effective candidates for a significant number of patients with respiratory diseases, including all CF patients, regardless of underlying mutations, and a large percentage of patients with COPD and severe asthma.

In April 2018 Enterprise closed an oversubscribed £29 million ($41 million USD) Series B round co-led by Versant Ventures and Novartis Venture Fund. The syndicate also included new investor Forbion, founding investor Epidarex Capital and existing investor IP Group.

Enterprise Therapeutics selected as a finalist for Scrip Awards and Lifestars Awards

  • Scrip Awards 2018 “Financing Deal of the Year- Private” category
  • Lifestars Awards 2018 “EU Private Finance Raise of the Year <£30M” category

Brighton, UK – 02 October 2018: Enterprise Therapeutics Ltd (Enterprise), a biopharmaceutical company dedicated to the research and development of novel therapies for the treatment of respiratory diseases, has recently been announced as a finalist in the “Financing Deal of the Year” category for the Scrip Awards, and the “EU Private Finance Raise of the Year- <£30M” category of LSX’s Lifestars Awards, based on its oversubscribed £29 million ($41 million USD) Series B funding round.

 

Enterprise announced the closing of its Series B round in April 2018. The round was co-led by Versant Ventures and Novartis Venture Fund, and the syndicate also included new investor Forbion, founding investor Epidarex Capital, and existing investor IP Group. The investment will fund the Company’s drug discovery pipeline of muco-regulatory therapies into clinical development, with the aim of enabling new treatment options for cystic fibrosis, chronic obstructive pulmonary disease and severe asthma.

 

The Scrip Awards recognise both individual and corporate achievement across a wide range of industry activities, including novel deals, new drug launches and technological breakthroughs in clinical trials. Winners will be announced on 28th November at the Gala Dinner in London.

 

The Lifestars Awards celebrate the success, innovations and transformational deals advancing the industry over the past year. The awards ceremony is run by LSX (formerly known as Biotech and Money) in association with Jefferies, on the evening of 13th November. The awards form the evening network function of the Jefferies Annual Healthcare Conference in London, Europe’s largest healthcare investment banking conference.

 

Dr John Ford, CEO, Enterprise Therapeutics, commented: “The Series B investment, along with the additional scientific, development and biotech expertise held by new directors, means that Enterprise is incredibly well positioned to achieve its longer-term goals of bringing new respiratory medicines to market. We are delighted that our significant progress has been recognised by these two very credible and prestigious industry awards.”

 

For more information about the Scrip Awards 2018, visit: https://pharmaintelligence.informa.com/events/awards/scrip-awards-2018

For more information about the Lifestars Awards 2018, visit: https://www.lsxleaders.com/lifestars-awards

Enterprise Therapeutics raises £29 million ($41 million USD) funding

Brighton, UK – 12 April 2018: Enterprise Therapeutics Ltd (Enterprise), a biopharmaceutical company dedicated to the research and development of novel therapies for the treatment of respiratory diseases, today announced that it has closed an oversubscribed £29 million ($41 million USD) Series B round co-led by Versant Ventures and Novartis Venture Fund. The
syndicate also included new investor Forbion, founding investor Epidarex Capital and existing investor IP Group.

The investment will fund the Company’s drug discovery pipeline of muco-regulatory therapies into clinical development, enabling new treatment options for cystic fibrosis, chronic obstructive pulmonary disease and severe asthma.

Enterprise Therapeutics is developing muco-regulatory therapies to treat patients with respiratory diseases of high unmet medical need, where mucus obstruction reduces lung function, leading to difficulty in breathing and recurrent lung infections. These novel therapies target the ion channels TMEM16A and ENaC, to increase the hydration and clearance of mucus. Enterprise Therapeutics has also identified novel targets and compounds that reduce mucus production, an approach that complements mucus hydration therapies.

Commenting on the fundraising, Dr John Ford, CEO, Enterprise Therapeutics, said: “Attracting this syndicate of leading life science investors is a reflection of what the Company has achieved to date and the strength of our pipeline. At Enterprise, we believe new therapies that target the underlying mechanisms of mucus congestion are essential to reduce the frequency of
lung infections and improve patient quality of life.”

Dr Alex Mayweg, Partner, Versant Ventures, commented: “We are very encouraged by progress the Enterprise team made in generating candidates for highly sought-after but challenging targets. This financing should enable introduction of novel therapeutics for patients with cystic fibrosis and other respiratory diseases.”

Dr David Morris, Venture Partner, Novartis Venture Fund, commented: “Our focus is on finding solutions for unmet needs and clinical impact, an area where Enterprise Therapeutics is making excellent progress for respiratory disease patients. We believe this additional round of funding puts the Company in a strong position as it continues to progress towards the clinic.”

“Forbion focuses on supporting novel approaches that have the potential to truly impact the future of medicine” commented Dr Geert-Jan Mulder, Managing Partner, Forbion. “As Enterprise Therapeutics has both a highly innovative R&D pipeline and an experienced leadership team, the Company in our view is well positioned to become a key player in the field of novel respiratory medicine.”

Enterprise Therapeutics receives funding from Cystic Fibrosis Trust

Award will support research into the use of bronchosphere technology as innovative treatment

Brighton, UK – 05 April, 2017: Enterprise Therapeutics Ltd, a drug discovery company dedicated to the research and development of novel therapies for the treatment of respiratory diseases, has won funding from the Cystic Fibrosis Trust to identify new drug mechanisms for the treatment of cystic fibrosis (CF).

The funding will be used to support pioneering research, leveraging Enterprise Therapeutics’ bronchosphere technology platform. Bronchospheres are a miniaturised model of the human airway. This innovative model can be used to support high-throughput drug and target discovery and will be used to facilitate the development of new classes of therapeutics for the treatment of cystic fibrosis.

Over 10,500 people are currently living with CF in the UK. This genetic condition causes the lungs to become clogged with mucus, making it difficult to breathe. People living with CF have a significantly reduced life-expectancy; median age of death is just 28 years. Quality of life is also extremely poor due to high treatment burden and susceptibility to chronic lung infections that result in frequent hospitalisations.

Commenting on this new partnership, Dr John Ford, CEO, Enterprise Therapeutics, said: “We are delighted to have the opportunity to collaborate with the Cystic Fibrosis Trust on this important project. The Trust’s funding will enable critical research to be undertaken to drive a greater scientific understanding of CF and support development of innovative treatments for this challenging genetic disease.”

Dr Janet Allen, Director of Strategic Innovation at the Cystic Fibrosis Trust commented: “This exciting approach will bring hope to the many people living with CF in the UK. We look forward to working with Enterprise Therapeutics and believe that their technology will bring us a step closer to identifying effective new medicines that may improve the lives of people with CF and those who care for them.”

Enterprise Therapeutics Announces £4 million ($5 million USD) Fundraising

Brighton, UK – 10th November, 2016: Enterprise Therapeutics Ltd, a drug discovery company dedicated to the research and development of novel therapies for the treatment of respiratory diseases, today announced that it has closed a £4 million ($5 million USD) financing. The funding was led by existing investors Epidarex Capital and Imperial Innovations and will be used to accelerate the Company’s three drug discovery projects towards drug candidate selection.

Enterprise Therapeutics is developing novel muco-regulatory therapies for patients suffering with cystic fibrosis (CF), Chronic Obstructive Pulmonary Disease (COPD) and severe asthma. To achieve this, the Company is targeting mechanisms that increase the clearance of mucus or reduce levels of mucus production in order to alleviate the symptoms and complications
associated with respiratory disease.

Commenting on the fundraising, Dr John Ford, CEO, Enterprise Therapeutics, said: “Respiratory disease remains an area of high unmet medical need especially in the area of CF where existing therapies do not treat all patients. This additional funding will allow us to advance our research projects and move closer towards our goal of evaluating the efficacy of muco-regulatory drugs in patients. I look forward to working with the team and our investors in this important next
stage of growth.”

Dr Liz Roper, Investor Director, Epidarex, commented: “Based on excellent achievement of project milestones across all three discovery projects at Enterprise Therapeutics we are providing this additional investment to put the company in a strong position as it continues to progress towards the clinic. We are committed to the novel muco-regulatory approach that
Enterprise is taking to combat respiratory diseases.”

Dr Rob Woodman, Investor Director, Imperial Innovations commented: “Enterprise Therapeutics has made significant progress in the 18 months since Imperial Innovations first invested. We believe that the Company has the potential to become a leading player in respiratory disease.”