We aim to improve health and quality of life for the millions of people affected by chronic respiratory diseases.
Our first-in-class development candidate, ensifentrine (RPL554), has the potential to provide relief for patients suffering from respiratory conditions such as chronic obstructive pulmonary disease (COPD), cystic fibrosis (CF) and asthma.
Ensifentrine is an inhaled therapy that acts as both a bronchodilator and an anti-inflammatory agent in one compound. This means, if approved, it would be the first single treatment that opens patients’ airways and reduces inflammation in the lungs. Ensifentrine is expected to enter Phase 3 clinical trials in 2020.
We are focused on developing our product candidate, ensifentrine, which we believe has the potential to become an important and novel treatment for chronic obstructive pulmonary disease (COPD), cystic fibrosis (CF) and other respiratory diseases, including asthma. We own the global commercialization rights to ensifentrine.
Rapidly advance nebulized ensifentrine into Phase 3 for COPD
Nebulized ensifentrine is expected to start Phase 3 clinical trials in 2020 as a maintenance treatment for moderate to severe COPD. Millions of COPD patients urgently need better treatments as they remain symptomatic despite maximum treatment with currently available therapies. In the US alone, total annual medical costs, indirect and direct, relating to COPD are projected to rise to about $50 billion in 2020. We believe ensifentrine will have an important role as an add-on therapy for dual/triple treated patients.
Develop formulations of ensifentrine for handheld inhalers
In addition to nebulized ensifentrine, we have developed dry powder inhaler (DPI) and pressurised metered dose inhaler (pMDI) formulations of ensifentrine for less severe patients. Initial results from Phase 2 trials for the maintenance treatment of COPD are promising. In the US, DPI and pMDI handheld inhalers are the most widely used option for medication in COPD, with an estimated 5.5 million people using them for COPD maintenance therapy. This market was valued at approximately $9 billion in 2018.
Seek strategic relationships
We may seek strategic collaborations with leading biopharmaceutical companies to develop and commercialize ensifentrine. These partnerships could provide significant funding to advance the development of ensifentrine while allowing us to benefit from the development and commercialization expertise of our collaborators.
Develop ensifentrine for cystic fibrosis and severe asthma
We may develop ensifentrine for the treatment of cystic fibrosis and severe asthma. CF is considered an orphan disease by both the US and European regulatory authorities. According to WHO estimates, 300 million people suffer from asthma and it is the most common chronic disease amongst children.
Pursue development of ensifentrine in additional territories
Initially, we are developing ensifentrine for the US and European markets. However, we may also develop ensifentrine for other markets such as China, which has more than 50 million COPD patients and is estimated to grow to 70 million in 2030.
Acquire or in-license product candidates for respiratory diseases
We are leveraging our expertise in respiratory diseases to in-license or acquire additional clinical-stage product candidates. We are seeking assets with the potential to become novel treatments for respiratory diseases with significant unmet medical needs.
Ensifentrine's unique mechanism of action has the potential to have a major impact on the treatment of respiratory diseases
Ensifentrine combines bronchodilator and anti-inflammatory properties in one compound and has the potential to be an effective treatment for COPD and other respiratory diseases, including asthma and cystic fibrosis.
Ensifentrine has been well tolerated in clinical trials involving over 1300 people. It is designed to maximize its effectiveness and reduce adverse events through:
high selectivity for PDE3 and PDE4 over other enzymes and receptors to minimize off-target effects;
direct delivery to the lungs by inhalation to maximize pulmonary exposure to ensifentrine while minimizing systemic distribution and potential adverse events.
NON-EXECUTIVE CHAIRMAN OF THE BOARD
Dr Ebsworth has as served as the Non-Executive Chairman of our board of directors since December 2014.
From October 2009 to August 2014, Dr Ebsworth served as Chief Executive Officer of Vifor Pharma, based in Zürich, the specialty pharma division of Galenica AG Group, a pharmaceutical wholesaler and retailer, and as a member of Galenica’s Executive Committee. In 2012, Dr Ebsworth was also named as Chief Executive Officer of Galenica and as Chairman of Galenica’s Executive Committee, positions he held until August 2014. Dr Ebsworth received a PhD in industrial relations from the University of Surrey.
PRESIDENT AND CHIEF EXECUTIVE OFFICER
Dr Zaccardelli was appointed as President and Chief Executive Officer and joined our board of directors in February 2020.
From December 2018 until its acquisition by Swedish Orphan Biovitrum for up to $915 million in November 2019, Dr Zaccardelli served as President and CEO of Dova Pharmaceuticals, a US company developing therapeutics for rare diseases. Previously, he was Acting CEO of Cempra, from December 2016 until the company’s merger with Melinta Therapeutics in November 2017. From 2004 until 2016, Dr Zaccardelli served in several senior management roles at United Therapeutics Corporation, including Chief Operating Officer, Chief Manufacturing Officer and Executive Vice President, Pharmaceutical Development and Operations. Prior to United Therapeutics, he founded and led a start-up company focused on contract research positions and held a variety of clinical research positions at Burroughs Wellcome & Co, Glaxo Wellcome, and Bausch & Lomb Pharmaceutical. Dr. Zaccardelli received a Pharm.D. from the University of Michigan.
Dr Cunningham has served as a Non-Executive Director on our board of directors since September 2015.
Dr Cunningham serves as the non-executive chairman of the board of directors of Abzena and non-executive member of the board of directors of Medherant. Dr Cunningham received a degree in medicine from St. Mary’s, Imperial College, London University.
Dr Edwards has served as an Independent Non-Executive Director on our board of directors since April 2019.
Dr Edwards has significant experience in investing and contributing to the development of biotech companies in both Europe and the US. Since 2003, Dr Edwards has held various positions at Novo Holdings, a life sciences investor, and is currently part-time Senior Partner. Dr Edwards serves on the boards of several biotech companies. Earlier in his career, he was Corporate VP and Global Head of Drug Development for Novo Nordisk, where he led all aspects of pre-clinical and clinical drug development. Dr Edwards trained in physiology and medicine at the University of Manchester. He is a Member of the Royal College of Physicians, a Member with distinction of the Royal College of General Practitioners, a Fellow of the Faculty of Pharmaceutical Medicine and holds a MBA from the University of Warwick.
Mr Gupta has served as a Non-Executive Director on our board of directors since July 2016.
Since 2002, Mr Gupta has held various positions at OrbiMed Advisors, a global healthcare investment firm, where he is currently a Private Equity Partner. Mr Gupta currently is a member of the board of directors of Avitide and Turnstone Biologics. Mr Gupta received an AB in biochemical sciences from Harvard College and a JD from Yale Law School.
Mahendra G. Shah
Dr Shah has served as a Non-Executive Director on our board of directors since July 2016.
Since March 2010, Dr Shah has served as a Managing Director of Vivo Capital, a healthcare investment firm. Dr Shah is also the founder and Executive Chair of Semnur Pharmaceuticals, a specialty pharmaceutical company. Dr Shah serves as a member of the board of directors of Fortis, a specialty pharmaceuticals company, Crinetics Pharmaceuticals, Soleno Therapeutics, Impel Neuropharma, and several other private companies in the biopharmaceutical and biotechnology industries. Dr Shah received his PhD in industrial pharmacy from St John’s University and a Master’s Degree in Pharmacy from LM College of Pharmacy in Gujarat, India.
Dr Sinclair has served as a Non-Executive Director on our board of directors since July 2016.
Since 2008, Dr Sinclair has held various positions at Abingworth LLP, a life sciences investment group, where he is currently a Partner and Portfolio Manager. Dr Sinclair received a PhD in chemistry and genetic engineering at the BBSRC Institute of Plant Science, Norwich, and a BSc in microbiology from King’s College London.
Mr Sinha has served as a Non-Executive Director on our board of directors since September 2016.
Mr Sinha is Chief Financial Officer and co-founder of ElevateBio, a holding company focused on building cell and gene therapy companies, which was formed in January 2018. He also serves as President and Chief Financial Officer of AlloVir, an ElevateBio portfolio company. From 2005 to 2016, he was the Chief Financial Officer of Alexion Pharmaceuticals, a biotechnology company. Mr Sinha holds a master's degree in business administration from the Asian Institute of Management. He is also a qualified Chartered Accountant from the Institute of Chartered Accountants of India and a Certified Public Accountant in the US.
Dr Ullman has served as a Non-Executive Director on our board of directors since September 2015.
Since 2016, he has served as Head of the COPD Centre at Sahlgrenska University Hospital, Sweden. From 2013 to 2014, Dr Ullman was Executive Vice President and Head of Research and Development in the BioScience business unit of Baxter International, a healthcare company that became Baxalta. From 2007 to 2013, Dr Ullman was Executive Vice President, Head of Research and Development at Nycomed Pharma, which was acquired by Takeda Pharmaceutical. Dr Ullman received a MD and a PhD in clinical pharmacology from the University of Gothenburg.
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